ProJenX wins $1M ALS Association grant to test prosetin in patients
Award supports 3rd part of Phase 1 trial testing the therapy in people with ALS
Biotechnology company ProJenX has received a $1 million grant from the ALS Association to support a portion of an ongoing clinical trial, which will test prosetin in people with amyotrophic lateral sclerosis (ALS).
The PRO-101 Phase 1 trial (NCT05279755) is testing prosetin in three parts. The first two included healthy volunteers and showed that the medication, given as single and multiple ascending doses, was generally safe and well tolerated.
The third part will now include ALS patients across the U.S., Canada, and Europe, with enrollment expected to begin in the coming months, according to ProJenX.
“By funding Part 1c of the PRO-101 trial, we are pleased to support the first dosing of prosetin in people living with ALS,” said Kuldip Dave, PhD, senior vice president of research at the ALS Association, in a press release.
The recent funding was given under the Hoffman ALS Clinical Trial Awards Program, created to support early clinical trials of investigational therapies for ALS. These trials are usually designed to produce data on treatment safety, dosing, and disease biomarkers.
“We are honored that the ALS Association recognizes the promise of prosetin and has selected ProJenX for a Hoffman Clinical Trial Award,” said Erin Fleming, ProJenX’s co-founder and chief operating officer. “Their support enables the rapid initiation of the first clinical study of prosetin in individuals living with ALS, which is a significant milestone for prosetin, ProJenX, and the broader ALS community that has supported this program since inception.”
Early work showed potential of prosetin in ALS
ALS is characterized by the abnormal folding of certain proteins, which clump together into toxic structures inside nerve cells.
While cells have some ability to reduce these abnormal proteins through the endoplasmic reticulum (ER) — a cellular compartment involved in protein production, folding, and modification — too many of these proteins can cause ER stress, which can contribute to nerve cell damage and death.
Prosetin is a first-in-class oral therapy designed to reduce ER stress and reduce damage in nerve cells by blocking a protein called mitogen-activated protein kinase kinase kinase kinase (MAP4K).
It was developed by ProJenX co-founders at Columbia University in collaboration with Project ALS, which demonstrated the therapy improved nerve cell survival in patient-derived cells and animal models.
“Decades of collaborative preclinical research funded by Project ALS at Columbia University pinpointed core mechanisms of ALS and resulted in the development of prosetin to counter ALS pathology across multiple forms of the disease,” said Jinsy Andrews, MD, ProJenX’s clinical advisor and director of neuromuscular clinical trials at Columbia University.
“I am excited to work with ProJenX to initiate Part 1c of the PRO-101 clinical trial, which will evaluate safety across multiple doses of prosetin and also explore biomarkers indicative of prosetin’s potential impact on ALS disease progression,” Andrews added.
Following the completion of Part 1c, patients may enroll in an open-label extension, where all will receive prosetin for at least one year. This study will assess long-term benefits of prosetin in biomarkers of disease progression, neuronal degeneration, and brain inflammation.
Prosetin was granted orphan drug designation by the U.S. Food and Drug Administration for the treatment of ALS. The designation is intended to accelerate the development of therapies for rare diseases and provides certain incentives to companies, including tax waivers and seven years of marketing exclusivity if the therapy is ultimately approved.
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