Drug intelligence / Profile preview

pentalafen

Development stage
Phase 2
Lead developer
Hemerion Therapeutics
Modality
Light/Condition-Responsive Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
Administration
Oral
01

Overview

Pentalafen is an oral small molecule drug containing 5-aminolevulinic acid hydrochloride (5-ALA HCl), developed specifically for use in patients with glioblastoma. It is designed as part of a combination therapy with the Heliance device to enable intraoperative photodynamic therapy (PDT). The mechanism involves two main steps: - First, after oral administration, Pentalafen accumulates selectively in tumor cells and enables fluorescence-guided surgery by highlighting tumor boundaries under specific light. - Second, following surgical resection of the tumor, residual cancer cells that have taken up Pentalafen are exposed to a specific wavelength of laser light via the Heliance device. This activates the compound within those cells and induces cytotoxic effects through photodynamic action, aiming to eradicate infiltrating glioblastoma cells left behind after surgery. The drug-device combination is currently being evaluated in phase 1/2 clinical trials for newly diagnosed grade IV glioblastoma. Early studies suggest it may improve progression-free and overall survival when added to standard-of-care treatment[1][2][3][8].

Brand names
Pentalafen
Other names
5-aminolevulinic acid hydrochloride5-ALA HCl

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