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Company profile / AGIO

Agios Pharmaceuticals

Clinaptis stanceNeutral
ConvictionHigh
Ticker
AGIO
Exchange
NASDAQ
Coverage
Standard profile
Updated
Oct 3, 2026
Rare Disease / Metabolism

Commercial-stage rare-disease company built around red-blood-cell pyruvate kinase activation. Mitapivat is marketed as PYRUKYND in pyruvate kinase deficiency and AQVESME in adult alpha- or beta-thalassemia, and is under FDA Priority Review for sickle cell disease. The pipeline extends into polycythemia vera, phenylketonuria and immune thrombocytopenia.

01 / Clinaptis view

The current read

Valuation — Neutral. Our central SOTP is approximately $27/share versus $33.07, within a broad ~$19–38 scenario range. The stock is not demonstrably expensive, but offers too little margin for correlated commercial assumptions: our carried SCD value is ~$526M, while the current price requires roughly ~$906M under the same conventions.

  1. Clinical — better than the tape implies. REIGNITE tests the randomized transfusion signal that survived RISE UP's pain-crisis miss, in a transfusion-enriched population. Our reconstruction and simulation produce approximately 72% predictive PoS. Clinical risk is not the principal reason we are unwilling to own the stock.
  2. Commercial — the unresolved variable. HIBISCUS shifts the debate to mitapivat's retained share once etavopivat is available. We carry ~$580–650M SCD peak sales versus ~$781M required to justify the current price. Even 100% retention in the strongest severe-anemia and transfusion-burden phenotypes falls short; broader retention is needed where etavopivat has the randomized VOC advantage.
  3. Security view — no position. We are comfortable missing initial clinical or regulatory de-risking and paying more if commercial evidence improves. RISE UP subgroup data, numerical HIBISCUS transfusion results, the FDA label and monitoring burden, and launch evidence on access, persistence and realized net price would change the view. A pullback toward the ~$27 carried value with fundamentals intact would also improve the setup.
02 / Consensus gap

Where the market may differ

The market may be focused on the wrong uncertainty. RISE UP's pain-crisis miss makes mitapivat's SCD program look clinically weaker than our work suggests. REIGNITE tests the randomized transfusion signal that survived RISE UP, and our modeling supports approximately 72% predictive PoS—less confirmatory clinical risk than a superficial reading implies.

  1. But higher clinical confidence does not make the stock cheap. HIBISCUS moves the harder question downstream: how much share can mitapivat retain once etavopivat enters with randomized anemia and VOC data? Our $580–650M SCD commercial centre remains below the $781M peak sales needed to justify $33.07 under our carried assumptions. Neither approval nor confirmatory PoS alone closes that gap.
  2. The disagreement is asymmetric. We are more constructive on durable SCD clinical benefit than the headline narrative suggests, but less confident that success yields enough steady-state patient-years at an attractive realized price. The 25,000-patient pool, 23% standalone share and ~$140k net price are assumptions public evidence cannot yet distinguish reliably.
  3. That leaves valuation without a clear edge. A favorable but plausible commercial case supports roughly $38/share; our carried SOTP is approximately $27, and the wider range is ~$19–38. At $33.07 the market needs no absurd assumption, but it needs several favorable ones together. The differentiated question is how much clinical success mitapivat can retain commercially once both PK activators are in market.
Coverage state
Standard profile
Work status
Complete
What the review turns on
Key variable

Whether AQVESME establishes a durable thalassemia revenue base while mitapivat's SCD label, monitoring burden and first-mover window permit commercially meaningful adoption before etavopivat

Next catalyst

PYRUKYND / AQVESME · 3Q26 launch revenue, patients on therapy and persistence · next quarterly results, date not confirmed; Mitapivat · FDA PDUFA decision for sickle cell disease · Nov 1, 2026

03 / Pipeline

Key programs

6 assets · 6 programs

AssetIndicationStageNext event
Mitapivat
Sickle cell disease
sNDA under Priority Review
FDA PDUFA decision on Nov. 1, 2026; label breadth, age floor and hepatic monitoring requirements are the critical readthroughs; REIGNITE confirmatory Phase 3 trial evaluates transfusion burden
PYRUKYND / AQVESME (mitapivat)
Pyruvate kinase deficiency / thalassemia
Commercial
3Q26 U.S. launch revenue, patients on therapy and persistence under the REMS; continued international expansion
AG-236
Polycythemia vera
Phase 2/3
Phase 2 portion expected to begin in 2H 2026
AG-181
Phenylketonuria
Phase 1b
Phase 1b safety and tolerability data in adults with PKU expected in 2H 2026; this is not an efficacy readout
Cevidoplenib
Immune thrombocytopenia
Phase 3-enabling / CMC development
Phase 3 initiation expected in 1H 2028 after additional CMC work; the program was licensed for $25 million upfront
Tebapivat
Sickle cell disease / lower-risk myelodysplastic syndromes
Discontinued
No further development planned after the SCD profile was judged insufficiently differentiated from other PK activators and the lower-risk MDS program did not meet the company's advancement threshold
04 / Connected research

Published on AGIO

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  1. 01

    Agios (AGIO): Mitapivat in Sickle Cell Disease — REIGNITE Is Not the Hard Part

    Our work supports ~72% predictive PoS for Phase 3 REIGNITE. The harder question is whether mitapivat can capture enough SCD share to justify $33/sh.

    Read