Syros Pharmaceuticals has received fast track designation from the US Food and Drug Administration (FDA) for tamibarotene to treat newly diagnosed acute myeloid leukaemia (AML) with RARA (retinoic acid receptor alpha) overexpression.

The treatment is intended for use along with azacitidine and venetoclax in patients aged 75 years or above with comorbidities that make intensive induction chemotherapy unsuitable.

An oral selective RARA agonist, tamibarotene is currently being assessed in the SELECT-AML-1 Phase II clinical trial in AML patients.

Syros reported preliminary randomised data from the SELECT-AML-1 trial in December 2023.

The results showed a 100% complete response/complete response with incomplete haematologic recovery (CR/CRi) rate in patients treated with a combination of tamibarotene, venetoclax and azacitidine versus a 70% rate in those treated with venetoclax and azacytidine alone.

All patients treated with the triplet regimen achieved a CR/CRi by the end of the first cycle, indicating a rapid median time to response.

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The tamibarotene combination regimen was generally well tolerated with a safety profile in line with previous clinical data.

The fast track process is an FDA initiative to facilitate the development and expedite the review of drugs that treat serious conditions and fill unmet medical needs.

The designation allows for more frequent interactions with the FDA regarding the development plan of the therapeutic candidate and may also lead to eligibility for priority review and accelerated approval based on clinical data.

Syros Pharmaceuticals chief medical officer David Roth stated: “This designation reflects the tremendous need for a safe and effective therapy, which can improve the clinical outcomes and prognosis among people diagnosed with AML, many of whom cannot tolerate intensive treatment.”

In February 2022, the FDA granted orphan drug designation to Syros’s tamibarotene to treat myelodysplastic syndrome.