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100+ Fabry Disease Patients Now on Reimbursed Galafold (migalastat) in the EU On Target to Reach 300 Patients on Galafold by Year-End 2017 CRANBURY, N.J., May 09, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of therapies for rare and orphan diseases, today announced financial results for the first quarter ended March 31, 2017. The Company also provided program updates and reiterated full-year 2017 financial guidance. “During the first quarter we continued to advance the international commercial launch of our oral precision medicine Galafold for Fabry disease, as we brought Galafold to more Fabry patients in Germany and other European markets with both new ERT switch patients as well as a notable proportion of ERT-naïve patients,” stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “We are extremely pleased with the progress of our Galafold launch in terms of patient and physician adoption, as well as pricing and reimbursement. With launch now just initiating this month in the UK, Italy, France, and additional markets, we are confident in achieving our target of 300 patients on Galafold by the end of this year. In addition to the launch, we have also made significant progress on our other four key strategic priorities for 2017: 1) targeting a Japanese new drug application (J-NDA) submission for migalastat in the second quarter, 2) establishing definitive clinical proof of concept for our novel Pompe treatment paradigm ATB200/AT2221, 3) completing our Phase 3 clinical study in patients with epidermolysis bullosa, and 4) maintaining our financial strength. We are particularly looking forward to sharing additional, important Pompe clinical data this quarter. We continue to believe that our Pompe program will be a crucial driver toward our vision of becoming one of the world’s leading global rare disease biotechnology companies.” Cash, cash equivalents, and marketable securities totaled $279.8 million at March 31, 2017. The Company continues to expect full-year 2017 net operating cash spend of between $175 million to $200 million and full-year 2017 total net cash spend (including third-party milestone payments and capital expenditures) of between $200 million and $225 million. The current cash position is anticipated to fund ongoing operations into the second half of 2018. Migalastat is an oral precision medicine intended to treat Fabry disease in patients who have amenable genetic mutations. As previously announced, the European Commission (EC) has granted full approval for migalastat under the trade name Galafold. The EC approval may serve as the basis for regulatory approvals in more than two-thirds of the global Fabry market that is outside the U.S. The Company has also defined a U.S. pathway to support full approval. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. Positive preliminary data were reported from a global Phase 1/2 clinical study (ATB200-02) to evaluate safety, tolerability, PK, and pharmacodynamics (PD) of ATB200/AT2221. The study is fully enrolled with 3 cohorts of patients, including ambulatory ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients (Cohort 2), and ERT-naïve patients (Cohort 3). SD-101 is a novel, late-stage, proprietary topical treatment and potential first-to-market therapy for EB. SD-101 is currently being investigated in a registration-directed Phase 3 study (ESSENCE, also known as SD-005) to support global regulatory submissions. SD-101 was granted FDA Breakthrough Therapy designation in 2013 based on results from a Phase 2a study for the treatment of lesions in patients suffering with EB. SD-101 is the first-ever treatment in clinical studies to show improvements in wound closure across all major EB types. Conference Call and Webcast Amicus Therapeutics will host a conference call and audio webcast today, May 9, 2017 at 8:30 a.m. ET to discuss first quarter 2017 financial results and corporate updates. Interested participants and investors may access the conference call by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international); participant code 16795077. An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/, and will be archived for 30 days. Web participants are encouraged to go to the web site 15 minutes prior to the start of the call to register, download and install any necessary software. A telephonic replay of the call will be available for seven days beginning at 11:30 a.m. ET today. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); participant code 16795077. Important Safety Information Treatment with GALAFOLD should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. GALAFOLD is not recommended for use in patients with a nonamenable mutation. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu. About Amicus Therapeutics Amicus Therapeutics (Nasdaq:FOLD) is a biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, financing plans, and the projected cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe or our other product candidates if and when approved; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; and the potential that we will need additional funding to complete all of our studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results for any of our product candidates. With respect to statements regarding projections of the Company's cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our previous filings with the SEC and in our Annual Report on Form 10-K for the year ended December 31, 2016. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.


News Article | May 10, 2017
Site: globenewswire.com

CRANBURY, N.J., May 10, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today announced that Chip Baird, Chief Financial Officer, will present at two upcoming investor conferences: A live webcast of both presentations can be accessed through the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/events.cfm, and will be archived for 90 days. About Amicus Therapeutics Amicus Therapeutics (Nasdaq:FOLD) is a global biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.


