GlobeNewswire Inc | Aug 14, 2020 07:00AM EDT

August 14, 2020

SERENITY I & II Phase 3 trials of BXCL501 achieved all primary and secondary endpoints; New Drug Application (NDA) submission to U.S. Food and Drug Administration (FDA) planned for Q1 2021

Initiating the third dose cohort, 90 mcg, of the TRANQUILITY trial in elderly dementia patients

Initiated a separate efficacy cohort in the Phase 2 trial of BXCL701 in combination with pembrolizumab (KEYTRUDA) to include patients with castrate-resistant prostate cancer (CRPC)

Strengthened balance sheet through follow-on offering raising approximately $200 million in gross proceeds

Company to host conference call today at 8:30 a.m. ET

NEW HAVEN, Conn., Aug. 14, 2020 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (BTI or Company) (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 second quarter ended June 30, 2020 and provided an update on key strategic and operational initiatives.

Since inception, this is one of the most exciting times in the Companys history, stated Vimal Mehta, Chief Executive Officer of BTI. First and foremost, we reported positive results from our two pivotal SERENITY trials last month that showed a robust treatment effect in reducing acute agitation in schizophrenia and bipolar disorder patients. We look forward to submitting an NDA for BXCL501 to the FDA in the first quarter of 2021 and, in preparation for a potential approval, we have made two key hires to lead commercialization and medical affairs. At the same time, we are advancing the TRANQUILITY and RELEASE trials in dementia and opioid withdrawal patients, respectively, and plan to initiate a trial in agitation associated with delirium patients this year, highlighting this candidates expected versatility across a wide range of diagnoses. We also continue to explore alternative indications associated with agitation, including alcohol withdrawal, post-traumatic stress disorder, traumatic brain injury and phobias. Finally, the successful completion of our recent follow-on equity offering puts us in a strong cash position to support BXCL501s commercial launch, if approved, and our aggressive indication expansion strategy.

Dr. Mehta continued, In addition, our immuno-oncology program is advancing. The Phase 2 efficacy trial of BXCL701 and KEYTRUDA for treatment emergent Neuroendocrine Prostate Cancer continues to progress on track, and we have expanded this study to include a separate cohort of men with CRPC. Our MD Anderson-led Phase 2 basket trial in advanced solid tumors is also making great strides, now having met the efficacy bar in both arms necessary for the study to proceed to completion. We believe BXCL701 has the potential to bridge innate and adaptive immunity, as demonstrated by the increase in IL-18 from baseline, and we look forward to reporting initial efficacydata from both trials later this year.

Second Quarter 2020 and Recent Highlights

BXCL501-Neuroscience Program

BXCL501 is an investigational sublingual thin film of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist, designed for the treatment of acute agitation. The Company believes BXCL501 may directly target a causal agitation mechanism.

