NEW HAVEN, Conn., Nov. 12, 2020 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence to identify improved therapies in neuroscience and immuno-oncology, today announced its quarterly results for the third quarter ended September 30, 2020 and provided an update on key strategic and operational initiatives.

“Building on the successful data readout from the Phase 3 SERENITY trials earlier this year, we have continued to make substantial progress with BXCL501’s development as a treatment for agitation across neuropsychiatric conditions,” stated Vimal Mehta, Chief Executive Officer of BTI. “Recently, we completed our pre-NDA meeting with the FDA and have initiated rolling submission of the NDA. This is a key milestone for our neuroscience franchise, laying a strong foundation for multiple follow-on indications and a catalyst for the ongoing build of our commercial infrastructure. While the TRANQUILITY and RELEASE trials advance, we are preparing to initiate a Phase 2 trial for agitation associated with delirium. Together, we believe that these trials, along with our strong cash position, will help to support our long-term strategy of establishing BXCL501 as an innovative treatment for agitation regardless of the patient’s underlying diagnosis.”

Dr. Mehta continued, “We are also making excellent strides advancing our immuno-oncology program, with two combination therapy trials progressing well. Earlier this week, we presented encouraging preliminary efficacy and safety data at SITC from both the Phase 1b/2 trial of BXCL701 and KEYTRUDA^® for the treatment of advanced prostate cancer and the MD Anderson-led Phase 2 basket trial in advanced solid tumors. Across both trials, we have already seen promising signals of activity in numerous difficult-to-treat tumors.”

Third Quarter 2020 and Recent Highlights

BXCL501-Neuroscience Program

BXCL501 is an investigational, proprietary, orally dissolving, sublingual thin film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist, designed for the treatment of agitation and opioid withdrawal symptoms. The Company believes BXCL501 may directly target a causal agitation mechanism.

• Following the recently announced positive topline results from the Phase 3 SERENITY trials, last month, BTI completed a successful pre-NDA meeting with the FDA for BXCL501 for the acute treatment of agitation in patients with schizophrenia and bipolar disorders. The FDA agreed to a rolling review of the NDA and BTI has already submitted part of the application to the FDA, with plans to submit the complete application in the first quarter of 2021.
• The Company initiated the third dose cohort (90 mcg) in the TRANQUILITY study, a Phase 1b/2 trial of BXCL501 for the acute treatment of agitation associated with dementia. Based on the findings, the Company expects to report topline results in the fourth quarter of 2020, or, if needed, proceed to an additional dose cohort.
• The RELEASE study, a Phase 1b/2 trial of BXCL501 for the treatment of opioid withdrawal symptoms, is ongoing, with enrollment of the dose cohorts progressing well. The Company expects to report topline results from the study in the first quarter of 2021.
• In October, BTI received FDA clearance of its Investigational New Drug (“IND”) application for BXCL501 for the treatment of hospitalized patients with agitation associated with delirium, including COVID-19 patients. The Company plans to initiate a Phase 2 trial within the next several months.
• BTI recently analyzed topline data from a cross-over study in healthy volunteers comparing the bioavailability of BXCL501 administered sublingually with the film placed drug-side down under the tongue, compared to the film placed drug-side up under the tongue, and administered buccally (between the lower lip and the gum). It was determined that all three administrations of BXCL501 are bioequivalent and were rapidly absorbed into the systemic circulation. Also, drinking water starting at 15 minutes following sublingual administration did not have any affect on bioavailability.
• The Company was issued U.S. patent No. 10,792,24 on October 6, 2020, which covers film formulations containing Dex and methods of treating agitation using such film formulations. The patent is expected to extend intellectual property (“IP”) protection until 2039.

BXCL701-Immuno-Oncology Program

BXCL701 is an orally-delivered small molecule, innate immunity activator designed to inhibit dipeptidyl peptidase (DPP) 8/9 and block immune evasion by targeting Fibroblast Activation Protein (FAP). It has shown single agent activity in melanoma and safety has been evaluated in more than 700 healthy subjects and cancer patients.

