Tenaya Therapeutics(TNYA) - 2025 Q2 - Quarterly Results

Executive Summary & Company Overview Tenaya Therapeutics announced Q2 2025 results, detailing clinical progress for gene therapies TN-201 and TN-401, and maintaining a cash runway into the second half of 2026 Introduction and Key Highlights Tenaya Therapeutics announced its Q2 2025 financial results and provided a business update, highlighting significant progress in its gene therapy clinical trials for MYBPC3-associated HCM (TN-201) and PKP2-associated ARVC (TN-401), with positive DSMB reviews enabling dose escalation and expansion; the company anticipates key data readouts for both programs in Q4 2025 and maintains a cash runway into the second half of 2026 - Enrollment completed in Cohorts 1 and 2 of MyPEAK™-1 Phase 1b/2 trial for TN-201 (MYBPC3-associated HCM); positive DSMB safety review enables enrollment of expansion cohorts[1][4] - Cohort 1 of RIDGE™-1 Phase 1b trial for TN-401 (PKP2-associated ARVC) enrolled, and first patient dosed in Cohort 2 following DSMB recommendation for dose escalation and expansion[1][4] - Data readouts from both TN-201 and TN-401 clinical programs are planned for the fourth quarter of 2025[1][4] - Tenaya Therapeutics has a cash runway into the second half of 2026[2][14] About Tenaya Therapeutics Tenaya Therapeutics is a clinical-stage biotechnology company dedicated to discovering, developing, and delivering curative therapies for heart disease, leveraging integrated internal capabilities for target validation, capsid engineering, and manufacturing - Tenaya Therapeutics is a clinical-stage biotechnology company focused on discovering, developing, and delivering potentially curative therapies for the underlying causes of heart disease[12] - The company's pipeline includes clinical-stage gene therapies TN-201 (for MYBPC3-associated HCM) and TN-401 (for PKP2-associated ARVC), a clinical-stage small molecule TN-301 (HDAC6 inhibitor for heart failure), and multiple early-stage preclinical programs[12] Business and Program Updates Tenaya Therapeutics provided updates on its clinical-stage gene therapy programs TN-201 and TN-401, highlighting trial progress, safety reviews, and upcoming data readouts, alongside advancements in research and manufacturing capabilities TN-201 Gene Therapy for MYBPC3-Associated Hypertrophic Cardiomyopathy (HCM) Tenaya Therapeutics has completed enrollment and dosing for Cohort 2 of the MyPEAK™-1 Phase 1b/2 trial for TN-201; a positive DSMB review confirmed an acceptable safety profile, allowing for dose expansion; interim data from Cohort 1 showed promising results, including robust transduction, increased RNA/protein expression, and clinical improvements in patients with severe disease - Completed enrollment and dosing of three patients at the 6E13 vg/kg dose level (Cohort 2) in the MyPEAK™-1 Phase 1b/2 clinical trial of TN-201 in May[5] - Independent DSMB determined TN-201 had an acceptable safety profile, enabling enrollment of dose expansion cohorts, with Tenaya anticipating enrolling patients in the 6E13 vg/kg dose expansion cohort[5] - Interim data from Cohort 1 (3E13 vg/kg) showed robust transduction and RNA expression with increasing RNA and protein levels over time; all three patients with severe disease at baseline achieved New York Heart Association Class I, and two experienced improvement in hypertrophy measures[6] - The MyClimb pediatric non-interventional natural history study has enrolled over 200 patients at 29 sites worldwide to characterize MYBPC3-associated HCM in patients under 18[9] TN-401 Gene Therapy for PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) Enrollment for Cohort 1 of the RIDGE™-1 Phase 1b trial for TN-401 was completed in April 2025; following a positive DSMB recommendation, enrollment for a higher dose Cohort 2 (6E13 vg/kg) has begun, with the first patient dosed; data from the RIDGE natural history study highlighted the high burden of arrhythmia and progressive structural changes in PKP2-associated ARVC patients, alongside low AAV9 immunity, suggesting broad eligibility for TN-401 - Enrollment of the first cohort of three patients receiving TN-401 at a dose level of 3E13 vg/kg in the RIDGE-1 Phase 1b clinical trial was completed in April 2025[9] - In July 2025, the RIDGE-1 DSMB issued a positive recommendation for both enrollment of a higher dose cohort (6E13 vg/kg) and additional patients at the 3E13 vg/kg dose; Cohort 2 enrollment is underway, and the first patient has been dosed at 6E13 vg/kg[9] - Data from the RIDGE natural history study (191 participants) indicated a high burden of arrhythmia (83% >500 PVCs/day, 49% history of VT) despite standard care, evidence of progressive structural changes, and low levels of preexisting immunity to AAV9 antibodies, making most participants eligible for TN-401[9] Research and Manufacturing Capabilities Tenaya's Research and Manufacturing teams presented advancements in core capabilities at the American Society for Gene Therapy Annual Meeting, focusing on novel capsid engineering, genetic medicine design, and AAV gene therapy manufacturing - In May 2025, Tenaya's Research and Manufacturing teams presented posters at the American Society for Gene Therapy 28th Annual Meeting[8] - Presentations detailed efforts to advance core capabilities in novel capsid engineering, identification, design and optimization of cardiomyocyte-targeting genetic medicines, and manufacturing of AAV gene therapies[8] Upcoming Events and Data Readouts Tenaya Therapeutics plans to release initial Cohort 2 data and an update on Cohort 1 for TN-401 in Q4 2025, along with meaningful new data for both TN-201 and TN-401; an abstract for the MyClimb study has been accepted for presentation at the ESC Annual Meeting, and the company will host a Virtual Key Opinion Leader event in August 2025 - Tenaya anticipates releasing initial Cohort 2 data and an update on Cohort 1 for TN-401 in the fourth quarter of 