Promising Phase I/II results for GenSight’s GS010 in LHON
Posted: 9 June 2016 | | No comments yet
The Phase I/II study was designed to demonstrate the safety and tolerability of GS010 in 15 patients with Leber’s Hereditary Optic Neuropathy…
GenSight has announced additional promising results from its Phase I/II study, designed to demonstrate the safety and tolerability of GS010 in 15 patients with Leber’s Hereditary Optic Neuropathy (LHON).
Each cohort of three patients was administered an escalating dose of GS010 through a single intravitreal injection in the eye most severely affected by the disease. Recruitment was completed in April 2015. These patients had an average onset of disease of 6 years.
At 48 weeks post-injection, in patients with an onset of disease of less than 2 years, a gain of +30 letters (-0.59 LogMAR) was observed in the treated eye and +13 letters (-0.25 LogMAR) in the untreated eye, a difference of 17 letters in favor of the treated eye. No significant difference was observed in patients with an onset of disease of more than two years.
The combined effect of the administered dose and the time from onset is noticeable at 36 weeks and stable after 48 weeks.
Results support GenSight’s MTS technology platform
Commenting on the results, Bernard Gilly, CEO and co-founder of GenSight, said: “This preliminary data is very encouraging and validates the design of the two Phase III studies currently ongoing in the US and Europe on GS010 for the treatment of Leber’s Hereditary Optic Neuropathy.”
Dr Gilly added: “These results support the potential of our mitochondrial targeting sequence technology platform or MTS, and allows us to envision other applications in diseases involving defects of the mitochondrion, in ophthalmology as well as in other therapeutic areas.”
GS010 targets LHON, a rare maternally inherited mitochondrial genetic disease, characterised by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. GS010 leverages a MTS proprietary technology platform, arising from research works conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produce the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function.