Patient Access and Reimbursement

Amylyx Pharmaceuticals Announces Plan to Submit New Drug Application (NDA) for AMX0035 for the Treatment of ALS in the Coming Months September 15, 2021

Amylyx Pharmaceuticals, Inc. today announced its intention to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for AMX0035 (sodium phenylbutyrate (PB) and taurursodiol (TURSO)) for the treatment of amyotrophic lateral sclerosis (ALS). The decision by the company to submit the application in the coming months follows recent discussions with the FDA, including a pre-NDA meeting held on July 15, 2021. “We are thrilled to move toward the U.S. submission of an NDA for AMX0035 and look forward to continuing to work with the FDA,” said Joshua Cohen, Co-CEO, Chairman and Co-Founder of Amylyx. “For those living with ALS, time is the most important resource, and we remain focused on advancing AMX0035 through the clinical development process as efficiently as possible,” added Justin Klee, Co-CEO, Director and Co-Founder of Amylyx. “We’re endlessly grateful for all of the support and efforts of ALS Finding a Cure, the ALS Association, I AM ALS, the Healey &

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Amylyx Pharmaceuticals Donates CENTAUR Clinical Trial Data and Participant Samples to Help Advance Science in ALS for Future Treatments and Discoveries September 13, 2021

– Amylyx Adds Data from CENTAUR to the Anonymized PRO-ACT Database, the Largest ALS Clinical Trial Dataset Ever Created – Company Also Donates Patient Samples from CENTAUR to the Northeast ALS Consortium (NEALS) Biorepository Housed at Mass General Hospital and Barrow Neurological Institute Amylyx Pharmaceuticals, Inc. today announced the donation of data from the CENTAUR clinical trial that evaluated the safety and efficacy of AMX0035 in participants with amyotrophic lateral sclerosis (ALS) to the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database led by Alex Sherman at the Healey and AMG Center and Neurological Clinical Research Institute (NCRI) at Mass General Hospital. Amylyx also donated participants’ samples to the Northeast ALS Consortium (NEALS) Biorepository housed at Mass General Hospital and Barrow Neurological Institute. “It is going to take a consolidated community effort to beat ALS. Sharing data and biological samples with integral databases and

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Amylyx Pharmaceuticals Announces Health Canada Accepts for Review the New Drug Submission for AMX0035 for the Treatment of ALS August 31, 2021

Amylyx Pharmaceuticals, Inc. today announced that Health Canada has accepted for review the New Drug Submission (NDS) for AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). “We are pleased that our submission has been accepted for review by Health Canada and we are committed to serving those affected by ALS in this country,” said Chris Aiello, General Manager and Head of Canada at Amylyx. “Today’s news marks another milestone in our efforts to bring a potential new treatment option to people with ALS. We look forward to working closely with Health Canada and the broader ALS community in Canada as we press ahead.” CENTAUR was a Phase 2, multicenter study in 137 participants with ALS encompassing a 6-month randomized placebo-controlled phase and an open-label long-term follow-up phase. As previously reported, the study met its primary efficacy endpoint of slowing ALS as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). In the study, AMX0035 showed numerical

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Amylyx Pharmaceuticals Closes $135M Series C Financing Led by Viking Global Investors to Support Late Stage Development of Lead Product Candidate AMX0035 for the Treatment of ALS July 20, 2021

Funding to support advancement of clinical development and potential launch planning of AMX0035 in ALS and further research and development in additional neurodegenerative diseases Amylyx Pharmaceuticals, Inc. today announced the closing of an oversubscribed $135 million Series C financing, led by Viking Global Investors, to support the clinical development and potential launch plans for lead product candidate AMX0035, for the potential treatment of ALS, and further research and development in other neurodegenerative diseases. The financing was led by Viking Global Investors with participation from Bain Capital Life Sciences, Perceptive Advisors, Rock Springs Capital, Woodline Partners, Marshall Wace, Tybourne Capital Management, Verition Fund Management, aMoon Fund and Falcon Edge. Existing investors, including Morningside Ventures, 683 Capital Management, Belinda Termeer and Polaris Founders Capital, also participated in the financing. “We have a clear mission at Amylyx: to develop

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Amylyx Pharmaceuticals Strengthens Board of Directors with Appointment of Daphne Quimi, Chief Financial Officer of Amicus Therapeutics June 29, 2021

