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News Article | December 23, 2016
Site: globenewswire.com

~ Expands Rare Disease Portfolio through Acquisition of U.S. Rights to KEVEYIS®, the First and Only FDA-Approved Treatment for Hyperkalemic, Hypokalemic, and Related Variants of Primary Periodic Paralysis ~ ~ Provides Update on Timelines and Strengthens COR-003 Clinical Development Program with Additional Phase 3 Study ~ ~ Announces $35 Million in Equity Financing; Finalizing $40 Million Credit Facility of which $20 Million to be Borrowed Initially, Extending Cash Runway at Least Through 2018 ~ DUBLIN, Ireland and TREVOSE, Pa., Dec. 23, 2016 (GLOBE NEWSWIRE) -- Strongbridge Biopharma plc, (Nasdaq:SBBP), a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs, today announced a corporate and financial update. “Throughout the second half of 2016, Strongbridge has been focused on building value in three key areas: enhancing the clinical development program and the probability of regulatory success for COR-003, pursuing a commercial-stage rare disease therapy resulting in the acquisition of the U.S. rights to KEVEYIS® (dichlorphenamide), and strengthening the financial condition of the Company,” said Matthew Pauls, president and chief executive officer of Strongbridge. “Executing on these initiatives strengthens our presence in rare diseases and sets the stage for a number of value-creating events in 2017, including commercial sales of KEVEYIS and completing enrollment in the SONICS study,” Pauls added. Expands Rare Disease Portfolio through Acquisition of U.S. Rights to KEVEYIS®, the First and Only FDA-Approved Treatment for Hyperkalemic, Hypokalemic, and Related Variants of Primary Periodic Paralysis Earlier today, Strongbridge announced in a separate press release that the Company has acquired the U.S. rights to KEVEYIS, the first and only U.S. Food and Drug Administration (FDA)-approved product to treat hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis, which is a group of rare hereditary disorders that causes potentially severe episodes of muscle weakness and/or paralysis. KEVEYIS is Strongbridge’s first commercial product, and expands the Company’s rare disease franchises to include both neuromuscular and endocrine diseases. Provides Update on Timelines and Strengthens COR-003 Clinical Development Program with Additional Phase 3 Study The global Phase 3 SONICS clinical trial evaluating COR-003 (levoketoconazole) for the treatment of endogenous Cushing’s syndrome is more than two-thirds enrolled. Based upon an analysis of recent enrollment trends, Strongbridge anticipates that the study will be fully enrolled in Q2 2017, with top-line data for the primary efficacy analysis available in Q1 2018. In October 2016, at the fourth semi-annual Data and Safety Monitoring Board (DSMB) review of the SONICS data, the DSMB recommended that the SONICS study continue as planned. In addition, based upon Strongbridge’s ongoing dialog with the FDA and observation of the current regulatory landscape, the Company plans to initiate LOGICS, a second global Phase 3 study of COR-003 for the treatment of endogenous Cushing's syndrome. The LOGICS study will supplement the long-term efficacy and safety data from the ongoing SONICS study in a randomized, double-blind, placebo-controlled study that will enroll approximately 35 patients, of which approximately two-thirds will have completed the SONICS study. LOGICS enrollment is anticipated to begin mid-year 2017 and top-line data are expected in Q3 2018. Additional detail regarding the LOGICS study will be provided upon initiation. “While recruitment in SONICS has been slower than anticipated, we have implemented several initiatives including additional site activations, new recruitment tactics, and protocol changes to improve enrollment rates,” said Fredric Cohen, M.D., chief medical officer of Strongbridge. “We believe that SONICS will provide scientifically-rigorous and clinically-relevant evidence characterizing the efficacy and safety of COR-003.  The addition of LOGICS will provide an opportunity to enhance the body of evidence for COR-003 by generating data in a well-controlled study,” Cohen added. COR-005 (veldoreotide) is a novel, investigational somatostatin analogue (SSA) with proven and differentiated pharmacology in pre-clinical models and patients with acromegaly. COR-005 may provide the acromegaly community with a new treatment option offering a unique clinical profile and delivery system. COR-005 has been granted orphan drug designation by the FDA and the European Medicines Agency (EMA). Strongbridge has completed the screening of potential long-acting release (LAR) technologies for COR-005, and selected a proprietary formulation based upon PLGA microspheres. PLGA is a well-known polymer, which has been widely applied in LAR formulations due to its biocompatibility, biodegradability, and favorable release kinetics. Announces $35 Million in Equity Financing; Finalizing $40 Million Credit Facility of which $20 Million to be Borrowed Initially, Extending Cash Runway at Least Through 2018 Strongbridge announced today in a separate press release that the Company has entered into a definitive securities purchase agreement for a  $35 million equity financing led by CAM Capital and Vivo Capital, with additional participation from Broadfin Capital, Boxer Capital of the Tavistock Group and HealthCap, as well as several new and existing institutional and individual investors. In addition to this equity financing, the Company is in the process of finalizing credit documentation for a $40 million credit facility, which the Company anticipates will close on or about December 28, 2016, and of which $20 million would be borrowed initially. Under the credit facility, the Company anticipates having access to two additional tranches of $10 million each, which would be available to the Company subject to the achievement of certain specified milestones. It is anticipated that the credit facility would mature after 48 months, provide interest-only payments initially for the first 18 months of the loan followed by an amortization period of 30 months, provide for a final payment fee equal to 8% of the amount borrowed, and bear interest at a rate equal to the sum of 8.22% plus the greater of 0.53% or the 30-day US LIBOR rate. It is also anticipated that the credit facility will provide that if the Company satisfies certain milestones and borrows the final $10 million tranche, the interest-only period would be extended by an additional six months and the amortization period would be 24 months. Under the credit facility, the Company would grant a security interest in substantially all of its existing and after-acquired assets, including intellectual property. The credit facility would contain facility and prepayment fees, and customary affirmative and negative covenants, and events of default. The Company’s pro forma cash and cash equivalents balance as of September 30, 2016 is $77.1 million, inclusive of estimated net proceeds from the equity financing and the initial $20 million borrowing under the credit facility. The Company believes it has sufficient financial resources, excluding any additional borrowings under the credit facility, to fund planned operations at least through 2018. For the three months ended June 30, 2016, net loss attributable to ordinary shareholders was $12.8 million, or $0.61 per basic and diluted share, compared to $17.1 million, or $1.19 per basic and diluted share, for the same period in the prior year. Research and development expenses were $4.6 million for the three months ended June 30, 2016, compared to $7.7 million for the same period in the prior year. Research and development expenses for the three months ended June 30, 2015 included $3.9 million of the $5.0 million in aggregate cash paid to Antisense Therapeutics upon entering into a license agreement in May 2015, with the remaining $1.1 million of cash paid recorded as the initial carrying value of our investment in the equity of Antisense Therapeutics. Research and development expenses for the three months ended June 30, 2016 included $1.8 million of increased expenses related to the global Phase 3 SONICS clinical trial and supporting development activities for COR‑003, formulation development activities for COR-005, and compensation and related personnel costs of increased employee count. These expenses were partially offset by a $1.0 million decrease in non-cash stock-based compensation costs resulting from the departure of certain research and development personnel. General and administrative expenses were $4.0 million for the three months ended June 30, 2016, compared to $9.5 million for the same period in the prior year. General and administrative expenses for the three months ended June 30, 2015 included $3.4 million of transaction fees and expenses related to the acquisition of COR-005 from Aspireo Pharmaceuticals, the license of COR-004 from Antisense Therapeutics, and other business development activities. General and administrative expenses for the three months ended June 30, 2015 also included $1.5 million of legal and accounting fees related to the indirect activities necessary to prepare the Company’s financial records for the U.S. initial public offering, which was completed in October 2015. General and administrative expenses for the three months ended June 30, 2016 included a $0.6 million decrease in non-cash stock based compensation costs due to liability-classified treatment of certain stock option awards in 2015. As a result of the termination of the license agreement between BioPancreate and Cornell Center for Technology Enterprise and Commercialization (CCTEC), we recorded an impairment charge related to the in-process research and development of $5.2 million for the three months ended June 30, 2016. For the three months ended September 30, 2016, net loss attributable to ordinary shareholders was $7.6 million, or $0.36 per basic and diluted share, compared to $9.7 million, or $0.51 per basic and diluted share, for the same period in the prior year. Research and development expenses were $4.5 million for the three months ended September 30, 2016, compared to $4.1 million for the same period in the prior year. The increase was primarily attributable to expenses related to the global Phase 3 SONICS clinical trial and supporting development activities for COR‑003, and compensation and related personnel costs of increased employee count. These expenses were partially offset by a decrease in development spending for the former COR-004 project. General and administrative expenses were $3.1 million for the three months ended September 30, 2016, compared to $5.5 million for the same period in the prior year.  General and administrative expenses for the three months ended September 30, 2015 included $1.7 million of legal and accounting fees related to the indirect activities necessary to prepare the Company’s financial records for the U.S. initial public offering, which was completed in October 2015. The remaining net decrease was primarily due to lower legal fees in support of general corporate matters, and lower employee recruiting fees and consulting fees for general business efforts. Partially offsetting these costs were higher cash compensation and non-cash stock based compensation costs due to increased employee count. For the nine months ended September 30, 2016, net loss attributable to ordinary shareholders was $32.7 million, or $1.54 per basic and diluted share, compared to $33.2 million, or $2.18 per basic and diluted share, for the same period in the prior year. Research and development expenses were $15.9 million for the nine months ended September 30, 2016, compared to $14.3 million for the same period in the prior year. Research and development expenses for the nine months ended September 30, 2015 included $3.9 million of the $5.0 million in aggregate cash paid to Antisense Therapeutics upon entering into a license agreement in May 2015, with the remaining $1.1 million of cash paid recorded as the initial carrying value of our investment in the equity of Antisense Therapeutics. The remaining net increase was primarily attributable to expenses related to the global Phase 3 SONICS clinical trial and supporting development activities for COR‑003, development spending for the former COR-004 project, formulation development activities for COR-005, and compensation and related personnel costs on increased employee count. These expenses were partially offset by a decrease in non-cash stock-based compensation costs due to the departure of certain research and development employees. General and administrative expenses were $11.3 million for the nine months ended September 30, 2016, compared to $18.2 million for the same period in the prior year. General and administrative expenses for the nine months ended September 30, 2015 included $3.7 million of legal and accounting fees related to the indirect activities necessary to prepare the Company’s financial records for the U.S. initial public offering, which was completed in October 2015. General and administrative expenses for the nine months ended September 30, 2015 also included $3.6 million of transaction fees and expenses related to the acquisition of COR-005 from Aspireo Pharmaceuticals, the license of COR-004 from Antisense Therapeutics, and other business development activities. The remaining net increase was primarily due to higher cash compensation and non-cash stock based compensation costs due to increased employee count, partially offset by decreased legal fees in support of general corporate matters, lower employee recruiting fees and consulting fees for general business efforts. As a result of the termination of the license agreement between BioPancreate and Cornell Center for Technology Enterprise and Commercialization (CCTEC), we recorded an impairment charge related to the In-process research and development of $5.2 million for the nine months ended September 30, 2016. About Strongbridge Biopharma Strongbridge Biopharma is a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs. Strongbridge's first commercial product is KEVEYIS® (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis. KEVEYIS has orphan drug exclusivity status in the U.S. through August 7, 2022. In addition to establishing this neuromuscular disease franchise, the Company has a clinical-stage pipeline of therapies for rare endocrine diseases. Strongbridge's lead compounds include COR-003 (levoketoconazole), a cortisol synthesis inhibitor currently being studied for the treatment of endogenous Cushing's syndrome, and COR-005, a next-generation somatostatin analog (SSA) being investigated for the treatment of acromegaly, with potential additional applications in Cushing's syndrome and neuroendocrine tumors. Both COR-003 and COR-005 have received orphan designation from the U.S. Food and Drug Administration and the European Medicines Agency. For more information, visit www.strongbridgebio.com. KEVEYIS® Indication KEVEYIS® (dichlorphenamide) is indicated for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants. KEVEYIS Important Safety Information In clinical studies, the most common side effects of KEVEYIS were a numbness or tingling, difficulty thinking and paying attention, changes in taste, and confusion. These are not all of the possible side effects that you may experience with KEVEYIS. Talk to your doctor if you have any symptoms that bother you or do not go away. KEVEYIS is not for everyone. Do not take KEVEYIS if you: Taking KEVEYIS may cause a drop in the amount of potassium (an electrolyte) in your body, which can lead to heart problems. Ask your doctor if you need to eat foods that contain high amounts of potassium while taking KEVEYIS. Your body may produce too much acid or may not be able to remove enough acid from body fluids while taking KEVEYIS. Your doctor will run tests on a regular basis to check for signs of acid buildup and may reduce your dose or stop your treatment with KEVEYIS. KEVEYIS may also increase the risk of falls, especially in elderly patients and patients taking high doses of KEVEYIS. Use caution when driving, operating machinery, or performing any other hazardous activities while taking KEVEYIS, as this medication may cause drowsiness. You are encouraged to report side effects to Strongbridge Biopharma at 1-855-324-8912, or to the FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch/. For more information, go to www.keveyis.com. For additional KEVEYIS important safety information, please see full prescribing information at www.keveyis.com. KEVEYIS® is a registered trademark licensed exclusively in the U.S. to Strongbridge Biopharma plc. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties.  All statements, other than statements of historical facts, contained in this press release, are forward-looking statements.  These statements relate to future events and involve known and unknown risks, including, without limitation, uncertainties regarding Strongbridge's strategy, plans, future financial position, anticipated investments, costs and results, outcomes of product development efforts, status and results of clinical trials and objectives of management for future operations.  The words "anticipate," "estimate," "expect," "intend," "may," "plan," "potential," "project," "target," "will," "would," or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.  These forward-looking statements are based on current expectations, estimates, forecasts and projections and are not guarantees of future performance or development and involve known and unknown risks, uncertainties and other factors.  The forward-looking statements contained in this press release are made as of the date of this press release, and Strongbridge Biopharma does not assume any obligation to update any forward-looking statements except as required by applicable law.


