Apellis scraps pegcetacoplan development in ALS after phase 2 trial flop

After Apellis Pharmaceuticals and partner Sobi ran into a clinical setback and ended the open-label extension arm of a study testing their amyotrophic lateral sclerosis (ALS) candidate, the companies have now scrapped development altogether.

The drug, Apellis’ C3 inhibitor pegcetacoplan, didn’t meet the mark in the phase 2 study called Meridian. The 52 week, 250-patient trial didn't meet its primary or secondary endpoints, Apellis said in a Thursday release.

“We are disappointed in the outcome of the MERIDIAN study, especially on behalf of the ALS community who has been waiting for new treatments for this complex and unrelenting disease,” Apellis’ chief development officer Jeffrey Eisele, Ph.D, said in the company’s statement.

“Our hope is that the data generated from this study will continue to support future research and development in ALS," Eisele added.

Apellis is analyzing the data and will present the findings at a future medical meeting, the company said.

The fail shouldn't come as a complete surprise. Late last Month, the open-label extension arm in the trial was called off after an independent data monitoring committee recommended its discontinuation.

Pegcetacoplan is already approved for paroxysmal nocturnal hemoglobinuria (PNH) and geographic atrophy (GA) and is branded as Empaveli and Syfovre, respectively, in those diseases. The geographic atrophy nod in March was a game-changer as the drug represented the first therapy approved for the condition. GA is an advanced stage of age-related macular degeneration.

On the ALS front, Biogen and Ionis’ tofersen, branded as Qalsody, bagged an accelerated FDA approval just days after Mizuho Securities analysts disclosed Apellis’ original trial setback. Qalsody is cleared to treat patients whose ALS is associated with a mutation in the SOD1 gene, a small subset of the overall patient population.

That approval came seven months after Amylyx’s Relyvrio broke into the tough-to-treat space as the first ALS treatment to show a significant slowing in disease progression, functional decline and extended survival. The FDA approved the drug after two advisory committee meetings, one with a negative outcome and another that yielded a positive vote.

Meanwhile, Apellis in early April was being eyed by larger drugmakers in a potential takeover, people with knowledge of the matter told Bloomberg. It was reportedly meeting with advisors to weigh its options, including potential partnerships or licensing agreements.