Enrollment Complete: 190 Patients Enrolled in SELECT-MDS-1 Phase 3 Trial

Syros Pharmaceuticals, a biopharmaceutical firm focused on pioneering new treatment protocols for hematologic malignancies, has announced the completion of patient enrollment for its Phase 3 clinical trial, SELECT-MDS-1. The study aims to assess the efficacy of tamibarotene in patients with newly diagnosed high-risk myelodysplastic syndrome (HR-MDS) who exhibit overexpression of the RARA gene. A total of 190 participants have been enrolled, which is crucial for the primary endpoint analysis of complete response (CR). The company anticipates unveiling key data in mid-2024.
According to David A. Roth, M.D., Syros' Chief Medical Officer, the completion of the enrollment is a significant milestone for the development of tamibarotene and brings the company closer to offering a new frontline treatment option for approximately half of HR-MDS patients with RARA overexpression. If the trial is successful, Syros plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA), potentially transforming the standard of care for hematologic malignancies.
The SELECT-MDS-1 trial is a double-blind, placebo-controlled study that randomizes newly diagnosed HR-MDS patients with RARA overexpression to receive either tamibarotene combined with azacitidine or azacitidine alone in a 2:1 ratio. The primary endpoint is the CR rate in the initial 190 patients, which, along with supporting data on durability, could lead to accelerated or full approval. The key secondary endpoint is overall survival (OS) in 550 patients. The study's design is intended to be efficient, allowing it to serve as a confirmatory trial if needed to transition from accelerated to full approval. Enrollment is ongoing to reach the target of 550 patients.
In addition, Syros is conducting the SELECT-AML-1 Phase 2 clinical trial, which combines tamibarotene with venetoclax and azacitidine for newly diagnosed unfit acute myeloid leukemia patients with RARA overexpression. Initial data from this trial showed a 100% CR/CRi rate in response-evaluable patients treated with the combination therapy, compared to 70% in patients treated with venetoclax and azacitidine alone, without increased toxicity. Syros expects to release more data from the SELECT-AML-1 trial in 2024.
Syros Pharmaceuticals is dedicated to establishing new standards of care for frontline treatment of patients with hematologic malignancies. The company is developing tamibarotene, an oral selective RARα agonist, for frontline patients with higher-risk myelodysplastic syndrome and acute myeloid leukemia with RARA gene overexpression.