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, today announced that
enrollment in REFLECT, a Phase III clinical trial of GS010 for the
treatment of Leber Hereditary Optic Neuropathy (LHON), was
successfully completed ahead of schedule.
REFLECT is a multi-center, randomized, double-masked,
placebo-controlled study to evaluate the efficacy and safety of
bilateral injections of GS010 in subjects with LHON due to the NADH
dehydrogenase 4 (ND4) mutation. Enrolling the target number of 90
subjects was originally anticipated to be completed in September
2019; instead the 98th subject enrolled in the trial was treated on
July 2.
“The conclusion of enrollment for the REFLECT study is a
milestone in the story of gene therapy for Leber Hereditary Optic
Neuropathy,” commented Nancy J. Newman, MD, LeoDelle Jolley
Professor of Ophthalmology and Neurology, Emory University School
of Medicine, Atlanta, USA, and Principal Investigator in REFLECT.
“The recruitment of nearly 100 patients in less than two years is a
tribute to the support of the LHON community in this partnership
among researchers, clinicians and patients trying to bring therapy
and hope to this blinding disease.”
The trial was designed and agreed under a Special Protocol
Assessment (SPA) with the Food and Drug Administration (FDA) in the
United States but is not required for the Marketing Authorization
Application (MAA) in the European Union, RESCUE and REVERSE being
considered by the EMA as pivotal for filing in the EU. The trial
enrolled subjects with vision loss up to 1 year in duration and is
underway across multiple centers in the United States, Europe, and
Taiwan. In the active arm, GS010 was administered as a single
intravitreal injection to both eyes of each subject. In the placebo
arm, GS010 was administered as a single intravitreal injection to
the first affected eye, while the fellow eye received a placebo
injection.
The primary endpoint for the REFLECT trial is the best corrected
visual acuity (BCVA) change from baseline reported in LogMAR at 52
weeks post-treatment in the second affected/not yet affected eye.
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, contrast sensitivity and quality of life.
The first subject was treated in March 2018; topline Week 52
results are expected to be available in the third quarter of
2020.
GS010 has Orphan Drug Designation both in the United States and
in Europe.
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. 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
About REFLECT
REFLECT is a multi-center,
randomized, double-masked, placebo-controlled study to evaluate the
efficacy and safety of bilateral injections of GS010 in subjects
with LHON due to the NADH dehydrogenase 4 (ND4)
mutation.
The trial enrolled 98 subjects
with vision loss up to 1 year in duration and is conducted in
multiple centers in Europe, in the US and in Taiwan.
In the active arm, GS010 was
administered as a single intravitreal injection to both eyes of
each subject. In the placebo arm, GS010 was administered as a
single intravitreal injection to the first affected eye, while the
fellow eye received 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. 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; topline Week 52 results are expected to
be available in the third quarter of 2020.
ClinicalTrials.gov Identifiers:
REFLECT: NCT03293524
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version on businesswire.com: https://www.businesswire.com/news/home/20190711005645/en/
GenSight Biologics Thomas Gidoin Chief Financial Officer
tgidoin@gensight-biologics.com +33 (0)1 76 21 72 20
RooneyPartners Media Relations Marion Janic
mailto:mjanic@rooneyco.com +1-212-223-4017
Solebury Trout US Investor Relations Chad Rubin
crubin@troutgroup.com +1-646-378-2947
James Palmer Europe Investor Relations
j.palmer@orpheonfinance.com +33 7 60 92 77 74
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