Pharming Group N.V. sees encouraging results from five patients with confirmed COVID-19 infections hospitalized with related severe pneumonia that were treated with Ruconest (recombinant human C1 inhibitor) under a compassionate use program at the University Hospital Basel, Switzerland.
Four male patients and one female patient (between 53-85 years of age) with COVID-19 and suffering from related severe pneumonia, who did not improve despite standard treatment, including hydroxychloroquine and lopinavir/ritonavir, were administered Ruconest at an initial dose of 8400 U, followed by 4200 U every 12 hours for three additional doses. No allergic reactions or drug-related adverse events were reported.
Following treatment with Ruconest, fever resolved in four of the five patients within 48 hours, and laboratory markers of inflammation decreased significantly (CRP, IL-6). Soon thereafter, the patients were discharged from the hospital as fully recovered. One patient had increased oxygen requirement and was temporarily transferred to the ICU for intubation, but over the subsequent days recovered and was released from the ICU.
Following these initial results, a multinational, randomized, controlled, investigator-initiated clinical trial with up to 150 patients with confirmed COVID-19 infections, requiring hospitalization due to significant COVID-19 related symptoms is planned. The study will be led by Dr. Michael Osthoff, University Hospital Basel, Switzerland.
Dr. Michael Osthoff, University Hospital Basel, Switzerland, and the treating physician, says:
“Although this is an uncontrolled, small treatment experience, the results demonstrate the potential effectiveness of using Ruconest as an anti-inflammatory approach to inhibit the complement and contact systems after SARS-CoV-2 infection. We are now in the midst of planning a multinational, randomized controlled trial in up to 150 patients to further understand the safety and efficacy of this approach in preventing deterioration in COVID-19 patients.”
Prof. Bruno Giannetti, Pharming’s Chief Medical Officer, comments: “Some of the dangerous biochemical processes occurring during the worsening of a COVID-19 infection towards life-threatening pneumonia are likely triggered by complement activation as part of a systemic hyperinflammatory syndrome, otherwise known as a ‘cytokine storm’. C1 inhibitor has numerous anti-1 inflammatory properties, including inhibition of the complement and contact systems. A compassionate treatment in a few patients suffering from COVID-19 pneumonia was, therefore, scientifically sound and these preliminary results are very encouraging. Amongst others, we need to better identify the best time point to start Ruconest treatment and the optimal dosing regimen. The planned multinational study under the leadership of the University of Basel is aimed at providing this information.”