ProJenX receives $15M in funding to further develop prosetin for ALS

Ongoing Phase 1 clinical trial to test prosetin in ALS patients in early 2024

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by Mary Chapman |

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The biotechnology company ProJenX recently received $15 million in funding to advance its experimental oral treatment prosetin for amyotrophic lateral sclerosis (ALS).

This Series A financing round is essentially the next round of funding after seed money was used to rapidly develop the investigational treatment.

Both rounds were led by Medical Excellence Capital, which created ProJenX last year in collaboration with Columbia University researchers and Project ALS, an organization that identifies and funds research for potential ALS therapies.

“We are focused on accelerating the development of prosetin to meet the critical need for new treatments for people living with ALS,” Stan Abel, ProJenX president and CEO, said in a company press release.

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Oral prosetin designed to reach brain and reverse stress in nerve cells

In ALS, certain proteins abnormally fold and form toxic clumps in nerve cells. This causes stress in certain cellular compartments, including in the endoplasmic reticulum (ER), a cell organelle involved in protein production, modification, and transport, and ultimately results in nerve cell damage and death.

ProJenX’s lead compound, prosetin, is a first-in-class oral therapy designed to reach the brain and reverse ER stress in nerve cells. It works by inhibiting the function of a protein called mitogen-activated protein kinase kinase kinase kinase (MAP4K).

Project ALS and Columbia University researchers developed the therapy candidate, which was found to potently block MAP4K in an ALS animal model, lower inflammation, and improve nerve cell survival.

Soon after ProJenX’s launch, a first-in-human Phase 1 clinical trial (NCT05279755) began testing prosetin in people with ALS and in healthy volunteers. In the trial’s initial two parts, researchers are assessing the safety, tolerability, and pharmacokinetics of single and ascending doses of the compound versus a placebo in healthy participants. Pharmacokinetics refers to the movement of drug into, through, and out of the body.

After those portions are completed, the same measures will be evaluated in ALS patients. The company is planning to launch this third portion in people with ALS early next year.

“We are encouraged by the therapeutic window emerging from our phase 1 trial and ongoing nonclinical studies and are looking forward to advancing prosetin to people living with ALS in early 2024,” Abel said.

The overall aim of the study is to establish the optimal prosetin dose and gather biomarker data that help inform the design of upcoming trials.

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Prosetin named orphan drug by FDA in 2020

Prosetin was granted orphan drug designation by the U.S. Food and Drug Administration for ALS in 2020. Such a designation offers a range of incentives to developers of medications for rare disorders, including seven years of market exclusivity if an approval is ultimately granted.

ProJenX also announced the board appointment of Rick Hartz, senior vice president, global pharma and human health business development at Merck (known as MSD outside North America).

“I am excited to join the ProJenX Board and contribute to its mission to advance promising therapies for ALS and other neurodegenerative diseases,” Hartz said. “With an orphan drug designation, prosetin is poised to advance toward a global pivotal trial next year and I look forward to working with my fellow board members and the ProJenX team to help guide the growth of the company.”