Roche to present new data on neuromuscular portfolio at MDA 2025, highlighting advances in SMA and DMD treatments

Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that it will present new data at the Muscular Dystrophy Association (MDA) conference, 16-19 March, 2025, in Dallas, Texas, from its neuromuscular portfolio, including 12 oral and poster presentations. These data demonstrate the breadth of Roche’s neuromuscular portfolio across spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD).

“These longer-term results are encouraging for people living with SMA and Duchenne, as they demonstrate sustained improvements or stabilisation in mobility and independence for those receiving our disease-modifying treatments,” said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development. “The positive SUNFISH five-year results encompass one of the broadest SMA populations to be studied to date. We are also very encouraged by the data being shared across our Elevidys studies, affirming the potential of the first gene therapy for the treatment of Duchenne to stabilise or slow disease progression.”

Five-year exploratory data from the pivotal SUNFISH study of Evrysdi in people with Types 2 or 3 SMA

After up to five years of treatment in the SUNFISH trial (NCT02908685, n=231), SMA patients on Evrysdi demonstrated overall long-term stabilisation of motor function improvements from baseline that were observed during the first year, as measured by Motor Function Measure 32 (MFM-32). This marks the final readout from SUNFISH, which has reinforced the efficacy, safety and longer-term impact of Evrysdi in a broad population of people with Types 2 and 3 SMA, aged 2-25 years at enrollment. Untreated natural history data presented together with the SUNFISH five-year data show that without treatment, patients from a similar population can experience a significant decline in motor function over the same five-year period.

Patients (>12 years of age) and caregivers both reported continuous improvement or stabilization in levels of independence for performing daily activities, such as dressing, picking up objects and washing, as measured by the SMA Independence Scale (SMAIS-ULM).

Additionally, mean treatment adherence with Evrysdi over the multi-year period was over 99%. The overall rates of adverse events (AEs) and serious adverse events (SAEs) were reflective of the underlying disease and were consistent with previous data. No treatment-related AEs led to withdrawal from the study.

Muscle health and longer-term functional outcomes from individuals with Duchenne’s disease after treatment with Elevidys, vs. well-matched external control groups

Elevidys showed statistically significant and clinically meaningful improvements across key measures of motor function two years after treatment in EMBARK (Phase III, NCT05096221) and three years after treatment in a pooled analysis of Study 101 ( Phase I/II, NCT03375164, n=4), Study 102 (Phase II, NCT03769116, n=26), and ENDEAVOR Cohort 1 (Phase Ib, NCT04626674,  n=20), compared to well-matched external control groups. Collectively, these data demonstrate that treatment with Elevidys results in long-term stabilisation or slowing of disease progression in individuals aged four through eight years of age at the time of treatment.

Topline data from year two of the EMBARK trial were announced in January 2025. No new safety signals were observed in the EMBARK study over the two-year duration.

To further evaluate the effect of Elevidys on disease progression, muscle health and changes in muscle pathology were assessed by magnetic resonance imaging (MRI) in a subset of individuals in EMBARK part one.  At week 52, results showed stabilisation or slowing of disease progression in Elevidys-treated patients compared to placebo-treated patients. At week 104, MRI changes from baseline continued to generally favour Elevidys versus placebo at week 52 across muscles and muscle groups.

 

Further information on the abstracts that Roche will present at MDA 2025 can be found HERE.