Session Information
Date: Thursday, June 8, 2017
Session Title: Parkinson’s Disease: Clinical Trials, Pharmacology And Treatment
Session Time: 1:15pm-2:45pm
Location: Exhibit Hall C
Objective: To evaluate PRX002 in patients with PD in a double-blind, placebo-controlled, phase 1b multiple ascending-dose study.
Background: PRX002 (RG7935) is an investigational monoclonal antibody designed to neutralize extracellular neurotoxic forms of alpha-synuclein. This may inhibit the cell-to-cell transmission of the aggregated, pathogenic form of alpha-synuclein and potentially modify disease progression associated with Parkinson’s disease (PD).
Methods: Patients with mild to moderate PD (Hoehn and Yahr stages 1-3), randomly assigned to six escalating-dose cohorts, received PRX002 or placebo (2:1 for 0.3, 1, 3, or 10 mg/kg [n=48] and 3:1 for 30 or 60 mg/kg [n=32]). Safety and tolerability (primary objectives), pharmacokinetics, immunogenicity, pharmacodynamic effects, and clinical effects were assessed.
Results: The intent-to-treat population (n=80) consisted predominantly of Caucasian (97.5%) males (80%) whose mean age was 58.3 years. PRX002 was well tolerated; no serious or severe treatment-emergent adverse events (TEAEs) were reported. TEAEs experienced by ≥5% of patients were constipation, infusion reaction (allergic), diarrhea, headache, peripheral edema, post-lumbar puncture headache, and upper respiratory infection. Central nervous system (CNS) penetration was demonstrated by a dose-dependent increase in PRX002 levels in the cerebral spinal fluid (CSF). Across all doses, the mean PRX002 concentration in CSF was 0.3% relative to serum. Results showed a rapid dose- and time-dependent mean reduction of free serum alpha-synuclein levels of up to 97% after a single dose (P<0.0001); this was consistently observed after two additional monthly doses.
Conclusions: PRX002 had an acceptable safety and tolerability profile, penetrated the CNS, and reduced free serum alpha-synuclein. A planned phase 2 study will evaluate PRX002 as a disease-modifying treatment for PD. These results were previously presented at the Alzheimer’s & Parkinson’s Disease Congress, AD/PD, Vienna, 2017.
To cite this abstract in AMA style:
J. Jankovic, I. Goodman, B. Safirstein, D. Schenk, G. Kinney, M. Koller, D. Ness, S. Griffith, M. Grundman, J. Soto, S. Ostrowitzki, F. Boess, M. Martin-Facklam, J. Quinn, S. Isaacson, D. Jennings, O. Omidvar, A. Ellenbogen. Results From a Phase 1b Multiple Ascending-Dose Study of PRX002, an Anti–Alpha-Synuclein Monoclonal Antibody, in Patients with Parkinson’s Disease [abstract]. Mov Disord. 2017; 32 (suppl 2). https://www.mdsabstracts.org/abstract/results-from-a-phase-1b-multiple-ascending-dose-study-of-prx002-an-anti-alpha-synuclein-monoclonal-antibody-in-patients-with-parkinsons-disease/. Accessed May 13, 2025.« Back to 2017 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/results-from-a-phase-1b-multiple-ascending-dose-study-of-prx002-an-anti-alpha-synuclein-monoclonal-antibody-in-patients-with-parkinsons-disease/