Drug Development

Catabasis shares are shredded after lead drug flops in PhII Duchenne MD study

Catabasis’ lead drug just flopped in a Phase II study for Duchenne muscular dystrophy, eviscerating its stock after yet another clinical setback raises questions about its competence and direction.

The drug, CAT-1004 (edasalonexent), failed to register a significant biomarker effect on muscle inflammation, looking very much like the placebo.

The edasalonexent 100 mg/kg/day treatment group “consistently showed numerical improvement versus placebo across multiple measures,” Catabasis reported, but none of them were statistically significant.

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%, eviscerating its market cap. The biotech also noted a setback last summer, though it wasn’t punished as badly.

Catabasis suffered its first big setback in the clinic last summer, writing off its midstage program for CAT-2054 in hypercholesterolemia after its trial flopped and sent its shares into a tailspin.

The news is also an issue for Sarepta, which agreed to test Exondys 51 as a combo drug with CAT-1004.

“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. 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 edasalonexent in DMD,” said Jill C. Milne, Chief Executive Officer of Catabasis. “We are enormously grateful to the boys and the families involved in the MoveDMD trial as well as the clinical trial site staff and patient groups who are making this trial possible.”

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