Longboard Pharmaceuticals(US:LBPH)2024-08-04 17:39Financial Data and Key Metrics Changes - The company ended Q2 2024 with approximately $305 million in cash and investments, reflecting a cash burn of about $19 million for the quarter [21] - Q2 expenses were higher than Q1 due to increased R&D activities primarily related to Bexicaserincaserin and a smaller amount for LP659 [21][22] Business Line Data and Key Metrics Changes - Bexicaserincaserin achieved a median reduction of countable motor seizures of approximately 60% in highly refractory participants [5] - The PACIFIC study demonstrated a sustained response over an approximate six-month period with continued favorable safety and tolerability [6][7] Market Data and Key Metrics Changes - The market for S1P receptor modulators is substantial, with existing treatments generating significant revenue in CNS applications, primarily in multiple sclerosis [9] - The company is preparing to initiate its Phase 3 program for Bexicaserincaserin, which will include pivotal studies for Dravet syndrome and other DEEs [7] Company Strategy and Development Direction - The company is committed to addressing the unmet medical needs for patients with DEEs and is moving forward with a global Phase 3 program [6][7] - LP659 is positioned as a next-generation S1P receptor modulator targeting CNS-related diseases with greater precision [14] Management's Comments on Operating Environment and Future Outlook - Management expressed excitement about the breakthrough therapy designation for Bexicaserincaserin, indicating a significant opportunity to address a large patient population [5][6] - The company plans to continue building its organization and infrastructure to support the global Phase 3 program for Bexicaserincaserin [22] Other Important Information - The Phase 1 SAD trial for LP659 demonstrated a favorable safety profile, with no serious adverse events reported [17] - The company is evaluating the risk-reward of additional studies for both programs to ensure efficient use of resources [23] Q&A Session Summary Question: Can you expand on the sizing of the Bexicaserincaserin pivotal programs? - Management indicated that they will provide a full disclosure of the plans during the R&D day scheduled for September 16 [25] Question: What is the market opportunity for DEEs? - Management believes the market is very large and is excited about the opportunity to address patients who lack access to the latest clinical trials and standard of care [27] Question: What formulations will be tested in the MAD study for LP659? - Management confirmed that formulation two is the path forward for the MAD study, as it shows better plasma absorption and less variability [28] Question: What is the status of discussions with global regulators regarding DEE? - Management stated that discussions with global regulatory authorities have been initiated and are expected to proceed positively [31] Question: What is the overall safety profile of LP659 compared to other S1P molecules? - Management noted that no abnormalities were observed in other organ systems typically seen with S1P modulators, but monitoring will continue [42]