Jan Smeitink: Serving the Mitochondrial Medicine Mission

Embarking on the journey of biopharmaceutical innovation demands more than just expertise—it requires an undying commitment to transforming the landscape of healthcare through perseverance and strategic foresight. Leading this dynamic field is Jan Smeitink, a prominent figure renowned for his contributions to mitochondrial medicine.

With an impressive career spanning over two decades, Smeitink’s dedication to improving the lives of individuals affected by inherited mitochondrial diseases is nothing short of exemplary. As the Founder and CEO of Khondrion, a leading clinical-stage biopharmaceutical enterprise, he epitomizes innovative leadership in the pursuit of pioneering therapies.

From his role as a distinguished Professor in Mitochondrial Medicine at Radboud University, The Netherlands, to heading the development of Khondrion, Jan Smeitink’s journey embodies a profound commitment to advancing the frontiers of medical science.

With a distinguished portfolio boasting over 400 peer-reviewed scientific publications and the supervision of numerous doctoral candidates, his scholarly contributions have not only expanded our understanding of mitochondria but also paved the way for transformative therapeutic innovations.

Honored with the prestigious ‘Knight in the Order of the Dutch Lion’ for his invaluable services to mitochondrial medicine, Smeitink’s indomitable spirit continues to inspire and catalyze progress within the field. His involvement in the development of groundbreaking compounds, including the revolutionary sonlicromanol, underscores his dedication to pioneering novel treatments for a myriad of mitochondrial diseases.

In our exclusive interview, we explore the transformative journey of Jan Smeitink—a thought leader whose strategic acumen and passion continue to shape the future of mitochondrial medicine.

Can you please introduce yourself and briefly describe your journey in the biopharma industry?

I am the founding CEO of Khondrion, a biopharmaceutical company focusing on the development of new treatments for patients suffering from primary mitochondrial disease. I have over 25 years of experience in mitochondrial medicine serving as a professor at the Radboud Centre for Mitochondrial Medicine Nijmegen being involved in patient care, diagnostics, counseling and applied and fundamental research. In 2012, I founded Khondrion realizing the unmet medical need for treatment development for patients with primary mitochondrial disease both privately and from a clinician’s perspective. In only 12 years, we have brought this business from start up to preparations for a Phase 3 registrational trial for our lead compound sonlicromanol.

Could you share some pivotal moments or experiences that have shaped your career trajectory in the industry?

The transition from full professor in mitochondrial medicine to founder and CEO of what is now an advanced clinical stage company was a huge step.

The ongoing support of the excellent team at Khondrion, our great network of experts in the industry as well as patient advocacy groups were crucial in getting where we are now as a company.

Could you provide an overview of your organization and its mission within the biopharma landscape?

Khondrion is a clinical stage biopharmaceutical company. Our mission is to bring a much-needed therapy to patients suffering from severe and debilitating primary mitochondrial diseases, often leading to death at young age with no treatments available for these multi-system diseases today. Khondrion will leave no stone unturned to reach this goal for patients and, we will do our utmost to build a sustainable R&D-driven mitochondrial disease company.

What sets your company apart from others in the industry, in terms of both innovation and impact?

Khondrion has a relatively small team of in-house experts with long-standing experience in multi-system mitochondrial diseases. Our lead compound sonlicromanol targets the key underlying disease mechanisms of a wide range of primary mitochondrial diseases, based on its unique triple mode of action. The development program is underpinned by a sound and reliable scientific data package and Khondrion has the unique advantage of being able to incorporate valuable insights of 52-78 weeks treatment data into the design of the upcoming Phase 3 study. Furthermore, we have built strong relationships with patient advocacy groups and KOLs in mitochondrial medicine across the globe, who support our mission and believe in the value of sonlicromanol as a best-in-class treatment for primary mitochondrial diseases.

How do you define your leadership style, and how has it contributed to your professional accomplishments?

I would consider my leadership style as strategic. This has contributed to making the most out of existing resources and the highly motivated Khondrion team members.

Can you highlight some of the key initiatives or strategies you’ve implemented as a leader to drive innovation and growth within your organization?

One of the key strategies was to use a phenotypic screening approach for the development of our lead compound, uniquely targeting the key underlying disease mechanisms for a broad range of mitochondrial diseases. Furthermore, we have decided to initially focus on m.3243A>G Spectrum Disorder patients (classical MELAS, MIDD and multi-system phenotypes), one of the most frequently observed groups of PMDs which is characterized by one specific genetic mutation. In doing so, we can make a more focused patient selection, improving the chance of success or our trials.

What are some of the most significant challenges you’ve faced in your role, and how have you addressed or overcome them?

By far the most significant challenge I am facing is the long duration and cost of operations it takes to bring our initial idea to regulatory authorization and market entry. I have, however, always maintained trust in the strategy that we have set and realize that in principle solutions exist for most if not all hurdles.

I always keep and signal a positive attitude towards patients and other stakeholders, and I am not afraid of asking obvious questions, to overcome the challenges we face.

What advice would you offer to aspiring leaders looking to make a difference in the biopharma sector?

Surround yourself with people smarter and more experienced than yourself and don’t be afraid to make mistakes. Realize from the start onwards that perseverance is key and the road to success is long. Keep focus on what patients really need and enjoy every day what you are doing.