Latest news with #RegenerativeMedicineAdvancedTherapy
Business Wire
2 days ago
- Business
- Business Wire
OS Therapies Submits Request for Regenerative Medicine Advanced Therapy (RMAT) Designation to U.S. FDA for OST-HER2 in the Prevention of Metastases in Recurrent, Fully-Resected, Lung Metastatic Pediatric Osteosarcoma
NEW YORK--(BUSINESS WIRE)-- OS Therapies Inc. (NYSE-A: OSTX) ('OS Therapies' or 'the Company'), a clinical-stage cancer immunotherapy and antibody drug conjugate biotechnology company, today announced it has submitted a request for Regenerative Medicine Advanced Therapy (RMAT) Designation to U.S. FDA for OST-HER2 in the prevention of metastases in recurrent, fully-resected, lung metastatic pediatric osteosarcoma. RMAT designations are granted to sponsors with regenerative medicine therapies for serious or life-threatening conditions and provide sponsors with various benefits, including eligibility for an accelerated Biologics License Application (BLA) review. OST-HER2 has already received Rare Pediatric Disease Designation (RPDD), Orphan Drug Designation (ODD) and Fast Track Designation (FTD) for osteosarcoma from the U.S. FDA. If OST-HER2 receives a conditional BLA via Accelerated Review prior to September 30, 2026, the Company will become eligible to receive a Priority Review Voucher (PRV) that it intends to immediately sell. The most recent publicly disclosed PRV sale, valued at $155 million, occurred in May 2025. The Company is awaiting feedback by mid-June 2025 from a Type D meeting with FDA regarding the statistical analysis plan to be used in an End of Phase 2 meeting for OST-HER2 in the prevention of metastases in recurrent, fully-resected, lung metastatic pediatric osteosarcoma. Upon receipt of the Type D Meeting feedback, the Company intends to promptly request the End of Phase 2 meeting with FDA in which it will be seek agreement to allow it to begin a rolling BLA submission in the third quarter of 2025. The grant of the RMAT designation in the third quarter of 2025 complements the company's parallel efforts in other major markets, including Europe and the United Kingdom, where the company plans to seek EMA PRIME Designation and Conditional Market Access (CMA) applications. In parallel to regulatory engagement and market access planning for OST-HER2, the Company is preparing for the late stage clinical development of other pipeline candidates. As such, the Company is well positioned for sustained growth across multiple therapeutic modalities. About OS Therapies OS Therapies is a clinical stage oncology company focused on the identification, development, and commercialization of treatments for osteosarcoma and other solid tumors. OST-HER2, the Company's lead asset, is an immunotherapy leveraging the immune-stimulatory effects of Listeria bacteria to initiate a strong immune response targeting the HER2 protein. OST-HER2 has received Rare Pediatric Disease Designation (RPDD) from the U.S. Food & Drug Administration and Fast-Track and Orphan Drug designations from the U.S. FDA and European Medicines Agency. The Company has demonstrated positive data in its Phase 2b clinical trial of OST-HER2 in recurrent, fully resected, lung metastatic osteosarcoma demonstrating statistically significant benefit in the 12-month event free survival (EFS) primary endpoint of the study. The Company anticipates submitting a BLA to the U.S. FDA for OST-HER2 in osteosarcoma in 2025 and, if approved, would become eligible to receive a Priority Review Voucher that it could then sell. OST-HER2 has completed a Phase 1 clinical study primarily in breast cancer patients, in addition to showing preclinical efficacy data in various models of breast cancer. OST-HER2 has been conditionally approved by the U.S. Department of Agriculture for the treatment of canines with osteosarcoma. In addition, OS Therapies is advancing its next-generation Antibody Drug Conjugate (ADC) and Drug Conjugates (DC), known as tunable ADC (tADC), which features tunable, tailored antibody-linker-payload candidates. This platform leverages the Company's proprietary silicone Si-Linker and Conditionally Active Payload (CAP) technology, enabling the delivery of multiple payloads per linker. For more information, please visit Forward-Looking Statements 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 within the meaning of the federal securities laws. These forward-looking statements and terms such as "anticipate," "expect," "intend," "may," "will," "should" or other comparable terms involve risks and uncertainties because they relate to events and depend on circumstances that will occur in the future. Those statements include statements regarding the intent, belief or current expectations of OS Therapies and members of its management, as well as the assumptions on which such statements are based. OS Therapies cautions readers that forward-looking statements are based on management's expectations and assumptions as of the date of this news release and are subject to certain risks and uncertainties that could cause actual results to differ materially, including, but not limited to the approval of OST-HER2 by the U.S. FDA and other risks and uncertainties described in 'Risk Factors' and 'Management's Discussion and Analysis of Financial Condition and Results of Operations' in the Company's most recent Annual Report on Form 10-K and other subsequent documents the Company files with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and, except as required by the federal securities laws, OS Therapies specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