-- In July 2020, BTI reported that the pivotal Phase 3 SERENITY trials for the acute treatment of agitation in patients with schizophrenia and bipolar disorder met the primary, key secondary and exploratory endpoints. BXCL501 was well tolerated, with rapid and durable reductions in agitation. The Company has a pre-NDA meeting with the FDA this October and plans to submit an NDA for both indications in the first quarter of 2021. -- The Company is initiating a 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. The adaptive trial is designed to identify the most effective and tolerable dose in this elderly patient population. BTI has successfully completed two lower-dose cohorts (30 mcg and 60 mcg) in a total of 30 patients. Based on findings from the 90 mcg cohort, the Company expects to report topline results in the fourth quarter of 2020, or, if needed, proceed to an additional dose cohort. -- In June 2020, the first patient was enrolled in the RELEASE study, a Phase 1b/2 trial of BXCL501 for the acute treatment of opioid withdrawal symptoms, with the third cohort currently enrolling (90 mcg twice a day, 12 hours apart). The Company expects to report topline results from the study in the first quarter of 2021. -- The Company expects to initiate a Phase 2 trial of BXCL501 in patients with agitation associated with delirium later this year. The planned study population will include ICU patients with or without COVID-19. This potential indication may offer synergy with the commercial infrastructure being developed to support our first NDA. -- The Company received a Notice of Allowance from the U.S. Patent and Trademark office for patient application No. 16/453,679 related to BXCL501. The patent is expected to cover film formulations containing Dex and methods of treating agitation using such film formulations. The patent, which is anticipated to be issued in the third quarter of 2020, is expected to extend 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) is advancing on track. Recently, BTI expanded the trial to include a separate cohort for CRPC (adenocarcinoma) patients who have failed taxane-based chemotherapy and up to two lines of second generation androgen pathway blockers, based on preliminary evidence of activity in this patient population in the safety cohort. The CRPC cohort is expected to run concurrently with the tNEPC cohort. Initial efficacy data from this trial are expected to be reported later this year. -- The open label Phase 2 basket trial evaluating the combination of BXCL701 and KEYTRUDA in patients with advanced solid cancers is proceeding, with early signs of clinical activity in several difficult-to-treat cancers. This study, which is being conducted at the MD Anderson Cancer Center, consists of two arms: checkpoint nave patients and patients who are refractory to checkpoint therapy. In June, the safety portion of the trial was completed and in August, the efficacy bar was met for both arms of the trial, allowing the study to proceed to completion. Initial efficacy data are expected to be presented at a scientific conference later this year. -- 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 is expected to begin following Nektar and Pfizers Phase 1b safety trial of a double combination of bempegaldesleukin and avelumab, pending the outcome of that trial.

Corporate Highlights

-- In July 2020, the Company raised gross proceeds of approximately $200 million in connection with its common stock offering. BTI believes that the proceeds from this offering, together with current reserves, provide cash runway well into 2022 to fund key clinical, regulatory, operational, and commercial activities. -- In June 2020, William Kane was appointed as Executive Vice President and Chief Commercial Officer of BTI. Mr. Kane brings over three decades of product commercialization experience in the pharmaceutical industry, with a proven track record in bringing neuropsychiatric drugs to market. -- In June 2020, Reina Benabou, M.D., Ph.D. was appointed as Senior Vice President and Chief Development Officer of BTI. Dr. Benabou has over 20 years of experience in directing drug development programs and implementing medical affairs strategies for product commercialization in neurology and psychiatry.

COVID-19

During the second quarter of 2020, the Company remained committed to ensuring the health and safety of its patients, investigators and employees. BTI continued to assess the impact of the COVID-19 pandemic to best mitigate risk, while continuing business operations. Beginning at the end of the second quarter, BTI began to slowly bring a limited number of staff back to the Companys office. This return to work is scheduled to be completed in September 2020. To date, the Companys business and operations has only been minimally impacted and have not experienced any significant delays to our ongoing or planned clinical trials, except for challenges in accessing elderly care facilities; however, this could rapidly change.

Second Quarter 2020 Financial Results

BTI reported a net loss of $21.4 million for the second quarter of 2020, compared to a net loss of $8.5 million for the same period in 2019. The second quarter 2020 results include approximately $2.0 million in non-cash stock-based compensation, compared to $1.0 million for the same period in 2019.

Research and development expenses were $17.9 million for the second quarter of 2020, compared to $6.5 million for the same period in 2019. The increase was primarily due to increases in clinical trial activity.

General and administrative expenses were $3.5 million for the second quarter of 2020, as compared to $2.1 million for the same period in 2019. The increase was primarily due to salaries, non-cash compensation costs and professional fees.

Total operating expenses for the second quarter of 2020 were approximately $21.4 million, compared to total operating expenses of approximately $8.6 million for the same period in 2019.

As of June 30, 2020, cash and cash equivalents totaled approximately $65.5 million. This does not include the $187.5 million in net proceeds generated from our equity offering that closed on July 31, 2020.

Conference Call:

BTI will host a conference call and webcast today at 8:30 a.m. ET. To access the call, please dial 877-407-2985 (domestic) and 201-378-4915 (international). A live webcast of the call will be available on the Investors sections of the BTI website at www.bioxceltherapeutics.com. The replay will be available through at least August 28, 2020.

