On May 9, 2024 BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience and immuno-oncology, reported its financial results for the first quarter of 2024 (Press release, BioXcel, MAY 9, 2024, View Source [SID1234642978]).
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"The fundamentals of our business are strong as we look to continue advancing and expanding our agitation portfolio," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. "We have designed two Phase 3 registrational programs for BXCL501 in addition to strengthening our balance sheet and intellectual property. We are intensely focused on the BXCL501 journey into the at-home setting and expansion into Alzheimer’s-related agitation with the goal of bringing new treatment options to larger numbers of patients while expanding the market potential of our lead neuroscience asset."
TRANQUILITY and SERENITY Clinical Programs
· Plans for two late-stage programs are advancing following recently announced designs of pivotal Phase 3 trials:
o TRANQUILITY In-Care trial: designed to evaluate the efficacy and safety of a 60 mcg dose of BXCL501 for agitation associated with Alzheimer’s dementia (AAD).
o SERENITY At-Home* safety trial: designed to evaluate the safety of a 120 mcg dose of BXCL501 in the at-home setting for agitation associated with bipolar disorders or schizophrenia.
· Study protocol submitted to FDA.
IGALMI Post-Marketing Requirement (PMR) Study
· Study was designed to evaluate whether tolerance, tachyphylaxis, or withdrawal occur following repeat dosing of IGALMI following seven days of repeated treatment.
o Completed enrollment of approximately 20 patients with frequent episodes of agitation for bipolar disorders or schizophrenia in an open-label study.
o Patients self-administered 180 mcg of IGALMI for repeated agitation episodes over the treatment period.
o Initiated data cleaning to enable database lock.
Corporate Updates
IGALMI Commercialization
· Net revenue grew 55% in Q1 2024 over the prior quarter driven largely by volume contracting, new customer acquisition, increased utilization among existing customers, and the permanent J-Code for IGALMI that became effective January 1, 2024.
Patent Portfolio
The Company continues to strengthen its intellectual property portfolio with over 30 granted or allowed patents and more than 140 additional patent applications in prosecution as of April 2024.
· Recently granted two new patents for BXCL501, in Japan and the U.S., with patent protection to 2039 and 2043, respectively.
· Eight currently listed U.S. patents for IGALMI in the United States Food and Drug Administration’s ("FDA") Approved Drug Products with Therapeutic Equivalence Evaluations (commonly known as the "Orange Book"), with two additional recently allowed patents that will be submitted for listing in the Orange Book once issued by the USPTO.
OnkosXcel Therapeutics
· Announced late-breaking abstract selected for presentation at 2024 ASCO (Free ASCO Whitepaper) Annual Meeting on preliminary findings from a Phase 2 investigator-sponsored trial of BXCL701 and KEYTRUDA (pembrolizumab) in metastatic pancreatic ductal adenocarcinoma (PDAC).
First Quarter 2024 Financial Results
Net Revenue: Net revenue from IGALMI was $582,000 for the first quarter of 2024, compared to $206,000 for the same period in 2023, representing a 182% increase. Sequential quarterly revenue increased 55% in Q1 2024 from the fourth quarter of 2023. The increased revenue for both periods was primarily attributable to increasing demand with existing customers, new customer orders, and volume-based contracting.
Research and Development (R&D) Expenses: R&D expenses were $11.4 million for the first quarter of 2024, compared to $27.8 million for the same period in 2023. The decreased expenses were primarily attributable to the wind-down of the SERENITY III and TRANQUILITY II and III trials, as well as decreased professional fees, personnel, and related costs.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $13.3 million for the first quarter of 2024, compared to $23.6 million for the same period in 2023. The reduced expenses were primarily attributable to a decrease in personnel and costs associated with the commercialization of IGALMI compared to the first quarter of 2023. The reduced expenses were partially offset by increased professional fees in the first quarter of 2024.
Net Loss: BioXcel Therapeutics had a net loss of $26.8 million for the first quarter of 2024, compared to a net loss of $52.8 million for the same period in 2023. The Company used $17.7 million in operating cash during the first quarter of 2024.
Cash and cash equivalents totaled $74.1 million as of March 31, 2024. This includes the $25 million from the registered direct offering announced on March 25, 2024.
Conference Call and Webcast
BioXcel Therapeutics will host a conference call and webcast today, May 9, 2024, at 8:00 a.m. ET to discuss its first quarter 2024 financial results. To access the call, please dial 877-407-5795 or 201-689-8722. A live webcast will be available on the Investors section of the corporate website, bioxceltherapeutics.com, and a replay will be available through August 9, 2024.
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 sign up to automatically receive email alerts and other information about the Company by visiting the "Email Alerts" option under the News/Events section of the Investors & Media website section and submitting your email address.
*SERENITY At-Home represents the redesigned SERENITY III trial.
