Cellivery, Milestone 3rd Efficacy Evaluation New Drug Substance... "Substance Delivered to Takeda"

[Asia Economy Reporter Hyunseok Yoo] Cellivery announced on the 11th that it delivered the new drug candidate 'CV-14' for pharmacokinetic/pharmacodynamic (PK/PD) analysis in animal models of ataxia and hypertrophic cardiomyopathy, the final milestone phase 3 of the 'Development of New Drug Candidates for Brain Neurological Disease Treatment' project, to Takeda Shonan Research Institute, a global pharmaceutical company.

The protein formulation CV-14 delivered to Takeda is a recombinant protein formulation with cell and tissue permeability, applying Cellivery's TSDT platform technology to a specific protein involved in energy synthesis present in the mitochondria of brain nerve cells and cardiac muscle cells. The company explained that it was developed as a new drug candidate. A deficiency of this protein causes Friedreich's ataxia (FRDA) as well as fatal inherited genetic diseases that lead to death by causing cardiac arrhythmia, heart failure, hypertrophic cardiomyopathy, and cardiac arrest.

The company stated, “Currently, there is no fundamental treatment for FRDA patients other than drugs such as antioxidants that temporarily alleviate symptoms. The only fundamental treatment is to directly deliver this deficient mitochondrial protein into brain nerve cells and cardiac muscle cells, and for this purpose, we are currently conducting joint new drug development with Takeda.”

At the end of last year, Takeda directly demonstrated the successful cell permeability and mitochondrial targeting ability of the CV-14 protein. Additionally, the quantitative cell permeability of CV-14, analyzed by confocal microscopy and flow cytometry as a secondary method, was proven to exceed the data performed by Cellivery. The company explained that pharmacokinetic/pharmacodynamic analysis and disease treatment efficacy evaluation tests in the final animal model are currently underway.

A company official emphasized, "If the safety and efficacy of CV-14, already proven by our research team (see therapeutic efficacy photos in the hypertrophic cardiomyopathy animal model), are successfully re-verified by Takeda, it is expected to have a significant impact on future business."

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