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 evaluated 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 sublingual thin film formulation in development for acute treatment of agitation resulting from neuropsychiatric disorders, and BXCL701, an investigational orally administered systemic innate immunity activator in development for treatment of a rare form of prostate cancer and for treatment of pancreatic cancer in combination with other immuno-oncology agents. For more information, please visit www.bioxceltherapeutics.com.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this press release include but are not limited to the timing and data from clinical development initiatives, applications and trials for BXCL501 and BXCL701, the Companys cash runway, the impact of the COVID-19 pandemic on the Companys business, financial results and financial condition, the Companys future growth, corporate strategy and position to execute on key milestones and the Companys intellectual property portfolio. When used herein, words including anticipate, being, will, plan, may, continue, and similar expressions are intended to identify forward-looking statements. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon BTI's current expectations and various assumptions. BTI believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain.

BTI may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, its limited operating history; its incurrence of significant losses; its need for substantial additional funding and ability to raise capital when needed; its limited experience in drug discovery and drug development; its dependence on the success and commercialization of BXCL501 and BXCL701 and other product candidates; the failure of preliminary data from its clinical studies to predict final study results; failure of its early clinical studies or preclinical studies to predict future clinical studies; its ability to receive regulatory approval for its product candidates; its ability to enroll patients in its clinical trials; undesirable side effects caused by BTIs product candidates; its approach to the discovery and development of product candidates based on EvolverAI is novel and unproven; its exposure to patent infringement lawsuits; its ability to comply with the extensive regulations applicable to it; impacts from the COVID-19 pandemic; its ability to commercialize its product candidates; and the other important factors discussed under the caption Risk Factors in its Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2020 as such factors may be updated from time to time in its other filings with the SEC, including, but limited to, its Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2020, which are accessible on the SECs website at www.sec.govand the Investors section of our website at www.bioxceltherapeutics.com.

These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While BTI may elect to update such forward-looking statements at some point in the future, except as required by law, it disclaims any obligation to do so, even if subsequent events cause our views to change. These forward-looking statements should not be relied upon as representing BTIs views as of any date subsequent to the date of this press release.

BIOXCEL THERAPEUTICS,INC.

BALANCE SHEETS

(amounts in thousands, except share and per share data)

June30, December31, 2020 2019 (unaudited) ASSETS Current assets Cash and cash equivalents $ 65,495 $ 32,426 Prepaid expenses and other current assets 2,818 1,681 Total current assets 68,313 34,107 Property and equipment, net 997 1,041 Operating lease right-of-use asset 1,101 1,193 Other assets 51 51 Total assets $ 70,462 $ 36,392 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities Accounts payable $ 10,434 $ 4,953 Accrued expenses 4,527 3,120 Due to Parent 72 64 Other current liabilities 1,128 331 Total current liabilities 16,161 8,468 Operating lease liability 907 1,029 Total liabilities 17,068 9,497 Stockholders' equity Preferred stock, $0.001 par value, 10,000,000shares authorized; no shares issued or ? ? outstandingCommon stock, $0.001 par value, 50,000,000 sharesauthorized; 20,352,913 and 18,087,382 shares 20 18 issued and outstanding as of June30,2020 andDecember31,2019, respectivelyAdditional paid-in-capital 146,392 83,565 Accumulated deficit (93,018 ) (56,688 )Total stockholders' equity 53,394 26,895 Total liabilities and stockholders' equity $ 70,462 $ 36,392

BIOXCEL THERAPEUTICS,INC.

STATEMENTS OF OPERATIONS

(amounts in thousands, except share and per share data)(unaudited)

Three Months Ended June30, Six Months Ended June30, 2020 2019 2020 2019Revenues $ ? $ ? $ ? $ ? Operatingcosts and expensesResearch and 17,906 6,506 30,277 12,180 developmentGeneral and 3,529 2,129 6,154 3,874 administrativeTotaloperating 21,435 8,635 36,431 16,054 expensesLoss from (21,435 ) (8,635 ) (36,431 ) (16,054 )operationsOther income Dividend andinterest 16 164 101 379 income, netNet loss $ (21,419 ) $ (8,471 ) $ (36,330 ) $ (15,675 ) Net loss pershareattributableto common $ (1.06 ) $ (0.54 ) $ (1.85 ) $ (1.00 )stockholdersbasic anddiluted Weightedaverage sharesoutstanding - 20,293,216 15,668,588 19,619,145 15,666,190 basic anddiluted

BIOXCEL THERAPEUTICS, INC.

