Agreement between Ascendis Pharma A/S and ST is for the exclusive distribution and commercialisation of three Ascendis Pharma endocrinology therapies

Ascendis Pharma A/S和ST之间的协议是三种Ascendis Pharma内分泌疗法的独家分销和商业化

Two of the therapies are already internationally approved, the third is in development following successful Phase 2 data

其中两种疗法已经获得国际批准，第三种疗法在第二阶段数据成功后正在开发中

ST's exclusive distribution agreement covers Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand and Vietnam

ST的独家经销协议涵盖澳大利亚、新西兰、新加坡、马来西亚、文莱、泰国和越南

SINGAPORE, Jan. 7, 2024 /PRNewswire/ -- Independent biopharmaceutical company Specialised Therapeutics Asia Pte Ltd (ST) has added three new endocrinology therapies to its specialist portfolio, following an exclusive distribution agreement with Danish company Ascendis Pharma A/S (NASDAQ: ASND).

新加坡，2024年1月7日/PRNewswire/--独立生物制药公司Specialized Therapeutics Asia Pte Ltd（ST）与丹麦Ascendis Pharma A/S（NASDAQ:ASND）达成独家经销协议后，在其专业投资组合中增加了三种新的内分泌疗法。

Under the terms of the agreement, ST will commercialise Ascendis Pharma's weekly injectable paediatric human growth hormone treatment SKYTROFATM (lonapegsomatropin), hypoparathyroidism treatment YORVIPATHTM (palopegteriparatide) and investigational achondroplasia therapy TransCon™ CNP (navepegritide)..

根据协议条款，ST将使Ascendis Pharma的每周可注射儿科人类生长激素治疗SKYTROFATM（lonapegsomatropin）、甲状旁腺功能减退治疗Yorvipathm（palopegteriparatide）和研究性软骨发育不全治疗TransCon商业化™ CNP（奈韦格列肽）。。

The agreement spans ST's key regions of Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam.

该协议涵盖澳大利亚、新西兰、新加坡、马来西亚、文莱、泰国和越南等ST的关键地区。

Two of the products included in this agreement are already internationally approved:

本协议中包含的两种产品已获得国际认可：

Once-weekly SKYTROFA is a human growth hormone (hGH) approved in the United States for the treatment of paediatric patients aged >1 years weighing >11.5 kg with growth failure due to inadequate secretion of endogenous growth hormone (GH)1 and in the European Union for growth failure in children and adolescents aged from 3 to 18 years due to insufficient endogenous growth hormone secretion (growth hormone deficiency [GHD]).2.

每周一次的SKYTROFA是一种人类生长激素（hGH），在美国被批准用于治疗1岁以上体重>11.5公斤的儿科患者，由于内源性生长激素（GH）1分泌不足而导致生长衰竭，在欧盟用于治疗3至18岁的儿童和青少年由于内源性生长激素分泌不足而导致的生长衰竭（生长激素缺乏症（GHD））。

YORVIPATH is a first-in-class parathyroid hormone (PTH) replacement therapy to treat chronic hypoparathyroidism, a rare and potentially serious condition where the body produces no or abnormally low levels of PTH. It is approved in the European Union for the treatment of adults with chronic hypoparathyroidism.3.

YORVIPATH是治疗慢性甲状旁腺功能减退症的一流甲状旁腺激素（PTH）替代疗法，慢性甲状旁腺功能减退症是一种罕见且可能严重的疾病，体内不会产生PTH或异常低水平的PTH。它被欧盟批准用于治疗成人慢性甲状旁腺功能减退症。

The third product – TransCon CNP – is in development by Ascendis Pharma for the treatment of achondroplasia (ACH), the most common genetic form of skeletal dysplasia and resulting disproportionate short stature, following successful Phase 2 trial results.4

Ascendis Pharma正在开发第三种产品TransCon CNP，用于治疗软骨发育不全（ACH），这是骨骼发育不良最常见的遗传形式，在2期试验结果成功后导致身材矮小。4

Australian endocrinologist Dr Veronica Preda noted that YORVIPATH would be the first specialist therapeutic option for Australian patients living with hypoparathyroidism.

