- Best-in-Class Functional Recovery and Survival Benefits seen in an Established Large Animal Model of Pre-existing Severe Non-Ischemic Dilated Cardiomyopathy (DCM) and Chronic Heart Failure (HF)
- Long-term Durability of Efficacy and Highly Favorable Safety Profile Observed in the Study
- IND-Enabling Development of Lead cBIN1 Program Underway, with IND filing Anticipated in 2025
SALT LAKE CITY & SAN FRANCISCO--(BUSINESS WIRE)--TikkunLev Therapeutics, a biotechnology company developing precision therapies targeting the causal disease biology of failing heart muscle, today announced the publication of a seminal efficacy and safety study of its cBIN1 precision therapy in Nature Regenerative Medicine. The study, conducted by TikkunLev’s Co-Founder and CSO, Dr. Robin Shaw, and his team at the Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI) at the University of Utah, demonstrates the therapeutic potential of cBIN1 precision therapy to repair the root cause of failing heart muscle, recover cardiac function, and improve outcomes. TikkunLev’s lead cBIN1 program, TLT-101, is in IND-enabling development for non-ischemic DCM and HF, with an anticipated IND filing in 2025.
The new long-term efficacy and safety study of cBIN1 precision therapy, conducted in minipigs with severe pre-existing non-ischemic DCM and chronic HF, demonstrated:
- Best-in-class recovery of cardiac function and reverse remodeling: A one-time IV delivered low dose of therapy repaired the underlying cardiac muscle pathology observed in HF, and resulted in best-in-class recovery of ejection fraction and reversed remodeling of failing hearts.
- Durable, long-term disease resolution with 100% survival: Long-term recovery of cardiac function, reverse remodeling, and 100% survival (vs. 0% survival in placebo-treated animals) seen at 6 months after a single low IV dose of therapy.
- Favorable safety profile: Safety, toxicology, and histopathology assessments continue to demonstrate a strong safety profile of cBIN1 precision therapy.
“The innovative nature of our approach is that our therapy is designed to correct the architecture of individual heart muscle cells which have become disorganized and inefficient as a result of heart failure,” said Robin Shaw, M.D., Ph.D. Dr. Shaw is also a cardiologist and Professor of Medicine/Cardiology and Director of the CVRTI at the University of Utah. “By rescuing failing heart muscle, we directly address the underlying pathology of heart failure. The unprecedented efficacy of the treatment validates that we have the right target.”
“This new study highlights the extraordinary potential of TLT-101 to dramatically improve the lives of patients living with non-ischemic DCM and HF, which is a major cause of morbidity and mortality worldwide,” said Matt Killeen, Ph.D., President of TikkunLev Therapeutics. “These findings not only validate cBIN1 as a critical therapeutic target in failing heart muscle, but also indicate that TLT-101 could be a revolutionary therapy in the heart failure field.”
Non-ischemic DCM is a common cause of HF, with an estimated prevalence of over 700,000 patients in the United States. The disease manifests as an enlarged and weakened heart and results in high rates of hospitalization and mortality. No currently available therapies address the root cause of failing heart muscle seen in DCM and HF, and patients can rapidly progress to severe disease and face the prospect of an artificial heart or heart transplant. TikkunLev’s lead cBIN1 program, TLT-101, is a first-in-class therapy designed to repair the root cause of failing heart muscle and is in IND-enabling development, with an anticipated IND filing in 2025.
About TikkunLev Therapeutics
TikkunLev Therapeutics is pioneering the development of precision therapies to reverse heart failure by targeting the causal disease biology of failing heart muscle. With a lead program in IND-enabling development for non-ischemic DCM and HF and a broad indication expansion pipeline focused on common, devastating forms of HF, TikkunLev is actively transforming the future treatment landscape for HF patients worldwide. For more information, visit www.tikkunlev.com and follow us on LinkedIn.
About the University of Utah’s CVRTI
The CVRTI is an independent 40,000 square foot cardiac research institute on the University of Utah Health Sciences campus that provides a highly integrated approach to the study of basic and translational cardiac biology. The Institute is the largest freestanding academic team of cardiac muscle biology, metabolism, vascular, and electrophysiology researchers in the country. Facilities include a fully equipped large animal operating room for preclinical studies and extensive on-site translational expertise.
Contacts
For more information, please contact: info@tikkunlev.com
