Synaptiq Therapeutics Launches with Acquisition of SYN-001, a Phase II-Ready Small Molecule for 22q11.2 Deletion Syndrome
核心洞察
Synaptiq Therapeutics (搜索) has launched as an Iceland-based clinical-stage biotech following the acquisition of SYN-001 (formerly NB-001) from Nobias Therapeutics (搜索) for neuropsychiatric symptoms associated with 22q11.2 Deletion Syndrome (搜索).
SYN-001, a small molecule modulator of metabotropic glutamate receptors (搜索), demonstrated favorable safety and statistically significant efficacy signals in a Phase II trial, with a Phase IIb study now planned across North America and Europe.
The FDA granted SYN-001 both Orphan Drug Designation and Rare Pediatric Disease Designation in 2024, and preliminary alignment on registrational endpoints was reported in 2025.
A consortium of European and US-based investors, together with Nobias Therapeutics (搜索), has announced the formation of Synaptiq Therapeutics (搜索), a privately held clinical-stage biotechnology company headquartered in Iceland. The new entity launches with the acquisition of Nobias Therapeutics' lead asset, NB-001—now renamed SYN-001—and a mandate to advance the compound through late-stage clinical development for neuropsychiatric symptoms associated with 22q11.2 Deletion Syndrome (搜索) (22q11DS). Patrick Dougherty has been appointed Chief Executive Officer of the newly formed company.
The founding investor group includes Investcorp-backed Sanos Group (搜索), a Denmark-headquartered specialized clinical research organization; an Icelandic investment fund managed by AxUM Securities; and Arctic Therapeutics (搜索), a clinical-stage biotechnology company with operations in Iceland and the United States. Nobias Therapeutics (搜索), which is majority-backed by Medical Excellence Capital, will retain an equity interest in Synaptiq Therapeutics (搜索) alongside the founding investors. Financial terms of the transaction were not disclosed.
"The creation of Synaptiq Therapeutics (搜索) marks an important milestone in the advancement of SYN-001," said Patrick Dougherty, Chief Executive Officer of Synaptiq Therapeutics. "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."
SYN-001: Mechanism and Clinical Data
SYN-001 is a novel small molecule modulator of metabotropic glutamate receptors (搜索) (mGluRs). Nobias Therapeutics (搜索) previously 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.
The upcoming Phase IIb study will incorporate a 22q11DS-specific clinical global impression (CGI) scale developed from insights gained during the completed Phase II trial. Synaptiq plans to conduct the study at leading medical centers across North America and Europe.
Regulatory Milestones
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 (搜索) announced preliminary alignment with the FDA on potential registrational endpoints to support future development. These designations and regulatory interactions position SYN-001 on a potentially expedited development pathway.
Disease Background and Unmet Need
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 globally. The condition is believed to be substantially underdiagnosed in certain patient subsets and in certain regions. Based on recent fertility statistics in Europe and the United States, this translates to approximately 1,000 to 2,000 children born with 22q11DS each year in each region. An estimated 65,000 people are living with the condition in the United States.
Individuals 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. There are currently no approved therapies specifically indicated for the neuropsychiatric manifestations of 22q11DS.
Strategic Platform and Future Direction
Headquartered in Iceland, Synaptiq Therapeutics (搜索) will leverage Arctic Therapeutics (搜索)' 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. Sanos Group (搜索) will contribute its clinical development and contract research capabilities, drawing on its network of approximately 200 employees across Denmark, the United States, Europe, and Hong Kong.
Ivar Hakonarson, Chief Executive Officer of Arctic Therapeutics (搜索) and Chairman of Synaptiq Therapeutics (搜索), stated: "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."
The company has indicated its intention to expand SYN-001 into additional neurodevelopmental and neuropsychiatric indications beyond the initial 22q11DS program, targeting multiple conditions with significant unmet medical needs.
"We are continually inspired by the resilience of individuals living with 22q11DS and by the dedication of their families, caregivers, and healthcare providers," Dougherty said. "Their experiences motivate our commitment to advancing innovative treatment options for this underserved patient community."