GenSight Biologics Announces Presentation of 72-Week Data From the REVERSE Phase III Trial of GS010 at the 45th Annual Meeting of NANOS

PARIS–(BUSINESS WIRE)–Regulatory News:

GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN: FR0013183985,
PEA-PME eligible), a biopharma company focused on discovering and
developing innovative gene therapies for retinal neurodegenerative
diseases and central nervous system disorders, announced today that the
72-week results from the REVERSE Phase III clinical trial of GS010 will
be presented at the 45th Annual Meeting of the North American
Neuro-Ophthalmology Society (NANOS), which will be held in Las Vegas, NV
from March 16 to 21, 2019. The results will be presented by Dr. Nancy J.
Newman, MD, LeoDelle Jolley Professor of Ophthalmology and Neurology at
the Emory University School of Medicine in Atlanta, GA, USA. Dr. Newman
is an International Principal Investigator and Site Principal
Investigator of the REVERSE trial. Widely recognized as one of the
leading authorities on Leber Hereditary Optic Neuropathy (LHON), Dr.
Newman has been integral to defining and describing LHON pathophysiology
and clinical declarations.

North American Neuro-Ophthalmology Society (NANOS)
16-21, 2019 – Las Vegas (NV, USA)

“rAAV2/2-ND4 Treatment
of Leber Hereditary Optic Neuropathy: 72-Week Data from the REVERSE
Phase III Clinical Trial”
presented by Nancy J. Newman, MD,
LeoDelle Jolley Professor of Ophthalmology and Neurology, Emory
University School of Medicine, Atlanta, GA

  • Scientific Platform Session III, Red Rock Ballroom
  • Tuesday, March 19, 10:30 am – 10:45 am PDT

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on
discovering and developing innovative gene therapies for retinal
neurodegenerative diseases and central nervous system disorders.
GenSight Biologics’ pipeline leverages two core technology platforms,
the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help
preserve or restore vision in patients suffering from blinding retinal
diseases. GenSight Biologics’ lead product candidate, GS010, is in Phase
III trials in Leber Hereditary Optic Neuropathy (LHON), a rare
mitochondrial disease that leads to irreversible blindness in teens and
young adults. Using its gene therapy-based approach, GenSight Biologics’
product candidates are designed to be administered in a single treatment
to each eye by intravitreal injection to offer patients a sustainable
functional visual recovery.

About GS010

GS010 targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a
mitochondrial targeting sequence (MTS) proprietary technology platform,
arising from research 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 produces the functional protein, which will
then be shuttled to the mitochondria through specific nucleotidic
sequences in order to restore the missing or deficient mitochondrial

About Leber Hereditary Optic Neuropathy (LHON)

Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited
mitochondrial genetic disease, characterized 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. LHON is associated with painless, sudden loss of
central vision in the 1st eye, with the 2nd eye
sequentially impaired. It is a symmetric disease with poor functional
visual recovery. 97% of patients have bilateral involvement at less than
one year of onset of vision loss, and in 25% of cases, vision loss
occurs in both eyes simultaneously. The estimated incidence of LHON is
approximately 1,400 to 1,500 new patients who lose their sight every
year in the United States and Europe.


RESCUE and REVERSE are two separate randomized, double-masked,
sham-controlled Phase III trials designed to evaluate the efficacy of a
single intravitreal injection of GS010 (rAAV2/2-ND4) in subjects
affected by LHON due to the G11778A mutation in the mitochondrial ND4

The primary endpoint will measure the difference in efficacy of GS010 in
treated eyes compared to sham-treated eyes based on Best-Corrected
Visual Acuity (BCVA), as measured with the ETDRS at 48 weeks
post-injection. The patients’ LogMAR (Logarithm of the Minimal Angle of
Resolution) scores, which are derived from the number of letters
patients read on the ETDRS chart, will be used for statistical purposes.
Both trials have been adequately powered to evaluate a clinically
relevant difference of at least 15 ETDRS letters between treated and
untreated eyes adjusted to baseline.

The secondary endpoints will involve the application of the primary
analysis to best-seeing eyes that received GS010 compared to those
receiving sham, and to worse-seeing eyes that received GS010 compared to
those that received sham. Additionally, a categorical evaluation with a
responder analysis will be evaluated, including the proportion of
patients who maintain vision (< ETDRS 15L loss), the proportion of
patients who gain 15 ETDRS letters from baseline and the proportion of
patients with Snellen acuity of >20/200. Complementary vision metrics
will include automated visual fields, optical coherence tomography, and
color and contrast sensitivity, in addition to quality of life scales,
bio-dissemination and the time course of immune response. By protocol,
readouts for these endpoints are at 48, 72 and 96 weeks after injection.

The trials are conducted in parallel, in 37 subjects for REVERSE and 39
subjects for RESCUE, in 7 centers across the United States, the UK,
France, Germany and Italy. Week 96 results are expected in 2019 for both
trials, after which patients will be transferred to a long-term
follow-up study that will last for three years.

ClinicalTrials.gov Identifiers:
REVERSE: NCT02652780


GenSight Biologics
Thomas Gidoin
Financial Officer

[email protected]
(0)1 76 21 72 20

Media Relations
Marion Janic
[email protected]

The Trout Group
US Investor Relations

[email protected]

James Palmer
Europe Investor Relations
[email protected]
7 60 92 77 74

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