Reported Promising Data for TN-201 and TN-401 Gene Therapies in Fourth Quarter of 2025; Additional Data Readouts and Pursuit of Regulatory Alignment for Each Program Planned in 2026

New Research Supports TN-301's Potential in Multiple Indications; Presented Preclinical Data for TN-301 in Duchenne Muscular Dystrophy Model

Entered into Multi-Target Research Collaboration with Alnylam Pharmaceuticals

December Financing with Net Proceeds of $55.8M and Anticipated Upfront Payment Extend Cash Runway into Second Half of 2027

SOUTH SAN FRANCISCO, Calif., March 11, 2026 (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, 2025, and provided a corporate update.

"As we enter 2026, we are energized by the momentum and clinical advances achieved over the past year," said Faraz Ali, Chief Executive Officer of Tenaya. "The encouraging data presented in 2025 from both of our lead gene therapy programs underscore the transformative potential of our science. In the first half of 2026, we expect to share additional updates, including longer-term follow-up data from the MyPEAK$(TM)$-1 clinical trial of TN-201 for MYBPC3-associated HCM, as well as one-year Cohort 1 data and early Cohort 2 data from the RIDGE(TM)-1 clinical trial of TN-401 for PKP2-associated ARVC. Over the course of the year, we also plan to pursue regulatory agency alignment on pivotal trial plans for both programs, a critical step toward accelerating the delivery of safe and effective gene therapies to patients with serious cardiac conditions."

Mr. Ali continued, "We are also excited to take modest but important steps to move TN-301 -- our clinical-stage, highly selective, small molecule HDAC6 inhibitor -- forward towards patients. New preclinical data in relevant DMD models that we presented at the recent MDA meeting adds to a growing body of external evidence supporting the potentially broad clinical utility of TN-301 in a range of cardiac and cardiac-adjacent indications with high unmet patient need and large market potential. The recently announced Alnylam collaboration further validates our platform capabilities that originally led to the discovery of TN-301. Together, these developments reflect the potential for Tenaya to add exciting new value drivers that are orthogonal to our portfolio of genetic medicines."

Business and Program Updates

TN-201 -- Gene Therapy for MYBPC3-Associated Hypertrophic Cardiomyopathy $(HCM)$

   -- In November 2025, Tenaya presented promising data from the MyPEAK-1 Phase 
      1b/2 clinical trial for the potential treatment of HCM due 
      to MYBPC3 mutations. The interim data reported included safety, biopsy 
      and leading indicators of efficacy results for the three patients who 
      each received a 3E13 vg/kg dose (Cohort 1) with follow-up ranging from 
      Week 52-78, as well as initial safety data and biopsy and efficacy 
      results for three patients who received a 6E13 vg/kg dose (Cohort 2) as 
      of the July 2025 data cut off. Key findings included: 
 
          -- TN-201 was generally well tolerated at both dose levels. No 
             dose-limiting toxicities were observed, and all patients had 
             successfully tapered off immunosuppressive medicine. 
 
          -- MyBP-C protein levels increased over time across patients in both 
             Cohorts, with a substantial increase in protein levels observed 
             commensurate with the higher dose in the first patient evaluable 
             from Cohort 2. 
 
          -- Multiple parameters associated with risk of complications and/or 
             survival improved among a majority of patients with greater than 
             26 weeks of follow-up, including circulating biomarkers of heart 
             muscle injury and measures of hypertrophy. All patients with 
             efficacy assessments improved to New York Heart Association Class 
             I, indicating no limitations to daily living due to symptoms. 
 
          -- These data were presented at the 2025 American Heart Association 
             Annual Scientific Sessions and simultaneously published 
             in Cardiovascular Research. 
 
   -- In January, following implementation of modest protocol amendments in 
      alignment with the U.S. Food and Drug Administration (FDA) input, Tenaya 
      resumed enrollment in MyPEAK-1 to generate additional safety and efficacy 
      data. 
 
