Expert Interview
A second look: Discussing Scholar Rock's SMA treatment apitegromab, a selective inhibitor of the activation of latent myostatin, and the TOPAZ and SAPPHIRE trials
Ticker(s): SRRKInstitution: Children's Hospital of Richmond (at VCU)
- Assistant Professor, Division of Child Neurology at VCU & Dual-board certified child neurologist and neuromuscular physician.
- Currently treats 60 to 70 patients with DMD.
- Clinical and research interests are in pediatric neuromuscular disorders with a particular focus on Duchenne Muscular Dystrophy; has served as the PI of several clinical trials in DMD and site investigator for Exon 51.
Roughly how many patients with type 2/3 SMA do you treat?
Added By: wilson_adminHow do you tend to treat type 2 patients? Type 3?
Added By: wilson_adminWhat are your high level thoughts on apitegromab?
Added By: wilson_admin Updated PhII SMA data from the TOPAZ study showed sustained functional improvement. What shall we expect at the 36 months follow up? Can one look for further improvement in function, beyond 4-5 average increase on average on top of Spinraza? Would it be a worrisome signal if we see a drop in functional improvement at 36 months? Anything particular regarding safety to look out for at 36 months disclosure
SRRK reported dose response is seen in pts treated at 2mg/kg at 1 year switching to 20mg/kg in year 2 -> what is the level of technical derisking that this data set brings? Shall we get excited about this intra-patient dose-effect?
Competitive landscape – how shall we think about the differentiation versus ROG (RO7204239) and BHVN (Taldefgrobep alfa) anti-myostatin programs? Roche started a Phase II/III SMA antimyostatin antibody (RO7204239) study on top of oral Risdiplam. It is encouraging to see Roche invest in anti-myostatin on top of an SMN corrector (talks that Big Pharma also sees here unmet medical need). We are not aware of any Phase I/II public data presentations for this program. Is there anything KOL can say about this ROG study?
Technical risk: level of confidence that the Apitegromab is going to work as an add-on across all approved therapies for SMA, as it was tested only on top of Spinraza in the TOPAZ study (PhII). Is there any reason to be concerned based on the biology of disease and mode of Action of approved drugs?
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