SELLAS Announces U.S. FDA Rare Pediatric Disease Designation (RPDD) Granted to SLS009 for the Treatment of Pediatric Acute Myeloid Leukemia

Core Insights - SELLAS Life Sciences Group, Inc. has received Rare Pediatric Disease Designation (RPDD) from the FDA for SLS009, a CDK9 inhibitor aimed at treating pediatric acute myeloid leukemia (AML) [1][5] - This designation follows a previous RPDD for pediatric acute lymphoblastic leukemia, highlighting the company's focus on addressing urgent needs in pediatric oncology [8] Company Overview - SELLAS is a late-stage clinical biopharmaceutical company dedicated to developing novel therapies for various cancer indications, including AML [6] - The company’s lead product candidate, GPS, targets the WT1 protein and has potential applications in both hematologic malignancies and solid tumors [6] Product Development - SLS009 is positioned as a potentially first-in-class small molecule CDK9 inhibitor, showing reduced toxicity and increased potency compared to existing CDK9 inhibitors [6] - The drug has demonstrated a high response rate in AML patients with unfavorable prognostic factors, such as ASXL1 mutations [6] Clinical Context - Acute myeloid leukemia is the second most common hematological malignancy in children, often associated with poor prognosis [5] - Current treatments for refractory and relapsed pediatric AML yield a 5-year overall survival rate of only 33%, with significantly lower rates for those with shorter remission periods [5] Regulatory Implications - If SLS009 receives FDA approval for pediatric AML, SELLAS may be eligible for a Priority Review Voucher (PRV), which can expedite the review process for future marketing applications [2][5] - PRVs have recently been valued at approximately $100 million, indicating significant financial implications for the company [2] Clinical Trials - The company has opened enrollment for pediatric AML patients in an ongoing Phase 2 clinical trial, emphasizing its commitment to developing SLS009 [8]