Rapport Therapeutics Conference Call Summary Company Overview - Rapport Therapeutics is focused on precision neuroscience, leveraging receptor-associated proteins to enhance treatment efficacy and tolerability for patients [2][3] Key Programs - The lead program, RAP-219, is a TARP gamma-8 AMPA modulator, showing promising results in epilepsy and ongoing programs in bipolar mania [2][3] - RAP-219 has demonstrated a 78% median reduction in clinical seizure frequency and a 24% seizure freedom rate in a proof of concept study [11][12] FDA Engagement and Study Plans - The company plans to meet with the FDA by the end of 2025 for an end of phase two meeting, aiming to start phase three studies by Q3 2026 [4][5] - Two parallel registrational studies are planned, following a standard protocol for focal epilepsy [4][5] Safety and Efficacy Insights - The biological thesis focuses on modulating the AMPA receptor through TARP gamma-8, which is expressed in key brain structures related to focal seizures [6][8] - The tolerability profile from phase two data showed a 10% discontinuation rate, indicating best-in-class tolerability for anti-seizure medications [9] - Concerns regarding adverse events (AEs) related to psychiatric disorders were addressed, with no significant findings in their trials [10] Patient Population and Efficacy Expectations - The proof of concept study involved a highly refractory patient population, with 70% on three or four anti-seizure medications [16] - The efficacy results are expected to translate well into the broader phase three patient population, despite potential variability [18][13] Bipolar Mania and Other Indications - The company is exploring RAP-219 for bipolar mania, supported by the drug's mechanism targeting excitatory processes in the limbic structure [24][26] - Enrollment for the bipolar trial is progressing well, with data expected in 2027 [26] Market Opportunity - The potential market opportunity for RAP-219 in epilepsy is estimated to be multi-billion dollars, with expected utilization similar to existing treatments like Keppra [29] Upcoming Milestones - Key updates expected include the end of phase two meeting with the FDA, additional data analyses at AES in December 2025, and the initiation of phase three studies in 2026 [30][31] - The company has sufficient cash reserves, approximately $500 million, to fund operations through 2029 [32]

Core Viewpoint - Rapport Therapeutics, Inc. (RAPP) shows significant upside potential with a mean price target of $52.14, indicating a 104.5% increase from the current trading price of $25.5 [1] Price Targets and Analyst Consensus - The average price target consists of seven estimates ranging from a low of $34.00 to a high of $80.00, with a standard deviation of $14.12, suggesting variability in analyst predictions [2] - The lowest estimate indicates a 33.3% increase, while the highest suggests a 213.7% upside [2] - A low standard deviation indicates strong agreement among analysts regarding the stock's price movement direction [9] Earnings Estimates and Analyst Optimism - Analysts have shown increasing optimism about RAPP's earnings prospects, as evidenced by a positive trend in earnings estimate revisions [11] - Over the last 30 days, the Zacks Consensus Estimate for the current year has increased by 5.2%, with three estimates moving higher and no negative revisions [12] - RAPP holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimates [13] Caution on Price Targets - While price targets are a common tool for investors, they can often mislead and should not be the sole basis for investment decisions [7][10] - Analysts may set overly optimistic price targets due to business incentives, which can inflate expectations [8]

