AstraZeneca triumphs in Phase III trials with a nanobody treatment for Myasthenia Gravis.
In a significant development for patients with myasthenia gravis, AstraZeneca's investigational treatment, gefurulimab, has demonstrated promising results in a Phase III trial. The drug, which targets the most common form of the disease, could potentially compete with Zilbrysq in the market.
Key Findings
The global, double-blind, placebo-controlled PREVAIL trial involved 260 patients across 20 countries. Participants were randomly assigned to receive either gefurulimab or a placebo for the initial 26 weeks, followed by an optional open-label extension period.
Gefurulimab met all primary and secondary endpoints in the PREVAIL trial, demonstrating a statistically significant and clinically meaningful improvement in patients' quality of life, as measured by the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score. This improvement was observed after 26 weeks of treatment.
In addition, gefurulimab was found to be safe and well-tolerated, with a profile consistent with previous C5 inhibitors. It showed statistically significant improvements in secondary endpoints like the Quantitative Myasthenia Gravis total score and the Myasthenia Gravis Composite total score.
Future Presentation of Detailed Information
More detailed results from the PREVAIL trial will be presented at a future medical meeting. AstraZeneca plans to discuss these findings with global regulatory authorities, which may lead to an application for FDA approval following the drug's existing orphan drug designation.
Market Implications
Myasthenia gravis is an autoimmune condition that breaks down communications between the brain and muscles. Currently, Zilbrysq is the only FDA-approved subcutaneous treatment for generalized myasthenia gravis (gMG). Argenx's Vyvgart, which received FDA approval in June 2023, currently leads in the $20 billion myasthenia gravis market due to its two-year head-start.
The minimal clinically important difference on the MG-ADL scale is considered to be ≥2 points from baseline. In a Phase III trial, a comparable approved daily treatment, UCB's Zilbrysq, got a 4.39-point reduction using the MG-ADL scale.
The PREVAIL announcement follows a week of newsmaking for AstraZeneca, which also announced a new $50 billion manufacturing push in the U.S., led by a plant in Virginia that will create the company's metabolic drugs aimed at cardiovascular conditions and obesity.
Dan Samorodnitsky reported this story for BioSpace. AstraZeneca will present more detailed information about gefurulimab at a future medical meeting, potentially at the Myasthenia Gravis Foundation of America (MFGA)'s sessions at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting on Oct. 29.
Source: BioSpace (dan.samorodnitsky@our website)
The promising results observed in the Phase III trial of AstraZeneca's investigational treatment, gefurulimab, for myasthenia gravis, suggest that science may contribute to the advancement of health-and-wellness for patients with neurological-disorders, such as myasthenia gravis, by offering a potential alternative to existing treatments like Zilbrysq. If the drug's safety and efficacy are confirmed through comprehensive analysis, it could carve out a significant niche in the $20 billion market for myasthenia gravis treatments.
