LONDON (ShareCast) - (Sharecast News) - AstraZeneca announced on Thursday that its investigational treatment 'gefurulimab' achieved statistically significant and clinically meaningful results in a phase three trial for generalised myasthenia gravis (gMG), a rare autoimmune neuromuscular disorder.
The FTSE 100 pharmaceuticals giant said the dual-binding nanobody met both its primary and secondary endpoints in the global 'PREVAIL' study.

It said the trial showed that gefurulimab delivered a significant improvement in the myasthenia gravis activities of daily living (MG-ADL) total score at week 26 compared to placebo.

The subcutaneous, once-weekly complement C5 inhibitor was also well tolerated, with no new safety signals reported.

"These positive results from the PREVAIL phase three trial demonstrate the potential for gefurulimab to offer rapid and sustained disease control for this patient community," said Marc Dunoyer, chief executive of Alexion, AstraZeneca Rare Disease.

"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."

The PREVAIL study enrolled 260 adults across North America, Europe, Asia and the Pacific region, all of whom had anti-acetylcholine receptor antibody-positive gMG.

Participants received either gefurulimab or placebo over a 26-week period, following an initial loading dose and subsequent once-weekly maintenance doses.

The primary endpoint assessed changes in MG-ADL, a patient-reported measure of daily functional ability.

Kelly Gwathmey, principal investigator of the trial, noted the potential impact 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," she said.

Gefurulimab, which has received orphan drug designation in the US for gMG, was designed to inhibit the terminal complement cascade by binding to the C5 protein and serum albumin.

The dual-binding mechanism allowed for an extended half-life and convenient dosing.

AstraZeneca said the treatment was aimed at reducing the immune system's overactivation that leads to muscle weakness in gMG.

It said the full data would be presented at a future medical meeting and submitted to global regulatory authorities.

An open-label extension study evaluating long-term safety and efficacy was ongoing.

At 0942 BST, shares in AstraZeneca were up 1.78% at 10.864p.

Reporting by Josh White for Sharecast.com.

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