Nesolicaftor

DB15239InvestigationalSmall molecule

Nesolicaftor (PTI-428) is under investigation in clinical trial NCT03258424 (Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO® as Background Therapy).

Mechanism: Pharmacologically active on Cystic fibrosis transmembrane conductance regulator
Curator reviewed
Formula / weight: C₁₈H₁₈N₄O₄ · 354.366 g/mol (avg)
Also known as: Nesolicitor
Code names: PTI-428

Resolves to

Compound

UNIIT2U4ZA62U4CAS1953130-87-4ChEMBLCHEMBL4297479

Structure

InChIKeyXPEHHUISIBFLHX-RAIGVLPGSA-NSMILESC[C@@H](O)C1=NN=C(O1)[C@H]1C[C@@H](C1)NC(=O)C1=CC(=NO1)C1=CC=CC=C1

Targets

P13569CFTR

What you can answer from here — as of July 08, 2026

1Protein targetEach mapped to UniProt, with action and pharmacological action

Listed above

4Clinical trialsPhase, status and sponsor resolved per trial

3+ReferencesStructured and connected to the statements they support

Which drugs share a target with Nesolicaftor, and which of those have an active Phase 3 trial?

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