Nobias Therapeutics, a clinical-stage company developing novel small-molecule therapeutics for rare diseases, today announced that it has secured preliminary alignment with the U.S. Food and Drug Administration (FDA) on registrational endpoints for its lead program, NB-001, for the treatment of neuropsychiatric symptoms associated with 22q11.2 deletion syndrome (22q11DS). This critical milestone clarifies the regulatory expectations for potential approval.
PHILADELPHIA, June 16, 2025 /PRNewswire-PRWeb/ -- Nobias Therapeutics, a clinical-stage company developing novel small-molecule therapeutics for rare diseases, today announced that it has secured preliminary alignment with the U.S. Food and Drug Administration (FDA) on registrational endpoints for its lead program, NB-001, for the treatment of neuropsychiatric symptoms associated with 22q11.2 deletion syndrome (22q11DS). This critical milestone clarifies the regulatory expectations for potential approval.
The FDA has agreed a global assessment of improvement plus a key secondary clinical efficacy outcome measure may be used as registrational endpoints in support of potential approval of NB-001.
For a global assessment, Nobias plans to use a disease-specific Clinical Global Impression - Improvement (CGI-I-22q) measure that it is developing based on guidance provided at its End of Phase 1 (EOP1) meeting with the FDA and in alignment with FDA guidance on rare disease trials and ISPOR task force recommendations and best practices for clinician-reported outcome (ClinRO) development. Nobias has discussed this CGI-I-22q with the FDA at a separate Type C meeting.
This novel global assessment focuses on specific symptom domains that are clinically relevant to patients with 22q11DS and most likely to be improved by NB-001. Nobias completed initial content validity work for the CGI-I-22q scale over the summer of 2024.
"Using a CGI-I scale is the most appropriate endpoint for this patient population," said Dr. Jacob Vorstman, MD, PhD, Professor of Psychiatry at the University of Toronto and Senior Scientist at the Sick Kids Research Institute, where he served as the primary investigator in Nobias' Phase 2 study. "This is based on a substantial body of evidence that shows symptoms may not reach categorical diagnosis in any single domain, but that the cumulative impact of sub-threshold symptoms across multiple domains can result in significant disease burden."
NB-001 is Nobias' most advanced clinical program and a small-molecule metabotropic glutamate receptor (mGluR) modulator. In late 2023, the company announced positive top line data from a multi-center, randomized, double blind, placebo-controlled Phase 2 clinical trial of NB-001. (More information about the Phase 2a study data is available here.) In late 2024, the program was granted both Orphan Drug Designation and Rare Pediatric Disease Designation by the FDA.
"Obtaining preliminary FDA alignment on registrational endpoints is a pivotal milestone in our development strategy," said Patrick Dougherty, CEO at Nobias Therapeutics. "Our recent, constructive interactions with the Agency have provided valuable guidance on many aspects of the development of NB-001, which we hope will become the first therapeutic agent developed specifically for children with 22q11.2 deletion syndrome. This feedback from the FDA clarifies our path forward and strengthens the program's value proposition for strategic partners and investors."
Next Steps: Nobias plans to conduct an independent study to generate preliminary evidence of the measurement properties of the CGI-I-22q. This will be followed by a Phase 2b study of NB-001, using mean change on the CGI-I-22q as the primary efficacy endpoint. The Phase 2b clinical study will also generate data on an objective measure of attention and executive function, as the company assesses options for the key secondary measure in a future registrational study.
About 22q11DS:
With an estimated prevalence of 1 in 2148 live births, approximately 1600 children are born with 22q11DS in the U.S. every year, affecting approximately 65,000 Americans and making it the most common human microdeletion. Children living with 22q11DS typically experience a distinctive mix of neuropsychiatric symptoms that include elements of attention deficit hyperactivity disorder (ADHD), anxiety, and autism spectrum disorder (ASD). Currently, there are no therapies that are specific for the 22q11DS population to treat these conditions, leaving many 22q11DS patients with inadequate treatment options and resulting in a significant burden for their parents and other caretakers.
About Nobias Therapeutics:
Nobias Therapeutics is a clinical-stage company developing novel small molecule therapeutics for rare diseases. Our unique access to rich genomic and health datasets and strong relationships with pediatric academic medical centers across North America allow us to rapidly advance drug discovery for genetically-defined rare diseases, with an initial focus on pediatric indications, but expansion opportunities in adults and non-rare indications. To learn more, please visit http://www.nobiastx.com and follow Nobias Therapeutics on LinkedIn.
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