Foralumab wins FDA fast track designation for nonactive SPMS
Tiziana Life Sciences has received fast track designation from the U.S. Food and Drug Administration (FDA) for its intranasal foralumab to treat nonactive secondary progressive multiple sclerosis (SPMS).
The FDA gives fast track status to experimental medicines that have the potential to fill unmet needs to treat serious disorders. The designation gives developing companies access to incentives such as more frequent meetings with the FDA, with the goal of speeding the development and review of potentially important medications.
The designation “is a testament to the innovative approach we are taking with foralumab,” William Clementi, Tiziana’s chief development officer, said in a company press release.
Gabriele Cerrone, chairman, acting CEO, and founder of Tiziana, said the FDA’s action “underscores the potential of foralumab to address critical unmet needs in the treatment of neurodegenerative diseases.”
Most people with multiple sclerosis (MS) are initially diagnosed with relapsing-remitting disease (RRMS), which is marked by relapses, or flares, when symptoms suddenly worsen, interspersed with periods of remission in which symptoms ease or disappear entirely. Over time, most people with RRMS eventually progress to SPMS, which is marked by symptoms that get continuously worse even in the absence of relapses.
Reducing inflammation
SPMS is deemed active or nonactive based on whether the patient experiences relapses. Historically, nonactive SPMS has been much harder to effectively treat than the active form, and only one treatment, mitoxantrone, exists for this disease type.
Foralumab is designed to reduce inflammation in the brain and spinal cord, which drives disease activity in MS. The therapy delivered intranasally, or via a nose spray.
The therapy specifically targets a protein called CD3 that is found at the surface immune T-cells. By targeting this molecule, foralumab aims to reduce the activity of inflammatory T-cells while simultaneously boosting the activity of anti-inflammatory T-cells. This is expected to reduce inflammation overall to help control MS disease activity.
“We believe that the intranasal delivery method offers a novel way to effectively target neuroinflammation, and we look forward to working closely with the FDA to bring this therapy to patients in need,” Clementi said.
Foralumab is being tested in 10 nonactive SPMS patients in an expanded access program, with the treatment given via nose sprays at a 50 microgram (mcg) dose. It’s administered in three-week cycles, consisting of sprays into each nostril three times a week for two weeks, followed by one week of rest.
Data have suggested that foralumab treatment reduced brain inflammation, with 80% of patients experiencing a reduction in the activity of brain resident immune cells called microglia. It also eased fatigue in 70% of patients.
A Phase 2a clinical trial (NCT06292923) testing foralumab against a placebo in nonactive SPMS is ongoing. The trial, which dosed its first participant late last year, is open to adults with nonactive SPMS who have experienced continual disease progression despite treatment with available therapies.
A total of 54 patients are expected to enroll. They will receive foralumab, at a dose of 50 mcg or 100 mcg, or a placebo, on a schedule similar to that of the expanded access program.
The main goals are to assess the treatment’s safety and impact on microglial activity. Other outcome measures will include disability progression and changes in fatigue, inflammatory brain lesions, and brain volume. The trial is expected to conclude in November.
“We are committed to advancing this promising therapy as quickly as possible to benefit patients,” Cerrone said.
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