AG-236 / Alnylam, Agios Pharmaceuticals - LARVOL DELTA

A Study to Determine the Safety and Tolerability of AG-236 and How it is Absorbed, Broken Down, and Eliminated From the Body in Healthy Participants (clinicaltrials.gov) - P1 | N=24 | Recruiting | Sponsor: Agios Pharmaceuticals, Inc. | Trial completion date: Jan 2026 ➔ Aug 2026 | Trial primary completion date: Dec 2025 ➔ Mar 2026

Trial completion date • Trial primary completion date

Anticipated 2026 Milestones (The Manila Times) - "Enrollment in the Phase 2b LR-MDS trial of tebapivat was completed in 2025. Agios expects to report topline results from this trial in the first half of 2026....Agios expects to report topline results from a Phase 1 healthy volunteer trial of AG-236, a small interfering RNA (siRNA) targeting TMPRSS6 as a potential treatment for PV, in the first half of 2026."

P1 data • P2b data • Myelodysplastic Syndrome • Polycythemia Vera

A Study to Determine the Safety and Tolerability of AG-236 and How it is Absorbed, Broken Down, and Eliminated From the Body in Healthy Participants (clinicaltrials.gov) - P1 | N=24 | Recruiting | Sponsor: Agios Pharmaceuticals, Inc.

New P1 trial

Second Quarter 2025 Financial Results (The Manila Times) - "Net product revenue from sales of PYRUKYND for the second quarter of 2025 was $12.5 million, compared to $8.6 million for the second quarter of 2024...Research and Development (R&D) Expenses were $91.9 million for the second quarter of 2025, compared to $77.4 million for the second quarter of 2024. The year-over-year increase was primarily attributed to a $10.0 million regulatory milestone payment to Alnylam associated with our agreement to develop and commercialize AG-236, an siRNA targeting TMPRSS6, intended for the treatment of polycythemia vera."

Commercial • Beta-Thalassemia • Polycythemia Vera • Sickle Cell Disease

Second Quarter 2025 and Recent Corporate Highlights (The Manila Times) - "Early Pipeline Investigational New Drug (IND) clearance received for AG-236, an siRNA targeting TMPRSS6 intended for the treatment of polycythemia vera (PV)."

IND • Polycythemia Vera

Key Upcoming Milestones & Priorities (GlobeNewswire) - "LR-MDS: Complete patient enrollment in the Phase 2b study of tebapivat for LR-MDS in late 2025; Early-Stage Pipeline: File an Investigational New Drug Application for AG-236, an siRNA targeting TMPRSS6 intended for the treatment of polycythemia vera, in mid-2025."

Enrollment status • IND • Myeloproliferative Neoplasm • Polycythemia Vera

Agios Reports Fourth Quarter and Full Year 2024 Financial Results and Recent Business Highlights (GlobeNewswire) - "'Backed by a strong balance sheet and a highly experienced team, Agios is focused on maximizing the potential PYRUKYND launches in thalassemia and sickle cell disease in 2025 and 2026, respectively'...PYRUKYND Revenues: Generated $10.7 million in net revenue for the fourth quarter of 2024, a 20 percent increase from the third quarter of 2024, primarily driven by year-end stocking and adjustments to certain revenue reserves...Announce topline results from the Phase 3 RISE UP study of mitapivat in sickle cell disease in late 2025...Additionally, begin patient enrollment for the Phase 2 study of tebapivat in sickle cell disease in mid-2025. LR-MDS: Complete patient enrollment in the Phase 2b study of tebapivat for LR-MDS in late 2025. Early-Stage Pipeline: File an Investigational New Drug Application for AG-236, a siRNA targeting TMPRSS6 intended for the treatment of polycythemia vera, in mid-2025."

Commercial • Enrollment status • IND • Launch US • P3 data: top line • Anemia • Beta-Thalassemia • Hematological Disorders • Myelodysplastic Syndrome • Polycythemia Vera • Sickle Cell Disease

Agios Announces Key 2025 Milestones for Innovative Rare Disease Portfolio (GlobeNewswire) - "Anticipated 2025 Milestones: Lower-Risk Myelodysplastic Syndromes (LR-MDS): Complete patient enrollment in the Phase 2b study of tebapivat for LR-MDS in late 2025. Early-Stage Pipeline: File an Investigational New Drug Application for AG-236, a siRNA targeting TMPRSS6 intended for the treatment of polycythemia vera, in mid-2025."

Enrollment status • IND • Myelodysplastic Syndrome • Polycythemia Vera

Agios Announces Exclusive Worldwide License Agreement for Novel siRNA for the Potential Treatment of Polycythemia Vera (GlobeNewswire) - "Agios Pharmaceuticals, Inc...has entered into an exclusive worldwide license agreement with Alnylam Pharmaceuticals, Inc. under which Agios will acquire the rights to develop and commercialize Alnylam’s novel preclinical siRNA targeting TMPRSS6, as a potential disease-modifying treatment for patients with polycythemia vera (PV)....Under the terms of the agreement, Agios will make an upfront payment of $17.5 million to Alnylam for an exclusive global license to the TMPRSS6 siRNA program. In addition, Alnylam is eligible to receive up to $130 million in potential development and regulatory milestone payments, in addition to sales milestones and tiered royalties. Agios will assume sole responsibility for all development, regulatory, and commercial activities and costs related to the program. Alnylam will provide manufacturing support for Phase 1, after which Agios will assume full responsibility for manufacturing."

Licensing / partnership • Hematological Malignancies • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera