Agios Pharmaceuticals Inc

AGIO

Equity

ISIN null / Valor 21630028

NASDAQ (2025-11-20)

USD 24.57+9.98%

Agios Pharmaceuticals Inc
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About company

Agios Pharmaceuticals Inc is a biopharmaceutical company that specializes in the discovery and development of novel therapies for rare diseases, with a particular emphasis on hemolytic anemias. Leveraging its expertise in cellular metabolism, Agios has made significant strides in the field, marked by the FDA approval of its first rare disease therapy for adults with pyruvate kinase (PK) deficiency. The company's pipeline includes mitapivat, its lead product candidate currently undergoing late-stage clinical trials for thalassemia and sickle cell disease. Additionally, Agios is working on AG-946, a new PK activator aimed at treating hemolytic anemias among other conditions. Beyond these leading programs, Agios is actively engaged in preclinical development, working on advancing a range of investigational therapies that promise to further its mission of addressing unmet medical needs in rare disease communities.

Summarized from source with an LLMView SourceSector: Technology

Revenue Growth

Net product revenue from PYRUKYND® (mitapivat) reached $12.5 million in Q2 2025, up from $8.6 million in Q2 2024, driven by a 6% increase in unique patient enrollments and a 4% rise in patients on therapy.

Regulatory Progress

The supplemental New Drug Application (sNDA) for PYRUKYND® in thalassemia is under active FDA review with a PDUFA goal date of September 7, 2025. Additionally, topline results from the RISE UP Phase 3 trial in sickle cell disease are on track for year-end, with a potential U.S. commercial launch in 2026.

R&D Advancements

Agios dosed the first patient in the Phase 2 tebapivat trial for sickle cell disease and received IND clearance for AG-236, targeting polycythemia vera. The company also continues to expand its clinical pipeline with ongoing trials in lower-risk myelodysplastic syndromes (LR-MDS).

Financial Position

As of June 30, 2025, Agios held $1.3 billion in cash, cash equivalents, and marketable securities. Despite increased research and development and selling, general and administrative expenses, the company expects its financial resources to support product launches and pipeline advancement.

Summarized from source with an LLMView Source

Key figures

-55.0%1Y

-12.9%3Y

-43.6%5Y

Performance

55.6%1Y

49.2%3Y

53.7%5Y

Volatility