Migraine Pipeline 2026: Drugs in Trials & Targets

Remission & chronicity

Spontaneous remission

Migraine frequency fluctuates; spontaneous remission (no attacks for 12+ months) occurs in ~25% over 10-year follow-up; 40% of migraine sufferers qualify for preventive therapy but only 3-13% receive it

Best-intervention remission

CGRP monoclonal antibodies (erenumab, fremanezumab, galcanezumab) achieve ≥50% reduction in monthly migraine days in 50-70% of patients; complete response (near-zero days) in 10-30%; acute triptans achieve 2-hour pain freedom in 30-40%

Gap size

Large — primarily an access/utilization gap for preventive therapy

Primary barrier

practice/access

Only 3-13% of the ~10 million US adults who qualify for preventive therapy receive it; prior authorization barriers for CGRP mAbs are substantial; widespread underdiagnosis; opioid prescribing for migraine in ED associated with medication overuse headache

Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: Clinical > Mechanistic > Correlative.

CACNA1A 18 CALCA 27 CALCRL 159 HTR1B 198 HTR1D 153 HTR1F 139 NOS3 0 RAMP1 144 TRPV1 160

CACNA1A

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
PREGABALIN	~ modulates	Mechanistic		DGIdb
TERODILINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
GABAPENTIN	~ modulates	Mechanistic		DGIdb
CELECOXIB	? unclear	Mechanistic		DGIdb
ELPETRIGINE	↓ inhibits	Mechanistic		DGIdb
NISOLDIPINE	? unclear	Mechanistic		DGIdb
BEPRIDIL	? unclear	Mechanistic		DGIdb
IMAGABALIN	~ modulates	Mechanistic		DGIdb
ATAGABALIN	~ modulates	Mechanistic		DGIdb
ANTIDEPRESSANT AGENT	? unclear	Mechanistic		DGIdb
PHLOROGLUCINOL	↓ inhibits	Mechanistic		DGIdb
GABAPENTIN ENACARBIL	~ modulates	Mechanistic		DGIdb
SULOCTIDIL	↓ inhibits	Mechanistic		DGIdb
BEPRIDIL HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
FLUNARIZINE	? unclear	Mechanistic		DGIdb
TERODILINE	? unclear	Mechanistic		Open Targets
CHEMBL2206242	↑ activates	Mechanistic	8800.0 nM (EC50)	ChEMBL
CHEMBL2017291	↓ inhibits	Mechanistic	1260.0 nM (IC50)	ChEMBL

CALCA

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Source
NATEGLINIDE	? unclear	Mechanistic	DGIdb
BUDIODARONE	? unclear	Mechanistic	DGIdb
CIZOLIRTINE	? unclear	Mechanistic	DGIdb
IH636 GRAPE SEED PROANTHOCYANIDIN EXTRACT	? unclear	Mechanistic	DGIdb
AMIODARONE HYDROCHLORIDE	? unclear	Mechanistic	DGIdb
INCOBOTULINUMTOXINA	? unclear	Mechanistic	DGIdb
RANITIDINE HYDROCHLORIDE	? unclear	Mechanistic	DGIdb
AMIFAMPRIDINE	? unclear	Mechanistic	DGIdb
DRONEDARONE	? unclear	Mechanistic	DGIdb
CHEMBL:CHEMBL42661	? unclear	Mechanistic	DGIdb
AZIMILIDE	? unclear	Mechanistic	DGIdb
PINACIDIL	? unclear	Mechanistic	DGIdb
FENTANYL CITRATE	? unclear	Mechanistic	DGIdb
TESTOSTERONE PROPIONATE	? unclear	Mechanistic	DGIdb
FREMANEZUMAB	↓ inhibits	Mechanistic	DGIdb
SULCARDINE SULFATE	? unclear	Mechanistic	DGIdb
PREDNISONE	? unclear	Mechanistic	DGIdb
DOFETILIDE	? unclear	Mechanistic	DGIdb
ANDOLAST	? unclear	Mechanistic	DGIdb
IBUTILIDE	? unclear	Mechanistic	DGIdb
EPTINEZUMAB	↓ inhibits	Mechanistic	DGIdb
CHEMBL:CHEMBL1563246	? unclear	Mechanistic	DGIdb
MINOXIDIL	? unclear	Mechanistic	DGIdb
DALFAMPRIDINE	? unclear	Mechanistic	DGIdb
BIMAKALIM	? unclear	Mechanistic	DGIdb
GALCANEZUMAB	↓ inhibits	Mechanistic	DGIdb
DIAZOXIDE	? unclear	Mechanistic	DGIdb

