CARLSBAD, Calif., Feb. 1, 2024
/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 Rare Pediatric Disease (RPD) Designation to TYRA-300, an
oral FGFR3 selective inhibitor, 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 foramen magnum
and spinal stenosis, hydrocephalus and sleep apnea. A specific DNA
mutation in FGFR3 causes an estimated 99% of achondroplasia.
TYRA is planning to submit an Investigational New Drug
application (IND) to the FDA in the second half of 2024
for the initiation of a randomized Phase 2 clinical trial
evaluating multiple dose cohorts of TYRA-300 for children
with achondroplasia.
"The Rare Pediatric Disease Designation recognizes the severity
of complications associated with achondroplasia in childhood, and
underscores our opportunity with TYRA-300 to develop a highly
selective, oral medication that benefits the achondroplasia
community," said Hiroomi Tada, M.D.
Ph.D., Chief Medical Officer of TYRA. "Children with
achondroplasia face a significant unmet need – and currently, there
are no approved treatment options that address the immediate and
long-term health complications associated with this
condition. We look forward to advancing TYRA-300 to the
clinic and pursuing our goal to bring a much-needed therapy to
these children."
Rare Pediatric Disease (RPD) Designation is granted by the FDA
to investigational drugs and biologics designed to address serious
or life-threatening diseases which affect fewer than 200,000 people
in the United States, or for which
there is no reasonable expectation that the cost of developing and
making the drug or biologic available in the U.S. for the
applicable disease or condition will be recovered from sales in the
U.S., and in which the serious or life-threatening manifestations
primarily affect individuals less than 18 years of age. If a New
Drug Application (NDA) for TYRA-300 to treat achondroplasia is
approved by the FDA, including pediatric populations, TYRA may be
eligible to receive a Priority Review Voucher that can be redeemed
to receive a priority review for any subsequent marketing
application or may be sold or transferred. The FDA has implemented
this program to encourage development of new drugs for treatment of
rare pediatric diseases.
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. In oncology, 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 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 in the second half of 2024 for the initiation of a Phase 2
clinical study in pediatric achondroplasia. TYRA-300 has received
Orphan Drug Designation and Rare Pediatric Designation for the
treatment of achondroplasia from the FDA.
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; the expected timing, design (including dosing levels) and
phase of clinical development of TYRA-300, including timing of a
submission of an IND for TYRA-300 in pediatric achondroplasia; the
potential benefits of Rare Pediatric Disease Designation; 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 TYRA-300 and
TYRA-200 for oncology 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; interim
results of a clinical trial do not predict final results and
clinical outcomes may materially change as patient enrollment
continues, following more comprehensive reviews of the data, and as
more patient data become available; 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; 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; the FDA may not approve an NDA for
TYRA-300 in pediatric achondroplasia, and we may not receive a
Priority Review Voucher (PRV) as a result; an accelerated
development or approval pathway may not be available for TYRA-300
or other product candidates and any such pathway, including any
priority review through the use of a PRV, may not lead to a faster
development process; we may not be able to sell or transfer any
unused PRV for any expected or adequate value or at all; we may not
realize the benefits associated with Orphan Drug Designation,
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; 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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