Alexion Pharmaceuticals is expanding the therapeutic potential of its rare disease drug Ultomiris (ravulizumab) into the COVID-19 arena, launching a phase 3 clinical trial for patients with severe complications of the disease. The initiative comes as pharmaceutical companies worldwide mobilize their resources to combat the SARS-CoV-2 coronavirus pandemic.
The Massachusetts-based biotechnology company received rapid FDA clearance to investigate Ultomiris in patients hospitalized with severe pneumonia or acute respiratory distress syndrome (ARDS) due to COVID-19. The trial, set to begin in May, will enroll approximately 270 patients across regions with high infection rates.
Scientific Rationale and Mechanism of Action
The scientific basis for testing Ultomiris in COVID-19 stems from its mechanism as a complement inhibitor. Preclinical evidence suggests that blocking terminal complement activation could reduce inflammatory signaling associated with severe COVID-19 cases. The complement system, typically involved in immune response, is believed to contribute to the severe pneumonia observed in critical COVID-19 patients.
Trial Design and Objectives
The phase 3 study will evaluate several critical outcomes comparing Ultomiris to best supportive care:
- Overall survival rate
- Duration of mechanical ventilation
- Length of hospital stay
Building on Existing Evidence
The decision to proceed with a late-stage trial is supported by encouraging clinical data from compassionate use of Soliris (eculizumab), Alexion's first-generation complement inhibitor. These preliminary results suggest complement inhibition may help mitigate COVID-19-related lung injury.
Strategic Advantages of Ultomiris
Ultomiris offers several advantages over its predecessor Soliris for COVID-19 treatment:
- Extended duration of action requiring less frequent dosing
- Weight-based dosing schedule
- Enhanced manufacturing capacity potential
- Improved hospital administration efficiency
Current Applications and Cost Considerations
Ultomiris is currently approved for treating rare conditions including Atypical Haemolytic Uremic Syndrome (aHUS) and Paroxysmal Nocturnal Haemoglobinuria. While Alexion has confirmed its ability to scale production if trials prove successful, the current cost of Ultomiris in rare disease treatment - approximately $458,000 annually for aHUS maintenance therapy - raises questions about pricing strategy for potential COVID-19 applications.
Parallel Research Initiatives
Multiple independent studies investigating Alexion's complement inhibitors in COVID-19 patients are already underway or planned. These parallel research efforts will provide additional data on the potential role of complement inhibition in treating severe COVID-19 complications.
