RARE Daily

Praxis Raises $200 Million in Public Offering

April 2, 2024

Rare Daily Staff

Praxis Precision Medicines raised $200 million through a public offering to advance its clinical stage genetic medicines pipeline for central nervous system disorders characterized by neuronal excitation-inhibition imbalance.

The company sold 3.3 million shares at a price of $56.50 and prefunded warrants to purchase up to an aggregate of 221,238 shares of common stock.

The latest offering comes after the company raised $150 million in January through a separate public offering. The activity reflects the move of biotechs to capitalize on more welcoming public markets after a few years in which they struggled to raise money.

All shares and pre-funded warrants in the offering are being offered by Praxis. In addition, Praxis has granted the underwriters a 30-day option to purchase up to 530,973 additional shares of common stock at the public offering price, less underwriting discounts and commissions.

Praxis develops its experimental candidates using two proprietary platforms—one for small molecule drugs and one for antisense oligonucleotides. Its lead small molecule candidate ulixacaltamide is in registrational enabling studies for the treatment of essential tumors.

The company is advancing 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.

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