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Pipeline
Expanding the pipeline beyond Ruconest®
With validation secured from the approval of the first product from our transgenic platform, we will now seek to initiate new projects on this platform.
Our transgenic platform remains the only technology that to date can deliver recombinant versions of certain complex human proteins in an economically viable way: this is a result of the low cost of capital investment required to start up a suitable founder herd, the fact that the herd is easily scalable and the high yield of product obtained.
Our goal is to focus on indications that have a high unmet medical need using biologics to help patients that otherwise have limited choice in treatment.
Expanding Beyond The Pipeline
rhC1INH (Ruconest)
Recombinant human C1 inhibitor (rhC1INH) is a recombinant human C1 inhibitor protein. Natural C1 inhibitor DNA from a human source is used in Pharming’s protein production technology to ensure expression of the C1 inhibitor protein. Human C1 inhibitor is a human plasma protein involved in the regulation of the complement and contact parts of the immune system. It is a controlling protein in early stages of these cascades and in that way a regulating factor of the immune system.
By inhibiting these systems, the abnormal inflammation can be controlled. This is also the mechanism by which an HAE attack can be treated with C1 inhibitor.
rhFVIII (Factor VIII)
Recombinant human Factor VIII is a natural human blood clotting factor and is in early-stage development for treatment of Haemophilia A. Haemophilia A is a hereditary disorder caused by defects in the Factor VIII gene. Lack of functional Factor VIII diminishes the body’s clotting ability, which in turn can lead to damaging- or fatal bleeding episodes. On this project, Pharming has a service agreement with Renova Life to provide Pharming with founder rabbits.
Haemophilia A is an X chromosome linked hereditary disorder caused by defects in the Factor VIII (FVIII) gene that lead to lower levels of the functional FVIII protein. Lack of functional FVIII diminishes the body’s clotting ability, which in turn can lead to damaging or fatal bleeding episodes.
rhC1INH (Ruconest)
Potential additional indications for Ruconest (rhC1INH) in development stage
Recombinant human C1 inhibitor (rhC1INH) is a recombinant human C1 inhibitor protein. Natural C1 inhibitor DNA from a human source is used in Pharming’s protein production technology to ensure expression of the C1 inhibitor protein. Human C1 inhibitor is a human plasma protein involved in the regulation of the complement and contact parts of the immune system. It is a controlling protein in early stages of these cascades and in that way a regulating factor of the immune system. By inhibiting these systems, the abnormal inflammation can be controlled. This is also the mechanism by which an HAE attack can be treated with C1 inhibitor.
Acute pancreatitis
Acute pancreatitis (AP) is an acute inflammatory disorder of the pancreas for which there are currently no approved medical therapies. With approximately 300,000 hospitalizations per year (an increase of more than 2-fold since 1988), AP represents the single most frequent gastrointestinal cause of hospital admissions in the US.
Based on the broad anti-inflammatory properties of C1INH, pdC1INH and rhC1INH have been studied in a variety of clinical conditions and animal models of numerous conditions involving contact and complement system activation with a vascular/capillary leak component. These studies have included models of pancreatitis, sepsis, thermal injury, xenotransplantation, and ischemia-reperfusion injury (e.g., myocardial infarction, stroke, delayed graft function in transplantation). Together, this experience suggests that rhC1INH may be able to interrupt the pro-inflammatory processes in patients with AP, and thereby resolve the ongoing SIRS to ultimately prevent organ failure.
rhFVIII (Factor VIII)
Factor VIII (rhFVIII)
Haemophilia A is an X chromosome linked hereditary disorder caused by defects in the Factor VIII (FVIII) gene that lead to lower levels of the functional FVIII protein. Lack of functional FVIII diminishes the body’s clotting ability, which in turn can lead to damaging or fatal bleeding episodes.
Recombinant human Factor VIII is a natural human blood clotting factor and is in early-stage development for treatment of Haemophilia A. Haemophilia A is a hereditary disorder caused by defects in the Factor VIII gene. Lack of functional Factor VIII diminishes the body’s clotting ability, which in turn can lead to damaging- or fatal bleeding episodes.
As the first step in the assessment of develop-ability, Pharming signed a service agreement with Renova Life (RLI). The agreement covers the development and supply of founder transgenic rabbits from RLI to Pharming. The founder rabbits will enable Pharming to start the commercial production breeding process.
Collaborative Product development: Strategic Collaborations with SIPI
In addition recently we have entered a strategic collaboration for the development, manufacture and commercialisation of new products based on the Pharming technology platform with The Shanghai Institute of Pharmaceutical Industry (SIPI), a Sinopharm Company based in China.
sipi logo
Under the terms of the agreement, Pharming will transfer the Pharming technology platform and manufacturing know- how to SIPI, such that joint global development for new products will take place at SIPI’s facilities in Shanghai and benefit from both the cost advantages of the Pharming platform and the competitive development and manufacturing costs structures at SIPI.
The first projects to be jointly developed and manufactured at SIPI will be recombinant C1-inhibitor (conestat alfa) and Factor VIII.
Products
Ruconest EU_Flag
Ruconest (conestat alfa) is a recombinant human C1 esterase inhibitor approved for the treatment of angioedema attacks in patients with HAE in the USA, Israel, all 28 EU countries plus Norway, Iceland and Liechtenstein.
Ruconest is distributed in the EU by Swedish Orphan Biovitrum. Ruconest is partnered with Salix Pharmaceuticals Inc. in North America.
Medical need
C1 esterase inhibitor (C1INH) is a protein that naturally occurs in the human body. It belongs to the class of serine-protease inhibitors or serpins. It regulates several inflammatory pathways in the body by inhibiting certain proteins (proteases) that are part of the human immune system. Deficiency of functional C1 inhibitor leads to excessive activation of the complement system and other immunological and haemostatic pathways, giving cause to angioedema attacks. These attacks are characterized by acute and painful swellings of soft tissues. Administration of C1 inhibitor protein can normalize the low C1 INH levels and stop the angioedema attacks.
Pharming’s therapy
Patients who suffer from hereditary angioedema (HAE) have a deficiency of functional C1INH resulting from a mutation in the C1 inhibitor gene. For them, Pharming’s Ruconest provides a causal treatment with an excellent efficacy and safety profile. The GMP conform proprietary production and purification technology allows for the commercial manufacturing of large quantities of high-grade product of outstanding quality. Ruconest does not carry the risk of transmission of plasma related human infectious agents, as it is not isolated from human blood sources.