GNT Pharma Co., Ltd.

** IND submitted to the FDA showing significantly improved patient condition when administered within 70 minutes of arrival at the Emergency Room ** *Multinational Phase 3 Clinical Trial protocols confirmed and initiated with leading global stroke research centers * SEOUL, South Korea, Dec. 11, 2024 /PRNewswire/ -- GNT Pharma Co. Ltd, a late-stage pharmaceutical company founded in 1998 with offices in Seoul Korea, Italy and USA, announced today the results of its latest Phase II ('SONIC') and Phase III ('Rodin') clinical trials study showing that stroke patients who received early administration of Nelonemdaz in combination with endovascular thrombectomy, demonstrated significant improvements in outcomes. These results were presented by Professor Jin Soo Lee, Chair of the Department of Neurology at Ajou University Medical Center at the recent International Conference Stroke Update 2024 (ICSU 2024). Dr BJ Gwag, Chairman and CEO of GNT Pharma commented "we are extremely encouraged with the SONIC and RODIN clinical trials results and excited to announce we have initiated multinational Phase 3 Clinical Trials (RENEW) with world class partners, including more than 20 top university hospitals in Korea as well as UCLA and UPMC Stroke Centers in the USA." Nelonemdaz is the world's first "multi-target" neuroprotection drug designed to reduce brain damage after a stroke or cardiac arrest. It offers superior efficacy compared to single-target neuroprotectants offering dual target action: (1) Inhibition of NMDA receptor subtype NR2B to safely reduce excitotoxicity; and (2) Removal of free radicals to reduce oxidative stress – two major causes of brain cell death following a stroke. Unlike non-selective NMDA receptor inhibitors, Nelonemdaz does not cause adverse effects and its safety has been verified in two Phase 1 trials, three Phase 2 trials, and two Phase 3 trials. There are over 5 million stroke-related deaths globally each year, with a further 5 million survivors annually left with permanent disabilities. This puts a huge strain on healthcare systems worldwide - the World Stoke Organization estimates total stroke-related costs to tip US$1 trillion by 2030. Moreover, stroke frequency is not abating, with the USA seeing an increase of 60% over the past 30 years. Almost 80% of all strokes are Ischemic Strokes and require emergency medical treatment, typically thrombolysis or endovascular thrombectomy. Given these alarming statistics, there is a global imperative to further improve the efficacy of current stroke treatments. Dr Gwag adds: "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," The prior Phase 2 SONIC trial results, published in Stroke Journal in 2022, showed the promising efficacy and safety of Nelonemdaz administered to stroke patients receiving thrombectomy. In the latest 498 patient Phase 3 RODIN Trial, administration of Nelonemdaz within 12 hours of ischemic stroke onset did not show significant efficacy in patients treated with thrombectomy. However, post-hoc analyses revealed potential interactions between treatment effects and time-related metrics. The RODIN results are to be published in the 'JAMA' (Journal of the American Medical Association) Network Open. The interactions were shown in the pooled analysis of stroke patients participating in the SONIC and RODIN trials, and in the subsequent subgroup analysis, the first infusion time of Nelonemdaz for improved functional outcome was established to be within 70 minutes from ER arrival. The efficacy of Nelonemdaz was assessed using the modified Rankin Scale (mRS), an ordinal scale ranging from 0 (normal) to 6 (death), at 12 weeks post-treatment. Compared to the placebo, the nelonemdaz caused a significantly beneficial shift in the mRS distribution (common odds ratio 2.2, p<0.05). The proportion of patients in mRS 0-2 corresponding to functional independence was 50.0% in the placebo group and improved to 68.6% in the Nelonemdaz group (p=0.059). Dr Lee adds " "Our post-hoc analyses unveiled a best clinical trial condition that showed a significant benefit of Nelonemdaz, and a protocol for the global phase 3 RENEW trial was established to confirm beneficial effects of Nelonemdaz in ischemic stroke patients in combination with endovascular thrombectomy." Dr. Lee will lead the RENEW trial alongside principal USA investigators Dr. Raul Nogueira, director of the UPMC Stroke Institute and