Elamipretide

Summary

Elamipretide (CHEMBL3833370) is a phase-3 clinical-stage protein; indicated across 10 conditions including age-related macular degeneration and inborn mitochondrial myopathy.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Protein
• Indications: 10 conditions
• Clinical trials: 20
• Chemistry: 639.8 Da · C32H49N9O5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3833370
Name	Elamipretide
Type	Protein
Max phase	3
FDA approved	no
PubChem CID	11764719
Molecular formula	C32H49N9O5
Molecular weight	639.8
InChIKey	SFVLTCAESLKEHH-WKAQUBQDSA-N

SMILES: CC1=CC(=CC(=C1C[C@@H](C(=O)N[C@@H](CCCCN)C(=O)N[C@@H](CC2=CC=CC=C2)C(=O)N)NC(=O)[C@@H](CCCN=C(N)N)N)C)O

IUPAC name: (2S)-6-amino-2-[[(2S)-2-[[(2R)-2-amino-5-(diaminomethylideneamino)pentanoyl]amino]-3-(4-hydroxy-2,6-dimethylphenyl)propanoyl]amino]-N-[(2S)-1-amino-1-oxo-3-phenylpropan-2-yl]hexanamide

Also known as: Elamipretida, Elamipretide, MTP 131, MTP-131, MTP131, Ocuvia, RX 31, RX-31, RX31, SS 31, SS-31, SS31

Parent form; salt/anhydrous children: CHEMBL4298161

Patent coverage: 309 distinct patent families (722 SureChEMBL compound mentions), from 2 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

9 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
age-related macular degeneration	3	MONDO:0005150	EFO:0001365
inborn mitochondrial myopathy	3	MONDO:0009637	Orphanet:206966
heart failure	2	MONDO:0005252	EFO:0003144
congestive heart failure	2	MONDO:0005009	EFO:0000373
Barth syndrome	2	MONDO:0010543	MONDO:0010543
Leber hereditary optic neuropathy	2	MONDO:0010788	MONDO:0010788
inborn mitochondrial metabolism disorder	2	MONDO:0004069	MONDO:0044970
kidney disorder	1	MONDO:0005240	EFO:0003086
Friedreich ataxia	1	MONDO:0100339	MONDO:0100339

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 20.

Phase distribution

Phase	Trials
PHASE2	7
PHASE1	5
PHASE3	3
PHASE1/PHASE2	2
PHASE4	1
PHASE2/PHASE3	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07531251	PHASE4	NOT_YET_RECRUITING	Clinical Trial in Patients With Barth Syndrome- 4TAZPower
NCT06373731	PHASE3	ACTIVE_NOT_RECRUITING	ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD)
NCT03098797	PHASE2/PHASE3	COMPLETED	A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome
NCT03323749	PHASE3	TERMINATED	A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension
NCT05162768	PHASE3	COMPLETED	Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)
NCT02245620	PHASE2	COMPLETED	A Phase 2 Study to Evaluate the Impact of MTP-131 (Bendavia™) on Skeletal Muscle Function in Elderly
NCT02314299	PHASE1/PHASE2	COMPLETED	A Study of MTP-131 Topical Ophthalmic Solution in Subjects With Diabetic Macular Edema and Non-Exudative Intermediate Age-related Macular Degeneration
NCT02693119	PHASE2	COMPLETED	A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber’s Hereditary Optic Neuropathy
NCT02788747	PHASE2	COMPLETED	Effect of Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Reduced Ejection Fraction
NCT02805790	PHASE2	COMPLETED	Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study
NCT02814097	PHASE2	COMPLETED	A Study to Evaluate the Effects of 4 Weeks Treatment With Subcutaneous Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Preserved Ejection Fraction
NCT02914665	PHASE2	COMPLETED	A Phase 2 Study to Evaluate the Cardiac and Renal Effects of Short Term Treatment With Elamipretide in Patients Hospitalized With Congestion Due to Heart Failure
NCT02976038	PHASE2	TERMINATED	Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)
NCT05168774	PHASE1/PHASE2	COMPLETED	FRDA Investigator Initiated Study (IIS) With Elamipretide
NCT01115920	PHASE1	COMPLETED	Study to Evaluate Safety, Tolerability, and Pharmacokinetics (PK) of Intravenous (IV) Infusion of MTP-131 (Bendavia™) in Healthy Adults
NCT02388464	PHASE1	COMPLETED	A Study Investigating the Safety, Tolerability, and Pharmacokinetics of MTP-131 in Subjects With Congestive Heart Failure
NCT02388529	PHASE1	WITHDRAWN	A Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Bendavia™ (MTP-131) in Patients With Heart Failure
NCT02436447	PHASE1	COMPLETED	A Phase 1 Study Investigating the Safety and Pharmacokinetics of Repeat-dose Intravenous Infusion of MTP-131 in Subjects With Impaired Renal Function
NCT02848313	PHASE1	COMPLETED	An Open-Label, Phase 1 Clinical Study to Evaluate the Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Intermediate Age-Related Macular Degeneration
NCT04689360	Not specified	AVAILABLE	An Intermediate Size Expanded Access Protocol of Elamipretide

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).