What is PRAX-562?

Praxis is currently developing an experimental medicine, called PRAX-562. It is currently in a Phase 1 healthy volunteer clinical trial. PRAX-562 is a persistent sodium current blocker being developed to treat some genetic epilepsies, such as epilepsy caused by gain-of-function mutations in SCN2A, SCN8A, and other indications. More information about PRAX-562 is coming soon.

 

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. At this time, we are in the preclinical testing stage. If the results of these tests are positive, we plan to work with regulatory authorities like the FDA to initiate clinical trials as quickly as possible in patients with gain-of-function SCN2A mutations.

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 oligoneucleotide, 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. It is our hope that PRAX-222, an investigational treatment that works in a similar way, may help children with SCN2A disorders.

 

 

Where are PRAX-222 and PRAX-562 in the drug development process?

PRAX-222 is currently in the preclinical phase of testing, meaning it is being studied in animals to determine safety, side effects, and the best doses to use. If the initial data looks promising, Praxis will work with regulatory authorities like the FDA to seek approval to initiate a clinical trial in children with gain-of-function SCN2A mutation as quickly as possible. PRAX-562 is currently in a Phase 1 healthy volunteer clinical trial.

 

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