Tenaya Therapeutics Announces Late Breaker and New Data Presentations at the American College of Cardiology’s Annual Scientific Session

Rhea-AI Summary

Tenaya Therapeutics (NASDAQ: TNYA) announced upcoming presentations of new clinical data for its MYBPC3-associated hypertrophic cardiomyopathy (HCM) program at the American College of Cardiology's Annual Scientific Session (March 29-31, 2025).

The key highlight will be a late-breaker presentation featuring one-year assessment data from the first two patients and six-month data from a third patient in Cohort 1 (3E13 vg/kg dose) of the MyPEAK-1 Phase 1b/2 trial of TN-201, their AAV9-based gene therapy. This follows encouraging early data shared in December 2024.

Additionally, a poster presentation will showcase findings from the SHaRe registry about disease burden differences in adults with MYBPC3-associated HCM. Dr. Milind Desai from Cleveland Clinic will present the clinical trial data, while Dr. Whit Tingley will present the registry findings.

Positive

• Early data from December 2024 was described as encouraging
• Trial progressing with multiple timepoints (6-month and 1-year) data to be presented
• Late-breaker presentation slot indicates potentially significant results

Negative

• None.

Insights

Biotechnology Research Analyst

Tenaya's upcoming data presentation at ACC represents a significant clinical development milestone for their gene therapy program in hypertrophic cardiomyopathy. The designation as a "late-breaker" presentation is particularly notable, as these slots are typically reserved for groundbreaking or highly compelling clinical results.

The company will present expanded follow-up data from the first cohort of their Phase 1b/2 MyPEAK-1 trial, including one-year assessments from two patients and six-month data from a third patient receiving TN-201 gene therapy at the 3E13 vg/kg dose. This builds upon what was characterized as "early encouraging data" shared in December 2024.

For context, TN-201 is an AAV9-based gene therapy targeting MYBPC3-associated HCM, a genetic heart condition caused by insufficient myosin-binding protein C production. The therapy aims to address the underlying genetic cause rather than just managing symptoms.

The scheduled presentation by Dr. Milind Desai, a prominent cardiologist from Cleveland Clinic, adds credibility to the data. Additionally, the company will present findings from the Sarcomeric Human Cardiomyopathy Registry, providing important context about disease burden in the target patient population.

For a clinical-stage biotech focused on heart disease therapies, these data presentations represent important validation points for their lead program. Positive durability data at one year would significantly strengthen TN-201's clinical profile and development pathway.

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Tenaya Therapeutics has announced upcoming presentations at the American College of Cardiology's Annual Scientific Session, featuring new clinical data from their gene therapy program for hypertrophic cardiomyopathy (HCM). The company will present updated results from the first cohort of their MyPEAK-1 Phase 1b/2 trial evaluating TN-201, an AAV9-based gene therapy for MYBPC3-associated HCM. Most notably, this will include one-year assessment data from the first two patients treated and six-month data from a third patient at the 3E13 vg/kg dose level. The presentation is designated as a late-breaker, suggesting potentially significant findings, and will build upon what the company described as "early encouraging data" shared in December 2024. The presentation will be delivered by Dr. Milind Desai, a prominent cardiologist from Cleveland Clinic who serves as an investigator on the trial. Additionally, Tenaya will present findings from the Sarcomeric Human Cardiomyopathy Registry describing the disease burden in adults with MYBPC3-associated HCM, providing important contextual information about the target patient population. For a clinical-stage biotech company focused on genetic heart disease, these data presentations represent important validation points that could significantly impact the perceived value and development pathway of their lead therapeutic program.

Late Breaker Oral Presentation to Include New Data from Cohort 1 of the MyPEAK™-1 Phase 1b/2 Clinical Trial of TN-201

SOUTH SAN FRANCISCO, Calif., March 19, 2025 (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 that new clinical and disease burden data pertaining to the company’s MYBPC3-associated hypertrophic cardiomyopathy (HCM) program will be presented at the upcoming American College of Cardiology’s Annual Scientific Session being held March 29-31, 2025 in Chicago, IL.

