ALEXION PHARMACEUTICALS, INC.

Vor Bio acquired exclusive global rights (excluding Greater China) to develop and commercialize telitacicept, a dual-target fusion protein approved in China for multiple autoimmune diseases including generalized myasthenia gravis.

The UK High Court has ruled that biosimilar versions of Soliris (eculizumab) developed by Amgen and Samsung Bioepis do not infringe on Alexion's patent for paroxysmal nocturnal hemoglobinuria treatment.

Multiple CDMOs secured significant contract manufacturing opportunities as regulatory bodies approved new indications for established drugs, particularly in oncology and rare diseases.

• Alexion Pharmaceuticals is initiating a Phase 3 clinical trial of Ultomiris (ravulizumab) in 270 patients with severe COVID-19 pneumonia or acute respiratory distress syndrome. • The study will evaluate Ultomiris's impact on patient survival, ventilation duration, and hospital stay length, targeting the complement system's role in severe COVID-19 complications. • The decision follows promising compassionate use results with Soliris and leverages Ultomiris's longer-lasting formulation and weight-based dosing schedule for optimal hospital administration.

Teva and Samsung Bioepis have launched Epysqli (eculizumab-aagh), a biosimilar to Alexion's Soliris, offered at a 30% discount off the reference product's wholesale acquisition cost.

• Regeneron's Veopoz, combined with an siRNA drug from Alnylam, demonstrated superior disease control compared to Ultomiris in paroxysmal nocturnal hemoglobinuria (PNH). • In follicular lymphoma, Regeneron's bispecific antibody Ordspono achieved a 100% complete response rate in previously untreated patients in a Phase 3 study. • Regeneron is also developing other drugs for PNH, with the potential to disrupt the market with improved efficacy and convenient administration. • Ordspono's efficacy in late-line follicular lymphoma showed an 80% objective response rate, with 73% achieving complete remission, suggesting best-in-class potential.

Several therapies are under evaluation in clinical trials for geographic atrophy (GA) secondary to age-related macular degeneration (AMD), addressing the unmet need for effective treatments.

• The FDA has approved Epysqli (eculizumab-aagh) as a biosimilar to Soliris for treating paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). • Epysqli's approval was based on analytical, non-clinical, and clinical data demonstrating its similarity to Soliris in safety, purity, and potency. • Clinical trials, including a Phase 3 study in PNH patients, showed clinical equivalence between Epysqli and Soliris in efficacy, safety, pharmacokinetics, and immunogenicity. • Epysqli aims to reduce healthcare costs and improve access to biologic medicines for rare diseases, already approved in Europe and Korea.