LONDON, Oct. 2, 2019 /PRNewswire/ -- ReNeuron Group plc
(AIM: RENE), a global leader in the development of cell-based
therapeutics, is pleased to announce new positive data in the
Company's ongoing Phase 1/2a clinical trial of its human retinal
progenitor cell (hRPC) therapy candidate in the blindness-causing
disease, retinitis pigmentosa (RP).
At most recent follow-up in the Phase 2a segment of the study,
the mean improvements from baseline in visual acuity as
measured by the number of letters read on the ETDRS
chart (the standardized eye chart used to measure visual
acuity in clinical trials) were as follows:
Months
post-treatment
|
Mean improvement in
visual acuity in treated eye
|
Mean improvement in
visual acuity in treated eye (excluding two patients with
procedure-related vision loss)
|
Mean change in visual
acuity in untreated eye
|
1
|
+8.3 letters
(n=8)
|
+14.5 letters
(n=6)
|
+ 1.6 letters
(n=8)
|
2
|
+5.4 letters
(n=8)
|
+13.0 letters
(n=6)
|
+ 2.8 letters
(n=8)
|
3
|
+6.1 letters
(n=8)
|
+17.8 letters
(n=6)
|
+ 6.8 letters
(n=8)
|
6
|
+18.5 letters
(n=4)
|
+28.7 letters
(n=3)
|
+ 7.8 letters
(n=4)
|
9
|
+12.0 letters
(n=1)
|
+12.0 letters
(n=1)
|
- 1.0 letter
(n=1)
|
The observed efficacy was rapid and profound in the first three
patients in the Phase 2a segment of the study. Efficacy in
subsequent patients has also been seen but at a lower rate and
magnitude (improvement in visual acuity ranging from +5 to +11
letters in the treated eye 3 months post-treatment), with the
ultimate extent of improvement not yet known as the study
is ongoing. Overall, the improvements to date represent a clear
signal of efficacy in a patient population where inexorable disease
progression is the norm.
A total of 22 patients have now been treated in the Phase 1/2a
study and a good safety profile has been established, with no
patients experiencing product-related serious adverse events and
two patients experiencing surgical procedure-related loss of
vision.
These latest results will be presented in further detail by
Pravin Dugel M.D., Managing Partner,
Retinal Consultants of Arizona,
Phoenix, Arizona, and Clinical
Professor, Roski Eye Institute, USC
Keck School of Medicine, Los Angeles,
California, at the American Academy of Ophthalmology Annual
Meeting (AAO) in San Francisco,
taking place on October 12-15, 2019.
Dr. Dugel will present in a Late Breaking Developments
session at the meeting on October 12,
2019.
Olav Hellebø, Chief Executive Officer of ReNeuron, will give a
presentation in the Gene and Cell Therapy Spotlight session during
the Ophthalmology Innovation Summit (OIS@AAO) on October 10, 2019, preceding the AAO meeting.
"I am looking forward to presenting and discussing these
encouraging data at the upcoming AAO meeting in San
Francisco. I believe that we are seeing a clear signal of
efficacy in this patient population where any gain in vision, let
alone the levels seen in some of these patients, is so hard to come
by and so very much appreciated," commented Dr.
Pravin Dugel.
RP is a group of hereditary diseases of the eye that lead to
progressive loss of sight due to cells in the retina becoming
damaged and eventually dying. The Company's RP
clinical program has been granted Orphan Drug Designation in both
Europe and the U.S., as well as
Fast Track Designation from the U.S. Food and Drug Administration
(FDA).
The Phase 1/2a clinical trial is an open-label study to evaluate
the safety, tolerability and preliminary efficacy of ReNeuron's
hRPC stem cell therapy candidate in patients with advanced
RP. The Phase 2a segment of the study, which uses a
cryopreserved hRPC formulation, enrolls subjects with some
remaining retinal function and is being conducted at two clinical
sites in the U.S. – Massachusetts Eye and Ear in Boston and Retinal Research Institute in
Phoenix, Arizona.
ReNeuron's hRPC cell therapy candidate offers a number of
potential advantages over alternative approaches to the treatment
of RP. Firstly, the cell therapy candidate is independent of
the many specific genetic defects that collectively define RP as a
disease, thereby allowing a much broader potential patient
population to be eligible for the treatment. Secondly, the
cells are cryopreserved, enabling on-demand shipment and use at
local surgeries and hospitals. Finally, the cells are
injected directly to the site of retinal degeneration, allowing a
greater chance of anatomic restoration of photoreceptor
function.
The Company will continue to generate further longer-term follow
up data from the ongoing Phase 1/2a study. In parallel, the
Company will consult with its advisers and regulatory authorities
in Europe and the US in order to
design and agree the future clinical development program for its
hRPC cell therapy candidate for the treatment of RP.
"We are greatly encouraged by the latest efficacy
data from the ongoing Phase 1/2a clinical study of our hRPC cell
therapy candidate in patients with RP," commented Olav
Hellebø, Chief Executive Officer of ReNeuron. "It is
especially gratifying to see positive data given the inherent
challenge in addressing a disease as complex as RP and we look
forward to further progressing the clinical development of this
promising cell therapy candidate."
About ReNeuron
ReNeuron is a global leader in cell-based therapeutics,
harnessing its unique stem cell technologies to develop 'off the
shelf' stem cell treatments, without the need for immunosuppressive
drugs. The Company's lead clinical-stage candidates are in
development for the blindness-causing disease, retinitis
pigmentosa, and for disability as a result of stroke.
ReNeuron is also advancing its proprietary exosome technology
platform as a potential delivery system for drugs that would
otherwise be unable to reach their site of action. ReNeuron's
shares are traded on the London AIM market under the symbol RENE.L.
For further information visit www.reneuron.com.
Contacts:
ReNeuron
|
+44 (0) 20 3819
8400
|
Olav Hellebø, Chief
Executive Officer
|
Michael Hunt, Chief
Financial Officer
|
Buchanan (UK
Media/Investor Relations)
|
+44 (0) 20 7466
5000
|
Mark Court, Sophie
Wills, Tilly Abraham
|
Argot Partners
(US Media/Investor Relations)
Claudia Styslinger,
David Rosen
|
+1 212 600
1902
|
|
|
Stifel Nicolaus
Europe Limited (NOMAD and Joint Broker)
Jonathan Senior,
Stewart Wallace, Ben Maddison
|
+44 (0) 20 7710
7600
|
N+1 Singer
(Joint Broker)
Aubrey Powell, James
Moat, Mia Gardner
|
+44 (0) 20 7496
3000
|
|
|
View original
content:http://www.prnewswire.com/news-releases/reneuron-announces-clinical-update-and-conference-presentations-300929243.html
SOURCE ReNeuron Group plc