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Scholar Rock Holding Corp (SRRK)·Q1 2025 Earnings Summary

Executive Summary

  • Q1 2025 printed as expected for a pre-commercial biotech: no revenue, net loss of $74.7M ($0.67/share) vs. ($56.9M) ($0.59/share) in Q1 2024; results were roughly in line with S&P consensus EPS of ($0.66), a modest miss of $0.01 per share. Management increased operating spend to fund launch readiness and inventory build-out . Consensus values marked with an asterisk are from S&P Global.
  • Regulatory momentum strengthened: FDA accepted apitegromab’s BLA with Priority Review and set a PDUFA action date of September 22, 2025; EMA validated the MAA. Management now “anticipate[s]” a U.S. launch beginning in Q3 2025, earlier than prior 4Q 2025 expectations, with Europe to follow in 2026 .
  • Commercial build-out progressing: payer outreach underway, ~50 field hires targeted by mid‑2025, and home infusion option planned; cash/marketable securities of $364.4M support operations “into 2027,” up from prior guidance of into 4Q 2026 .
  • Pipeline catalysts: Phase 2 EMBRAZE obesity readout in June 2025 (lean mass preservation on top of tirzepatide), and SRK‑439 IND filing targeted for Q3 2025; OPAL Phase 2 in SMA patients <2 years starts Q3 2025 .

What Went Well and What Went Wrong

  • What Went Well

    • FDA Priority Review with a PDUFA date (Sept 22, 2025) and EMA MAA validation, de-risking the near-term regulatory path; CEO: “our BLA was granted priority review…with a September 22 PDUFA date” .
    • Commercial readiness accelerated: “hiring and onboarding our customer-facing team of roughly 50…personnel is well underway…fully staffed by mid-2025,” and plan to be “out there the next day” post-approval, with ample supply .
    • Strengthened leadership bench (CEO, President of R&D, COO, CFO) to scale for launch; Hallal: “we joined…at a time of great strength and opportunity as we scale for the next phase of growth” .

  • What Went Wrong

    • Higher OpEx to support launch drove a larger quarterly loss: net loss widened to $74.7M vs $56.9M YoY; R&D and G&A rose on commercial manufacturing/launch and headcount .
    • EPS slightly missed S&P consensus by ~$0.01 in Q1 2025; sequential loss per share increased vs Q4 2024 as spending stepped up for commercialization (consensus/actual detail below). Values from S&P Global.
    • Still no product revenue; margin metrics remain not meaningful until commercialization; management refrained from providing quantitative launch guidance and pricing details, citing early stage in payer negotiations and evolving U.S. policy context .

Financial Results

Q1 headline P&L and cash (company-reported)

  • No revenue; net loss $74.7M; EPS ($0.67); OpEx $77.1M; cash/marketable securities $364.4M (Mar 31, 2025) vs $437.3M (Dec 31, 2024) .

Revenue and EPS vs estimates and prior periods

MetricQ3 2024Q4 2024Q1 2025
Revenue ($USD Millions)$0.0*$0.0*$0.0*
Revenue Consensus ($USD Millions)$0.0*$0.0*$0.0*
Diluted EPS ($)($0.66) ($0.61)*($0.67)
EPS Consensus Mean ($)($0.60)*($0.60)*($0.66)*

Values marked with * retrieved from S&P Global.

Operating metrics (YoY and sequential context)

MetricQ1 2024Q4 2024Q1 2025
Research & Development Expense ($M)$43.1 n/a$48.7
General & Administrative Expense ($M)$15.3 n/a$28.4
Total Operating Expenses ($M)$58.4 n/a$77.1
Net Loss ($M)$56.9 n/a$74.7
Cash, Cash Equivalents & Marketable Securities ($M)n/a$437.3 (Dec 31, 2024) $364.4 (Mar 31, 2025)

Notes:

  • Company recorded no revenue in Q1 2025 and Q1 2024; FY 2024 also had no revenue .
  • Gross margin and net margin % are not meaningful without revenue.

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
U.S. launch timing (apitegromab)2025Anticipated 4Q 2025 launch “Anticipate…commence in the U.S. in Q3” (pending approval) Raised (pulled forward)
FDA review2025BLA submitted Jan 2025 (no PDUFA yet) Priority Review; PDUFA 9/22/2025 New milestone/date set
EU review2025–26MAA on track for 1Q 2025 submission MAA validated; EU launch anticipated 2026 Progressed to validation
Cash runwayMulti-yearInto 4Q 2026 Into 2027 Raised
OPAL (SMA <2 yrs)StartMid‑2025 Q3 2025 Maintained/refined
EMBRAZE toplineQ2 2025Q2 2025 June 2025 Maintained/specified
SRK‑439 IND (obesity)Q3 2025Q3 2025 Q3 2025 Maintained