Yahoo
30-05-2025
- Business
- Yahoo
FDA Grants Orphan Drug Status to Beam Therapeutics Inc. (BEAM)' AATD Gene Therapy
Beam Therapeutics Inc. (NASDAQ:BEAM) has secured U.S. FDA orphan drug designation for BEAM-302, its pioneering genetic medicine targeting alpha-1 antitrypsin deficiency (AATD), a rare inherited disorder that can cause severe lung and liver disease. BEAM-302, delivered via liver-targeting lipid nanoparticles, is designed to correct the underlying DNA mutation responsible for AATD, offering hope for a one-time, potentially curative treatment. This regulatory milestone follows the recent Regenerative Medicine Advanced Therapy (RMAT) designation, underscoring the therapy's promise and the urgent need for effective AATD solutions. A biotechnologist in a lab coat discussing a therapeutic antibody with a colleague. Preliminary data from ongoing Phase 1/2 trials show BEAM-302 is well tolerated and achieves dose-dependent, durable correction of the disease-causing mutation, with protein levels exceeding therapeutic thresholds in the 60 mg dose group. Dosing in higher cohorts is underway, with more results from Beam Therapeutics Inc. (NASDAQ:BEAM) expected later in 2025. Orphan drug designation brings significant benefits, including tax credits, user fee exemptions, and up to seven years of market exclusivity post-approval, accelerating development for this first-in-class base editing therapy for AATD, a condition with no current curative treatments. While we acknowledge the potential of BEAM to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than BEAM and that has 100x upside potential, check out our report about this READ NEXT: and Disclosure: None. 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
30-05-2025
- Business
- Yahoo
Candel Therapeutics, Inc. (CADL)'s CAN-2409 Advances with FDA RMAT Status for Localized Prostate Cancer
Candel Therapeutics, Inc. (NASDAQ:CADL) has secured FDA Regenerative Medicine Advanced Therapy (RMAT) designation for its lead immunotherapy candidate, CAN-2409 (aglatimagene besadenovec), targeting newly diagnosed, localized prostate cancer in intermediate-to-high-risk patients. The designation, based on positive phase III trial results, aims to expedite the development and review of regenerative medicine therapies that address serious or life-threatening conditions with unmet medical needs. CAN-2409, an off-the-shelf adenovirus delivering the HSV-tk gene, is administered with the prodrug valacyclovir alongside standard radiation therapy. The phase III study met its primary endpoint, showing a significant improvement in disease-free survival compared to standard care alone, with a favorable safety profile. A technician in a lab coat adjusting a MRI Radiation Therapy system in a hospital clinic. The RMAT status provides Candel Therapeutics, Inc. (NASDAQ:CADL) with enhanced FDA guidance and the possibility of accelerated or priority review for its future Biologics License Application, which the company plans to submit by the end of 2026. CAN-2409 had previously received Fast Track designation for the same indication. Following the announcement, Candel Therapeutics, Inc. (NASDAQ:CADL)'s shares rose by over 6%. The company's CEO, Dr. Paul Peter Tak, highlighted the designation as validation of CAN-2409's potential to transform prostate cancer treatment. While we acknowledge the potential of CADL to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than CADL and that has 100x upside potential, check out our report about this READ NEXT: and Disclosure: None. Sign in to access your portfolio
Yahoo
20-05-2025
- Business
- Yahoo
Longeveron® to Attend BIO International Convention 2025