STATEMENTS OF CHANGES IN STOCKHOLDERS EQUITY

(amounts in thousands, except shares)

(unaudited)

Additional CommonStock Paidin Accumulated Shares Amount Capital Deficit TotalBalance as ofDecember 31, 15,663,221 $ 16 $ 62,593 $ (23,720 ) $ 38,889 2018Stock-based ? ? 682 ? 682 compensationExercise of 2,581 ? 1 ? 1 stock optionsNet loss ? ? ? (7,204 ) (7,204 )Balance as ofMarch 31, 15,665,802 $ 16 $ 63,276 $ (30,924 ) $ 32,368 2019 Issuance ofcommonshares, net 21,744 ? 230 ? 230 of issuancecosts of $11Stock-based ? ? 1,030 ? 1,030 compensationNet loss ? ? ? (8,471 ) (8,471 )Balance as of 15,687,546 $ 16 $ 64,536 $ (39,395 ) $ 25,157 June 30, 2019 Balance as ofDecember 31, 18,087,382 $ 18 $ 83,565 $ (56,688 ) $ 26,895 2019Issuance ofcommon stock,net of 2,300,000 2 68,809 68,811 issuancecosts of$4,789Purchase andcancellationof shares (300,000 ) ? (9,024 ) (9,024 )from BioXcelCorporationStock-based ? ? 776 ? 776 compensationExercise of 95,000 ? 39 ? 39 stock optionsNet loss ? ? ? (14,911 ) (14,911 )Balance as ofMarch 31, 20,182,382 $ 20 $ 144,165 $ (71,599 ) $ 72,586 2020 Stock-based ? 1,956 1,956 compensationExercise of 170,531 ? 271 271 stock optionsNet loss ? (21,419 ) (21,419 )Balance as of 20,352,913 $ 20 $ 146,392 $ (93,018 ) $ 53,394 June 30, 2020

BIOXCEL THERAPEUTICS,INC.

STATEMENTS OF CASH FLOWS

(amounts in thousands)

(unaudited)

Six months ended June30, 2020 2019CASH FLOWS FROM OPERATING ACTIVITIES: Net loss $ (36,330 ) $ (15,675 )Reconciliation of net loss to net cash used in operating activitiesDepreciation and amortization 95 122 Stock-based compensation expense 2,732 1,712 Changes in operating assets and liabilities: Prepaid expenses and other assets (1,137 ) (1,009 )Accounts payable, accrued expenses and other 7,663 3,153 Net cash used in operating activities (26,977 ) (11,697 ) CASH FLOWS FROM INVESTING ACTIVITIES: Purchases of equipment and leasehold improvements (51 ) (825 )Net cash used in investing activities (51 ) (825 ) CASH FLOWS FROM FINANCING ACTIVITIES: Proceeds from issuance of common stock, net of 68,811 230 issuance costsPurchase and cancellation of shares from BioXcel (9,024 ) ? CorporationDeferred offering expense ? (378 )Due to Parent ? 69 Exercise of options 310 1 Net cash provided by financing activities 60,097 (78 ) Net (decrease) increase in cash and cash 33,069 (12,600 )equivalents Cash and cash equivalents, beginning of theperiod 32,426 42,565 Cash and cash equivalents, end of theperiod $ 65,495 $ 29,965 Supplemental cash flow information: Interest paid $ 18 $ 29

Contact Information:BioXcel Therapeutics, Inc.www.bioxceltherapeutics.com

Investor Relations:John Grazianojgraziano@troutgroup.com1.646.378.2942

Media:Julia Deutschjdeutsch@troutgroup.com1.646.378.2967