subslover
7 months ago
BioXcel Therapeutics Receives FDA Fast Track Designation for BXCL701 for Treatment of Small Cell Neuroendocrine Prostate Cancer (SCNC)
Designation for BXCL701 in combination with a checkpoint inhibitor (CPI) for treatment of patients with metastatic SCNC with progression on chemotherapy and no evidence of microsatellite instability
BXCL701 is an investigational, oral innate immune activator designed to inflame the tumor microenvironment and augment CPI activity
Company to discuss registration path at upcoming meeting with FDA
NEW HAVEN, Conn., Feb. 12, 2024 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience and immuno-oncology, today announced that the U.S. Food and Drug Administration (FDA) has designated as a Fast Track development program the investigation of BXCL701 in combination with a CPI for the treatment of patients with metastatic small cell neuroendocrine prostate cancer (SCNC) with progression on chemotherapy and no evidence of microsatellite instability. The FDA grants Fast Track designation to facilitate the development and expedite the review of medicines to treat serious conditions, fill unmet medical needs, and bring promising medicines to patients more quickly. Therapies granted this designation are given the opportunity for more frequent interactions with the FDA, a rolling review, and potential eligibility for accelerated approval and priority review.
“The FDA’s Fast Track designation for the investigation of BXCL701 in SCNC is an important recognition of our most advanced immuno-oncology asset and an acknowledgment of its potential to address the considerable unmet medical need in these patients. At the same time, it further validates the unique AI-based drug re-innovation approach that we used to discover this asset,” said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. “BXCL701 has already demonstrated considerable potential in our clinical trials to date, and we plan to further define its development path while exploring strategic options for our OnkosXcel Therapeutics subsidiary.”
SCNC, classified as a “cold” tumor, represents an underserved, growing patient population, with cases increasing due to earlier and more widespread use of androgen receptor inhibitors. In 2024, the American Cancer Society estimates that 299,0101 men will be diagnosed with prostate cancer in the United States, with approximately 20% expected to progress to the more aggressive metastatic castration-resistant form, including an estimated 11,960 patients expected to progress to SCNC2.
“SCNC is characterized by poor prognosis and a low survival rate, and current treatment options are suboptimal,” said Vincent J. O’Neill, M.D., Executive Vice President, Chief of Product Development and Medical Officer of BioXcel Therapeutics. “We are encouraged by the potential of BXCL701, which has demonstrated clinical proof of concept in both SCNC and adenocarcinoma. Following the positive survival results from our Phase 2 trial that we reported at the end of last year, we look forward to further discussing the registration path at an upcoming meeting with the FDA.”
jgrabar
10 months ago
https://grabarlaw.com/the-latest/bioxcel-shareholder-investigation/
A recently filed securities fraud class action complaint alleges that BioXcel Therapeutics Inc. (NASDAQ: BTAI), via certain officers and directors, made materially false and/or misleading statements, as well as failed to disclose material adverse facts about the Company’s business, operations, and prospects. Specifically, the complaint alleges Defendants failed to disclose to investors: (1) that the Company lacked adequate internal controls over protocol adherence and data integrity; (2) that, as a result, the Company’s principal investigator failed to adhere to the informed consent form approved by the Institutional Review Board; (3) that the Company’s principal investigator failed to maintain adequate case histories for certain patients whose records were reviewed by the FDA; (4) that the Company’s principal investigator fabricated email correspondence with a pharmacovigilance safety vendor that was then provided to the FDA; (5) that the foregoing would negatively impact the Company’s ability to obtain regulatory approval of BXCL501 for the treatment of agitation associated with dementia in patients with probable Alzheimer’s disease; and (6) that, as a result of the foregoing, Defendants’ positive statements about the Company’s business, operations, and prospects were materially misleading and/or lacked a reasonable basis.