Amylyx Pharmaceuticals, Inc. today announced the addition of Daphne Quimi to its Board of Directors. Ms. Quimi brings more than 25 years of executive experience in the pharmaceutical and biotechnology industries with expertise in global finance operations, company building, and rare disease drug commercialization. She currently serves as Chief Financial Officer (CFO) of Amicus Therapeutics. “We seek the strongest, most experienced, and patient-centric industry professionals to join our Board of Directors to help Amylyx serve people living with ALS and other neurodegenerative diseases around the world. Daphne is the epitome of these qualities and more and will be a valuable advisor as we seek to advance our pipeline,” said Joshua Cohen, Co-CEO, Chairman and Co-Founder of Amylyx. “Daphne’s longtime financial leadership and acumen, combined with her dedication at Amicus to helping people living with rare diseases around the world, make her a wonderful addition to our Board. On behalf of

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Amylyx Pharmaceuticals Files New Drug Submission for AMX0035 for the Treatment of ALS in Canada June 14, 2021

– Submission Supported by Data from the CENTAUR Trial – CENTAUR Evaluated the Safety and Efficacy of AMX0035 in People with ALS and Demonstrated Statistically Significant Benefit in Function and Survival with Similar Rates of Adverse Events between AMX0035 and Placebo Arms Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease and other neurodegenerative diseases, today announced it has submitted a New Drug Submission (NDS) to Health Canada for AMX0035 (sodium phenylbutyrate (PB)-taurursodiol (TURSO)) for the treatment of ALS. “The submission of the NDS in Canada represents a significant milestone in our efforts to develop a new treatment option for people living with ALS who have no time to wait,” said Joshua Cohen, Co-CEO, Chairman and Co-Founder of Amylyx. “We’d like to thank the people who participated in CENTAUR, trial investigators, the ALS community and our partners and team, who

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Amylyx Pharmaceuticals Presents Trial Design of Global Phase 3 PHOENIX Study of AMX0035 in ALS at the European Network to Cure ALS (ENCALS) Meeting 2021 May 12, 2021

– Building on Statistically Significant Findings from Phase 2 CENTAUR Trial, PHOENIX to Include 600 People with ALS and Broader Inclusion Criteria – Trial in Europe and U.S. Expected to Begin in Q3 2021 to Support Regulatory Submissions Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease and other neurodegenerative diseases, today announced it will present the trial design of its Phase 3 study (A35-004 PHOENIX) of AMX0035 (sodium phenylbutyrate (PB)-taurursodiol (TURSO)) for the treatment of people with ALS at the European Network to Cure ALS (ENCALS) Meeting 2021, to be held virtually May 12-14, 2021. The study will assess the safety and efficacy of AMX0035 in an international population of people with ALS that will build upon findings from the CENTAUR trial. “We are pleased to present the Phase 3 trial design at this year’s ENCALS meeting,” said Leonard H. van den Berg, M.D., Ph.D.,

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Amylyx Pharmaceuticals Provides Global Regulatory Update on AMX0035 for ALS April 14, 2021

– Marketing Authorization Application (MAA) Submission in Europe Planned for Q4 2021 – Phase 3 Trial in Europe and U.S. Expected to Begin in Q3 2021 to Support Regulatory Submissions Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease and other neurodegenerative diseases, today provided an update on its plans to advance AMX0035 through the clinical development process for the treatment of ALS. The company intends to submit a Marketing Authorization Application (MAA) for AMX0035 to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) by the end of 2021. As previously reported, the company plans to submit a marketing application with Health Canada Q2 2021. The FDA has expressed an interest in seeing data from an additional placebo-controlled clinical trial prior to receiving a New Drug Application (NDA), the vehicle through which a pharmaceutical

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Amylyx Pharmaceuticals to Present CENTAUR Long-Term Survival Data of AMX0035 for ALS in Plenary Session at 2021 Virtual American Academy of Neurology (AAN) Annual Meeting April 13, 2021

– Abstract Awarded Merit of Distinction and Selected for Oral Presentation in Contemporary Clinical Issues Plenary Session – Abstract Will Be Featured at AAN’s Top Science Press Conference Ahead of Annual Meeting on April 13 Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease and other neurodegenerative diseases, today announced that long-term survival data from the CENTAUR trial of AMX0035 in patients with ALS were selected for an oral presentation in the Contemporary Clinical Issues Plenary Session on April 19 at the 2021 American Academy of Neurology (AAN) Annual Meeting. The meeting is being held virtually on April 17-22. The abstract, “Long-Term Survival of Participants in the CENTAUR Trial of AMX0035 for ALS,” was one of 26 abstracts awarded the merit of distinction by the American Academy of Neurology and the Science Committee. The distinction is awarded to the abstract deemed

