Core Insights - Rapport Therapeutics (RAPP) shares increased by 61.6% within a week following the announcement of positive top-line data from a mid-stage study of its lead candidate, RAP-219, for drug-resistant focal onset seizures (FOS) [1][6] Study Details - The phase IIa RAP-219-FOS-201 study evaluated the efficacy, safety, and tolerability of RAP-219 in adults with drug-resistant FOS, enrolling thirty patients who received escalating doses over an eight-week treatment period [2][3] - The study met its primary endpoint, demonstrating a statistically significant reduction in long episodes (LEs) associated with seizure activity compared to baseline [3] Efficacy Results - During the eight-week treatment, 85.2% of patients experienced a ≥30% reduction in LEs, 72% achieved a ≥50% reduction in clinical seizures, and 24% attained seizure freedom [4] - RAP-219 was well-tolerated, with most adverse events being mild to moderate in severity, supporting its potential as a new anti-seizure medication [4] Future Plans - The company plans to engage with the FDA in late 2025 to discuss the phase IIa study results and aims to initiate pivotal studies for RAP-219 in Q3 2026 [8] - A long-term safety study of RAP-219 is set to launch by the end of 2025, allowing patients from the initial study to continue treatment, with preliminary data expected in H2 2026 [9] Broader Development - Rapport Therapeutics is expanding RAP-219's clinical program to include additional indications, with a phase II study for bipolar mania currently underway and results anticipated in H1 2027 [10]

Core Insights - Rapport Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing small molecule precision medicines for neurological and psychiatric disorders [3] - The company will participate in the TD Cowen's 5th Annual Novel Mechanisms in Neuropsychiatry & Epilepsy Summit on September 17, 2025 [2] Company Overview - Rapport Therapeutics is dedicated to discovering and developing precision medicines, leveraging its RAP technology platform based on pioneering discoveries related to receptor associated proteins (RAPs) in the brain [3] - The lead investigational drug, RAP-219, targets specific regions of the brain and is being pursued for drug-resistant focal onset seizures, bipolar mania, and diabetic peripheral neuropathic pain [3] - The company is also engaged in preclinical and late-stage discovery programs targeting chronic pain and hearing disorders [3]

Core Insights - Rapport stock (NASDAQ: RAPP) experienced a significant decline of 18% on Wednesday, indicating a negative market reaction to its latest funding strategy [1] Company Summary - The market's response to Rapport's funding strategy was both predictable and painful, suggesting that investors had anticipated potential issues with the approach taken by the company [1]

Core Viewpoint - Rapport Therapeutics, Inc. has announced a public offering of 9,615,385 shares at a price of $26.00 per share, aiming to raise approximately $250 million in gross proceeds before expenses [1][3]. Company Overview - Rapport Therapeutics is a clinical-stage biotechnology company focused on developing small molecule precision medicines for neurological and psychiatric disorders [5]. - The company utilizes a RAP technology platform based on pioneering discoveries related to receptor associated proteins (RAPs) in the brain, which allows for a differentiated approach in drug discovery [5]. - The lead investigational drug, RAP-219, targets specific regions of the brain and is being pursued for conditions such as drug-resistant focal onset seizures, bipolar mania, and diabetic peripheral neuropathic pain [5]. Offering Details - The offering includes a 30-day option for underwriters to purchase an additional 1,442,307 shares at the public offering price [1]. - The offering is expected to close around September 11, 2025, pending customary closing conditions [1]. - Goldman Sachs, Jefferies, TD Cowen, and Stifel are acting as joint book-running managers for the offering [2].

