The final patient has begun dosing in a trial of a nasal therapy for non-active secondary progressive multiple sclerosis.
The phase 2a study is evaluating the safety, tolerability and efficacy of foralumab compared with a placebo, an inactive treatment.
Headline results are expected later this year and are planned for presentation at the joint ACTRIMS-ECTRIMS meeting in Toronto, Canada, in October.
ACTRIMS and ECTRIMS are major MS research organisations in the Americas and Europe.
Tiziana Life Sciences’ INFORM-MS trial is evaluating up to 48 adults at seven clinical sites in the US, with dosing under way at multiple locations.
Non-active secondary progressive multiple sclerosis, or SPMS, is a form of MS in which disability gradually worsens without relapses or new signs of disease activity on MRI scans.
It can develop after relapsing-remitting MS.
Ivor Elrifi, chief executive of Tiziana Life Sciences, said: “We are thrilled to reach this important milestone in our Phase 2 programme.
“The successful initiation of dosing in all patients underscores the strong execution by our clinical teams and the enthusiasm from investigators and patients for this novel intranasal approach.”
More than 20 disease-modifying therapies are approved for relapsing forms of MS, including relapsing-remitting MS, but options are much more limited once someone develops non-active SPMS.
Mitoxantrone is the only treatment approved for these patients, but it is rarely used because of its side effects and increased risk of serious complications.
Foralumab is an antibody designed to target CD3, a protein found on the surface of immune cells called T-cells. These cells contribute to the inflammation and damage associated with MS.
By binding to CD3, the treatment is expected to suppress the activity of inflammatory T-cells while increasing regulatory T-cells, which help keep immune responses in check.
Participants in the INFORM-MS trial were randomly assigned to receive either 50 or 100 micrograms of intranasal foralumab per dose, or a placebo, for about three months.
Treatment is given three times a week for two weeks, followed by one week without treatment, in repeating three-week cycles.
Participants who complete the randomised phase may have the option to receive foralumab for a further six months so researchers can study its longer-term effects.
The study’s main goal is to determine whether the treatment is safe and whether it can reduce microglial activity, which is believed to contribute to the progression of non-active SPMS.
Secondary and exploratory goals include changes in standard measures of disability and fatigue, as well as changes in inflammatory brain lesions and brain volume.
Foralumab is also being given to some people with non-active SPMS through an open-label expanded access programme, which provides access to the experimental medication outside clinical trials.
Data from 14 patients in the programme showed that disability remained stable or improved for most participants after about three years of follow-up. One patient experienced sustained worsening in disability scores.
Around two-thirds of the patients also showed improvements in fatigue that were considered clinically meaningful.
PET imaging data from ten patients showed that eight experienced reduced microglial activity after six months of treatment.
The placebo-controlled INFORM-MS trial is designed to test these early findings in a larger group.
Tanuja Chitnis, a neurologist at Mass General Brigham Neuroscience Institute and principal investigator in the trial, said: “Intranasal foralumab represents a promising and innovative therapeutic strategy for patients with non-active secondary progressive MS.
“We are encouraged by the smooth initiation of dosing across sites and look forward to evaluating its impact on microglial activation, and then further clinical outcomes during the open label extension in this underserved patient population.”
