The U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Syros Pharmaceuticals’ SY-5609 for the treatment of pancreatic cancer. SY-5609, a highly selective and potent oral cyclin-dependent kinase 7 (CDK7) inhibitor, is currently being evaluated in combination with chemotherapy for the treatment of patients with relapsed metastatic pancreatic cancer.
“This orphan drug designation underscores the urgency of our efforts to develop SY-5609 for patients with pancreatic cancer, one of the most devastating and difficult to treat malignancies,” said David A. Roth, M.D., chief medical officer of Syros.
“Based on the early data we reported last year, which demonstrated single-agent activity in heavily pre-treated patients, as well as compelling preclinical data and a strong mechanistic rationale, we believe SY-5609 could deliver meaningful benefit to people with pancreatic cancer, whose tumors have otherwise eluded therapeutic intervention. We look forward to sharing initial data from the safety lead-in portion of our ongoing Phase 1 study later this year.”
The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.
Syros’ ongoing Phase 1 trial is evaluating SY-5609 in combination with chemotherapy in pancreatic cancer patients who have progressed following treatment with FOLFIRINOX. Patients were randomized to receive either SY-5609 in combination with gemcitabine, or SY-5609 in combination with gemcitabine and nab-paclitaxel, at the approved doses of the combination agents. The study is assessing safety and tolerability, as well as efficacy measures such as disease control rate and progression free survival. Safety and clinical activity data from the safety lead-in portion of the trial are expected in the second half of 2022.
Under an existing clinical supply agreement with Roche, Syros is also supplying SY-5609 for a combination dosing cohort in Roche’s ongoing Phase 1/1b INTRINSIC trial. This cohort is evaluating the combination of SY-5609 and atezolizumab in patients with BRAF-mutant colorectal cancer. Roche is the sponsor of the INTRINSIC trial.