News Article | December 23, 2016
Site: globenewswire.com

~ Expands Rare Disease Portfolio through Acquisition of U.S. Rights to KEVEYIS®, the First and Only FDA-Approved Treatment for Hyperkalemic, Hypokalemic, and Related Variants of Primary Periodic Paralysis ~ ~ Provides Update on Timelines and Strengthens COR-003 Clinical Development Program with Additional Phase 3 Study ~ ~ Announces $35 Million in Equity Financing; Finalizing $40 Million Credit Facility of which $20 Million to be Borrowed Initially, Extending Cash Runway at Least Through 2018 ~ DUBLIN, Ireland and TREVOSE, Pa., Dec. 23, 2016 (GLOBE NEWSWIRE) -- Strongbridge Biopharma plc, (Nasdaq:SBBP), a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs, today announced a corporate and financial update. “Throughout the second half of 2016, Strongbridge has been focused on building value in three key areas: enhancing the clinical development program and the probability of regulatory success for COR-003, pursuing a commercial-stage rare disease therapy resulting in the acquisition of the U.S. rights to KEVEYIS® (dichlorphenamide), and strengthening the financial condition of the Company,” said Matthew Pauls, president and chief executive officer of Strongbridge. “Executing on these initiatives strengthens our presence in rare diseases and sets the stage for a number of value-creating events in 2017, including commercial sales of KEVEYIS and completing enrollment in the SONICS study,” Pauls added. Expands Rare Disease Portfolio through Acquisition of U.S. Rights to KEVEYIS®, the First and Only FDA-Approved Treatment for Hyperkalemic, Hypokalemic, and Related Variants of Primary Periodic Paralysis Earlier today, Strongbridge announced in a separate press release that the Company has acquired the U.S. rights to KEVEYIS, the first and only U.S. Food and Drug Administration (FDA)-approved product to treat hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis, which is a group of rare hereditary disorders that causes potentially severe episodes of muscle weakness and/or paralysis. KEVEYIS is Strongbridge’s first commercial product, and expands the Company’s rare disease franchises to include both neuromuscular and endocrine diseases. Provides Update on Timelines and Strengthens COR-003 Clinical Development Program with Additional Phase 3 Study The global Phase 3 SONICS clinical trial evaluating COR-003 (levoketoconazole) for the treatment of endogenous Cushing’s syndrome is more than two-thirds enrolled. Based upon an analysis of recent enrollment trends, Strongbridge anticipates that the study will be fully enrolled in Q2 2017, with top-line data for the primary efficacy analysis available in Q1 2018. In October 2016, at the fourth semi-annual Data and Safety Monitoring Board (DSMB) review of the SONICS data, the DSMB recommended that the SONICS study continue as planned. In addition, based upon Strongbridge’s ongoing dialog with the FDA and observation of the current regulatory landscape, the Company plans to initiate LOGICS, a second global Phase 3 study of COR-003 for the treatment of endogenous Cushing's syndrome. The LOGICS study will supplement the long-term efficacy and safety data from the ongoing SONICS study in a randomized, double-blind, placebo-controlled study that will enroll approximately 35 patients, of which approximately two-thirds will have completed the SONICS study. LOGICS enrollment is anticipated to begin mid-year 2017 and top-line data are expected in Q3 2018. Additional detail regarding the LOGICS study will be provided upon initiation. “While recruitment in SONICS has been slower than anticipated, we have implemented several initiatives including additional site activations, new recruitment tactics, and protocol changes to improve enrollment rates,” said Fredric Cohen, M.D., chief medical officer of Strongbridge. “We believe that SONICS will provide scientifically-rigorous and clinically-relevant evidence characterizing the efficacy and safety of COR-003.  The addition of LOGICS will provide an opportunity to enhance the body of evidence for COR-003 by generating data in a well-controlled study,” Cohen added. COR-005 (veldoreotide) is a novel, investigational somatostatin analogue (SSA) with proven and differentiated pharmacology in pre-clinical models and patients with acromegaly. COR-005 may provide the acromegaly community with a new treatment option offering a unique clinical profile and delivery system. COR-005 has been granted orphan drug designation by the FDA and the European Medicines Agency (EMA). Strongbridge has completed the screening of potential long-acting release (LAR) technologies for COR-005, and selected a proprietary formulation based upon PLGA microspheres. PLGA is a well-known polymer, which has been widely applied in LAR formulations due to its biocompatibility, biodegradability, and favorable release kinetics. Announces $35 Million in Equity Financing; Finalizing $40 Million Credit Facility of which $20 Million to be Borrowed Initially, Extending Cash Runway at Least Through 2018 Strongbridge announced today in a separate press release that the Company has entered into a definitive securities purchase agreement for a  $35 million equity financing led by CAM Capital and Vivo Capital, with additional participation from Broadfin Capital, Boxer Capital of the Tavistock Group and HealthCap, as well as several new and existing institutional and individual investors. In addition to this equity financing, the Company is in the process of finalizing credit documentation for a $40 million credit facility, which the Company anticipates will close on or about December 28, 2016, and of which $20 million would be borrowed initially. Under the credit facility, the Company anticipates having access to two additional tranches of $10 million each, which would be available to the Company subject to the achievement of certain specified milestones. It is anticipated that the credit facility would mature after 48 months, provide interest-only payments initially for the first 18 months of the loan followed by an amortization period of 30 months, provide for a final payment fee equal to 8% of the amount borrowed, and bear interest at a rate equal to the sum of 8.22% plus the greater of 0.53% or the 30-day US LIBOR rate. It is also anticipated that the credit facility will provide that if the Company satisfies certain milestones and borrows the final $10 million tranche, the interest-only period would be extended by an additional six months and the amortization period would be 24 months. Under the credit facility, the Company would grant a security interest in substantially all of its existing and after-acquired assets, including intellectual property. The credit facility would contain facility and prepayment fees, and customary affirmative and negative covenants, and events of default. The Company’s pro forma cash and cash equivalents balance as of September 30, 2016 is $77.1 million, inclusive of estimated net proceeds from the equity financing and the initial $20 million borrowing under the credit facility. The Company believes it has sufficient financial resources, excluding any additional borrowings under the credit facility, to fund planned operations at least through 2018. For the three months ended June 30, 2016, net loss attributable to ordinary shareholders was $12.8 million, or $0.61 per basic and diluted share, compared to $17.1 million, or $1.19 per basic and diluted share, for the same period in the prior year. Research and development expenses were $4.6 million for the three months ended June 30, 2016, compared to $7.7 million for the same period in the prior year. Research and development expenses for the three months ended June 30, 2015 included $3.9 million of the $5.0 million in aggregate cash paid to Antisense Therapeutics upon entering into a license agreement in May 2015, with the remaining $1.1 million of cash paid recorded as the initial carrying value of our investment in the equity of Antisense Therapeutics. Research and development expenses for the three months ended June 30, 2016 included $1.8 million of increased expenses related to the global Phase 3 SONICS clinical trial and supporting development activities for COR‑003, formulation development activities for COR-005, and compensation and related personnel costs of increased employee count. These expenses were partially offset by a $1.0 million decrease in non-cash stock-based compensation costs resulting from the departure of certain research and development personnel. General and administrative expenses were $4.0 million for the three months ended June 30, 2016, compared to $9.5 million for the same period in the prior year. General and administrative expenses for the three months ended June 30, 2015 included $3.4 million of transaction fees and expenses related to the acquisition of COR-005 from Aspireo Pharmaceuticals, the license of COR-004 from Antisense Therapeutics, and other business development activities. General and administrative expenses for the three months ended June 30, 2015 also included $1.5 million of legal and accounting fees related to the indirect activities necessary to prepare the Company’s financial records for the U.S. initial public offering, which was completed in October 2015. General and administrative expenses for the three months ended June 30, 2016 included a $0.6 million decrease in non-cash stock based compensation costs due to liability-classified treatment of certain stock option awards in 2015. As a result of the termination of the license agreement between BioPancreate and Cornell Center for Technology Enterprise and Commercialization (CCTEC), we recorded an impairment charge related to the in-process research and development of $5.2 million for the three months ended June 30, 2016. For the three months ended September 30, 2016, net loss attributable to ordinary shareholders was $7.6 million, or $0.36 per basic and diluted share, compared to $9.7 million, or $0.51 per basic and diluted share, for the same period in the prior year. Research and development expenses were $4.5 million for the three months ended September 30, 2016, compared to $4.1 million for the same period in the prior year. The increase was primarily attributable to expenses related to the global Phase 3 SONICS clinical trial and supporting development activities for COR‑003, and compensation and related personnel costs of increased employee count. These expenses were partially offset by a decrease in development spending for the former COR-004 project. General and administrative expenses were $3.1 million for the three months ended September 30, 2016, compared to $5.5 million for the same period in the prior year.  General and administrative expenses for the three months ended September 30, 2015 included $1.7 million of legal and accounting fees related to the indirect activities necessary to prepare the Company’s financial records for the U.S. initial public offering, which was completed in October 2015. The remaining net decrease was primarily due to lower legal fees in support of general corporate matters, and lower employee recruiting fees and consulting fees for general business efforts. Partially offsetting these costs were higher cash compensation and non-cash stock based compensation costs due to increased employee count. For the nine months ended September 30, 2016, net loss attributable to ordinary shareholders was $32.7 million, or $1.54 per basic and diluted share, compared to $33.2 million, or $2.18 per basic and diluted share, for the same period in the prior year. Research and development expenses were $15.9 million for the nine months ended September 30, 2016, compared to $14.3 million for the same period in the prior year. Research and development expenses for the nine months ended September 30, 2015 included $3.9 million of the $5.0 million in aggregate cash paid to Antisense Therapeutics upon entering into a license agreement in May 2015, with the remaining $1.1 million of cash paid recorded as the initial carrying value of our investment in the equity of Antisense Therapeutics. The remaining net increase was primarily attributable to expenses related to the global Phase 3 SONICS clinical trial and supporting development activities for COR‑003, development spending for the former COR-004 project, formulation development activities for COR-005, and compensation and related personnel costs on increased employee count. These expenses were partially offset by a decrease in non-cash stock-based compensation costs due to the departure of certain research and development employees. General and administrative expenses were $11.3 million for the nine months ended September 30, 2016, compared to $18.2 million for the same period in the prior year. General and administrative expenses for the nine months ended September 30, 2015 included $3.7 million of legal and accounting fees related to the indirect activities necessary to prepare the Company’s financial records for the U.S. initial public offering, which was completed in October 2015. General and administrative expenses for the nine months ended September 30, 2015 also included $3.6 million of transaction fees and expenses related to the acquisition of COR-005 from Aspireo Pharmaceuticals, the license of COR-004 from Antisense Therapeutics, and other business development activities. The remaining net increase was primarily due to higher cash compensation and non-cash stock based compensation costs due to increased employee count, partially offset by decreased legal fees in support of general corporate matters, lower employee recruiting fees and consulting fees for general business efforts. As a result of the termination of the license agreement between BioPancreate and Cornell Center for Technology Enterprise and Commercialization (CCTEC), we recorded an impairment charge related to the In-process research and development of $5.2 million for the nine months ended September 30, 2016. About Strongbridge Biopharma Strongbridge Biopharma is a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs. Strongbridge's first commercial product is KEVEYIS® (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis. KEVEYIS has orphan drug exclusivity status in the U.S. through August 7, 2022. In addition to establishing this neuromuscular disease franchise, the Company has a clinical-stage pipeline of therapies for rare endocrine diseases. Strongbridge's lead compounds include COR-003 (levoketoconazole), a cortisol synthesis inhibitor currently being studied for the treatment of endogenous Cushing's syndrome, and COR-005, a next-generation somatostatin analog (SSA) being investigated for the treatment of acromegaly, with potential additional applications in Cushing's syndrome and neuroendocrine tumors. Both COR-003 and COR-005 have received orphan designation from the U.S. Food and Drug Administration and the European Medicines Agency. For more information, visit www.strongbridgebio.com. KEVEYIS® Indication KEVEYIS® (dichlorphenamide) is indicated for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants. KEVEYIS Important Safety Information In clinical studies, the most common side effects of KEVEYIS were a numbness or tingling, difficulty thinking and paying attention, changes in taste, and confusion. These are not all of the possible side effects that you may experience with KEVEYIS. Talk to your doctor if you have any symptoms that bother you or do not go away. KEVEYIS is not for everyone. Do not take KEVEYIS if you: Taking KEVEYIS may cause a drop in the amount of potassium (an electrolyte) in your body, which can lead to heart problems. Ask your doctor if you need to eat foods that contain high amounts of potassium while taking KEVEYIS. Your body may produce too much acid or may not be able to remove enough acid from body fluids while taking KEVEYIS. Your doctor will run tests on a regular basis to check for signs of acid buildup and may reduce your dose or stop your treatment with KEVEYIS. KEVEYIS may also increase the risk of falls, especially in elderly patients and patients taking high doses of KEVEYIS. Use caution when driving, operating machinery, or performing any other hazardous activities while taking KEVEYIS, as this medication may cause drowsiness. You are encouraged to report side effects to Strongbridge Biopharma at 1-855-324-8912, or to the FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch/. For more information, go to www.keveyis.com. For additional KEVEYIS important safety information, please see full prescribing information at www.keveyis.com. KEVEYIS® is a registered trademark licensed exclusively in the U.S. to Strongbridge Biopharma plc. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties.  All statements, other than statements of historical facts, contained in this press release, are forward-looking statements.  These statements relate to future events and involve known and unknown risks, including, without limitation, uncertainties regarding Strongbridge's strategy, plans, future financial position, anticipated investments, costs and results, outcomes of product development efforts, status and results of clinical trials and objectives of management for future operations.  The words "anticipate," "estimate," "expect," "intend," "may," "plan," "potential," "project," "target," "will," "would," or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.  These forward-looking statements are based on current expectations, estimates, forecasts and projections and are not guarantees of future performance or development and involve known and unknown risks, uncertainties and other factors.  The forward-looking statements contained in this press release are made as of the date of this press release, and Strongbridge Biopharma does not assume any obligation to update any forward-looking statements except as required by applicable law.


