Tenaya Therapeutics NASDAQ: TNYA expects to provide regulatory and development updates in the fourth quarter for its two cardiac gene therapy programs and its small-molecule candidate TN-301, Chief Financial Officer Eric Hyllengren said during a Canaccord discussion.
Hyllengren, who said he had been in the CFO role for nearly a month, described the company’s strategy as developing precision medicines for heart disease using multiple therapeutic approaches, including gene therapy and small molecules. He said the company is focused on addressing the underlying causes of disease rather than solely treating symptoms.
TN-201 Program Targets MYBPC3-Associated HCM
Tenaya’s TN-201 is a gene therapy candidate for MYBPC3-associated hypertrophic cardiomyopathy, or HCM. Hyllengren said the condition affects approximately 120,000 people in the United States, representing about 20% of the overall HCM population.
In these patients, the left ventricle can thicken, reducing the heart’s ability to pump blood effectively, he said. Tenaya is evaluating reductions in left ventricular mass index, or LVMI, as an important outcome measure, while discussing appropriate endpoints with regulators.
The ongoing expansion cohort is evaluating the higher 6E13 dose of TN-201, compared with a dose half that level in the first cohort. Hyllengren said the treatment has been well tolerated at both doses and that prior data showed meaningful LVMI reductions in most patients. He also said many patients experienced improvements in symptoms and function.
The company is conducting a pediatric natural history study, citing limited existing data in younger patients. Hyllengren said earlier symptom onset is associated with greater risk of later complications, and Tenaya sees a potential future opportunity to study TN-201 in pediatric patients. Discussions with U.S. and European regulators are ongoing for both adult and pediatric development paths, with an update expected in the fourth quarter.
While Hyllengren said the pediatric development strategy remains to be determined, he noted that the unmet need may be particularly high because there are no effective treatment options for pediatric patients in this setting.
TN-401 Shows PVC Reductions in Early Data
Tenaya’s second gene therapy candidate, TN-401, is being developed for PKP2-associated arrhythmogenic right ventricular cardiomyopathy, or ARVC. Hyllengren estimated that roughly 70,000 to 75,000 patients in the U.S. have the condition.
The program is focused on reducing electrical instability in the heart, including premature ventricular contractions, or PVCs. Hyllengren said about 90% of patients have more than 500 PVCs per day. He said reducing PVCs could potentially improve outcomes and help prevent serious events, including sudden cardiac death.
According to Hyllengren, TN-401 has maintained a clean safety profile in the clinical data presented so far. He said the company has observed PVC reductions of approximately 60% to 67% across patients, with data extending to 52 weeks in some cases. He added that key opinion leaders have characterized a roughly 50% PVC reduction as clinically meaningful and said Tenaya plans to provide additional durability data and a regulatory update for TN-401 in the fourth quarter.
Although pediatric development remains possible for TN-401, Hyllengren said the company would likely focus more heavily on adult patients for that program.
TN-301 Returns to Development Focus
Tenaya is also advancing TN-301, an HDAC6 inhibitor that had previously been deprioritized while the company focused resources on gene therapy. Hyllengren said the candidate has generated encouraging preclinical and Phase 1 data.
He said the Phase 1 study, which included 72 patients, indicated that TN-301 was safe and well tolerated, engaged its intended target, and had pharmacokinetic and pharmacodynamic characteristics consistent with potential daily dosing.
The company is conducting Phase 2-enabling work, including toxicology studies and trial design planning. Potential indications under consideration include heart failure with preserved ejection fraction, or HFpEF, and Duchenne muscular dystrophy, or DMD, though Hyllengren said other indications could also be evaluated. Tenaya plans to outline its Phase 2 plans in the fourth quarter and aims to begin a Phase 2 study in the second half of next year.
Cash Runway and Alnylam Collaboration
Hyllengren said Tenaya ended the second quarter with approximately $78 million in cash, which the company expects to fund operations through the third quarter of next year. He cautioned that this runway does not include the costs of potential pivotal studies for TN-401 or Phase 2 trials for TN-301.
Once the company has greater regulatory clarity and a clearer view of study designs and costs, it may seek additional financing, he said. Tenaya is also evaluating non-dilutive funding options and could consider partnerships for TN-301 following internally conducted Phase 2 work, particularly if the program advances into large indications requiring late-stage studies involving thousands of patients.
Hyllengren also discussed Tenaya’s recently announced collaboration with Alnylam. The arrangement includes a $10 million upfront payment, research funding and more than $1 billion in potential milestone payments. He said the collaboration is intended to identify novel genetic targets in cardiology, with Alnylam receiving rights to develop and commercialize resulting programs.
About Tenaya Therapeutics (NASDAQ:TNYA)
Tenaya Therapeutics is a clinical‐stage biotechnology company focused on the discovery and development of gene therapy solutions for cardiovascular diseases. Leveraging a proprietary adeno‐associated virus (AAV) platform, the company aims to deliver durable, one‐time treatments for patients suffering from genetic cardiomyopathies and other inherited heart disorders. Its research programs center on optimizing vector design, delivery methods and manufacturing processes to enhance tissue specificity and minimize immune responses.
Founded in 2018 and headquartered in San Carlos, California, Tenaya has built a diversified pipeline of product candidates targeting conditions such as hypertrophic cardiomyopathy and other genetically driven forms of heart disease.
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