AGIOS PHARMACEUTICALS, INC. (AGIO)·Q1 2025 Earnings Summary

Executive Summary

• Q1 2025 revenue of $8.7M and EPS of $(1.55) reflected a sequential step-down from Q4 due to year-end stocking and reserve adjustments in Q4, but modest growth year/year; revenue missed consensus while EPS beat as operating losses were lower than feared . Consensus: revenue $9.68M*, EPS $(1.75). Actuals: revenue $8.73M, EPS $(1.55). Result: revenue miss, EPS beat. Values retrieved from S&P Global.
• Patient funnel and persistence improved: 234 unique prescription enrollment forms (+5% q/q) and 136 patients on therapy (+5% q/q), supporting a healthier base ahead of potential thalassemia launch .
• Key catalysts intact: thalassemia sNDA under active FDA review with no advisory committee planned and a PDUFA goal date of Sep 7, 2025; Phase 3 RISE UP sickle cell readout remains on track for late 2025 (potential U.S. launch in 2026) .
• Balance sheet strength continues to be a strategic advantage ($1.42B cash and securities), enabling “financial independence” to fund launches and pipeline; Mgmt expects 2025 PK deficiency revenue to be “relatively flat” as teams focus on thalassemia commercialization readiness .

What Went Well and What Went Wrong

• What Went Well

  • Regulatory momentum: FDA review for thalassemia is “progressing as expected,” with no advisory committee planned; PDUFA Sep 7, 2025 .
  • Commercial readiness: Enrollment forms and on-therapy patients grew 5% q/q; sales force doubled vs PKD footprint; payer engagement ongoing with positive feedback on unmet need/product profile .
  • Pipeline execution: RISE UP Phase 3 in sickle cell fully enrolled; Phase 2 tebapivat in LR-MDS progressing; mid-’25 start of tebapivat Phase 2 in SCD on track; positive ACTIVATE‑Kids topline for pediatric PK deficiency .

• What Went Wrong

  • Revenue miss and sequential decline: $8.7M vs $9.68M consensus and down 19% q/q due to Q4 stocking/reserve adjustments; highlights ongoing variability at early-stage commercial scale . Values retrieved from S&P Global.*
  • Opex elevated into launch: SG&A up to $41.5M (from $31.0M y/y) driven by launch prep; R&D $72.7M with LR‑MDS and SCD study costs; sustaining near-term losses (net loss $(89.3)M) .
  • Visibility to 2025 revenue trajectory limited: Management expects PKD revenue “relatively flat” and cautions thalassemia’s initial contribution will be a partial Q4 due to payer setup and time from enrollment to therapy initiation .

Financial Results

Notes: Gross margin calculated from revenue and cost of sales using reported figures.

Versus estimates

• Q1 2025 Revenue: $8.73M vs $9.68M consensus → Miss*
• Q1 2025 EPS: $(1.55) vs $(1.75) consensus → Beat*
  Values retrieved from S&P Global.*

KPIs

Segment breakdown: Not applicable; revenue consists of PYRUKYND product revenue .

Guidance Changes

Earnings Call Themes & Trends

Management Commentary

• “We look at 2025 as a breakout year for Agios as we focus on three key priorities: first, maximizing the potential of the PYRUKYND franchise… second, advancing and diversifying our key pipeline programs; and third, strategically focusing our capital deployment to sustain and drive our growth.”
• “The FDA has communicated that at this time no advisory committee meeting is planned, and the review is ongoing.”
• “In the first quarter of 2025, we generated $8.7 million… revenues decreased by 19%, primarily due to the benefit of year-end stocking and adjustments to certain revenue reserves [in Q4].”
• “Gross to net has generally been and is expected to be in the 10% to 20% range on an annual basis… with quarter-to-quarter variability.”
• “We have strategically grown the sales organization to approximately twice [the PKD] size… [and] are actively engaging and educating payers… Feedback… has been positive.”

Q&A Highlights

• FDA process: Management reiterated consistent engagement; no Ad Comm planned to date; labeling negotiations typically later in the cycle; cannot rule in/out REMS until approval .
• SCD landscape after Oxbryta withdrawal: No observed changes in trial conduct; underscores unmet need and supports Agios’ view that the market can absorb multiple therapies .
• ex-U.S. strategy/economics: GCC prioritized via NewBridge with revenue-split arrangement; expect EU structure “very similar” (capital efficient) .
• Pricing: Specifics to come post-approval; anchored to value/label; payers recognize unmet need; non-managed rare disease category expected .
• Portfolio strategy: Start tebapivat Phase 2 in SCD before RISE UP readout to accelerate proof-of-concept and future Phase 3 dose selection; no liver signal observed to date for tebapivat .

Estimates Context

• Q1 2025 vs consensus: Revenue $8.73M vs $9.68M (Miss); EPS $(1.55) vs $(1.75) (Beat).* Values retrieved from S&P Global.*
• Recent history vs consensus:
  • Q4 2024: Revenue $10.73M vs $9.35M (Beat); EPS $(1.74) vs $(1.69) (Miss).* Values retrieved from S&P Global.*
  • Q1 2024: Revenue $8.19M vs $8.37M (Miss); EPS $(1.45) vs $(1.65) (Beat).* Values retrieved from S&P Global.*

Note: Asterisked figures are S&P Global consensus estimates. Values retrieved from S&P Global.*

Where estimates may adjust

• 2025 PKD revenue likely to be reset flat; thalassemia initial contribution limited to partial Q4 2025; modelers should push thalassemia ramp into 2026 and incorporate medical-exception dynamics and payer setup lag .
• Gross-to-net assumptions anchored to 10%–20% annually with intra-year variability .
• SG&A trajectory elevated into thalassemia launch; R&D spend tied to LR‑MDS/SCD studies; EBITDA loss modestly better than prior consensus this quarter may temper near-term loss forecasts.* Values retrieved from S&P Global.*

Key Takeaways for Investors

• The quarter’s revenue miss vs consensus was largely a function of Q4 pull-forward dynamics; patient funnel/therapy persistence and operational readiness are trending positively into the thalassemia PDUFA .
• The regulatory path for thalassemia remains favorable (no Ad Comm planned; PDUFA 9/7/25), with detailed commercial groundwork (expanded field force, payer education) to support rapid post-approval execution .
• SCD readout by year-end 2025 is a pivotal second leg of the story; tebapivat adds optionality in both LR‑MDS and SCD with Phase 2 start mid‑’25 and no liver signal to date .
• Expect near-term financials to reflect launch investment and clinical execution; balance sheet (~$1.4B) affords independence and discipline on BD .
• Modeling implications: keep 2025 PKD flat, minimal thalassemia in Q4 2025 (partial quarter), 10–20% gross-to-net, and sustained SG&A into launch; larger thalassemia contribution begins 2026 .
• Stock catalysts: FDA thalassemia decision (Sep 7, 2025), RISE UP topline (late 2025), and updates on ex-U.S. partnerships (GCC ramp, EU approach) .
• Watch macro sensitivities (tariffs) and payer policy formation; management does not anticipate material tariff impact at this time .