1. Tell us all about your career before joining Khondrion
I studied pharmacology and molecular biology at the University of Strasbourg in France, where I obtained my PhD degree. At that time, my work was mainly focused on the discovery and preclinical development of therapies for cancer. During my studies, I became intrigued by the field of system biology, which was just emerging at that time, and I jumped at the opportunity to join the center for system biology and bioenergetics at the RadboudUMC in Nijmegen as a postdoctoral fellow. I started working on mitochondrial disease and that is where I first met Khondrion’s Founder and CEO Pr. Jan
2. What made you join the company?
During my postdoctoral fellowship I was trying to develop new cellular models to study mitochondrial disease and evaluate potential therapies. It was similar to Khondrion’s early work and, together with my background in translational drug development, I thought that I could be of help to the team and play a role in the company’s vision.During my fellowship, I also had the opportunity to meet with mitochondrial disease patients, which was a significant source of motivation for me. The opportunity to participate in Khondrion’s mission to bring potentially transformative therapies to these patients was what ultimately compelled me to join the company. It was a “no brainer”.
3. What excites you about Khondrion?
Both the past and the future. Looking back, as a former lab rat, I am amazed by what the Khondrion team has achieved in such a short period of time, bringing an initial idea through to clinical testing. Looking to the future, there are exciting times ahead with a new Phase IIb clinical trial for our lead asset currently underway, and a number of new early assets in the pipeline. I especially enjoy the continuous evolution and maturing of the company through its mission and development.
4. What does a typical day at Khondrion look like for you?
I don’t think I have any one typical day; my role is pretty diverse and changes with the development of our products and the overall company. I try to participate and facilitate all aspects of development, from research to business. One constant is that I am learning new things every day.
5.What are you hoping to achieve at Khondrion?
One clear goal is to bring our first drug candidate, sonlicromanol, to patients suffering from mitochondrial disease as quickly as possible. Another goal is to keep bridging the gap between academic research with clinical application and continue to grow the company so that we are in a good position to translate even more innovative approaches and ideas into potential therapies for patients with mitochondrial disease.
6.Tell us about the trial design for the ongoing phase IIb dosing study, KHENERGYZE.
The ongoing Phase IIb is a double-blind, randomized, placebo-controlled study examining the sonlicromanol dose-effect on cognitive function in 27 adult patients with mitochondrial DNA mutation m.3243A>G. This means patients participating will undergo three consecutive periods of 28-days of placebo, sonlicromanol 100mg or sonlicromanol 50mg. With this particular design, each patient is his own control, reducing variability and thus increasing the chances of observing true effects between treatment periods. The study will take place in three different mitochondrial references centers in Europe.
7. What do you believe makes Khondrion special in the field of mitochondrial medicine, and how do you ensure you are constantly evolving?
A key differentiator is our strong connection with patient organizations, academic and clinical centers. Khondrion was founded by Prof. Jan Smeitink, a key opinion leader in the field of mitochondrial disease, to accelerate the development of therapies and directly answer the high unmet needs of his patients, but also patients around the world. As a result, Khondrion is a truly complementary player within a wider network of academic research, diagnostic and clinical care in the field of mitochondrial disease. In my view, this is a rather unique situation and the optimal way to serve patients.
8. What do you see as the biggest challenges in the mitochondrial disease field?
Mitochondria, being under the dual control of nuclear and mitochondrial DNA, are certainly the most complex organelles within our cells, orchestrating and involved in a multitude of vital functions. Mitochondria functions also vary from organ to organ, which brings another level of complexity when developing therapies.The genetic and clinical heterogeneity of the disease also makes the clinical development of new therapies rather challenging. The regulatory pathway to drug approval for mitochondrial disease is still in its infancy and we are very much paving the way.
9. How important are patient organizations in developing new medicines for mitochondrial disease and how does Khondrion work with these organizations on drug development?
Medicines are developed for patients and together with patients. Patient organizations are central to the development of new medicines for any disease.We, as researchers or doctors, put a lot of work and thinking into the development. Patients, not only contribute themselves by participating in clinical trials, but are vital in increasing awareness for mitochondrial disease. They are a critical source of knowledge to us, as they are the ones who really know what mitochondrial disease is and what the burdens are. They are also extremely important in raising awareness with politicians and regulators.We all share the same goal and play our own part, but patients are without a doubt at the center of medicine development. For those reasons Khondrion has been directly supported by different patient organizations to accelerate the development of sonlicromanol.We are grateful for these collaborations and are convinced patients will ultimately benefit. Development of mitochondrial medicine is in its infancy and I believe that working together with all the players (drug developers, patient organizations, regulatory bodies etc.) is the most efficient way to solving the challenges to reach our common goal.
10. How hopeful are you about the future of mitochondrial disease?
One can only be positive looking at the recent efforts in translating research findings into potential therapies taking place around the world. Although there is still much to learn in mitochondrial biology and the patho-mechanisms underlying of mitochondrial disease, it is necessary to start bridging the gap from bench to bedside – this will ultimately accelerate the R&D wheel.