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AGIOS PHARMACEUTICALS, INC. (AGIO)·Q2 2025 Earnings Summary

Executive Summary

  • Q2 2025 net product revenue was $12.46M, up 45% y/y and 44% q/q, and above Wall Street consensus of $9.52M; diluted EPS was -$1.93 vs consensus -$1.79, reflecting higher R&D (incl. a $10M milestone) and SG&A ahead of thalassemia launch. CFO cited an extra ordering week and more specialty pharmacy units as drivers of the revenue strength. Bold: revenue beat; EPS miss . Estimates marked with asterisk; values retrieved from S&P Global.
  • Management reiterated near‑term catalysts: PYRUKYND sNDA for thalassemia (PDUFA Sep 7, 2025) and RISE UP Phase 3 sickle cell topline by year‑end 2025; Avanzanite partnership expands European commercialization .
  • Operating expenses elevated: R&D $91.94M (incl. $10M regulatory milestone to Alnylam) and SG&A $45.87M; cash, cash equivalents and marketable securities at $1.339B (June 30) vs $1.425B (March 31) and $1.532B (Dec 31) .
  • Commercial KPIs strengthened: 248 unique patient enrollment forms (+6% q/q) and 142 patients on therapy (+4% q/q), positioning for thalassemia launch preparedness .

What Went Well and What Went Wrong

What Went Well

  • PYRUKYND revenue grew to $12.46M, +45% y/y and +44% q/q, with management attributing strength to ordering dynamics and specialty pharmacy processing volume .
  • Thalassemia launch preparedness: CEO emphasized “2025 is shaping up to be a breakout year” and that the team is “less than 40 days” from PDUFA, with robust KOL and advocacy engagement; CCO detailed focus on ~4,000 actively managed adult patients in the U.S. .
  • Ex‑U.S. leverage: Agios signed an exclusive pan‑European commercialization pact with Avanzanite to distribute PYRUKYND across the EEA, UK, and Switzerland, enabling capital‑efficient expansion .

What Went Wrong

  • EPS missed consensus: diluted EPS was -$1.93 vs -$1.79*; net loss widened q/q to $112.02M as R&D rose on a $10M milestone and SG&A scaled ahead of thalassemia launch .
  • Near‑term product demand caution: CFO expects q/q variability in PYRUKYND net revenues and “softer PKD demand” during U.S. field transition to thalassemia, limiting 2025 revenue uplift (only “modest growth” vs 2024) .
  • Safety monitoring burden persists: monthly LFTs for six months across protocols following hepatocellular injury observations in thalassemia; risk language will be updated in final label, maintaining a warning/precautions posture .

Financial Results

MetricQ4 2024Q1 2025Q2 2025
Net Product Revenue ($USD Millions)$10.70 $8.73 $12.46
Cost of Sales ($USD Millions)$1.30 $1.09 $1.70
Gross Margin %87.9% (calc. from revenue & CoS) 87.6% (calc.) 86.3% (calc.)
R&D Expense ($USD Millions)$82.80 $72.74 $91.94
SG&A Expense ($USD Millions)$51.70 $41.53 $45.87
Interest Income, net ($USD Millions)N/A$16.09 $14.51
Net Loss ($USD Millions)$96.50 $89.29 $112.02
Diluted EPS ($)N/A-$1.55 -$1.93
KPIQ4 2024Q1 2025Q2 2025
Unique Patient Enrollment Forms223 234 248
Patients on PYRUKYND Therapy130 136 142
Balance SheetDec 31, 2024Mar 31, 2025Jun 30, 2025
Cash, Cash Equivalents & Marketable Securities ($USD Millions)$1,532.03 $1,424.63 $1,339.40
Total Assets ($USD Millions)$1,663.20 $1,555.52 $1,471.24
Stockholders’ Equity ($USD Millions)$1,540.96 $1,466.53 $1,369.56
Segment BreakdownQ4 2024Q1 2025Q2 2025
PYRUKYND Net Product Revenue ($USD Millions)$10.70 $8.73 $12.46

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Net RevenuesFY 2025Not provided (numeric) “Modest growth vs 2024”; q/q variability; thalassemia revenues not material in 2025 New qualitative guidance
SG&AFY 2025Scaling ahead of launch Bulk built; expect some additional launch‑related expenses upon approval Raised (launch‑related)
R&DFY 2025Ongoing trials (tebapivat, RISE UP) Q2 included a $10M milestone to Alnylam for AG‑236; continued investment in pipeline Elevated in Q2
Thalassemia sNDA (U.S.)2025PDUFA Sep 7, 2025 PDUFA Sep 7, 2025; team launch‑ready Maintained
SCD Phase 3 (RISE UP)2025Topline late 2025; potential U.S. launch 2026 Topline by year‑end; potential U.S. launch 2026 Maintained
Cash RunwayMulti‑yearSufficient to fund launches & pipeline $1.339B cash; “financial independence” reiterated Maintained

