Treatment cuts exacerbation, rescue therapy risk in generalized MG

Uplizna (inebilizumab) reduced the frequency of disease exacerbations, or periods when symptoms worsen, and the need for rescue therapy in people with generalized myasthenia gravis (gMG).

That’s according to a prespecified analysis of data from the global Phase 3 MINT clinical trial (NCT04524273), which showed that after six months of treatment, the risk of an exacerbation was 59% lower and the risk of requiring rescue therapy was 66% lower with Uplizna than with a placebo.

“By minimizing exacerbations and [rescue therapy] use, [Uplizna] may lead to improved gMG outcomes and reduced overall health system burden,” the researchers wrote.

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Prespecified analysis examines exacerbations and rescue therapy

The results were described in the study “Management of Exacerbations and Rescue Therapy in the Phase 3 Myasthenia Gravis Inebilizumab Trial: A Prespecified Analysis of a Randomized Clinical Trial,” published in JAMA Neurology.

Myasthenia gravis (MG) is an autoimmune disease driven by self-reactive antibodies that target proteins involved in communication between nerves and muscles, leading to symptoms such as muscle weakness and fatigue. These antibodies most commonly target acetylcholine receptors (AChRs), but they can also target other proteins, such as muscle-specific kinase (MuSK).

Uplizna is an antibody-based therapy approved for adults with gMG and antibodies against AChR or MuSK. Given by infusion into the bloodstream, it is designed to reduce the number of B cells, immune cells that produce antibodies, including the self-reactive antibodies that drive MG.

The treatment’s approvals were based on top-line results from the MINT trial, which enrolled 238 adults with gMG. Most participants had anti-AChR antibodies (AChR group) and were randomly assigned to Uplizna or a placebo during a 52-week, or one-year, randomized period. Those with anti-MuSK antibodies (MuSK group) were randomly assigned to Uplizna or a placebo during a 26-week, or six-month, randomized period.

Participants taking more than 5 mg per day of corticosteroids, a type of anti-inflammatory and immunosuppressive medication that is linked to serious side effects, began a planned taper at week 4, with the goal of reducing their daily dose to 5 mg or less by week 24.

MINT trial tested Uplizna alongside a corticosteroid taper

Earlier six-month results showed that Uplizna significantly reduced MG Activities of Daily Living (MG-ADL) scores, a patient-reported measure of MG severity, compared with a placebo, meeting the trial’s main goal. Uplizna also reduced Quantitative MG (QMG) scores, a clinician-rated measure of muscle weakness, compared with a placebo, meeting a key secondary goal.

Now, researchers reported results from a prespecified analysis of secondary trial endpoints related to gMG exacerbations, including rescue therapy use. Exacerbations were defined as rescue therapy use, myasthenic crisis, or significant symptom worsening that met specific criteria on the MG-ADL scale. A myasthenic crisis is a severe complication in which muscle weakness affects the muscles needed for breathing.

In the two years prior to the study, 51.7% of participants were hospitalized due to MG and 31.4% had received rescue therapy to rapidly control symptoms. The most commonly used rescue therapies during that period were intravenous immunoglobulin (18.6%), plasma exchange (13.6%), and high-dose corticosteroids (4.7%).

Data showed that after six months, fewer participants receiving Uplizna experienced an exacerbation (16% vs. 35%) or received rescue therapy (8.4% vs. 23.9%) compared with those given a placebo. In addition, 1.7% of Uplizna-treated participants experienced both significant symptom worsening and rescue therapy use, compared with 10.3% in the placebo group.

At six months, the percentages of participants who experienced exacerbations or received rescue therapy were lower with Uplizna than with a placebo in both the AChR and MuSK groups.

The differences were also seen through one year among participants with anti-AChR antibodies. By week 52, 20% of those receiving Uplizna had experienced an exacerbation, compared with 45.2% of those receiving a placebo. Rescue therapy was used by 11.6% of Uplizna-treated participants versus 35.5% of those given a placebo.

Uplizna lowers exacerbation risk across antibody groups

Further analyses demonstrated that Uplizna treatment significantly reduced the risk of exacerbation by 59% in the combined population after six months, by 61% in the AChR group after one year, and by 79% in the MuSK group after six months.

The therapy was also linked to a significantly lower risk of rescue therapy use, by 66% in the combined population after six months and by 70% in the AChR group after one year. The risk was also lower with Uplizna in the MuSK group after six months, but the difference did not reach statistical significance, meaning it could have been due to chance.

Additionally, among participants taking more than 5 mg per day of corticosteroids, 87.4% of those receiving Uplizna and 84.6% of those receiving a placebo reached the target dose of 5 mg per day or less by week 24. Few exacerbations occurred during the steroid taper initiation period, and exacerbations also occurred in participants whose steroid dose was unchanged or increased.

“Reducing the frequency of exacerbations and the need for [rescue therapy] has important implications for both patients and health care resource utilization,” the researchers wrote, as “exacerbations of gMG, particularly myasthenic crises, are associated with significant [illness], often requiring hospitalization and intensive care support.”