CALCRL

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
DAVALINTIDE	↑ activates	Mechanistic		DGIdb
FREMANEZUMAB	? unclear	Mechanistic		DGIdb
MK-3207	? unclear	Mechanistic		DGIdb
ZAVEGEPANT HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
TELCAGEPANT	↓ inhibits	Mechanistic		DGIdb
RIMEGEPANT	↓ inhibits	Mechanistic		DGIdb
CALCITONIN GENE-RELATED PEPTIDE	? unclear	Mechanistic		DGIdb
ERENUMAB	↓ inhibits	Mechanistic		DGIdb
OLCEGEPANT	↓ inhibits	Mechanistic		DGIdb
BI-44370	↓ inhibits	Mechanistic		DGIdb
RIMEGEPANT SULFATE	↓ inhibits	Mechanistic		DGIdb
MK3207	↓ inhibits	Mechanistic		DGIdb
ATOGEPANT	↓ inhibits	Mechanistic		DGIdb
UBROGEPANT	↓ inhibits	Mechanistic		DGIdb
GALCANEZUMAB	? unclear	Mechanistic		DGIdb
ZAVEGEPANT	↓ inhibits	Mechanistic		DGIdb
CHEMBL329678	↓ inhibits	Mechanistic	41000.0 nM (IC50)	ChEMBL
CHEMBL316211	↓ inhibits	Mechanistic	96000.0 nM (IC50)	ChEMBL
CHEMBL88111	↓ inhibits	Mechanistic	74000.0 nM (IC50)	ChEMBL
CHEMBL88441	↓ inhibits	Mechanistic	12000.0 nM (IC50)	ChEMBL
CHEMBL313797	↓ inhibits	Mechanistic	17000.0 nM (IC50)	ChEMBL
CHEMBL87174	↓ inhibits	Mechanistic	33000.0 nM (IC50)	ChEMBL
CHEMBL419684	↓ inhibits	Mechanistic	5900.0 nM (IC50)	ChEMBL
CHEMBL314992	↓ inhibits	Mechanistic	5200.0 nM (IC50)	ChEMBL
CHEMBL89148	↓ inhibits	Mechanistic	9900.0 nM (IC50)	ChEMBL
CHEMBL88582	↓ inhibits	Mechanistic	37000.0 nM (IC50)	ChEMBL
CHEMBL3037882	↓ inhibits	Mechanistic	2.0 nM (IC50)	ChEMBL
CHEMBL1794015	↓ inhibits	Mechanistic	185.0 nM (IC50)	ChEMBL
CHEMBL2369742	↓ inhibits	Mechanistic	4.0 nM (IC50)	ChEMBL
CHEMBL2369452	↓ inhibits	Mechanistic	9.0 nM (IC50)	ChEMBL
CHEMBL2369457	↓ inhibits	Mechanistic	11.2 nM (IC50)	ChEMBL
CHEMBL2369465	↓ inhibits	Mechanistic	25.0 nM (IC50)	ChEMBL
CHEMBL2369463	↓ inhibits	Mechanistic	2.3 nM (IC50)	ChEMBL
CHEMBL525571	↓ inhibits	Mechanistic	2.3 nM (IC50)	ChEMBL
CHEMBL2369458	↓ inhibits	Mechanistic	6.9 nM (IC50)	ChEMBL
CHEMBL2369459	↓ inhibits	Mechanistic	2.0 nM (IC50)	ChEMBL
CHEMBL2369464	↓ inhibits	Mechanistic	20.2 nM (IC50)	ChEMBL
CHEMBL2369454	↓ inhibits	Mechanistic	3.0 nM (IC50)	ChEMBL
CHEMBL404977	↓ inhibits	Mechanistic	24000.0 nM (IC50)	ChEMBL
CHEMBL216710	↓ inhibits	Mechanistic	1200.0 nM (IC50)	ChEMBL

Showing top 40 of 159 agents ranked by evidence tier.

