Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Partnerships BioMarin: license covering TA-ERT technology and patents. Eli Lilly: license agreement (May 2016)
Indications
Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)(Phase 3)
Catalysts
— None
Status note Investigational enzyme replacement therapy for MPS IIIB; company focus since November 2024. FDA confirmed (Type C meeting, March 2024) that CSF HS-NRE is a surrogate biomarker reasonably likely to predict clinical benefit for accelerated approval and that completed clinical/nonclinical studies suffice for a BLA submission. A confirmatory Phase 3 placebo-controlled 5-year study (14 patients) must be initiated.
Tildacerfont
Phase 2 · ActiveModality: small molecule
Target CRF1 receptor
Partnerships HMNC Brain Health: license, development and option agreement (May 2024); HMNC funded and conducted the TAMARIND study
Indications
Major depressive disorder (Cortibon-positive patients)(Phase 2)
Status note Oral, potent and selective small-molecule antagonist of the CRF1 receptor. Phase 2 proof-of-concept TAMARIND study in Cortibon-positive MDD patients was discontinued in Q1 2026 following a serious adverse event (elevated liver enzymes); the filing does not state a program-level discontinuation.
SPR202
Stage not disclosed · ActiveModality: monoclonal antibody
Target CRF
Partnerships HBM Alpha Therapeutics: license to the anti-CRH monoclonal antibody (rights outside mainland China, Taiwan, Hong Kong, Macau)
Indications
Congenital adrenal hyperplasia (CAH)(Stage not disclosed)
Status note Anti-CRH monoclonal antibody targeting CRF for CAH and other indications; filing states no trial phase.
Per-asset detail extracted from the 10-Q filed 2026-08-12. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (1 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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