Praxis(US:PRAX)2021-11-07 08:46Financial Data and Key Metrics Changes - As of the end of Q3 2021, Praxis had $314 million in cash and investments, down from $339 million at the end of Q2 2021, representing a quarter-over-quarter decrease of approximately $25 million [49] - Operating expenses for Q3 were approximately $44.8 million, an increase of roughly $8.3 million from the prior quarter, primarily driven by higher R&D expenses [51][52] Business Line Data and Key Metrics Changes - The company is advancing multiple CNS drug programs, with a focus on PRAX-114 for major depressive disorder (MDD) and PRAX-944 for essential tremor (ET) [10][14] - The ongoing PRAX-114 registration trial and the initiation of Phase 2 studies for PRAX-562 and PRAX-944 are key developments in the pipeline [18][24] Market Data and Key Metrics Changes - The company anticipates a catalyst-rich period with multiple clinical trial readouts expected in the coming year, including studies for PRAX-114 in PTSD and ET [19][25] - The market for treatments addressing essential tremor and PTSD is significant, with an estimated 11 million adults in the U.S. affected by PTSD alone [31] Company Strategy and Development Direction - Praxis emphasizes a foundation approach to drug development based on four pillars: validating targets through genetics, leveraging translational tools, applying rigorous development paths, and implementing patient-guided strategies [10][14] - The company aims to bring its first ASO (antisense oligonucleotide) to the clinic early next year, marking a significant milestone in its development strategy [102] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing progress of their clinical programs and the potential for significant advancements in treating CNS disorders [102] - The company is focused on maintaining a strong cash position to support its growing pipeline and operational needs into 2023 [49][50] Other Important Information - Praxis plans to host a Movement Disorder Day in December 2021 to discuss ongoing efforts in essential tremor and the potential of PRAX-944 [20][21] - The company has surpassed 100 full-time employees and moved to new headquarters in Boston, reflecting its growth and operational expansion [103] Q&A Session Summary Question: Inquiry about endpoints in the Aria and Acapella study - Management clarified that they do not plan to change the endpoints based on preclinical and clinical data, emphasizing the importance of maintaining patients on the drug for longer periods to ensure benefits [58] Question: Safety profile and sedation rates in the Aria study - Management indicated that the safety profile of PRAX-114 is well tolerated, with no significant next-day somnolence observed in trials [64] Question: Expectations for upcoming open label data from PRAX-944 - Management expects to present data focusing on upper limb tremor, with a goal of achieving similar or improved results compared to previous trials [67] Question: Powering assumptions for the Acapella study - Management explained that Acapella is not powered for accuracy but aims to explore lower doses that may generate similar effects with fewer side effects [73] Question: Nausea rates in the faster titration Phase 1 study for PRAX-944 - Management reported that nausea rates were similar across different titration schedules, indicating a consistent safety profile [79] Question: Rationale for flexed dosing in the PTSD study - Management stated that the flexed dosing approach allows for better understanding of patient responses and tolerability, which is crucial for developing effective treatment strategies [98]