Apellis Drops ALS Candidate After Disappointing Phase II Results

brain scans

Pictured: Scans of human brain /iStock, Nur Ceren Demir

Apellis Pharmaceuticals is the latest company to fall short in amyotrophic lateral sclerosis. On Thursday, the Massachusetts-based biopharma announced it is dropping its experimental drug, pegcetacoplan, in ALS after results from the Phase II MERIDIAN trial showed it failed to meet both its primary endpoint and key secondary efficacy endpoints. 

The mid-stage trial, conducted alongside Apellis’ partner Sobi, enrolled 250 adult ALS patients and compared pegcetacoplan to a placebo. Trial data showed the investigational therapy did not demonstrate a statistically significant difference in the time taken for disease progression between the treatment and placebo groups. 

Pegcetacoplan, a targeted C3 therapy, has shown promise in treating other disorders in the past and has been previously approved to treat paroxysmal nocturnal hemoglobinuria and geographic atrophy. According to Apellis’ press release, it is also currently being studied in several rare diseases across hematology, nephrology and neurology.

The trial failure, while disappointing, was likely somewhat expected. In April, Apellis discontinued the open-label portion of the MERIDIAN trial at the recommendation of an independent monitoring committee. 

A neurodegenerative disease with limited treatment options, ALS has long befuddled the industry, and there have been few wins in the space since Amylyx’s Relyvrio scored FDA approval and became the third approved drug on the market for ALS in September 2022.

Apellis is the second biopharma company this week to fall short in its attempt to develop an effective treatment. On Tuesday, Wave Life Sciences announced its antisense candidate being developed for ALS and frontotemporal dementia failed to show clinical benefit in a Phase Ib/IIa trial. Based on these data, the company decided to drop the asset from its pipeline. 

As for Apellis, the company stated it plans to conduct a thorough analysis of the data to gain insights into the trial results and present the findings at a future medical meeting.

Jeffrey Eisele, chief development officer at Apellis said in a statement that the company hopes the data generated from the MERIDIAN study “will continue to support future research and development in ALS.” 

Rosemary Scott is a content editor at BioSpace, focusing on the job market and career development for professionals in the life sciences. You can reach her at rosemary.scott@biospace.com and on LinkedIn.

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