Exceptional long-term responses to Pepaxti – new publication in Journal of Cancer Research and Clinical Oncology
Oncopeptides AB today announces the publication of three case reports in the Journal of Cancer Research and Clinical Oncology, demonstrating long-lasting clinical responses to melflufen (branded in Europe as Pepaxti) in patients with relapsed or refractory multiple myeloma (RRMM).
The peer-reviewed article, authored by European investigators involved in the pivotal OCEAN and HORIZON studies, describes three heavily pretreated patients who achieved unusually durable responses – including complete and stringent complete responses lasting more than four years – while receiving melflufen plus dexamethasone.
“These exceptional cases illustrate that melflufen can induce deep and durable remissions in selected patients, even in the context of advanced disease,” says Dr. Elena Zamagni, first author of the publication and Associate Professor at the University of Bologna, Italy. “Although such long-term outcomes are rare, they highlight the drug’s potential role as a meaningful option in the modern myeloma treatment landscape.”
“These data complement the clinical evidence base from the OCEAN and HORIZON studies and underscore Pepaxti’s relevance for patients who may not be eligible for, or have access to, novel immunotherapies,” says Stefan Norin, Chief Medical Officer at Oncopeptides. “While these are individual observations, they demonstrate that Pepaxti can deliver sustained benefit in real-world settings for certain patient subgroups.”
The article, “Exceptional long-term responses from OCEAN and HORIZON trials: melflufen-dexamethasone as an expansion of treatment options for relapsed/refractory multiple myeloma in the era of new immunotherapies”, is available online.
Below you will also find a Q&A for investors.
Q&A for Investors
What is this?
A peer-reviewed publication presenting three long-term case reports from the OCEAN and HORIZON trials of melflufen plus dexamethasone in relapsed/refractory multiple myeloma.
Why is this important?
The article documents exceptional responders with prolonged responses exceeding four years, supporting the continued clinical relevance of Pepaxti in select patient populations.
What are the key takeaways?
- Durability of response: Exceptional cases achieved complete or stringent complete responses lasting 4+ years.
- Patient profiles: All were heavily pretreated, consistent with the inclusion criteria for the HORIZON and OCEAN studies.
- Tolerability: Dose reductions were common but enabled long-term treatment continuation.
- Clinical context: The findings illustrate that meaningful and durable remissions remain achievable with Pepaxti even as immunotherapies dominate the field.
Does this change the evidence base or label?
No. These are anecdotal case reports that complement but do not replace prospective data. The publication does not change Pepaxti’s approved indication in the EU or UK.
How does this impact Oncopeptides?
The paper reinforces Pepaxti’s scientific credibility and visibility in peer-reviewed literature, supporting ongoing engagement with clinicians and highlighting its continued relevance for eligible RRMM patients.