Parkinson's disease and related neurological disorders are associated with the buildup of misfolded proteins in the brain. While existing therapies can manage symptoms, a significant need is still out there for treatments that can better manage symptoms while also addressing underlying disease progression.
Why are Phase 1 clinical trials critical to drug development?
Phase 1 clinical trials provide the first opportunity to evaluate an investigational therapy in humans. These studies are designed to assess safety and tolerability, while also generating critical pharmacokinetic data that shows how a drug is absorbed, distributed, metabolized, and eliminated by the body. They generate the foundational data needed to advance promising therapies into patient studies.
Researchers may also evaluate factors such as the effect of food on drug exposure and determine dosing strategies for future studies.
Moving a promising Parkinson's therapy into clinical testing
Between 2020-2021, Quotient Sciences conducted an early phase clinical trial, with a goal of evaluating an investigational therapy for Parkinson’s disease, in partnership with MODAG Gmbh, later acquired by Teva.
MODAG and Quotient Sciences investigated anle138b, an experimental therapy designed to target the toxic protein aggregates believed to contribute to neurodegeneration. To evaluate whether anle138b could safely advance into patient studies, a randomized, double-blind, placebo-controlled Phase 1 trial was performed.
Setting an important milestone for follow-up clinical trials in patients
Using a healthy volunteer study, MODAG and Quotient Sciences could asses how the drug was absorbed, distributed, and processed in the body across a range of dose levels. The food impact on drug exposure was also reviewed in order to inform later dosing strategies.
The study found that anle138b was generally well tolerated, with adverse events reported as mostly mild and resolved during the study period. Importantly, the drug achieved exposure levels in humans that exceeded those associated with therapeutic effects in preclinical Parkinson's disease models.
The research demonstrates how a carefully designed Phase 1 study in healthy volunteers was used to assess safety, tolerability, and drug exposure. Download the publication, which was featured in The Lancet (June 2022), to see how Quotient Sciences supports sponsors through early clinical development to bring innovative therapies closer to patients.