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Rapport Therapeutics (RAPP) FY Conference Transcript
2025-06-09 15:00
Rapport Therapeutics (RAPP) FY Conference Summary Company Overview - **Company**: Rapport Therapeutics (RAPP) - **Industry**: Biotechnology, specifically focusing on epilepsy and related neurological disorders Key Points and Arguments Clinical Development Progress - Rapport has made significant progress since going public a year ago, focusing on the clinical development of its lead program, RAP 219 [2][3] - Completed phase one studies, including multiple ascending dose studies and a human PET study, which have increased confidence in the pharmacology and specificity of RAP 219 [3][27] - Currently conducting a phase 2a proof of concept study in refractory focal epilepsy patients, utilizing an implantable neurostimulation device for diagnostics [3][6] Study Design and Rationale - The study design aims to produce compelling proof of concept results that are translatable to a phase three registration setting [5] - The study leverages an EEG biomarker correlated with clinical seizures, addressing the limitations of traditional clinical seizure diaries [6][9] - The trial includes an eight-week retrospective data collection period followed by a 28-day prospective period and an eight-week treatment phase [7][31] Data Expectations and Efficacy Measures - Data readout is expected in September, with a focus on achieving a 30% reduction in long episodes for 40% of participants, correlating to a 50% reduction in clinical seizures [19][22] - The study is designed to ensure a robust set of baseline characteristics, with a median clinical seizure count of 10, which positions the study favorably compared to other trials [48] Pipeline and Future Development - Rapport is also developing RAP 219 for bipolar disorder and neuropathic pain, with plans to initiate a bipolar study in Q3 [34][36] - The neuropathic pain program is currently on clinical hold due to requested protocol changes by the FDA [36][38] - A long-acting injectable formulation of RAP 219 is being explored, which could transform treatment for epilepsy patients [42][45] Financial Position - The company reported a cash position of $285 million, sufficient to fund activities through at least the end of 2026, covering the upcoming phase three trials and ongoing bipolar studies [51] Risks and Confidence - Confidence in the upcoming data readout is supported by a robust preclinical package and a high concordance rate between long episodes and electrographic seizures [47][49] - The company is optimistic about the translatability of the phase 2a results to phase three studies, with plans for an end-of-phase two meeting with the FDA following positive results [24][33] Additional Important Insights - The study design has received positive feedback from the epilepsy community, highlighting its objective analysis capabilities [9][17] - The long half-life of RAP 219 (8-14 days) is seen as beneficial for patient compliance, reducing the risk of breakthrough seizures [32] - The company aims to differentiate RAP 219 from existing treatments through its efficacy, dosing flexibility, and lack of sedation [23][27]
Rapport Therapeutics (RAPP) 2025 Conference Transcript
2025-05-08 18:30
Summary of Rapport Therapeutics Conference Call Company Overview - **Company**: Rapport Therapeutics - **Focus**: Developing precision neuroscience therapeutics, particularly for focal epilepsy with lead program RAP219 [1][2] - **Background**: Company spun out from technology developed by Dr. David Brett, with a history at UCSF and Eli Lilly [3] Key Points Financials and Development Timeline - **Public Offering**: Company went public in June of the previous year [3] - **Cash Position**: Ended the year with approximately $285 million, sufficient to fund operations through at least the end of the next year [4] - **Upcoming Milestones**: - Phase two trial readout for focal onset patients expected in Q3 [4] - Initiation of bipolar mania trial in Q3 with data expected in early 2027 [4] Scientific Approach - **Technology**: Focus on receptor-associated protein technology to target specific areas in the brain, enhancing drug precision [3] - **Lead Program**: RAP219 targets AMPA receptors through the accessory protein gamma-eight TARP, aiming to reduce side effects associated with non-selective targeting [5][8] - **Preclinical and Phase One Data**: - Strong preclinical data supporting the targeting approach, with a focus on receptor occupancy and therapeutic index [14][15] - Phase one PET study confirmed receptor occupancy exceeding 80% at therapeutic concentrations [15][17] Clinical Trial Design - **Innovative Trial Design**: - Utilizes an RNS device to track long episodes of epileptic activity, providing objective data [20][25] - Aims to correlate long episode frequency with clinical seizure frequency, with a target of a 30% decrease in long episodes correlating with a 50% decrease in clinical seizures [34] - **Success Metrics**: - Aiming for a responder rate of 40% or greater in the trial participants [36] - Combination of clinical seizure diary data and objective electrographic biomarkers to validate findings [39] Market Opportunity - **Focal Epilepsy Market**: Approximately 1.8 million patients with focal epilepsy, with 40% still experiencing seizures, indicating a significant market opportunity for new treatments [49] - **Pipeline Potential**: Plans to initiate a phase two study in bipolar disorder, leveraging the mechanism of action that targets hyperactivity in the limbic system [50][51] Additional Insights - **Community Engagement**: The trial design was influenced by feedback from the medical community, emphasizing the need for more efficient and predictive drug development methods [21][30] - **Regulatory Perspective**: The FDA is seen as potentially supportive of the innovative trial design, although it remains early in the process [45] This summary encapsulates the key aspects of the conference call, highlighting the company's strategic direction, scientific innovations, and market potential.