HTR1B

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
FLUPHENAZINE HYDROCHLORIDE	? unclear	Mechanistic		DGIdb
ALMOTRIPTAN MALATE	↑ activates	Mechanistic		DGIdb
TRELANSERIN	↓ inhibits	Mechanistic		DGIdb
SUMATRIPTAN	↑ activates	Mechanistic	7.7 nM (Ki)	DGIdb
RIZATRIPTAN BENZOATE	↑ activates	Mechanistic		DGIdb
DONITRIPTAN	↑ activates	Mechanistic		DGIdb
AMISULPRIDE	↓ inhibits	Mechanistic		DGIdb
NXN-188	? unclear	Mechanistic		DGIdb
ELTOPRAZINE	? unclear	Mechanistic		DGIdb
FROVATRIPTAN	? unclear	Mechanistic		DGIdb
CITALOPRAM	? unclear	Mechanistic		DGIdb
NARATRIPTAN HYDROCHLORIDE	↑ activates	Mechanistic		DGIdb
FROVATRIPTAN SUCCINATE	↑ activates	Mechanistic		DGIdb
ALMOTRIPTAN	? unclear	Mechanistic		DGIdb
ZOLMITRIPTAN	↑ activates	Mechanistic	6.166 nM (IC50)	DGIdb
ELETRIPTAN	↑ activates	Mechanistic		DGIdb
ERGOTAMINE	? unclear	Mechanistic		DGIdb
GSK163090	↓ inhibits	Mechanistic		DGIdb
NEFAZODONE	? unclear	Mechanistic		DGIdb
LITHIUM	? unclear	Mechanistic		DGIdb
CLOMIPRAMINE	? unclear	Mechanistic		DGIdb
ERGOLOID MESYLATES, USP	↑ activates	Mechanistic		DGIdb
ASENAPINE	? unclear	Mechanistic		DGIdb
DIHYDROERGOTAMINE MESYLATE	? unclear	Mechanistic		DGIdb
PAROXETINE HYDROCHLORIDE, HEMIHYDRATE	? unclear	Mechanistic		DGIdb
LIOTHYRONINE	? unclear	Mechanistic		DGIdb
VORTIOXETINE HYDROBROMIDE	? unclear	Mechanistic		DGIdb
NARATRIPTAN	? unclear	Mechanistic		DGIdb
ZIMELDINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
DEXFENFLURAMINE HYDROCHLORIDE	↑ activates	Mechanistic		DGIdb
DRUGSATFDA.NDA:016727	? unclear	Mechanistic		DGIdb
OLANZAPINE	? unclear	Mechanistic		DGIdb
ELZASONAN HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
FLUOXETINE HYDROCHLORIDE	? unclear	Mechanistic		DGIdb
ELZASONAN	? unclear	Mechanistic		Open Targets
ELETRIPTAN HYDROBROMIDE	? unclear	Mechanistic		Open Targets
RIZATRIPTAN	? unclear	Mechanistic		Open Targets
DEXFENFLURAMINE	? unclear	Mechanistic		Open Targets
ERGOLOID MESYLATES	? unclear	Mechanistic		Open Targets
ZIMELDINE	? unclear	Mechanistic		Open Targets

Showing top 40 of 198 agents ranked by evidence tier.

