Latest news with #OrphanDrug
Yahoo
2 days ago
- Business
- Yahoo
Hemispherian receives positive opinion for Orphan Medicinal Product Designation in the EU for GLIX1 in glioma
OSLO, Norway, June 6, 2025 /PRNewswire/ -- Hemispherian AS, a pioneering biotech company developing next-generation therapeutics for aggressive cancers, announced today that the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending Orphan Drug Designation (ODD) for GLIX1, the company's lead molecule, for the treatment of glioma, one of the most devastating and lethal brain cancers. Significant clinical benefit beyond current therapies. The designation marks a major regulatory milestone for Hemispherian, recognizing both the urgent unmet medical need in glioma and the potential of GLIX1 to offer significant clinical benefit beyond current therapies. "We are proud to have received this recommendation for an Orphan Drug Designation from the EMA Committee. This validates our scientific approach and supports our mission to transform treatment for patients with glioma, who currently face extremely limited and ineffective options," said Zeno Albisser, CEO of Hemispherian. About the Designation Following a detailed review, the COMP determined that GLIX1 meets the criteria for orphan designation under Regulation (EC) No 141/2000. In particular: Glioma is a life-threatening and chronically debilitating disease affecting approximately 2.6 in 10,000 people in the EU. Existing therapies for Glioblastoma (a form of Glioma) offer only limited survival benefits, with median overall survival typically less than 15 months. Non-clinical studies with GLIX1 demonstrated significant tumor reduction and extended survival in validated animal models, including cases of complete tumor eradication. These results suggest that GLIX1 offers a clinically relevant advantage over existing treatments, fulfilling the EMA's criteria for "significant benefit." Benefits of Orphan Drug Designation- Faster market Access- 10 Years of market Exclusivity Orphan Drug Designation by the EMA provides Hemispherian with a range of development and commercial incentives, including: 10 years of market exclusivity in the EU upon approval. Protocol assistance and regulatory guidance from EMA during clinical development. Eligibility for fee reductions for regulatory submissions, including marketing authorization. These incentives are designed to encourage the development of innovative treatments for rare diseases with high unmet need. About Hemispherian Hemispherian is an Oslo-based pharmaceutical company focused on the development of a novel class of small-molecule drugs targeting glioblastoma and other aggressive cancers. The company's proprietary GLIX platform is based on unique DNA-targeting technology aimed at improving patient survival and quality of life. So far, the treatment shows impressive effects, with limited to no side effects, and with no damage to healthy tissue. Contact: Zeno Albisser, CEO – zeno@ +47 40603455 View original content: SOURCE Hemispherian AS Error 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
Yahoo
2 days ago
- Business
- Yahoo
Hemispherian receives positive opinion for Orphan Medicinal Product Designation in the EU for GLIX1 in glioma
OSLO, Norway, June 6, 2025 /PRNewswire/ -- Hemispherian AS, a pioneering biotech company developing next-generation therapeutics for aggressive cancers, announced today that the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending Orphan Drug Designation (ODD) for GLIX1, the company's lead molecule, for the treatment of glioma, one of the most devastating and lethal brain cancers. Significant clinical benefit beyond current therapies. The designation marks a major regulatory milestone for Hemispherian, recognizing both the urgent unmet medical need in glioma and the potential of GLIX1 to offer significant clinical benefit beyond current therapies. "We are proud to have received this recommendation for an Orphan Drug Designation from the EMA Committee. This validates our scientific approach and supports our mission to transform treatment for patients with glioma, who currently face extremely limited and ineffective options," said Zeno Albisser, CEO of Hemispherian. About the Designation Following a detailed review, the COMP determined that GLIX1 meets the criteria for orphan designation under Regulation (EC) No 141/2000. In particular: Glioma is a life-threatening