Key Takeaways EMA granted ODD to efdoralprin alfa after the phase II ElevAATe study met endpoints.The study showed Q3W and Q4W dosing significantly improved outcomes versus weekly plasma-derived therapy.SNY added efdoralprin alfa via the Inhibrx acquisition and the drug already holds FDA Fast Track and ODD.Sanofi (SNY) announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (“ODD”) to efdoralprin alfa (SAR447537, formerly known as INBRX-101), an investigational biologic drug f ...

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 ...

Core Insights - Can-Fite BioPharma Ltd. announced a patient treated with Namodenoson has achieved an overall survival of 9 years with a complete response to treatment [1][2] Company Overview - Can-Fite BioPharma Ltd. is a biotechnology company focused on developing proprietary small molecule drugs for oncological and inflammatory diseases [1][7] - The company is advancing its lead drug candidate, Namodenoson, which is currently in a pivotal Phase III clinical study for advanced hepatocellular carcinoma (HCC) [3][7] Clinical Development - Namodenoson has shown promising results in a completed Phase II study, with a patient remaining cancer-free for 9 years [2][4] - The drug is being evaluated in multiple clinical trials, including a Phase IIb trial for Metabolic Dysfunction-associated Steatohepatitis (MASH) and a Phase IIa study in pancreatic cancer [6][7] - The U.S. FDA and European Medicines Agency have granted Namodenoson Orphan Drug status and Fast Track status for HCC treatment [3][7] Market Potential - The global market for HCC treatments is projected to reach $6.1 billion by 2027 for the G8 countries, driven by the need for effective and safe treatment options [5] - Liver cancer accounts for over 700,000 deaths globally each year, highlighting the urgent need for new therapies [5] Drug Mechanism - Namodenoson is a small orally bioavailable drug that selectively binds to the A3 adenosine receptor (A3AR), which is highly expressed in diseased cells, contributing to its excellent safety profile [6]

Core Insights - Moleculin Biotech, Inc. is progressing with its pivotal Phase 2B/3 "MIRACLE" study of Annamycin for treating adult patients with relapsed or refractory acute myeloid leukemia (AML), with 60% of the target enrollment completed as of November 4, 2025 [1][2][4] - The company expects to complete treatment for the first 45 subjects by Q1 2026, with initial unblinded data anticipated shortly thereafter [1][4][5] - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for AML treatment, and it is designed to avoid common cardiotoxicity associated with existing anthracyclines [7][9] Enrollment and Study Progress - The MIRACLE study is a global, multi-center, randomized, double-blind, placebo-controlled trial, combining data from its Phase 2B and Phase 3 portions to measure primary efficacy endpoints [3][10] - The first unblinding will involve 30 subjects treated with Annamycin in combination with HiDAC and 15 subjects treated with HiDAC plus placebo [3] - Recruitment is ongoing, with subjects enrolled from five countries, although some European sites are experiencing bed shortages affecting enrollment [5][2] Future Expectations - The company anticipates a second unblinding in the first half of 2026, following the initial unblinding of data [4] - The accelerated timeline for recruitment is attributed to positive feedback from potential investigators [4] - Annamycin is positioned as a potentially safer and more effective treatment option for AML, addressing a significant gap in current therapies [2][4]