Latest news with #DSMB
Yahoo
27-05-2025
- Business
- Yahoo
BioXcel Therapeutics Receives Positive Recommendation from Data Safety Monitoring Board (DSMB) to Continue SERENITY At-Home Pivotal Phase 3 Safety Trial for Acute Treatment of Agitation Associated with Bipolar Disorders or Schizophrenia
DSMB recommended the continuation of trial as planned Topline data expected in Q3 2025 NEW HAVEN, Conn., May 27, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced that an independent Data Safety Monitoring Board (DSMB) recommended that the SERENITY At-Home pivotal Phase 3 safety trial of BXCL501 for acute treatment of agitation associated with bipolar disorders or schizophrenia continue without modification. The DSMB recommendation followed a review of unblinded safety data from the first 115 patients dosed as of the May 2, 2025 cutoff date. The trial is fully enrolled and collection of data over the 12-week period is continuing. 'We are pleased with the favorable DSMB meeting outcome and are excited about the upcoming data readout for our first at-home trial with BXCL501,' said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. 'Results are intended to help support a potential sNDA submission for label expansion of IGALMI® in the at-home setting — a sizeable unmet medical need given there are no FDA-approved therapies for bipolar or schizophrenia-related agitation in this environment.' The SERENITY At-Home Phase 3 trial is designed as a double-blind, placebo-controlled study to evaluate the safety of a 120 mcg dose of BXCL501 in 200 patients for acute treatment of agitation associated with bipolar disorders or schizophrenia in the at-home setting. Trial enrollment is complete: More than 205 patients have been dosed. More than 150 patients have received multiple doses for agitation over the 12-week trial period. Topline data expected in Q3 2025. Additional information on the SERENITY At-Home trial is included in a corporate presentation in the Investors section of the Company's website: About BXCL501Outside of its approved indication by the U.S. Food and Drug Administration as IGALMI® (dexmedetomidine) sublingual film, BXCL501 is an investigational proprietary, orally dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. BXCL501 is under investigation by BioXcel Therapeutics for the acute treatment of agitation associated with Alzheimer's dementia 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 the SERENITY At-Home Phase 3 TrialThe SERENITY At-Home Phase 3 trial is a double-blind, placebo-controlled study designed to evaluate the safety of a 120 mcg dose of BXCL501 for the acute treatment of agitation associated with bipolar disorders or schizophrenia in the at-home setting. The trial is designed to evaluate 200 patients with a history of agitation episodes residing at home either alone or with caregivers/informants. Patients are self-administering 120 mcg of BXCL501 or placebo when agitation episodes occur over the 12-week trial period, with safety data (adverse events) collected during the trial. In addition, patients or caregivers/informants will complete a modified global impression of severity (mCGIs) and a clinical global impression of change (mCGI-C) two hours after dosing as an exploratory endpoint to evaluate use in the outpatient setting. 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 or call 1-800-FDA-1088. You can also contact BioXcel Therapeutics, Inc. at 1-833-201-1088 or medinfo@ Please see full prescribing information at About BioXcel Therapeutics, Therapeutics, Inc. (Nasdaq: BTAI) is a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience. Its wholly owned subsidiary, OnkosXcel Therapeutics, is focused on the development of medicines in immuno-oncology. The Company's drug re-innovation approach leverages existing approved drugs and/or clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic indications. For more information, please visit Forward-Looking StatementsThis press release includes 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended and Section 21E of the Securities Exchange Act of 1934, as amended. All statements contained in this press release other than statements of historical fact should be considered forward-looking statements, including, without limitation, statements related to: the Company's planned advancement of its SERENITY trial; potential market opportunity for BXCL501; completing enrollment and release of topline data from the ongoing SERENITY trial; submission of an sNDA; the potential for the results from the Company's completed, ongoing and proposed clinical trials to support regulatory approvals for its product candidates. When used herein, words including 'anticipate,' 'believe,' 'can,' 'continue,' 'could,' 'designed,' 'estimate,' 'expect,' 'forecast,' 'goal,' 'intend,' 'may,' 'might,' 'plan,' 'possible,' 'potential,' 'predict,' 'project,' 'should,' 'target,' 'will,' 'would' and similar expressions are intended to identify forward-looking statements, though not all forward-looking statements use these words or expressions. 