SOUTH SAN FRANCISCO, Calif., March 5, 2025 – Tenaya Therapeutics (NASDAQ: TNYA), a biotechnology company focused on developing genetic medicines for heart disease, has announced the pricing of its public offering of 75 million shares, expected to generate approximately $52.5 million in gross proceeds. The company plans to allocate the net funds toward advancing its clinical-stage gene therapy candidates, TN-201 and TN-401. Following the announcement, shares in Tenaya saw a sharp decline in trading.
Tenaya’s research is centered on developing targeted therapies for inherited and acquired cardiovascular diseases. Its pipeline includes TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), and TN-301, a small-molecule HDAC6 inhibitor for heart failure with preserved ejection fraction (HFpEF), along with multiple preclinical programs.
Progress in Gene Therapy Programs
TN-201 is an adeno-associated virus serotype 9 (AAV9)-based gene therapy designed to deliver a functional MYBPC3 gene directly to heart muscle cells via a single intravenous infusion. The therapy aims to restore MyBP-C protein levels, potentially slowing or reversing disease progression in patients with HCM, a genetic disorder that leads to abnormal thickening of the heart muscle.
Similarly, TN-401 targets PKP2-associated ARVC, a severe arrhythmic disorder caused by mutations in the PKP2 gene. This therapy, also delivered using an AAV9 vector, introduces a functional PKP2 gene to restore normal protein levels, aiming to improve cardiac function and reduce disease severity. Preclinical studies have shown promising results, including improvements in heart rhythm, disease markers, and survival rates.
Clinical Development and 2025 Outlook
In its latest corporate update, Tenaya outlined its clinical milestones for 2025:
- TN-201 (HCM program): The Phase 1b/2 clinical trial is expected to yield additional data in the first half of 2025, including safety and biopsy results from the first patient cohort. Interim data from late 2024 indicated that TN-201 was well-tolerated at the initial dose, with evidence of transgene RNA expression and stable or improving cardiac biomarkers. The first patient at a higher dose was dosed in 2024, and enrollment for the second cohort is ongoing, with completion expected in the first half of 2025. Full cohort data is anticipated in the second half of the year.
- TN-401 (ARVC program): The first cohort of the Phase 1b RIDGE-1 clinical trial is expected to complete enrollment in the first half of 2025. Initial safety and biopsy data are anticipated in the second half of the year. The first patient was dosed in late 2024, and Tenaya plans to expand the trial internationally, with its first non-U.S. site expected to open in 2025.
“2024 was a pivotal year for Tenaya, marked by strong operational execution across our clinical-stage gene therapy pipeline,” said Tenaya CEO Faraz Ali. “As we move into 2025, we are focused on accelerating enrollment and reporting safety, biopsy, and initial clinical endpoint data from both TN-201 and TN-401 programs.”
CIRM Grant Supports TN-401 Development
In addition to its public offering, Tenaya was recently awarded an $8 million CLIN2 grant from the California Institute for Regenerative Medicine (CIRM) to support its RIDGE-1 clinical trial for TN-401. The funding will help advance the development of TN-401 as a potential treatment for PKP2-associated ARVC, a progressive condition that affects an estimated 70,000 people in the U.S.
“Funding from CIRM will play a key role in supporting RIDGE-1, which is enrolling patients at leading cardiac centers,” said Ali. “We look forward to sharing initial data from the low-dose cohort in the second half of the year.”
With a strong focus on advancing its gene therapy programs, Tenaya aims to deliver transformative treatments for patients with inherited cardiac disorders. The company’s ongoing efforts, supported by both public and non-dilutive funding, position it to make meaningful progress in the field of genetic cardiology.
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