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Synaptiq Therapeutics launches with acquisition of small-molecule rare disease therapy

Synaptiq Therapeutics, an Iceland-based clinical-stage biotechnology company, launched through the acquisition of SYN-001, a small molecule already in...

Synaptiq Therapeutics launches with acquisition of small-molecule rare disease therapy

The Iceland-based Synaptiq Therapeutics has launched to advance SYN-001 (previously NB-001), a Phase II-ready small molecule for neuropsychiatric symptoms associated with 22q11.2 Deletion Syndrome, following its acquisition from Pennsylvania-based Nobias Therapeutics. Nobias retains an equity stake in Synaptiq alongside a consortium of founding investors. Financial terms were not disclosed.

Founding investors include Investcorp-backed Sanos Group, AxUM Securities, and Arctic Therapeutics. Nobias, majority-backed by Medical Excellence Capital, is not named as a founding investor but holds continuing equity in Synaptiq. Patrick Dougherty has been named Chief Executive Officer; no prior affiliations were disclosed for him. Arctic Therapeutics CEO Ivar Hakonarson serves as Synaptiq's chairman, linking the new company to Arctic's genomics research ties and drug development infrastructure.

SYN-001 is a small molecule modulator of metabotropic glutamate receptors. In a randomized, double-blind, placebo-controlled Phase II trial run by Nobias, the compound showed a favorable safety and tolerability profile along with statistically significant efficacy improvements in certain patient subgroups. Synaptiq plans to initiate a Phase IIb trial at centers across North America and Europe, incorporating a 22q11DS-specific clinical global impression scale developed from the earlier study's findings. SYN-001 holds Orphan Drug and Rare Pediatric Disease designations from the FDA, granted in 2024, and Nobias reported preliminary alignment with the agency on potential registrational endpoints in 2025.

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22q11.2 Deletion Syndrome affects an estimated one in every 2,000 to 3,300 live births globally, with roughly 65,000 people living with the condition in the United States. Patients frequently experience anxiety, ADHD and autism spectrum-related symptoms, but no therapy is currently approved specifically for these neuropsychiatric manifestations. Sanos Group will supply clinical trial operations and contract research support through its Phase I clinic network and biostatistics unit, while Arctic Therapeutics contributes its genomics-linked development platform. The company said it intends to expand SYN-001 into additional neurodevelopmental and neuropsychiatric indications beyond its initial 22q11DS program.


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