1. Tell us about your career before joining Khondrion.

I qualified as a Medical Doctor from the University of Utrecht and worked clinically in the Netherlands for a number of years. Being on the frontline helping patients and working with many amazing colleagues was good but I became interested in how I could use my skills differently, to help patients on a much larger scale. That’s how I moved into the pharmaceutical industry, working for companies including Novartis, Organon and Roche, in many therapeutic areas especially the central nervous system (CNS) field on treatments for conditions such as dementia, Parkinson’s disease and epilepsy but also oncology, urology, anesthesia and sleep medicine. It is very rewarding to be part of a team that’s responsible for developing and launching a medicine, then seeing the positive impact it can have for millions of patients around the world. While working I have also been lucky enough to continue my studies and hold a specialist certification in Pharmaceutical Medicine from the Faculty of Pharmaceutical Medicine, UK and an MBA from the University of Amsterdam.

2. Why did you decide to make the move from Big Pharma to a smaller company?

Having worked for major global pharmaceutical companies for many years, I gained a great deal of experience in strategic and operational global drug development, medical affairs, pharmacovigilance and regulatory affairs. I was excited to join a more agile and nimble biotech company like Khondrion, where projects have the potential to move much faster and where I have a broader scope of responsibility. I enjoy the entrepreneurial spirit that Jan and the team are fostering here and in my role, there is potential to make a real impact on the business and its strategy going forward. Working with such a collaborative team here at Khondrion has also brought me closer to other interesting elements of business such as fundraising and looking for potential partners who can help us to deliver the medicines we’re developing to patients.

3. What excites you about Khondrion?

The field of mitochondrial disease is still relatively unexplored and there are very little, if any, established regulatory paths to bring important new treatments to patients suffering from these rare diseases. I enjoy the pioneering work for the Khondrion team to collaborate closely with the regulatory authorities and patient groups and define the best routes to patients. The Khondrion team, with its combined experience and expertise, has real potential to make a significant impact and meaningfully change the lives of patients awaiting treatments for mitochondrial diseases. These conditions are devastating for patients and, for most, there are no treatment options available. That’s a very important driver for all of us working at Khondrion. In addition, the concept of a virtual development team, where external experts rather than in-house departments collaborate to create a medicine, adds dynamism to everything we do.

4. What does a typical day at Khondrion look like for you?

Well certainly no one day at Khondrion is the same! But I like that. As Chief Medical Officer, I have a very broad scope and each day is diverse with different balls to juggle and tasks to complete. I am constantly switching from a very broad strategic overview of the business to the intricacies of our science and the minutiae of implementation, intermixing medical and scientific discussions with other business aspects to find the best and quickest way forward.

5. What are you hoping to achieve at Khondrion?

My hope is that we can bring real change to patients with mitochondrial disease who are desperately in need. Right now, our focus is on bringing our lead drug candidate, sonlicromanol, through development and to patients as quickly as possible, and for as large a patient population as could benefit. Developments like this have the potential to significantly change the field of mitochondrial disease, which has been devoid of new approved treatments for too long.

6. What do you see as the biggest challenges in the mitochondrial disease space?

The huge heterogeneity between patients who, despite the same genetic make-up, vastly differ in symptoms and severity. This presents huge challenges in identifying the right target patient population, the right assessments to demonstrate the effect of your medicine and achieving the numbers of patients needed to make clinical trials interpretable. This, of course, is further complicated by the rarity of these genetic defects. Furthermore, the knowledge about pathophysiology, i.e. how a genetic defect precisely leads to a particular symptom, remains incomplete which adds to the challenge of predicting drug efficacy.

7. How hopeful are you about the future of mitochondrial disease?

Hope and expectation are what drives people, businesses and governments to progress. I expect that persistent and thorough research – sometimes aided by a bit of serendipity! – ultimately will pay off and lead to new medicines for patients. With novel technologies accelerating and our insights about disease processes growing every day, I am more hopeful than ever this will happen sooner rather than later.