Current BioXcel shareholders who have held BioXcel stock since prior to December 15, 2021, can seek corporate reforms, the return of funds spent defending litigation back to the company, and a court approved incentive award at no cost to them whatsoever.
realfast95
1 year ago
BioXcel Therapeutics' shares slumped Thursday after warning that prospects for regulatory approval for its investigational acute treatment for agitation in Alzheimer's patients could be hampered by a fraud investigation at a big study site.
In morning trading, the biopharmaceutical company's shares were 59% lower at $7.31 , having earlier in the day hit a fresh 52-week low of $7.09 . The stock is now down 66% so far this year..
BioXcel reported positive topline results from a late-stage trial that could support the use of BXCL501, an orally dissolving film formulation of dexmedetomidine, as a potential treatment for acute episodes of agitation in patients with mild to moderate Alzheimer's disease.
However, in a regulatory filing with the Securities and Exchange Commission , the company said it had confirmation that the principal investigator at a site that enrolled about 40% of the subjects in a Phase 3 clinical trial allegedly fabricated email correspondence. That came after BioXcel said it launched an investigation after learning in May the principal investigator in the so-called Tranquility II trial may have made up email correspondence purporting to demonstrate that the it timely submitted to the company's pharmacovigilance safety vendor a report of a serious adverse event from a different subject than one cited in a FDA form and purporting to show that the vendor had confirmed receipt.
The Food and Drug Administration inspected the clinical trial site in December, after which it issued a form identifying three observations related to the principal investigator's failure to adhere to an approved informed consent form for a limited number of subjects whose records the FDA reviewed, maintain adequate case histories for certain patients, and adhere to the investigational plan in certain instances.
BioXcel in its filing said it was made aware that fabricated email correspondence was provided to the FDA by the principal investigator's employer during the on-site inspection in December, and after unblinding the data it determined that the serious adverse event that was the subject of the correspondence between the principal investigator and pharmacovigilance vendor occurred in a subject in the placebo arm. It said the investigator hasn't participated in any other clinical trial sponsored or conducted by the company.
Meanwhile, BioXcel said topline results from the trial of BXCL501 were positive and the study met its primary efficacy endpoint. The 60 mcg dose also met the first key secondary endpoint of reducing agitation symptoms at one hour during the first episode of agitation, but didn't meet the other key secondary endpoint of change from baseline in PEC score at 30 minutes, the company said.
realfast95
1 year ago
BioXcel Shares Drop Premarket Following Results for Phase 3 Trial of BXCL501
6:58 am ET June 29, 2023 (Dow Jones) Print
By Robb M. Stewart
BioXcel Therapeutics' shares were down sharply ahead of the bell after a late-stage trial of the biopharmaceutical company's investigation treatment for Alzheimer's disease-related agitation met its main target but missed one of the key secondary goals.
In premarket trading, the shares were 36% lower after ending Wednesday at $17.67, down 18% so far this year.
BioXcel said the topline results from its Phase 3 trial of BXCL501, an orally dissolving film formulation of dexmedetomidine, were positive and the study met its primary efficacy endpoint. The 60 mcg dose also met the first key secondary endpoint of reducing agitation symptoms at one hour during the first episode of agitation, but didn't meet the other key secondary endpoint of change from baseline in PEC score at 30 minutes, the company said.
Efficacy for the dose was supported by a number of secondary measures, and most patients responded to the first 60 mcg dose and were determined to be "very much" or "much improved" compared to 50% with placebo, BioXcel. It added the primary endpoint wasn't met for the 40 mcg dose.
Chief Executive Vimal Mehta said the results represent a milestone, and data from the Tranquility II trial show that BXCL501 has the potential to treat acute episodes of agitation in patients with mild to moderate Alzheimer's disease, if approved.
BioXcel said it plans to develop a path to potential supplemental new drug application submission for the acute treatment of agitation associated with Alzheimer's disease in the second half of the year, subject to further discussions with the Food and Drug Administration.
