Public offering supports trials of gene therapies targeting hypertrophic cardiomyopathy and arrhythmogenic right ventricular cardiomyopathy.
Heart disease focused biotech Tenaya Therapeutics has announced the pricing of its public offering of 75 million new shares, expected to generate approximately $52.5 million in gross proceeds. Shares in the company fell sharply on the news, as it plans to allocate the net proceeds toward the advancement of clinical stage gene therapy candidates, TN-201 and TN-401.
Tenaya’s research is centered on addressing the underlying genetic causes of various heart conditions. 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), alongside several preclinical programs.
TN-201, an adeno-associated virus serotype 9 (AAV9)-based gene therapy, aims to deliver a functional MYBPC3 gene to heart muscle cells via a single intravenous infusion. The goal is to restore MyBP-C protein levels, potentially halting or reversing disease progression.
Similarly, TN-401 is designed to treat ARVC caused by PKP2 gene mutations, which lead to deficient protein levels necessary for heart cell structure and communication. Delivered using an AAV9 capsid, TN-401 introduces a functional PKP2 gene to restore protein levels, potentially slowing or reversing disease progression. Preclinical studies have demonstrated promising results, showing improvements in heart rhythm, disease markers and survival.
Tenaya recently outlined its strategic focus for 2025, with its Phase 1b/2 clinical trial for TN-201 expected to yield additional data in the first half of 2025, including safety and biopsy results from its first cohort. Initial interim data released in late 2024 indicated that TN-201 was generally well-tolerated at the starting 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 its second cohort remains ongoing, with completion anticipated in the first half of 2025. Data from both cohorts is expected in the second half of the year.
For TN-401, enrollment in the first cohort of a Phase 1b clinical trial is expected to be completed in the first half of 2025. Initial safety and biopsy data from patients is anticipated in the second half of the year. The first patient was dosed in late 2024, and Tenaya aims to expand the trial internationally with its first non-US site opening in 2025.
“2024 was a pivotal year for Tenaya, marked by important operational execution across our clinical-stage pipeline of gene therapy candidates with the potential to target and address the underlying drivers of heart disease,” said Tenaya CEO Faraz Ali. “As we enter 2025, we are eager to build on this momentum to accelerate enrollment and report on safety, biopsy and initial clinical endpoints from both our TN-201 and TN-401 programs throughout the year.”
Last month, the company was awarded an $8 million CLIN2 grant from the California Institute for Regenerative Medicine (CIRM). These funds will contribute to the ongoing costs of the Phase 1b trial, supporting the development of TN-401 as a potential treatment for PKP2-associated ARVC.
“Funds from this grant will support our ongoing RIDGE-1 clinical trial of TN-401, a potential best-in-class gene therapy for the treatment of PKP2-associated ARVC, a severe, progressive disease affecting an estimated 70,000 people in the US,” said Ali. “RIDGE-1 is actively enrolling patients at leading centers, and we look forward to sharing initial data from the low-dose cohort in the second half of this year.”