HAYWARD, Calif., Dec. 06, 2016 (GLOBE NEWSWIRE) -- Anthera Pharmaceuticals, Inc. (Nasdaq:ANTH) today announced positive trends from the Week 48 analysis of the Phase 2 BRIGHT-SC proof-of-concept study in 57 patients with IgA nephropathy. The effects of blisibimod versus placebo were assessed through at least the 48 week time point in all patients in the BRIGHT-SC study.   Patients enrolled in the BRIGHT-SC study had biopsy-proven IgA nephropathy with a mean proteinuria level of 2.4 grams and an estimated glomerular filtration rate of less than 70 mL/min/1.73m2 – indicative of stage 2 chronic kidney disease per the National Kidney Foundation.  Patients were on background angiotensin converting enzyme inhibitors or receptor blockers, but corticosteroids were not permitted during the trial. A positive trend on proteinuria of blisibimod in patients with biopsy-proven IgA nephropathy was observed. Consistent with the previously announced Week 24 analysis, blisibimod treated-patients over time demonstrated stable to slightly decreasing levels of estimated 24 hour urinary protein excretion, as assessed by urinary protein to creatinine ratio (PCR), as compared to slowly increasing levels of proteinuria in the placebo group. 44 of the original 57 patients had a Week 48 observation and 22 patients had a Week 96 observation at the time of this analysis. Total B cell counts and serum immunoglobulins IgA, IgG, and IgM, continued to demonstrate marked reduction, consistent with data from the Week 24 analysis as well as other studies with blisibimod. No safety or tolerability concerns were observed with blisibimod during routine reviews of the unblinded trial data by the study’s independent Data and Safety Monitoring Board. “The sustained effects of blisibimod versus placebo on proteinuria after up to 2 years of treatment are very encouraging for IgA nephropathy patients, for whom no treatments exist,” said Jonathan Barratt, MD, Reader in the Department of Infection, Immunity & Inflammation, University of Leicester, Honorary Consultant Nephrologist at Leicester General Hospital, and Head of the Postgraduate Specialty School of Clinical Academic Training at Health Education East Midlands. “We look forward to receiving longer term data from the study which will provide more conclusive assessment of blisibimod on the progression of kidney disease.” “The Week 48 observations from the BRIGHT-SC study continue to demonstrate consistent pharmacological effects on B cells and serum immunoglobulins, and suggest an effect on proteinuria,” said William Shanahan, MD, Anthera’s Chief Medical Officer. “We believe proteinuria is a robust surrogate biomarker for IgA nephropathy disease activity, and we remain optimistic that a longer-term evaluation in greater numbers of patients could provide further validation of blisibimod’s effects on IgA nephropathy by demonstrating continuing effects on proteinuria and preservation of glomerular function.” The full dataset from this study will be submitted and presented at an upcoming scientific conference. The study enrolled 57 patients, 44 of whom completed assessments through a minimum of 48 weeks. Patients with persistent proteinuria (1-6 g/24hrs) prior to enrollment were randomized to receive either blisibimod (300mg/wk for 8 weeks and 200mg/wk thereafter) or matching placebo over background angiotensin converting enzyme inhibitors (ACEi) or angiotensin receptor blockers (ARB) for up to 104 weeks and are followed thereafter in the absence of study drug to assess longer term outcome. All patients were treated with an optimal dose of ACEi or ARB for a minimum of 90 days prior to randomization and this therapy was continued throughout the trial as background medication for all patients. Steroid intervention was not permitted during the trial. The BRIGHT-SC study was originally designed as a phase 2/3 study with a target enrollment of 200 patients with renal biopsy verified IgA nephropathy, 24 hour urine protein excretion of 1-6 grams by urinary protein to creatinine ratio (PCR), and an estimated glomerular filtration rate (eGFR) of more than 30 ml/min/1.73 m2 of body surface area.  The original primary endpoint of the study was the number of patients who achieved a partial or complete response in urinary protein excretion at Week 24. A partial response is defined as achieving proteinuria ≤1g/24hrs, and a complete response as follows: for patients with baseline proteinuria ≥1g/24hrs but ≤2g/24hrs, achievement of proteinuria ≤1.0g/24hr AND a 50% reduction from baseline at 2 consecutive visits; for patients with baseline proteinuria >2g/24hrs, achievement of proteinuria ≤1.0g/24hr OR a 50% reduction from baseline at 2 consecutive visits. Due to slow recruitment, enrollment was curtailed at 57 and the study was converted to phase 2. An observed case analysis was conducted when all patients had the opportunity to complete Week 24 and topline results were previously announced.  Based upon the Week 24 results, applications for Orphan Drug and Breakthrough Status have been filed with the FDA, with responses expected by late 1Q2017. IgA nephropathy (IgAN, also known as Berger’s disease) is the most common cause of primary glomerulonephritis worldwide, occurring more frequently in Asia than in Europe or North America. Patients express under-glycosylated immunoglobulin A1 (IgA1) which is immunogenic and targeted by other immunoglobulins.  The resulting IgA-containing immune complexes are deposited in the kidney, causing inflammation with consequent blood and protein leakage into the urine. The disease typically progresses slowly, but as many as 40-50% of adults will eventually develop end-stage-renal disease and require dialysis or kidney transplant. The current management of IgAN is non-specific treatment aimed at blood pressure control and reduction of proteinuria with angiotensin converting enzyme inhibitors (ACEI) and/or angiotensin II receptor blockers (ARBs); corticosteroids and immunosuppressive therapy are used in some patients but benefits are uncertain. Anthera Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing products to treat serious and life-threatening diseases, including lupus, lupus with glomerulonephritis, IgA nephropathy, and exocrine pancreatic insufficiency due to cystic fibrosis. Additional information on the Company can be found at www.anthera.com. Any statements contained in this press release that refer to future events or other non-historical matters, including statements that are preceded by, followed by, or that include such words as "estimate," "intend," "anticipate," "believe," "plan," "goal," "expect," "project," or similar statements, are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are based on Anthera's expectations as of the date of this press release and are subject to certain risks and uncertainties that could cause actual results to differ materially as set forth in Anthera's public filings with the SEC, including Anthera's Quarterly Report on Form 10-Q for the quarter ended September 30, 2016. Anthera disclaims any intent or obligation to update any forward-looking statements, whether because of new information, future events or otherwise, except as required by applicable law.


HAYWARD, Calif., Dec. 06, 2016 (GLOBE NEWSWIRE) -- Anthera Pharmaceuticals, Inc. (Nasdaq:ANTH) today announced positive trends from the Week 48 analysis of the Phase 2 BRIGHT-SC proof-of-concept study in 57 patients with IgA nephropathy. The effects of blisibimod versus placebo were assessed through at least the 48 week time point in all patients in the BRIGHT-SC study.   Patients enrolled in the BRIGHT-SC study had biopsy-proven IgA nephropathy with a mean proteinuria level of 2.4 grams and an estimated glomerular filtration rate of less than 70 mL/min/1.73m2 – indicative of stage 2 chronic kidney disease per the National Kidney Foundation.  Patients were on background angiotensin converting enzyme inhibitors or receptor blockers, but corticosteroids were not permitted during the trial. A positive trend on proteinuria of blisibimod in patients with biopsy-proven IgA nephropathy was observed. Consistent with the previously announced Week 24 analysis, blisibimod treated-patients over time demonstrated stable to slightly decreasing levels of estimated 24 hour urinary protein excretion, as assessed by urinary protein to creatinine ratio (PCR), as compared to slowly increasing levels of proteinuria in the placebo group. 44 of the original 57 patients had a Week 48 observation and 22 patients had a Week 96 observation at the time of this analysis. Total B cell counts and serum immunoglobulins IgA, IgG, and IgM, continued to demonstrate marked reduction, consistent with data from the Week 24 analysis as well as other studies with blisibimod. No safety or tolerability concerns were observed with blisibimod during routine reviews of the unblinded trial data by the study’s independent Data and Safety Monitoring Board. “The sustained effects of blisibimod versus placebo on proteinuria after up to 2 years of treatment are very encouraging for IgA nephropathy patients, for whom no treatments exist,” said Jonathan Barratt, MD, Reader in the Department of Infection, Immunity & Inflammation, University of Leicester, Honorary Consultant Nephrologist at Leicester General Hospital, and Head of the Postgraduate Specialty School of Clinical Academic Training at Health Education East Midlands. “We look forward to receiving longer term data from the study which will provide more conclusive assessment of blisibimod on the progression of kidney disease.” “The Week 48 observations from the BRIGHT-SC study continue to demonstrate consistent pharmacological effects on B cells and serum immunoglobulins, and suggest an effect on proteinuria,” said William Shanahan, MD, Anthera’s Chief Medical Officer. “We believe proteinuria is a robust surrogate biomarker for IgA nephropathy disease activity, and we remain optimistic that a longer-term evaluation in greater numbers of patients could provide further validation of blisibimod’s effects on IgA nephropathy by demonstrating continuing effects on proteinuria and preservation of glomerular function.” The full dataset from this study will be submitted and presented at an upcoming scientific conference. The study enrolled 57 patients, 44 of whom completed assessments through a minimum of 48 weeks. Patients with persistent proteinuria (1-6 g/24hrs) prior to enrollment were randomized to receive either blisibimod (300mg/wk for 8 weeks and 200mg/wk thereafter) or matching placebo over background angiotensin converting enzyme inhibitors (ACEi) or angiotensin receptor blockers (ARB) for up to 104 weeks and are followed thereafter in the absence of study drug to assess longer term outcome. All patients were treated with an optimal dose of ACEi or ARB for a minimum of 90 days prior to randomization and this therapy was continued throughout the trial as background medication for all patients. Steroid intervention was not permitted during the trial. The BRIGHT-SC study was originally designed as a phase 2/3 study with a target enrollment of 200 patients with renal biopsy verified IgA nephropathy, 24 hour urine protein excretion of 1-6 grams by urinary protein to creatinine ratio (PCR), and an estimated glomerular filtration rate (eGFR) of more than 30 ml/min/1.73 m2 of body surface area.  The original primary endpoint of the study was the number of patients who achieved a partial or complete response in urinary protein excretion at Week 24. A partial response is defined as achieving proteinuria ≤1g/24hrs, and a complete response as follows: for patients with baseline proteinuria ≥1g/24hrs but ≤2g/24hrs, achievement of proteinuria ≤1.0g/24hr AND a 50% reduction from baseline at 2 consecutive visits; for patients with baseline proteinuria >2g/24hrs, achievement of proteinuria ≤1.0g/24hr OR a 50% reduction from baseline at 2 consecutive visits. Due to slow recruitment, enrollment was curtailed at 57 and the study was converted to phase 2. An observed case analysis was conducted when all patients had the opportunity to complete Week 24 and topline results were previously announced.  Based upon the Week 24 results, applications for Orphan Drug and Breakthrough Status have been filed with the FDA, with responses expected by late 1Q2017. IgA nephropathy (IgAN, also known as Berger’s disease) is the most common cause of primary glomerulonephritis worldwide, occurring more frequently in Asia than in Europe or North America. Patients express under-glycosylated immunoglobulin A1 (IgA1) which is immunogenic and targeted by other immunoglobulins.  The resulting IgA-containing immune complexes are deposited in the kidney, causing inflammation with consequent blood and protein leakage into the urine. The disease typically progresses slowly, but as many as 40-50% of adults will eventually develop end-stage-renal disease and require dialysis or kidney transplant. The current management of IgAN is non-specific treatment aimed at blood pressure control and reduction of proteinuria with angiotensin converting enzyme inhibitors (ACEI) and/or angiotensin II receptor blockers (ARBs); corticosteroids and immunosuppressive therapy are used in some patients but benefits are uncertain. Anthera Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing products to treat serious and life-threatening diseases, including lupus, lupus with glomerulonephritis, IgA nephropathy, and exocrine pancreatic insufficiency due to cystic fibrosis. Additional information on the Company can be found at www.anthera.com. Any statements contained in this press release that refer to future events or other non-historical matters, including statements that are preceded by, followed by, or that include such words as "estimate," "intend," "anticipate," "believe," "plan," "goal," "expect," "project," or similar statements, are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are based on Anthera's expectations as of the date of this press release and are subject to certain risks and uncertainties that could cause actual results to differ materially as set forth in Anthera's public filings with the SEC, including Anthera's Quarterly Report on Form 10-Q for the quarter ended September 30, 2016. Anthera disclaims any intent or obligation to update any forward-looking statements, whether because of new information, future events or otherwise, except as required by applicable law.