HTR1D

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
FROVATRIPTAN	? unclear	Mechanistic		DGIdb
TONABERSAT	? unclear	Mechanistic		DGIdb
SUMATRIPTAN	↑ activates	Mechanistic	39.81 nM (IC50)	DGIdb
ELZASONAN HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
ZOLMITRIPTAN	↑ activates	Mechanistic		DGIdb
ELETRIPTAN	↑ activates	Mechanistic		DGIdb
ALMOTRIPTAN MALATE	↑ activates	Mechanistic		DGIdb
ERGOTAMINE	? unclear	Mechanistic		DGIdb
ERGOTAMINE TARTRATE	↑ activates	Mechanistic		DGIdb
ALMOTRIPTAN	? unclear	Mechanistic		DGIdb
NARATRIPTAN	? unclear	Mechanistic		DGIdb
OLANZAPINE	? unclear	Mechanistic		DGIdb
NARATRIPTAN HYDROCHLORIDE	↑ activates	Mechanistic		DGIdb
DONITRIPTAN	↑ activates	Mechanistic		DGIdb
RIZATRIPTAN BENZOATE	↑ activates	Mechanistic		DGIdb
CHEMBL:CHEMBL490417	? unclear	Mechanistic		DGIdb
FENFLURAMINE HYDROCHLORIDE	↑ activates	Mechanistic		DGIdb
AMISULPRIDE	↓ inhibits	Mechanistic		DGIdb
PIROMELATINE	? unclear	Mechanistic		DGIdb
GSK163090	↓ inhibits	Mechanistic		DGIdb
FROVATRIPTAN SUCCINATE	↑ activates	Mechanistic		DGIdb
NXN-188	? unclear	Mechanistic		DGIdb
ZIMELDINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
DEXFENFLURAMINE HYDROCHLORIDE	↑ activates	Mechanistic		DGIdb
ZIPRASIDONE	? unclear	Mechanistic		DGIdb
DIHYDROERGOTAMINE MESYLATE	↑ activates	Mechanistic		DGIdb
DEXFENFLURAMINE	? unclear	Mechanistic		Open Targets
ELETRIPTAN HYDROBROMIDE	? unclear	Mechanistic		Open Targets
DIHYDROERGOTAMINE	? unclear	Mechanistic		Open Targets
SUMATRIPTAN SUCCINATE	? unclear	Mechanistic		Open Targets
RIZATRIPTAN	? unclear	Mechanistic	79.43 nM (IC50)	Open Targets
FENFLURAMINE	? unclear	Mechanistic		Open Targets
ELZASONAN	? unclear	Mechanistic		Open Targets
ZIMELDINE	? unclear	Mechanistic		Open Targets
GR-127935	↓ inhibits	Mechanistic	0.7 nM (Ki)	ChEMBL
CHEMBL321818	↓ inhibits	Mechanistic	2.0 nM (Ki)	ChEMBL
CHEMBL292779	↓ inhibits	Mechanistic	0.6 nM (IC50)	ChEMBL
CHEMBL325131	↓ inhibits	Mechanistic	340.0 nM (Ki)	ChEMBL
CHEMBL107772	↓ inhibits	Mechanistic	3.6 nM (Ki)	ChEMBL
CHEMBL304857	↓ inhibits	Mechanistic	2.2 nM (IC50)	ChEMBL

Showing top 40 of 153 agents ranked by evidence tier.

HTR1F

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
LASMIDITAN SUCCINATE	↑ activates	Mechanistic		DGIdb
SUMATRIPTAN	? unclear	Mechanistic	25.7 nM (Ki)	DGIdb
ZOLMITRIPTAN	? unclear	Mechanistic		DGIdb
AMISULPRIDE	↓ inhibits	Mechanistic		DGIdb
ALMOTRIPTAN MALATE	↑ activates	Mechanistic		DGIdb
[3H]LY334370	? unclear	Mechanistic		DGIdb
DEXFENFLURAMINE HYDROCHLORIDE	↑ activates	Mechanistic		DGIdb
ELETRIPTAN	↑ activates	Mechanistic		DGIdb
ZIMELDINE HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
NARATRIPTAN	? unclear	Mechanistic		DGIdb
LASMIDITAN	↑ activates	Mechanistic	87.84 nM (IC50)	DGIdb
ELETRIPTAN HYDROBROMIDE	? unclear	Mechanistic		Open Targets
ALMOTRIPTAN	? unclear	Mechanistic		Open Targets
ZIMELDINE	? unclear	Mechanistic		Open Targets
DEXFENFLURAMINE	? unclear	Mechanistic		Open Targets
CHEMBL292759	↓ inhibits	Mechanistic	316.23 nM (Ki)	ChEMBL
CHEMBL29846	↓ inhibits	Mechanistic	251.19 nM (Ki)	ChEMBL
CHEMBL29433	↓ inhibits	Mechanistic	316.23 nM (Ki)	ChEMBL
CHEMBL282971	↓ inhibits	Mechanistic	199.53 nM (Ki)	ChEMBL
CHEMBL292779	↓ inhibits	Mechanistic	3800.0 nM (IC50)	ChEMBL
CHEMBL178066	↓ inhibits	Mechanistic	8.2 nM (Ki)	ChEMBL
CHEMBL177258	↓ inhibits	Mechanistic	3.1 nM (Ki)	ChEMBL
CHEMBL369705	↓ inhibits	Mechanistic	2.9 nM (Ki)	ChEMBL
CHEMBL177238	↓ inhibits	Mechanistic	3.0 nM (Ki)	ChEMBL
CHEMBL101690	↓ inhibits	Mechanistic	1.6 nM (Ki)	ChEMBL
CHEMBL103479	↓ inhibits	Mechanistic	7.6 nM (Ki)	ChEMBL
CHEMBL369391	↓ inhibits	Mechanistic	13.5 nM (Ki)	ChEMBL
CHEMBL173949	↓ inhibits	Mechanistic	2.3 nM (Ki)	ChEMBL
CHEMBL2110300	↓ inhibits	Mechanistic	363.08 nM (IC50)	ChEMBL
CHEMBL432713	↓ inhibits	Mechanistic	3.162 nM (Ki)	ChEMBL
CHEMBL92139	↓ inhibits	Mechanistic	3.9 nM (Ki)	ChEMBL
CHEMBL76237	↓ inhibits	Mechanistic	2500.0 nM (Ki)	ChEMBL
CHEMBL263700	↓ inhibits	Mechanistic	29.0 nM (Ki)	ChEMBL
CHEMBL126340	↓ inhibits	Mechanistic	570.0 nM (Ki)	ChEMBL
CHEMBL125745	↓ inhibits	Mechanistic	47.0 nM (Ki)	ChEMBL
CHEMBL317535	↓ inhibits	Mechanistic	2000.0 nM (Ki)	ChEMBL
CHEMBL93868	↓ inhibits	Mechanistic	770.0 nM (Ki)	ChEMBL
CHEMBL321133	↓ inhibits	Mechanistic	16.0 nM (Ki)	ChEMBL
CHEMBL104753	↓ inhibits	Mechanistic	7.4 nM (Ki)	ChEMBL
CHEMBL105478	↓ inhibits	Mechanistic	18.0 nM (Ki)	ChEMBL

