CEVEC Pharmaceuticals, the developer and licensor of the CAP® Technology, today announced a major milestone regarding its recombinant human CAP® cell derived C1 Esterase Inhibitor (C1Inh).
CEVEC´s CAP derived C1 Inhibitor material showed in a pivotal pharmacokinetic rat study a serum half life matching one of the two currently available, plasma derived treatments, Berinert®. All other known versions of recombinant C1 Inh, including e.g. Ruconest® from transgenic rabbits, displayed so far a significantly shorter half life than plasma derived C1 Inh. To achieve this excellent protein quality of the recombinant C1Inh, new glycosylation optimized CAP-GO cells were developed at CEVEC. This result, in combination with previously shown excellent specific activities of CAP derived C1 Inh and commercially attractive production yields using CAP-GO cells, paves now the way to develop a safer and more economic therapy for acute and prophylactic HAE indications.
Dr. Nicole Faust, CSO: “A human cell derived recombinant C1 Inhibitor is a long sought after product as an alternative to the currently available plasma derived therapies. Our promising results based on our fully scalable and cGMP compliant CAP suspension cells potentially open an entire new way to treat HAE."
Wolfgang Kintzel, CEO comments: “We are proud to offer interested parties a licensable human cell derived recombinant C1 Inhibitor with safety and production advantages after years of intense development. We’re convinced that this molecule has the potential to become the recombinant alternative of choice in the HAE therapy field. CEVEC has an increasing license customer portfolio for its CAP cell lines. Additionally, we offer a variety of biotherapeutics in pre-clinical stage. CEVEC´s customers can now also order pre-clinical, phase I and phase II material via our CMO business."
About Hereditary Angioedema (HAE)
A rare defect in the gene controlling the synthesis of the C1 Inhibitor leads to a life threatening disorder called Hereditary Angioedema (HAE), where patients suffer from episodes of edema (swelling) in various body parts including the hands, feet, face and airway. The leading current FDA approved treatments of the disease include intravenous injections of C1 Esterase Inhibitors that are purified from human serum. As with any blood or plasma derived product, these treatments are dependent on plasma supply and contain the risk of transmission of infectious agents like viruses or theoretically also the CJD agent.