On May 7, 2019 BioXcel Therapeutics, Inc. ("BTI" or "Company") (Nasdaq: BTAI), reported its quarterly results for the first quarter ended March 31, 2019 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, MAY 7, 2019, View Source [SID1234535818]). BTI is a clinical-stage biopharmaceutical development company utilizing novel artificial intelligence approaches to identify the next wave of medicines across neuroscience and immuno-oncology.

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First Quarter 2019 and Recent Highlights:

(BXCL501)- Neuroscience Program-

·Completed dosing of multiple cohorts in Phase 1 pharmacokinetic (bioavailability) and safety study of BXCL501; data announcement expected in coming weeks

·Finalized formulation development and transitioned to automated process for manufacturing of BXCL501 thin film formulation for pivotal study

· Announced proof-of-concept data demonstrating high response rates from three independent Phase 1 studies of intravenously administered dexmedetomidine (Dex) for acute treatment of agitation in patients with schizophrenia, Alzheimer’s disease / dementia and opioid withdrawal symptoms

(BXCL701)- Immuno-Oncology Program-

·Filed investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) on proposed clinical trial of BXCL701, NKTR-214 and avelumab triple combination for treatment of pancreatic cancer along with our partners Pfizer and Merck KGaA, Darmstadt, Germany, and Nektar Therapeutics

·Filed a clinical trial application (CTA) with U.K. health authorities to advance global development of BXCL701 and pembrolizumab (Keytruda) in neuroendocrine prostate cancer (tNEPC)

· Multiple sites opened for tNEPC clinical trial and two sites selected for BXCL701 proof of mechanism study for previously opened INDs

·Presented positive preclinical data on combination of BXCL701 with OX40 agonist at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, showcasing synergistic anti-tumor activity and survival benefit in tumor models

Dr. Vimal Mehta, Chief Executive Officer of BTI said, "During the first quarter of 2019 a number of positive initiatives continued to drive the ongoing clinical advancement of our lead programs, BXCL501 and BXL701."

"We anticipate reporting data from our first-in-human pharmacokinetic (bioavailability) and safety study of BXCL501 in the coming weeks. The dosing of multiple cohorts has been successfully completed. We believe that these results will help lay the groundwork for launching future registration studies as well as supporting BTI’s first New Drug Application (NDA) expected to be filed with the FDA in 2020. Additionally, in order to support the Phase 2/3 registration studies planned to begin in second half of 2019, the process development for BXCL501 sublingual thin film has now been transitioned to an automated robotic aided platform. We intend to employ an automated and scalable manufacturing process for clinical and commercial supply. BXCL501 has demonstrated promising potential with intravenously administered Dex in Phase 1 trials for the acute treatment of agitation in patients with schizophrenia, Alzheimer’s disease/ dementia and opioid withdrawal symptoms, where new effective therapies are desperately needed. We continue to believe that BXCL501 has the potential to be applied to a wide number of neuropsychiatric disorders and look forward to exploring opportunities as we advance development in our initial target indications.

We are pleased to report that we have filed an IND with the FDA for the proposed clinical trial of BXCL701, NKTR-214 and avelumab triple combination for treatment of pancreatic cancer with our partners Pfizer and Merck KGaA and Nektar. The triple combination therapy can potentially be effective by activating both the innate and adaptive immunity in pancreatic cancer patients. We are diligently working with our partners to progress the development of this triple combination therapy. Additionally, our ongoing Phase 1b/2 US study of BXCL701 and pembrolizumab in tNEPC is open for enrollment in multiple clinical trial sites. With the goal of expanding the clinical development of this combination globally, we have filed a CTA with U.K. health authorities. Additionally, we have selected two sites for BXCL701 proof of mechanism study in pancreatic cancer patients.

In March of this year, we presented positive findings at the AACR (Free AACR Whitepaper) Annual Meeting from a preclinical study evaluating the combination of BXCL701 with an OX40 agonist. The findings demonstrated that the combination significantly elevated anti-cancer activity in tumor models compared to the control. These results also provide important validation on BXCL701’s ability to stimulate both innate and adaptive immunity and we expect to further investigate the applicability of this combination."

Dr. Mehta concluded, "We continued to strengthen our management team with the appointment of industry veteran, Dr. Pascal Borderies, as the Vice President, Commercial Development and Medical Affairs who is responsible for designing and executing BTI’s global commercialization and medical affairs strategy, including sales and marketing efforts. We remain intently focused on progressing our assets through clinical development throughout the remainder of the year. We believe our sound strategy, highly skilled leadership team and solid pipeline candidates make us well-positioned for continued growth in 2019 and beyond."

First Quarter 2019 Financial Results

BTI reported a net loss of $7.2 million for the first quarter of 2019, compared to a net loss of $4.3 million for the same period in 2018.

Research and development expenses were $5.6 million for the first quarter of 2019, as compared to $2.9 million for the same period in 2018. The increase was primarily due to a ramp-up of research and development costs, along with increased personnel expenses associated with BTI’s two main drug candidates.

General and administrative expenses were $1.7 million for the first quarter of 2019, as compared to $1.3 million for the same period in 2018. The increase was primarily due to additional payroll and payroll-related expenses and costs associated with operating as a public company.

These results include approximately $682,000 in non-cash share -based compensation charges.

As of March 31, 2019, cash and cash equivalents totaled $36.3 million.

Conference Call:

BTI will host a conference call and webcast today at 8:30 a.m. ET. To access the call please dial (888) 394-8218 (domestic) and (323) 794-2588 (international) and provide the passcode 9482739. A live webcast of the call will be available on the Investors sections of the BTI website at www.bioxceltherapeutics.com. The archived webcast will be available through June 7, 2019.

Upcoming investor conferences:

· UBS Global Healthcare Conference – May 20-22, 2019, New York City

· BXCL501 Investor Day – May 22, 2019, New York City

· BMO Capital Markets Prescription for Success Healthcare Conference – May 25, 2019, New York City

· Jefferies Global Healthcare Conference – June 4-7, 2019, New York City

About BXCL501:

BXCL501 is a first in class, sublingual film of dexmedetomidine, a selective alpha 2a receptor agonist for the treatment of acute agitation. BTI believes that BXCL501 directly targets a causal agitation mechanism and using IV (intravenous) Dex has demonstrated anti-agitation effects in

both preclinical and clinical studies. There is precedent for FDA approval and reimbursement of a non-invasive therapy for the acute treatment of agitation in patients with schizophrenia and bipolar disease, evidenced by regulatory approval of Adasuve, an inhaled version of the antipsychotic loxapine.

About BXCL701:

BXCL701 is a first in class oral immunotherapy with dual mechanisms of action, with an established safety profile from 700 healthy subjects and cancer patients. Designed to stimulate both the innate and acquired immune systems, BXCL701 works by inhibiting dipeptidyl peptidase (DPP) 8/9 and blocking immune evasion by targeting fibroblast activation protein (FAP). Preclinical combination data evaluating BXCL701, a checkpoint inhibitor and other IO agents has demonstrated encouraging anti-tumor activity in multiple tumor types and formation of functional immunological memory. It is under development for tNEPC and pancreatic cancer.