The ALS Association announced the awarding of an ALS Association-Initiated Grant to David Ennist, Ph.D., M.B.A, chief scientific officer of Origent Data Sciences, Inc. based in Vienna, VA for $497,433 over 34 months that will enable a research partnership with Cytokinetics Inc. to improve clinical trial design.

The topic of Dr. Ennist's project is "Prospective Validation of ALS Disease Models and Tools." This collaboration will refine and prospectively validate an Origent computer model to predict the course of ALS disease progression leveraging data from Cytokinetics' clinical trials of tirasemtiv, the first of its kind in a clinical trial setting.

"We are extremely excited to see this collaboration get underway," said ALS Association chief scientist Lucie Bruijn, Ph.D., M.B.A. "This tool has the potential to accelerate clinical trials for ALS and the Cytokinetics phase III trial provides an excellent opportunity to validate the disease progression algorithm."

ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure, and only one drug approved by the U.S. Food and Drug Administration (FDA) modestly extends survival.

The ALS Association-Initiated awards support collaborative projects that address research gaps, areas of high risk-high reward and/or areas that provide novel opportunities. Investigators submit proposals for peer-review in response to topics The ALS Association and its advisors have determined are priority areas of research.

Under Dr. Ennist's leadership, Origent Data Sciences, Inc. has previously won the DREAM Phil Bowen ALS Prediction Prize4Life Challenge to develop an ALS disease progression algorithm based on the PRO-ACT database. The PRO-ACT database is powerful as it contains more than 8 million data points extracted from the de-identified records of 8,500 patients who participated in 17 ALS phase II & III clinical trials.

Previously, the Origent models, predicting both function and survival of people living with ALS, have been validated using internal and retrospective external datasets. The goal of this study is to make prospective model validations. In collaboration with Cytokinetics, Inc., they will first confirm the retrospective external validation of their existing predictive models (including the ALSFRS-R, respiratory, gross, fine, and bulbar sub-scores, SVC and survival models) using the recently completed phase IIb BENEFIT-ALS tirasemtiv trial. Once the retrospective validations are confirmed, they will move to prospectively validating the models in parallel with the VITALITY-ALS phase III tirasemtiv trial. If validated, the models will allow for faster, less expensive ALS clinical trials that enroll fewer patients, resulting in a quicker path to new ALS therapeutics.

"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," said Dave Ennist, chief science officer, Origent Data Sciences. "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."

"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, M.D., Cytokinetics' senior director clinical research and development and head of Neuromuscular Therapeutics.

"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."