BlackThorn Therapeutics Initiates Phase 1 Study of BTRX-335140, an Investigational Kappa Opioid Receptor (KOR) Antagonist

Selective KOR Antagonist Represents BlackThorn’s Second Molecule to Enter Clinic

San Francisco – June 6, 2018 – BlackThorn Therapeutics, a clinical-stage biopharmaceutical company discovering and developing targeted treatments for neurobehavioral disorders, today announced enrollment of the first healthy volunteers in a Phase 1 study of BTRX-335140, a kappa opioid receptor (KOR) antagonist that targets brain circuits that modulate the negative effects of stress on behavior.

“KOR historically has been a challenging target so the initiation of this first-in-human study of BTRX-335140 marks an important milestone for clinical development focused on this receptor,” said Annette Madrid, M.D., chief medical officer of BlackThorn Therapeutics. “Advances in the field have been limited by an inability to design a KOR antagonist that is potent, selective for the receptor and reversible, with limited off-target activity. BTRX-335140 has been shown in preclinical studies to have all of these properties. We look forward to further evaluating BTRX-335140 in our Phase 1 program and expect data in the first half of 2019.”

KORs are located within brain regions that regulate stress and have emerged as a novel therapeutic target for a variety of disorders. For example, evidence suggests that blocking the receptor could be therapeutically useful for disorders such as anxiety and migraine, as well as symptoms of schizophrenia and subtypes of depression.

“KOR activation has been shown to produce dysphoria and anxiety in people. This provides evidence that blocking KOR has the potential for significant clinical benefit,” said Greg Vontz, chief executive officer of BlackThorn Therapeutics. “Advancing this molecule in healthy volunteers is the first step toward demonstrating clinical utility in areas of significant unmet clinical need.”

About the Phase 1 Program

The Phase 1 program includes a single and multiple ascending dose study that will evaluate the dose-exposure relationship and safety and tolerability of BTRX-335140 in healthy volunteers. Additional studies will determine BTRX-335140 receptor occupancy and circuit engagement.

About BTRX-335140

In partnership with The Scripps Research Institute, BlackThorn discovered BTRX-335140, a potent, selective and reversible KOR antagonist. BlackThorn is developing BTRX-335140 as a therapeutic for neurobehavioral disorders associated with dysregulation of brain circuits comprised of key cortical and subcortical brain regions in which KORs are expressed.

About BlackThorn Therapeutics, Inc.

BlackThorn Therapeutics is a clinical-stage biopharmaceutical company dedicated to transforming the lives of people with neurobehavioral disorders. BlackThorn has created a robust pipeline of first-in-class and potentially best-in-class treatments. The company uses novel technologies and insights from data science to uniquely understand the connection between brain and behaviors to enable targeted drug discovery and development. BlackThorn Therapeutics is headquartered in San Francisco, Calif. For more information, please visit http://www.blackthornrx.com/.

# # #

Contact:
Julie Normart
W2O pure
jnormart@w2ogroup.com
415-946-1087