澳大利亚内分泌学家Veronica Preda博士指出，YORVIPATH将是澳大利亚甲状旁腺功能减退症患者的第一个专业治疗选择。

'Hypoparathyroidism can seriously impact quality of life and has potentially life-threatening consequences,' Dr Preda said.

Preda博士说：“甲状旁腺功能减退会严重影响生活质量，并可能危及生命。”。

'To have an option that is able to treat the underlying cause of the disease, moving beyond standard oral calcium and active Vitamin D, is a great step forward.'

“除了标准的口服钙和活性维生素D之外，拥有一种能够治疗疾病根本原因的选择，是向前迈出的一大步。”

Announcing the partnership, ST Chief Executive Officer Carlo Montagner said this agreement was an important company milestone, signalling ST's expansion into both endocrinology and paediatric medicine.

ST首席执行官卡罗·蒙塔格纳（CarloMontagner）在宣布合作关系时表示，这项协议是公司的一个重要里程碑，标志着ST将扩展到内分泌学和儿科医学。

Mr Montagner commented: 'We are delighted to have been selected as Ascendis Pharma's exclusive partner for commercialising their portfolio in Oceania and these South-East Asia countries and look forward to launching these critical endocrinology products in our regions as soon as possible.

蒙塔格纳先生评论道：“我们很高兴被选为Ascendis Pharma的独家合作伙伴，将其在大洋洲和东南亚国家的投资组合商业化，并期待着尽快在我们的地区推出这些关键的内分泌产品。

'All three products are valuable inclusions to our broad therapeutic pipeline and our international business, as we continue to leverage our substantial experience commercialising specialist medicines across multiple regions.

“这三种产品都是我们广泛的治疗渠道和国际业务的宝贵组成部分，因为我们继续利用我们在多个地区将专业药物商业化的丰富经验。

'We look forward to working with endocrinologists across our territories to make these endocrine therapies available to all eligible patients who may benefit.'

“我们期待着与我们各地的内分泌学家合作，将这些内分泌疗法提供给所有可能受益的符合条件的患者。”

Ascendis Pharma Executive Vice President and Chief Commercial Officer Camilla Harder Hartvig said ST had been selected to launch the endocrinology portfolio in these countries based on its strong track record commercialising specialist products in multiple regions.

Ascendis Pharma执行副总裁兼首席商务官卡米拉·哈德·哈特维格（CamillaHardwig）表示，ST凭借其在多个地区将专业产品商业化的良好业绩，被选中在这些国家推出内分泌产品组合。

'We are delighted to partner with Specialised Therapeutics to broaden the reach of our endocrinology rare disease portfolio, contributing to our shared goal of making a meaningful difference for patients facing unmet medical needs,' she said.

她说：“我们很高兴与专业治疗学合作，扩大我们的内分泌罕见病组合的覆盖面，为我们的共同目标做出贡献，为面临未满足医疗需求的患者做出有意义的改变。”。

About Specialised Therapeutics

关于专业治疗学

Founded in 2007, Specialised Therapeutics is the region's largest independent specialty pharmaceutical company, providing new therapies and technologies to patients in Australia, New Zealand and across Southeast Asia. Headquartered in Singapore, ST partners with global pharmaceutical, biotech and diagnostic companies to bring novel healthcare opportunities to patients who are impacted by a range of diseases.

Specialized Therapeutics成立于2007年，是该地区最大的独立专业制药公司，为澳大利亚、新西兰和整个东南亚的患者提供新的疗法和技术。ST总部位于新加坡，与全球制药、生物技术和诊断公司合作，为受一系列疾病影响的患者带来新的医疗保健机会。

ST has built a strong track record of success, navigating complex regulatory, reimbursement and commercialisation environments in its diverse regions. The ST mission is to provide specialty therapies where there is an unmet need. The company's broad therapeutic portfolio currently includes novel agents in oncology, haematology, CNS, neurology, endocrinology, ophthalmology and supportive care, although it is not confined to these areas.

