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GenSight Biologics Announces Presentation of 72-Week Data from the REVERSE & RESCUE Phase III Trials of GS010 at the Annual Meeting of ARVO

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 and RESCUE Phase III clinical trials of
GS010 will be presented at the 2019 Association for Research in Vision
and Ophthalmology (ARVO) Annual Meeting in Vancouver, Canada, from April
28 to May 2, 2019. The results will be presented by Dr. Patrick
Yu-Wai-Man, MD, PhD, Cambridge Centre for Brain Repair, Department of
Clinical Neurosciences, and MRC Mitochondrial Biology Unit, University
of Cambridge, United Kingdom; NIHR Biomedical Research Centre at
Moorfields Eye Hospital and UCL Institute of Ophthalmology, London,
United Kingdom. Dr. Yu-Wai-Man is also the International Coordinating
Investigator of REVERSE.

Association for Research in Vision and Ophthalmology (ARVO)
April
28 – May 2, 2019 – Vancouver (BC, Canada)

“rAAV2/2-ND4 for the Treatment of Leber Hereditary Optic Neuropathy
(LHON): 72-Week Data from the REVERSE Phase III Clinical Trial”
presented
by Patrick Yu-Wai-Man, MD, PhD, Cambridge Centre for Brain
Repair, Department of Clinical Neurosciences, and MRC Mitochondrial
Biology Unit, University of Cambridge, United Kingdom; NIHR Biomedical
Research Centre at Moorfields Eye Hospital and UCL Institute of
Ophthalmology, London, United Kingdom.

  • Scientific Section: Eye Movements, Strabismus, Amblyopia,
    Neuro-Ophthalmology
  • Location: West 221/222
  • Session: Optic Neuropathy, Paper Session
  • Wednesday, May 1, 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
function.

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.

About RESCUE and REVERSE

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
gene.

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
RESCUE:
NCT02652767

Contacts

GenSight Biologics
Thomas Gidoin
Chief
Financial Officer

[email protected]
+33
(0)1 76 21 72 20

RooneyPartners
Media Relations
Marion Janic
[email protected]
+1-212-223-4017

The Trout Group
US Investor Relations
Chad
Rubin

[email protected]
+1-646-378-2947

James Palmer
Europe Investor Relations
[email protected]
+33
7 60 92 77 74

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