[PART I. FINANCIAL INFORMATION](index=9&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements (Unaudited)](index=9&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Presents unaudited condensed consolidated financial statements, including balance sheets, operations, equity, cash flows, and detailed notes [Condensed Consolidated Balance Sheets](index=9&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Summarizes the company's assets, liabilities, and equity at specific points in time **Condensed Consolidated Balance Sheets (in thousands)** | Metric | June 30, 2025 | December 31, 2024 | | :---------------------------------- | :-------------- | :---------------- | | Cash and cash equivalents | $55,067 | $56,805 | | Short-term investments | $205,380 | $248,475 | | Total current assets | $270,242 | $309,802 | | Total assets | $285,495 | $314,933 | | Total current liabilities | $11,879 | $8,767 | | Total liabilities | $21,983 | $9,506 | | Total stockholders' equity | $263,512 | $305,427 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Details the company's revenues, expenses, and net loss over specific periods **Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except per share data)** | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :------------------------------------------------ | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Research and development expenses | $22,680 | $15,689 | $42,252 | $28,193 | | General and administrative expenses | $6,816 | $5,111 | $14,352 | $9,701 | | Total operating expenses | $29,496 | $20,800 | $56,604 | $37,894 | | Loss from operations | $(29,496) | $(20,800) | $(56,604) | $(37,894) | | Interest income | $2,764 | $2,679 | $5,809 | $4,494 | | Change in fair value of preferred stock tranche right liability | $0 | $0 | $0 | $(7,390) | | Net loss | $(26,732) | $(18,121) | $(50,795) | $(40,790) | | Net loss per share, basic and diluted | $(0.75) | $(1.70) | $(1.44) | $(6.42) | | Weighted-average common shares outstanding | 35,444,635 | 10,666,528 | 35,356,098 | 6,356,700 | | Comprehensive loss | $(26,713) | $(18,144) | $(50,349) | $(40,977) | [Condensed Consolidated Statements of Convertible Preferred Stock and Stockholders' Equity (Deficit)](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Convertible%20Preferred%20Stock%20and%20Stockholders'%20Equity%20(Deficit)) Outlines changes in convertible preferred stock and stockholders' equity over reporting periods - Total stockholders' equity decreased from **$305.4 million** at December 31, 2024, to **$263.5 million** at June 30, 2025, primarily due to net loss and stock-based compensation[30](index=30&type=chunk) - Stock-based compensation expense for the six months ended June 30, 2025, was **$8.4 million**, contributing to the change in additional paid-in capital[30](index=30&type=chunk) - The company reported a net loss of **$26.7 million** for the three months ended June 30, 2025, and **$50.8 million** for the six months ended June 30, 2025, increasing the accumulated deficit[24](index=24&type=chunk)[30](index=30&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=12&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Reports cash inflows and outflows from operating, investing, and financing activities **Condensed Consolidated Statements of Cash Flows (in thousands)** | Activity | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :------------------------------------------ | :----------------------------- | :----------------------------- | | Net cash used in operating activities | $(45,307) | $(33,967) | | Net cash provided by (used in) investing activities | $43,627 | $(149,030) | | Net cash (used in) provided by financing activities | $(58) | $223,012 | | Net (decrease) increase in cash, cash equivalents, and restricted cash | $(1,738) | $40,015 | | Cash, cash equivalents, and restricted cash at end of period | $55,172 | $110,269 | [Notes to Unaudited Condensed Consolidated Financial Statements](index=13&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) Provides detailed explanations and breakdowns for figures presented in the financial statements - Rapport Therapeutics, Inc. is a clinical-stage biotechnology company focused on neurological or psychiatric disorders, incorporated in February 2022[34](index=34&type=chunk) - The company has incurred recurring losses since inception, with an accumulated deficit of **$174.5 million** as of June 30, 2025, and expects to continue generating operating losses[37](index=37&type=chunk) - Cash, cash equivalents, and short-term investments totaled **$260.4 million** as of June 30, 2025, expected to fund operations for at least 12 months from the financial statement issuance date[37](index=37&type=chunk) - The company completed its IPO and concurrent private placement in June 2024, raising net proceeds of **$157.6 million**[37](index=37&type=chunk) - Significant estimates in financial statements include research and development expenses, valuation of common stock and stock-based awards, and preferred stock tranche right liability[45](index=45&type=chunk) - Short-term investments, classified as available-for-sale debt securities, consist of U.S. Treasury bills, government securities, and government agency securities, carried at fair market value[48](index=48&type=chunk) **Short-term investments (in thousands)** | Type | June 30, 2025 Fair Value | December 31, 2024 Fair Value | | :-------------------------- | :----------------------- | :------------------------- | | U.S. Treasury bills | $26,867 | $28,723 | | Government securities | $91,979 | $136,514 | | Government agency securities | $86,534 | $83,238 | | **Total** | **$205,380** | **$248,475** | - The Series B preferred stock tranche right liability was settled in March 2024, resulting in no balance as of June 30, 2024, and no further changes in fair value recognized[73](index=73&type=chunk) **Property and equipment, net (in thousands)** | Category | June 30, 2025 | December 31, 2024 | | :-------------------------- | :-------------- | :---------------- | | Lab equipment | $4,261 | $3,716 | | Computer equipment | $93 | $67 | | Leasehold improvements | $281 | $281 | | Construction in process | $0 | $430 | | Total property and equipment | $4,635 | $4,494 | | Less: Accumulated depreciation | $(1,463) | $(965) | | **Net property and equipment** | **$3,172** | **$3,529** | **Accrued expenses and other current liabilities (in thousands)** | Category | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :-------------- | :---------------- | | Research and development | $2,974 | $1,664 | | Professional fees | $759 | $235 | | Employee related | $3,141 | $3,708 | | Accrued other | $70 | $469 | | **Total** | **$6,944** | **$6,076** | - Prior to the June 2024 IPO, all outstanding Series A and Series B convertible preferred stock converted into **22,146,816 shares** of common stock[79](index=79&type=chunk) - In June 2024, the company issued and sold **9,200,000 shares** of common stock in its IPO and **1,058,824 shares** in a concurrent private placement, generating **$140.9 million** and **$16.7 million** in net proceeds, respectively[94](index=94&type=chunk)[95](index=95&type=chunk) - As of June 30, 2025, **9,096,166 shares** of common stock were reserved for issuance under the 2022 Plan, 2024 Plan, and 2024 ESPP[96](index=96&type=chunk) - Stock-based compensation expense for stock options was **$3.8 million** and **$7.2 million** for the three and six months ended June 30, 2025, respectively[119](index=119&type=chunk) - The company entered into new operating leases in San Diego, CA (commenced Jan 2025) and Boston, MA (commenced June 2025) for laboratory and office space[121](index=121&type=chunk)[123](index=123&type=chunk) - Related party transactions with Janssen Pharmaceutical NV and Third Rock Ventures LLC decreased