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 ...

Core Insights - FibroGen's roxadustat has received Orphan Drug Designation from the FDA for treating myelodysplastic syndromes (MDS), indicating a significant treatment gap in this area [2][3] - The company plans to submit the Phase 3 protocol for roxadustat in the fourth quarter of 2025 [1][2] Company Overview - FibroGen, Inc. is a biopharmaceutical company focused on developing novel therapies for cancer and anemia [7][8] - Roxadustat is already approved in Europe, Japan, and other countries for treating anemia in chronic kidney disease (CKD) patients [6][8] Treatment Landscape - Approximately 58,000 patients in the U.S. are diagnosed with lower-risk MDS (LR-MDS), with 85% suffering from anemia [2] - Current first-line treatments achieve transfusion independence in less than 50% of patients, highlighting the need for more effective options [2][4] - Roxadustat has shown benefits in transfusion independence compared to placebo in patients with high transfusion burden [2][4] Drug Mechanism - Roxadustat is an oral medication that promotes red blood cell production by increasing endogenous erythropoietin, improving iron absorption, and downregulating hepcidin [5] Market Potential - The FDA's Orphan Drug Designation provides benefits such as market exclusivity for seven years post-approval, which could enhance the commercial prospects for roxadustat [3]

Core Insights - GSK plc announced that the FDA has granted Orphan Drug Designation to its investigational drug GSK'227, now renamed risvutatug rezetecan, for treating small-cell lung cancer (SCLC) [1][7] - The Orphan Drug Designation was based on positive results from the phase I ARTEMIS-001 study, which demonstrated durable responses in patients with extensive-stage SCLC [2] Drug Development and Designations - A phase III study for risvutatug rezetecan in relapsed SCLC commenced in October 2025, with additional phase III studies evaluating the drug for osteosarcoma and early-stage studies for other cancers [3] - Risvutatug rezetecan has received multiple expedited designations, including Breakthrough Therapy Designation from the FDA and PRIME designation from the EMA for various cancer treatments [4] Market Context - Small-cell lung cancer accounts for approximately 13% of all lung cancers in the U.S. and has one of the poorest prognoses among major cancers, with most patients experiencing relapse after initial treatment [5] - GSK's shares have increased by 41.7% over the past year, outperforming the industry average rise of 11.6% [3]

As filed with the U.S. Securities and Exchange Commission on December 8, 2025. Registration No. 333-291598 UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 AMENDMENT NO. 1 TO FORM F-1 REGISTRATION STATEMENT UNDER THE SECURITIES ACT OF 1933 Biodexa Pharmaceuticals PLC (Exact name of registrant as specified in its charter) (State or Other Jurisdiction of Incorporation or Organization) England and Wales 2834 Not Applicable (Primary Standard Industrial Classification Code Number) (IRS Emp ...

Registration No. 333-291598 UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 AMENDMENT NO. 1 TO FORM F-1 As filed with the U.S. Securities and Exchange Commission on December 8, 2025. REGISTRATION STATEMENT UNDER THE SECURITIES ACT OF 1933 Biodexa Pharmaceuticals PLC (Exact name of registrant as specified in its charter) (State or Other Jurisdiction of Incorporation or Organization) England and Wales 2834 Not Applicable (Primary Standard Industrial Classification Code Number) (IRS Emp ...

Core Insights - MediciNova, Inc. has provided an update on its Phase 2b/3 clinical trial of MN-166 (ibudilast) for Amyotrophic Lateral Sclerosis (ALS), known as the COMBAT-ALS study, with results presented at the 36th International Symposium on ALS/MND [1][2] Company Overview - MediciNova, Inc. is a clinical-stage biopharmaceutical company focused on developing novel small molecule therapies for inflammatory, fibrotic, and neurodegenerative diseases, with a late-stage pipeline including MN-166 (ibudilast) and MN-001 (tipelukast) [4][6] Clinical Trial Update - The COMBAT-ALS trial has successfully randomized a total of 234 participants, completing enrollment in September 2025 [5] - The baseline characteristics of participants include a mean age of 60.6 years, with 36.8% female and 63.2% male, and a racial distribution predominantly Caucasian (90.2%) [5] - The mean ALSFRS-R score at screening was 40.6, indicating the severity of the disease among participants [5] Drug Development and Designation - MN-166 (ibudilast) has received Orphan Drug Designation and Fast Track Designation from the FDA, as well as Orphan Designation from the European Commission for ALS treatment [3][2] - The drug is also in late-stage development for other neurodegenerative diseases and conditions, including progressive multiple sclerosis and glioblastoma [3][4] Future Expectations - Top-line data from the COMBAT-ALS study is anticipated by the end of 2026, with the company expressing hope that MN-166 will provide a significant therapeutic advance for ALS patients [2]

As filed with the Securities and Exchange Commission on November 28, 2025 Registration No. [*] UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM S-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) (I.R.S. Employer Identification Number) 3475 Edison ...