100+ Fabry Disease Patients Now on Reimbursed Galafold (migalastat) in the EU On Target to Reach 300 Patients on Galafold by Year-End 2017 CRANBURY, N.J., May 09, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of therapies for rare and orphan diseases, today announced financial results for the first quarter ended March 31, 2017. The Company also provided program updates and reiterated full-year 2017 financial guidance. “During the first quarter we continued to advance the international commercial launch of our oral precision medicine Galafold for Fabry disease, as we brought Galafold to more Fabry patients in Germany and other European markets with both new ERT switch patients as well as a notable proportion of ERT-naïve patients,” stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “We are extremely pleased with the progress of our Galafold launch in terms of patient and physician adoption, as well as pricing and reimbursement. With launch now just initiating this month in the UK, Italy, France, and additional markets, we are confident in achieving our target of 300 patients on Galafold by the end of this year. In addition to the launch, we have also made significant progress on our other four key strategic priorities for 2017: 1) targeting a Japanese new drug application (J-NDA) submission for migalastat in the second quarter, 2) establishing definitive clinical proof of concept for our novel Pompe treatment paradigm ATB200/AT2221, 3) completing our Phase 3 clinical study in patients with epidermolysis bullosa, and 4) maintaining our financial strength. We are particularly looking forward to sharing additional, important Pompe clinical data this quarter. We continue to believe that our Pompe program will be a crucial driver toward our vision of becoming one of the world’s leading global rare disease biotechnology companies.” Cash, cash equivalents, and marketable securities totaled $279.8 million at March 31, 2017. The Company continues to expect full-year 2017 net operating cash spend of between $175 million to $200 million and full-year 2017 total net cash spend (including third-party milestone payments and capital expenditures) of between $200 million and $225 million. The current cash position is anticipated to fund ongoing operations into the second half of 2018. Migalastat is an oral precision medicine intended to treat Fabry disease in patients who have amenable genetic mutations. As previously announced, the European Commission (EC) has granted full approval for migalastat under the trade name Galafold. The EC approval may serve as the basis for regulatory approvals in more than two-thirds of the global Fabry market that is outside the U.S. The Company has also defined a U.S. pathway to support full approval. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. Positive preliminary data were reported from a global Phase 1/2 clinical study (ATB200-02) to evaluate safety, tolerability, PK, and pharmacodynamics (PD) of ATB200/AT2221. The study is fully enrolled with 3 cohorts of patients, including ambulatory ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients (Cohort 2), and ERT-naïve patients (Cohort 3). SD-101 is a novel, late-stage, proprietary topical treatment and potential first-to-market therapy for EB. SD-101 is currently being investigated in a registration-directed Phase 3 study (ESSENCE, also known as SD-005) to support global regulatory submissions. SD-101 was granted FDA Breakthrough Therapy designation in 2013 based on results from a Phase 2a study for the treatment of lesions in patients suffering with EB. SD-101 is the first-ever treatment in clinical studies to show improvements in wound closure across all major EB types. Conference Call and Webcast Amicus Therapeutics will host a conference call and audio webcast today, May 9, 2017 at 8:30 a.m. ET to discuss first quarter 2017 financial results and corporate updates. Interested participants and investors may access the conference call by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international); participant code 16795077. An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/, and will be archived for 30 days. Web participants are encouraged to go to the web site 15 minutes prior to the start of the call to register, download and install any necessary software. A telephonic replay of the call will be available for seven days beginning at 11:30 a.m. ET today. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); participant code 16795077. Important Safety Information Treatment with GALAFOLD should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. GALAFOLD is not recommended for use in patients with a nonamenable mutation. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu. About Amicus Therapeutics Amicus Therapeutics (Nasdaq:FOLD) is a biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, financing plans, and the projected cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe or our other product candidates if and when approved; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; and the potential that we will need additional funding to complete all of our studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results for any of our product candidates. With respect to statements regarding projections of the Company's cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our previous filings with the SEC and in our Annual Report on Form 10-K for the year ended December 31, 2016. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.


News Article | May 10, 2017
Site: globenewswire.com

CRANBURY, N.J., May 10, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today announced that Chip Baird, Chief Financial Officer, will present at two upcoming investor conferences: A live webcast of both presentations can be accessed through the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/events.cfm, and will be archived for 90 days. About Amicus Therapeutics Amicus Therapeutics (Nasdaq:FOLD) is a global biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.


News Article | May 10, 2017
Site: globenewswire.com

CRANBURY, N.J., May 10, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today announced that Chip Baird, Chief Financial Officer, will present at two upcoming investor conferences: A live webcast of both presentations can be accessed through the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/events.cfm, and will be archived for 90 days. About Amicus Therapeutics Amicus Therapeutics (Nasdaq:FOLD) is a global biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.


