New Data from Both Cohorts of the MyPEAK™-1 Phase 1b/2 Trial of TN-201 for Adults with MYBPC3-Associated HCM Expected in the Second Quarter 2026
Preclinical Data at MDA 2026 Highlighted TN-301's Activity in Duchenne Muscular Dystrophy Disease Models; Distinct Mechanism of HDAC6 Inhibition Supportive of TN-301's Potential in Multiple Indications
Entered Research Collaboration with Alnylam to Identify and Validate Genetic Targets for Cardiovascular Conditions
SOUTH SAN FRANCISCO, Calif., May 06, 2026 (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 announced financial results for the first quarter ended March 31, 2026, and provided a corporate update.
"We are entering a catalyst-rich period for Tenaya, with multiple clinical milestones expected across our lead gene therapy programs throughout 2026. Building on the encouraging initial readouts we reported in 2025, we believe the additional data expected this year from both TN-201 and TN-401 may support alignment on registrational pathways for these novel gene therapies," said Faraz Ali, Chief Executive Officer of Tenaya.
Mr. Ali continued, "While our focus remains on the advancement of TN-201 and TN-401 for patients suffering from these genetic cardiomyopathies, we also announced meaningful steps in the direction of our next horizon of opportunities to address unmet patient needs and to create value for stockholders. We presented new preclinical data for TN-301, our clinical-stage small molecule candidate, in Duchenne muscular dystrophy adding to the body of compelling preclinical evidence for the broad clinical utility of this molecule in multiple prevalent and rare cardiac and cardiac-adjacent indications. Advancing TN-301 toward a trial in patients reflects our commitment to building a diversified portfolio grounded in mechanistic insight and translational rigor. The recently announced collaboration with Alnylam also reinforces the strength of Tenaya's innovation engine and expands the reach and impact of our modality-agnostic research capabilities."
Business and Program Updates
TN-201, Gene Therapy for MYBPC3-Associated Hypertrophic Cardiomyopathy (HCM)
On May 9, 2026, at the upcoming European Society of Cardiology (ESC) Heart Failure Conference, in Barcelona, Spain, Milind Desai, M.D., Director of the Hypertrophic Cardiomyopathy Center, Vice Chair of the Heart, Vascular & Thoracic Institute at Cleveland Clinic, and principal investigator for the MyPEAK-1 clinical trial, will present insights that emerged early in the trial that enabled reductions in the cumulative dose and duration of immune suppressive medications, even when TN-201 is administered at the higher dose.
Per the MyPEAK-1 protocol, sirolimus and prednisone are administered prophylactically in patients receiving TN-201 gene therapy, accompanied by post dose tapering in conjunction with monitoring of liver enzyme levels, an early indicator of potential complement system activation. Investigators found that minor adjustments, including administering sirolimus earlier, reducing the starting dose of prednisone and monitoring patients weekly, led to faster tapering and an overall decrease in the burden of immunosuppression.
The ESC-HF presentation offers more detail on the immunosuppressive regimen results previously reported in November 2025 and includes safety data for the first seven patients enrolled in MyPEAK-1. The optimized regimen and monitoring protocols that were successfully deployed in MyPEAK-1 are also being utilized in the RIDGE-1 clinical trial of TN-401.
Tenaya expects to report interim MyPEAK-1 data for Cohort 2 (6E13 vg/kg) and updates from Cohort 1 (3E13 vg/kg) in the second quarter of 2026.
In January, Tenaya resumed enrollment and screening in MyPEAK-1 following implementation of modest protocol amendments in alignment with U.S. Food and Drug Administration (FDA) input.
At the American Society of Gene and Cell Therapies (ASGCT) Annual Meeting, taking place May 11-15, 2026, in Boston, MA, Tenaya will present results from a survey exploring parental perceptions of gene therapy treatment for children with cardiomyopathies. This work was conducted by Tenaya in partnership with DDC Clinic and Children's Cardiomyopathy Foundation. The poster presentation is scheduled for May 12, 2026.
TN-401, Gene Therapy for PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
Data from the ongoing RIDGE-1 Phase 1b/2 clinical trial of TN-401 in adults with ARVC due to variants in the PKP2 gene have been accepted as a late-breaking presentation at the upcoming ASGCT Annual Meeting. The presentation, scheduled for Friday, May 15, is expected to include one-year data for Cohort 1 (3E13 vg/kg) and initial Cohort 2 (6E13 vg/kg).
Tenaya management plans to conduct a webcast conference call on Friday, May 15, 2026, at 10:30 a.m. EDT / 7:30 am PDT following the Late Breaker Session. The webcast conference call, including an accompanying slide presentation, will be accessible from the Investor section of the Tenaya website at www.tenayatherapeutics.com.
In January, the RIDGE-1 data and safety monitoring board (DSMB) reviewed all available data for the six patients that have received TN-401 gene therapy. 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.
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
In March 2026, Tenaya presented encouraging preclinical data comparing TN-301, the company's highly selective HDAC6 inhibitor, with givinostat, an approved pan-HDAC inhibitor, in well-established preclinical models of Duchenne muscular dystrophy (DMD) at the Muscular Dystrophy Association's 2026 Clinical and Scientific Congress.
Results of the study showed that:
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.
TN-301 was granted both Rare Pediatric Disease Designation and Orphan Drug Designation for the treatment of DMD from U.S. Food and Drug Administration.
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.
Research
In March 2026, Tenaya entered into a multi-target research collaboration with Alnylam Pharmaceuticals to identify and validate novel genetic targets aimed at treating cardiovascular disease.
Under the terms of the collaboration agreement, in April 2026, Tenaya received an upfront payment of $10.0M and may be eligible for future development, regulatory and sales-based milestones totaling up to $1.1 billion, in addition to reimbursement of associated research costs.
Updated results of preclinical studies characterizing TN-501, a gene editing therapeutic candidate intended for the treatment of PLN-R14del-associated dilated cardiomyopathy (DCM) will be presented at ASGCT on Thursday, May 14, 2026. TN-501 is designed to specifically inactivate the pathogenic phospholamban (PLN) R14del allele while preserving healthy function.
First Quarter 2026 Financial Highlights
Cash: As of March 31, 2026, cash and cash equivalents were $80.9 million. Tenaya expects that such resources, along ...
