Agios Pharmaceuticals, Inc. (AGIO): A Bull Case Theory

 We came across a bullish thesis on Agios Pharmaceuticals, Inc. on BioEquity Watch’s Substack. In this article, we will summarize the bulls’ thesis on AGIO. Agios Pharmaceuticals, Inc.'s share was trading at $27.80 as of January 15th. AGIO’s trailing and forward P/E were 3.54, respectively according to Yahoo Finance.

Agios Pharmaceuticals is a clinical-stage biotech company focused on cellular metabolism through oral, small-molecule allosteric activators of the pyruvate kinase (PK) enzyme. Its lead product, mitapivat, is marketed as PYRUKYND for PK deficiency and received FDA approval in December 2025 under the brand name AQVESME for alpha- and beta-thalassemia. By enhancing PK activity, mitapivat increases ATP levels and reduces 2,3-DPG accumulation in red blood cells, improving cell survival and reducing hemolysis, the main driver of anemia.

Phase 3 trials in thalassemia demonstrated strong efficacy, with ENERGIZE showing a 42.3% hemoglobin response in non-transfusion-dependent patients and ENERGIZE-T achieving a 30.4% transfusion reduction in dependent patients. In sickle cell disease, the RISE UP trial met hemoglobin endpoints but showed only favorable trends in pain crisis reduction. Agios is also advancing tebapivat, which achieved proof-of-concept in lower-risk myelodysplastic syndromes, with 40% of patients reaching transfusion independence.

Financially, Agios is well-capitalized, holding approximately $1.3 billion in cash and marketable securities as of Q3 2025, sufficient to fund launches and clinical programs without dilutive financing. PYRUKYND revenues grew 44% year-over-year, though net losses remain due to high R&D and SG&A expenses. The thalassemia approval provides a first-mover advantage in a larger patient population, while the upcoming sickle cell opportunity could expand its market further. Agios differentiates from competitors like Reblozyl, gene therapies, and other oral therapies by offering a twice-daily pill with broad accessibility.

Led by CEO Brian Goff and an experienced commercial and clinical team, the company is positioned to scale globally. Catalysts in early 2026 include the AQVESME U.S. launch, FDA discussions for sickle cell indications, and Phase 2b tebapivat data. With multiple approved indications, a strong balance sheet, and scalable platform, Agios offers a compelling investment opportunity, with upside potential from successful commercial execution and global expansion.

Previously, we covered a bullish thesis on Relay Therapeutics, Inc. (RLAY) by Steve Wagner | Invest in December 2024, which highlighted promising interim data for RLY-2608, strong tolerability, and an undervalued market cap. RLAY’s stock price has appreciated by approximately 59.62% since our coverage due to positive clinical updates. Agios Pharmaceuticals, Inc. (AGIO) shares a similar bullish perspective but emphasizes multiple approved indications, a validated platform, and upcoming commercial launches in rare diseases.

Agios Pharmaceuticals, Inc. is not on our list of the 30 Most Popular Stocks Among Hedge Funds. As per our database, 36 hedge fund portfolios held AGIO at the end of the third quarter which was 29 in the previous quarter. While we acknowledge the potential of AGIO as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock.

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Disclosure: None.