Today (July 24), AstraZeneca announced that its Gefurulimab achieved significant efficacy in the Phase III PREVAIL clinical trial for adult patients with generalized myasthenia gravis (gMG).

Generalized myasthenia gravis (gMG) is a rare, debilitating, chronic autoimmune neuromuscular disease characterized by loss of muscle function and severe weakness. Gefurulimab is a mini bispecific antibody (25kD) that contains an antibody heavy chain variable region targeting C5 and an antibody fragment that specifically binds to albumin. Its smaller molecular weight gives it better tissue penetration, and binding to albumin can prolong its half-life, making it a once-weekly subcutaneous injection therapy.

In the Phase III PREVAIL clinical trial, Gefurulimab showed significant efficacy in adult patients with gMG who were positive for anti-acetylcholine receptor (AChR) antibodies. Compared with placebo, Gefurulimab showed statistically significant and clinically meaningful improvements in the total score of the Myasthenia Gravis Activities of Daily Living Scale (MG-ADL) at week 26.

In addition, Gefurulimab was well tolerated in the trial, with a safety profile consistent with that observed in previous gMG C5 inhibitor trials, and no new safety signals were found. These data will be presented at an upcoming medical meeting and will be submitted to global regulatory authorities.

Gefurulimab, as a once-weekly, self-administered C5 inhibitor treatment, will provide patients with greater convenience and independence in managing their condition, helping them better control their own treatment. The emergence of this innovative therapy is expected to improve the treatment experience of gMG patients, reduce the treatment burden, and bring patients a higher quality of life.

It is worth mentioning that C5 inhibitors are a class of drugs that inhibit inflammatory and immune responses by blocking the activation of C5 protein in the complement system.

C5 protein plays a key role in a variety of autoimmune and inflammatory diseases. Therefore, C5 inhibitors have important potential in the treatment of diseases such as myasthenia gravis (gMG), paroxysmal nocturnal hemoglobinuria (PNH), and neuromyelitis optica (NMOSD).

AstraZeneca is one of the pioneers in the field of C5 inhibitors. Its reslizumab has been approved in China for the treatment of adult generalized myasthenia gravis (gMG) patients with positive anti-acetylcholine receptor (AChR) antibodies. In addition, AstraZeneca announced today that its Gefurulimab showed significant efficacy in the PREVAIL Phase III clinical trial for adult generalized myasthenia gravis (gMG) patients, and is expected to become a once-weekly subcutaneous injection therapy. AstraZeneca has a deep R&D foundation and technical accumulation in the field of C5 inhibitors. Its products have shown good efficacy and safety in clinical trials, and are expected to further consolidate its leading position in the treatment of rare diseases.

At the same time, Astellas entered the field of senile degenerative eye diseases through the acquisition of Iveric Bio, and its core product Izervay became the world's first long-term complement C5 inhibitor. Astellas has rich experience and technical accumulation in the field of ophthalmic disease treatment, and the approval of Izervay provides strong support for its position in the complement inhibitor market.

Novartis is focusing on the oral factor B inhibitor LPN023, which is currently undergoing a phase 2 trial for the treatment of PNH. Novartis has extensive experience and technical accumulation in the treatment of rare diseases, and the development of its oral factor B inhibitor LPN023 will provide a new treatment option for PNH patients.

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