Foralumab nasal spray joins pathway to HEALEY platform trial
Tiziana's Phase 2 study accepted into Healey ALS MyMatch program
A Phase 2 clinical trial to investigate Tiziana Life Sciences’ intranasal foralumab for the treatment of amyotrophic lateral sclerosis (ALS) has been accepted into the Healey ALS MyMatch program at Massachusetts General Hospital.
The program, located at the Sean M. Healey & AMG Center for ALS, is a precision medicine initiative that conducts a series of Phase 1b/2a clinical trials, matching patients to experimental treatments based on their genetic information and specific biomarkers.
If data from MyMatch trials are positive, the drugs may be considered for inclusion in the HEALEY platform trial (NCT04297683), a Phase 2/3 trial that simultaneously tests multiple ALS therapies against a shared placebo group, or may advance directly into a Phase 3 trial.
Advancing the trial quickly
The upcoming Phase 2a trial, supported by a grant from the ALS Association, will be led by principal investigators Suma Babu and James Berry, MD, at Mass General Brigham.
Participants will be recruited at multiple rapid-enrolling U.S. centers within the NEALS Consortium, a nonprofit organization comprising approximately 150 research centers that collaborate to accelerate the development of new ALS therapies.
“The rapid progression to trial activation reflects the urgency of the ALS community and the compelling science behind nasal foralumab,” Ivor Elrifi, PhD, Tiziana’s CEO, said in a company press release. “By leveraging the immune-modulatory potential of the nasal route, we aim to deliver meaningful clinical impact with a non-invasive therapy.”
Foralumab is an antibody-based therapy that binds CD3, a protein found at the surface of immune T-cells. This is designed to reduce the activity of pro-inflammatory T-cells while increasing the number and activity of immunosuppressive T-cells.
By helping rebalance immune activity, foralumab may indirectly reduce the activation of microglia — the brain’s resident immune cells, which become abnormally active and harmful in several neurodegenerative conditions. This may potentially help slow neurodegeneration.
Delivered as a nasal spray, foralumab is intended to modulate immune responses in the brain without causing body-wide immunosuppression, which may help minimize side effects.
“Foralumab’s ability to expand regulatory T cells and dampen [brain and spinal cord] inflammation offers a biologically rational approach to slowing progression in a genetically defined ALS population. We are eager to translate these mechanisms into patient benefit,” Elrifi said.
Trial design and biomarkers
Foralumab’s trial will assess the safety and efficacy of two doses of the experimental medication (50 and 100 micrograms) administered via nasal spray in 20 people with ALS.
Participants will be randomly assigned to one of the foralumab doses or a placebo, given in three-week cycles. After six months of treatment, researchers will assess changes in clinical parameters, microglia activation, and other disease biomarkers.
“This innovative multi-site randomized placebo-controlled trial will integrate innovative nasal immunology with cutting-edge blood, spinal fluid and cellular and advanced brain imaging markers to understand the effect of the drug,” said Babu, co-director of the Neurological Clinical Research Institute at Massachusetts General Hospital.
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