Today, PhenoNet, Inc., an advanced clinical-stage biotechnology company developing therapies for neurodegenerative diseases, announced the completion of recruitment and randomization on August 27, 2026, in its Phase IIb clinical trial evaluating PHENOGENE-1A as an adjunctive therapy for patients with mild-to-moderate Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease. Completing enrollment marks a key milestone in the company’s clinical development program and advances the study toward topline data.

The ongoing clinical trial, “A Phase IIb Randomized, Double-Blind, Placebo-Controlled, Multi-Dose Study to Evaluate the Effects of PHENOGENE-1A (Cromolyn) as an Adjuvant Treatment in Subjects with Mild-to-Moderate ALS,” is being conducted at clinical sites across the United States and Europe. The enthusiasm for the project from the investigators is reflected in the brisk pace of enrollment. The study already reached an important operational milestone when the first participant completed the treatment period on July 2, 2026.

PHENOGENE-1A is a novel investigational therapy that combines the established safety profile of cromolyn sodium with an advanced targeted drug-delivery platform. The platform is designed to enhance bioavailability and achieve therapeutically relevant concentrations in the blood, brain, and CSF, targeting key mechanisms of neuroinflammation and neurodegeneration.

PHENOGENE-1A is being developed as a potential disease-modifying treatment intended to complement existing standards of care and slow disease progression in ALS. Preclinical studies and an earlier clinical cohort have provided the evidence to support its continued clinical development and evaluation in the ongoing Phase IIb trial. If the trial demonstrates efficacy, PHENOGENE-1A could improve patient outcomes and reduce the burden of ALS on patients, families, and caregivers.

“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.”

“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,” said Atul Gupta, M.D., Chief Medical Officer of PhenoNet, Inc. and medical and safety monitor for the clinical program. “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.”

“I am pleased to serve as Principal Investigator for this Phase IIb study, which is evaluating a promising investigational approach for patients with ALS,” said Professor Björn Oskarsson, M.D., a neurologist and ALS specialist at Mayo Clinic in Florida. “The study has the potential to contribute meaningful clinical information to the evolving ALS treatment landscape. The Safety Review Committee will continue to evaluate accumulating safety data as the trial progresses.”

“Completion of recruitment and randomization represents an important milestone in the development of PHENOGENE-1A,” said Peter S. Conti, M.D., Ph.D., Professor of Radiology, Biomedical Engineering and Pharmaceutical Sciences at the University of Southern California (USC). “The results of this trial will help inform the design of the company’s future clinical and regulatory development program.” Dr. Conti serves as a scientific advisor to PhenoNet, Inc., contributing his extensive expertise in molecular imaging, pharmacology, and the early diagnosis of neurodegenerative diseases to support the company’s scientific and clinical development strategy.

If the results of the Phase IIb study are supportive, PhenoNet, Inc. intends to discuss an appropriate registrational development pathway with the U.S. Food and Drug Administration, including the potential use of the 505(b)(2) pathway, a Special Protocol Assessment, and an orphan-drug designation.

About PhenoNet, Inc.

PhenoNet, Inc. is a privately held biotechnology company headquartered in Cambridge, Massachusetts. The company is developing PHENOGENE-1A as a potential treatment for neurodegenerative diseases, including ALS and Alzheimer’s disease (AD).

PhenoNet’s clinical-development programs are designed to address neuroinflammation and other biological mechanisms associated with neurodegeneration and disease progression. The company’s objective is to develop therapies for patients with serious diseases and substantial unmet medical needs.

Additional information is available at www.phenonet.us.

Forward-Looking Statements

This press release contains forward-looking statements regarding, among other matters, the clinical development, safety, potential efficacy, regulatory pathway, and possible commercialization of PHENOGENE-1A. These statements are based on PhenoNet’s current expectations and are subject to risks and uncertainties that could cause actual results to differ materially. These risks and uncertainties include, but are not limited to, the timing, conduct, and results of clinical trials; the interpretation of clinical data; regulatory requirements and decisions; the company’s ability to complete development of its product candidates; the availability of financing; manufacturing and supply considerations; competing products and technologies; market conditions; and other events that may affect the development or commercialization of PHENOGENE-1A. PHENOGENE-1A is an investigational therapy and has not been approved by the U.S. Food and Drug Administration or any other regulatory authority for the treatment of ALS, AD, or any other condition. PhenoNet undertakes no obligation to update these forward-looking statements except as required by applicable law.

Media gallery

About The Author
Business Wire