Vaxxinity, Inc. announced that its investigational synthetic peptide vaccine for Parkinson’s, UB-312, met the primary endpoints of a Phase 1 clinical trial designed to assess its tolerability and efficacy in creating an antibody response against toxic aggregates of alpha-synuclein. In a preclinical trial last year, UB-312 prevented the accumulation of alpha-synuclein and improved motor function in a mouse model of synucleinopathies.
The placebo-controlled, double-blind Phase 1 trial comprised two parts. Part A, whose results were published in Movement Disorders, was a dose-escalation study of UB-312 vs placebo in 50 healthy participants. The vaccine was well-tolerated and produced an antibody response in all participants who receive the 300 μg dosage strength.
Part B tested two dosage strengths of UB-312 vs placebo in 20 patients with early Parkinson’s. It consisted of a 20-week treatment period followed by 24 weeks of observation. The majority (12/13) of patients who received UB-312 developed anti-alpha-synuclein antibodies, and the treatment was considered safe and well-tolerated. Further, UB-312 was detected in participants’ cerebrospinal fluid, indicating it is able to cross the blood-brain barrier.
“These positive Phase 1 results demonstrate several important features necessary for an immunotherapy against Parkinson’s disease and other synucleinopathies to be successful…UB-312 was observed to safely break immune tolerance, inducing antibodies against toxic aggregated forms of alpha-synuclein. Importantly, these antibodies crossed the blood brain barrier, and the data also suggest potential target engagement in the periphery, where pathological alpha-synuclein is known to be concentrated. Together these results support the further development of UB-312 in a Phase 2 clinical trial. We continue to view UB-312 as a promising candidate for the prevention or disease modification of Parkinson’s disease globally,” said Vaxxinity CEO Mei Mei Hu. Vaxxinity plans to move UB-312 into a Phase 2 trial based on these positive results.