Highlights New analysis of ATH434-201 double blind trial strengthens efficacy signal at high dose levelPositive data from ATH434-202 open-label trial demonstrates similar treatment effect in advanced MSA as observed in earlier stage patients in double-blind studyIndependent commercial assessment indicates USD $2.4 billion dollar potential worldwide peak sales opportunity in MSA for ATH434Cash balance on 30 September 2025 of A$54.56M MELBOURNE, Australia and SAN FRANCISCO, Oct. 31, 2025 (GLOBE NEWSWIRE) -- A ...

Core Insights - Annovis Bio, Inc. announced new results indicating that its drug candidate, buntanetap, can reduce inflammation and improve cellular health in Alzheimer's patients, suggesting potential disease-modifying effects beyond just symptomatic relief [1][2]. Group 1: Clinical Study Results - The Phase 2/3 study analysis showed that buntanetap effectively targets key biomarkers of neuroinflammation and neurodegeneration, leading to reductions in inflammatory markers such as IL-5, IL-6, S100A12, IFN-γ, and IGF1R compared to placebo [2]. - Buntanetap also decreased levels of neurofilament light chain (NFL), a marker of neuronal damage, indicating improved cellular integrity and neuronal health, particularly in patients with positive pTau217 plasma levels [2][3]. Group 2: Mechanism and Therapeutic Potential - The drug's ability to target multiple neurotoxic proteins simultaneously is highlighted as a significant advantage, potentially interrupting the toxic cascade associated with Alzheimer's disease [3]. - Previous clinical benefits were noted in mild Alzheimer's patients, who experienced significant cognitive improvements, reinforcing the potential of buntanetap as a disease-modifying therapy [3]. Group 3: Ongoing Research and Future Outlook - Buntanetap is currently undergoing a pivotal Phase 3 trial in early Alzheimer's disease, with a 6-month readout focused on symptomatic improvement and an 18-month readout aimed at assessing disease modification [3][4]. - The full biomarker data will be presented at the Clinical Trials on Alzheimer's Disease (CTAD) conference in December 2025, which is expected to provide further insights into the drug's efficacy [4].

Zorevunersen for Dravet Syndrome - Stoke Therapeutics is developing zorevunersen as a potential disease-modifying medicine for Dravet syndrome [16] - Initial 70mg doses of zorevunersen demonstrated substantial and sustained reductions in convulsive seizure frequency [18] - Ongoing zorevunersen treatment showed substantial and durable reductions in major motor seizure frequency in OLE studies [21] - 36-month OLE data shows continuing improvements in cognition and behavior with zorevunersen treatment [56] - The EMPEROR Phase 3 study is designed to assess disease modification in Dravet syndrome with a dosing regimen of 2x70mg followed by 2x45mg over a 52-week treatment period [31, 32] - The EMPEROR Phase 3 study has dosed its first patient in August 2025 and anticipates data in 2H 2027, randomizing approximately 170 patients across ~70 global sites [35] STK-002 for Autosomal Dominant Optic Atrophy (ADOA) - Approximately 65-90% of ADOA cases are caused by mutations in one allele of the OPA1 gene, leading to haploinsufficiency [64] - Up to 46% of ADOA patients are registered as legally blind [65] - Preclinical findings support further development of STK-002, showing increased OPA1 protein and ATP-linked respiration [70] - A Phase 1 study of STK-002 in ADOA patients is underway in the UK, involving 21 patients aged ≥18 to <55 years receiving single ascending doses (0.1, 0.3, 0.5, & 0.7 mg/eye) [76]

Core Insights - Scinai Immunotherapeutics Ltd. has published new peer-reviewed research confirming the efficacy and safety of PC111, a human anti-Fas Ligand monoclonal antibody, in treating pemphigus and other skin conditions [1][2][3] Scientific and Clinical Findings - The new study validates PC111's ability to prevent blister formation in humanized models of pemphigus by targeting soluble Fas Ligand, which is crucial for keratinocyte apoptosis [2][3] - PC111 has shown significant reduction in disease activity in a proprietary humanized FasL mouse model, confirming its non-immunosuppressive action at the keratinocyte level [3] - The research also indicates PC111's potential in treating Stevens-Johnson syndrome/toxic epidermal necrolysis (SJS/TEN), a severe dermatological condition [4] Market Opportunity - Current treatments for pemphigus and SJS/TEN involve chronic immunosuppression, which can have serious side effects; PC111 offers a differentiated, fast-acting, and safer therapeutic profile [5][6] - The combined market opportunity for pemphigus and SJS/TEN is estimated to exceed one billion dollars annually [6] Development Strategy - Scinai plans to advance PC111 through IND-enabling studies and clinical development, with orphan drug designation already granted in the EU and similar status sought in the U.S. [7] - The companies are exploring accelerated approval pathways that could facilitate market entry within three to four years [7] Program Structure and Leadership - The PC111 program is led by a team of experts including Prof. Carlo Pincelli and Dr. Antonino Amato, supported by Scinai's Scientific Advisory Board [8] - The efficacy of PC111 is backed by multiple peer-reviewed publications, enhancing its credibility [8] - Scinai may receive up to 80% cost coverage through a €15 million FENG grant to support development [8] Regulatory and Strategic Outlook - The combination of orphan designation, high unmet medical need, and a well-defined mechanism of action supports a realistic path toward Breakthrough Therapy designation and/or Accelerated Approval [9]