The Evolving Landscape of Myasthenia Gravis Therapies: A Q&A With Miriam Freimer, MD

Author(s):

Miriam Freimer, MD, clinical professor of neurology at The Ohio State University Wexner Medical Center, reflects on how findings from the RAISE-XT trial potentially translate to real-world practice and looks to the future of treatment for the autoimmune neuromuscular disorder.

A follow-up study presented at the MGFA 15th International Conference on Myasthenia and Related Disorders examined corticosteroid tapering among patients with myasthenia gravis who were currently being treated with zilucoplan. The RAISE-XT analysis (NCT04225871) was the long-term extension, over 120 weeks, of the RAISE trial (NCT04115293). In this final part of her interview with The American Journal of Managed Care^® (AJMC^®), study author Miriam Freimer, MD, clinical professor of neurology at The Ohio State University Wexner Medical Center, reflects on these findings and how they potentially translate to real-world practice, as well as looks to the future of treatment for the autoimmune neuromuscular disorder.

Findings from the long-term study show that close to 30% of patients were able to lower or stop their nonsteroidal immunosuppressive therapy by week 120.

Freimer also currently directs Ohio State’s Myasthenia Gravis Clinic and Electromyography Laboratory.

Revisit these earlier segments of Freimer's interview:

Part 1: Understanding How Complement C5 Inhibitors Work in MG: Miriam Freimer, MD

Part 2: Zilucoplan’s Edge in Complement Inhibition in Myasthenia Gravis: Miriam Freimer, MD

This transcript was lightly edited for clarity.

AJMC: In real-world practice, what challenges might arise when tapering therapies and how must doctors proceed to not cause flare-ups?

Freimer: Let’s start with steroids, because I think that how patients respond to tapering is different. Some of it for prednisone depends on how long the patient’s been on the drug. One of the things that happens with prednisone is that you can cause the adrenal glands, which are the gland body, which makes endogenous cortisol, you can cause those glands to not work as well outside steroids, and so they don’t need to produce as much for the body. Patients become very dependent on being on steroids—so if it’s been a long period of time that someone’s been on steroids, it can be harder to taper prednisone off, and it can vary from patient to patient in who has had their adrenal glands stop producing steroids.

Patients like the way they feel a lot of times. But what I’ve had patients say to me is, “I didn't have any muscle aches or pains. My joints didn’t hurt when I was on prednisone,” and that’s one of the things that prednisone helps with. So all of a sudden you start tapering prednisone, and symptoms of arthritis start to emerge, and patients don’t necessarily like that, understandably. Sometimes it can be hard to completely stop the prednisone in terms of tapering the nonsteroidal immunosuppressants. Those medications stay in the system for a long time in terms of their impact on white blood cell function, so you can’t taper those very quickly because you don’t know what that patient’s immune system is going to do. We tend to taper them more slowly, and you want to wait a few months to 6 months to make sure someone’s not worsening, because all of a sudden you’ve improved how their immune system works.

I’m not sure that’s any different than how it was done in the study. I don’t think anybody aggressively tapered medications, but we were allowed to do it at our own pace, and so it was very much tailored to the individual patient. And patients want to get off these medications. They’re tired of taking medications, and sometimes they want to get off really quickly, and sometimes quickly is not the best way to do it

AJMC: With the recent approval of nipocalimab, how do you view the future of drug development for patients living with myasthenia gravis?

Freimer: Because there’s success in medications, other companies have been very interested in developing approaches. There are several more complement inhibitors that are being evaluated that impact the complement system a little bit differently. There’s a class of drugs, and the FcRns [Fc receptor blockers] There’s a few more. Another one just came on the market, and there’s another one being studied. There are also medications that take totally different approaches. In these that are being looked at, one which may get approved in the next 6 to 12 months, takes a totally different approach. I think this is really exciting for patients with myasthenia gravis. There are going to be lots of options that are approved by the FDA, which means, hopefully, that insurance companies will pay for them.

Nipocalimab was just approved. There’s also a B-cell inhibitor that had very positive results. That’s the MINT trial (NCT04524273). I don’t know about the process and where it is in terms of FDA approval, but there are some other drugs those categories, too. It’s a very promising and exciting time.

Reference

Hewamadduma C, Freimer M, Genge A, et al. Corticosteroid dose tapering during treatment with zilucoplan in patients with generalised myasthenia gravis: 120-week followup of RAISE-XT. Presented at: MGFA 15th International Conference on Myasthenia and Related Disorders, May 13-15, 2025; Den Haag, The Netherlands.

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