The data were presented at the International Congress of Parkinson’s Disease and Movement Disorders (MDS), held Oct. 5-9, in Hong Kong, China.
Parkinson’s disease and progressive supranuclear palsy have overlapping symptoms, such as slowed movements and gait difficulty, which hinders accurate diagnosis. However, while the hallmark of Parkinson’s disease is the toxic accumulation of alpha-synuclein proteins, progressive supranuclear palsy is characterized by the build-up of tau proteins.
BIIB054, developed by Biogen as a potential therapy for Parkinson’s disease, is an antibody that binds to clumps of alpha-synuclein, preventing it from spreading.
A recent Phase 1 pilot study (NCT02459886) in healthy participants and participants with early Parkinson’s disease showed that a range of single doses of BIIB054 have favourable safety, tolerability, and pharmacokinetics profiles, supporting its further clinical development.
Pharmacokinetics refers to the compound’s absorption, bioavailability, distribution, metabolism, and excretion in the body.
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This led to the design of a Phase 2 study (NCT03318523) called SPARK, to evaluate the safety, pharmacokinetics, and potential effectiveness of three doses of BIIB054 in Parkinson’s patients. The study was presented in a scientific poster titled “Design and Status of the BIIB054 SPARK Trial.”
This multicenter, randomized, double-blind, placebo-controlled study is currently recruiting patients with early, untreated Parkinson’s at 90 international sites. The first patient was enrolled in January 2018, and enrollment is expected to continue through the end of the year.
In North America, the study is being developed in cooperation with the Parkinson Study Group.
Participants with will be randomized to receive monthly either one of three doses of BIIB054 (250 mg, 1,250 mg, or 3,500 mg) or a placebo, directly into the blood, for two years.
The primary objective is to evaluate BIIB054’s safety, while secondary goals include the assessment of BIIB054’s effects in dopamine-producing nerve cells — the most affected cells in Parkinson’s disease — and in clinical symptom burden.
Interim data of the ongoing Phase 1 clinical study (NCT02658916) evaluating the safety of three doses (150, 700 or 2100 mg) of BIIB092 — Biogen’s experimental therapy for the treatment of diseases with toxic aggregates of tau protein — in patients with progressive supranuclear palsy also were presented.
The poster, “Open-label extension (OLE) of a multiple ascending dose (MAD) study of BIIB092 in participants with PSP: Safety analysis,” showed that all three doses of BIIB092 were safe and well-tolerated.
Treatment-related side effects occurred in 11 (23%) patients — with no apparent association with dose — and eight patients had 10 serious side effects, which researchers considered to be non-related to BIIB092 treatment.
This data supports further studies on the safety and effectiveness of BIIB092 in people with progressive supranuclear palsy.
Biogen also presented specific data of the ongoing five-year observational study called Parkinson’s Progression Markers Initiative (PPMI) study (NCT01141023). The poster was titled “Effect of PD medication on disease progression as measured by rate of change in MDS_UPDRS and DaT SBR in PPMI study.”
Because during clinical trials for new Parkinson’s therapies it is common for patients to start to take approved symptomatic Parkinson’s medications, researchers evaluated the effects of those treatments in disease progression. This knowledge may help to improve Parkinson’s clinical trials’ design.
Among the 423 participants who entered the study, up to 55% of them started Parkinson’s medications — dopamine agonists, levodopa, or MAO-B inhibitors — by one year after entering, and up to 78% of them by three years after enrollment.
The results showed that patients had a higher rate of disease progression — assessed through MDS-UPDRS, the most widely used scale to assess Parkinson’s progression — before starting medication than after starting it. Also, patients who started levodopa had slightly more severe disease at study entry (before starting medication) than those who started other Parkinson’s medications.
“The data … are a testament to Biogen’s commitment to its goal of delivering innovative therapies for people living with complex, neurodegenerative conditions like PSP [progressive supranuclear palsy] and PD [Parkinson’s disease],” Kate Dawson, MD, vice president of Biogen’s late-stage clinical development, in a press release.
“We look forward to advancing the clinical development programs to help better understand these challenging conditions,” she added.
Five additional posters related to Parkinson’s disease and progressive supranuclear palsy were presented by Biogen during the congress.