St George’s and Orphan Technologies to develop drugs for rare disorders
Based on a cell-based enzyme-replacement technology called Erythrocyte Encapsulated (EE)
St George’s, University of London, Tooting, UK has signed an exclusive worldwide licensing agreement with Swiss biopharmaceutical firm Orphan Technologies to develop new therapies for deadly metabolic disorders. No financial details have been disclosed.
The partners aim to develop therapies based on a cell-based enzyme-replacement technology called Erythrocyte Encapsulated (EE). Their primary goal is to accelerate the development of an investigational therapy for mitochondrial neurogastrointestinal encephalomyopathy (MNGIE), a progressive disease that kills patients at an average age of 38. If approved, the new therapy would be the first drug for a mitochondrial disease.
Globally, only 200 cases of MNGIE have been identified, but these are increasing and experts believe it has been underdiagnosed. The only current potential cure is a stem cell transplant technique, but this carries a high risk of death and most patients are ineligible as recruitment is restricted only to those with an optimal matched donor and without irreversible end-stage disease.
EE-TP has shown very promising clinical data results in compassionate use treatment for MNGIE patients
The investigational therapy for MNGIE is called Erythrocyte Encapsulated Thymidine Phosphorylase (EE-TP), which is based on introducing TP directly into patients' red blood cells (erythrocytes). EE-TP was reported to be effective in reducing or eliminating the elevated plasma and urine concentrations of thymidine and deoxyuridine, toxic substances that accumulate in tissues of MNGIE patients. Clinical improvements have also been reported, with a reduction in the number of nausea and vomiting attacks, a reduction in weight loss, and an improvement in distal sensation in hands and fingers.
Dr Bridget Bax, Head of the St George’s team and a leading authority on MNGIE, said: ‘MNGIE is a relentlessly progressive degenerative disease and for a majority of patients there are no treatment options other than supportive care. Our team is committed to addressing the unmet needs of these patients.
‘Under the licensing agreement with Orphan, we intend to combine our strengths to accelerate the regulatory development of EE-TP. Our ultimate hope is that EE-TP will benefit patients with MNGIE and that patients globally have equitable access.’
Josef Rosenberg, director of Orphan Technologies, said: ‘EE-TP has shown very promising clinical data results in compassionate use treatment for MNGIE patients. The St George's team, led by Dr Bridget Bax, brings unique experience with the EE-TP technology; we are proud to be working with them and together aim to bring hope to the MNGIE population.’
EE-TP has been granted orphan drug status in Europe and the US.
Under the agreement with St George’s, Orphan Technologies has also licensed rights for the development of a new enzyme-replacement therapy for severe combined immunodeficiency (SCID) due to adenosine deaminase deficiency (ADA-SCID), another rare, fatal metabolic disease. The novel therapy, Erythrocyte Encapsulated Adenosine Deaminase (EE-ADA), has been proved efficient and safe, as reported in the treatment of an ADA-SCID patient for more than 15 years.
