Lipoxen Release: Positive Phase I Clinical Trial Results for ErepoXen
Published: May 06, 2009
- Data supports once monthly dosing (current EPO patients require up to three doses per week at a cost of approximately $9k per patient per year in the UK)
- Significant red blood cell response which lasted between 14 and 20 days after dosing
- Sustained rise in haemoglobin levels for 28 days after dosing
- Drug well tolerated with no safety issues
- Phase II studies will commence in this quarter in India and a Phase I/II study in Canada in H2 09
- Product scheduled for market launch in Russia in 2011
- Discussions continuing with potential licensing partners
This large Phase I trial was a randomised double-blind placebo-controlled single dose study and comprised 64 healthy adult males, 48 of whom received ErepoXen® while 16 received a placebo. The subjects who received ErepoXen® were assigned to four dose cohorts: 0.5, 1.5, 3.0 and 4.5 micrograms/kg respectively. The trial was designed to assess the safety, pharmacokinetics and pharmacodynamics of ErepoXen®, the polysialylated EPO, formulated using Lipoxen’s proprietary PolyXen® technology. This technology is based on polysialic acid (PSA), (a polymer occurring naturally in humans) which extends a protein’s active life, is biodegradable, non-immunogenic and non-toxic, and which is also therefore expected to avoid the toxicity attributed to polyethylene glycol in PEGylated protein drug candidates.
The pharmacodynamic data showed that ErepoXen® exerts a dose-dependent increase in reticulocyte (immature red blood cell) count. This increase was particularly prominent in the subjects receiving the two higher doses of ErepoXen®, with maximum reticulocyte count reached seven days after dosing. These subject’s reticulocyte counts then took between 14 and 20 days to return to baseline values, suggesting that ErepoXen® could exert a long-acting effect on erythropoiesis (red blood cell production). This effect is comparable to that observed with CERA (Continuous Erythropoietin Receptor Activator), a Roche product, and is significantly longer than that observed with other shorter acting currently marketed EPOs. Subjects receiving ErepoXen® also saw an increase in haemoglobin levels when compared to baseline and these effects lasted up to 28 days after dosing.
Phase II studies with ErepoXen® are planned to commence in India this quarter and market launch is expected in Russia in 2011. A Phase I/II study in Canada will be conducted according to FDA guidelines and will also commence before the end of 2009. These clinical studies will be funded by SIIL, India’s largest biotech company.
Commenting on this announcement, M. Scott Maguire, CEO of Lipoxen, said: “We are very excited about this new clinical data which indicates that Lipoxen’s ErepoXen® candidate has the potential to provide an improved and more convenient dosing regime for patients plus significant economic benefits for healthcare providers compared to current EPO products. The revenues for current EPOs (injected once to three times per week) totalled $9 billion in 2007 and thus we look forward to moving this product, which is funded by our partner, the Serum Institute of India, forward and into Phase II clinical trials next month. We have already begun discussions with potential commercial partners around this candidate and expect to sign a significant licensing deal in due course. Today’s new data, together with the exciting flu vaccine data released last week, shows that Lipoxen’s technologies are addressing pressing healthcare needs.”
Adding to this announcement, the Phase I study’s Principal investigator, Dr. Mikhail Ashraf*, Consultant Nephrologist and Senior Medical Tutor, Swansea University, said: “I am pleased that the results we have announced today continue to confirm the excellent safety profile of polysialylated EPO. The overall data, particularly the mean reticulocyte and haemoglobin response, suggests that this novel EPO preparation would be suitable for once monthly administration.”
*Dr. Ashraf, an advisor to major multinational companies, has participated extensively in clinical trials of EPOs. He is an expert in anaemia management and has a special interest in erythropoiesis, having conducted phase-II and phase-III clinical trials for short and long acting ESAs.