News Article | May 10, 2017
Site: globenewswire.com

CRANBURY, N.J., May 10, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, today announced that Chip Baird, Chief Financial Officer, will present at two upcoming investor conferences: A live webcast of both presentations can be accessed through the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/events.cfm, and will be archived for 90 days. About Amicus Therapeutics Amicus Therapeutics (Nasdaq:FOLD) is a global biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.


News Article | May 12, 2017
Site: www.prnewswire.com

Financial Results for the Three Months Ended March 31, 2017 Total operating expenses for the first quarter of 2017 were $3.9 million, compared to $2.1 million of operating expenses in 2016. The increase in operating expenses in the first quarter of 2017 was primarily due to increased research and development costs to execute PhaseRx's product development plan of its lead drug candidate PRX-OTC, increased costs associated with being a publicly-traded company and an increase in non-cash stock-based compensation expenses. The net loss for the quarter ended March 31, 2017 was $4.1 million, or $0.35 per share, compared to a net loss of $2.2 million, or $4.19 per share, for the quarter ended March 31, 2016. As of March 31, 2017, the company had cash, cash equivalents, and marketable securities of $11.9 million. PhaseRx is a biopharmaceutical company dedicated to developing mRNA products for the treatment of children with inherited enzyme deficiencies in the liver using intracellular enzyme replacement therapy (i-ERT). PhaseRx's initial product development focus is on urea cycle disorders, a group of rare genetic diseases that generally present before the age of twelve and are characterized by the body's inability to remove ammonia from the blood with potentially devastating consequences for patients. The company's i-ERT approach is enabled by its proprietary Hybrid mRNA Technology™ platform. PhaseRx is headquartered in Seattle. For more information, please visit www.phaserx.com. This press release contains "forward-looking statements." 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 not guarantees of future performance, 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 fact that the company has incurred significant losses since its inception and anticipates that it will continue to incur significant losses for the foreseeable future, (ii) the company being dependent on technologies it has licensed and that it may need to license in the future, (iii) the fact that the company will need to raise substantial additional funding to develop its planned products, (iv) the fact that the company's Hybrid mRNA Technology has not previously been tested beyond company preclinical studies, and that mRNA-based drug development is unproven, (v) the fact that all of the company's programs are in preclinical studies or early stage research and it is uncertain that any company product candidates will receive regulatory approval or be commercialized, (vi) the fact that development of the company's product candidates will be expensive, time-consuming and subject to regulatory approval, (vii) the company expecting to continue to incur significant research and development expenses, (viii) the company becoming dependent on collaborative arrangements for the development and commercialization of its products, (ix) the company's ability to adequately protect its proprietary technology from legal challenges, infringement or alternative technologies and (x) the biotechnology and pharmaceutical industries being intensely competitive. 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 most recent annual report on Form 10-K and its quarterly reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC's web site at http://www.sec.gov. The company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise. To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/phaserx-reports-first-quarter-2017-financial-results-and-provides-corporate-update-300456584.html


