Synaptiq Therapeutics通过收购临床阶段 SYN-001项目正式启动-动脉网

Synaptiq Therapeutics Launches with Acquisition of Clinical-Stage SYN-001 Program

PR Newswire 等信源发布 2026-07-23 20:51

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Company formed to advance SYN-001, an FDA-designated orphan and rare pediatric disease therapeutic candidate acquired from Nobias Therapeutics

公司成立旨在推进 SYN-001，这是一种从 Nobias Therapeutics 收购的、被美国食品药品监督管理局（FDA）指定为孤儿药及罕见儿科疾病治疗候选药物。

Phase IIb trial planned to further evaluate SYN-001 for the treatment of neuropsychiatric symptoms associated with 22q11.2 Deletion Syndrome (22q11DS)

计划开展IIb期临床试验，以进一步评估SYN-001用于治疗与22q11.2缺失综合征（22q11DS）相关的神经精神症状

AKUREYRI, Iceland and PHILADELPHIA

冰岛阿库雷里和费城

，

JULY 23, 2026

2026年7月23日

/PRNewswire/ -- A consortium of European and US-based investors and Nobias Therapeutics today announced the launch of Synaptiq Therapeutics, a privately held, clinical-stage biotechnology company dedicated to advancing SYN-001 for the treatment of neuropsychiatric symptoms associated with 22q11DS. With the acquisition of Nobias Therapeutics' lead asset, NB-001 (now SYN-001), Synaptiq brings together experienced investors, proven clinical development capabilities and operational expertise to advance SYN-001 through late-stage clinical development and toward potential regulatory approval.

/美通社/ -- 一个由欧洲和美国的投资者组成的财团与Nobias Therapeutics今天宣布成立Synaptiq Therapeutics，这是一家私人控股的临床阶段生物技术公司，致力于推进SYN-001用于治疗与22q11DS相关的神经精神症状。通过收购Nobias Therapeutics的核心资产NB-001（现更名为SYN-001），Synaptiq汇聚了经验丰富的投资者、经过验证的临床开发能力以及运营专长，以推动SYN-001进入后期临床开发阶段并朝着潜在的监管批准迈进。

Patrick Dougherty has been appointed Chief Executive Officer..

帕特里克·多尔蒂已被任命为首席执行官。

Founding investors include Investcorp-backed Sanos Group, a specialized, clinical research organization (CRO) headquartered in Denmark, an Icelandic investment fund by AxUM Securities, and Arctic Therapeutics, a clinical-stage biotechnology company with operations in Iceland and the US. Nobias Therapeutics, which is majority-backed by Medical Excellence Capital, will retain an equity interest in Synaptiq Therapeutics alongside the founding investors.

创始投资者包括由Investcorp支持的Sanos Group（一家总部位于丹麦的专业化临床研究组织[CRO]）、AxUM Securities旗下的冰岛投资基金，以及在冰岛和美国开展业务的临床阶段生物技术公司Arctic Therapeutics。由Medical Excellence Capital控股的Nobias Therapeutics将与创始投资者一同保留Synaptiq Therapeutics的股权权益。

The companies did not disclose the financial terms of the transaction..

这些公司未披露该交易的财务条款。

'The creation of Synaptiq Therapeutics marks an important milestone in the advancement of SYN-001,'

“Synaptiq Therapeutics 的成立标志着 SYN-001 研发进程中的一个重要里程碑，”

said Patrick Dougherty, Chief Executive Officer of Synaptiq Therapeutics

Synaptiq Therapeutics 首席执行官 Patrick Dougherty 表示

. 'The program has already generated impressive Phase II data. By bringing together dedicated investors, experienced development partners and a focused operating company, we are well positioned to advance SYN-001 through Phase IIb and move closer to delivering the first approved therapy specifically indicated for individuals living with 22q11DS.'.

“该计划已经产生了令人印象深刻的II期数据。通过汇聚专注的投资者、经验丰富的开发合作伙伴以及精干的运营公司，我们处于有利地位，能够推动SYN-001进入IIb期临床试验，并朝着为22q11缺失综合征（22q11DS）患者提供首款获批的特定适应症疗法的目标更进一步。”

Headquartered in Iceland, Synaptiq Therapeutics will leverage Arctic Therapeutics' established drug development platform, experienced clinical operations team, scientific expertise and deep research relationship with the US-based Center for Applied Genomics at the Children's Hospital of Philadelphia.

Synaptiq Therapeutics 总部位于冰岛，将利用 Arctic Therapeutics 成熟的药物开发平台、经验丰富的临床运营团队、科学专业知识，以及与美国费城儿童医院应用基因组学中心的深厚研究合作关系。

Sanos Group will contribute its clinical development and contract research capabilities as the company prepares to initiate a Phase IIb clinical trial at leading medical centers across North America and Europe. SYN-001 has the potential to become the first therapy specifically developed for the treatment of neuropsychiatric symptoms associated with 22q11DS..

