MGAM

Peer Reviewed Results from PROPEL Show Treatment with AT-GAA Provided Clinically Meaningful Improvements Over Standard of Care, including ERT Experienced Patients with High Unmet Need AT-GAA Deemed to Provide a Differentiated Mechanism of Action and Potential Alternative Treatment Option for People Living with Late-onset Pompe Disease PHILADELPHIA, Nov. 18, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, announced today that the data from the Phase 3 PROPEL pivotal trial, assessing the efficacy, safety and tolerability of AT-GAA in adults with late-onset Pompe disease compared to the standard of care, alglucosidase alfa, were published online in The Lancet Neurology. The manuscript includes data on the primary and key secondary endpoints, which were previously reported, as well as additional secondary endpoints. Based on the outcomes observed in the key domains of Pompe disease (muscle strength, pulmonary and motor function, patient-reported outcomes and biomarkers), the peer reviewed results determined the PROPEL data showed clinically meaningful improvements over standard of care, even among those who had been receiving approved therapy for at least 2 years, a subgroup that has been shown to plateau or decline after several years on treatment. AT-GAA is a two-component therapy consisting of cipaglucosidase alfa, an enhanced phosphorylated enzyme and miglustat, an enzyme stabilizer, thereby providing a different mechanism of action compared with alglucosidase alfa. “Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives. There is significant unmet medical need that persists within Pompe disease and patients are in need of new, effective and safe therapies. The outcomes of the PROPEL study demonstrate meaningful improvements in motor and respiratory functions in patients who are living with Pompe disease,” stated Prof. Benedikt Schoser, Professor of Neurology at Ludwig-Maximillians-University of Munich LMU Department of Neurology. “Results are particularly encouraging for the population switching from standard of care ERT to AT-GAA, who saw positive improvements in key manifestations of the disease.” John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, stated, “We are pleased that The Lancet Neurology has published our PROPEL pivotal data in adults living with late-onset Pompe disease. Based on the clinical data we have seen from the Phase 3 trial, in addition to the body of data that we have accumulated over nearly a decade of preclinical and clinical studies, we have generated a compelling data set that supports the potential of AT-GAA becoming the next standard of care in the treatment of Pompe disease. We are honored by this publication and would like to thank all of the investigators, patients and families who participated in the study.” The U.S. Food and Drug Administration (FDA) previously granted Breakthrough Therapy designation for AT-GAA and accepted for review the Biologics License Application (BLA) and the New Drug Application (NDA). The FDA has set a Prescription Drug User Fee Act action date of May 29, 2022 for the NDA and July 29, 2022 for the BLA. In the EU, the Marketing Authorization Applications for AT-GAA have been submitted in the fourth quarter of 2021. About AT-GAA AT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa (ATB200), a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly bis-phosphorylated mannose-6 phosphate (bis-M6P) glycans, to enhance uptake into cells, administered in conjunction with miglustat (AT2221), a stabilizer of cipaglucosidase alfa. In preclinical studies, AT-GAA was associated with increased levels of the mature lysosomal form of GAA and reduced glycogen levels in muscle, alleviation of the autophagic defect and improvements in muscle strength. In addition, Amicus is enrolling an open-label, uncontrolled, multicenter study to evaluate the PK, safety, efficacy, and PD of AT-GAA in pediatric patients aged 0 to 18 years with LOPD (ATB200-04). More information, including a list of participating sites, is available at : NCT03911505 About Pompe Disease Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA levels lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide. About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the company’s website at , and follow us on Twitter and LinkedIn. Forward Looking Statement This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to top-line data from a global Phase 3 study to investigate AT-GAA for the treatment of Pompe Disease, the potential implications on these data for the future advancement and development of AT-GAA, and anticipated regulatory submissions. There can be no assurance that the FDA will accept a BLA submission or if accepted will grant approval for AT-GAA. Words such as, but not limited to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” "confidence," "encouraged," “potential,” “plan,” “targets,” “likely,” “may,” “will,” “would,” “should” and “could,” and similar expressions or words identify forward-looking statements. The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors, including that the Company will not be able to successfully complete the development of, obtain regulatory approval for, or successfully manufacture and commercialize AT-GAA. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended December 31, 2020. As a consequence, actual results may differ materially from those set forth in this press release. You are cautioned not to place undue reliance on these forward-looking statements, which speak only of the date hereof. All forward looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise this press release to reflect events or circumstances after the date hereof. CONTACT: Investors: Andrew Faughnan Executive Director, Investor Relations afaughnan@amicusrx.com (609) 662-3809 Media: Diana Moore Head of Global Corporate Affairs & Communications dmoore@amicusrx.com (609) 662-5079 FOLD–G

