PXT 3003

DB16745InvestigationalSmall molecule

PXT 3003 is in phase 3 clinical trials for the treatment of Charcot-Marie-Tooth disease type 1A in adults.

Chemical structure of PXT 3003
Formula / weight
C21H27N3O2 · 353.466 g/mol (avg)
Code names
PXT-3003

Resolves to

Compound
CAS1467047-91-1
Structure
InChIKeyWRUIDZKNUAHKTR-UHFFFAOYSA-NSMILESCCOC(=O)C1=CC(NCC2=CC=CC=C2)=C(NC2CCCCC2)N=C1

What you can answer from here — as of April 23, 2025

5Clinical trialsPhase, status and sponsor resolved per trial
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Which companies are running trials of PXT 3003, in which indications and phases, and which of those programs are still active?

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