Release Date: August 08, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Scholar Rock Holding Corp (SRRK, Financial) is on track to achieve all key milestones for 2024, including the upcoming top-line results for the SAPPHIRE Phase 3 study in Q4.
- The company has demonstrated sustained clinical benefits and a favorable safety profile for apitegromab in SMA patients over 48 months, reinforcing confidence in its potential.
- SRRK's SRK-439 program in obesity shows promising preclinical data, suggesting best-in-class potential for preserving muscle mass during GLP-1-induced weight loss.
- The company has a robust pipeline with innovative and differentiated products, including SRK-181 for immuno-oncology and SRK-373 for fibrosis, showcasing its deep structural insights and antibody engineering expertise.
- Scholar Rock's research is gaining recognition in the global scientific community, with data featured at major conferences like the American Society of Clinical Oncology and the American Diabetes Association.
Negative Points
- The competitive landscape for SMA treatments is intensifying, with other companies like Roche also advancing similar therapies, potentially impacting market dynamics.
- The obesity program's success is contingent on proving the clinical benefit of muscle preservation, which remains a challenge in the regulatory landscape.
- The company faces the typical risks associated with drug development, including potential delays in clinical trials and regulatory approvals.
- There is uncertainty regarding payer dynamics and market access for apitegromab, which could affect its commercial success.
- The development of SRK-373 and other pipeline assets requires significant investment and resources, which could strain the company's financials if not managed effectively.
Q & A Highlights
Q: How does Scholar Rock view the competition with Roche in the SMA space, and are there any differences in study design or readout timing?
A: Jay Backstrom, President and CEO, explained that Roche is still in the early phases of their study, focusing on dose and effect, with their top-line Phase 3 trial results expected around 2026 or later. Scholar Rock's SAPPHIRE study is agnostic to both nusinersen and risdiplam, unlike Roche, which is only studying with risdiplam.
Q: Regarding apitegromab and SMA, is there any effect attributed to children aging, or is the differential still significant compared to historical clinical studies?
A: Jay Backstrom noted that the safety and tolerability of apitegromab allow for chronic treatment, which is beneficial as children grow. Jing Marantz, Chief Medical Officer, added that the patient population in the TOPAZ study is similar to those in historical studies, reinforcing the observed benefits.
Q: As SAPPHIRE data approaches, what are the potential access or payer dynamics for apitegromab?
A: Jay Backstrom mentioned preliminary payer interactions have been positive, with recognition of the need for additional care for SMA patients. The sustained benefit shown in the TOPAZ data is expected to support payer discussions.
Q: Can you provide more details on the distribution of Hammersmith scores in the 48-month data, and what percent of patients saw at least a 3-point improvement?
A: Jay Backstrom stated that detailed data would be presented at a future medical meeting. The focus is currently on the SAPPHIRE study, with the 48-month data serving as a supportive snapshot.
Q: Will the SAPPHIRE Phase 3 study's label potentially include longer-term data from TOPAZ, and how soon after EMBRAZE data will the IND for SRK-439 be filed?
A: Jay Backstrom indicated that the TOPAZ data will contribute to the safety profile for regulatory review and payer interactions. The IND for SRK-439 is on track for mid-2025, with EMBRAZE data expected to support the filing.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
