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Who leads BlackThorn Therapeutics?
BlackThorn was founded in 2015 by Alex Tkachenko (CEO) and Bill Martin (CSO then CEO) to bring machine learning to neuropsychiatric drug development. They built this company just as large biopharma was reducing the footprint of their neuroscience programs. A biochemist by training, Tkachenko previously founded Siluria Technologies in the natural gas industry and worked at Genentech focusing on companion diagnostics. He was also VP of Business Development at Hana Bioscience, which was focused on ALL drug development. Tkachenkso served as a squad leader in the Soviet Army seeing combat in Afghanistan. Martin worked at Theravance Biopharma focusing on drug development for CNS disorders. He started his career at Merck in the neuroscience division after a postdoc at UCSF at the Keck Center for Integrative Neuroscience.
The genesis of BlackThorn started off at The Scripps Research Institute led by Hugh Rosen and Edward Roberts. The research project was focused on discovering selective kappa opioid receptor (KOR) antagonists. The goal was to discover drug candidates to treat neurobehavioral disorders like depression. KOR has been found to be activated in a wide range of psychiatric and neurological disorders. BlackThorn spun off from Scripps with a lead asset that inhibits KOR, BTRX-335140, from academic research and built a platform around the asset to create new medicines for neuropsychiatry.
What does BlackThorn Therapeutics do?
The company is centered around their INFORM (Integrated Functional Objective Research Measures) platform. The technical premise is to connect brain physiology and genomics with behavior. This type of screening method in models could have a better chance at identifying useful markers for the pathology of neurobehavioral disorders. So INFORM has 5 main parts to it:
Identify new targets with brain imaging and sequencing. BlackThorn’s focus is on receptors that are found in dysregulated brain circuits connected to a disease.
Validate new biomarkers, both genomic and digital
Combine the first 2 to focus on particular patient populations based on the effects of these targets on the pathology and a biomarker’s ability to stratify patients
Select patients based on these features
Develop new behavioral endpoints to execute clinical trials
The idea is to bring precision to mental health to increase the odds of success during the drug development process and run smaller, more efficient clinical trials. BlackThorn’s vision of bringing personalized medicine to mental health patients requires a platform to collect and analyze very large amounts of data spanning sequencing and brain images to behavior. The company has likely built the largest database connecting neuropsychiatric phenotypes to genotypes. All of this computational work and multimodal data analysis is powered by PathFinder, which is the backend tooling for INFORM.
What makes BlackThorn Therapeutics unique?
Millions, if not over a hundred million, of patients around the world suffer from depression and many more from other mental health disorders. A historical challenge for drug developers in this field has been the patient heterogeneity. The tools to measure longitudinal behavior of patients and sequence them have only been available over the last decade or so. BlackThorn built a company to bring a suite of technologies together that really hadn’t ever been combined, to my knowledge, before at the time.
To accomplish this goal, the company has built a unique team blending neuroscience and clinical research with machine learning and scaling cloud-based infrastructure. BlackThorn is one of the few neuro companies with the ability to analyze complex datasets and have the capabilities to translate this computational work into new medicines.
Another important part of BlackThorn is its pipeline. Rather than starting off as a platform and developing new drugs from day one, the company in-licensed BTRX-335140 from Scripps at their founding. BTRX-335140 is a selective and reversible KOR antagonist in phase 2 clinical trials in patients with major depressive disorder. Even though this drug candidate didn’t come out of INFORM, it helps BlackThorn create value sooner and actually give them a buffer to invest in their platform before deploying it into the clinic. Recursion Pharmaceuticals also executed a similar strategy. Now BlackThorn has a second drug candidate, BTRX-323511, which inhibits the vasopressin 1a receptor to potentially treat autism.
Why I like what BlackThorn Therapeutics is doing?
BlackThorn got started on working on mental health drug development just as others were pulling out. The company has spent over 5 years building a powerful platform and is positioned to drive more drug candidates through clinical development. Traditional approaches in neuropsychiatry have relied on categorical diagnosis of disorders. BlackThorn has the toolkit to take a circuit-based approach, which can lead to a more precise understanding of the pathology and improve patient selection for clinical trials and use.
The company’s unique computational psychiatry platform is bringing precision medicine to mental health. With so much unmet need, any new company has the opportunity to build an enduring business and define the regulatory pathway for every other drug development company.
You can find BlackThorn Therapeutics here.