ST在其不同地区的复杂监管、报销和商业化环境中取得了巨大的成功。ST的使命是在需求未得到满足的地方提供专业治疗。该公司广泛的治疗组合目前包括肿瘤学，血液学，中枢神经系统，神经病学，内分泌学，眼科和支持治疗方面的新药，尽管它并不局限于这些领域。

ST is a member of the World Orphan Drug Alliance (WODA)..

ST是世界孤儿药联盟（WODA）的成员。。

Additional information: www.stbiopharma.com

其他信息：www.stbiopharma.com

About Ascendis Pharma

关于Ascendis Pharma

Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients' lives. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new and potentially best-in-class therapies.

Ascendis Pharma正在应用其创新的TransCon技术平台，建立一家领先的、全面整合的生物制药公司，专注于为患者的生活带来有意义的改变。在患者、科学和激情的核心价值观的指导下，该公司利用其TransCon技术创造了新的和潜在的一流疗法。

Please visit https://ascendispharma.com to learn more..

请访问https://ascendispharma.com了解更多。。

About SKYTROFA (lonapegsomatropin)

SKYTROFA™（单克隆生长激素）

SKYTROFA™ (lonapegsomatropin, developed as TransCon™ hGH) is a prodrug of somatropin, designed to provide sustained release of unmodified somatropin. The unmodified, unbound somatropin released from lonapegsomatropin has the same 191 amino acid sequence and size as endogenous GH.5 TransCon hGH is approved and marketed as SKYTROFA (lonapegsomatropin-tcgd) in the United States1 and as SKYTROFA (lonapegsomatropin) in the European Union2 as a once-weekly treatment for children and adolescents with GHD..

SKYTROFA公司™（lonapegsomatropin，开发为TransCon™ hGH）是生长激素的前药，旨在提供未修饰的生长激素的持续释放。从lonapegsomatropin释放的未修饰的未结合生长激素具有与内源性GH相同的191个氨基酸序列和大小。TransCon hGH在美国被批准并作为SKYTROFA（lonapegsomatropin tcgd）销售1，在欧盟被批准为SKYTROFA（lonapegsomatropin）销售2，作为GHD儿童和青少年的每周一次治疗。。

SKYTROFA single-use, prefilled cartridges are manufactured in nine dosage strengths, allowing for convenient dosing flexibility. They are designed for use only with the SKYTROFA Auto-Injector and may be stored at room temperature for up to six months. The recommended dose of SKYTROFA for treatment-naïve patients and patients switching from daily somatropin is 0.24 mg/kg body weight, administered once weekly.1,2 The dose may be adjusted based on the child's weight and insulin-like growth factor-1 standard deviation score (IGF-1 SDS).2.

SKYTROFA一次性预填充药筒具有九种剂量强度，可以方便地灵活给药。它们仅设计用于SKYTROFA自动注射器，可在室温下保存长达六个月。对于未接受治疗的患者和改用每日生长激素的患者，SKYTROFA的推荐剂量为0.24 mg/kg体重，每周一次。1,2剂量可根据儿童体重和胰岛素样生长因子-1标准差评分（IGF-1 SDS）进行调整。

TransCon hGH was studied in over 300 children with GHD across the Phase 3 program, which consisted of the Height Trial5 (for treatment-naïve patients), the Flight Trial6 (for treatment-experienced patients), and the Enlighten Trial7 (a long-term extension trial). Patients who completed the Height or Flight Trials were able to continue in Enlighten, with some on lonapegsomatropin treatment for over four years..

