With what analysts are calling “strong” data, Amgen plans to file a regulatory submission for Uplizna, currently approved for a rare ocular autoimmune disorder, in myasthenia gravis, in the first half of 2025.
Amgen’s anti-CD19 antibody Uplizna maintains and improves treatment response in patients with generalized myasthenia gravis through 52 weeks of follow-up, according to a Phase III readout posted Wednesday.
These findings, revealed in an abstract for the upcoming meeting of the American Academy of Neurology (AAN), “do look strong,” William Blair analysts wrote in an investor note on Wednesday evening, noting that they “solidify a meaningful role for Uplizna in the treatment of myasthenia gravis,” a chronic autoimmune disorder that causes muscle weakness.
Amgen said it will seek myasthenia gravis approval for Uplizna, with a filing planned for the first half of 2025.
Wednesday’s data come from a subpopulation analysis of the Phase III MINT trial, which enrolled 238 patients with myasthenia gravis, of whom 190 were positive for acetylcholine receptor (AChR) antibodies, which attack the receptors critical in nerve-to-muscle signaling. The results showed that Uplizna significantly eased the impact of the disease on daily living in AChR+ patients as compared with placebo. Over 52 weeks of observation, AChR+ patients in the Uplizna group saw significantly stronger reductions in disease severity versus placebo.
Along with a previous 26-week readout in September 2024, Wednesday’s results from MINT underscore Uplizna’s “strong efficacy with impressive durability,” the William Blair analysts said, adding that the drug’s “infrequent every-six-month dosing interval” will also give it an edge over other therapies, which are dosed weekly, monthly or once every other month.
William Blair expects Amgen to share full 52-week MINT data at the company’s AAN presentation in April.
Uplizna, approved in June 2020 for the treatment of a rare ocular autoimmune condition called neuromyelitis optica spectrum disorder, is a humanized IgG1 monoclonal antibody that works by targeting the CD19 cell surface antigen, typically found on immature and mature B cells. In myasthenia gravis, Uplizna’s mechanism of action could help deplete the cells that produce the disease-causing auto-antibodies.
In addition to myasthenia gravis, Amgen is also seeking to expand Uplizna into IgG4-related disease, for which an FDA review is currently ongoing with a target action date of Apr. 3, according to a company presentation last month.
