ProJenX Receives $1M Hoffman ALS Trial Award from ALS Association

ProJenX, Inc., a biotechnology firm at the forefront of developing brain-penetrant treatments for amyotrophic lateral sclerosis (ALS) and other severe neurological disorders, has announced the receipt of a significant grant. The ALS Association has awarded ProJenX a $1 million Hoffman ALS Clinical Trials Award, which will support the development of prosetin, a novel MAP4 kinase (MAP4K) inhibitor. This funding is specifically allocated for Part 1c of the PRO-101 clinical trial, focusing on patients living with ALS.

The PRO-101 trial is designed to assess prosetin's safety, tolerability, pharmacokinetics, and pharmacodynamics. To date, ProJenX has successfully completed Parts 1a and 1b, which involved healthy volunteers undergoing a randomized, double-blind, placebo-controlled study to evaluate single and multiple doses of prosetin. The favorable results from these initial phases have paved the way for the upcoming Part 1c, which is expected to commence in the third quarter of 2024 and will involve ALS patients.

Erin Fleming, Co-Founder and Chief Operating Officer of ProJenX, expressed gratitude for the ALS Association's recognition and support. "Their endorsement allows us to initiate the first clinical study of prosetin in ALS patients swiftly. This is a significant milestone for prosetin, ProJenX, and the broader ALS community," Fleming stated.

Kuldip Dave, Senior Vice President of Research at the ALS Association, emphasized the importance of funding early-stage clinical trials. "By supporting Part 1c of the PRO-101 trial, we are facilitating the first dosing of prosetin in ALS patients. Our goal is to accelerate the development of therapies that can transform ALS into a manageable condition," Dave noted.

Jinsy Andrews, MD, MSc, clinical advisor to ProJenX and Director of Neuromuscular Clinical Trials at Columbia University Irving Medical Center, highlighted the collaborative preclinical research that led to prosetin's development. "Years of research funded by Project ALS at Columbia University identified key ALS mechanisms, culminating in the creation of prosetin. I am eager to work with ProJenX on Part 1c of the PRO-101 trial, which will explore the safety of multiple doses and potential biomarkers for prosetin’s impact on ALS progression," Andrews explained.

The upcoming Part 1c of the PRO-101 trial will delve into the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in ALS patients. Following this phase, a long-term Open Label Extension (OLE) will be conducted over 12 or more months. This extension will allow for the observation of prosetin's long-term effects on ALS patients, using biomarkers to measure disease progression, neurodegeneration, and neuroinflammation in plasma.

The ALS Association, the world's leading philanthropic funder of ALS research, is dedicated to supporting global collaborations, providing care through a nationwide network, and advocating for better policies for ALS patients. The association aims to make ALS a livable disease while continuing the urgent search for new treatments and a cure.

ProJenX, emerging from a collaboration between Project ALS and Columbia University researchers, focuses on developing targeted, brain-penetrant therapies for ALS and other brain diseases. The company employs an innovative, patient-specific, cell-based drug discovery platform to advance research and drug development for debilitating neurological conditions.

As ProJenX embarks on this crucial phase of clinical trials, the support from the ALS Association signifies a hopeful step forward in the quest to find effective treatments for ALS. The outcome of these trials could potentially transform the lives of those living with this debilitating disease.