- Apellis Pharmaceuticals on Thursday became the latest drugmaker to give up on a potential treatment for amyotrophic lateral sclerosis, a neurodegenerative disorder that has frustrated researchers for decades.
- The decision came after a Phase 2 trial showed no benefit for the drug, known as systemic pegcetacoplan, compared with a placebo. It failed to meet the primary endpoint, measured by a statistical tool called the Combined Assessment of Function and Survival, as well as secondary goals assessing overall function, survival, lung function and muscle strength.
- The final results weren’t a total surprise; an independent board monitoring the study had already advised the company not to start a second, “open-label” part of the trial that would have offered the medicine to all participants after the initial 52-week research period. Apellis executives had also signaled to analysts that the trial had a low chance of success.
While the study failure may have little impact on Apellis shares, it’s another disappointment for patients with the devastating condition, also known as Lou Gehrig’s disease. The disorder attacks nerve cells in the brain and spinal cord, causing a loss of muscle control that eventually affects the whole body, even robbing a person of the ability to breathe. The average survival time after diagnosis is three years.
In 2017, researchers documented more than 60 molecules that had been studied as potential treatments for ALS. The “overwhelming majority” resulted in disappointment. In recent years, Biogen, Cytokinetics, Alexion Pharmaceuticals and Biohaven Pharmaceuticals have all announced study failures in ALS. And just two days ago, Wave Life Sciences joined the list.
In part spurred by appeals from patients, the Food and Drug Administration and National Institutes of Health last year launched an initiative focused on ALS and other rare brain conditions. The FDA has also shown a willingness to advance treatments, clearing an ALS medicine from Amylyx Pharmaceuticals in September and a Biogen drug in April for a small subset of ALS patients with a specific genetic mutation.
None of the approved drugs can reverse or cure the condition, at best giving patients a chance to slow their decline and live longer. Before the approval of Amylyx’s Relyvrio, only two medicines were available specifically to address the progression of ALS. The first, Rilutek, won FDA clearance in 1995. Another, Mitsubishi Tanabe Pharma’s Radicava, reached the market in 2017.
The drug developed by Apellis is designed to regulate the “complement cascade,” a part of the innate immune system that can essentially overreact and mistakenly attack healthy cells and tissue. Apellis and its partner, Swedish Orphan Biovitrum (Sobi), are also studying systemic pegcetacoplan for other uses in hematology, nephrology and neurology.
Apellis already has approval to sell the medicine in a different form for geographic atrophy, a common eye condition that causes vision loss. For that use, the company sells the drug as an injection under the brand name Syfovre. The drug is also sold to treat a rare blood disease, for which it’s sold as Empaveli.