Praxis Precision Medicines Updates and Reports Q2 2024 Financial Results

Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical firm focused on developing treatments for central nervous system (CNS) disorders, provided updates on its portfolio and financial status for the second quarter of 2024.

The company aims to have four programs at the registrational phase by 2025. Among its key initiatives, the Essential3 program, which investigates ulixacaltamide, is set for an interim analysis in Q4 2024. This analysis is part of the plan to support a New Drug Application (NDA) submission in 2025. The EMBOLD study for relutrigine (formerly PRAX-562), targeting pediatric patients with SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs), is expected to yield topline results in Q3 2024. Additionally, Praxis plans to commence the PRAX-628 Phase 2/3 POWER1 study for epilepsy in Q4 2024, with other related studies to follow, generating key data necessary for regulatory submissions.

Financially, Praxis reported $434 million in cash and investments as of June 30, 2024, a significant increase from $81.3 million at the end of 2023. This enhanced financial position was attributed to proceeds from public offerings and stock sales, offset by operational expenses. The company’s cash resources are projected to support its operations into 2027.

CEO Marcio Souza highlighted the advancement across Praxis' portfolio and the company's strategic plans to achieve regulatory milestones. He emphasized the progressing Essential3 program and its potential NDA submission in 2025. The EMBOLD study for relutrigine could pave the way for addressing more DEEs. Moreover, the PRAX-628 program is positioned to build on encouraging preliminary data, with additional studies set to start in the near future.

Key highlights and upcoming milestones for Praxis include:

1. Ulixacaltamide for Essential Tremor (ET):
- Topline results of the Essential3 Phase 3 program are expected in late 2024.
- An interim analysis for Study 1 is scheduled for Q4 2024.

2. PRAX-628 for Epilepsy:
- Initiation of four studies under the PRAX-628 ENERGY program.
- EMPOWER, an observational study, is set to begin in Q3 2024.
- RADIANT, a Phase 2 study for focal and generalized epilepsy, will start in the second half of 2024, with results expected in the first half of 2025.
- POWER1 and POWER2, Phase 2/3 studies for focal onset seizures, are expected to start in late 2024 and early 2025, respectively.

3. Relutrigine (PRAX-562) for DEEs:
- Phase 2 EMBOLD study results for SCN2A and SCN8A DEEs are anticipated in Q3 2024.
- The study evaluates safety, efficacy, and pharmacokinetics in pediatric patients.

4. Elsunersen (PRAX-222) for SCN2A Developmental Epilepsies:
- The first arm of the global confirmatory study has commenced in Brazil.
- The program is on track for advancement in the US and expansion to Europe later in 2024.

Financial results for Q2 2024 showed a net loss of $32.7 million, slightly lower than the $34.3 million loss in the same period the previous year. Research and development expenses rose to $27.3 million, primarily driven by increased spending on the Cerebrum™ platform and personnel costs, offset by decreased spending on the Solidus™ platform. General and administrative expenses also saw a modest increase to $10.6 million due to higher personnel-related costs.

Praxis continues to focus on its diverse CNS portfolio, leveraging genetic insights to develop innovative therapies for neurological disorders. With a robust pipeline and strong financial footing, the company is well-positioned to advance its programs toward regulatory approval and commercialization.

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