SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Septerna, a clinical-stage biotechnology company pioneering a new era of GPCR-targeted drug discovery, presented new preclinical data from its parathyroid hormone 1 receptor (PTH1R) program in a poster presentation at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting held from September 27-30, 2024, in Toronto, ON, Canada..

加利福尼亚州南旧金山（商业新闻短讯）--Septerna是一家临床阶段生物技术公司，开创了GPCR靶向药物发现的新时代，在2024年9月27日至30日于加拿大安大略省多伦多举行的美国骨与矿物质研究学会（ASBMR）2024年年会上的海报展示中，展示了其甲状旁腺激素1受体（PTH1R）计划的新临床前数据。。

The data demonstrate that Septerna’s PTH1R small molecule agonists activate PTH1R and elicit downstream effects on bone and kidney by regulating key genes important for calcium homeostasis in a manner similar to native parathyroid hormone (PTH). In addition, building on data presented at the Endocrine Society’s Annual Meeting (ENDO), Septerna’s PTH1R agonists demonstrated sustained control of serum calcium and phosphate levels and upregulation of bone turnover biomarkers and increases in active Vitamin D levels over 28 days with daily oral administration.

数据表明，Septerna的PTH1R小分子激动剂通过以类似于天然甲状旁腺激素（PTH）的方式调节对钙稳态重要的关键基因，激活PTH1R并引发对骨骼和肾脏的下游作用。此外，根据内分泌学会年会（ENDO）上提供的数据，Septerna的PTH1R激动剂在28天内持续控制血清钙和磷酸盐水平，上调骨转换生物标志物，并增加活性维生素D水平，每天口服给药。

Septerna is dosing healthy volunteers in a Phase 1 clinical trial to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of its lead oral small molecule PTH1R agonist product candidate, SEP-786..

Septerna正在一期临床试验中给健康志愿者服用，以评估其口服小分子PTH1R激动剂候选产品SEP-786的安全性、耐受性、药代动力学和药效学。。

“We are pleased to highlight data from the preclinical studies of our PTH1R small molecule agonists demonstrating activity similar to native PTH, which support the initiation of our clinical trial and affirm our belief that our lead product candidate, SEP-786, has the potential to reshape the treatment paradigm for patients with hypoparathyroidism,” said Jeffrey Finer, M.D., Ph.D., Chief Executive Officer and Co-founder of Septerna.

Septerna首席执行官兼联合创始人Jeffrey Finer博士说：“我们很高兴强调PTH1R小分子激动剂的临床前研究数据，这些数据表明其活性与天然PTH相似，这支持了我们临床试验的启动，并证实了我们的信念，即我们的主要候选产品SEP-786有可能重塑甲状旁腺功能减退症患者的治疗模式。”。

“The progress of this program is an excellent illustration of the ability of our Native Complex Platform™ to unlock difficult-to-drug GPCRs and discover novel small molecules with the potential to address numerous disorders in endocrinology and beyond.”.

“该计划的进展很好地说明了我们的Native Complex Platform™能够解锁难以药物治疗的GPCR，并发现新的小分子，这些小分子有可能解决内分泌学等多种疾病。”。

Data Summary

数据摘要

The poster, entitled “Characterization of a Novel Oral Small Molecule PTH1R Agonist: Sustained Control of Serum Calcium and Modulation of Bone Biomarkers in TPTx Rats,” is summarized below.

题为“新型口服小分子PTH1R激动剂的表征：TPTx大鼠血清钙的持续控制和骨生物标志物的调节”的海报总结如下。

Primary human renal proximal epithelial cells and a human osteoblast cell line treated with PTH and a small molecule agonist of PTH1R suggest similar pathway engagement.

用PTH和PTH1R的小分子激动剂处理的原代人肾近端上皮细胞和人成骨细胞系表明类似的途径参与。

Comparable on target bone activities (anabolic and catabolic) were observed in mouse bones treated with PTH peptide and a PTH1R small molecule agonist.

在用PTH肽和PTH1R小分子激动剂处理的小鼠骨骼中观察到可比较的靶向骨活性（合成代谢和分解代谢）。

Daily oral administration of a PTH1R agonist resulted in sustained, dose dependent regulation of serum calcium and phosphate levels over a 28-day period.

每天口服PTH1R激动剂可在28天内持续剂量依赖性地调节血清钙和磷酸盐水平。

New data were presented demonstrating that daily dosing of a PTH1R agonist upregulated bone turnover biomarkers and active Vitamin D levels in a dose-dependent manner over 28 days.

提供的新数据表明，PTH1R激动剂的每日剂量在28天内以剂量依赖性方式上调骨转换生物标志物和活性维生素D水平。

About SEP-786 and Hypoparathyroidism

关于SEP-786和甲状旁腺功能减退

Septerna is developing SEP-786, a novel, potent and selective oral small molecule parathyroid hormone 1 receptor (PTH1R) agonist for the treatment of patients with hypoparathyroidism, a rare endocrine disease characterized by a deficiency of the parathyroid hormone (PTH). Hypoparathyroidism results in a wide range of debilitating symptoms, including muscle cramps, fatigue, cognitive dysfunction and life-threatening complications, such as cardiac arrhythmias, seizures, and renal failure.

Septerna正在开发SEP-786，这是一种新型，有效且选择性的口服小分子甲状旁腺激素1受体（PTH1R）激动剂，用于治疗甲状旁腺功能减退症患者，甲状旁腺功能减退症是一种罕见的内分泌疾病，其特征是甲状旁腺激素（PTH）缺乏。甲状旁腺功能减退会导致多种衰弱症状，包括肌肉痉挛，疲劳，认知功能障碍和危及生命的并发症，例如心律不齐，癫痫发作和肾衰竭。

Currently available treatments include supplements that only partially address PTH deficiency, or PTH peptide replacements, which require daily injections. Preclinical safety studies support that SEP-786 has demonstrated the ability to normalize serum calcium and was generally well-tolerated in 28-day Good Laboratory Practice toxicology studies.

目前可用的治疗方法包括仅部分解决PTH缺乏症的补充剂，或需要每日注射的PTH肽替代品。临床前安全性研究支持SEP-786已证明血清钙正常化的能力，并且在28天的良好实验室实践毒理学研究中通常具有良好的耐受性。

SEP-786 has the potential to be a differentiated treatment for hypoparathyroidism..

SEP-786有可能成为甲状旁腺功能减退症的分化治疗方法。。

About Septerna

关于Septerna

Septerna, Inc. is a clinical-stage biotechnology company pioneering a new era of G protein-coupled receptor (GPCR) oral small molecule drug discovery powered by its proprietary Native Complex Platform™. Its industrial-scale platform aims to unlock the full potential of GPCR therapies and has led to the discovery and development of its deep pipeline of product candidates focused initially on treating patients in three therapeutic areas: endocrinology, immunology and inflammation, and metabolic diseases.

Septerna，Inc.是一家临床阶段的生物技术公司，开创了G蛋白偶联受体（GPCR）口服小分子药物发现的新时代，该药物由其专有的Native Complex Platform™提供支持。其工业规模的平台旨在释放GPCR疗法的全部潜力，并导致其深层候选产品的发现和开发，最初专注于治疗三个治疗领域的患者：内分泌学，免疫学和炎症以及代谢疾病。

Septerna was launched in 2022 by preeminent drug discovery company builders and scientific leaders in the biochemistry, structural biology, and pharmacology of GPCRs. For more information, please visit www.septerna.com..

Septerna于2022年由GPCR的生物化学，结构生物学和药理学领域的杰出药物发现公司建设者和科学领导者推出。欲了解更多信息，请访问www.septerna.com。。