The Canadian Neuromuscular Disease Registry and Amylyx Pharmaceuticals Partner on Real-World Evidence Generation for a Novel Investigational Therapy in ALS

The Canadian Neuromuscular Disease Registry and Amylyx Pharmaceuticals (NASDAQ: AMLX) today announced they are partnering on a real‐world evidence generation initiative for Amylyx’ novel investigational therapy AMX0035.

Feb. 3, 2022 13:00 UTC

CALGARY, Alberta & TORONTO--(BUSINESS WIRE)-- The Canadian Neuromuscular Disease Registry (CNDR), an independent, academic registry based out of the University of Calgary, and Amylyx Pharmaceuticals (NASDAQ: AMLX) today announced they are partnering on a real‐world evidence generation initiative for Amylyx’ novel investigational therapy AMX0035 (sodium phenylbutyrate (PB) and ursodoxicoltaurine (TURSO; also known as taurursodiol)), for the treatment of amyotrophic lateral sclerosis (ALS).

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The CNDR’s principal investigator, Dr. Lawrence Korngut, in collaboration with co-principal investigators, Drs. Agessandro Abrahao and Lorne Zinman from the Sunnybrook Health Science Centre and the University of Toronto, will partner with neuromuscular centers across Canada, including the pan-provincial ALS clinics that are part of the Canadian ALS Research Network (CALS), to follow consenting participants and collect key demographic, clinical, functional and survival data related to people living with ALS taking AMX0035. The CNDR-Amylyx partnership has the potential to generate the first global real‐world evidence on AMX0035 for scientific dissemination.

“We believe this collaboration, which brings together the real‐world data capability of the CNDR and the industry expertise of Amylyx Pharmaceuticals, will provide an opportunity for Canadians living with ALS and their loved ones to gain important insights on the impact of AMX0035,” said Dr. Lawrence Korngut. “The advantage of CNDR is that it enables the generation of real‐world evidence for decision‐making, while prioritizing patient treatment and access to investigational therapy. We hope that with this partnership, we can provide insights and guidance for best practices in ALS treatment.”

The ALS Society of Canada welcomes today’s announcement as an important step forward for the ALS community.

“Introducing new investigational therapies for people living with ALS in a timely manner is critical, however we need to better understand how patients respond to investigational therapies in a real‐world context,” said Tammy Moore, CEO of the ALS Society of Canada. “Collaborations between industry and academia are important to ensure that Canadians living with ALS have timely access to the best possible evidence‐based treatments. This partnership could also lay the framework for the timely introduction of future ALS therapies.”

“This investment and partnership with the CNDR is an extension of our long‐standing commitment to the ALS community, as we continue to uncover the full potential of AMX0035 for the benefit of people living with ALS around the world and their loved ones,” said Joshua Cohen, Co‐CEO, Chairman and Co‐Founder of Amylyx.

Justin Klee, Co‐CEO, Director and Co‐Founder of Amylyx added, “Canada has been the site of many first milestones for Amylyx, from setting up a manufacturing partnership in Ontario, to filing the first new drug submission with Health Canada to bring AMX0035 to Canadians living with ALS, and now, this unique collaboration with the CNDR. While this partnership gets underway, we will continue to work expeditiously through the Canadian approval processes required by federal, provincial and territorial governments.”


The Canadian Neuromuscular Disease Registry (CNDR) is a Canada‐wide registry of individuals diagnosed with a neuromuscular disease including ALS. The CNDR collects important medical information from individuals across the country to improve the understanding of neuromuscular disease and to accelerate the development of new therapies.

Currently, CNDR is working with 36 neuromuscular and 14 ALS clinical sites across Canada. With over 4600 patients registered and an extensive clinical‐investigator network, the CNDR has helped facilitate over 160 Canadian led projects using registry data.

The CNDR aims to change the landscape of neuromuscular disease to the benefit of affected individuals and families across Canada.


Amylyx Pharmaceuticals, Inc. is a clinical‐stage biopharmaceutical company working on developing a novel therapeutic for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. For more information, visit and follow us on LinkedIn and Twitter. For investors please visit


AMX0035 is a proprietary oral fixed-dose combination of two small molecules: sodium phenylbutyrate (PB), which is a small molecular chaperone designed to reduce the unfolded protein response (UPR), preventing cell death resulting from the UPR, and ursodoxicoltaurine (TURSO; also known as taurursodiol), which is a Bax inhibitor designed to reduce cell death through apoptosis. PB and TURSO were combined in a fixed-dose formulation in an effort to reduce neuronal death and dysfunction. AMX0035 is designed to target the endoplasmic reticulum and mitochondrial-dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases.


Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements”, within the meaning the of Private Securities Litigation Reform Act of 1995, as amended, including with respect to the offering. Such statements include, but are not limited to, statements regarding the potential approval of for the treatment of ALS; the potential of AMX0035 as a treatment for ALS; and the success of the partnership between Amylyx and CNDR in generating evidence and insights into the potential impact of AMX0035. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Any forward-looking statements in this statement are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include those risks and uncertainties set forth in Amylyx’ regulatory filings with the United States Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date on which they were made. Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.



Amylyx Pharmaceuticals
Becky Gohsler
Finn Partners
(646) 307‐6307

Lindsey Allen
Amylyx Pharmaceuticals, Inc.
(857) 320-6222

Source: Amylyx Pharmaceuticals, Inc. and The Canadian Neuromuscular Disease Registry (CNDR)