Showing top 40 of 139 agents ranked by evidence tier.

NOS3

UniProt Ensembl DGIdb Open Targets

Pipeline results not yet available for this target. Run the pipeline to populate.

RAMP1

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
DAVALINTIDE	↑ activates	Mechanistic		DGIdb
ZAVEGEPANT	↓ inhibits	Mechanistic		DGIdb
PRAMLINTIDE ACETATE	↑ activates	Mechanistic		DGIdb
PRAMLINTIDE	? unclear	Mechanistic	23.44 nM (EC50)	DGIdb
CHEMBL264010	↓ inhibits	Mechanistic	14.0 nM (Ki)	ChEMBL
CHEMBL406459	↓ inhibits	Mechanistic	76.8 nM (Ki)	ChEMBL
CHEMBL2371892	↓ inhibits	Mechanistic	70.5 nM (Ki)	ChEMBL
CHEMBL2371890	↓ inhibits	Mechanistic	6.31 nM (Kd)	ChEMBL
CHEMBL385986	↓ inhibits	Mechanistic	25.12 nM (Kd)	ChEMBL
CHEMBL386763	↓ inhibits	Mechanistic	10.0 nM (Kd)	ChEMBL
CHEMBL2371891	↓ inhibits	Mechanistic	31.62 nM (Kd)	ChEMBL
CHEMBL427759	↓ inhibits	Mechanistic	0.7 nM (Ki)	ChEMBL
CHEMBL208148	↓ inhibits	Mechanistic	4250.0 nM (Ki)	ChEMBL
CHEMBL381585	↓ inhibits	Mechanistic	83.0 nM (Ki)	ChEMBL
CHEMBL381798	↓ inhibits	Mechanistic	2370.0 nM (Ki)	ChEMBL
CHEMBL381710	↓ inhibits	Mechanistic	61.0 nM (Ki)	ChEMBL
CHEMBL424864	↓ inhibits	Mechanistic	44.0 nM (Ki)	ChEMBL
CHEMBL380514	↓ inhibits	Mechanistic	1060.0 nM (Ki)	ChEMBL
CHEMBL211052	↓ inhibits	Mechanistic	897.0 nM (Ki)	ChEMBL
CHEMBL207186	↓ inhibits	Mechanistic	2200.0 nM (Ki)	ChEMBL
CHEMBL379686	↓ inhibits	Mechanistic	8400.0 nM (Ki)	ChEMBL
OLCEGEPANT	↓ inhibits	Mechanistic	0.1 nM (IC50)	ChEMBL
CHEMBL235457	↓ inhibits	Mechanistic	35.0 nM (Ki)	ChEMBL
CHEMBL235456	↓ inhibits	Mechanistic	266.0 nM (Ki)	ChEMBL
CHEMBL238011	↓ inhibits	Mechanistic	65.0 nM (IC50)	ChEMBL
CHEMBL392132	↓ inhibits	Mechanistic	29.0 nM (Ki)	ChEMBL
CHEMBL394229	↓ inhibits	Mechanistic	29.0 nM (Ki)	ChEMBL
CHEMBL235237	↓ inhibits	Mechanistic	2.0 nM (Ki)	ChEMBL
CHEMBL429262	↓ inhibits	Mechanistic	25.0 nM (Ki)	ChEMBL
CHEMBL235015	↓ inhibits	Mechanistic	5828.0 nM (Ki)	ChEMBL
CHEMBL393823	↓ inhibits	Mechanistic	31.0 nM (Ki)	ChEMBL
CHEMBL428547	↓ inhibits	Mechanistic	689.0 nM (Ki)	ChEMBL
CHEMBL395599	↓ inhibits	Mechanistic	934.0 nM (Ki)	ChEMBL
CHEMBL397054	↓ inhibits	Mechanistic	1993.0 nM (Ki)	ChEMBL
CHEMBL236936	↓ inhibits	Mechanistic	11100.0 nM (Ki)	ChEMBL
CHEMBL393523	↓ inhibits	Mechanistic	1800.0 nM (Ki)	ChEMBL
CHEMBL234989	↓ inhibits	Mechanistic	7800.0 nM (Ki)	ChEMBL
CHEMBL238203	↓ inhibits	Mechanistic	3700.0 nM (Ki)	ChEMBL
CHEMBL236516	↓ inhibits	Mechanistic	471.0 nM (IC50)	ChEMBL
CHEMBL2035986	↓ inhibits	Mechanistic	0.25 nM (Ki)	ChEMBL

