Tyra Biosciences Receives IND Clearance from FDA to Proceed with Phase 2 Study of TYRA-300 in Pediatric Achondroplasia (BEACH301)
Tyra Biosciences received FDA clearance for its IND application to proceed with BEACH301, a Phase 2 clinical trial of TYRA-300 in pediatric achondroplasia. TYRA-300 is the first oral FGFR3-selective inhibitor well-tolerated in clinical studies. The trial will evaluate children ages 3-10 with achondroplasia, enrolling up to 10 participants per dose level in treatment-naïve and previously treated cohorts. The study aims to assess safety, tolerability, and changes in growth velocity. First patient dosing is expected in Q1 2025. TYRA-300 received both Orphan Drug and Rare Pediatric Designations from the FDA for achondroplasia treatment.
Tyra Biosciences ha ricevuto l'approvazione della FDA per la sua domanda IND per procedere con BEACH301, uno studio clinico di Fase 2 su TYRA-300 per l'achondroplasia pediatrica. TYRA-300 è il primo inibitore orale selettivo del FGFR3 ben tollerato negli studi clinici. Lo studio valuterà bambini di età compresa tra 3 e 10 anni con achondroplasia, arruolando fino a 10 partecipanti per livello di dosaggio in coorti naive al trattamento e precedentemente trattate. L'obiettivo dello studio è valutare la sicurezza, la tollerabilità e le variazioni nella velocità di crescita. Il primo dosaggio per il paziente è previsto per il primo trimestre del 2025. TYRA-300 ha ricevuto dalla FDA sia la designazione di Farmaco Orfano che di Designazione Pediatrica Rara per il trattamento dell'achondroplasia.
Tyra Biosciences recibió la aprobación de la FDA para su solicitud IND para proceder con BEACH301, un ensayo clínico de Fase 2 de TYRA-300 en achondroplasia pediátrica. TYRA-300 es el primer inhibidor oral selectivo de FGFR3 que ha mostrado buena tolerancia en estudios clínicos. El ensayo evaluará a niños de 3 a 10 años con achondroplasia, inscribiendo hasta 10 participantes por nivel de dosis en cohortes naïve al tratamiento y previamente tratadas. El estudio tiene como objetivo evaluar la seguridad, la tolerancia y los cambios en la velocidad de crecimiento. La primera dosificación del paciente se espera para el primer trimestre de 2025. TYRA-300 recibió tanto la designación de Medicamento Huérfano como la Designación Pediátrica Rara de la FDA para el tratamiento de la achondroplasia.
타이라 바이오사이언스는 소아 왜소증 치료를 위한 BEACH301의 IND 신청서가 FDA 승인을 받았습니다. TYRA-300은 임상 연구에서 잘 견딜 수 있는 첫 번째 경구 FGFR3 선택적 억제제입니다. 이 시험은 3세에서 10세 사이의 왜소증 아동을 평가하며, 치료를 받지 않은 그룹과 이전에 치료를 받은 그룹에서 각 용량 수준 당 최대 10명을 등록합니다. 이 연구의 목표는 안전성, 내약성 및 성장 속도의 변화를 평가하는 것입니다. 첫 환자 투여는 2025년 1분기에 예상됩니다. TYRA-300은 왜소증 치료를 위해 FDA로부터 고아약 및 희귀 소아 디자인ation을 받았습니다.
Tyra Biosciences a reçu l'autorisation de la FDA pour sa demande IND afin de procéder avec BEACH301, un essai clinique de Phase 2 de TYRA-300 chez des enfants atteints d'achondroplasie. TYRA-300 est le premier inhibiteur oral sélectif de FGFR3 bien toléré dans les études cliniques. L'essai évaluera les enfants âgés de 3 à 10 ans atteints d'achondroplasie, en recrutant jusqu'à 10 participants par niveau de dose dans des cohortes naïves de traitement et précédemment traitées. L'étude vise à évaluer la sécurité, la tolérance et les modifications de la vitesse de croissance. Le premier dosage du patient est prévu pour le premier trimestre 2025. TYRA-300 a reçu de la FDA à la fois la désignation de médicament orphelin et la désignation de pédiatrie rare pour le traitement de l'achondroplasie.
Tyra Biosciences erhielt die Zulassung der FDA für seinen IND-Antrag, um mit BEACH301, einer Phase-2-Studie zu TYRA-300 bei pädiatrischer Achondroplasie, fortzufahren. TYRA-300 ist der erste oral gut verträgliche FGFR3-selektive Inhibitor, der in klinischen Studien getestet wurde. Die Studie wird Kinder im Alter von 3 bis 10 Jahren mit Achondroplasie evaluieren und bis zu 10 Teilnehmer pro Dosierungsstufe in behandlungsnaiven und zuvor behandelten Gruppen einschreiben. Ziel der Studie ist es, Sicherheit, Verträglichkeit und Veränderungen in der Wachstumsgeschwindigkeit zu bewerten. Die erste Patientenbehandlung wird im ersten Quartal 2025 erwartet. TYRA-300 erhielt sowohl die Orphan Drug- als auch die Rare Pediatric Designation von der FDA für die Behandlung der Achondroplasie.
- FDA clearance received for Phase 2 clinical trial
- TYRA-300 is first-in-class oral FGFR3-selective inhibitor
- Dual FDA designations: Orphan Drug and Rare Pediatric
- Potential to improve upon current injection-based therapy
- First patient dosing not until Q1 2025
- Efficacy and safety in pediatric population yet to be proven
Insights
The FDA clearance for TYRA-300's Phase 2 trial in pediatric achondroplasia represents a significant milestone in rare disease treatment development. This oral FGFR3-selective inhibitor shows promise in targeting the specific genetic mutation present in nearly all achondroplasia cases, potentially offering advantages over the current injectable standard of care.
The trial design is comprehensive, with planned enrollment of up to 80 patients across multiple dose levels, including both treatment-naïve and previously treated children. The study's focus on both growth velocity and functional outcomes (spine health, mobility, quality of life) suggests a holistic approach to treatment evaluation. The dual-cohort structure, including a safety sentinel cohort, demonstrates a careful approach to pediatric drug development.
The existing safety data from SURF301 oncology trials at higher doses provides encouraging preliminary safety insights, though pediatric safety profiles will need careful monitoring. The Orphan Drug and Rare Pediatric Designations could accelerate development and provide market advantages if approved.
This development positions TYRA strategically in the $1.5+ billion achondroplasia market. The potential for an oral therapy represents a significant competitive advantage over BioMarin's injectable Voxzogo, the current market leader. The comprehensive trial design targeting both treatment-naïve and previously treated patients could maximize market penetration if successful.
The Q1 2025 timeline for first patient dosing provides a clear catalyst for investors to monitor. The expansion into skeletal dysplasias beyond achondroplasia (including hypochondroplasia) suggests multiple potential revenue streams. The dual-purpose development strategy - with TYRA-300 also being studied in urothelial cancer - offers risk mitigation and multiple paths to market value.
– 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 –
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
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
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
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
View original content to download multimedia:https://www.prnewswire.com/news-releases/tyra-biosciences-receives-ind-clearance-from-fda-to-proceed-with-phase-2-study-of-tyra-300-in-pediatric-achondroplasia-beach301-302288159.html
SOURCE Tyra Biosciences
FAQ
What is the purpose of Tyra Biosciences' BEACH301 Phase 2 trial for TYRA-300?
When will Tyra Biosciences (TYRA) begin dosing patients in the BEACH301 trial?
What FDA designations has TYRA-300 received for achondroplasia treatment?
