Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Partnerships Ligand: worldwide sublicense to develop/manufacture/commercialize sparsentan (2012). CSL Vifor: license and collaboration (Sep 2021); CSL Vifor responsible for commercialization in licensed territories; EC conditional MA Apr 2024. Chugai (via Renalys, merged): exclusive license (Jan 2024)
Indications
Primary IgA nephropathy (IgAN) at risk of disease progression(Approved)
Focal segmental glomerulosclerosis (FSGS) without nephrotic syndrome (adults and pediatric 8+)(Approved)
Status note FDA accelerated approval Feb 2023 and full approval Sep 2024 for IgAN (to slow kidney function decline); full traditional approval Apr 13, 2026 for FSGS (to reduce proteinuria). Available under a REMS for liver monitoring.
pegtibatinase
Phase 3 · ActiveModality: investigational human enzyme replacement therapy
Target CBS
Indications
Classical homocystinuria (HCU)(Phase 3)
Status note Granted Rare Pediatric Disease, Fast Track and Breakthrough Therapy designations by the FDA plus orphan designations. In Sep 2024 the company announced a voluntary pause of enrollment in the pivotal Phase 3 HARMONY study because the desired drug substance profile was not achieved in the initial scale-up process; no discontinuation disclosed.
Thiola
Approved · Active
Aliases tiopronin
Target Cystine (thiol-disulfide exchange)
Partnerships Mission Pharmacal (Mission): exclusive, royalty-bearing license (2014) to market, sell and commercialize Thiola in the US and Canada
Indications
Cystinuria (prevention of cystine kidney stones)(Approved)
Status note Marketed licensed product; not a company-developed pipeline candidate.
Per-asset detail extracted from the 10-Q filed 2026-08-04. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (6 as sponsor, 4 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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