Bioxcel Therapeutics Inc. said a third-party audit of data from its Tranquility II phase III study came back clean, potentially positioning the firm to submit the statistically significant results in a supplemental NDA seeking approval of BXCL-501 in treating agitation associated with dementia in probable Alzheimer’s disease.
Although the highest dose of Bioxcel Therapeutics Inc.’s BXCL-501 hit the primary endpoint in the phase III Tranquility II trial for acute agitation in Alzheimer’s disease patients, shares fell dramatically on June 29 by 64% when investors learned the company received an FDA warning letter over infractions at a trial site.
Mixed phase III study results have Bioxcel Therapeutics Inc. moving ahead to complete the clinical trial while withstanding a hammering from investors. BXCL-501 (dexmedetomidine), a sublingual film being developed to treat bipolar disorders- or schizophrenia-associated agitation, produced clinically meaningful efficacy results in part 1 of the pivotal study with half of the approved dose, but the primary efficacy endpoint was not statistically significant at two hours (p=0.077). BXCL-501 separated from placebo at four hours (p=0.049).
Just two weeks after winning U.S. FDA approval for the first orally dissolving formulation of dexmedetomidine for agitation in schizophrenia and bipolar disorders, Bioxcel Therapeutics Inc. has secured commitments for up to $260 million to support the product’s commercial launch, slated for this quarter in addition to a planned European marketing application.
Drug "re-innovation" specialist Bioxcel Therapeutics Inc. is gearing up to discuss a pivotal phase III trial for BXCL-501 with the FDA after top-line results of a phase Ib study showed it reduced schizophrenia-associated agitation while calming trial participants without excessive sedation. H.C. Wainwright & Co. analyst Raghuram Selvaraju called the results "highly favorable" and suggested the drug, a sublingual-formulated version of dexmedetomidine, could reach market as soon as 2021.