Iceland-based Synaptiq launches with Nobias’ rare disease drug

The Iceland-headquartered company has renamed Nobias’ NB-001 programme SYN-001 and plans to test it in a Phase 2b trial for neuropsychiatric symptoms associated with 22q11.2 deletion syndrome, also known as DiGeorge syndrome.

Why it matters: Synaptiq is giving the drug, called fasoracetam, yet another corporate home after repeated attempts to repurpose a molecule originally developed – and abandoned – for dementia.

• The common thread is Hakon Hakonarson, CEO of Arctic Therapeutics and director of the Center for Applied Genomics at the Children’s Hospital of Philadelphia. His research helped establish the genetic rationale for repurposing fasoracetam, and he will now chair the company responsible for its latest clinical attempt.

Zoom in: Synaptiq was formed by a group of European and U.S. investors that includes Arctic Therapeutics, Danish clinical research organisation Sanos Group and an Icelandic investment fund managed by AxUM Securities.

• Patrick Dougherty, who became Nobias’ CEO in 2024, will lead the new company. Nobias will retain an equity stake, but its website now says substantially all its assets have been acquired by Synaptiq. Financial terms and the size of Synaptiq’s financing were not disclosed.

How it works: Fasoracetam is a small molecule that modulates metabotropic glutamate receptors, which help regulate signalling between nerve cells.

• Synaptiq is targeting people with 22q11.2 deletion syndrome, a genetic condition associated with developmental abnormalities and a high incidence of anxiety, ADHD, autism-related symptoms and other psychiatric disorders.
• There is currently no drug approved specifically for the neuropsychiatric symptoms associated with the condition.

A long journey: Fasoracetam was originally developed by Japan’s Nippon Shinyaku as NS-105 and reached Phase 3 testing for vascular dementia in the 1990s. Development was discontinued after the drug failed to show sufficient efficacy.

• The programme returned years later through Hakonarson’s work on genetic alterations affecting metabotropic glutamate receptor, or mGluR, signalling in neuropsychiatric disorders. Hakonarson founded neuroFix Therapeutics to develop fasoracetam in genetically selected patients. Medgenics acquired neuroFix in 2015 and renamed the programme NFC-1 before changing its own name to Aevi Genomic Medicine.
• At Aevi, the drug became AEVI-001 and was studied in children with ADHD whose genetic profiles suggested disruption of the mGluR pathway. The company also explored its use in 22q11.2 deletion syndrome.
• The ADHD programme did not produce convincing efficacy results. Aevi was later acquired by Cerecor, now Avalo Therapeutics, although public disclosures do not clearly explain how the fasoracetam rights subsequently moved to Nobias. The drug eventually resurfaced as NB-001, becoming Nobias’ principal clinical asset.

Also notable: Nobias completed a randomized crossover Phase 2 study involving 37 children and adolescents with 22q11.2 deletion syndrome. The study showed a favourable safety and tolerability profile and produced positive efficacy signals, including statistically significant improvements in clinically relevant patient subgroups.

What’s next: Synaptiq is preparing a Phase 2b trial at medical centres in North America and Europe. The study will use a clinical global impression scale developed specifically for 22q11.2 deletion syndrome, drawing on insights from the completed Phase 2 trial.

• Synaptiq’s shareholder Sanos Group will provide clinical development and contract research support, while Arctic Therapeutics will contribute its drug development platform, clinical operations capabilities and research network, including its relationship with the Center for Applied Genomics at the Children’s Hospital of Philadelphia.