   -- Tenaya outlined anticipated milestones associated with the TN-201 program 
      for 2026, which include: 
 
          -- Enrolling additional patients in the 6E13 vg/kg expansion cohort 
             of MyPEAK-1 over the course of the year 
 
          -- Reporting interim MyPEAK-1 data for Cohort 2 and updates from 
             Cohort 1 in the first half of 2026 
 
          -- Presenting one-year Cohort 2 data and two-year Cohort 1 data in 
             the second half of 2026 
 
          -- Providing an update on its progress in pursuing regulatory 
             alignment for TN-201 pivotal plans in the second half of the year. 

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

   -- In December 2025, Tenaya reported positive initial data in the ongoing 
      RIDGE-1 Phase 1b/2 clinical trial of TN-401 gene therapy for the 
      potential treatment of ARVC caused by mutations in the plakophilin-2 
      (PKP2) gene. The data reported included safety, biopsy and arrhythmia 
      results from the first three patients to receive TN-401 at a dose of 3E13 
      vg/kg (Cohort 1) as of the October 2025 data cut off, with follow-up 
      ranging from 20-40 weeks post-dose. Key findings included: 
 
          -- TN-401 was generally well tolerated at the 3E13 vg/kg dose, and no 
             dose-limiting toxicities were observed. Adverse events (AEs) were 
             generally mild, asymptomatic and manageable and deemed unrelated 
             to TN-401 treatment. Enrollment and dosing of Cohort 2 was 
             completed with no new serious AEs reported. 
 
          -- Biopsies taken at eight weeks post-treatment demonstrated robust 
             transduction and RNA expression in all patients. PKP2 protein 
             levels increased by an average of 10 percent compared to baseline 
             in the first two patients dosed. 
 
          -- Clinically meaningful improvements in measures of electrical 
             instability (premature ventricular contractions and non-sustained 
             ventricular tachycardias) were observed in the first two patients 
             with greater than six months follow-up after TN-401 dosing. 
 
   -- In January, the RIDGE-1 data and safety monitoring board (DSMB) reviewed 
      all available data for the six patients to have received either a 3E13 
      vg/kg (Cohort 1) or 6E13 vg/kg (Cohort 2) dose of TN-401. The DSMB 
      determined that TN-401 had an acceptable safety profile and endorsed 
      continued enrollment of patients in RIDGE-1 expansion cohorts at either 
      dose. 
 
   -- In 2026, Tenaya expects to achieve the following milestones associated 
      with the TN-401 program's advancement: 
 
          -- Enrolling patients in 6E13 vg/kg expansion cohort of RIDGE-1 
             throughout the year 
 
          -- Presenting one-year data for Cohort 1 and initial Cohort 2 data in 
             the first half of 2026 
 
          -- Reporting interim Cohort 2 data in the second half of 2026 
 
          -- Pursuing regulatory alignment on TN-401 pivotal plans and sharing 
             an update by year-end, as available. 

TN-301 -- Small Molecule HDAC6 Inhibitor for the Potential Treatment of Heart Failure with Preserved Ejection Fraction (HFpEF) and Related Cardiac, Metabolic, or Muscular Diseases

   -- Tenaya presented encouraging preclinical data comparing TN-301, the 
      company's highly selective HDAC6 inhibitor, with givinostat, an approved 
      pan-HDAC inhibitor, in a well-established mdx mouse model of Duchenne 
      muscular dystrophy (DMD) at the Muscular Dystrophy Association's 2026 
      Clinical and Scientific Congress. 
 
   -- Results of the study showed that in mdx mice: 
 
          -- TN-301 treatment at doses as low as 3 mg/kg improved grip strength 
             to wild-type levels within five weeks, whereas mdx mice treated 
             with givinostat (10 mg/kg, approximating clinical exposures) 
             failed to reach wild-type performance. 
 
          -- TN-301-mediated functional improvements were accompanied by 
             reductions in circulating creatine kinase and favorable changes in 
             gene expression, indicating reduced muscle cell injury. 
 
          -- In cardiomyocytes derived from human DMD-induced pluripotent stem 
             cells, TN-301 corrected calcium handling abnormalities and 
             mitochondrial dysfunction, while givinostat exacerbated these 
             established drivers of DMD cardiomyopathy. 
 
   -- In 2026, Tenaya plans to advance TN-301 toward clinical trials in 
      patients in order to generate proof-of-activity data, with HFpEF and DMD 
      being among the most promising potential indications identified to date. 

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March 11, 2026 16:05 ET (20:05 GMT)