Drug Development · global
Rare Disease Drug Changes Hands for a New Start: Synaptiq Takes Over 22q11.2 Deletion Syndrome Candidate SYN-001
The new company will advance an mGluR modulator that has completed a small Phase 2 trial into a multinational Phase 2b study; the asset and clinical capabilities have been reorganized, but efficacy still needs to be demonstrated in a larger, more clearly designed study.
22q11.2 deletion syndrome can affect physical development, cognition, and mental health at the same time, yet patients have no approved drug specifically targeting its neuropsychiatric symptoms. Newly established Synaptiq Therapeutics has now taken over a clinical-stage drug candidate, aiming to advance the signals left by early human research into a more robust multinational trial.
Synaptiq was jointly formed by European and US investors and has acquired Nobias Therapeutics’ lead asset, NB-001, renaming it SYN-001. The transaction value and other financial terms were not disclosed; Nobias retained an equity stake in the new company. Founding investors include Sanos Group, an Icelandic fund managed by AxUM Securities, and Arctic Therapeutics.
SYN-001 is the oral small-molecule drug fasoracetam monohydrate, whose mechanism of action is described as modulating metabotropic glutamate receptors rather than acting as a central nervous system stimulant. Synaptiq plans to use it to treat anxiety, attention-deficit/hyperactivity disorder, and autism spectrum-related manifestations associated with 22q11.2 deletion syndrome; these symptoms often overlap and can also affect learning and social functioning.
ClinicalTrials.gov data show that the Phase 2 study previously completed by Nobias enrolled 37 children and adolescents aged 6 to 17. It used a randomized, placebo-controlled, crossover design with quadruple masking and a daily oral dose of 400 mg. Safety and tolerability were the primary outcomes, while the Clinical Global Impression–Improvement scale was listed as a secondary outcome; the trial was completed in June 2023, and results were first posted in February 2025.
The company said the existing study showed favorable safety and tolerability, with efficacy signals also observed in some clinical subgroups. However, the sample size of 37 was limited, and subgroup findings are particularly susceptible to chance differences; currently available public information is also insufficient to determine the magnitude and duration of the effect or whether different symptom groups benefited consistently. These signals are therefore more appropriately viewed as a basis for the next-stage trial rather than as established efficacy.
Synaptiq will next advance a Phase 2b trial in North America and Europe and introduce a Clinical Global Impression scale designed for 22q11.2 deletion syndrome. Sanos Group will provide clinical development and contract research capabilities; Patrick Dougherty has been appointed CEO, and Arctic Therapeutics CEO Ivar Hakonarson will serve as chairman. SYN-001 received US FDA Orphan Drug and Rare Pediatric Disease designations in 2024, but these designations do not signify recognition of efficacy; whether the new trial can confirm reproducible, clinically meaningful improvement is the real question this asset restructuring is intended to answer.