Agios' PYRUKYND Faces FDA Review In September - Expansion Opportunity Ahead?

(RTTNews) - Shares of Agios Pharmaceuticals Inc. (AGIO) have retreated approximately 43% from their 52-week high of $62.58, reported last November, and trade near the $35 level.

The company's flagship drug, PYRUKYND, has demonstrated strong year-over-year sales growth since its initial approval in February 2022. The drug is currently indicated for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency.

Pyruvate kinase (PK) deficiency is a rare disease caused by inherited mutations in the PKLR gene, resulting in impaired cellular energy production and reduced red blood cell survival. People with PK deficiency can experience a range of signs and symptoms, including anemia, debilitating fatigue, exercise intolerance, jaundice, memory loss, and difficulty concentrating.

Looking to significantly broaden its therapeutic label, Agios has proposed an expanded indication for PYRUKYND as a treatment for adult patients with both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The FDA is slated to announce its decision on the proposed indication on September 7, 2025.

PYRUKYND is also being explored as a treatment for sickle cell disease patients who are 16 years of age or older in a phase III trial, dubbed RISE UP. The study is fully enrolled with over 200 patients, with topline results expected in late 2025.

The company has also successfully completed two pivotal phase III trials of PYRUKYND in pediatric populations - ACTIVATE-Kids and ACTIVATE-KidsT.

ACTIVATE-Kids is a phase III trial of PYRUKYND in children aged 1 to less than 18 years with PK deficiency who are not regularly transfused. ACTIVATE-KidsT is a phase III trial of PYRUKYND in children aged 1 to less than 18 years with PK deficiency who are regularly transfused.

As mentioned earlier, PYRUKYND has demonstrated consistent year-over-year revenue growth - $11.74 million in 2022, $26.82 million in 2023, and $36.5 million in 2024. This momentum has continued into 2025, with the drug generating $8.7 million in net revenue for the first quarter, compared to $8.2 million in the first quarter of 2024.

Another clinical drug candidate of the company is Tebapivat, which is under a phase IIb study in lower-risk myelodysplastic syndromes. Patient enrolment in this study is expected to be completed in late 2025.

Lower-risk myelodysplastic syndromes (LR-MDS) are a subset of myelodysplastic syndromes - a group of blood cancers characterized by ineffective blood cell production (hematopoiesis) leading to low blood counts (cytopenias) and a risk of progression to acute myeloid leukemia.

The company plans to initiate a phase II study of Tebapivat in sickle cell disease by mid-2025.

Cash Position

The company's cash, cash equivalents, and marketable securities as of March 31, 2025, were $1.4 billion.

Agios' common stock commenced trading on the NASDAQ Global Select Market, under the ticker symbol "AGIO", on July 24, 2013, priced at $18 per share.

AGIO has traded in a range of $23.42 to $62.58 in the last 1 year. The stock closed yesterday's trading at $35.75, up 4.23%.