verdiperstat

Biohaven‘s therapy candidate verdiperstat did not significantly slow functional decline among people with amyotrophic lateral sclerosis (ALS) in the HEALEY ALS platform trial, failing to meet its primary goal, data show. Key secondary efficacy goals, such as survival and changes in lung function and muscle strength, also were…

Note: This story was updated June 16, 2022, to reflect that competitor Kyle Brown was not disqualified, but did not finish the swim portion of the Ironman World Championship within the time limit and was not able to move forward in the competition.   Patrick Harfield was on his last legs.

The HEALEY ALS platform trial, the first study to test several therapy candidates for amyotrophic lateral sclerosis (ALS) simultaneously, has completed enrollment for at least two of its first three regimens. These include Clene Nanomedicine’s CNM-Au8 (NCT04414345) and Biohaven Pharmaceuticals’ verdiperstat (NCT04436510). The recruitment status…

The HEALEY ALS platform trial, which is evaluating the safety and efficacy of several potential treatments for amyotrophic lateral sclerosis (ALS), has passed the 50% participant enrollment target for its first three regimens. The investigational treatments are UCB’s zilucoplan, Biohaven Pharmaceuticals’ verdiperstat, and Clene Nanomedicine’s CNM-Au8. A fourth candidate…

The first platform trial for amyotrophic lateral sclerosis (ALS), designed to assess multiple treatment candidates simultaneously with a goal of accelerating the development of ALS therapies, has enrolled its first patients. The HEALEY ALS Platform Trial (NCT04297683) will start by evaluating UCB’s…

The U.S. Food and Drug Administration (FDA) has authorized the evaluation of the first three candidate therapies for amyotrophic lateral sclerosis (ALS) in the HEALEY ALS Platform Trial. This trial model, designed to accelerate the development of ALS therapies by assessing multiple treatment candidates simultaneously, is the first of…

To help accelerate development of effective treatments for amyotrophic lateral sclerosis (ALS), the ALS Association is investing $3 million in the disease’s first platform trial. The award is $1 million annually for three years to support the platform investigation, a type of clinical study that evaluates the…