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 |
Rapport Therapeutics, Inc.(RAPP) - 2025 Q2 - Quarterly Results