GenSight Biologics to Host KOL Breakfast on May 23, 2019 in New York

PARIS–(BUSINESS WIRE)–Regulatory News:

GenSight Biologics (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 that it will host a KOL
breakfast today, May 23, 2019 in New York City from 8:30 a.m. – 10:30
a.m. EST. Management and Key Opinion Leaders in the gene therapy and
ophthalmology space will present and discuss recent results and findings
from the REVERSE & RESCUE Phase III clinical trials of GS010 for the
treatment of Leber Hereditary Optic Neuropathy (LHON). Discussion will
also focus on the regulatory strategy for GS010.

Speakers will include:

  • David J. Calkins, PhD, O’Day Professor, Vice Chair and Director
    for Research Vanderbilt Eye Institute, Vanderbilt University Medical
    Center, Nashville, United-States
  • Sean Donahue, MD, PhD, Coleman Professor of Ophthalmology,
    Neurology and Pediatrics; Vice Chair of Clinical Affairs,
    Ophthalmology Vanderbilt University Medical Center, Nashville,
  • Mark Moster, MD, Neuro-Ophthalmology, Wills Eye Hospital and
    Professor of Neurology and Ophthalmology at Thomas Jefferson
    University, Philadelphia, United-States (Investigator in REVERSE
    and RESCUE trials)
  • José-Alain Sahel, MD, Director of the Institut de la Vision
    (Sorbonne-Université/Inserm/CNRS), Paris; Chairman of the Department
    of Ophthalmology at Centre Hospitalier National d’Ophtalmologie des
    , Paris, France; Professor and Chairman of the Department of
    Ophthalmology at University of Pittsburgh School of Medicine and UPMC
    (University of Pittsburgh Medical Center), United-States, and
    Co-Founder of GenSight Biologics
  • Robert C. Sergott, MD, Director, Neuro-Ophthalmology, Wills Eye
    Hospital; Director, William H. Annesley, Jr, EyeBrain Center, and
    Professor of Neurology and Ophthalmology at Thomas Jefferson
    University, Philadelphia, United-States

The presentation will be webcast live at
For those not available to attend or listen to the live broadcast, a
replay will be archived for 3 months and available at

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. 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. Identifiers:

REVERSE: NCT02652780

RESCUE: NCT02652767


REFLECT is a multi-center, randomized, double-masked, placebo-controlled
study to evaluate the safety and efficacy of bilateral injections of
GS010 in subjects with LHON due to the NADH dehydrogenase 4 (ND4)

The trial is planned to enroll 90 patients with vision loss up to 1 year
in duration and will be conducted in multiple centers in Europe and in
the US.

In the active arm, GS010 will be administered as a single intravitreal
injection to both eyes of each subject. In the placebo arm, GS010 will
be administered as a single intravitreal injection to the first affected
eye, while the fellow eye will receive a placebo injection.

The primary endpoint for the REFLECT trial is the BCVA reported in
LogMAR at 1-Year post-treatment in the second-affected/not-yet-affected
eye. The change from baseline in second-affected/not-yet-affected eyes
receiving GS010 and placebo will be the primary response of interest.
The secondary efficacy endpoints include: BCVA reported in LogMAR at
2-Years post-treatment in the second-affected/not-yet-affected eye
compared to both placebo and the first-affected eye receiving GS010, OCT
and contrast sensitivity and quality of life scales. The first subject
was treated in March 2018. Identifiers:

REFLECT: NCT03293524


GenSight Biologics
Thomas Gidoin
Financial Officer
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Media Relations
Marion Janic

Solebury Trout
US Investor Relations

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Europe Investor Relations
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