Latest news with #RarePediatricDiseaseDesignation
Yahoo
3 days ago
- Business
- Yahoo
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
If awarded, OST-HER2 would become the first listeria investigational medicinal product to be awarded the RMAT designation RMAT designation reduces BLA application review time and permits augmented interactions with FDA to inform market access NEW YORK, June 06, 2025--(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. View source version on Contacts OS Therapies Contact Information: Jack Doll410-297-7793Irpr@
Yahoo
15-05-2025
- Business
- Yahoo
SELLAS Life Sciences Announces First Pediatric AML Patient Dosed in the Ongoing Phase 2 Trial of SLS009 r/r AML
- First ASXL1 Pediatric Acute Myeloid Leukemia (AML) Patient Dosed at MD Anderson Cancer Center: Program Supported by Rare Pediatric Disease Designation (RPDD) NEW YORK, May 15, 2025 (GLOBE NEWSWIRE) -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ('SELLAS'' or the 'Company'), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced that the first pediatric AML patient has been dosed in the ongoing Phase 2 trial of SLS009 (tambiciclib), a highly selective CDK9 inhibitor, in relapsed/refractory acute myeloid leukemia (r/r AML). 'Building upon our promising Cohort 3 data, we are pleased to dose our first pediatric AML patient as part of the ongoing Phase 2 trial,' said Dragan Cicic, MD, Chief Development Officer of SELLAS. 'This milestone reflects our commitment to addressing critical unmet needs in hematologic disorders as we develop treatments for the most difficult to treat patients, particularly pediatric patients, with very few available options, including multi-hit TP53 mutation, failure of azacitidine and venetoclax, failure of transplant, and almost all available high-intensity chemotherapies. With the Rare Pediatric Disease Designation already in place, we are hopeful that our work will bring meaningful progress and potential regulatory advantages as we continue to advance this important program.' SELLAS was granted the FDA RPDD for the treatment of pediatric AML in July 2024. If, in the future, a New Drug Application (NDA) for SLS009 for the treatment of pediatric AML is approved by the FDA, SELLAS will be eligible to receive a Priority Review Voucher (PRV) that could be redeemed to receive a priority review for any subsequent marketing application. PRVs may be used by the sponsor or sold to another sponsor for their use and have recently sold for approximately $100 million. The Phase 2 clinical trial of SLS009 is an open-label, single-arm, multi-center study designed to evaluate the safety, tolerability, and efficacy of SLS009 in combination with venetoclax and azacitidine at two dose levels, 45 and 60 mg. In the 60 mg dose cohort, patients were treated at either a 60 mg dose once per week or a 30 mg dose two times per week. The trial was expanded to include two additional cohorts, one with ASXL1-mutated AML patients and one with patients with myelodysplasia-related molecular abnormalities other than ASXL1. The target response rate at the optimal dose level is 20% with a target median survival of at least 3 months. In addition, the study aims to identify biomarkers for the target patient population and enrichment for further trials. For more information on the study, visit identifier NCT04588922. About SELLAS Life Sciences Group, Inc. SELLAS is a late-stage clinical biopharmaceutical company focused on the development of novel therapeutics for a broad range of cancer indications. SELLAS' lead product candidate, GPS, is licensed from Memorial Sloan Kettering Cancer Center and targets the WT1 protein, which is present in an array of tumor types. GPS has the potential as a monotherapy and combination with other therapies to address a broad spectrum of hematologic malignancies and solid tumor indications. The Company is also developing SLS009 (tambiciclib) - potentially the first and best-in-class differentiated small molecule CDK9 inhibitor with reduced toxicity and increased potency compared to other CDK9 inhibitors. Data suggests that SLS009 demonstrated a high response rate in AML patients with unfavorable prognostic factors including ASXL1 mutation, commonly associated with poor prognosis in various myeloid diseases. For more information on SELLAS, please visit Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical facts are 'forward-looking statements,' including those relating to future events. In some cases, forward-looking statements can be identified by terminology such as 'plan,' 'expect,' 'anticipate,' 'may,' 'might,' 'will,' 'should,' 'project,' 'believe,' 'estimate,' 'predict,' 'potential,' 'intend,' or 'continue' and other words or terms of similar meaning. These statements include, without limitation, statements related to the GPS clinical development program, including the REGAL study and the timing of future milestones related thereto. These forward-looking statements are based on current plans, objectives, estimates, expectations, and intentions, and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties with oncology product development and clinical success thereof, the uncertainty of regulatory approval, and other risks and uncertainties affecting SELLAS and its development programs as set forth under the caption 'Risk Factors' in SELLAS' Annual Report on Form 10-K filed on March 20, 2025 and in its other SEC filings. Other risks and uncertainties of which SELLAS is not currently aware may also affect SELLAS' forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof. SELLAS undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations, or other circumstances that exist after the date as of which the forward-looking statements were made. Investor Contact Bruce Mackle Managing Director LifeSci Advisors, LLC SELLAS@
