BlackThorn Therapeutics raised $76 million to push at least one of its targeted treatments for neurobehavioral disorders into phase 2 trials and to keep working on the machine learning-based technology that underlies its clinical pipeline.
Neurobehavioral disorders are traditionally diagnosed through qualitative means—that is, a physician’s observation of the presence or absence of symptoms. Poor understanding of particular forms of disease such as treatment-resistant depression and their underlying biology results in treatments that don’t work well, BlackThorn told FierceBiotech in a previous interview. The biology behind each patient’s disease is different, but they are treated with a one-size-fits-all approach.
Founded in 2016, BlackThorn aims to create better treatments for disorders such as depression, schizophrenia and obsessive-compulsive disorder by better understanding the link between biological changes in the brain and patient symptoms. To that end, it has developed an artificial intelligence and machine learning-based platform that gathers, integrates and analyzes data to identify brain circuits that have gone awry and generate biomarkers that help in the development of targeted treatments.
Dubbed PathFinder, the cloud-based technology uses these “neuromarkers” to identify drug targets, come up with clinical trial endpoints and divide patients into “neurobiologically-defined patient populations.” The hope is, by identifying patients whose diseases share the same underlying biological causes, BlackThorn will be able to create drug candidates with a higher likelihood of success.
“BlackThorn was founded to bring new therapies to patients by applying advances in computational sciences to address patient heterogeneity, one of the biggest historical challenges in the field of neuropsychiatric drug development,” said Bill Martin, Ph.D., BlackThorn's chief operating officer, in a statement. “Three years later, insights from our data-driven approaches are yielding patient enrichment strategies that could increase probability of clinical trial success and improve patient outcomes.”
“We have pioneered methodologies using 'XAI' (explainable AI) to generate predictive models that can be used to inform patient enrichment strategies for clinical development. In this way, we can identify patient subgroups using baseline characteristics to predict who is more likely to respond to drug or placebo. This could result in more efficient clinical trials and better patient outcomes. The 'XAI' component is key because it allows us to generate interpretable models that a clinician can use to select patients,” Martin told FierceBiotech in an email.
BlackThorn’s lead drug BTRX-335140 is a kappa opioid receptor antagonist for mood disorders slated to enter phase 2 by the end of the year. Its second program, BTRX-323511, is an antagonist of the vasopressin 1a receptor in the works for social-emotional disorders such as autism spectrum disorder. The company plans to file its IND early next year.
In a January R&D update, the company announced that a third program, an antagonist of the nociceptin receptor, missed its endpoints in a phase 2a study testing it in major depressive disorder. BlackThorn said at the time that “further analyses” were underway but that it had deprioritized the program, originally licensed from Eli Lilly.
In addition to funding the phase 2 trial for BlackThorn’s mood disorder program, the funding will also support efforts to “build out the computational and data aspects” of the company’s technology. The capital came from a laundry list of investors, including newcomers Polaris Partners, Premier Partners, Scripps Research and Vertex Ventures, as well as existing backers Alexandria Venture Investments, Altitude Life Science Ventures, ARCH Venture Partners, Biomatics Capital, GV, Johnson & Johnson Innovation and Mercury Fund.
“Our continued growth is supported by our growing team, which includes appointments to our executive management team today,” Martin said. BlackThorn added Dr. Jane Tiller as chief medical officer and Laura Hansen, Ph.D., as vice president of corporate affairs.