Canadian Registry, Amylyx Working to Collect Real-life Data on AMX0035

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by Mary Chapman |

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The Canadian Neuromuscular Disease Registry (CNDR) and Amylyx Pharmaceuticals are collaborating on an initiative that could produce the first real-world evidence on AMX0035, Amylyx’s investigational therapy to slow functional decline in amyotrophic lateral sclerosis (ALS) patients.

The two, in partnership with neuromuscular centers across Canada, will collect demographic, clinical, functional, and outcomes data from ALS patients using the experimental treatment. These centers include clinics that are part of the Canadian ALS Research Network.

“We believe this collaboration, which brings together the real-world data 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,” Lawrence Korngut, MD, the registry’s lead investigator, said a press release.

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AMX0035 is an oral and fixed-dose combination therapy containing two small molecules — sodium phenylbutyrate and tauroursodeoxycholic acid — in clinical use and with a good safety profile. They work together to minimize cellular mechanisms linked to cell death in ALS.

The therapy is under regulatory review for possible approval in Canada and in the U.S., where a U.S. Food and Drug Administration decision is expected by June 29. A request for review has also been filed in Europe.

These submissions were based on top-line results from the CENTAUR Phase 2/3 clinical trial (NCT03127514), which showed that AMX0035 slowed functional decline and extended survival compared with placebo in patients with rapidly advancing disease.

CNDR, a Canada-wide registry of people with ALS and other neuromuscular diseases, aims to improve the understanding of these disorders to advance new treatments. More than 4,600 patients are registered among its 36 neuromuscular and 14 ALS clinical sites.

“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,” Korngut said.

The ALS Society of Canada called this partnership with Amylyx 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, the society’s CEO.

“Collaborations between industry and academia are important … [and] this partnership could also lay the framework for the timely introduction of future ALS therapies,” Moore added.

“While this partnership gets underway, we will continue to work expeditiously through the Canadian approval processes required by federal, provincial and territorial governments,” said Justin Klee, co‐CEO, director and co‐founder of Amylyx.

An ongoing Phase 3 clinical trial — PHOENIX (NCT05021536) — that started in November is also investigating AMX0035 versus a placebo in about 600 ALS patients. The roughly one-year trial is expected to be enrolling eligible patients at sites across the U.S. and Europe; more information can be found here. Currently, only U.S. sites are listed.

Its main goal is changes in a joint assessment of functional decline — measured with the ALS Functional Rating Scale-Revised (ALSFRS-R) — and survival after 48 weeks. Secondary outcomes include changes in lung health, the need for ventilation, and quality of life.

AMX0035 is reported to work to prevent nerve cell death by blocking stress signals within mitochondria — the cells’ powerhouses — and the endoplasmic reticulum, a cellular organelle involved in protein production, modification, and transport.