First Patient Dosed in Phase 1 Trial of Protollin for Early Alzheimer’s
I-Mab Biopharma announced that the first patient has been dosed in the U.S.-based Phase 1 trial of its intranasal immunotherapy agent Protollin — an investigational medication being developed to treat those with early Alzheimer’s disease.
The U.S. Food and Drug Administration approved the company’s request for this safety and early efficacy study in July.
“This is an important milestone in the quest to develop novel treatments for patients suffering from Alzheimer’s disease and we look forward to continuing to support this global effort to develop novel therapies,” Jingwu Zang, MD, PhD, founder, chairman, and director of I-Mab, said in a press release.
Protollin is made up of proteins derived from the outer membranes of certain bacteria. It is designed to stimulate the innate immune system — the body’s first line of defense — to potentially clear amyloid-beta plaques and tau protein tangles, two hallmarks of Alzheimer’s disease. By clearing such clumps, the therapy could prevent or slow disease progression.
It is delivered as a nasal spray that enables it to reach the brain, where it is expected to mount an immune response.
Preclinical studies in mouse models have demonstrated that nasal administration of Protollin activated microglia — immune cells found in the brain — and prevented the accumulation of amyloid-beta plaques in young mice. Protollin also was shown to decrease amyloid-beta accumulation and improve memory in an aged mouse model of Alzheimer’s.
The Phase 1 trial, co-sponsored by I-Mab and Jiangsu Nhwa Pharmaceutical, is being conducted by Brigham and Women’s Hospital in Boston. It is evaluating the safety, tolerability, and immune effects of Protollin in patients, ages 60-85, with early to mild symptomatic Alzheimer’s and being treated at the Boston hospital. More information can be found here.
“We are pleased to be working with Brigham and Women’s Hospital and our other partners to advance Protollin into the clinic,” Zang said.
The trial’s single-ascending dose design — two doses given each participant one week apart — will allow the researchers to determine the highest dose of Protollin that maintains its safety and potential efficacy. Evidence of treatment effectiveness will be measured through patients’ immune response, including its effects on white blood cells and through examinations of cell surface markers, Brigham and Women’s Hospital reported in a separate release.
The Boston hospital and Inspirevax (formerly Biodextris) initially worked to develop Protollin. They granted I-Mab and Nhwa exclusive licenses to further develop, manufacture and, if approved, commercialize the therapy.
I-Mab will develop and commercialize Protollin outside of the Greater China area, which includes mainland China, Hong Kong, Macau, and Taiwan, while Nhwa will develop and commercialize the medication within those areas.
“If clinical trials in humans show that Protollin is safe and effective, this could represent a novel therapy for people with Alzheimer’s, and it could also be given early to help prevent Alzheimer’s disease in people at risk,” said Howard L. Weiner, MD, co-director of the Ann Romney Center for Neurologic Diseases at the Brigham hospital.