BioArctic Expands Exidavnemab Phase 2a Study to Include MSA Patients in Spain and Poland
In a significant development for the treatment of neurodegenerative diseases, BioArctic AB has announced the regulatory approval to expand its Exidavnemab Phase 2a study to include patients with Multiple System Atrophy (MSA). This notable change has been sanctioned by regulatory authorities in both Spain and Poland. MSA, a rare and rapidly progressive condition, affects the central and autonomic nervous systems, leading to severe impairment in balance, movement, and other essential functions.
The ongoing Phase 2a study, officially known as the EXIdavnemab Synucleinopathy Trial (EXIST), initially focused solely on patients with Parkinson's disease. The study utilizes a randomized, double-blind, placebo-controlled design to evaluate the safety and tolerability of Exidavnemab, an innovative antibody targeting alpha-synuclein aggregates known for their involvement in various neurodegenerative disorders.
With the recent protocol modification approved, an additional cohort of 12 MSA patients will now be included in the study alongside the existing 24 participants with mild to moderate Parkinson's disease. This inclusion allows for the evaluation of the underlying mechanisms of Exidavnemab across both conditions, which share characteristics of alpha-synuclein pathology.
The principal aim of the EXIST study is to establish the safety profile and tolerability of Exidavnemab, along with analyzing a diverse range of biomarkers. These include samples from plasma, cerebrospinal fluid (CSF), and digital measures, providing comprehensive insights into treatment effects and disease progression.
MSA remains a condition with substantial unmet medical needs due to the absence of effective treatments to alleviate its progression. The condition is marked by a pathological aggregation of alpha-synuclein, contributing to the demise of nerve cells responsible for critical bodily functions such as respiration, digestion, and autonomic regulation. Currently, patients diagnosed with MSA typically face a grim prognosis, with most surviving only a few years following initial symptom onset.
Exidavnemab represents a promising avenue for the treatment of synucleinopathies. Specifically designed as a monoclonal antibody, it selectively binds to aggregated forms of alpha-synuclein, including oligomers, protofibrils, and fibrils. By promoting their clearance, Exidavnemab has the potential to mitigate the adverse effects of alpha-synuclein and preserve neuronal function.
The ongoing research into Exidavnemab signifies a pivotal moment in drug development targeting neurodegenerative diseases. The implications of successful outcomes from the EXIST trial could herald a new era in the management of both MSA and Parkinson's, addressing critical therapeutic gaps that have historically challenged healthcare providers and patients alike.
As a notable milestone, the U.S. FDA's Office of Orphan Products Development granted orphan drug designation to Exidavnemab for treating MSA in March 2025, underscoring the drug's potential in the therapeutic landscape.
BioArctic AB aims to leverage its extensive research portfolio in neurodegenerative disease management, which includes the notable success of Leqembi® (lecanemab) for Alzheimer’s disease. With innovative technologies like the proprietary BrainTransporter™, the company is committed to discovering treatments that not only delay disease progression but also improve patients' quality of life.
For further updates on the trial and BioArctic's advancements, interested parties can explore the company’s official channels or consult with media representatives at the provided contact points.
The ongoing Phase 2a study, officially known as the EXIdavnemab Synucleinopathy Trial (EXIST), initially focused solely on patients with Parkinson's disease. The study utilizes a randomized, double-blind, placebo-controlled design to evaluate the safety and tolerability of Exidavnemab, an innovative antibody targeting alpha-synuclein aggregates known for their involvement in various neurodegenerative disorders.
With the recent protocol modification approved, an additional cohort of 12 MSA patients will now be included in the study alongside the existing 24 participants with mild to moderate Parkinson's disease. This inclusion allows for the evaluation of the underlying mechanisms of Exidavnemab across both conditions, which share characteristics of alpha-synuclein pathology.
The principal aim of the EXIST study is to establish the safety profile and tolerability of Exidavnemab, along with analyzing a diverse range of biomarkers. These include samples from plasma, cerebrospinal fluid (CSF), and digital measures, providing comprehensive insights into treatment effects and disease progression.
MSA remains a condition with substantial unmet medical needs due to the absence of effective treatments to alleviate its progression. The condition is marked by a pathological aggregation of alpha-synuclein, contributing to the demise of nerve cells responsible for critical bodily functions such as respiration, digestion, and autonomic regulation. Currently, patients diagnosed with MSA typically face a grim prognosis, with most surviving only a few years following initial symptom onset.
Exidavnemab represents a promising avenue for the treatment of synucleinopathies. Specifically designed as a monoclonal antibody, it selectively binds to aggregated forms of alpha-synuclein, including oligomers, protofibrils, and fibrils. By promoting their clearance, Exidavnemab has the potential to mitigate the adverse effects of alpha-synuclein and preserve neuronal function.
The ongoing research into Exidavnemab signifies a pivotal moment in drug development targeting neurodegenerative diseases. The implications of successful outcomes from the EXIST trial could herald a new era in the management of both MSA and Parkinson's, addressing critical therapeutic gaps that have historically challenged healthcare providers and patients alike.
As a notable milestone, the U.S. FDA's Office of Orphan Products Development granted orphan drug designation to Exidavnemab for treating MSA in March 2025, underscoring the drug's potential in the therapeutic landscape.
BioArctic AB aims to leverage its extensive research portfolio in neurodegenerative disease management, which includes the notable success of Leqembi® (lecanemab) for Alzheimer’s disease. With innovative technologies like the proprietary BrainTransporter™, the company is committed to discovering treatments that not only delay disease progression but also improve patients' quality of life.
For further updates on the trial and BioArctic's advancements, interested parties can explore the company’s official channels or consult with media representatives at the provided contact points.
Topics Health）
【About Using Articles】
You can freely use the title and article content by linking to the page where the article is posted.
※ Images cannot be used.
【About Links】
Links are free to use.