Alexion trials rare disease drug Ultomiris in severe COVID-19 cases
Pharma is pretty much throwing everything it has at COVID-19 and US rare disease firm Alexion has joined the fray, testing its Ultomiris in a subset of severely ill patients.
A biologic medicine, Ultomiris (ravulizumab) works by inhibiting the complement system, which is thought to play a role in the severe pneumonia that can occur in patients with serious SARS-CoV-2 coronavirus infections.
Massachusetts-based Alexion said it will begin a phase 3 study investigating UItomirus in COVID-19 patients hospitalised with severe pneumonia or acute respiratory distress syndrome (ARDS).
The study is expected to enrol about 270 patients across countries with high numbers of diagnosed cases, beginning in May.
It will test the impact of Ultomiris on survival, duration of mechanical ventilation, and hospital stay compared with best supportive care.
This follows a rapid review by the FDA of Alexion’s filing to begin clinical trials of Ultomiris for severe COVID-19.
Ultomiris is already approved for the rare diseases Atypical Haemolytic Uremic Syndrome (aHUS), and Paroxysmal Nocturnal Haemoglobinuria, which at first glance would seem a million miles away from a viral infection like COVID-19.
But Alexion said the decision to press ahead with a late stage trial in coronavirus patients is based on preclinical data suggesting that inhibiting terminal complement can lower levels of chemical signals that drive inflammation associated with severe cases of the diseases.
Ultomiris is a longer lasting version of Alexion’s Soliris (eculizumab) complement inhibitor, which has produced “promising” evidence in the clinic through a compassionate use programme suggesting that this class of drugs may relieve injury to the lungs caused by the coronavirus.
There are already ongoing or planned independent studies of Alexion’s complement inhibitors in patients with COVID-19.
Alexion said that outcomes reported to data support a controlled clinical programme with Ultomiris the preferred option of Soliris thanks to its less frequent, weight-based dosing schedule.
This means that it could be easier to administer in hospitals and could be manufactured at a higher capacity.
Alexion has said it can ramp up production if the drug works in trials but what it has not mentioned is its cost: Ultomiris is hugely expensive when used in rare diseases, costing several thousand dollars for a single vial and about $458,000 per year in aHUS when given as a maintenance therapy.
Gilead has already faced criticism for what was seen as an attempt to cash in on the COVID-19 crisis with its potential therapy remdesivir, and Alexion will likely have similar issues to consider with Ultomiris if trials are successful.
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