诺华第三期 HARBOR 临床试验结果：FSHD 社区最新进展

The FSHD Society extends our support to the myotonic dystrophy community after the announcement that Novartis’ Phase 3 HARBOR trial of del-desiran in myotonic dystrophy did not meet its primary endpoint. A late-stage clinical trial brings incredible hope for a potential new treatment while also requiring tremendous dedication and courage from trial participants and their families. Just as we have experienced in FSHD, disappointments like this are profoundly painful for a community waiting for treatments.

It’s important for our community to understand that del-desiran and del-brax are distinct therapies acting on different biological targets (DMPK RNA in myotonic dystrophy versus DUX4 RNA in FSHD). As shared in their update, “Novartis is planning to meet with the FDA on next steps for delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD) based on recent positive Phase I/II biomarker data.” This reflects that program moving forward on its own path. As the Myotonic Dystrophy Foundation shared in their statement, “Every clinical trial teaches us something. Sometimes the path to a treatment is direct; more often, it is built through one piece of knowledge at a time.” This is a sentiment that we know well from our own trial history. Although del-desiran did not meet its primary endpoint, there is plenty yet to learn as researchers examine the full HARBOR dataset and work to understand how it can be used to guide next steps in the search for effective therapies.

Similarly, our work under the FSHD Industry Collaborative project and in collaboration with trial sponsors like Novartis is focused on learning from community input and previous trial data, including that from the Fulcrum Therapeutics trial of losmapimod in FSHD which failed to meet its primary endpoint. The voices, contributions, and hope of the courageous people who volunteer to participate in clinical trials are more important than ever as we work to use these data to inform the best paths forward.

Most importantly, what connects myotonic dystrophy and FSHD is the strength of our resolve. To the myotonic dystrophy community, know that we see you and we are with you, just as you have stood with us through our own disappointments. We remain hopeful about the road ahead, grounded not in any one result but in steady progress and our shared, unwavering commitment toward effective treatments for our families.