Khondrion Completes Enrollment in Phase IIb KHENERGYZE Trial Evaluating Sonlicromanol in Adult Patients with MELAS Spectrum Disorders

Khondrion complete registration in KHENERGYZE Phase IIb test assess sonlicromanol in adult patients with MELAS spectrum disorders

NIJMEGEN, THE NETHERLANDS – JANUARY 10, 2022: Khondrion, a clinical-stage biopharmaceutical company that discovers and develops therapies targeting primary mitochondrial diseases, today announced that the latest patient has received a dose of sonlicromanol as part of the ‘KHENERGYZE phase IIb clinical study. Sonlicromanol is the main 100% owned ingredient in Khondrion, developed to treat a range of mitochondrial diseases in children and adults. KHENERGYZE topline data is expected in the third quarter of 2022.

The primary objective of the study is to assess the dose effect of sonlicromanol on the attention domain score of cognitive functioning, as assessed by the Cogstate computerized visual identification test. Cognitive impairment is increasingly recognized in patients with mitochondrial diseases and can have a significant and debilitating impact on many aspects of their lives. The potential of sonlicromanol to counter cognitive decline is supported by preclinical research and the results of a completed Phase IIa study, which showed significant improvement in attention and mood outcomes in patients treated with sonlicromanol compared to placebo.

Khondrion Managing Director Prof Jan Smeitink said: End of registration in our Phase IIb study is an important step and brings us one step closer to providing an urgent and much needed disease modifying therapy for patients with severe and debilitating mitochondrial diseases. I would like to express my gratitude to all patients and their families for their participation in this trial, as well as to the investigators and the broader team, whose dedication has been vital in advancing this important clinical programmme. WWe are looking forward to to receive results of the trial later in the year. ”

Sonlicromanol is a first-class oral small molecule targeting key underlying mechanisms of mitochondrial disease based on its unique triple mode of action: redox modulation to help restore cell metabolism, radical scavenging preventing ferroptotic cell death and inhibition of mPGES-1 resulting in anti-inflammatory effects. In phase I and phase IIa studies, sonlicromanol has shown a good safety and tolerability profile well beyond the target therapeutic doses.

KHENERGYZE is a double-blind, randomized, placebo-controlled, multicenter, three-way crossover study examining cognitive function in adult patients with a specific genetic mutation of DNA in mitochondrial transfer RNA.Leu(UUR) (MT-TL1m.3243A> G). This mutation is responsible for disorders of the MELAS spectrum, in particular MELAS syndromes (mitochondrial encephalomyopathy, lactic acidosis and stroke), MIDD syndromes (diabetes and deafness of maternal origin) and mixed phenotypes.

Sonlicromanol’s development program also includes two other ongoing clinical studies: the open-label KHENEREXT Phase IIb extension study, examining the long-term safety and efficacy of sonlicromanol in adult patients who have completed the KHENERGYZE study, and the KHENERGYC Phase II study in children.

Further details on the KHENERGYZE study are available at clinicaltrials.gov.

– ENDS

Contacts:

Khondrion BV
Teacher. Dr. Jan Smeitink, CEO
Email: [email protected]
Phone: + 31-24-7635000
www.khondrion.com

Strategic Communication Consilium
Mary-Jane Elliott, David Daley, Melissa Gardiner
Email: [email protected]
Phone. : +44 20 3709 5700

About Khondrion
Khondrion is a clinical-stage biopharmaceutical company developing therapies for patients with inherited mitochondrial diseases. Based on proprietary science and a deep biological understanding of mitochondrial dysfunction, the company is advancing its lead drug candidate, sonlicromanol, a first-class oral small molecule targeting key underlying mechanisms of mitochondrial disease based on the drug’s unique triple mode of action.

One of the most advanced disease-modifying drug candidates for developing mitochondrial disease, sonlicromanol is currently being tested in a phase IIb trial and a 12-month open-label extension study in adult patients with MELAS (mitochondrial encephalomyopathy, lactic acidosis and stroke -like episodes) of spectrum disorders, as well as in a 6-month phase II study in children with genetically confirmed primary mitochondrial diseases and who suffer from motor symptoms. The compound has received orphan drug designations for the treatment of MELAS, Leigh’s disease, and patients with maternal inherited diabetes and deafness (MIDD) in Europe, and for all inherited disorders of the mitochondrial respiratory chain in the States. -United. It has also received a rare pediatric disease designation in the United States for the treatment of MELAS. Sonlicromanol and other compounds from Khondrion’s proprietary library have the potential to be developed for a wide range of diseases and conditions to benefit patients whose daily lives are severely affected by mitochondrial impairment.

For more information, visit www.khondrion.com.

About mitochondrial disease
Mitochondrial disease occurs when the mitochondria, which are present in all cells of the human body and responsible for producing the energy necessary for cells to function, fail. This can lead to a wide range of serious and debilitating illnesses occurring soon after birth or later in life. Signs and symptoms of these may include cognitive problems, learning disabilities, blindness, deafness, heart failure, diabetes, fatigue, exercise intolerance, muscle weakness, and gait problems, and stunted growth. Orphan diseases of the oxidative phosphorylation system such as Leigh’s disease, MELAS spectrum disorders (mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes), including MIDD (maternal inherited diabetes and deafness), and Other respiratory chain disorders / oxidative phosphorylation, are all examples of mitochondrial disease. MELAS spectrum disorders are among the most frequently observed primary mitochondrial diseases, in which all patients are characterized by an underlying point mutation (m.3243A> G) in the MTTL1 gene inherited from the mother.


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