Mitsubishi Tanabe Pharma America announced Oxford BioDynamics has joined the REFINE-ALS study, which was designed to identify and measure specific biomarkers in people with amyotrophic lateral sclerosis (ALS). Using a technology platform to evaluate epigenetic and protein biomarkers, the company's analyses may offer insights on disease progression and treatment effect.
"ALS is a complex disease and the specific causes of disease onset and progression are not fully understood," said Stephen Apple, M.D., Senior Director of Medical Affairs, MTPA. "Through this biomarker study we are seeking to enhance our understanding of RADICAVA therapy in ALS. We are proud to announce Oxford BioDynamics has joined us in this effort and we look forward to seeing the data we gain from their technologies."
REFINE-ALS is sponsored by MTPA and led by Massachusetts General Hospital (MGH) Neurological Clinical Research Institute (NCRI). Biomarkers including oxidative stress, inflammation, neuronal injury/death and muscle injury, as well as clinical assessments, will be obtained from up to 300 patients prior to initiating treatment with edaravone, at start of treatment and at pre-specified time points for 24 weeks.
Oxford BioDynamics will evaluate biomarker panels utilizing EpiSwitch™. This proprietary technology platform assesses a novel class of epigenetic biomarkers known as chromosome conformation signatures, which are aimed at understanding the rate of disease progression.
"ALS is a challenging disease and we're excited to apply the EpiSwitch technology to uncover more information about its progression," said Alexandre Akoulitchev, Chief Scientific Officer, Oxford BioDynamics. "We are very pleased that OBD has been selected for this pivotal prospective trial led by the world leaders in ALS therapeutic development and patient care. As an extension of our previous work in ALS based on collaborations with ALS experts, this is an acknowledgment of the utility and value that EpiSwitch offers. We look forward to collaborating with our study partners to help disease understanding and clinical care for the entire ALS community."
At least 30 genes are believed to play a role in ALS, but more information is needed on their potential impact in ALS. This, combined with the lack of reliable laboratory tests to identify ALS, contributes to poor disease diagnosis, which may impact treatment.
All participants in the prospective, observational, longitudinal, multicenter trial will be newly prescribed commercially available RADICAVA® (edaravone). Patient biomarker data and disease progression assessments will be compared to samples stored at biorepositories and progression models, respectively. The study is expected to begin in late spring of 2019, with early interim analyses planned for later in the year.