Chief Executive Officer of Khondrion, Prof. Dr. Jan Smeitink, reflects on the Mitochondrial Medicine conference held from 11-13 December 2019 at the Welcome Genome Campus in Cambridge, UK.
Having returned from this year’s Mitochondrial Medicine Conference, I must commend the 2019 Scientific Programme Committee, Patrick Chinnery, Marni Falk and Vamsi Mootha on a remarkable job. The conference aimed to bring together leaders in mitochondrial medicine and the organizers succeeded in connecting all stakeholders involved in this field.
I was most impressed by the active participation of representatives from the patient organizations working both nationally and together under the umbrella of the International Mito Patient Organization (www.mitopatients.org). The representatives stressed the importance of developing much needed clinically relevant therapies and the combined responsibility we all have in reaching this goal. Having worked on both sides, as a Mitochondrial Medicine Professor and now as the CEO of a clinical stage pharmaceutical company, I have seen both the needs of patients and their families as well as the progress that has been made in the field. I am confident that by all stakeholders (patient groups, academia and industry) working together on new initiatives we will succeed in achieving our goal of combating mitochondrial disease.
At this year’s conference we learned about the latest state-of-the-art technological developments, the recent breakthroughs in our understanding of mitochondrial diseases; and the exciting progress in an increasing number of clinical trials investigating future therapies designed to fight these rare diseases. This was particularly significant for me as the Khondrion team nears the start of our phase IIb trial investigating our lead asset, Sonlicromanol, as a potential treatment for a range of mitochondrial diseases, including MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders.
Attending events like the Mitochondrial Medicine Conference and the upcoming June 2020 Euromit Conference organized by the NewCastle group further reinforces the need for continued research into mitochondrial diseases in the face of such a huge patient burden. Yet
the conference had a positive atmosphere with lively discussions during the scientific meetings and attendees spilling into the corridors of the conference centre as well! It was great to meet with so many colleagues and old friends, and to make new friends – all dedicated to building bridges, not only between scientific disciplines but also between all stakeholders involved, from across the globe.
I am already looking forward to next year’s Mitochondrial Medicine Conference, to learn even more about the research within this field and, I hope, to share Khondrion’s progress in helping to achieve a better future for our patients.
Khondrion is a clinical-stage pharmaceutical company discovering and developing therapies targeting mitochondrial disease. The company is advancing its proprietary science through a wholly owned clinical and preclinical small molecule pipeline of potential medicines.
Khondrion’s lead asset, Sonlicromanol a redox-modulator with anti-inflammatory properties, is a potential first-in-class oral small molecule in phase IIb clinical development to treat a range of mitochondrial diseases including m.3243A>G spectrum disorders covering MELAS, MIDD and mixed phenotypes. It has been granted Orphan Drug Designation for MELAS spectrum disorders Leigh disease and MIDD in Europe and for all inherited mitochondrial respiratory chain disorders in the USA.
The company’s in-house discovery engine is using unique live-cell imaging technologies, patient-derived cell lines and predictive cell-based disease models to build a portfolio of promising compounds. Active discovery programmes are underway developing new therapies, biomarkers and new read-out technologies in the field of mitochondrial diseases.
To accelerate the discovery and development of its potential medicines for mitochondrial diseases, Khondrion collaborates with a global clinical and academic network and patient organisations internationally. Khondrion is headquartered in Nijmegen, The Netherlands. For more information visit www.khondrion.com
About mitochondrial disease
Mitochondrial disease occurs when mitochondria, found within all cells of the human body and responsible for producing the energy necessary for life, are defective. This can result in a wide range of serious and debilitating illnesses, signs and symptoms of which can include: cognitive problems, learning disabilities, blindness, deafness, heart failure, diabetes, fatigue, intolerance to exercise, muscle weakness and gait problems, and stunted growth. Orphan diseases of the oxidative phosphorylation system like Leigh disease, MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders, MIDD (maternally inherited diabetes and deafness), LHON (Leber’s hereditary optic neuropathy) and other respiratory chain/ oxidative phosphorylation disorders, are all examples of mitochondrial disease.