US-based biotechnology company Syros Pharmaceuticals has enrolled 190 subjects in the initial cohort of the SELECT-MDS-1 Phase III clinical trial of tamibarotene to treat newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS).
The double-blind, placebo-controlled study is designed to assess tamibarotene in combination with azacitidine, compared with azacitidine alone, in HR-MDS patients with retinoic acid receptor alpha (RARA) gene overexpression.
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This initial portion will aid in the analysis of the trial’s primary endpoint of complete response (CR).
Syros expects to publish the initial data by the middle of the fourth quarter of this year.
The CR rate among the initial 190 subjects will be assessed, along with supporting durability findings, which may lead to expedited or full regulatory approval.
The trial’s key secondary endpoint is the overall survival (OS) across a larger cohort of 550 patients.
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By GlobalDataSyros Pharmaceuticals chief medical officer David Roth said: “This marks an important step in advancing tamibarotene through late-stage clinical development and brings us closer to delivering our RARα agonist as a frontline treatment option for the approximately 50% of HR-MDS patients with RARA overexpression.
“We look forward to reporting pivotal data later this year which, if successful, will allow us to file our first New Drug Application (NDA) with the US Food and Drug Administration (FDA) and, ultimately, execute our vision of fundamentally changing the standard of care in haematologic malignancies.”
In addition to SELECT-MDS-1, Syros is investigating tamibarotene in the SELECT-AML-1 Phase II trial in unfit acute myeloid leukaemia patients newly diagnosed with RARA overexpression.
According to initial data from the SELECT-AML-1 trial, treatment with a combination of tamibarotene, venetoclax and azacytidine caused a 100% complete response/complete response with incomplete haematologic recovery (CR/CRi) rate, compared with a 70% CR/CRi rate in venetoclax plus azacitidine.
In addition, no increased toxicity was seen in the tamibarotene combination arm.