Financial Performance - The company has not generated any revenue from product sales since its inception in February 2022 and has incurred significant operating losses, with net losses of $26.9 million and $17.5 million for the three months ended September 30, 2025 and 2024, respectively[146][150]. - The net loss for the three months ended September 30, 2025, was $26.925 million, compared to a net loss of $17.537 million for the same period in 2024, indicating a $9.388 million increase in losses[177]. - Net loss for the nine months ended September 30, 2025, was $77.7 million, compared to a net loss of $58.3 million in 2024, an increase of $19.4 million[183]. - Interest income decreased to $3.061 million for the three months ended September 30, 2025, down from $4.103 million in 2024, a decline of $1.042 million[177]. - Other income decreased to $3.1 million for the three months ended September 30, 2025, down from $4.1 million in 2024, primarily due to reduced interest income from lower cash and investment balances[179]. Cash and Financing - As of September 30, 2025, the company had raised aggregate gross proceeds of $711.9 million from financings and had cash, cash equivalents, and short-term investments of $513.0 million, excluding restricted cash[149]. - The company completed an underwritten public offering in September 2025, raising approximately $269.4 million in net proceeds after deducting offering costs[149]. - Net cash provided by financing activities was $270.5 million for the nine months ended September 30, 2025, primarily from the September 2025 Offering[195]. - The company believes its existing cash and cash equivalents will fund operations into the second half of 2029[153]. - The company expects its cash and cash equivalents will be sufficient to fund operating expenses and capital expenditures for at least the next 12 months[197]. Research and Development - The company plans to initiate an open-label long-term safety trial for RAP-219 by the end of 2025, with preliminary results expected in the second half of 2026[142]. - The Phase 2a trial of RAP-219 in drug-resistant focal onset seizures met its primary and secondary endpoints, demonstrating a statistically significant reduction in clinical seizures compared to baseline[142]. - The company is developing a long-acting injectable formulation of RAP-219 to improve patient adherence and expand clinical utility across indications[144]. - The Investigational New Drug application for a Phase 2 trial of RAP-219 for diabetic peripheral neuropathic pain is on clinical hold pending additional information requested by the FDA[143]. - Research and development expenses increased to $22.3 million for the three months ended September 30, 2025, compared to $15.5 million for the same period in 2024, representing a $6.7 million increase[177]. - The increase in research and development expenses included a $3.1 million rise in RAP-219 program costs, driven by clinical trial costs for the Phase 2a trial and open-label long-term safety trial[178]. - The company expects research and development expenses to increase substantially as it advances RAP-219 through clinical development and pursues regulatory approval[164]. - Research and development expenses rose to $64.5 million for the nine months ended September 30, 2025, compared to $43.7 million in 2024, marking an increase of $20.8 million[184]. - The RAP-219 program costs increased by $12.0 million, driven mainly by clinical trial costs for various Phase 2 trials[184]. Operating Expenses - Total operating expenses for the three months ended September 30, 2025, were $29.986 million, up from $21.640 million in 2024, reflecting an increase of $8.346 million[177]. - Total operating expenses for the nine months ended September 30, 2025, were $86.6 million, up from $59.5 million in 2024, reflecting a $27.1 million increase[183]. - General and administrative expenses increased to $7.7 million for the three months ended September 30, 2025, up from $6.1 million in the same period of 2024, representing a $1.6 million increase[179]. - The company anticipates that general and administrative expenses will increase due to hiring additional personnel and compliance-related costs associated with being a public company[170]. Collaborations and Agreements - The company has a collaboration agreement with Janssen Pharmaceutical NV, which includes potential milestone payments of up to $76.0 million for development and $40.0 million for sales of TARPg8 products[157]. - The company made a non-refundable payment of $1.0 million to Janssen and an additional option fee of $4.0 million, with potential milestone payments totaling up to $141.0 million for product development and sales[203]. - The company has entered into a master services agreement with NeuroPace Inc., which includes a payment of up to $5.3 million over approximately four years for services related to the RAP-219 clinical trials[161]. - Cumulative expenses incurred under the NeuroPace agreement amount to $3.4 million through September 30, 2025, with a total payment commitment of up to $5.3 million over four years[204]. Future Outlook and Risks - The company expects its expenses and operating losses to increase substantially as it continues clinical trials and research activities[151]. - The company anticipates substantial increases in expenses related to ongoing preclinical studies and clinical trials, with funding needs dependent on various factors[199]. - The company may need to raise additional capital through equity offerings or debt financings, which could dilute ownership interests and impose restrictions on operations[199]. - Future minimum operating lease payments under non-cancelable leases amount to $14.0 million, with a weighted average remaining lease term of 4.8 years[200][202]. - The company has recorded a full valuation allowance against its net deferred tax assets, indicating uncertainty about the realization of these assets[175]. - As of December 31, 2024, the company had federal net operating loss carryforwards of approximately $13.7 million and state net operating loss carryforwards of approximately $12.0 million[174]. Accounting and Compliance - The company has not experienced material changes to its critical accounting policies and estimates during the nine months ended September 30, 2025[207]. - The company has elected not to "opt out" of the extended transition period for new accounting standards, which may affect comparability with other public companies[208][209]. - The company is classified as a smaller reporting company and is not required to provide certain market risk disclosures[211].

