Leiden, The Netherlands, 10 December 2020: Pharming Group N.V. (“Pharming” or “the Company”) (Euronext Amsterdam: PHARM) today announced that the first patient has been enrolled in a randomized, open label, parallel group, controlled, pilot clinical trial in up to 120 patients hospitalized with confirmed COVID-19 treated with RUCONEST® (recombinant human C1 inhibitor) for the prevention of severe SARS-CoV-2 infections at the Valley Hospital in Ridgewood, New Jersey in the United States.
Initially based at Valley Hospital in Ridgewood, New Jersey, this trial is planned to include patients at multiple centers in the US. This clinical trial follows an ongoing investigator-initiated, multinational, multicenter study, led by Dr. Michael Osthoff from the University Hospital Basel, into the use of RUCONEST® in the prevention of severe SARS-CoV-2 infections in patients hospitalized with related severe pneumonia. Pharming announced the enrolment of the first patient in that trial in August this year.
RUCONEST® and COVID-19
RUCONEST® is a recombinant C1 esterase inhibitor (C1INH) approved for the treatment of hereditary angioedema (HAE) in Europe and the US. C1INH is a protein that naturally occurs in the human body. It regulates several inflammatory pathways in the body by inhibiting certain proteins that are part of the human immune system. In diseases like HAE, deficiency of functional C1 inhibitor leads to excessive activation of the complement system and other immunological and haemostatic pathways, giving cause to angioedema attacks. In HAE, these attacks are characterized by acute and painful swellings of soft tissues. Administration of C1 inhibitor can normalize the low C1INH levels and stop angioedema attacks.
Systemic hyperinflammation is a hallmark of more severe stages of COVID-19 leading to acute respiratory distress syndrome, mechanical ventilation and ultimately death. Treatment with RUCONEST® may; 1) dampen uncontrolled complement activation and collateral lung damage; 2) reduce capillary leakage and subsequent pulmonary edema by direct inhibition of the kallikrein-kinin system; and 3) reduce the generation of microthrombi by inhibiting MASP-1 induced clot formation and factor XII amplified thrombo-inflammation.
C1 inhibitor is an acute phase reactant, meaning that the body naturally increases production during inflammatory conditions, such as infections. Despite this, a relative deficiency may occur and complement activation continues unchecked, often leading to a cytokine storm, a dangerous biochemical process that worsens the complications of COVID-19 infection, such as organ failure and death.
These clinical studies in hospitalized patients with confirmed COVID-19 seek to identify if the administration of additional C1INH can control or stop the systemic hyperinflammation syndrome or cytokine storm. Headline data will be made publicly available following either an interim analysis or after all patients have been treated.