Sweden-based Oncopeptides AB (Nasdaq Stockholm: ONCO) reported detection of active compound in resected brain tumor tissue from all three evaluated patients receiving the peptide drug conjugate melflufen (melphalan flufenamide; Pepaxti, OP-701) in the Phase I INSULA window-of-opportunity study in recurrent glioblastoma. The company said the findings met the primary objective of this part of the study and indicated that its peptide drug conjugate technology could deliver active compound across the human blood-brain barrier.
The open-label study at Oslo University Hospital administers melflufen before planned tumor resection to assess drug uptake and activity in tumor tissue. Oncopeptides said intratumoral drug concentrations in the first three evaluated patients were consistent with its preclinical models. The first treated patient has received five monthly doses through the study's open-label extension without major drug-related toxicity. Postoperative paresis attributed to surgery was reported to be improving. Recruitment continues toward approximately 10 patients to further characterize pharmacokinetics and pharmacodynamics.
Melflufen is approved in the EU under the brand name Pepaxti for certain patients with relapsed or refractory multiple myeloma. Oncopeptides is evaluating the drug in INSULA as part of its efforts to extend its peptide drug conjugate platform into brain tumors. The study enrolled its first patient in June 2026. Existing treatment options for recurrent glioblastoma include the anti-VEGF antibody bevacizumab (Avastin), which received accelerated US FDA approval for the indication in 2009.
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