Tenaya is advancing TN-201, an AAV9-based gene therapy for the potential treatment of MYBPC3-associated HCM, a condition caused by insufficient levels of myosin-binding protein C (MyBP-C). As part of the late-breaking Clinical and Investigative Horizons session on Monday, March 31, data from the first cohort of adult patients enrolled in the MyPEAK-1 Phase 1b/2 clinical trial will be featured. Building on early encouraging data shared in December 2024, the presentation at ACC will include results from one-year assessments of the first two patients to receive TN-201 gene therapy, and baseline biopsy and six-month assessments from the third patient in the 3E13 vg/kg cohort. These data will be presented by Milind Desai, M.D., M.B.A, Haslam Family Endowed Chair in Cardiovascular Medicine, Vice Chair, Heart Vascular Thoracic Institute, Director of the Hypertrophic Cardiomyopathy Center at the Cleveland Clinic, and an investigator for the MyPEAK-1 Phase 1b/2 clinical trial.

A poster presentation on Sunday, March 30, will detail findings from SHaRe (Sarcomeric Human Cardiomyopathy Registry), describing differences in disease burden among adults with HCM caused by MYBPC3 mutations.

Details of the presentations are as follows:

Sunday, March 30, 2025

• Poster: Differences in Patient Characteristics and Burden of Disease in Adults with MYBPC3-Associated HCM (#129)
• Presenting author: Whit Tingley, M.D., Ph.D., Tenaya Therapeutics
• Session 1152: Heart Failure and Cardiomyopathies
• Time and location: 10:30 am – 11:30 am CT; South Hall

Monday, March 31, 2025 - ACC Late-breaking presentation

• Presentation: First Report of Phase Ib/2a Study Evaluating Safety and Early Efficacy of TN-201, an Adeno-Associated Virus Serotype 9 Gene Replacement Therapy, in Adults with MYBPC3-Associated Hypertrophic Cardiomyopathy (abstract ##)
• Presenting author: Dr. Milind Desai, M.D., M.B.A., Cleveland Clinic
• Session 402: Heart Failure and Cardiomyopathies
• Time and location: 9:00 am – 10:00 am CT; S100B

To view full event programming, please visit the ACC.25 website. Following the conference, Tenaya’s presentations will be available in the “Our Science” section of the company’s website.

About Tenaya Therapeutics
Tenaya Therapeutics is a clinical-stage biotechnology company committed to a bold mission: to discover, develop and deliver potentially curative therapies that address the underlying drivers of heart disease. Leveraging integrated proprietary core capabilities enabling target identification and validation, design of AAV-based genetic medicines and in-house manufacturing the company is advancing a pipeline of novel therapies with diverse treatment modalities for rare genetic cardiovascular disorders and more prevalent heart conditions. Tenaya’s most advanced candidates include 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 being initially developed for heart failure with preserved ejection fraction (HFpEF). Tenaya also has multiple early-stage programs progressing through preclinical development. For more information, visit www.tenayatherapeutics.com.

Contact
Michelle Corral
VP, Corporate Communications and Investor Relations
Tenaya Therapeutics
IR@TenayaThera.com

Investors
AnneMarie Fields
Stern IR
AnneMarie.Fields@SternIR.com

Media
Wendy Ryan
Ten Bridge Communications
wendy@tenbridgecommunications.com

FAQ

What new data will be presented for TNYA's TN-201 gene therapy at ACC 2025?

One-year data from two patients and six-month data from a third patient in Cohort 1 (3E13 vg/kg dose) of the MyPEAK-1 Phase 1b/2 trial will be presented as a late-breaker on March 31, 2025.

When and where will Tenaya Therapeutics (TNYA) present at the ACC 2025 conference?

TNYA will present on March 30 (poster session) and March 31 (late-breaker presentation) at the ACC conference in Chicago, IL.

What is the target indication for TNYA's TN-201 gene therapy?

TN-201 is being developed for MYBPC3-associated hypertrophic cardiomyopathy (HCM), a condition caused by insufficient levels of myosin-binding protein C.

What additional research will TNYA present about MYBPC3-associated HCM at ACC 2025?

TNYA will present findings from the SHaRe registry describing differences in disease burden among adults with HCM caused by MYBPC3 mutations.