On November 12, 2024 Syros Pharmaceuticals (NASDAQ:SYRS), a biopharmaceutical company committed to advancing new standards of care for the frontline treatment of hematologic malignancies, reported that the SELECT-MDS-1 Phase 3 trial evaluating tamibarotene in combination with azacitidine in newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) patients with RARA gene overexpression did not meet its primary endpoint of complete response (CR) rate (Press release, Syros Pharmaceuticals, NOV 12, 2024, View Source [SID1234648215]). Tamibarotene is Syros’ proprietary oral, selective, retinoic acid receptor alpha (RARα) agonist.
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In the first 190 enrolled patients, the CR rate by intent-to-treat (ITT) in the tamibarotene/azacitidine treatment arm was 23.8% (n=126; 95% CI: 16.7%-32.2%) compared to a CR rate of 18.8% (n=64; 95% CI: 10.1%-30.5%) in the placebo/azacitidine control arm and was not statistically significant (p-value = 0.2084). In the safety analysis of all enrolled patients (n=245), tamibarotene in combination with azacitidine (n=160) appeared to be generally well-tolerated, with an adverse event profile that was similar to that seen in earlier Syros-sponsored studies.
Syros also reported that, as previously disclosed in its filings with the SEC, the failure of the SELECT-MDS-1 trial to achieve its primary endpoint constitutes an event of default under its secured loan facility with Oxford Finance LLC.
"We are deeply disappointed by this outcome, particularly for the HR-MDS patients who are seeking a new treatment option for this challenging disease," said Conley Chee, Chief Executive Officer of Syros. "We plan to stop the study, review the clinical data more thoroughly, and evaluate the next steps. We want to express our sincere appreciation for the patients, caregivers and healthcare professionals who took part in the SELECT-MDS-1 trial and to all the employees of Syros for their exceptional work on the tamibarotene program."
About SELECT-MDS-1 Phase 3 Trial
SELECT-MDS-1 is a multinational Phase 3 randomized, double-blind, placebo-controlled trial evaluating the safety and efficacy of tamibarotene in combination with azacitidine compared to placebo and azacitidine in HR-MDS patients with RARA overexpression. The primary endpoint of the trial was the CR rate in the first 190 patients.