Agios Pharmaceuticals AGIO announced that the FDA has accepted its supplemental new drug application (sNDA) seeking label expansion for its sole marketed drug, Pyrukynd, in thalassemia indication.

A final decision is expected by Sep. 7, 2025. If approved, Pyrukynd will become the first oral therapy for use in all thalassemia subtypes. A commercial launch in this indication is expected before this year’s end.

The sNDA is supported by data from two phase III studies, ENERGIZE and ENERGIZE-T, which evaluated Pyrukynd for alpha or beta-thalassemia patients who are non-transfusion-dependent and transfusion-dependent, respectively. Both studies achieved their primary and key secondary endpoints. Similar regulatory filings have been submitted by Agios in the European Union, Kingdom of Saudi Arabia and United Arab Emirates, supported by data from the two studies.

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

AGIO Stock’s Performance

In the past year, Agios’ shares have surged nearly 50% against the industry’s 15% decline.

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Factors Responsible for the Uptick in AGIO Stock

This surge in share price began at the onset of 2024 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.

In the third quarter of 2024, management significantly boosted its cash resources when AGIO added $1.1 billion from Royalty Pharma RPRX 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 agreement between the two companies, Servier made a milestone payment of $200 million to Agios for this approval.

In May, Agios inked 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.

However, some of the stock gains were pared last month after Agios revealed that two thalassemia patients who received Pyrukynd across the ENERGIZE and ENERGIZE-T studies reported hepatocellular (liver) injury in the first six months of treatment. The regulatory filings by management identify this injury as a potential risk in thalassemia patients and propose monthly liver test monitoring during the first six months of treatment.

AGIO’s Zacks Rank

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

Agios Pharmaceuticals, Inc. Price

Agios Pharmaceuticals, Inc. price | Agios Pharmaceuticals, Inc. Quote

Our Key Picks Among Biotech Stocks

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

Bottom-line estimates for Castle Biosciences have improved from a loss of 8 cents per share to earnings of 34 cents for 2024 in the past 60 days. During the same timeframe, loss per share estimates for 2025 have narrowed from $1.88 to $1.84. In the past year, shares of Castle Biosciences have surged 56%.

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 18 cents to 5 cents. Estimates for 2025 loss per share have narrowed from 55 cents to 35 cents during the same timeframe. CTMX’s shares have lost 38% in the past year.

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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