Expert Interview
A third 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: University College London
- Director of the Dubowitz Neuromuscular Centre, a leading clinical and research institution for children affected by neuromuscular disorders in the UK
- His center cares for over 300 patients with DMD
- Currently PI on 9 clinical trials in DMD, SMA, and Myotubular Myopathy
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?
Competitive landscape - are you aware of another phase 2 muscle-specific activating or enhancing agent called NMD670, a ClC-1 ion channel inhibitor? It's trial design also includes adding on top of oral risdiplam or nusinersen, and potentially on top of Zolgensma for added muscle strength and reducing fatigue. This agent has positive initial Phase 2a data in myasthenia gravis added to conventional treatment. What are your thoughts on the success of that approach in SMA in comparison to this approach? Is there differentiation between the two?
Added By: djbnmdAre You Interested In These Questions?
Slingshot Insights Explained
Expert research benefits investors by giving them timely access to unbiased real world perspectives on highly specialized topics. Slingshot Insights' crowdfunded model makes this access available at a fraction of the cost of other expert networks.
Reason
*Slingshot Insights provides access to information, not investment advice. We work to support you and facilitate access to experts; however we are not responsible for monitoring calls for the disclosure of MNPI. You should obtain financial, legal and tax advice from your qualified and licensed advisers before deciding to invest in any security.
