On March 10, 2022 Gritstone bio, Inc. (Nasdaq: GRTS), a clinical-stage biotechnology company developing the next generation of cancer and infectious disease immunotherapies, reported financial results for the fourth quarter and full year ended December 31, 2021 and provided recent clinical and corporate updates (Press release, Gritstone Oncology, MAR 10, 2022, View Source [SID1234609883]).

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"2021 was a transformational year for Gritstone, as we extended our unique understanding of T cell antigens and proprietary vaccine delivery platforms to infectious disease, expanding our pipeline to include novel programs in COVID-19 (CORAL) and human immunodeficiency virus (HIV). Furthermore, we made significant progress with our tumor-specific neoantigen oncology programs, GRANITE and SLATE, which are advancing into later-stage trials," said Andrew Allen, M.D., Ph.D., Co-Founder, President and Chief Executive Officer of Gritstone. "In CORAL, we have demonstrated the potential for our T cell enhanced samRNA vaccine candidates to drive broad and durable immune responses in humans against both current and future variants against COVID-19, a credible first step toward developing a pan-coronavirus vaccine given the high conservation of many T cell antigens across different coronaviruses. Within oncology, we have continued to demonstrate the ability to drive a large and sustained T cell response, including the challenging CD8+ killer T cells, against tumor-specific neoantigens. These T cell responses are potentially driving the molecular and radiological responses observed in our "off the shelf" program, SLATE, which is focused on KRAS mutations, and also appear to be driving molecular response and extended overall survival in the end-stage colorectal cancer patients treated in our individualized program, GRANITE."

"Our collective experience with cancer immunotherapy suggests that as these programs move into earlier stages of disease, immune responses may be stronger and the potential benefits of our approach will be further accentuated," Dr. Allen continued. "Consequently, we are excited to have initiated randomized Phase 2/3 trials with GRANITE in colorectal cancer (CRC) patients where unmet need persists and checkpoint inhibitor therapy has had little impact to date in the vast majority of patients. We believe Gritstone is well-positioned to continue advancing our programs and platforms, and look forward to sharing updates as the year progresses."

Clinical Program Updates

Tumor-Specific Neoantigen (TSNA) Oncology Programs

GRANITE – Individualized, tumor specific neoantigen (TSNA)-directed immunotherapy using an adenoviral priming vector and self-amplifying mRNA boost vector to deliver relevant neoantigens

To date, an ongoing Phase 1/2 study assessing GRANITE in combination with an anti-PD-1 checkpoint inhibitor, nivolumab, and subcutaneous anti-CTLA-4 antibody, ipilimumab, has demonstrated a favorable safety and tolerability profile for patients treated in the study and demonstrated consistent induction of neoantigen-specific CD8+ T cells, and reduction in circulating tumor DNA (ctDNA), an increasingly recognized biomarker within utility in immunotherapy in advanced solid tumors.

Additionally, colorectal cancer patients who demonstrated a molecular response (ctDNA reduction >50% from baseline) had median overall survival of >17 months (median not reached) whereas those without molecular response exhibited a median overall survival of 7.8 months, the latter being consistent with expected outcome in 3rd line treatment of metastatic, microsatellite-stable colorectal cancer (MSS-CRC) patients. These data support the potential for GRANITE to offer benefit in a disease setting such as MSS-CRC where patients do not traditionally respond to checkpoint inhibitor therapy.

Given the results of the Phase 1/2 study, Gritstone has initiated two studies assessing GRANITE in earlier stages of disease: GRANITE-CRC-1L (a Phase 2/3 randomized, controlled trial evaluating the individualized neoantigen vaccine GRANITE in combination with immune checkpoint blockade for the first line (1L) maintenance treatment of newly diagnosed patients with metastatic MSS-CRC) and GRANITE-CRC-ADJUVANT (Randomized, controlled phase 2 trial of adjuvant GRANITE immunotherapy in high risk MSS-CRC patients with stage II/III disease who are ctDNA+ after definitive surgery). In January 2022, Gritstone announced the first patient was enrolled for inclusion in the GRANITE-CRC-1L trial. As of March 2022, the first patient was enrolled for inclusion in the GRANITE–CRC-ADJUVANT study.
SLATE – "Off-the-shelf" shared neoantigen-directed immunotherapy using an adenoviral priming vector and self-amplifying mRNA boost vector to deliver a cassette of shared TSNA

