Praxis uncloaks with $100M to push precision medicines for brain disorders

DNA genomics precision medicine
Praxis' pipeline targets genes that control the imbalance in neuronal signaling that underlies CNS disorders. (Pixabay)

Praxis Precision Medicines is coming out of the shadows with $100 million from the likes of Blackstone Life Sciences and a pipeline of central nervous system (CNS) programs. The cash will bankroll a pivotal study for a depression drug as well as push earlier stage programs into the clinic.

As suggested by its name, Praxis aims to do for brain disorders what precision medicine has done for cancer treatment. Its pipeline targets genes that control the imbalance in neuronal signaling that underlies CNS disorders, both prevalent and rare. Since its inception, the Cambridge, Massachusetts-based biotech has banked $100 million from the likes of Novo Holdings, Vida Ventures and Eventide.

Praxis has two candidates in phase 2: PRAX-114, a positive allosteric modulator of GABAA receptors in development for major depressive disorder and perimenopausal depression, and PRAX-944, which blocks a type of calcium channel to treat essential tremor.

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“As was achieved in oncology decades ago, recent genetic insights have presented meaningful opportunities to treat brain disorders in entirely different and targeted ways based on the specific genetically validated pathways driving a patient’s disease,” said Kiran Reddy, M.D., a venture partner at Blackstone Life Sciences, Praxis’ founding investor. “We are reducing these insights to practice, to create novel medicines that could fundamentally alter the treatment path and outcomes for patients with brain disorders.”

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Reddy’s fellow founders include Chief Scientific Officer Steven Petrou, Ph.D., director of the Florey Institute of Neuroscience and Mental Health at the University of Melbourne in Australia, and David Goldstein, Ph.D., who leads the Institute for Genomic Medicine at Columbia University.

At the helm is Marcio Souza, who joined from PTC Therapeutics, where he has worn several hats since 2014, most recently serving as its chief operating officer.

Over the next year, Praxis plans to start a pivotal study of PRAX-114 in major depressive disorder and release proof-of-concept data for PRAX-944 in essential tremor. It also aims to push its earlier-stage programs into the clinic, including treatments for rare epilepsies.