Tenaya Therapeutics Q1 2024 Financial Results and Business Update

Tenaya Therapeutics, Inc., a clinical-stage biotechnology firm focused on discovering, developing and delivering therapies aimed at the root causes of heart disease, has announced its financial results for the first quarter ending March 31, 2024, along with several significant corporate updates. The company has reported strong initial progress for the year, underscored by strategic actions to ensure sustained advancement in its clinical programs and financial health.

For the TN-201 program, which targets MYBPC3-associated hypertrophic cardiomyopathy (HCM), Tenaya continues to enroll patients in the MyPEAK-1 Phase 1b trial. This multi-center, open-label dose-escalation study aims to evaluate the safety, tolerability, and clinical efficacy of a single intravenous infusion of TN-201. Initial data from the first cohort of patients, encompassing safety, biopsy, and biomarker information, is anticipated in the latter half of 2024. Additionally, a non-interventional study assessing adenovirus-associated virus serotype 9 (AAV9) antibody prevalence among adults with MYBPC3-associated HCM has completed enrollment with over 100 patients across 12 U.S. HCM centers. Preliminary findings indicate that a majority of these patients are eligible for the MyPEAK-1 trial.

In another significant development, Tenaya has activated the first two clinical sites for its RIDGE-1 Phase 1b trial of TN-401, aimed at treating arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by PKP2 gene mutations. The RIDGE-1 trial is structured similarly to MyPEAK-1, focusing on assessing the safety and efficacy of a single intravenous infusion of TN-401. The company plans to commence patient dosing in the second half of 2024. Concurrently, Tenaya is enrolling patients for a global, non-interventional seroprevalence and natural history study of PKP2-associated ARVC at 18 sites in the U.S. and Europe. Notably, preclinical studies on TN-401, published in Nature Communications Medicine, demonstrated efficacy in a Pkp2-deficient mouse model, highlighting improvements in heart function and survival.

Furthermore, Tenaya is advancing TN-301, a small molecule HDAC6 inhibitor targeting heart failure with preserved ejection fraction (HFpEF). Preclinical research on TN-301 was featured in Nature Communications, indicating the potential of HDAC6 inhibition in treating HFpEF. This condition affects more than three million individuals in the U.S. alone.

Tenaya’s research and manufacturing teams have also made strides in gene and cell therapy innovations. At the American Society for Gene and Cell Therapy (ASGCT) meeting, researchers presented posters on advancements in AAV-based drug design, capsid engineering, and gene editing. One significant highlight included a self-inactivating CRISPR Cas9 vector designed to target the phospholamban (PLN) R14del mutation, which is linked to dilated cardiomyopathy. Additionally, studies on capsid engineering revealed the superior performance of the AAV9 serotype in cardiomyocyte transgene expression.

On the corporate front, Tenaya has undertaken cost containment measures to align with its focus on clinical-stage data generation. This includes a planned workforce reduction of approximately 22%, expected to be completed in the second quarter of 2024. The company also appointed Dr. Whedy Wang as Senior Vice President of Biometrics, bringing over 30 years of industry experience to the role.

Financially, Tenaya raised $47 million in net proceeds from a first-quarter financing, extending its cash runway into the second half of 2025. As of March 31, 2024, the company held $122.2 million in cash, cash equivalents, and marketable securities. Research and development expenses were $25.1 million, and general and administrative expenses were $8.7 million for the quarter. The net loss for the period was $32.2 million, or $0.40 per share.

Tenaya Therapeutics continues to make significant progress in its mission to develop therapies for heart disease, leveraging its integrated platforms in gene therapy, cellular regeneration, and precision medicine. The company's advanced therapeutic candidates—TN-201, TN-401, and TN-301—are at the forefront of its pipeline, with multiple early-stage programs also in development.