Syros Pharmaceuticals (SYRS) Doses First Patient in SY-1425 Phase 2 in AML, MDS

September 22, 2016 8:06 AM EDT
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Syros Pharmaceuticals (Nasdaq: SYRS) announced that the first patient has been dosed in the Phase 2 clinical trial of its lead drug candidate, SY-1425, a first-in-class selective retinoic acid receptor alpha (RARα) agonist, in genomically defined subsets of patients with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) identified using a novel biomarker discovered by its gene control platform.

“This is an important milestone for Syros and for patients,” said David A. Roth, M.D., Chief Medical Officer of Syros. “There has been little improvement in the treatment of AML and MDS for the past 20 years, and survival rates for these patients lag behind many other blood cancers. Treatment with SY-1425 represents a promising therapeutic strategy for subsets of AML and MDS patients with a novel biomarker that we discovered using our gene control platform. Our pioneering approach is designed to advance a new wave of medicines to control the expression of disease-driving genes in genomically defined subsets of patients to provide them with a profound and durable clinical benefit. Our goal is to rapidly advance this first-in-class therapy for these currently underserved patients."

Using its gene control platform, Syros discovered subsets of AML and MDS patients whose tumors have a highly specialized regulatory region of non-coding DNA, known as a super-enhancer, that is associated with the RARA gene, which codes for the RARα transcription factor. The super-enhancer is believed to lead to over-production of the RARα transcription factor, locking cells in an immature, undifferentiated and proliferative state. Syros further investigated this unique biology directly in patient tissues and conducted preclinical studies showing that the RARA super-enhancer is predictive of response to treatment with SY-1425 in preclinical models of AML. Based on that data, Syros is implementing a biomarker strategy for its Phase 2 trial that selects a subset of approximately 25 percent of AML and MDS patients who may respond to treatment with SY-1425.

“The prognosis for these patients is poor, and targeted approaches like SY-1425 offer hope for much-needed new therapies that attack the underlying biology of the disease and hopefully allow patients to live longer without the toxicities of traditional chemotherapy,” said Rachel J. Cook, M.D., M.S., assistant professor of medicine at Oregon Health & Science University and an investigator in the trial. “We’re pleased to have enrolled the first patient in this clinical trial and look forward to further investigating SY-1425 for this newly identified subset of AML and MDS patients.”

The Phase 2 clinical trial is a multi-center, open-label trial exploring safety and efficacy in relapsed or refractory AML or high-risk MDS patients who have been prospectively selected using the Company's biomarker strategy. The trial is expected to enroll approximately 40 patients, and the primary endpoint of the trial will be overall response rate. The trial will also assess pharmacodynamic biomarkers, duration of response, safety and tolerability, and survival. Additional details about the trial can be found using the identifier NCT02807558 at

SY-1425 is approved in Japan as Amnolake (tamibarotene) to treat a different form of AML known as acute promyelocytic leukemia (APL), in which it has a well-established efficacy and safety profile. Syros in-licensed SY-1425 to develop and commercialize it in North America and Europe for all cancers.

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