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Paul Fonteyne Joins Amylyx Pharmaceuticals Board of Directors March 29, 2021

Former Boehringer Ingelheim U.S. CEO Adds More than Thirty Years of Executive Leadership Experience and Commercial Expertise to the Amylyx Board of Directors Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced the appointment of Paul Fonteyne to its Board of Directors. Mr. Fonteyne’s deep commercialization and leadership experience will support the company’s global plans to bring new treatments to people with neurodegenerative diseases. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210329005384/en/ Paul Fonteyne, Amylyx Board of Directors member (Photo: Business Wire) “Paul is a true leader in the health care industry with a proven record of success in building effective teams, launching global products and running commercial organizations focused on delivering for

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Amylyx Pharmaceuticals to Present New Long-Term Data from the Pivotal CENTAUR Trial in ALS at the Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference March 18, 2021

– Significantly Lower Long-Term Risk of Death or Permanent Assisted Ventilation and Risk of First Hospitalization Among Participants Originally Randomized to AMX0035 Versus Placebo – Data were Consistent with CENTAUR and CENTAUR OLE Primary Outcome Measures of Statistically Significant Slowing of Functional Decline and Long-Term Survival Results Associated with the Administration of AMX0035 CAMBRIDGE, Mass. […]

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Amylyx Pharmaceuticals Plans Regulatory Filing of AMX0035 for the Treatment of ALS in Canada March 9, 2021

– Marketing Application in Canada (New Drug Submission) Planned for First Half of 2021 – Amylyx Also Plans to Pursue Early Access Options in Canada Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease and other neurodegenerative diseases, today announced plans to submit a New Drug Submission (NDS) for AMX0035 for the treatment of ALS with Health Canada in the first half of 2021. The decision to submit is based on the positive clinical data from the CENTAUR trial and feedback from Health Canada. In addition, Amylyx will explore options for early access to AMX0035 in Canada, including a potential special access program (SAP) in collaboration with the Canadian ALS Research Network, and aims to finalize plans by the end of the second quarter of 2021. “Results from CENTAUR demonstrated that AMX0035 has both functional and long-term survival benefits which were seen on top of baseline use of

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Amylyx Supports Rare Disease Day® and #ShowYourStripes Campaign to Raise Awareness for Rare Disease Communities February 22, 2021

#ShowYourStripes In Community Solidarity by Using New Facebook Filter or Downloadable Video Conferencing Background Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease and other neurodegenerative diseases, has joined forces with the National Organization for Rare Disorders (NORD®) to help raise awareness for this year’s Rare Disease Day® on February 28 th . Rare Disease Day is an annual awareness day dedicated to elevating public understanding of rare diseases and emphasizing the special challenges the community faces. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210222005597/en/ #ShowYourStripes Video Conferencing Background Available for Download (Graphic: Business Wire) Amylyx is honored to be a Rare Disease Day “Campaign Supporter” and participate in NORD’s #ShowYourStripes campaign, which aims to empower advocates,

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James Frates Joins Amylyx Pharmaceuticals as Chief Financial Officer January 14, 2021

– Former Alkermes Chief Financial Officer Expands Amylyx Executive Leadership Team as the Company Grows and Scales Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease and other neurodegenerative diseases, today announced the appointment of James Frates as Chief Financial Officer (CFO), effective Jan. 25, 2021. Mr. Frates joins Amylyx following his tenure as CFO at Alkermes, since 1998. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210114005141/en/ James Frates, Amylyx Chief Financial Officer (Photo: Business Wire) “We are so excited for Jim to join us at this critical juncture for Amylyx,” said Joshua Cohen, Co-CEO, Chairman and Co-Founder of Amylyx. “Jim’s depth and breadth of experience as a biopharmaceutical executive coupled with his financial acumen and personal mission for bringing promising treatments to patients

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Amylyx Pharmaceuticals Appoints Erin Whitney Head of Global Clinical Operations, Shauna Horvath Head of Global Marketing and Keith White Head of Global Market Access December 21, 2020

Amylyx Continues to Expand Leadership Team to Support Clinical Development and Commercial Growth Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s Disease and other neurodegenerative diseases, today announced the appointments of Erin Whitney to Head of Global Clinical Operations, Shauna Horvath, previously a brand strategy consultant to Amylyx, to Head of Global Marketing and Keith White to Head of Global Market Access. These key additions to the Amylyx team are integral to support the company’s continued growth and future commercialization plans. “We are thrilled to have these highly skilled leaders join the Amylyx team at this critical time of growth as we continue our efforts to advance the development of AMX0035, our investigational product, in multiple neurodegenerative diseases,” said Joshua Cohen, Co-CEO, Chairman and Co-Founder of Amylyx. “Erin’s deep experience in complex clinical

