Cambridge, Mass. — PhenoNet, Inc., a clinical-stage biotechnology company developing therapies for neurodegenerative diseases, said it has completed recruitment and randomization in a Phase IIb clinical trial evaluating PHENOGENE-1A as an adjunctive treatment for patients with mild-to-moderate amyotrophic lateral sclerosis, or ALS.
The company completed enrollment on Aug. 27, moving the study closer to topline results. The randomized, double-blind, placebo-controlled, multi-dose trial is being conducted at clinical sites across the United States and Europe. The first participant completed the treatment period on July 2.
PHENOGENE-1A is an investigational therapy that combines cromolyn sodium with a targeted drug-delivery platform designed to increase bioavailability and achieve therapeutically relevant concentrations in the blood, brain and cerebrospinal fluid.
The therapy is being developed as a potential disease-modifying treatment intended to complement existing standards of care and slow ALS progression. PhenoNet said preclinical studies and an earlier clinical cohort supported advancing the candidate into the Phase IIb study.
“Addressing both the triggers and progression of ALS through a multifunctional therapeutic approach may offer a new strategy for modifying the course of the disease,” said David R. Elmaleh, Ph.D., Founder and Chairman of PhenoNet. “Early intervention, beginning with the first clinical signs of muscle function decline, may be critical for delaying disease progression in ALS. Our findings from in vitro studies, in vivo ALS animal models, and a Phase IIa cohort supported advancing PHENOGENE-1A to this Phase IIb trial. We are pleased to have completed recruitment and randomization and to have achieved the important milestone of the first participant completing the treatment period.”
Atul Gupta, M.D., Chief Medical Officer of PhenoNet and medical and safety monitor for the clinical program, said no safety findings have been reported to date that required changes to the study protocol or conduct.
“Disease-modifying therapies that can be administered alongside the standard of care and that address the underlying pathophysiology of ALS could add significant value to current treatment options,” Gupta said. “To date, no safety findings have been reported that resulted in a change to the protocol or the conduct of the study. ALS remains one of the most devastating diagnoses in neurology, with limited options that address the underlying disease. A therapy that safely slows progression would be transformative for patients and the people who care for them.”
Professor Björn Oskarsson, M.D., a neurologist and ALS specialist at Mayo Clinic in Florida and Principal Investigator of the study, said the trial could contribute additional clinical information to the ALS treatment landscape. The study’s Safety Review Committee will continue evaluating accumulating safety data.
PhenoNet said that if the Phase IIb results are supportive, it plans to discuss a potential registrational development pathway with the U.S. Food and Drug Administration, including possible use of the 505(b)(2) pathway, a Special Protocol Assessment and orphan-drug designation.
