Company Overview - Tenaya Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering, developing, and delivering potentially curative therapies for heart disease [5] - The company employs integrated capabilities such as target validation, capsid engineering, and manufacturing to create genetic medicines for both rare genetic disorders and common heart conditions [5] Proposed Offering - Tenaya intends to offer and sell units consisting of common stock and Series A and Series B warrants, which allow the purchase of common stock [1] - The offering may also include pre-funded units with pre-funded warrants at a purchase price of $0.001 per share, which will be immediately exercisable and will not expire [1] - The offering is subject to market conditions, and there is no assurance regarding its completion or the actual size and terms [1] Management and Regulatory Aspects - Leerink Partners and Piper Sandler are acting as joint bookrunning managers for the proposed offering [2] - The offering is being conducted under a Registration Statement on Form S-3, previously filed and declared effective by the SEC [2] Pipeline and Product Development - Tenaya's pipeline includes several gene therapies: TN-201 for MYBPC3-associated hypertrophic cardiomyopathy, TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy, and TN-301, a small molecule HDAC6 inhibitor for heart failure with preserved ejection fraction [5] - The company also has multiple early-stage programs in preclinical development [5]

Core Insights - Tenaya Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing potentially curative therapies for heart disease [1][3] - The company will participate in two upcoming healthcare conferences, showcasing its commitment to advancing its innovative therapies [1][2] Company Overview - Tenaya Therapeutics aims to discover, develop, and deliver therapies targeting the underlying causes of heart disease [3] - The company utilizes integrated capabilities such as target validation, capsid engineering, and manufacturing to create genetic medicines [3] - Tenaya's pipeline includes: - TN-201: Gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM) - TN-401: Gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC) - TN-301: Small molecule HDAC6 inhibitor for heart failure with preserved ejection fraction (HFpEF) - Multiple early-stage programs in preclinical development [3]

Core Insights - Tenaya Therapeutics has received an $8.0 million CLIN2 grant from the California Institute for Regenerative Medicine (CIRM) to support the ongoing Phase 1b RIDGE-1 clinical trial of TN-401 gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC) [2][4] - The RIDGE-1 trial aims to evaluate the safety, tolerability, and preliminary clinical efficacy of TN-401, which is designed to deliver a functional PKP2 gene to heart muscle cells [3][4] - Initial data from the RIDGE-1 trial is expected in the second half of 2025 [1][4] Company Overview - Tenaya Therapeutics is a clinical-stage biotechnology company focused on developing potentially curative therapies for heart disease [12] - The company is advancing a portfolio of genetic medicines, including TN-401 for PKP2-associated ARVC and TN-201 for MYBPC3-associated hypertrophic cardiomyopathy [12] Clinical Trial Details - The RIDGE-1 clinical trial is an open-label, dose-escalation study enrolling symptomatic adults diagnosed with PKP2-associated ARVC [3][4] - TN-401 utilizes an adeno-associated virus serotype 9 (AAV9) vector for gene delivery, which has a strong safety record and targets heart muscle cells effectively [7][9] Disease Background - PKP2 mutations are the most common genetic cause of ARVC, affecting approximately 70,000 people in the U.S. [5][6] - Current treatments for ARVC do not address the underlying genetic cause, highlighting the potential significance of TN-401 as a novel therapeutic approach [6][9] Funding and Support - CIRM has been instrumental in funding regenerative medicine research, having provided billions since its establishment in 2004 [10][11] - The grant awarded to Tenaya Therapeutics is part of CIRM's efforts to accelerate the development of transformative treatments for serious medical conditions [4][10]

