The FDA really, really, really doesn’t like Chiasma’s Phase III study for its lead drug, octreotide. And the biotech isn’t making much headway in finding a way around the regulatory roadblock without satisfying the agency’s demand for a new, far better designed Phase III trial.
Regulators at the agency batted down the acromegaly drug, dubbed MyCapssa, last April after concluding that the biotech simply didn’t prove that the pill works with the single-arm Phase III on offer. The oral drug is intended to replace the injected therapies currently used to treat the rare hormone ailment, in which the pituitary gland generates too much hormone. And regulators don’t appear to be too receptive to any alternative plans that Chiasma has in mind. From the biotech’s 10Q:
In the End of Review meeting minutes, the FDA reiterated its strong recommendation for a randomized, double-blind and controlled trial, and introduced the concept of a placebo control as a design element that could address some of the FDA’s concerns. While we acknowledge this feedback, we continue to evaluate various potential paths forward, including a determination as to whether we can produce data sufficient to satisfy the FDA of the efficacy and safety of Mycapssa in adult patients with acromegaly. The FDA stated that it considers pathways alternative to its recommendations to be less ideal and ultimately more risky to our efforts to secure approval of our NDA for octreotide capsules in acromegaly. The FDA strongly recommended that we work with the FDA to reach a common understanding of expectations prior to initiating and executing any alternative plans.
Chiasma — a U.S./Israeli hybrid with corporate headquarters in Waltham, MA — does have another Phase III running for the planned European application, but it’s another open-label study that also doesn’t meet the U.S. agency’s demands. Their drug is a mimic of somatostatin.
Interestingly, Chiasma had good cause to wonder how the FDA would handle the NDA before it sent it in. In its Q2 statement, the biotech acknowledged that Roche had bowed out of its collaboration after conferring with regulators about the study. In a subsequent meeting with the FDA:
The FDA also informed us that, in its view, a single-arm study was not as informative as a controlled study such as an active control trial using a non-inferiority design, and that the interpretability of the efficacy findings we submitted in our NDA from our single-arm study, and whether these findings would be robust enough to warrant approval, would be review issues as the agency evaluated our NDA.
The bottom line is that Chiasma — run by longtime biotech vet Mark Leuchtenberger — is caught with dwindling resources at a time its stock has been beaten down to a fraction of its 12-month high. The company laid off about a third of its staff after the rejection and has enough money to make it through 2017. But it also acknowledges that it doesn’t have enough cash for a new study that would meet the FDA’s demands.
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