and chronically debilitating disease affecting approximately 2.6 in 10,000 people in the EU. Existing therapies for Glioblastoma (a form of Glioma) offer only limited survival benefits, with median overall survival typically less than 15 months. Non-clinical studies with GLIX1 demonstrated significant tumor reduction and extended survival in validated animal models, including cases of complete tumor eradication. These results suggest that GLIX1 offers a clinically relevant advantage over existing treatments, fulfilling the EMA's criteria for "significant benefit." Benefits of Orphan Drug Designation- Faster market Access- 10 Years of market Exclusivity Orphan Drug Designation by the EMA provides Hemispherian with a range of development and commercial incentives, including: 10 years of market exclusivity in the EU upon approval. Protocol assistance and regulatory guidance from EMA during clinical development. Eligibility for fee reductions for regulatory submissions, including marketing authorization. These incentives are designed to encourage the development of innovative treatments for rare diseases with high unmet need. About Hemispherian Hemispherian is an Oslo-based pharmaceutical company focused on the development of a novel class of small-molecule drugs targeting glioblastoma and other aggressive cancers. The company's proprietary GLIX platform is based on unique DNA-targeting technology aimed at improving patient survival and quality of life. So far, the treatment shows impressive effects, with limited to no side effects, and with no damage to healthy tissue. Contact: Zeno Albisser, CEO – zeno@ +47 40603455 View original content: SOURCE Hemispherian AS
Yahoo
23-05-2025
- Business
- Yahoo
Sol-Gel Reports First Quarter 2025 Results
NESS ZIONA, Israel, May 23, 2025 (GLOBE NEWSWIRE) -- Sol-Gel Technologies, Ltd. (NASDAQ: SLGL), a clinical-stage dermatology company, today announced financial results for the first quarter ended March 31, 2025. Financial Results for the First Quarter Year Ended March 31st, 2025 Total revenue in the first quarter was $1 million compared to $0.5 million revenues for the same period in 2024. Research and development expenses were $8.8 million compared to $5.3 million in the same period in 2024. The increase of $3.5 million was primarily attributed to an increase of $3.6 million due to expenses associated with supplier-led manufacturing development to support future commercialization of SGT-610, an increase of $0.5 million related to the commercialization of EPSOLAY and TWYNEO in territories outside of the U.S., offset by a decrease of $0.5 million in clinical trial expenses related to SGT-610. General and administrative expenses were $1.3 million compared to $1.8 million for the same period in 2024. The decrease of $0.5 million was mainly attributed to a decrease in payroll and stock-based compensation expenses due to cost measures being taken in 2024. Sol-Gel reported a net loss of $8.8 million for the first quarter of 2025 and of $3.2 basic and diluted loss per share, compared to a net loss of $6.3 million and a loss of $2.3 per basic and diluted share for the same period in 2024. As of March 31, 2025, Sol-Gel had $16.9 million in cash, cash equivalents, and deposits and no marketable securities for a total balance of $16.9 million. The Company expects its cash resources to fund cash requirements into the first quarter of 2027. About Sol-Gel Technologies Sol-Gel Technologies, Ltd. is a dermatology company focused on identifying, developing and commercializing or partnering drug products for the treatment of skin diseases. Sol-Gel developed TWYNEO which is approved by the FDA for the treatment of acne vulgaris in adults and pediatric patients nine years of age and older; and EPSOLAY, which is approved by the FDA for the treatment of inflammatory lesions of rosacea in adults. The Company's pipeline includes Orphan Drug candidate, SGT-610 under investigation for the prevention of new basal cell carcinomas in Gorlin syndrome patients, and also includes topical drug candidate SGT-210 under investigation for the treatment of rare skin keratodermas. For additional information, please visit Forward Looking Statements This press release contains 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, but not limited to our expected cash runway. In some cases, you can identify forward-looking statements by terminology such as 'believe,' 