Professor of Neurology and Dr. David S. Liebeskind, Director of the UCLA Stroke Center and Professor of Neurology. About GNT Pharma: GNT Pharma Co., Ltd ('GNTP') was founded in April 1998 by eight highly accomplished professors in subjects ranging from neuroscience and pharmacology to cell biology researching neuronal cell death. Since then, GNTP has discovered and developed an enviable pipeline of late stage drug candidates from nervous system disorders to chronic inflammatory diseases. As well as Nelonemdaz, GNTP's other flagship drug candidates include: Crisdesalazine for the treatment of Alzheimer's and neurodegenerative diseases and depression; and Flusalazine for inflammatory and respiratory diseases including COPD and Asthma. GNTP also has two commercial products in market, including Gedacure (Crisdesalazine) for the treatment of CDS or 'doggy dementia' that shares similar pathology to Alzheimers; and (2) Radipair, skin care products. Forward-Looking Statements Notice: This news release contains statements about expectations, plans, projections, and strategies of GNTP that constitute forward-looking statements to shareholders, investors, and partners. Statements included in this document that are not historical data and fact constitute forward-looking statements about future clinical and financial results. FURTHER INFORMATION: Media Inquiries: Byung Chul Kim, (M) +82-318005-9910; (E) [email protected] Investors/ Korea: Daeyong Kim, (M) +82-704261-0788; (E) [email protected] Investors /International: Sam Cho, (M) + 82-108948-7142; (E) [email protected] SOURCE GNT Pharma WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Promising efficacy and safety profiles were observed in prior Phase II studies in acute ischemic stroke patients treated with recanalization therapies. The ENIS-3 trial has achieved >33% of target enrollment (948 patients). The independent data monitoring committee (IDMC) has completed interim analysis and indicated that the trial should continue. The RODIN trial has achieved >45% of target enrollment (496 patients) in 7 months. YONGIN, South Korea, Sept. 19, 2022 (GLOBE NEWSWIRE) -- GNT Pharma Inc. announces that the independent data monitoring committee (IDMC) for ENIS-3 (Efficacy and safety of Nelonemdaz for Ischemic Stroke) has completed a pre-specified interim analysis encompassing 227 acute ischemic stroke patients treated with placebo or nelonemdaz within 8 hours of onset in China. The IDMC concluded that the ENIS-3 trial should continue as planned without modification. In the prior phase II trial in China (ENIS, 237 patients), administration of nelonemdaz reduced disability primarily in moderate to severe ischemic stroke patients treated with a thrombolytic drug. The ENIS-3 trial was then designed to examine the efficacy and safety of nelonemdaz in 946 patients with moderate to severe acute ischemic stroke treated with tPA within 8 hours of stroke onset. In South Korea, another multicenter, double-blinded phase III trial (RODIN: Rescue on Reperfusion Damage in Cerebral Infarction by Nelonemdaz) has been initiated to further explore the functional and neuroprotection efficacy of nelonemdaz in acute cerebral ischemia and reperfusion, aiming to enroll a total of 496 patients with moderate to severe ischemic stroke who receive endovascular thrombectomy within 12 hours of stroke onset. The RODIN trial has now enrolled more than 224 patients over the last 7 months and is expected to complete enrollment in the middle of 2023. In the prior phase II study published online in the peer-reviewed journal Stroke in September, nelonemdaz treatment was associated with better functional outcomes than placebo in moderate to severe ischemic stroke patients who received endovascular thrombectomy within 8 hours of stroke onset. Good functional outcomes were analyzed by a fraction of 0 (no disability symptoms) – 2 (slight disability, able to look after own affairs without assistance) on the modified Rankin Scale (mRS) and a Barthel index of >90 (functional independence in activities of daily living) at 12 weeks. An mRS score of 0–2 was achieved in 54.1% (33/66) and 63.2% (36/57) patients in the placebo and high-dose groups treated with 5,250 mg nelonemdaz, respectively. The common odds ratio (90% confidence interval) indicating a favorable shift in mRS scores was 1.61 (0.94–2.76) between the placebo and high-dose groups. A Barthel index of >90 was observed in 24 (43.6%) and 34 (63.0%) patients in the placebo and high-dose groups, respectively. An overall trend towards excellent outcome (mRS score of 0 and BI of >90) and reduced mortality at 12 weeks, was observed in the nelonemdaz treatment groups compared to placebo. Dr. Dennis W. Choi, Professor of Neurology, Stony Brook Medicine, and a pioneer in identifying pathological mechanisms of ischemic brain injury said, “Phase III studies of nelonemdaz appear to be progressing well. These trials represent a key shot on the worldwide goal of developing an effective neuroprotective treatment for ischemic stroke.” Nelonemdaz is a multi-target neuroprotectant acting as both a moderate NR2B-selective N-methyl d-aspartate (NMDA) receptor antagonist and a potent spin trapper that ameliorates both fast NMDA receptor-mediated neuronal death and the delayed free-radical mediated brain cell degeneration occurring hours-to-days after ischemic stroke. In animal models of stroke, superior safety and efficacy was observed for nelonemdaz treatment compared with NMDA antagonists or antioxidants alone. In prior phase I and phase II studies, administration of nelonemdaz was well-tolerated, with no appearance of the psychotic symptoms seen in humans exposed to previous NMDA receptor antagonist drugs. “Recent advances in recanalization therapy remarkably improve functional outcomes after acute ischemic stroke and open up new opportunities for neuroprotection therapy based on early delivery and reperfusion,” said Dr. Byoung Joo Gwag, founder and CEO of GNT Pharma. “Nelonemdaz is safe and shows promising efficacy profiles in patients receiving thrombolytic or endovascular therapy. When combined with recanalization therapies, nelonemdaz will hopefully break new ground in treating ischemic brain injury, reducing consequent disability and mortality.” About nelonemdaz Nelonemdaz is a breakthrough multi-target neuroprotectant drug candidate, acting both as a moderately powerful, NR2B subtype-selective NMDA receptor antagonist, and a powerful antioxidant. It is designed to safely ameliorate NMDA receptor-mediated excitotoxicity while also reducing free radical toxicity, two major routes of brain cell death after stroke. About the phase II ENIS Results In China, a phase II study of nelonemdaz (ENIS trial) has been completed in acute ischemic stroke patients receiving a thrombolytic drug within 8 hours of stroke onset (n=237). Administration of nelonemdaz for 5 days was well-tolerated and showed promising efficacy in moderate to severe stroke patients with initial National Institutes of Health Stroke Scale (NIHSS) scores of ≥6 on admission. The fraction of recovery to normal in placebo-treated group was 13%, 16%, and 26% at 14 days, 30 days, and 90 days, respectively, which improved to 25%, 34% and 44% in the group treated with high dose nelonemdaz. About the phase II SONIC Results In South Korea, a phase II, double-blind, randomized, placebo-controlled, multi-center study (SONIC trial) has been conducted to assess the safety and efficacy of nelonemdaz in acute ischemic stroke patients treated with endovascular recanalization within 8 hours of stroke onset. SONIC enrolled 208 acute ischemic stroke patients with NIHSS score of ≥8. Patients randomly received infusions of placebo, 500 mg nelonemdaz (low-dose group), or 750 mg nelonemdaz (high-dose group) prior to thrombectomy. Low-dose and high-dose nelonemdaz groups then received 9 consecutive infusions of 250 mg and 500 mg every 12 hours, respectively. The primary outcome was the percentage of patients achieving at least a good functional outcome 12 weeks later. Good functional outcome was defined as 0 (no disability symptoms) – 2 (slight disability, able to look after own affairs without assistance) on the modified Rankin Scale (mRS), the most widely used outcome measure in stroke research. In the full analysis set encompassing 183 patients, an mRS score of 0–2 at 12 weeks was achieved in 54.1% (33/66), 61.5% (40/65), and 63.2% (36/57) patients in the placebo, low-dose, and high-dose groups, respectively. In secondary outcomes, the common odds ratio (90% confidence interval) indicating a favorable shift in mRS scores at 12 weeks was 1.55 (0.92–2.60) between the placebo and low-dose groups and 1.61 (0.94–2.76) between the placebo and high-dose groups. A Barthel index of >90 (functional independence in activities of daily living) at 12 weeks was observed in 24 (43.6%), 