CAMBRIDGE, Massachusetts, August 3, 2022–(BUSINESS WIRE)–Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of drugs that regulate gene expression, today announced that the European Medicines Agency (EMA) has issued a positive opinion on the application of the orphan drug company drug designation for tamibarotene for the treatment of myelodysplastic syndrome (MDS). Tamibarotene, an oral first-class selective retinoic acid receptor alpha (RARα) agonist, is currently being evaluated in combination with azacitidine in the SELECT-MDS-1 phase 3 trial for RARA-positive patients with newly diagnosed higher-risk MDS (HR-MDS ).
Previously, the US Food and Drug Administration (FDA) granted orphan drug designation to tamibarotene in MDS in February 2022.
“We are pleased that the EMA has issued a positive opinion on the orphan drug designation for tamibarotene, as it represents an important milestone for MDS patients, who urgently need effective, acceptable and convenient treatment options,” said David A. Roth, MD, Chief Medical Officer of Syros. “We believe that tamibarotene has the potential to change the current standard of care and become the first therapy for a specific population in HR-MDS. We continue to advance our SELECT-MDS-1 trial and look forward to providing critical data by the end of 2023 or early 2024.”
Orphan medicinal product designation in the European Union (EU) is granted by the European Commission on the basis of a positive opinion from the EMA Committee for Orphan Medicinal Products. EMA orphan drug designation is available to companies developing treatments for life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the EU. Medicines that meet EMA’s orphan designation criteria are eligible for financial and regulatory incentives, including a 10-year marketing exclusivity period in the EU after product approval, EMA protocol support at reduced fees during the product development phase and access to centralized marketing authorizations .
The ongoing SELECT-MDS-1 Phase 3 clinical trial is evaluating the safety and efficacy of tamibarotene in combination with azacitidine for RARA-positive patients with newly diagnosed HR-MDS. Data from the pivotal study is expected in the fourth quarter of 2023 or the first quarter of 2024, with a possible filing of a new drug application in 2024.
Syros is also evaluating tamibarotene in combination with azacitidine and venetoclax for RARA-positive patients with newly diagnosed unsuitable acute myeloid leukemia (AML), for which tamibarotene had previously received orphan drug designation from both the FDA and EMA. Data from the preliminary safety portion of the SELECT-AML-1 Phase 2 trial is expected in the second half of this year.
About Syros Pharmaceuticals
Syros redefines the power of small molecules to control gene expression. Based on its unique ability to elucidate regulatory regions of the genome, Syros aims to develop drugs that offer significant benefit to patients with diseases that have eluded other genomics-based approaches. Syros is advancing a robust clinical phase pipeline, including: tamibarotene, a first-class oral selective RARα agonist in RARA-positive patients with higher-risk myelodysplastic syndromes and acute myeloid leukemia; SY-2101, a new oral form of arsenic trioxide in patients with acute promyelocytic leukemia; and SY-5609, a highly selective and potent oral CDK7 inhibitor in patients with selected solid tumors. Syros also has multiple preclinical and discovery programs in oncology and monogenic diseases. For more information, visit www.syros.com and follow us on Twitter (@SyrosPharma) and LinkedIn.
Warning Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including but not limited to statements regarding Syros’ clinical development plans related to tamibarotene, the potential of tamibarotene for RARA-positive HR-MDS patients and to become the first approved therapy in a target population in HR-MDS, the timing of expected data readouts and potential regulatory filings of Syros’s clinical trials, and the potential for Syros’ product candidates to gain regulatory approval. In addition, a positive opinion from the EMA on Syros’s application for orphan designation for tamibarotene for the treatment of MDS does not guarantee approval of an orphan drug designation. The words ”anticipate” ”believe” ”continue” ”can” ”estimate” ”expect” ”hope” ”intend” ”can,” ”plan” ”potential” ”forecast” ”project” ”target” ”should” ”should” 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 expressed in these forward-looking statements as a result of several important factors, including Syros’ ability to: Promote the development of its programs, including tamibarotene, within timelines it projects into current and future clinical trials; demonstrate the required safety, efficacy and compatibility of its drug candidates in all current and future clinical studies; support the response rates and durability of response observed to date with its drug candidates; successfully develop a companion diagnostic test to identify patients with the RARA biomarker; obtaining and maintaining patent protection for its drug candidates and the freedom to operate under the intellectual property of third parties; obtaining and maintaining the necessary regulatory approvals; identifying, entering into and maintaining cooperation agreements with third parties; manage competition; manage expenses; raising the substantial additional capital required to achieve its business objectives; attract and retain qualified personnel; and successfully execute its business strategies; risks described under the heading “Risk Factors” in Syros’ Annual Report on Form 10-K for the year ended December 31, 2021 and Quarterly Report on Form 10-Q for the quarter ended March 31, 2022, each on file with the Securities and Exchange Commission; and risks described in other filings Syros makes in the future with the Securities and Exchange Commission. In addition, the extent to which the COVID-19 pandemic continues to affect Syros’ workforce and its clinical trials activities, as well as the activities of the third parties that Syros relies on, will depend on future developments, which are highly uncertain and cannot be predicted with confidence, including the duration and severity of the pandemic, additional or amended government actions, and the actions that may be needed to contain the virus or treat its impact. Any forward-looking statements in this press release speak only as of the date hereof, and Syros expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.
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Contacts
Media contact
Courtney Solberg
Syros Pharmaceuticals
917-698-9253
csolberg@syros.com
Contact for investors
Hannah Deresiewicz
Stern Investor Relations, Inc.
212-362-1200
hannah.deresiewicz@sternir.com