Sanos Group将贡献其临床开发和合同研究能力，为公司准备在北美和欧洲的领先医疗中心启动IIb期临床试验。SYN-001有望成为首个专门开发用于治疗与22q11DS相关的神经精神症状的疗法。

'This transaction creates an entity dedicated to realizing the potential of SYN-001,'

“此次交易创建了一个专门致力于实现SYN-001潜力的实体，”

said Ivar Hakonarson, Chief Executive Officer of Arctic Therapeutics and Chairman of Synaptiq Therapeutics.

北极疗法公司首席执行官兼Synaptiq疗法公司董事长伊瓦尔·哈卡松表示。

'By combining Arctic Therapeutics' development capabilities with an experienced investor group, an outstanding management team and strategic partners, we have created the right platform to accelerate development of this important program. We are delighted to welcome Patrick Dougherty as Chief Executive Officer and look forward to working together to bring a much-needed treatment option to patients and families affected by 22q11DS.'.

“通过将 Arctic Therapeutics 的开发能力与经验丰富的投资者群体、卓越的管理团队及战略合作伙伴相结合，我们打造了加速推进这一重要项目开发的理想平台。我们很高兴欢迎 Patrick Dougherty 担任首席执行官，并期待携手合作，为受 22q11DS 影响的患者和家庭带来亟需的治疗选择。”

22q11DS, also known as DiGeorge syndrome, is one of the most common rare genetic disorders, affecting approximately one in every 2,000 to 3,300 live births globally¹˒⁵. The condition is believed to be substantially underdiagnosed in certain patient subsets and in certain regions. Based on recent fertility statistics in Europe² and the United States³, this translates to approximately 1,000 to 2,000 children born with 22q11DS each year in each region.

22q11缺失综合征（22q11DS），又称迪乔治综合征，是最常见的罕见遗传性疾病之一，全球每2,000至3,300名活产婴儿中约有1人受累¹˒⁵。据信，在某些患者亚群和某些地区，该病的诊断率严重不足。根据欧洲²和美国³最近的生育统计数据，这意味着每个地区每年约有1,000至2,000名儿童患有22q11DS出生。

It is estimated that around 65,000 people are living with the condition in the United States⁴˒⁵..

据估计，美国约有65,000人患有该疾病⁴˒⁵。

SYN-001 is a novel small molecule modulator of metabotropic glutamate receptors (mGluRs). Nobias Therapeutics evaluated the compound in a multi-center, randomized, double-blind, placebo-controlled Phase II clinical trial which demonstrated a favorable safety and tolerability profile. The study also generated positive efficacy signals, including statistically significant improvements in clinically relevant patient subgroups.

SYN-001 是一种新型代谢型谷氨酸受体（mGluRs）小分子调节剂。Nobias Therapeutics 在一项多中心、随机、双盲、安慰剂对照的 II 期临床试验中对该化合物进行了评估，试验结果显示其具有良好的安全性和耐受性。该研究还产生了积极的疗效信号，包括在临床相关的患者亚组中观察到统计学上的显著改善。

The upcoming Phase IIb study will incorporate a 22q11DS-specific clinical global impression (CGI) scale that is based on insights from this completed trial..

即将开展的IIb期研究将纳入一种基于本项已完成试验见解的、针对22q11缺失综合征（22q11DS）的特异性临床总体印象（CGI）量表。

In 2024, SYN-001 received both Orphan Drug Designation and Rare Pediatric Disease Designation from the US Food and Drug Administration (FDA). In 2025, Nobias Therapeutics announced preliminary alignment with the FDA on potential registrational endpoints to support future development.

2024年，SYN-001获得了美国食品药品监督管理局（FDA）授予的孤儿药认定和罕见儿科疾病认定。2025年，Nobias Therapeutics宣布与FDA就支持未来开发的潜在注册终点达成初步一致。

Individuals living with 22q11DS frequently experience a combination of neuropsychiatric symptoms, including anxiety, attention-deficit/hyperactivity disorder (ADHD), and autism spectrum disorder-related manifestations. These symptoms can have a profound impact on quality of life, educational achievement, and social functioning, while placing a significant burden on families and caregivers.

患有22q11缺失综合征（22q11DS）的个体常常经历一系列神经精神症状，包括焦虑、注意缺陷多动障碍（ADHD）以及自闭症谱系障碍相关表现。这些症状可能对生活质量、教育成就和社会功能产生深远影响，同时给家庭和照护者带来沉重负担。

There are currently no approved therapies specifically indicated for the neuropsychiatric manifestations of 22q11DS..

