In two out of the first three patients, TN-201 was able to increase MyBP-C protein levels from 56% to 59% in one, and 62% to 64% in the other after 52 weeks. Credit: Shutterstock / TEEREXZ
Three patients living with objectively severe heart disease have had their limitations on physical activity lifted following a single dose of Tenaya Therapeutics’ gene therapy TN-201, according to interim data from the company’s Phase Ib/2 trial.
Unveiling the interim MYPEAK-1 trial (NCT05836259) data during a late-breaker presentation at the 2025 American College of Cardiology (ACC) conference in Chicago, California-based Tenaya detailed how its therapy for hypertrophic cardiomyopathy (HCM) had shown to be safe and well tolerated in the first three patients dosed in the first cohort.
HCM is caused by a deficiency of myosin-binding protein C (MyBP-C). In two out of the first three patients, TN-201 was able to increase MyBP-C protein levels from 56% to 59% in one, and 62% to 64% in the other after 52 weeks.
Meanwhile, all three patients in the first cohort have seen their New York Heart Association classification level, a scale used to measure the severity of heart failure, drop from Class two or three down to one.
Researchers also found that enlargement of the heart, known as hypertrophy, improved in two patients while left ventricular posterior wall thickness also decreased in two patients by up to 40% and into a normal range.
The MyPEAK-1 trial investigator Milind Desai said: “TN-201 is the first gene therapy to be tested in HCM patients whose disease is caused by mutations to the MYBPC3 gene. For patients with this mutation, their disease is often more aggressive in its progression, and they are at higher risk of serious – sometimes fatal – complications.
“I look forward to continuing to study and evaluate this to see if this patient population is one that could be treated with gene therapy, as we have seen with other diseases.”
GlobalData’s Pharmaceutical Intelligence Center estimates that TN-201 could bring in as much as $74m for Tenaya Therapeutics by the end of 2028, climbing to $419m by the end of the decade. By the end of next year, the market for hypertrophic cardiomyopathy is set to bring in more than $1.5bn, with Bristol-Myers Squibb’s Camzyos accounting for more than $1.2bn.
GlobalData is the parent company of Clinical Trials Arena.
Elsewhere at ACC 2025, Cleerly announced interim results from a trial that found women living with chronic coronary artery disease (CAD) are at higher risk of major adverse coronary events. Meanwhile, AstraZeneca’s AZD0780 has demonstrated a 50.7% reduction in LDL-C after 12 weeks when combined with standard statin therapy.
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