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 the Company's current expectations and various assumptions. The Company believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. The Company 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; the impact of the reprioritization; its significant indebtedness, ability to comply with covenant obligations and potential payment obligations related to such indebtedness and other contractual obligations; the Company has identified conditions and events that raise substantial doubt about its ability to continue as a going concern; its limited experience in drug discovery and drug development; risks related to the TRANQUILITY program; its dependence on the success and commercialization of IGALMI®, BXCL501, BXCL502, BXCL701 and BXCL702 and other product candidates; the number of episodes of agitation and the size of the Company's total addressable market may be overestimated, and approval that the Company may obtain may be based on a narrower definition of the patient population; its lack of experience in marketing and selling drug products; the risk that IGALMI or the Company's product candidates may not be accepted by physicians or the medical community in general; the Company still faces extensive and ongoing regulatory requirements and obligations for IGALMI; 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 the Company's product candidates; its novel approach to the discovery and development of product candidates based on EvolverAI; the significant influence of and dependence on BioXcel LLC; its exposure to patent infringement lawsuits; its reliance on third parties; its ability to comply with the extensive regulations applicable to it; impacts from data breaches or cyber-attacks, if any; risks associated with the increased scrutiny relating to environmental, social and governance (ESG) matters; risks associated with federal, state or foreign health care 'fraud and abuse' laws; and its ability to commercialize its product candidates, as well as the important factors discussed under the caption 'Risk Factors' in its Annual Report on Form 10-K for the fiscal year ended December 31, 2024, as such factors may be updated from time to time in its other filings with the SEC, which are accessible on the SEC's website at and the Investors section of the Company's website at 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 management's estimates as of the date of this press release. While the Company 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 the Company's views as of any date subsequent to the date of this press release. Contact Information Corporate/InvestorsBioXcel Therapeutics Erik Kopp 1.203.494.7062 MediaRusso PartnersDavid Schull 1.858.717.2310 Source: BioXcel Therapeutics, is a registered trademark of BioXcel Therapeutics, in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Insider
14-05-2025
- Business
- Business Insider
Belite Bio reports Q1 EPS (45c), consensus (37c)
As of March 31, 2025, the Company had $157.4 million in cash, liquidity funds, time deposits, and U.S treasury bills. 'We continue to advance the clinical development of Tinlarebant, reaching a major milestone with the favorable interim analysis of our Phase 3 DRAGON trial earlier this year,' said Dr. Tom Lin, Chairman and CEO of Belite Bio (BLTE). 'We are excited by the encouraging feedback from the DSMB on the safety and efficacy outcomes in DRAGON as we work toward trial completion by the end of 2025. We are focused on maintaining strong execution across our late-stage clinical programs as we aim to deliver new treatment options for people living with degenerative retinal diseases, where there is significant unmet need.' Protect Your Portfolio Against Market Uncertainty
Yahoo
14-05-2025
- Business
- Yahoo
Belite Bio Reports First Quarter 2025 Financial Results and Provides Corporate Update