About IGALMI (dexmedetomidine) sublingual film
INDICATION
IGALMI (dexmedetomidine) sublingual film is a prescription medicine, administered under the supervision of a health care provider, that is placed under the tongue or behind the lower lip and is used for the acute treatment of agitation associated with schizophrenia and bipolar disorder I or II in adults. The safety and effectiveness of IGALMI has not been studied beyond 24 hours from the first dose. It is not known if IGALMI is safe and effective in children.
IMPORTANT SAFETY INFORMATION
IGALMI can cause serious side effects, including:
· Decreased blood pressure, low blood pressure upon standing, and slower than normal heart rate, which may be more likely in patients with low blood volume, diabetes, chronic high blood pressure, and older patients. IGALMI is taken under the supervision of a healthcare provider who will monitor vital signs (like blood pressure and heart rate) and alertness after IGALMI is administered to help prevent falling or fainting. Patients should be adequately hydrated and sit or lie down after taking IGALMI and instructed to tell their healthcare provider if they feel dizzy, lightheaded, or faint.
· Heart rhythm changes (QT interval prolongation). IGALMI should not be given to patients with an abnormal heart rhythm, a history of an irregular heartbeat, slow heart rate, low potassium, low magnesium, or taking other drugs that could affect heart rhythm. Taking IGALMI with a history of abnormal heart rhythm can increase the risk of torsades de pointes and sudden death. Patients should be instructed to tell their healthcare provider immediately if they feel faint or have heart palpitations.
· Sleepiness/drowsiness. Patients should not perform activities requiring mental alertness, such as driving or operating hazardous machinery, for at least 8 hours after taking IGALMI.
· Withdrawal reactions, tolerance, and decreased response/efficacy. IGALMI was not studied for longer than 24 hours after the first dose. Physical dependence, withdrawal symptoms (e.g., nausea, vomiting, agitation), and decreased response to IGALMI may occur if IGALMI is used longer than 24 hours.
The most common side effects of IGALMI in clinical studies were sleepiness or drowsiness, a prickling or tingling sensation or numbness of the mouth, dizziness, dry mouth, low blood pressure, and low blood pressure upon standing.
These are not all the possible side effects of IGALMI. Patients should speak with their healthcare provider for medical advice about side effects.
Patients should tell their healthcare provider about their medical history, including if they suffer from any known heart problems, low potassium, low magnesium, low blood pressure, low heart rate, diabetes, high blood pressure, history of fainting, or liver impairment. They should also tell their healthcare provider if they are pregnant or breastfeeding or take any medicines, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Patients should especially tell their healthcare provider if they take any drugs that lower blood pressure, change heart rate, or take anesthetics, sedatives, hypnotics, and opioids.
Everyone is encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You can also contact BioXcel Therapeutics, Inc. at 1-833-201- 1088 or [email protected].
Please see full Prescribing Information at igalmi.com.
About BXCL501
In indications other than those approved by the U.S. Food and Drug Administration (FDA) as IGALMI (dexmedetomidine) sublingual film, BXCL501 is an investigational proprietary, orally dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. BioXcel Therapeutics believes that BXCL501 potentially targets an important mediator of agitation, and the Company has observed anti-agitation results in multiple clinical trials across several neuropsychiatric disorders. BXCL501 is under investigation by BioXcel Therapeutics for the acute treatment of agitation associated with dementia due to probable Alzheimer’s disease and for the acute treatment of agitation associated with bipolar I or II disorder or schizophrenia in the at-home setting. The safety and efficacy of BXCL501 for these investigational uses have not been established. BXCL501 has been granted Breakthrough Therapy designation by the FDA for the acute treatment of agitation associated with dementia and Fast Track designation for the acute treatment of agitation associated with schizophrenia, bipolar disorders, and dementia.
About BXCL701
BXCL701 is an investigational, oral innate immune activator designed to initiate inflammation in the tumor microenvironment. Approved and experimental immunotherapies often fail to address cancers that appear "cold." Therefore, BXCL701 is being evaluated to determine if it can render "cold" tumors "hot," making them more detectable by the adaptive immune system and thereby facilitating the development of a strong anticancer immune response. OnkosXcel Therapeutics’ preclinical data support BXCL701’s potential synergy with both current checkpoint inhibitors and emerging immunotherapies directed to activate T-cells. BXCL701 is currently being developed as a potential therapy for the treatment of aggressive forms of prostate cancer and advanced solid tumors that are refractory or treatment naïve to checkpoint inhibitors. BXCL701 has received Orphan Drug Designation from the U.S. Food and Drug Administration in four indications: acute myelogenous leukemia, pancreatic cancer, stage IIb to IV melanoma, and soft tissue sarcoma. The U.S. Food and Drug Administration (FDA) designated as a Fast Track development program the investigation of BXCL701 in combination with a checkpoint inhibitor for treatment of patients with metastatic small cell neuroendocrine prostate cancer (SCNC) with progression on chemotherapy and no evidence of microsatellite instability. An 800+-subject clinical database, with data collected by the Company and others, supports the ongoing development of BXCL701.