What failure? Catabasis shares soar after execs tout open-label data, PhIII plans

Before reviewing Catabasis’ second attempt at making a good first impression with its Phase II study of the Duchenne muscular dystrophy drug edasalonexent (CAT-1004), it’s important to remember what happened the first time around.

It flopped — badly — at helping patients, as researchers reported in early February.  The drug failed to register a significant biomarker effect on muscle inflammation, looking very much like the placebo. And its stock $CATB was shredded.

Today, the biotech claimed that if you look at the extended, open-label data from the study now, you can tease out positive results. The 24 and 36-week data offer “clinically meaningful evidence that edasalonexent substantially slowed the progression of Duchenne muscular dystrophy.” And there’s nary a word about the stage B clinical failure in their statement.

Catabasis says that based on these results, they’re going into a pivotal Phase III trial.

That broadcast pushed up shares more than 50% this morning. But it didn’t last. By mid-day that gain had been pared down to a more modest 14%.

There’s a big wing of the biotech industry that doesn’t think much of mounting pivotal trials for drugs that fail Phase II — or of the companies that wait for new data to try and piece together a more positive picture. Particularly open-label data.

In DMD in particular, developers have been accused of all kinds of shenanigans, with Sarepta winning an approval for eteplirsen even though the label clearly states there’s no solid evidence of efficacy.

Catabasis and Sarepta and collaborating on their work in the field.

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VP Oncology Biology
Skyhawk Therapeutics Waltham, MA
Associate Director CMC
Elektroki Boston, MA
Director Process Development
Elektroki Boston, MA
Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT

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