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FDA Wants More Safety Info on Psych Drug, Intra-Cellular Shares Drop

Xconomy New York — 

Despite mixed data from clinical trials, Intra-Cellular Therapies plans to seek FDA approval for an experimental schizophrenia drug, its only therapy even close to market. But that plan got more difficult this morning as the company revealed the FDA has some questions about the drug’s safety, causing shares to slide to their lowest levels since the company went public three years ago.

Intra-Cellular (NASDAQ: ITCI) said that it plans to file for approval of lumateperone by mid-2018. But first, the FDA wants more information showing that long-term use of the New York company’s drug doesn’t lead to any safety problems. The FDA flagged a potential problem in a preclinical toxicology study of lumateperone in dogs, and Intra-Cellular has to answer the agency’s questions before it can move forward. On a conference call Monday morning, CEO Sharon Mates said the company will have responses for the agency “very shortly,” but didn’t provide any more specifics. “We’re going as fast as we can,” Mates said.

On the call, Intra-Cellular senior vice president of clinical development Kimberly Vanover said that in the study the FDA referenced animals were exposed to a much higher dose than Intra-Cellular has tested in humans. She added humans and dogs break down lumateperone differently. In humans, the drug is cleared from the body quickly. In dogs, a “substantial” amount of drug and drug-related material is retained for an extended period of time, she said. In human studies so far, the drug, Mates said, has shown a safety profile “similar to placebo.”

Intra-Cellular is gearing up to run a long-term safety study of lumateperone anyway, but Vanover said that the company doesn’t believe that it needs to complete a new trial before it can file for approval. “We believe the data we have in hand will address [this],” she said.

Intra-Cellular is making a risky bet. 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. Intra-Cellular plans to move forward and file for FDA approval anyway. 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. A number of psychiatric drugs to have tripped up in late-stage testing have been doomed for similar reasons, but Intra-Cellular still thinks, failed trial aside, it has accrued enough good data on lumateperone to bring to the FDA. “We believe our schizophrenia clinical development program collectively provides evidence for the efficacy and safety of lumateperone for the treatment of schizophrenia,” Mates said on today’s call.

In a statement, the company said the data it has accrued so far “does not preclude” the company from filing for approval. But today’s disclosure on potential safety issues adds more uncertainty as to whether lumateperone can make it to market. Shares of Intra-Cellular fell more than 36 percent, to $8.74 apiece, in early trading. Since Intra-Cellular went public at $17.50 per share in 2014, shares haven’t closed below $11.36. In a research note, Leerink Partners analyst Seamus Fernandez called the share move a “clear sign that investors are not confident that lumateperone will have a clear path for [an approval] filing.”