Generated static export from Obsidian-friendly vault markdown
ClinPharm Vault

Rilzabrutinib

Overview

  • Priority class: BTK
  • Mechanistic bucket: Bruton's tyrosine kinase pathway program
  • Sponsor: Sanofi
  • Development focus: Completed or historical urticaria development represented in current raw-source layer
  • Indications represented in current raw sources: Chronic Spontaneous Urticaria

Strategy readout

  • Headline: More focused BTK challenger strategy in CSU, with a thinner visible development stack than remibrutinib.
  • Current strategic read: In the current local evidence layer, rilzabrutinib looks like a narrower CSU-centered BTK program rather than a broad urticaria franchise. The strategic posture appears more concentrated and later-entry than remibrutinib’s multi-branch stack.
  • Highest visible phase in current registry: Phase 2
  • Strategy confidence in current local layer: Medium

Why this looks like the strategy

  • The current local layer shows one clear CSU trial and one primary manuscript, without the same visible extension, CIndU, pediatric, or real-world lattice seen for remibrutinib.
  • That makes the program easier to describe, but also indicates less disclosed breadth in the current source cache.

What to watch next

  • Whether additional sponsor materials reveal broader lifecycle or subtype expansion plans beyond the currently visible CSU focus.

Operational study design view

Trial Arms in registry Active dose regimens Total enrollment Per-arm sample size summary
NCT05107115 4 3 161 PMCID full text directly supports randomized sizes of 400 mg/d n=38, 800 mg/d n=41, 1200 mg/d n=41, and placebo n=40 overall; it also reports primary-analysis sample sizes of placebo n=36, 400 mg/d n=37, 800 mg/d n=35, and 1200 mg/d n=35.

Study Inventory

Completed / historical studies

  • NCT05107115 - Phase 2; COMPLETED; Chronic Spontaneous Urticaria

Active / recruiting studies

  • None in current registry

Other registry entries

  • None in current registry

Program Results Summary

Trials with source-backed results in the current local layer: 1 of 1

NCT05107115 (Published) - Primary endpoint (ISS7 change at week 12, 1200 mg/d versus placebo, omalizumab-naive): LS mean -9.21 versus -5.77; difference -3.44 (95% CI -6.25 to -0.62; P = 0.02). - Favorable risk-benefit; no dose-dependent AEs; most frequent AEs: diarrhea and nausea (mild). - Sources: PMID 40266575; PMCID PMC12019677

Sponsor-sourced result evidence

Sponsor press release and conference poster provide additional detail beyond the primary manuscript for RILECSU Phase 2.

Sanofi RILECSU Phase 2 press release (February 2024) (Sponsor press release) - Rilzabrutinib 400 mg TID (ITT population): significant ISS7 reduction at week 12 (LSM -9.58 vs -6.31 placebo; P = 0.0181). - Significant UAS7 reduction at week 12 (LSM -17.95 vs -11.20; P = 0.0116). - Significant HSS7 reduction at week 12 (LSM -8.31 vs -4.89; P < 0.01). - Significant ISS7 changes observed as early as week 1. - Safety: No events of cytopenia, bleeding, or atrial fibrillation (distinguishing from older BTK inhibitors). - Safety: Most common TEAEs (TID): diarrhea 29.3%, nausea 19.5%, headache 9.8%. - Source(s): raw/sponsors/btk/rilzabrutinib/phase-2-csu-results-press-release.md

RILECSU Phase 2 hives poster (AAAAI 2024) (Conference poster) - Significant and sustained improvements in UAS7 through week 12 with rilzabrutinib 1200 mg/day. - Nominally significant improvements in percent change in HSS7 as early as week 1 with all rilzabrutinib doses. - Source(s): raw/sponsors/btk/rilzabrutinib/rilecsu-phase-2-hives-poster.md

Longitudinal efficacy view

  • Plotted page: Rilzabrutinib longitudinal UAS7
  • Shared comparison page: Cross-program longitudinal UAS7 comparison
  • Current explicit numeric plotting coverage: randomized-arm UAS7 change-from-baseline landmarks at weeks 4 and 12, plus 1200 mg/day versus placebo UAS7 difference landmarks at weeks 1, 4, and 12.
  • Current responder plotting coverage: week 12 UAS7 <= 6 and UAS7 = 0 snapshot for placebo versus rilzabrutinib 1200 mg/day.
  • Current main gap: the current local extraction still does not support a safely tabulated four-arm week-by-week UAS7 curve or richer long-term CSU durability numerics.
  • Local data backbone: data/rilzabrutinib_longitudinal_uas7.json and data/rilzabrutinib_longitudinal_uas7_notes.md.

Evidence Coverage

  • CT.gov trials in registry: 1
  • Sponsor artifacts in registry: 3
  • Primary publications in registry: 1
  • Supporting publications in registry: 1
  • Publication status: primary_manuscripts_found
  • Publication summary: One clear primary CSU efficacy manuscript was identified, plus later non-primary drug-review coverage.

Primary publications

  • PMID 40266575 (2025, JAMA dermatology): Rilzabrutinib in Antihistamine-Refractory Chronic Spontaneous Urticaria: The RILECSU Phase 2 Randomized Clinical Trial.
  • Role: phase_2_core
  • Linked trial IDs: NCT05107115
  • Local cache: raw/publications/pubmed/markdown/PMID40266575.md

Supporting evidence

  • PMID 41359083: Rilzabrutinib: First Approval. (raw/publications/pubmed/markdown/PMID41359083.md)
  • Sponsor artifact: Sanofi pipeline page (Sanofi)
  • raw/sponsors/btk/rilzabrutinib/sanofi-pipeline-page.html
  • raw/sponsors/btk/rilzabrutinib/sanofi-pipeline-page.md
  • Sponsor artifact: Phase 2 CSU results press release (Sanofi)
  • raw/sponsors/btk/rilzabrutinib/phase-2-csu-results-press-release.pdf
  • raw/sponsors/btk/rilzabrutinib/phase-2-csu-results-press-release.txt
  • raw/sponsors/btk/rilzabrutinib/phase-2-csu-results-press-release.md
  • Sponsor artifact: RILECSU Phase 2 hives poster (Sanofi)
  • raw/sponsors/btk/rilzabrutinib/rilecsu-phase-2-hives-poster.pdf
  • raw/sponsors/btk/rilzabrutinib/rilecsu-phase-2-hives-poster.txt
  • raw/sponsors/btk/rilzabrutinib/rilecsu-phase-2-hives-poster.md

Interpretation

  • Verified facts: the current v2 registry tracks 1 CT.gov entries for this program and links them conservatively to sponsor and publication evidence where explicit identifiers are available.
  • Interpretation: this program already has enough linked manuscript or registry support for a source-first derived program page, but individual study pages should still be reviewed and enriched over time.
  • Open questions:
  • Unclassified publication PMIDs remain in the search set: 38141832, 35175630, 40074986, 39598410, 40326848, 35166638, 41587611, 41937093
  • Several sponsor artifacts remain program-level because no explicit study identifier was captured in cached metadata.

Provenance

  • Primary source(s):
  • ../inventories/source_registry.json
  • ../inventories/source_registry.md
  • Supporting source(s):
  • ../inventories/ctgov_priority_trials.json
  • ../inventories/publication_priority_curation.json
  • ../inventories/sponsor_priority_sources.json
  • Last verified: 2026-04-08
  • Verification status: Partial

Change Log

  • 2026-04-08: Generated or refreshed this program page from the v2 source registry and local source caches.