34 (54.8%), and 34 (63.0%) patients in the placebo, low-dose, and high-dose groups, respectively. An overall trend towards excellent outcome (mRS score of 0 and BI of >90) and reduced mortality at 12 weeks, was observed in the nelonemdaz treatment groups compared to placebo. No serious adverse events were observed in nelonemdaz treatment groups. The SONIC trial results have been published in the journal Stroke in September. About the phase III ENIS-3 Trial Based upon the encouraging results observed in the ENIS trial, Apeloa Pharmaceutical of Hengdian Group, GNTP’s Chinese partner, initiated in December 2020 an ongoing phase III study of nelonemdaz in 948 patients with moderate to severe (9≤NIHSS≤22) acute ischemic stroke (ENIS III), engaging 40 Chinese university stroke centers. All patients receive a thrombolytic drug tPA prior to administration of placebo or nelonemdaz within 8 hours of stroke onset. About the phase III RODIN Trial In September 2021, the Korea Ministry of Food and Drug Safety (MFDS) approved an Investigational New Drug Application (IND) for a phase III study of nelonemdaz in acute ischemic stroke patients (RODIN trial). RODIN is designed to investigate the safety and efficacy of 5,250 mg nelonemdaz in 496 patients with severe ischemic stroke receiving endovascular thrombectomy within 12 hours of stroke onset. Patients will be enrolled at 23 university stroke centers and evaluated by mRS, NIHSS, BI, and MRI over 90 days following stroke. At present, RODIN has enrolled 224 patients at 21 university stroke centers including Seoul Asan Medical Center. About GNT Pharma: GNT Pharma was founded in April 1998 by eight professors in the fields of neuroscience, pharmacology, medicinal chemistry, psychology, ophthalmology, and cell biology who had all been investigating mechanisms of neuronal cell death. Since then, GNTP has discovered and developed several innovative drug candidates for the treatment of nervous system disorders such as stroke and Alzheimer’s disease. Disclosure Notice This press release includes forward-looking statements about GNT Pharma’s efforts to treat acute ischemic stroke and the investigational anti-stroke drug nelonemdaz that are based on management’s beliefs and assumptions, expectations, estimates, and projections as well as facts and factors currently known by GNT Pharma. Statements included in this release that are not historical data and facts, including the words “plans”, “expects”, or similar expressions and statements constitute forward-looking statements. Such statements are subject to certain risks and uncertainties that could cause actual results to differ materially from those expressed in these forward-looking statements. Risks and uncertainties include those associated with the impact COVID-19 could have on our business, and including but not limited to, those inherent in research and development, including completion dates of clinical trials, final data from clinical trials, and the regulatory process. You are cautioned not to rely on these forward-looking statements as there are important factors that could cause actual results to differ materially from forward-looking statements. GNT Pharma undertakes no obligation to update forward-looking statements. Contacts 1. Eun Chan Park, PhD (US Office): ecpark@gntpharma.com (1-848-228-6841) 2. Chang Geun Kim, PhD: cgkim@gntpharma.com (+82-70-4261-0786) 3. Byung Chul Kim, Public Relations Director: bckim@gntpharma.com (+82-70-4262-4755)

Los Angeles, USA , April 21, 2021 (GLOBE NEWSWIRE) -- Spinal Cord Injury Clinical Trials Review: A Multitude of Emerging Drugs for Better Treatment Around 40+ notable companies such as Kringle Pharma, Eusol Biotech, Pharmicell, Pharmazz, AbbVie, Acorda Therapeutics, Geron Corporation, ReNetX Bio, GNT Pharma, AlaMab Therapeutics, Mitsubishi Tanabe Pharma, Kancera, Olatec Therapeutics, Scholar Rock, AXONIS Therapeutics, K-STEMCELL, Athersys, AmKor Pharma, Histocell, Lineage Cell Therapeutics, and others have their Spinal Cord Injury drug candidates in the pipeline to influence treatment scenario. DelveInsight’s “Spinal Cord Injury Pipeline Insight” report provides comprehensive insights about 40+companies and 40+ pipeline drugs in the Spinal Cord Injury pipeline landscapes. It comprises Spinal Cord Injury pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Spinal Cord Injury therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive Spinal