Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Congenital hyperinsulinism (congenital HI)(Stage not disclosed)
Status note Lead investigational asset; first-in-class GLP-1 receptor antagonist. LUCIDITY (NCT06747468): 78-participant Phase 3 in PBH post-RYGB at 21 U.S. sites. Expanded Access Program initiated May 2026 for U.S. adults with PBH post-RYGB.
AMX0035
Phase 2 · ActiveModality: oral fixed-dose combination of sodium phenylbutyrate and taurursodiol
Target ER stress and mitochondrial dysfunction
Indications
Wolfram syndrome(Phase 2)
Progressive supranuclear palsy (PSP)(Phase 2)
Status note HELIOS open-label Phase 2 in Wolfram syndrome ongoing; company working with FDA on a Phase 3. Phase 2b/3 in PSP discontinued and winding down.
Status note LUMINA (NCT06665165): multinational multiple-ascending-dose Phase 1 in ALS; Cohort 1 biomarker data presented Jun 2026. FDA Fast Track Designation.
Partnerships Gubra A/S: collaboration and license agreement (Dec 2024) for long-acting GLP-1 receptor antagonist R&D; second research collaboration Jul 2026 for a rare endocrine disease
Indications
PBH and other rare diseases(Preclinical)
Status note Selected as development candidate Jan 2026 after preclinical evaluation; IND-enabling studies planned/ongoing.
Per-asset detail extracted from the 10-Q filed 2026-08-06. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (3 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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