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Trial Testing Rare Disease Drug for Covid-19 Pneumonia

Human lungs illustration

(NIH.gov)

11 Aug. 2020. A clinical trial is underway assessing a drug approved for a rare inherited disease as a treatment for people hospitalized with pneumonia from Covid-19 infections. The study is testing the drug Ruconest, made by Pharming Group N.V. in Leiden, the Netherlands, enrolling the first Covid-19 patient at University Hospital Basel in Switzerland.

Pharming Group is a biotechnology company designing biologic drugs produced in genetically-engineered animals from proteins expressed only in the milk of the animals. The company’s lead product is Ruconest, approved in Europe, the U.S., Israel, and South Korea to treat hereditary angioedema, a rare but life-threatening inherited condition. Hereditary angioedema occurs in about 1 in 30,000 individuals and causes swelling of hands, feet, face, and airways, as well as severe pain and vomiting.

Ruconest is an engineered C1 esterase inhibitor protein that in most people regulates several inflammatory pathways, but when mutated can trigger excessive swelling characteristic of hereditary angioedema. C1 esterase inhibitor is also part of the complement system, a series of proteins in blood plasma that help the immune system fight infections.

In April, doctors at University Hospital Basel gave Ruconest to five patients with severe pneumonia complications from Covid-19 infections, who did not respond to other treatments including hydroxychloroquine and the AIDS drugs lopinavir/ritonavir. Pharming Group says within 48 hours, fever and inflammation biomarkers in four of the five patients decreased, who were discharged from the hospital soon thereafter. The fifth patient required supplemental oxygen and intensive care, but has since been released. All five patients are now fully recovered.

The clinical trial plans to enroll up to 150 patients in a randomized, controlled study testing Ruconest as a treatment for patients with pneumonia resulting from Covid-19 infections. Other sites are expected to join University Hospital Basel. The study is not yet listed at U.S. or European clinical trial registries.

Michael Osthoff, the study team leader at University Hospital Basel, says in a Pharming Group statement, “After the encouraging results observed in five patients treated with Ruconest in our clinic, it is justified to investigate this drug and its unique mode of action of targeting several inflammatory cascades in a clinical trial with a large number of patients. We will gather precious information about efficacy, safety and appropriate dosing of the drug in the treatment and prevention of the severe complications of Covid-19.”

Pharming Group says if the trial is successful, the company plans to test Ruconest as a treatment for other hyper-inflammatory outcomes of Covid-19 infections in some patients, including blood clots throughout the body and cytokine storms that lead to organ failure and death.

“We have learned that cytokine storms caused by complement system activation cannot be controlled by targeted anti-inflammatory therapies,” notes Bruno Giannetti, Pharming Group’s chief medical officer. “Instead, broad anti-inflammatory agents are required to stop the activation of multiple inflammation pathways. Ruconest’s multiple interactions with key inflammation pathways therefore make it a promising candidate to prevent the severe complications observed in Covid-19 patients.”

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