Syros Announces Completion of Enrollment of 190 Patients Necessary to Support Primary Endpoint Analysis in SELECT-MDS-1 Phase 3 Trial

2024-03-25

临床2期临床3期临床结果申请上市

-- On track to report pivotal complete response data by mid-4Q 2024 -- CAMBRIDGE, Mass.--(BUSINESS WIRE)--Syros Pharmaceuticals (NASDAQ:SYRS), a biopharmaceutical company committed to advancing new standards of care for the frontline treatment of hematologic malignancies, today announced that the enrollment of 190 patients has been completed in the SELECT-MDS-1 Phase 3 clinical trial evaluating tamibarotene in newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) patients with RARA gene overexpression. This initial cohort of 190 patients is necessary to support the complete response (CR) primary endpoint analysis. Syros expects to report these pivotal data by the middle of the fourth quarter of 2024. “We are pleased to announce the completion of enrollment of the 190 patients necessary to support the primary CR endpoint in SELECT-MDS-1. This marks an important step in advancing tamibarotene through late-stage clinical development and brings us closer to delivering our RARα agonist as a frontline treatment option for the approximately 50 percent of HR-MDS patients with RARA overexpression,” said David A. Roth, M.D., Chief Medical Officer of Syros. “We look forward to reporting pivotal data later this year which, if successful, will allow us to first New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) and, ultimately, execute on our vision of fundamentally changing the standard of care in hematologic malignancies.” The Phase 3 SELECT-MDS-1 clinical trial is a double-blind, placebo-controlled study evaluating tamibarotene in newly diagnosed HR-MDS patients with RARA overexpression randomized 2:1 to receive tamibarotene in combination with azacitidine or azacitidine alone. The primary endpoint is CR rate in the first 190 patients enrolled in the trial which, together with supporting durability data, can serve as the basis for accelerated approval or full approval; the key secondary endpoint in SELECT-MDS-1 is overall survival (OS) in a total of 550 patients. This study design reflects an efficient “one-trial” approach and could allow SELECT-MDS-1 to serve as a confirmatory study, if needed, to convert from accelerated to full approval. Enrollment is ongoing to reach the 550-patient target. Syros is also evaluating tamibarotene in combination with venetoclax and azacitidine in the SELECT-AML-1 Phase 2 clinical trial in newly diagnosed unfit acute myeloid leukemia patients with RARA gene overexpression. Syros previously reported initial data from the study, observing a 100% CR/CRi (complete response/complete response with incomplete hematologic recovery) rate in response-evaluable patients treated with the triplet regimen of tamibarotene, venetoclax and azacitidine without increased toxicity, as compared to 70% among patients treated with venetoclax and azacitidine alone. Syros expects to report additional data from SELECT-AML-1 in 2024. Read more here. About Syros Pharmaceuticals Syros is committed to developing new standards of care for the frontline treatment of patients with hematologic malignancies. Driven by the motivation to help patients with blood disorders that have largely eluded other targeted approaches, Syros is developing tamibarotene, an oral selective RARα agonist in frontline patients with higher-risk myelodysplastic syndrome and acute myeloid leukemia with RARA gene overexpression. For more information, visit and follow us on Twitter (@SyrosPharma) and LinkedIn. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including without limitation statements regarding Syros’ clinical development plans, the progression of its clinical trials, the timing to report clinical data, and the ability to commercialize tamibarotene and deliver benefit to patients. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “hope,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “target,” “should,” “would,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including Syros’ ability to: advance the development of its programs under the timelines it projects in current and future clinical trials; demonstrate in any current and future clinical trials the requisite safety, efficacy and combinability of its drug candidates; sustain the response rates and durability of response seen to date with its drug candidates; successfully develop a companion diagnostic test to identify patients with the RARA biomarker; obtain and maintain patent protection for its drug candidates and the freedom to operate under third party intellectual property; obtain and maintain necessary regulatory approvals; identify, enter into and maintain collaboration agreements with third parties; manage competition; manage expenses; raise the substantial additional capital needed to achieve its business objectives; attract and retain qualified personnel; and successfully execute on its business strategies; risks described under the caption “Risk Factors” in Syros’ Annual Report on Form 10-K for the year ended December 31, 2022 and Quarterly Reports on Form 10-Q for the quarters ended March 31, 2023, June 30, 2023 and September 30, 2023, each of which is on the Securities and Exchange Commission; and risks described in other filings that Syros makes with the Securities and Exchange Commission in the future.