Following a pre-specified interim analysis, an independent Data Safety Monitoring Board (DSMB) recommended the pivotal Phase 3 trial (DRAGON) of Tinlarebant in adolescent Stargardt disease (STGD1) patients proceed without any modification; trial completion expected Q4 2025 (including a three-month follow-up period) DSMB also recommended the Company submit the interim data for further regulatory review for drug approval A pivotal global Phase 3 trial (PHOENIX) of Tinlarebant in geographic atrophy (GA) patients is ongoing with 464 of targeted 500 subjects enrolled Raised $15 million in gross proceeds in a registered direct offering on February 5, 2025 Conference call and webcast on Wednesday, May 14, 2025, at 4:30 p.m. ET SAN DIEGO, May 13, 2025 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced its financial results for the first quarter ended March 31, 2025, and provided a business update. 'We continue to advance the clinical development of Tinlarebant, reaching a major milestone with the favorable interim analysis of our Phase 3 DRAGON trial earlier this year,' said Dr. Tom Lin, Chairman and CEO of Belite Bio. 'We are excited by the encouraging feedback from the DSMB on the safety and efficacy outcomes in DRAGON as we work toward trial completion by the end of 2025. We are focused on maintaining strong execution across our late-stage clinical programs as we aim to deliver new treatment options for people living with degenerative retinal diseases, where there is significant unmet need.' First Quarter 2025 Business Highlights and Upcoming Milestones: Clinical Highlights Tinlarebant is an oral, once-daily, potent retinol binding protein 4 (RBP4) antagonist that decreases RBP4 levels in the blood and reduces vitamin A (retinol) delivery to the eye without disrupting systemic retinol delivery to other tissues. Vitamin A is critical for normal vision but can accumulate as toxic byproducts (bisretinoids) in individuals affected with STGD1 and GA, the advanced form of dry age-related macular degeneration (AMD), leading to retinal cell death and loss of vision. Stargardt disease (STGD1): Accumulation of cytotoxic bisretinoids compounds has been implicated in the onset and progression of STGD1, for which there are no approved treatments. Tinlarebant has been granted Fast Track and Rare Pediatric Disease Designations in the U.S.; Orphan Drug Designation in the U.S., Europe, and Japan; and Sakigake (Pioneer Drug) Designation in Japan for the treatment of STGD1. DRAGON Trial: Ongoing, 24-month, randomized (2:1, active: placebo), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in adolescent STGD1 patients Following a pre-specified interim analysis, an independent DSMB recommended trial continuation without modifications, maintaining a sample size of 104 subjects In addition, the DSMB recommended submitting the data for further regulatory review for drug approval Primary efficacy endpoint is the growth rate of atrophic lesions; safety and tolerability will also be assessed Trial completion expected by Q4 2025 (including a three-month follow-up period) DRAGON II Trial: Combination of a Phase 1b open-label trial to evaluate the pharmacokinetics and pharmacodynamics of Tinlarebant in adolescent Japanese STGD1 patients and a Phase 2/3, 24-month, randomized (1:1, active: placebo), double-masked, placebo-controlled, multicenter trial in adolescent STGD1 patients Enrolled 16 subjects in the Phase 2/3 trial, with a target enrollment of approximately 60 subjects, aged 12 to 20 years old, including approximately 10 Japanese subjects; data from the Japanese subjects is intended to facilitate a future new drug application in Japan Primary efficacy endpoint is the growth rate of atrophic lesions; safety and tolerability will also be assessed Geographic Atrophy (GA): GA is a chronic degenerative disease of the retina that leads to blindness in the elderly. Accumulation of cytotoxic vitamin A byproducts (bisretinoids) has been implicated in the progression of GA. There are currently no FDA-approved, orally administered treatments for GA. PHOENIX Trial: Ongoing, 24-month, randomized (2:1, active: placebo), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in GA patients 464 of the targeted 500 subjects have been enrolled to date Primary efficacy endpoint is the growth rate of atrophic lesions; safety and tolerability will also be assessed Company expects to conduct an interim analysis Corporate Highlights In February 2025, Belite completed a registered direct offering priced at the market, raising gross proceeds of $15 million, with the potential for additional proceeds of approximately $15 million from the exercise of five-year warrants issued in the offering. First Quarter 2025 Financial Results: Current Assets: As of March 31, 2025, the Company had $157.4 million in cash, liquidity funds, time deposits, and U.S treasury bills. R&D Expenses: For the three months ended March 31, 2025, research and development expenses were $9.4 million compared to $6.8 million for the same period in 2024. The