Preliminary data from an ongoing Phase 1/2 trial in advanced disease patients indicated a favorable safety and tolerability profile and induction of CD8+ T cells against multiple KRAS TP53 driver mutations. Clinical activity was observed in NSCLC patients who had all progressed on prior chemoimmunotherapy, including 6 NSCLC patients with the G12C KRAS mutation. Among these G12C patients, ctDNA responses were observed in 2 of 3 eligible for analysis, which correlated with clinical benefit, and a RECIST radiologic response (unconfirmed) was observed in one 2nd line patient with aggressive disease who had progressed after 3 months of 1st line chemo-immunotherapy. There were no safety signals of note; the most common adverse events being low grade, self-limiting fever and injection site reactions. An optimized SLATE cassette (SLATE-KRAS, v2), which exclusively includes KRASmut epitopes, exhibited immunogenic superiority over version 1 in preclinical studies. This next-generation SLATE-KRAS cassette is now in Phase 2 testing in patients with advanced non-small cell lung cancer (NSCLC) and CRC, and initial data are expected in 2H 2022.
Infectious Disease Programs

CORAL – second-generation SARS-CoV-2 vaccine program delivering both spike and highly conserved non-spike T cell epitopes (TCEs) with a focus on the samRNA vector. This approach offers potential for more durable clinical protection and broader immunity against SARS-CoV-2 variants than first generation products by inducing potent CD8+ T cells in addition to neutralizing antibody responses.

To date, the CORAL program has demonstrated positive preclinical and clinical data supporting the approach of a T cell enhanced samRNA vaccine against COVID-19, indicating a favorable safety profile and induction of both neutralizing antibodies and T cells.

In January 2022, Gritstone announced positive clinical data from the first cohort of CORAL-BOOST, a Phase 1 study evaluating the safety, reactogenicity, and immunogenicity of a samRNA vaccine directed against Spike and highly conserved non-Spike TCEs as a booster against SARS-CoV-2 in healthy adults ≥60 years who previously received two doses of AstraZeneca’s first-generation COVID-19 vaccine AZD1222 (Vaxzevria). Enrollment of the second cohort of CORAL-BOOST has since concluded and based on the favorable immunogenicity and reactogenicity seen with the 10µg dose, the study is proceeding to additional cohorts utilizing this dose.

Two additional Phase 1 trials, CORAL-CEPI and CORAL-IMMUNOCOMPROMISED, were initiated in 1Q 2022. The CORAL-CEPI trial, which is evaluating T cell enhanced omicron- and beta-spike constructs in virus-naïve, convalescent, and HIV+ patients, is being run in South Africa with support from the Coalition for Epidemic Preparedness Innovations (CEPI). The CORAL-IMMUNOCOMPROMISED trial, which is evaluating T cell enhanced samRNA and chimpanzee adenovirus (ChAd) vaccines in B cell deficient subjects, is being run in the United Kingdom.

CORAL-NIH, a Phase 1 study evaluating the immunogenicity and safety of samRNA and/or ChAd vaccines in healthy adult subjects that is supported by the National Institute of Allergy and Infectious Diseases (NIAID) is being conducted through their Infectious Diseases Clinical Research Consortium (IDCRC). Initial data is expected in 1H 2022.

All four clinical trials within the CORAL program are now enrolling and Gritstone expects to provide data updates on each of these trials throughout the remainder of 2022.
HIV – Collaboration with Gilead Sciences, Inc (Gilead) under their HIV Cure Program to research and develop vaccine-based HIV immunotherapy treatment

Following initiation of the collaboration with Gilead in early 2021, an IND was submitted and subsequently cleared in December 2021.
2021 Funding Highlights and Recent Corporate Updates

