biOasis begins MPS II trial with Lysosomal storage disease expert Dr. Maurizio Scarpa and The Brains for Brain Foundation

The study will be conducted by way of a biOasis grant to the Brains for Brain Foundation ("B4B") of Padova, Italy, headed by renowned Lysosomal Storage Disease expert, Dr. Maurizio Scarpa, Founder and President of the Foundation. The study will be performed at the "Diagnosis and Therapy of Lysosomal Disorders" research facility of the University of Padova, and will be coordinated by Dr. Rosella Tomanin.

The human enzyme to be administered in the study, Idursulfase (I2S), is missing or functionally deficient in human sufferers of Hunter Syndrome. The study will use a knockout animal model that has shown the same characteristics as in the human form of MPS II.

The study will demonstrate whether a therapeutic quantity of I2S can be delivered in the animal’s brain using biOasis’ fusion proteins, MTf-I2S and the Transcend peptide-based MTfp-I2S, and whether the proteins can restore normal I2S function after intravenous administration.

Through the mechanism of Receptor Mediated Transcytosis, the biOasis fusion proteins, MTfp-I2S and MTf-I2S, are designed to cross the BBB and to allow the I2S to localize in the lysosomes of brain cells. The study will allow biOasis and B4B to not only study the effects of the fusion MTfp and MTf proteins on the restoration of enzymatic activity, but also the effect of the proteins’ administration in brain parenchyma cells and in cells from various other tissues.

Preliminary experiments using this animal model of MPS II have suggested that Enzyme Replacement Therapy with recombinant I2S may be effective in reducing the build-up of GAG stores that result from the I2S enzyme deficiency.

The results of the study will be used to support the design of clinical studies to evaluate the efficacy of MTfp-I2S and MTf-I2S in MPS II patients.

"The delivery of enzymes to the brain in a safe and efficient manner would offer clinicians a way to treat patients suffering from this dreadful disease," said Dr. Scarpa, President of B4B. "A number of invasive techniques have been deployed to deliver these enzymes into the brain and these techniques have demonstrated in humans that when the enzymes get there, they can be effective. The biOasis technology that we are going to study may offer a non-invasive, broad solution to the disorders."

The Brains for Brain Foundation was founded and also funded in 2007 by Dr. Maurizio Scarpa and the Foundation’s Vice-President, Dr. David Begley. B4B’s stated mission is the following: "B4B aims to develop new and innovative therapeutic strategies to cross the Blood-Brain Barrier, a capillary system which shields and defends the CNS from circulating neurotoxin compounds. It has a very important filtering protective function but unfortunately also prevents access to the brain by most candidate therapeutic drugs under development for CNS diseases.

B4B mission consists of the promotion of an International Network of health professionals in rare disorders, specifically in the field of rare neurological paediatric diseases. The purposes of the Foundation are to support the following activities in the field of rare Neurological Disorders: scientific research, knowledge dissemination, social and socio-medical assistance and health assistance."

Rob Hutchison, biOasis’ CEO, commented, "biOasis chose to work with Dr. Scarpa and the B4B Foundation he founded for a number of reasons. Dr. Scarpa has dedicated his career to helping children fight these dreadful diseases. He and Dr. Begley of the Foundation are world leading experts in the field. Dr. Scarpa, together with Dr. Tomanin, also guides a research facility at the University of Padova and they have several years of expertise with this animal model. We expect to have this study completed later this year with evaluation of data to follow."