Business Wire
27-04-2025
- Business
- Business Wire
Innorna Announces FDA Rare Pediatric Disease and Orphan Drug Designations Granted to IN013 for Treatment of Wilson Disease
BOSTON & HONG KONG--(BUSINESS WIRE)-- Innorna, a clinical-stage biotechnology company revolutionizing mRNA therapeutics with its innovative lipid nanoparticle (LNP) delivery technology, today announced the U.S. Food and Drug Administration (FDA) has granted both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) to its investigational mRNA therapy, IN013, for treating Wilson Disease (WD). This dual achievement accelerates the clinical development of IN013, advancing Innorna's mission to deliver transformative therapies for WD patients. Innorna receives dual FDA designations for IN013, marking a critical milestone in developing an mRNA therapy for Wilson Disease. About FDA Designations The Rare Pediatric Disease Designation (RPDD) incentivizes therapies for serious or life-threatening diseases affecting fewer than 200,000 U.S. patients, primarily those aged 18 or younger. Sponsors may qualify for a Priority Review Voucher (PRV) upon approval to expedite FDA review of a subsequent drug application. The Orphan Drug Designation (ODD) supports therapies targeting rare diseases (affecting fewer than 200,000 U.S. patients) by providing seven years of market exclusivity, tax credits for clinical trials, and waiver of certain FDA fees. About Wilson Disease (WD) and IN013 Wilson Disease (WD), or Hepatolenticular Degeneration (HLD), is a rare and life-threatening autosomal recessive disorder caused by mutations in the ATP7B gene, leading to toxic copper accumulation in the liver, brain, kidneys, and other organs. Symptoms include hepatic and neurological damage, ocular abnormalities, kidney injury, hemolysis, and skeletal complications. Current treatments offer limited efficacy and carry risks such as hepatorenal toxicity or exacerbated neurological symptoms. IN013, developed using Innorna's mRNA-LNP platform, targets the root cause of WD by restoring functional ATP7B protein levels. By reducing systemic copper buildup, IN013 aims to alleviate multi-organ damage and deliver disease-modifying benefits. About Innorna Founded in 2019, Innorna is committed to developing state-of-the-art LNP delivery systems and RNA therapies to address unmet medical needs. Its proprietary Diversity-Oriented Lipid Library —with over 5,000 ionizable lipids—enables breakthroughs in mRNA vaccines, gene editing, and cell therapies. With its innovative mRNA-LNP platform, Innorna has built a robust pipeline targeting infectious diseases, rare genetic disorders, and cancer immunotherapy. Beyond its internal R&D efforts, the company collaborates with global-leading biotechnology partners to expand the reach of its transformative technology. Innorna's achievements have earned recognition as one of MIT Technology Review's Global Top 50 Smartest Companies and Fortune China's Most Socially Influential Startups. Guided by INNOVATION, INTEGRITY, EFFICIENCY, and OPENNESS, Innorna drives its mission to revolutionize mRNA applications and make a global impact.
Associated Press
27-04-2025
- Business
- Associated Press
Innorna Announces FDA Rare Pediatric Disease and Orphan Drug Designations Granted to IN013 for Treatment of Wilson Disease
BOSTON & HONG KONG--(BUSINESS WIRE)--Apr 27, 2025-- Innorna, a clinical-stage biotechnology company revolutionizing mRNA therapeutics with its innovative lipid nanoparticle (LNP) delivery technology, today announced the U.S. Food and Drug Administration (FDA) has granted both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) to its investigational mRNA therapy, IN013, for treating Wilson Disease (WD). This dual achievement accelerates the clinical development of IN013, advancing Innorna's mission to deliver transformative therapies for WD patients. About FDA Designations The Rare Pediatric Disease Designation (RPDD) incentivizes therapies for serious or life-threatening diseases affecting fewer than 200,000 U.S. patients, primarily those aged 18 or younger. Sponsors may qualify for a Priority Review Voucher (PRV) upon approval to expedite FDA review of a subsequent drug application. The Orphan Drug Designation (ODD) supports therapies targeting rare diseases (affecting fewer than 200,000 U.S. patients) by providing seven years of market exclusivity, tax credits for clinical trials, and waiver of certain FDA fees. About Wilson Disease (WD) and IN013 Wilson Disease (WD), or Hepatolenticular Degeneration (HLD), is a rare and life-threatening autosomal recessive disorder caused by mutations in the ATP7B gene, leading to toxic copper accumulation in the liver, brain, kidneys, and other organs. Symptoms include hepatic and neurological damage, ocular