AIM ImmunoTech Conference Summary Company Overview - AIM ImmunoTech is a late-stage immunopharma company focused on the drug Ampligen (rintatolimod), which has shown efficacy and safety in various solid tumors, particularly in oncology since its repurposing from chronic fatigue syndrome in 2016-2017 [1][2] Core Points and Arguments - **Pancreatic Cancer Focus**: The company is prioritizing the approval of Ampligen for pancreatic cancer, having initiated a program in 2017 with a Dutch government-approved early access program for late-stage patients [2][4] - **Clinical Trials**: Currently conducting a Phase I-II trial combining Ampligen with AstraZeneca's Imfinzi (durvalumab) at Erasmus Medical Center, with approximately 75 subjects treated so far [2][6] - **Mechanism of Action**: Ampligen is described as converting "cold tumors" into "hot tumors" by altering the tumor microenvironment, specifically the ratio of suppressor T cells to killer T cells [3][4] - **Safety Profile**: The drug has a well-established safety profile with over 100,000 IV doses administered, and approvals for various administration methods [4][5] - **Market Opportunity**: The company sees a significant opportunity in the pancreatic cancer market, supported by patent and orphan drug designations that provide market exclusivity [5][6] Key Data and Results - **Survival Benefits**: In the early access program, the median overall survival for 57 subjects was 19.7 months, compared to 12.5 months for the standard of care, resulting in an 8.6-month survival benefit [8][17] - **Quality of Life**: Patients reported significant improvements in quality of life, which is crucial given the poor quality of life associated with late-stage pancreatic cancer [8][19] - **Immunological Markers**: Specific biomarkers, such as the neutrophil-lymphocyte ratio and CA19-9 levels, have been identified as predictive of Ampligen's response and overall survival [9][10] Future Milestones - **DURIPANC Program**: The program aims to treat 25 subjects, with 18 currently in treatment. The last subject is expected to be treated by the end of the year, which will be a significant milestone [20][21] - **Phase III Trial Planning**: The company is already planning for a Phase III trial, with the goal of demonstrating continued positive impacts on survival and quality of life [21][22] Additional Important Information - **Patent Protection**: A recent U.S. patent covering Ampligen in combination with PD-L1 for various cancers is valid until 2039, enhancing the company's market position [5][6] - **Orphan Drug Designation**: This designation provides up to 10 years of market protection in the EU, commencing upon drug approval, which is critical for the company's strategy [6][7] This summary encapsulates the key points from the AIM ImmunoTech conference, highlighting the company's focus on pancreatic cancer, the efficacy and safety of Ampligen, and the strategic planning for future clinical trials and market opportunities.

SHELTON, CONNECTICUT / ACCESS Newswire / February 10, 2026 / NanoViricides, Inc., a publicly traded company (NYSE American:NNVC) (the "Company"), and a clinical stage, leading global pioneer in the development of broad-spectrum antivirals based on host-mimetic nanomedicine technology that viruses cannot escape, announced today that it has filed an application for "Orphan Drug Designation" (ODD) for NV-387 as a Treatment for Measles with the US FDA Office of Orphan Products Development (OOPD). If approved, o ...

Core Insights - Sanofi's rilzabrutinib has received breakthrough therapy designation from the FDA for treating warm autoimmune hemolytic anemia (wAIHA) and orphan drug designation in Japan for the same condition [1][2][3] Group 1: Designations and Studies - The breakthrough therapy designation is based on clinical data from the ongoing LUMINA 2 phase 2b study, which assesses the efficacy and safety of rilzabrutinib for wAIHA patients [2] - A new LUMINA 3 phase 3 study is also underway, comparing rilzabrutinib with placebo in wAIHA patients [2] - There are currently no approved treatments specifically targeting the underlying cause of wAIHA, which can lead to anemia and serious organ damage [2] Group 2: Rilzabrutinib Overview - Rilzabrutinib is a novel oral, reversible Bruton's tyrosine kinase (BTK) inhibitor that aims to restore immune balance through multi-immune modulation [7][8] - It is already approved in the US, EU, and UAE for treating immune thrombocytopenia (ITP) and is under regulatory review for ITP in Japan [4][8] - Rilzabrutinib has received multiple designations, including orphan drug status for autoimmune hemolytic anemia and other rare diseases [5] Group 3: About wAIHA - wAIHA is a rare autoimmune disorder characterized by the destruction of red blood cells, affecting 4 to 24 people per 100,000 in the US and EU, and 3 to 10 people per million in Japan [6] - Symptoms include fatigue, dizziness, palpitations, and shortness of breath, with potential complications such as thromboembolism [6] Group 4: Company Overview - Sanofi is an R&D driven biopharma company focused on improving lives through innovative medicines and vaccines, with a commitment to addressing urgent healthcare challenges [10]

