Scholar Rock Holding Corp Q4 FY2024 earnings call

Management Statement and Operational Highlights

• Regulatory Milestones: Submitted BLA for apitegromab in the U.S. in January, on track for MAA submission in the EU in March. SAPPHIRE data to be presented at the MDA annual meeting in Dallas on March 19th. OPAL study for SMA under 2 years old to open in Q3.
• Commercial Preparations: Launched 'Life Takes Muscle' SMA disease education campaign. Engaging with top U.S. commercial and federal payers ahead of potential Q4 launch. Scaling customer-facing team in late Q2/early Q3 and preparing home infusion option for launch.
• Cardiometabolic Progress: EMBRAZE study goals include preserving lean muscle mass, safe combination with Tirzepatide, comparable weight loss at week 24, blunting fat regain, and gaining experience in obesity for SRK-439 development. Non-clinical data for SRK-439 shows potential to preserve lean mass and improve metabolic parameters.

Segment Performance

• SMA Program: Submitted BLA in the U.S. for apitegromab in January and is on track to submit the MAA in the EU in March. The SAPPHIRE results showed apitegromab plus standard-of-care delivered a 1.8-point improvement in the Hammersmith Functional Motor scale at week 52, with consistency across age groups 2 through 21. The OPAL study for those under 2 years of age in SMA is set to open in the third quarter.
• Cardiometabolic Program: On track to share top-line data from EMBRAZE in Q2 and file the IND for SRK-439 in the third quarter. EMBRAZE is a Phase 2 proof-of-concept study comparing Tirzepatide plus apitegromab to Tirzepatide plus placebo in obese/overweight adults, focusing on preserving lean muscle mass, weight loss, and metabolic parameters.

Guidance

• Submit BLA in the U.S. for apitegromab in January, and MAA in the EU in March.
• Share top-line data from EMBRAZE in Q2, and file IND for SRK-439 in Q3.
• Open OPAL study for SMA under 2 years old in Q3.

Risks

• Regulatory challenges in obtaining approvals for apitegromab and SRK-439.
• Market competition in the weight management and neuromuscular disease spaces.

Question and Answer

Q: One on SMA, discussing SAPPHIRE data presentation and EMBRAZE primary endpoint.

A: Jay discusses the consistency of SAPPHIRE data across age groups and the importance of lean mass preservation in EMBRAZE.

Q: On obesity readout, thoughts on weight loss biology.

A: Jay states weight loss at week 24 is expected to be comparable, with potential for additional weight loss over time.

Q: Exploration of additional neuromuscular indications for apitegromab.

A: Jay discusses ongoing work with experts, translational models, and the framework for further investment in indications.

Q: FDA guidance on obesity clinical trials and impact on cardiometabolic program.

A: Jay discusses FDA guidance, importance of body composition, and potential for SRK-439 to demonstrate metabolic benefits.

Q: EMBRAZE lean mass preservation and 439 performance, payer discussions, pricing.

A: Jay talks about 439 potential, Tracey discusses payer receptiveness and pricing expectations.

Q: Future development path for muscle preservation drug, strength endpoints.

A: Jay discusses functional measures, regulatory paths, and the stair-stepping approach for 439 development.