XLRN - Don't Dismiss Acceleron Despite Its Recent Clinical Setback
Getting new drugs to market can be a tough business. Acceleron Pharma (NASDAQ: XLRN) is learning this the hard way, scrapping plans to continue development for its experimental drug, ACE-083, in treating patients who have facioscapulohumeral muscular dystrophy (FSHD).
The drug showed initial promise for FSHD patients after it proved to increase muscle strength in patients who have disorders that impair muscle strength. Just over a year ago, in May 2018, the company was given fast-track status by the FDA to expedite development and bring the drug to FSHD patients, who currently don't have any available drug therapies to inhibit muscle impairment.
However, earlier this month, Acceleron announced that in phase 2 trials, ACE-083 did not "achieve functional secondary endpoints." In other words, while it increases muscle volume, it's not actually helping FSHD patients by improving muscle function. This was, of course, a huge blow to the small company, which has invested a lot of time and energy into the product and has only a few candidates in its pipeline.