Khondrion Receives Rare Pediatric Disease Designation for Sonlicromanol from US FDA

Khondrion Receives Rare Pediatric Disease Designation for Sonlicromanol from US FDA

Designation granted for the treatment of MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) syndrome

NIJMEGEN, the Netherlands - Monday, September 28, 2020: Khondrion, a clinical-stage biopharmaceutical company discovering and developing therapies targeting mitochondrial disease, today announces that it received a rare pediatric disease (RPD) designation from the United States (US) Food and Drug Administration (FDA) for sonlicromonal for the treatment of patients with MELAS syndrome.

Sonlicromanol is Khondrion's wholly-owned, potentially first-in-class oral small molecule and one of the most clinically-advanced disease-modifying drug treatments for mitochondrial disease in development. Currently in Phase IIb clinical development, sonlicromanol has already been granted Orphan Drug Designations for MELAS, Leigh disease and patients with maternally inherited diabetes and deafness (MIDD) syndrome in Europe, and for all inherited mitochondrial respiratory chain disorders in the US.

The FDA grants RPD designation 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. Subject to FDA approval of sonlicromanol for the treatment of MELAS syndrome, Khondrion may be granted a priority review voucher from the FDA that can be redeemed to obtain priority review for any subsequent marketing application or be sold or transferred to another company.

Prof. Dr. Jan Smeitink, Chief Executive Officer at Khondrion, said: " We are delighted to have received this rare pediatric disease designation for our lead clinical stage development program. It signals 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".

A Phase IIb study (KHENERGYZE) of sonlicromanol in MELAS spectrum disorders is currently ongoing across a number of internationally recognised mitochondrial disease centres in Europe. Khondrion intends to submit a pre-IND (Investigational New Drug) request in the short term to further discuss the development plan for sonlicromanol in the US.

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About Khondrion

Khondrion is a privately held clinical-stage biopharmaceutical 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.

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 MELAS syndrome

Mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes syndrome belongs to a clinical spectrum of mitochondrial diseases most frequently caused by a mutation (m.3243A>G) in the MT-TL1 gene in mitochondrial DNA (mtDNA). MELAS syndrome begins in childhood, usually between the ages of two and fifteen years, with approximately 75 percent of cases reporting an onset of the disorder before the age of 20 years. Although rare, it is one of the most common types of mitochondrial diseases caused by mutations in the mtDNA, with an estimated prevalence for MELAS spectrum disorders in adults and children of risk of 4.4 in 100,000 people. MELAS syndrome is a progressive and often early fatal disorder affecting organs and tissues with a high-energy demand, such as the brain and skeletal muscle. Current existing therapies only manage the symptoms and don't target the underlying cause of the disease.

About mitochondrial disease

Mitochondrial disease occurs when mitochondria, found within all cells of the human body and responsible for producing the energy necessary for cells to function, are defective. This can result in a wide range of serious and debilitating illnesses occurring shortly after birth or later in life. Signs and symptoms of these 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.

Contacts:
Khondrion BV Prof. Dr. Jan Smeitink, CEO E-mail: [e-mail] (mailto:[e-mail]) Tel: [phone] www.khondrion.com (http://www.khondrion.com/)	Consilium Strategic Communications Mary-Jane Elliott, David Daley, Melissa Gardiner E-mail: [e-mail] (mailto:[e-mail]) Tel: [phone]