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Amylyx Pharmaceuticals to Present Positive Functional and Survival Data and New Predictive Modeling Results from the CENTAUR Trial in People with ALS at the 31st International Symposium on ALS/MND December 9, 2020

– Administration of AMX0035 Is Associated with a Statistically Significant Retention of Functional Abilities and Long-Term Survival Benefit in People Living with ALS – New Analysis Using ENCALS Survival Prediction Model Demonstrates Longer Overall Survival Resulting from AMX0035 Use Compared to Predicted Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced the presentation of results from the CENTAUR and open-label extension (OLE) trials demonstrating positive results from administration of AMX0035 in the retention of functional abilities and survival for people with ALS at the 31 st International Symposium on ALS/MND. CENTAUR was a 24-week placebo-controlled study of 137 participants with ALS that evaluated the safety and efficacy of AMX0035. The study was led by Drs. Sabrina Paganoni and Merit Cudkowicz from the Healey Center for

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Amylyx Pharmaceuticals Announces FDA Granted Orphan Drug Designation for AMX0035 for the Treatment of Wolfram Syndrome November 16, 2020

Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AMX0035 for the treatment of Wolfram syndrome. “There is a critical unmet need for people living with Wolfram syndrome, a rare genetic disease in which many patients die prematurely with severe neurological disabilities,” said Wolfram syndrome expert Fumihiko Urano, M.D., Ph.D., a Professor of Medicine and of Pathology and Immunology at Washington University School of Medicine in St. Louis. “Endoplasmic reticulum (ER) dysfunction is a critical component of Wolfram syndrome. AMX0035 is designed to target the ER stress, and preclinical data suggest that it may be a promising approach to halt the irreversible progression of optic nerve atrophy in patients with Wolfram syndrome.” The FDA may grant

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Amylyx Pharmaceuticals Announces Last Patient Completes Final Study Visit in PEGASUS Trial of AMX0035 in Alzheimer’s Disease November 9, 2020

Topline Results Expected in the First Half of 2021 Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced that the last participant has completed the planned 24 weeks in the Phase II PEGASUS trial assessing the safety and biological activity of AMX0035 administration in people with Alzheimer’s disease. “Alzheimer’s is a complex, progressive brain disease defined by amyloid and tau pathologies, but we now realize that there are multiple pathways driving the disease,” said Steven E. Arnold, M.D., Professor and Translational Neurology Head of the Interdisciplinary Brain Center at Massachusetts General Hospital and Harvard Medical School and the PEGASUS trial principal investigator. “The PEGASUS trial design and broad inclusion criteria will allow us to assess whether AMX0035 can prevent neurodegeneration in people with Alzheimer’s via MR

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Amylyx Pharmaceuticals Announces Publication of CENTAUR Survival Data Demonstrating Statistically Significant Survival Benefit of AMX0035 for People with ALS October 16, 2020

Data From CENTAUR Trial Published in Muscle & Nerve Demonstrates That Administration of AMX0035 Results in Longer Survival for People With ALS Early Administration of AMX0035 Reduced the Risk of Death by 44% Over the Duration of Follow-Up AMX0035 is the First ALS Therapeutic to Demonstrate Both a Statistically Significant Survival and Functional Benefit in ALS in a Randomized Controlled Trial Majority (77%) of Patients Were Receiving One or More Approved ALS Therapies at or Before Study Entry Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced the publication of an overall survival analysis from the CENTAUR trial evaluating AMX0035 in people with ALS in the neuromuscular disease journal Muscle & Nerve . The survival analysis followed each CENTAUR participant for up to three years from the start of the trial regardless of

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Amylyx Pharmaceuticals Highlights New Long-Term Safety Data from CENTAUR Study of AMX0035 in People with ALS at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2020 Virtual Meeting October 9, 2020

Data from New Long-Term Safety Analysis Show No New Safety Concerns with AMX0035 Identified in the Open-Label Extension Study Data Were Consistent with Primary Outcome of Statistically Significant Slowing of the Functional Decline Associated with the Administration of AMX0035 Company Also Presented Secondary Outcome Results Demonstrating the Administration of AMX0035 Resulted in Statistically Significant Slower […]

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Get the latest news and updates from Amylyx, delivered right to your inbox.

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