Khondrion’s Lead Candidate Therapy for Mitochondrial Disease Wins New Patents

Khondrion has secured three new patents that protect several products, including the Dutch pharmaceutical company’s lead candidate, KH176, to treat mitochondrial disease and related conditions.

KH176 is a small molecule that targets reactive oxygen species, or byproducts of mitochondria. The compound belongs to a new class of drugs that are used to control oxidative and redox pathologies.

“We are extremely satisfied with the granting of this important asset within our IP portfolio,” Khondrion CEO Jan Smeitink said in a press release. “The grant of these patents brings further recognition of the quality of the innovation being carried out by the Khondrion team. We are actively working on the development of our pipeline.”

The three patents protect the library of chromanyl-derivative compounds in the United States, Europe and Hong Kong, offering Khondrion exclusive rights for KH176 and other compounds until at least 2033.

The European Medicines Agency granted KH176 its orphan drug designation to treat Leigh disease and MELAS (mitochondrial myopathy, encephalopathy, lactic acidosis and stroke) syndrome in Europe. Likewise, the U.S. Food and Drug Administration’s gave it similar designation to treat all inherited mitochondrial respiratory chain disorders.

The study, “KH176 under development for rare mitochondrial disease: a first in man randomized controlled clinical trial in healthy male volunteers” — published in the Orphanet Journal of Rare Diseases — suggest that KH176 was well tolerated in single doses up to 800 mg for seven days, as well as in multiple doses of 400 mg administered twice a day.

The most common adverse effects were headaches, reported in both the active group receiving KH176 and in the placebo group. The drug’s pharmacokinetic profile supported the twice-daily dosing. More about this study can be found here.

KH176 is now being studied in the Phase 2 KHENERGY clinical trial (NCT02909400) in patients with MELAS, MIDD (maternally inherited diabetes and deafness) and mixed phenotypes. Results are expected in the first quarter of 2018.