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AGIOS PHARMACEUTICALS, INC. (AGIO)·Q3 2025 Earnings Summary

Executive Summary

  • Q3 2025 net product revenue was $12.88M, up 44% year over year and 3% sequentially, while diluted EPS was $(1.78); revenue and EPS beat consensus estimates of $10.61M and $(1.90), respectively, reflecting continued commercial execution in PK deficiency ahead of potential thalassemia approval . Revenue consensus $10.61M* and EPS consensus $(1.90); Actual EPS $(1.78) vs consensus $(1.90); Actual revenue $12.88M vs consensus $10.61M* (Values retrieved from S&P Global).
  • FDA extended PYRUKYND thalassemia sNDA PDUFA goal date by three months to December 7, 2025 due to a REMS request, not new efficacy/safety data; management reiterated launch preparedness and confidence in benefit-risk profile .
  • CHMP adopted a positive opinion for PYRUKYND in thalassemia; EC decision expected by early 2026; Saudi Arabia approval secured with early patient access initiated via NewBridge .
  • RISE UP Phase 3 sickle cell topline remains guided “by year-end,” a key binary catalyst alongside the Dec 7 PDUFA; management emphasized strong balance sheet of ~$1.26B to fund launches and pipeline .

What Went Well and What Went Wrong

What Went Well

  • Strong PYRUKYND commercial execution: Q3 net revenue rose to $12.88M (+44% YoY; +3% QoQ), with 262 unique patient enrollment forms (+6% QoQ) and 149 patients on therapy (+5% QoQ) .
  • Regulatory momentum outside U.S.: CHMP positive opinion for thalassemia (EU EC decision early 2026) and Saudi Arabia approval, with launch activities underway via NewBridge .
  • Pipeline progress: Completed enrollment in Phase 2b tebapivat for LR-MDS; topline expected early 2026; continued progress on AG-181 (PKU) and AG-236 (PV) programs .

Quoted management lines:

  • “We have multiple high-value catalysts…position PYRUKYND…to achieve its multi-billion dollar potential across PK deficiency, thalassemia, and sickle cell disease.” — Brian Goff, CEO .
  • “Our strong balance sheet with approximately $1.3 billion…positions us to invest…to support potential U.S. launches and advance our rare disease pipeline.” — Brian Goff .
  • “Providers…do not view a potential REMS program as a barrier to prescribing.” — Tsveta Milanova, CCO .

What Went Wrong

  • Wider operating loss driven by R&D and SG&A: Q3 net loss $(103.43)M; R&D $86.80M (+$14.34M YoY) primarily due to PK activation franchise trials; SG&A $41.27M (+$2.74M YoY) ahead of thalassemia launch .
  • U.S. regulatory delay: PDUFA extension to Dec 7, 2025 linked to REMS for potential hepatocellular injury risk; extension not due to new efficacy/safety data but introduces a timing overhang .
  • Revenue base remains small, limiting scale near term; management framed growth as robust but acknowledged small base and sequential increases are modest .

Financial Results

P&L Summary vs Prior Periods

MetricQ1 2025Q2 2025Q3 2025
Net Product Revenue ($USD Millions)$8.73 $12.46 $12.88
Cost of Sales ($USD Millions)$1.09 $1.70 $1.68
Total Operating Expenses ($USD Millions)$115.36 $139.51 $129.75
Net (Loss) Income ($USD Millions)$(89.29) $(112.02) $(103.43)
Diluted EPS ($USD)$(1.55) $(1.93) $(1.78)

Notes:

  • YoY Q3 comparison: Revenue $12.88M vs $8.96M (+44%) .

Balance Sheet Liquidity

MetricDec 31, 2024Mar 31, 2025Jun 30, 2025Sep 30, 2025
Cash, Cash Equivalents & Marketable Securities ($USD Millions)$1,532.03 $1,424.63 $1,339.40 $1,257.20

KPIs (Commercial)

KPIQ1 2025Q2 2025Q3 2025
Unique Prescription Enrollment Forms234 248 262
Patients on Therapy (U.S.)136 142 149

Actuals vs Wall Street Consensus (S&P Global)

MetricConsensusActualSurprise
Revenue ($USD)$10.61M*$12.88M Bold beat
Primary EPS ($USD)$(1.90)*$(1.78) Bold beat

Estimates: Primary EPS Consensus Mean $(1.8961); Revenue Consensus Mean $10.61468M; # of estimates: 8 for revenue and EPS*. Values retrieved from S&P Global.

