- Research agreement focuses on evaluating a potential treatment for a lysosomal storage disorder using NanoMedSyn’s proprietary AMFA technology
- Research reinforces Shire’s genetic diseases franchise’s leadership in rare diseases innovation
Cambridge, MA and Montpellier, France – March 26, 2018 – Shire plc (LSE: SHP, NASDAQ: SHPG), the global biotechnology leader in rare diseases, and NanoMedSyn, a biotechnology company dedicated to innovation in enzyme replacement therapy (ERT), today 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.1
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.2,3,4 Preclinical data demonstrate that AMFA has a high affinity for binding to the M6P receptor.4 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.4
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.
About NanoMedSyn and AMFA
NanoMedSyn is a private biotech company based in Montpellier (France) which was created to develop the innovative AMFA technology. NanoMedSyn holds the exclusive worldwide rights on the technology. The technological platform of AMFA compounds have the potential to target various proteins or drugs to tissues and cells expressing the mannose 6-phosphate receptors in order to facilitate their cellular entrance and eventual lysosomal uptake. In September 2016, the European Medicines Agency (EMA) granted orphan drug designation for NanoMedSyn’s first compound, recombinant acid alpha-glucosidase conjugated with AMFA for the potential treatment of Pompe disease, a lysosomal storage disorder.
About Shire’s Genetic Disease Franchise
Shire is dedicated to helping patients with inherited illnesses. Shire’s genetic disease franchise has a strong legacy in developing therapies for LSDs, with a portfolio that includes commercial products, late-stage investigational therapies, and pipeline candidates, as well as a robust R&D program. Therapeutic areas in which Shire is working include Hunter Syndrome, Type 1 Gaucher Disease and Fabry Disease.
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NOTES TO EDITORS
Shire is the global leader in serving patients with rare diseases. We strive to develop best-in-class therapies across a core of rare disease areas including hematology, immunology, genetic diseases, neuroscience, and internal medicine with growing therapeutic areas in ophthalmics and oncology. Our diversified capabilities enable us to reach patients in more than 100 countries who are struggling to live their lives to the fullest.
We feel a strong sense of urgency to address unmet medical needs and work tirelessly to improve people’s lives with medicines that have a meaningful impact on patients and all who support them on their journey.
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a further list and description of risks, uncertainties and other matters can be found in Shire’s most recent Annual Report on Form 10-K and in Shire’s subsequent Quarterly Reports on Form 10-Q, in each case including those risks outlined in “ITEM1A: Risk Factors”, and in Shire’s subsequent reports on Form 8-K and other Securities and Exchange Commission filings, all of which are available on Shire’s website.
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1 Bellettato CM et al. Pediatr Clin North Am. 2018 Apr;65(2):353-373. doi: 10.1016/j.pcl.2017.11.011.
2 Coutinho MF, Prata MJ and Alves S. Mol Gen Metab. 2012 Apr;105(4):542-550. doi: 10.1016/j.ymgme.2011.12.012
3 Staudt C, Puissant E and Boonen M. Int J Mol Sci. 2016 Dec;18(1). pii: E47. doi: 10.3390/ijms18010047.
4 Cheikh et al. Angew Chem Int Ed Engl. 2016 Oct;55(47):14774-14777. doi: 10.1002/anie.201607824.