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

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 theNortheast 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 biorepositories can play an important role in treatment and care breakthroughs if they are continuously updated,” said Joshua Cohen, Co-CEO, Chairman and Co-Founder of Amylyx. “Keeping data fresh will give the ALS community the information it needs to identify new pathways and approaches to developing new treatments. We’re grateful to each CENTAUR participant who donated invaluable time and energy to the clinical trial, and without whom this resource would not be possible,” added Justin Klee, Co-CEO and Co-Founder of Amylyx.

The PRO-ACT database, which is currently sponsored by The ALS Association and managed by the NCRI, houses the largest ALS clinical trials dataset, containing nearly 11,000 ALS patient records from 23 completed clinical trials. The platform harmonizes and merges anonymized data from existing publicly and privately conducted ALS clinical trials to generate a unique, freely available resource for the scientific community to find cures for ALS. The PRO-ACT platform was selected as the Bio-IT World’s Best Practices Awards winner in 2013 and The Clinical Informatics News Best Practices winner in Clinical Data Intelligence category in 2015.

The PRO-ACT platform was created by Prize4Life Israel, a non-profit organization, in partnership with NEALS and the NCRI at Mass General Hospital, and with initial funding from The ALS Therapy Alliance, Prize4Life, NCRI, and The ALS Association. To date, PRO-ACT has served as the primary data source for more than 70 publications and has been critical for numerous others. The platform has allowed researchers to better understand disease heterogeneity, develop novel predictive models of disease progression and has been a critical tool to support the design of several ALS clinical trials.

“The PRO-ACT platform is only as valuable as the data that is fed into it,” said Avi Kremer, Founder of Prize4Life, an ALS Association consultant, and a person living with ALS. “Having data that take into account the evolving approaches to ALS treatment and results from trials allows the community to build on past work and move us closer to a cure for this devastating disease.”

“Data that would otherwise normally sit with a company and not be accessible to researchers have become an invaluable resource, and most importantly, a freely shared one,” said Alex Sherman, Director of the Center for Innovation and Biomedical Informatics (CIB) at the Neurological Clinical Research Institute (NCRI) and Healey & AMG Center at Mass General, Principal Associate in Neurology at Harvard Medical School, leader of the team that created the PRO-ACT database, and the current Principal Investigator of the PRO-ACT platform. “Each learning brings us one step closer to better understanding, better diagnosing, and ultimately finding new or better treatments for ALS. We hope to see this momentum continue and more companies will follow Amylyx in data donations.”

In a continued effort to help foster collaboration and innovation among the ALS community, Amylyx has also donated participants’ biological samples from the CENTAUR trial to the NEALS Biorepository. These biospecimens are available to the research community for the purpose of furthering the understanding of ALS or motor neuron disease, testing the effects of potential drug treatments, and developing disease biomarkers. The NEALS Biorepository is funded by The ALS Association, Project ALS, ALS Finding a Cure, and donors to the Healey and AMG Center at Mass General.

“We commend Amylyx for its commitment to transparency with the broader research community and allowing scientists across the world to do deeper analyses with the shared samples and datasets,” said Neil Thakur, Ph.D., Chief Mission Officer at The ALS Association.

“In recent years, we have seen shifts in research that could change the treatment trajectory of ALS. New findings in this complex disease are increasingly helpful. I am delighted that, thanks to this donation, clinical data and biological samples obtained in CENTAUR will continue to contribute to ALS research, thereby amplifying the impact of this trial even further,” said Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR study, investigator at the Healey & AMG Center for ALS at Mass General and member of the Executive Committee of NEALS. “We are indebted to the trial participants whose generous contributions have made progress in ALS research possible.”

“Biomarker development and new target identification is critical to understanding ALS and accelerating clinical trials,” said Merit Cudkowicz, MD, co-principal investigator of the CENTAUR trial and co-founder of the Northeast ALS Consortium, Director of the Healey & AMG Center for ALS and Chair of Neurology at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “Such incredible progress has been made, but there’s more work to do and we encourage all companies to donate clinical data and samples whenever possible in the hopes of gaining new insights that can change the course of this terrible disease.”

