Ionis and Biogen Drop ALS Drug After Early-to-Mid-Stage Study Failu

After the disappointing outcome of early-stage trials for their antisense drug BIIB078 in 2022, Ionis and Biogen have faced another setback with the results of their early-to-mid-stage trial for the amyotrophic lateral sclerosis (ALS) treatment BIIB105.

While the trial achieved its target of reducing levels of a protein called ataxin-2 in ALS patients, it failed to impact the reduction of the neurofilament light chain. This failure to significantly affect the neurofilament light chain, which is crucial in positively impacting key health measures such as strength, breathing, and overall functioning as ALS progresses, rendered the results insufficient to warrant further development of the drug.

Findings from the nearly 100-person trial revealed that BIIB105 significantly decreased ataxin-2 levels compared to a placebo over a six-month period. However, upon completion of this period, when participants entered an “open-label extension” phase where everyone received the drug, the study data provided by Ionis and Biogen showed no benefits regarding clinical outcome measures or effectiveness in neurofilament reduction.

Stephanie Fradette, head of Biogen’s Neuromuscular Development Unit, stated, “While BIIB105 lowered ATXN2 protein, it did not reduce neurofilament, which gives us confidence that BIIB105 did not slow the disease process.”

The decision to terminate the development of their experimental treatment caused shares of Biogen and Ionis to fall by nearly 2% in premarket trading.

Recently, Amylyx Pharmaceuticals also had to withdraw from the race for an ALS drug. Just over a month ago, the company announced that it would be withdrawing its ALS drug, Relyvrio, from markets in Canada and the U.S. after the drug performed poorly in a key late-stage trial.

Currently, the two drugs competing in the ALS market are Radicava from the Japanese firm Mitsubishi Tanabe and Qalsody, which is notably a product of the collaboration between Ionis and Biogen.

In April of last year, Qalsody received accelerated approval in the United States based on preliminary data. The introduction of Qalsody changed research parameters within the ALS community. Although the therapy failed to hit its central goal, it reduced the production of an ALS-associated protein called SOD1. This allowed the FDA to understand that while the drug may not be better than a placebo at slowing disease progression, it can fix a key biological marker of neurological damage by lowering the neurofilament light chain.

This decision established the importance for drugmakers to demonstrate an effect on neurofilament in their clinical tests and provided a new pathway for ALS drug approvals.

Approximately 60,000 people in the U.S. and Europe are affected by ALS. According to U.S. government data, most patients who get the disease die within five years of diagnosis.

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