AFFiRiS, a clinical-stage biotechnology company developing novel disease-modifying specific active immunotherapies (SAITs), today announced the publication of positive results of a phase 1 randomized, double-blind, placebocontrolled trial in Multiple System Atrophy (MSA) in the peer-reviewed Movement Disorders Journal (https://doi.org/10.1002/mds.28218).
MSA is a rare and fatal neurodegenerative disease that is caused by the progressive loss of nerve cells in the brain and spinal cord. Only very limited and symptomatic treatment options are available for patients with MSA. Symptoms worsen continuously with rapid progression after the onset of disease and a mean survival time of 6-10 years.
To address this urgent unmet medical need, AFFiRiS has developed AFFITOPE®s PD01A and PD03A, two novel immunotherapy candidates designed to induce a sustained antibody response following immunization by specifically targeting the alpha-synuclein (aSyn) epitope. The accumulation of aSyn in oligodendrocytes in the brain is believed to play a central role in the neurodegenerative process in MSA.
The results of this 52-week phase 1 trial in 30 patients with early MSA showed that both immunotherapies, PD01A and PD03A, were safe and well tolerated after five subcutaneous injections. Treatment-related adverse events were similar to site reactions typically observed in vaccination trials. Immunization with PD01A resulted in a significant increase in IgG titers against the immunizing PD01 peptide, and 89% of treated patients developed a PD01-specific antibody response after receiving all injections. Induced antibodies displayed clear reactivity to the aSyn target epitope. Moreover, the antibody response increased rapidly following a booster injection, suggesting that the priming immunizations produced a significant memory effect, and antibody levels persisted until the end of the study. Titers and antibody responder rate (58%) were lower in the PD03A treated group. No significant changes were found in clinical rating scale scores during the study period in all groups. However, the study was not designed to determine efficacy effects.
“Current treatment options for MSA are limited and provide only some symptomatic relief. Disease modifying therapies remain therefore an urgent unmet need,” says Wassilios Meissner, Professor of Neurology and Chairman of the French Reference Center for MSA at the University Hospital Bordeaux, principal investigator of the trial. “The results of this trial in terms of safety, tolerability and immune reaction following repeated administrations of two active immunotherapies, PD01A and PD03A, warrant further investigation of SAITs in MSA patients.”
“We are encouraged by these clinical results which support aSyn targeting therapies as a promising approach to modify disease progression in MSA. Planning activities are currently ongoing for future clinical studies in MSA,” adds Noel Barrett, Ph.D., CEO of AFFiRiS AG. “With our Specific Active Immunotherapies, we aim to harness the power of the body’s own immune system to target and fight the root causes of neurodegenerative diseases, rather than just treating symptoms. Based on our AFFITOME® Technology, AFFiRiS has developed a pipeline of preclinical and clinical stage immunotherapy candidates designed to effectively treat neurodegenerative diseases such as MSA, Parkinson’s and dementia with Lewy bodies, in order to improve the lives of severely ill patients.”
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 active immunotherapies (SAIT). 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.