increase in research and development expenses was primarily attributable to (i) share-based compensation granted in the third quarter of 2024 and first quarter of 2025, (ii) slightly higher clinical trial expenses related to the PHOENIX trial. G&A Expenses: For the three months ended March 31, 2025, general and administrative expenses were $6.1 million compared to $1.6 million for the same period in 2024. The increase resulted primarily from an increase in share-based compensation granted in the third quarter of 2024 and first quarter of 2025. Other Income: For the three months ended March 31, 2025, other income was $1.2 million compared to $0.5 million for the same period in 2024. The increase in other income was attributable to accrued interest from time deposits and U.S. treasury bills. Net Loss: For the three months ended March 31, 2025, the Company reported a net loss of $14.3 million, compared to a net loss of $7.9 million for the same period in 2024. Webcast Information Belite Bio will host a webcast on Wednesday, May 14, 2025, at 4:30 p.m. Eastern Time to discuss the Company's financial results and provide a business update. To join the webcast, please visit A replay will be available for approximately 90 days following the event at the Company's Investor Relations website at About Belite Bio Belite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical need, such as Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD), in addition to specific metabolic diseases. Belite's lead candidate, Tinlarebant, an oral therapy intended to reduce the accumulation of toxins in the eye, is currently being evaluated in a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) in adolescent STGD1 patients and a Phase 3 study (PHOENIX) in patients with GA. For more information, follow us on X, Instagram, LinkedIn, Facebook or visit us at Important Cautions Regarding Forward Looking Statements This press release contains forward-looking statements about future expectations and plans, as well as other statements regarding matters that are not historical facts. These statements include but are not limited to statements regarding the potential implications of clinical data for patients, and Belite Bio's advancement of, and anticipated preclinical activities, clinical development, regulatory milestones, and commercialization of its product candidates, and any other statements containing the words 'expect', 'hope' and similar expressions. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including but not limited to Belite Bio's ability to demonstrate the safety and efficacy of its drug candidates; the clinical results for its drug candidates, which may not support further development or regulatory approval; the timing to complete relevant clinical trials and/or to receive the interim/final data of such clinical trials; the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approval of Belite Bio's drug candidates; the potential efficacy of Tinlarebant, as well as those risks more fully discussed in the 'Risk Factors' section in Belite Bio's filings with the U.S. Securities and Exchange Commission. All forward-looking statements are based on information currently available to Belite Bio, and Belite Bio undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law. BELITE BIO, INC UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (Amounts in thousands of US Dollars, except share and per share amounts) For the Three MonthsEnded March 31, 2024 2025 Expenses Research and development 6,765 9,396 General and administrative 1,563 6,121 Total operating expenses 8,328 15,517 Loss from operations (8,328 ) (15,517 ) Other income: Total other income, net 463 1,240 Loss before income tax (7,865 ) (14,277 ) Income tax expense 6 - Net loss (7,871 ) (14,277 ) Other comprehensive income (loss) Foreign currency translation adjustments, net of nil tax (96 ) 18 Total comprehensive loss (7,967 ) (14,259 ) Weighted average number of ordinary shares used in per share calculation: - Basic and Diluted 29,677,173 32,084,106 Net loss per ordinary share - Basic and Diluted $ (0.27 ) $ (0.45 ) BELITE BIO, INC UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS (Amounts in thousands of US Dollars, except share amounts) December 31, March 31, 2024 2025 Current assets $ 147,073 $ 159,287 Other assets 5,059 4,914 TOTAL ASSETS $ 152,132 $ 164,201 TOTAL LIABILITIES $ 6,311 $ 6,131 TOTAL SHAREHOLDERS' EQUITY 145,821 158,070 TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY $ 152,132 $ 164,201 Ordinary shares authorized 400,000,000 400,000,000 Ordinary shares issued 31,857,802 32,595,001 Ordinary shares outstanding 31,826,549 32,544,784 Media and Investor Relations Contact: Jennifer Wu ir@ Julie Fallon belite@ in to access your portfolio
