RARE Daily

Praxis Precision Medicines Launches with More Than $100 Million to Target Brain Disorders

May 4, 2020

Rare Daily Staff

Praxis Precision Medicines launched with more than $100 million to develop a clinical stage pipeline of therapies that target the underlying causal mechanisms of both prevalent and rare brain disorders with overlapping disease biology.

Founding investor Blackstone Life Sciences (via prior Clarus funds in 2016) led the financing with participation by Novo Holdings, Vida Ventures, Eventide, and other prominent funds.

“Praxis aims to leverage the recent breakthroughs in genetics to develop innovative medicines that can improve the lives of the many patients who need them,” said Nicholas Galakatos, chairman of the Praxis board of directors and global head of Blackstone Life Sciences.

Praxis is leveraging recent discoveries in the genetics of epilepsy, which have elucidated genes that when dysregulated, drive a range of neuropsychiatric and movement disorders. Using these insights, Praxis is rapidly advancing a pipeline of treatments that specifically address genes controlling the imbalance of excitation and inhibition of neuronal circuitry at the core of multiple CNS disorders.

“Neurology and psychiatry are finally primed for a revolution in how new therapies are discovered and developed, and Praxis’ approach has the potential to change the treatment landscape,” said Marcio Souza, president and chief executive officer of Praxis.

The company’s lead drugs in development include PRAX-114, a GABAA positive allosteric modulator in phase 2 development for the treatment of major depressive disorder and perimenopausal depression, and PRAX-944, a T-type calcium channel blocker in phase 2 development for the treatment of essential tremor.

Within the next year, Praxis plans to initiate its first pivotal trial for PRAX-114 in major depressive disorders, report proof-of-concept data for PRAX-944 in essential tremor, and advance its earlier stage programs into clinical development for rare epilepsies and other neurological disorders with genetically validated mechanisms.

“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, co-founder and member of Praxis’ board of directors. “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.”

Praxis’ three founders bring pedigree as scientists and clinicians. Reddy is a neurologist and venture partner at Blackstone Life Sciences, and previously on the corporate strategy leadership team at Biogen.

David Goldstein is the director of The Institute for Genomic Medicine, and professor in the department of Genetics and Development, at Columbia University. His work focuses on broad aspects of precision medicine and includes the Epi4K consortium that discovered and characterized novel epilepsy genes.

Steven Petrou, chief scientific officer at Praxis, is the director of the Florey Institute of Neuroscience and Mental Health, head of the Department of the Florey Institute at the University of Melbourne in Australia. Petrou is a recognized leader in the field of ion channel neuropathies in rare pediatric epilepsies and other neurodevelopmental disorders.

Souza was at PTC Therapeutics, most recently serving as chief operating officer, prior to joining Praxis. He also served in leadership roles in the U.S. and globally at NPS Pharmaceuticals, Shire and Genzyme.

Photo: Marcio Souza, CEO of Praxis

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