News Article | February 23, 2017
Site: globenewswire.com

TEL-AVIV, Israel, Feb. 23, 2017 (GLOBE NEWSWIRE) -- RedHill Biopharma Ltd. (NASDAQ:RDHL) (TASE:RDHL) (“RedHill” or the “Company”), a specialty biopharmaceutical company primarily focused on the development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for gastrointestinal and inflammatory diseases and cancer, today reported its financial results for the fourth quarter and full-year ended December 31, 2016. Revenues for the fourth quarter of 2016 were $0.1 million, compared to immaterial revenues for the fourth quarter of 2015. Research and Development Expenses for the fourth quarter of 2016 were $7.5 million, up 51% compared to the fourth quarter of 2015. The increase was mainly due to the ongoing Phase III and Phase II studies with BEKINDA® for gastroenteritis and IBS-D, respectively, the ongoing Phase III study with RHB-104 for Crohn’s disease and ongoing studies with YELIVA® for multiple indications. General, Administrative and Business Development Expenses for the fourth quarter of 2016 were $1.6 million, down 6.9% compared to the fourth quarter of 2015. The decrease was mainly due to a decrease in professional services. Operating Loss for the fourth quarter of 2016 was $9 million, up 33% compared to the fourth quarter of 2015. The increase was mainly due to an increase in research and development expenses, as detailed above. Financial Income, net for the fourth quarter of 2016 was $0.6 million, up 214%, compared to the fourth quarter of 2015. The increase was mainly due to a fair value gain on derivative financial instruments. Net Cash Used in Operating Activities for the fourth quarter of 2016 was $10.1 million, up 69% compared to the fourth quarter of 2015. The increase was mainly due to the increase in operating loss, as detailed above. Net Cash Provided by Investment Activities for the fourth quarter of 2016 was $21.3 million, up 206% compared to the fourth quarter of 2015. The increase was mainly due to maturity of bank deposits. Net Cash Provided by Financing Activities for the fourth quarter of 2016 was $35.9 million compared to an immaterial amount for the fourth quarter of 2015. The increase was mainly due to the December 2016 public offering. Revenues for 2016 were $0.1 million, compared to immaterial revenues in 2015. Research and Development Expenses for 2016 were $25.2 million, up 42% compared to 2015. The increase was mainly due to the ongoing Phase III MAP US study with RHB-104 for Crohn's disease, the ongoing Phase III and Phase II studies with BEKINDA® for gastroenteritis and IBS-D, respectively, and the ongoing studies with YELIVA® for multiple indications. General, Administrative and Business Development Expenses for 2016 were $5.4 million, up 31% compared to 2015. The increase was mainly due to an increase in professional services, compensation and other operating expenses. Operating Loss for 2016 was $30.5 million, up 39% compared to 2015. The increase was mainly due to an increase in research and development expenses, as detailed above. Financial Income, net for 2016 was $1.2 million, up 29% compared to 2015. The increase was mainly due to a fair value gain on derivative financial instruments. Net Cash Used in Operating Activities for 2016 was $28.2 million, up 59% compared to 2015. The increase was mainly due to an increase in operating loss, as detailed above. Net Cash Provided by Investment Activities for 2016 was $24.5 million, up 215% compared to 2015. The difference was mainly due to maturity of bank deposits. Net Cash Provided by Financing Activities for 2016 was $36 million, down 34% compared to 2015. The decrease resulted primarily from the two public offerings in February and July 2015 of the comparable period. Cash Balance3 as of December 31, 2016 was $66.3 million, an increase of $8.2 million compared to $58.1 million as of December 31, 2015 and an increase of $25.8 million compared to $40.5 million as of September 30, 2016. Micha Ben Chorin, RedHill’s CFO, said: “Our strong cash position of approximately $66 million at the end of 2016 should allow us to continue to execute our strategic plans for 2017. We are looking forward to an important year ahead, including the planned initiation of a confirmatory Phase III study with RHB-105 for H. pylori infection, a second independent DSMB meeting for the ongoing MAP US Phase III study with RHB-104 for Crohn’s disease, top-line results from the ongoing Phase III and Phase II studies with BEKINDA® for gastroenteritis and IBS-D, respectively, and commencement of our promotional activities in the U.S. with Donnatal®." The Company will host a conference call on Thursday, February 23, 2017, at 9:00 am EST to review the financial results and business highlights. To participate in the conference call, please dial the following numbers 5-10 minutes prior to the start of the call: United States: +1-877-280-1254; International: +1-646-254-3366; and Israel: +972-3-763-0145. The access code for the call is 4402478. The conference call will be broadcasted live and available for replay on the Company's website, http://ir.redhillbio.com/events.cfm, for 30 days. Please access the Company's website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software. RHB-105 - H. pylori bacterial infection (confirmatory Phase III) (QIDP status)         Following the announcement of the successful final results from a first Phase III clinical study with RHB-105 for the eradication of H. pylori infection (the ERADICATE Hp study) in March 2016, RedHill concluded two positive Type B meetings with the U.S. Food and Drug Administration (FDA) regarding RHB-105. The first meeting, announced in April 2016, confirmed the path to marketing approval of RHB-105 and the planned confirmatory Phase III study. A second Type B meeting, announced in November 2016, discussed the chemistry, manufacturing and controls (CMC) aspects of the RHB-105 Phase III development program towards filing the CMC package as part of the potential U.S. New Drug Application (NDA) to be submitted for RHB-105, subject to successful completion of the planned confirmatory Phase III study. The two-arm, randomized, double-blind, active comparator confirmatory Phase III study, comparing RHB-105 against a dual therapy amoxicillin and omeprazole regimen at equivalent doses, is planned to be initiated in the second quarter of 2017, subject to the successful completion of the ongoing supportive pharmacokinetic (PK) program and submission of the Clinical Study Report to the FDA. The confirmatory Phase III study is planned to enroll approximately 440 patients in up to 55 clinical sites in the U.S. In October 2016, RedHill provided an update on the RHB-104 Phase III Crohn’s disease development program, planned enhancements to the ongoing MAP US Phase III study and expected milestones, including an increase in the total number of patients planned to be enrolled in the MAP US study from 270 to 410, and the addition of an open-label extension study offering patients who complete 26 weeks of study participation and remain out of remission (Crohn’s disease active index (CDAI) >150) the opportunity to receive treatment with RHB-104 for a 52-week period. The open-label extension study is expected to be initiated in the coming weeks. Following a pre-planned review of safety data from its ongoing MAP US study by an independent Data and Safety Monitoring Board (DSMB), RedHill announced in December 2016 that it had received a unanimous recommendation to continue the MAP US study as planned. A second independent DSMB meeting of the MAP US study, expected in the second quarter of 2017, will include an interim efficacy analysis and will evaluate the option for an early stop for success for overwhelming efficacy, according to a pre-specified statistical significance threshold. Taking into account the increase in the total number of patients planned in the MAP US study, and assuming the MAP US study is not stopped for success or inefficacy following the independent DSMB meeting in the second quarter of 2017, completion of recruitment is expected by the end of 2017. In December 2016, RedHill announced encouraging top-line final results of a Phase IIa, proof-of-concept clinical study, evaluating RHB-104 as an add-on therapy to interferon beta-1a in patients treated for relapsing remitting multiple sclerosis (the CEASE MS study). The top-line final results (48 weeks) were consistent with previously announced interim results, suggesting meaningful positive safety and clinical signals upon 24 weeks of treatment with RHB-104 as an add-on therapy, thereby supporting further clinical development. In January 2017, RedHill announced that RHB-104 had been granted Qualified Infectious Disease Product (QIDP) designation by the FDA for the treatment of nontuberculous mycobacteria (NTM) infections. RedHill plans to consult with the FDA regarding the RHB-104 development program for NTM infections. In February 2017, RedHill announced that the last patient enrolled in the randomized, double-blind, placebo-controlled Phase III clinical study with BEKINDA® 24 mg in the U.S. for acute gastroenteritis and gastritis (the GUARD study) had completed the treatment course and observation period for the primary endpoint evaluation. Top-line results are expected in the second quarter of 2017. A randomized, double-blind, placebo-controlled Phase II clinical study with BEKINDA® 12 mg for the treatment of diarrhea-predominant irritable bowel syndrome (IBS-D) is ongoing in the U.S. with top-line results expected in mid-2017. In June 2016, RedHill announced positive final results from a Phase I study with YELIVA® in patients with advanced solid tumors. The Phase I study, conducted at the Medical University of South Carolina Hollings Cancer Center, successfully met its primary and secondary endpoints, demonstrating that the drug is well-tolerated and can be safely administered to cancer patients at doses predicted to have therapeutic activity. In September 2016, RedHill announced a research collaboration with Stanford University School of Medicine for the evaluation of YELIVA®. The research collaboration is intended to complement RedHill’s planned Phase Ib clinical study to evaluate YELIVA® as a radioprotectant for prevention of mucositis in head and neck cancer patients undergoing therapeutic radiotherapy. The Phase Ib study is planned to be initiated in mid-2017. In October 2016, RedHill announced the initiation of a Phase II clinical study with YELIVA® for advanced hepatocellular carcinoma at the Medical University of South Carolina. In December 2016, RedHill announced that the first patient was dosed in a Phase Ib/II study with YELIVA® for refractory or relapsed multiple myeloma, conducted at Duke University Medical Center. A Phase I/II clinical study evaluating YELIVA® in patients with refractory/relapsed diffuse large B-cell lymphoma is ongoing at the Louisiana State University Health Sciences Center and was recently amended to address overall recruitment prospects and to include Kaposi sarcoma patients in the study. A Phase II study to evaluate the efficacy of YELIVA® in patients with moderate to severe ulcerative colitis is planned to be initiated in the second half of 2017.    