Yahoo
13-05-2025
- Business
- Yahoo
COSCIENS Biopharma Inc. Reports First Quarter 2025 Financial Results and Provides a Corporate Update
Repositioned as a pure-play natural-based product company following strategic review and pipeline prioritization Efforts continue to gain efficiencies through streamlining and cost cutting measures; Company ended the quarter with US$13.8 million in cash Advancing Phase 2 clinical trial with Avenanthramide product Appoints Global Consumer Products and Biosciences Executive, Anna Biehn as Chief Executive Officer TORONTO, ONTARIO, May 13, 2025 (GLOBE NEWSWIRE) -- COSCIENS Biopharma Inc. (NASDAQ: CSCI) (TSX: CSCI) ('COSCIENS' or the 'Company'), a life sciences company which develops and commercializes a diversified portfolio of cosmeceutical, nutraceutical and pharmaceutical products, today reported its financial and operating results for the first quarter ended March 31, 2025 and provided a corporate update. Pharmaceuticals: Avenanthramides Tablets in Clinical Development as an Anti-Inflammatory Product: During the first quarter of 2025, the Company announced successful Phase 1 results from the Phase 1 clinical study of its Avenanthramides tablets. That study involved 72 healthy subjects. There were no significant clinical adverse events observed from ascending doses ranging from 30 mg to 960 mg of the Company's Avenanthramides tablets in that Phase 1 clinical study. As a result of the successful completion of that Phase 1 clinical study, the Company launched a Phase 2a clinical efficacy study of its Avenanthramides tablets on March 15, 2025. That study includes 20 patients divided in two cohorts. The first cohort of 10 patients who received a daily dose of 480 mg is completed. Subsequent to the end of the first quarter, data from those 10 patients was reviewed by independent members of the Data and Safety Monitoring Board (DSMB). The DSMB recommended that the study continue as planned with a second cohort of 10 patients who will receive a daily dose of 960 mg. The Company's goal remains to complete the Phase 2a Clinical Efficacy Study during Q3, 2025. Nutraceuticals: The COSCIENS team is working to commercialize a new portfolio of nutraceuticals leveraging the deep understanding and expertise in the active ingredients category and marking an important chapter in the Company's journey toward promoting wellness while expanding its business model. Those include: Oat Beta Glucan (OBG) Chewable Bar – Cholesterol Reduction: We have successfully developed a unique, standardized formulation for a healthy confection which includes a high concentration of OBG with daily dosage according to approved OBG product monograph in 10 developed countries. Yeast Beta Glucan (YBG) Powder - Immune Booster: Our YBG product has been successfully manufactured as part of our PGX scale-up project in Edmonton, Alberta. Our YBG product is being finalized in capsule form with the goal to commercialize it as an immune booster. Technology: Pressurized Gas eXpanded Technology (PGX Technology): Edmonton Main Facility PGX Scale Up 50 Liters Vessel: The project is completed and the equipment is ready to produce YBG at a small-scale commercial level. Summary of First Quarter 2025 Financial Results All amounts are in U.S. Company had $13.8 million in cash and cash equivalents at March 31, the three-month period ended March 31, 2025, we reported a consolidated net loss of $3.7 million, or $1.16 loss per common share, as compared with a consolidated net loss of $1.4 million, or $0.76 loss per common share for the same period in 2024. The $2.3 million increase in net loss is primarily due to increases in operating expenses of $1.4 million, a $0.5 million decrease in gross margin, and a decrease of $0.4 million in income tax recoveries. Revenues Our total revenue for the three-month period ended March 31, 2025, was $1.5 million as compared to $2.1 million for the same period in 2024, a decrease of $0.6 million. This decrease was primarily due to a $0.7 million decrease in sales of Avenanthramides, Beta Glucan, and Oat Oil from prior period, offset by $0.1 million in Macrilen revenue. Operating Expenses Our total operating expenses for the three-month period ended March 31, 2025, were $4.0 million as compared with $2.7 million for the same period in 2024. This increase of $1.3 million was due to higher selling, general and administrative costs of $1.2 million due primarily to the merger completed in June 2024 between Aeterna (now COSCIENS) and Ceapro, as well as a slight increase in research and development costs