CARLSBAD, Calif., Aug. 1, 2023
/PRNewswire/ -- Tyra Biosciences, Inc. (Nasdaq: TYRA), a
clinical-stage biotechnology company focused on developing
next-generation precision medicines that target large opportunities
in Fibroblast Growth Factor Receptor (FGFR) biology, today
announced that the U.S. Food and Drug Administration (FDA) has
granted Orphan Drug Designation (ODD) to its lead precision
medicine program, TYRA-300, for the treatment of
achondroplasia.
Achondroplasia is the most common form of dwarfism with limited
therapeutic options. People living with achondroplasia may
experience severe skeletal complications including cranial and
spinal stenosis, hydrocephalus and sleep apnea. A specific mutation
in FGFR3 causes over 97% of achondroplasia. TYRA-300 is an oral
FGFR3 selective inhibitor whose design may have a meaningful impact
on achondroplasia and other skeletal dysplasias.
"People living with achondroplasia can have significant health
complications that are not adequately addressed with currently
available therapies. Our goals with TYRA-300 in
achondroplasia are to address not only height, but the long-term
health complications associated with this condition," said
Hiroomi Tada, M.D. Ph.D., Chief
Medical Officer of TYRA. "The FDA's decision to grant Orphan
Drug Designation to TYRA-300 is an important recognition of the
potential of our approach to deliver benefit to the achondroplasia
community. We remain on track to submit an IND to the FDA to
enable a Phase 2 study of TYRA-300 in pediatric achondroplasia in
2024."
The FDA's Office of Orphan Products Development grants orphan
designation status to drugs and biologics that are intended for
treatment, diagnosis or prevention of rare diseases and conditions
that affect fewer than 200,000 people in the U.S. Orphan Drug
Designation provides certain benefits, including tax credits for
qualified clinical trials and exemption from certain user fees to
support clinical development and the potential for up to seven
years of market exclusivity in the U.S. upon regulatory
approval.
TYRA also announced today the appointment of Michael Bober, M.D. Ph.D., as Vice
President, Clinical Development and Medical Affairs, to lead the
skeletal dysplasia program. Dr. Bober is a leader in the
diagnosis and management of skeletal dysplasia. He served on
numerous scientific and medical advisory boards within the skeletal
dysplasia community. Dr. Bober joins TYRA following a
distinguished career as the Medical Director of the Skeletal
Dysplasia Program, Nemours Children's Hospital, Delaware.
Dr. Bober added, "I am excited to join TYRA and contribute to
the team working to develop TYRA-300 into a therapy for the patient
community which I care so much about. I believe TYRA-300 has the
potential to improve function and quality of life in
achondroplasia."
About TYRA-300
TYRA-300 is the Company's lead precision medicine program
stemming from its in-house SNÅP platform. TYRA-300 is an
investigational, oral, FGFR3-selective inhibitor currently in
development for the treatment of cancer and skeletal dysplasias
including achondroplasia. TYRA-300 is being evaluated in a
multi-center, open label Phase 1/2 clinical study, SURF301
(Study in Untreated and Resistant
FGFR3+ Advanced Solid Tumors). SURF301 (NCT05544552) was
designed to determine the optimal and maximum tolerated doses (MTD)
and the recommended Phase 2 dose (RP2D) of TYRA-300, as well as to
evaluate the preliminary antitumor activity of TYRA-300.
SURF301 is currently enrolling adults with advanced urothelial
carcinoma and other solid tumors with FGFR3 gene alterations.
In skeletal dysplasias, TYRA-300 has demonstrated positive
preclinical results and the Company expects to submit an IND for
the initiation of a Phase 2 clinical study in pediatric
achondroplasia in 2024.
About Tyra Biosciences
Tyra Biosciences, Inc. (Nasdaq: TYRA) is a clinical-stage
biotechnology company focused on developing next-generation
precision medicines that target large opportunities in FGFR
biology. The Company's in-house precision medicine platform, SNÅP,
enables rapid and precise drug design through iterative molecular
SNÅPshots that help predict genetic alterations most likely to
cause acquired resistance to existing therapies. TYRA's initial
focus is on applying its accelerated small molecule drug discovery
engine to develop therapies in targeted oncology and genetically
defined conditions. TYRA is based in Carlsbad, CA. For more information about our
science, pipeline and people, please visit
www.tyra.bio and engage with us on LinkedIn.
Forward-Looking Statements
TYRA cautions you that statements contained in this press
release regarding matters that are not historical facts are
forward-looking statements. The forward-looking statements are
based on our current beliefs and expectations and include, but are
not limited to: the potential to develop next-generation precision
medicines and the potential safety and therapeutic benefits of
TYRA-300 and other product candidates, including the potential for
TYRA-300 to become a treatment option for achondroplasia; the
expected timing and phase of clinical development of TYRA-300,
including timing of a submission of an IND for TYRA-300 in
pediatric achondroplasia; and the potential for SNÅP to enable
rapid and precise drug design. Actual results may differ from those
set forth in this press release due to the risks and uncertainties
inherent in our business, including, without limitation: we are
early in our development efforts, have only recently begun testing
our lead product candidate in clinical trials and the approach we
are taking to discover and develop drugs based on our SNÅP platform
is novel and unproven and it may never lead to product candidates
that are successful in clinical development or approved products of
commercial value; potential delays in the commencement, enrollment,
and completion of preclinical studies and clinical trials; results
from preclinical studies or early clinical trials not necessarily
being predictive of future results; our dependence on third parties
in connection with manufacturing, research and preclinical testing;
acceptance by the FDA of INDs or of similar regulatory submissions
by comparable foreign regulatory authorities for the conduct of
clinical trials of TYRA-300 in pediatric achondroplasia; an
accelerated development or approval pathway may not be available
for TYRA-300 or other product candidates and any such pathway may
not lead to a faster development process; unexpected adverse side
effects or inadequate efficacy of our product candidates that may
limit their development, regulatory approval, and/or
commercialization; the potential for our programs and prospects to
be negatively impacted by developments relating to our competitors,
including the results of studies or regulatory determinations
relating to our competitors; we may not realize the benefits
associated with ODD, including that orphan drug exclusivity may not
effectively protect a product from competition and that such
exclusivity may not be maintained; regulatory developments in
the United States and foreign
countries; we may use our capital resources sooner than we expect;
unstable market and economic conditions and adverse developments
with respect to financial institutions and associated liquidity
risk may adversely affect our business and financial condition and
the broader economy and biotechnology industry; and other risks
described in our prior filings with the Securities and Exchange
Commission (SEC), including under the heading "Risk Factors" in our
annual report on Form 10-K and any subsequent filings with the SEC.
You are cautioned not to place undue reliance on these
forward-looking statements, which speak only as of the date hereof,
and we undertake no obligation to update such statements to reflect
events that occur or circumstances that exist after the date
hereof. All forward-looking statements are qualified in their
entirety by this cautionary statement, which is made under the safe
harbor provisions of the Private Securities Litigation Reform Act
of 1995.
Contact:
Amy Conrad
aconrad@tyra.bio
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SOURCE Tyra Biosciences