Core Insights - Tenaya Therapeutics is advancing its gene therapy programs, TN-201 and TN-401, targeting cardiomyopathies, with significant milestones expected in 2025 [1][2][3] Program Updates - **TN-201**: A gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM) is currently in the MyPEAK-1 Phase 1b/2 clinical trial. Additional data from Cohort 1 is anticipated in the first half of 2025, including safety assessments and biopsy results from the first three patients dosed [3][6] - **TN-401**: A gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC) is in the RIDGE-1 Phase 1b clinical trial. Initial clinical data from the first cohort is expected in the second half of 2025 [3][7] Clinical Trial Details - The MyPEAK-1 trial for TN-201 has shown promising initial results, with the first two patients demonstrating detectable vector DNA and transgene RNA expression. The trial is expanding to include adults with obstructive forms of HCM [3][4] - The RIDGE-1 trial for TN-401 has enrolled over 100 participants, with initial seroprevalence data indicating low antibody levels to AAV9, which is crucial for eligibility [10] Strategic Priorities - Tenaya plans to enhance its capabilities in drug discovery and genetic medicine delivery, focusing on both rare genetic disorders and prevalent heart conditions in 2025 [5][8] - The company aims to complete enrollment for both Cohort 1 of RIDGE-1 and Cohort 2 of MyPEAK-1 in the first half of 2025, with data presentations planned for the second half of the year [3][10] Market Context - MYBPC3 mutations are the most common genetic cause of HCM, affecting approximately 120,000 patients in the U.S. alone, while PKP2 mutations are linked to over 70,000 ARVC cases [6][7] - Both TN-201 and TN-401 have received Fast Track and Orphan Drug Designations from the FDA, indicating their potential significance in treating these conditions [6][7]

Core Insights - Tenaya Therapeutics reported early data from the MyPEAK-1 clinical trial of TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), indicating that TN-201 was well tolerated and showed promising early efficacy [2][3][4] Group 1: Clinical Trial Results - Preliminary data from three patients in the first dose cohort (3E13 vg/kg) showed TN-201 was generally well tolerated, with detectable vector DNA in the heart and increasing levels of TN-201 mRNA and MyBP-C protein over time [3][6] - Circulating biomarkers of cardiac muscle strain and injury remained stable, with some clinical measures showing improvement from baseline, particularly in Patients 1 and 2 [9][10] - The MyPEAK-1 trial is designed to assess the safety, tolerability, and clinical efficacy of TN-201, with a focus on symptomatic adults diagnosed with MYBPC3-associated HCM [12] Group 2: Safety Profile - TN-201 exhibited a manageable safety profile with no observed cardiac toxicities or serious adverse events related to the treatment [6][7] - Isolated elevations in liver enzymes were noted but were well managed and not indicative of liver damage [7] - Most adverse events were mild and transient, typical of AAV-based gene therapies [7] Group 3: Future Outlook - Tenaya plans to continue monitoring the initial patients and anticipates additional data readouts from Cohort 1 and a higher dose cohort in 2025 [3][4] - The company expressed confidence in TN-201's potential based on early evidence of transgene expression and cardiac transduction [10][11] - The MyPEAK-1 trial may expand to enroll up to 24 patients with MYBPC3-associated HCM in planned dose expansion cohorts [12]

Core Insights - Tenaya Therapeutics reported early data from the MyPEAK-1 clinical trial of TN-201 gene therapy, showing it was well tolerated and demonstrated promising early efficacy in treating MYBPC3-associated hypertrophic cardiomyopathy (HCM) [2][3][4] Group 1: Clinical Trial Results - Preliminary data from three patients in the first dose cohort (3E13 vg/kg) indicated that TN-201 was generally well tolerated, with detectable vector DNA in the heart and increasing levels of TN-201 mRNA and MyBP-C protein over time [3][6] - Clinical measures of HCM mostly remained stable or showed improvement from baseline, with some patients experiencing better NYHA classification scores [10][11] - The trial is ongoing, with additional data readouts expected from Cohort 1 and a higher dose cohort anticipated in 2025 [3][4] Group 2: Safety Profile - TN-201 exhibited a manageable safety profile, with no observed cardiac toxicities or serious adverse events related to the treatment [6][7] - Isolated elevations in liver enzymes were noted, which were well managed and consistent with known side effects of AAV-based gene therapies [6][7] Group 3: Mechanism and Efficacy - TN-201 achieved robust transduction into cardiomyocytes, with measurable transgene RNA expression and a 50% increase in TN-201 mRNA levels observed at Week 52 for one patient [8][9] - Total levels of MyBP-C protein showed a 3% increase from Weeks 8 to 52, indicating successful transcription and expression of the gene therapy [9] Group 4: Future Outlook - The company expressed confidence in TN-201's potential based on the early data, with plans to continue monitoring patients and gathering more data [12] - The MyPEAK-1 trial is designed to assess the safety and efficacy of TN-201, with a focus on symptomatic adults diagnosed with MYBPC3-associated HCM [14][15]