to zero for the six months ended June 30, 2025, from **$69 thousand** and **$0.1 million**, respectively, in 2024[124](index=124&type=chunk)[125](index=125&type=chunk) - The company entered into a Master Services Agreement with NeuroPace Inc. in November 2023 for data services related to the RAP-219 Phase 2a clinical trial, with payments up to **$3.7 million**[128](index=128&type=chunk)[131](index=131&type=chunk) - The company operates as a single operating and reportable segment, focused on developing medicines for neurological or psychiatric disorders in the United States[132](index=132&type=chunk)[133](index=133&type=chunk)[134](index=134&type=chunk) - Basic and diluted net loss per common share was **$(0.75)** and **$(1.44)** for the three and six months ended June 30, 2025, respectively[138](index=138&type=chunk) - On July 1, 2025, the company filed an S-3 registration statement for up to **$400.0 million** in securities, including an at-the-market (ATM) offering program for up to **$150.0 million** in common stock[140](index=140&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=38&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Analyzes Rapport Therapeutics' financial condition, operations, clinical progress, product candidates, and future funding needs [Overview](index=38&type=section&id=Overview) Provides a high-level summary of the company's business, lead product candidates, and financial performance - Rapport Therapeutics is a clinical-stage biotechnology company developing small molecule precision medicines for neurological or psychiatric disorders using its RAP technology platform[144](index=144&type=chunk) - RAP-219, the lead product candidate, is an AMPA receptor negative allosteric modulator (NAM) targeting TARPγ8 for focal onset seizures, bipolar disorder, and peripheral neuropathic pain[145](index=145&type=chunk) - Phase 2a proof-of-concept trial in drug-resistant focal onset seizures is on track for topline results in September 2025[147](index=147&type=chunk) - Phase 2 proof-of-concept trial in bipolar mania has been initiated, with topline results expected in the first half of 2027[147](index=147&type=chunk) - The IND for a Phase 2a trial of RAP-219 for diabetic peripheral neuropathic pain (DPNP) was placed on clinical hold by the FDA in Q4 2024, pending additional information[147](index=147&type=chunk) - The company has incurred significant operating losses since inception, with net losses of **$26.7 million** and **$50.8 million** for the three and six months ended June 30, 2025, respectively, and an accumulated deficit of **$174.5 million**[150](index=150&type=chunk) - As of June 30, 2025, cash, cash equivalents, and short-term investments totaled **$260.4 million**, expected to fund operations through the end of 2026[149](index=149&type=chunk)[155](index=155&type=chunk) [License and Collaboration Agreements](index=42&type=section&id=License%20and%20Collaboration%20Agreements) Details key agreements with third parties for product development and data services - The company holds an exclusive option and license agreement with Janssen Pharmaceutical NV for TARPγ8 AMPAR products and a non-exclusive license for nACh products[156](index=156&type=chunk) - Payments to Janssen include a **$1.0 million** upfront payment, a **$4.0 million** option fee, and potential future development and sales milestone payments up to **$76.0 million** and **$40.0 million**, respectively, for the lead TARPγ8 product, plus royalties[157](index=157&type=chunk)[158](index=158&type=chunk) - A Master Services Agreement with NeuroPace Inc. provides data services for the RAP-219 Phase 2a clinical trial in drug-resistant focal onset seizures, with payments up to **$3.7 million** over two years[160](index=160&type=chunk)[161](index=161&type=chunk) [Components of Results of Operations](index=44&type=section&id=Components%20of%20Results%20of%20Operations) Explains the primary drivers and nature of the company's operating expenses, interest income, and other financial items - Research and development expenses include personnel, external costs (CMOs, CROs), intellectual property licenses, lab supplies, and facility costs, and are expected to increase substantially[162](index=162&type=chunk)[163](index=163&type=chunk)[165](index=165&type=chunk) - General and administrative expenses cover personnel, legal, professional, office, IT, and facility costs, and are anticipated to rise due to public company operations and growth[167](index=167&type=chunk)[168](index=168&type=chunk) - Interest income is derived from cash, cash equivalents, and short-term investments[169](index=169&type=chunk) - The Series B preferred stock tranche right liability was settled in March 2024, eliminating future changes in fair value recognition[171](index=171&type=chunk) - No income tax provision was recorded for the periods, with a full valuation allowance against net deferred tax assets[172](index=172&type=chunk) [Results of Operations](index=47&type=section&id=Results%20of%20Operations) Compares the company's financial performance across different reporting periods, highlighting key changes in expenses and net loss **Results of Operations (Three Months Ended June 30, in thousands)** | Metric | 2025 | 2024 | Change | | :-------------------------- | :--- | :--- | :----- | | Research and development | $22,680 | $15,689 | $6,991 | | General and administrative | $6,816 | $5,111 | $1,705 | | Total operating expenses | $29,496 | $20,800 | $8,696 | | Loss from operations | $(29,496) | $(20,800) | $(8,696) | | Interest income | $2,764 | $2,679 | $85 | | Net loss | $(26,732) | $(18,121) | $(8,611) | **Results of Operations (Six Months Ended June 30, in thousands)** | Metric | 2025 | 2024 | Change | | :------------------------------------------------ | :--- | :--- | :----- | | Research and development | $42,252 | $28,193 | $14,059 | | General and administrative | $14,352 | $9,701 | $4,651 | | Total operating expenses | $56,604 | $37,894 | $18,710 | | Loss from operations | $(56,604) | $(37,894) | $(18,710) | | Interest income | $5,809 | $4,494 | $1,315 | | Change in fair value of preferred stock tranche right liability | $0 | $(7,390) | $7,390 | | Net loss | $(50,795) | $(40,790) | $(10,005) | - The increase in R&D expenses for both periods was primarily driven by higher RAP-219 program costs (clinical trials, manufacturing) and increased personnel-related costs, partially offset by decreased preclinical program costs[178](index=178&type=chunk)[185](index=185&type=chunk) - General and administrative expenses increased due to higher workforce expenses (salaries, bonuses, stock-based compensation) and administrative costs associated with public company operations, partially offset by reduced professional and consulting fees post-IPO[177](index=177&type=chunk)[182](index=182&type=chunk)[184](index=184&type=chunk)[186](index=186&type=chunk)[187](index=187&type=chunk) - Interest income increased due to higher cash, cash equivalents, and short-term investment balances following the IPO and Series B financing[179](index=179&type=chunk)[188](index=188&type=chunk) [Liquidity and Capital Resources](index=52&type=section&id=Liquidity%20and%20Capital%20Resources) Assesses the company's ability to meet its short-term and long-term financial obligations and its funding strategies - As of June 30, 2025, the company had **$260.4 million** in cash, cash equivalents, and short-term investments, excluding restricted cash[190](index=190&type=chunk) - Historically, operations have been funded by convertible notes, preferred stock, and common stock (IPO and private placement), totaling **$424.4 million** in gross proceeds[190](index=190&type=chunk) - The company filed an S-3 registration statement for up to **$400.0 million** in securities, including an ATM program for up to **$150.0 million** in common stock, to fund future operations[191](index=191&type=chunk) **Cash Flows Summary (Six Months Ended June 30, in thousands)** | Activity | 2025 | 2024 | | :------------------------------------------ | :--- | :--- | | Net cash used in operating activities | $(45,307) | $(33,967) | | Net cash provided by (used in) investing activities | $43,627 | $(149,030) | | Net cash (used in) provided by financing activities | $(58) | $223,012 | | Net increase (decrease) in cash, cash equivalents and restricted cash | $(1,738) | $40,015 | - Operating activities used **$45.3 million** in cash in H1 2025, an increase from **$34.0 million** in H1 2024, primarily due to net loss and increased prepaid expenses[193](index=193&type=chunk)[194](index=194&type=chunk) - Investing activities provided **$43.6 million** in cash in H1 2025, a significant shift from using **$149.0 million** in H1 2024, driven by maturities of short-term investments[195](index=195&type=chunk)[196](index=196&type=chunk) - Financing activities used **$0.1 million** in cash in H1 2025, compared to providing **$223.0 million** in H1 2024, reflecting deferred offering costs in 2025 versus IPO and preferred stock proceeds in 2024[197](index=197&type=chunk)[198](index=198&type=chunk) - Existing cash, cash equivalents, and short-term investments are projected to fund operating expenses and capital expenditure requirements through the end of 2026[199](index=199&type=chunk) - Future funding requirements are substantial and depend on the progress of product candidates, clinical trials, manufacturing, regulatory approvals, and commercialization efforts[200](index=200&type=chunk)[205](index=205&type=chunk) - Contractual obligations include **$14.2 million** in future minimum operating lease payments over a weighted average remaining lease term of 5.0 years[203](index=203&type=chunk) - The Janssen License includes potential milestone payments up to **$76.0 million** for development and **$40.0 million** for sales, plus tiered royalties[204](index=204&type=chunk) - The NeuroPace Agreement involves payments up to **$3.7 million** over approximately two years for services and milestone achievement[206](index=206&type=chunk) [Critical Accounting Policies and Estimates](index=58&type=section&id=Critical%20Accounting%20Policies%20and%20Estimates) Highlights key accounting policies and estimates that require significant judgment and can materially impact financial reporting - There were no material changes to the critical accounting policies and estimates previously disclosed in the Annual Report on Form 10-K for the year ended December 31, 2024[210](index=210&type=chunk) [Emerging Growth Company and Smaller Reporting Company Status](index=58&type=section&id=Emerging%20Growth%20Company%20and%20Smaller%20Reporting%20Company%20Status) Discusses the company's status as an emerging growth company and smaller reporting company, and the associated reduced disclosure requirements - The company qualifies as an 'emerging growth company' (EGC) and has elected to use the extended transition period for complying with new or revised accounting standards[211](index=211&type=chunk)[507](index=507&type=chunk) - As an EGC, the company benefits from reduced reporting requirements, including fewer years of audited financial statements and reduced executive compensation disclosures[505](index=505&type=chunk) - The company also qualifies as a 'smaller reporting company,' allowing for scaled disclosures[213](index=213&type=chunk)[508](index=508&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=59&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a smaller reporting company, detailed quantitative and qualitative disclosures about market risk are not required - The company is a smaller reporting company and is not required to provide the information required by this Item[213](index=213&type=chunk) [Item 4. Controls and Procedures](index=59&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls and procedures were effective as of June 30, 2025, with no material changes in internal control - Management, including the principal executive officer and principal financial officer, concluded that disclosure controls and procedures were effective as of June 30, 2025, at the reasonable assurance level[215](index=215&type=chunk) - There have been no material changes in internal control over financial reporting during the quarter ended June 30, 2025[216](index=216&type=chunk) [PART II. OTHER INFORMATION](index=60&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=60&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in legal proceedings expected to have a material adverse effect on its business - The company is not currently a party to any litigation or legal proceedings that are probable to have a material adverse effect on its business[219](index=219&type=chunk) [Item 1A. Risk Factors](index=60&type=section&id=Item%201A.%20Risk%20Factors) Outlines risks and uncertainties impacting Rapport Therapeutics, covering operations, finances, product development, third parties, and intellectual property [Risks Related to Our Limited Operating History, Financial Condition and Need for Additional Capital](index=60&type=section&id=Risks%20Related%20to%20Our%20Limited%20Operating%20History,%20Financial%20Condition%20and%20Need%20for%20Additional%20Capital) Highlights risks associated with the company's early stage, recurring losses, and substantial future funding requirements - As a clinical-stage biotechnology company formed in February 2022, Rapport Therapeutics has a limited operating history and has not generated any revenue from product sales[222](index=222&type=chunk) - The company has incurred significant net losses since inception, totaling **$50.8 million** for the six months ended June 30, 2025, and an accumulated deficit of **$174.5 million**[223](index=223&type=chunk) - Additional funding will be required to finance operations and product development, and the inability to raise capital on acceptable terms could force delays or elimination of programs[227](index=227&type=chunk)[231](index=231&type=chunk) - Raising additional capital through equity or convertible debt securities will dilute existing stockholders' ownership interests[232](index=232&type=chunk) - Obligations under the Janssen License, including substantial contingent milestone payments, could be a significant drain on cash resources or necessitate incurring debt[238](index=238&type=chunk) [Risks Related to Our Business](index=66&type=section&id=Risks%20Related%20to%20Our%20Business) Covers risks concerning product development, resource allocation, acquisitions, data security, and the use of new technologies - The business is highly dependent on the successful clinical development, regulatory approval, and commercialization of product candidates, particularly RAP-219 for focal onset seizures[239](index=239&type=chunk) - The successful development of pharmaceutical products is a lengthy, expensive, and highly uncertain process, with many product candidates failing to reach the market[242](index=242&type=chunk) - The FDA placed a clinical hold on the Phase 2a proof-of-concept trial of RAP-219 for diabetic peripheral neuropathic pain (DPNP) in Q4 2024, requesting additional information[242](index=242&type=chunk) - Due to significant resource requirements, the company must prioritize certain product candidates, potentially diverting resources from more profitable opportunities[248](index=248&type=chunk)[249](index=249&type=chunk) - Growth through acquisitions or investments in new businesses, products, or technologies carries numerous risks, including integration difficulties, unanticipated costs, and diversion of management attention[250](index=250&type=chunk)[251](index=251&type=chunk) - The company is subject to