Company Overview - Rapport Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing small molecule precision medicines for central nervous system (CNS) disorders [6] - The company utilizes a unique RAP technology platform that targets receptor associated proteins (RAPs) in the brain, aiming to create precision small molecule candidates that address limitations in conventional neurology drug discovery [6] Leadership Appointment - Dr. Jeffrey Sevigny has been appointed as the chief medical officer (CMO) of Rapport Therapeutics, bringing over 15 years of experience in translational and clinical drug development [1][2] - Dr. Sevigny previously served as CMO at Prevail Therapeutics and held senior roles at Eli Lilly, where he contributed to significant corporate successes including financing rounds, an IPO, and an acquisition [2][4] Clinical Strategy and Pipeline - As CMO, Dr. Sevigny will oversee the clinical strategy and advancement of Rapport's pipeline, particularly the RAP-219 program, which is currently in clinical trials for conditions such as focal epilepsy, diabetic peripheral neuropathic pain, and bipolar mania [3][6] - The RAP-219 program is noted for its compelling Phase 1 data and aims to achieve neuroanatomical specificity by selectively targeting RAPs expressed in specific brain regions [5][6] Company Vision and Goals - The CEO of Rapport, Abraham N. Ceesay, expressed confidence in Dr. Sevigny's ability to lead the clinical development team and emphasized the importance of collaboration with regulatory authorities and patient advocacy organizations [4] - Dr. Sevigny highlighted the company's strong scientific foundation and the potential of its clinical candidates to provide transformative treatment options for patients with neurological disorders [5]

Company Overview - Rapport Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing small molecule precision medicines for central nervous system (CNS) disorders [3] - The company utilizes a unique RAP technology platform based on pioneering discoveries related to receptor associated proteins (RAPs) in the brain, allowing for a differentiated approach in drug discovery [3] - The precision neuroscience pipeline includes the lead clinical program RAP-219, which targets specific RAPs in discrete brain regions and is currently in trials for focal epilepsy, diabetic peripheral neuropathic pain, and bipolar mania [3] Upcoming Events - Management will present at the TD Cowen 45 Annual Health Care Conference in Boston on March 4, 2025, from 3:10 to 3:40 PM EST [1] - The company will also participate in the Stifel 2025 Virtual CNS Forum on March 19, 2025, from 10:30 to 10:55 AM EST [2] - Live and archived webcasts of these presentations will be available on the company's website under the "Investors" section [2]

Core Insights - Rapport Therapeutics, Inc. announced positive results from its PET and MAD-2 trials for RAP-219, indicating its potential as a treatment for focal epilepsy and other CNS disorders [1][2][3] Trial Results - In the PET trial, RAP-219 achieved target receptor occupancy (RO) associated with maximal efficacy within five days of dosing, confirming the neuroanatomical expression of TARPγ8 [1][10] - The MAD-2 trial demonstrated that RAP-219 was generally well tolerated, with no serious adverse events and favorable tolerability across various dosing regimens [2][11] - The ongoing Phase 2a trial of RAP-219 in focal epilepsy is on track, with topline data expected in mid-2025 [1][7] Drug Profile - RAP-219 is designed as a selective AMPA receptor negative allosteric modulator targeting TARPγ8, which is enriched in the hippocampus and cerebral cortex, potentially leading to improved tolerability compared to traditional treatments [8][9] - The drug has a long half-life of 8-14 days and minimal drug-drug interactions, making it suitable for patients on multiple medications [8] Company Overview - Rapport Therapeutics focuses on developing small molecule precision medicines for CNS disorders, leveraging its RAP technology platform to create differentiated product candidates [9] - The company is advancing RAP-219 for multiple indications, including focal epilepsy, diabetic peripheral neuropathic pain, and bipolar mania [9]

