Catabasis Pharmaceuticals Inc. (NASDAQ: CATB) saw its shares skyrocket on Tuesday after the firm announced new results from its Duchene muscular dystrophy (DMD) study with edasalonexent. Overall the trial showed consistent improvements in all assessments of muscle function after more than a year of edasalonexent.
Additionally, supportive changes in non-effort-based measures of muscle health were seen, with significant longer-term reductions in muscle enzymes and C-reactive protein, supporting the durability of edasalonexent treatment effects.
At the same time, edasalonexent continued to be well tolerated with no safety signals observed in the trial. More data will be presented on Saturday, February 17, at the XVI International Conference on Duchenne and Becker Muscular Dystrophy in Italy.
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 thrilled to see this preservation of muscle function and substantial slowing of disease progression in boys following more than a year of edasalonexent treatment. This effect has the potential to be extremely impactful for boys affected by Duchenne. Building on the results previously reported for edasalonexent treatment in patients up to 36 weeks, these new data at 48 and 60 weeks show that edasalonexent continued to slow progression of the disease. We look forward to advancing edasalonexent in a single global Phase 3 trial later this year with the goal of improving the quality and length of life for those affected by Duchenne.
Excluding Tuesday’s move, Catabasis had underperformed the broad markets with its stock down 16% year to date. Over the past 52 weeks, the stock was only up 2.5%.
Shares of Catabasis were last seen up about 60% at $2.00 on Tuesday, with a consensus analyst price target of $4.50 and a 52-week range of $1.09 to $3.78.