Core Viewpoint - Quoin Pharmaceuticals has received confirmation from the Japanese MHLW that its lead product candidate QRX003 qualifies for both Orphan Drug Designation and Fast Track regulatory review in Japan, aiming to self-commercialize the product if approved [1][3]. Group 1: Regulatory Designation - QRX003 has been granted Orphan Drug Designation by both the U.S. FDA and the European Medicines Agency in 2025, and is now seeking similar status in Japan [1][2]. - The MHLW's Orphan Drug Designation program offers benefits such as R&D subsidies, tax credits for clinical testing, reduced application fees, priority review, and ten years of market exclusivity if approved [2]. Group 2: Clinical Development - QRX003 lotion (4%) is currently being evaluated in two late-stage pivotal clinical trials for Netherton Syndrome, with enrollment expected to be completed in the first half of 2026 [3]. - Top-line data from these trials is anticipated in the second half of 2026, with a New Drug Application (NDA) submission planned for late 2026 or early 2027 [3]. Group 3: Company Strategy - The company plans to establish its own commercial infrastructure in Japan, which is one of the three core territories for QRX003 and other pipeline products [3]. - Quoin Pharmaceuticals is committed to completing the clinical development of QRX003 urgently to address the needs of patients suffering from Netherton Syndrome [3].

Core Insights - OSE Immunotherapeutics acknowledges the FDA's Orphan Drug Designation for pegrizeprument (VEL-101), aimed at preventing organ rejection in liver transplant patients [1][3] Group 1: Product Development - Pegrizeprument is a novel investigational immunomodulatory monoclonal antibody fragment, originally developed by OSE Immunotherapeutics and licensed to Veloxis Pharmaceuticals in 2021 for all transplant-related indications [2][6] - The drug is designed to block CD28-mediated T cell activation while allowing CTLA-4 mediated immunosuppressive functions, indicating a dual mechanism of action [5] Group 2: Market Implications - The Orphan Drug Designation highlights the need for improved treatment options in solid organ transplantation, marking a significant milestone in the development of pegrizeprument [3][4] - Veloxis Pharmaceuticals is responsible for the global development, manufacturing, and commercialization of pegrizeprument, which is currently being developed for kidney transplantation [2][6] Group 3: Company Overview - OSE Immunotherapeutics is a biotech company focused on developing first-in-class assets in immuno-oncology and immuno-inflammation, partnering with leading institutions to address unmet patient needs [7]

Core Insights - Dyne Therapeutics has received Orphan Drug designation in Japan for zeleciment basivarsen (z-basivarsen) to treat myotonic muscular dystrophy type 1 (DM1), highlighting the urgent need for new therapies in this area [1][2] - Z-basivarsen has shown early and sustained improvements in myotonia, muscle strength, and function, with a favorable safety profile, and has also received similar designations in the U.S. and Europe [2][4] - The ACHIEVE trial is a Phase 1/2 clinical trial evaluating z-basivarsen, with a registrational dose of 6.8 mg/kg administered every eight weeks, aiming for potential regulatory submissions [3][4] Company Overview - Dyne Therapeutics focuses on delivering functional improvement for individuals with genetically driven neuromuscular diseases, including DM1 and Duchenne muscular dystrophy (DMD) [6][7] - The company is advancing clinical programs targeting muscle and the central nervous system to address the root causes of these diseases [7] Clinical Trial Details - The ACHIEVE trial is a global, randomized, placebo-controlled, double-blind study assessing the safety, tolerability, and efficacy of z-basivarsen in DM1 patients [3] - The primary endpoint for the registrational expansion cohort is the change in middle finger myotonia measured by video hand opening time (vHOT) at 6 months compared to placebo [3] Disease Context - Myotonic dystrophy type 1 (DM1) is a rare genetic neuromuscular disease affecting approximately 40,000 people in the U.S. and 55,000 in the EU, characterized by high morbidity and early mortality [5] - Symptoms of DM1 can vary widely and include muscle weakness, myotonia, cognitive impairments, and other systemic issues, with no approved disease-modifying treatments currently available [5]

