Oncopeptides announced that initial data from three patients in its ongoing INSULA study showed that its peptide drug conjugate platform crossed the human blood-brain barrier and reached glioblastoma tissue. The study examines a central challenge in brain cancer treatment: whether a systemically administered drug can reach the tumor.
Following preoperative treatment with melflufen, analysis of surgically removed tumor samples consistently detected the active compound in all 3 evaluated patients. Drug concentrations were in line with preclinical findings, providing initial human data to inform further development of the company’s brain cancer program.
Sofia Heigis, CEO of Oncopeptides, said:
“Reaching our primary objective to confirm that our PDC technology successfully crosses the human blood-brain barrier is a major breakthrough for Oncopeptides.”
Dr. Heigis highlighted the longstanding challenge posed by the blood-brain barrier in glioblastoma treatment. She said that detecting meaningful drug penetration in surgically removed brain tumors after a single dose supported the platform’s capabilities and provided a clinical basis for advancing the company’s brain cancer program.
Key Findings From the First Three Patients
- Blood-brain barrier penetration: Analysis of surgically removed glioblastoma tissue consistently detected the active compound in all three evaluated patients. This met the primary endpoint for this part of the INSULA study and, according to Oncopeptides, supported the platform’s ability to cross the human blood-brain barrier after a single preoperative dose.
- Drug concentrations in tumor tissue: Pharmacokinetic measurements showed that concentrations within the patients’ tumor samples were consistent with preclinical results, supporting the anticipated drug-delivery behavior of the peptide drug conjugate platform.
- Initial safety and tolerability: Treatment was reported to be well tolerated across evaluated patients, with no major drug-related toxicity observed at the time of the announcement.
- Continued treatment in the first patient: The first treated patient entered the open-label extension and received five monthly doses. No new tumor-related neurological symptoms were reported. Postoperative paresis attributed to surgery was observed and was improving during ongoing treatment.
- Surgical complications: No unexpected surgical complications were reported.
These initial findings concern drug delivery and tolerability. The study evaluates uptake, tissue distribution, and cytotoxic mechanisms rather than progression-free or overall survival. Recruitment continues toward approximately 10 patients.
Assessing Drug Delivery Before Surgery
The open-label Window-of-Opportunity study at Oslo University Hospital in Norway administers melflufen before surgery for recurrent glioblastoma. Researchers examine removed tissue to assess drug uptake, distribution, and cytotoxic activity.
The active compound was detected consistently in all three patients’ tumor samples, meeting the primary endpoint for this study component.
Glioblastoma and Unmet Treatment Needs
Glioblastoma is the most common and aggressive form of primary brain cancer, with a median overall survival of 12-15 months. It highlights substantial unmet medical needs and limited effective treatment options.
One challenge is delivering systemic therapies across the blood-brain barrier into tumor tissue. Oncopeptides’ ongoing INSULA study addresses this question in patients undergoing surgery for recurrent glioblastoma, assessing drug uptake and biological activity in removed tumor tissue rather than long-term survival outcomes.
The Blood-Brain Barrier in Glioblastoma Treatment
The blood-brain barrier is a major obstacle to delivering systemic cancer treatments to brain tumors. According to Oncopeptides’ release, more than 90% of conventional systemic cancer drugs fail to reach the brain because they cannot cross this barrier.
The INSULA study examines whether the company’s peptide drug conjugate platform can deliver an active compound into glioblastoma tissue. Patients receive melflufen before surgery, allowing researchers to analyze drug uptake directly in the removed tumor.
In the first 3 evaluated patients, tissue analysis consistently detected the active compound, with concentrations matching preclinical findings. Oncopeptides described these results as evidence supporting the platform’s ability to cross the human blood-brain barrier.
The study focuses on drug delivery, tissue distribution, and cytotoxic mechanisms. These initial findings do not establish improvements in progression-free or overall survival; further enrollment will help characterize the platform’s pharmacokinetic, biological, and safety profiles.
Prof. Petter Brandal, Oslo University Hospital, and primary investigator in the WoO study, said:
“Crossing the blood-brain barrier in patients diagnosed with Glioblastoma is an important step for any systemic brain cancer therapy.”
About Oncopeptides
Oncopeptides is a Swedish biotechnology company focused on researching, developing, and commercializing therapies for difficult-to-treat cancers. Its proprietary Peptide Drug Conjugate (PDC) platform is designed to deliver cytotoxic agents rapidly and selectively into cancer cells. The company is also developing compounds based on its PDC and SPiKE technology platforms.
Oncopeptides’ flagship drug is being commercialized in Europe, with partnership agreements covering South Korea, the Middle East, Africa, and other markets. Founded in 2000, the company has approximately 70 employees and operations in Sweden, Germany, Austria, Spain, and Italy. Oncopeptides is listed on Nasdaq Stockholm under the ticker ONCO.
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