Earnings Call Themes & Trends

TopicPrevious Mentions (Q4 2024, Q1 2025)Current Period (Q2 2025)Trend
Thalassemia Regulatory & Launch PrepFiled sNDA across U.S., EU, KSA, UAE; PDUFA Sep 7, 2025 “Breakout year”; <40 days to PDUFA; detailed U.S. targeting of ~4,000 actively managed adult patients Steady progress; operational readiness rising
Sickle Cell Disease (RISE UP)Completed Phase 3 enrollment; topline late 2025 On track for topline by year‑end; dual primary endpoints; consistent trial design across phases Maintained timelines; reinforced design rigor
Ex‑U.S. CommercializationGCC and EU filings; partnerships being formed Avanzanite exclusive pan‑Europe deal; GCC launch path via NewBridge Acceleration; capital‑efficient ex‑U.S.
Safety/Label (Hepatocellular Injury)PKD label includes warning; thalassemia observations informed PKD Monthly LFT monitoring for 6 months; label updates at approval; expectation warning/precautions posture continues Risk management sustained
SG&A BuildoutSG&A elevated for launch prep Bulk built last year; incremental launch expenses to come on approval Near‑term step‑up likely
Commercial KPIsQ4 2024: 223 forms, 130 treated; Q1 2025: 234, 136 Q2 2025: 248 forms, 142 treated; variable ordering expected Steady growth; variability flagged

Management Commentary

  • CEO: “2025 is shaping up to be a breakout year for Agios… we are now less than 40 days from our September 7 PDUFA goal date” .
  • CFO: “Sequential net revenue growth reflects… an extra week of ordering in the second quarter and an increase in the number of units processed directly by the specialty pharmacy… we expect continued quarter‑on‑quarter variability” .
  • CFO: “Pending approval for thalassemia in the U.S., we expect softer PKD demand… on a full‑year basis we expect net revenues in 2025 to show modest growth compared to 2024” .
  • CCO: “Our initial launch is focused on the 4,000 patients that are actively managed… includes both transfusion and non‑transfusion dependent patients who experience complications” .
  • CMO: “We do at minimum anticipate updates in the PKD label to reflect the indication statement and the dose to be changed to thalassemia… final label… we will know at the PDUFA date” .

Q&A Highlights

  • Safety and label: No new safety updates outside thalassemia; hepatocellular injury risk management continues; label update anticipated at PDUFA, likely remaining in warnings/precautions .
  • SG&A trajectory: Most infrastructure built; expect additional launch‑related spend post‑approval .
  • Target population and uptake: U.S. adult thalassemia ~6,000 diagnosed; ~4,000 actively managed prioritized at launch; claims data and ICD‑10 enable precise account targeting .
  • Pediatric strategy: Adult benefit‑risk first; pediatric trials to follow and support broader filings over time .
  • Ex‑U.S. commercialization: GCC approvals pursued with NewBridge; pan‑Europe via Avanzanite; country‑by‑country strategies tailored to prevalence and access .

Estimates Context

MetricQ2 2025 ConsensusQ2 2025 ActualSurprise
Revenue ($USD)$9.52M*$12.46M Bold: beat
Primary EPS ($)-$1.79*-$1.93 Bold: miss
# of EPS Estimates7*N/A—
# of Revenue Estimates8*N/A—

Values retrieved from S&P Global.

Management attribution: revenue upside driven by ordering calendar (extra week) and specialty pharmacy unit processing; EPS pressure from $10M regulatory milestone (AG‑236) and commercial build ahead of thalassemia launch .

Key Takeaways for Investors

  • Revenue beat with clear operational drivers (ordering cadence, specialty pharmacy), but management warns of intra‑quarter variability and softer PKD demand during the thalassemia pivot .
  • EPS miss reflects planned investment: $10M AG‑236 milestone and scaled SG&A for launch readiness; expect incremental launch spend if approval arrives in September .
  • U.S. thalassemia launch poised to target ~4,000 actively managed adult patients; payers engaged and sales force doubled to ~40—near‑term revenue contribution in 2025 expected to be immaterial due to timing .
  • Ex‑U.S. commercialization de‑risked via Avanzanite (Europe) and NewBridge (GCC), supporting capital‑efficient global scale as regulatory decisions roll out .
  • Safety/label watch: hepatocellular injury risk language expected to be addressed in the final thalassemia label; monthly LFT monitoring embedded across protocols—label outcome is a key sentiment driver around PDUFA .
  • Cash of $1.339B provides ample runway to fund launches and pipeline, with reiterated “financial independence” to advance programs and opportunistically expand assets .
  • Tactical setup: near‑term catalysts (PDUFA Sep 7, RISE UP topline by year‑end) are likely stock movers; sentiment will hinge on label details, launch execution, and SCD efficacy endpoints .

Appendix: Additional Contextual Press Releases

  • Q2 press release reiterating revenue and pipeline milestones (duplicate of Exhibit 99.1 content) .
  • Q2 webcast notice (structural; no financial changes) .
  • Pan‑European Avanzanite partnership details (June 9) .