In 2016, RedHill and its co-development partner, IntelGenx Corp., entered into exclusive license agreements for the commercialization of RIZAPORT® oral thin-film for acute migraines with Grupo JUSTE S.A.Q.F (now Exeltis Healthcare, S.L.) for Spain and with Pharmatronic Co. for South Korea. Re-submission of the RIZAPORT® NDA to the FDA is expected in the third quarter of 2017. In January 2017, RedHill announced the signing of a new collaboration agreement with the Department of Molecular Biology and Genetics of Denmark-based Aarhus University for the evaluation of RedHill’s Phase II-stage oncology drug candidate, MESUPRON. The new research collaboration follows previous non-clinical studies conducted with Denmark’s Aarhus University and is designed to identify additional high affinity molecular targets of MESUPRON. Further evaluation of MESUPRON, together with Aarhus University, may allow for selection of appropriate sub-populations of patients toward demonstrating the activity of MESUPRON in planned clinical trials. RedHill is currently preparing a protocol for a Phase I/II study of the safety, efficacy and dose evaluation of MESUPRON in combination with chemotherapy in patients receiving adjuvant chemotherapy for resected pancreatic cancer. The Phase I/II study is expected to be initiated in the second half of 2017 in up to six sites in Germany. As part of RedHill’s strategic initiative to become a revenue-generating, gastrointestinal-focused, specialty pharmaceutical company with a commercial presence in the U.S., the Company entered in January 2017 into an exclusive co-promotion agreement with a subsidiary4 of Concordia International Corp., granting RedHill certain U.S. promotional rights for Donnatal®, a prescription oral drug used with other drugs in the treatment of irritable bowel syndrome (irritable colon, spastic colon, mucous colitis) and acute enterocolitis (inflammation of the small bowel)5. RedHill expects to initiate promotion of Donnatal® in the coming months. In December 2016, RedHill closed an underwritten public offering and a registered direct offering of American Depositary Shares (ADSs) and warrants to purchase ADSs for aggregate net proceeds, after deducting underwriting discounts and commissions, placement agent fees and other offering expenses, of $35.9 million. Investors in the public offering included, among others, Sabby Management, LLC, DAFNA Capital Management, Rosalind Advisors, Inc., Koramic Holding, Lincoln Park Capital, and Nexthera Capital LP. About Donnatal®: Donnatal® (Phenobarbital, Hyoscyamine Sulfate, Atropine Sulfate, Scopolamine Hydrobromide), a prescription drug, is classified as possibly effective as an adjunctive therapy in the treatment of irritable bowel syndrome (irritable colon, spastic colon, mucous colitis) and acute enterocolitis. Donnatal® slows the natural movements of the gut by relaxing the muscles in the stomach and intestines and acts on the brain to produce a calming effect. Donnatal® comes in two formulations: immediate release Donnatal® Tablets and immediate release Donnatal® Elixir, a fast acting liquid. Donnatal® is contraindicated in patients who have glaucoma, obstructive uropathy, obstructive disease of the gastrointestinal tract, paralytic ileus, unstable cardiovascular status, severe ulcerative colitis, myasthenia gravis, hiatal hernia with reflux esophagitis, or known hypersensitivity to any of the ingredients. Patients who are pregnant or breast-feeding or who have autonomic neuropathy, hepatic or renal disease, hyperthyroidism, coronary heart disease, congestive heart failure, cardiac arrhythmias, tachycardia or hypertension should notify their doctor before taking Donnatal®. Side effects may include: dryness of the mouth, urinary retention, blurred vision, dilation of pupils, rapid heartbeat, loss of sense of taste, headache, nervousness, drowsiness, weakness, dizziness, insomnia, nausea, vomiting and allergic reactions which may be severe. Further information, including prescribing information, can be found on www.donnatal.com. Please see the following website for important safety information about Donnatal®:           http://www.donnatal.com/professionals/important-safety-information/ About RedHill Biopharma Ltd.: RedHill Biopharma Ltd. (NASDAQ:RDHL) (TASE:RDHL) is a specialty biopharmaceutical company headquartered in Israel, primarily focused on the development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for the treatment of gastrointestinal and inflammatory diseases and cancer. RedHill has a U.S. co-promotion agreement with Concordia for Donnatal®, a prescription oral adjunctive drug used in the treatment of IBS and acute enterocolitis. RedHill’s clinical-stage pipeline includes: (i) RHB-105 - an oral combination therapy for the treatment of Helicobacter pylori infection with successful results from a first Phase III study; (ii) RHB-104 - an oral combination therapy for the treatment of Crohn's disease with an ongoing first Phase III study, a completed proof-of-concept Phase IIa study for multiple sclerosis and QIDP status for nontuberculous mycobacteria (NTM) infections; (iii) BEKINDA® (RHB-102) - a once-daily oral pill formulation of ondansetron with an ongoing Phase III study for acute gastroenteritis and gastritis and an ongoing Phase II study for IBS-D; (iv) RHB-106 - an encapsulated bowel preparation licensed to Salix Pharmaceuticals, Ltd.; (v) YELIVA® (ABC294640) - a Phase II-stage, orally-administered, first-in-class SK2 selective inhibitor targeting multiple oncology, inflammatory and gastrointestinal indications; (vi) MESUPRON - a Phase II-stage first-in-class, orally-administered uPA inhibitor, targeting gastrointestinal and other solid tumors and (vii) RIZAPORT® (RHB-103) - an oral thin film formulation of rizatriptan for acute migraines, with a U.S. NDA currently under discussion with the FDA and marketing authorization received in Germany in October 2015. More information about the Company is available at: www.redhillbio.com. This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements may be preceded by the words “intends,” “may,” “will,” “plans,” “expects,” “anticipates,” “projects,” “predicts,” “estimates,” “aims,” “believes,” “hopes,” “potential” or similar words. Forward-looking statements are based on certain assumptions and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company’s control, and cannot be predicted or quantified and consequently, actual results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, risks and uncertainties associated with (i) the initiation, timing, progress and results of the Company’s research, manufacturing, preclinical studies, clinical trials, and other therapeutic candidate development efforts; (ii) the Company’s ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; (iii) the extent and number of additional studies that the Company may be required to conduct and the Company’s receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings, approvals and feedback; (iv) the manufacturing, clinical development, commercialization, and market acceptance of the Company’s therapeutic candidates; (v) the Company’s ability to successfully market Donnatal®, (vi) the Company’s ability to establish and maintain corporate collaborations; (vii) the Company's ability to acquire products approved for marketing in the U.S. that achieve commercial success and build its own marketing and commercialization capabilities; (viii) the interpretation of the properties and characteristics of the Company’s therapeutic candidates and of the results obtained with its therapeutic candidates in research, preclinical studies or clinical trials; (ix) the implementation of the Company’s business model, strategic plans for its business and therapeutic candidates; (x) the scope of protection the Company is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; (xi) parties from whom the Company licenses its intellectual property defaulting in their obligations to the Company; and (xii) estimates of the Company’s expenses, future revenues capital requirements and the Company’s needs for additional financing; (xiii) competitive companies and technologies within the Company’s industry. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the Securities and Exchange Commission (SEC), including the Company's Annual Report on Form 20-F filed with the SEC on February 25, 2016. All forward-looking statements included in this Press Release are made only as of the date of this Press Release. We assume no obligation to update any written or oral forward-looking statement unless required by law. 1 All financial highlights are approximate and are rounded to the nearest hundreds of thousands. 2 All financial highlights are approximate and are rounded to the nearest hundreds of thousands. 5 Donnatal® (Phenobarbital, Hyoscyamine Sulfate, Atropine Sulfate, Scopolamine Hydrobromide) is a prescription drug, classified as possibly effective as an adjunctive therapy in the treatment of irritable bowel syndrome (irritable colon, spastic colon, mucous colitis) and acute enterocolitis.  For more information, please see the prescribing information: http://www.donnatal.com/wp-content/uploads/2015/02/2015-02-18-Risk-Benefit-information-DTC-REV.-SE.pdf.


