Pharmaceuticals and Medical Devices Agency

Tokyo, Japan

Pharmaceuticals and Medical Devices Agency

Tokyo, Japan

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‒ NeoCart® Phase 3 Clinical Trial Enrollment Near Completion ‒ ‒ NeoCart Approval Pathway in Japan Defined After Successful Conclusion of Discussions with the Japan Pharmaceuticals and Medical Devices Agency ‒ ‒ Publication of Additional Data to Support Potential Approval and Commercialization of NeoCart ‒ ‒ Company to Host Conference Call and Webcast Today at 8:30 a.m. EDT ‒ WALTHAM, Mass., May 11, 2017 (GLOBE NEWSWIRE) -- Histogenics Corporation (Histogenics) (Nasdaq:HSGX), a regenerative medicine company focused on developing and commercializing products in the musculoskeletal space, announced its financial and operational results for the quarter ended March 31, 2017. “In the first quarter of 2017, we continued to execute on our important near- and long-term business objectives.  Importantly, we enrolled an additional 23 patients in the NeoCart Phase 3 clinical trial, and completed a series of productive formal meetings with the PMDA in Japan where we determined a near-term regulatory pathway for NeoCart to gain full Marketing Authorization in the Japanese market,” stated Adam Gridley, President and Chief Executive Officer of Histogenics.  “We are pleased to report continued strength in our enrollment of the clinical trial, which is expected to conclude this quarter.  We also continue to build on our strong portfolio of bench and clinical data with several new publications on the clinical and biomechanical performance of NeoCart.  This contributes to our goal of a rapid BLA filing in the second half of 2018, and helps prepare us for the potential commercialization of NeoCart in 2019, pending positive results from the NeoCart Phase 3 clinical trial and FDA approval,” continued Mr. Gridley. Financial Results for the First Quarter of 2017 Loss from operations was $(6.8) million in the first quarter of 2017, compared to $(7.8) million in the first quarter of 2016.  The decrease in operating expenses was driven by a reduction in research and development expenses and partially offset by a small increase in general and administrative expenses. Research and development expenses were $4.5 million in the first quarter of 2017, compared to $5.6 million in the first quarter of 2016.  The decrease was primarily due to a reduction in consulting and temporary labor costs and patient recruiting expenses related to the NeoCart Phase 3 clinical trial.  This decrease was partially offset by an increase in hiring fees in the first quarter of 2017.  General and administrative expenses were $2.3 million in the first quarter of 2017, compared to $2.2 million in the first quarter of 2016.  The increase was primarily due to increases in stock-based compensation and repairs and maintenance expense that were partially offset by a reduction in hiring fees and consulting costs. Net loss attributable to common stockholders was $(5.8) million in first quarter of 2017, or $(0.27) per share, compared to $(7.9) million, or $(0.60) per share, in the first quarter of 2016.  The decrease in net loss attributable to common stockholders is primarily due to lower operating expenses and the allocation of a portion of the net loss to the Series A Preferred Stock. As of March 31, 2017, Histogenics had cash, cash equivalents and marketable securities of $24.4 million, compared to $31.9 million at December 31, 2016.  Histogenics believes its current cash position will be sufficient to fund its operations into the middle of 2018. Histogenics’ management will host a conference call on Thursday, May 11, 2017 at 8:30 a.m. EDT.  A question-and-answer session will follow Histogenics’ remarks.  To participate on the live call, please dial (877) 930-8064 (domestic) or (253) 336-8040 (international) and provide the conference ID “97247103” five to ten minutes before the start of the call. A live audio webcast of the presentation will be available via the “Investor Relations” page of the Histogenics website, www.histogenics.com. A replay of the webcast will be archived on Histogenics’ website for approximately 45 days following the presentation. Histogenics is a leading regenerative medicine company developing and commercializing novel tissue therapies that may offer more rapid and durable recoveries for patients with pain and loss of function due to musculoskeletal conditions.  Histogenics’ regenerative medicine platform combines expertise in cell processing, scaffolding, tissue engineering and bioadhesives to create tissue ex-vivo.  Histogenics’ first investigational product candidate, NeoCart is designed to treat cartilage defects in the knee and is currently in Phase 3 clinical development.  NeoCart is designed to exhibit characteristics of articular, hyaline cartilage prior to and upon implantation into the knee and therefore does not rely on the body to make new cartilage.  As a result, NeoCart is the only product in development or on the market with a one-year primary superiority endpoint as compared to the standard of care.  There are more than 500,000 or more knee cartilage procedures in the United States each year, with many healthy active adults avoiding treatment as they seek other alternatives.  Left untreated, even a small cartilage defect can expand in size and progress to debilitating osteoarthritis, ultimately necessitating a joint replacement procedure.  Osteoarthritis is more common in adults over the age of 50, but the condition and precursors of the condition can be observed much earlier, and cartilage damage is believed to be one of the leading contributors of this disease.  For more information, please visit www.histogenics.com. Various statements in this release are “forward-looking statements” under the securities laws. Words such as, but not limited to, “anticipate,” “believe,” “can,” “could,” “expect,” “estimate,” “design,” “goal,” “intend,” “may,” “might,” “objective,” “plan,” “predict,” “project,” “target,” “likely,” “should,” “will,” and “would,” or the negative of these terms and similar expressions or words, identify forward-looking statements. Forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Important factors that could cause actual results to differ materially from those reflected in Histogenics’ forward-looking statements include, among others:  the timing and success of Histogenics’ NeoCart Phase 3 clinical trial; possible delays in enrolling the NeoCart Phase 3 clinical trial; the ability to obtain and maintain regulatory approval of NeoCart or any product candidates, and the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing Histogenics’ product candidates; the ability to obtain and maintain regulatory approval regarding the comparability of critical NeoCart raw materials; the size and growth of the potential markets for Histogenics’ product candidates and the ability to serve those markets; Histogenics’ expectations regarding its expenses and revenue; the sufficiency of Histogenics’ cash resources and the availability of additional financing on commercially reasonable terms; the early stage of development of the technologies on which Histogenics’ channel partnering agreement with Intrexon Corporation is based; the additional expenses that Histogenics will incur in connection with its exclusive channel collaboration agreement with Intrexon Corporation and other factors that are described in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of Histogenics’ Annual Report on Form 10-K for the year ended December 31, 2016 which is on file with the SEC and available on the SEC’s website at www.sec.gov.  Additional factors may be set forth in those sections of Histogenics’ Quarterly Report on Form 10-Q for the quarter ended March 31, 2017, to be filed with the SEC in the second quarter of 2017.  In addition to the risks described above and in Histogenics’ annual report on Form 10-K and quarterly reports on Form 10-Q, current reports on Form 8-K and other filings with the SEC, other unknown or unpredictable factors also could affect Histogenics’ results. There can be no assurance that the actual results or developments anticipated by Histogenics will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Histogenics.  Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved. All written and verbal forward-looking statements attributable to Histogenics or any person acting on its behalf are expressly qualified in their entirety by the cautionary statements contained or referred to herein.  Histogenics cautions investors not to rely too heavily on the forward-looking statements Histogenics makes or that are made on its behalf.  The information in this release is provided only as of the date of this release, and Histogenics undertakes no obligation, and specifically declines any obligation, to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.


‒ NeoCart® Phase 3 Clinical Trial Enrollment Near Completion ‒ ‒ NeoCart Approval Pathway in Japan Defined After Successful Conclusion of Discussions with the Japan Pharmaceuticals and Medical Devices Agency ‒ ‒ Publication of Additional Data to Support Potential Approval and Commercialization of NeoCart ‒ ‒ Company to Host Conference Call and Webcast Today at 8:30 a.m. EDT ‒ WALTHAM, Mass., May 11, 2017 (GLOBE NEWSWIRE) -- Histogenics Corporation (Histogenics) (Nasdaq:HSGX), a regenerative medicine company focused on developing and commercializing products in the musculoskeletal space, announced its financial and operational results for the quarter ended March 31, 2017. “In the first quarter of 2017, we continued to execute on our important near- and long-term business objectives.  Importantly, we enrolled an additional 23 patients in the NeoCart Phase 3 clinical trial, and completed a series of productive formal meetings with the PMDA in Japan where we determined a near-term regulatory pathway for NeoCart to gain full Marketing Authorization in the Japanese market,” stated Adam Gridley, President and Chief Executive Officer of Histogenics.  “We are pleased to report continued strength in our enrollment of the clinical trial, which is expected to conclude this quarter.  We also continue to build on our strong portfolio of bench and clinical data with several new publications on the clinical and biomechanical performance of NeoCart.  This contributes to our goal of a rapid BLA filing in the second half of 2018, and helps prepare us for the potential commercialization of NeoCart in 2019, pending positive results from the NeoCart Phase 3 clinical trial and FDA approval,” continued Mr. Gridley. Financial Results for the First Quarter of 2017 Loss from operations was $(6.8) million in the first quarter of 2017, compared to $(7.8) million in the first quarter of 2016.  The decrease in operating expenses was driven by a reduction in research and development expenses and partially offset by a small increase in general and administrative expenses. Research and development expenses were $4.5 million in the first quarter of 2017, compared to $5.6 million in the first quarter of 2016.  The decrease was primarily due to a reduction in consulting and temporary labor costs and patient recruiting expenses related to the NeoCart Phase 3 clinical trial.  This decrease was partially offset by an increase in hiring fees in the first quarter of 2017.  General and administrative expenses were $2.3 million in the first quarter of 2017, compared to $2.2 million in the first quarter of 2016.  The increase was primarily due to increases in stock-based compensation and repairs and maintenance expense that were partially offset by a reduction in hiring fees and consulting costs. Net loss attributable to common stockholders was $(5.8) million in first quarter of 2017, or $(0.27) per share, compared to $(7.9) million, or $(0.60) per share, in the first quarter of 2016.  The decrease in net loss attributable to common stockholders is primarily due to lower operating expenses and the allocation of a portion of the net loss to the Series A Preferred Stock. As of March 31, 2017, Histogenics had cash, cash equivalents and marketable securities of $24.4 million, compared to $31.9 million at December 31, 2016.  Histogenics believes its current cash position will be sufficient to fund its operations into the middle of 2018. Histogenics’ management will host a conference call on Thursday, May 11, 2017 at 8:30 a.m. EDT.  A question-and-answer session will follow Histogenics’ remarks.  To participate on the live call, please dial (877) 930-8064 (domestic) or (253) 336-8040 (international) and provide the conference ID “97247103” five to ten minutes before the start of the call. A live audio webcast of the presentation will be available via the “Investor Relations” page of the Histogenics website, www.histogenics.com. A replay of the webcast will be archived on Histogenics’ website for approximately 45 days following the presentation. Histogenics is a leading regenerative medicine company developing and commercializing novel tissue therapies that may offer more rapid and durable recoveries for patients with pain and loss of function due to musculoskeletal conditions.  Histogenics’ regenerative medicine platform combines expertise in cell processing, scaffolding, tissue engineering and bioadhesives to create tissue ex-vivo.  Histogenics’ first investigational product candidate, NeoCart is designed to treat cartilage defects in the knee and is currently in Phase 3 clinical development.  NeoCart is designed to exhibit characteristics of articular, hyaline cartilage prior to and upon implantation into the knee and therefore does not rely on the body to make new cartilage.  As a result, NeoCart is the only product in development or on the market with a one-year primary superiority endpoint as compared to the standard of care.  There are more than 500,000 or more knee cartilage procedures in the United States each year, with many healthy active adults avoiding treatment as they seek other alternatives.  Left untreated, even a small cartilage defect can expand in size and progress to debilitating osteoarthritis, ultimately necessitating a joint replacement procedure.  Osteoarthritis is more common in adults over the age of 50, but the condition and precursors of the condition can be observed much earlier, and cartilage damage is believed to be one of the leading contributors of this disease.  For more information, please visit www.histogenics.com. Various statements in this release are “forward-looking statements” under the securities laws. Words such as, but not limited to, “anticipate,” “believe,” “can,” “could,” “expect,” “estimate,” “design,” “goal,” “intend,” “may,” “might,” “objective,” “plan,” “predict,” “project,” “target,” “likely,” “should,” “will,” and “would,” or the negative of these terms and similar expressions or words, identify forward-looking statements. Forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Important factors that could cause actual results to differ materially from those reflected in Histogenics’ forward-looking statements include, among others:  the timing and success of Histogenics’ NeoCart Phase 3 clinical trial; possible delays in enrolling the NeoCart Phase 3 clinical trial; the ability to obtain and maintain regulatory approval of NeoCart or any product candidates, and the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing Histogenics’ product candidates; the ability to obtain and maintain regulatory approval regarding the comparability of critical NeoCart raw materials; the size and growth of the potential markets for Histogenics’ product candidates and the ability to serve those markets; Histogenics’ expectations regarding its expenses and revenue; the sufficiency of Histogenics’ cash resources and the availability of additional financing on commercially reasonable terms; the early stage of development of the technologies on which Histogenics’ channel partnering agreement with Intrexon Corporation is based; the additional expenses that Histogenics will incur in connection with its exclusive channel collaboration agreement with Intrexon Corporation and other factors that are described in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of Histogenics’ Annual Report on Form 10-K for the year ended December 31, 2016 which is on file with the SEC and available on the SEC’s website at www.sec.gov.  Additional factors may be set forth in those sections of Histogenics’ Quarterly Report on Form 10-Q for the quarter ended March 31, 2017, to be filed with the SEC in the second quarter of 2017.  In addition to the risks described above and in Histogenics’ annual report on Form 10-K and quarterly reports on Form 10-Q, current reports on Form 8-K and other filings with the SEC, other unknown or unpredictable factors also could affect Histogenics’ results. There can be no assurance that the actual results or developments anticipated by Histogenics will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Histogenics.  Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved. All written and verbal forward-looking statements attributable to Histogenics or any person acting on its behalf are expressly qualified in their entirety by the cautionary statements contained or referred to herein.  Histogenics cautions investors not to rely too heavily on the forward-looking statements Histogenics makes or that are made on its behalf.  The information in this release is provided only as of the date of this release, and Histogenics undertakes no obligation, and specifically declines any obligation, to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.


