Long-term clinical data continue to support favorable safety and tolerability; new preclinical data show that prosetin corrects TDP-43-related dysfunction in vitro

NEW YORK, Oct. 27, 2026 /PRNewswire/ — ProJenX, a clinical-stage biotechnology company developing novel, targeted, brain-penetrant therapies for the treatment of amyotrophic lateral sclerosis (ALS) and other debilitating brain diseases, today announced it will present updates on the prosetin development program at the 2026 Network for Excellence in ALS (NEALS) Annual Meeting, taking place Oct 27 – 30 in Clearwater, FL and online. Prosetin is a potent, selective, CNS-penetrant MAP4K inhibitor currently being evaluated in participants with ALS in the Phase 1b study PRO-101.

In addition to sharing long-term clinical data from PRO-101, presentations will highlight preclinical characterization of prosetin and the MAP4K mechanism, including new data demonstrating that prosetin broadly corrects TDP-43-related mis-splicing in human stem cell-derived motor neurons. ProJenX will also present an update on the critical role of its Community Advisory Board across the prosetin development program.

“The body of data we’ve generated strengthens the case for MAP4K as an important target in ALS and related neurodegenerative diseases,” said Stan Abel, Chief Executive Officer of ProJenX. “In PRO-101, we reached target therapeutic exposures, saw target engagement that tracks with exposure, and maintained a favorable safety profile over long-term dosing. Together, these results show for the first time that CNS-penetrant MAP4K inhibition is a viable clinical strategy. We’re grateful to the participants, investigators, and community advisors who made this possible.”

Presentation Details

Wednesday, October 28
Poster Presentation, 4:50 pm ET, Poster #56
Preclinical characterization of prosetin, a novel, CNS-penetrant MAP4K inhibitor developed for the treatment of ALS

Thursday, October 29
Oral Presentation, 9:50 am ET
Session: A Collaborative Ecosystem for Clinical Trial Innovation
Early Incorporation of Lived Experience in the Prosetin Development Program

Poster Presentation, 4:50 pm ET, Poster #207
Results from PRO-101, a Phase 1b study evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in participants with ALS

ABOUT PROSETIN

Prosetin is a potent, selective, CNS-penetrant, orally available MAP4K inhibitor under evaluation for the potential treatment of ALS. While MAP4K was initially identified as a critical regulator of proteotoxic stress-mediated motor neuron loss in a human stem cell-based discovery platform developed by ProJenX scientific founders at Columbia University, more recent internal and independent data have demonstrated that MAP4K inhibition confers broad neuroprotective effects in diverse preclinical ALS models, including significant rescue of TDP-43 pathology in vitro. Prosetin is an investigational new drug and has not been approved by the FDA.

ABOUT PROJENX

ProJenX is a clinical-stage biotechnology company developing 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 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 neurodegenerative diseases. For more information, visit projenx.com.

View original content to download multimedia:https://www.prnewswire.com/news-releases/projenx-to-present-data-update-for-the-prosetin-development-program-at-the-2026-neals-annual-meeting-302920232.html

SOURCE ProJenX