Syros Pharmaceuticals (SYRS) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Company overview and strategy

• Focuses on small molecule medicines targeting gene expression in blood disorders, leveraging discoveries in super enhancers over 11 years.

• Portfolio includes a phase II-ready CDK7 therapy, a phase III oral arsenic trioxide program, and lead candidate tamibarotene.

• Leadership team has deep experience in drug development and U.S. launches, preparing for potential product introduction.

Lead program: Tamibarotene for high-risk MDS

• Tamibarotene, a RARA agonist, is in pivotal phase III for high-risk MDS, addressing a large unmet need with 18,500 annual cases in the U.S. and Europe.

• High-risk MDS patients have poor prognosis and limited treatment options, with current standard azacitidine yielding a 17% CR rate.

• Tamibarotene targets RARA overexpression, present in 50% of high-risk MDS patients, offering a broad target population.

Clinical data and trial design

• Single-agent tamibarotene showed a 60% hematologic response rate in relapsed/refractory MDS; combination with azacitidine in AML showed a 67% CR/CRI rate.

• In AML patients with low blast counts (similar to high-risk MDS), a 67% CR rate was observed, with deep molecular responses.

• Responses were rapid (median 1.2 months) and consistent across mutation/cytogenetic risk; 72% achieved transfusion independence.

• Adverse events were highly manageable, with no additive myelosuppression, supporting use in elderly, community-treated patients.

• Phase III is a global, double-blind, placebo-controlled trial (azacitidine ± tamibarotene), primary endpoint CR, readout expected mid-Q4 2024.