TransCon hGH在第三阶段计划中对300多名GHD儿童进行了研究，其中包括HEIGH试验5（对于未接受治疗的患者），Flight试验6（对于有治疗经验的患者）和INDIGINT试验7（长期延长试验）。完成身高或飞行试验的患者能够继续接受启蒙治疗，其中一些患者接受了超过四年的lonapegsomatropin治疗。。

Ascendis Pharma is also conducting the ongoing global Phase 3 Foresight Trial of TransCon hGH in adults with GHD.8

Ascendis Pharma还正在对患有GHD的成年人进行TransCon hGH的全球3期前瞻性试验。8

About Paediatric Growth Hormone Deficiency

关于小儿生长激素缺乏症

Paediatric GHD is a serious orphan disease caused when the pituitary gland does not produce enough GH. Children with GHD are not only characterised by short stature; they also may experience metabolic abnormalities, psychosocial challenges, and an overall poor quality of life. For decades, the standard of care for GHD has been a daily subcutaneous injection of hGH to improve growth and overall endocrine health..

小儿GHD是一种严重的孤儿疾病，由垂体产生的GH不足引起。患有GHD的儿童不仅身材矮小；他们还可能经历代谢异常，心理社会挑战和总体生活质量差。几十年来，GHD的护理标准是每天皮下注射hGH，以改善生长和整体内分泌健康。。

About YORVIPATH (palopegteriparatide)

关于YORVIPATH（palopegteriparatide）

YORVIPATH™ (palopegteriparatide, developed as TransCon™ PTH) is a once-daily prodrug with sustained release of active PTH approved by the European Union as a PTH replacement therapy for the treatment of adults with chronic hypoparathyroidism. Treatment should be initiated and monitored by physicians or qualified healthcare professionals experienced in the diagnosis and management of patients with hypoparathyroidism.3.

约维派教徒™（palopegteriparatide，开发为TransCon™ PTH）是一种每日一次的前药，可持续释放活性PTH，经欧盟批准作为PTH替代疗法，用于治疗成人慢性甲状旁腺功能减退症。治疗应由在甲状旁腺功能减退症患者的诊断和管理方面经验丰富的医生或合格的医疗保健专业人员发起和监测。

TransCon PTH met all primary and key secondary endpoints in the Phase 3 Pathway Trial, demonstrating a response rate of 78.7% compared to 4.8% for control (p-value <0.0001) for the primary composite endpoint, and statistically significant improvements compared to control on all key secondary endpoints, which included measures evaluating patient-reported disease symptoms and impacts.9.

TransCon PTH在3期Pathway试验中达到了所有主要和关键次要终点，显示有效率为78.7%，而主要复合终点的对照组为4.8%（p值<0.0001），并且与所有关键次要终点的对照组相比，统计学显着改善，其中包括评估患者报告的疾病症状和影响的措施。

About Hypoparathyroidism

关于甲状旁腺功能减退症

Hypoparathyroidism is an endocrine disease caused by insufficient levels of PTH, the primary regulator of calcium/phosphate balance in the body, acting directly on bone and kidneys and indirectly on intestines. Hypoparathyroidism is considered chronic if it persists >6 months following surgery. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment.9.

甲状旁腺功能减退症是一种内分泌疾病，由PTH水平不足引起，PTH是体内钙/磷平衡的主要调节剂，直接作用于骨骼和肾脏，间接作用于肠道。如果甲状旁腺功能减退症在手术后持续>6个月，则被认为是慢性的。甲状旁腺功能减退症患者可能会出现一系列严重且可能危及生命的短期和长期并发症，包括神经肌肉过敏，肾脏并发症，骨骼外钙化和认知障碍。

About TransCon CNP (navepegritide)

关于TransCon CNP（naveperigritide）

TransCon™ CNP (navepegritide) is an investigational long-acting prodrug of C-type natriuretic peptide (CNP), designed to provide continuous exposure of CNP at safe, therapeutic levels, via a single, weekly subcutaneous dose, for the treatment of children with ACH.4

TransCon公司™ CNP（navepegritide）是一种C型利钠肽（CNP）的研究性长效前药，旨在通过单次，每周皮下剂量，以安全，治疗水平持续暴露CNP，用于治疗ACH患儿

The Phase 2 Accomplish Trial, a randomised, double-blind, placebo-controlled, dose-escalation trial evaluating the safety and efficacy of once-weekly TransCon CNP compared to placebo in prepubertal children with ACH aged 2 to 10 years old, met its primary objectives, and demonstrated that TransCon CNP at 100 µg/kg/week was superior to placebo for the primary efficacy endpoint of annualised growth velocity (AGV) at 52 weeks4..

