Oncopeptides AB (publ), a biotech company specializing in difficult-to-treat cancers, today announces data from the first patients in its ongoing clinical Window-of-Opportunity (WoO) study, OP-701 (INSULA), evaluating its proprietary Peptide Drug Conjugate (PDC) platform in glioblastoma. Data from the first three treated patients demonstrate successful penetration of the human blood-brain barrier (BBB) with intra-tumoral drug concentrations in line with preclinical models, alongside good tolerability where the first patient has received five monthly doses to date without any major drug-related toxicity.

The blood-brain barrier represents one of the greatest hurdles in neuro-oncology, causing more than 90 percent of conventional systemic cancer drugs to fail because they cannot reach the brain. By confirming meaningful, BBB penetration and delivery of a cytotoxic payload into brain tumor cells in the first patients, the findings provide a strong indication of a clinical proof-of-concept for the PDC platform’s mechanism in solid brain tumors.

“Reaching our primary objective to confirm that our PDC technology successfully crosses the human blood-brain barrier is a major breakthrough for Oncopeptides,” says Sofia Heigis, CEO of Oncopeptides. “For decades, the blood-brain barrier has locked out effective treatments for glioblastoma patients. Demonstrating meaningful drug penetration in resected brain tumors in patients after a single dose validates the fundamental strength and versatility of our platform, transforming our brain cancer program from an ambitious basic scientific premise into a true, clinically justified, opportunity.”