ProJenX Launches, Will Develop ALS Therapy Prosetin

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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A company called ProJenX has been launched and will focus on the clinical development of treatments for brain diseases, starting with prosetin, its lead candidate therapy for amyotrophic lateral sclerosis (ALS).

ProJenX was created in collaboration with Project ALS, researchers at Columbia University, and Medical Excellence Capital, which invested $5.1 million in seed funding for the company.

“Since 1998, Project ALS has led aggressive, collaborative research to unlock the mysteries of ALS and the brain — the body’s most complex organ. We are immensely excited that ProJenX will take on the critical task of translating these hard-won discoveries to people with ALS — beginning with prosetin,” Valerie Estess said in a press release. Estess is the co-founder and director of research at Project ALS and ProJenX’s co-founder.

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ALS is characterized by the toxic buildup of misfolded proteins in nerve cells. These proteins accumulate in the endoplasmic reticulum (ER) — a cell compartment involved in protein folding — that can cause excessive ER stress and cellular dysfunction.

Prosetin is an oral therapy developed by scientists at Project ALS and Columbia University. It was designed to enter the brain and reverse ER stress through the inhibition of a protein called MAP4 kinase (MAP4K).

When tested in an ALS mouse model, the therapy showed an ability to enter the brain where it potently inhibited MAP4K, reduced inflammation, and improved the survival of motor neurons.

“Prosetin was developed out of a decades-long collaboration between the labs of Hynek Wichterle and Brent R. Stockwell at Columbia University and Project ALS to build more predictive models of ALS — and then use them to identify better treatments. We have seen promising effects of prosetin in a range of laboratory models and are hopeful for its potential to improve the lives of many people with ALS and other devastating neurological conditions,” said Erin Fleming, co-founder and senior director of operations at ProJenX.

The promising data led the U.S. Food and Drug Administration to grant prosetin orphan drug designation in 2020, which is intended to speed progress toward the development of therapies for rare diseases.

Researchers first identified MAP4 kinases as a promising therapeutic target using a screening platform that tested the ability of several compounds to prevent ER stress and related neurodegeneration in rat and human neurons. They found that most of the successful compounds targeted MAP4Ks.

ProJenX believes its screening platform eventually can be used to identify therapies for other brain diseases.

“Medical Excellence is delighted to have played a central role in the formation of ProJenX. Patients with ALS desperately need new therapies. We believe that the patient-specific, cell-based discovery platform at the heart of ProJenX’s approach is an exciting opportunity for the creation of transformative neuroscience medicines,” said Eric Heil, interim CEO of ProJenX and a managing partner at Medical Excellence Capital.

ProJenX is named in honor of Project ALS co-founder, Jenifer Estess, who was diagnosed with ALS at age 35.