Showing top 40 of 144 agents ranked by evidence tier.

TRPV1

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
STREPTOZOCIN	? unclear	Mechanistic		DGIdb
BENZYL ISOTHIOCYANATE	? unclear	Mechanistic		DGIdb
ACETAMINOPHEN	↑ activates	Mechanistic		DGIdb
POLYGODIAL	? unclear	Mechanistic		DGIdb
PROPOFOL	? unclear	Mechanistic		DGIdb
SAF312	↓ inhibits	Mechanistic		DGIdb
GRC-15300	? unclear	Mechanistic		DGIdb
AZD1386	↓ inhibits	Mechanistic		DGIdb
TIVANISIRAN	↓ inhibits	Mechanistic		DGIdb
MAVATREP	↓ inhibits	Mechanistic		DGIdb
MR-1817	↓ inhibits	Mechanistic		DGIdb
FERRUGINENE B	? unclear	Mechanistic		DGIdb
CAPSAICIN	↑ activates	Mechanistic	19.95 nM (EC50)	DGIdb
RECOMBINANT TUMOR NECROSIS FACTOR FAMILY PROTEIN	? unclear	Mechanistic		DGIdb
PHENETHYLISOTHIOCYANATE	? unclear	Mechanistic		DGIdb
VOACANGINE	? unclear	Mechanistic		DGIdb
RESINIFERATOXIN	↑ activates	Mechanistic	23.1 nM (Ki)	DGIdb
DWP05195	↓ inhibits	Mechanistic		DGIdb
XEN-D0501	↓ inhibits	Mechanistic		DGIdb
PAC-14028	↓ inhibits	Mechanistic		DGIdb
SB-705498	↓ inhibits	Mechanistic		DGIdb
MASOPROCOL	? unclear	Mechanistic		DGIdb
ALLYL ISOTHIOCYANATE	? unclear	Mechanistic		DGIdb
NGD-8243	↓ inhibits	Mechanistic		DGIdb
GRC-6211	↓ inhibits	Mechanistic		DGIdb
RESERPINE	? unclear	Mechanistic		DGIdb
NABIXIMOLS	? unclear	Mechanistic		DGIdb
A425619	? unclear	Mechanistic		DGIdb
PHE377	↓ inhibits	Mechanistic		DGIdb
ANTISENSE OLIGONUCLEOTIDES	? unclear	Mechanistic		DGIdb
WORTMANNIN	? unclear	Mechanistic		DGIdb
JTS-653	↓ inhibits	Mechanistic		DGIdb
LEVODOPA	? unclear	Mechanistic		DGIdb
AMG517	↓ inhibits	Mechanistic		DGIdb
CAPSAZEPINE	? unclear	Mechanistic	120.0 nM (Ki)	DGIdb
ZUCAPSAICIN	↑ activates	Mechanistic		DGIdb
INDOMETHACIN	? unclear	Mechanistic		DGIdb
CHEMBL:CHEMBL191534	? unclear	Mechanistic		DGIdb
ABT-102	↓ inhibits	Mechanistic		DGIdb
ISOPROPYL ISOTHIOCYANATE	? unclear	Mechanistic		DGIdb

