SELLAS Life Sciences Group, a clinical-stage biopharmaceutical company specializing in cancer therapies, has received Orphan Drug Designation (ODD) from the European Commission for SLS009, a novel CDK9 inhibitor aimed at treating acute myeloid leukemia (AML). This designation, based on a positive opinion from the European Medicines Agency (EMA), underscores SELLAS' commitment to advancing innovative treatments for challenging cancer indications.
CEO Dr. Angelos Stergiou expressed enthusiasm over the ODD, highlighting its alignment with recent Phase 2 data showing promising results for SLS009 in treating AML. This follows earlier ODD recognition from the FDA, reinforcing the drug's potential therapeutic value.
Orphan Drug Designation in the EU is granted for therapies addressing life-threatening or debilitating conditions affecting fewer than five in 10,000 individuals. Benefits include 10 years of market exclusivity post-approval, regulatory fee reductions, and EMA protocol assistance. These incentives support expedited development and potential approval pathways.
Andrew Elnatan, VP of Regulatory Affairs at SELLAS, emphasized the clinical benefits observed in AML patients, particularly those relapsed or refractory to venetoclax regimens. He underscored the importance of EMA's recognition and collaboration in defining regulatory strategies for European approval, complementing efforts with the FDA for potential US approval.
The ongoing Phase 2a trial assesses SLS009's safety, tolerability, and efficacy in combination with aza/ven at varying doses. Notably, the 60 mg dose cohort is evaluating response rates and survival outcomes, targeting a 20% response rate and over three months median survival. The trial continues enrolling patients with specific mutations associated with myelodysplastic syndromes, including ASXL1 mutations.
SELLAS also focuses on another lead candidate, GPS, targeting the WT1 protein across various cancers. SLS009 (formerly GFH009) stands out as a differentiated CDK9 inhibitor, demonstrating potent efficacy with reduced toxicity. Early clinical results highlight its potential to address unmet needs in AML, particularly among patients with adverse prognostic factors like ASXL1 mutations.