of $0.1 million related mainly to timing and residual expenses associated with certain post-trial activities related to the DETECT trial. Since the merger, we have successfully reduced overall spend by leveraging restructuring initiatives and operational synergies. This is reflected in our quarter-over-quarter reduction in cash spend, decreasing from $3.6M in Q4 2024 to $2.6M in Q1 2025. Consolidated Financial Statements and Management's Discussion and Analysis For reference, the Management's Discussion and Analysis of Financial Condition and Results of Operations for the first quarter of 2025, as well as the Company's consolidated financial statements as of March 31, 2025, will be available on the Company's website ( in the Investors section or at the Company's SEDAR+ and EDGAR profiles at and respectively. About COSCIENS Biopharma is a life sciences company which develops and commercializes a diversified portfolio of cosmeceutical, nutraceutical and pharmaceutical products. Our technology includes proprietary extraction technology, which is applied to the production of active ingredients from renewable plant resources currently used in cosmeceutical products (i.e., oat beta glucan and avenanthramides which are found in leading skincare product brands like Aveeno and Burt's Bees formulations) and being developed as potential nutraceuticals and/or pharmaceuticals. Our consolidated portfolio also includes macimorelin (Macrilen®; Ghryvelin®), the first and only U.S. FDA and European Medicines Agency approved oral test indicated for the diagnosis of adult growth hormone deficiency ('AGHD'). The company is listed on the NASDAQ Capital Market and the Toronto Stock Exchange, and trades on both exchanges under the ticker symbol "CSCI". For more information, please visit COSCIENS' website at Statements Certain statements in this news release, referred to herein as "forward-looking statements", constitute "forward-looking statements" within the meaning of the United States Private Securities Litigation Reform Act of 1995, as amended, and "forward-looking information" under the provisions of Canadian securities laws. All statements, other than statements of historical fact, that address circumstances, events, activities, or developments that could or may or will occur are forward-looking statements. When used in this news release, words such as "anticipate", "assume", "believe", "could", "expect", "forecast", "future", "goal", "guidance", "intend", "likely", "may", "would" or the negative or comparable terminology as well as terms usually used in the future and the conditional are generally intended to identify forward-looking statements, although not all forward-looking statements include such words. Forward-looking statements in this news release include, but are not limited to, statements relating to: our plans for the Phase 2a Clinical Efficacy Study of its Avenanthramides tablets, our goal to commercialize our nutraceutical products (including our OBG chewable bar and YBG powder), the potential of our PGX technology and our goals and expectations regarding our other plans related to the development, manufacture or commercialization of our products. Forward-looking statements are necessarily based upon a number of factors and assumptions that, while considered reasonable by the Company as of the date of such statements, are inherently subject to significant business, economic, operational and other risks, uncertainties, contingencies and other factors, including those described below, which could cause actual results, performance or achievements of the combined Company to be materially different from results, performance or achievements expressed or implied by such forward-looking statements and, as such, undue reliance must not be placed on them. Forward-looking statements involve known and unknown risks and uncertainties which include, among others: the combined Company's present and future business strategies; operations and performance within expected ranges; anticipated future cash flows; local and global economic conditions and the environment in which the combined Company operates; anticipated capital and operating costs; uncertainty in our revenue generation from our marketed products, product development and related clinical trials and validation studies; results from our products under development may not be successful or may not support advancing the product; the failure of the DETECT-trial to achieve its primary endpoint in children (CGHD) may impact the market for macimorelin (Macrilen®; Ghryvelin®) in adults (AGHD) and the existing relationships we have for that product; ability to raise capital