Financial Performance - Tenaya reported a net loss of $20.3 million, or $0.12 loss per share, for Q3 2025, compared to a net loss of $25.6 million, or $0.30 per share, in Q3 2024[10]. - For the three months ended September 30, 2025, total operating expenses were $20,936,000, a decrease from $26,711,000 in the same period of 2024, representing a reduction of approximately 21.6%[16]. - The net loss for the three months ended September 30, 2025, was $20,275,000, compared to a net loss of $25,634,000 for the same period in 2024, indicating an improvement of about 20.5%[16]. - The company reported a net loss per share of $0.12 for the three months ended September 30, 2025, compared to a net loss per share of $0.30 for the same period in 2024[16]. - Interest income for the three months ended September 30, 2025, was $658,000, compared to $1,080,000 for the same period in 2024, showing a decrease of approximately 39.0%[16]. - Total other income, net, for the three months ended September 30, 2025, was $661,000, down from $1,077,000 in the same period of 2024, reflecting a decline of about 38.7%[16]. Expenses - Research and Development (R&D) expenses were $15.4 million for Q3 2025, down from $20.4 million in the same period of 2024[10]. - General & Administrative (G&A) expenses decreased to $5.6 million in Q3 2025 from $6.4 million in Q3 2024[10]. - Research and development expenses for the nine months ended September 30, 2025, totaled $53,809,000, down from $68,054,000 in 2024, reflecting a decrease of approximately 21%[16]. Cash and Assets - As of September 30, 2025, Tenaya had cash, cash equivalents, and investments totaling $56.3 million, expected to support operations into the second half of 2026[10]. - Cash, cash equivalents, and marketable securities as of September 30, 2025, were $56,312,000, a decrease from $61,446,000 as of December 31, 2024[18]. - Total assets decreased to $104,980,000 as of September 30, 2025, down from $119,940,000 as of December 31, 2024, representing a decline of approximately 12.5%[18]. - Total liabilities decreased to $22,112,000 as of September 30, 2025, from $27,086,000 as of December 31, 2024, indicating a reduction of about 18.4%[18]. Clinical Trials and Research - The MyPEAK-1 trial for TN-201 gene therapy showed robust transduction and durable expression with dose-dependent increases in MyBP-C protein[4]. - Tenaya completed dosing in Cohort 2 of the RIDGE-1 trial for TN-401, with three patients enrolled at a dose of 6E13 vg/kg[5]. - A seroprevalence study indicated that nearly 95% of MYBPC3-associated HCM patients had low or absent pre-existing immunity to AAV9, meeting eligibility criteria for the MyPEAK-1 trial[6]. - The independent Data Safety Monitoring Board (DSMB) for the MyPEAK-1 trial confirmed an acceptable safety profile for TN-201, allowing for enrollment in dose expansion cohorts[6]. - Interim data from the MyClimb study indicated that 93% of pediatric participants had the nonobstructive HCM phenotype, with no approved treatment options available[6]. - Tenaya's ongoing clinical trials and research initiatives are focused on transformative treatments for serious genetic cardiomyopathies[3].

Core Insights - Tenaya Therapeutics announced significant advancements in its gene therapy programs, TN-201 and TN-401, aimed at treating serious genetic cardiomyopathies, with positive recommendations from Data Safety Monitoring Boards for both products [2][3]. Business and Program Updates - TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), showed robust transduction and durable expression, with a dose-dependent increase in MyBP-C protein. Cohort 1 patients exhibited decreases in circulating biomarkers and reductions in left ventricular hypertrophy over time [3]. - The MyPEAK-1 trial for TN-201 is currently on clinical hold, with the company working with the FDA to resolve the issue and resume dosing [4]. - TN-401, targeting PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), has completed dosing in Cohort 2 of the RIDGE-1 trial, with initial safety and biopsy data expected to be shared by year-end 2025 [5][6]. Research and Development Updates - A seroprevalence study indicated that nearly 95% of MYBPC3-associated HCM patients had low pre-existing immunity to AAV9, suggesting a favorable patient eligibility for the MyPEAK-1 trial [7]. - The MyClimb study, focusing on pediatric patients with MYBPC3-associated HCM, revealed that 93% of participants had the nonobstructive HCM phenotype, highlighting the need for treatment options [7]. - Tenaya presented new preclinical data on cardiac function improvement in a pig model of ischemic heart failure, achieved through a proprietary in vivo reprogramming cocktail [7]. Financial Highlights - As of September 30, 2025, Tenaya reported cash, cash equivalents, and marketable securities totaling $56.3 million, sufficient to support operations into the second half of 2026 [12]. - Research and Development (R&D) expenses for Q3 2025 were $15.4 million, down from $20.4 million in Q3 2024. General and Administrative (G&A) expenses also decreased to $5.6 million from $6.4 million in the same period [12][16]. - The net loss for Q3 2025 was $20.3 million, or $0.12 per share, compared to a net loss of $25.6 million, or $0.30 per share, in Q3 2024 [12][16].