‒ NeoCart® Phase 3 Clinical Trial Enrollment Near Completion ‒ ‒ NeoCart Approval Pathway in Japan Defined After Successful Conclusion of Discussions with the Japan Pharmaceuticals and Medical Devices Agency ‒ ‒ Publication of Additional Data to Support Potential Approval and Commercialization of NeoCart ‒ ‒ Company to Host Conference Call and Webcast Today at 8:30 a.m. EDT ‒ WALTHAM, Mass., May 11, 2017 (GLOBE NEWSWIRE) -- Histogenics Corporation (Histogenics) (Nasdaq:HSGX), a regenerative medicine company focused on developing and commercializing products in the musculoskeletal space, announced its financial and operational results for the quarter ended March 31, 2017. “In the first quarter of 2017, we continued to execute on our important near- and long-term business objectives.  Importantly, we enrolled an additional 23 patients in the NeoCart Phase 3 clinical trial, and completed a series of productive formal meetings with the PMDA in Japan where we determined a near-term regulatory pathway for NeoCart to gain full Marketing Authorization in the Japanese market,” stated Adam Gridley, President and Chief Executive Officer of Histogenics.  “We are pleased to report continued strength in our enrollment of the clinical trial, which is expected to conclude this quarter.  We also continue to build on our strong portfolio of bench and clinical data with several new publications on the clinical and biomechanical performance of NeoCart.  This contributes to our goal of a rapid BLA filing in the second half of 2018, and helps prepare us for the potential commercialization of NeoCart in 2019, pending positive results from the NeoCart Phase 3 clinical trial and FDA approval,” continued Mr. Gridley. Financial Results for the First Quarter of 2017 Loss from operations was $(6.8) million in the first quarter of 2017, compared to $(7.8) million in the first quarter of 2016.  The decrease in operating expenses was driven by a reduction in research and development expenses and partially offset by a small increase in general and administrative expenses. Research and development expenses were $4.5 million in the first quarter of 2017, compared to $5.6 million in the first quarter of 2016.  The decrease was primarily due to a reduction in consulting and temporary labor costs and patient recruiting expenses related to the NeoCart Phase 3 clinical trial.  This decrease was partially offset by an increase in hiring fees in the first quarter of 2017.  General and administrative expenses were $2.3 million in the first quarter of 2017, compared to $2.2 million in the first quarter of 2016.  The increase was primarily due to increases in stock-based compensation and repairs and maintenance expense that were partially offset by a reduction in hiring fees and consulting costs. Net loss attributable to common stockholders was $(5.8) million in first quarter of 2017, or $(0.27) per share, compared to $(7.9) million, or $(0.60) per share, in the first quarter of 2016.  The decrease in net loss attributable to common stockholders is primarily due to lower operating expenses and the allocation of a portion of the net loss to the Series A Preferred Stock. As of March 31, 2017, Histogenics had cash, cash equivalents and marketable securities of $24.4 million, compared to $31.9 million at December 31, 2016.  Histogenics believes its current cash position will be sufficient to fund its operations into the middle of 2018. Histogenics’ management will host a conference call on Thursday, May 11, 2017 at 8:30 a.m. EDT.  A question-and-answer session will follow Histogenics’ remarks.  To participate on the live call, please dial (877) 930-8064 (domestic) or (253) 336-8040 (international) and provide the conference ID “97247103” five to ten minutes before the start of the call. A live audio webcast of the presentation will be available via the “Investor Relations” page of the Histogenics website, www.histogenics.com. A replay of the webcast will be archived on Histogenics’ website for approximately 45 days following the presentation. Histogenics is a leading regenerative medicine company developing and commercializing novel tissue therapies that may offer more rapid and durable recoveries for patients with pain and loss of function due to musculoskeletal conditions.  Histogenics’ regenerative medicine platform combines expertise in cell processing, scaffolding, tissue engineering and bioadhesives to create tissue ex-vivo.  Histogenics’ first investigational product candidate, NeoCart is designed to treat cartilage defects in the knee and is currently in Phase 3 clinical development.  NeoCart is designed to exhibit characteristics of articular, hyaline cartilage prior to and upon implantation into the knee and therefore does not rely on the body to make new cartilage.  As a result, NeoCart is the only product in development or on the market with a one-year primary superiority endpoint as compared to the standard of care.  There are more than 500,000 or more knee cartilage procedures in the United States each year, with many healthy active adults avoiding treatment as they seek other alternatives.  Left untreated, even a small cartilage defect can expand in size and progress to debilitating osteoarthritis, ultimately necessitating a joint replacement procedure.  Osteoarthritis is more common in adults over the age of 50, but the condition and precursors of the condition can be observed much earlier, and cartilage damage is believed to be one of the leading contributors of this disease.  For more information, please visit www.histogenics.com. Various statements in this release are “forward-looking statements” under the securities laws. Words such as, but not limited to, “anticipate,” “believe,” “can,” “could,” “expect,” “estimate,” “design,” “goal,” “intend,” “may,” “might,” “objective,” “plan,” “predict,” “project,” “target,” “likely,” “should,” “will,” and “would,” or the negative of these terms and similar expressions or words, identify forward-looking statements. Forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Important factors that could cause actual results to differ materially from those reflected in Histogenics’ forward-looking statements include, among others:  the timing and success of Histogenics’ NeoCart Phase 3 clinical trial; possible delays in enrolling the NeoCart Phase 3 clinical trial; the ability to obtain and maintain regulatory approval of NeoCart or any product candidates, and the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing Histogenics’ product candidates; the ability to obtain and maintain regulatory approval regarding the comparability of critical NeoCart raw materials; the size and growth of the potential markets for Histogenics’ product candidates and the ability to serve those markets; Histogenics’ expectations regarding its expenses and revenue; the sufficiency of Histogenics’ cash resources and the availability of additional financing on commercially reasonable terms; the early stage of development of the technologies on which Histogenics’ channel partnering agreement with Intrexon Corporation is based; the additional expenses that Histogenics will incur in connection with its exclusive channel collaboration agreement with Intrexon Corporation and other factors that are described in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of Histogenics’ Annual Report on Form 10-K for the year ended December 31, 2016 which is on file with the SEC and available on the SEC’s website at www.sec.gov.  Additional factors may be set forth in those sections of Histogenics’ Quarterly Report on Form 10-Q for the quarter ended March 31, 2017, to be filed with the SEC in the second quarter of 2017.  In addition to the risks described above and in Histogenics’ annual report on Form 10-K and quarterly reports on Form 10-Q, current reports on Form 8-K and other filings with the SEC, other unknown or unpredictable factors also could affect Histogenics’ results. There can be no assurance that the actual results or developments anticipated by Histogenics will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Histogenics.  Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved. All written and verbal forward-looking statements attributable to Histogenics or any person acting on its behalf are expressly qualified in their entirety by the cautionary statements contained or referred to herein.  Histogenics cautions investors not to rely too heavily on the forward-looking statements Histogenics makes or that are made on its behalf.  The information in this release is provided only as of the date of this release, and Histogenics undertakes no obligation, and specifically declines any obligation, to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.


News Article | May 11, 2017
Site: www.businesswire.com

WASHINGTON--(BUSINESS WIRE)--DIA, (founded as the Drug Information Association), announced today keynote speaker Alexander Tsiaras, 11 tracks, more than 160 sessions, and a featured career fair at the DIA 2017 Global Annual Meeting, June 18 to 22 in Chicago, IL. Appropriately themed “Driving Insights to Action”, the DIA 2017 Global Annual Meeting is designed to foster the international exchange of actionable insights to improve health globally through the advancement of lifesaving medicines and technologies. An integral part of the health and life sciences community, this the platform on which all global stakeholders in the drug product development life cycle have an opportunity to converse, debate, and raise questions about the evolution of novel therapies, the pharmaceutical research and development (R&D) landscape, and regulatory challenges to drive action throughout health care product development. With traditional educational sessions and innovative learning experiences, the program includes themed tracks with scientific sessions focused on today’s hottest topics ranging from big data, medical affairs, and clinical operations to patient engagement, clinical trial safety, and value and access. In the keynote address, Interactive Storytelling and Personal Health Data Drives Unprecedented Patient Empowerment, Alexander Tsiaras will share how the fusion of personal stories of disease, powerful patient data, and visceral interactive experiences can drive greater patient engagement and outcomes. “With the changes in governance around the world, the continuing economic uncertainties, the ever-increasing consumer involvement, the regulatory challenges, plus the continuing pressure for even higher quality and innovative products, the life sciences industry is facing myriad challenges,” said Barbara Lopez Kunz, Global Chief Executive, DIA. “It’s important for all stakeholders in this arena to openly discuss these challenges, find ways to overcome them and take action to help move our ecosystem forward. Our global annual meeting provides the essential platform by which to do just that. Insights and outcomes garnered at this meeting will advance innovation to deliver safe, effective, and accessible healthcare products to patients.” DIA 2017 will host over 7,000 professionals from the pharmaceutical, biotechnology and medical device communities from over 50 countries for the five-day conference, which also features a three-day expo with more than 450 exhibiting companies. In addition to health care and life sciences industry professionals, academia, and students, representatives from global regulatory bodies such as: Food and Drug Administration (FDA), European Medicines Agency (EMA), International Coalition of Medicines Regulatory Authorities (ICMRA), Pharmaceuticals and Medical Devices Agency (PMDA), Health Canada, The Brazilian Health Regulatory Agency (Anvisa), National Center for Advancing Translational Sciences (NCATS) and Agency for Healthcare Research and Quality (AHRQ) will be in attendance. More importantly, these same groups of people will serve as session chairs and speakers, igniting questions, inspiring thought, and driving action. To learn more and register for DIA 2017, visit www.DIAglobal.org/DIA2017. For live updates, attendees and the public should follow and join the conversation on Twitter, @DrugInfoAssn using the hashtag #DIA2017. DIA (founded as the Drug Information Association) is an international, nonprofit, multidisciplinary member association that provides health care product development professionals a neutral and transparent forum for collaboration and the exchange of insights to improve health globally through the advancement of lifesaving medicines and technologies. DIA builds knowledge through, learning solutions (digital and in person training), conferences and insights in the areas of Regulatory Science, Translational Medicine, Patient Engagement and Value and Access for professionals in the pharmaceutical, biotechnology, and medical device communities. DIA is based in Washington, DC (US) with regional offices representing the Americas (Horsham, PA, US); Europe, the Middle East and Africa, (Basel, Switzerland); and Asia (Beijing and Shanghai, China; Mumbai, India; and Tokyo, Japan). For more information, visit www.DIAglobal.org or connect with us on Twitter, LinkedIn, Facebook, and Instagram.