Focus on potential partnership and strategic opportunities for the Company's stem cell therapy program for Alzheimer's disease Recent Type B meeting with FDA provided alignment on a planned single, pivotal Phase 2/3 clinical trial, which if positive, would be acceptable for Biological License Application (BLA) submission for Alzheimer's disease Initiation of planned single, pivotal Phase 2/3 clinical trial in mild Alzheimer's disease anticipated in 2H 2026, contingent upon obtaining non-dilutive funding and/or partnering support Results from the Phase 2a clinical trial (CLEAR MIND) evaluating laromestrocel as a potential treatment for Alzheimer's disease were published in the peer reviewed journal Nature Medicine Laromestrocel is, to Company's knowledge, the first cellular therapeutic candidate to receive FDA Regenerative Medicine Advanced Therapy (RMAT) designation for Alzheimer's disease MIAMI, May 20, 2025 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN), a clinical stage regenerative medicine biotechnology company developing cellular therapies for life-threatening and chronic aging-related conditions, today announced that the Company will participate in the BIO International Convention taking place June 16-19, 2025 at the Boston Convention & Exhibition Center. At the conference, members of the Longeveron management team will host meetings with global pharmaceutical company executives to explore potential partnership and strategic opportunities for the Company's Alzheimer's disease program, which has generated positive data in successful Phase 1 and Phase 2 clinical trials. Results from the Phase 2a clinical trial (CLEAR MIND), which support the therapeutic potential of laromestrocel in the treatment of mild Alzheimer's disease, were published in the peer reviewed journal Nature Medicine in March 2025. Longeveron's lead investigational therapeutic candidate is laromestrocel, a proprietary, scalable, allogenic cellular therapy. In the completed Phase 2a clinical trial (CLEAR MIND), laromestrocel treated patients showed an overall slowing/prevention of disease worsening compared to placebo. The trial achieved the primary safety and secondary efficacy endpoints and showed statistically significant improvements in pre-specified clinical and biomarker endpoints in specific laromestrocel groups compared to placebo. Longeveron previously announced top-line results for the CLEAR MIND Phase 2a clinical trial on October 5, 2023, and reported additional clinical data and imaging biomarker results from CLEAR MIND on December 20, 2023. Full results from the CLEAR MIND study were presented in a featured research oral presentation at the 2024 Alzheimer's Association International Conference (AAIC). The FDA has granted laromestrocel both Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation for the treatment of mild Alzheimer's disease, which allow greater access to FDA interaction during laromestrocel's development for Alzheimer's disease. Laromestrocel is, to Company's knowledge, the first cellular therapeutic candidate to receive FDA RMAT designation for Alzheimer's disease. To connect with Longeveron during the conference, please request a meeting via the BIO International Conference meeting portal or contact info@ About Longeveron is a clinical stage biotechnology company developing regenerative medicines to address unmet medical needs. The Company's lead investigational product is laromestorcel, an allogeneic mesenchymal stem cell (MSC) therapy product isolated from the bone marrow of young, healthy adult donors. Laromestrocel has multiple potential mechanisms of action encompassing pro-vascular, pro-regenerative, anti-inflammatory, and tissue repair and healing effects with broad potential applications across a spectrum of disease areas. Longeveron is currently pursuing three pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer's disease, and Aging-related Frailty. Laromestrocel development programs have received five distinct and important FDA designations: for the HLHS program - Orphan Drug designation, Fast Track designation, and Rare Pediatric Disease designation; and, for the AD program - Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation. For more information, visit or follow Longeveron on LinkedIn, X, and Instagram. Forward-Looking StatementsCertain statements in this press release that are not historical facts are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, which reflect management's current expectations, assumptions, and estimates of future operations, performance and economic conditions, and involve known and unknown risks, uncertainties, and other important factors that could cause actual results, performance, or achievements to differ materially from those anticipated, expressed, or implied by the statements made herein. Forward-looking statements are generally identifiable by the use of forward-looking terminology such as 'anticipate,' 'believe,' 'contemplate,' 'continue,' 'could,' 'estimate,' 'expects,' 'intend,' 'looks to,' 'may,' 'on condition,' 'plan,' 'potential,' 'predict,' 'preliminary,' 'project,' 'see,' 'should,' 'target,' 'will,' 'would,' or the negative thereof or comparable terminology, or by discussion of strategy or goals or other future events, circumstances, or effects and include, but are not limited to, statements about the various below-listed factors. Factors that could cause actual results to differ materially from those expressed or implied in any forward-looking statements in this release include, but are not limited to, our cash position and need to raise