2025[9] - Meaningful new data readouts for both TN-201 and TN-401 clinical programs are planned for the fourth quarter of 2025[4] - An abstract regarding Tenaya's pediatric non-interventional natural history study, MyClimb, has been accepted for presentation at the upcoming European Society of Cardiology (ESC) Annual Meeting (August 29-September 1)[9] - Tenaya plans to host a Virtual Key Opinion Leader event, 'Measuring Protein Expression in Cardiac Gene Therapy,' on August 19, 2025[14] Financial Results Tenaya Therapeutics reported a reduced net loss for Q2 2025 and H1 2025, driven by lower operating expenses, and maintained a cash position of $71.7 million, extending its runway into the second half of 2026 Second Quarter 2025 Financial Highlights Tenaya Therapeutics reported a net loss of $23.3 million for Q2 2025, an improvement from $29.4 million in Q2 2024, driven by reduced R&D and G&A expenses; the company ended the quarter with $71.7 million in cash, cash equivalents, and marketable securities, providing a cash runway into the second half of 2026 - Cash, cash equivalents and investments in marketable securities were $71.7 million as of June 30, 2025[14] - The company expects its current funds to support planned operations into the second half of 2026[14] Key Financial Highlights (Q2 2025 vs. Q2 2024) | Metric | Q2 2025 (in millions USD) | Q2 2024 (in millions USD) | Change (QoQ, millions USD) | | :----------------------- | :------------------------ | :------------------------ | :------------------------- | | Research & Development | $17.4 | $22.6 | -$5.2 | | General & Administrative | $6.7 | $8.2 | -$1.5 | | Net Loss | $(23.3) | $(29.4) | +$6.1 | | Net Loss Per Share | $(0.14) | $(0.34) | +$0.20 | Condensed Statements of Operations For the second quarter of 2025, Tenaya Therapeutics reported a net loss of $23.3 million, a decrease from $29.4 million in the prior year, primarily due to lower operating expenses; for the six months ended June 30, 2025, the net loss was $50.1 million, an improvement from $61.7 million in the same period of 2024 Condensed Statements of Operations (Three Months Ended June 30) | Metric | Q2 2025 (in thousands USD) | Q2 2024 (in thousands USD) | Change (QoQ, thousands USD) | | :----------------------- | :------------------------- | :------------------------- | :-------------------------- | | Research and development | $17,370 | $22,649 | -$5,279 | | General and administrative | $6,712 | $8,174 | -$1,462 | | Total operating expenses | $24,082 | $30,823 | -$6,741 | | Loss from operations | $(24,082) | $(30,823) | +$6,741 | | Interest income | $815 | $1,393 | -$578 | | Net loss | $(23,283) | $(29,431) | +$6,148 | | Net loss per share | $(0.14) | $(0.34) | +$0.20 | | Weighted-average shares | 162,791,579 | 85,706,501 | +77,085,078 | Condensed Statements of Operations (Six Months Ended June 30) | Metric | H1 2025 (in thousands USD) | H1 2024 (in thousands USD) | Change (YoY, thousands USD) | | :----------------------- | :------------------------- | :------------------------- | :-------------------------- | | Research and development | $38,446 | $47,704 | -$9,258 | | General and administrative | $13,174 | $16,881 | -$3,707 | | Total operating expenses | $51,620 | $64,585 | -$12,965 | | Loss from operations | $(51,620) | $(64,585) | +$12,965 | | Interest income | $1,449 | $2,845 | -$1,396 | | Net loss | $(50,147) | $(61,659) | +$11,512 | | Net loss per share | $(0.37) | $(0.74) | +$0.37 | | Weighted-average shares | 136,476,623 | 83,344,414 | +53,132,209 | Condensed Balance Sheet Data As of June 30, 2025, Tenaya Therapeutics reported an increase in cash, cash equivalents, and marketable securities to $71.7 million from $61.4 million at the end of 2024; total assets slightly increased, while total liabilities decreased over the same period Condensed Balance Sheet Data (As of June 30, 2025 vs. December 31, 2024) | Metric | June 30, 2025 (in thousands USD) | December 31, 2024 (in thousands USD) | Change (thousands USD) | | :--------------------------------- | :--------------------------------- | :--------------------------------- | :--------------------- | | Cash, cash equivalents and marketable securities | $71,667 | $61,446 | +$10,221 | | Total assets | $122,151 | $119,940 | +$2,211 | | Total liabilities | $22,323 | $27,086 | -$4,763 | | Total liabilities and stockholders' equity | $122,151 | $119,940 | +$2,211 | Legal and Contact Information This section provides standard disclaimers regarding forward-looking statements, outlining inherent risks and uncertainties, and lists key contact information for Tenaya Therapeutics Forward-Looking Statements This section provides a standard disclaimer regarding forward-looking statements, emphasizing that they are based on current expectations and involve inherent risks and uncertainties; it outlines various factors that could cause actual results to differ materially from projections, including clinical trial outcomes, regulatory approvals, funding, and market conditions - The press release contains forward-looking statements based on current expectations, involving assumptions, risks, and uncertainties that may cause actual results to differ[13][15] - Key risks include the timing and progress of clinical trials, unexpected safety events, potential failure of product candidates, differences from expected results, ability to obtain approvals, funding needs, reliance on third parties, competition, and intellectual property protection[15] - Tenaya assumes no obligation to update or revise any forward-looking statements, except as required by law[15] Tenaya Contacts This section provides contact information for Tenaya Therapeutics' corporate communications, investor relations, and media inquiries - Contact information is provided for Michelle Corral (VP, Corporate Communications and Investor Relations), Anne-Marie Fields (Precision AQ for Investors), and Wendy Ryan (Ten Bridge Communications for Media)[16]