Core Insights - Tenaya Therapeutics, Inc. is set to announce initial data from the MyPEAK-1 Phase 1b/2 clinical trial of TN-201 gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM) on December 17, 2024 [1] Company Overview - Tenaya Therapeutics is a clinical-stage biotechnology company focused on discovering, developing, and delivering potentially curative therapies for heart disease [4] - The company utilizes integrated capabilities such as target validation, capsid engineering, and manufacturing to create genetic medicines for both rare genetic disorders and common heart conditions [4] - Tenaya's pipeline includes several therapies: TN-201 for MYBPC3-associated HCM, TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), and TN-301, a small molecule HDAC6 inhibitor for heart failure with preserved ejection fraction (HFpEF) [4] Upcoming Events - A live webcast and conference call will be held on December 17, 2024, at 8:00 a.m. ET to review the initial data from the MyPEAK-1 trial [2] - The webcast will be accessible on Tenaya's Investor Relations page [2][3]

Financial Performance - Total operating expenses for Q3 2024 were $26.7 million, down from $30.9 million in Q3 2023, reflecting a decrease of $4.2 million, or 14%[66]. - The net loss for Q3 2024 was $25.6 million, compared to a net loss of $29.1 million in Q3 2023, representing an improvement of $3.5 million, or 12%[66]. - Net loss for the nine months ended September 30, 2024, was $87.3 million, compared to a net loss of $94.1 million for the same period in 2023, reflecting an improvement of $6.8 million or 7%[77]. - Total operating expenses for the nine months ended September 30, 2024, were $91.3 million, down from $99.7 million in 2023, a decrease of $8.5 million or 8%[72]. - Cash, cash equivalents, and investments in marketable securities totaled $79.5 million as of September 30, 2024, with an accumulated deficit of $490.6 million[78]. - Net cash used in operating activities for the nine months ended September 30, 2024, was $72.1 million, compared to $80.1 million in 2023, indicating a reduction of $8.0 million[86]. - Net cash provided by financing activities for the nine months ended September 30, 2024, was $47.4 million, primarily from a follow-on offering, compared to $3.7 million in 2023[88]. Research and Development - Research and development expenses decreased by $2.7 million, or 12%, from $23.1 million in Q3 2023 to $20.4 million in Q3 2024, primarily due to a workforce reduction plan and lower costs for outside lab services[69]. - Research and development expenses decreased to $68.1 million for the nine months ended September 30, 2024, from $75.2 million in 2023, a reduction of $7.1 million or 9%[74]. - The company continues to invest in new technologies and optimization of existing capabilities to support the development of gene therapies[63]. - The company has initiated the MyPEAK-1 Phase 1b/2 clinical trial for TN-201, with initial data expected in December 2024[62]. - TN-401 received FDA clearance for clinical testing and patient dosing is planned to begin in Q4 2024[63]. - TN-301 showed positive data in a Phase 1 clinical trial, indicating it was well tolerated and demonstrated dose proportionality[63]. - The company has received rare pediatric disease designation and orphan drug designation for TN-201 from the FDA[63]. - The Genetic Medicines Manufacturing Center is strategically located to support clinical studies and enhance manufacturing productivity[65]. Expenses and Cost Management - General and administrative expenses decreased by $1.5 million, or 19%, from $7.8 million in Q3 2023 to $6.4 million in Q3 2024, driven by reductions in employee-related costs and lower professional fees[70]. - General and administrative expenses were $23.2 million for the nine months ended September 30, 2024, down from $24.6 million in 2023, a decrease of $1.3 million or 5%[75]. - The company expects to continue incurring operating losses in the foreseeable future, with operating expenses anticipated to remain relatively flat for the next twelve months[83]. Company Classification and Compliance - The company is classified as an emerging growth company under the JOBS Act, remaining so until it exceeds $1.235 billion in annual revenue or meets other specified criteria[93]. - The company is also a smaller reporting company, with a market value of stock held by non-affiliates below $700 million and annual revenue under $100 million in the most recently completed fiscal year[93]. - The company may continue to qualify as a smaller reporting company if its market value remains below $250 million or if it maintains annual revenue below $100 million[93]. - As a smaller reporting company, the company can present only the two most recent fiscal years of audited financial statements in its Annual Report on Form 10-K[93]. - The company has elected to use an extended transition period for complying with new or revised accounting standards, delaying adoption until it is no longer classified as an emerging growth company[93]. - The financial statements of the company may not be comparable to those of companies that comply with new accounting pronouncements as of public company effective dates[93]. - The company is not required to provide quantitative and qualitative disclosures about market risk due to its status as a smaller reporting company[94]. Funding and Financial Resources - The company has the right to draw down $20.0 million under a Loan Agreement with Silicon Valley Bank, with potential additional funds available upon meeting certain milestones[79][80]. - Interest income for the nine months ended September 30, 2024, was $3.9 million, down from $5.6 million in 2023, representing a decrease of $1.7 million or 30%[76].

