Nobias Therapeutics Announces Phase 2 Clinical Trial Results for NB-001 at 52nd Child Neurology Society Annual Meeting

Trial met its primary endpoint showing NB-001 is safe and well-tolerated; efficacy measures support progression to registrational trial.

MOUNTAIN VIEW, CA, AND PHILADELPHIA, PA – Oct 10th, 2023 – Nobias Therapeutics, a biotechnology company pioneering AI-based deep phenotyping techniques to discover advanced therapeutics, announced topline data from a Phase 2 clinical trial of NB-001 (fasoracetam), a treatment for the neuropsychiatric symptoms associated with 22q11.2 deletion syndrome ("22q11DS") in children. The data in the late-breaking abstract and poster presentation "A Randomized Double-Blind Placebo-Controlled Phase 2 Clinical Trial of NB-001 (Fasoracetam) for Neuropsychiatric Symptoms in Children with 22q11.2 Deletion Syndrome" were presented at the 52nd Child Neurology Society (CNS) Annual Meeting in Vancouver, BC, on October 5, 2023.

The trial included participants aged 6 to 17 years old with molecularly confirmed 22q11DS and a Clinical Global Impression (CGI)-Severity Scale score of ≥ 4 at screening. The patients also had either (1) psychiatric symptoms in the clinical range for anxiety disorder, attention deficit hyperactivity disorder (ADHD), or autism spectrum disorder, or (2) psychiatric symptoms in the subclinical range for at least 2 out of 3 of these neuropsychiatric disorders. The presence of a history of psychosis was an exclusion criterion for this trial.

Results from the multi-center, randomized, placebo-controlled crossover trial demonstrate the safety and tolerability of NB-001, as well as robust efficacy trends that further support evaluation in a registrational clinical trial.

"We are encouraged by the progress of NB-001 and its potential to help children who have 22q11DS," said Neil Inala, President and CEO of Nobias Therapeutics. "The data further increase our confidence in our NB-001 program, particularly given the small sample size and limited treatment period of this trial. These findings also underscore the value of the genomic dataset and associated electronic health record (EHR) data around which we are building our AI-based deep phenotyping drug discovery engine."

In this crossover trial, patients with 22q11DS received two 6-week treatment periods separated by a 1-week washout period. Each patient was randomly assigned to one of two treatment sequences: NB-001 followed by placebo or placebo followed by NB-001. Evidence from prior studies shows that one mechanistic effect of NB-001 is non-stimulant activation of multiple metabotropic glutamate receptors (mGluRs).

The primary endpoint of the trial was to determine the safety and tolerability of NB-001. The most common adverse events in the 37 patients treated were fatigue and nasopharyngitis in 9 percent of participants, while 6 percent experienced epistaxis, pyrexia, and/or vomiting. All adverse events were graded mild to moderate and there were no serious adverse events attributable to treatment.

The trial's key secondary endpoint was to assess the efficacy of NB-001 using the CGI-Improvement Scale (CGI-I). The least squares mean CGI-I for the treatment period was 3.34 (standard error: 0.15) versus 3.69 (0.15) for placebo (delta=-0.36 (0.20), p=0.07). Investigators further found that NB-001 treatment resulted in improved CGI-I scores for all three symptom groups (ADHD, anxiety, and autism) as compared to their individual baseline scores. Proportionally, the number of patients responding to treatment was 1.4 to 1.7 times higher as compared with placebo. The trial was intended to be signal-finding and therefore was not powered for efficacy endpoint statistical significance.

"Many children with 22q11DS have symptoms of inattention, anxiety, and difficulties with social communication, and very often have symptoms in more than one of these domains. This constellation of symptoms often results in significant functional impairment. The results of this important trial demonstrate that NB-001 holds promise for the high unmet medical need in this patient group and support further clinical development of NB-001 in a registrational-intent Phase 3 clinical trial," said Dr. Bridget Martell, M.D., Advising CMO for Nobias Therapeutics.

About 22q11DS
22q11.2 deletion syndrome ("22q11DS"), also known as DiGeorge syndrome, velocardiofacial syndrome (VCFS), conotruncal anomaly face syndrome, Takao syndrome, and Shprintzen syndrome, is the most common human deletion syndrome and is associated with delayed or incomplete development of several body structures or systems. The syndrome is also strongly associated with neuropsychiatric conditions, including attention deficit hyperactivity disorder (ADHD), anxiety, autism, and schizophrenia/psychosis.

About Nobias Therapeutics
Nobias Therapeutics is a biotechnology company pioneering deep phenotyping to discover advanced therapeutics. Nobias uses its cutting-edge, applied AI platform to distill disease drivers and optimal points for therapeutic intervention from rich clinical and genetic datasets, and to accelerate the development of novel therapies for a broad range of disorders. To learn more, please visit and follow Nobias Therapeutics on LinkedIn.

Nobias Therapeutics Media Contact:
Virgie Townsend
Nobias Therapeutics
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