Double Whammy For ALS As AbbVie/Calico And Denali Drugs Fail

 
• By Kevin Grogan

Two treatments that target elF2B, a regulator of the integrated stress response that appears to be overactive in the progressive motor neurone disease, have missed their primary and secondary endpoints in a landmark platform trial.

Physiotherapist assisting a patient with Amyotrophic Lateral Sclerosis
• Source: Alamy

The amyotrophic lateral sclerosis (ALS) pipeline has suffered a series of setbacks recently and two separate candidates from AbbVie/Calico Life Sciences and Denali Therapeutics that have the same of mechanism of action are the latest to fall by the wayside.

The bad news began when Denali announced that DNL343, a small molecule that targets elF2B, a central regulator of the integrated stress response which appears to be overactive in ALS, failed to meet its primary endpoint in the Phase II/III HEALEY ALS platform trial. The initiative, which is led by the Sean M

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