Core Viewpoint - Rapport Therapeutics, Inc. announced positive results from its PET and MAD-2 trials for RAP-219, indicating its potential as a differentiated treatment for focal epilepsy with a favorable tolerability profile and broad therapeutic index [1][2][3]. Group 1: Trial Results - In the PET trial, RAP-219 achieved target receptor occupancy (RO) associated with maximal efficacy within five days of dosing, confirming the neuroanatomical expression of TARPγ8 [1][10]. - The MAD-2 trial demonstrated that RAP-219 was generally well tolerated, with no serious adverse events and a favorable tolerability profile consistent with prior Phase 1 trials [2][11]. - Data from both trials support the ongoing Phase 2a trial in focal epilepsy, with topline results expected in mid-2025 [1][7]. Group 2: Drug Profile - RAP-219 is designed as a clinical-stage AMPA receptor negative allosteric modulator, selectively targeting TARPγ8 to achieve neuroanatomical specificity, which may enhance tolerability compared to traditional treatments [8][12]. - The drug has a long half-life of 8-14 days and minimal drug-drug interactions, making it suitable for patients on multiple medications [8]. - The selective targeting of TARPγ8 may provide a pipeline-in-a-product opportunity for various neurological disorders, including focal epilepsy, diabetic peripheral neuropathic pain, and bipolar mania [8][12]. Group 3: Company Overview - Rapport Therapeutics is a clinical-stage biotechnology company focused on developing small molecule precision medicines for central nervous system disorders [9]. - The company utilizes its RAP technology platform to generate differentiated product candidates, aiming to improve therapeutic outcomes for patients suffering from CNS disorders [9].

Core Insights - Rapport Therapeutics announced positive results from the PET and MAD-2 trials for RAP-219, indicating its potential for treating focal epilepsy and demonstrating a favorable tolerability profile [1][2][3] Group 1: Trial Results - In the PET trial, RAP-219 achieved target receptor occupancy (RO) associated with maximal efficacy within five days of dosing, confirming the neuroanatomical expression of TARP8 [1][12] - The MAD-2 trial showed RAP-219 was generally well tolerated, with no serious adverse events and a favorable tolerability profile across various dosing regimens [2][13] - Both trials underscored the potential broad therapeutic index of RAP-219 and its dosing flexibility, with topline data from the ongoing Phase 2a trial expected in mid-2025 [1][8] Group 2: Drug Profile - RAP-219 is designed as a selective AMPA receptor negative allosteric modulator targeting TARP8, which is enriched in the hippocampus and cerebral cortex, potentially leading to improved tolerability compared to traditional treatments [9][10] - The drug has a long half-life of 8-14 days and minimal drug-drug interactions, making it suitable for patients on multiple medications [9] - The company is pursuing RAP-219 for various conditions, including focal epilepsy, diabetic peripheral neuropathic pain, and bipolar mania, indicating a broad application potential [9][10] Group 3: Company Developments - Rapport Therapeutics is a clinical-stage biotechnology company focused on developing small molecule precision medicines for CNS disorders, leveraging its RAP technology platform [10] - The company has conducted four Phase 1 trials with 100 healthy volunteers, demonstrating the safety and tolerability of RAP-219 [2][10] - A leadership change was announced with the resignation of the chief medical officer, although the company expressed confidence that this would not disrupt ongoing clinical programs [3]

Core Insights - Rapport Therapeutics announced positive results from the PET and MAD-2 trials for RAP-219, demonstrating its potential for treating focal epilepsy and confirming its dosing regimen for the ongoing Phase 2a trial [1][2][3] Trial Results - In the PET trial, RAP-219 achieved target receptor occupancy (RO) associated with maximal efficacy within five days of dosing, exceeding the target range of 50%-70% [10] - The MAD-2 trial showed RAP-219 was generally well tolerated, with no serious adverse events and no treatment emergent adverse events greater than Grade 2 [2][11] - The trials confirmed the neuroanatomical specificity of RAP-219 through selective targeting of TARP8, which is enriched in the hippocampus and cerebral cortex [8][10] Dosing and Tolerability - The ongoing Phase 2a trial is on track, with topline data expected in mid-2025, and the dosing regimen has been supported by the trial results [1][3][7] - RAP-219 demonstrated favorable tolerability across various dosing regimens, with a long half-life of 8-14 days and minimal drug-drug interactions, making it suitable for polypharmacy [8][12] Company Overview - Rapport Therapeutics is focused on developing small molecule precision medicines for CNS disorders, leveraging its RAP technology platform to create differentiated product candidates [9] - The company is advancing RAP-219 as a treatment for focal epilepsy, diabetic peripheral neuropathic pain, and bipolar mania, with additional programs targeting chronic pain and hearing disorders [12]

