Denali-Sanofi's ALS drug fails to meet mid-stage trial goal

[February 17, 2024]  (Reuters) -Drug developer Denali Therapeutics said on Friday its and partner Sanofi's experimental drug for a fatal neurodegenerative disease failed to slow decline of motor function in a mid-stage study.

Shares of Denali were down nearly 8% in morning trading.

This marks the latest setback in the long list of roadblocks in the path to develop effective treatments against amyotrophic lateral sclerosis (ALS), a condition which affects 16,000 to 32,000 people in the United States, and almost completely paralyzed late British physicist Stephen Hawking.

The U.S. FDA has given traditional approval for three drugs — Japanese firm Mitsubishi Tanabe's Radicava, generic drug Riluzole and Amylyx Pharmaceuticals' Relyvrio — for the treatment of ALS to date.

Biogen's Qalsody received the agency's accelerated approval in April last year.

Denali said their drug did not meet the main goal of change in the ALS functional rating scale, which measures deviations from "normal" motor functioning as caused by ALS.

Their ALS drug works by inhibiting increased activity of a protein that is thought to contribute to neurodegeneration.

Sanofi logo at the company's headquarters during the annual results news conference in Paris, France, February 4, 2022. REUTERS/Benoit Tessier/ File photo

ALS is a rare neurological disease that can break down nerve cells in the brain and spinal cord responsible for muscle movements, leading to progressive paralysis and death.

Sanofi, however, will continue to conduct a mid-stage trial evaluating the drug in participants with multiple sclerosis, a disease of the central nervous system.

Sanofi and Denali had entered into a partnership in 2018, where Sanofi agreed to conduct trials to test therapies developed by Denali for neurological and inflammatory diseases.

(Reporting by Christy Santhosh; Editing by Shilpi Majumdar)

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