AFFiRiS, a clinical-stage biotechnology company developing novel disease-modifying specific immunotherapies, announces today the presentation of in vivo data with its antibody mAB C6-17 against the mutant huntingtin protein (mtHTT) at the 16th Annual Huntington’s Disease Therapeutics Conference. The Conference will be held virtually April 27 – 29, 2021 and will cover all aspects of drug discovery and development for Huntington’s disease (HD).
HD is a hereditary neurodegenerative disorder characterized by changes in personality, cognitive impairment and loss of motor function, leading to death over a period of 10 to 30 years. The disease is caused by a mutation in the gene encoding for the huntingtin protein (HTT), and the resulting mtHTT is ubiquitously expressed and exhibits the ability to propagate from cell to cell to disseminate the pathology. Thus, immunotherapy targeting mtHTT may provide a viable approach to neutralize the defective protein in the extracellular space and modify disease progression.
AFFiRiS has developed mAB C6-17, a monoclonal antibody targeting a particularly exposed region of the HTT protein. In vitro testing has already shown that mAB C6-17 is able to significantly inhibit mtHTT uptake in cultured cells (Bartl et al. 2020).
Affiris’ poster presentation, “In vivo targeting and reduction of mtHTT protein by passive immunization with the monoclonal antibody C6-17,” reports results from a new study with mAB C6-17. The study showed that the monoclonal antibody quickly distributes in the body, including into peripheral organs and the central nervous system (CNS). As a result, mAB C6-17-treated animals exhibited significantly reduced mtHTT levels in peripheral organs and the CNS as well as displaying improved motor performance.
“Interference with the extracellular mutant huntingtin protein by a specific antibody could reduce and block intercellular mtHTT transmission and spread of HD pathology. This is expected to modify disease progression in the brain and periphery,” explained Günther Staffler, PhD, Chief Technology Officer of AFFiRiS AG. “Our previous findings supported mAB C6-17 as a potential passive immunotherapy to treat features of Huntington’s disease. These new in vivo data are further evidence of the potential of this monoclonal antibody in fighting this disease.”
“RNA/DNA targeted approaches focusing on the CNS may not be sufficient to treat a full body disease like Huntington disease, as they could leave a quantity of mtHTT still capable of inducing pathology in the periphery and also the CNS,” commented Prof. Amber Southwell, Assistant Professor, Burnett School of Biomedical Sciences, Division of Neuroscience, UCF, Orlando FL, and Principal Investigator. “Our findings demonstrate that mAB C6-17 treatment may slow progression of motor deficits by inducing degradation of extracellular mtHTT protein. Immunotherapies such as mAB C6-17 could therefore be an useful approach in combination with other HTT-lowering interventions to obtain a more complete depletion of mtHTT and to achieve a more comprehensive benefit in the treatment of HD.”
About AFFiRiS AG:
AFFiRiS is a clinical-stage biotechnology company located in Vienna, Austria, with a vision of using the immune system to identify and target human proteins central to the development and progression of neurodegenerative diseases, based on its proprietary patented AFFITOME® technology. The Company’s ultimate goal is to improve the lives of patients suffering from these diseases by providing disease-modifying specific immunotherapies. With its lead candidate, AFFITOPE® PD01, AFFiRiS is the leader in active immunotherapies for Parkinson’s disease. For further information, please visit www.affiris.com and follow us on LinkedIn and Twitter.