SELLAS Life Sciences Group announced the first pediatric patient with acute myeloid leukemia (AML) has been dosed in a Phase 2 trial of SLS009 (tambiciclib), a CDK9 inhibitor. This trial builds on promising data from a previous cohort and expands the study to include patients with ASXL1-mutated AML. The company previously received Rare Pediatric Disease Designation (RPDD) from the FDA for SLS009 in pediatric AML, potentially qualifying them for a Priority Review Voucher upon successful NDA approval.
This development is important because it addresses the significant unmet need for effective treatments in pediatric AML, particularly for patients with ASXL1 mutations, who often face poor prognoses and limited therapeutic options. This trial offers a potential new treatment avenue for these vulnerable patients, especially those who have failed other therapies including transplants and intensive chemotherapy. The potential for accelerated review via a PRV underscores the FDA’s recognition of this unmet medical need and could expedite the availability of SLS009 if proven successful.
The Phase 2 trial is evaluating SLS009 in combination with venetoclax and azacitidine at different dose levels and schedules. The study aims to determine safety, tolerability, and efficacy, with a target response rate of 20% and a median survival of at least three months. Furthermore, the trial seeks to identify biomarkers that could help select patients most likely to benefit from SLS009 in future studies.
The dosing of the first pediatric patient is a significant step forward for SELLAS and the development of SLS009. Positive results from this pediatric cohort could significantly strengthen the drug’s potential, leading to accelerated regulatory review and ultimately providing a much-needed treatment option for children with this aggressive form of leukemia. Further, a successful trial could validate the company’s focus on CDK9 inhibition as a therapeutic strategy in AML and potentially other cancers.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
