It’s the final nail in the coffin for Catabasis Pharmaceuticals’ Duchenne muscular dystrophy (DMD) hopeful as edasalonexent has once again failed to deliver.
The results look like this: The phase 3 test, known as PolarisDMD, saw edasalonexent miss both its primary and secondary endpoints.
That primary endpoint was a change from baseline in the North Star Ambulatory Assessment (NSAA) over one year of edasalonexent compared to placebo. The secondary endpoint, timed function tests (time to stand, 10-meter walk/run and four-stair climb), also did not show statistically significant improvements.
That’s a massive fail and adds to the previous flop in 2017. Edasalonexent is an oral NF-kB inhibitor and, unlike other DMD treatments such as Sarepta's Exondys 51, could potentially have been used in patients regardless of their underlying mutation.
But that dream is now dead. “Catabasis is stopping activities related to the development of edasalonexent including the ongoing GalaxyDMD open-label extension trial,” the biotech said in a statement.
On top of this, the dreaded external advisers are being drafted in “to explore and evaluate strategic options going forward.” This comes two years after the company slashed its staffers after the first 2017 failure of the drug and threw most of its weight behind edasalonexent.
Shares were in free-fall on the news, down 66%. At the end of last month, it had around $53 million, but nearly all of its bets were on edasalonexent. The only other asset listed as in its pipeline is a preclinical cystic fibrosis drug.
“We are deeply saddened and disappointed by the results of our Phase 3 PolarisDMD trial,” said Jill Milne, Ph.D., CEO of Catabasis.
“I want to sincerely thank all of the boys, their families and caregivers, investigators and the trial sites that participated in and enabled this program. The entire Catabasis team has worked tirelessly to find a treatment for this progressive disease. We hope that our data and work to date can be used to benefit ongoing and future research in DMD.”