The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) for Syros Pharmaceuticals’ SY-5609 for the treatment of pancreatic cancer. 

An oral inhibitor of cyclin-dependent kinase 7 (CDK7), SY-5609 is presently being analysed alongside chemotherapy as a treatment for relapsed metastatic pancreatic cancer patients.

The Phase I clinical trial that is underway is assessing SY-5609 plus chemotherapy in subjects with pancreatic cancer whose disease has advanced after Folfirinox treatment. 

In the trial, subjects were randomised to take either SY-5609 plus gemcitabine or SY-5609 plus gemcitabine along with nab-paclitaxel at dosage levels approved for these combination therapies. 

Tolerability and safety, as well as efficacy parameters, such as disease control rate and progression-free survival, will be analysed in the trial. 

The company anticipates safety and clinical activity findings from the trial’s safety lead-in segment in the second half of the year.

Syros has a clinical supply agreement in place with Roche and is delivering SY-5609 for a combination dosing cohort in the latter company’s ongoing Phase I/Ib INTRINSIC trial. 

This cohort is assessing a combination of SY-5609 and atezolizumab in BRAF-mutant colorectal cancer patients. 

Syros Pharmaceuticals chief medical officer David Roth said: “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. 

“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 tumours have otherwise eluded therapeutic intervention.”

In February, the company received ODD from the FDA for its tamibarotene oral therapy for the treatment of myelodysplastic syndrome.