abnormalities, kidney injury, hemolysis, and skeletal complications. Current treatments offer limited efficacy and carry risks such as hepatorenal toxicity or exacerbated neurological symptoms. IN013, developed using Innorna's mRNA-LNP platform, targets the root cause of WD by restoring functional ATP7B protein levels. By reducing systemic copper buildup, IN013 aims to alleviate multi-organ damage and deliver disease-modifying benefits. About Innorna Founded in 2019, Innorna is committed to developing state-of-the-art LNP delivery systems and RNA therapies to address unmet medical needs. Its proprietary Diversity-Oriented Lipid Library —with over 5,000 ionizable lipids—enables breakthroughs in mRNA vaccines, gene editing, and cell therapies. With its innovative mRNA-LNP platform, Innorna has built a robust pipeline targeting infectious diseases, rare genetic disorders, and cancer immunotherapy. Beyond its internal R&D efforts, the company collaborates with global-leading biotechnology partners to expand the reach of its transformative technology. Innorna's achievements have earned recognition as one of MIT Technology Review's Global Top 50 Smartest Companies and Fortune China's Most Socially Influential Startups. Guided by INNOVATION, INTEGRITY, EFFICIENCY, and OPENNESS, Innorna drives its mission to revolutionize mRNA applications and make a global impact. View source version on CONTACT: Innorna Co. Ltd. [email protected] KEYWORD: NORTH AMERICA UNITED STATES ASIA PACIFIC EUROPE HONG KONG MASSACHUSETTS INDUSTRY KEYWORD: BIOTECHNOLOGY TECHNOLOGY FDA HEALTH PHARMACEUTICAL HEALTH TECHNOLOGY RESEARCH NANOTECHNOLOGY GENETICS SCIENCE CLINICAL TRIALS SOURCE: Innorna Co. Ltd. Copyright Business Wire 2025. PUB: 04/27/2025 05:36 AM/DISC: 04/27/2025 05:36 AM
Yahoo
12-03-2025
- Business
- Yahoo
Atsena Therapeutics Granted U.S. FDA Fast Track Designation for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis
Marks third FDA designation for ATSN-201, which has also received Rare Pediatric Disease Designation and Orphan Drug Designation DURHAM, N.C., March 12, 2025 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, leverages the company's novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment. 'We are pleased that the FDA has granted Fast Track designation to ATSN-201, reinforcing its potential to address the significant unmet need in XLRS, a rare inherited retinal disease with no approved treatments,' said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. 'This designation, along with the previously granted Orphan Drug and Rare Pediatric Disease designations, marks an important milestone in advancing the development of ATSN-201. The Atsena team remains dedicated to developing transformative gene therapies and improving the quality of life of individuals suffering from XLRS and other inherited retinal diseases.' Fast Track designation is granted to treatments intended to address serious or life-threatening diseases that have demonstrated the potential to meet an unmet medical need. Treatments that receive Fast Track designation may benefit from more frequent interactions with the FDA throughout drug development. In addition, the Fast Track program allows for Priority Review, if relevant criteria are met. Currently, there are no approved treatments for XLRS, which is typically diagnosed in early childhood and affects approximately 30,000 males in the U.S. and the EU. The safety and tolerability of ATSN-201 is currently being evaluated in the LIGHTHOUSE study, a Phase I/II, dose-escalation and dose-expansion clinical trial in male patients ages six and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. Enrollment for this study is ongoing. For more information, visit (Identifier: NCT05878860). About X-linked Retinoschisis (XLRS)XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss and ultimately blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments. About one of Atsena's novel capsids, spreads laterally beyond the subretinal injection site to enable safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that promotes transgene expression well beyond subretinal injection bleb margins. This is in contrast to benchmark AAV vectors, which remain confined to the original bleb margins. At clinically relevant doses, efficiently transduces foveal cones without the need for surgical detachment and has a favorable safety profile relative to benchmark capsids. For more information about the preclinical study and how works, visit About Atsena TherapeuticsAtsena Therapeutics ('Atsena') is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company's lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a genetic condition that is typically diagnosed in childhood and leads to blindness later in life. ATSN-101, Atsena's first-in-class, investigational gene therapy for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children, has completed a Phase 1 / 2 trial with positive results ( Atsena is advancing ATSN-101 toward the initiation of a global pivotal trial as part of its exclusive strategic collaboration with Nippon Shinyaku Co., Ltd. Atsena's pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit Media Contact:Gina Mangiaracina6 Degrees(917) 797-7904gmangiaracina@ Business Contact:info@ in to access your portfolio
