First Oral Presentation of the Tanruprubart Real-World Evidence (RWE) Study by International Guillain-Barré Syndrome Outcomes Study (IGOS) Researchers Highlights Benefits over Current Standard of Care in Matched Patient Populations Poster Presentations on New Pivotal Phase 3 Analyses Underscore Tanruprubart’s Rapid and Sustained Treatment Effect and Improvement in Quality of Life Compared to Placebo BRISBANE, Calif., May 19, 2025 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a biopharmaceutical company ...

Financial Data and Key Metrics Changes - The company is focused on executing the clinical development plan for NurOwn and has received FDA clearance to initiate a pivotal Phase 3b trial for ALS [6][10] - Financial constraints have been acknowledged, but the company has made significant strides in trial preparations despite limited resources [13][25] Business Line Data and Key Metrics Changes - The NurOwn technology platform is central to the company's strategy, with ongoing clinical trials aimed at demonstrating its therapeutic benefits [6][12] - The company is actively negotiating clinical trial agreements with approximately 15 leading clinical centers across the U.S. for the Phase 3b trial [10][25] Market Data and Key Metrics Changes - The ALS community's strong interest in NurOwn is highlighted by the engagement of renowned ALS clinicians and researchers [10] - The company participated in the annual ALF Drug Development Summit, indicating its active involvement in the ALS therapeutic development landscape [12] Company Strategy and Development Direction - The company aims to secure strategic funding to support the trial, with a focus on partnerships and non-dilutive grants [14][26] - Expansion of manufacturing capabilities in the U.S. is a key strategic objective, with plans to announce a letter of intent with a U.S.-based facility [30] Management's Comments on Operating Environment and Future Outlook - Management emphasized the urgency for innovative therapeutic options in ALS and the commitment to executing the trial with scientific rigor [11][12] - The company is optimistic about the potential of NurOwn and is preparing for various funding opportunities to ensure timely trial commencement [14][26] Other Important Information - The trial, named "Endurance," is designed to resonate with the ALS community, reflecting the strength and resilience of ALS patients [27] - The company is encouraged by progress in its exosome program, which shows potential in treating respiratory and inflammatory diseases [34] Q&A Session Summary Question: Can you start the trial without proper funding? - Management stated that while significant progress has been made, proper funding is essential to commence the trial [25][26] Question: What is the meaning of the trial name "Endurance"? - The name was chosen to resonate with the ALS community and reflects the company's commitment to delivering a therapeutic option for ALS [27] Question: Will the company also be producing in the U.S.? - The company confirmed plans to expand its manufacturing footprint in the U.S. and will announce a letter of intent with a U.S.-based facility [30] Question: Can you update on any advances in the exosome program? - Management expressed encouragement regarding the exosome program's progress and is preparing to advance it towards clinical development [34] Question: Have you had any communications with FDA regarding stratification by UNC13A? - Management indicated that while the FDA has not approved any biomarker as a surrogate, they are excited about the genetic discoveries and will explore them further [39][40] Question: Can the mechanism of action be used as part of the data package for the next BLA? - Management confirmed that the protective effect observed in cell cultures could support the data package for future filings [43] Question: How many clinical trial sites are you looking to open? - Management confirmed plans to open approximately 15 clinical trial sites and is finalizing agreements to begin patient enrollment [48][50]

NRx Pharmaceuticals (NRXP) Q1 2025 Earnings Call May 15, 2025 04:30 PM ET Company Participants Matthew Duffy - Chief Business Officer & Co-CEO of Hope TherapeuticsJonathan Javitt - Founder, Chairman, Chief Scientist & Co-CEO of Hope TherapeuticsMichael Abrams - CFO Conference Call Participants Thomas Shrader - MD & Healthcare AnalystEdward Woo - Director of Research & Senior Analyst Operator Good afternoon, ladies and gentlemen, and welcome to the NRx Pharmaceuticals First Quarter twenty twenty five Earning ...

NRx Pharmaceuticals (NRXP) Q1 2025 Earnings Call May 15, 2025 04:30 PM ET Speaker0 Good afternoon, ladies and gentlemen, and welcome to the NRx Pharmaceuticals First Quarter twenty twenty five Earnings Call. At this time, all lines are in a listen only mode. Following the presentation, we will conduct a question and answer session. If at any time during this call, you require immediate assistance, please press 0 for the operator. This call is being recorded on Thursday, 05/15/2025. I would now like to turn ...

Company has continued to advance its previously announced plan to obtain FDA approval for two new drugs and to develop a network of clinics focused on neuroplastic therapies to treat severe and suicidal depression, PTSD, and related conditions. Clinic acquisition financing is at the HOPE Therapeutics level and non-dilutive to NRXP shareholders In January 2025 completed the third tranche of a convertible note offering and registered direct equity offering to an institutional investor on favorable terms to t ...

