Shire and NanoMedSyn Enter Into Research Agreement for Potential Lysosomal Storage Disorder Treatment

Shire plc and NanoMedSyn announced that the companies have entered into a preclinical research collaboration to evaluate a potential ERT using NanoMedSyn’s proprietary synthetic derivatives named AMFA.

“NanoMedSyn has demonstrated innovation in advancing the next generation of enzyme replacement therapy, and Shire is pleased to enter this research agreement with NanoMedSyn” said Andreas Busch, Ph.D., Head of Research and Development and Chief Scientific Officer at Shire. “The novel design of AMFA and the promising biological activity demonstrated in preclinical models makes this program an exciting opportunity for Shire to further expand its commitment to evaluating potential advancements in lysosomal storage disorder treatments.”

“This agreement provides the opportunity to further evaluate molecules based on our proprietary AMFA technology, which may potentially benefit patients with lysosomal storage disorders that are currently treated with the traditional enzyme replacement therapies,” said Henry-Vincent Charbonné, Chief Executive Officer and Chairman of NanoMedSyn. “As a global biotech leader in the development and commercialization of biologic therapeutics, Shire is an ideal research partner, particularly given their extensive expertise in the area of lysosomal storage disorders.”

Lysosomal storage disorders are inherited metabolic disorders that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies.1 There are more than 50 of these disorders altogether, and they may affect different parts of the body, including the central nervous system.

The AMFA compound is designed for the targeting of a specific membrane lectin, the mannose 6-phosphate (M6P) receptor, a major intracellular lysosomal trafficking pathway. Preclinical data demonstrate that AMFA has a high affinity for binding to the M6P receptor. Additionally, in preclinical models the AMFA compound leads to increased lysosomal exposure and enhanced activity of enzyme replacement therapy compared to a current available ERT.

Under the terms of the agreement, the parties will perform preclinical evaluations of AMFA conjugated to recombinant enzyme. Shire will provide funding to NanoMedsyn under the agreement. Further terms of the agreement were not disclosed.