2021 Funding Highlights

Gritstone entered into a funding agreement of up to $20.6 million with the Coalition for Epidemic Preparedness Innovations (CEPI) to advance the clinical development of Gritstone’s CORAL second generation mRNA COVID-19 vaccine program.
Gritstone secured $55 million Private Placement led by Frazier Life Sciences Public Fund, Redmile Group and Gilead Sciences.
Gritstone secured $60 million payment from Gilead at closing of HIV program collaboration, consisting of a $30 million upfront cash payment and a $30 million equity investment at a premium.
Recent corporate updates

The funding agreement with CEPI was expanded for up to $5 million to advance the clinical development of Gritstone’s CORAL second generation mRNA COVID-19 vaccine program targeting the omicron variant in South Africa (December 2021).
Clare Fisher, Senior Vice President of Business Development and Mergers & Acquisitions at BeiGene, was appointed to the Gritstone Board of Directors effective as of January 1, 2022 (December 2021).
Gritstone was added to the Nasdaq Biotechnology Index (December 2021).
Full Year 2021 Financial Results

Cash, cash equivalents, marketable securities and restricted cash were $223.5 million as of December 31, 2021, compared to $172.1 million as of December 31, 2020.
Research and development expenses were $97.5 million for the year ended December 31, 2021, compared to $88.6 as of December 31, 2020. The increase was primarily due to increases in personnel-related expenses and clinical trial expenses.
General and administrative expenses were $25.9 million for the year ended December 31, 2021, compared to $21.4 million as of December 31, 2020. The increase was primarily attributable to an increase in personnel-related costs as we expanded our headcount, and an increase in outside services for legal, finance, recruiting and other professional services to support our ongoing operations and operate as a public company.
Collaboration and license revenue was $46.7 million for the year ended December 31, 2021, compared to $3.5 million for the prior year. During the year ended December 31, 2021, we recorded $38.6 million in license revenue and $5.1 million in collaboration revenue related to the Gilead Collaboration Agreement, and $3.0 million in collaboration revenue related to the 2seventy bio Agreement.

On March 10, 2022 Galera Therapeutics, Inc. (Nasdaq: GRTX), a clinical-stage biopharmaceutical company focused on developing and commercializing a pipeline of novel, proprietary therapeutics that have the potential to transform radiotherapy in cancer, reported financial results for the fourth quarter and year ended December 31, 2021 and provided recent corporate updates (Press release, Galera Therapeutics, MAR 10, 2022, View Source [SID1234609899]).

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"In 2021, we were pleased to report positive data from our lead program evaluating avasopasem for the treatment of severe oral mucositis, a radiotherapy-induced toxicity, in patients with head and neck cancer," said Mel Sorensen, M.D., Galera’s President and CEO. "The Phase 3 ROMAN trial demonstrated a statistically significant reduction in the incidence of SOM, the primary endpoint, and in the number of days patients experienced SOM. This data is in line with the topline results observed in the Phase 2a EUSOM trial and our previously completed randomized Phase 2b trial, reinforcing avasopasem’s potentially transformative clinical benefit for patients undergoing radiotherapy for head and neck cancer. With this robust package of data in hand, we are preparing to meet with the FDA to discuss the potential NDA submission. Concurrently, we remain on track to report data from our Phase 2a AESOP trial evaluating avasopasem in esophagitis in the first half of this year."

Dr. Sorensen continued: "We also continue to progress our Phase 1/2 GRECO-1 and Phase 2b GRECO-2 clinical trials evaluating our second product candidate, rucosopasem, in increasing the anti-cancer efficacy of higher daily doses of radiotherapy in patients with non-small cell lung cancer and locally advanced pancreatic cancer, respectively. We look forward to reporting initial data from our GRECO-1 trial in the first half of this year."

Recent Corporate Updates

Radiotherapy-Induced Toxicity Programs:

Severe Oral Mucositis (SOM)

