- 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
“The continued expansion of our neuromuscular portfolio demonstrates our commitment to developing innovative medicines for a range of neurological disorders,” said
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,
Additional data from Evrysdi’s comprehensive clinical development programme will also be presented, including:
- Preliminary efficacy and safety data from the RAINBOWFISH study, in pre-symptomatic babies from birth to 6 weeks of age (at first dose), which showed that all babies treated with Evrysdi for one-year or more were alive without permanent ventilation, maintained swallowing and feeding abilities, and had not required hospitalisation.
- Three-year data from the SUNFISH study, further highlighting the long-term efficacy and safety profile of Evrysdi in a broad population of children, teenagers and adults with SMA.
- Three-year pooled safety and efficacy data from Part 1 and Part 2 of the FIREFISH study which showed that after three years of treatment at the pivotal dose (n=58), 84% of infants were alive and did not require permanent ventilation. Overall, babies maintained or improved their motor skills in terms of developmental milestones and motor function between Month 24 and Month 36.
Duchenne muscular dystrophy (DMD)
DMD is a rare X-linked, progressive neuromuscular disease caused by mutations in the DMD gene that disrupts the production of functional dystrophin protein, leading to a loss of muscle function and premature death. It is one of the most common fatal genetic disorders, affecting approximately one in every 3,500 to 5,000 male births worldwide. Results from three ongoing clinical trials of gene therapy delandistrogene moxeparvovec in DMD will be presented:
- Long-term, four-year data from Study 101, an open-label Phase 1/2a study evaluating the safety of delandistrogene moxeparvovec in four ambulatory participants aged between 4-8 years old with DMD. The enduring response and safety observed in the study support the continuation of clinical trials assessing delandistrogene moxeparvovec using single dose gene therapy in patients with DMD.
- One-year data from ENDEAVOR, an open-label Phase 1b study evaluating the expression and safety of commercially representative delandistrogene moxeparvovec in four cohorts of DMD patients, representing different stages of disease progression. One-year safety and functional data and 12-week expression data from Cohort 1 will be presented. These data reinforce our confidence in the probability of success of EMBARK, our first Phase 3 study using intended commercial process material, which is now fully enrolled.
- Integrated analyses comparing data from patients treated with delandistrogene moxeparvovec with an external comparator cohort will be presented, including collective safety data from Study 101, Study 102 and ENDEAVOR.
Generalised Myasthenia Gravis (gMG)
Myasthenia gravis is a rare chronic autoimmune, neuromuscular disease that causes weakness in skeletal muscles. It most commonly affects the muscles that control the eyes and eyelids, facial expressions, chewing, swallowing and speaking as well as limb musculature and muscles of respiration.
Facioscapulohumeral Muscular Dystrophy (FSHD)
FSHD is a rare autosomal dominant genetic disorder characterised by progressive weakening of the skeletal muscles in the face, shoulders, arms, trunk and limbs. There is currently no approved therapy for this disease.
The full range of data from Roche’s clinical development programme in neuromuscular disease being presented at WMS 2022 include:
Medicine | Abstract Title | Presentation Number (type), Presentation Date, Time |
Evrysdi™ (risdiplam) for spinal muscular atrophy | RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic SMA | FP.24 Wednesday 12th October 16.30-16.45 Ballroom |
FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam in Type 1 SMA | P.109 Wednesday 12th October 16.00-17.30 Poster area - Ballroom B1-B2 | |
JEWELFISH: 24-month safety and pharmacodynamic data in non-treatment-naïve patients with SMA | P.110 Wednesday 12th October 16.00-17.30 Poster area - Ballroom B1-B2 | |
SUNFISH Parts 1 and 2: 3-year efficacy and safety of risdiplam in Types 2 and 3 SMA | P.114 Wednesday 12th October 16.00-17.30 Poster area - Ballroom B1-B2 | |
Real-world experience after one year treating SMA children with risdiplam | P.51 Wednesday 12th October 14.30-16.00 Poster area - Ballroom B1-B2 | |
Safety update: Risdiplam clinical trial development program | P.113 Wednesday 12th October 16.00-17.30 Poster area - Ballroom B1-B2 | |
The importance of bulbar and respiratory symptoms in spinal muscular atrophy: Results from interviews with patients, caregivers, and healthcare providers | P.40 Wednesday 12th October 16.00 - 17.30 Poster area - Ballroom B1 - B2 | |
Delandistrogene moxeparvovec for Duchenne muscular dystrophy | Integrated analyses of data from clinical trials of delandistrogene moxeparvovec (SRP-9001) in DMD | P.128 Friday 14th October 14.30-16.00 Poster area - Ballroom B1-B2 |
One-year data from ENDEAVOR, a Phase 1b trial of delandistrogene moxeparvovec (SRP-9001) in boys with DMD | P.129 Friday 14th October 14.30-16.00 Poster area - Ballroom B1-B2 | |
Phase 1/2a trial of delandistrogene moxeparvovec (SRP-9001) in patients with DMD: 4-year update | P.134a Friday 14th October 14.30-16.00 Poster area - Ballroom B1-B2 | |
A Phase 2 clinical trial evaluating the safety and efficacy of delandistrogene moxeparvovec (SRP-9001) in patients with DMD | LSVP.36 Friday 14th October Virtual platform and E-posters | |
Satralizumab for generalised myasthenia gravis | LUMINESCE: Phase 3 study of satralizumab, a therapeutic recycling antibody targeting the IL-6 receptor, in patients with generalised myasthenia gravis | P.92 Wednesday 12th October 16.00-17.30 Poster area - Ballroom B1-B2 |
GYM329 (RO7204239) for facioscapulohumeral muscular dystrophy | MANOEUVRE study design: A study of GYM329 (RO7204239) in patients with FSHD | P.141 Friday 14th October 14.30-16.00 Poster area - Ballroom B1-B2 |
Full session details and data presentations listing for the WMS 2022
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About Evrysdi (risdiplam)
Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube.
Evrysdi is designed to treat SMA by increasing and sustaining the production of the SMN protein in the central nervous system (CNS) and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.
Evrysdi was granted PRIME designation by the
About delandistrogene moxeparvovec
Delandistrogene moxeparvovec (SRP-9001; rAAVrh74.MHCK7.micro-dystrophin) is an investigational gene therapy aimed to deliver the micro-dystrophin-encoding transgene directly to the skeletal and cardiac muscle for the targeted production of the SRP-9001-dystrophin protein to enable a durable clinical response. In
About ENSPRYNG® (satralizumab)
ENSPRYNG, which was designed by Chugai, a member of the
Positive Phase III results for ENSPRYNG, as both monotherapy and in combination with baseline immunosuppressive therapy, demonstrate that IL-6 inhibition is an effective therapeutic approach for neuromyelitis optica spectrum disorder ( NMOSD). ENSPRYNG is currently approved for NMOSD in 72 countries with further applications under review with numerous regulators.
ENSPRYNG has been designated as an orphan drug for gMG, MOGAD, AIE (NMDAR) and NMOSD in
About GYM329 (RO7204239)
GYM329 is an investigational anti-myostatin antibody that is designed to increase muscle growth by binding to inhibiting myostatin, a natural negative regulator of muscle growth. By binding to the protein, GYM329 was created with the aim of blocking the function of myostatin, therefore helping muscles to grow in size and strength.
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