AGIO

Financial Performance: Agios Pharmaceuticals reported $12.9 million in net revenues for PYRUKYND in Q3 2025, a 44% increase from the previous year, while facing a net loss of $103.4 million due to increased R&D and operational expenses.

Regulatory Updates: The FDA has extended the PDUFA goal date for PYRUKYND's sNDA in thalassemia to December 7, 2025, while the CHMP has issued a positive opinion for its approval in Europe, with a final decision expected by early 2026.

Clinical Trials: The RISE UP Phase 3 trial results for PYRUKYND in sickle cell disease are anticipated by year-end 2025, and the Phase 2b trial for tebapivat in lower-risk MDS has been fully enrolled, with results expected in early 2026.

Cash Position: As of September 30, 2025, Agios holds $1.3 billion in cash and marketable securities, which is expected to support upcoming commercial launches and ongoing clinical programs.

Investigation Announcement: Pomerantz LLP is investigating potential securities fraud and unlawful business practices by Agios Pharmaceuticals and its executives, urging affected investors to reach out for assistance.

Stock Price Impact: Following the FDA's extension of the review date for Agios's drug PYRUKYND®, the company's stock price dropped by over 11% on September 4, 2025, reflecting investor concerns over the delay.

Partnership Announcement: Avanzanite entered an exclusive partnership with Agios in June 2025 to manage the commercialization of PYRUKYND across Europe, targeting thalassemia as a new indication pending European Commission approval.

Positive CHMP Opinion: The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for PYRUKYND, which is currently approved for PK deficiency, with a final decision from the European Commission expected by early 2026.