Core Insights - Tenaya Therapeutics presented new interim safety and efficacy data for TN-201 during the AHA Scientific Sessions 2025, indicating promising results for patients with MYBPC3-associated hypertrophic cardiomyopathy (HCM) [1][3][7] Group 1: Clinical Trial Overview - The MyPEAK-1 Phase 1b/2a clinical trial is assessing the safety and efficacy of TN-201, a gene therapy for HCM, with two cohorts receiving different doses [9] - Cohort 1 patients showed consistent improvements in hypertrophy measures over a follow-up period of 52 to 78 weeks, while initial data from Cohort 2 indicated early dose-responsive increases in TN-201 transduction and MyBP-C protein expression [1][4][5] Group 2: Safety and Tolerability - TN-201 was generally well tolerated at both tested doses (3E13 vg/kg and 6E13 vg/kg), with no dose-limiting toxicities observed [2][6] - The most common treatment-related adverse events were reversible, asymptomatic liver enzyme elevations, with no signs of cardiotoxicities reported [6][12] Group 3: Efficacy Results - Significant reductions in cardiac biomarkers were observed, with Cardiac Troponin I levels declining by 48% to 74% in Cohort 1 patients, indicating improved cardiac health [6][5] - All patients in Cohort 1 experienced notable reductions in left ventricular posterior wall thickness (LVPWT) by 21% to 39% and improvements in NYHA classification, with all now classified as NYHA Class I [6][5][4] Group 4: Future Plans and Regulatory Status - Tenaya plans to continue long-term follow-up of patients and periodically report additional results to inform the late-stage development of TN-201 [6][8] - The FDA has placed the MyPEAK-1 trial on clinical hold, and Tenaya is working to address the agency's concerns [9][12]

Core Insights - Tenaya Therapeutics is set to present interim safety and efficacy results from the MyPEAK-1 Phase 1b/2a clinical trial for TN-201, a gene therapy targeting MYBPC3-associated hypertrophic cardiomyopathy, at the AHA 2025 Scientific Sessions [1][2] - The company will also showcase results from a preclinical study on cellular reprogramming gene therapy in a pig model of ischemic heart failure [1][2] Company Overview - Tenaya Therapeutics is a clinical-stage biotechnology company focused on developing potentially curative therapies for heart disease [4] - The company's pipeline includes TN-201 for MYBPC3-associated hypertrophic cardiomyopathy and TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy [4][5] - Tenaya employs integrated capabilities such as target validation and capsid engineering to develop novel medicines based on genetic insights [5] Upcoming Events - The AHA presentation will occur on November 8, 2025, featuring Dr. Milind Y Desai as the presenting author [2] - A second presentation on cellular reprogramming will take place on November 9, 2025, led by Kathy Ivey [2] - Tenaya management will host a conference call on November 10, 2025, to discuss the TN-201 data presented at the AHA sessions [3]

