Pabinafusp Alfa
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Also known as Jr-141JR141
Summary
Pabinafusp Alfa (CHEMBL4594565) is a phase-3 clinical-stage protein (ATC A16AB27) targeting TFRC; indicated across 1 condition including mucopolysaccharidosis type 2.
At a glance
- Status: Max clinical phase 3 (not approved)
- Modality: Protein
- ATC class: A16AB27
- Targets: 1 (TFRC)
- Indications: 1 condition
- Clinical trials: 7
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL4594565 |
| Name | Pabinafusp Alfa |
| Type | Protein |
| Max phase | 3 |
| ATC | A16AB27 |
Also known as: Jr-141, JR-141, JR141, Pabinafusp alfa, PABINAFUSP ALFA
Targets
Targets
Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).
| Gene | Target | Action | pAffinity | Cancer dependency | UniProt |
|---|---|---|---|---|---|
| TFRC | transferrin receptor (CD71) | Binding | 71.8% | P02786 |
Bioactivity
No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).
Target pathways
Aggregated over 1 target gene(s): TFRC.
Top Reactome pathways
29 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Developmental Biology | 1 | TFRC |
| Signal Transduction | 1 | TFRC |
| Signaling by Rho GTPases | 1 | TFRC |
| Membrane Trafficking | 1 | TFRC |
| trans-Golgi Network Vesicle Budding | 1 | TFRC |
| Transport of small molecules | 1 | TFRC |
| Golgi Associated Vesicle Biogenesis | 1 | TFRC |
| Vesicle-mediated transport | 1 | TFRC |
| Cargo recognition for clathrin-mediated endocytosis | 1 | TFRC |
| Clathrin-mediated endocytosis | 1 | TFRC |
| RHOA GTPase cycle | 1 | TFRC |
| RHO GTPase cycle | 1 | TFRC |
| RHOB GTPase cycle | 1 | TFRC |
| RHOC GTPase cycle | 1 | TFRC |
| CDC42 GTPase cycle | 1 | TFRC |
| RAC1 GTPase cycle | 1 | TFRC |
| RAC2 GTPase cycle | 1 | TFRC |
| RHOQ GTPase cycle | 1 | TFRC |
| RHOH GTPase cycle | 1 | TFRC |
| RHOG GTPase cycle | 1 | TFRC |
| RHOJ GTPase cycle | 1 | TFRC |
| RAC3 GTPase cycle | 1 | TFRC |
| Iron uptake and transport | 1 | TFRC |
| Transferrin endocytosis and recycling | 1 | TFRC |
| RND2 GTPase cycle | 1 | TFRC |
| RND1 GTPase cycle | 1 | TFRC |
| Signaling by Rho GTPases, Miro GTPases and RHOBTB3 | 1 | TFRC |
| Differentiation of Keratinocytes in Interfollicular Epidermis in Mammalian Skin | 1 | TFRC |
| Developmental Cell Lineages | 1 | TFRC |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| response to hypoxia | 1 |
| iron ion transport | 1 |
| intracellular iron ion homeostasis | 1 |
| receptor-mediated endocytosis | 1 |
| acute-phase response | 1 |
| cell surface receptor signaling pathway | 1 |
| response to nutrient | 1 |
| response to iron ion | 1 |
| response to manganese ion | 1 |
| positive regulation of gene expression | 1 |
| negative regulation of mitochondrial fusion | 1 |
| osteoclast differentiation | 1 |
| positive regulation of B cell proliferation | 1 |
| positive regulation of protein-containing complex assembly | 1 |
| receptor internalization | 1 |
Indications & clinical
Indications
1 disease 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 |
|---|---|---|---|
| mucopolysaccharidosis type 2 | 3 | MONDO:0010674 | MONDO:0010674 |
Clinical trials
Total trials: 7.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2/PHASE3 | 2 |
| PHASE3 | 2 |
| PHASE2 | 2 |
| PHASE1/PHASE2 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT04348136 | PHASE2/PHASE3 | ACTIVE_NOT_RECRUITING | An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II |
| NCT04573023 | PHASE3 | ACTIVE_NOT_RECRUITING | A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) |
| NCT05594992 | PHASE3 | ENROLLING_BY_INVITATION | An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects |
| NCT03568175 | PHASE2/PHASE3 | COMPLETED | A Study of JR-141 in Patients With Mucopolysaccharidosis II |
| NCT03708965 | PHASE2 | ACTIVE_NOT_RECRUITING | An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II |
| NCT03128593 | PHASE1/PHASE2 | COMPLETED | A Study of JR-141 in Patients With Mucopolysaccharidosis Type II |
| NCT03359213 | PHASE2 | COMPLETED | A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21) |
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).
Related Atlas pages
- Genes: TFRC
- In clinical trials for: mucopolysaccharidosis type 2