March 8, 2022: “Roche announced that new data from its growing neuromuscular portfolio will be presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, 13 – 16 March 2022.
Presentations include eight abstracts from its spinal muscular atrophy (SMA) programme and three from its Duchenne muscular dystrophy (DMD) programme, demonstrating Roche’s commitment to advancing clinical understanding of these conditions with the aim of treating a broad range of people living with neuromuscular disorders.
“These new data further highlight the compelling body of evidence for Evrysdi across infants, children and adults living with SMA,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development.
“The results from our Duchenne Muscular Dystrophy gene therapy programme are encouraging and represent the potential to provide transformative outcomes for people living with this condition.
We are grateful for the partnerships that can help us accelerate progress across SMA and DMD.”
Spinal Muscular Atrophy (SMA)
Six abstracts from the Evrysdi® (risdiplam) clinical development programme will be presented.
This includes 3-year SUNFISH Part 1 and 2 efficacy and safety data, as well as a comparison of 2-year SUNFISH Part 2 data with an untreated external control group, both of which highlight the long-term efficacy and safety profile of Evrysdi in a broad population of people aged 2-25 years with Type 2 or Type 3 SMA.
The data also demonstrate sustained increase in motor function over time, compared to an untreated external control group at 2-years.
Updated interim data from the RAINBOWFISH study will report updated safety data in enrolled infants and efficacy data in infants who have received risdiplam for at least 12 months.
Roche will also be presenting data from four other SMA studies including:
- 24-month data from FIREFISH Part 1 and 2 in SMA Type 1, exploring the safety and efficacy of Evrysdi in infants treated with Evrysdi for 2-years
- 12-month data from the JEWELFISH study, exploring the safety, tolerability and pharmacokinetic/pharmacodynamic (PD) relationship of Evrysdi in a broad age range of patients (6 months to 60 years) who have previously received RG7800 (RO6885247), nursinersen, olesoxime or onasemnogene abeparvovec
- Design of new MANATEE trial, a multi-centre, randomised, placebo-controlled, double-blind study investigating the effect of GYM329, an investigational anti-myostatin, in combination with Evrysdi
- Data on the demographics and clinical characteristics of people with SMA enrolled in the Muscular Dystrophy Association Neuromuscular Observational Research (MOVR) hub
- Using baseline data from FIREFISH, SUNFISH, JEWELFISH and NAtHis-SMA studies, a validated algorithm for estimating the weight of patients with SMA based on few input parameters
Duchenne Muscular Dystrophy (DMD)
Roche has partnered with Sarepta to accelerate access to investigational gene therapy, delandistrogene moxeparvovec (SRP-9001), for DMD outside of the United States upon US Food and Drug Adminstration (FDA) approval.
Sarepta, will present three year data from Study 101, an open-label Phase 1/2a study evaluating the safety of SRP-9001 in four ambulatory participants aged between 4-7 years old with DMD.
The results show an acceptable safety profile, and all patients demonstrated a clinically meaningful improvement in their North Star Ambulatory Assessment (NSAA) from baseline.
In addition, Sarepta will also present results from Study 102 which continue to support SRP-9001’s clinical profile and reinforce a potential benefit risk profile.
Study 102 is a three-part, Phase 2 clinical study evaluating the safety and efficacy of delandistrogene moxeparvovec in patients with DMD.
Analysis of Part 2 of the study will be presented.
Other presentations include:
- The study design and methodology of the first delandistrogene moxeparvovec global Phase III gene therapy trial, EMBARK, assessing the safety and efficacy of commercially representative delandistrogene moxeparvovec material in ambulatory boys with a confirmed DMD mutation aged 4 to 7 years old.
The full range of data from Roche’s clinical development programme in neuroscience being presented at MDA 2022 include:
| Medicine | Abstract Title | Presentation Number (type), Presentation Date, Time |
| Evrysdi® (risdiplam) for spinal muscular atrophy | SUNFISH Parts 1 and 2: 3-year efficacy and safety of risdiplam in Types 2 and 3 SMA | Data presentation Wednesday 16th March 8:20 – 8:35 am CT (14:20 – 14:35 CET) |
| SUNFISH Part 2: 24-month efficacy of risdiplam compared with external control comparators | Posters Sunday 13th March 18:00 – 20:00 CT (02:00 – 04:00 am CET) Monday 14th March 10:00 am – 20:00 CT (16:00 – 02:00 am CET) Tuesday 15th March 10:00 am – 20:00 CT (16:00 – 02:00 am CET) Dedicated Poster sessions between 6:00-8:00pm daily | |
| MANATEE: A study of GYM329 (RO7204239) in combination with risdiplam treatment in pediatric patients with SMA | ||
| Demographics and clinical characteristics of individuals with spinal muscular atrophy enrolled in the Muscular Dystrophy Association MOVR data hub | ||
| FIREFISH Parts 1 and 2: 24-month safety and efficacy of risdiplam in infants with Type 1 SMA | ||
| JEWELFISH: Safety, pharmacodynamic and exploratory efficacy data in non-naïve patients with SMA receiving treatment with risdiplam | ||
| RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic SMA | ||
| Statistical modelling to estimate patients’ weight in Types 1−3 SMA | ||
| SRP-9001 for Duchenne muscular dystrophy | EMBARK study design: Phase 3 trial evaluating the safety and efficacy of delandistrogene moxeparvovec (SRP-9001) in Duchenne muscular dystrophy | |
| Phase 1/2a trial of delandistrogene moxeparvovec (SRP-9001) in patients with Duchenne muscular dystrophy: 3-year safety and functional outcomes | ||
| A Phase 2 clinical trial evaluating the safety and efficacy of delandistrogene moxeparvovec (SRP-9001) in patients with Duchenne muscular dystrophy |
Full session details and data presentations listing for the MDA Clinical and Scientific Conference can be found at the meeting website: https://mdaconference.org/
Follow Roche on Twitter via @Roche and keep up to date with MDA 2022 Conference news and updates by using the hashtag #RocheAtMDA2022, #MDA and #neuromuscular”
