ProJenX Receives Health Canada Clinical Trial Application Authorization for First Study of Prosetin in People Living with ALS

NEW YORK, Nov. 29, 2023 /PRNewswire/ -- ProJenX, 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 authorization of its clinical trial application (CTA) by Health Canada for study PRO-101, a global, hybrid Phase 1 clinical trial evaluating prosetin—a first-in-class MAP4K inhibitor—in healthy volunteers and participants with ALS. The Health Canada CTA authorization supports initiation of Part 1c of PRO-101, designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in participants with ALS.

"Today's announcement is an important milestone for the prosetin program and our ProJenX community," said Erin Fleming, Co-Founder and Chief Operating Officer at ProJenX. "Following Health Canada's CTA authorization, we will be able for the first time to evaluate prosetin in people living with ALS. We are encouraged by the safety and tolerability profile that has emerged from completed healthy volunteer studies of prosetin, and we are laser-focused on initiating ALS patient enrollment in Canada as quickly as possible."

Prosetin is a selective, oral, brain-penetrant, MAP4K inhibitor developed by ProJenX co-founders at Columbia University for the treatment of ALS. Following the discovery that MAP4K inhibition confers potent motor neuron protection across multiple patient stem cell-derived models of ALS, prosetin was optimized for potency against MAP4Ks, efficacy in motor neuron rescue, and preferential distribution to the CNS.

"Despite scientific advances, ALS remains a fatal, devastating neurodegenerative disease that demands more meaningful treatment options," said Angela Genge, MD, FRCP(C), eMBA, Associate Professor, Department of Neurology and Neurosurgery at McGill University, Director of the ALS Centre of Excellence for Research and Patient Care at the Montreal Neurological Institute (The Neuro), and PRO-101 study investigator. "ProJenX has generated compelling preclinical data showing that prosetin may protect motor neurons against well-established pathological mechanisms in ALS, and I look forward to working with their team on the first-ever study evaluating its activity in ALS patients."

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. 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.

SOURCE ProJenX