Amyloidosis in Transthyretin (TTR); Amyloidosis, Familial; Amyloid Cardiomyopathy
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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 Next-generation oral small molecule near-complete TTR stabilizer. FDA approved November 22, 2024 for cardiomyopathy of wild-type or variant ATTR amyloidosis; EC approved February 10, 2025 (as Beyonttra) for ATTR-CM.
BBP-418
Phase 3 · ActiveModality: oral small molecule
Target FKRP
Indications
limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9)(Phase 3)
Catalysts
— November 27, 2026
Status note NDA submitted to FDA March 30, 2026; accepted with Priority Review. Designed to supply supra-physiological levels upstream to drive residual activity of the mutant FKRP enzyme.
encaleret
Phase 3 · ActiveModality: oral small molecule
Target CaSR (calcium-sensing receptor)
Indications
autosomal dominant hypocalcemia type 1 (ADH1)(Phase 3)
chronic hypoparathyroidism (CHP)(Stage not disclosed)
Catalysts
— May 8, 2027
Status note Negative allosteric modulator of the calcium-sensing receptor. NDA submitted to FDA May 12, 2026 and accepted for filing.
low-dose infigratinib
Phase 3 · ActiveModality: oral small molecule
Target FGFR1-3
Indications
achondroplasia(Phase 3)
hypochondroplasia(Phase 3)
Status note Selective FGFR1-3 tyrosine kinase inhibitor; gain-of-function FGFR3 mutations cause achondroplasia/hypochondroplasia. NDA submitted to FDA for achondroplasia; ongoing Phase 2 and Phase 3 trials.
BBP-812
Phase 1 · ActiveModality: gene therapy (IV AAV9)
Indications
Canavan disease(Phase 1)
Status note Phase 1/2 CANaspire study ongoing; designed as a single registrational study using a biomarker for accelerated approval. Orphan, Fast Track, RMAT and Rare Pediatric Disease designations from FDA.
antibody deplete program
Stage not disclosed · ActiveModality: antibody
Indications
ATTR-CM(Stage not disclosed)
Status note Company references a new antibody deplete program for ATTR-CM; clinical timeline referenced in filing but no named candidate or stage stated.
Per-asset detail extracted from the 10-Q filed 2026-08-10. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (14 as sponsor, 3 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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