RARE Daily

Catabasis Shares Tumble as DMD Drug Fails in Late-Stage Study

October 27, 2020

Rare Daily Staff

Catabasis Pharmaceuticals said the phase 3 PolarisDMD trial of its experimental drug edasalonexent failed to meet its primary and secondary endpoints in patients with the progressive neuromuscular disease Duchenne muscular dystrophy.

Catabasis is stopping activities related to the development of edasalonexent including the ongoing GalaxyDMD open-label extension trial. The company plans to work with external advisors to explore and evaluate strategic options going forward.

Shares of Catabasis were down as much as 70 percent in intraday trading Tuesday to $1.625 from a previous close at $5.36 on the news. The primary endpoint in the study was a change from baseline in the North Star Ambulatory Assessment (NSAA) over one year of edasalonexent compared to placebo. The secondary endpoint of timed function tests (time to stand, 10-meter walk/run and 4-stair climb) also did not show statistically significant improvements.

Edasalonexent was observed to be generally safe and well tolerated in this trial.

Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by progressive muscle deterioration and weakness that primarily affects boys with symptoms beginning as early as three years of age. It is the most common type of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Progressive muscle weakness in the lower limbs spreads to the arms, neck and other areas of the body. The condition is universally fatal, and death usually occurs before the age of 30 generally due to respiratory or cardiac failure. DMD occurs in about one out of every 3,600 male infants worldwide.

Edasalonexent is an experimental small molecule that was being developed as a treatment for DMD, regardless of the underlying mutation driving the disease. Edasalonexent inhibits NF-kB, a protein that is activated in DMD and drives inflammation and fibrosis, muscle degeneration, and suppresses muscle regeneration.

The phase 3 trial was a one-year placebo-controlled trial designed to evaluate the safety and efficacy of edasalonexent in boys ages 4-7 (up to eighth birthday) with DMD. The global trial enrolled 131 boys across eight countries, with any mutation type, who were not on steroids.

Data from the PolarisDMD trial will be further analyzed and are expected to be presented at an upcoming scientific conference, and published.

The company expects to report Q3 2020 financials in November of 2020. As of September 30, 2020, Catabasis had cash and cash equivalents of approximately $52.9 million.

“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,” said Jill Milne, CEO of Catabasis. “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.”

Photo: Jill Milne, CEO of Catabasis.


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