SELLAS Gains FDA RPDD for SLS009 in Pediatric AML Treatment

SELLAS Life Sciences Group, Inc. (NASDAQ: SLS), a biopharmaceutical company in the advanced stages of clinical development, announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to their drug SLS009 for the treatment of pediatric acute myeloid leukemia (AML). SLS009 is known as a highly selective CDK9 inhibitor. The FDA has previously awarded this drug Orphan Drug and Fast Track Designations for AML treatment.

Dr. Angelos Stergiou, President and CEO of SELLAS, emphasized the significance of this achievement by highlighting that SLS009 has been recognized for its potential to transform treatment for pediatric AML patients. He noted that this designation underscores the urgent necessity to develop therapies for children with treatment-resistant forms of AML, a disease often associated with limited treatment options. This recognition follows a similar designation for pediatric acute lymphoblastic leukemia received last month.

AML in children typically presents a challenging prognosis, particularly in cases where the disease is refractory or relapsed. Survival rates remain low under current treatments. A representative study indicated that the 5-year overall survival rate for relapsed pediatric AML was 33% across all patients, plummeting to 15.7% for those whose remission lasted less than a year. Alarmingly, patients who did not achieve complete remission after the initial chemotherapy had a 0% 5-year overall survival rate. Relapse occurs in about half of pediatric AML cases, with bone marrow transplants often being the only curative option for these patients. Chemotherapy's primary goal in such scenarios is to achieve remission to make transplantation viable.

The FDA's Rare Pediatric Disease Designation is reserved for serious or life-threatening diseases impacting fewer than 200,000 individuals in the U.S., primarily affecting those under 18. Should SELLAS receive approval for an NDA for SLS009, the company could obtain a Priority Review Voucher (PRV). This voucher allows for expedited review of any subsequent marketing application and is a valuable asset, as PRVs can be sold to other sponsors for significant sums, with recent sales reaching approximately $100 million.

SELLAS Life Sciences Group positions itself as an innovative player in cancer therapeutics, focusing on the development of new treatments for various cancer indications. Their lead product, GPS, licensed from Memorial Sloan Kettering Cancer Center, targets the WT1 protein found in multiple tumor types. GPS shows promise both as a standalone treatment and in combination with other therapies, addressing a diverse range of hematologic malignancies and solid tumors.

SLS009, formerly known as GFH009, is a small molecule CDK9 inhibitor that stands out for its reduced toxicity and enhanced potency compared to other inhibitors in its class. Preliminary data indicates that SLS009 has a high response rate in AML patients with negative prognostic factors, such as the ASXL1 mutation, often linked with poor outcomes in various myeloid diseases.

SELLAS continues to advance the clinical development of its novel therapies, aiming to improve treatment outcomes for patients with rare and challenging cancers, including pediatric AML.