ProJenX and Unlearn Partner to Enhance ALS Trial PRO-101 with Digital Twin Model

ProJenX, Inc., a biotechnology company focused on developing new treatments for amyotrophic lateral sclerosis (ALS) and other severe brain diseases, has announced a collaboration with Unlearn to enhance its Phase 1 clinical trial, PRO-101, for the drug prosetin. Prosetin is a brain-penetrant kinase inhibitor designed to target MAP4 kinase (MAP4K). This partnership aims to utilize Unlearn's advanced artificial intelligence (AI) technology to create digital twins of the clinical trial participants.

Unlearn, a leader in the field of machine learning and AI, will employ its ALS-Digital Twin Generator (ALS-DTG) to generate these digital twins. These digital twins are essentially probabilistic forecasts of the future health outcomes of the trial participants and will serve as placebos for those dosed with prosetin. The aim is to evaluate the clinical outcomes of ALSFRS-R, Slow Vital Capacity, and plasma neurofilament light over a 52-week open-label extension.

The ALS-DTG model is trained on patient-level data and is capable of generating longitudinal clinical placebo outcomes, biomarkers, and lab results from baseline data collected at the start of the trial. This approach allows for a more accurate comparison of the drug's effects by addressing critical challenges in clinical development. The digital twins are delivered through Unlearn's Platform, augmenting the evaluation process of prosetin's effectiveness in ALS patients.

Stan Abel, the president and CEO of ProJenX, remarked that this collaboration could yield valuable insights, expediting clinical trials and more efficiently evaluating prosetin's impact on ALS patients. He emphasized that the digital twins enable a direct comparison between ALS patients dosed with prosetin and their digitally generated placebo versions based on initial baseline measurements. This methodology aims to improve the interpretation of prosetin's effects, supporting a more efficient and data-driven clinical development program.

Steve Herne, CEO of Unlearn, highlighted the urgency of advancing research in ALS, given the substantial number of people affected by this debilitating disease. He expressed confidence in the collaboration's potential to support the development of effective therapies that could significantly improve patients' lives.

The PRO-101 trial is a hybrid Phase 1 clinical trial designed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in both healthy volunteers and ALS patients. The trial is divided into several parts: Parts 1a and 1b, which have already been completed, involved a randomized, double-blind, placebo-controlled, dose-escalating study to assess the safety and pharmacokinetics of single and multiple doses of prosetin in healthy volunteers. Parts 1c and 1d will focus on evaluating these parameters in ALS patients.

ProJenX, born out of a research collaboration between Project ALS and Columbia University, aims to quickly develop and commercialize prosetin for ALS patients. The company uses an innovative, patient-specific, cell-based drug discovery platform that can be applied to ALS and other severe brain diseases.

Unlearn, on the other hand, is dedicated to transforming medical research by leveraging digital twins—advanced computational models of patients—to overcome significant challenges in clinical development. Their mission is to eliminate trial and error in medicine, thereby accelerating the path to effective treatments.

Overall, the partnership between ProJenX and Unlearn represents a significant stride in the quest to find effective treatments for ALS, combining cutting-edge AI technology with innovative drug development strategies to potentially improve clinical outcomes for patients suffering from this debilitating disease.