A New Approach to Treating ALS

ProJenX is on a mission to make prosetin, a scientifically-validated, first-in-class MAP4 kinase inhibitor, available to people with ALS

About ProJenX

ProJenX is a clinical-stage biotechnology company with novel, brain penetrant therapies to target biologically-defined pathways for the treatment of debilitating brain diseases, with an initial focus on amyotrophic lateral sclerosis (ALS).

ProJenX was created out of a long-term research collaboration between Project ALS and Columbia University in order to rapidly develop and commercialize its lead therapeutic 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 to identify targeted, small molecule therapies for ALS and other currently untreatable brain diseases.

Prosetin: Changing the Trajectory of ALS

Prosetin is the first brain-penetrant MAP4K inhibitor therapy to enter clinical trials. ProJenX scientific co-founders at Columbia University have shown that prosetin is highly effective across multiple laboratory models of ALS.

What sets prosetin apart?

Penetrating the
Blood-Brain Barrier

Even though ALS is a brain disease, most drugs developed for ALS cannot cross the “blood-brain barrier” to reach motor neurons. We developed prosetin to preferentially reach the brain and target motor neurons directly.

Leveraging
ALS in a Dish

Prosetin’s inventors at Columbia University were the first to identify how to make a motor neuron from a skin or blood sample from someone with ALS. Their world-leading research means that prosetin was discovered using patient-derived laboratory models, and developed specifically to treat ALS.

Blocking
MAP4K

While ALS is a complicated, heterogeneous disease, endoplasmic reticulum (ER) stress is a well-established common feature across ALS subtypes. By blocking MAP4K, we have shown that prosetin can rescue ER stress-mediated motor neuron loss in ALS models.

Pipeline Development:

Novel, brain-penetrant kinase inhibitors

Developing prosetin for people with ALS is our top priority. At the same time, we are exploring the role of MAP4K inhibition in treating other neurodegenerative disorders that trigger endoplasmic reticulum (ER) stress-mediated degeneration of neurons, and in other debilitating brain diseases in which this pathway play an established biological role.

Target	Candidate	Indication
		ALS
MAP4K	Prosetin	Parkinson’s disease
		Glioblastoma
		Alzheimer’s disease
Undisclosed	Undisclosed	ALS

A Deeply Personal Mission

ProJenX spun out of a long-term collaboration between the ALS research nonprofit Project ALS and researchers at Columbia University, who have worked together for over twenty years toward a shared goal of developing the first effective treatments, and ultimately a cure, for ALS.

Project ALS, founded in 1998 by Jenifer, Meredith, and Valerie Estess after Jenifer’s ALS diagnosis at 35, began with a powerful call to arms: I do not find this acceptable. There were no treatments for ALS, and no dedicated research efforts to understand this complex brain disease. Project ALS, with research partners at Columbia and beyond, set out to change this paradigm.

ProJenX, named for Jenifer, was launched in this same spirit. We have come this far guided by scientific rigor, by powerful advocacy, and most of all, by love—and we will not stop until people diagnosed with ALS have access to meaningful treatments.