'may,' 'estimate,' 'continue,' 'anticipate,' 'intend,' 'should,' 'plan,' 'expect,' 'predict,' 'potential,' or the negative of these terms or other similar expressions. Forward-looking statements are based on information we have when those statements are made or our management's current expectations and are subject to risks and uncertainties that could cause actual performance or results to differ materially from those expressed in or suggested by the forward-looking statements. Important factors that could cause such differences include, but are not limited to, our ability to enter into further collaborations, lower than anticipated annual revenue income from new collaborations, a delay in the timing of our clinical trials, the success of our clinical trials, and an increase in our anticipated costs and expenses, as well as the following factors: (i) the adequacy of our financial and other resources, particularly in light of our history of recurring losses and the uncertainty regarding the adequacy of our liquidity to pursue our complete business objectives; (ii) our ability to complete the development of our product candidates; (iii) our ability to find suitable co-development partners; (iv) our ability to obtain and maintain regulatory approvals for our product candidates in our target markets, the potential delay in receiving such regulatory approvals and the possibility of adverse regulatory or legal actions relating to our product candidates even if regulatory approval is obtained; (v) our collaborators' ability to commercialize our pharmaceutical product candidates; (vi) our ability to obtain and maintain adequate protection of our intellectual property; (vii) our collaborators' ability to manufacture our product candidates in commercial quantities, at an adequate quality or at an acceptable cost; (viii) our collaborators' ability to establish adequate sales, marketing and distribution channels; (ix) acceptance of our product candidates by healthcare professionals and patients; (x) the possibility that we may face third-party claims of intellectual property infringement; (xi) the timing and results of clinical trials that we may conduct or that our competitors and others may conduct relating to our or their products; (xii) intense competition in our industry, with competitors having substantially greater financial, technological, research and development, regulatory and clinical, manufacturing, marketing and sales, distribution and personnel resources than we do; (xiii) potential product liability claims; (xiv) potential adverse federal, state and local government regulation in the United States, China, Europe or Israel; and (xv) loss or retirement of key executives and research scientists; (xvi) general market, political and economic conditions in the countries in which the Company operates; and, (xvii) the current war between Israel and Hamas and any deterioration of the war in Israel into a broader regional conflict involving Israel with other parties. These factors and other important factors discussed in the Company's Annual Report on Form 20-F filed with the Securities and Exchange Commission ('SEC') on April 29, 2025, and our other reports filed with the SEC, could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Except as required by law, we undertake no obligation to update any forward-looking statements in this press release. SOL-GEL TECHNOLOGIES LTD. CONDENSED CONSOLIDATED BALANCE SHEETS (U.S. dollars in thousands) (Unaudited) December 31, March 31, 2024 2025 Assets CURRENT ASSETS: Cash and cash equivalents $ 19,489 $ 16,890 Bank deposits 12 12 Marketable securities 4,425 - Accounts receivables 3,595 3,883 Prepaid expenses and other current assets 3,774 2,647 TOTAL CURRENT ASSETS 31,295 23,432 NON-CURRENT ASSETS: Restricted long-term deposits and cash equivalents 1,291 1,293 Long-term receivables 1,024 518 Property and equipment, net 202 183 Operating lease right-of-use assets 1,426 1,332 Other long-term assets 13 - Funds in respect of employee rights upon retirement 595 305 TOTAL NON-CURRENT ASSETS 4,551 3,631 TOTAL ASSETS $ 35,846 $ 27,063 Liabilities and shareholders' equity CURRENT LIABILITIES: Accounts payable $ 1,265 $ 438 Other accounts payable 3,590 3,702 Current maturities of operating leases 430 433 TOTAL CURRENT LIABILITIES 5,285 4,573 LONG-TERM LIABILITIES: Operating leases liabilities 878 746 Liability for employee rights upon