According to a story from BioPharma Dive, Apellis Pharmaceuticals will now join the ranks of pharma companies that have seen an investigational therapy being developed for amyotrophic lateral sclerosis falter in a clinical trial. The findings from the company’s phase 2 clinical trial failed to demonstrate any benefit from systemic pegcetacoplan when measured alongside placebo. The trend of clinical trial failure in amyotrophic lateral sclerosis (ALS) suggests that the scientific understanding of the disease still has some major gaps.

About Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic lateral sclerosis, otherwise known as Lou Gehrig’s disease, is a rare, degenerative disease that causes the death of nerve cells associated with the voluntary muscles. Little is known about the origins of amyotrophic lateral sclerosis, with no definitive cause in about 95 percent of cases. The remaining five percent appear to inherit the disease from their parents. Symptoms initially include loss of coordination, muscle weakness and atrophy, muscle stiffness and cramping, and trouble speaking, breathing, or swallowing. These symptoms worsen steadily over time; most patients die because of respiratory complications. Treatment is mostly symptomatic and the medication riluzole can prolong life. Life expectancy after diagnosis ranges from two to four years, but some patients can survive for substantially longer. To learn more about amyotrophic lateral sclerosis, click here.

ALS Confounds the Pharma Industry

The drug failed to meet the study’s primary endpoint, which was a statistics tool called the Combined Assessment of Function and Survival. Secondary endpoints included assessments of overall survival, functionality, muscle strength, and lung function, which were also not achieved.

Apellis was not taken by surprise as an independent board that was charged with monitoring the study course already urged the company to abandon the open label portion of the trial, which would have offered the drug to all participants after the 52-week mark. The disappointing result follows a discouraging trend in the development of treatments for the neurodegenerative disease. Very few ALS drug trials end in success, leaving this patient community with a severe unmet need and little chance for prolonged survival or slowed disease progression. 

Other companies that have tried and failed to develop a therapy for this disease include:

• Biohaven Pharmaceuticals
• Biogen
• Cytokinetics
• Alexion Pharmaceuticals
• Wave Life Sciences