Praxis Raises $150 Million to Advance Clinical Pipeline of Rare CNS Disease Therapeutics

Rare Daily Staff

Praxis Precision Medicines raised $150 million in an underwritten public offering to advance its clinical stage genetic medicines pipeline for central nervous system disorders characterized by neuronal excitation-inhibition imbalance.

The company priced 3.2 million shares of its common stock at $35.50 a share and, in lieu of shares of common stock, pre-funded warrants to purchase up to an aggregate of one million shares of common stock at a purchase price of $35.4999 per pre-funded warrant, which equals the public offering price per share of the common stock less the $0.0001 per share exercise price of each pre-funded warrant. The gross proceeds from the offering are expected to be approximately $150 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by Praxis. In addition, Praxis has granted the underwriters a 30-day option to purchase up to 633,750 additional shares of common stock at the public offering price, less the underwriting discount and commission.

Praxis develops its investigational candidates using two proprietary platforms—one for small molecule drugs and one for antisense oligonucleotides (ASOs).

The company is currently completing regulatory interactions to advance lead ASO candidate PRAX-222 towards a pivotal study for the treatment of SCN2A gain-of-function developmental epilepsies (SCN2A-DEE). SCN2A related disorders are a group of epilepsy and neurodevelopmental disorders, each caused by mutations in a gene called SCN2A. Children with SCN2A-DEE often experience seizures that start early in infancy, sometimes even shortly after birth. These seizures may be difficult to control with anti-seizure medications. Children with SCN2A-developmental and epileptic encephalopathy often have delays in reaching developmental milestones and may have several types of seizures during their disease course.

PRAX-222 is designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early-seizure-onset SCN2A-DEE to treat seizures and other symptoms in patients with gain-of-function SCN2A mutations. In vitro studies of PRAX-222 have demonstrated reduction in both SCN2A gene expression and protein levels.

In vivo, PRAX-222 has demonstrated significant, dose-dependent reduction in seizures, improvement in behavioral and locomotor activity and increased survival in SCN2A mouse models, with potential to be the first disease-modifying treatment. The PRAX-222 program is ongoing under a collaboration with Ionis Pharmaceutics and RogCon. Praxis expects topline results from the EMBOLD study in pediatric patients with SCN2A-DEE and SCN8A-DEE in the first half of 2024.