BlackThorn Therapeutics

JLABS alumnus BlackThorn aims to succeed in the neuro territory by using physiological measures to better define the diseases and their populations.
Fierce 15

This clinical-stage biopharmaceutical company is looking to innovate in the neglected space of neurobehavioral disorders.

CEO: Charles Gregory Vontz
Based: San Francisco
Founded: 2016
Clinical focus: Neurobehavioral disorders, including autism spectrum disorder, obsessive-compulsive disorder, depression and schizophrenia, have typically been described in ways that don’t necessarily relate directly to disease pathology. BlackThorn hopes to change that by creating precise, physiologically based segmentation of these groups that could enable more targeted and less expensive candidate development.

The scoop: JLABS alumnus BlackThorn is another of our winners this year seeking to be a hybrid biotech-medtech company, translating deep machine learning into new therapies in the oft-neglected space of depression, schizophrenia and other associated disorders.

What makes BlackThorn Fierce: For the past few years, Fierce 15 has focused on oncology and rare diseases, predominately because that's where the next-gen therapy race is typically being staged.

BlackThorn, however, is looking to create a new wave of innovation in the neglected space of neurobehavioral disorders, such as treatment-resistant depression, components of obsessive-compulsive disorder, autism and dimensions of schizophrenia.

Its CEO Charles Gregory Vontz and CSO Bill Martin say they don’t need to waste time explaining the significance of these diseases to society, and Vontz points out that most of these disorders are poorly treated with low-efficacy therapies, and they've not seen the kind of breakthroughs achieved in oncology or rare diseases.

“What makes BlackThorn unique, unlike what has historically been done here by large pharma companies (which have largely targeted neurotransmitters)," Vontz says, "is that BlackThorn is specifically focused on key neuropeptides that are regulating known targets and circuits linked to the behaviors that are needing to be regulated."

The company has "a very broad and rich pipeline" of modulators focused on nociceptin, including the midstage BTRX-246040, a first-in-class antagonist of the nociceptin receptor that was in-licensed from Eli Lilly, he added.

With its extended $54 million series A, and with a series B on the horizon, BlackThorn has another trio of early candidates. They're all based on the work of Edward Roberts and Hugh Rosen, the Scripps researchers behind Receptos (now owned by Celgene), and formerly involved with Merck and Roche; they are now scientific co-founders of BlackThorn.

Its work is based on the INFORM platform, which aims to use advanced functional imaging and objective assessment tools to quantify emotion, behavior and cognition to better define neurobehavioral disorders. These diagnoses are more traditionally based on qualitative clinical observations.

Equally important, Vontz and Martin say, is the understanding that, as there is not one cancer, but many cancers, there are many different treatment approaches, and scientists are using diagnostics, biomarkers and personalized targeting to seek them out. So too should there be a targeted approach to disorders such as depression, and not a one-size fits all approach, they say.

“In the case of [central nervous system] disorders, for many years we didn’t really understand the drugs we were using to treat patients, and then when we started with the molecular revolution we started to say well, OK, we’re starting to understand the mechanism, but we don’t understand the patient very well," Martin says. "So, for us, what we see is a lot of opportunity based on technical and conceptual advances to really, and for the first time, put together a set of approaches that helps create a deeper understanding of the patient.”

This is vital, he says, as the failures in treating conditions such as major depressive disorder really stem from a poor relationship between a particular subtype of that disease and the underlying biology. The key is to find the molecule, understand the patient and the biology, and use these links to find drug targets.

To that end, BlackThorn built out an informatics base that integrates components from data science, signal image processing and others to create cloud-based tools that allow them to analyze a variety of data and generate an understanding of the patients and their disease.

They’re using this platform now to help predict a patient’s symptoms and severity, as well as the potential for treatment efficacy and symptom improvement. Martin says the company is unique in that it combines the biology with IT. “Some IT companies would work on the tech but have no real interest or ability in therapeutics, and then therapeutic companies, and those still remaining in this CNS space, would see data science as a bit of an afterthought. We’re integrating the two, and that only makes us stronger.”

Investors: Arch Venture Partners with participation from Johnson & Johnson Innovation – JDC, Altitude Life Science Ventures, Mercury Fund, Alexandria Real Estate Equities, GV (formerly Google Ventures), Biomatics Capital and an undisclosed crossover fund.

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BlackThorn Therapeutics