Intra-Cellular Therapeutics is digging in deep to defend the troubling late-stage data its researchers gathered on a lead drug for schizophrenia. But now the FDA has raised questions about tox issues seen in an animal study, and the biotech execs appear to have lost another big group of once-faithful investors.
The biotech’s share price $ITCI was already subjected to a severe beating last fall, when company execs tried to explain why their second Phase III study for lumateperone (ITI-007) flopped badly against a placebo and an active control arm. Part of their case centered on the improved safety profile they touted at the time.
Now the biotech says that in meetings with the FDA, regulators raised a red flag on a toxicity issue seen in an animal safety study. The biotech is planning a long-term safety study in humans, but says the FDA may put that on clinical hold if the company can’t satisfy their safety queries.
The company’s share price took a fresh plunge, sliding 37%. The stock is down 80% from its last close before the biotech revealed its Phase III failure last September.
From their statement:
We are preparing responses to the FDA’s request for additional information and intend to proceed with our long-term safety study of lumateperone in patients with schizophrenia. If the FDA deems our responses regarding the nonclinical findings to be sufficient, we intend to submit an NDA for lumateperone for the treatment of schizophrenia by mid-year 2018 supported by the efficacy studies we have conducted to date. If the FDA deems our responses insufficient, they may place our long-term safety study on a clinical hold. The results of the long-term safety study will be required to support an NDA approval for a chronic condition such as schizophrenia.
Intra-Cellular’s drug actually did just a tad worse than placebo, and significantly worse than the risperidone control arm in its second Phase III. “ITI-007 60 mg and 20 mg demonstrated a change from baseline on the PANSS total score of -14.6 points and -15.0 points respectively versus a -15.1 point change in placebo,” the company reported. “Risperidone, the active control, demonstrated a change from baseline on the PANSS total score of -20.5 points.”
But the company says an earlier, positive Phase III combined with other positive data provided a sufficient basis for an NDA. As for the safety data from a dog study, the company explained that the drug is metabolized differently in dogs and humans. But the FDA isn’t quite satisfied with that.
Right now, that’s an argument that Intra-Cellular execs are finding a tough slog.
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