Science is leading the way
I remember it as if it was yesterday. It was February 26, 2021, and we were expecting an approval of Pepaxto in the US, twenty years after the discovery of the molecule. For a small Swedish biotech company, it is a major accomplishment to take a product all the way from early discovery to commercialization in the US. When we finally got the approval letter it was close to midnight in Sweden, and the excitement was huge.
The commercialization of Pepaxto started in mid-March, shortly after the approval. Early on Pepaxto was included in the national treatment guidelines, which spurred patient access. Almost every day, we received feedback from doctors across the US that Pepaxto was making a difference for patients. This made me immensely proud. We were about to live our vision, Bringing hope through science.
Sales took off early on, and the entire organization was so excited and we were all set for a successful launch. However, during the summer we experienced some setbacks in the dialogue with the FDA, that ultimately led to a voluntary withdrawal of Pepaxto. This forced us to make tough decisions, close business operations, dismantle the organization, and refocus on R&D.
Looking in the mirror, 2021 has been extraordinary challenging for Oncopeptides and our shareholders. I have not experienced anything similar in my entire career. It has been like riding a never-ending rollercoaster; with ups and downs, and U-turns.
In April 2021 we submitted the application for marketing authorization of melflufen in the EU, to the European Medicines Agency, EMA.
COMPLEX STUDY RESULTS
In April 2021 we completed the phase 3 OCEAN study, a post-approval commitment under the accelerated approval program, in the US. We analyzed the OCEAN data in a small team, spending hours, days, and weeks in a bunker, trying to get a good understanding of the data. When we announced the results in May, we had strong confidence in the data, even though we realized that the data was complex, and would take time to fully interpret.
The OCEAN study met its primary endpoint of superior progression free survival and demonstrated a better overall response rate in patients who were refractory to lenalidomide with 2-4 prior lines of therapy. However, the overall survival (OS) hazard ratio in the intent to treat (ITT) population was 1.1 in favor of pomalidomide. We emphasized that the negative trend in ITT OS result, by large, was driven by other factors than treatment with melflufen, however the FDA requested a partial clinical hold of the development program and issued a safety alert due to a potential detriment. In August, the FDA announced that they considered holding a public oncology drugs advisory committee (ODAC) meeting to discuss patient safety and the continued marketing authorization of Pepaxto. After an intensified dialogue with the FDA during the fall, it became evident that the FDA did not accept OCEAN as a confirmatory study. That left us with no other option than to withdraw the product from the market.
Consequently, we decided to refocus Oncopeptides on R&D, continue to support the EMA-submission for marketing authorization of melflufen in Europe, discontinue parts of the clinical development program of melflufen and focus on developing the next generation of drug candidates from the PDC-platform. Shortly after the withdrawal we closed the business operations in the US and Europe and scaled down the Sweden based organization. We made a decision to reduce the number of employees globally from 330 FTEs to approximately 50. It has been a very challenging task, and I am so impressed by all employees who have contributed relentlessly throughout this process, even though many of them knew that they potentially would lose their job. These measures led to a cash position of SEK 362 M by year-end 2021. Assuming that the operations' re-structuring continues according to plan, the Group will have the necessary liquidity for operations to continue for at least the next 12 months.
SCIENCE WILL PREVAIL
Even though we made a voluntary decision to withdraw Pepaxto from the US market, we have never lost confidence in our data. For us, this is all about science. That is why we have continued to review and analyze data from OCEAN and other relevant trials. Our findings have convinced us to reconsider our previous decision and rescind our October 22 letter requesting withdrawal of Pepaxto in the US.
Oncopeptides has discontinued the marketing of Pepaxto in the US and does not intend to market Pepaxto in the US at this time. The company has initiated a dialogue with the FDA to review the new data. It is currently premature to talk about if, when, and how patients in the US could get access to Pepaxto again. We believe that melflufen may become an important treatment option for patients with relapsed refractory multiple myeloma (RRMM), and that the comprehensive data further support the pending European Medicines Agency’s (EMA) review of melflufen.
OCEAN DATA PUBLISHED
At the International Myeloma Working Group meeting in Vienna on September 11, Dr Fredrik Schjesvold from Oslo Myeloma Center presented the full data set from OCEAN. Data from prespecified subgroups showed that the main benefit of melflufen on progression free survival was driven by patients without a prior autologous stem cell transplant (ASCT) and that the potential detriment of Pepaxto in the ITT population was isolated to patients with a prior ASCT.
In January 2022, we reached an important milestone when the results from the OCEAN study were published in Lancet Haematology. Getting our data accepted for publication in this reputable journal is an acknowledgement of the value of our data. The authors of the publication emphasized that the OS results seem to be driven by unusual large heterogeneity in predefined subgroups. Age, gender, and a previous autologous stem cell transplant (ASCT), all showed significantly different outcomes in the subgroups.
MOVING INTO 2022
Even though 2021 took a direction that neither of us could anticipate, I am confident that 2022 will be an exciting and challenging year for Oncopeptides. We do not know what the rescission of the withdrawal of Pepaxto in the US will lead to, however, we believe that by leveraging science and data, while continuing to make innovative medicines available to patients, we will thrive as a company.
We continue our interactions with EMA and look forward to the CHMP opinion around mid-year. Currently, almost 70 patients are treated with
melflufen through the Early Access Program in the EU, which is showing the unmet medical need for patients with relapsed refractory multiple myeloma. In 2022 we also intend to advance our portfolio of drug candidates from our proprietary PDC-platform.
2021 was an extraordinarily challenging year, but it is with growing enthusiasm and confidence we now move into 2022. I would like to thank all
employees for your dedicated contributions, and all our shareholders for your continued belief in Oncopeptides. What is good for patients will be good for Oncopeptides.
Stockholm, April 13, 2022
Jakob Lindberg, CEO