100+ Fabry Disease Patients Now on Reimbursed Galafold (migalastat) in the EU On Target to Reach 300 Patients on Galafold by Year-End 2017 CRANBURY, N.J., May 09, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of therapies for rare and orphan diseases, today announced financial results for the first quarter ended March 31, 2017. The Company also provided program updates and reiterated full-year 2017 financial guidance. “During the first quarter we continued to advance the international commercial launch of our oral precision medicine Galafold for Fabry disease, as we brought Galafold to more Fabry patients in Germany and other European markets with both new ERT switch patients as well as a notable proportion of ERT-naïve patients,” stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “We are extremely pleased with the progress of our Galafold launch in terms of patient and physician adoption, as well as pricing and reimbursement. With launch now just initiating this month in the UK, Italy, France, and additional markets, we are confident in achieving our target of 300 patients on Galafold by the end of this year. In addition to the launch, we have also made significant progress on our other four key strategic priorities for 2017: 1) targeting a Japanese new drug application (J-NDA) submission for migalastat in the second quarter, 2) establishing definitive clinical proof of concept for our novel Pompe treatment paradigm ATB200/AT2221, 3) completing our Phase 3 clinical study in patients with epidermolysis bullosa, and 4) maintaining our financial strength. We are particularly looking forward to sharing additional, important Pompe clinical data this quarter. We continue to believe that our Pompe program will be a crucial driver toward our vision of becoming one of the world’s leading global rare disease biotechnology companies.” Cash, cash equivalents, and marketable securities totaled $279.8 million at March 31, 2017. The Company continues to expect full-year 2017 net operating cash spend of between $175 million to $200 million and full-year 2017 total net cash spend (including third-party milestone payments and capital expenditures) of between $200 million and $225 million. The current cash position is anticipated to fund ongoing operations into the second half of 2018. Migalastat is an oral precision medicine intended to treat Fabry disease in patients who have amenable genetic mutations. As previously announced, the European Commission (EC) has granted full approval for migalastat under the trade name Galafold. The EC approval may serve as the basis for regulatory approvals in more than two-thirds of the global Fabry market that is outside the U.S. The Company has also defined a U.S. pathway to support full approval. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. Positive preliminary data were reported from a global Phase 1/2 clinical study (ATB200-02) to evaluate safety, tolerability, PK, and pharmacodynamics (PD) of ATB200/AT2221. The study is fully enrolled with 3 cohorts of patients, including ambulatory ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients (Cohort 2), and ERT-naïve patients (Cohort 3). SD-101 is a novel, late-stage, proprietary topical treatment and potential first-to-market therapy for EB. SD-101 is currently being investigated in a registration-directed Phase 3 study (ESSENCE, also known as SD-005) to support global regulatory submissions. SD-101 was granted FDA Breakthrough Therapy designation in 2013 based on results from a Phase 2a study for the treatment of lesions in patients suffering with EB. SD-101 is the first-ever treatment in clinical studies to show improvements in wound closure across all major EB types. Conference Call and Webcast Amicus Therapeutics will host a conference call and audio webcast today, May 9, 2017 at 8:30 a.m. ET to discuss first quarter 2017 financial results and corporate updates. Interested participants and investors may access the conference call by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international); participant code 16795077. An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/, and will be archived for 30 days. Web participants are encouraged to go to the web site 15 minutes prior to the start of the call to register, download and install any necessary software. A telephonic replay of the call will be available for seven days beginning at 11:30 a.m. ET today. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); participant code 16795077. Important Safety Information Treatment with GALAFOLD should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. GALAFOLD is not recommended for use in patients with a nonamenable mutation. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu. About Amicus Therapeutics Amicus Therapeutics (Nasdaq:FOLD) is a biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, financing plans, and the projected cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe or our other product candidates if and when approved; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; and the potential that we will need additional funding to complete all of our studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results for any of our product candidates. With respect to statements regarding projections of the Company's cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our previous filings with the SEC and in our Annual Report on Form 10-K for the year ended December 31, 2016. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.


