NEW YORK, March 1, 2022 /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 initiation of dosing for PRO-101, a Phase 1 clinical study of prosetin in healthy volunteers and people with ALS.

“We are excited to advance prosetin, a potential first-in-class treatment for ALS and related neurodegenerative diseases, into the clinic,” said Erin Fleming, Co-Founder and Senior Director of Operations at ProJenX. “PRO-101 will provide essential safety, pharmacokinetic, and biomarker data about prosetin to inform our decisions on future clinical testing, including potential registrational studies.”

PRO-101, the first-in-human study of prosetin, is a three-part, hybrid Phase 1 trial. Parts 1a and 1b will evaluate safety, tolerability, and pharmacokinetics of prosetin in healthy volunteers, and Part 1c is planned to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in people with ALS. Designed after consulting the Food and Drug Administration (FDA), leading ALS clinicians, and ALS community advisors, PRO-101 aims to efficiently establish the safety and biomarker data necessary for rational dose selection and late-phase study design. Prosetin Community Advisory Board member Michele Stellato said, “Soon after my ALS diagnosis, I learned about prosetin, and was energized by the promising results of this new drug candidate. I admire the transparency and urgency with which prosetin has been developed. Going to clinical trial is an exciting milestone for prosetin and a true beacon of hope for the ALS community.”

Prosetin is a potent, oral, brain-penetrant, mitogen-activated protein kinase kinase kinase 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. Prosetin is an investigational new drug and has not been approved by the FDA.

Preclinical development of prosetin was funded by the nonprofit organization Project ALS. Valerie Estess, Project ALS Co-Founder and Director of Research, and ProJenX Co-Founder and Board Director, commented, “Today marks an important first for the Project ALS family. We are confident that the ProJenX team will take prosetin to the next level clinically with the determination, spirit, and scientific rigor that marks Project ALS research.”

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 consist 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. Following analysis of data from these parts, Part 1c is planned to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in ALS patients.

For more information, contact [email protected].

About ProJenX

ProJenX is a clinical-stage biotechnology company with novel, brain-penetrant, targeted therapies to address debilitating 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 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.