retirement 833 363 Other long-term Liability 1,209 TOTAL LONG-TERM LIABILITIES 1,711 2,318 TOTAL LIABILITIES 6,996 6,891 SHAREHOLDERS' EQUITY: Ordinary shares, NIS 0.1 par value – authorized: 5,000,000 as of December 31, 2024 and March 31, 2025, respectively; issued and outstanding: 2,785,762 as of December 31, 2024 and March 31, 2025, respectively (*) 774 774 Additional paid-in capital 258,959 259,089 Accumulated deficit (230,883) (239,691) TOTAL SHAREHOLDERS' EQUITY 28,850 20,172 TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY $ 35,846 $ 27,063 (*) All share amounts have been retroactively adjusted to reflect a 1-for-10 reverse share split. SOL-GEL TECHNOLOGIES LTD. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (U.S. dollars in thousands) (Unaudited) Three months endedMarch 31 2024 2025 LICENSE REVENUES $ 466 $ 1,031 RESEARCH AND DEVELOPMENT EXPENSES 5,345 8,843 GENERAL AND ADMINISTRATIVE EXPENSES 1,833 1,257 OPERATING LOSS 6,712 9,069 FINANCIAL INCOME,net (368) (261) LOSS FOR THE PERIOD 6,344 8,808 BASIC AND DILUTED LOSS PER ORDINARY SHARE 2.3 3.2 WEIGHTED AVERAGE NUMBER OF SHARES*OUTSTANDING USED IN COMPUTATION OF BASIC AND DILUTED LOSS PER SHARE (*) 2,785,762 2,785,762 (*)All share amounts have been retroactively adjusted to reflect a 1-for-10 reverse share split. Sol-Gel Contact:Eyal Ben-OrChief Financial Officerir@ Sol-Gel Technologies in to access your portfolio
Yahoo
15-05-2025
- Business
- Yahoo
BrainStorm Reschedules First Quarter 2025 Financial Results Release to Post-Market Close Today and Sets Investor Call for Monday, May 19
NEW YORK, May 15, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced that it has rescheduled the release of its financial results for the first quarter ended March 31, 2025, to post-market close today, May 15. In addition, the Company has rescheduled its previously announced investor conference call, which will now take place on Monday, May 19, 2025, at 8:30 a.m. Eastern Time. BrainStorm's President & Chief Executive Officer, Chaim Lebovits, will present a corporate update to be followed by Q&A. Joining Mr. Lebovits to answer investment community questions will be Haro Hartounian, Ph.D., Chief Operating Officer, Bob Dagher, M.D., Chief Medical Officer, and Alla Patlis, CPA, MBA, Interim Chief Financial Officer. Participants are encouraged to submit their questions in advance of the call by sending them to: q@ Questions should be submitted by 5:00 p.m. Eastern Time on Friday, May 16, 2025. Investors may access the conference call by dialing the following numbers: Participant Numbers: Toll Free 888-506-0062 International 973-528-0011 Participant Access Code 621608 Webcast The replay of the conference call can be accessed by dialing the numbers below and will be available for 14 days. Replay Numbers: Toll Free 877-481-4010 International 919-882-2331 Reply Passcode 52457 About Brainstorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn® platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection. NurOwn® is BrainStorm's lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). A Phase 3 trial in ALS (NCT03280056) has been completed, and a confirmatory Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA. The NurOwn clinical program has generated valuable insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A genotype, biomarker data collected at seven longitudinal time points, and a comprehensive analysis of the "Floor Effect" — a critical challenge in measuring clinical outcomes in advanced ALS. BrainStorm has published its findings in multiple peer-reviewed journals. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society. BrainStorm is also advancing a proprietary, allogeneic exosome-based platform designed to deliver therapeutic proteins and nucleic acids. The company recently received a Notice of Allowance from the U.S. Patent and Trademark Office for a foundational patent covering its exosome technology, further strengthening BrainStorm's growing IP portfolio in this emerging area of regenerative learn more, visit Notice Regarding Forward-Looking Statements This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), ADCOM meeting related to NurOwn, the timing of a PDUFA action date for the BLA for NurOwn, the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. CONTACTSInvestors:Michael WoodPhone: +1 646-597-6983mwood@ Logo - View original content: SOURCE BrainStorm Cell Therapeutics Inc.