Showing top 40 of 160 agents ranked by evidence tier.

Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is Migraine: recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

Efficacy and Safety of Erenumab in Pediatric Subjects With Chronic Migraine Completed

NCT03832998 Phase: 3 Started: 2019-09-05

This study will evaluate the efficacy and safety of erenumab in migraine prevention in children (6 to \<12 years) and adolescents (12 to \<18 years) with chronic migraine. The study hypothesis is that in pediatric participants with chronic migraine, the combined erenumab dose group has a greater red...

A Study to Investigate the Safety and Efficacy of MEDI0618 Compared to Placebo in Adult Participants With Episodic Migraine Active Not Recruiting

NCT06602479 Phase: 2 Started: 2024-10-07

The purpose of this Phase 2 study is to evaluate the safety and efficacy of SC MEDI0618 compared to placebo in participants with episodic migraine.

A Prospective, Open-Label Study Evaluating the Effectiveness of Gepants for Acute Treatment of Migraine in the Emergency Department Recruiting

NCT07667621 Phase: 1 Started: 2026-09-15

Prospective, single-site, open-label, non-controlled pilot study consisting of two parallel trials. Participants will receive rimegepant or zavegepant for acute migraine treatment during a single Emergency Department visit, with post-discharge follow-up through 28 days. Participants will be allocate...

Long-term Safety Study of Rimegepant in Pediatric Subjects for the Acute Treatment of Migraine Recruiting

NCT04743141 Phase: 3 Started: 2021-04-28

The purpose of this study is to test the long-term safety of rimegepant in the acute treatment of migraine in children and adolescents (≥ 6 to \< 18 years of age).

Randomized Study in Children and Adolescents With Migraine: Acute Treatment Recruiting

NCT04649242 Phase: 3 Started: 2021-01-15

The purpose of this study is to test the safety and efficacy of BHV-3000 versus placebo in the acute treatment of moderate or severe migraine in children and adolescents.

Efficacy and Safety of Rizatriptan-Naproxen (10/550 mg) in the Acute Treatment of Migraine Completed

NCT04384367 Phase: 3 Started: 2022-12-30

A phase III study, multicenter, double-blind, double-dummy, randomized, single-dose, placebo-controlled study to evaluate the efficacy and safety of Rizatriptan-Naproxen (10/550 mg) in the acute treatment of migraine. ⚠️Study will only be conducted in research centers in Brazil (please do not send e...

A Study to Learn About the Study Medicine Called Rimegepant in Adolescents With Frequent Migraine Active Not Recruiting

NCT06616194 Phase: 3 Started: 2024-11-15

The purpose of this study is to learn about the effect of a study medicine called rimegepant in adolescents who have frequent migraine attacks. Rimegepant is a tablet that disintegrates when you put it on or under your tongue. The study will enroll participants who have headache for 15 days (or more...

Efficacy and Safety of BR005-036C Tablets for the Acute Treatment of Migraine in Adults Recruiting

NCT07792681 Phase: 2/3 Started: 2026-09-30

The purpose of this study is to compare the efficacy and safety of BR005-036C versus placebo in subjects with Acute Migraines

A Phase IIb, Randomized, Double-Blind, Placebo-Controlled Study of Elismetrep (K-304) in the Treatment of Migraine Completed

NCT06848075 Phase: 2 Started: 2025-03-11

This is a double-blind, randomized, multicenter, outpatient evaluation of the safety and efficacy of elismetrep as compared to placebo in the acute treatment of migraine.

