Research shows that Pepaxti is effective in high-risk myeloma
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| Land | Sverige |
|---|---|
| Lista | Small Cap Stockholm |
| Sektor | Hälsovård |
| Industri | Bioteknik |
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Stockholm, December 29, 2025 – Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a biotech company focused on difficult-to-treat cancers, today announces new research demonstrating that Pepaxti (melflufen) maintains its anti-myeloma activity in patients with high-risk genetic features, including deletion 17p (del(17p)) and TP53 mutations. These abnormalities are known to be linked to more aggressive disease being less responsive to treatment. The data has been published in an article in Experimental Hematology & Oncology.
The findings come from a collaborative study conducted by researchers in Finland, Germany and Sweden, using patient samples, disease models and clinical data. The research supports earlier observations from the phase 3 OCEAN study.
Key highlights from the study
- Consistent activity in high-risk patients:
In laboratory testing of bone marrow samples from 24 myeloma patients, Pepaxti showed strong activity across all genetic subgroups, including those with del(17p) and TP53 mutations. This was in clear contrast to standard alkylating agents. - Mechanism of action maintained despite loss of functional TP53:
Pepaxti triggered rapid cell death and DNA damage in both normal and TP53-deficient myeloma models, indicating a mode of action that does not rely on functional TP53. - Clinical relevance confirmed in OCEAN:
A post-hoc analysis of the OCEAN study showed that patients with del(17p) treated with Pepaxti and dexamethasone achieved longer progression-free survival compared to pomalidomide-based treatment.
“These results help explain why Pepaxti performs well even in some of the most challenging forms of myeloma,” says Caroline Heckman, Senior Author and Research Director, Institute for Molecular Medicine Finland-FIMM, HiLIFE-Helsinki Institute of Life Science, University of Helsinki, Finland. “Understanding how Pepaxti works in these high-risk patients gives physicians additional confidence when evaluating treatment options.”
“The study adds to a growing body of evidence supporting Pepaxti and the PDC platform´s differentiation versus conventional alkylators and reflects what we have seen in both clinical trials and real-world patients,” says Stefan Norin, Chief Medical Officer at Oncopeptides. “We remain committed to expanding scientific knowledge and supporting the myeloma community with high-quality research. In addition we use our expanding scientific knowledge to further develop our PDC pipeline.”
The full research article, Efficacy of Melflufen in Multiple Myeloma with Mutated or Deleted TP53, is available through this link.
More information, including questions and answers for investors, will be posted on oncopeptides.com.