TN-201 / Tenaya Therap - LARVOL DELTA

Low seroprevalence of neutralizing antibodies to adeno-associated virus serotype 9 in preparation for MyPEAK-1 study of TN-201 for individuals with MYBPC3-associated hypertrophic cardiomyopathy (HEART FAILURE 2025) - "Results suggest that MYBPC3+ HCM patients in the US have low levels of pre-existing AAV9 NAbs and that most would meet current NAb eligibility for clinical studies of TN-201 gene therapy. Tenaya is enrolling patients in MyPEAK-1, a Phase 1b/2a trial evaluating the safety, tolerability, and exploratory efficacy measures of TN-201 in MYBPC3+ HCM."

Clinical • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy • Non-obstructive Hypertrophic Cardiomyopathy

Tenaya Therapeutics Publishes Preclinical Data Demonstrating TN-201 Enhances Cardiac Function and Survival in MYBPC3 Cardiomyopathy Models (GlobeNewswire) - "Preclinical results published in Nature Communications show that Tenaya’s MYBPC3 gene replacement therapy achieved dose-dependent increases in MyBP-C protein, improving multiple parameters of cardiac function at protein levels well below wild-type with doses as low as 1x1013 vg/kg. Of note, treatment with Tenaya’s MYBPC3 gene therapy reversed left ventricular hypertrophy, a hallmark of HCM, as evidenced by decreases in posterior wall thickness relative to vehicle and normalization of left ventricular mass relative to body weight."

Preclinical • Cardiomyopathy

Tenaya Therapeutics Announces Late Breaker and New Data Presentations at the American College of Cardiology’s Annual Scientific Session (GlobeNewswire) - "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."

P1/2 data • Hypertrophic Cardiomyopathy

AAV9-mediated MYBPC3 gene therapy with optimized expression cassette enhances cardiac function and survival in MYBPC3 cardiomyopathy models. (PubMed, Nat Commun) - "Dose-ranging efficacy studies exhibit restoration of wild-type MYBPC3 protein levels and saturation of cardiac improvement at the clinically relevant dose of 3E13 vg/kg, outperforming a previously published construct. These findings suggest that TN-201 may offer therapeutic benefits in MYBPC3-associated cardiomyopathy, pending further validation in clinical settings."

Journal • Cardiomyopathy • Cardiovascular • Gene Therapies • Hypertrophic Cardiomyopathy

MyPEAK-1: Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM (clinicaltrials.gov) - P1/2 | N=30 | Recruiting | Sponsor: Tenaya Therapeutics | Phase classification: P1 ➔ P1/2 | N=15 ➔ 30 | Trial completion date: Dec 2029 ➔ Aug 2032 | Trial primary completion date: Dec 2025 ➔ Aug 2027

Enrollment change • Phase classification • Trial completion date • Trial primary completion date • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy • TNNI3

Low Seroprevalence Of Neutralizing Antibodies To Adeno-associated Virus Serotype 9 (AAV9) In Preparation For Mypeak-1, The First-in-Human Study Of TN-201, An Investigational AAV9-mediated Gene Therapy For Individuals With MYBPC3-associated Hypertrophic Cardiomyopathy (HCM) (HFSA 2024) - "Results suggest that MYBPC3+ HCM patients in the US have low levels of pre-existing AAV9 NAbs and that most would meet current NAb eligibility for clinical studies of TN-201 gene therapy. Tenaya is enrolling patients in MyPEAK-1, a Phase 1b/2a trial evaluating the safety, tolerability, and exploratory efficacy measures of TN-201 in MYBPC3+ HCM."

Clinical • Gene therapy • P1 data • Cardiomyopathy • Cardiovascular • Gene Therapies • Hypertrophic Cardiomyopathy • Inflammation • Non-obstructive Hypertrophic Cardiomyopathy

MyPEAK-1: Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM (clinicaltrials.gov) - P1 | N=15 | Recruiting | Sponsor: Tenaya Therapeutics

Trial completion date • Trial primary completion date • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy • TNNI3

Tenaya Therapeutics Doses First Patient in the MyPeak-1 Phase 1b Clinical Trial of TN-201 for the Treatment of MYBPC3-Associated Hypertrophic Cardiomyopathy (GlobeNewswire) - "Tenaya Therapeutics...today announced that the first patient has been dosed with TN-201 gene therapy for the treatment of Myosin Binding Protein C3 (MYBPC3)-associated HCM in the MyPeak-1 Phase 1b clinical trial at the Cleveland Clinic, Cleveland, Ohio. Tenaya anticipates sharing initial data from the MyPeak-1 trial in 2024....The trial will initially seek to enroll at least six symptomatic (New York Heart Association Class II or III) adults who have been diagnosed with MYBPC3-associated nonobstructive HCM and have an implantable cardioverter defibrillator, and potentially treat up to 15 subjects in total from the U.S. and outside the U.S."

P1 data • Trial status • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy

Tenaya Therapeutics to Present Data for TN-301 and TN-201 at Upcoming Medical Conferences (GlobeNewswire) - "Tenaya Therapeutics...announced today that poster presentations related to its TN-201 and TN-301 programs will be presented at the 2023 Hypertrophic Cardiomyopathy Medical Society (HCMS) Scientific Sessions taking place October 6, 2023, and at the Heart Failure Society of America (HFSA) Annual Scientific Meeting occurring October 6-9, 2023....Data to be presented at HFSA will include results from the company’s Phase 1 clinical trial of TN-301 in healthy adult participants....The company will also present new data from preclinical studies examining the effects of its HDAC6 inhibitor in combination with an approved HFpEF treatment in a model of disease....Tenaya will highlight the clinical trial design for its Phase 1b clinical trial in nonobstructive patients with MYBPC3-associated HCM as well as sharing interim data from an ongoing seroprevalence study."

P1 data • Preclinical • Cardiomyopathy • Cardiovascular • Heart Failure • Hypertrophic Cardiomyopathy

Tenaya Therapeutics Reports Second Quarter 2023 Financial Results and Provides Business Update (GlobeNewswire) - "Tenaya anticipates dosing its first patient in the 'MyPeak-1' Phase 1b multi-center, open-label, dose-escalation clinical trial of TN-201 during the third quarter of 2023....Initial data from the MyPeak-1 clinical trial are anticipated in 2024."

P1 data • Trial status • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy

MyPEAK-1: Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM (clinicaltrials.gov) - P1 | N=15 | Recruiting | Sponsor: Tenaya Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy

MyPEAK-1: Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM (clinicaltrials.gov) - P1 | N=15 | Not yet recruiting | Sponsor: Tenaya Therapeutics

New P1 trial • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy

Tenaya Therapeutics Receives FDA Fast Track Designation for TN-201 (GlobeNewswire) - "Tenaya Therapeutics, Inc...today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its gene therapy product candidate, TN-201, being developed for the treatment of Myosin Binding Protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM)."

Fast track designation • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy

Tenaya Therapeutics Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Update (GlobeNewswire) - "TN-201 – MYBPC3 Gene Therapy Program for Genetic Hypertrophic Cardiomyopathy (HCM)....Tenaya plans to commence a Phase 1b multi-center, open-label, dose-escalation study to assess the safety, tolerability and pharmacodynamics of a one-time intravenous infusion of TN-201 in symptomatic adults with MYBPC3-associated HCM in the third quarter of 2023. Initial data from the Phase 1b trial are anticipated in 2024."

New P1 trial • P1 data • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy

Tenaya Therapeutics Announces TN-201 IND Clearance and Anticipated 2023 Milestones (GlobeNewswire) - "Tenaya Therapeutics...announced today that the U.S. Food and Drug Administration (FDA) has provided clearance of its Investigational New Drug (IND) application to initiate clinical testing of TN-201. In addition, Tenaya shared anticipated 2023 program milestones and updated cash runway guidance....'We are pleased to start 2023 with clearance of the IND for our TN-201 gene therapy program and look forward to dosing patients with MYBPC3-associated HCM in our Phase 1b study in the coming months. We are also starting 2023 with confirmation of target engagement in our TN-301 Phase 1 study and look forward to reporting clinical data for this program later this year.'"

IND • New P1 trial • P1 data • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy

Tenaya Therapeutics Reports Third Quarter 2022 Financial Results and Provides Business Update (GlobeNewswire) - "TN-201 – MYBPC3 Gene Therapy Program for Genetic Hypertrophic Cardiomyopathy (HCM): Tenaya plans to submit an Investigational New Drug (IND) application for TN-201 to the U.S. Food and Drug Administration (FDA) by year end 2022. The company expects to provide an update on the status of the TN-201 IND in early 2023....Tenaya received notification from the U.S. Patent and Trademarks Office (USPTO) of the issuance of patent No. 11,446,397. This patent provides composition of matter protections for TN-201, covering a recombinant adeno-associated viral (AAV)-based therapy containing MYBPC3. This is the second patent granted by the USPTO related to the TN-201 product candidate. This new patent is expected to expire no earlier than 2041."

IND • Patent • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy

Tenaya Therapeutics to Participate in Inaugural Hypertrophic Cardiomyopathy Medical Society’s 2022 Scientific Sessions (GlobeNewswire) - "Encore Presentation of Lead Gene Therapy TN-201 Preclinical Data to be Featured in Late-Breaking Trials Session....Tenaya Therapeutics...announced today that it is scheduled to participate in the Hypertrophic Cardiomyopathy Medical Society’s (HCMS) inaugural 2022 Scientific Sessions taking place September 30, 2022...will present preclinical data for Tenaya’s TN-201, a gene therapy candidate intended to correct the underlying genetic cause of HCM, MYBPC3 gene mutations. Variants in the MYBPC3 gene are the most common genetic cause of HCM, believed to contribute to approximately 20 percent of all HCM cases."

Late-breaking abstract • Preclinical • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy

Tenaya Therapeutics Launches Operations of New Genetic Medicines Manufacturing Center to Support the Development of Potentially First-In-Class Cardiovascular Therapeutics (Businesswire) - "TN-201 is being developed for the treatment of genetic hypertrophic cardiomyopathy (HCM) due to MYBPC3 gene mutations. Tenaya plans to submit an Investigational New Drug (IND) application for TN-201 to the U.S. Food and Drug Administration (FDA) in the second half of this year."

IND • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy

Tenaya Therapeutics Reports First Quarter 2022 Financial Results and Provides Business Update (Businesswire) - "TN-201 – MYBPC3 Gene Therapy Program for Genetic Hypertrophic Cardiomyopathy (HCM): TN-201 received Orphan Medicinal Product designation from the European Commission for the treatment of HCM due to mutations in the MYBPC3 gene."

European regulatory • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy

2 more biotech companies, backed by industry pioneers, plot IPOs (Business Journals) - "Heart-focused Tenaya Therapeutics Inc., a South San Francisco-based company launched from the Gladstone Institutes with the support of Genentech Inc.'s first scientist, Dave Goeddel, bookmarked its IPO at $100 million....Tenaya, founded by scientists from San Francisco-based Gladstone and the University of Texas Southwestern Medical Center, is focused on gene therapies, cellular regeneration and precision medicine approaches to rebuild damaged heart tissue. It plans to seek approval from the Food and Drug Administration next year to take its gene therapy TN-201 into a clinical trial to treat genetic hypertrophic cardiomyopathy."

Financing • New trial • Hypertrophic Cardiomyopathy

[VIRTUAL] Prevention of Premature Lethality and Reversal of Cardiac Hypertrophy with an Optimized MYBPC3 Gene Therapy (ESGCT 2021) - "Thus, we have successfully engineered an AAV vector (TN201) with superior properties for selective restoration of MYBPC3 to cardiomyocytes upon systemic delivery...Further, pilot safety studies in adult and infant mice injected with >10X an efficacious dose exhibited no clinical observations and no alterations in cardiac function. Finally, we have established stable cardiac benefit for greater than one year postinjection, as well as reversal of cardiac dysfunction even in latestage homozygote disease."

Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Gene Therapies • Heart Failure • Hypertrophic Cardiomyopathy

Tenaya Therapeutics to Present Preclinical Data on Its Gene Therapy Programs at the ESGCT 28th Annual Congress (Businesswire) - "Tenaya Therapeutics...announced today that it will present new preclinical data at the virtual 28th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), October 19 - 22, 2021. In an oral presentation, Tenaya will present key data on TN-201, its AAV-based gene therapy product candidate for patients carrying mutations of the MYBPC3 gene, the most common genetic cause of hypertrophic cardiomyopathy (HCM) that affects more than 115,000 patients in the United States alone. TN-201 is in IND-enabling studies, and Tenaya expects to file an IND in 2022."

IND • Preclinical • Cardiomyopathy • Hypertrophic Cardiomyopathy

Tenaya Therapeutics Reports Second Quarter 2021 Financial Results and Provides Business Updates (Businesswire) - "TN-201 – MYBPC3 Gene Therapy Program for Genetic HCM (gHCM): Tenaya is expected to initiate a global natural history study for patients with MYBPC3 mutations in the second half of 2021. Tenaya has initiated IND-enabling activities and intends to submit an investigational new drug (IND) application or clinical trial application (CTA) to the FDA or European Medicine Agency (EMA), respectively, in 2022."

European regulatory • IND • New trial • Cardiomyopathy • Hypertrophic Cardiomyopathy

Tenaya Therapeutics catches IPO window months after crossover round (Endpoints News) - "A good chunk of the IPO funding ($35 million to $40 million) will go straight into Tenaya’s lead gene therapy candidate, which targets genetic hypertrophic cardiomyopathy due to mutations in the MYBPC3 gene. That candidate’s headed for the clinic in 2022, according to the S-1/A."

Clinical • Financing • Cardiomyopathy • Hypertrophic Cardiomyopathy

Heart Disease Company Tenaya Therapeutics Files For $100M US IPO (Yahoo Finance) - "...it plans INDs for its gene therapy and precision medicine platforms in 2022."

IND • Cardiomyopathy • Hypertrophic Cardiomyopathy