BlackThorn Therapeutics Advances Phase 2 Clinical Development for Selective KOR Antagonist, BTRX-140, in Neuropsychiatric Disorders

SAN FRANCISCO--(BUSINESS WIRE)-- BlackThorn Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing targeted medicines to treat central nervous system (CNS) disorders, today announced the advancement of Phase 2 clinical development for its lead drug candidate, BTRX-335140 (BTRX-140), a selective antagonist for the kappa opioid receptor (KOR). Dosing has now begun in a Phase 2 proof-of-concept trial for BTRX-140 in a subpopulation of patients with major depressive disorder.

The KOR neuropeptide system is an important mediator of the negative effects of stress-induced alterations on mood, emotional regulation and motivation. KOR antagonism is a novel therapeutic approach, which offers the potential to address key drivers of disability, such as depressed mood, anxiety, anhedonia and cognitive impairment. These clinical symptoms commonly occur in multiple CNS disorders, but are not adequately addressed by currently available medications for many people seeking therapy.

“BlackThorn is focused on developing a new generation of medicines for CNS disorders by leveraging our proprietary computational psychiatry platform,” said Bill Martin, Ph.D., BlackThorn’s president and chief executive officer. “Independent clinical research has shown that blocking KOR modulates key brain circuits and reduces symptoms in patients with mood and anxiety disorders.1 Using our precision approach, we have gained novel insights about the patients most likely to benefit from KOR antagonist therapy. In this Phase 2 trial for BTRX-140, we are applying our data-driven insights intended to further increase confidence in a clinically proven mechanism that is differentiated from current treatments for depression.”

“BlackThorn’s clinical pipeline is designed to restore balance to dysregulated brain circuits that are relevant to multiple CNS disorders,” said Jane Tiller, MBChB, FRCPsych, BlackThorn’s chief medical officer. “By targeting the KOR system, BTRX-140 has the potential to address clinical symptoms shared across multiple disorders. The BTRX-140 Phase 2 development program is supported by positive Phase 1 results, which confirmed that BTRX-140 engages KORs in the brain at levels that are projected to be effective in treating symptoms relevant to depression and other CNS disorders.”

The Phase 2 proof-of-concept trial is a double-blind, placebo-controlled, randomized trial designed to evaluate the efficacy, safety and tolerability of BTRX-140 in approximately 120 patients with major depressive disorder. The primary endpoint of the trial is change in Hamilton Rating Scale for Depression (HAM-D) total score in the BTRX-140 treatment group compared to the placebo group following eight weeks of treatment.

About BlackThorn’s Lead Clinical Program: BTRX-140

BTRX-140 is a potent, selective KOR antagonist being investigated for the treatment of neurobehavioral disorders, including major depressive disorder. Activation of the KOR system under conditions of stress and disease can contribute to symptoms associated with depressed mood, anhedonia, anxiety and cognitive impairment, which are key drivers of disability in numerous psychiatric and neurological disorders. BTRX-140 is intended to reduce stress responses and restore dysregulated brain networks underlying these symptom domains, providing a novel therapeutic approach to treating neurobehavioral disorders. BTRX-140 was discovered as part of a research collaboration with The Scripps Research Institute.

About BlackThorn Therapeutics

BlackThorn Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery and development of targeted medicines for the treatment of central nervous system (CNS) disorders. With pathfinder™, our computational psychiatry platform, our approach is based on generating a quantitative, brain-based understanding of neurobehavioral disorders. These data-driven insights are applied to create treatments targeted to biologically-based subtypes of patients to increase the precision of therapeutic intervention. Our clinical pipeline is designed to restore balance to dysregulated brain circuits and empower individuals with CNS disorders to live better lives. BlackThorn is headquartered in San Francisco, California. For more information, please visit


  1. Krystal A, Neuropsychopharmacology 43, 1–76 (2018)



Laura Hansen, Ph.D., VP, Corporate Affairs
tel: 650-703-6523


Source: BlackThorn Therapeutics, Inc.

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