Dosing Begins in Phase 1 Trial of Prosetin

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by Teresa Carvalho |

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A Phase 1 clinical trial investigating ProJenX‘s oral therapy prosetin in healthy volunteers and people with amyotrophic lateral sclerosis (ALS) has started dosing participants.

The first-in-human PRO-101 trial is divided into three parts. In parts 1a and 1b, researchers will investigate the safety, tolerability, and pharmacokinetics of single and ascending doses of prosetin versus a placebo in healthy volunteers. Pharmacokinetics refers to the movement of a drug into, through, and out of the body.

After those parts are completed, Part 1c will evaluate the same measures in people with ALS. According to an August webinar by Project ALS, this portion of the trial will take place at Columbia University Medical Center, in New York.

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The study’s overall goal is to determine the optimal dose of prosetin and acquire biomarker data that helps inform the design of late-stage trials.

ProJenX was created recently as part of a long-term collaboration between Project ALS and researchers at Columbia University, to accelerate the development and commercialization of treatments for brain diseases, starting with prosetin.

“Today marks an important first for the Project ALS family. We are confident that the ProJenX team will take prosetin to the next level clinically with the determination, spirit, and scientific rigor that marks Project ALS research,” Valerie Estess said in a press release. Estess is Project ALS co-founder and director of research, and ProJenX co-founder and board director.

“We are excited to advance prosetin, a potential first-in-class treatment for ALS and related neurodegenerative diseases, into the clinic,” said Erin Fleming, co-founder and senior director of operations at ProJenX.

“PRO-101 will provide essential safety, pharmacokinetic, and biomarker data about prosetin to inform our decisions on future clinical testing, including potential registrational studies,” Fleming added.

ALS is charaterized by the abnormal folding of certain proteins, which accumulate and form 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 leads to nerve cell damage and death.

Prosetin is an oral therapy designed to enter the brain and reverse ER stress by blocking a protein called mitogen-activated protein kinase kinase kinase kinase (MAP4K).

It was developed by scientists at Project ALS and Columbia University, who showed that prosetin potently inhibited MAP4K in an animal model of ALS, reduced inflammation, and improved nerve cell survival.

“Soon after my ALS diagnosis, I learned about prosetin, and was energized by the promising results of this new drug candidate. I admire the transparency and urgency with which prosetin has been developed. Going to clinical trial is an exciting milestone for prosetin and a true beacon of hope for the ALS community,” said Michele Stellato, prosetin community advisory board member.

Prosetin was designated an orphan drug by the U.S. Food and Drug Administration in 2020 for the treatment of ALS. This designation provides a number of financial incentives, including tax waivers and seven years of marketing exclusivity upon approval.