2022 – A Year in Review

As 2022 draws to a close, Khondrion’s CEO Jan Smeitink reflects on the significant progress achieved by Khondrion in its development of new treatment options for patients suffering from inherited mitochondrial diseases and looks ahead at what is to come in 2023.

2022 was an important year for Khondrion, continuing to build on the progress we made in 2021. I am incredibly proud of the team’s achievements this past year as we get closer to our goal of bringing a much-needed treatment option to patients with primary mitochondrial diseases.

The year culminated in the announcement of promising results from the Phase IIb development programme for our wholly owned lead asset, sonlicromanol, in patients with MELAS spectrum disorders – a rare and progressive mitochondrial disease. While sonlicromanol’s effect did not meet the primary endpoint in the 28-day Phase IIb KHENERGYZE study, broader results from the Phase II program, including an interim analysis of the open-label extension 52-week KHENEREXT study, provide, in our view, unequivocal evidence of the positive longer-term clinical impact and important patient benefit that sonlicromanol can provide to patients. We are particularly encouraged by improvement in patients’ quality of life, including overall disease severity, mood, cognition, fatigue, balance and pain, which are some of the most burdensome symptoms patients with MELAS spectrum disorders experience in their daily lives. 

These data support advancing sonlicromanol into Phase III development, for which we are currently making good progress. We are looking forward to discussing our plans with European and U.S. regulators soon, with the aim of initiating a Phase III registrational study in late 2023. 

Additionally, to support the Phase III development of sonlicromanol and continue developing our pipeline and expanding our clinical capabilities, we strengthened our team with the appointment of Dr. Tom Pulles as Chief Medical Officer in June. 

Patients are at the heart of what we do at Khondrion, and I am pleased that we have been fortunate enough to continue our ‘Patient Perspective’ series this past year, highlighting stories from the selfless individuals leading mitochondrial disease patient advocacy groups, ensuring patients’ voices remain a key part of the conversation. Their inspiring stories can be accessed and read on our website.   

Collaboration with patients, physicians and the scientific community remain vital in the pursuit of much-needed treatment approaches to tackling rare diseases, such as mitochondrial disease, and we were delighted to be able to return to in-person events and conferences this year. The team presented at a number of international conferences, including mitoNice in September, the World Mitochondria Society’s Targeting Mitochondria 2022 Congress in October, and Wellcome’s Mitochondrial Medicine – Therapeutic Development Conference in November, where we presented the results from our Phase IIb sonlicromanol clinical programme. Opportunities to share knowledge, including the latest research in mitochondrial disease and the current understanding around potential pathways towards diagnosis and treatment are critical to bringing meaningful change to the lives of patients with mitochondrial diseases.

I would like to take this opportunity to express my heartfelt thanks to the patients and their families who have taken part in these studies, and to the patient advocacy groups, the clinical study teams, and Foundations and Governmental bodies for their contributions to our research.

Looking ahead to 2023, we will continue build on the success and insights gained from this past year and I look forward to sharing Khondrion’s progress as we continue into our next phase of clinical development for sonlicromanol. 

I would also like to extend a huge thank you to our devoted and talented team at Khondrion for their continued hard work, dedication, and commitment to furthering this field of research and to developing new and much needed treatments for patients with mitochondrial diseases. 

I wish you all the best for the holidays and a happy New Year. 

Jan Smeitink

CEO & Founder