Financial Performance - Rapport Therapeutics reported a net loss of $26.9 million for Q3 2025, compared to a net loss of $17.5 million in the same period last year, representing a 53.4% increase in losses[12] - General and Administrative (G&A) expenses increased to $7.7 million in Q3 2025 from $6.1 million in Q3 2024, reflecting a 26.2% increase[17] - Net cash used in operating activities increased to $17.465 million in Q3 2025 from $16.415 million in Q3 2024[26] - Net cash used in investing activities rose to $56.776 million in Q3 2025 compared to $53.041 million in Q3 2024[26] - Net cash provided by financing activities significantly increased to $270.533 million in Q3 2025, contrasting with a cash outflow of $1.394 million in Q3 2024[26] - The net increase in cash, cash equivalents, and restricted cash was $196.292 million in Q3 2025, compared to a decrease of $70.850 million in Q3 2024[26] Research and Development - Research and Development (R&D) expenses for Q3 2025 were $22.3 million, up from $15.5 million in Q3 2024, indicating a 43.5% increase year-over-year[12] - The Phase 2a clinical trial of RAP-219 showed a 77.8% median reduction in clinical seizures, with 24% of patients achieving seizure freedom during the 8-week treatment period[6] - Approximately 85.2% of patients in the Phase 2a trial achieved a ≥30% reduction in long episodes of seizures compared to baseline, with a p-value of <0.0001[6] - The company expects to initiate two Phase 3 pivotal trials of RAP-219 in focal onset seizures in Q3 2026[11] - The Phase 2 trial of RAP-219 in bipolar mania is currently enrolling patients, with topline results expected in the first half of 2027[7] - The company plans to develop a long-acting injectable formulation of RAP-219, with initial pharmacokinetics results expected in 2027[11] Capital and Funding - The company completed a public offering in September 2025, raising approximately $269.4 million in net proceeds[9]

Core Insights - Rapport Therapeutics reported positive Phase 2a results for RAP-219, indicating a potential best-in-class profile for treating drug-resistant focal onset seizures with a nearly 78% median reduction in clinical seizures and seizure freedom in nearly 24% of patients [2][5] - The company has a strong financial position with approximately $269.4 million in net proceeds from a recent public offering, ending the quarter with $513.0 million in cash, which is expected to fund operations into the second half of 2029 [4][11] Business Highlights - The Phase 2a clinical trial of RAP-219 met its primary endpoints with high statistical significance, showing 85.2% of patients achieving a ≥30% reduction in long episodes [5] - The company plans to initiate two Phase 3 pivotal trials of RAP-219 in focal onset seizures in Q3 2026 and expects topline results from the bipolar mania trial in the first half of 2027 [4][6] - An open-label long-term safety trial for patients from the Phase 2a trial is planned to begin by the end of 2025 [5] Financial Results - For Q3 2025, the company reported a net loss of $26.9 million, compared to a net loss of $17.5 million in the same period last year [11] - Research and Development (R&D) expenses increased to $22.3 million from $15.5 million year-over-year, driven by clinical development costs [11] - General and Administrative (G&A) expenses rose to $7.7 million from $6.1 million, reflecting business growth [11] Cash Position - The company ended Q3 2025 with $513.0 million in cash, cash equivalents, and short-term investments, a significant increase from $260.4 million as of June 30, 2025 [11] - The cash runway is expected to support operations and capital expenditures into the second half of 2029 [11]

Core Insights - Rapport has acquired Aquifer Motion, marking a significant advancement in the field of AI technology by becoming the first company to provide real-time, AI-powered avatars with full-body animation [1] Company Summary - The acquisition positions Rapport as a leader in the AI avatar market, enhancing its product offerings and technological capabilities [1] - The integration of Aquifer Motion's technology is expected to drive innovation and expand the potential applications of AI avatars across various industries [1] Industry Summary - The development of real-time, AI-powered avatars represents a growing trend in the technology sector, indicating increased demand for advanced animation and virtual interaction solutions [1] - This acquisition highlights the competitive landscape of the AI industry, where companies are increasingly seeking to differentiate themselves through unique technological advancements [1]

Group 1 - Rapport Therapeutics, Inc. (NASDAQ:RAPP) is considered one of the best fast money stocks to buy, with JMP Securities reaffirming a 'Market Outperform' rating and a price target of $77, indicating a potential increase of approximately 174% [1] - The company has outperformed the market by 45% year-to-date, with a significant market of 1.8 million patients in the U.S., 40% of whom are treatment-resistant [2] - The leadership expressed enthusiasm about RAP-219 during a presentation, viewing it as a multibillion-dollar market opportunity, supported by its novel mechanism of action and best-in-class efficacy [3] Group 2 - Rapport Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing therapies for central nervous system disorders, and was incorporated in 2022 [4]