Amicus Therapeutics is launching a next-generation genetic medicine company called Caritas Therapeutics, Inc. through a merger deal with ARYA Sciences Acquisition Corp IV, a special purpose acquisition company sponsored by Perceptive Advisors. Shares of Amicus are up 10% in premarket trading following the announcement. The new company launches with a pipeline led by two Batten disease programs with clinical proof of concept in CLN6 and CLN3. Caritas also has six active preclinical programs, including ones for Fabry disease, Pompe disease, and CDKL5 deficiency disorder. Additionally, the new company has multiple discovery programs. Caritas will have exclusive, global rights to gene therapy programs for nearly 50 rare genetic diseases, including next-generation lysosomal disease programs and 11 prevalent rare disorders. Among these are Angelman Syndrome, Duchenne Muscular Dystrophy, Rett Syndrome, Myotonic Dystrophy and select other muscular dystrophies. Amicus will retain control of its gene therapy properties for Fabry disease and Pompe disease. The company will also maintain a focus on rare diseases and late-stage development. The deal will result in two independent publicly traded companies. Both companies are expected to have stand-alone investment pro Amicus will become the largest shareholder in Caritas with an ownership stake of about 36%. The current Amicus Chairman and Chief Executive Officer John F. Crowley will helm Caritas as chairman and CEO when the merger is complete. He will also become the chairman emeritus and chief strategic advisor for Amicus. Current Amicus President and Chief Operating Officer Bradley Campbell will be named the CEO of Amicus. Crowley called the formation of Caritas Therapeutics a “massive step forward” to treat rare genetic conditions. He said the new entity will accelerate funding for the company’s next-generation gene therapy pipeline and strengthen both companies’ financial pro “In a single stroke with the formation and funding of Caritas, we will create what will be one of the world’s preeminent next-generation genetic medicines companies. We strongly believe that separating our business into two highly focused, stand-alone companies is the best way to unlock significant value for Amicus shareholders and to advance our medicines and technologies to people living with rare diseases around the world,” Crowley said in a statement. Campbell, the new head of Amicus, said the company will be laser-focused on maintaining the growth of Galafold and executing on the anticipated global launch of AT-GAA. At the same time Amicus announced the business restructuring, the company also announced that the U.S. Food and Drug Administration (FDA) had accepted its Biologics License Application for cipaglucosidase alfa and the New Drug Application (NDA) miglustat for AT-GAA, the company’s investigational two-component therapy for the treatment of Pompe disease. The FDA set a Prescription Drug User Fee Act action date of May 29, 2022, for the NDA and July 29, 2022, for the BLA. Amicus’ AT-GAA Pompe disease treatment is a two-part approach that includes two-component therapy that consists of cipaglucosidase alfa, a recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures designed to enhance uptake into cells. It is administered in conjunction with miglustat, a stabilizer of cipaglucosidase alfa. Data supporting the BLA and NDA include the Phase I/II and Phase III Propel studies and data from an open-label extension study. Crowley touted the acceptance of the filings, calling it an “immensely important step forward for people living with Pompe disease.” He said Amicus would work with the FDA as they review the data. Additionally, the company will be seeking approval for AT-GAA outside of the U.S. Pompe disease is a rare genetic enzyme deficiency that leads to muscle and respiratory issues in patients. The disease can cause premature death in patients. AT-GAA was granted Breakthrough Therapy Designation by the FDA for treatment of late-onset Pompe disease based on clinical efficacy results from the Phase I/II clinical study. Amicus also announced today that a syndicate of investors, including Redmile Group, Avoro Capital Advisors, Perceptive Advisors, Invus, Sphera Healthcare, and Janus Henderson Investors infused the company with a private investment of approximately $200 million. Amicus expects to use the net proceeds to further fund initiatives in the global commercialization of Galafold and the anticipated global launch of AT-GAA and, in connection with the business combination, to invest $50 million in cash in Caritas in exchange for additional equity.

