axelvento
3月前
Experimental therapy shows promise in pancreatic cancer clinical trial-
It acts by inflaming the microenvironment surrounding the tumour and thereby augmenting the activity of immunotherapies like pembrolizumab. BXCL701 has received Orphan Drug Designation from the U.S. Food and Drug Administration in pancreatic cancer and three other cancer types: acute myelogenous leukaemia, stage IIb to IV melanoma, and soft tissue sarcoma.
hree patients had significant reductions in a marker that indicates the presence of pancreatic cancer, CA19-9, with 100%, 97% and 73% reductions. Weinberg says that CA19-9 is the best marker they have in pancreatic cancer, but it is not as good a screening test as PSA is for prostate cancer. He believes, however, that it can be useful for monitoring disease activity in patients with advanced cancers.
“Historically, no one has responded to immunotherapy in pancreatic cancer so even one response and another with stable disease could be early signs of efficacy,” says Weinberg. “A third patient had an initial drop in their CA19-9 marker even though they had disease progression on imaging, and this may be more early evidence that this drug combination has anti-cancer effects.”
https://ecancer.org/en/news/24822-asco-2024-experimental-therapy-shows-promise-in-pancreatic-cancer-clinical-trial
subslover
11月前
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
1年前
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
2年前
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
2年前
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年前
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年前
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.
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