BlackThorn Therapeutics To Receive Up to $8 Million in NIH Blueprint Neurotherapeutics Network Funding to Advance Kappa Opioid Receptor (KOR) Antagonist Program

South San Francisco, Calif. – June 20, 2017 – BlackThorn Therapeutics, a clinical-stage biopharmaceutical company discovering and developing targeted treatments for neurobehavioral disorders, today announced it has received a grant from the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH). The grant is funded as part of the Blueprint Neurotherapeutics Network (BPN) of the NIH Blueprint for Neuroscience Research, a collaboration of NIH Institutes and Centers that supports research on the nervous system with the goal of developing new neurotherapeutic drugs. This award is worth up to $8 million to support BlackThorn’s development of kappa opioid receptor (KOR) antagonists.

“Recent research suggests that KOR is a novel therapeutic target for disorders associated with stress, including migraine,” said Bill Martin, Ph.D., chief scientific officer of BlackThorn. “For KOR antagonists to have therapeutic potential, they must be potent and highly selective and have reversible pharmacology and excellent brain penetration. To date, BlackThorn’s KOR antagonists have demonstrated these properties, which helped the company receive this prestigious grant award. Research support from the NINDS will help us discover and evaluate KOR antagonists with the goal of developing them for multiple therapeutic applications, including migraine prophylaxis.”

“Migraine is a prevalent and disabling neurological disorder that severely impacts patients’ quality of life,” said Charles L. Cywin, Ph.D., program director of BPN. “We look forward to building on BlackThorn’s capabilities in working with the KOR pathway and to potentially targeting the KOR to develop treatments for migraine relief.”

“BlackThorn has worked on the KOR pathway since its inception and is well positioned to leverage its medicinal chemistry expertise toward migraine prophylaxis,” said Rob Jones, Ph.D., the principal investigator on the grant, who will work in collaboration with co-investigators Professor Edward Roberts, Ph.D., and Professor Hugh Rosen, M.D., Ph.D., both of The Scripps Research Institute, and Professor Frank Porreca, Ph.D., of the University of Arizona Health Sciences. Dr. Jones added, “Preliminary data suggest that stress can induce features that are consistent with clinical observations of migraine pain in a novel animal model of medication overuse headache induced by a period of prior exposure to triptan drugs. Central KOR blockade appears to counteract this effect in part via a CGRP-dependent mechanism.”

Approximately 40 million Americans suffer from migraines, and the disease costs the United States about $36 billion annually in healthcare costs and lost productivity, according to the Migraine Research Foundation.

About the KOR Pathway in Neurological Disorders

The KOR pathway – located within brain regions that regulate the negative effects of stress on behavior – recently has emerged as a novel therapeutic target for the treatment of disorders related to stress, including depression, substance abuse disorder and migraine. The anatomical and physiological roles of the KOR suggest that blocking it could be therapeutically useful for these disorders.

About BlackThorn’s KOR Antagonist Program

BlackThorn is building a proprietary KOR antagonist program through a partnership with The Scripps Research Institute. Preclinical studies of the compounds in BlackThorn’s KOR program have shown potency, selectivity and reversibility, and additional studies are ongoing. BlackThorn plans to use the Blueprint grant to develop novel compounds and optimize their KOR affinity, selectivity, reversibility, oral availability, brain/plasma ratio, and drug metabolism and pharmacokinetic profiles for potential therapeutic use in stress-related migraine prophylaxis, alongside BlackThorn’s other KOR programs, which are targeting neurobehavioral and neurologic disorders.

About the NIH Blueprint for Neuroscience Research

The NIH Blueprint for Neuroscience Research is a collaborative framework that includes the NIH Office of the Director and 15 NIH Institutes and Centers that support research on the nervous system. By pooling resources and expertise, Blueprint identifies cross-cutting areas of research, and confronts challenges too large for any single Institute or Center. One of Blueprint’s Grand Challenges, launched to catalyze research with the potential to transform basic understanding of the brain and approaches to treating brain disorders, is the Blueprint Neurotherapeutics Network. The Network provides funding to researchers and biopharmaceutical companies for small molecule drug discovery and development. For more information, please visit

About BlackThorn Therapeutics, Inc.

BlackThorn Therapeutics is a clinical-stage biopharmaceutical company dedicated to transforming the lives of people with neurobehavioral disorders through the discovery and development of novel, targeted treatments. BlackThorn has a robust pipeline of first- and best-in-class treatments. The company approaches drug discovery and development by linking brain physiology to behavior. Through BlackThorn’s network of collaborations with leading academic investigators and technology alliances, the company is developing and deploying a set of technologies that will enable focused, rapid, resource-efficient translational studies for drug development. The company is headquartered in South San Francisco, Calif. For more information, please visit

About The Scripps Research Institute

The Scripps Research Institute (TSRI) is one of the world’s largest independent, not-for-profit organizations focusing on research in the biomedical sciences. TSRI is internationally recognized for its contributions to science and health, including its role in laying the foundation for new treatments for cancer, rheumatoid arthritis, hemophilia and other diseases. An institution that evolved from the Scripps Metabolic Clinic founded by philanthropist Ellen Browning Scripps in 1924, the Institute now employs more than 2,500 people on its campuses in La Jolla, Calif., and Jupiter, Fla., where its renowned scientists – including two Nobel laureates and 20 members of the National Academies of Science, Engineering or Medicine – work toward their next discoveries. The institute’s graduate program, which awards Ph.D. degrees in biology and chemistry, ranks among the top ten of its kind in the nation. In October 2016, TSRI announced a strategic affiliation with the California Institute for Biomedical Research (Calibr), representing a renewed commitment to the discovery and development of new medicines to address unmet medical needs. For more information, please visit

About the University of Arizona Health Sciences

The University of Arizona Health Sciences is the statewide leader in biomedical research and health professions training. The UA Health Sciences includes the UA Colleges of Medicine (Phoenix and Tucson), Nursing, Pharmacy and Mel and Enid Zuckerman College of Public Health, with main campus locations in Tucson and the growing Phoenix Biomedical Campus in downtown Phoenix. From these vantage points, the UA Health Sciences reaches across the state of Arizona and the greater Southwest to provide cutting-edge health education, research, patient care and community outreach services. A major economic engine, the UA Health Sciences employs almost 5,000 people, has nearly 1,000 faculty members and garners more than $126 million in research grants and contracts annually. For more information, please visit Follow: Facebook | Twitter | YouTube | LinkedIn

Julie Normart
Pure Communications

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