Financial Position - As of September 30, 2024, Tenaya Therapeutics reported cash, cash equivalents, and investments in marketable securities of $79.5 million, sufficient to support operations into the second half of 2025[6]. - In August 2024, Tenaya entered into a $45 million credit facility with Silicon Valley Bank, which has not been drawn upon[5]. Expenses - Research and Development (R&D) expenses for Q3 2024 were $20.4 million, a decrease from $23.1 million in Q3 2023, while General & Administrative (G&A) expenses were $6.4 million, down from $7.8 million in the same period[6]. Net Loss - The net loss for Q3 2024 was $25.6 million, or $0.30 per share, compared to a net loss of $29.1 million, or $0.39 per share, for Q3 2023[6]. Clinical Trials - Tenaya completed dosing of the first three patients at the 3E13 vg/kg dose in the MyPEAK-1 clinical trial with no unexpected events or toxicities observed[3]. - The Data Safety and Monitoring Board (DSMB) endorsed the dose escalation to 6E13 vg/kg for Cohort 2 in the MyPEAK-1 study[3]. - Tenaya anticipates sharing interim results from MyPEAK-1, including safety and tolerability analyses, in December 2024[3]. - Tenaya activated six clinical sites for the RIDGE-1 trial, assessing TN-401 for ARVC, and has over 20 sites activated in six countries for the RIDGE seroprevalence and natural history study[4]. Regulatory Designation - The TN-201 gene therapy received rare pediatric disease designation from the FDA for treating MYBPC3-associated HCM in individuals under 18[3]. Intellectual Property - The U.S. Patent and Trademark Office issued U.S. Patent Number 12,104,165 for a method of treating ARVC with Tenaya's PKP2 gene therapy, expected to expire no earlier than 2040[4].

Data Safety and Monitoring Board Endorsed Dose Escalation and Broadening of Inclusion Criteria in the MyPEAKTM-1 Phase 1b/2 Trial of TN-201 Gene Therapy Initial TN-201 Data from Cohort 1 of MyPEAK-1 to be Reported in December 2024 SOUTH SAN FRANCISCO, Calif., Nov. 06, 2024 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today ...