Rosiptor

Summary

Rosiptor (CHEMBL3989954) is a phase-3 clinical-stage small molecule targeting INPP5D; indicated across 4 conditions including interstitial cystitis and atopic eczema.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 1 (INPP5D)
• Indications: 4 conditions
• Clinical trials: 7
• Chemistry: 321.5 Da · C20H35NO2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3989954
Name	Rosiptor
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	76965484
Molecular formula	C20H35NO2
Molecular weight	321.5
InChIKey	MDEJTPWQNNMAQF-BVMLLJBZSA-N

SMILES: C[C@@]1(CC[C@@H](C[C@@H]1CO)O)[C@H]2CC[C@]3([C@H]([C@@H]2CN)CCC3=C)C

IUPAC name: (1S,3S,4R)-4-[(3aS,4R,5S,7aS)-4-(aminomethyl)-7a-methyl-1-methylidene-3,3a,4,5,6,7-hexahydro-2H-inden-5-yl]-3-(hydroxymethyl)-4-methylcyclohexan-1-ol

Also known as: Aqx-1125, AQX-1125, AQX-1125 free base, Rosiptor, ROSIPTOR

Parent form; salt/anhydrous children: CHEMBL3989953

Patent coverage: 42 distinct patent families (118 SureChEMBL compound mentions), from 1 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

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
INPP5D	INPP5D	Activation		0%	Q92835

Bioactivity

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

Target pathways

Aggregated over 1 target gene(s): INPP5D.

Top Reactome pathways

20 total, by targets touching each:

Pathway	Targets	Genes
Hemostasis	1	INPP5D
Cytokine Signaling in Immune system	1	INPP5D
Adaptive Immune System	1	INPP5D
Metabolism	1	INPP5D
Inositol phosphate metabolism	1	INPP5D
PI Metabolism	1	INPP5D
Phospholipid metabolism	1	INPP5D
Synthesis of PIPs at the plasma membrane	1	INPP5D
Immune System	1	INPP5D
Synthesis of IP3 and IP4 in the cytosol	1	INPP5D
TCR signaling	1	INPP5D
Downstream TCR signaling	1	INPP5D
Cell surface interactions at the vascular wall	1	INPP5D
PECAM1 interactions	1	INPP5D
Signaling by Interleukins	1	INPP5D
Interleukin-2 family signaling	1	INPP5D
Interleukin-3, Interleukin-5 and GM-CSF signaling	1	INPP5D
Metabolism of lipids	1	INPP5D
Interleukin receptor SHC signaling	1	INPP5D
Signaling by CSF1 (M-CSF) in myeloid cells	1	INPP5D

Dominant GO biological processes

GO term	Targets
phosphatidylinositol biosynthetic process	1
phosphate-containing compound metabolic process	1
apoptotic process	1
signal transduction	1
determination of adult lifespan	1
negative regulation of signal transduction	1
immunoglobulin mediated immune response	1
negative regulation of B cell proliferation	1
negative regulation of interleukin-6 production	1
intracellular signal transduction	1
positive regulation of apoptotic process	1
positive regulation of B cell differentiation	1
positive regulation of erythrocyte differentiation	1
negative regulation of monocyte differentiation	1
negative regulation of neutrophil differentiation	1

Indications & clinical

Indications

4 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
interstitial cystitis	3	MONDO:0018301	EFO:0008507
atopic eczema	2	MONDO:0004980	EFO:0000274
chronic obstructive pulmonary disease	2	MONDO:0005002	EFO:0000341

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

Clinical trials

Total trials: 7.

Phase distribution

Phase	Trials
PHASE2	4
PHASE1	2
PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02858453	PHASE3	UNKNOWN	Efficacy and Safety of 2 Doses of AQX-1125 in Subjects With Interstitial Cystitis / Bladder Pain Syndrome
NCT01882543	PHASE2	COMPLETED	Efficacy and Safety of AQX-1125 in IC/BPS
NCT01954628	PHASE2	COMPLETED	Efficacy and Safety of AQX-1125 in Unstable COPD
NCT02324972	PHASE2	COMPLETED	Pilot Efficacy and Safety Study of AQX-1125 in Atopic Dermatitis
NCT03500159	PHASE2	TERMINATED	Efficacy and Safety of AQX-1125 in Subjects With Chronic Prostatitis/Chronic Pelvic Pain Syndrome
NCT03185195	PHASE1	COMPLETED	Phase 1 Absorption Distribution Metabolism and Excretion of AQX-1125
NCT03478020	PHASE1	COMPLETED	Oral AQX-1125 and Combination Oral Contraceptive Drug-Drug Interaction Study

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

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

3 molecules share ≥1 primary target. Top 3 by shared-target count:

Molecule	Source	Status	Shared targets
MOLIBRESIB	ChEMBL	Phase 2	INPP5D
Ephedrine	PubChem	Approved	INPP5D
Pseudoephedrine	PubChem	Approved	INPP5D