The U.S. Food and Drug Administration (FDA) granted fast track designation to Syros Pharmaceuticals’ (NASDAQ:SYRS) tamibarotene to treat higher-risk myelodysplastic syndrome (HR-MDS).

MDS are a group of cancers in which immature blood cells in the bone marrow do not mature or become healthy blood cells.

Tamibarotene, an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, is currently being evaluated in combination with azacitidine in a phase 3 trial, dubbed SELECT-MDS-1, to treat patients with newly diagnosed HR-MDS with RARA gene overexpression.

Syros expects to complete patient enrollment in in the trial in Q4 2023, with pivotal data expected in Q3 2024.

“No new therapies beyond hypomethylating agents have been approved since 2006, and approximately half of all patients diagnosed with HR-MDS patients ultimately progress to AML,” said Syros Chief Medical Officer.

Syros is also evaluating tamibarotene in combination with venetoclax and azacitidine in newly diagnosed unfit patients with AML having RARA overexpression. Initial data from the randomized portion of the phase 2 trial called SELECT-AML-1 is expected in Q4 2023 and additional data in 2024.

Tamibarotene had received the FDA’s orphan drug status in February 2022 to treat MDS.

SYRS +4.50% to $4.64 premarket Jan. 26