and obtain financing to continue our currently planned operations; our now heavy dependence on sales by and revenue from our main distributor of our legacy Ceapro products and its customers, the continued availability of funds and resources to successfully commercialize our products; the ability to secure strategic partners for late stage development, marketing, and distribution of our products; our ability to enter into out-licensing, development, manufacturing, marketing and distribution agreements with other pharmaceutical companies and keep such agreements in effect; our ability to protect and enforce our patent portfolio and intellectual property; and our ability to continue to list our common shares on the NASDAQ Capital Market. Investors should consult our quarterly and annual filings with the Canadian and U.S. securities commissions for additional information on risks and uncertainties, including those discussed in our Annual Report on Form 20-F and MD&A filed under the Company's profile on SEDAR+ at and on EDGAR at We disclaim any obligation to update any such risks or uncertainties or to publicly announce any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, unless required to do so by a governmental authority or applicable law. No securities regulatory authority has either approved or disapproved of the contents of this news release. The Toronto Stock Exchange accepts no responsibility for the adequacy or accuracy of this news release. Issuer: Anna BiehnChief Executive OfficerE: ABiehn@ Investor Contact:Jenene ThomasJTC Team T: (US): +1 (908) 824-0775E: csci@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
13-05-2025
- Business
- Business Wire
MaaT Pharma Provides Business Update and Reports Financial Results for the First Quarter 2025
LYON, France--(BUSINESS WIRE)--Regulatory News: The first months of 2025 have marked a pivotal moment for MaaT Pharma with the positive results from our Phase 3 trial of MaaT013, our lead asset. Share MaaT Pharma (EURONEXT: MAAT – the 'Company'), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem Therapies TM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, today provided a business update and reported its cash position as of March 31, 2025. 'The first months of 2025 have marked a pivotal moment for MaaT Pharma with the positive results from our Phase 3 trial of MaaT013, our lead asset. This milestone enables us to advance toward a Market Authorization submission in Europe and reflects over a decade of dedication and close collaboration with physicians. We are deeply grateful for the trust and participation of patients, whose support has been essential in achieving this progress,' said Hervé Affagard, CEO and co-founder of MaaT Pharma. 'We are also making strong progress across our pipeline, with encouraging outcomes for MaaT033 and MaaT034. Additionally, by extending our cash runway to October 2025, we are well-positioned to deliver on our upcoming value-creating milestones.' Pipeline highlights In Hemato-Oncology Acute Graft-versus-Host Disease (aGvHD) – MaaT013 In January 2025, the Company announced positive topline results from the pivotal Phase 3 ARES Study evaluating MaaT013 in aGvHD. The study met its primary endpoint with a significant gastrointestinal overall response rate at Day 28 of 62% and demonstrates the unprecedented efficacy of MaaT013 as third-line treatment of aGvHD with gastrointestinal involvement (GI-aGvHD) consistent with previously communicated EAP results. The Company anticipates MAA submission in Europe in June 2025. In March 2025, the Company received positive opinion from EMA Pediatric Committee on the Pediatric Investigation Plan for MaaT013, a key milestone achieved towards the MAA submission to the EMA. In March 2025, the Company received a positive outcome from the final DSMB meeting on ARES Phase 3 trial, confirming the remarkable efficacy results and positive risk/benefit profile of MaaT013 in third-line aGvHD. Earlier this year, the Company announced its intent to partner the distribution of MaaT013 in Europe, while retaining MaaT013 rights in the U.S. Allogenic Hematopoietic Stem Cell Transplant (allo-HSCT) - MaaT033 In January 2025, the Company announced that the DSMB completed its second safety assessment of the Phase 2b trial PHOEBUS and recommended continuation of the trial without modification. In April 2025, the Company announced the positive outcome of a key DSMB safety interim analysis for the Phase 2b trial PHOEBUS. As a result of their unblinded analysis, the DSMB recommended the trial to proceed as planned, showing no excessive mortality related to MaaT033 as of today. This additional positive