Core Viewpoint - Rapport Therapeutics, Inc. has initiated an underwritten public offering of $250 million in common stock, with an additional option for underwriters to purchase up to $37.5 million more [1][2]. Company Overview - Rapport Therapeutics is a clinical-stage biotechnology company focused on developing small molecule precision medicines for neurological and psychiatric disorders [5]. - The company utilizes a RAP technology platform based on pioneering discoveries related to receptor associated proteins (RAPs) in the brain, aiming to create precision small molecule candidates that address limitations in conventional neurology drug discovery [5]. - The lead investigational drug, RAP-219, targets specific regions of the brain and is being pursued for conditions such as drug-resistant focal onset seizures, bipolar mania, and diabetic peripheral neuropathic pain [5]. Offering Details - The public offering is subject to market conditions, and there is no guarantee regarding its completion or the final terms [1]. - The offering is conducted under an effective shelf registration statement filed with the U.S. Securities and Exchange Commission (SEC) [3].

Core Insights - Rapport stock (NASDAQ: RAPP) experienced a significant surge, climbing nearly 200% in a single trading session [1] - The increase in stock price was driven by excitement surrounding upcoming clinical trial data and a growing confidence in the company's prospects [1] Company Summary - The stock's dramatic rise indicates strong market interest and investor sentiment towards Rapport, likely influenced by anticipated positive outcomes from clinical trials [1] - The confidence in the company's future performance suggests potential for further growth and investment opportunities in the biotech sector [1]

Rapport Therapeutics Conference Call Summary Company Overview - **Company**: Rapport Therapeutics (NasdaqGM:RAPP) - **Focus**: Development of RAP-219 for focal onset seizures and other indications Key Industry Insights - **Market Need**: Despite over 30 approved anti-seizure medications, up to 40% of patients continue to experience seizures, indicating a significant unmet need in the focal epilepsy market [5][6] - **Potential Market Size**: RAP-219 is believed to represent a multi-billion dollar commercial opportunity, subject to approval [6][19] Core Findings from the Phase 2A Trial of RAP-219 - **Efficacy Results**: - 78% reduction in clinical seizure frequency [4][14] - 24% seizure freedom rate [4][23] - 71% median reduction in long episode frequency from baseline [12] - 85.2% of patients achieved a 30% or greater reduction in long episodes [13] - 72% of patients achieved a 50% or greater reduction in clinical seizures [15] - **Safety Profile**: RAP-219 was generally well tolerated, with common treatment-emergent adverse events (TEAEs) being dizziness, headache, and fatigue [16] - **Dosing Convenience**: Once-daily dosing and low risk of drug-drug interactions enhance the potential for widespread adoption among healthcare providers [6][18] Development Plans and Future Steps - **Next Steps**: Plans to meet with the FDA for an end-of-Phase 2 meeting in Q4 2025 and initiate Phase 3 trials in Q3 2026 [17][19] - **Long-Acting Injectable (LAI)**: Development of an LAI formulation is ongoing, which could improve patient adherence and expand clinical utility [18] - **Bipolar Mania Trials**: Ongoing Phase 2 trial in bipolar mania, with expectations for data in early 2027 [52] Additional Insights - **Physician Feedback**: Positive reception from physicians regarding RAP-219's profile, with expectations for it to be used in both refractory settings and earlier in treatment algorithms [64][66] - **Translatability to Phase 3**: Confidence in the translatability of Phase 2 results to Phase 3 trials, with a baseline seizure frequency of 10 being representative of typical disease burden [22][24] - **Market Dynamics**: The competitive context and the drug's profile are expected to facilitate efficient patient recruitment for Phase 3 trials [41][42] Conclusion - RAP-219 demonstrates promising efficacy and safety profiles, positioning it as a potential best-in-class treatment for focal onset seizures and possibly other indications. The company is well-prepared to advance its development plans and capitalize on the significant market opportunity.