stringent and evolving privacy and data security laws (e.g., CCPA, GDPR, NIS 2), with potential for significant fines, penalties, and business disruption from non-compliance or breaches[253](index=253&type=chunk)[254](index=254&type=chunk)[260](index=260&type=chunk)[262](index=262&type=chunk)[266](index=266&type=chunk)[270](index=270&type=chunk) - Information technology systems and data are vulnerable to cyber-attacks, cybersecurity incidents, and breaches, which could result in substantial costs, revenue loss, and operational disruption[272](index=272&type=chunk)[278](index=278&type=chunk) - The use of new technologies like AI/ML introduces risks related to intellectual property, compliance with evolving regulations (e.g., EU AI Act), and potential for reputational harm or liability[281](index=281&type=chunk)[282](index=282&type=chunk) [Risks Related to the Discovery and Development of Our Current or Future Product Candidates](index=81&type=section&id=Risks%20Related%20to%20the%20Discovery%20and%20Development%20of%20Our%20Current%20or%20Future%20Product%20Candidates) Addresses challenges in regulatory approval, clinical trials, potential side effects, market acceptance, patient enrollment, and reliance on third-party manufacturing - The regulatory approval processes of the FDA, EMA, MHRA, and other authorities are lengthy, time-consuming, and inherently unpredictable, with no guarantee of approval[285](index=285&type=chunk) - Clinical trials are expensive, difficult to design, and have a high failure rate; positive results from early stages may not be replicated in later-stage trials[286](index=286&type=chunk)[293](index=293&type=chunk)[295](index=295&type=chunk) - Product candidates may cause undesirable side effects or have other properties that could delay or prevent regulatory approval or limit commercialization[303](index=303&type=chunk)[305](index=305&type=chunk) - Even if approved, product candidates may fail to achieve sufficient market acceptance by physicians, patients, and third-party payors, hindering commercial success[322](index=322&type=chunk) - Concentrating R&D efforts on nervous system disorders presents challenges due to reliance on subjective patient-reported outcomes and the placebo effect[315](index=315&type=chunk) - Difficulties in enrolling patients in clinical trials, influenced by eligibility criteria, competition, and patient drop-out rates, could significantly delay or adversely affect development activities[317](index=317&type=chunk)[318](index=318&type=chunk)[321](index=321&type=chunk) - Reliance on third parties (CROs, CMOs, NeuroPace) for clinical trials and manufacturing introduces risks related to control, timing, expense, quality, and compliance with regulatory requirements[351](index=351&type=chunk)[353](index=353&type=chunk)[354](index=354&type=chunk)[358](index=358&type=chunk)[361](index=361&type=chunk)[366](index=366&type=chunk) - Geopolitical events and trade policies, particularly concerning manufacturers located outside the United States (e.g., China), could disrupt supply chains and increase costs[365](index=365&type=chunk) - Establishing collaborations on commercially reasonable terms is challenging, and reliance on collaborators' efforts and resources poses risks to product development and commercialization[370](index=370&type=chunk)[371](index=371&type=chunk)[376](index=376&type=chunk) - Scaling up manufacturing production or changes in product candidate manufacturing methods/formulation may result in additional costs or delays[379](index=379&type=chunk)[380](index=380&type=chunk) [Risks Related to Government Regulation](index=111&type=section&id=Risks%20Related%20to%20Government%20Regulation) Examines regulatory approval complexities, ongoing compliance, expedited pathways, funding impacts, healthcare laws, reimbursement, and international regulations - Obtaining regulatory approval in one jurisdiction does not guarantee approval in others, and foreign regulatory requirements can be more extensive and time-consuming[381](index=381&type=chunk)[382](index=382&type=chunk) - Approved products are subject to ongoing regulatory obligations, including manufacturing, labeling, safety reporting, and compliance with cGMP and GCP, with potential penalties for non-compliance[383](index=383&type=chunk)[386](index=386&type=chunk) - While the company may seek expedited approval pathways (e.g., Fast Track, Breakthrough Therapy), there is no assurance of obtaining such designations or realizing their intended benefits[389](index=389&type=chunk)[390](index=390&type=chunk)[391](index=391&type=chunk)[392](index=392&type=chunk) - Accelerated approval pathways require confirmatory trials, and failure to verify clinical benefit or comply with post-marketing requirements could lead to withdrawal of approval[395](index=395&type=chunk)[396](index=396&type=chunk) - Inadequate funding for the FDA or other government agencies could hinder their ability to review and approve new products in a timely manner, negatively impacting the business[398](index=398&type=chunk)[401](index=401&type=chunk) - Relationships with healthcare providers and payors are subject to anti-kickback, fraud and abuse, and other healthcare laws, risking criminal sanctions, civil penalties, and reputational harm[403](index=403&type=chunk)[405](index=405&type=chunk) - Coverage and adequate reimbursement from third-party payors are critical for product success, but may be limited or unavailable, making profitable sales difficult[406](index=406&type=chunk)[408](index=408&type=chunk)[409](index=409&type=chunk) - Ongoing healthcare legislative and regulatory reforms, such as the Inflation Reduction Act and OBBBA, could reduce Medicare/Medicaid spending, impact drug pricing, and adversely affect revenue and commercial prospects[414](index=414&type=chunk)[415](index=415&type=chunk)[416](index=416&type=chunk)[418](index=418&type=chunk) - Employee misconduct or off-label use of product candidates could lead to regulatory sanctions, product liability suits, and reputational harm[422](index=422&type=chunk)[423](index=423&type=chunk) - EU drug marketing and reimbursement regulations, including governmental price controls and anti-bribery laws, could materially affect the ability to market and receive coverage for products[424](index=424&type=chunk)[425](index=425&type=chunk)[428](index=428&type=chunk) - The company is subject to export/import controls, economic sanctions, and anti-corruption laws (e.g., FCPA), with potential for criminal liability and other serious consequences for violations[429](index=429&type=chunk) - Failure to comply with environmental, health, and safety laws by the company or third-party manufacturers could result in fines, penalties, or liabilities[430](index=430&type=chunk)[433](index=433&type=chunk) [Risks Related to Our Intellectual Property](index=126&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) Details risks concerning in-licensed IP, patent protection, challenges, foreign laws, term extensions, infringement claims, trade secrets, and trademarks - The company depends on in-licensed intellectual property, and failure to comply with license obligations could lead to the loss of important rights[435](index=435&type=chunk) - Inability to obtain and maintain broad patent protection for product candidates, or challenges to existing patents, could allow competitors to commercialize similar products[437](index=437&type=chunk)[438](index=438&type=chunk)[439](index=439&type=chunk)[443](index=443&type=chunk) - Changes to patent law in the United States and foreign jurisdictions could diminish the value of patents and impair the ability to protect product candidates[455](index=455&type=chunk)[456](index=456&type=chunk) - The company may not be able to protect its intellectual property rights throughout the world, as