Associated Press
15-05-2025
- Business
- Associated Press
Cadrenal Therapeutics Announces Tecarfarin Manufacturing Progress in Support of Clinical Trial Readiness
PONTE VEDRA, Fla.--(BUSINESS WIRE)--May 15, 2025-- Cadrenal Therapeutics, Inc. (Nasdaq: CVKD), a biopharmaceutical company developing therapeutics for patients with cardiovascular disease, today announced manufacturing and supply chain milestones for its lead drug candidate, tecarfarin, a novel oral vitamin K antagonist (VKA) anticoagulant that is designed to address unmet needs in anticoagulation therapy. Cadrenal completed the technical transfer and manufacturing of its tecarfarin drug substance in accordance with current good manufacturing practices (cGMP) earlier this year at a U.S. site of a leading global Contract Development and Manufacturing Organization (CDMO). Manufacturing of the tecarfarin drug product candidate is currently underway. 'We are pleased with the important progress we have made with the supply chain and cGMP manufacturing process for tecafarin,' said Quang X. Pham, Chairman & CEO. 'This is a key milestone as we support our clinical development strategy and tecarfarin's potential to provide clinical benefits for patients with cardiovascular disease who require chronic VKA anticoagulation.' About Cadrenal Therapeutics, Inc. Cadrenal Therapeutics, Inc. is a biopharmaceutical company developing therapeutics for patients with cardiovascular disease. Cadrenal's lead investigational product is tecarfarin, a novel oral vitamin K antagonist anticoagulant that addresses unmet needs in anticoagulation therapy. Tecarfarin is a reversible anticoagulant (blood thinner) designed to prevent heart attacks, strokes, and deaths due to blood clots in patients requiring chronic anticoagulation. Although warfarin is widely used off-label for several indications, extensive clinical and real-world data have shown it can have significant, serious side effects. Cadrenal is pursuing a pipeline-in-a-product approach with tecarfarin. Tecarfarin received Orphan Drug designation (ODD) for advanced heart failure patients with implanted mechanical circulatory support devices, including Left Ventricular Assisted Devices (LVADs). The Company also received ODD and fast-track status for tecarfarin in end-stage kidney disease and atrial fibrillation (ESKD+AFib). Cadrenal is opportunistically pursuing business development initiatives with a longer-term focus on creating a pipeline of cardiovascular therapeutics. For more information, visit and connect with us on LinkedIn. Safe Harbor Any statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute 'forward-looking statements.' The words 'anticipate,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'plan,' 'potentially,' 'predict,' 'project,' 'should,' 'target,' 'will,' 'would' and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These statements include statements regarding tecarfarin's potential to provide clinical benefits for patients with cardiovascular disease who require chronic VKA anticoagulation and pursuing business development initiatives with a longer-term focus on creating a pipeline of cardiovascular therapeutics. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the ability of tecarfarin to provide clinical benefits for patients with cardiovascular disease who require chronic VKA anticoagulation, the ability of Cadrenal to build a pipeline of specialized cardiovascular therapeutics and other assets and the other risk factors described in the Company's Annual Report on Form 10-K for the year ended December 31, 2024, and the Company's subsequent filings with the Securities and Exchange Commission, including subsequent periodic reports on Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Any forward-looking statements contained in this press release speak only as of the date hereof and, except as required by federal securities laws, the Company specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise. View source version on CONTACT: Corporate and Investor Relations Paul Sagan LaVoieHealthScience (617) 865-0041 [email protected] Andrew Korda LaVoieHealthScience (617) 865-0043 [email protected] KEYWORD: UNITED STATES NORTH AMERICA FLORIDA INDUSTRY KEYWORD: HEALTH OTHER HEALTH GENERAL HEALTH CLINICAL TRIALS PHARMACEUTICAL CARDIOLOGY BIOTECHNOLOGY SOURCE: Cadrenal Therapeutics, Inc. Copyright Business Wire 2025. PUB: 05/15/2025 07:30 AM/DISC: 05/15/2025 07:29 AM
