Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Status note Lead program. Phase 2 initiated Nov 2024 (ongoing open-label trial in adults with APOL1 high-risk genotype proteinuric CKD); topline proof-of-concept data announced Mar 2026; enrollment continuing.
MZE782
Phase 2 · ActiveModality: oral small molecule
Target SLC6A19
Indications
Phenylketonuria (PKU)(Phase 2)
Chronic kidney disease (CKD)(Stage not disclosed)
Catalysts
— 2027
— H1 2027
Status note Phase 1 in healthy volunteers completed Sep 2025. Phase 2 proof-of-concept trial in PKU initiated Aug 2026; Phase 2 proof-of-concept trial in CKD planned for H1 2027.
MZE001
Phase 2 · ActiveModality: oral small molecule
Aliases S606001
Target GYS1 (glycogen synthase 1)
Partnerships Shionogi & Co., Ltd.: exclusive worldwide license (Mar 2024); $150M upfront, up to $275M in clinical/regulatory milestones, up to $330M in sales milestones, tiered royalties; $20M clinical milestone achieved Mar 2026
Indications
Pompe disease(Phase 2)
Status note Partnered program fully licensed to Shionogi. Shionogi-led Phase 2 ongoing; first patient dosed Mar 2026.
Per-asset detail extracted from the 10-Q filed 2026-08-11. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (2 as sponsor, 0 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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