The FDA has just lowered a key barrier standing between Intra-Cellular Therapies and potential approval of an experimental schizophrenia drug. Now the question is whether the mixed set of data the company has accrued to support the program will be good enough for regulators.
Intra-Cellular (NASDAQ: ITCI) said the FDA has finished looking over responses the New York company provided about a preclinical toxicology study of its drug, lumateperone, in dogs. The FDA was concerned that some potential safety hazards seen in that study might predict similar issues in humans who take the drug chronically, but Intra-Cellular was able to convince the regulators otherwise. Still, the company will add new monitoring measures to an ongoing, long-term safety study it has been ramping up.
The news clears Intra-Cellular to file for approval for lumateperone, and it aims to do so by mid-2018. Shares climbed 33 percent in pre-market trading Wednesday morning on the news, but the future is still uncertain for lumateperone.
Lumatepereone, a small molecule drug meant to act on serotonin, dopamine, and glutamate levels in the brain, succeeded in one Phase 3 trial in schizophrenia but failed a second study. The company blamed the failed study on an “unusually high placebo response at certain sites…disproportionately affected the trial results,” leading to a different outcome than it has seen in previous trials of lumateperone, and plans to file for FDA approval anyway.
A number of psychiatric drugs tripped up in late-stage testing—among them a drug from now-shuttered Forum Pharmaceuticals, of Waltham, MA—have been doomed for similar reasons, but Intra-Cellular still thinks, failed trial aside, it has enough good data on lumateperone to win FDA approval. The agency’s response so far, according to Intra-Cellular, has been that the data accrued “does not preclude” the company from filing for approval. Time will tell whether the gamble pays off.
“We are very pleased with the outcome of our discussions with the FDA and look forward to progressing our schizophrenia program,” Intra-Cellular CEO and chairman Sharon Mates said in a statement.