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Biotech, A.I. Companies Model Animal Studies/Clinical Trials


(Lynn Ketchum, Oregon State University, Flickr.

18 Aug. 2023. A discoverer of neuroscience therapies and a genomics data science company are developing machine learning models to predict clinical trial outcomes for ADHD drugs. Financial and intellectual property details of the collaboration between 3Z Pharmaceuticals in Reykjavík, Iceland and in Potsdam, Germany — the company name is spelled in all lower-case — are not disclosed.

3Z Pharmaceuticals discovers new treatments for central nervous system disorders by screening existing drugs with genetic models in zebrafish, a tropical fish in the minnow family. Zebrafish have a 70 percent genetic similarity to humans, with an array of organs also similar to humans including brain and spinal cord. 3Z Pharma says zebrafish brains express the same neurotransmitters as humans, which allows for real-time live animal screening of drug candidates. The company says it tracks behaviors of genetically-engineered zebrafish affected by drugs, with models linking those behaviors to schizophrenia, Parkinson’s disease, sleep disorders, and attention-deficit/hyperactivity disorder or ADHD.

3Z Pharma says it repurposes currently approved drugs since they often have known safety profiles, which require fewer early-stage clinical trials and thus faster market entry. The company now has four therapies for ADHD in its pipeline, with an insomnia treatment, all in preclinical stages.

Link clinical trial success to zebrafish screenings is a six year-old enterprise building machine-learning models for identifying causal biomarkers for drug discovery. The company says its algorithms are based on data from 28,500 drugs and 2 million genomes linked to 12,000 diseases, and 500 disease-related traits. says its models run in the cloud with a capacity of one million experiments per day. The company works for drug makers and biotechnology companies to discover causal biomarkers to design clinical trials, repurpose existing drugs, and better understand therapies’ mechanisms of action. One of its customers is a developer of treatments for Alzheimer’s disease.

In their collaboration, and 3Z Pharmaceuticals seek to develop algorithms for predicting the clinical trial success of ADHD drug candidates from real-world data generated by zebrafish screenings. The companies expect the algorithms to be based on genomic models to predict likely effects on treating ADHD in patients, evaluate metabolic impacts, and assess effects on other disorders. The data will then predict outcomes in a simulated mid-stage clinical trial. 3Z Pharma believes the findings can reduce the risk of developing new uses for current drugs by finding direct links between preclinical animal tests and clinical trial results.

“We are entering a new era in drug discovery and development,” says 3Z Pharmaceuticals CEO Karl Karlsson in a company statement released through Cision, “where AI has the potential to revolutionize the industry. The animal models we develop focus on disorders and diseases that manifest as dysregulation within neuronal networks, which is probably the most challenging aspect to simulate successfully. However, we can model the effects of the drugs we identify on specific genetic targets, enabling us to obtain critical information and significantly reduce the risk of failure going forward.”

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