‒ NeoCart® Phase 3 Clinical Trial Enrollment Near Completion ‒ ‒ NeoCart Approval Pathway in Japan Defined After Successful Conclusion of Discussions with the Japan Pharmaceuticals and Medical Devices Agency ‒ ‒ Publication of Additional Data to Support Potential Approval and Commercialization of NeoCart ‒ ‒ Company to Host Conference Call and Webcast Today at 8:30 a.m. EDT ‒ WALTHAM, Mass., May 11, 2017 (GLOBE NEWSWIRE) -- Histogenics Corporation (Histogenics) (Nasdaq:HSGX), a regenerative medicine company focused on developing and commercializing products in the musculoskeletal space, announced its financial and operational results for the quarter ended March 31, 2017. “In the first quarter of 2017, we continued to execute on our important near- and long-term business objectives.  Importantly, we enrolled an additional 23 patients in the NeoCart Phase 3 clinical trial, and completed a series of productive formal meetings with the PMDA in Japan where we determined a near-term regulatory pathway for NeoCart to gain full Marketing Authorization in the Japanese market,” stated Adam Gridley, President and Chief Executive Officer of Histogenics.  “We are pleased to report continued strength in our enrollment of the clinical trial, which is expected to conclude this quarter.  We also continue to build on our strong portfolio of bench and clinical data with several new publications on the clinical and biomechanical performance of NeoCart.  This contributes to our goal of a rapid BLA filing in the second half of 2018, and helps prepare us for the potential commercialization of NeoCart in 2019, pending positive results from the NeoCart Phase 3 clinical trial and FDA approval,” continued Mr. Gridley. Financial Results for the First Quarter of 2017 Loss from operations was $(6.8) million in the first quarter of 2017, compared to $(7.8) million in the first quarter of 2016.  The decrease in operating expenses was driven by a reduction in research and development expenses and partially offset by a small increase in general and administrative expenses. Research and development expenses were $4.5 million in the first quarter of 2017, compared to $5.6 million in the first quarter of 2016.  The decrease was primarily due to a reduction in consulting and temporary labor costs and patient recruiting expenses related to the NeoCart Phase 3 clinical trial.  This decrease was partially offset by an increase in hiring fees in the first quarter of 2017.  General and administrative expenses were $2.3 million in the first quarter of 2017, compared to $2.2 million in the first quarter of 2016.  The increase was primarily due to increases in stock-based compensation and repairs and maintenance expense that were partially offset by a reduction in hiring fees and consulting costs. Net loss attributable to common stockholders was $(5.8) million in first quarter of 2017, or $(0.27) per share, compared to $(7.9) million, or $(0.60) per share, in the first quarter of 2016.  The decrease in net loss attributable to common stockholders is primarily due to lower operating expenses and the allocation of a portion of the net loss to the Series A Preferred Stock. As of March 31, 2017, Histogenics had cash, cash equivalents and marketable securities of $24.4 million, compared to $31.9 million at December 31, 2016.  Histogenics believes its current cash position will be sufficient to fund its operations into the middle of 2018. Histogenics’ management will host a conference call on Thursday, May 11, 2017 at 8:30 a.m. EDT.  A question-and-answer session will follow Histogenics’ remarks.  To participate on the live call, please dial (877) 930-8064 (domestic) or (253) 336-8040 (international) and provide the conference ID “97247103” five to ten minutes before the start of the call. A live audio webcast of the presentation will be available via the “Investor Relations” page of the Histogenics website, www.histogenics.com. A replay of the webcast will be archived on Histogenics’ website for approximately 45 days following the presentation. Histogenics is a leading regenerative medicine company developing and commercializing novel tissue therapies that may offer more rapid and durable recoveries for patients with pain and loss of function due to musculoskeletal conditions.  Histogenics’ regenerative medicine platform combines expertise in cell processing, scaffolding, tissue engineering and bioadhesives to create tissue ex-vivo.  Histogenics’ first investigational product candidate, NeoCart is designed to treat cartilage defects in the knee and is currently in Phase 3 clinical development.  NeoCart is designed to exhibit characteristics of articular, hyaline cartilage prior to and upon implantation into the knee and therefore does not rely on the body to make new cartilage.  As a result, NeoCart is the only product in development or on the market with a one-year primary superiority endpoint as compared to the standard of care.  There are more than 500,000 or more knee cartilage procedures in the United States each year, with many healthy active adults avoiding treatment as they seek other alternatives.  Left untreated, even a small cartilage defect can expand in size and progress to debilitating osteoarthritis, ultimately necessitating a joint replacement procedure.  Osteoarthritis is more common in adults over the age of 50, but the condition and precursors of the condition can be observed much earlier, and cartilage damage is believed to be one of the leading contributors of this disease.  For more information, please visit www.histogenics.com. Various statements in this release are “forward-looking statements” under the securities laws. Words such as, but not limited to, “anticipate,” “believe,” “can,” “could,” “expect,” “estimate,” “design,” “goal,” “intend,” “may,” “might,” “objective,” “plan,” “predict,” “project,” “target,” “likely,” “should,” “will,” and “would,” or the negative of these terms and similar expressions or words, identify forward-looking statements. Forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Important factors that could cause actual results to differ materially from those reflected in Histogenics’ forward-looking statements include, among others:  the timing and success of Histogenics’ NeoCart Phase 3 clinical trial; possible delays in enrolling the NeoCart Phase 3 clinical trial; the ability to obtain and maintain regulatory approval of NeoCart or any product candidates, and the labeling for any approved products; the scope, progress, expansion, and costs of developing and commercializing Histogenics’ product candidates; the ability to obtain and maintain regulatory approval regarding the comparability of critical NeoCart raw materials; the size and growth of the potential markets for Histogenics’ product candidates and the ability to serve those markets; Histogenics’ expectations regarding its expenses and revenue; the sufficiency of Histogenics’ cash resources and the availability of additional financing on commercially reasonable terms; the early stage of development of the technologies on which Histogenics’ channel partnering agreement with Intrexon Corporation is based; the additional expenses that Histogenics will incur in connection with its exclusive channel collaboration agreement with Intrexon Corporation and other factors that are described in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of Histogenics’ Annual Report on Form 10-K for the year ended December 31, 2016 which is on file with the SEC and available on the SEC’s website at www.sec.gov.  Additional factors may be set forth in those sections of Histogenics’ Quarterly Report on Form 10-Q for the quarter ended March 31, 2017, to be filed with the SEC in the second quarter of 2017.  In addition to the risks described above and in Histogenics’ annual report on Form 10-K and quarterly reports on Form 10-Q, current reports on Form 8-K and other filings with the SEC, other unknown or unpredictable factors also could affect Histogenics’ results. There can be no assurance that the actual results or developments anticipated by Histogenics will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Histogenics.  Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved. All written and verbal forward-looking statements attributable to Histogenics or any person acting on its behalf are expressly qualified in their entirety by the cautionary statements contained or referred to herein.  Histogenics cautions investors not to rely too heavily on the forward-looking statements Histogenics makes or that are made on its behalf.  The information in this release is provided only as of the date of this release, and Histogenics undertakes no obligation, and specifically declines any obligation, to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.


