Dear Cure JM Community,

You may have heard by now that the FDA has just approved the first-ever effective drug for myositis treatment. The drug is brepocitinib, now to be marketed as Lisraya, and it works by blocking cellular signals in myositis that lead to inflammation, muscle weakness, and skin rash.

In clinical trials, Lisraya was also shown to reduce or eliminate a patient’s need for daily steroids. As you know all too well, steroids suppress the immune system and can have damaging cumulative side effects. JAK inhibitors like Lisraya, however, provide targeted disease control for both skin and muscle symptoms.

Lisraya represents a class of drug known as JAK inhibitors. Advancing research that leads to a better understanding of JAKs (there are several) and how they work to block inflammation in myositis has long been a priority within Cure JM’s research program, and has contributed to the Lisraya breakthrough.

What’s next? Lisraya is approved for adults (anyone over the age of 18) with myositis and Cure JM is working with all due speed to provide clinical data to the FDA in support of pediatric approval. Concurrently, we are working with our pharma partner, Priovant Therapeutics, to educate and inform JM clinicians on Lisraya’s safety profile and mechanism of action in preparation for pediatric approval. I want to thank Priovant for its dedication to pediatrics as we move toward fast-track clinical review with the FDA.

And what about a cure?

I have also been sharing with you progress of another JM clinical trial for a treatment known as CAR-T therapy. In CAR-T therapy, a JM patient’s own cells—the cells that fight inflammation—are genetically modified to do their job of destroying the aberrant cells that cause inflammation in JM. This clinical trial is fully enrolled with JM patients, some younger than 10 years old.

We know that CAR-T can be an effective therapy and has been used safely in oncology for blood cancers for years. But will it work in inflammatory autoimmune diseases like JM? Early evidence strongly suggests that it does, and the objective of the current clinical trial is to provide medical proof leading to FDA approval.

CAR-T therapy is yet another area where Cure JM’s partnership with a pharmaceutical leader—Cabaletta Bio—will, we believe, result in a treatment that is potentially curative and appropriate for refractory or difficult-to-treat cases of JM. Cabaletta is on the cutting edge of several pharmaceutical companies testing CAR-T products. Their dedication to pediatrics as a Cure JM partner is unmatched, and the company’s intention is to file for FDA approval in pediatric and adult indications simultaneously in 2027. This is an example of pediatrics leading the way!

A Cure Starts With You

You have brought us to this moment.

Cure JM has made extraordinary progress without direct government funding. And

when I say Cure JM, I mean you. I mean the patients, parents, grandparents, and

families who have provided the determination and resources to build our research

network, advance clinical trials, and pursue breakthroughs for children with JM.

None of this progress would have been possible without your commitment to our

mission.

The Cure JM 2026 Holiday Challenge is how that commitment becomes action. Every individual and family who participates provides critical resources to pursue the next study, further the next treatment, and move closer to a cure.

We have come this far because of you. What we accomplish next will depend on you, too.

Please join this year’s Holiday Challenge and help improve the lives of JM kids everywhere.

With gratitude,