PharmaEssentia's novel pegylation technology platform has yielded our lead product, Ropeginterferon alfa-2b, which is currently being explored in several indications. Ropeginterferon alfa-2b is a novel engineered interferon alpha 2b, designed to be the most purer interferon alpha designed. In the field of protein drug design, the process of pegylation of the therapeutic protein preserves its biological activity by targeting the PEG polymer (polyethylene glycol) at a specific and defined region on the protein. Our pegylation technology platform is designed to increase the protein drug's efficacy by prolonging its circulation in the bloodstream. Utilizing its innovative 40K PEG and its novel pegylation technology platform by combining protein engineering and PEG-related chemistry, we creates novel products for better disease treatment. Compared with the similar drugs on the market, including PEG-Intron (Merck) and Pegasys (Roche), Ropeginterferon alfa-2b shows not only the most effective PK/PD data but also reduces severe side effects.
Our Ropeginterferon alfa-2b offers the following advantages:
Polycythemia vera (PV) is a rare, chronic disorder characterized by overproduction (proliferation) of red blood cells in the bone marrow. PV is typically associated with an elevated white blood cell count, an elevated platelet count, and an enlarged spleen (splenomegaly). The cause of PV is not completely understood, but it may be caused by genetic mutation. Nearly all PV patients have a mutation called “JAK2 V617F” (found in the JAk2 gene) in blood-forming cells. While Essential thrombocythemia (ET) is a chronic MPN previously called hemorrhagic thrombocythemia, is characterized by a sustained clonal proliferation of megakaryocytes in the bone marrow. The proliferation of megakaryocytes is primarily caused by clonal stem cells, as confirmed by enzyme and genetic analysis.
There is currently no cure for PV, currently available treatments includes phlebotomy, antiplatelet therapy, managing risk factors and cytoreductive therapies such as hydroxyurea (HU) and PEG-INF α-2a; meanwhile Hydroxyurea (HU) is generally considered the first-line therapy for ET. HU is very effective to the diseases, however many patients do not tolerate HU or suffer from anemia, leukopenia, skin lesions, or gastrointestinal symptoms, as the result interferon is another option for treatments.
Unfortunately the main problem with the current pegylated interferons (PegIntron and Pegasys) are patient tolerability, as these drugs induce severe flu-like symptoms and neuropsychiatric side effects. To solve the problems we develop Ropeginterferon alfa-2b which presents a much purer pegylated interferon (>90% 1 predominant isomer vs. PegIntron’s 14 isomers and Pegasys’ 8 isomers). As a result of our purer profile, we have seen data that indicates improved tolerability, compliance and thus long-term treatment outcomes. We are able to administer our product once every two weeks (vs. every week for other pegylated interferons) for enhanced tolerability.
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