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Home Neuroscience MDA 2023
Roche and Genentech at

MDA 2023

March 19 - March 22
Texas, USA/ Virtual (Hybrid)
mdaconference.org
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Mar 22 / Roche and Genentech
One-year data from ENDEAVOR, a Phase 1b trial of delandistrogene moxeparvovec in patients with DMD
Delandistrogene moxeparvovec is an investigational gene transfer therapy developed for targeted skeletal and cardiac muscle expression of micro-dystrophin (a shortened, functional dystrophin protein) that is being studied in patients with Duchenne muscular dystrophy (DMD). ENDEAVOR (NCT04626674) is a two-part, open-label, Phase 1b study assessing the expression and safety (over 260 weeks) of commercially representative delandistrogene moxeparvovec material in four cohorts of boys with DMD. We present 1-year safety and functional data and 12-week expression data from all patients in Cohort 1 (n=20) following treatment with commercially representative delandistrogene moxeparvovec material.
Neuroscience Duchenne Muscular Dystrophy MDA-2023

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Mar 19 / Roche and Genentech
Safety update: Risdiplam▼ clinical trial program for spinal muscular atrophy (SMA)
Safety data were obtained from FIREFISH (NCT02913482) in infants with Type 1 SMA, SUNFISH (NCT02908685) in patients with Types 2/3 SMA and JEWELFISH (NCT03032172) in patients with SMA who have previously received other disease-modifying therapies. Data were pooled and analyzed per SMA type. Data from RAINBOWFISH (NCT03779334) were collected from presymptomatic patients and presented separately. Collective safety data were analyzed to determine the longer-term safety profile of risdiplam▼ in patients with SMA who have participated in risdiplam▼ clinical trials.
Neuroscience Spinal Muscular Atrophy MDA-2023

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Mar 19 / Roche and Genentech
Integrated analyses of data from clinical trials of delandistrogene moxeparvovec in DMD
Delandistrogene moxeparvovec is an investigational gene transfer therapy developed for targeted skeletal and cardiac muscle expression of micro-dystrophin (a shortened, functional dystrophin protein) that is being studied in patients with Duchenne muscular dystrophy (DMD). The objective of this integrated analysis is to evaluate functional data from patients (≥4 to ≤8 years old) with DMD who have participated in the delandistrogene moxeparvovec studies and compare these data with a control cohort of propensity-matched external control patients. Here we present an integrated analysis of functional data from the 52 patients assessed to date from Study 101 (N=4), Parts 1 and 2 of Study 102 (n=28) who received the target dose (1.33x1014 vg/kg by linear qPCR) of delandistrogene moxeparvovec over 1 year, and patients from Cohort 1 of ENDEAVOR (n=20). Collective safety data from all patients in Study 101, Study 102, and all cohorts of ENDEAVOR (N=84) are also presented.
Neuroscience Duchenne Muscular Dystrophy MDA-2023
12:00 PM
Duration 15mins Chantilly Ballroom West
One-year data from ENDEAVOR, a Phase 1b trial of delandistrogene moxeparvovec in patients with DMD
CM Proud, CM Zaidman, CM McDonald, S Mason, M Guridi, S Wang, C Reid, E Darton, C Wandel, S Lewis, J Malhotra, DA Griffin, RA Potter, LR Rodino-Klapac, JR Mendell
Neuroscience | Duchenne Muscular Dystrophy

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This website is intended for healthcare professionals outside the United Kingdom (UK) and Australia. Registration status and prescribing information of medicinal products may differ between countries. Please refer to local product information for any medicinal products mentioned on this website.