- Catabasis Pharmaceuticals' drug for the rare disease Duchenne muscular dystrophy (DMD) missed its primary endpoint in the second portion of a mid-stage study, according to top-line results the company released Tuesday.
- Part B of the three-part MoveDMD study enrolled 31 boys ages four to seven with DMD who were then given either one of two doses of the drug in question, edasalonexent, or a placebo over a 12-week period. Results showed that while the drug had a solid safety profile, it did not lead to significantly improved muscle composition or reduced inflammation — the primary endpoints of the study.
- Catabasis said it will continue with Part C of the study, an open-label expansion test, with interim results expected during the second quarter and fuller readouts later this year.
Edasalonexent's failure is another let down in the DMD space, where only one drug — Sarepta's Exondys 51 (eteplirsen) — has gained approval from the Food and Drug Administration. Sarepta's win wasn't pretty either. Lengthy delays preceded the FDA's final decision, while the drug's effectiveness and the potential political motives behind its approval are still a point of controversy.
Catabasis now joins others who have had setbacks with their own DMD drugs. Back in July, the FDA shot down Santhera Pharmaceuticals' motion for accelerated approval of Raxone (idebenone). Three months later, PTC Therapeutics revealed the agency dismissed an appeal from the company requesting a review of Translarna (ataluren).
Meanwhile, BioMarin won't see an approval decision on Brineura (cerliponase alfa) until late April, after the FDA requested additional time to review new data added to the initial Biologics License Application.
In the second portion of its MoveDMD study, Catabasis tested its drug in two doses: 67mg/kg and 100 mg/kg, which were administered daily to their respective patient groups. The drug's effects on patient muscles were assessed through magnetic resonance imaging (MRI), the North Star Ambulatory Assessment (NSAA), as well as multiple "function tests" that measured how long it took a patient to walk 10 meters or go up four stairs.
While the lower dose had mixed results, boys receiving the higher one did outperform placebo-taking patients in the timed tests, though not significantly.
"We observed potential treatment-associated effects at 12 weeks in the 100 mg/kg/day treatment group, which we believe warrant further evaluation to see if the signals strengthen in the longer-term data from the ongoing open-label extension," Catabasis CEO Jill Milne said in a Jan. 31 statement. "Following additional data analysis from the open-label extension, we will determine the next steps for edasalonexent in DMD."