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BlackThorn Therapeutics Announces Positive Phase 1 Results for Its Selective KOR Antagonist, BTRX-33

Phase 1 results and new translational data supporting the development of BTRX-335140 for neurobehavioral disorders presented at the Kappa Therapeutics 2019 Conference

San Francisco – April 1, 2019 – BlackThorn Therapeutics, a neurobehavioral health company leveraging its proprietary computational psychiatry platform to develop targeted therapeutics, today announced positive Phase 1 results and new translational data supporting development of BTRX-335140, the company’s selective kappa opioid receptor (KOR) antagonist, for neurobehavioral disorders. The findings were presented at the Kappa Therapeutics 2019 Conference that took place March 28 – 30 in Seattle, Washington.

The KOR system is an important mediator of the negative effects of stress-induced alterations on mood, emotional regulation and motivation. This system has been a target for drug development in neurobehavioral disorders where dysregulation of stress signaling is associated with increased vulnerabilities.

BlackThorn reported results from a Phase 1 trial showing that BTRX-335140 exhibited favorable pharmacokinetics and was well tolerated in healthy human subjects. Preclincial data also showed that BTRX-335140 has a favorable drug profile, including brain penetration, potency, selectivity and a medication-like duration of action.

BlackThorn also presented translational data generated with GEMINI-DOT™, the company’s proprietary technology designed to identify genes expressed in brain circuits linked to behavioral symptoms. Using GEMINI-DOT, the company identified a robust correlation between expression of the KOR gene and a brain circuit involved in cognitive function.

The activity of BTRX-335140 on this cognitive circuit was tested in a preclinical model. Results showed that BTRX-335140 conferred resiliency to stress-induced cognitive performance deficits. Future clinical research in this area may leverage MINT-COG™, BlackThorn’s proprietary digital task that assesses motivation and cognition.

“Successful completion of this Phase 1 trial is a significant clinical milestone for our BTRX-335140 development program,” said Bill Martin, Ph.D., BlackThorn’s President and Chief Operating Officer. “In addition, the translational data support our approach of using behavioral biomarkers to identify patient subtypes most likely to respond to treatment. As part of Phase 2 readiness with BTRX-335140, we look forward to completing a PET imaging study in mid-2019.”

About BlackThorn Therapeutics, Inc. BlackThorn Therapeutics is a neurobehavioral health company developing targeted therapeutics, informed by a deep understanding of brain and behavior relationships, to significantly improve patient outcomes. The company has pioneered a proprietary computational psychiatry platform, which leverages bioinformatics, neuroinformatics and artificial intelligence technologies, with the goal of determining a patient’s distinct neuroprint to inform the proper on-target treatment option for biologically defined patient subtypes. BlackThorn Therapeutics has completed a Phase 1 trial of BTRX-335140, a selective kappa opioid receptor antagonist, and is conducting IND-enabling studies for BTRX-323511, a selective vasopressin 1a receptor antagonist. The company also has a drug discovery program directed at novel therapeutic targets for neurobehavioral disorders. BlackThorn Therapeutics is headquartered in San Francisco, California. For more information, please visit

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