2020 – A Year in Review

As a new year approaches, Khondrion’s CEO Jan Smeitink reflects on 2020 and the progress Khondrion has made in its research and the development of new treatment options for patients suffering from mitochondrial disease. 

2020 has been a year like no other – never before has the spotlight been so focused on the life sciences sector and what it can achieve for humankind. Beyond the companies researching much needed vaccines and therapeutics against COVID-19, this global industry has continued to progress its potentially life-saving research and deliver new treatments to patients. Despite the hurdles 2020 has thrown our way, the healthcare industry has shown how resilient, collaborative and committed to patients it can be.

 At Khondrion I am thankful that, over the past 12 months, we have been able to make significant progress in our research developing innovative treatment options for patients with inherited mitochondrial diseases. We kick-started this year with the initiation of our phase 2b trial in adult MELAS spectrum disorder patients. The study’s key objective is to reconfirm and expand on the positive phase 2a results of sonlicromanol, our wholly-owned lead asset, on cognitive function, which is becoming increasingly recognised as a significant and debilitating aspect of mitochondrial disease. Despite experiencing some delays from the pandemic, I am pleased this trial is progressing well and we expect data next year which should support us to advance sonlicromanol towards a pivotal trial.

 Our lead asset’s potential in childhood mitochondrial diseases was also under the spotlight this year when the US Food and Drug Administration (FDA) granted a Rare Paediatric Disease Designation for sonlicromanol for the treatment of patients with MELAS syndrome. The FDA grants RPD designations for serious and life-threatening diseases that primarily affect children aged 18 years or younger and fewer than 200,000 people in the United States. This milestone signalled the FDA’s recognition that MELAS syndrome presents a serious and unmet medical need and highlights the potential of sonlicromanol to become the first disease-modifying treatment for this devastating mitochondrial disease. Developing sonlicromanol for a paediatric population is a key part of our research here at Khondrion and I hope to share more news on that work in Europe early next year.

 Over the past year we have continued to strengthen the Khondrion team, welcoming experts who will be key in helping us accomplish our mission of bringing new, clinically meaningful medicines to patients living with mitochondrial diseases. Firstly, we welcomed Dr. Rob van Maanen, who was appointed Chief Medical Officer in February. Rob is already playing a leading role in the late stage development of our pipeline and the expansion of Khondrion’s clinical capabilities building on his many years of experience as a drug developer in several leading pharma companies. Dr. Herma Renkema, already part of Khondrion’s scientific team since 2016, was appointed Chief Early Development Officer in July. Herma is now responsible for our early-stage pipeline, overseeing Khondrion’s preclinical development strategy and managing the day-to-day operations within the company’s laboratories.

We also took an important step this year in establishing the company’s Scientific Advisory Board, welcoming Prof. Sir Doug Turnbull as its Chairman, alongside Prof. Marni Falk, Prof. Michelangelo Mancuso, Prof. Akira Ohtake, Prof. Shamima Rahman and Prof. Anu Suomalainen Wartiovaara. I have known all of them professionally for a number of years and I have great admiration for their scientific expertise and commitment to furthering their fields of mitochondrial research. It is a testament to the strength of the work that everyone is doing at Khondrion that these leaders in their fields have chosen to join us. Khondrion’s pipeline is at a critical stage and their guidance will be invaluable as we progress sonlicromanol through clinical development.

 While Khondrion’s focus throughout 2020 has remained steadfast in its pursuit of new treatments for mitochondrial disease, as a science-driven company it was impossible to ignore the global call issued early in the year for research that might contribute to the fight against COVID-19. I was very proud of our team’s determination exploring a scientific hypothesis born out of data generated during sonlicromanol’s development and its effects on the body’s inflammatory responses. Whether there is an opportunity to further develop sonlicromanol in this setting remains to be seen but it warrants further investigation by the scientific community.

Despite the unprecedented challenges faced globally the commitment and agility of all patient organisations have continued to provide invaluable platforms for patients and researchers over the past year. I saw, first-hand, just a fraction of their hard work at a number of the virtual events that took place this year, allowing the important conversations and interactions between academia, industry and the patient communities to continue. For me, these events highlight the strength of the patient community and the commitment of all stakeholders to progress mitochondrial disease research.

To close, I would like to thank our talented team who continues working with such passion and dedication to finding treatments that can make a substantial impact on the lives of mitochondrial disease patients. And to everyone who has continued to support Khondrion with that mission. I hope that 2021 will be a pivotal year for both Khondrion and the mitochondrial disease community as a whole.

Wishing you all the very best for the holidays and the New Year.

Jan Smeitink

CEO & Founder