outcome further reinforces MaaT033's safety profile and supports MaaT033's integration in the allo-HSCT setting without significant risks of severe adverse events. In Immuno-Oncology MaaT034 - Next-generation drug candidates with co-cultured technology (MET-C platform) In April 2025, MaaT Pharma presented promising preclinical data for MaaT034 at the American Association for Cancer Research (AACR) Annual Meeting 2025, demonstrating strong anti-tumor efficacy and immune activation in germ-free mice. MaaT013– Proof-of-Concept trials with donor derived drugs (MET-N platform) MaaT013 is currently being evaluated in a Phase 2a randomized clinical trial (NCT04988841) (PICASSO) sponsored by AP-HP and in collaboration with INRAE and Institut Gustave Roussy, in combination with immune checkpoint inhibitors (ICI), ipilimumab (Yervoy®) and nivolumab (Opdivo®), in metastatic melanoma patients. The Company provided its MaaT013 drug candidate and placebo and contributes to the microbiome profiling of patients using its proprietary gutPrint® AI research engine, while the trial investigator sponsor handled recruitment, treatment and oversee data collection and analysis. Data readout is expected in H2 2025. In Neurodegenerative Diseases Amyotrophic Lateral Sclerosis (ALS) - MaaT033 In May 2025, MaaT Pharma announced positive final Phase 1b results for MaaT033 in ALS, showing a favorable safety and tolerability profile supported by biomarker and microbiome analyses. Rapid and sustained microbial engraftment was observed, along with a slower rate of disease progression (ALSFRS-R slope to be interpreted with caution. The Company is seeking a partner to further advance clinical evaluation in ALS. Corporate update In April 2025, the Company announced the initiation of coverage of its stock by H.C. Wainwright & Co. With a research report named ' In With the Gut and Out With the Bad in GvHD; Initiating at Buy With a €21 PT', H.C. Wainwright & Co initiated a Buy recommendation and a Target Price of €21. Cash position 1 As of March 31, 2025, total cash and cash equivalents were EUR 24.4 million, as compared to EUR 20.2 million as of December 31, 2024. The net increase in cash position of EUR 4.2 million during the first quarter of 2025 includes the capital increase of €13 million supported by historical shareholders, while investment in R&D activities continued across the pipeline. The Company believes it has sufficient cash to cover its current needs and planned development programs into October 2025 and is exploring several options to further extend its cash horizon. Revenues in Q1 2025 1 MaaT Pharma reported revenues of EUR 1.1 million for the first quarter of 2025 compared with EUR 0.8 million for the same period of 2024 representing a constant growth, quarter to quarter, year to year. Upcoming financial communications* June 20, 2025: Annual General Meeting September 16, 2025: Publication of H1 2025 results November 4, 2025: Publication of revenues & cash for Q3 2025 *Indicative calendar that may be subject to change. Upcoming investor and business conferences participation June 12-15 – European Hematology Association (EHA) Congress, Milan June 16-19, 2025 – Bio International Convention, Boston, MA June 18-19, 2025 – Portzamparc Conference Mid & Small Caps 2025, Paris September 25, 2025 – KBC Healthcare Conference, Brussels --- About MaaT Pharma MaaT Pharma is a leading, late-stage clinical company focused on developing innovative gut microbiome-driven therapies to modulate the immune system and enhance cancer patient survival. Supported by a talented team committed to making a difference for patients worldwide, the Company was founded in 2014 and is based in Lyon, France. As a pioneer, MaaT Pharma is leading the way in bringing the first microbiome-driven immunomodulator in oncology. Using its proprietary pooling and co-cultivation technologies, MaaT Pharma develops high diversity, standardized drug candidates, aiming at extending life of cancer patients. MaaT Pharma has been listed on Euronext Paris (ticker: MAAT) since 2021. Forward-looking Statements All statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Company's control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as 'target,' 'believe,' 'expect,' 'aim', 'intend,' 'may,' 'anticipate,' 'estimate,' 'plan,' 'project,' 'will,' 'can have,' 'likely,' 'should,' 'would,' 'could' and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company's control that could cause the Company's actual results or performance to be materially different from the expected results or performance expressed or implied by such forward-looking statements. 1 unaudited financial results
