What happened
Shares of BioXcel Therapeutics (BTAI -1.54%) were down more than 19% early Thursday afternoon after the biotech company released the first part of a phase 3 trial for the therapy BXCL501 (dexmedetomidine) to treat acute agitation in bipolar disorders or schizophrenia.
So what
The study was based on at-home use of the therapy on patients by their caregivers. The drug, marketed under the name Igalmi (dexmedetomidine), was approved by the Food and Drug Administration (FDA) last April in a sublingual film to treat bipolar and schizophrenia patients in medical settings, but the additional indication was for at-home use. The part of the study that worried investors is the therapy didn't meet its primary endpoint. Measured at two hours after the dose, the change in Positive and Negative Syndrome Scale, Excited Component, though it differed from that with placebo, did not reach statistical significance, the company said.
The drug is also in trials to treat acute agitation in major depressive disorder and Alzheimer's disease.
Now what
The efficacy of the drug has already been proven, so the disappointing trial isn't that big a deal. BioXcel said it will continue the study with the hope to get approval for the additional at-home indication for BXCL501. The key part to figure out will be the safety profile of the allowed dose. It's important for patients, though. BioXcel said there are roughly 39 million annual incidents of agitation connected with schizophrenia in adults in the U.S., and 60% of them occur outside of a medical setting. An additional approval would likely more than double the drug's sales, BioXcel said.
In the first quarter, the company only had $206,000 in revenue, but Igalmi's sales are expected to grow. The company also lost $52.8 million in the quarter and had $165.5 million in cash, enough to fund operations into 2025.
