Investigational Therapy SkQ1 Shows Vision Benefits in LHON Patients, Russian Study Finds

Investigational Therapy SkQ1 Shows Vision Benefits in LHON Patients, Russian Study Finds

Use of eye drops to deliver an investigational compound called SkQ1 can help reduce the central vision symptoms of patients with mitochondrial disease Leber’s Hereditary Optic Neuropathy (LHON), results from a Phase 2 clinical study show.

LHON is a genetic condition characterized by the occurrence of mutations in mitochondrial genes. These genetic mutations disrupt the respiratory chain of mitochondria and cause accumulation of reactive oxygen species (ROS), leading to energy production impairment in the cells involved in image formation in the eye. The first symptoms commonly appear in young adults, but this condition is mainly asymptomatic until it leads to visual impairment or blindness.

Mitotech has developed a new compound, SkQ1, that captures oxygen-reactive elements, blocking their damaging effects. This candidate therapy is being tested in two drug formulations, Visomitin (SkQ1 eye drops) and Plastomitin (SkQ1 pill), to treat different ophthalmology and neurodegenerative conditions.

The company conducted a Phase 2 clinical trial in Russia to test the effectiveness and safety of Visomitin for treatment of central vision loss in LHON patients. The study included 20 participants carrying any of three LHON-causing mutations and who had vision-loss symptoms at least 12 months before the trial started. The patients were treated with SkQ1 eye drops for about 30 months.

The participants showed significant visual improvement after just six months of treatment. This beneficial effect was sustained through the study. Although the study did not include a placebo-treated control group, it found no severe adverse effects were noted during treatment.

“It is encouraging to see such a rapid onset of action for our drug and its continued effect at advanced stages of the disease,” Natalia Perekhvatova, CEO of Mitotech, said in a press release. “SkQ1 was designed to counter excess of ROS in mitochondria and these data demonstrated relevancy of our approach for Leber’s Hereditary Optic Neuropathy … . This result is extremely valuable for our clinical development of SkQ1 for LHON and potentially for other mitochondrial disorders in the U.S., Europe and other markets,” she said.

The company successfully completed a Phase 2 clinical trial for dry eye indication in the United States and has received marketing approval for Visomitin in Russia for that condition.


  1. Sharon Draper for Brian Weeden says:

    My son has LHON and would be interested in any trials he would be eligible for. Mutation 14484. He is totally blind and can be reached at 518-774-0318 if someone could contact him

  2. Christian says:

    My husband has LHON and has been blind for 15 years and he is very willing to be a part of any trials to help his vision and anyone who is diagnosed with any macular degeneration issues.

    • Alice Melão says:

      Dear Hasan,
      We at Mitochondrial Disease News are not involved in the research or development of the therapies that we present. Additional studies may still be required further consolidate the SkQ1 therapeutic value for LOHN patients. We will keep providing information on new advances made on the field.

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