On December 14, 2021, the Company announced corrected topline efficacy results from the Phase 3 ROMAN trial of avasopasem for the reduction of SOM in patients with locally advanced head and neck cancer (HNC). The Company had previously announced topline results from the ROMAN trial on October 19, 2021. Upon further analysis following the October announcement, an error by the contract research organization was identified in the statistical program. Correction of this error resulted in improved p-values for the primary and secondary endpoints. The corrected results demonstrated efficacy across multiple SOM endpoints with a statistically significant reduction on the primary endpoint of incidence of SOM and the secondary endpoint of number of days of SOM, more than halving the median number of days a patient experienced SOM. Avasopasem appeared to be generally well tolerated compared to placebo. The Company announced that it plans to meet with the U.S. Food and Drug Administration (FDA) in 2022 to discuss a potential New Drug Application (NDA) submission.
On December 14, 2021, the Company also reported topline data from the Phase 2a EUSOM multicenter trial of avasopasem in Europe in patients with HNC undergoing standard-of-care radiotherapy. Avasopasem appeared to be generally well tolerated and the incidence and number of days of SOM was in line with the avasopasem data reported in the Phase 3 ROMAN trial.
Esophagitis

The Company expects to report topline data from the Phase 2a AESOP trial of avasopasem evaluating its ability to reduce the incidence of esophagitis induced by radiotherapy in patients with lung cancer in the first half of 2022.
Anti-Cancer Programs:

Locally Advanced Pancreatic Cancer (LAPC)

Enrollment is ongoing in the Phase 2b, 160-patient randomized, multicenter, placebo-controlled GRECO-2 trial of rucosopasem, Galera’s second dismutase mimetic product candidate, in combination with stereotactic body radiation therapy (SBRT) in patients with LAPC. The primary endpoint of the trial is overall survival.
Non-Small Cell Lung Cancer (NSCLC)

Enrollment is ongoing in the Phase 1/2 GRECO-1 trial of rucosopasem in combination with SBRT in patients with NSCLC. The Company expects to report initial data from this trial in the first half of 2022.
Fourth Quarter 2021 Financial Highlights

Research and development expenses were $9.2 million in the fourth quarter of 2021, compared to $14.6 million for the same period in 2020. The decrease was primarily attributable to a decrease in avasopasem development costs, partially offset by an increase in rucosopasem development costs.
General and administrative expenses were $5.3 million in the fourth quarter of 2021, compared to $4.3 million for the same period in 2020. The increase was primarily attributable to employee-related costs from increased headcount and share-based compensation expense, and increased insurance expense and professional fees.
Galera reported a net loss of $(16.8) million, or $(0.64) per share, for the fourth quarter of 2021, compared to a net loss of $(20.1) million, or $(0.80) per share, for the same period in 2020.
As of December 31, 2021, Galera had cash, cash equivalents and short-term investments of $71.2 million. Galera expects that its existing cash, cash equivalents and short-term investments will enable Galera to fund its operating expenses and capital expenditure requirements into the second half of 2023.
Full Year 2021 Financial Highlights

Research and development expenses were $52.4 million for the year ended December 31, 2021, compared to $54.8 million for the year ended December 31, 2020. The decrease was primarily attributable to a decrease in avasopasem development costs, partially offset by an increase in rucosopasem development costs.
General and administrative expenses were $21.0 million for the year ended December 31, 2021, compared to $15.7 million for the year ended December 31, 2020. The increase was primarily attributable to employee-related costs from increased headcount and share-based compensation expense, increased expenses related to preparation for potential commercialization of avasopasem, and increased insurance expense and professional fees.
Galera reported a net loss of $(80.5) million, or $(3.12) per share, for the year ended December 31, 2021, compared to a net loss of $(74.2) million, or $(2.98) per share, for the year ended December 31, 2020.

On March 10, 2022 Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing therapies for the treatment of refractive and retinal eye disorders, reported that Mina Sooch, MBA, Founder and CEO will present a corporate overview at the Oppenheimer 32nd Annual Healthcare Conference on Wednesday, March 16 at 8:00-8:30 AM EDT (Press release, Ocuphire Pharma, MAR 10, 2022, View Source [SID1234609916]).

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Oppenheimer 32nd Annual Healthcare Conference – March 15-17, 2022

Title: Ocuphire Pharma (OCUP) Company Presentation
Date: Wednesday, March 16, 2022
Time: 8:00 AM EDT
Presenter: Mina Sooch, CEO
Webcast Link: Register here
If you are interested in arranging a 1X1 meeting request or listening live or to a replay of the company presentation, please contact your bank conference representative or [email protected]. To access the archived recording for replay, please see the Investors and Events section of Ocuphire’s corporate website.

