Vaxxinity Announces UB-312 Successfully Met Primary Objectives of Phase 1 Clinical Trial in Parkinson’s Disease - Seite 2

Geert Jan Groeneveld, MD, PhD, Professor of Clinical Neuropharmacology at Leiden University Medical Center, CMO/CSO of CHDR, and principal investigator of the Phase 1 trial, said, “A vaccine against alpha-synuclein is a revolutionary concept that can be of immense impact in treating neurodegenerative diseases such as Parkinson’s disease and synucleinopathies.”

Part B consisted of a 20-week treatment period followed by 24 weeks of observation. This study was conducted to evaluate the safety, tolerability and immunogenicity of UB-312 in patients with PD. Part B end-of-study results are as follows:

• The primary objectives were met.
• 92% of patients (12 out of 13) who completed dosing with UB-312 developed anti-aSyn antibodies.
• UB-312 was generally safe and well-tolerated with overall adverse event profile similar across UB-312 and placebo groups. Two patients experienced serious adverse events (SAEs). Only one event was deemed possibly related to the trial, and all SAEs were resolved.
• Antibodies were also detectable in the CSF.

The trial included exploratory measures of Parkinson’s disease progression, including UPDRS Parts II and III, and the Montréal Cognitive Assessment; however, the trial was not designed or powered to detect differences between UB-312 and placebo on these measures.

Additionally, The Michael J. Fox Foundation (“MJFF”) is funding a 2-year collaborative project between Vaxxinity, the Mayo Clinic, and the University of Texas Houston using CSF collected from patients who participated in Part B of the trial. This work is evaluating the potential of protein misfolding cyclic amplification (“PMCA”) to assess target engagement and will also aim to characterize the anti-aSyn antibodies produced after UB-312 administration.

More information about the Phase 1 trial is available at https://clinicaltrials.gov/ct2/show/NCT04075318.

About Parkinson’s Disease

Parkinson’s disease (PD) affects approximately one million people in the United States and more than 10 million people worldwide. PD is a chronic and progressive neurodegenerative disorder that affects predominately dopamine-producing (“dopaminergic”) neurons in the substantia nigra area of the brain. While today’s approved products are aimed at providing symptomatic relief, they often produce significant side effects and lose their beneficial effects over time. There are no currently approved disease-modifying therapeutics for PD. Alpha-synuclein (aSyn) is a protein highly expressed in neurons, mostly at presynaptic terminals, suggesting a role in synaptic vesicle trafficking, synaptic functions and in regulation of neurotransmitter release at the synapse. Mutations in the gene encoding aSyn are known to cause or increase the risk of developing PD or dementia with Lewy bodies (DLB) and have been shown to alter the secondary structure of aSyn, resulting in misfolded and aggregated forms of the protein (i.e., pathological forms). While mutations in the aSyn gene are rare, aggregates of aSyn in the form of Lewy bodies (LB) and Lewy neurites are common neuropathological hallmarks of both familial and sporadic PD, suggesting a key role of aSyn in PD neuropathogenesis. Immunotherapy approaches targeting aSyn have been shown to ameliorate aSyn pathology as well as functional deficits in mouse models of PD and are now being investigated in the clinic.