About the AMX-3500 (CENTAUR) Study

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. CENTAUR assessed the impact of AMX0035 on disease progression as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) when compared to placebo. CENTAUR also assessed the effects of AMX0035 on other measures that are critical to people with ALS, including muscle strength, lung vital capacity, and biomarkers of neuronal degeneration. CENTAUR was conducted by investigators at 25 top ALS medical centers through the Northeast ALS (NEALS) Consortium and led by Drs. Sabrina Paganoni and Merit Cudkowicz from the Healey & AMG Center at Mass General.

CENTAUR was the recipient of the ALS ACT grant, and is supported by The ALS AssociationALS Finding a Cure, a program of The Leandro P. Rizzuto Foundation, the Northeast ALS ConsortiumSean M. Healey & AMG Center for ALS at Mass General, and was funded in part by the ALS Ice Bucket Challenge.

More information on the CENTAUR trial can be found at www.amylyx.com/pipeline/ or www.clinicaltrials.gov, NCT03127514 or NCT03488524.

About AMX0035

AMX0035 is an investigational product comprised of two complementary active agents, sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine), which were combined in a co-formulation to reduce neuronal death and dysfunction. AMX0035 targets endoplasmic reticulum and mitochondrial dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.

About Prize4Life

Prize4Life is a nonprofit organization whose mission is to accelerate the discovery of treatments and a cure for ALS (Amyotrophic Lateral Sclerosis, also known as Lou Gehrig’s disease) by using powerful incentives to attract new people and drive innovation. Prize4Life believes that solutions to some of the biggest challenges in ALS research will require out-of-the-box thinking, and that some of the most critical discoveries may come from unlikely places. Founded in 2007 by Avi Kremer, who was diagnosed with ALS at the age of 29, Prize4Life encourages and rewards creative approaches that will yield real results for ALS patients. For more information, visit www.prize4life.il.

About Neurological Clinical Research Institute at Mass General Hospital

The Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital accelerates translational research in neurological disorders through initiating and testing novel therapies. The NCRI has an extensive history in leading clinical research to find new treatments for neurological diseases including Amyotrophic Lateral Sclerosis (ALS), myasthenia gravis, diabetic neuropathy, stroke, multiple sclerosis, Parkinson’s disease, and Huntington’s disease. For more information, visit www.NCRInstitute.org or www.data4cures.org.

About The ALS Association

The ALS Association is the largest private funder of ALS research in the world. The Association funds global research collaborations, provides assistance for people with ALS and their families through our nationwide network of chapters and certified clinical care centers, and advocates for better public policies for people with ALS. The ALS Association builds hope and enhances quality of life while urgently searching for new treatments and a cure. For more information about The ALS Association, visit our website at www.als.org.

About the Northeast ALS Consortium

The Northeast ALS Consortium (NEALS) is an international, independent, non-profit group of over 130 research sites around the world who collaboratively conduct clinical research in Amyotrophic Lateral Sclerosis (ALS) and other motor neuron diseases. The mission of NEALS is to translate scientific advances into new treatments for people with ALS and motor neuron disease as rapidly as possible. The NEALS network is governed by an Executive Committee and the Consortium research activities are advised by an experienced Scientific Advisory Board. Each Leadership team member brings a depth of experience and a unique perspective to the governance of NEALS. Through this leadership, active oversight is provided for ongoing operations along with new approaches to trial design evaluated and implemented. For more information, visit www.neals.org.

About Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc. is a pharmaceutical company working on developing a novel therapeutic for Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases. For more information, visit www.amylyx.com and follow us on LinkedIn and Twitter.


Merissa Muller

Finn Partners

(617) 778-6633

Merissa.Muller@finnpartners.com

Source: Amylyx Pharmaceuticals, Inc.

Stay Connected

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

By clicking SUBMIT, you confirm that you have read Amylyx's Privacy Policy and that you agree to receive the electronic communications from Amylyx that you've selected. You can withdraw your consent at any time.

Stay Connected

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

By clicking SUBMIT, you confirm that you have read Amylyx's Privacy Policy and that you agree to receive the electronic communications from Amylyx that you've selected. You can withdraw your consent at any time.