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ALS Therapy Start-Up Gains $5.1M in Seed Funds

Neurons

(commonfund.nih.gov)

16 Feb. 2022. A start-up enterprise is developing a treatment for amyotrophic lateral sclerosis or ALS, spun off from university research labs and an ALS advocacy group. The new company, ProJenX in New York, is raising $5.1 million in seed funds to advance work begun by neuroscience researchers at Columbia University and the organization Project ALS.

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder where neurons or nerve cells controlling muscles in the body begin to waste away, and can no longer send or receive signals from the brain or spinal cord. As the nerve cells stop functioning, muscles in the limbs, and later speech and breathing muscles, begin weakening and eventually stop functioning. Most people with the disease die of respiratory failure. According to Johns Hopkins University, ALS affects some 30,000 people in the U.S., with 5,000 new cases reported each year.

ProJenX seeks to advance a treatment candidate for ALS called prosetin into clinical trials. Prosetin is an optimized small-molecule compound discovered by researchers from Columbia University’s Project ALS Therapeutics Core, a joint initiative with Project ALS that advocates and raises money for research on ALS. In a paper published in Oct. 2019, researchers from Columbia and Project ALS identified prosetin as a synthesized compound that blocks actions of damaging enzymes known as mitogen-activated protein or MAP4 kinases. By blocking MAP4 kinases, prosetin reverses endoplasmic reticulum stress, pressure exerted on cells causing misfolded proteins, which in nerve cells can build-up and become toxic.

Granted orphan drug status

The Columbia/Project ALS team tested prosetin in lab mice, and found the compound penetrates the blood-brain barrier to block MAP4 kinases and protect nerve cells. In Aug. 2020, Food and Drug Administration designated prosetin as an orphan drug, which provides for extended market exclusivity, tax credits for clinical trials, and waiver of user fees for FDA review. In Aug. 2021, Project ALS announced plans for an early-stage clinical trial of prosetin, testing primarily for safety and optimal dose.

Erin Fleming, ProJenX co-founder and operations director says in a company statement released through Cision, “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.”

Medical Excellence Capital is providing ProJenX with $5.1 million in seed funds as well as its managing partner Eric Heil to serve as interim CEO. Medical Excellence is an early-stage life sciences venture investor that formed and incubated ProJenX. With the funds, ProJenX plans to start clinical studies of prosetin, hire more executives, and support preclinical work on other therapies.

“Patients with ALS desperately need new therapies,” notes Heil. “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.”

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