ALS drug pulled from market in Canada, U.S. after failing late-stage clinical trial

Amylyx Pharmaceuticals said Thursday it will with‐ draw its amyotrophic later‐ al sclerosis (ALS) drug from the market in Canada and the U.S. following its failure in a late-stage clinical trial.

The ALS drug, branded Al‐ brioza in Canada and Re‐ lyvrio in the U.S., was ap‐ proved in both countries in 2022.

ALS causes progressive paralysis and death. Each year about 1,000 Canadians die from the disease and about the same number are diagnosed, according to the ALS Society of Canada's web‐ site. ALS affects roughly 60,000 people in the U.S. and Europe.

There are few options to treat the potentially fatal dis‐ ease.

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The medication is Amy‐ lyx's only product on the market. The drug will no longer be available to new patients.

"Patients currently on therapy in the U.S. and Cana‐ da who, in consultation with their physician, wish to stay on treatment can be transi‐ tioned to a free drug pro‐ gram," the Cambridge, Mass., company said in a release.

The drug failed to outper‐ form a placebo on a scale that measures the ability of patients to perform tasks such as walking, breathing, speaking and swallowing.

The company did not pro‐ vide detailed results, but the numbers cited suggest the drug provided no benefit.

Dr. Angela Genge, a neu‐ rologist in Montreal and di‐ rector of the Amyotrophic Lateral Sclerosis (ALS) clinic at The Neuro, called the an‐ nouncement disappointing but not discouraging.

"We were very hopeful that this was a new product that would help us in the fight against ALS, but pleased that the company kept their word in the decision to take the drug off the market if the confirmatory study was neg‐ ative," Genge said in an inter‐ view.

Genge said the ALS com‐ munity is using and testing several new pills and injec‐ tions targeted to what hap‐ pens in the disease.

Lack of 'substantially persuasive' data

Amylyx also announced a 70 per cent reduction in its workforce. At the end of 2023, the company had 384 full-time employees.

Approval followed a rare turnaround by the U.S. Food and Drug Administration's advisers. They backed the drug months after rejecting it for a lack of "substantially persuasive" data.

The approval was based on mid-stage trial data in 137 patients that showed the treatment slowed disease progression and extended life expectancy.

However, a larger latestage study failed to confirm the reported benefit of slow‐ ing disease progression, with no significant difference in patients treated with Relyvrio and a placebo.

The drug, which has a list price of $158,000 US per year in the U.S., generated sales of about $381 million US in 2023.

Amylyx said it plans to present the data from its Phoenix clinical trial at an up‐ coming medical meeting and will publish the results in a medical journal later this year.