Predictive Model of ALS Progression Being Tested with Support of ALS Association
Origent Data Sciences, Inc., and Cytokinetics, Inc., announced a new research partnership to further refine and validate an Origent computer model to predict the progression of amyotrophic lateral sclerosis (ALS) in patients — a progression that widely varies, making effective clinical trials difficult and costly — by leveraging data from previous and ongoing studies of a Cytokinetics’ ALS treatment called tirasemtiv.
The research project, funded by Origent through an ALS Association research grant, will be the first prospective validation of a predictive model in the context of clinical trials. Origent models were previously validated through internal and retrospective external data sets.
“We are extremely grateful to The ALS Association for this generous grant and to Cytokinetics for the opportunity to access their robust clinical trial data,” Dave Ennist, chief scientific officer at Origent, said in a press release. “If validated, the models we have developed will be submitted to the FDA and may enable more nimble, cost-effective execution of ALS clinical trials, resulting in a potentially quicker path to new medicines.”
Under the partnership, Origent will seek retrospective external validation of existing predictive models using baseline data from BENEFIT-ALS, a Phase 2b trial of tirasemtiv (a fast skeletal muscle activator). If confirmed, Origent will then prospectively validate the models with completed data from VITALITY-ALS, Cytokinetics’ ongoing Phase 3 trial comparing the effects of tirasemtiv to placebo on Slow Vital Capacity (SVC) rates and other measures of skeletal muscle strength in ALS patients.
“We are extremely excited to see this collaboration get underway,” said Lucie Brujin, PhD, MBA, and the ALS Association’s chief scientist. “This tool has the potential to accelerate clinical trials for ALS and the Cytokinetics’ Phase 3 trial provides an excellent opportunity to validate the disease progression algorithm.”
While the average life expectancy of an ALS patient is between three and five years after diagnosis, some survive only a few months while others live for decades. The disease’s heterogeneity sets considerable challenges for clinical trial design, as patients progress at highly variable rates, confusing statistical analyses. Origent’s statistical models aim to relevantly identify progression in individual patients, and so reduce those statistical complexities for future clinical studies.
“This unique collaboration between Origent Data Sciences, Cytokinetics and The ALS Association reflects another step in our collective efforts to accelerate the clinical trial process and make new medicines available to people with ALS in desperate need of new therapeutic options,” said Jinsy Andrews, MD, senior director of Clinical Research & Development and head of Neuromuscular Therapeutics at Cytokinetics. “We are pleased to join with Origent to potentially validate their predictive model using datasets from BENEFIT-ALS and VITALITY-ALS which we hope may then facilitate the use of this novel technology to positively impact the design and conduct of future ALS clinical trials.”