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Abstract Details

JEWELFISH: Safety and Pharmacodynamic Data in Non-Naïve Patients with Spinal Muscular Atrophy (SMA) Receiving Treatment with Risdiplam
Child Neurology and Developmental Neurology
Child Neurology and Developmental Neurology Posters (7:00 AM-5:00 PM)
064

To determine the safety, tolerability and pharmacokinetics/pharmacodynamics (PK/PD) relationship of risdiplam in non-naïve (previously treated with other SMA therapies) patients with spinal muscular atrophy (SMA).

SMA is a severe, progressive neuromuscular disease caused by reduced levels of survival of motor neuron (SMN) protein due to deletions and/or mutations of the SMN1 gene. A second SMN gene, SMN2, produces only low levels of functional SMN protein. Risdiplam is a centrally and peripherally distributed oral SMN2 pre-mRNA splicing modifier that increases levels of functional SMN protein. Risdiplam (EVRYSDI™) has been approved by the FDA for the treatment of patients with SMA, aged 2 months and older.
JEWELFISH (NCT03032172) is an ongoing, multicenter, open-label study evaluating the safety, tolerability and PK/PD relationship of daily oral risdiplam in non-naïve patients with SMA (inclusion criteria 6 months–60 years at enrollment). JEWELFISH participants previously received RG7800 (RO6885247), nusinersen (SPINRAZA®), olesoxime or onasemnogene abeparvovec-xioi (ZOLGENSMA®).

We have previously presented safety data from 173 patients with SMA (data-cut: 31st January 2020) who received risdiplam for up to 32.8 months (13 patients previously received RG7800, 76 patients received nusinersen, 14 received onasemnogene abeparvovec and 70 patients received olesoxime). No treatment-related safety findings leading to withdrawal were reported. The overall AE profile of risdiplam treatment in non-naïve patients was consistent with that of treatment-naïve patients. Previously presented JEWELFISH PD data showed a ≥2-fold increase in median SMN protein levels versus baseline (data-cut: 01 June 2020), which was consistent with PD data from the SUNFISH study (NCT02908685) in treatment-naïve patients with Type 2/3 SMA.

We will present updated safety and PD data from patients in the JEWELFISH study
The JEWELFISH study is ongoing in sites across Europe and the US and will provide important data on the safety and PD of risdiplam in non-naïve patients with SMA.
Authors/Disclosures
Claudia A. Chiriboga, MD, FÂ鶹´«Ã½Ó³»­ (Columbia University)
PRESENTER 		Dr. Chiriboga has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentec. Dr. Chiriboga has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. The institution of Dr. Chiriboga has received research support from Roche. The institution of Dr. Chiriboga has received research support from Avexis/Novartis. The institution of Dr. Chiriboga has received research support from Biogen. The institution of Dr. Chiriboga has received research support from NIH. The institution of Dr. Chiriboga has received research support from Biohaven. The institution of Dr. Chiriboga has received research support from Genentec. Dr. Chiriboga has received publishing royalties from a publication relating to health care.
Claudio Bruno Claudio Bruno has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Claudio Bruno has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Claudio Bruno has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta.
Tina Duong Tina Duong has received personal compensation for serving as an employee of Roche. Tina Duong has received personal compensation for serving as an employee of Novartis. Tina Duong has received personal compensation for serving as an employee of Scholar Rock. Tina Duong has received personal compensation for serving as an employee of Genentech. Tina Duong has received personal compensation for serving as an employee of Novartis. Tina Duong has received personal compensation for serving as an employee of Sarepta. Tina Duong has received personal compensation for serving as an employee of Biogen. Tina Duong has received personal compensation for serving as an employee of Audentes. Tina Duong has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Tina Duong has received personal compensation in the range of $0-$499 for serving as a Consultant for Audentes. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Genentech. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta. Tina Duong has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for biogen. The institution of Tina Duong has received research support from biogen. The institution of Tina Duong has received research support from scholar rock.
Dirk Fischer The institution of Dirk Fischer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Hoffmann La Roche AG. The institution of Dirk Fischer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hoffmann La Roche AG.
Janbernd Kirschner No disclosure on file
No disclosure on file
No disclosure on file
Ksenija Gorni Dr. Gorni has received personal compensation for serving as an employee of Hoffman La Roche.
Heidemarie Kletzl Heidemarie Kletzl has received personal compensation for serving as an employee of Hoffmann-La Roche.
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file