Cord Injury pipeline products. Some of the key takeaways of the Spinal Cord Injury Pipeline Report Get an overview of pipeline landscape @ Spinal Cord Injury Clinical Trials Analysis Spinal Cord Injury is traumatic damage or nerves at the end of the spinal canal. This affects the conduction of sensory and motor signals across the site of the lesion. This disease is a debilitating neurological condition with a tremendous socio-economic impact on affected individuals and the health care system. Spinal Cord Injuries patients require multidisciplinary care management to achieve optimal health outcomes. Treatments for chronic Spinal Cord Injury focus on avoiding or improving distinct pathophysiological mechanisms, such as glial scar formation, demyelination, and astrogliosis. Treatment strategies for acute Spinal Cord Injury are limited to preventing further damage; the therapeutic approach for chronic Spinal Cord Injury focuses on promoting neuronal regeneration and treating accompanying symptoms of chronic complications. Spinal Cord Injury Emerging Drugs HGF (KP-100IT) protects neurons, promotes axons’ extension, and can be developed as a potential treatment for Spinal Cord Injury. It is currently in Phase III and is produced by Kringle Pharma. Research and Development Phase III NCT04475224: In July 2020, Kringle Pharma initiated A Non-randomized, Multicenter, and Confirmatory Study by Intrathecal Administration of KP-100IT in Subjects with Acute Spinal Cord Injury. This study is a non-randomized, multicenter, confirmatory study by intrathecal administration of KP-100IT, code of HGF (Hepatocyte Growth Factor) formulation for intrathecal injection, in subjects with acute spinal cord injury. The trial is expected to get completed by September 2022 with expected 25 enrolled participants. Results: Primary endpoints in Phase I/II, the safety and tolerability of HGF administration to acute Spinal Cord Injury patients, were confirmed. Although there was no significant difference in the change in ASIA motor score from baseline at week 24, which was used as the primary endpoint of efficacy, the HGF-treated group showed a consistent functional recovery trend throughout the 6-month observation period compared with the placebo group. Moreover, the two groups significantly differed about the change in the ASIA motor score from baseline at week 20, which was the secondary endpoint of efficacy. Thus, the proof of concept (POC) for HGF treatment in Acute Spinal Cord Injury was established. ES135 is a recombinant human acidic fibroblast growth factor (rhFGF1) with 135 amino acids. ES135 promote neurite outgrowth. The sequence of ES135 has been patented in Taiwan, the EU, China and the United States. ES135 is undergoing a multi-centre, double-blind, randomised, placebo-control study as the pivotal Phase 3 clinical trial for Spinal Cord Injury. For further information, refer to the detailed report @ Spinal Cord Injury Pipeline Therapeutics Scope of Spinal Cord Injury Pipeline Drug Insight Key Questions regarding Current Spinal Cord Injury Treatment Landscape and Emerging Therapies Answered in the Pipeline Report Table of Contents Get a customised pipeline report @ Spinal Cord Injury Drugs Pipeline Report Related Reports DelveInsight's Spinal Cord Injury (SCI)- Market Insights, Epidemiology and Market Forecast - 2030 report provides a detailed overview of the disease and a depth understanding of historical and forecasted epidemiology. DelveInsight's Spinal Cord Injury (SCI) - Epidemiology Forecast 2030 report delivers an in-depth understanding of the disease, historical, and forecasted epidemiology of the disease. DelveInsight's Chronic Spinal Cord Injury - Epidemiology Forecast 2030 report delivers an in-depth understanding of the disease, historical, and forecasted epidemiology of Chronic Spinal Cord Injury in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan. DelveInsight' s Spinal Fusion - Market Insights, Epidemiology and Market Forecast - 2030 report provides a detailed overview of the disease and a depth understanding of historical and forecasted epidemiology. DelveInsight' s Spinal Muscular Atrophy (SMA) - Market Insights, Epidemiology and Market Forecast - 2030 report provides a detailed overview of the disease and an in-depth understanding of historical and forecasted epidemiology. About DelveInsight DelveInsight is a leading Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.