Comparative Effectiveness of Migraine Preventive Medications: The APT Comparison Study Recruiting

NCT06972056 Phase: 4 Started: 2025-07-09

This goal of this study is to compare three medications used for migraine preventive treatment. This study will compare atogepant, a newer migraine preventive medication, with two older preventive medications, topiramate and propranolol. It will be determined if one works better and is more tolerabl...

A Study With Eptinezumab in Adolescents (12-17 Years) With Chronic Migraine Recruiting

NCT04965675 Phase: 3 Started: 2021-06-30

To find out if eptinezumab is better than placebo (normal saline solution) in lowering the number of days with migraine in young people ages 12 to 17 with chronic migraine.

A Long-Term Safety Study of Elismetrep (K-304) in the Acute Treatment of Migraine Recruiting

NCT07674654 Phase: 3 Started: 2026-08-03

This is a long-term safety study to evaluate the safety and tolerability of intermittent use of elismetrep in the acute treatment of migraine attacks.

A Study to Find Out if the Medicine Called Rimegepant is Safe for Adults in India Recruiting

NCT07685769 Phase: 4 Started: 2026-07-24

This is a multicenter, single-arm, open-label Phase 4 study evaluating the safety and tolerability of rimegepant 75 mg orally disintegrating tablet (ODT) for the acute treatment of migraine (with or without aura) in adult patients in India who have had an insufficient response, intolerance, or contr...

A Study to Learn About the Study Medicine Called Rimegepant in Adults When Used for the Prevention of Chronic Migraine Recruiting

NCT07729462 Phase: 3 Started: 2026-08-26

Rimegepant is a calcitonin gene-related peptide (CGRP) receptor antagonist approved for the acute treatment of migraine attacks in adults and for the preventive treatment of episodic migraine in adults when administered every other day. This study evaluates once daily rimegepant for the preventive t...

A Study With Eptinezumab in Children and Adolescents (6 to 17 Years) With Chronic or Episodic Migraine Recruiting

NCT05164172 Phase: 3 Started: 2021-12-01

The main goal of the study is to assess the long-term safety of eptinezumab on children and adolescents ages 6 to 17 with chronic or episodic migraine.

Agents Found By Disease-Level Search

Searched directly from “Migraine” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 9-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

37 agents shown (approved or with trial evidence) — 30 not found by the gene-target search above.

Dihydroergotamine

Approved · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT05337254 NCT05337254 NCT05337254

Rimegepant

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT06439628 NCT03237845

Frovatriptan

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00644033 NCT00644033

PRT-064040 nasal sprayNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT07016516 NCT07016516

SPHERENOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02710942

PRISMNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02710942

MAbsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06549270

topiramateNOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00210873

"Investigational"Auto-injector (sumatriptan succinate)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00510419

IbudilastNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01389193

almotriptan malate

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00210509

Rizatriptan 5 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01286207

Rizatriptan 10 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01286207

Standard CareNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01286207

Galcanezumab

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02163993

fremanezumab (TEV-48125)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT03303092

KetamineNOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02697071

erenumab (AMG334)NOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov
NCT03927144

Oral ProphylacticNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03927144

Fremanezumab

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT04464707

erenumab-aooeNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06150781

Rimegepant 75 MGNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05709106

DFN-02NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02279082

Rimegepant/BHV3000NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04649242

Antipaletet drugNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06953622

Patent Foramen Ovale ClosureNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06953622

Botulinum toxin type ANOT in gene-target list

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT03381261

Lidocaine 2 % PharmacopunctureNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07067853

propranolol 20mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07067853

Palmitoylethanolamide sold as Levagen +NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05046522

Lasmiditan 50mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04218162

Lasmiditan 100mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04218162

OnabotulinumtoxinANOT in gene-target list

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01071096

DFN-15 Dose A (treatment A)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02472418

DFN-15 Dose B (treatment B)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02472418

Eptinezumab

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT07035197

Active drug for chronic migraine treatment.NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07071506

Migraine

Remission & chronicity

Biomarker Targets & Therapeutic Agents

CACNA1A

CALCA

CALCRL

HTR1B

HTR1D

HTR1F

NOS3

RAMP1

TRPV1

Clinical Trials

Agents Found By Disease-Level Search

The research patients need, made open source.

Conditions

Data

Tools

Foundation