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. 

加利福尼亚州卡尔斯巴德，2024年2月1日/PRNewswire/--Tyra Biosciences，Inc.（纳斯达克：Tyra），一家临床阶段生物技术公司，专注于开发针对成纤维细胞生长因子受体（FGFR）生物学大量机会的下一代精准药物，今天宣布美国食品和药物管理局（FDA）已将罕见儿科疾病（RPD）指定为Tyra-300，一种口服FGFR3选择性抑制剂，用于治疗软骨发育不全。

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.

软骨发育不全是侏儒症最常见的形式，治疗选择有限。软骨发育不全患者可能会出现严重的骨骼并发症，包括枕骨大孔和椎管狭窄，脑积水和睡眠呼吸暂停。FGFR3中的特定DNA突变导致估计99%的软骨发育不全。

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. 

TYRA计划在2024年下半年向FDA提交一份研究性新药申请（IND），以启动一项随机2期临床试验，评估TYRA-300对软骨发育不全儿童的多剂量组群。

'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.'

TYRA首席医疗官Hiroomi Tada医学博士说：“罕见的儿科疾病名称认识到儿童期软骨发育不全相关并发症的严重程度，并强调了我们与TYRA-300合作开发一种高选择性口服药物的机会，这种药物对软骨发育不全社区有益。”“软骨发育不全的儿童面临着巨大的未满足需求，目前还没有批准的治疗方案来解决与这种情况相关的即时和长期健康并发症。我们期待着将TYRA-300推进诊所，并追求我们的目标，为这些儿童带来急需的治疗。”

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.

美国食品和药物管理局（FDA）将罕见儿科疾病（RPD）指定为研究药物和生物制剂，旨在解决影响美国不到20万人的严重或危及生命的疾病，或者没有合理的预期在美国开发和制造药物或生物制剂的成本。

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.

对于适用的疾病或状况，将从美国的销售中恢复，其中严重或危及生命的表现主要影响18岁以下的个人。如果TYRA-300治疗软骨发育不全的新药申请（NDA）得到FDA（包括儿科人群）的批准，TYRA可能有资格获得优先审查凭证，该凭证可以兑换以接受任何后续营销申请的优先审查，也可以出售或转让。

The FDA has implemented this program to encourage development of new drugs for treatment of rare pediatric diseases..

FDA实施了这一计划，以鼓励开发治疗罕见儿科疾病的新药。

About TYRA-300

关于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).

TYRA-300是该公司的领先精准医学计划，源自其内部SNÅP平台。TYRA-300是一种研究性口服FGFR3选择性抑制剂，目前正在开发中，用于治疗癌症和骨骼发育不良，包括软骨发育不全。在肿瘤学方面，TYRA-300正在一项多中心，开放标签的1/2期临床研究SURF301（未经治疗和耐药的FGFR3+晚期实体瘤研究）中进行评估。

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.

SURF301（NCT05544552）旨在确定TYRA-300的最佳和MTD以及推荐的2期剂量（RP2D），并评估TYRA-300的初步抗肿瘤活性。SURF301目前正在招募患有晚期尿路上皮癌和其他具有FGFR3基因改变的实体瘤的成年人。

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..

在骨骼发育不良中，TYRA-300已显示出积极的临床前结果，该公司预计将在2024年下半年提交IND，以启动小儿软骨发育不全的2期临床研究。TYRA-300已获得FDA关于治疗软骨发育不全的孤儿药和罕见儿科药物。。

About Tyra Biosciences

关于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 Biosciences，Inc.（纳斯达克股票代码：Tyra）是一家临床阶段生物技术公司，专注于开发针对FGFR生物学巨大机遇的下一代精准药物。该公司的内部精密医学平台SNÅP通过迭代分子SNÅPshots实现了快速而精确的药物设计，这些分子SNÅPshots有助于预测最有可能导致对现有疗法产生获得性耐药的基因改变。

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..

TYRA最初的重点是应用其加速的小分子药物发现引擎来开发靶向肿瘤学和基因定义条件下的疗法。TYRA总部位于加利福尼亚州卡尔斯巴德。

Forward-Looking Statements

前瞻性声明

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.

TYRA提醒您，本新闻稿中关于非历史事实的声明是前瞻性声明。前瞻性声明基于我们目前的信念和期望，包括但不限于：开发下一代精准药物的潜力以及TYRA-300的潜在安全性和治疗益处；TYRA-300的预期时间，设计（包括剂量水平）和临床开发阶段，包括在小儿软骨发育不全中提交TYRA-300 IND的时间；罕见儿科疾病指定的潜在益处；以及SNÅP实现快速准确药物设计的潜力。

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.

由于我们业务中固有的风险和不确定性，实际结果可能与本新闻稿中的结果有所不同，包括但不限于：我们的开发工作刚刚起步，最近才开始在临床试验中测试TYRA-300和TYRA-200的肿瘤学，我们正在基于我们的SNÅP平台发现和开发药物的方法是新颖且未经证实的，它可能永远不会导致候选产品在临床开发或批准的商业价值产品中取得成功；临床前研究和临床试验的开始，注册和完成可能延迟；临床试验的中期结果不能预测最终结果，随着对数据进行更全面的审查以及更多患者数据的可用性，随着患者登记的继续，临床结果可能会发生重大变化；临床前研究或早期临床试验的结果不一定能预测未来的结果；我们在制造、研究和临床前测试方面依赖第三方；验收人。