A biotech company that markets drugs for rare blood disorders plans to bring its most recently approved product into a Phase III clinical trial for Covid-19.
Boston-based Alexion Pharmaceuticals said Monday that it would start a global Phase III study of Ultomiris (ravulizumab-cwvz) in adults with Covid-19 who are hospitalized with severe pneumonia or acute respiratory distress syndrome, also known as ARDS. The announcement comes nearly a month after the company said it was looking into another, older drug, Soliris (eculizumab), for the same indication.
Ultomiris is a C5 complement inhibitor approved for treating paroxysmal nocturnal hemoglobinuria. It belongs to the same class as Soliris, but is designed to be used less frequently.
“Alexion has been in close contact with physicians and global health authorities in an effort to rapidly evaluate the potential of C5 inhibition in treating patients with severe Covid-19,” said Alexion executive VP John Orloff, in a statement. “Based on early anecdotal information available from compassionate use cases in multiple countries, we are launching a controlled clinical trial to evaluate the potential of Ultomiris in mitigating the severe pneumonia and lung injury caused by the virus.”
The company said the announcement follows the Food and Drug Administration’s rapid review and acceptance of its investigational new drug application for Ultomiris in severe Covid-19. The trial is planned to have 270 patients, though it is not yet listed on ClinicalTrials.gov.
Much of the news media’s attention has focused on potential Covid-19 antivirals like Gilead Sciences’ remdesivir and also the antimalarial drugs hydroxychloroquine and chloroquine. But a significant amount of the drug development in Covid-19 consists of existing medicines being repurposed as treatments for the inflammation from hyperactive immunity that leads to much of the morbidity and mortality associated with the disease. These include monoclonal antibodies for autoimmune diseases that target IL-6, as well as small-molecule drugs like JAK and BTK inhibitors. On Friday, Incyte said it initiated a Phase III study of its drug, the JAK inhibitor Jakafi (ruxolitinib), in cytokine storm associated with Covid-19.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Ultomiris and Soliris work by targeting the complement system, which itself is part of the immune system and enhances its ability to clear the body of pathogens and damaged cells, but has also long been known to have a role in pneumonia and ARDS. The drugs work by preventing the complement C5 protein from being cleaved into two parts and preventing formation of a part of the complement system known as the membrane attack complex.
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