目前尚无专门针对22q11缺失综合征（22q11DS）神经精神表现的获批疗法。

'We are continually inspired by the resilience of individuals living with 22q11DS and by the dedication of their families, caregivers, and healthcare providers

“我们不断受到22q11DS患者坚韧精神的鼓舞，也受到他们的家人、护理人员和医疗保健提供者奉献精神的激励。”

,' Dougherty said.

“，”多尔蒂说。

'Their experiences motivate our commitment to advancing innovative treatment options for this underserved patient community,'

“他们的经历激励我们致力于推进针对这一患者群体的创新治疗方案。”

he added

他补充道

。

About Synaptiq Therapeutics

关于Synaptiq Therapeutics

Synaptiq Therapeutics is a privately held clinical-stage biotechnology company dedicated to advancing the clinical development of SYN-001 for multiple neurodevelopmental and neuropsychiatric indications with significant unmet medical needs. SYN-001 is a novel small molecule modulator of metabotropic glutamate receptors (mGluRs) initially being developed for the treatment of neuropsychiatric symptoms associated with 22q11.2 deletion syndrome (22q11DS).

Synaptiq Therapeutics 是一家私营的临床阶段生物技术公司，致力于推进 SYN-001 在多种存在重大未满足医疗需求的神经发育和神经精神适应症中的临床开发。SYN-001 是一种新型代谢型谷氨酸受体（mGluRs）小分子调节剂，最初开发用于治疗与 22q11.2 缺失综合征（22q11DS）相关的神经精神症状。

The company is preparing to initiate a Phase IIb clinical trial across leading centers in North America and Europe. For more information, please visit .

该公司正准备在北美和欧洲的领先中心启动IIb期临床试验。欲了解更多信息，请访问。

www.synaptiqtx.com

。

About Arctic Therapeutics

关于北极疗法公司

ATx is a clinical-stage biopharmaceutical company, established in 2015 as a spin-off from the US-based Center for Applied Genomics (CAG), a research center at the Children's Hospital of Philadelphia. The company's diversified pipeline reflects a deep commitment to address diseases with a high unmet need, by translating genetic discoveries into safe and effective treatments.

ATx 是一家处于临床阶段的生物制药公司，于2015年从美国应用基因组学中心（CAG）分拆成立，该中心是费城儿童医院旗下的一家研究机构。公司拥有多元化的产品管线，致力于将遗传学发现转化为安全有效的治疗方法，以应对存在巨大未满足医疗需求的疾病。

The company has operations in Iceland, the US and multiple collaborations across Europe. For more information, please visit .

该公司在冰岛和美国设有业务，并在欧洲开展多项合作。欲了解更多信息，请访问。

www.arctictherapeutics.com

and follow us on

并在以下平台关注我们

领英

。

About Sanos Group

关于Sanos集团

Sanos Group is a leading specialist provider of clinical study services in the global pharma and biotech industry. The group consists of around 200 employees, mainly in Denmark and the United States, but also in Europe and Hong Kong, and is headquartered in Søborg outside Copenhagen. The group includes Sanos Clinic, which consists of four private research clinics in Denmark, including a Phase 1 unit, the two global full-service CROs NBCD and Blueskin, and Omicron specializing in biostatistics.

Sanos Group 是全球制药和生物科技行业中领先的临床研究服务专业提供商。该集团拥有约200名员工，主要分布在丹麦和美国，同时在欧洲和香港也有布局，总部设在哥本哈根郊区的索博格（Søborg）。集团旗下包括 Sanos Clinic，其在丹麦设有四家私人研究诊所，其中包括一个I期临床试验单元；两家提供全球全方位服务的合同研究组织（CRO）NBCD 和 Blueskin；以及专注于生物统计学的 Omicron。

Studies&Me is a unique digital recruitment platform and tech support for clinical trials. Sanos Supply offers contract manufacturing as well as packaging, labeling, storage and distribution of medicines for clinical studies. Please find more information.

Studies&Me 是一个独特的数字化招聘平台，并为临床试验提供技术支持。Sanos Supply 提供合同制造服务，以及面向临床研究的药品包装、标签、储存和分发服务。请查阅更多信息。

在

www.sanos.com

。

About AxUM Securities

关于 AxUM 证券

AxUM Securities Ltd. (i.e AxUM Verðbréf hf.) is an Icelandic licensed alternative investment fund (AIF) management company, located in Akureyri, Iceland. AxUM Verðbréf hf. manages funds for professional private investors.

AxUM Securities Ltd.（即 AxUM Verðbréf hf.）是一家位于冰岛阿克雷里的持牌另类投资基金（AIF）管理公司。AxUM Verðbréf hf. 为专业私人投资者管理基金。

For more information, visit:

更多信息，请访问：

https://www.axum.is/um-okkur

https://www.axum.is/关于我们

。

References:

参考文献：

22q11 Europe. General Information. Available at:

22q11 欧洲。一般信息。获取网址：

https://22q11europe.org/portfolio/general-information/

Eurostat. Fertility Statistics. Available at: