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ALS drug from Denali, Sanofi falls short in mid-stage study

Dive Brief:

  • An experimental drug being developed by Denali Therapeutics and partner Sanofi for ALS failed to meet its goal in a Phase 2 trial, the biotechnology company disclosed Thursday via a regulatory filing.
  • The trial, dubbed Himalaya, tested the companies’ drug on a rating scale known as the ALSFRS-R, which measures physical functions like walking and speaking. According to Denali, Sanofi reported results that didn’t meet the primary endpoint of change in ALSFRS-R.
  • Denali had previously highlighted the readout as one of four to watch this year, and shares sank by more than 6% Friday morning on the news. Sanofi is also testing the drug in people with multiple sclerosis.

Dive Insight:

Denali and Sanofi’s partnership dates back to 2018, when they agreed to work together to develop drugs for autoimmune and neurological disorders. The deal, which handed Denali $125 million upfront, focused on small molecules that target a protein called RIPK1.

The ALS drug, known alternatively as SAR443820 or DNL788, was a product of that research. In Sanofi’s latest earnings report, the pharmaceutical firm had earmarked the drug for advancement into Phase 3 testing later this year, pending the outcome of the Phase 2 study that just read out results.

Those plans are now in question, although Jefferies analyst Michael Yee wrote in an investor note Friday that expectations were “very low.”

Denali didn’t indicate whether ALS development would be discontinued, only noting that Sanofi would present the study’s efficacy and safety results at a “future scientific forum.”

Despite the setback, Sanofi is continuing to evaluate the drug in multiple sclerosis, another nervous system disorder involving damage to nerves.

Denali is also testing another drug for ALS, or amyotrophic lateral sclerosis. That candidate, dubbed DNL343, works by activating a protein called eIF2B to block what’s known as “integrated stress response.” Per Denali, this response is implicated in nerve cell death.

DNL343 is currently being tested in a high-profile “platform” study being run by the Sean M. Healey & AMG Center at Massachusetts General Hospital.