Mitotech’s SkQ1 Improved Visual Acuity in Leber’s Hereditary Optic Neuropathy (LHON) Patients

Share Article

Mitotech’s SkQ1 improved visual acuity in Leber’s Hereditary Optic Neuropathy (LHON) patients in an open label clinical study.

Mitotech S.A, a Luxembourg based clinical stage biotechnology company, announced positive results of a clinical study in Leber’s Hereditary Optic Neuropathy (LHON) patients. LHON is a rare devastating genetic disease that leads to a sudden and rapid loss of central vision. Mitotech studied efficacy of SkQ1 eye drops in a group of 20 patients carrying 11778, 14484 and 3460 mutations and having experienced the onset of symptoms more than 12 months before enrollment. In the study, patients treated with Mitotech’s SkQ1 –a small molecule engineered to reduce oxidative stress inside mitochondria – demonstrated statistically significant average improvement of visual acuity of 0.3 logMAR over the first 6 months of treatment with even stronger response over the next two years of 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,” said Natalia Perekhvatova, CEO of Mitotech S.A. “SkQ1 was designed to counter excess of ROS in mitochondria and these data demonstrated relevancy of our approach for Leber’s Hereditary Optic Neuropathy – a genetic disease causing blindness. 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.”

The study was conducted in Russia and was designed as an open label study enrolling LHON patients with specific point mutations. Enrolled patients topically administered SkQ1 ophthalmic solution for 30 months TID. Data analysis for 20 patients enrolled more than 12 months after the onset of vision loss demonstrated statistically significant improvement of Best Corrected Visual Acuity (BCVA) after just 6 months of treatment with further sustained improvement of BCVA over the remaining two years of the study. On average, BCVA was improved by 0.3 logMAR after the first 6 months of treatment. Enrolled patients were predominantly male averaging 29 years of age with 1 to 18 years since the onset of vision loss. Mitotech’s study did not have a control group, but it is worth noting that previous studies conducted by other research groups have demonstrated no statistically significant BCVA improvement for LHON patients on placebo treatment.

"The study started in 2013 and took three and a half years from start to finish,” said Elena Karger, Mitotech’s study director “We are excited to see that SkQ1 demonstrated a really unique effect of improving vision acuity in LHON patients with established vision loss. The study also provided valuable long-term safety data for ophthalmic use of SkQ1. We did not observe any notable treatment-related adverse events over the 30-month treatment course. This result also serves as evidence of SkQ1’s ability to reach back of the eye in therapeutic amounts. That was a limited study, but it is a great first step to possible approval of our drug for LHON.”

About Mitotech S.A

Mitotech S.A. is a Luxembourg-based biotechnology company developing novel drugs for the treatment of predominantly age-related disorders. The core technology behind Mitotech products is based on a novel class of small molecules – mitochondria targeting cardiolipin peroxidation inhibitors. Company’s lead compound SkQ1 is being developed in several drug formulations covering a variety of therapeutic areas with major focus on ophthalmology and neurodegenerative diseases. Mitotech successfully completed Phase 2 clinical study for Dry Eye indication in the U.S. with other indications also approaching clinical stage of development.

Share article on social media or email:

View article via:

Pdf Print

Contact Author

Natalia Perekhvatova
Mitotech
+352 271127060
Email >
Visit website