News Article | February 23, 2017
Site: globenewswire.com

TEL-AVIV, Israel, Feb. 23, 2017 (GLOBE NEWSWIRE) -- RedHill Biopharma Ltd. (NASDAQ:RDHL) (TASE:RDHL) (“RedHill” or the “Company”), a specialty biopharmaceutical company primarily focused on the development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for gastrointestinal and inflammatory diseases and cancer, today reported its financial results for the fourth quarter and full-year ended December 31, 2016. Revenues for the fourth quarter of 2016 were $0.1 million, compared to immaterial revenues for the fourth quarter of 2015. Research and Development Expenses for the fourth quarter of 2016 were $7.5 million, up 51% compared to the fourth quarter of 2015. The increase was mainly due to the ongoing Phase III and Phase II studies with BEKINDA® for gastroenteritis and IBS-D, respectively, the ongoing Phase III study with RHB-104 for Crohn’s disease and ongoing studies with YELIVA® for multiple indications. General, Administrative and Business Development Expenses for the fourth quarter of 2016 were $1.6 million, down 6.9% compared to the fourth quarter of 2015. The decrease was mainly due to a decrease in professional services. Operating Loss for the fourth quarter of 2016 was $9 million, up 33% compared to the fourth quarter of 2015. The increase was mainly due to an increase in research and development expenses, as detailed above. Financial Income, net for the fourth quarter of 2016 was $0.6 million, up 214%, compared to the fourth quarter of 2015. The increase was mainly due to a fair value gain on derivative financial instruments. Net Cash Used in Operating Activities for the fourth quarter of 2016 was $10.1 million, up 69% compared to the fourth quarter of 2015. The increase was mainly due to the increase in operating loss, as detailed above. Net Cash Provided by Investment Activities for the fourth quarter of 2016 was $21.3 million, up 206% compared to the fourth quarter of 2015. The increase was mainly due to maturity of bank deposits. Net Cash Provided by Financing Activities for the fourth quarter of 2016 was $35.9 million compared to an immaterial amount for the fourth quarter of 2015. The increase was mainly due to the December 2016 public offering. Revenues for 2016 were $0.1 million, compared to immaterial revenues in 2015. Research and Development Expenses for 2016 were $25.2 million, up 42% compared to 2015. The increase was mainly due to the ongoing Phase III MAP US study with RHB-104 for Crohn's disease, the ongoing Phase III and Phase II studies with BEKINDA® for gastroenteritis and IBS-D, respectively, and the ongoing studies with YELIVA® for multiple indications. General, Administrative and Business Development Expenses for 2016 were $5.4 million, up 31% compared to 2015. The increase was mainly due to an increase in professional services, compensation and other operating expenses. Operating Loss for 2016 was $30.5 million, up 39% compared to 2015. The increase was mainly due to an increase in research and development expenses, as detailed above. Financial Income, net for 2016 was $1.2 million, up 29% compared to 2015. The increase was mainly due to a fair value gain on derivative financial instruments. Net Cash Used in Operating Activities for 2016 was $28.2 million, up 59% compared to 2015. The increase was mainly due to an increase in operating loss, as detailed above. Net Cash Provided by Investment Activities for 2016 was $24.5 million, up 215% compared to 2015. The difference was mainly due to maturity of bank deposits. Net Cash Provided by Financing Activities for 2016 was $36 million, down 34% compared to 2015. The decrease resulted primarily from the two public offerings in February and July 2015 of the comparable period. Cash Balance3 as of December 31, 2016 was $66.3 million, an increase of $8.2 million compared to $58.1 million as of December 31, 2015 and an increase of $25.8 million compared to $40.5 million as of September 30, 2016. Micha Ben Chorin, RedHill’s CFO, said: “Our strong cash position of approximately $66 million at the end of 2016 should allow us to continue to execute our strategic plans for 2017. We are looking forward to an important year ahead, including the planned initiation of a confirmatory Phase III study with RHB-105 for H. pylori infection, a second independent DSMB meeting for the ongoing MAP US Phase III study with RHB-104 for Crohn’s disease, top-line results from the ongoing Phase III and Phase II studies with BEKINDA® for gastroenteritis and IBS-D, respectively, and commencement of our promotional activities in the U.S. with Donnatal®." The Company will host a conference call on Thursday, February 23, 2017, at 9:00 am EST to review the financial results and business highlights. To participate in the conference call, please dial the following numbers 5-10 minutes prior to the start of the call: United States: +1-877-280-1254; International: +1-646-254-3366; and Israel: +972-3-763-0145. The access code for the call is 4402478. The conference call will be broadcasted live and available for replay on the Company's website, http://ir.redhillbio.com/events.cfm, for 30 days. Please access the Company's website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software. RHB-105 - H. pylori bacterial infection (confirmatory Phase III) (QIDP status)         Following the announcement of the successful final results from a first Phase III clinical study with RHB-105 for the eradication of H. pylori infection (the ERADICATE Hp study) in March 2016, RedHill concluded two positive Type B meetings with the U.S. Food and Drug Administration (FDA) regarding RHB-105. The first meeting, announced in April 2016, confirmed the path to marketing approval of RHB-105 and the planned confirmatory Phase III study. A second Type B meeting, announced in November 2016, discussed the chemistry, manufacturing and controls (CMC) aspects of the RHB-105 Phase III development program towards filing the CMC package as part of the potential U.S. New Drug Application (NDA) to be submitted for RHB-105, subject to successful completion of the planned confirmatory Phase III study. The two-arm, randomized, double-blind, active comparator confirmatory Phase III study, comparing RHB-105 against a dual therapy amoxicillin and omeprazole regimen at equivalent doses, is planned to be initiated in the second quarter of 2017, subject to the successful completion of the ongoing supportive pharmacokinetic (PK) program and submission of the Clinical Study Report to the FDA. The confirmatory Phase III study is planned to enroll approximately 440 patients in up to 55 clinical sites in the U.S. In October 2016, RedHill provided an update on the RHB-104 Phase III Crohn’s disease development program, planned enhancements to the ongoing MAP US Phase III study and expected milestones, including an increase in the total number of patients planned to be enrolled in the MAP US study from 270 to 410, and the addition of an open-label extension study offering patients who complete 26 weeks of study participation and remain out of remission (Crohn’s disease active index (CDAI) >150) the opportunity to receive treatment with RHB-104 for a 52-week period. The open-label extension study is expected to be initiated in the coming weeks. Following a pre-planned review of safety data from its ongoing MAP US study by an independent Data and Safety Monitoring Board (DSMB), RedHill announced in December 2016 that it had received a unanimous recommendation to continue the MAP US study as planned. A second independent DSMB meeting of the MAP US study, expected in the second quarter of 2017, will include an interim efficacy analysis and will evaluate the option for an early stop for success for overwhelming efficacy, according to a pre-specified statistical significance threshold. Taking into account the increase in the total number of patients planned in the MAP US study, and assuming the MAP US study is not stopped for success or inefficacy following the independent DSMB meeting in the second quarter of 2017, completion of recruitment is expected by the end of 2017. In December 2016, RedHill announced encouraging top-line final results of a Phase IIa, proof-of-concept clinical study, evaluating RHB-104 as an add-on therapy to interferon beta-1a in patients treated for relapsing remitting multiple sclerosis (the CEASE MS study). The top-line final results (48 weeks) were consistent with previously announced interim results, suggesting meaningful positive safety and clinical signals upon 24 weeks of treatment with RHB-104 as an add-on therapy, thereby supporting further clinical development. In January 2017, RedHill announced that RHB-104 had been granted Qualified Infectious Disease Product (QIDP) designation by the FDA for the treatment of nontuberculous mycobacteria (NTM) infections. RedHill plans to consult with the FDA regarding the RHB-104 development program for NTM infections. In February 2017, RedHill announced that the last patient enrolled in the randomized, double-blind, placebo-controlled Phase III clinical study with BEKINDA® 24 mg in the U.S. for acute gastroenteritis and gastritis (the GUARD study) had completed the treatment course and observation period for the primary endpoint evaluation. Top-line results are expected in the second quarter of 2017. A randomized, double-blind, placebo-controlled Phase II clinical study with BEKINDA® 12 mg for the treatment of diarrhea-predominant irritable bowel syndrome (IBS-D) is ongoing in the U.S. with top-line results expected in mid-2017. In June 2016, RedHill announced positive final results from a Phase I study with YELIVA® in patients with advanced solid tumors. The Phase I study, conducted at the Medical University of South Carolina Hollings Cancer Center, successfully met its primary and secondary endpoints, demonstrating that the drug is well-tolerated and can be safely administered to cancer patients at doses predicted to have therapeutic activity. In September 2016, RedHill announced a research collaboration with Stanford University School of Medicine for the evaluation of YELIVA®. The research collaboration is intended to complement RedHill’s planned Phase Ib clinical study to evaluate YELIVA® as a radioprotectant for prevention of mucositis in head and neck cancer patients undergoing therapeutic radiotherapy. The Phase Ib study is planned to be initiated in mid-2017. In October 2016, RedHill announced the initiation of a Phase II clinical study with YELIVA® for advanced hepatocellular carcinoma at the Medical University of South Carolina. In December 2016, RedHill announced that the first patient was dosed in a Phase Ib/II study with YELIVA® for refractory or relapsed multiple myeloma, conducted at Duke University Medical Center. A Phase I/II clinical study evaluating YELIVA® in patients with refractory/relapsed diffuse large B-cell lymphoma is ongoing at the Louisiana State University Health Sciences Center and was recently amended to address overall recruitment prospects and to include Kaposi sarcoma patients in the study. A Phase II study to evaluate the efficacy of YELIVA® in patients with moderate to severe ulcerative colitis is planned to be initiated in the second half of 2017.    In 2016, RedHill and its co-development partner, IntelGenx Corp., entered into exclusive license agreements for the commercialization of RIZAPORT® oral thin-film for acute migraines with Grupo JUSTE S.A.Q.F (now Exeltis Healthcare, S.L.) for Spain and with Pharmatronic Co. for South Korea. Re-submission of the RIZAPORT® NDA to the FDA is expected in the third quarter of 2017. In January 2017, RedHill announced the signing of a new collaboration agreement with the Department of Molecular Biology and Genetics of Denmark-based Aarhus University for the evaluation of RedHill’s Phase II-stage oncology drug candidate, MESUPRON. The new research collaboration follows previous non-clinical studies conducted with Denmark’s Aarhus University and is designed to identify additional high affinity molecular targets of MESUPRON. Further evaluation of MESUPRON, together with Aarhus University, may allow for selection of appropriate sub-populations of patients toward demonstrating the activity of MESUPRON in planned clinical trials. RedHill is currently preparing a protocol for a Phase I/II study of the safety, efficacy and dose evaluation of MESUPRON in combination with chemotherapy in patients receiving adjuvant chemotherapy for resected pancreatic cancer. The Phase I/II study is expected to be initiated in the second half of 2017 in up to six sites in Germany. As part of RedHill’s strategic initiative to become a revenue-generating, gastrointestinal-focused, specialty pharmaceutical company with a commercial presence in the U.S., the Company entered in January 2017 into an exclusive co-promotion agreement with a subsidiary4 of Concordia International Corp., granting RedHill certain U.S. promotional rights for Donnatal®, a prescription oral drug used with other drugs in the treatment of irritable bowel syndrome (irritable colon, spastic colon, mucous colitis) and acute enterocolitis (inflammation of the small bowel)5. RedHill expects to initiate promotion of Donnatal® in the coming months. In December 2016, RedHill closed an underwritten public offering and a registered direct offering of American Depositary Shares (ADSs) and warrants to purchase ADSs for aggregate net proceeds, after deducting underwriting discounts and commissions, placement agent fees and other offering expenses, of $35.9 million. Investors in the public offering included, among others, Sabby Management, LLC, DAFNA Capital Management, Rosalind Advisors, Inc., Koramic Holding, Lincoln Park Capital, and Nexthera Capital LP. About Donnatal®: Donnatal® (Phenobarbital, Hyoscyamine Sulfate, Atropine Sulfate, Scopolamine Hydrobromide), a prescription drug, is classified as possibly effective as an adjunctive therapy in the treatment of irritable bowel syndrome (irritable colon, spastic colon, mucous colitis) and acute enterocolitis. Donnatal® slows the natural movements of the gut by relaxing the muscles in the stomach and intestines and acts on the brain to produce a calming effect. Donnatal® comes in two formulations: immediate release Donnatal® Tablets and immediate release Donnatal® Elixir, a fast acting liquid. Donnatal® is contraindicated in patients who have glaucoma, obstructive uropathy, obstructive disease of the gastrointestinal tract, paralytic ileus, unstable cardiovascular status, severe ulcerative colitis, myasthenia gravis, hiatal hernia with reflux esophagitis, or known hypersensitivity to any of the ingredients. Patients who are pregnant or breast-feeding or who have autonomic neuropathy, hepatic or renal disease, hyperthyroidism, coronary heart disease, congestive heart failure, cardiac arrhythmias, tachycardia or hypertension should notify their doctor before taking Donnatal®. Side effects may include: dryness of the mouth, urinary retention, blurred vision, dilation of pupils, rapid heartbeat, loss of sense of taste, headache, nervousness, drowsiness, weakness, dizziness, insomnia, nausea, vomiting and allergic reactions which may be severe. Further information, including prescribing information, can be found on www.donnatal.com. Please see the following website for important safety information about Donnatal®:           http://www.donnatal.com/professionals/important-safety-information/ About RedHill Biopharma Ltd.: RedHill Biopharma Ltd. (NASDAQ:RDHL) (TASE:RDHL) is a specialty biopharmaceutical company headquartered in Israel, primarily focused on the development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for the treatment of gastrointestinal and inflammatory diseases and cancer. RedHill has a U.S. co-promotion agreement with Concordia for Donnatal®, a prescription oral adjunctive drug used in the treatment of IBS and acute enterocolitis. RedHill’s clinical-stage pipeline includes: (i) RHB-105 - an oral combination therapy for the treatment of Helicobacter pylori infection with successful results from a first Phase III study; (ii) RHB-104 - an oral combination therapy for the treatment of Crohn's disease with an ongoing first Phase III study, a completed proof-of-concept Phase IIa study for multiple sclerosis and QIDP status for nontuberculous mycobacteria (NTM) infections; (iii) BEKINDA® (RHB-102) - a once-daily oral pill formulation of ondansetron with an ongoing Phase III study for acute gastroenteritis and gastritis and an ongoing Phase II study for IBS-D; (iv) RHB-106 - an encapsulated bowel preparation licensed to Salix Pharmaceuticals, Ltd.; (v) YELIVA® (ABC294640) - a Phase II-stage, orally-administered, first-in-class SK2 selective inhibitor targeting multiple oncology, inflammatory and gastrointestinal indications; (vi) MESUPRON - a Phase II-stage first-in-class, orally-administered uPA inhibitor, targeting gastrointestinal and other solid tumors and (vii) RIZAPORT® (RHB-103) - an oral thin film formulation of rizatriptan for acute migraines, with a U.S. NDA currently under discussion with the FDA and marketing authorization received in Germany in October 2015. More information about the Company is available at: www.redhillbio.com. This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements may be preceded by the words “intends,” “may,” “will,” “plans,” “expects,” “anticipates,” “projects,” “predicts,” “estimates,” “aims,” “believes,” “hopes,” “potential” or similar words. Forward-looking statements are based on certain assumptions and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company’s control, and cannot be predicted or quantified and consequently, actual results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, risks and uncertainties associated with (i) the initiation, timing, progress and results of the Company’s research, manufacturing, preclinical studies, clinical trials, and other therapeutic candidate development efforts; (ii) the Company’s ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; (iii) the extent and number of additional studies that the Company may be required to conduct and the Company’s receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings, approvals and feedback; (iv) the manufacturing, clinical development, commercialization, and market acceptance of the Company’s therapeutic candidates; (v) the Company’s ability to successfully market Donnatal®, (vi) the Company’s ability to establish and maintain corporate collaborations; (vii) the Company's ability to acquire products approved for marketing in the U.S. that achieve commercial success and build its own marketing and commercialization capabilities; (viii) the interpretation of the properties and characteristics of the Company’s therapeutic candidates and of the results obtained with its therapeutic candidates in research, preclinical studies or clinical trials; (ix) the implementation of the Company’s business model, strategic plans for its business and therapeutic candidates; (x) the scope of protection the Company is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; (xi) parties from whom the Company licenses its intellectual property defaulting in their obligations to the Company; and (xii) estimates of the Company’s expenses, future revenues capital requirements and the Company’s needs for additional financing; (xiii) competitive companies and technologies within the Company’s industry. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the Securities and Exchange Commission (SEC), including the Company's Annual Report on Form 20-F filed with the SEC on February 25, 2016. All forward-looking statements included in this Press Release are made only as of the date of this Press Release. We assume no obligation to update any written or oral forward-looking statement unless required by law. 1 All financial highlights are approximate and are rounded to the nearest hundreds of thousands. 2 All financial highlights are approximate and are rounded to the nearest hundreds of thousands. 5 Donnatal® (Phenobarbital, Hyoscyamine Sulfate, Atropine Sulfate, Scopolamine Hydrobromide) is a prescription drug, classified as possibly effective as an adjunctive therapy in the treatment of irritable bowel syndrome (irritable colon, spastic colon, mucous colitis) and acute enterocolitis.  For more information, please see the prescribing information: http://www.donnatal.com/wp-content/uploads/2015/02/2015-02-18-Risk-Benefit-information-DTC-REV.-SE.pdf.