Summary of Tenaya Therapeutics FY Conference Call Industry Overview - The conference focused on the cardiac gene therapy industry, highlighting both advancements and setbacks in the field. [1] - There is a supportive regulatory environment for gene therapy, particularly in areas of high unmet medical need, alongside a growing momentum for precision medicine. [1] Company Insights Tenaya Therapeutics - Tenaya Therapeutics is advancing gene therapy for BAG3 dilated cardiomyopathy, targeting heart failure in younger patients. [4] - The company is utilizing a novel cardiotropic AAV capsid designed to transduce nearly 100% of cardiac cells at a significantly lower dose compared to conventional capsids. [17] - An Investigational New Drug (IND) application is expected to be filed later this quarter, with clinical results anticipated in early next year. [5] AskBio - AskBio is focusing on gene therapy for both common and rare diseases, with lead programs in Parkinson's disease and heart failure currently in Phase II trials. [7] - The company emphasizes the importance of local delivery methods to minimize doses and avoid immunosuppression. [31] - AskBio's global Phase II trial for congestive heart failure will involve over 80 patients, supported by Bayer's expertise. [25] Lexeo Therapeutics - Lexeo Therapeutics is developing gene therapies for rare cardiac diseases, including Friedreich's ataxia and arrhythmogenic cardiomyopathy. [9] - The company is utilizing non-invasive methods to measure therapeutic benefits, including cardiac MRI and various biomarkers. [10] - Lexeo is preparing for pivotal studies and aims to demonstrate clinical efficacy through established endpoints. [12] Key Points and Arguments - **Safety and Efficacy**: The panelists discussed the importance of low dosing in gene therapy to mitigate safety risks, with a consensus that lower doses correlate with fewer adverse events. [29][41] - **Regulatory Environment**: There is optimism regarding the FDA's flexibility in approving gene therapies, especially for rare diseases, with a focus on demonstrating clear clinical benefits. [69] - **Commercialization Challenges**: The need for successful commercial launches in gene therapy was emphasized, with a call for companies to prepare for market uptake and pricing strategies. [44][56] - **Manufacturing Improvements**: Advances in manufacturing processes are expected to lower costs and improve yields, making gene therapies more commercially viable. [52][55] Additional Important Insights - The discussion highlighted the evolving landscape of gene therapy, with a shift towards precision medicine in cardiovascular treatments, similar to trends seen in oncology. [58] - The panelists expressed confidence in the future of gene therapy, citing recent clinical successes and the potential for new products to emerge from ongoing research. [66] - The importance of patient selection and trial design was noted, as these factors significantly impact the success of clinical trials and the overall perception of gene therapy safety. [42] This summary encapsulates the key discussions and insights from the Tenaya Therapeutics FY Conference Call, providing a comprehensive overview of the current state and future outlook of the cardiac gene therapy industry.

Summary of Tenaya Therapeutics Conference Call (October 14, 2025) Company Overview - **Company**: Tenaya Therapeutics (NasdaqGS:TNYA) - **Focus**: Genetic medicine, specifically gene therapies for cardiomyopathies Key Assets - **TN-201**: Targets MYBPC3-positive hypertrophic cardiomyopathy (HCM) patients - **TN-401**: Targets PKP2-positive arrhythmogenic right ventricular cardiomyopathy (ARVC) patients - Both therapies utilize AAV9-based vectors and are currently in clinical trials [3][4][5] Core Points and Arguments TN-201 (HCM) - **Mechanism**: AAV9 vector delivering a full-length MYBPC3 gene with a cardiomyocyte-specific promoter [4] - **Unique Selling Proposition**: Addresses the underlying genetic cause of HCM, particularly for non-obstructive forms, which represent 70% of the patient population [9][10] - **Market Context**: Current therapies like Camzyos are not approved for non-obstructive HCM, highlighting a significant unmet need [10] - **Clinical Data**: Positive interim data from the MyPeak-1 study showed safety and tolerability, with two out of three patients experiencing significant improvements in cardiac biomarkers [14][15][18] - **Durability of Effect**: All patients in the first dose cohort reached New York Heart Association Class I, indicating no disease impact on daily living [18] TN-401 (ARVC) - **Mechanism**: AAV9 vector delivering a full-length PKP2 gene [29] - **Unmet Need**: ARVC is a severe condition with no approved therapies addressing its genetic cause, leading to high morbidity and mortality [30] - **Preclinical Data**: Demonstrated prevention of arrhythmia and cardiac remodeling in severe preclinical models [32] - **Clinical Trial**: Ongoing Phase 1b trial (RIDGE-1) with expected data release in Q4 2025 [33] Additional Important Insights - **Safety Database**: AAV9 has the largest safety database among AAV vectors, enhancing confidence in its use [6] - **Innovative Engineering**: The company has developed unique promoters and molecular designs to optimize gene delivery [4][5] - **Data Presentation**: Upcoming data at the AHA conference is highly anticipated, with a focus on safety and efficacy [22][20] - **Comparison with Peers**: The company is mindful of how its data compares with other gene therapy programs, emphasizing the importance of robust protein measurement methods [35][36] Conclusion - **Future Outlook**: Tenaya Therapeutics is positioned to address significant unmet needs in cardiomyopathy with its innovative gene therapies, backed by promising clinical data and a strong safety profile [13][20]

Core Insights - Tenaya Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing potentially curative therapies for heart disease [1][3] - The CEO, Faraz Ali, will participate in a fireside chat at the H.C. Wainwright Genetic Medicines Virtual Conference on October 14, 2025, with virtual 1x1 meetings scheduled for October 15, 2025 [1][2] Company Overview - Tenaya Therapeutics is dedicated to discovering and delivering therapies that target the underlying causes of heart disease [3] - The company's pipeline includes clinical-stage candidates such as TN-201 for MYBPC3-associated hypertrophic cardiomyopathy and TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy [3] - Tenaya employs integrated capabilities like target validation and capsid engineering to develop novel medicines, including TN-301, a small molecule HDAC6 inhibitor for heart failure [3]

