Praxis Precision Medicines Q3 2024 Update and Financial Results

Praxis Precision Medicines, Inc. released its third-quarter 2024 financial results and provided updates on its pipeline advancements. The company, which focuses on developing therapies for central nervous system (CNS) disorders, reported significant progress in several of its key programs.

One of the highlights was their molecule ulixacaltamide, which is being developed for the treatment of essential tremor (ET). Praxis has confirmed that interim analysis for Study 1 of the Essential3 Phase 3 program will be completed by the first quarter of 2025. The company plans to file a New Drug Application (NDA) in 2025 based on these findings. Ulixacaltamide is seen as a promising treatment option due to its potential to address an unmet need in ET, a condition where 85% of patients see their neurologists primarily to seek new treatments, according to recent surveys.

In the realm of epilepsy, Praxis is making strides with vormatrigine (PRAX-628) which is undergoing studies for both focal onset seizures and generalized epilepsy. The POWER1 study in focal epilepsy and the RADIANT study in generalized epilepsy are scheduled for topline results in 2025. The company is also considering expanding the use of vormatrigine to treat pain, given its potent inhibition of Nav 1.7 and 1.8 channels.

Praxis also reported promising results for relutrigine (PRAX-562) in developmental and epileptic encephalopathies (DEEs). The Phase 2 EMBOLD study showed that patients experienced substantial seizure reduction, with some achieving complete seizure freedom. These results have prompted the initiation of a second cohort in the registrational EMBOLD study, aiming to enroll 80 patients. This expansion is expected to bolster the data required for regulatory approval.

Elsunersen (PRAX-222) is another significant candidate in Praxis' pipeline, targeting early-seizure-onset SCN2A developmental epilepsies. The company has started dosing patients in Brazil as part of the EMBRAVE study and is working towards expanding the trial to include the U.S. and Europe.

Financially, Praxis reported having $411.2 million in cash and marketable securities as of September 30, 2024, a significant increase from $81.3 million at the end of 2023. This boost primarily stems from public offerings and sales of common stock earlier in the year. However, the company also noted increased expenditures in research and development, which totaled $41.9 million for the quarter, a rise attributed to the expansion of their Cerebrum™ platform.

The company’s general and administrative expenses were also higher, reaching $15.3 million, primarily due to increased personnel and professional expenses. As a result, Praxis reported a net loss of $51.9 million for the quarter.

Praxis remains committed to advancing its diverse portfolio of CNS therapies. The company is leveraging genetic insights to drive the discovery and development of treatments for both rare and more common neurological disorders. With four clinical-stage products and a robust pipeline, Praxis is well-positioned to continue its progress in the coming years, with a financial runway extending into 2027.