Primary Hyperoxaluria (PH) is a rare autosomal recessive inborn error of glyoxylate metabolism, with a prevalence of 1-3 per million. The disease is characterised by severe hyperoxaluria, i.e. excessively high levels of oxalate in the urine of the patient and excessively high oxalate levels in the plasma of patients suffering from chronic renal failure.
The disease is present at birth, and most patients develop kidney stones and nephrocalcinosis at very young ages, most often under the age of 10. Progression of renal failure is followed by systemic deposition of CaOx and premature death. Overall, the risk of end-stage renal disease (ESRD) is 50% by age 15 and 80% by age 30. The median age at death is around 30. (Hoppe et al., 2009).
There is currently no approved pharmaceutical therapy for this devastating disease. The only curative therapy, applicable for PH type I, is eventually a combined kidney and liver transplantation at end stage renal disease (Hoppe, 2009).
The ELIMOX Partnership
ELIMOX, Biopharmaceutical therapy for treatment of Primary Hyperoxaluria, is a project funded by FP7-SME-2013 Research for the benefit of SMEs program. The project is co-ordinated by OxThera AB, Stockholm, Sweden.
The ELIMOX Consortium has three SME-partners; Oxthera, Cobra Biologics and SymbioPharma, plus nine Research, Technology & Development (RTD) partners.
The project is truly international with funding from the European Union´s Seventh Framework Programme managed by REA-Research Executive Agency (FP7/2007-2013) under grant agreement no FP7-SME-2013.
The use of naturally occurring bacteria from the human gut as pharmaceutical drugs has gained increased interest, as knowledge about the human microbiota and its role in health and disease has advanced considerably with new technology and computational techniques.
The primary aim of the ELIMOX project is to develop an innovative and unique bacterial pharmaceutical product for the treatment of a severe and debilitating inherited disease, Primary Hyperoxaluria (PH).
Our approach is to use the metabolic potential of a naturally occurring microbe, Oxalobacter formigenes to eliminate toxic compounds from the blood. The potential clinical applications for such a probiotic drug are many, including treatment of disorders where enteric elimination would be an alternative metabolic pathway as well as for malabsorptive disorders. The SME’s in this project will be pioneers in this area of research and technological development.
To achieve this, the following will be implemented:
1. The manufacture of an anaerobic bacterial product for administration in the gut.
2. The development of analytical tools to verify the quality, delivery and activity of the bacterial product.
3. The clinical development of the bacterial product to verify clinical efficacy and safety.
The activities are divided into five Work Packages detailed in the pdf download.
Download the full case study here: