Biopharma company, Khondrion, has marked announced improvements from patients with MELAS spectrum disorders taking its lead asset, sonlicromanol.
Updates were provided by the company from a 28-day double-blind, randomized placebo-controlled, three-way cross-over phase 2b study called KHNERGYZE in which 27 took part. It also reported updates on the ongoing, open-label extension KHEREREXT phase 2b study in seven patients for up to 52 weeks. Both studies are jointly called the phase 2b program.
Sonlicormanol for MELAS syndrome
People with MELAS syndrome have an accumulation of lactic acid in the blood (lactic acidosis), that can lead to vomiting, abdominal pain, fatigue, muscle weakness and difficulty breathing. This accumulation of lactic acid has also been observed in the spinal fluid and in the brain.
In line with previous studies, sonlicromanol was found to be safe and well tolerated in the phase 2b program, with no serious adverse effects. The overall evaluation of the shorter and longer term data from the program shows patient benefits in several domains (cognition and mood, pain, fatigue and balance control).
Progression to phase 3 study
Marked improvements in global health and quality of life assessments, supporting sonlicromanol’s progression into phase 3 clinical development in patients with MELAS spectrum disorders were also noted. In line with expectations in these slowly progressing rare diseases, analysis of the data shows more pronounced treatment effects after 52 weeks.
These longer-term patient data are, therefore, instrumental in providing valuable insights for the design of the upcoming phase 3 trial.
Patients want to continue with sonlicromanol
Dr. Mirian Janssen, one of the principal investigators, Radboud Centre for mitochondrial medicine the Netherlands, said: ‘’I am encouraged by these promising results, especially the improvement in quality of life measurements and NMDAS score over a longer period. The fact that the study-patients wish to continue using sonlicromanol and their personal motivation are also positive signs.
“I have had the privilege to work closely with many of these patients for many years and I am pleased to observe tangible improvements in those patients who have now been treated with sonlicromanol for 52 weeks. I praise Khondrion for its pursuit of mitochondrial disease innovation, care to the patients in its trials and commitment to this development program.”
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