additional capital, the difficulties we may face in obtaining access to capital, and the dilutive impact it may have on our investors; our financial performance, and ability to continue as a going concern; the period over which we estimate our existing cash and cash equivalents will be sufficient to fund our future operating expenses and capital expenditure requirements; the ability of our clinical trials to demonstrate safety and efficacy of our product candidates, and other positive results; the timing and focus of our ongoing and future preclinical studies and clinical trials, and the reporting of data from those studies and trials; the size of the market opportunity for certain of our product candidates, including our estimates of the number of patients who suffer from the diseases we are targeting; our ability to scale production and commercialize the product candidate for certain indications; the success of competing therapies that are or may become available; the beneficial characteristics, safety, efficacy and therapeutic effects of our product candidates; our ability to obtain and maintain regulatory approval of our product candidates in the U.S. and other jurisdictions; our plans relating to the further development of our product candidates, including additional disease states or indications we may pursue; our plans and ability to obtain or protect intellectual property rights, including extensions of existing patent terms where available and our ability to avoid infringing the intellectual property rights of others; the need to hire additional personnel and our ability to attract and retain such personnel; and our estimates regarding expenses, future revenue, capital requirements and needs for additional financing. Further information relating to factors that may impact the Company's results and forward-looking statements are disclosed in the Company's filings with the Securities and Exchange Commission, including Longeveron's Annual Report on Form 10-K for the year ended December 31, 2024, filed with the Securities and Exchange Commission on February 28, 2025, its Quarterly Reports on Form 10-Q, and its Current Reports on Form 8-K. The Company operates in highly competitive and rapidly changing environment; therefore, new factors may arise, and it is not possible for the Company's management to predict all such factors that may arise nor assess the impact of such factors or the extent to which any individual factor or combination thereof, may cause results to differ materially from those contained in any forward-looking statements. The forward-looking statements contained in this press release are made as of the date of this press release based on information available as of the date of this press release, are inherently uncertain, and the Company disclaims any intention or obligation, other than imposed by law, to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise. Investor and Media Contact:Derek ColeInvestor Relations Advisory 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
15-05-2025
- Health
- Business Wire
FDA Grants Regenerative Medicine Advanced Therapy Designation for BrainChild Bio's B7-H3 CAR T-cell Therapy for Incurable Pediatric Brain Tumors
SEATTLE & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- BrainChild Bio, Inc., a clinical-stage biotechnology company developing CAR T-cell therapies to treat tumors in the central nervous system (CNS), today announced that the investigational B7-H3 targeting autologous CAR T-cell therapy has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S Food and Drug Administration (FDA) for the treatment of diffuse intrinsic pontine glioma (DIPG), an incurable pediatric brain tumor. The use of a regenerative medicine, specifically a CAR T-cell therapy, offers the potential to overcome barriers for other drug modalities to be effective in addressing DIPG, including the precarious location of the DIPG tumor in the brainstem, the infiltrative growth of the tumor throughout normal brainstem functional anatomy, and the blood brain barrier that remains intact during tumor progression. BrainChild Bio has designed BCB-276 to be administered by locoregional delivery of targeted CAR T-cells directly into the cerebrospinal fluid, permitting infused CAR T-cells to directly access the tumor bed, using an indwelling reservoir-catheter device. This approach to administering an autologous B7-H3 CAR T-cell therapy has been successfully implemented and resulted in the promising overall survival benefit in patients with brain tumors observed in the BrainChild-03 Phase 1 trial (NCT04185038), conducted by BrainChild Bio's academic partner, Seattle Children's Research Institute, and recently published in Nature Medicine. 