#AAN2018: Biogen’s BIIB054 Shows Positive Data in Phase 1 Parkinson’s Trial

Parkinson’s treatment candidate BIIB054 was well-tolerated and had a favorable pharmacological profile in preliminary Phase 1 clinical results.

Biogen will present those results at the 2018 American Academy of Neurology ANN Annual Meeting in Los Angeles, California, April 21-27. Parkinson’s News Today will be covering the conference.

The presentation “Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study of AntiAlpha-Synuclein Antibody BIIB054 in Patients with Parkinson’s Disease,” will be made at 3:30 p.m.  PST April 24.

People with Parkinson’s disease often exhibit clumps mainly composed of the aggregated form of a protein called alpha-synuclein. These clumps result from incorrect folding of alpha-synuclein into its 3-D structure and severely impair nerve cell communication. Scientists believe that alpha-synuclein aggregates spread from diseased to healthy cells, dictating Parkinson’s progression.

BIIB054 is a human-derived antibody that binds to aggregated alpha-synuclein, preventing the clumped protein from spreading.

Biogen conducted a placebo-controlled Phase 1 trial (NCT02459886) to evaluate the safety, tolerability and pharmacokinetics of a range of intravenous, single BIIB054 doses, in healthy participants and patients with early Parkinson’s disease. Pharmacokinetics refers to a drug’s absorption, bioavailability, distribution, metabolism, and excretion in the body.

Scientists also analyzed BIIB054’s pharmacokinetics in participants’ blood, along with the investigational compound’s immunogenicity, or its ability to induce an immune response in the body.

A total of 18 Parkinson’s patients (aged 47-75, 13 men) randomly received a range of single doses of BIIB054 (15-45 mg/kg) or placebo. Subjects were evaluated for 16 weeks with clinical, brain imaging, electrocardiogram (measuring the heart’s electrical activity), and laboratory assessments.

BIIB054’s blood half-life — the time required for a 50 percent reduction in the amount of a compound —  was approximately 30 days in Parkinson’s patients. The average amount of BIIB054 in the cerebrospinal fluid, which fills the brain and spinal cord, was 0.4% relative to that of the blood.

PK data of healthy volunteers and Parkinson’s patients were similar. BIIB054-synuclein complexes were found in the blood, confirming the molecule’s binding to alpha-synuclein.

Most adverse events were mild and unrelated to treatment with BIIB054.

“Preliminary results from the first study of BIIB054 in patients with [Parkinson’s] shows a favorable [pharmacokinetics], safety and tolerability profiles, providing rationale for further clinical development,” researchers wrote.