Third indication beckons for Amgen's Uplizna
As Amgen waits for an FDA decision on a second indication for its B cell-depleting therapy Uplizna, it has new data suggesting a third may be coming down the pipe.
New data from the phase 3 MINT trial in adults living with autoimmune disorder generalised myasthenia gravis (gMG) – which can lead to muscle weakness, breathing troubles, difficulty swallowing and impaired speech and vision – will be used to support a filing with the FDA in the first half of this year.
Amgen acquired CD19-directed antibody Uplizna (inebilizumab) as part of its $27.8 billion takeover of Horizon Therapeutics in 2023, and already has approval for the drug in AQP4 antibody-positive neuromyelitis optica spectrum disorder (NMOSD), generating $379 million in sales last year.
The FDA is currently reviewing the drug as a treatment for immunoglobulin G4-related disease (IgG4-RD), another rare disease that causes inflammation and fibrosis around the body, with a decision due on 3rd April.
Approval in gMG would open up a market larger than the other two combined in terms of the number of patients in the US and would help Amgen achieve its objective of building multibillion-dollar sales for Uplizna.
The new MINT data builds on earlier results reported at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) congress last year, which revealed that the trial met its primary objective: a significant improvement in myasthenia gravis activities of daily living (MG-ADL) disease scores at 26 weeks compared to placebo in patients with acetylcholine receptor autoantibody-positive (AChR+) or muscle-specific kinase autoantibody-positive (MuSK+) gMG.
The new 52-week data from the study only applies to the AChR+ cohort, as the smaller MuSK+ group did not receive treatment beyond 26 weeks. After a year, 72.3% of the Uplizna group had three-point or greater improvement in the MG-ADL score, compared to 45.2% of patients receiving placebo.
Amgen's head of R&D Jay Bradner said Uplizna could offer a "new standard of care" in gMG and a simpler treatment regimen than current drugs for the disease, thanks to a twice-yearly dosing regimen after an initial loading dose.
Other options include complement C5 inhibitors like AstraZeneca/Alexion's Soliris (eculizumab) and Ultomiris (ravulizumab) – given intravenously every two and eight weeks, respectively – as well as Argenx's Vyvgart Hytrulo (efgartigimod alfa) and UCB's Rystiggo (rozanolixizumab), both subcutaneously injected FcRn blockers, and Argenx's older IV version Vyvgart.
