Cellivery Receives Analysis Results of New Ataxia Drug Substance from Takeda... 20% Increase in Activity

[Asia Economy Reporter Hyunseok Yoo] Cellivery announced on the 9th that it received pharmacokinetic and pharmacodynamic analysis results from Takeda regarding the 'development of new drug candidates for neurodegenerative disease treatment' project, evaluating the therapeutic efficacy for ataxia and hypertrophic cardiomyopathy.

According to the company, the pharmacodynamic efficacy evaluation, objectively conducted by a third-party Contract Research Organization (CRO), showed that the biological activity of the heart increased by 20% after just a single administration. The pharmacokinetic results indicated that Cellivery’s TSDT platform normalized brain function deep within the cerebral cortex as well as restored heart function by delivering the new drug candidate, as specified in Takeda’s analysis report. Both companies stated that these results will greatly aid in designing upcoming clinical trials involving human subjects.

At the 11th video conference, Takeda’s chief development officer explained, "The delivery protein was sufficiently detected in the heart and brain, organs where drugs typically have poor penetration, and we were particularly surprised by the detection within the brain in under an hour." The company emphasized that this experiment was very important because if the project succeeds, not only will the licensing out of this new drug candidate be possible, but Cellivery’s TSDT platform technology could also become a licensing out target.

Starting in August, therapeutic efficacy verification tests will be conducted on animal models of brain disease (ataxia) and heart disease (hypertrophic cardiomyopathy) in Japan and the United States, respectively. These tests will reproduce the protocols previously conducted internally by Cellivery to verify reproducibility. The therapeutic efficacy results presented at the recent video conference showed that intravenous injections three times a week at a specific concentration led to a 30% recovery in body weight, a 60% increase in survival rate, and a 73% recovery in heart rate. Additionally, by inhibiting cardiac cell fibrosis and preventing cardiac cell necrosis, heart function was normalized, ultimately demonstrating a life-preserving effect, according to the company.

Cellivery stated, "If the therapeutic efficacy in heart and brain disease animal models, which has already been proven multiple times by our research team, is finally revalidated by Takeda, we expect to enter licensing deal negotiations."

The core of the protein therapy jointly developed with Takeda is that a deficiency of a specific protein in the brain and heart causes Friedreich’s ataxia (FRDA). This leads to fatal inherited genetic diseases causing cardiac arrhythmia, heart failure, hypertrophic cardiomyopathy, and cardiac arrest, resulting in death. The protein drug developed to treat this intractable disease is a cell-penetrating protein formulation whose pharmacological efficacy was proven through pharmacokinetic and pharmacodynamic experiments conducted by Takeda on Cellivery’s development.

The company explained, "Currently, there is no fundamental treatment for FRDA patients other than drugs such as antioxidants that temporarily alleviate symptoms. The only fundamental treatment is directly delivering this deficient protein into brain nerve cells and heart muscle cells. For this purpose, we are currently conducting joint new drug development with Takeda and have succeeded at every stage so far."