DBV Technologies, BioNet-Asia and Geneva University Hospitals Complete Dosing in First Cohort of Phase I Study of Viaskin rPT for Booster Vaccination Against Pertussis Following positive DSMB review, dosing with Viaskin rPT 50 µg has been initiated PARIS, BANGKOK and GENEVA November 17, 2016 - DBV Technologies (Euronext: DBV - ISIN: FR0010417345 - Nasdaq Stock Market: DBVT), the Geneva University Hospitals (HUG) and BioNet-Asia Co. Ltd today announced that in a planned interim assessment of the Phase I trial of Viaskin rPT for booster immunization against Bordetella pertussis, the independent Data and Safety Monitoring Board (DSMB) concluded that there were no safety concerns with the administration of Viaskin rPT 25 mcg in the first subject cohort. Based on this review, enrollment in the trial has continued as planned, with dosing of Viaskin rPT 50 mcg commencing in the second subject cohort. The Viaskin rPT pertussis booster vaccination program intends to test the ability of DBV's needleless and adjuvant-free patch technology, Viaskin, to epicutaneously deliver two different doses of BioNet's genetically detoxified, recombinant pertussis toxin for boosting immunity against whooping cough. In the first dosing cohort, subjects received two applications of either Viaskin rPT 25 mcg or placebo. Following the DSMB's positive recommendation, a second cohort of subjects will receive two applications of Viaskin rPT 50 mcg or placebo at two-week interval. This Phase I proof of concept study is being conducted under the supervision of Professor Claire-Anne Siegrist from the Clinical Research Center of HUG and is sponsored by DBV Technologies. About the Phase I Viaskin rPT Trial This Phase I dose-escalation, randomized, double-blind, placebo-controlled safety and immunogenicity study is assessing the safety of BioNet's genetically-detoxified recombinant pertussis toxin administered by DBV's Viaskin patches in 60 young healthy adults. Secondary endpoints will assess the patients' humoral responses elicited by Viaskin rPT 25 mcg and 50 mcg compared to placebo. Immune cellular responses will also be monitored as exploratory endpoints. The trial is being conducted in the Clinical Research Center of the Geneva University Hospitals. Men and women aged 18 to 40 years who have been vaccinated during childhood against pertussis will be randomized into two cohorts of 30 subjects each. The Viaskin patches will be applied for 48 hours, with a two-week interval between applications. Four weeks after the second Viaskin application, participants will receive one dose of Boostrix® dTpa vaccine to ensure the recall of immunity against diphtheria, tetanus and the three pertussis antigens (only a single antigen will be delivered through Viaskin rPT). All subjects will be observed after each application. Local and systemic adverse events will be monitored. The first cohort has received two applications of Viaskin rPT 25 mcg or placebo. Following a positive DSMB review, dosing in the second patient cohort, which is expected to receive two applications of Viaskin rPT 50 mcg or placebo has commenced. About Bordetella Pertussis Pertussis, commonly known as whooping cough, is a highly contagious respiratory illness caused by a type of bacteria known as Bordetella pertussis. Pertussis vaccination is recommended as part of routine childhood immunization. Although the incidence of pertussis has declined as a result of immunization of infants and young children, vaccine-induced immunity does not persist for long.  This phenomenon, known as waning immunity, has increased since the introduction of acellular pertussis vaccines in 1996, which tend to provide short-lived protection against the Bordetella pertussis bacteria. According to the U.S. Centers for Disease Control and Prevention (CDC), there are 16 million pertussis cases worldwide each year, mainly in adolescents and adults who often can infect infants who have not yet completed their pertussis immunization. In these young patients, pertussis can be severe and fatal. Booster immunizations are now recommended for adolescents and adults, but compliance is not always high. A new vaccine technology that is patient-friendly, painless and non-invasive could help increase the compliance for booster immunization against whooping cough. About DBV Technologies  DBV Technologies is developing Viaskin®, a proprietary technology platform with broad potential applications in immunotherapy. Viaskin is based on epicutaneous immunotherapy, or EPIT®, DBV's method of delivering biologically active compounds to the immune system through intact skin. With this new class of self-administered and non-invasive product candidates, the company is dedicated to safely transforming the care of food allergic patients, for whom there are no approved treatments. DBV's food allergies programs include ongoing clinical trials of Viaskin Peanut and Viaskin Milk, and preclinical development of Viaskin Egg. DBV is also pursuing a human proof-of-concept clinical study of Viaskin Milk for the treatment of Eosinophilic Esophagitis, and exploring potential applications of its platform in vaccines and other immune diseases. DBV Technologies has global headquarters in Montrouge, France and New York, NY. Company shares are traded on segment B of Euronext Paris (Ticker: DBV, ISIN code: FR0010417345), part of the SBF120 index, and traded on the Nasdaq Global Select Market in the form of American Depositary Shares (each representing one-half of one ordinary share) (Ticker: DBVT). For more information on DBV Technologies, please visit our website: www.dbv-technologies.com About Geneva University Hospitals The Geneva University Hospitals (HUG), reference academic institution at both national and international level, gather eight public hospitals of Geneva. Their centres of excellence cover hepato-biliary and pancreatic diseases, cardiovascular diseases, oncology, musculoskeletal and sports medicine, old age medicine, genetic medicine and vaccinology. Its Center of Vaccinology, led by Professor Claire-Anne Siegrist, gained international recognition through the performance of a large first-in-humans Phase I randomized clinical trial that enrolled 115 subjects to characterize the safety and immunogenicity of the VSV-ZEBOV Ebola vaccine candidate. With their 10,500 employees, the HUG welcome each year 60,000 hospitalized patients and assure 91,000 emergencies, 990,000 consultations or ambulatory care and 26,000 surgical procedures. More than 800 physicians, 3,000 interns and 150 apprentices perform their training here. The HUG are working closely with the Faculty of Medicine of the University of Geneva and WHO in various training and research projects. They develop partnerships with CHUV, EPFL, CERN and other actors from the Lemanic Health Valley. More information on: www.hug-ge.ch About BioNet-Asia BioNet-Asia offers access to vaccine and technology through biotech innovation and partnering networks. BioNet has built several international partnerships fostering vaccine self-reliance and leading to the supply of billions of doses of vaccines worldwide. BioNet has also a broad pipeline of vaccines in R&D and clinical stages. BioNet most advanced program is the development of a new generation of pertussis vaccines aimed at overcoming the waning immunity observed with the conventional acellular pertussis vaccines. BioNet's pertussis vaccines are produced from a new proprietary Bordetella pertussis strain expressing genetically detoxified Pertussis Toxin (PTgen(TM)). The unique properties of BioNet's PTgen enables the vaccines to induce superior anti-PT immune response. For additional information, please visit www.bionet-asia.com Forward Looking Statements This press release contains forward-looking statements, including statements about the potential safety and efficacy of Viaskin as a means of delivering recombinant pertussis toxin to boost immunity against Bordetella pertussis.  These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties.  The Company's product candidates have not been approved for sale in any jurisdiction. Among the factors that could cause actual results to differ materially from those described or projected herein include uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals, the risk that historical preclinical results may not be predictive of future clinical trial results, and the risk that historical clinical trial results may not be predictive of future trial results.  A further list and description of these risks, uncertainties and other risks can be found in the Company's regulatory filings with the French Autorité des Marchés Financiers, the Company's Securities and Exchange Commission filings and reports, including in the Company's Annual Report on Form 20-F for the year ended December 31, 2015 and future filings and reports by the Company.  Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. DBV Technologies undertakes no obligation to update or revise the information contained in this Press Release, whether as a result of new information, future events or circumstances or otherwise. Geneva University Hospitals Contact Professor Claire-Anne Siegrist, Director of the Center of Vaccinology +41(22) 379 57 77 claire-anne.siegrist@unige.ch


News Article | February 21, 2017
Site: www.eurekalert.org

The National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), has launched a Phase 1 clinical trial to test an investigational vaccine intended to provide broad protection against a range of mosquito-transmitted diseases, such as Zika, malaria, West Nile fever and dengue fever, and to hinder the ability of mosquitoes to transmit such infections. The study, which is being conducted at the NIH Clinical Center in Bethesda, Maryland, will examine the experimental vaccine's safety and ability to generate an immune response. The investigational vaccine, called AGS-v, was developed by the London-based pharmaceutical company SEEK, which has since formed a joint venture with hVIVO in London. The consulting group Halloran has provided regulatory advice to both companies. Unlike other vaccines targeting specific mosquito-borne diseases, the AGS-v candidate is designed to trigger an immune response to mosquito saliva rather than to a specific virus or parasite carried by mosquitoes. The test vaccine contains four synthetic proteins from mosquito salivary glands. The proteins are designed to induce antibodies in a vaccinated individual and to cause a modified allergic response that can prevent infection when a person is bitten by a disease-carrying mosquito. "Mosquitoes cause more human disease and death than any other animal," said NIAID Director Anthony S. Fauci, M.D. "A single vaccine capable of protecting against the scourge of mosquito-borne diseases is a novel concept that, if proven successful, would be a monumental public health advance." Led by Matthew J. Memoli, M.D., director of the Clinical Studies Unit in NIAID's Laboratory of Infectious Diseases, the clinical trial is expected to enroll up to 60 healthy adults ages 18 to 50 years. Participants will be randomly assigned to receive one of three vaccine regimens. The first group will receive two injections of the AGS-v vaccine, 21 days apart. The second group will receive two injections of AGS-v combined with an adjuvant, 21 days apart. The adjuvant is an oil and water mixture commonly added to vaccines to enhance immune responses. The third group will receive two placebo injections of sterile water 21 days apart. Neither the study investigators nor the participants will know who is assigned to each group. Participants will be asked to return to the clinic twice between vaccinations and twice after the second vaccination to undergo a physical exam and to provide blood samples. Study investigators will examine the blood samples to measure levels of antibodies triggered by vaccination. Each participant also will return to the Clinical Center approximately 21 days after completing the vaccination schedule to undergo a controlled exposure to biting mosquitoes. The mosquitoes will not be carrying viruses or parasites, so the participants are not at risk of becoming infected with a mosquito-borne disease. Five to 10 female Aedes aegypti mosquitoes from the insectary in NIAID's Laboratory of Malaria and Vector Research will be put in a feeding device that will be placed on each participant's arm for 20 minutes. The mosquitoes will bite the participants' arms through the netting on the feeding devices. Afterward, investigators will take blood samples from each participant at various time points to see if participants experience a modified response to the mosquito bites as a result of AGS-v vaccination. Investigators also will examine the mosquitoes after the feeding to assess any changes to their life cycle. Scientists suspect that the mosquitoes who take a blood meal from ASG-v-vaccinated participants may have altered behavior that could lead to early death or a reduced ability to reproduce. This would indicate that the experimental vaccine could also hinder disease transmission by controlling the mosquito population. All participants will be asked to return to the clinic for follow-up visits every 60 days for five months following the mosquito feeding. A final clinic visit to assess long-term safety will take place approximately 10 months after the mosquito feeding. Throughout the trial, an independent Data and Safety Monitoring Board will review study data to evaluate participant safety and the overall conduct of the study. A medical monitor from NIAID's Office of Clinical Research Policy and Regulatory Operations will also perform routine safety assessments. The study is expected to be completed by summer 2018. For more information about the trial, see ClinicalTrials.gov using the trial identifier NCT03055000. NIAID conducts and supports research--at NIH, throughout the United States, and worldwide--to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID website. About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www. .