On March 10, 2022 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported its financial results for the fourth quarter and year ended December 31, 2021 and provided a corporate update (Press release, Regulus, MAR 10, 2022, View Source [SID1234609932]).

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"Last year was a transformational year for Regulus with the accomplishments in our ADPKD program including clinical validation of the target and the prioritization and advancement of our next generation compound RGLS8429," commented Jay Hagan, CEO of Regulus Therapeutics. "We are on track with an IND filing and initiation of our Phase 1 clinical study and with the recent equity financing we believe the Company is poised and capitalized to deliver on upcoming milestones."

Program Updates

RGLS8429 for ADPKD: In January 2022, the Company announced the successful completion of a pre-investigational new drug (Pre-IND) meeting with the U.S. Food and Drug Administration (FDA). The FDA provided overall agreement with the trial design and length of the Phase 1 study. Based on this feedback, the company is on track to submit an IND application in the second quarter of 2022 to obtain clearance for the initiation of the Phase 1 clinical trial. The Company anticipates reporting top-line data from the healthy volunteer portion of the study in the second half of 2022, and top-line biomarker data in the first cohort of RGLS8429-treated patients with ADPKD in the first half of 2023.

Lademirsen (RG-012) for Alport syndrome: In February 2022, the Company announced completion of enrollment in the Phase 2 HERA clinical study evaluating lademirsen for the treatment of adult patients with Alport Syndrome under the Company’s Collaboration and License Agreement with Sanofi. Final data is expected in the first half of 2023 and if successful could provide further validation of the Company’s platform technology designed to address genetic kidney diseases and earn the Company a $25 million milestone, furthering the cash runway into 2024.

Corporate Highlights

Closed $34.6 Million Private Placement: In November 2021, the Company announced the closing of a private placement of equity. The financing was led by the Federated Hermes Kaufmann Funds and New Enterprise Associates (NEA), with participation from other new and existing investors. The Company received gross proceeds of approximately $34.6 million from the sale of 58,923,352 shares of the Company’s common stock ("Common Stock") at a purchase price of $0.36 per share. In addition, the Company sold 3,725,720 shares of non-voting Class A-4 convertible preferred stock, in lieu of shares of Common Stock, at a price of $3.60 per share. Each share of non-voting Class A-4 convertible preferred stock is convertible into 10 shares of Common Stock, subject to certain beneficial ownership conversion limitations. Net proceeds from the transaction will be used for non-clinical and clinical development activities for the Company’s product candidates and general corporate purposes. SVB Leerink acted as the lead placement agent for the financing and H.C. Wainwright and Co. acted as co-placement agent.

Expanded Team: In December 2021, the Company announced the appointment of Mohammad Ahmadian, Ph.D., as Vice President, Chemistry and Pharmaceutical Development. Dr. Ahmadian has worked for various life sciences companies in research and managerial capacities. Most recently, he was Vice President & Resident Director of Kinovate Life Sciences, Inc.

Financial Results

Cash Position: As of December 31, 2021, Regulus had $60.4 million in cash and cash equivalents.

Research and Development (R&D) Expenses: Research and development expenses were $4.4 million and $17.8 million for the fourth quarter and year ended December 31, 2021, respectively, compared to $4.0 million and $15.3 million for the same periods in 2020, respectively. These amounts reflect internal and external costs associated with advancing our clinical and preclinical pipeline.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.6 million and $10.0 million for the fourth quarter and year ended December 31, 2021, respectively, compared to $2.1 million and $8.8 million for the same periods in 2020, respectively. These amounts reflect personnel-related and ongoing general business operating costs.

Net Loss: Net loss was $7.1 million, or $0.07 per share (basic and diluted), and $27.8 million, or $0.32 per share (basic and diluted), for the fourth quarter and year ended December 31, 2021, respectively, compared to $1.3 million, or $0.03 per share (basic and diluted), and $15.7 million, or $0.45 per share (basic and diluted), respectively, for the same periods in 2020.

