PROVIDENCE, R.I., May 18, 2018 /PRNewswire/ -- The National Institutes of Health (NIH) has awarded EpiVax, Inc. ("EpiVax"), experts in the field of immunogenicity risk assessment, a $324,980 Small Business Innovation Research (SBIR) grant. The funding will enable EpiVax to further develop the new Personalized Immunogenicity Assessment (PIMA) tool, for the individualized risk assessment of enzyme replacement therapy for Pompe Disease patients. This innovative tool has the capacity to identify patients who are at risk of developing immune responses to this life-saving treatment, based on their own specific genotype. Annie De Groot, MD, CEO/CSO of EpiVax will direct the development and validation of the PIMA tool with her team at EpiVax, in collaboration with Priya Kishnani, MD; Zoheb Kazi, MBBS; and other colleagues at the Alice and YT Chen Pediatric Genetics and Genomics Research Center (Department of Pediatrics, Duke University).
Many, but not all Pompe Disease patients are prone to developing anti-therapeutic antibodies (ATA) to their enzyme replacement therapy, even if they have residual natural enzyme expressed in their bodies. ATA can reduce the efficacy of enzyme replacement therapy. While Immune Tolerance Induction (ITI) treatments can be used to ameliorate this problem, accurate identification of patients who may be at risk is not currently possible. EpiVax has developed the PIMA tool to assess individual risk based upon patient human leukocyte antigen (HLA) haplotype and endogenous enzyme mutations. Prospective validation of the tool will be carried out in collaboration with Duke University.
"A personalized prediction tool could potentially enable clinicians to more effectively manage enzyme replacement therapy for patients with Pompe disease, leading to improved quality of life and efficacy for this otherwise lethal disorder," said Kishnani, one of the world's experts on Pompe Disease. "Proof of principle may pave the way for this personalized risk assessment to be applied to related enzyme deficiencies," said De Groot, "such as Niemann-Pick disease, Gaucher disease, and Fabry disease."
About Pompe Disease
Pompe Disease is a metabolic disorder caused by the deficiency of the acid alpha-glucosidase (GAA) enzyme, which requires life-long treatment with enzyme replacement therapy. Enzyme replacement therapy can lead to the development of anti-therapeutic antibodies (ATA), which decrease the efficacy of treatment, resulting poor outcomes for many Pompe Disease patients.
EpiVax is a world leader in the fields of immunogenicity risk assessment and vaccine and biologics design. EpiVax's tools are used by a global roster of companies to design and optimize therapeutic proteins and vaccines. For more information visit www.epivax.com.
Research reported in this publication was supported by the National Center for Advancing Translational Sciences of the NIH under Award Number R43TR002441. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.