Oncopeptides today announces the publication of a new real-world study in the peer-reviewed European Journal of Haematology. The study, conducted at the IRCCS Azienda Ospedaliero-Universitaria di Bologna, Italy, confirms the efficacy and safety of Pepaxti (melflufen) plus dexamethasone. The heavily pretreated patients, including those refractory to novel immunotherapies, benefitted from Pepaxti’s strong efficacy, the overall response rate (ORR) was 41% with a median progression-free survival (mPFS) of 9.0 months.
The retrospective analysis evaluated 17 patients with relapsed/refractory multiple myeloma (RRMM) treated in a real-world setting outside of clinical trials. Despite a highly refractory population—where 88% were triple-class refractory and 41% were penta-drug refractory—Pepaxti demonstrated a clinically meaningful overall response rate (ORR) of 41%.
Key Study Highlights:
- Strong efficacy in late-line settings: The ORR was 41%, with a median progression-free survival (mPFS) of 9.0 months in responding patients.
- Preserved access to future therapies: Notably, 100% of patients who received subsequent novel immunotherapies (such as CAR-T or bispecific antibodies) after Pepaxti achieved a partial response or better, with the majority reaching a very good partial response (VGPR) or better.
- Manageable safety in frail populations: The safety profile was consistent with previous clinical trials, despite a high portion (41%) of the cohort being >75 years old and many presented with reduced renal function.
- Strategic positioning: The data suggests Pepaxti serves as an effective “bridging” or interval therapy, maintaining disease control without compromising the success of subsequent T-cell redirecting treatments.
“Collectively, our data confirmed the efficacy previously reported with melflufen–dexamethasone and its manageable safety profile, also in elderly patients that likely are more fragile and more heavily pretreated than those included in the trials,” says Dr. Elena Zamagni, Associate Professor at the University of Bologna, Italy, and lead author of the study. “Melflufen–dexamethasone may represent a treatment option, especially for patients refractory to novel immunotherapies or those who are not ideal candidates to receive such treatments while still preserving access to subsequent T-cell redirecting therapies”.
“This independent real-world evidence from a leading European center is highly encouraging as we continue the commercial roll-out of Pepaxti in Italy and across Europe,” says Stefan Norin, Chief Medical Officer at Oncopeptides. “The 100% response rate observed in subsequent immunotherapies is particularly significant for clinicians, as it addresses the critical question of treatment sequencing and confirms that Pepaxti can be used effectively to bridge patients to the next generation of care”.
The full article, titled “Positioning of Melflufen in Heavily Pretreated RRMM Patients: Real-World Evidence in a Rapidly Evolving Therapeutic Landscape,” is available online through this link.
Q&A for Investors
More about how the study was conducted
The study analyzed 17 RRMM patients treated between December 2021 and July 2025 at the Seràgnoli Institute of Hematology in Bologna. The patient population was characterized by high-risk features, including high-risk cytogenetics (41%) and advanced age (median 71 years). Patients had received a median of four prior lines of therapy.
How does this study impact the commercial potential of Pepaxti in Italy?
Italy is a key market for Oncopeptides, following the formal reimbursement approval in early 2025. Real-world data from a prestigious Italian institution like Bologna builds significant clinician confidence and supports the drug’s uptake in routine practice.
Why is the “sequencing” data important?
A major question has been whether alkylating agents like Pepaxti might hinder the effectiveness of later-line T-cell therapies. This study provides indicates that Pepaxti does not preclude high-quality responses in subsequent lines, positioning it as a versatile tool in the modern “treatment algorithm” rather than a treatment of last resort.
Does this data support use in a broader patient population?
Yes. By including patients with poor renal function ($< 45$ mL/min) and those over 75, the study demonstrates Pepaxti’s utility in a “less selected” and more realistic patient population than typically seen in phase III trials.