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
PYRUKYND Thalassemia U.S. PDUFA2025Sep 7, 2025 Dec 7, 2025 Extended
PYRUKYND Thalassemia EU2026Ongoing review CHMP positive; EC decision early 2026 Advanced (positive opinion)
Saudi Arabia Thalassemia2025Under review Approved; early patient access underway Approved/launch initiated
RISE UP Phase 3 (SCD) Topline2025Late 2025/By year-end By year-end Maintained
Tebapivat LR-MDS Topline2026Enrollment completion by end-2025 Topline early 2026 Updated
Commercial Revenue CommentaryQ4 2025N/AQ4 benefits from an additional ordering week; continued focus on PKD Commentary (no numeric range)

Earnings Call Themes & Trends

TopicPrevious Mentions (Q1 & Q2)Current Period (Q3 2025)Trend
U.S. Thalassemia sNDA/PDUFAPDUFA Sept 7; launch prep underway PDUFA extended to Dec 7 due to REMS; launch readiness affirmed Timing extended; confidence maintained
EU/Intl StrategyEU under review; signed Avanzanite; GCC reviews CHMP positive opinion; Saudi approval; early access via NewBridge Regulatory momentum improving
PKD Commercial ExecutionSteady revenue growth; enrollment/therapy up Q3 revenue +3% QoQ; +44% YoY; further account engagement Gradual growth on small base
REMS/Liver MonitoringNot highlighted earlierREMS triggered PDUFA extension; monthly LFT monitoring in Saudi label; providers experienced with REMS Managed risk; operational readiness
RISE UP (SCD) Design/TimingEnrollment completed (Oct 2024); late-2025 topline Topline by year-end; trial positive if either primary endpoint met Near-term binary catalyst
LR-MDS TebapivatEnrollment progression Phase 2b enrollment completed; topline early 2026 Advancing to data in 2026
Capital Allocation/BDCash ~$1.4B-$1.3B; pipeline expansion ~$1.26B cash & investments; BD not tied to readouts; rare disease focus Optionality preserved

Management Commentary

  • “We have multiple high-value catalysts in the coming months…position PYRUKYND…to achieve its multi-billion dollar potential across PK deficiency, thalassemia, and sickle cell disease.” — Brian Goff, CEO .
  • “Third quarter net PYRUKYND revenue was $12.9 million…an increase of 44% YoY and 3% QoQ.” — Cecilia Jones, CFO .
  • “Providers…do not view a potential REMS program as a barrier to prescribing.” — Tsveta Milanova, CCO .
  • “The trial is positive if statistical significance is achieved on either one of the endpoints.” — Dr. Sarah Gheuens, CMO, on RISE UP .

Q&A Highlights

  • REMS specifics: Label details not disclosed during review, but REMS requested for hepatocellular injury; anticipate monitoring and education components .
  • Liver monitoring requirements: Saudi label includes monthly monitoring for first six months; EU details finalized post EC decision .
  • Commercial impact of REMS: Management does not expect REMS to be a barrier; prescribers have substantial REMS experience .
  • International demand cadence: Saudi early access proceeds case-by-case; national procurement may take “a couple of years,” with strong clinician interest .
  • BD strategy: Ongoing pursuit of best-in-class rare disease assets; not tied to RISE UP timing .

Estimates Context

  • Q3 2025 revenue beat: Actual $12.88M vs consensus $10.61M*; EPS beat: Actual $(1.78) vs consensus $(1.90)* . Consensus metrics from S&P Global: Revenue Consensus Mean $10.61468M*, Primary EPS Consensus Mean $(1.8961); # of estimates: 8 for each. Values retrieved from S&P Global.
  • Implications: Modest but clear beat likely driven by incremental patient growth and ongoing PKD execution; near-term estimate revisions may reflect sustained PKD trajectory, with the major estimate reset tied to thalassemia label (Dec 7) and RISE UP outcome by year-end .

Key Takeaways for Investors

  • Two near-term catalysts: Dec 7 PDUFA for thalassemia and RISE UP topline by year-end; stock setup is binary/ event-driven in the next weeks .
  • Commercial base strengthening: Q3 revenue $12.88M (+44% YoY; +3% QoQ) with KPIs trending positively (enrollment +6%, therapy +5% QoQ) .
  • REMS overhang manageable: Extension due to REMS, not efficacy/safety data; provider familiarity reduces adoption risk post-approval .
  • Ex-U.S. optionality: CHMP positive opinion and Saudi approval diversify geographic exposure; Europe commercialization via Avanzanite and GCC via NewBridge .
  • Balance sheet strength: ~$1.26B cash, cash equivalents and marketable securities to fund launches and pipeline; interest income offsets some operating burn .
  • 2026 pipeline visibility: Tebapivat LR-MDS topline early 2026 and potential U.S. launch in SCD 2026 (pending RISE UP) broaden revenue drivers beyond PKD .
  • Trading lens: Expect heightened volatility into PDUFA and RISE UP; favorable REMS/label outcome and at least one positive primary endpoint on RISE UP would be material positive catalysts .

S&P Global disclaimer: Asterisk-marked values are consensus estimates retrieved from S&P Global.