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Here's Why Agios Stock Plummeted More Than 20% on Monday

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Shares of Agios Pharmaceuticals (AGIO - Free Report) lost 21% yesterday after management revealed that two thalassemia patients who received its sole-marketed drug Pyrukynd (mitapivat) across two late-stage studies reported hepatocellular (liver) injury in the first six months of treatment.

This information was disclosed by Agios as part of an oral presentation at the ASH 2024 annual meeting when it presented detailed results from two phase III studies, ENERGIZE-T and ENERGIZE. While the ENERGIZE-T study evaluated Pyrukynd in adult participants with transfusion-dependent (TD) alpha or beta-thalassemia, the ENERGIZE study evaluated the drug in non-transfusion-dependent (NTD) thalassemia patients.

Alongside the oral presentation, Agios also disclosed that it has submitted regulatory filings in the United States, European Union, Kingdom of Saudi Arabia and United Arab Emirates seeking label expansion for Pyrukynd in both NTD and TD thalassemia patients.

The above submissions also identify hepatocellular injury as a potential risk in thalassemia patients and propose monthly liver test monitoring during the first six months of treatment.

Pyrukynd is currently approved by the FDA to treat adults with pyruvate kinase deficiency, a rare and debilitating blood disorder. The drug is currently the sole marketed product in the company’s portfolio.

AGIO Stock’s Performance

The results came as a shock to investors as both the ENERGIZE-T and ENERGIZE studies had already achieved their respective primary and key secondary endpoints. Though the side-effect was observed in a very small number of patients, they raised concerns about Pyrukynd’s potential across various other diseases in which the company seeks label expansion.

Year to date, Agios’ shares have skyrocketed 118.4% against the industry’s 7.4% decline.

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Factors Responsible for the YTD Surge in AGIO Stock

This surge in share price began earlier this year when Agios reported that the ENERGIZE study achieved its primary endpoint of hemoglobin response, as 42.3% of patients who received the drug achieved a hemoglobin response compared with 1.6% for placebo.

Later in June, management reported similar results from the ENERGIZE-T study which also achieved its primary endpoint of reduction in transfusion burden, as 30.4% of patients in the Pyrukynd arm achieved a transfusion reduction response compared with 12.6% in the placebo arm.

Per management, the above results strengthen Pyrukynd’s potential to become the first oral therapy for TD and NTD thalassemia patients. If approved, a commercial launch in this indication is expected next year.

In the third quarter of 2024, management significantly boosted its cash resources when the company added $1.1 billion from Royalty Pharma (RPRX - Free Report) and France-based Servier following the FDA’s approval of vorasidenib for a brain tumor called IDH-mutant diffuse glioma in August.

Vorasidenib was a part of Agios’ oncology business that was sold to Servier in 2021. Per the terms of the sale executed between the two companies, Servier paid a milestone payment of $200 million to Agios for this approval. In May, Agios entered into a deal with Royalty Pharma to sell its royalty rights on potential net sales of vorasidenib in exchange for an upfront payment of $905 million contingent on the FDA’s approval for the drug. Management intends to utilize the above funds to expand its pipeline and support potential product launches.

AGIO’s Zacks Rank

Agios currently carries a Zacks Rank #3 (Hold).

Key Picks Among Biotech Stocks

Some top-ranked stocks from the sector are Castle Biosciences (CSTL - Free Report) and CytomX Therapeutics (CTMX - Free Report) , each sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, 2024 estimates for Castle Biosciences have improved from a loss of 58 cents per share to earnings of 34 cents. During the same timeframe, loss per share estimates for 2025 have narrowed from $2.13 to $1.84. Year to date, shares of Castle Biosciences have surged 43.2%.

CSTL’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 172.72%.

In the past 60 days, estimates for CytomX Therapeutics’ 2024 loss per share have narrowed from 29 cents to 5 cents. Estimates for 2025 loss per share have narrowed from 56 cents to 35 cents during the same timeframe. Year to date, CTMX stock has lost 18.7%.

CytomX’s earnings beat estimates in two of the trailing four quarters and missed the mark in the other two, delivering an average surprise of 115.70%.

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