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Acceleron shelves drug after disappointing readout

Acceleron Pharma won’t continue work on one of its experimental drugs after receiving disappointing results from a mid-stage study.The study tested whether Acceleron’s drug, known as ACE-083, would be a useful treatment for patients with Charcot-Marie-Tooth disease, which causes nerve damage and muscle weakness in the limbs.According to a Monday release, the drug actually hit the study’s main goal by showing a statistically significant increase in mean total muscle volume compared to placebo. Yet that added muscle didn’t translate into significant functional or quality of life improvements, according to Acceleron. As such, the company has decided to discontinue development and focus resources around its other drugs.

Pharmaceutical companies don’t often shelve a drug right after it achieves the primary goal of a clinical trial. But in the case of ACE-083, Acceleron isn’t convinced of the drug’s utility after it failed to hit any of the study’s secondary endpoints measuring muscle function and quality of life.

“Unfortunately, over the course of multiple clinical trials, our myostatin-plus hypothesis has not resulted in the functional outcomes necessary to provide clinically meaningful benefits for patients with neuromuscular diseases,” Habib Dable, Acceleron’s CEO, said in a March 9 statement.

Like all of Acceleron’s drugs, ACE-083 targets a large family of proteins called transforming growth factor beta, which regulates immune system responses and affects cell growth, differentiation and death. This drug in particular was meant to inhibit certain proteins within the family, namely myostatin, that stifle muscle growth.

Acceleron plans to present for detailed results from the Phase 2 study in April, at the American Academy of Neurology’s annual meeting. In the meantime, the company is ending further study of ACE-083, including the Charcot-Marie-Tooth trial as well a separate mid-stage extension study.

A spokesperson said the company is winding down that latter study “as quickly as possible.” Patients won’t receive the drug anymore, the spokesperson added, but may have follow-up visits.

Dable said his company will now focus resources around hematology and pulmonary conditions.

In late January, the company’s experimental drug sotatercept scored positive Phase 2 data in a study of patients with pulmonary arterial hypertension. The data sparked a nearly 50% spike in share value for Acceleron and ambitious forecasts from sell-side analysts who claimed sotatercept could bring in hundreds of millions of dollars by 2030.

That readout also came just weeks after Acceleron and partner Bristol Myers Squibb received U.S. approval for Reblozyl, a treatment for anemia associated with a blood disorder called beta-thalassemia.

Accleron shares were up 2% in late morning trading Tuesday. Over the past year, shares of the Cambridge, Massachusetts-based pharma have almost doubled in price.