Strong real-world efficacy and safety data with Pepaxti published in the European Journal of Haematology
Oncopeptides AB today announces that a new real-world study on melflufen (melphalan flufenamide, branded in Europe as Pepaxti) plus dexamethasone in patients with relapsed, refractory multiple myeloma (RRMM) has been published in the peer-reviewed journal European Journal of Haematology.
The study, authored by researchers at the Dana-Farber Cancer Institute in Boston, Massachusetts, USA found that melflufen demonstrated clinically meaningful efficacy, with an overall response rate (ORR) of 55%, including complete responses in 27% of patients. The safety profile was consistent with previous clinical trials, with manageable hematologic toxicities. The findings indicate that melflufen is an effective treatment option for patients with heavily pretreated RRMM in the real-world setting.
The article, titled “Outcomes of Melflufen Treatment in Patients with Relapsed/Refractory Multiple Myeloma,” evaluates real-world data from patients treated at the Dana-Farber Cancer Institute. The study provides additional clinical insights into the efficacy and safety of melflufen outside of the controlled clinical trial setting, reinforcing its potential role in the treatment of RRMM.
“Our real-world experience has strengthened our understanding of melflufen’s important role in treating relapsed and refractory multiple myeloma, as reflected by our data,” says Paul G. Richardson, MD, Clinical Program Leader and Director of Clinical Research at Dana-Farber Cancer Institute and senior author of the article. “The results indicate that melflufen’s efficacy and safety profile observed in clinical trials have favorably translated into real-world practice. In my view, this offers hope to our patients who may have limited remaining treatment options, and in particular need therapeutic strategies that circumvent immune exhaustion with a novel mechanism of action, as well as having the advantage of outpatient, “off the shelf” administration and low rates of infection.”
The full article can be accessed here.
About the Study
The retrospective study analyzed 12 patients with RRMM treated with melflufen plus dexamethasone at the Dana-Farber Cancer Institute. The cohort had a median of 5.5 prior lines of therapy, with a significant proportion exhibiting high-risk disease characteristics. Key findings include:
- 55% ORR, with 27% achieving complete response (CR)
- • 21.3-week median duration of response
- Manageable safety profile, with primarily hematologic adverse events
- No new safety signals compared to clinical trial data
Reasons for treatment discontinuation included disease progression (42%), drug withdrawal from the US market (33%), adverse events (17%), and one unrelated sudden death (8%).
Q&A for Investors
What is this?
Medical data and research regarding our science is from time to time published in established scientific journals such as European Journal of Haematology. These publications are aimed at healthcare professionals to provide critical insights into treatment efficacy and safety.
What does this data mean for physicians?
This study confirms that melflufen maintains strong efficacy and safety in real-world clinical practice, particularly for heavily pretreated patients with RRMM. The findings support its use as a treatment option in routine oncology practice.
Why is this result important?
Real-world evidence complements clinical trial data by providing insights into drug performance in broader, more diverse patient populations where patients usually are of higher age compared to clinical studies. This study demonstrates that melflufen remains effective in real-world settings, reinforcing its value in RRMM management.
How does this data impact Oncopeptides?
These findings validate melflufen’s clinical utility and may support its uptake among healthcare professionals treating RRMM. Real-world data is crucial for regulatory and market access discussions as well as for further clinical development.
Do these publications affect product development?
The data from research and clinical trials, when published, can provide insights into the development of new drugs or new indications for existing ones. They can also guide the direction of future research projects.
How do healthcare professionals use these publications?
Healthcare professionals rely on independent medical publications to stay updated on the latest research, innovative techniques, and best practices. This knowledge helps them make informed decisions about patient care and treatment choices.
How was the study funded?
This study was funded by Dana Farber Cancer Institute, in part supported by the RJ Corman Multiple Myeloma Research Fund and an unrestricted grant from Oncopeptides.
Can the general public access these publications?
It depends on the publication. While many medical journals require subscriptions, there are open-access journals and resources where research is freely available. The article in question is free to access.
Why is the peer-review process in medical publications crucial?
Peer review ensures that the research presented in the publication is of high quality, scientifically sound, and free from biases. Experts in the field evaluate the research, which helps maintain the credibility and trustworthiness of the findings.
How do medical publications benefit patients?
By driving advancements in medical science, these publications play a role in the development of newer, safer, and more effective treatments. When healthcare companies share their findings, it speeds up the dissemination of crucial information, ultimately leading to better patient outcomes.
Where can I find more information?
The full article is available here.