What is PRAX-222?

Praxis is developing an experimental new drug for gain-of-function SCN2A mutations using an antisense oligonucleotide (ASO), currently referred to as PRAX-222. An ASO is a synthetic, short segment of genetic sequence that can be designed to selectively modulate a target gene expression to help restore its normal function. PRAX-222 has been designed to selectively decrease SCN2A gene expression, which we believe directly targets the underlying cause of gain-of-function SCN2A epilepsy and other related symptoms. The program is being developed in collaboration with Ionis Pharmaceuticals Inc. and RogCon Inc.

How does PRAX-222 work?

Mutations in SCN2A alter the sodium channel function, leading to a group of brain disorders. There have been significant advances in our understanding of how different mutations in SCN2A contribute to the different forms of brain disorders. PRAX-222 is a selective treatment called an antisense oligonucleotide, or ASO. An ASO is a synthetic, short segment of genetic sequence that can be designed to selectively modulate a target gene expression to help restore its normal function. In the case of PRAX-222, it selectively decreases SCN2A gene expression. ASO medications have been recently approved for other rare neurological conditions in children, including spinal muscular atrophy and Duchenne muscular dystrophy.1 It is our hope that PRAX-222, an investigational treatment that works in a similar way, may help children with SCN2A disorders.

Where is EMBRAVE (PRAX-222) in the drug development process?

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References

  1. Practical Neurology. FDA approves antisense oligonucleotide therapy golodirsen for Duchenne muscular dystrophy subtype. Accessed April 1, 2022. https://practicalneurology.com/news/fda-approves-antisense-oligonucleotide-therapy-for-duchenne-muscular-dystrophy-subtype