Scienza e Tecnologia
Pharming announces enrolment of first patient in multinational clinical trial for the treatment of COVID-19 with RUCONEST®
Pharming Group N.V. ("Pharming" or "the Company") (Euronext Amsterdam: PHARM) today announced that the first patient has been enrolled in a randomized, controlled, investigator-initiated clinical trial in up to 150 patients for the treatment with RUCONEST® (recombinant human C1 inhibitor) of patients with confirmed COVID-19 (SARS-CoV-2) infections hospitalised with related severe pneumonia at the University Hospital Basel in Basel, Switzerland .
In April 2020 , Pharming reported encouraging results from a compassionate use programme at the University Hospital Basel, Switzerland , in which four male patients and one female patient (between 53-82 years of age) with COVID-19, suffering from related severe pneumonia, who did not improve despite standard treatment, including hydroxychloroquine and lopinavir/ritonavir, had been administered RUCONEST®. Following treatment, fever resolved in four of the five patients within 48 hours, and laboratory markers of inflammation decreased significantly (CRP, IL-6). Soon thereafter, four of the five patients were discharged from the hospital as fully recovered. One patient had increased oxygen requirement and was eventually transferred to the ICU for intubation but has also since made a full recovery.
Following these encouraging results, the Company, in partnership with treating physician Dr Michael Osthoff from the University Hospital of Basel, Switzerland , saw potential for a larger investigator-initiated, multinational, multicentre study to investigate the full extent of the role of RUCONEST® in the treatment of severe pneumonia related to a COVID-19 infection. If successful, the clinical trial could also lead to additional studies in patients suffering from other diseases with severe respiratory or other organ failure complications driven by activation of the complement system as part of a systemic hyperinflammatory syndrome, also known as a 'cytokine storm'.
RUCONEST® is a recombinant C1 esterase inhibitor (C1INH) approved for the treatment of hereditary angioedema (HAE) in the EU and 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 characterised by acute and painful swellings of soft tissues. Administration of C1 inhibitor can normalise the low C1 INH 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 and 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.
This clinical study in hospitalised patients with COVID-19 seeks to identify if the administration of additional C1 INH can control or stop the systemic hyperinflammation syndrome or cytokine storm. Once results from either an interim analysis or after all patients have been treated, headline data will be made publicly available.
Pharming is a specialty pharmaceutical company developing innovative products for the safe, effective treatment of rare diseases and unmet medical needs. Pharming's lead product, RUCONEST® (conestat alfa) is a recombinant human C1 esterase inhibitor approved for the treatment of acute attacks of angio-edema in patients with hereditary angioedema (HAE) in Europe ; for adults, adolescents and children from two years of age, in the US, for adults and adolescents and in Israel and South Korea . The product is available on a named-patient basis in other territories where it has not yet obtained marketing authorisation.
RUCONEST® is commercialised by Pharming in the US and in Europe , and the Company holds all other commercialisation rights in other countries not specified below. In some of these other countries distribution is made in association with the HAEi Global Access Program (GAP). RUCONEST® is distributed in Argentina , Colombia , Costa Rica , the Dominican Republic , Panama , and Venezuela by Cytobioteck, in South Korea by HyupJin Corporation and in Israel by Kamada.
RUCONEST® is also being evaluated for various additional indications. Pharming's technology platform includes a unique production process that has proven capable of producing industrial quantities of pure high quality recombinant human proteins in a more economical and less immunogenic way compared with current cell-line based methods.
Leads for enzyme replacement therapy ("ERT") for Pompe and Fabry's diseases are also being produced and optimised respectively at present.
Pharming has recently in-licensed leniolisib from Novartis, a small molecule and selective PI3Kδ inhibitor, which is in a registrational study for activated PI3K-delta syndrome (APDS), a rare form of Primary Immunodeficiency.
Pharming has a long-term partnership with the China State Institute of Pharmaceutical Industry ("CSIPI"), a Sinopharm company, for joint global development of new products, starting with recombinant human Factor VIII for the treatment of Haemophilia A. Preclinical development and manufacturing will take place to global standards at CSIPI and its affiliates and are funded by CSIPI. Clinical development will be shared between the partners with each partner taking the costs for their territories under the partnership.
Sijmen de Vries, CEO: T: +31 71 524 7400
Susanne Embleton , Investor Relations Manager: +31 71 524 7400/investor@pharming.com
Victoria Foster Mitchell / Mary Whittow ,
T: +44 203 727 1000
Leon Melens
T: +31 6 53 81 64 27
E: pharming@lifespring.nl