BXCL501 has been granted a breakthrough therapy designation for the acute treatment of dementia-related agitation based on Tranquility I data.
MDizzle
2 years ago
BioXcel Therapeutics to Ring Nasdaq Stock Market Closing Bell Today
NEW HAVEN, Conn., Feb. 21, 2023 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, today announced that Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics, additional members of the management team, and other BioXcel team members will ring the closing bell of the Nasdaq Stock Market today, Tuesday, February 21, 2023.
"At BioXcel Therapeutics, we are building a disruptive biopharmaceutical business model to successfully drive accelerated AI-enabled drug development and commercialization," said Dr. Mehta. "Today we are celebrating a year of significant accomplishments that include FDA approval and launch of our first product, IGALMI(TM) sublingual film, for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults. In addition, we look forward to market-expansion opportunities for our overall neuropsychiatric program, with two pivotal study readouts planned for BXCL501 in the first half of 2023. Recently, we also reported positive Phase 2 data for our lead immuno-oncology program, BXCL701, in combination with KEYTRUDA(R), which is in development for the treatment of a rare, aggressive form of prostate cancer. As BioXcel advances its lead drug candidates in two therapeutic areas, we appreciate Nasdaq's recognition of our progress through today's closing bell ceremony."
The ceremony will take place at the Nasdaq MarketSite, 4 Times Square, New York, beginning at 3:45 PM ET. It can be viewed at https://livestream.com/accounts/27896496/events/10752444.
MDizzle
2 years ago
First Patients Dosed in SERENITY III Phase 3 Trial for Acute Treatment of Agitation in Adults with Bipolar I or II Disorder or Schizophrenia
BioXcel Therapeutics Announces First Patients Dosed in SERENITY III Phase 3 Trial for Acute Treatment of Agitation in Adults with Bipolar I or II Disorder or Schizophrenia
SERENITY III will evaluate the efficacy and safety of BXCL501 for at-home use
Top-line pivotal data expected in 1H 2023
Estimated 23 million annual agitation episodes in the home-setting would more than double current market opportunity for BXCL501 in the U.S. (1) (-4)
NEW HAVEN, Conn., Nov. 30, 2022 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, today announced that the first 13 patients have been dosed in Part 1 of the pivotal Phase 3 SERENITY III trial investigating at-home use of BXCL501 (dexmedetomidine) sublingual film, the Company's proprietary, orally dissolving film, for agitation associated with bipolar I or II disorder or schizophrenia.
"Evaluating BXCL501 for at-home use is an exciting and important milestone that potentially expands the market opportunity for the treatment of agitation and drives the growth of our neuroscience franchise," said Robert Risinger, M.D., Chief Medical Officer, Neuroscience of BioXcel Therapeutics. "We anticipate top-line data from SERENITY III as well as our TRANQUILITY II trial for Alzheimer's-related agitation in the first half of 2023. These two near-term pivotal data readouts further reinforce the potential of BXCL501 to address the unmet medical needs of millions of patients."
The treatment of agitation represents a significant market opportunity. There are approximately 39 million reported agitation episodes that occur in the U.S. each year related to bipolar disorders and schizophrenia.(1) (-4) 23 million of these episodes occur outside of the institutional setting, potentially more than doubling the current market opportunity for BXCL501.
SERENITY III is a two-part, double-blinded, placebo-controlled pivotal study designed to evaluate BXCL501 60mcg dose for at-home use. This strategic trial design follows a Type B meeting with the U.S. Food and Drug Administration and observed dose-dependent responses in a prior Phase 1/2b study assessing a range of doses. The first part of the study is similar to SERENITY I and II and designed to assess the efficacy and safety of a 60mcg dose in acutely agitated patients with bipolar disorder or schizophrenia in a monitored setting. The primary efficacy endpoint is the change from baseline in Positive and Negative Syndrome Scale-Excitatory Component (PEC) total score at two hours after dosing compared to placebo. In Part 1, approximately 200 patients will be enrolled at up to approximately 20 clinical sites in the U.S. The second part of the study is designed to assess the safety of 60mcg dose compared to placebo when self-administered at home. SERENITY III will utilize many of the same investigators and clinical sites as SERENITY I and II.
realfast95
2 years ago
BioXcel Therapeutics Reports Second Quarter 2022 Financial Results and Recent Operational Highlights
August 09 2022 - 07:00AM
BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a commercial-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, today announced its financial results for the second quarter ended June 30, 2022 and provided an update on key strategic initiatives.
