Small Molecule · BHV-2100

BHV-2100

Phase 2Biohaven Ltd. (BHVN)

Potential first-in-class, potent, selective, orally administered TRPM3 (transient receptor potential melastatin 3) channel antagonist in development as a non-opioid, non-sedating treatment for acute migraine and pain. Originated through Biohaven's license and research collaboration with KU Leuven.

Also known as: BHV-2100

Data current as of

Key facts

Modality
Small molecule
Mechanism
TRPM3 antagonist
Highest phase
Phase 2
Lead indication
Migraine
Developer
Biohaven Ltd. (BHVN)
Trials
1 tracked · 60 sites

Mechanism of action#

BHV-2100 is a first-in-class, orally bioavailable antagonist of TRPM3, a calcium-permeable cation channel expressed in trigeminal- and dorsal-root-ganglion sensory neurons and implicated in nociceptive/neuroinflammatory pain signaling and CGRP release. It inhibited human, mouse and rat TRPM3 with IC50 values in the 1-10 nM range and inhibited pregnenolone-sulfate-evoked calcium influx in human iPSC-derived sensory neurons with an IC50 of ~10.9 nM, with >1000-fold selectivity over a broad panel of other ion channels and receptors.

TargetActionAffinity
TRPM3primaryTRPM3AntagonistIC50 10.9 nMⓘ

Formulations#

FormulationRouteRegimenPharmacokinetics
BHV-2100 oral
Oral single doses; Phase 2 acute-migraine study evaluates 75 mg and 150 mg single doses. Phase 1 SAD tested single doses up to 500 mg in healthy adults.
OralSingle doset½ 10 h · Tmax 1.75 h

Development timeline#

Jul 22Jan 23Jul 23Jan 24Jul 24Jan 25Phase 24 Jun 2025 — readout (missed) — Phase 2 acute migraine trial of BHV-2100 detects no efficacy signal on the 2-hour pain-freedom and MBS co-primary endpoints24 Mar 2025 — phase change — NCT06603623 reached primary completion (CT.gov primary completion date 2025-03-24; overall status Completed) with 647 actual participants enrolled. Topline efficacy not yet publicly reported in primary sources as of 2025-05-12.10 Oct 2024 — phase change — Biohaven initiated the pivotal Phase 2 acute migraine trial (NCT06603623); study start date 2024-10-10. Announced 2024-09-30 as ~575 patients across ~60 US sites at 75 mg and 150 mg vs placebo.10 Jan 2022 — Licensing deal — Biohaven in-licenses TRPM3 antagonist platform (BHV-2100) from KU Leuven
Phase change Readout Event UpcomingHover a marker for details.
Phase 2Jan 2022 – Jun 2025
  1. missedPhase 2 acute migraine trial of BHV-2100 detects no efficacy signal on the 2-hour pain-freedom and MBS co-primary endpoints↗
  2. NCT06603623 reached primary completion (CT.gov primary completion date 2025-03-24; overall status Completed) with 647 actual participants enrolled. Topline efficacy not yet publicly reported in primary sources as of 2025-05-12.
  3. Biohaven initiated the pivotal Phase 2 acute migraine trial (NCT06603623); study start date 2024-10-10. Announced 2024-09-30 as ~575 patients across ~60 US sites at 75 mg and 150 mg vs placebo.↗
  4. Licensing dealBiohaven in-licenses TRPM3 antagonist platform (BHV-2100) from KU Leuven↗

BHV-2100 for Migraine#

Phase 2ActiveMigraine indication →

BHV-2100 is in a pivotal, registration-enabling Phase 2 trial (NCT06603623) for the acute treatment of migraine, evaluating 75 mg and 150 mg oral doses vs placebo with FDA-accepted co-primary endpoints of pain freedom and freedom from most bothersome symptom at 2 hours. The trial reached primary completion on 2025-03-24; topline efficacy had not been publicly reported as of Biohaven's Q1 2025 update (2025-05-12), which guided migraine proof-of-concept data for 1H 2025. Status set to active pending the readout.

Readouts

  • 2025-06-04ReportedTopline datamissedNCT06603623

    Phase 2 acute migraine trial of BHV-2100 detects no efficacy signal on the 2-hour pain-freedom and MBS co-primary endpoints ↗

Clinical trials#

NCT06603623Phase 2Completedn=647

Phase 2 Double-Blind, Randomized, Placebo Controlled, Efficacy and Safety Trial of BHV-2100 for the Acute Treatment of Migraine

Started Oct 2024· Primary completion Mar 2025· 60 sites across 1 country

United States

Sources#

  1. BHV-2100, A First-In-Class TRPM3 Antagonist for the Treatment of Pain (AAN 2024 oral presentation) — Biohaven Ltd. / American Academy of Neurology 2024
  2. Biohaven Enters Exclusive License with KU Leuven for TRPM3 Antagonists — Biohaven Ltd. (PR Newswire)
  3. Biohaven Initiates Pivotal Trial of Novel Investigational Drug for Treatment of Migraine — Biohaven Ltd. / PR Newswire (2024-09-30)
  4. Biohaven Ltd. Form 8-K — June 2025 Investor Presentation (BHV-2100 milestones: migraine proof-of-concept completed, no efficacy signal detected) — Biohaven Ltd. (SEC EDGAR)
  5. Efficacy and Safety Trial of BHV-2100 for the Acute Treatment of Migraine (NCT06603623) — ClinicalTrials.gov / U.S. National Library of Medicine