Gefurulimab found to ease gMG symptoms in global Phase 3 trial
Self-administered therapy may provide 'rapid and sustained disease control'
Treatment with gefurulimab, an experimental self-administered injection therapy, outperformed a placebo in easing symptoms of generalized myasthenia gravis (gMG) among people with the chronic autoimmune disease, the results of a Phase 3 clinical trial show.
The trial met both its primary and secondary endpoints, or goals, with treatment resulting in a “statistically significant and clinically meaningful reduction in disease severity” among gMG patients after six months, according to Alexion, Astrazeneca Rare Disease, the therapy’s developer.
“These positive results … demonstrate the potential for gefurulimab to offer rapid and sustained disease control for this patient community,” Marc Dunoyer, CEO of Alexion, said in a company press release.
Dubbed PREVAIL (NCT05556096), the global trial involved more than 250 people with gMG.
Dunoyer said “these data, reflecting patient participation across 20 countries, reinforce the established safety profile and efficacy of C5 inhibition and show the potential for gefurulimab as a first line biologic, with the convenience of a self-administered option.”
An autoimmune neuromuscular condition, MG is caused by self-reactive antibodies that disrupt the communication between nerve and muscle cells, resulting in symptoms like muscle weakness and fatigue. When MG-driving antibodies bind to their targets, they can activate the complement cascade, a group of immune proteins that further contribute to damage the point of contact between nerve and muscle cells.
Gefurulimab is designed to block the activation of the complement cascade by targeting a protein called C5.
Gefurulimab meets goals in trial spanning 20 countries
Two other C5-targeting treatments developed by Alexion — Ultomiris (ravulizumab-cwvz) and Soliris (eculizumab) — are already approved for gMG. Both are indicated for patients who are positive for antibodies targeting the acetylcholine receptor (AChR), the most common type of MG-causing antibody.
But while Ultomiris and Soliris are given by intravenous, or into-the-vein, infusions that must be administered under the guidance of a healthcare provider, gefurulimab is given by subcutaneous, or under-the-skin, injection, and can be self-administered by patients or given by caregivers who have had adequate training. According to the developer, this mode of administration may ease the treatment burden for patients.
A once-weekly, self-administered C5 treatment option would offer patients greater convenience and independence in managing their condition, empowering them to have more control over their therapy.
“Rapidly fluctuating symptoms and the unpredictable disability associated with gMG can affect nearly every aspect of a patient’s life, making early intervention and sustained disease control a critical treatment goal,” said Kelly Gwathmey, MD, principal investigator of the trial at Virginia Commonwealth University. “A once-weekly, self-administered C5 treatment option would offer patients greater convenience and independence in managing their condition, empowering them to have more control over their therapy.”
The Phase 3 trial tested gefurulimab against a placebo in 260 people with gMG who were positive for anti-AChR antibodies. The study’s main goal was to assess the impact of treatment on the scores of the MG Activities of Daily Living (MG-ADL) — a scale that assesses the extent to which MG symptoms are causing problems in day-to-day life — after 26 weeks, or about six months.
According to Alexion, the results showed that gefurulimab was significantly better than the placebo at improving MG-ADL scores after 26 weeks. Scores on the Quantitative MG (QMG) scale, another measure of disease severity, also improved significantly more with gefurulimab than with the placebo.
The results also indicated that the therapy’s safety profile is similar to that of other C5-targeting therapies.
Alexion didn’t provide further details on the findings, but noted that in-depth results will be presented at future scientific meetings and shared with regulatory authorities.
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