“BioXcel Therapeutics made tremendous progress in its journey to becoming a fully integrated AI-driven commercial-stage company with the potential to transform the agitation treatment landscape,” said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. “This transformation is being driven by the continued execution of our land and expand strategy within our neuroscience franchise. We are focused on the commercial launch for our recently FDA approved drug IGALMI™ while significantly increasing the opportunity for BXCL501 through at-home, medical setting expansion and the pursuit of multiple additional indications for our BXCL501 franchise. We believe we are well-positioned and have laid a strong foundation to drive long-term sustainable growth.”
Company Highlights
Neuroscience Franchise
IGALMI™ (dexmedetomidine) sublingual film
IGALMI was approved by the U.S. Food and Drug Administration (FDA) on April 5, 2022 for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults.2 Within these two patient populations in the U.S., up to 25 million agitation episodes occur each year.3-5
Trade and Market Access: Key focus on unlocking access and supporting unencumbered demand generation for IGALMI.
IGALMI made available in 120mcg and 180mcg doses through the Company’s third-party logistics provider and for order through wholesalers as of early July.
Engagement underway with 59 high-value Integrated Delivery Networks (IDNs) and their affiliated hospitals.
Discussions and negotiations ongoing with major national Group Purchasing Organizations (GPOs), representing over 90% of beds in target hospitals.
Institutional Sales Force: Deployed first phase of our national sales force to target high-priority accounts in late May, with expansion of commercial footprint in process. Engaging multiple stakeholders in target hospitals and progressing P&T formulary review discussions. Gathered field level market intelligence which further reinforces and augments current and future commercial strategy.
Early response to the IGALMI value proposition exceeding expectations across full spectrum of institutional and healthcare stakeholders.
Market dynamics are rapidly evolving, favoring a less invasive and voluntary approach to treating agitation.
Marketing: Strategic marketing focused on branded campaigns to support sales force activities.
Peer-to-peer speaker programs educating healthcare providers (HCPs) of the importance of early intervention to mitigate agitation episodes.
Digital marketing efforts driving significant engagement resulting in over 100,000 visits to IGALMI HCP website.
Investigating Potential Expansion of BXCL501 for At-Home Use for Agitation Associated with Bipolar Disorders and Schizophrenia: Planning SERENITY III double-blinded, placebo-controlled, pivotal study designed to evaluate BXCL501 60mcg dose for at-home use. This strategic trial decision follows successful completion of Type B meeting with FDA and observed dose-dependent responses in a prior Phase 1/2b study assessing 60mcg, 80mcg, 120mcg, and 180mcg doses. Of the approximately 25 million agitation episodes that occur in the U.S. each year related to schizophrenia and bipolar disorders, approximately one-third occur outside of the institutional setting.6
SERENITY III will consist of two parts:
First part of SERENITY III is similar to the SERENITY I and II pivotal trials and designed to assess efficacy and safety in acutely agitated bipolar and schizophrenia patients.
Primary efficacy endpoint is change from baseline in Positive and Negative Syndrome Scale-Excitatory Component (PEC) total score at two hours after dosing compared to placebo.
Second part of SERENITY III designed to assess safety compared to placebo when self-administered at home.
SERENITY III will utilize many of the same investigators and clinical sites as SERENITY I and II and is expected to initiate in 2H 2022.
