Release Date: August 13, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Praxis Precision Medicines Inc (PRAX, Financial) has made significant progress in 2024, positioning up to four programs in the registrational phase by 2025.
- The company is fully funded through several key readouts, with a strong cash position of $433.8 million, ensuring financial stability into 2027.
- The Essential3 trials for Ulixacaltamide in essential tremor are progressing well, with topline results expected later this year.
- The PRAX-628 program in epilepsy is advancing with multiple studies, including the RADIANT study, which is expected to provide important safety, PK, and efficacy information.
- The EMBOLD Study of relutrigine (PRAX-562) in pediatric patients with developmental and epileptic encephalopathies is on track, with topline results expected this quarter.
Negative Points
- Operating expenses increased to $37.8 million in Q2, up from $20.9 million in Q1, reflecting higher activity levels.
- The Essential3 trials for Ulixacaltamide may face potential delays if an interim analysis necessitates an increase in sample size.
- The company acknowledges the challenges and uncertainties associated with being the first in the essential tremor space, which could impact the success of their programs.
- The RADIANT study for PRAX-628 is not placebo-controlled, which may limit the robustness of the efficacy data compared to the POWER1 and POWER2 studies.
- The EMBOLD Study involves very severe patients, which could complicate the interpretation of efficacy and safety results.
Q & A Highlights
Q: Could you comment on the enrollment status for the Essential3 trials and the interim analysis you mentioned?
A: Enrollment is progressing as planned, and we have built in an interim analysis to potentially adjust the sample size if needed. This analysis is scheduled for Q4 2024 and aims to ensure the highest probability of success for the trial.
Q: How predictive will the RADIANT study be for the POWER1 and POWER2 studies?
A: RADIANT will provide important safety, PK, and efficacy data, which will be valuable for the POWER1 and POWER2 studies. It will help us understand the broader population and refine our approach for these subsequent trials.
Q: What are the expectations for the relutrigine data in DEE, and how does it translate to other DEEs?
A: We expect a 20-30% seizure reduction, which would be significant given the severity of the patients. This data will help us understand the broader applicability of relutrigine across other DEEs, potentially opening up a large market opportunity.
Q: Can you explain the differences in the study design for Essential3 and how it impacts your confidence in the readout?
A: Essential3 includes both a parallel group and a randomized withdrawal design. The parallel group helps us understand the heterogeneous patient population, while the randomized withdrawal design mimics real-world use, increasing our confidence in the readout.
Q: What are the next updates we can expect from the Essential3 program?
A: The next updates will include the completion of planned enrollment and the results of the interim analysis. We will communicate any decisions made based on the interim analysis, such as sample size adjustments.
Q: What is the gating factor for initiating a pivotal program for generalized epilepsy with PRAX-628?
A: The RADIANT study will inform the dose and provide initial efficacy data for generalized epilepsy. Depending on the results, we may initiate a pivotal program for generalized epilepsy in the near future.
Q: How does age and baseline severity impact seizure reduction expectations for PRAX-562?
A: Younger patients with higher seizure burdens are expected to show significant reductions, which will be crucial for their development and quality of life. This data will help us understand the broader applicability of PRAX-562 across other DEEs.
Q: What feedback did you receive from global regulators for initiating the pivotal study for Elsunersen in Brazil?
A: We have received positive feedback from Brazilian regulators and are in the process of obtaining similar feedback from European and U.S. regulators. This will allow us to expand the program globally.
Q: Why not conduct the RADIANT study exclusively with generalized epilepsy patients?
A: We wanted to understand the impact of PRAX-628 on both focal and generalized epilepsy. Depending on the results, we may initiate a separate study focused solely on generalized epilepsy.
Q: How will you prioritize focal patient enrollment between POWER1 and RADIANT?
A: There is no competition between the two studies. POWER1 will start first, followed by RADIANT, ensuring that both studies recruit efficiently without overlapping.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