第二阶段COMPLITE试验是一项随机，双盲，安慰剂对照，剂量递增试验，评估了每周一次TransCon CNP与安慰剂相比在2至10岁ACH青春期前儿童中的安全性和有效性，达到了其主要目标，并证明100µg/kg/周的TransCon CNP在52周时的年化生长速度（AGV）的主要疗效终点优于安慰剂4。。

All 57 randomised children completed the blinded portion of Accomplish and are currently continuing in the open label extension at the 100 μg/kg/week dose4.  Ascendis Pharma recently confirmed that these 57 clinical trial patients have all completed one year of treatment with TransCon CNP at 100 µg/kg/week, and announced that TransCon CNP is the first investigational product to demonstrate improvements in health-related quality of life and disease impacts in children with ACH.10.

所有57名随机儿童完成了COMPLETE的盲法部分，目前正在以100微克/千克/周的剂量继续开放标签扩展4。Ascendis Pharma最近证实，这57名临床试验患者均以100µg/kg/周的剂量完成了一年的TransCon CNP治疗，并宣布TransCon CNP是第一个证明与健康相关的生活质量改善的研究产品。ACH.10儿童的疾病影响。

About Achondroplasia

关于软骨发育不全

Achondroplasia is the most common genetic form of skeletal dysplasia and resulting disproportionate short stature, caused by a genetic mutation in the fibroblast growth factor receptor 3 (FGFR3). This leads to an imbalance between the stimulatory and inhibitory signaling pathways involved in regulating bone growth.

软骨发育不全是骨骼发育异常的最常见遗传形式，并导致不成比例的身材矮小，这是由成纤维细胞生长因子受体3（FGFR3）的基因突变引起的。这导致参与调节骨生长的刺激性和抑制性信号传导途径之间的不平衡。

People living with ACH may experience serious complications and comorbidities due to inhibited skeletal development. Complications may include sleep apnoea and respiratory problems, chronic back and leg pain from lower spine impingement, and sudden infant death from compression of the brain stem. Chronic ear infections due to eustachian tube problems can lead to hearing loss and speech delay.

由于骨骼发育受到抑制，患有ACH的人可能会出现严重的并发症和合并症。并发症可能包括睡眠呼吸暂停和呼吸系统问题，下脊柱撞击引起的慢性背部和腿部疼痛，以及脑干受压导致的婴儿猝死。咽鼓管问题引起的慢性耳部感染可导致听力下降和言语延迟。

Children with ACH may also experience social and emotional challenges..

患有ACH的儿童也可能经历社交和情绪挑战。。

References

参考文献

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SKYTROFA（lonapegsomatropin tcgd）美国处方信息。

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SKYTROFA（lonapegsomatropin）EU产品特性总结。

YORVIPATH (palopegteriparatide) EU Summary of Product Characteristics.

YORVIPATH（palopegteriparatide）EU产品特性总结。

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Khan AA, Rubin MR, Schwarz P, et al. J Bone Miner Res. 2023;38(1):14–25.

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Ascendis Pharma A/S, Significant Health and Quality of Life Improvements Achieved in Children with Achondroplasia Treated for One Year with TransCon™ CNP (Navepegritide) at 100 µg/kg/week, Press Release, 20 December 2023, https://investors.ascendispharma.com/news-releases/news-release-details/significant-health-and-quality-life-improvements-achieved..

Ascendis Pharma A/S，使用TransCon治疗一年的软骨发育不全儿童的健康和生活质量得到了显着改善™ CNP（Navepegritide），每周100微克/千克，新闻稿，2023年12月20日，https://investors.ascendispharma.com/news-releases/news-release-details/significant-health-and-quality-life-improvements-achieved..

SOURCE Specialised Therapeutics

来源专业治疗