Core Insights - Several clinical-stage biotech companies experienced significant after-hours trading momentum due to anticipated trial data releases and pipeline updates [1] Company Summaries - **MBX Biosciences Inc. (MBX)**: Shares surged 33% in after-hours trading, rising from $10.00 to $13.77, following the announcement of topline results from its Phase 2 trial of Canvuparatide, expected on September 22 [2][3] - **Structure Therapeutics Inc. (GPCR)**: Stock increased 11.37% to $26.26 after closing at $23.58, driven by interest in its lead candidate aleniglipron, with topline results from two Phase 2b trials expected by the end of 2025 [4] - **Cartesian Therapeutics Inc. (RNAC)**: Shares rose 5.51% to $9.96 after a decline during the day, with preliminary data from the Phase 2 trial of Descartes-08 expected in the second half of 2025 [5][6] - **Rapport Therapeutics Inc. (RAPP)**: Stock increased 3.60% to $27.31, following positive topline results from its Phase 2a trial for RAP-219, with Phase 3 trials expected to start in 2026 [9][10] - **Rezolute Inc. (RZLT)**: Shares rose 3.77% to $7.99 after a decline during the day, with topline data from the Phase 3 sunRIZE trial for congenital hyperinsulinism expected in December 2025 [11][12]

Core Insights - The company is excited about the recent Phase II data readout, which is significant for patients and indicates a positive outcome for their treatment approach [1][2]. Company Overview - The CEO expressed gratitude for the opportunity to present the company's story and recent data, highlighting the importance of the findings [2]. Market Context - The market for the company's treatment is substantial, with approximately 1.8 million patients in the U.S. affected, of which around 40% (approximately 560,000 patients) are treatment-resistant [3].

Rapport Therapeutics Conference Call Summary Company Overview - **Company**: Rapport Therapeutics (NasdaqGM:RAPP) - **Event**: FY Conference on September 17, 2025 Industry Context - **Industry**: Neuropsychiatry and Epilepsy - **Market Size**: Approximately 1.8 million patients in the U.S. with 560,000 treatment-resistant patients [2][27] Key Points and Arguments Phase 2 Trial Results - **Efficacy**: The Phase 2 trial demonstrated a median reduction of 78% in clinical seizures and a 24% seizure freedom rate, indicating a best-in-class profile for the anti-seizure medication RAP-219 [5][26] - **Patient Demographics**: The trial enrolled treatment-resistant focal epilepsy patients, with 70% on three or four anti-seizure medications, confirming the refractory nature of the population [7][8] - **Trial Design**: The trial utilized a novel design informed by the epilepsy community, leveraging an electrographic biomarker to predict clinical seizure reductions [4][9] Next Steps - **Phase 3 Plans**: Rapport Therapeutics plans to initiate two parallel Phase 3 studies in Q3 2026, following an end of Phase 2 meeting with the FDA [10][36] - **Translatability**: The results from the Phase 2 trial are believed to be highly translatable to registrational studies, improving the probability of success [6][9] Safety and Tolerability - **Tolerability Profile**: The trial reported a 10% discontinuation rate, one of the lowest in proof of concept studies for this patient population, with no severe adverse events greater than grade two [18][24] - **Comparison with Other Drugs**: RAP-219 showed a differentiated tolerability profile compared to Fycompa, with significantly lower rates of sedation and aggression [19][22] Commercial Potential - **Market Opportunity**: The peak sales opportunity for RAP-219 has shifted from an estimated $1.5 billion to potentially much higher, with expectations of broad adoption by general neurologists and internists [26][27] - **Long-Acting Injectable**: There is potential for developing a long-acting injectable formulation, which could transform patient management in epilepsy [29][30] Future Indications - **Bipolar and Pain**: The Phase 2 results have raised the probability of success for other indications like bipolar mania, due to confirmed target engagement and a favorable tolerability profile [39][40] Financial Position - **Cash Balance**: As of Q2, the company had $260 million, sufficient to fund operations through the end of 2029, including the initiation of Phase 3 trials and development of a long-acting injectable [37][38] Additional Important Insights - **Community Feedback**: The company has received positive feedback from the epilepsy community regarding the drug's profile and potential for adoption [26][28] - **Enrollment Strategy**: The company is focused on enrolling the right patient population for Phase 3 studies, considering competitive context and drug profile [32][33] This summary encapsulates the critical insights from the conference call, highlighting the promising developments for Rapport Therapeutics and its lead candidate RAP-219 in the epilepsy market.