Strong real-world efficacy and safety data with Pepaxti published in the European Journal of Haematology

Stockholm – February 25, 2025 – Oncopeptides AB (publ), a biotech company focused on difficult-to-treat cancers, 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%).
For more information, visit oncopeptides.com where you will be able to find question and answers for investors.

For more information, please contact:
David Augustsson, Director of IR and Communications, Oncopeptides AB (publ)
E-mail: david.augustsson@oncopeptides.com
Cell phone: +46 76 229 38 68

About Oncopeptides
Oncopeptides is a Swedish biotech company focusing on research, development and commercialization of targeted therapies for difficult-to-treat cancers. 

The company uses its proprietary Peptide Drug Candidate platform (PDC) to develop compounds that rapidly and selectively deliver cytotoxic agents into cancer cells. Its flagship drug is currently being commercialized in Europe with partnership agreements for South Korea, the Middle East and Africa and elsewhere.

Oncopeptides is also developing several new compounds based on its two proprietary technology platforms PDC and SPiKE.

The company was founded in 2000, has about 80 employees with operations in Sweden, Germany, Austria, Spain and Italy. Oncopeptides is listed on Nasdaq Stockholm with the ticker ONCO.

For more information see: www.oncopeptides.com

About Pepaxti
Pepaxti® (melphalan flufenamide, also called melflufen) has been granted Marketing Authorization, in the European Union, the EEA-countries Iceland, Lichtenstein and Norway, as well as in the UK. Pepaxti is indicated in combination with dexamethasone for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapies, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapy. For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation.