Scholar Rock (SRRK) Q2 2024 earnings summary

Executive summary

• Achieved or exceeded all key milestones in H1 2024, with pivotal SAPPHIRE Phase 3 data for apitegromab in SMA expected in Q4 2024 and potential commercial launch in 2025 if successful.

• Advanced a robust pipeline, including anti-myostatin and TGF-beta1 programs targeting SMA, obesity, immuno-oncology, and fibrosis, with multiple candidates in clinical and preclinical stages.

• EMBRAZE Phase 2 obesity study enrollment ahead of schedule, with top-line results expected in Q2 2025.

• Long-term data from the TOPAZ study show sustained motor function improvement and favorable safety for apitegromab in SMA over 48 months, with >90% patient retention.

• Preparing for commercialization and regulatory submissions, including PRIME pre-submission meeting with EMA and potential product launches through 2025.

Financial highlights

• Net loss for Q2 2024 was $58.5 million ($0.60/share), up from $37.9 million ($0.47/share) in Q2 2023, driven by increased R&D and G&A expenses.

• Operating expenses for Q2 2024 were $59.5 million, a 52% increase from Q2 2023, mainly due to higher clinical trial and employee costs.

• Cash, cash equivalents, and marketable securities totaled $190.5 million as of June 30, 2024, expected to fund operations into the second half of 2025.

• No product revenue was generated; all revenue to date has been from terminated collaborations.

• G&A expenses increased 40% year-over-year in Q2 2024, primarily from higher headcount and commercial readiness activities.

Outlook and guidance

• SAPPHIRE Phase 3 top-line results for apitegromab in SMA expected in Q4 2024, with potential launch in Q4 2025 if successful.

• EMBRAZE Phase 2 obesity study top-line data anticipated in Q2 2025.

• IND filing for SRK-439 in obesity planned for 2025; IND for SRK-373 in fibrosis expected in 2026.

• Existing cash is expected to fund operations into the second half of 2025, but additional capital will be required to complete clinical development and commercialization.

• Study in SMA patients under 2 years old planned for 2025, with design endorsed by EMA's pediatric committee.