ALS Association funds small clinical trial of intranasal foralumab in ALS
Study will explore safety, efficacy of two doses in 20 patients
The ALS Association has awarded a “groundbreaking” grant to Tiziana Life Sciences to support a small, early-stage clinical trial testing foralumab nasal spray for amyotrophic lateral sclerosis (ALS).
The upcoming trial intends to investigate the safety and effectiveness of two foralumab doses in 20 ALS patients. It builds on positive findings from multiple sclerosis (MS) patients, in whom the intranasal antibody therapy significantly decreased neuroinflammation and stabilized or reduced disability levels. Tiziana now wants to see if foralumab might also help in ALS.
Funding for the trial comes from the ALS Association’s Hoffman ALS Clinical Trial Awards Program, to which the company was invited to apply by the organization. The program, named after late philanthropist Hugh Hoffman, is designed to support early clinical trials that gather safety, dosing, and biomarker data on a small number of patients to advance the development of experimental therapies. Positive results may help attract additional funding for the next stage of drug development, typically larger clinical trials.
“We are honored to receive this prestigious grant from the ALS Association, which underscores the promising potential of our therapeutic platform in addressing the urgent needs of ALS patients,” Ivor Elrifi, CEO of Tiziana, said in a company press release.
“With this award, we are pleased to help advance the development of intranasal foralumab for ALS. By funding programs at this early stage, we hope to accelerate the development of therapeutic candidates that can help make ALS a livable disease,” said Kuldip Dave, PhD, senior vice president of research at the ALS Association.
The underlying cause of ALS isn’t fully known, but inflammation is believed to contribute to its onset and progression. This inflammation is driven, in part, by the excessive activity of immune T-cells and microglia.
Testing foralumab
Foralumab is an antibody-based therapy designed to modify the activity of T-cells by targeting a protein on their surface known as CD3. Animal models have shown this can reduce the activity of inflammatory T-cells while simultaneously boosting the activity of anti-inflammatory T-cells, which can act on other immune cell types to further reduce inflammation.
The therapy has been tested in 10 people with nonactive secondary progressive MS, which is mainly driven by chronic inflammation in the brain and spinal cord. While people with it normally have steady symptom worsening over time, all 10 patients who took foralumab under expanded access programs saw their disability levels stabilize or improve after at least six months. Eight of them also had reduced microglial activity in their brains, according to PET imaging data.
A Phase 2b trial (NCT06292923) is now comparing the safety and efficacy of foralumab relative to a placebo in the same MS population. Building on data from the expanded access programs, researchers will use PET imaging to examine the drug’s effect on markers of brain inflammation.
The small clinical trial in ALS will also measure brain inflammation with PET scans to see if foralumab can reduce harmful immune system activity in ALS patients.
“We will be using PET imaging to detect neuroinflammation in this study of patients with ALS. We hope to replicate the previous positive findings of the PET imaging approach that Tiziana has seen in studies of patients with multiple sclerosis, in this new potential indication for intranasal foralumab,” said James Berry, MD, and Suma Babu, director and co-director of the Neurological Clinical Research Institute at Mass General Hospital.
About the Author