Permits All Eligible Individuals with Late-Onset Pompe Disease Access to AT-GAA Prior to Marketing Authorization in the UK Positive Scientific Opinion Under Early Access to Medicines Scheme (EAMS) Recognizes High Unmet Medical Need in ERT Treated Late-Onset Pompe Disease Patients Positive Scientific Opinion Includes MHRA Review of AT-GAA Phase 3 PROPEL Study Data PHILADELPHIA and MARLOW, United Kingdom, June 08, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on discovering, developing and delivering novel medicines for rare diseases, today announced that the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) to AT-GAA, the Company’s investigational two-component therapy for the treatment of Pompe disease. This positive opinion means that eligible adults living with late-onset Pompe disease (LOPD) who have received alglucosidase alfa for at least 2 years can now switch and have access to AT-GAA prior to marketing authorization in the UK. The EAMS mechanism is intended to provide individuals in the UK who live with a life threatening or seriously debilitating condition early access to innovative and unlicensed medicines for which there is not yet marketing authorization and where there is a clear unmet medical need. John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, stated, “We are pleased that the MHRA has recognized the potential of AT-GAA for individuals living with Late-Onset Pompe disease. This positive scientific opinion reaffirms that there is significant unmet medical need in Pompe disease today, and supports our strategy of advancing AT-GAA as quickly as possible to as many patients as possible. We are privileged to offer eligible patients in the UK access to this novel medicine prior to marketing authorization, reinforcing our commitment to people living with Pompe and other rare diseases.” “The EAMS positive scientific opinion from the MHRA is an important development for people living with Late-Onset Pompe disease. Alternative treatment options that have the potential to have a meaningful impact in managing this devastating neuromuscular disease are desperately needed, and this decision will allow eligible patients access to AT-GAA at the earliest opportunity,” said Dr. Mark Roberts, Consultant Neurologist at the Greater Manchester Neurosciences Unit at Salford Royal NHS Foundation Trust. "I am very pleased that the MHRA has granted this positive opinion to AT-GAA in ERT treated late-onset Pompe disease patients," said Allan Muir, Chair of the Board of Trustees of Pompe Support Network. "With significant unmet needs and a lack of treatment choices for people living with Pompe disease in the UK, we are closer to a new option for patients. Amicus has been a true partner for the Pompe community for more than a decade, and I look forward to potentially having a new therapy available in the UK." The MHRA’s decision is based on the evaluation of the effects of AT-GAA in LOPD patients and its safety profile, including data from both the Phase 1/2 and Phase 3 PROPEL study. Results from the global Phase 3 PROPEL clinical study of AT-GAA were presented at the 17th Annual WORLDSymposium™ 2021, held virtually February 8-12, 2021. Marketing Authorization Applications for AT-GAA are expected to be submitted with the UK and EU, respectively, in the second half of 2021. For the full approval in the UK, Amicus will seek inclusion of the treatment population to all ERT-switch patients regardless of length of time on prior ERT, as well as the treatment naïve population. Future studies will also investigate the treatment of patients with Infantile Onset Pompe Disease. Previously, the MHRA has granted the PIM (Priority Innovative Medicines) designation to AT-GAA. Also, the U.S. FDA granted Breakthrough Therapy Designation to AT-GAA for the treatment of LOPD based on clinical efficacy results from the Phase 1/2 clinical study. A rolling Biologics License Application (BLA) for AT-GAA was initiated with the U.S. FDA in the fourth quarter of 2020 and is on track with submission of the final modules in the second quarter of 2021. About AT-GAAAT-GAA is an investigational two-component therapy that consists of cipaglucosidase alfa (ATB200), a recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly bis-phosphorylated mannose-6 phosphate (bis-M6P) glycans, to enhance uptake into cells, administered in conjunction with miglustat (AT2221), a stabilizer of cipaglucosidase alfa. In preclinical studies, AT-GAA was associated with increased levels of the mature lysosomal form of GAA and reduced glycogen levels in muscle, alleviation of the autophagic defect and improvements in muscle strength. About Pompe DiseasePompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA levels lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. The disease can be debilitating and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide. About Amicus TherapeuticsAmicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With a patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the company’s website at , and follow us on Twitter and LinkedIn. Forward Looking StatementThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to top-line data from a global Phase 3 study to investigate AT-GAA for the treatment of Pompe Disease and the potential implications on these data for the future advancement and development of AT-GAA. Words such as, but not limited to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” "confidence," "encouraged," “potential,” “plan,” “targets,” “likely,” “may,” “will,” “would,” “should” and “could,” and similar expressions or words identify forward-looking statements. The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors, including that the Company will not be able to successfully complete the development of, obtain regulatory approval for, or successfully manufacture and commercialize AT-GAA. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended December 31, 2020 and Quarterly Report on 10-Q for the Quarter ended March 31, 2021. As a consequence, actual results may differ materially from those set forth in this press release. You are cautioned not to place undue reliance on these forward looking statements, which speak only of the date hereof. All forward looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise this press release to reflect events or circumstances after the date hereof. CONTACTS: Investors:Andrew FaughnanSr. Director, Investor Relationsafaughnan@amicusrx.com(609) 662-3809 Media:Diana MooreHead of Global Corporate Communicationsdmoore@amicusrx.com(609) 662-5079 FOLD–G NP--UKI-00020621 June 2021