Latest news with #RPDD
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
