RARE Daily

ALS Association Gives ProJenX $1 Million Clinical Trial Award

June 20, 2024

Rare Daily Staff

ProJenX, the biotech created out of a collaboration between Columbia University and Project ALS, said it received a $1 million Hoffman ALS Clinical Trials Award from the ALS Association to help fund the development of prosetin, its experimental therapy for the rare and fatal neurodegenerative condition.

Prosetin is a novel, brain-penetrant, MAP4 kinase inhibitor, which is currently in the Part 1c portion of the PRO-101 clinical trial in participants living with ALS.

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.
Following the Part 1c, a long-term open label extension of 12 or more months will occur, allowing observation of the long-term behavior of prosetin in ALS patients, measured via biomarkers of disease progression, neurodegeneration, and neuroinflammation in plasma.

The ALS Association’s Hoffman Clinical Trial Awards Program supports early-stage clinical trials of new treatments that hold promise for those living with ALS.

“By funding programs at this critical stage, we hope to accelerate the development of therapeutic candidates that can help make ALS a livable disease,” said Kuldip Dave, senior vice president of research at the ALS Association.

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