Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Partnerships NewBridge Pharmaceuticals: distribution agreement for PYRUKYND in certain MENA territories; Avanzanite: commercialization access in EU/GB/Saudi Arabia/UAE
Indications
PK deficiency(Approved)
Thalassemia (alpha/beta)(Approved)
Sickle cell disease(Stage not disclosed)
Catalysts
— November 1, 2026
Status note Approved in US as AQVESME for anemia in adults with non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia and as PYRUKYND for hemolytic anemia in adults with PK deficiency; also approved in EU/GB/Saudi Arabia/UAE. Confirmatory REIGNITE phase 3 trial in SCD ongoing under accelerated-approval pathway.
Tebapivat
Phase 2 · Terminated
Target pyruvate kinase (PK)
Indications
Lower-risk myelodysplastic syndromes(Phase 2)
Sickle cell disease(Phase 2)
Status note Development discontinued: May 2026 — will not advance in LR-MDS (phase 2b did not meet predefined threshold); July 2026 — will not continue development in SCD (phase 2 did not establish sufficient differentiation from other PK activators).
AG-181
Phase 1 · Active
Target phenylalanine hydroxylase (PAH)
Indications
Phenylketonuria (PKU)(Phase 1)
Status note IND filed December 2023; phase 1 trial in healthy volunteers initiated Q1 2024, multiple ascending dose portion initiated Q2 2024.
AG-236
Preclinical · ActiveModality: siRNA
Target TMPRSS6
Partnerships Alnylam Pharmaceuticals: license agreement for development and commercialization of the TMPRSS6 siRNA
Indications
Polycythemia vera(Preclinical)
Status note In-licensed from Alnylam under July 28, 2023 license agreement ($17.5M upfront IP R&D recognized in 2023).
Cevidoplenib
Stage not disclosed · ActiveModality: small molecule
Target SYK (spleen tyrosine kinase)
Partnerships Oscotec Inc.: June 1, 2026 license agreement for worldwide rights (Oscotec retains opt-in for South Korea); $25M upfront IP R&D paid; up to $140M in development/regulatory milestones
Indications
Immune thrombocytopenia (ITP)(Stage not disclosed)
Other autoimmune indications(Stage not disclosed)
Catalysts
— first half of 2028
Status note Oral SYK inhibitor in-licensed from Oscotec; Agios will account for the deal as an asset acquisition.
Per-asset detail extracted from the 10-Q filed 2026-07-30. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (15 as sponsor, 2 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.
Institutional holder positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.
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