• The Phase 2 portion of the Phase 1b/2 trial of BXCL701 in combination with pembrolizumab (KEYTRUDA^®) for treatment emergent Neuroendocrine Prostate Cancer (“tNEPC”) and castrate-resistant prostate cancer (“CRPC”) is advancing. Data from the 1b portion of this trial was presented recently at SITC, with an additional efficacy update planned.
• The MD Anderson-led Phase 2 open label basket trial evaluating the combination of BXCL701 and KEYTRUDA^® in patients with advanced solid tumors is progressing well, and has already moved to the stage-2 efficacy phase. Preliminary efficacy data on 14 patients (5 patients in the checkpoint naïve cohort and 9 patients in the checkpoint pretreated cohort) was presented earlier this week at SITC.
• The BXCL701 phase of the triple combination study of BXCL701, bempegaldesleukin (NKTR-214, Nektar Therapeutics, Inc.) and BAVENCIO^® (avelumab, Merck KGaA, Darmstadt, Germany and Pfizer) in second line pancreatic cancer was planned to initiate following Nektar and Pfizer’s Phase 1B dose-escalation trial of bempegaldesleukin and avelumab, which was delayed. All parties have agreed to discontinue activities on the triple combination study and instead reallocate resources to other studies and development programs. The parties terminated their clinical trial collaboration agreement, effective November 10, 2020.

Corporate Highlight

• In July 2020, the Company raised net proceeds of approximately $187 million in connection with its common stock offering. BTI believes that the proceeds from this offering, together with current reserves, provide the cash runway to fund key clinical, regulatory, operational, and commercial activities well into 2022.

Third Quarter 2020 Financial Results

BTI reported a net loss of $24.8 million for the third quarter of 2020 compared to a net loss of $9.0 million for the same period in 2019. The third quarter 2020 results include approximately $5.3 million in non-cash stock-based compensation compared to $0.8 million for the same period in 2019.

Research and development expenses were $16.3 million for the third quarter of 2020, compared to $7.1 million for the same period in 2019. The increase was primarily attributable to increased clinical trial costs and professional research related to the acceleration of the Company’s research and development activities, primarily related to its SERENITY I and II clinical trials as well increased costs associated with its TRANQUILITY and RELEASE clinical trials for BXCL501. These amounts were partially offset by reduced costs related to the BXCL701 pancreatic cancer trial.

Personnel costs also increased, primarily related to the growth of BTI’s clinical team as the Company continues to expand its clinical programs, and in preparation of the potential commercial launch of BXCL501 in the U.S. Non-cash stock-based compensation also increased as result of the additional personnel combined with increased grant date fair values arising from higher market prices of the Company’s common stock.

General and administrative expenses were $8.5 million for the third quarter of 2020, compared to $2.0 million for the same period in 2019. The increase was primarily due to increased non-cash stock-based compensation and personnel costs related to the growth of BTI’s operations combined with increased grant date fair values arising from higher market prices of the Company’s common stock. Professional fees also increased which is primarily attributable to increased corporate legal and investor relations fees combined with increased insurance premiums.

Total operating expenses for the third quarter of 2020 were approximately $24.8 million, compared to total operating expenses of approximately $9.1 million for the same period in 2019.

As of September 30, 2020, cash and cash equivalents totaled approximately $233.4 million.

About BioXcel Therapeutics, Inc.:

BioXcel Therapeutics, Inc. is a clinical stage biopharmaceutical company focused on drug development that utilizes artificial intelligence to identify improved therapies in neuroscience and immuno-oncology. BTI's drug re-innovation approach leverages existing approved drugs and/or clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic indices. BTI's two most advanced clinical development programs are BXCL501, an investigational, proprietary, orally dissolving, sublingual thin film formulation of dexmedetomidine for the treatment of agitation and opioid withdrawal symptoms, and BXCL701, an investigational, orally administered, systemic innate immunity activator in development for the treatment of aggressive forms of prostate cancer and advanced solid tumors that are refractory or treatment naïve to checkpoint inhibitors. For more information, please visit www.bioxceltherapeutics.com.