foreign laws may offer less extensive protection[457](index=457&type=chunk)[459](index=459&type=chunk) - Failure to obtain patent term extension for product candidates could allow competitors to enter the market sooner[460](index=460&type=chunk) - Third parties may initiate legal proceedings alleging infringement or misappropriation of their intellectual property, leading to substantial damages, injunctions, or costly licensing requirements[461](index=461&type=chunk)[462](index=462&type=chunk)[465](index=465&type=chunk) - Intellectual property litigation is expensive, time-consuming, and can divert management's attention, potentially harming the business[471](index=471&type=chunk) - Inability to protect the confidentiality of trade secrets would harm the business and competitive position[473](index=473&type=chunk)[474](index=474&type=chunk) - Claims challenging inventorship or ownership of patents and other intellectual property could be expensive and time-consuming[472](index=472&type=chunk) - Inadequate protection of trademarks and trade names could hinder name recognition and adversely affect the business[476](index=476&type=chunk) - European patents and applications could be challenged in the Unified Patent Court (UPC), potentially leading to a loss of patent protection across multiple European countries[477](index=477&type=chunk) [Risks Related to Ownership of Our Common Stock](index=138&type=section&id=Risks%20Related%20to%20Ownership%20of%20Our%20Common%20Stock) Addresses stock price volatility, fluctuating operating results, analyst coverage, significant stockholder control, dilution, dividend policy, and anti-takeover provisions - The trading price of the common stock may be volatile due to factors such as clinical trial results, regulatory decisions, competition, and general market conditions[480](index=480&type=chunk)[481](index=481&type=chunk) - Quarterly and annual operating results may fluctuate significantly, making future predictions difficult and potentially causing results to fall below expectations[482](index=482&type=chunk) - Inaccurate or unfavorable research, or cessation of coverage by securities or industry analysts, could lead to a decline in stock price and trading volume[484](index=484&type=chunk) - Executive officers, directors, and principal stockholders own a significant percentage of common stock, allowing them to exert substantial control over corporate actions[487](index=487&type=chunk) - Sales of a substantial number of shares of common stock in the public market could cause the stock price to fall[488](index=488&type=chunk) - Future issuances of additional capital stock in connection with financings, acquisitions, or incentive plans will dilute existing stockholders' ownership interests[492](index=492&type=chunk) - The company does not currently intend to pay dividends on its common stock, meaning investment return will depend solely on stock price appreciation[493](index=493&type=chunk) - Provisions in corporate charter documents and Delaware law could make an acquisition of the company more difficult and prevent attempts to replace current directors and management[494](index=494&type=chunk)[495](index=495&type=chunk) - Bylaw provisions designating specific courts as the sole forum for certain actions may limit stockholders' ability to choose favorable judicial forums[496](index=496&type=chunk)[497](index=497&type=chunk) - Failure to continue satisfying Nasdaq listing requirements could lead to delisting, impairing stockholders' ability to trade common stock and affecting its market price[500](index=500&type=chunk) [Other General Risks](index=148&type=section&id=Other%20General%20Risks) Covers risks from global economic conditions, business interruptions, public company operations, internal controls, tax law changes, and various litigations - Unfavorable global economic conditions, political instability, and geopolitical events (e.g., conflicts, trade tensions, inflation) could adversely affect the business, financial condition, and stock price[501](index=501&type=chunk)[502](index=502&type=chunk)[503](index=503&type=chunk) - Natural disasters, public health crises, or other business interruptions could severely disrupt operations and delay clinical trials or regulatory approvals[504](index=504&type=chunk) - The company's status as an 'emerging growth company' and 'smaller reporting company' with reduced reporting requirements may make its common stock less attractive to investors[505](index=505&type=chunk)[506](index=506&type=chunk) - Operating as a public company incurs significant legal, accounting, and compliance costs, diverting management time and potentially increasing net loss[511](index=511&type=chunk)[512](index=512&type=chunk) - Failure to establish and maintain an effective system of internal control over financial reporting could lead to inaccurate financial reporting, fraud, and loss of investor confidence[513](index=513&type=chunk)[515](index=515&type=chunk) - The ability to use net operating loss carryforwards and other tax attributes may be limited by ownership changes or regulatory changes, even if profitability is achieved[517](index=517&type=chunk)[518](index=518&type=chunk) - Changes in tax law, such as the OBBBA or Section 174 of the Code, could adversely affect the business and financial condition[519](index=519&type=chunk) - Clinical trial and product liability lawsuits could divert resources, result in substantial liabilities, and limit commercialization of any developed products[520](index=520&type=chunk)[521](index=521&type=chunk) - General litigation, including securities class action lawsuits, could be expensive, divert management's attention, and harm the business[522](index=522&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=157&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) Confirms no unregistered equity sales and no material changes to IPO and private placement proceeds use - No unregistered sales of equity securities occurred during the period[524](index=524&type=chunk) - There has been no material change in the planned use of proceeds from the initial public offering and concurrent private placement[525](index=525&type=chunk) [Item 3. Defaults Upon Senior Securities](index=157&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) Reports no defaults upon senior securities during the period - No defaults upon senior securities were reported[527](index=527&type=chunk) [Item 4. Mine Safety Disclosures](index=157&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the registrant - This item is not applicable to the registrant[528](index=528&type=chunk) [Item 5. Other Information](index=157&type=section&id=Item%205.%20Other%20Information) No directors or officers adopted, modified, or terminated Rule 10b5-1 or non-Rule 10b5-1 trading arrangements - None of the directors or officers adopted, modified, or terminated a Rule 10b5-1 trading arrangement or non-Rule 10b5-1 trading arrangement during the quarter ended June 30, 2025[529](index=529&type=chunk) [Item 6. Exhibits](index=158&type=section&id=Item%206.%20Exhibits) Lists all exhibits filed as part of the Form 10-Q, including corporate governance documents, agreements, and certifications - Key exhibits filed include the Third Amended and Restated Certificate of Incorporation, Amended and Restated Bylaws, Specimen Common Stock Certificate, Amended and Restated Investors' Rights Agreement, and a Lease Agreement[531](index=531&type=chunk) - Certifications from the Principal Executive Officer and Principal Financial Officer pursuant to the Securities Exchange Act of 1934 and Sarbanes-Oxley Act of 2002 are included[531](index=531&type=chunk) [Signatures](index=159&type=section&id=Signatures) Contains required signatures of the CEO and CFO, certifying the accuracy and completeness of the quarterly report - The report is duly signed on August 7, 2025, by Abraham N. Ceesay, Chief Executive Officer and Director, and Troy Ignelzi, Chief Financial Officer (Principal Financial and Accounting Officer)[537](index=537&type=chunk)

