Chief Executive Officer of Khondrion, Prof. Dr. Jan Smeitink, reflects on the annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM):
Having returned from this year’s SSIEM symposium in Rotterdam, the Netherlands, I must commend Symposium President Prof. Dr. Ans van der Ploeg and her team on a remarkable job.
With near to 3,000 participants, the organisation definitely succeeded in Building Bridges – this year’s congress theme – between all stakeholders involved in the inborn errors of metabolism field.
I was lucky enough to chair a session in the plenary programme which, over four days and thanks to top-notch speakers, provided the community with an update on the latest information on a huge variety of inborn errors of metabolism, from molecule – to patient – to population. We learned about the latest state-of-the-art technological developments in areas such as metabolomics and systems biology; of breakthroughs in our understanding of organelle diseases like lysosomal and mitochondrial diseases; and of the exciting progress in an increasing number of clinical trials investigating future therapies designed to combat 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 (KH176), as a potential treatment for a range of mitochondrial diseases, including MELAS (mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) spectrum disorders.
Attending an event like SSIEM only reinforces the need for continued research into these diseases, in the face of such a huge patient burden. And yet the conference had a positive atmosphere with lively discussions during the scientific meetings and attendees spilling into the corridors 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 SSIEM, to learning more about the research within this field and, I hope, to sharing Khondrion’s progress in helping to achieve a better future for our patients.
Jan Smeitink
Khondrion is a clinical-stage pharmaceutical company discovering and developing therapies targeting mitochondrial disease. Founded by Prof. Jan Smeitink, a world-leader in mitochondrial medicine, 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, 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
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.
