ProJenX Establishes Clinical Advisory Board to Advance Prosetin in ALS
Key Insights
ProJenX (search) has formed a Clinical Advisory Board (ClAB) comprising experts in ALS (search) clinical trial design to guide the development of prosetin.
The ClAB will provide strategic guidance for Study PRO-101, a Phase 1 trial evaluating prosetin's safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers and ALS (search) patients.
Prosetin, a novel brain-penetrant MAP4 kinase (search) inhibitor, targets endoplasmic reticulum (ER) stress, a key factor in ALS (search) pathogenesis.
ProJenX (search), a biotechnology company focused on neurodegenerative diseases, particularly amyotrophic lateral sclerosis (search) (ALS (search)), has announced the formation of its Clinical Advisory Board (ClAB). This board comprises internationally recognized experts in ALS clinical trial strategy and design, and will provide guidance for the clinical development of ProJenX's lead therapeutic candidate, prosetin, and other pipeline programs.
Clinical Advisory Board Composition
The ClAB is chaired by Angela Genge, MD, FRCP(C), eMBA (McGill University), Director of the ALS (search) Global Centre of Excellence at the Montreal Neurological Institute and Professor of Neurology at McGill University. Other members include Jinsy Andrews, MD, MSc (Columbia University), Leonard van den Berg, MD, PhD (UMC Utrecht), and Merit Cudkowicz, MD, MSc (Massachusetts General Hospital).
Prosetin: A Novel Therapeutic Approach
Prosetin is a potent, oral, brain-penetrant, mitogen-activated protein kinase (MAP4K (search)) inhibitor targeting endoplasmic reticulum (ER) stress. ER stress is implicated in both sporadic and familial forms of ALS (search). MAP4Ks have been identified as critical regulators of ER stress-mediated motor neuron loss through a patient-specific, cell-based discovery platform developed by researchers at Columbia University.
Study PRO-101: Phase 1 Clinical Trial
Prosetin is currently under evaluation in Study PRO-101, a hybrid Phase 1 clinical trial designed to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics in both healthy volunteers and participants with ALS (search). Parts A and B of the study, which involved single and multiple ascending doses of prosetin in healthy volunteers, have been completed. The data from 48 healthy volunteers demonstrated positive safety, tolerability, and pharmacokinetic profiles, supporting the continued exploration of prosetin in ALS patients.
Expert Perspectives
Dr. Genge stated, "Improved clinical outcomes for ALS (search) 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."
Erin Fleming, Co-Founder and Chief Operating Officer at ProJenX (search), added, "With our advisors' leadership, ProJenX is poised to translate decades of scientific discovery to people with ALS (search)."