Term sheet with Universal Capital, LLC to fund HOPE Therapeutics clinic acquisition strategy Funding tied to already-announced acquisitions, with additional tranches contemplated for further growth, subject to standard due diligence Together with previously announced term sheet with a strategic investor, HOPE funding of $10.3 million is planned in the near term  MIAMI, May 15, 2025 /PRNewswire/ -- HOPE Therapeutics™, Inc. ("HOPE"), a multi-site clinical care delivery organization and wholly-owned subsidiar ...

Quince Therapeutics ( QNCX ) is pursuing the treatment of ataxia-telangiectasia, a rare neurodegenerative disorder, which starts in early childhood. I have covered the company in the summer of 2024, and the stock has performedI am working in the financial sector. My areas of interest are technology, biotech, pharmaceutical companies, banks and a bit of shipping when an actionable trade passes on my radar.Analyst’s Disclosure: I/we have a beneficial long position in the shares of QNCX either through stock ow ...

Group 1 - Proactive provides fast, accessible, informative, and actionable business and finance news content to a global investment audience [2] - The news team covers medium and small-cap markets, as well as blue-chip companies, commodities, and broader investment stories [3] - Proactive's content includes insights across various sectors such as biotech, pharma, mining, natural resources, battery metals, oil and gas, crypto, and emerging technologies [3] Group 2 - Proactive is committed to adopting technology to enhance workflows and content production [4] - The company utilizes automation and software tools, including generative AI, while ensuring all content is edited and authored by humans [5]

Core Viewpoint - Long COVID is increasingly recognized as a serious condition affecting millions, with no approved treatment options currently available, highlighting the urgent need for effective therapies [1][2][4] Group 1: Long COVID Overview - Long COVID impacts approximately 20 million adults in the U.S. and millions more globally, with 10-30% of COVID-19 patients experiencing lingering symptoms such as fatigue and cognitive impairment [2][5] - The economic impact of long COVID is significant, estimated at $3.7 trillion due to loss in quality of life, earnings, and increased medical costs [5] Group 2: Bezisterim Development - BioVie Inc. has initiated the Phase 2 ADDRESS-LC clinical trial to evaluate bezisterim (NE3107) for treating neurological symptoms associated with long COVID, with topline data expected in the first half of 2026 [1][3] - Bezisterim targets neuroinflammation, which is believed to play a central role in long COVID symptoms, and has shown potential in reducing chronic symptoms in trials for Alzheimer's and Parkinson's diseases [1][3][6] Group 3: Clinical Trial Details - The ADDRESS-LC study is a randomized, placebo-controlled trial involving approximately 200 patients with long COVID experiencing cognitive impairment and fatigue [3][8] - The trial is fully funded by a $13.13 million grant from the U.S. Department of Defense [10] Group 4: Mechanism of Action - Bezisterim is an orally bioavailable, blood-brain barrier-permeable compound that modulates inflammatory pathways implicated in chronic inflammation, potentially improving symptoms in long COVID patients [6][8] - The drug is designed to reduce neuroinflammation and address metabolic dysfunction, which may help restore normal function and improve quality of life for affected individuals [3][4] Group 5: Broader Implications - The persistent presence of SARS-CoV-2 proteins and the resulting inflammatory pathways are linked to prolonged neuroinflammation and symptoms in long COVID patients, indicating a need for targeted interventions [4][8] - BioVie is also conducting trials for bezisterim in Parkinson's and Alzheimer's diseases, demonstrating its broader therapeutic potential [7][9]

Core Insights - Masitinib demonstrates neuroprotective effects by lowering serum neurofilament light chain (NfL), a key biomarker for neurodegenerative disorders such as multiple sclerosis, amyotrophic lateral sclerosis, and Alzheimer's disease [1][4][6] Group 1: Research Findings - The study published in PLOS One shows that masitinib can significantly reduce serum NfL levels, indicating its potential neuroprotective effects [1][3] - At Day 8, masitinib treatment reduced NfL levels by 43% at a dosage of 50 mg/kg/day and by 60% at 100 mg/kg/day, demonstrating a dose-dependent response [5][11] - Absolute serum NfL concentrations were approximately 25% lower in both masitinib treatment groups compared to the EAE control group at Day 8, with further reductions observed at Day 15 [5][6] Group 2: Mechanism of Action - Masitinib targets the innate neuroimmune system, specifically mast cells and microglia, which are implicated in the pathophysiology of neurodegenerative diseases [4][7] - The drug has shown clinical benefits in previous trials for progressive multiple sclerosis, ALS, and mild-to-moderate Alzheimer's disease, reinforcing its therapeutic promise [4][6] Group 3: Functional Performance - Masitinib treatment improved grip strength in EAE mouse models, with treated mice recovering to baseline levels by Day 15, indicating a protective effect on motor function [11] - Both treatment groups exhibited significantly less relative deterioration in grip strength compared to the EAE control group (p < 0.001) [11]