UB-312 is first immunotherapy to show reduction of pathological alpha-synuclein in cerebrospinal fluid of Parkinson’s patients

LISBON, Portugal I March 07, 2024 I Vaxxinity, Inc. (NASDAQ: VAXX), a U.S. company pioneering the development of a new class of medicines, announced positive clinical data from its UB-312 program in Parkinson’s disease (PD) presented by Jean-Cosme Dodart, PhD, SVP of Research at Vaxxinity in an oral session at the AD/PD™ 2024 International Conference on Alzheimer’s and Parkinson’s Disease, held virtually and in Lisbon, Portugal from March 5 to March 9, 2024. UB-312 is the first active immunotherapy candidate to show reduction of pathological alpha-synuclein (aSyn) in cerebrospinal fluid (CSF) of PD patients.

UB-312 is designed to target aggregated forms of aSyn, the toxic species that underlies PD and other synucleinopathies. As part of the randomized, double-blind, placebo-controlled Phase 1 clinical trial, The Michael J. Fox Foundation (MJFF) funded a 2-year collaborative project between Vaxxinity, the Mayo Clinic, and UTHealth Houston to analyze CSF collected from patients, and to conduct exploratory research to assess target engagement.

The UB-312-induced antibodies showed preferential binding to aggregated aSyn and almost no binding to normal monomeric aSyn, as measured by dot blot. After a single priming regimen, those treated with UB-312 in the 300/100/100µg dosing group showed a 20% decrease from baseline in aggregated aSyn in the CSF compared to a 3% increase in the placebo group (p<0.05), as measured by a Seed Amplification Assay (SAA). Further, a post hoc analysis showed that patients with detectable UB-312-induced antibodies in the CSF exhibited improvement in activities of daily living as measured by the MDS-UPDRS II clinical scale (p<0.01). These data also suggest a correlation between reduction in aggregated aSyn in the brain and change in MDS-UPDRS II (R=0.52, p=0.001).

“What we see from our UB-312 program is the potential to change the whole conversation around Parkinson’s treatment and prevention,” says Lou Reese, Co-Founder and Executive Chairman of Vaxxinity. “Our findings suggest UB-312 could transform Parkinson’s care, offering hope for improved outcomes with a disease-modifying treatment. The future isn’t decades away: today’s Parkinson’s patients may have hope for the near, not distant future.”

The aforementioned results come from Part B of the Phase 1 clinical trial in 20 PD patients. Vaxxinity expects to publish these results in a peer reviewed scientific journal soon. Results from Part A of the trial in healthy volunteers were published in Movement Disorders in 2022 (Yu et al.). Both parts of the trial showed UB-312 to be generally well tolerated (most adverse events were mild and transient) and immunogenic, with antibodies detectable in serum and CSF.

“Currently, there are no treatments that address the underlying conditions of Parkinson’s, and we are very excited about this target engagement data. This provides us confidence that we are going after the right target and in a way that is statistically and clinically relevant to patients. There is new hope on the horizon,” said Jean-Cosme Dodart, PhD, SVP Research at Vaxxinity. “With Parkinson’s being the fastest growing neurodegenerative disease in the world, Vaxxinity remains committed to developing safe, convenient, and effective disease-modifying active immunotherapies for all.”

For more information about Vaxxinity, Inc., visit http://www.vaxxinity.com and follow us on social media @vaxxinity.

About Vaxxinity

Vaxxinity, Inc. is a purpose-driven biotechnology company committed to democratizing healthcare across the globe. The company is pioneering a new class of medicines aimed at disrupting the existing treatment paradigm for chronic disease, increasingly dominated by monoclonal antibodies, which suffer from prohibitive costs and cumbersome administration. The company’s proprietary technology platform has enabled the innovation of novel synthetic peptide immunotherapy candidates designed to bring the efficiency of vaccines to the treatment of chronic diseases, including Alzheimer’s disease, Parkinson’s disease, migraine, and hypercholesterolemia. The technology is also implemented as part of a COVID-19 vaccine program. Vaxxinity has optimized its pipeline to achieve a potentially historic, global impact on human health.