Tenaya Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Business Update

Dosing Initiated in Cohort 2 of the MyPEAK$(TM)$-1 Phase 1b/2 Clinical Trial of TN-201 for Treatment of MYBPC3-Associated Hypertrophic Cardiomyopathy

MyPEAK-1 Cohort 1 Data Accepted for Late-Breaker Presentation at the American College of Cardiology Meeting

Initial Data from RIDGE(TM)-1 Phase 1b Clinical Trial of TN-401 for PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy Expected in 2H25

Recent $48.9 Million Financing Extends Cash Runway into mid-2026

SOUTH SAN FRANCISCO, Calif., March 10, 2025 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. $(TNYA)$, 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 announced financial results for the fourth quarter and full year ended December 31, 2024, and provided a corporate update.

"Throughout 2024, Tenaya made important advances across our cardiovascular gene therapy development pipeline that have positioned us for a data-rich 2025. We look forward to sharing data from both the TN-201 MyPEAK-1 clinical trial for MYBPC3-associated HCM and the RIDGE-1 clinical trial of TN-401 for PKP2-associated ARVC in the months ahead," said Faraz Ali, Chief Executive Officer of Tenaya. "The recent financing with support from existing and new shareholders allows us to maintain our focus on driving our gene therapy programs for cardiomyopathies toward later-stage development. In the first half of next year, we expect to have greater than one-year of follow-up in the high-dose cohort of MyPEAK-1, as well as one-year follow-up for the first few patients enrolled in the RIDGE-1 clinical trial."

Business and Program Updates

TN-201 -- Gene Therapy for MYBPC3-Associated Hypertrophic Cardiomyopathy

   -- In December 2024, Tenaya reported promising early data from the first 
      cohort of patients in the MyPEAK-1 Phase 1b/2 clinical trial of TN-201 
      gene therapy. 
 
          -- Preliminary data from the first three patients dosed at 3E13 vg/kg 
             (Cohort 1) showed that TN-201 was generally well tolerated with 
             readily detectable vector DNA in the heart, evidence of transgene 
             RNA expression, and increasing TN-201 mRNA and MyBP-C protein 
             levels over time. Circulating biomarkers of cardiac muscle strain 
             and injury remained largely stable, and certain clinical markers 
             of disease remained stable or improved from baseline in the first 
             two individuals dosed, while other measures were not yet available, 
             interpretable or mixed. 
 
          -- MyPEAK-1 is a Phase 1b/2 multi-center, open-label, dose-escalation 
             trial designed to assess safety, tolerability and clinical 
             efficacy of a one-time intravenous infusion of TN-201 in treating 
             patients with HCM caused by mutations in the MYBPC3 gene. The 
             trial is being conducted in the U.S. with ten active clinical 
             sites. 
 
   -- Additional data from Cohort 1 of the MyPEAK-1 clinical trial has been 
      accepted as a late-breaker presentation at the American College of 
      Cardiology (ACC) Scientific Sessions taking place March 29-31 in Chicago. 
      These data are expected to include safety and available assessments from 
      the first three patients dosed, 52-week biopsy data for Patient 2, and 
      baseline and post-dose biopsy data for Patient 3. 
 
          -- Data from Cohort 1 of the MyPEAK-1 clinical trial of TN-201 will 
             be presented on Monday, March 31, 2025, at 9:00 am CT during the 
             Clinical and Investigative Horizons Session. 
 
   -- Tenaya is on track to complete enrollment in Cohort 2 of MyPEAK-1 in the 
      first half of 2025, with two participants having received TN-201 at the 
      6E13 vg/kg dose to date. 
 
          -- All patients in Cohort 2 will receive three biopsies: one at 
             baseline, one post-dose and one at 52 weeks. 
 
          -- In the second half of 2025, Tenaya plans to release initial Cohort 
             2 data, as well as further follow-up data from patients in Cohort 
             1. 
 
   -- Tenaya anticipates releasing data from its pediatric non-interventional 
      natural history study, known as MyClimb, in the second half of 2025. 
 
          -- MyClimb has enrolled more than 200 patients at 29 sites worldwide 
             in an effort to characterize the disease burden for MYBPC3 
             patients diagnosed before age 18 for whom there are currently no 
             approved therapeutic agents. 

TN-401 -- Gene Therapy for PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

   -- In November 2024, the first patient received TN-401 at a dose of 
      3E13vg/kg (Cohort 1) as part of the RIDGE-1 Phase 1b clinical trial. With 
      two participants dosed to date, Tenaya expects to complete enrollment of 
      Cohort 1 of the RIDGE-1 Phase 1b clinical trial in the first half of 
      2025. 
 
