Shire, NanoMedSyn Team Up to Research Potential Therapy for Pompe Disease

The biotech companies Shire and NanoMedSyn have launched a new research partnership to evaluate a potential treatment for lysosomal storage disorders including Pompe disease.

NanoMedSyn has developed an enzyme replacement therapy using proprietary technology called AMFA that was shown in preclinical models to have promising biological activity. The companies will now explore its potential in the treatment of these types of disorders.

“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,” Henry-Vincent Charbonné, chief executive officer and chairman of NanoMedSyn, said in a press release. “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 in which toxic materials accumulate in the body’s cells as a result of enzyme deficiencies. More than 50 of these types of disorders have been identified to date, all affecting different parts of the body.

NanoMedSyn is developing its AMFA technology to potentially target various proteins or treatments to tissues and cells that express mannose 6-phosphate (M6P) receptors. This would allow the treatments to enter cells and be taken up by lysosomes.

Lysosomes are sacs that exist inside our cells that are full of enzymes that digest molecules and waste products. In lysosomal storage disorders, the lysosomal function is defective, leading to the accumulation of unwanted substances. In Pompe disease, this substance is glycogen. Its accumulation is caused by the lack of an enzyme called acid alpha-glucosidase (GAA).

Preclinical studies have shown that AMFA has a high affinity for binding to the M6P receptor. In preclinical models, it has led to increased lysosomal uptake and enhanced activity of enzyme replacement therapies.

In September 2016, NanoMedSyn’s first compound using the AMFA technology received orphan drug designation from the European Medicines Agency for the potential treatment of Pompe disease.

NanoMedSyn’s first compound using AMFA technology is a version of the GAA enzyme designed to improve the uptake of GAA by muscle cells and to increase entry into the lysosomes, where it replaces the missing enzyme.

According to the new research agreement, Shire and NanoMedSyn will conduct preclinical evaluations of AMFA, with Shire funding the collaboration.