```markdown [Executive Summary](index=1&type=section&id=Executive%20Summary) [Company Introduction](index=1&type=section&id=Company%20Introduction) Rapport Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing small molecule precision medicines for neurological or psychiatric disorders - Rapport Therapeutics is a clinical-stage biotechnology company specializing in **small molecule precision medicines** for neurological or psychiatric disorders[2](index=2&type=chunk) [Key Highlights](index=1&type=section&id=Key%20Highlights) The company reported progress on its lead program RAP-219, with topline results for the Phase 2a trial in focal onset seizures expected in September 2025 and initiation of a Phase 2 trial in bipolar mania. Rapport ended the quarter with $260.4 million in cash, expected to fund operations through the end of 2026 - Topline results for RAP-219 Phase 2a trial in focal onset seizures are on track for **September 2025**[3](index=3&type=chunk)[4](index=4&type=chunk) - Phase 2 trial of RAP-219 in bipolar mania has been initiated and is enrolling patients, with topline results anticipated in the **first half of 2027**[3](index=3&type=chunk)[4](index=4&type=chunk) Cash Position and Runway | Metric | Value | | :-------------------------------- | :------------------- | | Cash, cash equivalents, and short-term investments (as of June 30, 2025) | $260.4 million | | Expected cash runway | Through the end of 2026 | [Business Highlights](index=2&type=section&id=BUSINESS%20HIGHLIGHTS) [RAP-219 Lead Program Updates](index=2&type=section&id=RAP-219%20Lead%20Program%20Updates) Rapport provided updates on the development of its lead program, RAP-219, including a consolidated Phase 1 safety summary and detailed expectations for the upcoming Phase 2a topline results in focal onset seizures - RAP-219 Phase 1 trials involved **100 healthy volunteers** across four studies, demonstrating differentiated tolerability with all treatment-emergent adverse events (TEAEs) being Grade 1 or 2, and no serious adverse events (SAEs)[6](index=6&type=chunk)[13](index=13&type=chunk) - The Phase 2a trial of RAP-219 in drug-resistant focal onset seizures is fully enrolled and on track for topline results in **September 2025**, utilizing intracranial electroencephalography (iEEG) data to assess effects on long episodes (LEs)[7](index=7&type=chunk) - Key endpoints for the Phase 2a topline data analysis include the proportion of patients achieving **≥30% reduction in LEs** and **≥50% reduction in clinical seizures**, along with TEAE incidence and grade[7](index=7&type=chunk) [Phase 1 Development Summary](index=2&type=section&id=RAP-219%20Phase%201%20Development%20Update) - Four Phase 1 trials (single ascending dose, two multiple ascending dose, and a multiple ascending dose human PET trial) have been conducted, exposing **100 healthy volunteers** to RAP-219[6](index=6&type=chunk) - Final aggregate data from multiple dose trials (n=64) showed all TEAEs were Grade 1 or 2, no SAEs, and no clinically significant laboratory, vital signs, or ECG abnormalities. Most common TEAEs included headache, dry mouth, brain fog, and fatigue[13](index=13&type=chunk) [Phase 2a Focal Onset Seizure Trial](index=2&type=section&id=Topline%20Results%20for%20RAP-219%20Phase%202a%20Focal%20Onset%20Seizure%20Trial%20Expected%20in%20September%202025) - The Phase 2a trial for drug-resistant focal onset seizures is fully enrolled, with topline results anticipated in **September 2025**[7](index=7&type=chunk) - The trial uses iEEG data from the RNS System to assess RAP-219's effect on long episodes (LEs), an objective biomarker correlating with clinical seizures[7](index=7&type=chunk) - Primary endpoint analysis will focus on the proportion of patients achieving **≥30% reduction in LEs** and median percent change in LE frequency. Key secondary endpoints include **≥50% reduction in clinical seizures** and median percent change in clinical seizure frequency[7](index=7&type=chunk) [Clinical Program Updates by Indication](index=3&type=section&id=Clinical%20Program%20Updates%20by%20Indication) Rapport provided updates on RAP-219's progress across various indications, including focal onset seizures, bipolar disorder, and peripheral neuropathic pain, highlighting key milestones and regulatory status [Focal Onset Seizures](index=3&type=section&id=Focal%20Onset%20Seizures) - Topline results for the Phase 2a trial in drug-resistant focal onset seizures are expected in **September 2025**[8](index=8&type=chunk) [Bipolar Disorder](index=3&type=section&id=Bipolar%20Disorder) - A Phase 2 trial for RAP-219 in bipolar mania has been initiated and is enrolling patients, following FDA acceptance of the IND application[9](index=9&type=chunk) - Topline results for the bipolar mania trial are expected in the **first half of 2027**[9](index=9&type=chunk) [Peripheral Neuropathic Pain](index=3&type=section&id=Peripheral%20Neuropathic%20Pain) - Plans for a Phase 2a trial in Diabetic Peripheral Neuropathic Pain (DPNP) are being finalized, with an update on the timeline expected in **2025**[10](index=10&type=chunk) - The IND for RAP-219 in DPNP was placed on clinical hold in Q4 2024, with the FDA requesting additional information and protocol design amendments[10](index=10&type=chunk) [Corporate Updates](index=3&type=section&id=CORPORATE%20UPDATES) Rapport Therapeutics hosted its inaugural Investor and Analyst Day in June 2025, featuring presentations from executives and a leading epilepsy expert - Rapport hosted its inaugural Investor and Analyst Day on **June 2, 2025**, including presentations by key executives and a fireside chat with epilepsy KOL, Jacqueline A. French, M.D[11](index=11&type=chunk) [Second Quarter 2025 Financial Results](index=3&type=section&id=SECOND%20QUARTER%202025%20FINANCIAL%20RESULTS) [Overview of Financial Performance](index=3&type=section&id=Overview%20of%20Financial%20Performance) Rapport Therapeutics reported an increased net loss for the second quarter of 2025 compared to the prior year period, primarily driven by higher operating expenses Net Loss (Q2 2025 vs Q2 2024) | Metric | Q2 2025 | Q2 2024 | | :------- | :-------- | :-------- | | Net Loss | $(26.7) million | $(18.1) million | [Operating Expenses](index=3&type=section&id=Operating%20Expenses) Both Research and Development (R&D) and General and Administrative (G&A) expenses increased in Q2 2025, reflecting increased clinical development activities, pipeline progression, business growth, and costs associated with operating as a public company [Research and Development (R&D) Expenses](index=3&type=section&id=Research%20and%20Development%20(R%26D)%20Expenses) R&D Expenses (Q2 2025 vs Q2 2024) | Metric | Q2 2025 | Q2 2024 | | :------- | :-------- | :-------- | | R&D Expense | $22.7 million | $15.7 million | - The increase in R&D expense was primarily due to operational costs related to clinical development and supporting the progression of the Company's overall pipeline[14](index=14&type=chunk)[15](index=15&type=chunk) [General and Administrative (G&A) Expenses](index=4&type=section&id=General%20and%20Administrative%20(G%26A)%20Expenses) G&A Expenses (Q2 2025 vs Q2 2024) | Metric | Q2 2025 | Q2 2024 | | :------- | :-------- | :-------- | | G&A Expense | $6.8 million | $5.1 million | - The increase in G&A expense was primarily driven by costs associated with business growth and the requirements of operating as a public company[19](index=19&type=chunk) [Cash Position and Runway](index=4&type=section&id=Cash%20Position) Rapport's cash, cash equivalents, and short-term investments