Synaptiq Therapeutics Launches with Orphan Drug Candidate

A new clinical-stage biopharmaceutical entity, Synaptiq Therapeutics, has officially launched, fueled by a strategic acquisition of a promising therapeutic candidate targeting 22q11.2 Deletion Syndrome (22q11DS). The company is set to advance SYN-001, a small molecule modulator of metabotropic glutamate receptors, into Phase IIb trials. This compound has already secured both Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA), underscoring its potential to address a significant unmet medical need.

The formation of Synaptiq Therapeutics represents a concerted effort by a syndicate of European and U.S. investors. Key founding backers include Sanos Group, a specialized clinical research organization backed by Investcorp; an Icelandic investment fund managed by AxUM Securities; and Arctic Therapeutics, a biotechnology firm with established operations in Iceland and the U.S. Nobias Therapeutics, the original developer of the asset and majority-backed by Medical Excellence Capital, will maintain an equity stake in the newly formed company.

Patrick Dougherty has been appointed Chief Executive Officer of Synaptiq Therapeutics. He highlighted the robust Phase II data already generated for SYN-001, stating, “By uniting dedicated investors, seasoned development partners, and a focused operational entity, we are strategically positioned to propel SYN-001 through Phase IIb and closer to delivering the first approved therapy specifically indicated for individuals living with 22q11DS.” The company’s operational hub will be in Iceland, leveraging Arctic Therapeutics’ drug development infrastructure and its research ties with the Center for Applied Genomics at the Children’s Hospital of Philadelphia.

The market for rare disease therapeutics is experiencing substantial growth, driven by advancements in genetic understanding and targeted drug development. 22q11DS, affecting an estimated 1 in 2,000 to 3,300 live births globally, presents a complex set of neuropsychiatric symptoms, including anxiety, ADHD, and autism spectrum disorder manifestations. Current treatment options are largely symptomatic, creating a clear demand for disease-modifying or targeted therapies. The estimated 65,000 individuals living with 22q11DS in the United States alone represent a significant patient population for whom novel treatments are critically needed.

Sanos Group will contribute its extensive clinical development and contract research expertise to support the upcoming Phase IIb study, which is slated to enroll patients at leading medical centers across North America and Europe. This collaborative approach aims to accelerate the development timeline for SYN-001. The previous Phase II trial demonstrated a favorable safety profile and yielded positive efficacy signals, particularly in specific patient subgroups. The design of the upcoming study will incorporate a 22q11DS-specific clinical global impression scale, refined based on insights from the prior trial.

The strategic acquisition and subsequent launch of Synaptiq Therapeutics underscore a growing trend of specialized entities being formed to advance promising assets in rare disease indications. This model allows for focused capital allocation and operational execution, crucial for navigating the complexities of late-stage clinical development and regulatory pathways. The involvement of experienced investors like Investcorp through Sanos Group, alongside dedicated biotech players like Arctic Therapeutics and specialized funds, signals strong confidence in the therapeutic potential of SYN-001 and the market opportunity it addresses.