Recognify Life Sciences announced that its Phase 2b clinical trial evaluating inidascamine (formerly RL-007) in patients with cognitive impairment associated with schizophrenia (CIAS) failed to achieve statistical significance on its primary endpoint, despite showing consistent numerical improvements across multiple cognitive measures.
The randomized, placebo-controlled, double-blind Phase 2b study enrolled 242 patients across the United States and Europe (NCT05686239) and evaluated the efficacy, safety, and tolerability of two doses of inidascamine versus placebo over a six-week treatment period.
Trial Results and Efficacy Measures
While the study did not achieve statistical significance on its primary endpoint of improvement on the Measurement and Treatment Research to Improve Cognition in Schizophrenia Consensus Cognitive Battery (MCCB) neurocognitive composite score at Week 6, inidascamine demonstrated modest but consistent numerical improvement across the overall MCCB neurocognitive composite and multiple individual subdomains, including Symbol Coding, Speed of Processing and Verbal Learning (immediate recall).
Directionally positive effects were also observed on the Virtual Reality Functional Capacity Assessment Tool (VRFCAT), a measure of real-world functional cognitive capacity.
"Although we are disappointed that the study did not reach statistical significance on the primary efficacy endpoint, we are encouraged by the consistency of improvement signals across multiple cognitive and functional measures as well as replication on specific subsets of the cognitive measures; namely, symbol coding and verbal memory," said Matt Pando, PhD, Chief Executive Officer and Co-Founder of Recognify Life Sciences.
Safety Profile
Inidascamine was well-tolerated, with a favorable safety profile consistent with previous studies. Importantly, no evidence of sedation, weight gain, or extrapyramidal symptoms was observed, which are side effects commonly associated with treatments used in people living with schizophrenia.
Expert Commentary
Keith Nuechterlein, PhD, Distinguished Professor in the UCLA Department of Psychiatry and Biobehavioral Sciences and co-chair of the MATRICS Neurocognition Committee, commented: "Developing treatments for cognitive impairment in schizophrenia is a complex challenge that requires persistence and innovation. I am encouraged by the inidascamine topline results showing consistent directional tendencies for improvement and look forward to seeing the full set of results."
Drug Mechanism and Development History
Inidascamine is an orally available compound with unique mechanisms of action for the treatment of cognitive impairment that modulates cholinergic, glutamatergic and GABA-B receptors, thereby putatively altering the excitatory/inhibitory balance in the brain to produce pro-cognitive effects. It has previously been evaluated in ten clinical studies, including one in the CIAS indication, with over 600 unique participants dosed to date. Clinical studies of inidascamine suggest improvement in cognitive performance, particularly for verbal learning and memory and processing speed.
Market Context and Unmet Need
Schizophrenia affects over 21 million people globally and approximately 2.4 million people in the United States, with around 300,000 new cases being diagnosed each year in the US. Cognitive deficits are present in approximately 80% of patients and represent a core feature of the illness that contributes significantly to long-term disability and impairments in daily functioning. There are presently no effective pharmaceutical treatments approved for CIAS, representing a high unmet clinical need.
Future Development Plans
A comprehensive analysis of remaining secondary and exploratory endpoints, including subgroup analyses, is ongoing to determine whether there are identifiable responder populations or mechanistic insights that may guide future development. Recognify plans to present additional results from the study at upcoming scientific meetings and will continue to evaluate strategic options for inidascamine based on the totality of data.
Srinivas Rao, MD, PhD, Chief Executive Officer and Co-Founder of atai Life Sciences, added: "CIAS remains a challenging therapeutic area with a significant unmet need. While we believe these results support the continued development of inidascamine by Recognify for CIAS as well as its potential application in other indications, as previously communicated, we intend to allocate atai's resources on our wholly owned pipeline of transformative psychedelic product candidates focused on affective disorders."