TEL-AVIV, Israel, Dec. 13, 2016 (GLOBE NEWSWIRE) -- RedHill Biopharma Ltd. (NASDAQ:RDHL) (TASE:RDHL) (“RedHill” or the “Company”), a biopharmaceutical company primarily focused on development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for gastrointestinal and inflammatory diseases and cancer, today reported that, following a pre-planned review of safety data from its ongoing Phase III study with RHB-104 for Crohn’s disease (the MAP US study) by an independent Data and Safety Monitoring Board (DSMB), it has received a unanimous recommendation to continue the study as planned, without any modifications. RHB-104 is a proprietary and potentially groundbreaking antibiotic combination therapy in oral capsule formulation, with potent intracellular, anti-mycobacterial and anti-inflammatory properties. The development of RHB-104 is based on increasing evidence supporting the hypothesis that Crohn’s disease, and potentially other autoimmune diseases, are related to Mycobacterium avium subspecies paratuberculosis (MAP) infection in susceptible patients. The development of RHB-104 is consistent with the growing awareness of the possibility that a bacterially-induced dysregulated immune system may contribute to the pathogenesis of various autoimmune diseases of unknown etiology. The ongoing MAP US study is a randomized, double-blind, placebo-controlled first Phase III study intended to evaluate the safety and efficacy of RHB-104 in patients with moderately to severely-active Crohn’s disease (defined as Crohn’s Disease Activity Index (CDAI) between 220 and 450). To date, 242 patients have been enrolled out of a planned total of 410 patients in up to 150 clinical sites in the U.S, Canada, Europe, Israel, Australia and New Zealand. Subjects enrolled in the MAP US study are randomized 1:1 to receive RHB-104 or a placebo, with a primary endpoint of disease remission, defined as reduction in CDAI to less than 150 at week 26. Secondary and exploratory endpoints include, among others, state of response at week 26, maintenance of remission through week 52, endoscopic evaluation of mucosal healing and efficacy outcome measures in relation to the presence of MAP bacterial infection. Additional studies will be required to support a U.S. New Drug Application (NDA) for RHB-104. Two additional independent DSMB meetings are expected to take place in the MAP US study after 50% and after 75% of the 410 patients planned to be enrolled in the study will complete 26 weeks of study participation. The second independent DSMB meeting is expected in the second quarter of 2017 after the first 205 patients are expected to complete 26 weeks of study participation (patient 205 was randomized in August 2016). The second DSMB meeting will include safety and interim efficacy analysis and could potentially provide the opportunity to expedite the data locking process for the final analysis, once the study is complete. Importantly, this independent DSMB meeting will evaluate the option of an early stop for success for overwhelming efficacy, according to a pre-specified statistical significance threshold for analysis of RHB-104 versus placebo in the primary endpoint. If the pre-specified threshold is met at the second DSMB meeting, the study could be stopped for efficacy or inefficacy. If the pre-specified threshold is not met during the interim analysis, the MAP US study is planned to continue through randomization of all 410 patients and follow-up at week 26. Taking into account the increase in the total number of patients planned in the MAP US study, and assuming the study is not stopped for success or inefficacy following the DSMB meeting in the second quarter of 2017, completion of recruitment is expected by the end of 2017. RedHill is advancing its preparation to initiate an open-label extension study for all patients who have completed 26 weeks of treatment in the MAP US study and failed to achieve remission at week 26, the study’s primary endpoint. Patients with a Crohn’s Disease Active Index (CDAI) score of greater than 150 at week 26 will be offered the opportunity to receive treatment with active drug (RHB-104) for a 52-week period. This study is considered separate from the MAP US study and data collected will be supplemental to the MAP US study data. About RHB-104: Currently in a first Phase III study for the treatment of Crohn’s disease (the MAP US study), RHB-104 is a proprietary and potentially groundbreaking oral antibiotic combination therapy, with potent intracellular, anti-mycobacterial and anti-inflammatory properties. RHB-104 is based on increasing evidence supporting the hypothesis that Crohn’s disease is caused by Mycobacterium avium subspecies paratuberculosis (MAP) infection in susceptible patients. Clinical trials conducted with earlier formulations of RHB-104 include an Australian Phase III study conducted by Pharmacia/Pfizer. RedHill has conducted several supportive studies with the current formulation of RHB-104 and a long-term population pharmacokinetic (pop-PK) study is ongoing as part of the Phase III MAP US study. RHB-104 is covered by several issued and pending patents. RedHill has also completed a Phase IIa, proof-of-concept clinical study, evaluating RHB-104 as an add-on therapy to interferon beta-1a in patients treated for relapsing-remitting multiple sclerosis (the CEASE MS study). Top-line final results from the CEASE MS study suggest meaningful positive safety and clinical signals upon 24 weeks treatment with RHB-104 as an add-on therapy, thereby supporting further clinical development. About RedHill Biopharma Ltd.: RedHill Biopharma Ltd. (NASDAQ:RDHL) (TASE:RDHL) is a biopharmaceutical company headquartered in Israel, primarily focused on the development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for the treatment of gastrointestinal and inflammatory diseases and cancer. RedHill’s pipeline of proprietary products includes: (i) RHB-105 - an oral combination therapy for the treatment of Helicobacter pylori infection with successful results from a first Phase III study; (ii) RHB-104 - an oral combination therapy for the treatment of Crohn's disease with an ongoing first Phase III study and a completed proof-of-concept Phase IIa study for multiple sclerosis; (iii) BEKINDA® (RHB-102) - a once-daily oral pill formulation of ondansetron with an ongoing Phase III study for acute gastroenteritis and gastritis and an ongoing Phase II study for IBS-D; (iv) RHB-106 - an encapsulated bowel preparation licensed to Salix Pharmaceuticals, Ltd.; (v) YELIVA® (ABC294640) - a Phase II-stage, orally-administered, first-in-class SK2 selective inhibitor targeting multiple oncology, inflammatory and gastrointestinal indications; (vi) MESUPRON - a Phase II-stage first-in-class, orally-administered uPA inhibitor, targeting gastrointestinal and other solid tumors and (vii) RIZAPORT® (RHB-103) - an oral thin film formulation of rizatriptan for acute migraines, with a U.S. NDA currently under discussion with the FDA and marketing authorization received in Germany in October 2015. This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements may be preceded by the words “intends,” “may,” “will,” “plans,” “expects,” “anticipates,” “projects,” “predicts,” “estimates,” “aims,” “believes,” “hopes,” “potential” or similar words. Forward-looking statements are based on certain assumptions and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company’s control, and cannot be predicted or quantified and consequently, actual results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, risks and uncertainties associated with (i) the initiation, timing, progress and results of the Company’s research, manufacturing, preclinical studies, clinical trials, and other therapeutic candidate development efforts; (ii) the Company’s ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; (iii) the extent and number of additional studies that the Company may be required to conduct and the Company’s receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings, approvals and feedback; (iv) the manufacturing, clinical development, commercialization, and market acceptance of the Company’s therapeutic candidates; (v) the Company’s ability to establish and maintain corporate collaborations; (vi) the Company's ability to acquire products approved for marketing in the U.S. that achieve commercial success and build its own marketing and commercialization capabilities; (vii) the interpretation of the properties and characteristics of the Company’s therapeutic candidates and of the results obtained with its therapeutic candidates in research, preclinical studies or clinical trials; (viii) the implementation of the Company’s business model, strategic plans for its business and therapeutic candidates; (ix) the scope of protection the Company is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; (x) parties from whom the Company licenses its intellectual property defaulting in their obligations to the Company; (xi) estimates of the Company’s expenses, future revenues capital requirements and the Company’s needs for additional financing; (xii) competitive companies and technologies within the Company’s industry; and (xiii) the impact of the political and security situation in Israel on the Company's business. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the Securities and Exchange Commission (SEC), including the Company's Annual Report on Form 20-F filed with the SEC on February 25, 2016. All forward-looking statements included in this Press Release are made only as of the date of this Press Release. We assume no obligation to update any written or oral forward-looking statement unless required by law.


TEL-AVIV, Israel, Dec. 13, 2016 (GLOBE NEWSWIRE) -- RedHill Biopharma Ltd. (NASDAQ:RDHL) (TASE:RDHL) (“RedHill” or the “Company”), a biopharmaceutical company primarily focused on development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for gastrointestinal and inflammatory diseases and cancer, today reported that, following a pre-planned review of safety data from its ongoing Phase III study with RHB-104 for Crohn’s disease (the MAP US study) by an independent Data and Safety Monitoring Board (DSMB), it has received a unanimous recommendation to continue the study as planned, without any modifications. RHB-104 is a proprietary and potentially groundbreaking antibiotic combination therapy in oral capsule formulation, with potent intracellular, anti-mycobacterial and anti-inflammatory properties. The development of RHB-104 is based on increasing evidence supporting the hypothesis that Crohn’s disease, and potentially other autoimmune diseases, are related to Mycobacterium avium subspecies paratuberculosis (MAP) infection in susceptible patients. The development of RHB-104 is consistent with the growing awareness of the possibility that a bacterially-induced dysregulated immune system may contribute to the pathogenesis of various autoimmune diseases of unknown etiology. The ongoing MAP US study is a randomized, double-blind, placebo-controlled first Phase III study intended to evaluate the safety and efficacy of RHB-104 in patients with moderately to severely-active Crohn’s disease (defined as Crohn’s Disease Activity Index (CDAI) between 220 and 450). To date, 242 patients have been enrolled out of a planned total of 410 patients in up to 150 clinical sites in the U.S, Canada, Europe, Israel, Australia and New Zealand. Subjects enrolled in the MAP US study are randomized 1:1 to receive RHB-104 or a placebo, with a primary endpoint of disease remission, defined as reduction in CDAI to less than 150 at week 26. Secondary and exploratory endpoints include, among others, state of response at week 26, maintenance of remission through week 52, endoscopic evaluation of mucosal healing and efficacy outcome measures in relation to the presence of MAP bacterial infection. Additional studies will be required to support a U.S. New Drug Application (NDA) for RHB-104. Two additional independent DSMB meetings are expected to take place in the MAP US study after 50% and after 75% of the 410 patients planned to be enrolled in the study will complete 26 weeks of study participation. The second independent DSMB meeting is expected in the second quarter of 2017 after the first 205 patients are expected to complete 26 weeks of study participation (patient 205 was randomized in August 2016). The second DSMB meeting will include safety and interim efficacy analysis and could potentially provide the opportunity to expedite the data locking process for the final analysis, once the study is complete. Importantly, this independent DSMB meeting will evaluate the option of an early stop for success for overwhelming efficacy, according to a pre-specified statistical significance threshold for analysis of RHB-104 versus placebo in the primary endpoint. If the pre-specified threshold is met at the second DSMB meeting, the study could be stopped for efficacy or inefficacy. If the pre-specified threshold is not met during the interim analysis, the MAP US study is planned to continue through randomization of all 410 patients and follow-up at week 26. Taking into account the increase in the total number of patients planned in the MAP US study, and assuming the study is not stopped for success or inefficacy following the DSMB meeting in the second quarter of 2017, completion of recruitment is expected by the end of 2017. RedHill is advancing its preparation to initiate an open-label extension study for all patients who have completed 26 weeks of treatment in the MAP US study and failed to achieve remission at week 26, the study’s primary endpoint. Patients with a Crohn’s Disease Active Index (CDAI) score of greater than 150 at week 26 will be offered the opportunity to receive treatment with active drug (RHB-104) for a 52-week period. This study is considered separate from the MAP US study and data collected will be supplemental to the MAP US study data. About RHB-104: Currently in a first Phase III study for the treatment of Crohn’s disease (the MAP US study), RHB-104 is a proprietary and potentially groundbreaking oral antibiotic combination therapy, with potent intracellular, anti-mycobacterial and anti-inflammatory properties. RHB-104 is based on increasing evidence supporting the hypothesis that Crohn’s disease is caused by Mycobacterium avium subspecies paratuberculosis (MAP) infection in susceptible patients. Clinical trials conducted with earlier formulations of RHB-104 include an Australian Phase III study conducted by Pharmacia/Pfizer. RedHill has conducted several supportive studies with the current formulation of RHB-104 and a long-term population pharmacokinetic (pop-PK) study is ongoing as part of the Phase III MAP US study. RHB-104 is covered by several issued and pending patents. RedHill has also completed a Phase IIa, proof-of-concept clinical study, evaluating RHB-104 as an add-on therapy to interferon beta-1a in patients treated for relapsing-remitting multiple sclerosis (the CEASE MS study). Top-line final results from the CEASE MS study suggest meaningful positive safety and clinical signals upon 24 weeks treatment with RHB-104 as an add-on therapy, thereby supporting further clinical development. About RedHill Biopharma Ltd.: RedHill Biopharma Ltd. (NASDAQ:RDHL) (TASE:RDHL) is a biopharmaceutical company headquartered in Israel, primarily focused on the development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for the treatment of gastrointestinal and inflammatory diseases and cancer. RedHill’s pipeline of proprietary products includes: (i) RHB-105 - an oral combination therapy for the treatment of Helicobacter pylori infection with successful results from a first Phase III study; (ii) RHB-104 - an oral combination therapy for the treatment of Crohn's disease with an ongoing first Phase III study and a completed proof-of-concept Phase IIa study for multiple sclerosis; (iii) BEKINDA® (RHB-102) - a once-daily oral pill formulation of ondansetron with an ongoing Phase III study for acute gastroenteritis and gastritis and an ongoing Phase II study for IBS-D; (iv) RHB-106 - an encapsulated bowel preparation licensed to Salix Pharmaceuticals, Ltd.; (v) YELIVA® (ABC294640) - a Phase II-stage, orally-administered, first-in-class SK2 selective inhibitor targeting multiple oncology, inflammatory and gastrointestinal indications; (vi) MESUPRON - a Phase II-stage first-in-class, orally-administered uPA inhibitor, targeting gastrointestinal and other solid tumors and (vii) RIZAPORT® (RHB-103) - an oral thin film formulation of rizatriptan for acute migraines, with a U.S. NDA currently under discussion with the FDA and marketing authorization received in Germany in October 2015. This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements may be preceded by the words “intends,” “may,” “will,” “plans,” “expects,” “anticipates,” “projects,” “predicts,” “estimates,” “aims,” “believes,” “hopes,” “potential” or similar words. Forward-looking statements are based on certain assumptions and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company’s control, and cannot be predicted or quantified and consequently, actual results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, risks and uncertainties associated with (i) the initiation, timing, progress and results of the Company’s research, manufacturing, preclinical studies, clinical trials, and other therapeutic candidate development efforts; (ii) the Company’s ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; (iii) the extent and number of additional studies that the Company may be required to conduct and the Company’s receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings, approvals and feedback; (iv) the manufacturing, clinical development, commercialization, and market acceptance of the Company’s therapeutic candidates; (v) the Company’s ability to establish and maintain corporate collaborations; (vi) the Company's ability to acquire products approved for marketing in the U.S. that achieve commercial success and build its own marketing and commercialization capabilities; (vii) the interpretation of the properties and characteristics of the Company’s therapeutic candidates and of the results obtained with its therapeutic candidates in research, preclinical studies or clinical trials; (viii) the implementation of the Company’s business model, strategic plans for its business and therapeutic candidates; (ix) the scope of protection the Company is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; (x) parties from whom the Company licenses its intellectual property defaulting in their obligations to the Company; (xi) estimates of the Company’s expenses, future revenues capital requirements and the Company’s needs for additional financing; (xii) competitive companies and technologies within the Company’s industry; and (xiii) the impact of the political and security situation in Israel on the Company's business. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the Securities and Exchange Commission (SEC), including the Company's Annual Report on Form 20-F filed with the SEC on February 25, 2016. All forward-looking statements included in this Press Release are made only as of the date of this Press Release. We assume no obligation to update any written or oral forward-looking statement unless required by law.

Loading Data and Safety Monitoring Board collaborators
Loading Data and Safety Monitoring Board collaborators