GNT Pharma's Nelonemdaz, a multi-target neuroprotection drug, has shown promising results in improving outcomes for stroke patients when administered early in combination with endovascular thrombectomy. The findings, presented at the International Conference Stroke Update 2024 (ICSU 2024) by Professor Jin Soo Lee, indicate a significant benefit when the drug is given within a critical 70-minute window of arrival at the emergency room.
Clinical Trial Results
The results are based on post-hoc analyses of the Phase 3 RODIN trial and data from the Phase 2 SONIC trial. While the initial RODIN trial, involving 498 patients, did not show significant efficacy when Nelonemdaz was administered within 12 hours of ischemic stroke onset in patients undergoing thrombectomy, further analysis revealed potential interactions between treatment effects and time-related metrics. The SONIC trial, published in Stroke Journal in 2022, had previously indicated the safety and efficacy of Nelonemdaz when used with thrombectomy.
A pooled analysis of data from both the SONIC and RODIN trials established that the first infusion of Nelonemdaz within 70 minutes of arrival at the emergency room was crucial for improved functional outcomes. The efficacy of Nelonemdaz was assessed using the modified Rankin Scale (mRS) at 12 weeks post-treatment. The mRS, ranging from 0 (normal) to 6 (death), showed a significantly beneficial shift in distribution with Nelonemdaz (common odds ratio 2.2, p<0.05). Specifically, the proportion of patients achieving functional independence (mRS 0-2) increased from 50.0% in the placebo group to 68.6% in the Nelonemdaz group (p=0.059).
Rationale for Nelonemdaz
Nelonemdaz is designed to reduce brain damage following a stroke or cardiac arrest through a dual-target mechanism. It inhibits the NMDA receptor subtype NR2B to reduce excitotoxicity and removes free radicals to reduce oxidative stress, both major causes of brain cell death after a stroke. According to GNT Pharma, Nelonemdaz's safety has been verified in multiple Phase 1, 2, and 3 trials.
Ongoing and Future Research
Based on these findings, GNT Pharma has initiated multinational Phase 3 RENEW clinical trials. These trials, led by Dr. Lee along with principal U.S. investigators Dr. Raul Nogueira (UPMC Stroke Institute) and Dr. David S. Liebeskind (UCLA Stroke Center), aim to confirm the beneficial effects of Nelonemdaz in ischemic stroke patients undergoing endovascular thrombectomy.
Dr. BJ Gwag, Chairman and CEO of GNT Pharma, expressed enthusiasm about the results and the initiation of the RENEW trials with world-class partners. Dr. Lee added that the post-hoc analyses revealed a specific clinical trial condition showing significant benefit with Nelonemdaz, leading to the establishment of the RENEW trial protocol.
Stroke Statistics and Current Treatment Landscape
Globally, stroke is a significant health burden, with over 5 million stroke-related deaths annually and another 5 million survivors left with permanent disabilities. The World Stroke Organization estimates stroke-related costs to reach US$1 trillion by 2030. In the United States, stroke frequency has increased by 60% over the past 30 years. Ischemic strokes account for approximately 80% of all strokes, requiring emergency medical treatment such as thrombolysis or endovascular thrombectomy.
"In 2015, fast endovascular thrombectomy was proven to improve functional outcome in acute ischemic stroke patients and is the de facto standard for stroke care across the world. It is therefore encouraging that the outcome of stroke patients is further improved by the fast administration of Nelonemdaz along with rapid thrombectomy," Dr. Gwag commented.
