SELLAS Gets EMA Orphan Drug Status for SLS009 in AML Treatment

On July 8, 2024, SELLAS Life Sciences Group, Inc. (NASDAQ: SLS), a clinical-stage biopharmaceutical company specializing in novel cancer therapies, announced that the European Commission, following a favorable opinion from the European Medicines Agency (EMA), has granted Orphan Drug Designation (ODD) to SLS009, a novel and selective CDK9 inhibitor intended for the treatment of acute myeloid leukemia (AML).

Angelos Stergiou, MD, ScD h.c., President and CEO of SELLAS, expressed excitement over the EMA's ODD approval for AML treatment. He emphasized that this recognition, along with promising preliminary Phase 2 data and prior FDA ODD designation, underscores the firm's ongoing commitment to SLS009's development. Stergiou affirmed that the company remains on schedule to release additional data on SLS009 in the third quarter of the year and will continue collaborating closely with the EMA and FDA to bring this treatment to patients in need.

In the European Union (EU), orphan drug designation is granted by the European Commission based on a positive recommendation from the EMA's Committee for Orphan Medicinal Products. This designation is intended for treatments targeting life-threatening or chronically debilitating conditions affecting fewer than five in 10,000 individuals within the EU. Benefits of this designation include financial and regulatory incentives, such as a 10-year market exclusivity post-approval, reduced fees for protocol assistance from the EMA during the product development phase, and access to centralized marketing authorization. The treatment must also demonstrate a significant benefit to those suffering from the condition.

Andrew Elnatan, Vice President of Regulatory Affairs at SELLAS, also welcomed the EMA’s designation, noting that it complements the earlier FDA ODD. He highlighted that ongoing clinical trial data supports SLS009's significant benefit for AML patients who have relapsed after or are refractory to venetoclax treatments. Elnatan detailed that this designation will help pave the way toward regulatory approval in the EU and potential approval in the U.S.

The Phase 2a clinical trial of SLS009 is designed as an open-label, single-arm, multi-center study to assess the safety, tolerability, and efficacy of SLS009 in combination with aza/ven at two dose levels—45 mg and 60 mg. Within the 60 mg dose cohort, patients were randomized to receive either a 60 mg dose once per week or a 30 mg dose twice weekly. The trial aims for a 20% response rate at the optimal dose level and a target median survival exceeding three months. The ASXL1 mutation, identified as a promising target mutation due to its biological characteristics and the SLS009 mechanism of action, has so far shown favorable clinical results. The trial continues to enroll patients in two cohorts, one with ASXL1 mutations and another with other myelodysplasia-related mutations. Further information on the study can be found at clinicaltrial.gov with identifier NCT04588922.

SELLAS Life Sciences is a late-stage clinical biopharmaceutical company dedicated to developing innovative therapies for various cancer indications. Another key product candidate from SELLAS is GPS, licensed from Memorial Sloan Kettering Cancer Center. GPS targets the WT1 protein prevalent in numerous tumor types and holds potential both as a monotherapy and in combination therapies for a wide range of hematologic malignancies and solid tumors. Additionally, SELLAS is advancing SLS009, previously known as GFH009, which may become the leading small molecule CDK9 inhibitor due to its reduced toxicity and increased potency compared to other inhibitors. Preliminary data suggests a high response rate for SLS009 in AML patients with poor prognostic factors, including the ASXL1 mutation.

SELLAS continues its mission to develop and deliver novel cancer therapeutics, fostering hope and potential treatment options for patients worldwide.