Enrollment complete in Phase 2b trial of add-on therapy for adults with ALS
PHENOGENE-1A is being tested alongside riluzole over 24 weeks
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- Enrollment is complete in a Phase 2b trial of PHENOGENE-1A for mild-to-moderate ALS.
- ALS is a progressive disorder in which motor neurons die, with nervous system inflammation thought to play a role.
- PHENOGENE-1A contains cromolyn and is being tested with riluzole; cell studies suggest cromolyn may support nerve branch growth when used with nerve growth factor.
Enrollment of adults with mild-to-moderate amyotrophic lateral sclerosis (ALS) has been completed in a clinical trial testing PHENOGENE-1A as an add-on treatment for the neurodegenerative disease.
Enrollment of about 105 participants in the Phase 2b trial (NCT07142291) wrapped up in late August, and they were randomly assigned to receive one of two doses of PHENOGENE-1A or a placebo by dry powder inhaler twice daily for 24 weeks. All are also being treated with riluzole tablets, an approved ALS treatment previously sold as Rilutek (now only available as generics).
In addition, the first participant completed their 24-week treatment period in early July.
Enrollment and first treatment completion mark trial milestones
“We are pleased to have completed recruitment and randomization and to have achieved the important milestone of the first participant completing the treatment period,” David R. Elmaleh, PhD, founder and chairman of Phenonet, said in a company press release.
The study’s main goals are to evaluate PHENOGENE-1A’s safety and changes in participants’ ability to perform everyday activities, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), after 24 weeks.
“The results of this trial will help inform the design of the company’s future clinical and regulatory development program,” said Peter S. Conti, MD, PhD, a professor at the University of Southern California and a scientific advisor to Phenonet.
If trial findings are supportive, Phenonet plans to discuss with the U.S. Food and Drug Administration an appropriate path for further clinical and regulatory development.
“To date, no safety findings have been reported that resulted in a change to the protocol or the conduct of the study,” said Atul Gupta, MD, Phenonet’s chief medical officer and the medical and safety monitor for the clinical program.
ALS is a progressive disorder in which motor neurons, the nerve cells that control movement, become damaged and die. “ALS remains one of the most devastating diagnoses in neurology, with limited options that address the underlying disease,” Gupta said.
The causes of ALS are not fully understood, but abnormal inflammation in the nervous system is thought to play a role in the disease’s development and progression.
“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,” Elmaleh said.
PHENOGENE-1A uses cromolyn in targeted delivery platform
PHENOGENE-1A is a formulation of cromolyn, an anti-inflammatory molecule that has been used since the 1970s to treat asthma and other inflammatory disorders. In lab-grown cells, cromolyn was shown to enhance the growth of nerve cell branches when given together with nerve growth factor, a protein that helps nerve cells grow.
“Early intervention, beginning with the first clinical signs of muscle function decline, may be critical for delaying disease progression in ALS,” Elmaleh said. “Our findings from in vitro [lab-grown cell] studies, … ALS animal models, and a Phase IIa cohort supported advancing PHENOGENE-1A to this Phase IIb trial.”
Björn Oskarsson, MD, principal investigator of the Phase 2b study at Mayo Clinic in Florida, said the trial “has the potential to contribute meaningful clinical information to the evolving ALS treatment landscape.”
In addition to ALS, Phenonet is developing PHENOGENE-1A as a potential treatment for Alzheimer’s disease.
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