LONDON, Feb. 24, 2020 /PRNewswire/ -- ReNeuron Group
plc (AIM: RENE), a global leader in the development of cell-based
therapeutics, is pleased to announce positive long-term data from
the ongoing Phase 1/2a clinical trial of its hRPC stem cell therapy
candidate in retinitis pigmentosa (RP) and plans to expand the
ongoing study. RP is a group of hereditary diseases of the
eye that lead to progressive loss of sight and blindness.
In October 2019, positive interim
efficacy data from patients treated in the Phase 2a segment of the
ongoing Phase 1/2 study were announced by the Company and
subsequently presented by Dr. Pravin
Dugel at the American Academy of Ophthalmology Meeting in
San Francisco. The data showed a group of subjects who had
had a successful surgical procedure with sustained clinically
relevant improvements in visual acuity compared with baseline, as
measured by the number of letters read on the ETDRS chart (the
standardized eye chart used to measure visual acuity in clinical
trials).
Subsequent long-term efficacy data from the study continue to
show a meaningful clinical effect from the therapy at all time
points out to twelve months post-treatment. As previously
reported, the degree of efficacy observed varies between patients,
with mean results as set out in the table below:
Months post-
treatment
|
Mean change
from
baseline in visual acuity
in treated eye*
|
Mean change from
baseline in visual acuity
in untreated eye*
|
Difference in
mean
change between
treated eye and
untreated eye*
|
1
|
+11.4 letters
(n=8)
|
+ 0.3 letters
(n=8)
|
+ 11.1 letters
(n=8)
|
2
|
+10.8 letters
(n=8)
|
+ 1.6 letters
(n=8)
|
+ 9.2 letters
(n=8)
|
3
|
+14.0 letters
(n=8)
|
+ 5.1 letters
(n=8)
|
+ 8.9 letters
(n=8)
|
6
|
+15.7 letters
(n=6)
|
+ 6.5 letters
(n=6)
|
+ 9.2 letters
(n=6)
|
9
|
+16.5 letters
(n=4)
|
+ 6.0 letters
(n=4)
|
+ 10.5 letters
(n=4)
|
12
|
+14.3 letters
(n=3)
|
+ 7.0 letters
(n=3)
|
+ 7.3 letters
(n=3)
|
* In patients with a successful surgical
procedure
The equivalent data set announced by the Company in October 2019 is as follows:
Months post-
treatment
|
Mean change from
baseline in visual acuity
in treated eye*
|
Mean change from
baseline in visual acuity
in untreated eye*
|
Difference in
mean
change between
treated eye and
untreated eye*
|
1
|
+14.5 letters
(n=6)
|
+1.5 letters
(n=6)
|
+13.0 letters
(n=6)
|
2
|
+13.0 letters
(n=6)
|
+3.5 letters
(n=6)
|
+9.5 letters
(n=6)
|
3
|
+17.8 letters
(n=6)
|
+8.3 letters
(n=6)
|
+9.5 letters
(n=6)
|
6
|
+28.7 letters
(n=3)
|
+9.0 letters
(n=3)
|
+19.7 letters
(n=3)
|
9
|
+12.0 letters
(n=1)
|
-1.0 letters
(n=1)
|
+13.0 letters
(n=1)
|
* In patients with a successful surgical procedure
The Company has submitted a protocol amendment to the US FDA to
expand the Phase 1/2a study to treat up to a further nine patients
in the Phase 2a segment of the study with a dose of two million
hRPC cells, which compares with the dose of one million cells used
in the study thus far. The amended clinical trial protocol
also allows for a greater range of pre-treatment baseline visual
acuity in patients and includes changes that enhance the ability to
use microperimetry testing to measure and detect changes in retinal
sensitivity in patients treated.
The Company and its clinical advisers believe that this protocol
amendment will enable the efficacy signal observed in the study
thus far to be both better delineated and magnified when
demonstrated in a larger and more closely defined group of RP
patients.
In addition, the Company has submitted an application to the
MHRA to open the ongoing trial to a highly experienced UK clinical
site, the Oxford Eye Hospital, with Professor Robert MacLaren, a world-renowned leader in the
treatment of retinal diseases, as Principal Investigator.
The Company expects to present further data from the expanded
Phase 1/2a clinical trial during the course of 2020 and expects to
have sufficient data from the study to enable it to seek approval
in the first half of 2021 to commence a pivotal clinical study with
its hRPC cell therapy candidate in RP.
ReNeuron's clinical program in RP has been granted Orphan Drug
Designation in both Europe and the
US, as well as Fast Track designation from the FDA in the US.
Orphan Drug Designation provides the potential for a significant
period of market exclusivity once the therapy is approved in those
territories. Fast Track designation provides eligibility
for an accelerated approval and priority review process by the
FDA.
"We remain greatly encouraged by the data from the Phase 1/2a
clinical study of our hRPC cell therapy candidate in patients with
RP. The longer-term follow-up data are particularly
noteworthy, demonstrating that the therapy appears to maintain its
beneficial effects out to at least one year post-treatment,"
commented Olav Hellebø, Chief Executive Officer of
ReNeuron.
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.
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SOURCE ReNeuron Group plc