Phase I trial of PD 01A vaccine for Parkinsons meets primary endpoint - AFFiRiS

AFFiRiS AG has presented data from a Phase I clinical trial of PD 01A, a vaccine approach that aims to slow or stop Parkinson's disease progression. In this study, two different doses of PD 01A were safe and well tolerated, meeting the primary endpoint of the trial. Secondary endpoints of the study included the induction of an alpha-synuclein-specific antibody response. A hallmark pathology of Parkinson's disease is aggregates of protein - chiefly alpha-synuclein - called Lewy bodies that accummulate in brain cells, leading to cell degeneration and cell death. Researchers hypothesize that reducing alpha-synuclein accumulation will be neuroprotective; AFFiRiS is using active immunotherapy to test that theory and develop a disease-modifying treatment. PD 01A was applied at two different doses (15 �g and 75 �g) to 12 patients per group.

All received four vaccinations in monthly intervals, and all completed the study. Eight patients on best medical care, including standard symptomatic medication, served as a control group. Each patient was regularly seen and evaluated during a 12-month period. Fifty percent of the vaccinated patients generated alpha-synuclein-specific antibodies as measured in serum samples. Additionally, vaccine-induced antibodies were detectable in cerebrospinal fluid. This induction of antibodies against alpha-synuclein is strong preliminary evidence in support of the principle of AFFiRiS' proprietary therapeutic vaccine. Furthermore, analysis of clinical endpoints revealed a trend, consistent over all parameters, towards functional stabilization of the vaccinated groups as compared to non-vaccinated control patients. The pharmacodynamic profile of PD 01A and its clinical effects will be the basis of later phase studies, should development continue. The next study will take place in Austria and focus on assessing the immunological and clinical effects of a boost vaccination. Recruitment is expected to begin September.