February 1, 2017
By Mark Terry, BioSpace.com Breaking News Staff
Cambridge, Mass.-based Catabasis Pharmaceuticals took a major dive after it announced the failure of its edasalonexent (CAT-1004) for Duchenne muscular dystrophy (DMD) drug. Shares are currently trading at $1.22, down from $4.04 yesterday.
DMD is a muscle wasting disease caused by mutations in the dystrophin gene. It is a progressive disease that usually causes death in early adulthood, with serious complications that include heart or respiratory-related problems. It mostly affects boys, about 1 in every 3,500 or 5,000 male children.
Catabasis announced top-line safety and efficacy results for Part B of the MoveDMD trial of edasolonexent. The objective was to determine the effects of the drug using magnetic resonance imaging (MRI) T2 as a biomarker at 12 weeks. The primary efficacy end point was average change from baseline to week 12. There was no particular change compared to placebo.
The company also said that “The edasolonexent 100 mg/kg/day treatment group consistently showed numerical improvement versus placebo across multiple measures although the changes were not statistically significant.”
John Carroll, writing for Endpoints News, said, “The Cambridge, MA-based biotech also tried to reassure investors with some inconclusive signs of drug activity, but investors weren’t in an agreeable mood. The company’s stock swiftly plunged 73 percent, eviscerating its market cap. The biotech also noted a setback last summer, though it wasn’t punished as badly.”
That was on June 7, 2016, when the company announced that its CAT-2054 for hypercholesterolemia (high cholesterol) failed to meet its primary end point, reduction in LDL-cholesterol from baseline. That announcement caused the stock to drop from $7.12 on June 6, 2016 to $3.54 on July 5, 2016.
In addition, the company announced today that Rick Modi, its chief business officer, resigned.
It also creates some doubts as to a planned trial with Sarepta Therapeutics ' DMD drug, Exondys 51, as a combination treatment with CAT-1004. Sarepta currently has the only approved drug for DMD, although there’s quite a bit of debate among experts and within the U.S. Food and Drug Administration about just how effective it is. It underwent a dramatic rollercoaster ride of an approval process that took most of last year.
“Although we did not meet the MRI T2 composite end point, the continued safety, tolerability and plasma exposure data in Part B of the MoveDMD trial are reassuring,” said Jill Milne, Catabasis’ chief executive officer, in a statement. “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. Following additional data analysis from the open-label extension, we will determine the next steps for adasalonexent in DMD.”
Edasalonexent is an oral drug that inhibits NF-kB, a protein activated in DMD and that causes inflammation and fibrosis, muscle degeneration, and suppresses muscle regeneration. In animal models, it showed beneficial effects. The FDA had granted it orphan drug, fast track and rare pediatric disease designations. The European Commission also granted it orphan medicinal product designation.
“The top-line data results from Part B of the MoveDMD trial provide us with an early snapshot of the effects of edasalonexent in boys with DMD over a 12-week period,” said Richard Finkel, division chief, Division of Neurology, Department of Pediatrics at Nemours Children’s Health System and principal investigator of the study, in a statement. “Continuing the open-label extension of the MoveDMD trial will allow us to further evaluate the potential for edasalonexent to provide benefit in DMD. The unmet medical need in Duchenne is profound and potential therapies that benefit all patients are needed.”
