ProJenX, Inc.

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 or follow @UnlearnAI on Twitter/X and @unlearn-ai on LinkedIn. SOURCE ProJenX WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

NEW YORK, Aug. 27, 2024 /PRNewswire/ -- ProJenX, Inc., a clinical-stage biotechnology company developing novel, brain-penetrant therapies targeting biologically-defined pathways for the treatment of amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, today announced the formation of its Clinical Advisory Board (ClAB). The ClAB, which includes internationally renowned experts in clinical trial strategy and design for ALS and related neurodegenerative diseases, will provide strategic guidance for the clinical development of ProJenX's lead therapeutic candidate, prosetin—a novel, brain-penetrant, MAP4 kinase (MAP4K) inhibitor—and additional pipeline programs. Prosetin is being evaluated in Study PRO-101, a hybrid Phase 1 clinical trial designed to evaluate its safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers and participants with ALS. Parts A and B of the study, which assessed single and multiple ascending doses of prosetin in healthy volunteers, are complete, and positive safety, tolerability, and pharmacokinetic data from 48 healthy volunteers supports continued exploration of prosetin in ALS participants. The inaugural ClAB Chair is Angela Genge, MD, FRCP(C), eMBA (McGill University), who is joined by members Jinsy Andrews, MD, MSc (Columbia University), Leonard van den Berg, MD, PhD (UMC Utrecht), and Merit Cudkowicz, MD, MSc (Massachusetts General Hospital). Dr. Genge, Director of the ALS Global Centre of Excellence at the Montreal Neurological Institute and Professor of Neurology at McGill University, said, "Improved clinical outcomes for ALS patients will only be achieved through stewarding novel, scientifically rational investigational therapies supported by compelling and thorough preclinical data into the ALS clinical trial pipeline. Prosetin has been developed specifically for ALS and demonstrates robust neuroprotection in diverse ALS patient-derived stem cell models of the disease, and I am excited to guide Study PRO-101 as Chair of the ProJenX Clinical Advisory Board, as well as Principal Investigator of one of the trial study sites." Erin Fleming, Co-Founder and Chief Operating Officer at ProJenX, said, "As we initiate the first-ever clinical study of prosetin in people living with ALS, we are thrilled to convene a world-class Clinical Advisory Board, which will bring unrivaled experience and knowledge across ALS drug development and data-driven clinical trial design to the prosetin development program. With our advisors' leadership, ProJenX is poised to translate decades of scientific discovery to people with ALS." ProJenX's ClAB member biographies can be viewed here. About ProJenX ProJenX is a clinical-stage biotechnology company developing novel, brain-penetrant, targeted therapies to address neurodegenerative 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 Prosetin Prosetin is a potent, oral, brain-penetrant, mitogen-activated protein kinase (MAP4K) inhibitor targeting endoplasmic reticulum (ER) stress. ER stress is a common feature across sporadic and familial forms of ALS, and MAP4Ks emerged as the critical regulators of ER stress-mediated motor neuron loss in a patient-specific, cell-based discovery platform developed by researchers at Columbia University. ProJenX is currently conducting a three-part Phase 1 clinical trial, PRO-101, investigating prosetin in healthy individuals and people living with ALS. Prosetin is an investigational new drug and has not been approved by the FDA. SOURCE ProJenX

Funding will support the first study of prosetin in people living with ALS NEW YORK, June 20, 2024 /PRNewswire/ -- ProJenX, Inc., a clinical-stage biotechnology company developing novel, brain-penetrant therapies targeting biologically-defined pathways for the treatment of amyotrophic lateral sclerosis (ALS) and other debilitating brain diseases, today announced the receipt of a $1M Hoffman ALS Clinical Trials Award from the ALS Association. The grant will help fund the development of prosetin, a novel, brain-penetrant, MAP4 kinase (MAP4K) inhibitor, in the Part 1c portion of the PRO-101 clinical trial of prosetin in participants living with ALS. PRO-101 evaluates the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin. ProJenX has completed the Part 1a and Part 1b portions of PRO-101 in healthy volunteers. Part 1c of the PRO-101 clinical trial is expected to initiate enrollment in Q3 2024. "We are honored that the ALS Association recognizes the promise of prosetin and has selected ProJenX for a Hoffman Clinical Trial Award," said Erin Fleming, Co-Founder and Chief Operating Officer at ProJenX. "Their support enables the rapid initiation of the first clinical study of prosetin in individuals living with ALS, which is a significant milestone for prosetin, ProJenX, and the broader ALS community that has supported this program since inception." "The ALS Association's Hoffman Clinical Trial Awards Program supports early-stage clinical trials of new treatments that hold promise for those living with ALS," said Kuldip Dave, Senior Vice President of Research at the ALS Association. "By funding Part 1c of the PRO-101 trial, we are pleased to support the first dosing of prosetin in people living with ALS. By funding programs at this critical stage, we hope to accelerate the development of therapeutic candidates that can help make ALS a livable disease." "Decades of collaborative preclinical research funded by Project ALS at Columbia University pinpointed core mechanisms of ALS and resulted in the development of prosetin to counter ALS pathology across multiple forms of the disease," said Jinsy Andrews, MD, MSc, clinical advisor to ProJenX and Director of Neuromuscular Clinical Trials at the Columbia University Irving Medical Center. "I am excited to work with ProJenX to initiate Part 1c of the PRO-101 clinical trial, which will evaluate safety across multiple doses of prosetin and also explore biomarkers indicative of prosetin's potential impact on ALS disease progression." About PRO-101 PRO-101 is a three-part 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. Based on favorable safety, tolerability, and pharmacokinetic data from this portion of the study, Part 1c will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in ALS patients. Following the Part 1c, a long-term Open Label Extension (OLE) of 12 or more months will occur, allowing observation of the long-term behavior of prosetin in ALS patients, measured via biomarkers of disease progression, neurodegeneration, and neuroinflammation in plasma. For more information about PRO-101, contact [email protected]. About the ALS Association The ALS Association is the largest philanthropic funder of ALS research in the world. The Association funds global research collaborations, assists people with ALS and their families through its nationwide network of care and certified clinical care centers, and advocates for better public policies for people with ALS. The ALS Association is working to make ALS a livable disease while urgently searching for new treatments and a cure. For more information about The ALS Association, visit our website at als.org. 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. SOURCE ProJenX