GenSight Biologics Announces Positive Data Safety Monitoring Board Review and Continuation of PIONEER Phase I/II Clinical Trial of GS030 Combining Gene Therapy and Optogenetics for the Treatment of Retinitis Pigmentosa

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, today announced that the
independent Data Safety Monitoring Board (DSMB) completed its first
safety review of the ongoing PIONEER Phase I/II clinical trial of GS030
combining gene therapy and optogenetics for the treatment of Retinitis
Pigmentosa. The DSMB confirmed the absence of any safety issues for the
first cohort of three subjects who received a single intravitreal
injection of 5e10 vg combined with a wearable optronic visual
stimulation device. The DSMB recommended moving forward as planned
without any modification in the protocol and recruiting the second
cohort of three subjects receiving an escalating dose of 1.5e11 vg.

“We are pleased to be able to move forward GS030, our second clinical
stage program. We look forward to confirm the safety of GS030 at higher
doses and to demonstrate efficacy in restoring useful visual functions
in RP patients,” commented Bernard Gilly, Co-founder and
Chief Executive Officer of GenSight.

PIONEER is a first-in-man, multi-center, open label dose-escalation
study to evaluate the safety and tolerability of GS030 in 18 subjects
with Retinitis Pigmentosa. GS030 combines a gene therapy (GS030-DP)
administered via a single intravitreal injection with a wearable
optronic visual stimulation device (GS030-MD).

GS030 is based on the optogenetics technology platform developed by
GenSight, which uses gene therapy to introduce a gene encoding for a
light-sensitive protein into retinal ganglion cells by a single
intravitreal injection, making them responsive to light and bypassing
disease-destroyed photoreceptors.

Eligible patients in the first three cohorts will be those affected by
end-stage non-syndromic Retinitis Pigmentosa with no light perception
(NLP) or light perception (LP) levels of visual acuity. The extension
cohort will include patients with hand motion (HM) and counting fingers
(CF) levels of visual acuity.

As per protocol, three cohorts of three subjects each will be
administered an increasing dose of GS030-DP via a single intravitreal
injection in their worse affected eye. An extension cohort will receive
the highest tolerated dose. The DSMB will review safety data of all
treated subjects in each cohort and make recommendations before moving
to the next dose.

The primary outcome analysis will be the safety and tolerability at one
year post-injection.

GS030 was granted Orphan Drug Designation in the United States and
Europe. PIONEER is being conducted in three centers across the United
Kingdom, France and the United States.

GenSight expects to complete enrollment in the first half of 2020. Early
findings may be available and released in the second half of 2019, and
preliminary results are expected in the fourth quarter of 2020.

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 GS030

GS030 leverages GenSight’s optogenetics technology platform, a novel
approach to restore vision in patients by using gene therapy to
introduce a gene encoding for a light-sensitive protein into
specifically targeted cells of the retina by a single injection, thereby
making them responsive to light. An external wearable medical device to
specifically stimulate the transduced cells is developed to amplify the
light signal and further enable vision. Patients will need to wear the
external wearable device to enable optimal restoration of visual
function. Using this optogenetics technology platform, and with the
support of the Vision Institute in Paris and the team of Dr. Botond
Roska at the Friedrich Miescher Institute in Basel, GenSight is
developing its second product candidate, GS030, to restore vision in
patients suffering from Retinitis Pigmentosa, or RP. GenSight’s
optogenetics technology platform is independent of the specific genetic
mutations that lead to this family of disease. It is expected that GS030
would benefit patients from the early stages of RP. This technology
offers the possibility of application to other diseases of the retina
where photoreceptors have degenerated, and may be transferable to the
dry form of Age Related Macular Degeneration (dry-AMD).

About Optogenetics

Optogenetics is a biological technique that involves the transfer of a
gene encoding for a light sensitive protein to cause neuronal cells to
respond to light stimulation. As a result, it is a neuromodulation
method that can be used to modify or control the activities of
individual neurons in living tissue and even in-vivo, with a very high
spatial and temporal resolution. Optogenetics combines the use of gene
therapy methods to transfer a gene into target neurons with the use of
optics and electronics (optronics) to deliver the light to the
transduced cells. Optogenetics is widely used by research laboratories
throughout the world and holds clinical promise in the field of vision
impairment or degenerative neurological disorders.

About Retinitis Pigmentosa (RP)

Retinitis Pigmentosa (RP) is a family of orphan genetic diseases caused
by multiple mutations in numerous genes involved in the visual cycle.
Over 100 genetic defects have been implicated. RP patients generally
begin experiencing vision loss in their young adult years, with
progression to blindness by age 40. RP is the most widespread hereditary
cause of blindness in developed nations, with a prevalence of about 1.5
million people throughout the world. In Europe and the United States,
about 350,000 to 400,000 patients suffer from RP, and every year between
15,000 and 20,000 new patients with RP lose sight. There is currently no
existing curative treatment for RP.

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

Solebury
Trout
US Investor Relations
Chad Rubin
[email protected]
+1-646-378-2947

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