GNT Pharma proved the safety and disability improvement of its stroke candidate drug, Nelonemdaz, and announced its phase 3 clinical trial progress at the 2023 International Stroke Conference (ISC)  held last week in Dallas, U.S. 

The results of the phase 2 study showed that 208 moderate or higher-level stroke patients, who underwent thrombectomy within eight hours of onset and later received Nelonemdaz, improved their disability compared to placebo. Furthermore, high doses of approximately 5,250 mg of the drug had a more pronounced effect in patients with severe stroke.

The phase 3 clinical trial for stroke will enroll 496 patients who will be randomly assigned into the Nelonemdaz and placebo groups. Patients will be included if they have an acute ischemic stroke caused by intracranial large vessel occlusion in the anterior circulation and if they are expected to undergo endovascular thrombectomy within 12 hours after stroke onset.

The primary endpoint will be evaluated by a favorable shift in the modified Rankin Scale (mRS) score at 90 days after the first dose of the drug. 

Subsequently, the secondary endpoints include functional independence at 35 and 90 days, the favorable shift of mRS at 35 days, and the occurrence rates of symptomatic intracranial hemorrhage within seven days to evaluate the drug’s ability to protect the brain.

Professor Lee Jin-soo who leads the clinical trial study said, "As the rate of reperfusion increases with the development of intra-arterial thrombectomy, there is a trend of renewed increase in the development of therapeutics to protect the brain and Nelonemdaz is showing superiority.”

The phase 3 trials will be conducted at stroke centers at 24 university hospitals, including Asan Medical Center, said a company official.

"Nelonemdaz, the first multi-target brain cell protection drug optimized for stroke treatment, has a high chance of success in phase 3 clinical trials due to the excellent efficacy and safety confirmed in phase 2 clinical trials,” GNT Pharma CEO Gwag Byoung-joo said. “With patient enrollment in phase 3 clinical trial expected to be completed around April, the launch of our stroke drug Nelonemdaz could be available from as early as the second half of 2024.”