Catabasis Pharmaceuticals Inc. (NASDAQ: CATB) shares continued their incredible climb after the firm announced positive efficacy results from its MoveDMD trial in Duchenne muscular dystrophy (DMD). These results were presented in a late-breaking session at the World Muscle Society Conference in France.
As we noted in a review of catalysts coming in October that Catabasis was due for results, along with Sarepta Therapeutics (NASDAQ: SRPT), whose results are expected Thursday. There are a few other catalysts coming out this month that could see significant moves as well.
According to the trial results, across all key assessments of muscle function, improvements were observed in the rate of decline after 24 and 36 weeks of oral 100 mg/kg/day edasalonexent treatment compared to the rate of change in the control period for boys prior to receiving edasalonexent treatment. These results demonstrated meaningful evidence that edasalonexent substantially slowed the progression of DMD.
Apart from the main efficacy results, supportive changes in measures of muscle health were seen, consistent with positive edasalonexent treatment effects. Muscle enzymes significantly decreased compared to baseline at 12 weeks and later time points and lower leg muscle MRI T2 rate of change was significantly improved in comparison to progression during the control period. Edasalonexent continued to be well tolerated with no safety signals observed in the trial.
Looking ahead, Catabasis plans to initiate a single global Phase 3 trial with edasalonexent in patients with DMD regardless of mutation type in the first half of 2018, with top-line results expected in 2020.
Jill C. Milne, Ph.D., CEO of Catabasis, commented:
We are extremely excited to see edasalonexent change the trajectory of disease in the MoveDMD trial with substantially slowed disease progression. Boys treated with edasalonexent stabilized; they experienced meaningful improvements in muscle function compared to the rates of change observed during the control period. Importantly, other supportive positive measures of muscle health were observed. We look forward to advancing edasalonexent as a disease-modifying therapy in a single Phase 3 pivotal trial as soon as possible with the goal of providing a meaningful impact on disease progression for all boys affected by Duchenne.
Shares of Catabasis were last seen up 22% at $3.46, with a consensus analyst price target of $4.10 and a 52-week range of $1.08 to $5.74.
Sarepta shares were trading at $49.89. The stock has a 52-week range of $26.26 to $63.03 and a consensus price target of $67.40.