decreased to $260.4 million by the end of Q2 2025, primarily due to operating cash outflows, but are expected to fund operations through the end of 2026 Cash, Cash Equivalents, and Short-Term Investments | Metric | June 30, 2025 | March 31, 2025 | | :-------------------------------- | :-------------- | :------------- | | Cash, cash equivalents, and short-term investments | $260.4 million | $285.4 million | - The decrease in cash position was primarily due to cash outflows from operating activities in Q2 2025[19](index=19&type=chunk) - The Company expects its current cash position to fund operating expenses and capital expenditure requirements through the **end of 2026**[19](index=19&type=chunk) [About Rapport Therapeutics & RAP-219](index=4&type=section&id=About%20Rapport%20Therapeutics%20%26%20RAP-219) [About RAP-219](index=4&type=section&id=About%20RAP-219) RAP-219 is a clinical-stage AMPA receptor (AMPAR) negative allosteric modulator (NAM) designed for neuroanatomical specificity by selectively targeting TARPγ8, which is expressed in brain regions relevant to focal seizures, bipolar disorder, and peripheral neuropathic pain - RAP-219 is an AMPAR NAM that selectively targets TARPγ8, a receptor associated protein, to achieve neuroanatomical specificity[16](index=16&type=chunk) - TARPγ8 is expressed in discrete regions like the hippocampus and neocortex, where focal seizures originate, with minimal expression in the hindbrain, potentially offering a differentiated tolerability profile[16](index=16&type=chunk) - RAP-219 is being evaluated as a potential treatment for focal onset seizures, bipolar disorder, and peripheral neuropathic pain, demonstrating 'pipeline-in-a-product' potential[16](index=16&type=chunk) [About Rapport Therapeutics](index=4&type=section&id=About%20Rapport%20Therapeutics) Rapport Therapeutics is a clinical-stage biotechnology company leveraging its proprietary RAP technology platform to develop precision small molecule medicines for neurological or psychiatric disorders, with RAP-219 as its lead investigational drug and additional preclinical programs underway - Rapport Therapeutics is a clinical-stage biotechnology company focused on precision medicines for neurological or psychiatric disorders, built on pioneering discoveries related to receptor associated proteins (RAPs)[18](index=18&type=chunk) - The Company's RAP technology platform enables a differentiated approach to generate precision small molecule product candidates[18](index=18&type=chunk) - Rapport's pipeline includes RAP-219 for focal onset seizures, bipolar mania, and diabetic peripheral neuropathic pain, as well as other preclinical and late-stage discovery programs targeting chronic pain and hearing disorders[18](index=18&type=chunk)[20](index=20&type=chunk) [Forward-Looking Statements & Contact](index=5&type=section&id=Forward-Looking%20Statements%20%26%20Contact) [Forward-Looking Statements](index=5&type=section&id=Forward-Looking%20Statements) This section contains standard forward-looking statements regarding the clinical development of RAP-219, the potential of Rapport's technology platform, and financial expectations, subject to various risks and uncertainties - The press release includes forward-looking statements concerning the clinical development of RAP-219 for focal onset seizures, bipolar mania, and DPNP, including trial timing, progress, and results[21](index=21&type=chunk) - Statements also cover expectations for RAP-219's activity, tolerability, commercial potential, the RAP technology platform, and Rapport's financial outlook, including its cash runway[21](index=21&type=chunk) - These statements are based on current expectations and are subject to risks and uncertainties that could materially affect business, operating results, financial condition, and stock value, as detailed in SEC filings[22](index=22&type=chunk) [Contact Information](index=5&type=section&id=Contact) Contact information for investor relations and communications is provided - Contact for Communications & IR: Julie DiCarlo, jdicarlo@rapportrx.com[23](index=23&type=chunk) [Financial Statements](index=6&type=section&id=Financial%20Statements) [Condensed Consolidated Balance Sheet Data](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheet%20Data) The condensed consolidated balance sheet provides a snapshot of Rapport Therapeutics' financial position as of June 30, 2025, compared to December 31, 2024, showing changes in assets, liabilities, and stockholders' equity Condensed Consolidated Balance Sheet Data (Unaudited, In thousands) | | June 30, 2025 | December 31, 2024 | | :------------------------------------------ | :-------------- | :---------------- | | **Assets** | | | | Cash and cash equivalents | $55,067 | $56,805 | | Short-term investments | 205,380 | 248,475 | | Total current assets | 270,242 | 309,802 | | Total assets | $285,495 | $314,933 | | **Liabilities, Convertible Preferred Stock and Stockholders' Equity** | | | | Total current liabilities | 11,879 | 8,767 | | Total liabilities | 21,983 | 9,506 | | Total stockholders' equity | 263,512 | 305,427 | | Total liabilities, convertible preferred stock, and stockholders' equity | $285,495 | $314,933 | [Condensed Consolidated Statement of Operations](index=7&type=section&id=Condensed%20Consolidated%20Statement%20of%20Operations) The condensed consolidated statement of operations details Rapport Therapeutics' financial performance for the three months ended June 30, 2025, compared to the same period in 2024, highlighting operating expenses, loss from operations, and net loss Condensed Consolidated Statement of Operations (Unaudited, In thousands, except share and per share data) | | For the three months ended June 30, 2025 | For the three months ended June 30, 2024 | | :------------------------------------------ | :--------------------------------------- | :--------------------------------------- | | Operating expenses | | | | Research and development | $22,680 | $15,689 | | General and administrative | 6,816 | 5,111 | | Total operating expenses | 29,496 | 20,800 | | Loss from operations | (29,496) | (20,800) | | Interest income | 2,764 | 2,679 | | Net loss | $(26,732) | $(18,121) | | Net loss per share attributable to common stockholders, basic and diluted | $(0.75) | $(1.70) | | Weighted-average common shares outstanding, basic and diluted | 35,444,635 | 10,666,528 | [Condensed Consolidated Statements of Cash Flows](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) The condensed consolidated statements of cash flows present the cash movements from operating, investing, and financing activities for the three months ended June 30, 2025, compared to the prior year period Condensed Consolidated Statements of Cash Flows (Unaudited, In thousands) | | For the Three Months Ended June 30, 2025 | For the Three Months Ended June 30, 2024 | | :------------------------------------------ | :--------------------------------------- | :--------------------------------------- | | Net cash used in operating activities | $(25,070) | $(16,352) | | Net cash provided by (used in) investing activities | 22,596 | (107,104) | | Net cash (used in) provided by financing activities | (63) | 159,353 | | Net (decrease) increase in cash, cash equivalents and restricted cash | $(2,537) | $35,897 | ```

BOSTON and SAN DIEGO, Aug. 07, 2025 (GLOBE NEWSWIRE) -- Rapport Therapeutics, Inc. (Nasdaq: RAPP) ("Rapport" or the "Company"), a clinical-stage biotechnology company dedicated to the discovery and development of small molecule precision medicines for patients with neurological or psychiatric disorders, today reported financial results for the second quarter ending June 30, 2025, and provided a business update. "We remain on track to report topline results in September from our Phase 2a trial of RAP-219 in ...

