Amgen's recent Phase 3 trial results for Uplizna in generalized myasthenia gravis (gMG) have shown clinically meaningful and statistically significant efficacy, while data for rocatinlimab in atopic dermatitis has been met with skepticism by Wall Street analysts. The MINT trial results for Uplizna were presented at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting.
Uplizna's Positive Results in gMG
The Phase 3 MINT trial evaluated Uplizna (inebilizumab-cdon) in adults with gMG, a rare autoimmune disorder. The trial met its primary endpoint, demonstrating a statistically significant change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score for Uplizna (-4.2) compared with placebo (-2.2) (difference: –1.9, p<0.0001) at Week 26 for the combined study population. The study included patients who are acetylcholine receptor autoantibody-positive (AChR+) and those who are muscle-specific kinase autoantibody-positive (MuSK+).
Uplizna also demonstrated statistically significant and clinically meaningful changes from baseline compared to placebo for the first four key secondary endpoints:
- Significant change in Quantitative Myasthenia Gravis (QMG) score for the combined population (-4.8) compared to placebo (-2.3) at Week 26 (difference: -2.5, p=0.0002).
- In the AChR+ population, Uplizna demonstrated mean change from baseline (-4.2) in MG-ADL score at Week 26 compared to placebo (-2.4) (difference: -1.8, p=0.0015).
- In the AChR+ population, Uplizna demonstrated mean change from baseline (-4.4) in QMG score at Week 26 compared to placebo (-2.0) (difference -2.5, p=0.0011).
- In the MuSK+ population, Uplizna demonstrated mean change from baseline (-3.9) in MG-ADL score at Week 26 compared to placebo (-1.7) (difference -2.2, p=0.0297).
Notably, patients taking corticosteroids at the start of the study were tapered down to prednisone 5 mg per day by Week 24. No new safety signals were identified.
Richard J. Nowak, M.D., M.S., global principal study investigator and director of the Myasthenia Gravis Clinic at Yale University, emphasized the clinical benefits of Uplizna, noting that the trial also included a protocol-specified steroid taper, which is an important consideration for patients given the adverse effects of prolonged high-dose steroid use.
Amgen plans to file for approval of Uplizna in the U.S., followed by other key markets, based on the MINT trial results.
Rocatinlimab's Atopic Dermatitis Data Under Scrutiny
While Amgen is expanding the treatment options, its rocatinlimab, designed to treat atopic dermatitis, showed statistically significant improvement versus placebo. However, analysts have expressed concerns about its efficacy profile, especially when there are still questions about safety.
Nearly 33% of patients in the study witnessed a significant improvement in their eczema symptoms. Amgen is also studying the medicine in asthma and prurigo nodularis.
Analysts suggest the drug needed to show an especially high efficacy profile to counter safety concerns. The results are underwhelming, especially when compared to Sanofi and Regeneron Pharmaceuticals’ Dupixent.