          -- Initial data, including safety and post-dose biopsy results, from 
             the first cohort of patients receiving TN-401 at the 3E13 vg/kg 
             dose is anticipated in the second half of 2025. 
 
          -- RIDGE-1 is a multi-center, open-label, dose escalation trial 
             designed to assess the safety, tolerability and preliminary 
             clinical efficacy of a one-time intravenous infusion of TN-401 for 
             the treatment of plakophilin-2 (PKP2)-associated ARVC. Seven 
             clinical sites have been activated in the U.S. 
 
   -- Tenaya anticipates sharing data from the ongoing RIDGE seroprevalence and 
      natural history study. RIDGE has enrolled more than 100 adults with 
      PKP2-associated ARVC and is being conducted at more than 20 sites in six 
      countries. 
 
   -- In February 2025, Tenaya was awarded an $8 million clinical grant from 
      the California Institute for Regenerative Medicine (CIRM), which will 
      help fund clinical trial costs associated with the ongoing Phase 1b 
      RIDGE-1 clinical trial of TN-401. 

Leadership Team

   -- Hiro Higa has been promoted to the position of Senior Vice President, 
      Finance and serves as the company's interim principal accounting officer. 
      Mr. Higa joined Tenaya in 2020 bringing more than 20 years of experience 
      in finance-focused roles for biopharmaceutical companies. While at Tenaya, 
      he led the establishment of key financial systems and processes, 
      including the company's transition from private to public. Prior to 
      Tenaya, Mr. Higa held positions of increasing responsibility for CytomX 
      Therapeutics, OncoMed Pharmaceuticals, Amgen and Abgenix. Mr. Higa 
      received his B.A. in economics from the University of Chicago and an M.B. 
      A. from the University of Chicago Booth School of Business. 

Business Updates

   -- In March 2025, Tenaya completed an underwritten public offering with net 
      proceeds of approximately $48.9 million after discounts, commissions, and 
      other offering expenses. The offering consisted of 75 million units 
      priced at $0.70 per unit, consisting of one share of Tenaya common stock, 
      one warrant to purchase one share of Tenaya common stock at an exercise 
      price of $0.80 per share and one warrant to purchase one half of a share 
      of Tenaya common stock at an exercise price of $0.70 per share. The 
      warrants are immediately exercisable and expire five years from the date 
      of issuance and June 30, 2026, respectively. 
 
   -- In January 2025, the U.S. Patent and Trademark Office issued U.S Patent 
      Number 12,201,617 (the '617 patent). The '617 patent is directed to a 
      method of treating heart failure with preserved ejection fraction (HFpEF) 
      with Tenaya's proprietary HDAC6 inhibitors, including TN-301. The '617 
      patent provides method of treatment protection for Tenaya's TN-301 for 
      the treatment of HFpEF and is expected to expire no earlier than 2040. 

Fourth Quarter and Full 2024 Financial Highlights

   -- Cash Position and Guidance: As of December 31, 2024, cash, cash 
      equivalents and investments in marketable securities were $61.4 million, 
      compared to $104.6 million as of December 31, 2023. With the additional 
      estimated net proceeds of $48.9 million from the March 2025 public 
      offering, the company expects that its current funds are sufficient to 
      support planned company operations into mid-2026. Tenaya has not drawn on 
      the $45 million credit facility established with Silicon Valley Bank and 
      is not obligated to do so. 
 
   -- Research & Development (R&D) Expenses: R&D expenses were $18.7 million 
      for the fourth quarter and $86.7 million for the full year ended December 
      31, 2024, compared to $22.9 million and $98.0 million for the same 
      periods in 2023. Non-cash stock-based compensation included in R&D 
      expense was $1.9 million for the fourth quarter and $8.2 million for the 
      full year ended December 31, 2024. 
 
   -- General & Administrative (G&A) Expenses: G&A expenses were $6.0 million 
      for the fourth quarter and $29.2 million for the full year ended December 
      31, 2024, compared to $8.6 million and $33.2 million for the same periods 
      in 2023. Non-cash stock-based compensation included in G&A expense was 
      $1.8 million for the fourth quarter and $8.3 million for the full year 
      ended December 31, 2024. 
 
   -- Net Loss: Net loss was $23.8 million, or $0.28 per share for the fourth 
      quarter ended December 31, 2024. For the full year 2024, net loss was 
      $111.1 million, or $1.31 per share. 

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