Catabasis says Phase 3 trial results for its muscular dystrophy treatment Edasalonexent is expected in the 2Q of 2020

Catabasis Pharmaceuticals Inc (NASDAQ:CATB) said Wednesday that a pivotal Phase 3 trial will offer top-line results for its Duchenne muscular dystrophy treatment Edasalonexent in the second quarter of 2020.

The Cambridge, Massachusetts-based clinical-stage biopharma company shared the broad design of the PolarisDMD Phase 3 trial for Edasalonexent with the World Muscle Society Congress in Mendoza, Argentina.

The biotech’s lead product Edasalonexent has completed Phase II clinical trial for the treatment of Duchenne muscular dystrophy (DMD). Consistent improvements in all assessments of muscle function were observed after more than a year of treatment compared to the rate of change in the pre-specified control period.

DMD is a rare muscle-wasting disease that almost exclusively affects only young boys, for which there is currently no known cure.

“The PolarisDMD trial is expected to include approximately 40 clinical trial sites globally. The Phase 3 PolarisDMD trial is a one-year, randomized, double-blind, placebo-controlled trial,” said the company.

Catabasis plans to enroll 125 patients aged between 4 to 7 regardless of mutation type who have not been on steroids for at least 6 months.

“Top-line results from the Phase 3 PolarisDMD trial are expected in the second quarter of 2020,” said the biotech.

Shares of the biotech rose 6% in premarket trade, only to tumble nearly 8% to $0.78 in afternoon trading.

Catabasis also reported data Wednesday supporting the utility of magnetic resonance imaging (MRI T2) as a marker to predict disease progression in boys with Duchenne muscular dystrophy.

READ: Catabasis surges after successful mid-stage trial of its DMD treatment

Joanne Donovan, chief medical officer of Catabasis Pharmaceuticals, said the potential of MRI T2 to predict diseases progression and potential treatment benefits would hopefully lead to “improved” clinical management of boys living with the disease.

“This represents a promising step forward to better understand MRI endpoints and their utility in Duchenne to support our work to bring hope and life changing therapies to those affected by Duchenne,” said Donovan.

“We are excited to have recently initiated our Phase 3 PolarisDMD trial for edasalonexent based on the positive results observed in the MoveDMD trial.”

The utility of MRI T2 as a marker to predict disease progression in DMD was assessed in the ImagingDMD natural history database of more than 150 boys.

In April, Catabasis reported new positive magnetic resonance results showing slowed disease progression in boys with DMD in the MoveDMD trial through 48 weeks of edasalonexent treatment.

Contact Uttara Choudhury at [email protected]

Follow her on Twitter: @UttaraProactive 

Story by ProactiveInvestors