EVENITY is an investigational bone-forming agent that rapidly increases bone formation and reduces bone resorption simultaneously, increases bone mineral density and reduces the risk of fracture. In this study, women received subcutaneous injection of EVENITY monthly for 12 months followed by oral alendronate weekly for at least 12 months. At 24 months, women in the EVENITY treatment group experienced a statistically significant 50 percent reduction in the relative risk of a new vertebral (spine) fracture compared to those receiving alendronate alone. Women in the EVENITY treatment group also experienced a statistically significant 27 percent reduction in the relative risk of clinical fracture (non-vertebral fracture and clinical vertebral fracture) at the primary analysis. Additionally, non-vertebral fractures were statistically significantly reduced by 19 percent in the EVENITY treatment group, including a nominally significant reduction in hip fractures. "We are impressed with the statistically significant superior fracture risk reduction of EVENITY over alendronate, a current standard of care in osteoporosis. When we think that patients who have had a fracture are highly likely to suffer another one, the importance of post-fracture care cannot be emphasized enough," said Iris Loew-Friedrich, UCB's chief medical officer. "We are working on understanding the observed cardiovascular safety signal and will continue to discuss these results with global regulators and experts in the field." Overall adverse events and serious adverse events were generally similar between the treatment groups throughout the study and also in the initial 12-month EVENITY treatment period. In the initial 12-month EVENITY treatment period, the three most commonly reported adverse events in both arms were nasopharyngitis, back pain and arthralgia. Injection site reactions were reported in 4.4 percent of patients in the EVENITY treatment group and 2.6 percent in the alendronate group during the initial 12-month period. Most injection site reactions were reported as mild in severity. During the open-label alendronate period, there were two positively adjudicated events of osteonecrosis of the jaw, one in a patient treated with EVENITY followed by alendronate and one treated with alendronate alone. There were six patients with positively adjudicated events of atypical femoral fracture during the open-label alendronate period (two patients treated with EVENITY followed by alendronate and four treated with alendronate alone). The patient incidence of positively adjudicated cardiovascular serious adverse events at 12 months was 2.5 percent in the EVENITY group compared to 1.9 percent in the alendronate group. No imbalance in cardiovascular serious adverse events was seen in the 7,180-patient placebo-controlled FRAME study. Regulatory submissions for EVENITY based on the FRAME study results are currently under review with the U.S. Food and Drug Administration (FDA), Health Canada and the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan. Amgen has agreed with the FDA that the ARCH data should be considered in the regulatory review prior to the initial marketing authorization, and as a result the Company does not expect approval of EVENITY in the U.S. to occur in 2017.  Engagement with PMDA and Health Canada will occur as part of the ongoing review process. The preparation for the European regulatory submission will continue as planned. Further analysis of the Phase 3 ARCH study data is ongoing and will be submitted to a future medical conference and for publication. About EVENITY EVENITY is an investigational bone-forming monoclonal antibody and is not approved by any regulatory authority for the treatment of osteoporosis. It is designed to work by inhibiting the activity of sclerostin and has a dual effect on bone, increasing bone formation and decreasing bone resorption. EVENITY is being studied for its potential to reduce the risk of fractures in an extensive global Phase 3 program. This program includes two large fracture trials comparing EVENITY to either placebo or active comparator in more than 10,000 postmenopausal women with osteoporosis. Amgen and UCB are co-developing EVENITY. About the ARCH study ARCH (Active-contRolled FraCture Study in Postmenopausal Women with Osteoporosis at High Risk of Fracture) is a Phase 3 multicenter, international, randomized, double-blind, alendronate-controlled study of EVENITY in postmenopausal women with osteoporosis at high risk for fracture based on previous fracture history. The study evaluated 12 months of EVENITY treatment followed by at least 12 months of alendronate treatment, compared with alendronate treatment alone. The purpose of this study was to determine if EVENITY treatment is effective in reducing the incidence of clinical fracture (non-vertebral fracture and clinical vertebral fracture) and new vertebral fracture. The incidence of clinical fracture was event-driven and the primary analysis occurred when 330 fractures occurred or the last patient was on the study for 24 months, whichever was later. Patients (4,093) were randomized 1:1 to receive either 210 mg EVENITY subcutaneously every month or 70 mg alendronate orally every week for the duration of the 12-month double-blind alendronate-controlled study period. After the double-blind active-comparator study period, patients received alendronate while remaining blinded to their initial treatment assignment. About the FRAME study FRAME (FRActure study in postmenopausal woMen with ostEoporosis) is a multicenter, international, randomized, double-blind, placebo-controlled, parallel-group study in postmenopausal women with osteoporosis, defined as low bone mineral density at the total hip or femoral neck. The study evaluated the effectiveness of EVENITY treatment, compared with placebo, in reducing the risk of new vertebral fractures through 12 months. The study also further evaluated if EVENITY treatment for 12 months followed by denosumab treatment for 12 months, compared with placebo followed by denosumab treatment, was effective in reducing the risk of new vertebral fractures through 24 months. In addition, clinical fracture (a composite endpoint which encompasses all symptomatic fractures, both non-vertebral and painful vertebral fractures) risk reduction, non-vertebral fracture (fractures outside of the spine, excluding sites that are not considered osteoporotic, fractures due to high trauma or pathologic fractures) risk reduction and other endpoints were assessed at 12 and 24 months. 7,180 patients were randomized 1:1 to receive either 210 mg EVENITY subcutaneous (SC) monthly (QM) or placebo SC QM for the 12-month double-blind study period. After the placebo-controlled study period, patients entered the open-label phase where all patients received 60 mg denosumab SC every six months (Q6M) for 12 months, while remaining blinded to initial treatment. An additional 12 month extension period of open-label 60 mg denosumab SC Q6M is currently ongoing. About the Amgen and UCB Collaboration Since 2004, Amgen and UCB have been working together under a collaboration and license agreement to research, develop and market antibody products targeting the protein sclerostin. As part of this agreement, the two companies continue to collaborate on the development of EVENITY for the treatment of osteoporosis. This gene-to-drug project demonstrates how Amgen and UCB are joining forces to translate a genetic discovery into a new medicine, turning conceptual science into a reality. About Amgen Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology. Amgen focuses on areas of high unmet medical need and leverages its expertise to strive for solutions that improve health outcomes and dramatically improve people's lives. A biotechnology pioneer since 1980, Amgen has grown to be one of the world's leading independent biotechnology companies, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential. For more information, visit www.amgen.com and follow us on www.twitter.com/amgen. About UCB UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With more than 7 500 people in approximately 40 countries, the company generated revenue of € 4.2 billion in 2016. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news Amgen Forward-Looking Statements This news release contains forward-looking statements that are based on the current expectations and beliefs of Amgen. All statements, other than statements of historical fact, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial metrics, expected legal, arbitration, political, regulatory or clinical results or practices, customer and prescriber patterns or practices, reimbursement activities and outcomes and other such estimates and results. Forward-looking statements involve significant risks and uncertainties, including those discussed below and more fully described in the Securities and Exchange Commission reports filed by Amgen, including its most recent annual report on Form 10-K and any subsequent periodic reports on Form 10-Q and Form 8-K. Unless otherwise noted, Amgen is providing this information as of the date of this news release and does not undertake any obligation to update any forward-looking statements contained in this document as a result of new information, future events or otherwise. No forward-looking statement can be guaranteed and actual results may differ materially from those Amgen projects. Discovery or identification of new product candidates or development of new indications for existing products cannot be guaranteed and movement from concept to product is uncertain; consequently, there can be no guarantee that any particular product candidate or development of a new indication for an existing product will be successful and become a commercial product. Further, preclinical results do not guarantee safe and effective performance of product candidates in humans. The complexity of the human body cannot be perfectly, or sometimes, even adequately modeled by computer or cell culture systems or animal models. The length of time that it takes for Amgen to complete clinical trials and obtain regulatory approval for product marketing has in the past varied and Amgen expects similar variability in the future. Even when clinical trials are successful, regulatory authorities may question the sufficiency for approval of the trial endpoints Amgen has selected. Amgen develops product candidates internally and through licensing collaborations, partnerships and joint ventures. Product candidates that are derived from relationships may be subject to disputes between the parties or may prove to be not as effective or as safe as Amgen may have believed at the time of entering into such relationship. Also, Amgen or others could identify safety, side effects or manufacturing problems with its products after they are on the market. Amgen's results may be affected by its ability to successfully market both new and existing products domestically and internationally, clinical and regulatory developments involving current and future products, sales growth of recently launched products, competition from other products including biosimilars, difficulties or delays in manufacturing its products and global economic conditions. In addition, sales of Amgen's products are affected by pricing pressure, political and public scrutiny and reimbursement policies imposed by third-party payers, including governments, private insurance plans and managed care providers and may be affected by regulatory, clinical and guideline developments and domestic and international trends toward managed care and healthcare cost containment. Furthermore, Amgen's research, testing, pricing, marketing and other operations are subject to extensive regulation by domestic and foreign government regulatory authorities. Amgen or others could identify safety, side effects or manufacturing problems with its products after they are on the market. Amgen's business may be impacted by government investigations, litigation and product liability claims. In addition, Amgen's business may be impacted by the adoption of new tax legislation or exposure to additional tax liabilities. If Amgen fails to meet the compliance obligations in the corporate integrity agreement between it and the U.S. government, Amgen could become subject to significant sanctions. Further, while Amgen routinely obtains patents for its products and technology, the protection offered by its patents and patent applications may be challenged, invalidated or circumvented by its competitors, or Amgen may fail to prevail in present and future intellectual property litigation. Amgen performs a substantial amount of its commercial manufacturing activities at a few key manufacturing facilities and also depends on third parties for a portion of its manufacturing activities, and limits on supply may constrain sales of certain of its current products and product candidate development. In addition, Amgen competes with other companies with respect to many of its marketed products as well as for the discovery and development of new products. Further, some raw materials, medical devices and component parts for Amgen's products are supplied by sole third-party suppliers. Certain of Amgen's distributors, customers and payers have substantial purchasing leverage in their dealings with Amgen. The discovery of significant problems with a product similar to one of Amge's products that implicate an entire class of products could have a material adverse effect on sales of the affected products and on its business and results of operations. Amgen's efforts to acquire other companies or products and to integrate the operations of companies Amgen has acquired may not be successful. Amgen may not be able to access the capital and credit markets on terms that are favorable to it, or at all. Amgen is increasingly dependent on information technology systems, infrastructure and data security. Amgen's stock price may be volatile and may be affected by a number of events. Amgen's business performance could affect or limit the ability of the Amgen Board of Directors to declare a dividend or its ability to pay a dividend or repurchase its common stock. The scientific information discussed in this news release related to Amgen's product candidates is preliminary and investigative. Such product candidates are not approved by the U.S. Food and Drug Administration, and no conclusions can or should be drawn regarding the safety or effectiveness of the product candidates. UCB Forward-Looking Statements This press release contains forward-looking statements based on current plans, estimates and beliefs of management. All statements, other than statements of historical fact, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, political, regulatory or clinical results and other such estimates and results. By their nature, such forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and assumptions which could cause actual results to differ materially from those that may be implied by such forward-looking statements contained in this press release. Important factors that could result in such differences include: changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, product liability claims, challenges to patent protection for products or product candidates, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws and hiring and retention of its employees. UCB is providing this information as of the date of this press release and expressly disclaims any duty to update any information contained in this press release, either to confirm the actual results or to report a change in its expectations. There is no guarantee that new product candidates in the pipeline will progress to product approval or that new indications for existing products will be developed and approved. Products or potential products which are the subject of partnerships, joint ventures or licensing collaborations may be subject to differences between the partners. Also, UCB or others could discover safety, side effects or manufacturing problems with its products after they are marketed. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement. To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/amgen-and-ucb-announce-top-line-phase-3-data-from-active-comparator-study-of-evenity-romosozumab-in-postmenopausal-women-with-osteoporosis-300461160.html


