FDA casts doubts on Intra-Cellular safety data
- In a discussion with the Food and Drug Administration over potential data for submission of lumateperone for a New Drug Application (NDA), the regulator has raised questions about non-clinical animal toxicity studies and has requested additional information to rule out any potential issues over long-term exposure in humans.
- More positively for the company, the FDA has confirmed to Intra-Cellular Therapies that the results of Study ITI-007-302, where the lumateperone results did not show an advantage over placebo, do not preclude the company from submitting an NDA based on the efficacy studies conducted to date.
- The FDA's feedback chopped 24% of the company's value yesterday, cutting the share price down to $10.49; it fell a further 4.7% overnight, dropping to $10.00.
Despite Intra-Cellular Therapies' protestations that the pre-clinical results, which showed some toxicity in canine safety studies, have no relevance to the human use of lumateperone and are not indicative of risk because the human and dog metabolism of the drug differs widely, the Food and Drug Administration request for more data has knocked the already struggling company's value back.
This isn't the first time that Intra-Cellular has seen its share tumble on lumateperone study results. Data from a late-stage study of the drug, previously known as ITI-007, showed that neither dose of lumateperone studied proved better than placebo in patients with schizophrenia. This was blamed on an "an unusually high placebo response at certain sites," however, the comparator Risperdal (risperidone) arm seemed to show higher efficacy than placebo, which rather muddies the waters. At that point, Intra-Cellular's shares fell by more than $26 to just below $16.
Intra-Cellular is preparing its response for the FDA, and plans to submit it in the very near term, but wouldn't expand any further on the timing in the conference call. "The FDA requires additional information, but we believe that we have data in hand that will address the questions. We don't think any further studies are warranted," said Sharon Mates, chairman, president and CEO.
Following the resolution of the questions, Intra-Cellular will prepare an NDA based on the efficacy studies carried out to date.
"Our submission timeline has always depended on discussions with the FDA. We now expect to submit a New Drug Application by mid-2018, and will keep investors updated," says Sharon Mates, president and CEO.
Lumateperone is also in development for bipolar disorder and depression, and the ongoing studies should complete enrollment around mid-or late-2018. The FDA discussions should not have an impact on these indications.
Schizophrenia is a very competitive market place to enter, particularly with risperidone available as a generic, and while there is always room for drugs that meet unmet needs, lumateperone will have to show a clinical improvement against existing drugs to gain a foothold.
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