Candidate: Ultomiris® (ravulizumab-cwvz)
Type: Complement inhibitor indicated for adults with paroxysmal nocturnal hemoglobinuria (PNH), and adults and children one month of age and older with atypical hemolytic uremic syndrome (aHUS).
Status: In announcing third-quarter 2020 results on October 29, Alexion said a Phase III randomized controlled trial (NCT04369469) assessing Ultomiris in adults with COVID-19 who are hospitalized with severe pneumonia or acute respiratory distress syndrome was underway. The trial is designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of Ultomiris in adult patients with COVID-19 severe pneumonia, acute lung injury, or acute respiratory distress syndrome.
The trial is estimated to enroll 270 participants in the U.S. and four other countries (France, Japan, Spain, and the U.K.). Two-thirds of the participants will be randomized to Ultomiris, the other third to best supporting care Patients will be randomly assigned to receive ravulizumab in addition to best supportive care (BSC) alone.
Alexion announced plans for the Phase III trial on April 20, saying patients would be enrolled across countries with “high” numbers of diagnosed cases beginning in May. The company said the study would evaluate the impact of Ultomiris on survival, duration of mechanical ventilation, and hospital stay compared to best supportive care.
The company cited published preclinical data suggesting that inhibition of terminal complement can lower cytokine and chemokine levels and significantly reduce lung inflammation and pathology in animal models of viral pneumonia. Alexion also said elevated complement biomarkers and promising preliminary clinical evidence from patients who accessed Soliris (eculizumab) through the company’s compassionate use program suggested that complement inhibition may improve coronaviral-mediated lung injury.
COVID-19: 300 Candidates and Counting
To navigate through the >300 potential therapeutic and vaccine options for COVID-19, GEN has grouped the candidates into four broad categories based on their developmental and (where applicable) clinical progress:
● FRONT RUNNER – the most promising therapeutics/vaccines based on clinical progress, favorable data or both.
● DEFINITELY MAYBE – earlier phases with promising partners, or more advanced candidates in development that have generated uneven data.
● KEEPING AN EYE ON… – interesting technology, attracting notable partners, or both, but preliminary data.
● TOO SOON TO TELL – longshots pending additional experimental and/or clinical data.
GEN has also tagged the most common treatment types: