Syros Announces Completion of Enrollment of 190 Patients Necessary to Support Primary Endpoint Analysis in SELECT-MDS-1 Phase 3 Trial
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Insights
The completion of enrollment for the SELECT-MDS-1 Phase 3 clinical trial by Syros Pharmaceuticals is a significant milestone in the drug development process. The focus on tamibarotene as a potential treatment for higher-risk myelodysplastic syndrome (HR-MDS) patients with RARA gene overexpression is notable given the current treatment landscape. HR-MDS is a group of bone marrow disorders characterized by ineffective production of blood cells and a risk of progression to acute myeloid leukemia (AML). Current treatments include hypomethylating agents like azacitidine, which tamibarotene is being tested alongside.
The trial's design, with a 2:1 randomization and a primary endpoint of complete response (CR) rate, aligns with regulatory standards for assessing efficacy. The mention of a 'one-trial' approach suggests a strategic regulatory path that could expedite the drug's approval process if the data are favorable. Moreover, the ability of the trial to potentially serve as a confirmatory study for full approval transition from accelerated approval indicates a well-thought-out development plan. The observed 100% CR/CRi rate in the SELECT-AML-1 Phase 2 trial, although early, provides a positive signal for the drug's efficacy in a related blood cancer, which could bode well for the ongoing Phase 3 trial.
For investors, the announcement by Syros Pharmaceuticals represents a important point in the company's valuation. The successful enrollment of patients in a Phase 3 trial is often seen as reducing clinical development risk, potentially leading to positive stock market reactions. Investors will be closely monitoring the upcoming complete response data in 4Q 2024 as it could significantly impact the company's stock price.
The market for HR-MDS treatments is a niche but important one and successful trial outcomes could position Syros favorably in this space. The potential filing of a New Drug Application (NDA) with the FDA, contingent on successful trial results, would be a major catalyst for the company's financial prospects. However, it is essential to consider that the biopharmaceutical sector is highly volatile and outcomes are uncertain. Thus, while the news is promising, the actual impact on the company's financial health will depend on the trial results and subsequent regulatory decisions.
From a clinical perspective, the development of tamibarotene for HR-MDS patients is an important advancement. HR-MDS is a challenging condition to treat and the overexpression of the RARA gene presents an opportunity for targeted therapy. If tamibarotene demonstrates a significant CR rate without increased toxicity, it could become a valuable addition to the current standard of care. The combination therapy approach, using tamibarotene with azacitidine and venetoclax, reflects a trend in oncology towards personalized medicine and combination regimens to improve outcomes.
The secondary endpoint of overall survival (OS) is also critical, as it is the ultimate measure of a treatment's benefit. The trial's results will not only influence clinical practices but also have implications for patient quality of life and long-term health outcomes. The oncology community will be keenly awaiting the detailed data from this trial to assess the real-world applicability of tamibarotene for HR-MDS patients.
-- On track to report pivotal complete response data by mid-4Q 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 file our first New Drug Application (NDA) with the
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
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 www.syros.com 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 file with the Securities and Exchange Commission; and risks described in other filings that Syros makes with the Securities and Exchange Commission in the future.
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Syros Contact
Karen Hunady
Director of Corporate Communications & Investor Relations
1-857-327-7321
khunady@syros.com
Investor Contact
Hannah Deresiewicz
Stern Investor Relations, Inc.
212-362-1200
hannah.deresiewicz@sternir.com
Source: Syros Pharmaceuticals
FAQ
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