Guangzhou Magpie Pharmaceuticals presents Phase II Clinical Study Data of its Leading Drug Candidate TBN for Treatment of Amyotrophic Lateral Sclerosis at the 2023 XXVI World Congress of Neurology in Montreal, Canada

02 Oct 2023
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Phase 2Clinical Result
Guangzhou, China, October 2, 2023. Guangzhou Magpie Pharmaceuticals announced its submission of a poster presentation at the XXVI World Congress of Neurology (WCN 2023), showcasing Phase II data of their leading asset, TBN, and its significant success in improving key markers of ALS disease progression. One of these key markers, grip strength, is considered to be one of the most important markers of ALS disease severity and patients’ quality of life, but no drug currently on the market has been shown to significantly improve it. The World Federation of Neurology's XXVI World Congress of Neurology will be in Montreal, Canada, from October 15-19, 2023. The poster titled, “Phase II results for multi-functional tetramethylpyrazine nitrone (TBN) demonstrate significant efficacy improving key markers of ALS progression” is being presented by the study’s principal investigator, Professor Dongsheng Fan of the Department of Neurology at Peking University Third Hospital (Beijing, China). This Phase II trial was prospective, multi-center, randomized, double-blinded, and placebocontrolled. A total of 155 Chinese subjects, aged 45-70 years old, was randomly assigned (1:1:1) to receive TBN 600 mg, 1200 mg or placebo twice daily for 180 days. Eligible patients were within two years after symptom onset and had a decrease of 1-4 points in their ALS Functional Rating Scale–Revised (ALSFRS-R) score during a 12-week observation period before randomization. TBN was safe and well-tolerated, with no drug-related serious adverse events occurring during the study. These results were consistent with TBN safety data from previous clinical studies. The full analysis set (FAS) of the data consisted of 148 patients. The primary and secondary end points of the study, changes in the ALSFRS-R score and forced vital capacity (FVC), respectively, were not met; however, TBN both significantly and statistically improved Grip Strength (32.37%, P=0.037), which no other ALS drug has been shown to do. TBN was more effective in younger patients with slow disease progression—after analyzing a subgroup of 100 patients who were <65 years of age with a 1-2 point decrease in their ALSFRS- R score during a 12-week observation period before randomization. TBN dose-dependently improved the ALSFRS-R score and FVC, while at the same time improving grip strength as found in the FAS patient group. The magnitude of the change TBN brought was also significant: at a 1200 mg dose, compared to placebo, TBN improved ALSFRS-R scores, FVC and grip strength by 22.90%, 37.25% and 50.14% (P=0.011), respectively. TBN also markedly improved bulbar (64.71%, P=0.040) and respiratory function scores (85.92%, P=0.021), achieving statistically significant change at a clinically meaningful magnitude. The principal investigator of the study, Professor Dongsheng Fan, stated that as ALS is a fatal neurodegenerative disease without currently effective treatments, he was optimistic about TBN’s results. He found TBN’s efficacy results in the Phase II data promising, as TBN improved several important markers that have been proven to correlate with increased quality of life for patients, and noted the drug’s particular efficacy in younger patients with slow disease progression. He also stated that in the future, a longer trial is warranted to further evaluate the efficacy and safety of TBN in ALS patients. Dr. Yuqiang Wang, the founder and CEO of Guangzhou Magpie Pharmaceuticals, said, “We are pleased to see these significant results from our phase II clinical study of TBN in ALS patients. ALS is a deadly and ruthless disease. There are currently four US FDA-approved drugs available, but all are severely limited in efficacy and usage. Without a cure, ALS patients deserve more options, and that means medicines with new mechanisms that deliver both on efficacy and safety. This phase II study demonstrated that TBN has the potential to become a new treatment for ALS and we hope we can develop and deliver it to those who desperately need it.” The study was funded by Guangzhou Magpie Pharmaceuticals Co., Ltd., and was registered at the chinadrugtrials.org.cn (CTR20202126) About TBN TBN is a multi-functional experimental drug with novel mechanisms of action on multiple fronts to combat ALS. TBN acts by scavenging damaging ROS, including hydroxyl (•OH), superoxide anion (O2 •– ) and peroxynitrite (ONOO- ) radicals; up-regulating the AMPK/NRF2 pathway; inhibiting mTOR to activate autophagy; protecting mitochondria and promoting mitochondrial biogenesis; and inhibiting inflammation. In addition to treating ALS, TBN has undergone phase II clinical trials for acute ischemic stroke and diabetic kidney disease, both producing promising results. About Guangzhou Magpie Pharmaceuticals Co., Ltd. Guangzhou Magpie Pharmaceuticals Co., Ltd. is a clinical-stage drug discovery and development company. Their mission is to develop innovative medicines for unmet medical needs, with a particular focus on cerebro-cardiovascular, neurodegenerative, mitochondrial dysfunction, and diabetic kidney diseases. Their current projects include potential first-in-class medications targeting ALS, Alzheimer's disease, ischemic stroke, diabetic kidney disease, and pulmonary arterial hypertension, with each drug characterized by novel mechanisms of action. Forward Looking Statements This news release may contain statements that qualify as forward-looking, including descriptions of the Company's intentions, beliefs, or current expectations regarding its business operations and financial conditions. Such statements can be identified by terms like 'will,' 'expects,' 'anticipates,' 'future,' 'intends,' 'plans,' 'believes,' 'estimates,' 'confident,' and similar expressions. It's important to note that these forward-looking statements do not guarantee future performance and are subject to risks, uncertainties, and other factors, some of which are beyond the Company's control and unforeseeable. As a result, actual results may differ from those projected in these forward-looking statements due to various factors and assumptions, including changes and developments in our business, the competitive landscape, and political, economic, legal, and social conditions. The Company and its affiliates, directors, officers, advisors, and representatives have no obligation and do not intend to update these forward-looking statements to reflect new information, future events, or circumstances after the date of this news release, except as required by law. Investor Contact Menghan Zhang Guangzhou Magpie Pharmaceuticals Co., Ltd. menghanzhang@magpiepharma.cn +86 (20) 83980717/+86 13922700333
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