The patient engagement study is carried out at University of Californiaand will include up to 45 patients who will share their experiences of pain and care needs related to oral mucositis. The results will help OncoZenges fine-tune the design of its Clinical Trial Application (CTA), which the company aims to submit to the European Medicines Agency EMA before the end of the year. This application is required to obtain approval to initiate clinical trials in the EU. The study may also serve as background material for a future Investigational New Drug (IND) submission in the United States.
The launch marks OncoZenge’s first US-based activity and further strengthens the foundation for its phase III program, which will be conducted in Europe. The collaboration is also part of OncoZenge’s long-term plan to build an international network of clinical and scientific partners.
Parallel studies in the EU and U.S.?
Unlike a clinical trial, this is a so-called patient engagement study – an exploratory study led by UCLA. The format focuses on collecting self-reported data from patients and clinicians rather than evaluating treatment efficacy. OncoZenge provides expert input and guidance on study design, with the goal of gaining a deeper understanding of patient perspectives and optimizing the setup for the upcoming phase III trial.
As the study is not directly linked to the company's regulatory development program, it falls outside the formal registration process and therefore does not require approval from the EMA or MPA. This allows OncoZenge to simultaneously advance its European program, where the phase III is expected to begin in 2026.
A very painful side effect of cancer treatment
Oral mucositis is caused by damage to the mucous membranes of the mouth during radiation or chemotherapy and often leads to severe ulcers, difficulty swallowing, and an increased risk of infection. In severe cases, patients are forced to pause their cancer treatment. Despite the seriousness of the problem, effective therapies are still lacking.
BupiZenge was developed to offer local, long-acting, non-opioid pain relief directly at the site of pain. In the upcoming phase III trial, the company will also measure opioid use and patient weight as indicators of quality of life and nutritional status – two key factors in the treatment outcome of oral mucositis.
Q&A with CEO Stian Kildal
To learn more about the significance of the UCLA study and how the results will inform the European phase III design, we spoke with OncoZenge CEO Stian Kildal.
What does the inclusion of the first patient in the UCLA study mean for OncoZenge and the BupiZenge project?
– First of all, we are honored and delighted that such a respected organization as UCLA Health sees the potential and the need for BupiZenge and actively wants to support our project with the goal of bringing it to market. We have been in discussions for quite some time, and they have expressed great interest in contributing, including through clinical activities. The patient study is the first concrete step, and the timing is excellent. GCP guidelines increasingly encourage drug developers to integrate patient perspectives before starting clinical trials. Since BupiZenge’s purpose is largely to improve quality of life for cancer patients, it is only natural that this kind of patient feedback should help shape how the study is conducted and support our regulatory submission.
What specific parameters will the study investigate?
– In surveys and focus groups, cancer patients undergoing treatment will describe their experiences with oral mucositis – how current treatment options work for them, which side effects are hardest to manage, and what improvements they hope to see in future therapies. This will help support our positioning and value proposition for BupiZenge going forward. Other questions are aimed at gathering insights into how our clinical trial should be conducted.
How will the study results be used in the CTA submission and phase III design, and will they be ready in time?
– We will include the responses in our CTA submission. The context here is that our trial will offer BupiZenge to patients undergoing intensive cancer therapy. For OncoZenge, it is essential that we have all the data we need as the foundation for a future marketing authorization application. But it is equally important that we set up patient visits and pain reporting at a frequency that patients can realistically manage. The responses from the patient study will help validate the assumptions in our study protocol and guide any adjustments needed to reduce, for example, the risk of patient drop-outs. The UCLA project is designed to deliver results in time for our CTA submission.
You are currently conducting a feasibility study with Link Medical, the CRO that will run your phase III trial. What kind of interest have you received from hospitals invited to participate in the trial?
– The response has been very positive. We have received strong interest and encouraging feedback on patient recruitment from several hospitals in Norway, Sweden, Denmark, and Germany. The CRO is now working through questions, conducting site visits, and gathering feedback on the study. It is particularly encouraging that hospitals and investigators in Denmark who participated in the phase II study will also take part in phase III. Their experience has been shared during joint meetings and has further motivated sites in other countries.
You have previously stated that you intend to submit your CTA for the phase III trial in Europe before the end of the year. How are you progressing toward that goal, and what still needs to come together before you can submit the application?
– Work on the CTA is intense, but we’re still on track to submit later this year. We’re well prepared, but there are many issues – both large and small – that must be verified and documented with clear supporting documentation before submission.. In addition to materials on study design, relevant historical data, and product details, several supplier decisions still need to be finalized – for example, handling of our comparator lidocaine, PK analysis services, labeling and distribution, and insurance. Moreover, our Pediatric Investigative Plan (PIP) must be submitted before the CTA. It is a lot of work, but we have a fantastic team and we are on track.
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