Clinical Pipeline
BXCL501, a proprietary, sublingual film formulation of dexmedetomidine, has received Breakthrough Therapy and Fast Track designation for the acute treatment of agitation associated with dementia.
Indication Expansion
Alzheimer’s Disease-related Agitation: TRANQUILITY program is designed to capture Alzheimer’s-related agitation market opportunity. There are an estimated 100 million agitation episodes in Alzheimer’s patients occurring in the U.S. annually.7
TRANQUILITY II: top-line data readout expected in 1H 2023.
TRANQUILITY III: enrollment expected to begin in 2H 2022.
Adjunctive Treatment for Major Depressive Disorder (MDD): Ongoing Phase 1 trial evaluating BXCL501 daily dosing designed to inform dose selection in future proof-of-concept study evaluating daily BXCL501 dosing in MDD patients. Over 300 million antidepressant prescriptions are filled annually in the U.S. and current treatments are limited by slow onsets of action and incomplete responses.8
Double-blind, placebo-controlled, multiple ascending dose (MAD) selection trial in healthy volunteers includes two cohorts of 18 patients, each having completed seven days of daily dosing of 30mcg or 60mcg compared to placebo.
Dose escalation in current cohort also enrolling to evaluate 80mcg.
Additional cohorts planned to evaluate safety and assess tolerability of a range of doses administered once daily and twice daily.
Top-line results expected in 1H 2023.
OnkosXcel Therapeutics
Established a wholly owned subsidiary to focus on the sustained expansion and optimization of the Company’s immuno-oncology (I-O) franchise, including its most advanced I-O program, BXCL701. BXCL701 is an investigational orally administered, systemic innate immune activator in development for the treatment of aggressive forms of prostate cancer.
Strategic Advancements: Fully functioning subsidiary dedicated to executing focused strategy and developing innovative oncology pipeline. Evaluating strategic options, which may include third party investments, aimed to fully capture unique value creation opportunity in areas of high unmet medical need.
Metastatic Castration-Resistant Prostate Cancer (mCRPC) Program: Continued ongoing Phase 2 trial for BXCL701 in combination with KEYTRUDA® (pembrolizumab) in mCRPC patients with small cell neuroendocrine carcinoma (SCNC) or adenocarcinoma phenotype.
Expect to complete enrollment of 28-patient SCNC cohort in 2H 2022.
Continued enrollment in adenocarcinoma randomized trial expansion evaluating BXCL701 monotherapy vs. BXCL701-KEYTRUDA combination therapy.
Corporate Updates
Strengthened Board of Directors: Appointed Michael P. Miller to the Company’s Board of Directors, to provide strategic leadership and commercial growth expertise.
Enhanced Intellectual Property: Received two Notices of Allowance for patents (issued from patent application numbers 17/560,392 and 17/560,423) related to IGALMI to cover film formulations containing dexmedetomidine and methods of treating agitation using the films.
Second Quarter 2022 Financial Results
Research and Development Expenses: Research and development expenses were $17.9 million for the second quarter of 2022, compared to $13.5 million for the same period in 2021. The increased expenses were primarily attributable to clinical trial costs related to the Company’s TRANQUILITY program.
Selling, General and Administrative Expenses: Selling, general and administrative expenses were $18.4 million for the second quarter of 2022, as compared to $14.1 million for the same period in 2021. The increase was primarily due to personnel and costs related to the launch of IGALMI in the U.S.
Net Loss: BioXcel Therapeutics reported a net loss of $37.7 million for the second quarter of 2022, compared to a net loss of $27.6 million for the same period in 2021.
As of June 30, 2022, cash and cash equivalents totaled approximately $233.5 million. This excludes $30 million of contributions from the $260 million strategic financing announced in April. To date the Company has met the milestones and has received $100 million from the agreement.
Conference Call
BioXcel Therapeutics will host a conference call and webcast August 9, 2022, at 8:30 a.m., ET, to discuss its second quarter 2022 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 November 9, 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.
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