Revisiting BioXcel Therapeutics (BTAI)

(RTTNews) - Shares of BioXcel Therapeutics Inc. (BTAI) have gained nearly 86 percent over the last 1 month as the Company gears up for reporting trial results of one of its most advanced clinical development programs BXCL501.

BXCL501 is an investigational, sublingual thin film formulation of Dexmedetomidine for the acute treatment of agitation resulting from neuropsychiatric disorders like schizophrenia, bipolar disorder, and dementia.

The Company initiated the SERENITY program, comprising of two phase III studies of BXCL501 for the acute treatment of agitation in patients with schizophrenia and bipolar disorder, 18 to 75 years of age, dubbed SERENITY I and SERENITY II, last December.

SERENITY I is evaluating BXCL501 at 120 micrograms, 180 micrograms or placebo in patients with agitation associated with schizophrenia, while SERENITY II is evaluating patients with agitation associated with bipolar disorder.

The primary endpoint of the trials is reducing acute agitation measured by the Positive and Negative Syndrome Scale, examining the Excited Component ("PEC") change from baseline compared to placebo. A key secondary endpoint includes determining the earliest time where an effect on agitation is apparent as measured by the change from baseline in PEC total score.

Despite the current situation with COVID-19, the enrollment of the SERENITY studies is on track, according to the Company, and topline results from the studies are expected in the middle of this year.

BXCL501 is also being tested in a phase Ib/II study for the acute treatment of agitation in patients with dementia, dubbed TRANQUILITY. Initiated in January of this year, the TRANQUILITY study is designed to assess agitation as measured by the Pittsburgh Agitation Scale, a validated clinical instrument, as well as improvement in the modified Cohen Mansfield Agitation Inventory and Positive and Negative Syndrome Scale, Excitatory Component.

The Company expects to report data from the TRANQUILITY study also in mid-2020.

A phase II study initiated in February this year by researchers at Yale University to measure biomarkers associated with agitation in patients with schizophrenia and the response to treatment with BXCL501 is underway, with topline results expected this quarter (Q2, 2020).

The Company has also received FDA clearance to initiate a phase Ib/II trial for the treatment of opioid withdrawal symptoms, a potential fourth indication for BXCL501.

Also in the pipeline is BXCL701, an immune-oncology compound, which works by inhibiting dipeptidyl peptidase (DPP) 8/9 and blocking immune evasion by targeting Fibroblast Activation Protein (FAP).

-- BXCL701 in combination with Merck's KEYTRUDA is under a phase Ib/II trial for the treatment of neuroendocrine prostate cancer (tNEPC). The initial data readout from this study is expected in the first half of this year.

-- A triple combination of BXCL701, bempegaldesleukin and BAVENCIO is under a phase Ib/II study in pancreatic cancer.

-- A phase II study evaluating the combination of BXCL701 and KEYTRUDA in patients with advanced solid cancers, which is being conducted at MD Anderson, is also ongoing.

Cash position

The Company, which ended the year 2019 with cash and cash equivalents of approximately $32.4 million, raised net proceeds of $60 million in a stock offering in February of this year.

BioXcel expects the cash on hand to be sufficient to fund its operations into 2021.

BTAI was trading around $7 when we first alerted readers to this stock on March 8, 2019.

In the last 1 year, the stock has traded in a range of $3.76 TO $43.63. BTAI closed yesterday's trading at $27.50, up 14.97%.

Related Reading BioXcel Therapeutics' (BTAI) Journey From $7 To $41 In Less Than A Year