Health Canada to Review AMX0035 as ALS Treatment

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by Somi Igbene |

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AMX0035 for ALS

Health Canada has agreed to review Amylyx Pharmaceuticals‘ application seeking approval of 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 the country,” Chris Aiello, general manager and head of Canada at Amylyx, said in a press release.

This “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,” Aiello added.

Meanwhile, the company plans to submit a similar application in Europe and is in discussions with regulatory authorities worldwide to determine the best path for approval in each location.

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A Phase 3 clinical trial expected to support regulatory submission in the U.S. is planned to begin in the coming months. The PHOENIX trial will enroll up to 600 ALS patients at 55 sites across the U.S. and Europe.

AMX0035 is made of two small molecules, tauroursodeoxycholic acid and sodium phenylbutyrate, that protect nerve cells. Both compounds are in clinical use, and known to be generally safe and well-tolerated. They work to block stress signals within mitochondria — the powerhouses of cells — and the endoplasmic reticulum, a cellular organelle involved in protein production, modification, and transport.

The PHOENIX trial and regulatory fillings are based on data from CENTAUR, a Phase 2 clinical trial (NCT03127514) that evaluated the safety and effectiveness of AMX0035 in 137 adults who had a recent diagnosis of ALS and rapidly progressing disease.

Participants were assigned randomly to receive either AMX0035 or a placebo, twice daily for six months, after which most (92%) chose to enter an open-label extension part (NCT03488524), in which all are receiving AMX0035 for up to 30 months.

Top-line data from CENTAUR showed that AMX0035 significantly slowed patients’ functional decline — as assessed with the ALS Functional Rating Scale-Revised — and led to improvements in muscle strength, breathing, and hospitalization rates.

Patients who received the active medication during the randomized part also showed better survival rates than those on a placebo — median survival of 25 months versus 18.5 months, representing a 44% lower risk of death.

The combined risk of death, tracheostomy, permanent assisted ventilation, or first hospitalization was reduced by 44%. The therapy was considered generally safe and well-tolerated.

Despite the promising results, a petition asking for U.S. approval as quickly as possible, and a call to action from the The ALS Association, the U.S. Food and Drug Administration requested data from an additional controlled clinical trial — the PHOENIX trial — before considering AMX0035 for approval.