biOasis Provides Scientific Updates on Its Transcend Programs - Seite 2

The biOasis Lysosomal Storage Disease (LSD) Program

In late 2014, biOasis initiated a study using an animal model of Lysosomal Storage Disease with Dr. Maurizio Scarpa and the Brains for Brain Foundation in Padova, Italy. The purpose of the study is to investigate the ability of biOasis' Transcend BBB transport technology to deliver to the central nervous system the therapeutic enzyme required to correct the neurological disorder Mucopolysaccharidosis type II or Hunter syndrome. The study aims to determine whether a therapeutic quantity of idursulphase (I2S) can be delivered to the brain using biOasis's Transcend fusion proteins, MTf-I2S and the MTfp-I2S, and whether the fusion proteins can restore normal function after intravenous administration.

The company is pleased to report that the portion of the study performed in live animals is now complete and that the histopathology analyses are currently underway. Early results indicate that the animals given the fusion protein MTfp-I2S had levels of glycosaminoglycan similar to those seen in the liver of wild-type (normal) mice. Glycosaminoglycan levels are elevated in liver cell lysosomes of MPS II diseased mice.

The biOasis siRNA Program

On March 16, 2015, biOasis announced the results of an ischemic stroke model performed using the Transcend peptide carrier, MTfp coupled to a siRNA. In this study, the company demonstrated that the MTfp-siRNA therapeutic was successfully transported across the BBB, reduced the volume of the brain infarct, and improved overall brain function as determined by neurological scoring.

The ischemic stroke model was undertaken as a "proof-of-concept" study due to the clear data readout this model provides. The goal of this study was to provide a therapeutic efficacy study to support licensing of biOasis' vector MTfp to interested gene therapy companies for the treatment of CNS diseases such as Parkinson's disease and amyotrophic lateral sclerosis (ALS).

The biOasis-Medimmune Neuropathic Pain Program

Studies conducted by MedImmune, LLC. have produced data that illustrate certain desirable characteristics of the biOasis peptide carrier, MTfp. To validate the peptide (MTfp) licensing model, MedImmune was provided with the amino acid sequence of MTfp. Using the MTfp sequence, MedImmune designed and manufactured a fusion protein comprised of MTfp coupled to an antibody that targets a pain receptor in the brain. In an animal model, the fusion successfully crossed the BBB and also showed greater pain reduction than the currently used therapeutic.