ADDED
Heart disease remains the leading cause of death in the world.
In the U.S., one person dies from a cardiovascular-related health condition every 34 seconds, a gruesome statistic that translates to one in every three deaths in the U.S each year.
One in 20 adults suffer from congenital heart disease (CHD) and the picture is equally bleak at the other end of the age spectrum, as approximately 40,000 children are born in the U.S every year with CHD, the leading cause of birth defect-related morbidity and mortality.
We are primarily focused on advancing our clinical-stage gene therapy candidates, TN-201, for MYBPC3 -associated hypertrophic cardiomyopathy (HCM), and TN-401, for PKP2 -associated arrhythmogenic right ventricular cardiomyopathy (ARVC).
Each candidate is currently in Phase 1b/2 clinical testing to establish the safety and efficacy profile of two different doses.
We anticipate that data generated to date and over the course of 2026 will support pursuit of regulatory alignment on late-stage development for TN-201 and TN-401.
A third clinical-stage candidate discovered utilizing our targeted drug discovery capabilities is TN-301, a highly specific small molecule inhibitor of histone deacetylase 6 (HDAC6) with a unique multi-modal mechanism of action that has potentially broad utility in prevalent conditions such as heart failure with preserved ejection fraction (HFpEF), as well as other cardiac, metabolic, muscular and pulmonary diseases, including but not limited to genetic dilated cardiomyopathy (DCM), Duchenne muscular dystrophy (DMD) and pulmonary arterial hypertension (PAH).
Early on in our company history, we invested in differentiated capabilities to enable modality agnostic target identification and validation and product candidate optimization efforts anchored in human genetics and the use of human disease models.
These highly productive platform drug discovery capabilities directly contributed to the development of our three clinical-stage programs, as well as to several earlier-stage pipeline.
To support our initial focus on gene therapy candidates, we have internalized expertise in capsid engineering, promoters and regulatory elements and manufacturing science anchored on the use of adeno-associated viruses (AAVs) as the method of delivery to the heart.
REMOVED
In the U.S., one in every five deaths is attributable to heart disease, and an estimated 40,000 infants are born each year with congenital heart conditions.
We are primarily focused on advancing our lead investigational product candidates, TN-201, a gene therapy for MYBPC3 -associated hypertrophic cardiomyopathy (HCM) and TN-401, a gene therapy for PKP2 -associated arrhythmogenic right ventricular cardiomyopathy (ARVC).
Each candidate is currently in clinical testing to establish the safety profile of two different doses in adults with disease due to pathogenic/likely pathogenic mutations.
We anticipate that data generated in the next year will inform late-stage development by characterizing each candidates safety and tolerability profile, ability to transduce target heart cells, and produce the lacking protein underlying disease to slow or even halt disease progression.
Early on in our company history, we invested in differentiated capabilities to enable modality agnostic target identification, validation, anchored in human genetics and the use of human disease models.
Where our discovery efforts lead to product candidates intended for relatively prevalent indications, such as TN-301, our small molecule histone deacetylase-6 (HDAC6) inhibitor for heart failure with preserved ejection fraction (HFpEF), our strategy is to out-license or partner such programs.
TN-201 gene therapy for HCM caused by variants in the Myosin Binding Protein C3 (MYBPC3) gene: TN-201 is our AAV serotype 9 (AAV9)-based gene therapy being developed to treat the underlying cause of MYBPC3 -associated HCM by delivering a working MYBPC3 gene to specific cells of the heart via a single infusion.
We are currently conducting the Phase 1b/2 MyPEAK TM -1 clinical trial in symptomatic adults diagnosed with MYBPC3 -associated nonobstructive HCM, for which we presented promising initial data on the first cohort of patients in December 2024.
We plan to report additional Cohort 1 data in the first half of 2025.
We also anticipate completing Cohort 2 enrollment in the first half of 2025 and providing initial Cohort 2 data and a further update on Cohort 1 in the second half of 2025.