The Phase I trial, which was carried out at the Veeda Clinical Research Pvt in India, was a randomised double-blind placebo-controlled single dose study. The trial was designed to assess the safety, tolerability and efficacy of EPO formulated using Lipoxen’s proprietary PolyXen® technology. This technology is based on polysialic acid (PSA), a naturally occurring polymer which extends a protein’s active life, is biodegradable, non-immunogenic and non-toxic, and which is also expected to avoid the toxicity attributed to polyethylene glycol in PEGylated protein drug candidates. The trial recruited 32 healthy adult males, 24 of whom received ErepoXen® while eight received placebo, via subcutaneous injection. The patients who received ErepoXen® were assigned to two groups one of which received 0.5 micrograms/kg (body weight) and the other received 1.5 microgram/kg.
The initial results from the Phase I study show that ErepoXen® is safe and well tolerated with no adverse events attributed to the product being experienced in any of the treated patients. The trial also provided initial data on the product candidate’s pharmacokinetics. In the patients receiving the higher dose of ErepoXen® there was a clear reticulocyte (immature red blood cells) response which lasted for two weeks. In addition, there was a sustained rise in haemoglobin levels which lasted for 28 days in these patients.
Commenting on the results, Dr. Mikhail Ashraf, Consultant Nephrologist and Senior Medical Tutor, Swansea University, said, "These results from early cohorts are very encouraging. The 28 day sustained haemoglobin rise and the lasting reticulocyte response indicate that this preparation could be suitable for once monthly administration. With most current erythropoietin therapies administered between once and three times a week, ErepoXen® has the potential to fulfill a large unmet medical need in anaemia management and I look forward to following its development."
M. Scott Maguire, CEO of Lipoxen, said:
“We are extremely pleased that the initial results from the first Phase I trial with ErepoXen® have been so positive. The study has shown the product candidate to be safe and well tolerated, and also demonstrates that this long-acting erythropoietin, which has been formulated using our proprietary PolyXen® technology, has the potential to be administered on a once a month basis. This could be a key competitive advantage for ErepoXen® as there is a clear demand from patients for improved forms of EPO, which have, in particular, less frequent dosing and more patient convenience. In 2007, the overall global market for EPO was worth $9 billion.
Today’s announcement is a major milestone for Lipoxen’s business as it is the first human data showing that we can use our PolyXen® technology to improve the delivery of biological drugs, a growing and highly profitable segment of the overall pharmaceutical market. We are currently conducting a Phase I study with our long-acting insulin product SuliXen and I am confident that this study will also produce a positive outcome. With two high-value differentiated biologicals under development and a portfolio of delivery technologies which is attracting interest from a growing list of potential partners, I believe that Lipoxen is well positioned to generate significant shareholder value over the remainder of 2008."
