The pharmaceutical company now is planning to submit its application — some two years earlier than expected — to the European Medicines Agency (EMA) to request melflufen’s conditional approval.
The request was originally intended to be submitted only after the expected mid-2022 completion of OCEAN (NCT03151811), a Phase 3 trial currently investigating whether a combination therapy of melflufen and the corticosteroid dexamethasone might be superior to standard treatment with Pomalyst (pomalidomide) and dexamethasone.
However, after gathering feedback from European key opinion leaders and re-assessing the regulatory requirements, Oncopeptides decided to move forward and has now informed the EMA of its intentions to file a conditional marketing authorisation for melflufen. Once completed, data from OCEAN will be submitted to European regulatory authorities to potentially expand the therapy’s label, Oncopeptides said.
This type of conditional approval may be granted to medications that aim to treat or prevent debilitating or life-threatening diseases, provided that the current data support the developer’s claims that its benefits outweigh the potential risks.
“Key opinion leaders and clinics across Europe have gained extensive experience of melflufen from our clinical development program in multiple myeloma. We share a mutual interest to enable early access to this rapidly growing patient population in desperate need of new treatment options,” Klaas Bakker, chief medical officer of Oncopeptides, said in a press release.
Melflufen is a peptide-drug conjugate designed to rapidly deliver a cancer-killing agent — an alkylating agent — to myeloma cells that contain high levels of enzymes that break down peptides, called aminopeptidases. This mechanism allows the therapy to preferentially target malignant cells containing abnormally high levels of these enzymes, while sparing healthy cells.
This request for conditional approval will be supported by data from the pivotal HORIZON Phase 2 trial (NCT02963493), which is assessing the safety and effectiveness of melflufen in combination with dexamethasone. The ongoing trial, involving 157 relapsed or refractory (resistant) myeloma patients, is tracking participants for up to two years.
All patients enrolled in the study had received at least two prior therapies, including one immunomodulatory agent (IMiD) and one proteasome inhibitor, and failed to respond to Darzalex (daratumumab) and/or Pomalyst.
Top-line data from HORIZON showed that 29% of patients in the study’s overall population responded to treatment. Similar percentages of responders also were found among hard-to-treat patients, including those with triple-refractory disease (26%) and extramedullary disease or EMD (24%). Of note, EMD occurs when myeloma cells form tumors outside the bone marrow in the soft tissues or organs of the body, while patients with triple-refractory disease are those who fail to respond to treatment with at least one IMiD, one proteasome inhibitor, and one anti-CD38 monoclonal antibody.
Compared with those who failed to respond to treatment, responders in the overall population lived several more months without disease worsening (8.5 months vs. 4.4 months). Similar findings were shown in the subgroups of hard-to-treat patients.
Median survival was similar in the overall population (11.6 months) and in patients with triple-refractory disease (11.2 months). For those with EMD, it was 6.5 months.
The treatment’s safety profile also was found to be consistent with earlier reports, with no new safety concerns identified during the trial.
These findings also supported the submission of a new drug application to the U.S. Food and Drug Administration earlier this year requesting the accelerated approval of melflufen plus dexamethasone for hard-to-treat myeloma patients. The U.S. agency granted priority review to this application in August, and a final decision is expected in February 2021.
Oncopeptides, which specializes in developing targeted therapies for difficult-to-treat hematological diseases, also announced it has entered into a loan agreement with the European Investment Bank (EIB), which will enable the company to access a loan of €40 million (around $47 million) to support melflufen’s clinical development, and its transition to a global biopharmaceutical company.
“As the company is approaching a potential commercialization of its lead product melflufen, several new financing options become available,” Anders Martin-Löf, chief financial officer of Oncopeptides, said in a press release. “We are grateful for the support from the EIB and look forward to working together through the continued expansion of Oncopeptides.”
This loan will be divided into three tranches, each lasting up to five years, that will become available to Oncopeptides once the company achieves certain commercial milestones of melflufen in the U.S. and Europe.
If Oncopeptides decides to draw a portion of the loan, EIB will be entitled to receive a predetermined number of warrants that correspond to up to 0.7% of the total number of company shares.
“This is the kind of project that the Investment Plan for Europe was set up to support,” said Thomas Östros, vice president of EIB. “We are very happy to get behind yet another innovative Swedish company that has ground-breaking plans for the future.”
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