News Article | May 24, 2017
Site: www.businesswire.com

SAN DIEGO--(BUSINESS WIRE)--Quidel Corporation (NASDAQ: QDEL), a provider of rapid diagnostic testing solutions, cellular-based virology assays and molecular diagnostic systems, announced today it has received approval from Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for Quidel’s Sofia® Influenza A+B Fluorescent Immunoassay (FIA) to be used with the Sofia® Fluorescent Immunoassay Analyzer. Sofia is the brand name for Quidel’s instrumented immunoassay system. The easy-to-use Sofia Analyzer and Sofia Influenza A+B FIA combine unique software and fluorescent chemistry to yield an automatic, objective result that is readily available on the instrument’s screen, in a hard-copy printout, and in a transmissible electronic form that can network via an LIS system to hospital and medical center databases. The Sofia FIA employs advanced lateral flow and immunofluorescence technologies to provide enhanced clinical sensitivity for influenza A and B. The Sofia Analyzer provides for high-throughput batching methods, and other features designed to facilitate use in a variety of healthcare settings, including hospitals, medical centers, small clinics and doctors’ offices. These features help ensure a reliable, objective, rapid, and accurate diagnostic result. It is estimated that there were between 13 million to 16 million confirmed cases of Influenza in Japan every year between 2010 and 2014.1 “ We are pleased to have received approval in Japan for our Sofia Influenza A+B assay, and are excited about the opportunity to expand Sofia’s international footprint,” said Douglas Bryant, president and chief executive officer of Quidel Corporation. The Sofia® Fluorescent Immunoassay Analyzer, Sofia® Group A Strep Assay, and Sofia® RSV assay are also currently available for sale in Japan. Quidel Corporation serves to enhance the health and well-being of people around the globe through the development of diagnostic solutions that can lead to improved patient outcomes and provide economic benefits to the healthcare system. Marketed under the Sofia®, QuickVue®, D3® Direct Detection, Thyretain® and InflammaDry® leading brand names, as well as under the new Solana®, AmpliVue® and Lyra® molecular diagnostic brands, Quidel’s products aid in the detection and diagnosis of many critical diseases and conditions, including, among others, influenza, respiratory syncytial virus, Strep A, herpes, pregnancy, thyroid disease and fecal occult blood. Quidel’s research and development engine is also developing a continuum of diagnostic solutions from advanced lateral-flow and direct fluorescent antibody to molecular diagnostic tests to further improve the quality of healthcare in physicians’ offices and hospital and reference laboratories. For more information about Quidel’s comprehensive product portfolio, visit quidel.com. This press release contains forward-looking statements within the meaning of the federal securities laws that involve material risks, assumptions and uncertainties. Many possible events or factors could affect our future financial results and performance, such that our actual results and performance may differ materially from those that may be described or implied in the forward-looking statements. As such, no forward-looking statement can be guaranteed. Differences in actual results and performance may arise as a result of a number of factors including, without limitation, our reliance on development of new technologies, fluctuations in our operating results resulting from the timing of the onset, length and severity of cold and flu seasons, seasonality, government and media attention focused on influenza and the related potential impact on humans from novel influenza viruses, adverse changes in competitive conditions in domestic and international markets, the reimbursement system currently in place and future changes to that system, changes in economic conditions in our domestic and international markets, lower than anticipated market penetration of our products, the quantity of our product in our distributors’ inventory or distribution channels, changes in the buying patterns of our distributors, and changes in the healthcare market and consolidation of our customer base; our development and protection of proprietary technology rights; our development of new technologies, products and markets; our reliance on a limited number of key distributors; our reliance on sales of our influenza diagnostics tests; our ability to manage our growth strategy, including our ability to integrate companies or technologies we have acquired or may acquire; intellectual property risks, including but not limited to, infringement litigation; our inability to settle conversions of our Convertible Senior Notes in cash; the effect on our operating results from the trigger of the conditional conversion feature of our Convertible Senior Notes; our need for additional funds to finance our capital or operating needs; the financial soundness of our customers and suppliers; acceptance of our products among physicians and other healthcare providers; competition with other providers of diagnostic products; adverse actions or delays in new product reviews or related to currently-marketed products by the U.S. Food and Drug Administration (the “FDA”); changes in government policies; compliance with other government regulations, such as safe working conditions, manufacturing practices, environmental protection, fire hazard and disposal of hazardous substances; third-party reimbursement policies; our ability to meet demand for our products; interruptions in our supply of raw materials; product defects; business risks not covered by insurance and exposure to other litigation claims; interruption to our computer systems; competition for and loss of management and key personnel; international risks, including but not limited to, compliance with product registration requirements, exposure to currency exchange fluctuations and foreign currency exchange risk sharing arrangements, longer payment cycles, lower selling prices and greater difficulty in collecting accounts receivable, reduced protection of intellectual property rights, political and economic instability, taxes, and diversion of lower priced international products into U.S. markets; dilution resulting from future sales of our equity; volatility in our stock price; provisions in our charter documents, Delaware law and the indenture covering our Convertible Senior Notes that might delay or impede stockholder actions with respect to business combinations or similar transactions; and our intention of not paying dividends. Forward-looking statements typically are identified by the use of terms such as “may,” “will,” “should,” “might,” “expect,” “anticipate,” “estimate,” “plan,” “intend,” “goal,” “project,” “strategy,” “future,” and similar words, although some forward-looking statements are expressed differently. The risks described in reports and registration statements that we file with the Securities and Exchange Commission (the “SEC”) from time to time, should be carefully considered. You are cautioned not to place undue reliance on these forward-looking statements, which reflect management’s analysis only as of the date of this press release. Except as required by law, we undertake no obligation to publicly release the results of any revision or update of these forward-looking statements, whether as a result of new information, future events or otherwise.


Researchers at Okayama University report in the Journal of Artificial Organs the promising performance of a retinal prosthesis material that can convert external light stimuli into electric potentials that are picked up by neurons. The results are an important step towards curing certain hereditary eye diseases. Retinitis pigmentosa is a hereditary retinal disease, causing blindness due to dead photoreceptor cells but with other retinal neurons being alive. A potential remedy for patients diagnosed with this disease are prostheses replacing the non-functioning photoreceptor cells with artificial sensors, and making use of the functioning of the remaining, living neurons. Now, a team of researchers from Okayama University led by Toshihiko Matsuo & Tetsuya Uchida has quantitatively tested the response of a promising type of prosthesis, when implanted in rats, to external light flashes — a crucial step in the further development of retinal prostheses. The prosthesis tested by the researchers was developed earlier at Okayama University, and is known as Okayama University-type retinal prosthesis (OURePTM). The main component of OURePTM is a photoelectric dye: an organic molecule capable of converting light into electric potentials. Uchida and colleagues attached the dye molecules to a thin film of polyethylene — a safe and stable biocompatible material — and implanted the dye-coupled film (of size 1 mm x 5 mm) subretinally in the eyes of 10 male rats 6 weeks old. Cranial electrodes for registering potentials were attached 2–3 weeks later. The researchers also implanted plain, polyethylene films in 10 other rats for comparison purposes. The scientists tested the response of the implanted OURePTM sensors to flashing white light-emitting diodes (LEDs) placed on the surface of the rats’ corneas (the transparent front parts of the eyes) for different background-light conditions. The experiments were done with the rats anesthetized, and after appropriate periods of adaptation to dark or light conditions. Comparisons between results obtained with dye-coupled films and plain polyethylene films showed that the former led to visually evoked potentials — confirming the potential of OURePTM as a retinal prosthesis for treating diseases like retinitis pigmentosa. The use of OURePTM poses no toxicity issues and, because of the high density of the dye molecules on the polyethylene film, offers a high spatial resolution. Regarding tests on human eyes, Matsuo and colleagues point out that “a first-in-human clinical trial for OURePTM at Okayama University Hospital ... will be planned in consultation with [Japan’s] Pharmaceuticals and Medical Devices Agency.” Background OURePTM Okayama University-type retinal prosthesis (OURePTM), developed at Okayama University, is a material that mimics the function of photoreceptor cells present in mammalian eyes: phototransduction, the conversion of light into neuron signals triggering biological processes. The main component of OURePTM are 2-[2-[4-(dibutylamino)phenyl]ethenyl]-3-carboxymethylbenzothiazolium bromide molecules, photoelectric dye molecules that can convert light into electric potentials. These molecules are attached to the surface of a thin polyethylene film; the resulting dye-coupled OURePTM film can be used as an implant replacing non-functioning photoreceptor cells. OURePTM is a particularly promising prosthesis in situations — such as for the disease retinitis pigmentosa — where other retinal neuron cells, sending neuron signals to the brain, are still active. Competing strategies Other approaches for remedying dead photoreceptor cells rely on the use of a digital video camera integrated into glasses worn by the patient. The video images are converted to electrical signals transmitted to a receiver implanted in the patient’s body, after which electric currents are sent out of an array of electrodes implanted around the patient’s retina. Challenges associated with this type of approach, as e.g. implemented in the Argus IITM Retinal Prosthesis System (Second Sight, Inc.), include the miniaturization of the components, their biocompatibility, achieving high spatial resolution, and the need for an external power source. As demonstrated by the team led by Toshihiko Matsuo, the use of OURePTM overcomes these issues.. Reference Alamusi, Toshihiko Matsuo, Osamu Hosoya & Tetsuya Uchida. Visual evoked potential in RCS rats with Okayama University-type retinal prosthesis (OURePTM) implantation. Journal of Artificial Organs, 08 February, 2017. DOI:10.1007/s10047-016-0943-4 https://link.springer.com/article/10.1007%2Fs10047-016-0943-4


LYON, France--(BUSINESS WIRE)--POXEL SA (Euronext – POXEL - FR0012432516), a biopharmaceutical company focused on the development of innovative drugs to treat metabolic diseases, including type 2 diabetes, announced today positive top line Phase 2b data results for Imeglimin for the treatment of type 2 diabetes in Japan. The randomized, double-blind, placebo-controlled study of Imeglimin administered twice-daily for 24 weeks, demonstrated dose-dependent efficacy on two key measures of diabetes control in 299 Japanese patients. The trial achieved statistical significance (p<0.0001) for its primary endpoint of glycated hemoglobin A1c reduction versus placebo in all treatment groups at 24 weeks. In the study, hemoglobin A1c reduction was 0.52%, 0.94% and 1.00% for the 500 mg, 1000 mg and 1500 mg dose twice-daily, respectively. The level of reduction of hemoglobin A1c for Imeglimin was even more pronounced in the Japanese Phase 2b data than what has previously been shown in the U.S. and EU Phase 2b data. For the study’s key secondary endpoint of a decrease in Fasting Plasma Glucose (FPG), the top two Imeglimin doses of 1000 mg and 1500 mg achieved statistical significance (p<0.0001) versus placebo at 24 weeks. Analyses of data for the additional secondary endpoints are ongoing. In this study, Imeglimin was shown to be safe and well tolerated and the adverse event profile was consistent to what was observed in the U.S. and EU Phase 1 and 2 programs. The Company anticipates meeting with the Pharmaceuticals and Medical Devices Agency in Japan during the third quarter, and pending feedback from the meeting, the Company plans to be in the position to advance Imeglimin into a Phase 3 program in Japan during the fourth quarter of 2017. "The strong efficacy of Imeglimin observed in this study suggests that its innovative mechanism of action may play an important role in the treatment of type 2 diabetes and could be particularly well-suited for Japanese patients,” said Pascale Fouqueray, MD, PhD, Executive Vice President, Early Development & Translational Medicine of Poxel. “Imeglimin has been successfully studied in over 1,200 subjects and has shown a differentiated product profile combined with favorable safety, and we believe that it has the potential to address the large and growing needs of the type 2 diabetes market.” “We are very pleased with these results and are actively preparing for the Imeglimin Phase 3 program in Japan. The Phase 3 program is anticipated to include 3 pivotal trials as well as non-pivotal studies in specific patient populations to further differentiate Imeglimin from other approved therapies in Japan,” said Christophe Arbet-Engels, MD, PhD, MBA, Chief Medical Officer and Executive Vice President, Late Development & Medical Affairs of Poxel. “Diabetes is a growing health concern in Asia and with these data results we are encouraged that Imeglimin could become an innovative new treatment option for Japanese patients to manage their type 2 diabetes.” "This is a significant milestone for Poxel. Japan is a key focus and is an integral part of our business strategy, especially with the unique treatment paradigm for innovative therapies. In Japan, we believe Imeglimin may be a prime candidate for first-line treatment as monotherapy and as an add-on to other glucose lowering therapies for the treatment of patients with type 2 diabetes,” said Thomas Kuhn, CEO of Poxel. “Japan represents the second largest single market for type 2 diabetes and is expected to grow to approximately $6 billion in annual sales in 2020*. Asia, in broader terms, is considered the most important geographic location with regards to treating the diabetes pandemic in the future." Poxel anticipates presenting the full data results of the Phase 2b study in Japanese patients at scientific meetings during the second half of 2017. Poxel will host an investor conference call today to discuss the Phase 2b data results at 7 pm Central European Summer Time (1 pm Eastern Daylight Time). To participate in the call, please use the dial-in numbers below. About Imeglimin Imeglimin is the first clinical candidate in a new chemical class of oral agents called the Glimins. Imeglimin has a unique mechanism of action (MOA) that targets mitochondrial bioenergetics. Imeglimin acts on the three main target organs involved in glucose homeostasis: the liver, muscle, and the pancreas. This MOA has the potential for glucose lowering benefits, as well as the potential to prevent endothelial dysfunction, which can provide protective effects on micro- and macro-vascular defects induced by diabetes. The additional protective effect on beta-cell survival and function may lead to a delay in disease progression. This unique mode of action compared to existing treatments for type 2 diabetes makes Imeglimin a prime candidate in all stages of the current anti-diabetic treatment paradigm, including monotherapy or as an add-on to other glucose lowering therapies for the treatment of patients with type 2 diabetes. About Poxel SA Poxel uses its development expertise in metabolism to advance a pipeline of drug candidates focused on the treatment of metabolic disorders, including type 2 diabetes. We have successfully completed a Phase 2 clinical program for our first-in-class lead product, Imeglimin, which targets mitochondrial dysfunction, in the U.S., EU and Japan. Our second program, PXL770, a direct AMPK activator, is in Phase 1 development. We intend to generate further growth through strategic partnerships and pipeline development. (Euronext: POXEL, www.poxel.com)


LYON, France--(BUSINESS WIRE)--POXEL SA (Euronext – POXEL - FR0012432516), a biopharmaceutical company focused on the development of innovative treatments for metabolic disorders, including type 2 diabetes, announced today additional results from the Imeglimin Phase 2b study conducted in Japan. In addition to the highly statistically significant (p<0.0001) top-line results for the primary and key secondary endpoint, the Company is reporting additional data from this study. This press release is being issued in conjunction with a corporate presentation at the Jefferies global healthcare conference today at 4 pm ET. To access the webcast, please use the following link http://wsw.com/webcast/jeff105/poxel.pa. The Phase 2b randomized, double-blind, placebo-controlled study tested three doses of Imeglimin (500 mg, 1000 mg and 1500 mg) administered twice-daily for 24 weeks in 299 Japanese patients for the treatment of type 2 diabetes. In this study, a statistically significant (p<0.0001) decrease in the primary endpoint of HbA1c was observed along with consistent, statistically significant (p<0.0001) decreases in the key secondary endpoints of fasting plasma glucose, glycated albumin and percentage of patients reaching a target HbA1c of less than 7%. A statistically significant dose dependent (500 mg p=0.008,1000 mg p=0.0008, and 1500 mg p<0.0001) improvement of the homeostasis model assessment of beta-cell function (HOMA-B), a marker of beta cell function in fasting condition, was also observed and is consistent with previously published data. In addition, there was a significant decrease in two of the most relevant liver enzymes, alanine aminotransferase (ALT) and gamma-glutamyl transferase (GGT), which are considered biomarkers in liver disease. The ALT and GGT results reflect an improvement of liver function and are consistent with previously published data in animal models. These data support Imeglimin’s unique dual mechanism of action of improving both insulin secretion and sensitivity, which are the two key defects that cause type 2 diabetes. In this study, Imeglimin was safe and well tolerated and the adverse event profile was consistent to what was observed in the U.S. and EU Phase 1 and 2 programs. No serious adverse events related to Imeglimin were reported. There was no difference in the overall incidence of patients presenting with at least one treatment emergent adverse event between treatment and placebo groups. Of particular note, in this study, the safety and efficacy of Imeglimin in patients with mild to moderate chronic kidney disease was similar to patients with normal renal function. Furthermore, no weight gain from Imeglimin was observed. “Imeglimin’s effect on beta cell function through HOMA-B is very meaningful. We believe that Imeglimin could be particularly well-suited for Japanese patients with type 2 diabetes and this effect may be an important contributor to the robust efficacy seen in our Phase 2b study in Japanese patients,” said Pascale Fouqueray, MD, PhD, Executive Vice President, Early Development & Translational Medicine of Poxel. “The additional data demonstrating a decrease in liver enzymes are promising and could represent an added benefit to type 2 diabetes patients who are at a high risk for liver disease.” As previously reported, the primary endpoint of the trial achieved statistical significance (p<0.0001) for the change from baseline in HbA1c versus placebo in all treatment groups at 24 weeks. In the study, placebo adjusted HbA1c reduction was 0.52%, 0.94% and 1.00% for the 500 mg, 1000 mg and 1500 mg dose twice-daily, respectively. The Company anticipates meeting with the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan during the third quarter of this year, and pending feedback from the meeting, the Company anticipates advancing Imeglimin into a Phase 3 program in Japan during the fourth quarter of 2017. “Japan is a key focus and an integral part of our business strategy, especially with the unique treatment paradigm for innovative new therapies. Given Imeglimin’s novel mechanism of action coupled with the results of this Phase 2b trial, we believe Imeglimin is a prime candidate for first-line therapy either as monotherapy or as an add-on to other glucose lowering therapies for the treatment of patients with type 2 diabetes,” said Thomas Kuhn, CEO of Poxel. “We look forward to advancing Imeglimin into a Phase 3 program in Japan.” Imeglimin is the first clinical candidate in a new chemical class of oral agents called the Glimins. Imeglimin has a unique mechanism of action (MOA) that targets mitochondrial bioenergetics. Imeglimin acts on the three main target organs involved in glucose homeostasis: the liver, muscle, and the pancreas. This MOA has the potential for glucose lowering benefits, as well as the potential to prevent endothelial dysfunction, which can provide protective effects on micro- and macro-vascular defects induced by diabetes. The additional protective effect on beta-cell survival and function may lead to a delay in disease progression. This unique mode of action compared to existing treatments for type 2 diabetes makes Imeglimin a prime candidate in all stages of the current anti-diabetic treatment paradigm, including monotherapy or as an add-on to other glucose lowering therapies for the treatment of patients with type 2 diabetes. Poxel uses its development expertise in metabolism to advance a pipeline of drug candidates focused on the treatment of metabolic disorders, including type 2 diabetes. We have successfully completed a Phase 2 clinical program for our first-in-class lead product, Imeglimin, which targets mitochondrial dysfunction, in the U.S., EU and Japan. Our second program, PXL770, a direct AMPK activator, is in Phase 1 development. We intend to generate further growth through strategic partnerships and pipeline development. (Euronext: POXEL, www.poxel.com)

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