Company Overview - Tenaya Therapeutics is a clinical-stage biotechnology company focused on discovering, developing, and delivering potentially curative therapies for heart disease [3] - The company's pipeline includes clinical-stage candidates TN-201 for MYBPC3-associated hypertrophic cardiomyopathy (HCM) and TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC) [3] - Tenaya employs integrated internal capabilities such as target validation, capsid engineering, and manufacturing to create novel medicines based on genetic insights [3] Upcoming Conferences - Tenaya's CEO, Faraz Ali, will participate in the Cell & Gene Meeting on the Mesa on October 6, with a panel discussion on emerging trends in cell and gene therapy development [1] - A company presentation is also scheduled for October 6 at the same conference [1] - Additionally, Tenaya will be part of the Chardan Annual Genetic Medicines Conference on October 21, focusing on in vivo gene therapies for genetic cardiac diseases [2]

Core Insights - Tenaya Therapeutics has had a strong start to the year and is on track to meet its guidance for the year [2] - The company successfully dosed the high-dose cohort in its 201 lead gene therapy program for the leading genetic cause of hypertrophic cardiomyopathy (HCM) and received DSMB clearance, indicating safety [2] - Tenaya Therapeutics has released data from the largest natural history study for children with a specific mutation related to this severe disease [2] - The company is expected to deliver additional data in the second half of the year, with a narrowed guidance to Q4 [3]

Summary of Tenaya Therapeutics FY Conference Call Company Overview - **Company**: Tenaya Therapeutics (NasdaqGS:TNYA) - **Industry**: Biotechnology, specifically focusing on gene therapy for cardiac diseases Key Points and Arguments Company Performance and Milestones - Tenaya Therapeutics reported a strong start to the year and is on track to meet its guidance for 2025 [4][6] - The company successfully dosed the high-dose cohort for its lead gene therapy program, TN-201, targeting hypertrophic cardiomyopathy (HCM) [4][6] - Data from the largest natural history study for HCM, involving 190 patients, is expected to be released in Q4 2025 [7][27] - The company has been accepted for a late-breaker clinical presentation at the American Heart Association (AHA) conference, which is a significant event for Tenaya [6][27] Regulatory Environment - The CEO emphasized a positive environment at the FDA for gene therapy, particularly for rare diseases, with bipartisan support for innovative medicines [10][11] - The FDA has introduced new pathways for rare diseases, which could benefit Tenaya's programs [11] Safety and Immunosuppression - Recent incidents involving deaths in other gene therapy studies raised concerns about immunosuppressive regimens [13][14] - Tenaya's regimen includes prednisone and sirolimus, which has been deemed appropriate and effective [15] - The company has not experienced any significant safety issues, and the safety profile of its therapies remains favorable [27] Protein Measurement and Efficacy - Accurate measurement of protein expression is critical for assessing the efficacy of gene therapies [18][19] - Tenaya has partnered with experts to improve protein measurement techniques, utilizing mass spectrometry for better accuracy compared to traditional methods [20][21] - The company aims to demonstrate that even modest increases in protein expression can lead to significant clinical benefits [37][38] TN-201 Program Updates - Initial data from the TN-201 program showed that two out of three patients had normalized cardiac troponin I levels, indicating a positive response to treatment [27][28] - The upcoming AHA presentation will provide more detailed data on safety and efficacy, including baseline biopsies for all patients [30][31] TN-401 Program Overview - TN-401 targets arrhythmogenic cardiomyopathy, a severe condition affecting approximately 70,000 patients in the U.S. [45] - The program is designed similarly to TN-201, with a focus on safety and efficacy endpoints [48][49] - Initial phase one data is expected to be shared in Q4 2025, with a focus on arrhythmia metrics as key indicators of treatment impact [50][53] Future Directions - Tenaya plans to continue dosing patients and expanding its natural history study, which is the largest of its kind for PKP2 mutations [59][60] - The company is also exploring international expansion and regulatory pathways for accelerated approval based on protein expression and other biomarkers [40][59] Macro Considerations - The CEO views the rise of biotech innovation in China as a positive development for the industry, emphasizing collaboration over competition [63][64] - Tenaya is leveraging artificial intelligence in its drug discovery processes, particularly in capsid engineering and phenotypic screening [65][66] Other Important Content - The company is focused on maintaining a strong safety profile while advancing its gene therapy programs [27][28] - The CEO highlighted the importance of collaboration and data sharing within the biotech community to enhance patient outcomes [64][66]