Syros Receives FDA Orphan Drug Designation for Tamibarotene for the Treatment of MDS
February 02 2022 - 3:01PM
Business Wire
Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development
of medicines that control the expression of genes, today announced
that the U.S. Food and Drug Administration (FDA) has granted orphan
drug designation (ODD) to 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).
“The FDA’s orphan drug designation is an important milestone in
the development of tamibarotene as a treatment for MDS,” said David
A. Roth, M.D., Syros’ Chief Medical Officer. “We believe
tamibarotene’s novel mechanism of action, promising clinical
activity data, oral delivery, and favorable tolerability profile
supports a potential new option for the approximately 30% of HR-MDS
patients who are RARA-positive. We are focused on developing the
first potential therapy for a targeted population in HR-MDS as we
continue to advance our ongoing SELECT-MDS-1 pivotal trial.”
The FDA's Office of Orphan Drug Products grants orphan status to
support development of medicines for the treatment of rare diseases
that affect fewer than 200,000 people in the United States. Orphan
drug designation may provide certain benefits, including a
seven-year period of market exclusivity if the drug is approved,
tax credits for qualified clinical trials and an exemption from FDA
application fees.
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.
Safety lead-in data from the ongoing 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 and blood cancers. 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. 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, 2020 and Quarterly Report on
Form 10-Q for the quarter ended September 30, 2021, 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220202005820/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
Syros Pharmaceuticals (NASDAQ:SYRS)
Historical Stock Chart
From Apr 2024 to May 2024
Syros Pharmaceuticals (NASDAQ:SYRS)
Historical Stock Chart
From May 2023 to May 2024