Shares of BioXcel Therapeutics (BTAI 1.82%) are trading lower on Thursday after the company reported somewhat disappointing data from a clinical trial for one of its leading pipeline candidates, BXCL501, on Wednesday evening. The biotech's stock was down by 13.3% as of 2:31 p.m. EDT.
BXCL501 is a thin sublingual film being developed for the treatment of opioid withdrawal symptoms, among other conditions. In a phase 1b/2 study, BXCL501 was generally safe and well tolerated with no serious adverse reaction reported, which was the study's primary safety endpoint. However, it is worth noting that treatment adverse events were relatively frequent for the two highest doses of BXCL501 (180 micrograms and 240 micgrograms), at least when compared to the placebo, which recorded no treatment adverse events.
One of the study's secondary endpoints was an improvement in retention rates, which is a key goal of opioid withdrawal treatment. The 120 microgram and 180 microgram dose groups showed 42% and 52% retention rates, respectively, six days after treatment with BXCL501, compared to a 24% retention rate for the placebo group. However, these observations were not statistically significant. These mixed results prompted Truist analyst Robyn Karnauskas to lower her price target for BioXcel stock to $127, down from $150, although the analyst kept a buy rating on the shares. Given that BioXcel is currently trading for $37.39, it is clear that Karnauskas still sees a significant upside for the biotech stock.
BXCL501 is also being developed as a treatment for acute agitation associated with psychiatric conditions such as schizophrenia and bipolar disorder. The therapy could generate more than a billion dollars in sales from these indications, provided it earns regulatory approval. In other words, despite the relatively unimpressive results from this early stage study of BXCL501 as a treatment for opioid withdrawal symptoms, the medicine could still have a bright future ahead.