Michael Kelly, MD, MPH, MS, Syros Pharmaceuticals Senior Medical Director speaks about Syros Starts Phase 3 Clinical Trial of Targeted Investigational Therapy in Newly Diagnosed Higher-Risk MDS Patients.
Link to Abstract:
https://clinicaltrials.gov/ct2/show/NCT04797780
Syros Pharmaceuticals has begun a Phase 3 clinical trial of tamibarotene, a targeted experimental treatment, in a subset of newly diagnosed HR-MDS patients with RARA gene overexpression. RARA-positive persons account for around 30% of people with HR-MDS, and tamibarotene is the first focused treatment in clinical research for this patient population.
The SELECT-MDS-1 Phase 3 trial is a double-blind, placebo-controlled study that will recruit around 190 RARA-positive newly diagnosed HR-MDS patients. Tamibarotene with azacitidine or placebo plus azacitidine will be given to patients in a 2:1 ratio. The trial’s major aim will be to compare the rates of full remission between the two therapy groups. ClinicalTrials.gov, with the study number NCT04797780, has further information about the experiment.
Tamibarotene has shown activity in RARA-positive relapsed or refractory HR-MDS patients when used alone in previous clinical studies, and tamibarotene in combination with azacitidine led to a 67 percent composite complete response rate in RARA-positive patients with low blast count AML, which is similar to HR-MDS on the disease continuum. Furthermore, compared to either medication alone, the combination of tamibarotene and azacitidine has been well-tolerated, with no indication of enhanced toxicity, including myelosuppression.
Tamibarotene is also being tested in clinical trials for RARA-positive newly diagnosed AML patients who do not qualify for conventional intense chemotherapy. In the second half of 2021, Syros aims to start a Phase 2 study in that patient population to test tamibarotene in combination with venetoclax and azacitidine.