Highlights

  • BIOA B advances its Phase 2a EXIST trial after interim data shows exidavnemab is well tolerated.
  • The company decided to initiate two additional cohorts testing higher doses in Parkinson’s disease and MSA patients.
  • BIOA B exidavnemab targets aggregated alpha-synuclein, linked to synucleinopathy-related neurodegeneration.

BioArctic AB (publ) (Nasdaq Stockholm: BIOA B) based in Sweden, focuses on research and development of therapies aimed at neurodegenerative diseases. The company previously partnered with Eisai on the development of Leqembi® (lecanemab), an approved treatment for early Alzheimer’s disease. Beyond Alzheimer’s, BioArctic is pursuing research programs in conditions such as ALS, Parkinson’s disease, and enzyme deficiency disorders.

The company has announced that its clinical Phase 2a study, known as EXIST, will proceed to additional dosing cohorts following an interim safety review. The evaluation of the first cohort, involving a lower dose of its investigational monoclonal antibody exidavnemab in patients with Parkinson’s disease, indicated that the treatment was safe and well tolerated.

The EXIST trial is assessing exidavnemab as a potential disease-modifying therapy for synucleinopathies, including Parkinson’s disease and Multiple System Atrophy (MSA). Based on the safety data, two new cohorts will now be initiated to examine a higher dose of exidavnemab in patients with both conditions.

The EXIST (EXIdavnemab Synucleinopathy Trial) study is structured as a randomized, double-blind, placebo-controlled trial. Its primary objective is to evaluate the safety and tolerability of exidavnemab, as well as its pharmacokinetic properties. In addition to these endpoints, the trial will monitor a range of biomarkers using plasma, cerebrospinal fluid (CSF), and digital assessments to build a broader understanding of the drug’s potential efficacy.

Exidavnemab is a monoclonal antibody specifically engineered to target aggregated forms of alpha-synuclein, a protein known to accumulate abnormally in neurodegenerative disorders such as Parkinson’s disease and MSA. The drug is designed to distinguish pathological aggregates from the normal, physiological form of the protein. These aggregated forms are implicated in neuronal damage, and exidavnemab’s mechanism aims to clear them selectively, potentially slowing the progression of neurodegeneration.

BioArctic CEO Gunilla Osswald commented on the development, stating that the safety profile observed aligns with expectations and supports dose escalation in the next phase of the study. The company views this as a significant step in its broader effort to evaluate exidavnemab for diseases marked by pathological protein aggregation.

In recent months, exidavnemab has received orphan drug designation (ODD) in the United States and a positive opinion for orphan medicinal product designation (OD) in the European Union for the treatment of MSA. These designations are intended to encourage the development of therapies for rare diseases by providing incentives such as extended market exclusivity and reduced regulatory fees.

There remains a substantial unmet need for disease-modifying treatments in Parkinson’s disease and MSA, both of which are characterized by progressive motor and non-motor symptoms and currently lack curative therapies. Existing treatments largely manage symptoms without affecting the underlying disease mechanisms.