Scholar Rock Holding Corp (SRRK)·Q4 2024 Earnings Summary

Executive Summary

• SRRK delivered a catalyst-rich Q4: positive Phase 3 SAPPHIRE data in October, BLA submitted in January, MAA filing on track for 1Q25, and U.S. apitegromab launch targeted for 4Q25, positioning 2025 as a potential inflection year .
• Liquidity was significantly fortified: year-end cash, cash equivalents and marketable securities were ~$437.3M (benefiting from the upsized October equity raise), extending runway into 4Q26 .
• No product revenue; FY24 net loss widened to $246.3M ($2.47/sh) on stepped-up R&D and G&A to advance apitegromab and build commercial capabilities; management emphasized “financial strength” entering 2025 .
• Near-term stock reaction catalysts: detailed SAPPHIRE data presentations (MDA), 2Q25 EMBRAZE obesity readout, and continued commercial readiness milestones toward a 4Q25 U.S. launch .

What Went Well and What Went Wrong

What Went Well

• Pivotal win in SMA: SAPPHIRE met its primary endpoint (HFMSE) with a 1.8-point improvement (p=0.0192) for pooled doses; 30% achieved ≥3-point HFMSE gains vs 12.5% on placebo; safety favorable and consistent with prior experience .
• Regulatory momentum: BLA submitted to FDA in Jan-2025; MAA on track for 1Q25; management is “working with a sense of urgency” toward a 4Q25 U.S. launch if approved .
• Balance sheet strength: completed an upsized ~$300M equity offering in Oct-2024; year-end cash rose to ~$437.3M, supporting launch build and pipeline programs, with runway into 4Q26 .

Management quotes:

• “We submitted the BLA in the U.S. in January, and we remain on track to submit the MAA in the EU in March…planning for a 2025 launch” .
• “Scholar Rock has entered 2025 from a position of financial strength…well positioned to bring apitegromab to patients” .

What Went Wrong

• Continued losses and higher spend: FY24 net loss expanded to $246.3M (vs $165.8M FY23) as R&D rose to $184.6M and G&A to $67.5M with clinical and commercial investments .
• No product revenue yet; Q4 discrete financials not broken out (company reported full-year financials alongside Q4 update) .
• Obesity regulatory path remains evolving: FDA draft guidance maintains BMI/weight endpoints; while FDA encourages lean mass assessment, endpoints for combinations will require added clinical benefit (e.g., A1c or functional measures) .

Financial Results

Quarterly operating metrics

Notes: 9/30/24 pro forma cash including the October raise was ~$463.5M (disclosed for context but not period-end balance) .

Annual comparison

Cash trajectory (2024)

Segment and KPI notes: SRRK is pre-commercial; no revenue segments. Key KPIs are clinical/regulatory milestones and cash runway .

Versus Estimates

S&P Global consensus for Q4 2024 was unavailable at the time of our request. SRRK is pre-revenue; quarterly EPS is not provided separately in the FY release .

Guidance Changes

Earnings Call Themes & Trends

Management Commentary

• Strategic focus and urgency: “We are working with a sense of urgency to bring apitegromab to those living with SMA…submitted the BLA…on track to submit the MAA…planning for a 2025 launch” (Jay Backstrom, CEO) .
• Commercial build: “We will be scaling our customer-facing team of roughly 50 sales, reimbursement and patient support personnel in late Q2 and early Q3…home infusion as an option at launch” (Tracey Sacco, CCO) .
• EMBRAZE objective: “By reducing the amount of lean mass loss by 20% to 40%, we can preserve 1 to 2 kilograms of lean mass, which we believe will translate into clinically meaningful benefit” (Backstrom) .
• Financial footing: “Scholar Rock has entered 2025 from a position of financial strength…well positioned to bring apitegromab to patients” (Ted Myles, COO & CFO) .

Q&A Highlights

• Clinically meaningful lean mass preservation target: management framed 20–40% reduction in lean mass loss at Week 24 (~1–2 kg preserved) as a signal supportive of advancing SRK-439 dosing strategy .
• Weight loss expectations: at Week 24, expect comparable weight loss between apitegromab+tirzepatide and tirzepatide alone; over longer durations, possible incremental benefits via basal metabolic rate and composition .
• Obesity regulatory: FDA draft retains BMI/weight; encourages lean mass assessment; combinations must show added benefit (e.g., A1c reduction, functional improvements), aligning with SRK-439 development strategy .
• Indication expansion: company evaluating adjacent neuromuscular diseases (DMD, FSHD, Becker, ALS) with translational modeling and expert engagement to prioritize PoS and value .
• Payers/pricing: early payer conversations receptive; existing policies for “combination” post gene therapy suggest openness; pricing not disclosed .

Estimates Context

• S&P Global consensus for Q4 2024 revenue and EPS was unavailable at the time of our request; SRRK remains pre-revenue and did not disclose discrete Q4 EPS, providing only FY results .
• Given the lack of product revenue and the focus on clinical/regulatory milestones, near-term estimate revisions are more likely around operating expense trajectories and cash runway assumptions rather than revenue/EPS.

Key Takeaways for Investors

• Pivotal validation and regulatory execution de-risk SMA: positive SAPPHIRE, BLA submitted, MAA imminent; the narrative is now commercialization readiness and label/speed of review .
• 2025 is catalyst-rich: detailed SAPPHIRE data (near-term), EMBRAZE readout in 2Q25, SRK-439 IND in 3Q25, and potential U.S. launch in 4Q25 if approved—an unusually dense set of value events .
• Balance sheet can fund launch and pipeline through key inflections; YE cash ~$437M and runway into 4Q26 mitigate financing overhang near term .
• Obesity program strategy aligns with evolving FDA stance: aim to preserve lean mass and demonstrate added metabolic/functional benefit for combinations, informing SRK-439’s registration path .
• Execution watch items: payer engagement, field build-out, home infusion logistics, and potential OPAL initiation for <2-year-old SMA patients in 3Q25 .
• Risk factors: no revenue base; operating losses increased with commercialization prep; obesity regulatory endpoints and competitive landscape require precise clinical execution .

Supporting Documents and Data

• Q4/FY24 8-K (Item 2.02) and press release (Ex. 99.1): financials, cash, milestones .
• Q4 2024 earnings call transcript: regulatory progress, commercial plan, EMBRAZE/SRK-439 strategy and regulatory views .
• Q3 2024 press release: quarterly P&L, cash, SAPPHIRE topline, EMBRAZE enrollment .
• Q2 2024 press release: quarterly P&L, cash, SAPPHIRE timeline, EMBRAZE guidance .
• SAPPHIRE topline press release (Oct-2024): efficacy and safety details .
• Equity raise (Oct-2024): ~$300M offering pricing .

All figures are sourced from company filings and press releases as cited above.