Core Insights - Atossa Therapeutics has received Orphan Drug Designation from the FDA for (Z)-endoxifen to treat Duchenne muscular dystrophy (DMD), which is a significant milestone for the company in its development efforts for this serious disease [1][2] Group 1: Company Overview - Atossa Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies in oncology and other areas with high unmet medical needs [7] - The company's lead product candidate, (Z)-endoxifen, is being evaluated for its potential applications in oncology and rare diseases [5][7] - Atossa has a growing global intellectual property portfolio supporting the (Z)-endoxifen program, including multiple recently issued U.S. patents and numerous pending applications worldwide [6] Group 2: Product Information - (Z)-endoxifen is a potent Selective Estrogen Receptor Modulator/Degrader (SERM/SERD) with demonstrated activity across multiple mechanisms of interest, showing a favorable safety profile distinct from tamoxifen [5] - The drug is not yet approved for any indication, and the company plans to continue engaging with the FDA as it advances its development efforts [2][5] Group 3: Disease Context - Duchenne Muscular Dystrophy (DMD) is a rare, progressive, X-linked neuromuscular disorder caused by mutations in the dystrophin gene, leading to severe symptoms and a substantial unmet medical need for effective treatments [4]

As filed with the Securities and Exchange Commission on January 16, 2026 Registration No. 333-291852 UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM S-1 Amendment No. 3 REGISTRATION STATEMENT UNDER THE SECURITIES ACT OF 1933 Edison Oncology Holding Corp. (Exact name of registrant as specified in its charter) (State or other jurisdiction of incorporation or organization) (Primary Standard Industrial Nevada 2836 83-1614120 (I.R.S. Employer Identification Number) 3475 Edison Way, S ...

Core Insights - Sanofi (SNY) has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) for efdoralprin alfa, an investigational biologic drug aimed at treating alpha-1 antitrypsin deficiency (AATD) related emphysema in adults [1][8] Drug Development and Mechanism - Efdoralprin alfa is a recombinant human AAT protein designed to inhibit neutrophil elastases, which cause lung tissue damage in AATD patients [3] - The drug is currently in mid-stage development and was integrated into Sanofi's rare disease pipeline following the acquisition of Inhibrx in 2024 [4] Clinical Study Results - The ODD was granted based on positive results from the global phase II ElevAATe study, which showed that efdoralprin alfa administered every three weeks (Q3W) or four weeks (Q4W) significantly increased functional AAT levels compared to weekly plasma-derived therapy [7][8] - Treatment with efdoralprin alfa also resulted in a higher percentage of days with functional AAT levels above the normal range, meeting key secondary endpoints of the study [9] Market Performance - Over the past year, Sanofi's shares have increased by 1.3%, while the industry has seen a rise of 13.8% [6]

As filed with the Securities and Exchange Commission on December 16, 2025 Registration No. 333-291852 UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM S-1 Amendment No. 1 REGISTRATION STATEMENT UNDER THE SECURITIES ACT OF 1933 Edison Oncology Holding Corp. (Exact name of registrant as specified in its charter) (State or other jurisdiction of incorporation or organization) Nevada 2836 83-1614120 (Primary Standard Industrial Classification Code Number) 3475 Edison Way, Suite R Menl ...