Latest news with #FastTrackDesignation
Toronto Star
21-07-2025
- Business
- Toronto Star
ProMIS Neurosciences Granted Fast Track Designation by U.S. FDA for PMN310 in the Treatment of Alzheimer's Disease
flag wire: true flag sponsored: false article_type: Syndication : sWebsitePrimaryPublication : publications/toronto_star bHasMigratedAvatar : false : This press release contains forward-looking statements that are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Certain information in this news release constitutes forward-looking statements and forward-looking information (collectively, 'forward-looking information') within the meaning of applicable securities laws. In some cases, but not necessarily in all cases, forward-looking information can be identified by the use of forward-looking terminology such as 'plans', 'pleased to', 'look forward to', 'potential to', 'targets', 'expects' or 'does not expect', 'is expected', 'excited about', 'an opportunity exists', 'is positioned', 'estimates', 'intends', 'assumes', 'anticipates' or 'does not anticipate' or 'believes', or variations of such words and phrases or state that certain actions, events or results 'may', 'could', 'would', 'might', 'will' or 'will be taken', 'occur' or 'be achieved'. In addition, any statements that refer to expectations, projections or other characterizations of future events or circumstances contain forward-looking information. Specifically, this news release contains forward-looking information relating to the Company's progress and expectations for its Phase 1b clinical trial in AD patients, including planned timing for completion and anticipated data readout of interim and full results, the potential for such studies to provide the first proof-of-concept data for PMN310, the potential that PMN310 has the potential to positively benefit patients with AD and to be a more effective and well-tolerated option, the targeting of toxic misfolded proteins in neurodegenerative diseases that the Company believes may directly address fundamental AD pathology (including the belief and understanding that toxic oligomers of Aβ are a major driver of AD) and have greater therapeutic potential due to reduction of off-target activity and the Company's expectations regarding the benefits of Fast Track Designation. Statements containing forward-looking information are not historical facts but instead represent management's current expectations, estimates and projections regarding the future of our business, future plans, strategies, projections, anticipated events and trends, the economy and other future conditions. Forward-looking information is necessarily based on a number of opinions, assumptions and estimates that, while considered reasonable by the Company as of the date of this news release, are subject to known and unknown risks, uncertainties and assumptions and other factors that may cause the actual results, level of activity, performance or achievements to be materially different from those expressed or implied by such forward-looking information, including, but not limited to, the risk that clinical results or early results may not be indicative of future results, the Company's ability to retain and recognize the incentives conferred by Fast Track Designation for PMN310, the Company's ability to fund its operations and continue as a going concern, its accumulated deficit and the expectation for continued losses and future financial results. Important factors that could cause actual results to differ materially from those indicated in the forward-looking information include, among others, the factors discussed throughout the 'Risk Factors' section of the Company's most recently filed Annual Report on Form 10-K for the year ended December 31, 2024 and in its subsequent filings filed with the United States Securities and Exchange Commission. Except as required by applicable securities laws, the Company undertakes no obligation to publicly update any forward-looking information, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.
Associated Press
30-06-2025
- Business
- Associated Press
Vitalgen's Gene Therapy Drug VGN-R09b for Primary Parkinson's Disease Receives FDA Fast Track Designation
SHANGHAI, CHINA, June 30, 2025 / / -- Shanghai Vitalgen BioPharma Co., Ltd. ('Vitalgen') announced that its gene therapy drug, VGN-R09b, for the treatment of Primary Parkinson's Disease (PD), has received Fast Track Designation (FTD) from the U.S. Food and Drug Administration (FDA). VGN-R09b received IND approvals in China and the US in 2024, as a first-in-class recombinant adeno-associated virus (rAAV) gene therapy that targets two key pathways for the treatment of Parkinson's Disease (PD). The Phase I/II clinical trials have been ongoing in China since September, 2024, with all six patients dosed in the Phase I dose-escalation study. Safety data shows that the product is well-tolerated with no findings of VGN-R09b-related adverse events. Preliminary efficacy readouts show that VGN-R09b has a rapid onset of action, with significant gait improvements within two weeks post-treatment. The MDS-UPDRS Part III OFF scores decreased significantly after one month, with an average of 25-point improvements (approximately 50% improvements from the baseline) at six months. Overall, the Hoehn-Yahr (H-Y) scores progressively decreased, along with a reduction in oral medication dosages. The FTD indicates FDA's recognition of the clinical potential of VGN-R09b, marking an important milestone for Vitalgen's expansion into the chronic disease area, and it also represents a global breakthrough for a Chinese gene therapy company in the neurodegenerative field. Together with its FDA IND approval in July 2024, this FTD further validates VGN-R09b's global development potential and will provide expedited support for its future US clinical trials and regulatory filings. About Fast Track Designation (FTD) FTD is an FDA program designed to accelerate the development and approval of drugs that treat serious or life-threatening conditions and have the potential to address unmet medical needs. According to the performance report from the FDA's Center for Biologics Evaluation and Research (CBER), over 800 products have applied for FTD, with a success rate of approximately 68%. After receiving FTD, applicants are given the opportunity for frequent discussions with the FDA regarding their drug development plans, thereby increasing development efficiency. If the product meets certain criteria, it can qualify for accelerated approval, priority review, and rolling reviews, which can further accelerate development and market approval processes. About Parkinson's Disease (PD) Parkinson's Disease (PD) is the second most common neurodegenerative disease, with fast-growing incidences. It is projected that the global PD patient numbers will increase to more than 25 million by 2050. Current PD treatments are primarily small-molecule drugs for symptom relief. As the disease progresses, patients gradually lose response to oral medication, and significant motor complications persist despite fine-tuning the treatment regimens. There is currently no available treatment to delay disease progression or improve long-term outcomes. a: Projections for prevalence of Parkinson's disease and its driving factors in 195 countries and territories to 2050: modeling study of Global Burden of Disease Study 2021. BMJ. 2025;388:e080952. doi:10.1136/bmj-2024-080952. About Vitalgen Vitalgen was founded in March 2020 as a cell and gene therapy (CGT) company based in Shanghai, China, dedicated to translating cutting-edge CGT technologies into clinically accessible treatments for patients worldwide. With proprietary intellectual property rights covering three core CGT platforms: ViVec® (AAV manufacturing platform), ViCas® (CRISPR gene-editing platform) and ViLNP® (Lipid nanoparticle delivery platform), Vitalgen has developed a diversified portfolio of innovative CGTs targeting CNS diseases, ophthalmic conditions, metabolic and hematologic disorders, and oncology, including multiple potential First-in-Class (FIC) products. Up-to-date, Vitalgen has received multiple rounds of investment from renowned venture funds and has established CGT R&D labs and operational centers in the Zhangjiang High-tech Park and a GMP commercial production facility in the Waigaoqiao Free Trade Zone, Shanghai, China. David Wu Vitalgen BioPharma email us here Legal Disclaimer: EIN Presswire provides this news content 'as is' without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.
