NEW YORK, NY — 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 that it has received authorization in the European Union (EU) for study PRO-101, a hybrid Phase 1 clinical trial evaluating prosetin—a first-in-class MAP4K inhibitor—in healthy volunteers and participants with ALS. Following recent Health Canada authorization of this study, the European authorization will expand the global footprint of Part 1c of PRO-101, designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in participants with ALS.
“This EU clinical trial authorization will allow us to expand the first study of prosetin for people living with ALS to European participants,” said Erin Fleming, Co-Founder and Chief Operating Officer at ProJenX. “We are committed to initiating ALS patient enrollment 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.
“ALS is a devastating disease, and there is an immense unmet need for therapeutic options to slow or halt its rapid progression,” said Leonard H. van den Berg, MD, PhD, Professor of Neurology at UMC Utrecht, Chairman of the Treatment Research Initiative to Cure ALS (TRICALS), and PRO-101 study investigator. “MAP4K inhibition has been shown to protect motor neurons from endoplasmic reticulum stress and other well-established pathological mechanisms of ALS, and we are excited to partner with ProJenX on study PRO-101, the first clinical trial to explore the potential benefits of MAP4K inhibition for 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.
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.
Contact:
Margot Shanahan
(917) 423-6476
[email protected]