Clinical Trial Results of RAP-219 - The Phase 2a trial of RAP-219 in drug-resistant focal onset seizure patients achieved primary endpoints with high statistical significance, showing a 71% reduction in long episodes (LEs) and an 85.2% responder rate (≥30% LE reduction)[31] - Clinical seizure secondary endpoints achieved statistical significance with RAP-219, demonstrating a 77.8% clinical seizure reduction and 72% of patients achieving a clinically meaningful response (≥50% reduction)[34] - RAP-219 was generally well-tolerated in the Phase 2a trial, with 83.3% of patients reporting any treatment-emergent adverse events (TEAEs), the majority (79%) of which were mild, and a 10% discontinuation rate due to TEAEs[38] RAP-219 Development and Future Plans - The company plans to hold an End of Phase 2 FDA meeting in 4Q 2025 and initiate Phase 3 trials in 3Q 2026 for RAP-219 in focal onset seizures[43] - The company is advancing the first-ever long-acting injectable (LAI) formulation for epilepsy patients, which could provide IP extension and potential commercial upside for RAP-219[15] - The company anticipates multiple catalysts over the next 24 months, including the initiation of a RAP-219 focal onset seizure open-label extension trial in 2H 2025, a bipolar mania Phase 2 trial in 3Q 2025, and the initiation of two Phase 3 trials for focal onset seizures in 3Q 2026[51] Market and Financial Position - The company believes RAP-219 has an emerging best-in-class profile for focal onset seizures, representing a multi-billion dollar commercial opportunity if approved[14, 53] - As of June 30, 2025, the company has a cash balance of $260.4 million, which is expected to support operations through the end of 2026[52]

Core Insights - Rapport Therapeutics announced that the Phase 2a clinical trial of RAP-219 met its primary endpoint, showing a statistically significant reduction in long episodes (LEs) in patients with drug-resistant focal onset seizures [1][4][6] - The company plans to advance RAP-219 into two Phase 3 pivotal trials in Q3 2026, following the promising results from the trial [1][11] Efficacy Results - In the 8-week treatment period, 85.2% of patients achieved a ≥30% reduction in LEs from baseline (p<0.0001) [4][5] - 72.0% of patients achieved a ≥50% reduction in clinical seizures from baseline (p<0.0001) [4][7] - 24% of patients achieved seizure freedom during the treatment period (p<0.0001) [4][6] Trial Design - The Phase 2a trial was a proof-of-concept, multi-center, open-label study involving 30 patients with drug-resistant focal onset seizures [3][10] - Patients received escalating doses of RAP-219 over an 8-week period, starting with 0.75 mg daily for 5 days, followed by 1.25 mg daily [3] Safety and Tolerability - RAP-219 was generally well tolerated, with 78.5% of treatment-emergent adverse events (TEAEs) being mild [9][12] - No serious adverse events were reported, and the discontinuation rate due to TEAEs was low at 10% [12] Future Development Plans - The company plans to initiate an open-label long-term safety trial by the end of 2025 for patients who participated in the Phase 2a trial [13] - Rapport is also developing a long-acting injectable formulation of RAP-219 to improve patient adherence [14] Broader Applications - Beyond epilepsy, RAP-219 is being evaluated in a Phase 2 trial for bipolar mania, with topline results expected in the first half of 2027 [15]

Core Viewpoint - Rapport Therapeutics is set to announce topline results for the Phase 2a trial of its investigational drug RAP-219, aimed at treating drug-resistant focal onset seizures, during a conference call on September 8, 2025 [1]. Company Overview - Rapport Therapeutics is a clinical-stage biotechnology company focused on developing small molecule precision medicines for neurological and psychiatric disorders [3]. - The company’s founders have made significant discoveries regarding receptor associated proteins (RAPs) in the brain, which underpin the RAP technology platform [3]. - The lead investigational drug, RAP-219, is designed to achieve neuroanatomical specificity by selectively targeting a RAP expressed in specific brain regions [3]. - In addition to drug-resistant focal onset seizures, RAP-219 is being explored as a potential treatment for bipolar mania and diabetic peripheral neuropathic pain [3]. - The company is also engaged in preclinical and late-stage discovery programs targeting chronic pain and hearing disorders [3].