Foralumab eases inflammation in 80% of nonactive SPMS patients

Most patients with nonactive secondary progressive multiple sclerosis (SPMS) who took foralumab nasal spray for at least six months saw a decrease in microglial activity in their brains, according to its developer Tiziana Life Sciences.

The results were gleaned from PET imaging data from 10 nonactive SPMS patients who took part in an expanded access program (EAP), wherein patients with serious or life-threatening conditions have access to an experimental therapy outside of clinical trials.

Microglia are resident immune cells in the brain that are believed to play a role in driving inflammation and nerve damage in multiple sclerosis (MS). In the ongoing EAP, which includes two patients in single-patient access programs and eight in an intermediate-sized EAP, 80% of patients saw a reduction in microglia activity.

“I am thrilled that 80% of the na-SPMS patients who received intranasal foralumab treatment for at least [six] months have a qualitative reduction of microglial activity as confirmed in these latest PET images,” Gabriele Cerrone, chairman, acting CEO, and founder of Tiziana, said in a company press release.

Earlier data from the same 10 patients showed intranasal foralumab stabilized or improved disability levels in all of them and eased fatigue in 70%. The U.S. Food and Drug Administration (FDA) has allowed foralumab to be administered to 20 more patients in the EAP, based on the findings.

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Tiziana has also used EAP clinical data and animal model data to apply for orphan drug and fast track designations, the latter of which has been accepted for review by the FDA. The designations are meant to expedite the clinical development and approval of the therapy. Fast track status provides more frequent meetings with the FDA and discussions about the development plan and, if certain criteria are met, enables therapies to receive priority review and accelerated approval. Orphan drug status is given to drugs that treat rare diseases and provides development incentives such as reduced regulatory fees and seven years of market exclusivity if the treatment is approved.

“The progressive nature of [nonactive] SPMS and lack of FDA-approved therapies for this disease aligns with the Food and Drug Administration’s criteria for fast track designation. The increased interaction and partnership with the FDA afforded by this designation would be a tremendous asset to our foralumab development program, if granted,” Cerrone, said in another press release.

Foralumab is an antibody-based therapy designed to reduce MS-driving inflammation by targeting the CD3 protein at the surface of immune T-cells. This should block inflammatory T-cell activity, while boosting regulatory T-cells that help keep the immune system in check.

In the EAP, foralumab is being administered into the nose at a 50 microgram (mcg) dose. It’s given in three-week cycles, consisting of sprays into each nostril three times a week for two weeks, followed by a week without treatment.

After the promising findings in a patient population with few treatment options available, Tiziana launched a Phase 2a trial (NCT06292923) to evaluate the treatment in a placebo-controlled setting. In the trial, 54 patients with nonactive SPMS will be enrolled and randomly assigned to one of two foralumab doses (50 or 100 mcg) or a placebo for three months.

The main goals are to determine its safety and changes in microglial activation in PET scans after three months. Researchers will also look at disability and fatigue outcomes as secondary goals to confirm the benefits observed in the EAP.

“With the allowance of an additional 20 patients in the EA program, the application for orphan drug designation for [nonactive] SPMS, and the ongoing Phase 2a trial, Tiziana is rapidly progressing its intranasal foralumab program in multiple sclerosis,” Cerrone said.