Industry Overview - The biopharmaceuticals industry presents both exciting opportunities and significant risks for investors, with leading growth stocks often emerging from this sector due to substantial rallies following positive trial results or government drug approvals [1][2] - Many biopharma firms face constant threats of collapse due to insufficient revenue, making breakthrough developments critical for their survival [2] Investment Strategies - Investors can balance their risk tolerance by relying on Wall Street analysts who specialize in the healthcare sector, potentially identifying high-potential investments before they gain mainstream recognition [3] Company Highlights: Kymera Therapeutics - Kymera Therapeutics Inc. focuses on developing small-molecule therapeutics to selectively degrade disease-causing proteins, with positive Phase 1 trial results for its flagship drug KT-621, aimed at treating moderate to severe atopic dermatitis [4][6] - The stock forecast for Kymera indicates a 12-month price target of $59.82, representing a 25.65% upside from the current price of $47.61, with 17 out of 18 analysts rating it as a Buy [5][7] - Kymera benefits from a robust pipeline and a partnership with AI-powered biopharma developer Sanofi, along with substantial cash reserves expected to sustain operations through 2028 [7] Company Highlights: Vera Therapeutics - Vera Therapeutics Inc. specializes in treatments for immunological diseases, with its leading candidate atacicept recently completing a positive Phase 3 trial for IgAN, positioning the company for potential FDA approval and a commercial launch by 2026 [9][10] - The stock forecast for Vera suggests a 12-month price target of $65.00, indicating a 193.45% upside from the current price of $22.15, with 9 out of 10 analysts rating it as a Buy [10][12] Company Highlights: Rapport Therapeutics - Rapport Therapeutics Inc. develops medicines for neurological and psychiatric disorders, with its candidate RAP-219 showing positive trial data for refractory focal epilepsy and plans for trials in bipolar mania [13][14] - The stock forecast for Rapport indicates a 12-month price target of $32.67, representing a 191.41% upside from the current price of $11.21, with all four analysts rating it as a Buy [14][15]

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]

Summary of Rapport Therapeutics (RAPP) 2025 Conference Company Overview - **Company**: Rapport Therapeutics (RAPP) - **Industry**: Biotechnology, specifically in precision neuroscience and epilepsy treatment - **Key Personnel**: Abe Cisse (CEO), Choi Ignelzi (CFO) [1][2] Core Points and Arguments - **Vision**: Rapport aims to be a leading precision neuroscience company, focusing on receptor associated proteins to develop targeted therapies [4][5]. - **Lead Program**: RAP-two 19, an AMPA TARP gamma-eight modulator, targets specific brain areas to modulate AMPA receptors, aiming to reduce adverse effects (AEs) associated with traditional anti-seizure medications [5][6]. - **Clinical Trials**: - Currently conducting a Phase 2a proof of concept study for RAP-two 19 in focal epilepsy, fully enrolled with 30 patients, with data expected in September 2025 [7][8]. - Plans to explore RAP-two 19 in bipolar mania and peripheral neuropathic pain, with a long-acting injectable formulation also in development [8][9][10]. - **Differentiation**: RAP-two 19 is designed for once-daily dosing with a long half-life, aiming to minimize drug-drug interactions (DDIs) and improve patient adherence [12][14][15][16]. Important Data and Metrics - **Phase 1 Studies**: Completed four studies with 100 healthy volunteers, showing a favorable tolerability profile and high receptor occupancy (up to 85%) without significant sedation or motor impairment [23][24]. - **Efficacy Expectations**: The primary endpoint for the Phase 2a study is a 30% reduction in long episodes of seizures, with a target responder rate of at least 40% [38][39]. - **Safety Profile**: No serious adverse events (SAEs) reported; mild AEs observed were transient and resolved on their own [45][46]. Additional Insights - **Biomarker Utilization**: The study leverages an implantable RNS device to capture EEG activity, providing an objective measure of efficacy compared to traditional seizure diaries [28][30]. - **Regulatory Pathway**: The company plans to conduct two registrational trials for NDA submission, with the potential to start in 2026 [51][52]. - **Market Positioning**: The combination of efficacy, tolerability, and administration profile positions RAP-two 19 as a potentially best-in-class therapy for epilepsy [41][44]. Conclusion Rapport Therapeutics is advancing its lead program RAP-two 19 with a focus on precision neuroscience, aiming to address significant unmet needs in epilepsy treatment through innovative clinical trial designs and a differentiated safety profile. The upcoming data readout in September 2025 will be critical for the company's future development plans and market positioning.

Disclaimer This presentation contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identif ...

Summary of Rapport Therapeutics Investor and Analyst Day Company Overview - **Company**: Rapport Therapeutics - **Event**: Inaugural Investor and Analyst Day - **Focus**: Lead candidate RAP-two 19 for epilepsy treatment Key Points and Arguments Industry and Product Focus - **Lead Candidate**: RAP-two 19, a TARP gamma eight AMPA modulator, aimed at treating focal onset seizures in epilepsy [3][19] - **Market Size**: Focal epilepsy affects approximately 1.8 million patients in the U.S., with 30-40% being drug-resistant [32] - **Pipeline**: Fully enabled pipeline including RAP-two 19 and two late-stage discovery programs targeting chronic pain and hearing disorders [19][27] Clinical Trials and Results - **Phase Two Trial**: Ongoing trial for RAP-two 19 in focal onset seizure patients, fully enrolled with results expected in September [21][26] - **Phase One Results**: Completed four Phase I studies with 100 healthy volunteers; RAP-two 19 was safe and well-tolerated [52][53] - **Receptor Occupancy**: Achieved target receptor occupancy of 50-70% at lower doses than predicted, indicating potential for effective treatment [56][58] Scientific Insights - **Mechanism of Action**: RAP-two 19 selectively modulates AMPA receptors in specific brain regions, potentially reducing side effects associated with traditional anti-seizure medications [38][39] - **Preclinical Efficacy**: Demonstrated significant efficacy in animal models with a favorable safety profile compared to existing treatments [44][46] Management and Strategy - **Management Team**: Comprised of experienced professionals in neuroscience and drug development, emphasizing a strategic approach to building the company [15][17] - **Vision**: To create a leading precision neuroscience company, addressing the limitations of conventional small molecules in treating neurological disorders [24][25] Additional Important Content - **Unmet Needs**: Despite numerous treatment options, the number of treatment-resistant epilepsy patients remains unchanged, highlighting the need for innovative therapies [92][93] - **Patient Population**: The trial population is representative of refractory focal epilepsy patients, enhancing the translatability of results to broader populations [87] - **Long-Acting Injectable**: Potential development of a long-acting injectable formulation for RAP-two 19, which could transform treatment for epilepsy patients [29][30] Conclusion Rapport Therapeutics is positioned to address significant unmet needs in the epilepsy treatment landscape with its innovative lead candidate RAP-two 19, backed by a robust clinical and scientific foundation. The company's strategic focus on precision neuroscience and the experienced management team further enhance its potential for success in the market.

Core Viewpoint - Rapport Therapeutics is advancing its Phase 2a trial of RAP-219 in refractory focal epilepsy, which is fully enrolled and expected to report topline results in September 2025, marking a significant milestone for the company and its precision neuroscience approach [3][4][10] Group 1: Clinical Trial Updates - The Phase 2a trial of RAP-219 is designed with input from leading epilepsy experts and utilizes intracranial electroencephalography (iEEG) data to assess the drug's potential effect on long episodes (LEs), a biomarker correlated with clinical seizures (CSs) [4] - Preliminary baseline characteristics of the first 14 enrolled patients indicate that the trial population is representative of historical registrational refractory focal epilepsy trials, with a median age of 37 years and a balanced sex distribution of 7 females and 7 males [5][6] - The company anticipates providing detailed analyses of primary and secondary endpoints, treatment-emergent adverse events (TEAEs), and a consolidated safety summary from Phase 1 trials when topline results are reported [9][12] Group 2: Financial and Operational Insights - As of March 31, 2025, Rapport reported $285.4 million in cash, cash equivalents, and short-term investments, which are expected to support operations through the end of 2026 [10] - The company has conducted four Phase 1 trials involving 100 healthy volunteers, reinforcing RAP-219's differentiated tolerability profile [9][10] Group 3: Future Catalysts - Upcoming milestones include the topline readout for RAP-219 in focal epilepsy in September 2025, initiation of a Phase 2a trial in bipolar mania in Q3 2025, and updates on the diabetic peripheral neuropathic pain program in the second half of 2025 [13]

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 two upcoming investor conferences, providing opportunities for engagement with potential investors [1][2] Company Overview - Rapport Therapeutics specializes in discovering and developing small molecule 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 the treatment of refractory focal epilepsy, 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]