100+ Fabry Disease Patients Now on Reimbursed Galafold (migalastat) in the EU On Target to Reach 300 Patients on Galafold by Year-End 2017 CRANBURY, N.J., May 09, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of therapies for rare and orphan diseases, today announced financial results for the first quarter ended March 31, 2017. The Company also provided program updates and reiterated full-year 2017 financial guidance. “During the first quarter we continued to advance the international commercial launch of our oral precision medicine Galafold for Fabry disease, as we brought Galafold to more Fabry patients in Germany and other European markets with both new ERT switch patients as well as a notable proportion of ERT-naïve patients,” stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “We are extremely pleased with the progress of our Galafold launch in terms of patient and physician adoption, as well as pricing and reimbursement. With launch now just initiating this month in the UK, Italy, France, and additional markets, we are confident in achieving our target of 300 patients on Galafold by the end of this year. In addition to the launch, we have also made significant progress on our other four key strategic priorities for 2017: 1) targeting a Japanese new drug application (J-NDA) submission for migalastat in the second quarter, 2) establishing definitive clinical proof of concept for our novel Pompe treatment paradigm ATB200/AT2221, 3) completing our Phase 3 clinical study in patients with epidermolysis bullosa, and 4) maintaining our financial strength. We are particularly looking forward to sharing additional, important Pompe clinical data this quarter. We continue to believe that our Pompe program will be a crucial driver toward our vision of becoming one of the world’s leading global rare disease biotechnology companies.” Cash, cash equivalents, and marketable securities totaled $279.8 million at March 31, 2017. The Company continues to expect full-year 2017 net operating cash spend of between $175 million to $200 million and full-year 2017 total net cash spend (including third-party milestone payments and capital expenditures) of between $200 million and $225 million. The current cash position is anticipated to fund ongoing operations into the second half of 2018. Migalastat is an oral precision medicine intended to treat Fabry disease in patients who have amenable genetic mutations. As previously announced, the European Commission (EC) has granted full approval for migalastat under the trade name Galafold. The EC approval may serve as the basis for regulatory approvals in more than two-thirds of the global Fabry market that is outside the U.S. The Company has also defined a U.S. pathway to support full approval. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. Positive preliminary data were reported from a global Phase 1/2 clinical study (ATB200-02) to evaluate safety, tolerability, PK, and pharmacodynamics (PD) of ATB200/AT2221. The study is fully enrolled with 3 cohorts of patients, including ambulatory ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients (Cohort 2), and ERT-naïve patients (Cohort 3). SD-101 is a novel, late-stage, proprietary topical treatment and potential first-to-market therapy for EB. SD-101 is currently being investigated in a registration-directed Phase 3 study (ESSENCE, also known as SD-005) to support global regulatory submissions. SD-101 was granted FDA Breakthrough Therapy designation in 2013 based on results from a Phase 2a study for the treatment of lesions in patients suffering with EB. SD-101 is the first-ever treatment in clinical studies to show improvements in wound closure across all major EB types. Conference Call and Webcast Amicus Therapeutics will host a conference call and audio webcast today, May 9, 2017 at 8:30 a.m. ET to discuss first quarter 2017 financial results and corporate updates. Interested participants and investors may access the conference call by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international); participant code 16795077. An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/, and will be archived for 30 days. Web participants are encouraged to go to the web site 15 minutes prior to the start of the call to register, download and install any necessary software. A telephonic replay of the call will be available for seven days beginning at 11:30 a.m. ET today. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); participant code 16795077. Important Safety Information Treatment with GALAFOLD should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. GALAFOLD is not recommended for use in patients with a nonamenable mutation. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu. About Amicus Therapeutics Amicus Therapeutics (Nasdaq:FOLD) is a biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, financing plans, and the projected cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe or our other product candidates if and when approved; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; and the potential that we will need additional funding to complete all of our studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results for any of our product candidates. With respect to statements regarding projections of the Company's cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our previous filings with the SEC and in our Annual Report on Form 10-K for the year ended December 31, 2016. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.


News Article | May 9, 2017
Site: www.prweb.com

ERT, a global data and technology company that minimizes uncertainty and risk in clinical trials, today announced the acquisition of ImageIQ, originally established as Cleveland Clinic’s Biomedical Imaging and Analysis Center. The acquisition enables ERT to offer advanced, end-to-end clinical trial imaging analysis using best-in-class technology that delivers compliant data for use in clinical development. “After conducting an exhaustive industry search, we determined the cloud-based imaging technology invented at Cleveland Clinic is more advanced than traditional imaging solutions available today,” said James Corrigan, President and CEO of ERT. “At the core of this technology is the ability to capture high quality, compliant data without the dependency on human intervention and bias. Providing this level of accuracy will help our customers eliminate more risk and uncertainty from the development process.” Quantitative, objective software analysis enables ERT Imaging to deliver more accurate and verifiable imaging results than subjective readings commonly relied upon with standard scoring systems. Coupled with comprehensive clinical trial services, ERT will provide imaging solutions across key therapeutic areas to generate compliant, high-quality imaging results while reducing site and sponsor burden. “It’s extremely rewarding to extend the Cleveland Clinic’s innovative technology and expertise in custom imaging analysis to researchers around the world,” said Jack Miner, Managing Director at Cleveland Clinic Ventures. “We are pleased that ERT can now offer this next generation imaging solution to their global biopharmaceutical research and medical device clients to help them achieve high quality data and accelerate their clinical development programs.” For more information on ERT Imaging, visit ERT.com/imaging. About ERT ERT is a global data and technology company that minimizes uncertainty and risk in clinical trials so that our customers can move ahead with confidence. With more than 45 years of clinical and therapeutic experience, ERT balances knowledge of what works with a vision for what’s next, so it can adapt without compromising standards. Powered by the company’s EXPERT® technology platform, ERT’s solutions enhance trial oversight, enable site optimization, increase patient engagement, and measure the efficacy of new clinical treatments while ensuring patient safety. Over the past four years, more than half of all FDA drug approvals came from ERT-supported studies. Pharma companies, Biotechs, and CROs have relied on ERT solutions in 9,500+ studies spanning three million patients to date. By identifying trial risks before they become problems, ERT enables customers to bring clinical treatments to patients quickly – and with confidence. For more information, go to ert.com or follow us on LinkedIn and Twitter.

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