Can AI Tools, $76M Lead BlackThorn to Targeted Psych Drugs?

Can AI Tools, $76M Lead BlackThorn to Targeted Psych Drugs?

It’s notoriously tough for experimental psychiatric drugs to succeed in clinical tests. BlackThorn Therapeutics wants to show that an AI-driven, precision approach can make it easier, and today it’s added another $76 million to get the chance to prove it.

The new Series B round will give San Francisco-based BlackThorn—a startup spun out of the Scripps Research Institute in 2013—the backing to run human tests of two experimental treatments. One, headed for Phase 2 testing by next year, is being developed for mood or anxiety disorders. The other, for autism, could start a Phase 1 study in 2020.

Those plans aren’t so unusual. What’s unusual is how BlackThorn intends to enroll patients in these trials. It aims to combine a slew of collected psychiatric data with computing tools to find subsets of patients with neurobehavioral diseases—mental health problems like obsessive compulsive disorder and depression—most likely to respond. The hope is this effort will lead to “targeted” therapies that show a clear effect for these particular patient groups, says CEO Bill Martin.

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“We really want to arrive at a way of guiding patients [with neurobehavioral disorders] toward the therapies that they’ll benefit from,” he says.

Targeted drugs are common in fields like oncology, where drugs can be used against genetic mutations known to drive certain cancers. But similar progress hasn’t been made with neurobehavioral diseases due to significant roadblocks. Among them: unknowns about the underlying biology of such diseases; strong placebo effects that skew results; and a lack of reliable “biomarkers,” or biological indicators, that a patient has a disease or that a drug is helping.

With mood disorders, patients are diagnosed through psychiatric evaluations, and a drug’s effects in a clinical trial are measured by observations documented by clinicians or via standardized questionnaires. These methods are far less precise than, for example, a blood test to check for the presence of a disease, or a scan to see if a cancer drug shrinks a tumor.

BlackThorn has been aiming for a more targeted, predictive approach. It’s using a combination of brain imaging technology, behavioral evaluation techniques, and a cloud-based data collection tool called “PathFinder.” That has helped the company amass a library of information meant to connect behavioral symptoms with changes in the brain’s circuitry. BlackThorn plans to use this information to design its clinical trials and show that it can find subsets of patients—those exhibiting certain symptoms and neurological characteristics linked to those symptoms—who are most likely to respond to its drugs. (This paper, published in 2018 and presented at a medical conference earlier this year, explains its use of AI in more detail.)

Still, the first of these efforts, a potential depression drug licensed from Eli Lilly (NYSE: LLY), failed a Phase 2 trial. But Martin frames that failure as more of a fact-finding mission for BlackThorn. The company enrolled all types patients with major depressive disorder. It then tested the drug with the intent of using its technology to analyze the results and more carefully select patients in future studies, Martin says. “It was always conceptualized to be a platform enabling study,” he says.

BlackThorn now has the chance to prove that that is indeed the case. The Lilly drug aside (which Martin vows will have a new path forward), the two therapies it is advancing both resemble medicines currently being tested by larger firms in broader populations. One drug, BTRX-335140, is known as a kappa opioid receptor antagonist, just like a Johnson & Johnson (NYSE: JNJ) drug in Phase 2 testing for major depressive disorder. Another, BTRX-323511, has the same target—the vasopressin 1a receptor—as the Roche drug balovaptan, which is currently in late-stage testing for autism spectrum disorder. Martin won’t say how BlackThorn intends to design the coming trials for both drugs, just that the company intends to take “a more targeted approach to development” than Roche and J&J.

New investors Polaris Partners, Premier Partners, Scripps, and Vertex Ventures HC participated in the round alongside earlier backers Arch, Alexandria Venture Investments, Altitude Life Science Ventures, Biomatics Capital, GV, Johnson & Johnson Innovation, and Mercury Fund.

Here’s more on BlackThorn and some of the troubles companies have had developing psychiatric drugs.

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