Core Insights - Rapport Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing small molecule precision medicines for central nervous system disorders [1][6] - The company will present new data at the American Epilepsy Society Annual Meeting regarding the RNS® System and its correlation with seizure frequency reduction [2][3] Company Overview - Rapport Therapeutics specializes in precision medicines targeting CNS disorders, leveraging its RAP technology platform to create differentiated product candidates [6] - The lead product candidate, RAP-219, is a negative allosteric modulator designed to selectively target TARPγ8, which is expressed in specific brain regions [5][6] Clinical Trials and Research - A Phase 2a proof-of-concept trial for RAP-219 is underway, focusing on its efficacy as an anti-seizure treatment for drug-resistant focal epilepsy [2] - The trial aims to demonstrate a reduction in long episode frequency and clinically meaningful reduction in patient-reported seizures [2] Upcoming Presentations - Rapport will showcase multiple posters at the AES Annual Meeting, including studies on the antiseizure effects of TARPγ8 modulators and the pharmacokinetics of RAP-219 [3] - The presentations will cover the evolution of RAP-219 and its potential as a treatment for various CNS disorders [3]

Financial Performance - The company reported net losses of $17.5 million and $8.7 million for the three months ended September 30, 2024 and 2023, respectively, and $58.3 million and $21.3 million for the nine months ended September 30, 2024 and 2023, respectively[129]. - The net loss for the three months ended September 30, 2024, was $17.5 million, compared to a net loss of $8.7 million for the same period in 2023, representing an increase of $8.8 million[158]. - The company reported a net loss of $58.3 million for the nine months ended September 30, 2024, compared to a net loss of $21.3 million for the same period in 2023, indicating a $37.0 million increase in losses[163]. - The company incurred $50.4 million in cash used in operating activities during the nine months ended September 30, 2024, primarily due to a net loss of $58.3 million[171]. - Net cash used in operating activities for the nine months ended September 30, 2024, was $(50.4) million, compared to $(16.8) million for the same period in 2023[175]. - General and administrative expenses for the nine months ended September 30, 2024, were $15.8 million, up from $5.2 million in the same period of 2023, representing a $10.6 million increase[165]. Research and Development - The company completed a Phase 1 trial for RAP-219 and is currently recruiting for a Phase 2a trial in adult patients with drug-resistant focal epilepsy, expecting topline results in mid-2025[124]. - The company has initiated a second MAD clinical trial for RAP-219, with topline results expected in the first quarter of 2025[124]. - The company anticipates a substantial increase in research and development expenses as it advances RAP-219 through clinical development and regulatory approval[144]. - Research and development expenses for the three months ended September 30, 2024, were $15.5 million, an increase of $8.0 million from $7.6 million in the same period of 2023[158]. - Research and development expenses totaled $43.7 million for the nine months ended September 30, 2024, up from $16.2 million in the same period of 2023, reflecting a $27.5 million increase[164]. - During the nine months ended September 30, 2024, the company recognized $1.1 million in research and development expenses related to NeuroPace services[183]. Financial Position and Funding - As of September 30, 2024, the company had an accumulated deficit of $103.8 million and expects operating losses to increase substantially due to ongoing clinical trials and research activities[129]. - The company raised aggregate gross proceeds of $424.4 million from financings, with cash, cash equivalents, and short-term investments totaling $320.7 million as of September 30, 2024[128]. - Cash and cash equivalents as of September 30, 2024, were $39.3 million, with short-term investments totaling $281.3 million[170]. - As of September 30, 2024, the company had cash, cash equivalents, and short-term investments of $320.7 million, expected to fund operations through at least 12 months from the issuance date of the financial statements[178]. - Future funding requirements are anticipated to increase substantially due to ongoing activities, particularly in clinical trials and product development[178]. - The company may need to raise substantial additional capital in the future to support ongoing product development and commercialization efforts[179]. Expenses and Income - Total operating expenses for the three months ended September 30, 2024, were $21.6 million, compared to $9.6 million in the same period of 2023, resulting in an increase of $12.1 million[158]. - General and administrative expenses for the three months ended September 30, 2024, were $6.1 million, up from $2.0 million in the same period of 2023, reflecting an increase of $4.1 million[158]. - Interest income for the three months ended September 30, 2024, was $4.1 million, an increase of $3.2 million from $0.9 million in the same period of 2023[158]. - Interest income for the nine months ended September 30, 2024, was $8.6 million, compared to $1.2 million for the same period in 2023, indicating a $7.4 million increase[167]. Agreements and Collaborations - The company entered into a license agreement with Janssen Pharmaceutical NV, which includes potential milestone payments of up to $76 million for development and $40 million for sales of TARPg8 products[135]. - The NeuroPace Agreement allows the company to utilize data from RNS systems in clinical trials, with exclusivity provisions preventing NeuroPace from providing similar services to competitors[139][140]. - An upfront payment of $1.0 million was made to Janssen Pharmaceutical NV, with potential milestone payments totaling up to $141 million for product development and sales[181]. - The company expects to pay NeuroPace up to $3.7 million over two years for services related to the Phase 2a clinical trial of RAP-219[182]. - The change in fair value of preferred stock tranche right liability was $7.4 million for the nine months ended September 30, 2024, compared to $1.0 million for the same period in 2023, reflecting a $6.4 million increase[168].

