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PNM-201 is a targeted cytotoxin (immunotoxin) being developed by Precision NeuroMed for the treatment of glioblastoma (GBM). It consists of a recombinant fusion protein combining human interleukin-13 (IL13) with a truncated version of Pseudomonas exotoxin A (PE38). The drug specifically targets glioblastoma tumor stem cells (TSCs) that overexpress the IL13Ra2 receptor, which is minimally expressed in normal brain tissue. Upon binding to IL13Ra2, the fusion toxin is internalized, and the PE38 payload inhibits protein synthesis, leading to cell death. Precision NeuroMed has partnered with Brainlab to utilize precision drug delivery technologies for administration. The program received FDA Orphan Drug Designation in 2025 and is currently in the preclinical stage.
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