Conference Call and Webcast Information:
The Company will host a conference call and live audio webcast today at 5:00 p.m. ET to discuss its fourth quarter and year end 2021 financial results and corporate update. To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 3786683. To access the telephone replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), passcode ID 3786683. The webcast and telephone replay will be archived on the Company’s website at www.regulusrx.com following the call.

About ADPKD

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About RGLS8429

RGLS8429 is a novel, next generation oligonucleotide designed to inhibit miR-17 and to preferentially target the kidney. Administration of RGLS8429 has shown robust data in preclinical models, where clear improvements in kidney function, size, and other measures of disease severity, as well as a superior pharmacologic profile have been demonstrated. Regulus has nominated RGLS8429 as a clinical candidate for the treatment of ADPKD.

On March 10, 2022 BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, reported its financial results for the fourth quarter and full year ended December 31, 2021 and provided an update on key strategic initiatives (Press release, BioXcel Therapeutics, MAR 10, 2022, View Source [SID1234609868]).

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"2021 was marked by significant achievement across the business, most notably with the NDA submission for BXCL501, which concluded an unprecedented, less than three-year journey from first-in-human trials to regulatory submission," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. "As we near potential approval, we remain focused on executing our launch readiness plan. Matt Wiley, who recently joined as our Chief Commercial Officer, has, and will continue to play a pivotal role in developing our commercial and market access strategy and building out our field sales organization. In parallel, we continued to advance our ‘land and expand’ strategy by furthering our Alzheimer’s disease and MDD programs, both important components of our five-year vision to become the leading AI-enabled neuroscience Company."

Dr. Mehta added, "Our oncology franchise further progressed with encouraging composite response rates for BXCL701 in combination with KEYTRUDA for mCRPC patients with SCNC and adenocarcinoma phenotypes, both aggressive tumors with very few treatment options. We continue to develop BXCL701’s potential to convert cold tumors to hot as a novel systemic innate immune activator."

Company Highlights

Neuroscience Franchise

BXCL501 is an investigational, proprietary, orally dissolving, thin film formulation of the adrenergic receptor agonist dexmedetomidine for the treatment of agitation associated with neuropsychiatric disorders. BXCL501 has received U.S. Food & Drug Administration (FDA) Breakthrough Therapy designation for the acute treatment of agitation associated with dementia and FDA Fast Track designation for the acute treatment of agitation associated with schizophrenia, bipolar disorders I and Il, and dementia.

BXCL501 for the Acute Treatment of Agitation in Patients with Schizophrenia and Bipolar Disorders: The Company is gearing up to launch BXCL501 in the U.S. pending FDA approval by the April 5, 2022 PDUFA action date. This is a large addressable market, with over 7 million people in the U.S. diagnosed with schizophrenia or bipolar disorders1-3, totaling approximately 25 million annual agitation episodes.3

Data Published in Journal of the American Medical Association (JAMA) from SERENITY II Pivotal Phase 3 Trial Evaluating BXCL501 in Bipolar Disorders: Results demonstrate BXCL501 as a potential treatment for the millions of patients experiencing agitation, a difficult-to-manage symptom associated with many neuropsychiatric conditions.
Indication Expansion

BXCL501 for Acute Treatment of Agitation in Patients with Alzheimer’s Disease: Initiated pivotal Phase 3 program in Q4 2021 following alignment with FDA on key trial design features. Alzheimer’s disease is the most prevalent type of dementia in the U.S. and is expected to approximately double from 5.8 million patients in 2020 to 11.8 million patients by 2040.4

BXCL501 for Major Depressive Disorder (MDD): Submitted an Investigational New Drug (IND) application to evaluate BXCL501 as an adjunct treatment for MDD, the most common type of depression in the United States with approximately 27 million cases a year.5 There are over 300 million antidepressant prescriptions filled annually, with limitations of current treatment including slow onset of action and incomplete response.6
Geographic Expansion

International Growth: The Company expects to submit a Marketing Authorization Application to the European Medicines Agency (EMA) for BXCL501 in 1H 2022.
AI-driven Drug Discovery & Development

The Company continues its innovative approach to R&D by leveraging its proprietary artificial intelligence platform to expand its current neuroscience portfolio, including identification of the Company’s newest product candidate, BXCL502. The Company initiated formulation for this new product candidate for chronic treatment of agitation in dementia patients. BXCL502 is designed to be a potent and selective antagonist for a GPCR target affecting serotonergic signaling in the cerebral cortex.
BXCL501 Commercial Readiness Progress

Expanded Commercial Organization: Hired Chief Commercial Officer Matt Wiley to develop integrated commercial strategy for potential BXCL501 approval and beyond, and continue the buildout of the Company’s national sales force.