Earnings Call Themes & Trends

TopicPrevious Mentions (Q3 2024, Q4 2024)Current Period (Q1 2025)Trend
Regulatory pathway & labelSAPPHIRE met primary endpoint; plan for BLA/MAA; expectation of broad label across ages per FDA convention Priority Review; PDUFA 9/22/25; EMA MAA validated; interactions “extremely constructive,” still on track Positive momentum
U.S. launch readinessFoundation, payer team build, ~<50 FTEs field plan; home infusion option planned Hiring ~50 customer-facing roles by mid‑2025; payer meetings ongoing; “prepared to be out there the next day” post‑approval; ample supply Accelerating
Payer receptivity/combination therapyEarly payer education; community expects combo motor neuron + muscle approach Early discussions “positive”; payers already cover multiple SMN therapies for some patients Constructive
Pricing/policyNo specifics; value-based narrative building Too early for guidance; value to rarity/severity; MFN EO not altering plans Unchanged cautiously
EMBRAZE obesity PoCEnrollment completed; 24-week lean mass focus; see 20–40% preservation as meaningful Topline June; focus on 24‑week lean mass; 8-week post‑stop data informative but exploratory On track
SRK‑439 (anti‑myostatin, subcu)IND mid‑2025 planned; best‑in‑class preclinical profile IND Q3 2025; optionality across neuromuscular/cardiometabolic On track
Additional neuromuscular indicationsDMD/FSHD models supportive, planning expansion Expanding to DMD/FSHD; OPAL in infants/toddlers Q3 2025 Broadening

Management Commentary

  • “We were gratified that our BLA…was granted priority review by the FDA with a September 22 PDUFA date.” – Akshay Vaishnaw, President of R&D .
  • “Our process of hiring and onboarding our customer-facing team of roughly 50…is well underway. We expect to be fully staffed by mid‑2025…prepared to be out there the next day [post‑approval].” – COO Keith Woods .
  • “We ended the quarter with $364.4 million…We have an additional $100 million under our debt facility…bringing our anticipated runway into 2027.” – CFO Vikas Sinha .

Q&A Highlights

  • Payers: Early U.S. payer discussions “positive,” with recognition of residual muscle degeneration on SMN therapies; precedent exists for coverage of multiple SMN-directed agents; Europe to sequence starting with Germany in 2026 .
  • FDA process: Interactions “extremely constructive,” company remains on track to the PDUFA date; no signals of an AdCom disclosed .
  • Launch kinetics: Expect a steady, formulary-gated ramp; academic centers’ schedules and J‑code timing imply a measured adoption curve despite tailwinds (100% newborn screening; concentrated centers) .
  • Supply/readiness: Launch‑day readiness emphasized; ample supply at launch .
  • Obesity PoC expectations: Primary focus is 24‑week lean mass; anticipate ~25–30% of weight loss is lean mass on tirzepatide alone by week 24; 8‑week post‑treatment follow‑up informative but not definitive .
  • Europe partnering: Not a priority; intent to commercialize directly given team’s global launch experience .

Estimates Context

  • Q1 2025 EPS ($0.67) vs S&P consensus EPS ($0.66): ~($0.01) miss; revenue in line at $0.0 .
  • Trend: Q4 2024 EPS actual ($0.61)* to Q1 2025 ($0.67) reflects increased commercialization spending ahead of launch; Q3 2024 EPS was ($0.66) . Values retrieved from S&P Global.
MetricQ3 2024Q4 2024Q1 2025
EPS Actual ($)($0.66) ($0.61)*($0.67)
EPS Consensus ($)($0.60)*($0.60)*($0.66)*
Revenue Actual ($M)$0.0*$0.0*$0.0*
Revenue Consensus ($M)$0.0*$0.0*$0.0*

Values marked with * retrieved from S&P Global.

Key Takeaways for Investors

  • Near-term catalyst-rich: FDA Priority Review with a definitive Sept 22, 2025 PDUFA date and a June EMBRAZE readout should drive sentiment and volatility; any positive obesity signal (clinically meaningful lean mass preservation with acceptable safety) could expand optionality for SRK‑439 .
  • Launch now guided earlier (Q3 2025) vs prior Q4 2025, signaling confidence in regulatory timelines and operational readiness; expect a measured ramp given formulary/J‑code cycles despite structural tailwinds (concentrated centers, advocacy) .
  • Payer posture appears constructive for combination therapy addressing residual muscle degeneration; pricing will anchor to rarity/severity/value, but specifics likely closer to approval .
  • Investment phase visible in OpEx and sequential EPS; cash runway extended into 2027, plus $100M additional debt capacity pre-approval, providing funding through initial launch and pipeline steps .
  • Broader neuromuscular strategy (DMD/FSHD, OPAL in infants/toddlers) and ex‑U.S. roll-out (Germany first in 2026) support a multi-year growth narrative beyond initial U.S. SMA launch .
  • Risk checks: Regulatory (label/scope, potential AdCom though none signaled), payer/pricing execution, launch ramp cadence, and EMBRAZE translational read-through to SRK‑439 are key watch items .

Footnotes:

  • Company financials and operational details are cited to Scholar Rock’s Q1 2025 press release and call materials and Q1 2025 transcript [3:]. Regulatory press release cited for Priority Review and PDUFA date . Prior-period materials cited where used [6:] [8:] [36:] [26:*].
  • Values marked with * are retrieved from S&P Global (analyst consensus and point-in-time actuals where applicable).