Syros Receives Positive Opinion on Orphan Drug Designation from the European Medicines Agency for Tamibarotene for the Treatment of MDS
August 03 2022 - 4:01PM
Business Wire
Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development
of medicines that control the expression of genes, today announced
that the European Medicines Agency (EMA) issued a positive opinion
on the Company’s application for orphan drug designation for
tamibarotene for the treatment of myelodysplastic syndrome (MDS).
Tamibarotene, an oral first-in-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 U.S. 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 for
orphan drug designation for tamibarotene as it represents an
important milestone for MDS patients, who have an urgent need for
effective, tolerable, and convenient treatment options,” said David
A. Roth, M.D., Syros’ Chief Medical Officer. “We believe
tamibarotene has the potential to change the current standard of
care and become the first therapy for a targeted population in
HR-MDS. We continue to advance our SELECT-MDS-1 trial and are
looking forward to announcing pivotal data in late 2023 or early
2024.”
Orphan drug designation in the European Union (EU) is granted by
the European Commission based on a positive opinion issued by the
EMA Committee for Orphan Medicinal Products. The EMA’s orphan
designation is available to companies developing treatments for
life-threatening or chronically debilitating conditions that affect
fewer than five in 10,000 persons in the EU. Medicines that meet
the EMA’s orphan designation criteria qualify for financial and
regulatory incentives that include a 10-year period of marketing
exclusivity in the EU after product approval, protocol assistance
from the EMA at reduced fees during the product development phase
and access to centralized marketing authorization.
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 trial are expected in the fourth quarter of
2023 or the first quarter of 2024, with a potential new drug
application filing expected in 2024.
Syros is also evaluating tamibarotene in combination with
azacitidine and venetoclax for RARA-positive patients with newly
diagnosed unfit acute myeloid leukemia (AML), for which
tamibarotene had previously received orphan drug designation from
both the FDA and EMA. Data from the safety lead-in portion of the
SELECT-AML-1 Phase 2 trial is expected in the second half of this
year.
About Syros Pharmaceuticals
Syros is redefining the power of small molecules to control the
expression of genes. Based on its unique ability to elucidate
regulatory regions of the genome, Syros aims to develop medicines
that provide a profound benefit for patients with diseases that
have eluded other genomics-based approaches. Syros is advancing a
robust clinical-stage pipeline, including: tamibarotene, a
first-in-class oral selective RARα agonist in RARA-positive
patients with higher-risk myelodysplastic syndrome and acute
myeloid leukemia; SY-2101, a novel 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 select
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.
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 with respect to tamibarotene, the
potential of tamibarotene to benefit RARA-positive HR-MDS patients
and to become the first approved therapy in a targeted population
in HR-MDS, the timing of anticipated data readouts and potential
regulatory submissions from Syros’ clinical trials, and the
potential for Syros’s product candidates to obtain regulatory
approval. In addition, a positive opinion from the EMA on Syros’
application for orphan drug designation for tamibarotene for the
treatment of MDS is not a guarantee of approval of an orphan drug
designation. 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,
including tamibarotene, 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, 2021 and Quarterly Report on
Form 10-Q for the quarter ended March 31, 2022, 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. In addition, the extent to which
the COVID-19 pandemic continues to impact Syros’ workforce and its
clinical trial operations activities, and the operations of the
third parties on which Syros relies, 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 modified government actions, and the
actions that may be required to contain the virus or treat its
impact. Any forward-looking statements contained in this press
release speak only as of the date hereof, and Syros expressly
disclaims any obligation to update any forward-looking statements,
whether because of new information, future events or otherwise.
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version on businesswire.com: https://www.businesswire.com/news/home/20220803005673/en/
Media Contact Courtney Solberg Syros Pharmaceuticals
917-698-9253 csolberg@syros.com
Investor Contact Hannah Deresiewicz Stern Investor
Relations, Inc. 212-362-1200 hannah.deresiewicz@sternir.com
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