Financial Performance - The company reported a net loss of $17.5 million for Q3 2024, compared to a net loss of $8.7 million in the same period last year, reflecting a 100% increase in losses [15]. - Net loss for the three months ended September 30, 2024, was $17,537,000, compared to a net loss of $8,709,000 in the prior year, representing a 101% increase in losses [25]. - The company reported a net cash increase of $(70,850,000) for the three months ended September 30, 2024, compared to an increase of $79,884,000 in the same period of 2023 [26]. Expenses - Research and development expenses rose to $15.5 million in Q3 2024, up from $7.6 million in the prior year, indicating a 104% increase driven by clinical development costs [15]. - General and administrative expenses increased to $6.1 million in Q3 2024, compared to $2.0 million in the prior year, representing a 205% increase due to business growth and public company requirements [15]. - Operating expenses for the three months ended September 30, 2024, were $21,640,000, up from $9,564,000 in the same period of 2023, indicating a 126% increase [25]. - Research and development expenses rose to $15,543,000 for the three months ended September 30, 2024, compared to $7,580,000 in the same period of 2023, marking a 105% increase [25]. Cash and Assets - The company ended Q3 2024 with $320.7 million in cash, cash equivalents, and short-term investments, down from $336.1 million as of June 30, 2024, primarily due to cash outflows from operating activities [15]. - Cash and cash equivalents decreased to $39,314,000 as of September 30, 2024, down from $70,169,000 on December 31, 2023 [24]. - Total current assets increased to $325,939,000 as of September 30, 2024, compared to $150,872,000 on December 31, 2023, reflecting a significant growth [24]. - The company expects its current cash reserves to fund operations through the end of 2026 [15]. Clinical Trials and Development - Ongoing clinical trials for RAP-219 include a Phase 2a trial in focal epilepsy, with topline data expected in mid-2025 [9]. - The FDA placed the IND for a Phase 2a trial of RAP-219 in diabetic peripheral neuropathic pain on clinical hold, requiring additional protocol information [10]. - The company is conducting a second multiple ascending dose trial (MAD-2) of RAP-219, with topline results expected in Q1 2025 [8]. - The company plans to initiate a Phase 2a trial in bipolar disorder patients with acute mania in 2025 [13]. Corporate Governance - The company has appointed new board members with extensive biotech experience to support its growth and innovation strategy [3]. Shareholder Equity and Liabilities - Total stockholders' equity improved to $323,114,000 as of September 30, 2024, compared to a deficit of $(25,634,000) on December 31, 2023 [24]. - Total liabilities decreased to $8,030,000 as of September 30, 2024, from $14,479,000 on December 31, 2023 [24]. - Weighted-average common shares outstanding increased to 34,855,907 for the three months ended September 30, 2024, from 1,529,216 in the prior year [25].

Core Insights - Rapport Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing small molecule precision medicines for central nervous system (CNS) disorders [2] Group 1: Company Overview - The company is dedicated to discovering and developing transformational precision neuromedicines for patients with CNS disorders [2] - Rapport's founders have made pioneering discoveries related to receptor associated proteins (RAPs) in the brain, forming the basis of the company's RAP technology platform [2] - The precision neuroscience pipeline includes the lead clinical program, RAP-219, which targets a RAP expressed in specific brain regions [2] Group 2: Clinical Programs - RAP-219 is currently in clinical trials for focal epilepsy, peripheral neuropathic pain, and bipolar disorder [2] - Additional preclinical and late-stage discovery programs are targeting CNS disorders such as chronic pain and hearing disorders [2] Group 3: Upcoming Events - Management will participate in fireside chats at the Stifel Healthcare Conference on November 18, 2024, and the Jefferies London Healthcare Conference on November 20, 2024 [1]