Scholar Rock Key Takeaways on Apitegromab from SMA KOL Roundtable & Mgmt Catch Up

Research evidence excerpt

Scholar Rock Key Takeaways on Apitegromab from SMA KOL Roundtable & Mgmt Catch Up

ent group. Taking a step back, we believe that the BLA term positivity or caution based on a

for apitegromab should be approved on or before its PDUFA date (strengthened by specific catalyst. As such, it can differ from

the inclusion of a second U.S.-based fill-finish facility in the BLA; our note from the analysts’ published ratings, which are

early May here) and anticipate a strong launch, and ultimately, we see apitegromab relative to their coverage universe and six

as having multi-billion dollar revenue potential in SMA alone. Scholar Rock to 12 months in duration.

remains on our Positive Catalyst Watch.

• On the clinical data for apitegromab… He emphasized the integrity of the

phase 3 SAPPHIRE trial, calling it “fairly conducted”, and viewed the data as

robust and clear, showing a difference between the treatment and placebo arms

“whatever way you look at the population.” He added: “If you see it in the

younger, you see in the older, it doesn't seem to work even better in the younger

than the older, which is quite a surprise.” He did express that he sees the 10mg/

kg and 20mg/kg higher dosing as equal from a PK perspective.

• On expected uptake of apitegromab... Dr. Servais estimates that ~85%-90%

of eligible children and ~65%-70% of eligible adults will go on the drug, but

expects that not all patients will remain on the drug, potentially driven by some

patients being disappointed in the case that they perceive moreso stabilization

vs a massive functional improvement. However, based on the longer term data

we have seen thus far from the SAPPHIRE trial experience/long-term

extension patient retention rates, apitegromab’s benefit in improving muscle