- Tell us about your career before joining Khondrion
I started my career as a physician in the Dutch Navy, 22 years ago, following the completion of my medical degree at Utrecht University. From there I spent the next two decades working in global and medical affairs roles of increasing responsibility at pharma and biotech companies in the Netherlands and Switzerland, including Solvay Pharma, Sanofi-aventis, MEDCON international, and Shire Pharmaceuticals. Prior to joining Khondrion, I was Head of Medical Affairs and Patient Advocacy for the EMEA region at Ultragenyx, an (ultra-) rare genetic disease company, where I built the medical organisation in Europe.
Setting sail: The start of my career as a physician, in the Dutch Navy, on board Hr. Ms. Van der Zaan
- What led you to specialize in rare diseases?
While at Shire, I had the opportunity to start working in the rare disease field in 2012, at that time focusing on mucopolysaccharidosis (MPS) disorders and, later, other inborn errors of metabolism. I felt a huge connection with this field of medicine, it was the right personal thing for me to do, something in which I could really make a difference and have a positive impact on the lives of patients and their families. My work in patient advocacy, in particular, was very rewarding. Since then, a lot of my work has involved patient communities, who have been instrumental in driving research of rare diseases and the development of new medicines.
- How did you cross paths with Khondrion and what was your motivation in joining the company?
When I was first introduced to Jan, Khondrion’s CEO, I was immediately attracted to his personal mission and passion, which fit with my own values and mission. We had a great connection from the start and I knew that I could help Jan and Khondrion to advance the sonlicromanol clinical programme. The team here knows the enormous patient need driving mitochondrial research. That’s what is driving our work.
- As Chief Medical Officer, what are your current priorities at Khondrion?
As CMO, I oversee and manage the daily operational aspects of the clinical programmes at Khondrion, together with our Clinical Trial Manager. My role also involves reviewing and interpreting clinical data, working closely with Jan and Herma Renkema, Khondrion’s CSO, from our clinical trials – currently most notably our Phase IIb programme evaluating sonlicromanol in adults with MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like episodes) spectrum disorders.
We are at a pivotal time in progressing these broad Phase IIb clinical studies, with results expected later this year. That brings a significant amount of work for the team, though it’s very rewarding work because we know the potential that this programme could bring to patients.
My role also requires me to work with different external bodies and stakeholders on Khondrion’s broader clinical development programmes, such as the Scientific Advisory Board, Data Safety Monitoring Board, and external consultants where relevant. Their expertise and skills are all vital in our work.
- What do you see as the biggest challenges in the mitochondrial disease field?
The heterogeneity and multigenetic aspect of most mitochondrial diseases makes it very challenging to easily identify clear patient populations for clinical research. To date, not much research has been done in this field, making it very difficult to define appropriate and/or validated clinical endpoints in trial protocols.
Additionally, and as seen in all rare diseases, the low levels of awareness and delays in or incorrect diagnoses by physicians pose a huge challenge for this field. If medical professionals aren’t familiar with these diseases, it takes more time to diagnose patients appropriately and get them the care they need.
- Drug development in the field of mitochondrial disease is experiencing an increased focus, why do you think this is?
Fortunately for patients and families, there is certainly growing interest and activity in the field of mitochondrial diseases as more research is being funded by companies like Khondrion and patient advocacy groups. Over the past decade, we have seen the role of advocacy by patient groups grow exponentially as they connect patients with each other, clinicians and the wider industry via awareness events such as World Mitochondrial Disease Awareness Week and by participating in mitochondrial disease conferences. We can’t underestimate the impact this has had on driving innovation forward.
- What is your vision for the future of mitochondrial disease research and the treatment of mitochondrial disease patients?
I truly hope that there will be more disease-specific treatment options available for mitochondrial diseases so that patients and families have hope and a choice to improve their lives.
Of course, most of all, I do hope that sonlicromanol will prove to be safe and effective, approved by regulators and made available in the near future to all patients with primary mitochondrial diseases who could benefit and who currently have no disease-specific treatments available.