Medical Science Liaison and Medical Managed Care Teams Fully Deployed: Continued engagement with health care professionals (HCPs) and payers to provide key insights and support potential BXCL501 commercial launch, including planned participation at:
Academy of Managed Care Pharmacy annual meeting in March 2022
International Society of Pharmacoeconomics and Outcomes Research conference in May 2022
Immuno-Oncology Franchise

BXCL701 is an investigational, orally administered, systemic innate immune activator in development for treatment of aggressive forms of prostate cancer and advanced solid tumors that are refractory or treatment naïve to checkpoint inhibitors.

Metastatic Castration-Resistant Prostate Cancer (mCRPC) Program: Presented positive data from Phase 2 trial of BXCL701 in aggressive forms of prostate cancer at 2022 ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium in February. The presentations showed that BXCL701 plus KEYTRUDA (pembrolizumab) demonstrated encouraging composite response rates in patients with either adenocarcinoma (21% response rate) or SCNC (33% response rate) phenotype, supporting further evaluation of BXCL701’s potential to extend checkpoint inhibitor therapy into cold tumor settings.

Solid Tumors Program (Checkpoint Naïve and Refractory): Additional efficacy data from MD Anderson-led open-label Phase 2 basket trial of BXCL701 and KEYTRUDA expected in 2H 2022.
Fourth Quarter and Full Year 2021 Financial Results

Research and Development Expenses: Research and development expenses were $12.5 million for the fourth quarter of 2021, compared to $11.4 million for the same period in 2020. The increased expenses were primarily attributable to increased headcount and related costs during the three-month period of 2021.

Research and development expenses were $52.7 million for the full year 2021, as compared to $58.0 million for the same period in 2020. The decrease for the year ended December 31, 2021 was primarily attributable to decreased clinical trial expenses related to BXCL501, partially offset by increased costs related to BXCL701 clinical trials.

General and Administrative Expenses: General and administrative expenses were $13.6 million for the fourth quarter of 2021, as compared to $9.7 million for the same period in 2020. The increase was primarily due to increased headcount and related costs, including higher stock-based compensation, increased marketing and commercial costs related to the potential launch of BXCL501 in the U.S., as well as increased legal and professional fees, and insurance costs.

General and administrative expenses were $54.2 million for the full year 2021, as compared to $24.3 million for the same period in 2020. The increase for the year ended December 31, 2021 was primarily attributable to increased headcount and associated costs, including non-cash stock-based compensation. The Company also incurred significant commercial costs in preparation for a potential U.S. launch of BXCL501.

Net Loss: BioXcel Therapeutics reported a net loss of $26.1 million for the fourth quarter of 2021, compared to a net loss of $21.1 million for the same period in 2020. For the full year, BioXcel reported a net loss of $106.9 million, compared to a net loss of $82.2 million for the same period in 2020.

As of December 31, 2021, cash and cash equivalents totaled approximately $233.0 million.

Conference Call

BioXcel Therapeutics will host a conference call and webcast March 10, 2022 at 8:30 a.m. ET, to discuss its fourth quarter and full year 2021 financial results and provide an update on recent operational highlights. To access the call, please dial 877-407-5795 (domestic) and 201-689-8722 (international). A live webcast of the call will be available on the Investors section of the BioXcel website, www.bioxceltherapeutics.com, and a replay of the call will be available through at least April 10, 2022.

BioXcel Therapeutics may use its website as a distribution channel of material information about the Company. Financial and other important information regarding the Company is routinely posted on and accessible through the Investors sections of its website at www.bioxceltherapeutics.com. In addition, you may automatically receive email alerts and other information about the Company when you enroll your email address by visiting the "Email Alerts" option under the News/Events menu of the Investors & Media section of its website.