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Another micro-cap biotech has flunked a pivotal test for a regenerative cell therapy

Marc H. Hedrick

Today the failure belongs to Cytori Therapeutics $CYTX which says that the cells it extracted from patients’ fat failed to hit either the primary or secondary endpoints in a Phase III comparison with placebo in treating scleroderma. Most of the endpoint scores were far off from any indication of success.

Shares of Cytori were eviscerated on the news, dropping 60% into penny stock land.

The San Diego-based biotech, though, was able to extract some material to back its therapy Habeo, even though investors were not in any way reassured.

Looking just at a more severe form of the disease, diffuse cutaneous scleroderma, the biotech says it could see “improvements in the Cochin Hand Function Score and the Health Assessment Questionnaire-Disability Index (HAQ-DI), a measure of functional disability and an important secondary endpoint, met or exceeded the published criteria for minimally important clinical differences in these measures (6.5 points for Cochin1, 0.22 points for HAQ-DI2).”

Subgroup analysis, though, is a tricky field, particularly as the pivotal program included only 51 patients in that particular subgroup. On its website, Cytori also acknowledges that it is uncertain of the mechanism of action. Nevertheless, Cytori says it plans to take the data it has and show it to the FDA before determining next steps, if any.

Scleroderma is a connective tissue disease characterized by a hardening of the skin that can cripple hand function. About 300,000 Americans suffer from the disease.

“We are disappointed that the study missed the primary and secondary endpoints. However, we are very encouraged by the trends toward improved hand function and scleroderma-related health status in patients with diffuse cutaneous scleroderma,” said Cytori CEO Marc H. Hedrick.

Cell therapies like this were once the vogue in biotech, but a long slate of nonstarters has placed a cloud over the field. Cytori started today with a market cap of $36 million.


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RAPS Regulatory Convergence 2017