Novartis said its drug del-desiran did not reach its primary endpoint in the Harbor clinical trial, falling short of the target measure of time required for patients to open their hand—a standard gauge of muscle stiffness in myotonic dystrophy type 1.
The Phase 2 study ran for more than 54 weeks and enrolled roughly 150 patients. While the primary endpoint was missed, the company noted that secondary endpoints and supplementary analyses showed indications of clinical efficacy.
Myotonic dystrophy type 1 is a progressive disease affecting muscles and other organs, with no approved therapy currently available. Del-desiran is an antibody-oligonucleotide conjugate designed to target the underlying molecular cause of the condition.
Novartis said it would now conduct a full analysis of the study data and hold discussions with health authorities on how to proceed with the development program for del-desiran.
Despite the setback, the Basel-based drugmaker reaffirmed its revenue growth forecast of 5 to 6 percent annually for the period 2025 to 2030.
Development of other candidates from the same antibody-oligonucleotide conjugate class is advancing in parallel. The US Food and Drug Administration has opened accelerated review for del-zota, which is being developed for a specific form of Duchenne muscular dystrophy. For del-brax, intended to treat limb-girdle facioscapulohumeral muscular dystrophy, Novartis plans to request a meeting with the FDA to discuss next steps in development.
