Clinical Outcomes of ALSFRS-R, Slow Vital Capacity, and Plasma Neurofilament Light to be Evaluated in Placebo Digital Twins of Study Participants Dosed with Prosetin

NEW YORK, Sept. 26, 2024 /PRNewswire/ — ProJenX, Inc., a clinical-stage biotechnology company developing novel therapies targeting biologically defined pathways for the treatment of amyotrophic lateral sclerosis (ALS) and other debilitating brain diseases, today announced its partnership with Unlearn to augment PRO-101, its Phase 1 clinical trial of prosetin—a brain-penetrant, MAP4 kinase (MAP4K) inhibitor—with Unlearn’s advanced generative artificial intelligence (AI) technology.

Unlearn, a pioneering technology company innovating machine learning to revolutionize medical research, will implement its ALS-Digital Twin Generator (ALS-DTG) to produce digital twins of PRO-101 clinical trial participants living with ALS to evaluate the clinical outcomes of ALSFRS-R, Slow Vital Capacity, and plasma neurofilament light over a 52-week open-label extension. Digital twins will act as placebos of participants dosed with prosetin.

Unlearn’s ALS-DTG is an advanced machine-learning generative model trained on patient-level data to generate probabilistic forecasts of future health outcomes, known as digital twins, for individual clinical trial participants. At the start of a trial, baseline data are collected from all participants. The ALS-DTG uses these data to forecast longitudinal clinical placebo outcomes, biomarkers, and labs—regardless of the participant’s randomization assignment. Digital twins help address key challenges in clinical development and are delivered through Unlearn’s Platform.

“ProJenX and Unlearn’s collaboration aims to extract valuable insights that will help us expedite clinical trials and more efficiently evaluate prosetin in individuals living with ALS,” said Stan Abel, president and CEO at ProJenX. “Digital twins provide the opportunity for us to compare participants with ALS who are dosed with prosetin to their digitally generated placebo versions of themselves based on their baseline measurements. In PRO-101, this collaboration will enhance our interpretation of the effects of prosetin on ALS patients to support a more efficient and data-driven clinical development program.”

“With hundreds of thousands of people affected by ALS, the need to accelerate research that can transform treatment for this debilitating and paralyzing disease has never been more pressing,” said Steve Herne, CEO of Unlearn. “ProJenX is making significant strides in this area, and we are confident that our collaboration will continue to support their efforts to develop impactful therapies that improve the lives of patients everywhere.”

About PRO-101

PRO-101 is a hybrid phase 1 clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in healthy volunteers and patients with ALS. Parts 1a and 1b, which have been completed, consisted of a randomized, double-blind, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, and pharmacokinetics of single ascending and multiple doses of prosetin in healthy volunteers. Parts 1c and 1d will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in ALS patients. For more information about PRO-101, contact [email protected].

About ProJenX

ProJenX is a clinical-stage biotechnology company developing novel, brain-penetrant, targeted therapies to address untreatable brain diseases, with an initial focus on ALS. ProJenX was created out of a long-term research collaboration between Project ALS and researchers at Columbia University to rapidly develop and commercialize its lead therapy candidate, prosetin, for people living with ALS. At the heart of ProJenX’s approach is an innovative, patient-specific, cell-based drug discovery platform that can be leveraged for research and drug development for ALS and other debilitating brain diseases. For more information, visit projenx.com.

About Unlearn

Unlearn is a pioneering AI company with a product-driven focus, dedicated to transforming medicine by eliminating trial and error. Our Platform harnesses the power of digital twins—advanced computational models of patients—to empower pharmaceutical and biotech companies to overcome critical challenges in clinical development.

For more information, please visit www.unlearn.ai or follow @UnlearnAI on Twitter/X and @unlearn-ai on LinkedIn.