'We are very pleased to now also receive RMAT designation, less than one month after being granted Breakthrough Therapy designation from FDA for our lead CAR T therapy, BCB-276, for the treatment of DIPG. Receiving designations from two independent reviews within FDA further validates the positive CAR-T clinical results achieved by our team to date and the urgent need for a treatment for DIPG,' stated Michael Jensen, MD, Founder and Chief Scientific Officer of BrainChild Bio. 'Our team is keenly focused on initiating the pivotal Phase 2 trial by the end of this year and look forward to continuing to work with the FDA on an accelerated path forward to bring potential new CAR-T treatments for CNS brain tumors in children and adults.' FDA grants RMAT designation to investigational regenerative medicine therapies, including cell therapies, that are aimed at treating serious or life-threatening diseases have shown preliminary clinical evidence that the drug has the potential to address unmet medical needs for the disease. Investigational medicines with RMAT are provided intensive interactions with the FDA during the product candidate's development process in addition to being eligible for rolling submission and priority review of the marketing application. 'It's gratifying to see another important benchmark reached in our work to combat pediatric brain cancer,' said Dr. Jeff Sperring, Chief Executive Officer at Seattle Children's. 'Our research is the foundation of progress to bring potential therapies to kids as fast as we can – and we're excited about the possibilities afforded by this designation.' BrainChild Bio is preparing to advance BCB-276 in a Phase 2 multi-center, pivotal registration trial to support a potential Biologics License Application (BLA) to the FDA for the treatment of children and young adults with DIPG. This clinical plan is based on alignment between BrainChild Bio and FDA at a Type B meeting in late 2024. About Diffuse Intrinsic Pontine Glioma (DIPG) and Application of CAR T-cell Therapies Diffuse intrinsic pontine glioma (DIPG) is a primary high-grade brain tumor that arises in the pons and is uniformly fatal. DIPG affects approximately 300 children per year in the U.S. with the majority of diagnoses made in children between 5 and 10 years of age. Current standard-of-care treatment remains limited to palliative focal radiation therapy which results in a median overall survival of only about 11 months from diagnosis. 1 Barriers to effective therapies for DIPG include the precarious location of the tumor in the brainstem, the infiltrative growth of the tumor throughout normal brainstem functional anatomy, and the blood brain barrier that remains relatively intact during tumor progression preventing therapies from gaining access to the cancer. The barriers to effective therapies for DIPG can be effectively overcome by the locoregional delivery of appropriately targeted CAR T-cells directly into the cerebrospinal fluid via intracerebroventricular (ICV) dosing with an indwelling reservoir-catheter device. This enables the potential for extensive exposure of the pons to cerebrospinal fluid flow from the ventricular system, thus permitting infused CAR T-cells to directly access the tumor bed. This also allows for repetitive infusions of CAR T-cells to replenish the tumor bed, offering the potential for more durable and sustained efficacy. Additionally, with the blood brain barrier intact, this therapeutic approach can also minimize any on-target, off-tumor toxicities resulting from systemic exposure of CAR T-cells. About BrainChild Bio BrainChild Bio, Inc., is a kids-first, clinical-stage biotechnology company harnessing the power of CAR T-cell technology to treat tumors in the central nervous system, prioritizing pediatric indications with plans to expand into adult CNS tumors, specifically Glioblastoma and brain metastasis. BrainChild Bio was launched out of Seattle Children's Therapeutics program and founded on the work of Dr. Michael Jensen, a pioneer in the cancer immunotherapy field and previous Chief Therapeutics Officer at Seattle Children's. BrainChild Bio is advancing a next-generation CAR T-cell therapy platform for tumors of the CNS that weaves together synthetic technologies, including multiplex targeting and enhanced potency controls, to enable multiple targets in a single CAR T-cell therapy, novel transgenes to increase potency, delivery technology for durable efficacy, and streamlined CAR T-cell design and manufacturing. BrainChild Bio's lead drug candidate is BCB-276, an autologous CAR T-cell therapy that targets the immune checkpoint B7-H3, that is advancing in clinical trials for the treatment of diffuse intrinsic pontine glioma (DIPG), a pediatric cancer that forms in the brainstem which currently has no approved treatments. BrainChild Bio's cell therapy approach using autologous B7-H3 CAR T-cell therapy has received Breakthrough Therapy designation and Regenerative Medicine Advanced Therapy designation from the U.S. Food and Drug Administration (FDA). More information is available at _____________________________________________________________ 1 DIPG Registry.
