– TYRA-300 is the first oral FGFR-3 selective
inhibitor to be well-tolerated in clinical studies –
– First child with achondroplasia expected to
be dosed in Q1 2025 –
CARLSBAD, Calif., Oct. 28,
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, announced
today that the U.S. Food and Drug Administration (FDA) cleared its
Investigational New Drug (IND) application for TYRA-300 allowing
the company to proceed with a Phase 2 clinical trial of TYRA-300
for children with achondroplasia (BEACH301).
TYRA-300 is a potential first-in-class, investigational, oral,
FGFR3-selective inhibitor designed to avoid the toxicities
associated with inhibition of FGFR1, FGFR2 and FGFR4.
TYRA-300 is also being evaluated for metastatic urothelial cancer
in the ongoing SURF301 study, where interim clinical
proof-of-concept data were recently reported at the ENA 2024
meeting.
"IND clearance to proceed with BEACH301 is a significant
milestone for the achondroplasia community and for TYRA, as we move
into the clinic to treat our first rare skeletal dysplasia
indication," said Todd Harris, CEO of TYRA. "We believe
FGFR3 is the right target for achondroplasia, with almost one
hundred percent of cases being driven by a specific mutation in the
FGFR3 gene. TYRA-300 has the potential to precisely
engage FGFR3 to potentially achieve a higher annualized growth
velocity, and lead to important functional outcomes and clinical
benefits such as improvements in reach, gait and spinal
disease."
Mr. Harris continued, "The currently available therapy is a
once-daily injection that delivers modest increases in annualized
growth velocity. As a highly selective FGFR3 inhibitor, we
are hopeful that TYRA-300 may provide an improved therapeutic
option for achondroplasia, and we are excited about our opportunity
to potentially deliver improvements with our differentiated oral
therapy in BEACH301."
BEACH301 will be a Phase 2, multicenter, open-label,
dose-escalation/dose-expansion study evaluating TYRA-300 in
children ages 3 to 10 with achondroplasia with open growth plates.
The study will enroll children who are treatment-naïve (Cohort 1)
and those who have received prior growth-accelerating therapy
(Cohort 2) at multiple sites across the globe. Each of these
cohorts is expected to enroll up to 10 participants per dose level
(0.125, 0.25, 0.375, 0.50 mg/kg) for up to 12 months. Prior
to initiation of Cohorts 1 and 2, the study will enroll a safety
sentinel cohort of up to 3 treatment-naïve participants per dose
level in children ages 5 to 10.
The primary objectives of this study will be to assess safety
and tolerability in children with achondroplasia and evaluate
change from baseline in annualized growth velocity to determine the
dose(s) for further development. Secondary objectives will include
evaluating change from baseline in height z-score, proportionality
and pharmacokinetics (PK). TYRA is also planning exploratory
assessments of clinical outcomes such as functional improvements,
changes in the spine, and quality of life measures.
"We are excited to expand the clinical development of TYRA-300
into achondroplasia with BEACH301. Our existing database from
the SURF301 oncology study includes information on doses
significantly higher than what we are planning in
achondroplasia. We believe this information suggest TYRA-300
may be well tolerated at low doses in children," said Doug Warner, MD, Chief Medical Officer of
TYRA. "We are continuing to engage with the achondroplasia
community, including advocates and physicians, as we actively work
to initiate the BEACH301 study and commence dosing in the first
quarter of 2025."
In July 2023 and January 2024, the FDA granted
Orphan Drug Designation (ODD) and Rare Pediatric Designation (RPD)
to TYRA-300, respectively, for the treatment of achondroplasia.
TYRA is committed to exploring the potential of TYRA-300 for
functional impacts and quality of life measures in achondroplasia,
hypochondroplasia and other skeletal dysplasia.
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 dysplasia,
including achondroplasia and hypochondroplasia. 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)
(NCT05544552). The study is designed to determine the optimal
and the recommended Phase 2 dose (RP2D) of TYRA-300, as well as to
evaluate the preliminary antitumor activity of TYRA-300. Part
A of the study included patients with all solid tumors who are
FGFR3 +/-, and explored doses of TYRA-300 ranging from 10mg -120mg
once-daily (QD). Part A of SURF301 is complete. The
Company continues to advance TYRA-300 through dose expansion in
Part B, which includes patients with solid tumors who are
FGFR3+, to evaluate potentially therapeutic doses in
preparation for potential future Phase 2 studies in metastatic
urothelial carcinoma and non-muscle invasive bladder cancer.
In skeletal dysplasia, TYRA-300 has demonstrated positive
preclinical results in achondroplasia and hypochondroplasia and
TYRA has received IND clearance from the U.S FDA to proceed with
its BEACH301 clinical trial in children with achondroplasia.
About Achondroplasia
Achondroplasia is the most common form of dwarfism with limited
therapeutic options. It is estimated that 1 in 15,000 to
40,000 children are born have achondroplasia, with approximately
250,000 affected individuals worldwide. People living with
achondroplasia may experience severe complications including
foramen magnum and spinal stenosis, sleep apnea and
disproportionate short stature. An FGFR3 G380R gain of
function mutation accounts for approximately 99% of achondroplasia.
TYRA-300 is a first-in-class oral FGFR3 selective inhibitor whose
design may have a meaningful impact on achondroplasia and other
skeletal dysplasia.
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 expertise
in FGFR biology has created a differentiated pipeline with three
clinical-stage programs in targeted oncology and genetically
defined conditions. The Company's lead precision medicine
stemming from SNÅP, TYRA-300, is a potential first-in-class
selective FGFR3 inhibitor that is designed to avoid the toxicities
associated with inhibition of FGFR1, FGFR2 and FGFR4, while being
agnostic for the FGFR3 gatekeeper mutations. TYRA-300 is in
development for the treatment of cancer in the SURF301 Phase 1/2
study and for achondroplasia in the BEACH301 Phase 2 study.
TYRA is also developing TYRA-200, an investigational, FGFR1/2/3
inhibitor, in the SURF201 study for metastatic intrahepatic
cholangiocarcinoma, and TYRA-430, an investigational FGFR4/3-biased
inhibitor for FGF19+/FGFR4-driven cancers. 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: expected initiation of the BEACH301 study and the
timing thereof; the design and goals of the BEACH301 study; the
potential to develop next-generation precision medicines and the
potential safety and therapeutic benefits of TYRA-300; the
continued evaluation of TYRA-300 in SURF301; and the potential for
SNÅP to develop therapies in targeted oncology and genetically
defined conditions. 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: later developments
with the FDA may be inconsistent with prior feedback from the FDA,
including with respect to the proposed initiation and design of our
planned Phase 2 study of TYRA-300 in achondroplasia; 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, data readouts and completion of
preclinical studies and clinical trials; results from preclinical
studies or early clinical trials not necessarily being predictive
of future results; interim results of a clinical trial are not
necessarily indicative of final results and one or more of the
clinical outcomes may materially change as patient enrollment
continues, following more comprehensive reviews of the data, as
follow-up on the outcome of any particular patient continues and as
more patient data becomes available, including the risk that
unconfirmed responses may not ultimately result in confirmed
responses to treatment after follow-up evaluations; the potential
for proof-of-concept results to fail to result in successful
subsequent development of TYRA-300; 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; 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; unfavorable results from preclinical studies; 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, or
from the RPD Designation, including receipt of a Priority Review
Voucher or any value therefrom; regulatory developments in the
United States and foreign countries; our ability to obtain and
maintain intellectual property protection for our product
candidates and proprietary technologies; 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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