Roche to present new data from its expanding neuromuscular

• New positive data from Evrysdi, a treatment for spinal muscular atrophy (SMA), a progressive neuromuscular disease that can be fatal
• Data from the gene therapy programme for Duchenne muscular dystrophy (DMD), a progressive disease that leads to premature death, reinforce confidence in the most advanced Phase 3 study currently underway
• Study designs of two new trials in generalised myasthenia gravis (gMG), a rare chronic autoimmune disease, and facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder

Basel, 05 October 2022 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data from its industry-leading neuromuscular portfolio will be presented at the World Muscle Society (WMS) congress, 11th-15th October 2022. These data demonstrate Roche’s commitment to advancing clinical understanding and supporting the development of treatments for people living with neuromuscular disorders.

“The continued expansion of our neuromuscular portfolio demonstrates our commitment to developing innovative medicines for a range of neurological disorders,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. “Through our ongoing research and development and our strong partnerships with patient communities, we continue to address the needs of people with rare neuromuscular conditions.”

Spinal muscular atrophy (SMA)
SMA is a severe, progressive neuromuscular disease that can be fatal. It is the leading genetic cause of infant mortality, affecting approximately one in 10,000 babies. For the first time, Roche will present new 2-year exploratory efficacy data from the JEWELFISH study in a broad range of patients previously treated with an SMA-targeting therapy, including nusinersen (Spinraza^R) or onasemnogene abeparvovec (Zolgensma^R).

Additional data from Evrysdi’s comprehensive clinical development programme will also be presented, including:

• Preliminary efficacy and safety data from the…