AstraZeneca Prepares Showdown With JNJ, Amgen With Phase 3 Win For Myasthenia Gravis Drug Candidate

AstraZeneca Plc (NASDAQ:AZN) on Thursday reported high-level results from the PREVAIL Phase 3 trial evaluating the safety and efficacy of gefurulimab in adults with generalized myasthenia gravis (gMG). The trial enrolled 260 patients.

MG is a chronic autoimmune disorder that causes muscle weakness and fatigue. The disease is characterized by antibodies against the acetylcholine receptor, a protein found on the surface of nerve cells that plays a key role in muscle contraction.

In adults with anti-acetylcholine receptor (AChR) antibody-positive (Ab+) generalized myasthenia gravis, gefurulimab met its primary and all secondary endpoints.

Also Read: Johnson & Johnson’s New ‘Compelling Data’ Shows Sustained Symptom Improvement In Muscular Disease Patients

Data demonstrated a statistically significant and clinically meaningful improvement from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score at week 26 compared to placebo.

gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness.

Gefurulimab was well-tolerated, and the safety profile was consistent with previous trials of C5 inhibitors in gMG with no new safety signals observed. These data will be presented at a forthcoming medical meeting and shared with global regulatory authorities.

In April, Cartesian Therapeutics, Inc. (NASDAQ:RNAC) revealed 12-month efficacy and safety data from the Phase 2b trial of Descartes-08, the lead cell therapy candidate, in participants with generalized myasthenia gravis.

In March, Amgen Inc. (NASDAQ:AMGN) announced new data from the Phase 3 MINT trial of Uplizna (inebilizumab-cdon) for adult patients with generalized myasthenia gravis.

The results demonstrated the durable and sustained efficacy of Uplizna in patients with acetylcholine receptor autoantibody-positive (AChR+) gMG with two doses a year following an initial loading dose.

The trial demonstrated continued improvement in the efficacy of Uplizna compared to placebo as measured by the change in baseline of Myasthenia Gravis Activities of Daily Living (MG-ADL) score in the AChR+ subpopulation through week 52.

Price Action: AZN stock is up 1.45% at $$74.06 at the last check on Thursday.

Read Next:

• FDA Flags Major Safety Concerns For Argenx’s Nerve Disorder Drug, Stock Falls

Photo by JHVEPhoto via Shutterstock