LONDON, June 29, 2020 /PRNewswire/ -- ReNeuron Group plc
(AIM:RENE), a global leader in the development of cell-based
therapeutics, is pleased to announce further positive long-term
data from the ongoing Phase 2a clinical trial of its hRPC stem cell
therapy candidate in retinitis pigmentosa (RP). RP is a group
of hereditary diseases of the eye that lead to progressive loss of
sight and ultimately blindness.
On February 24, 2020, positive
interim efficacy data from patients treated in the Phase 2a study
were announced by the Company. The data continued to show a
meaningful clinical effect from the therapy at all time-points out
to twelve months post-treatment, as measured by the number of
letters read on the ETDRS chart (the standardised eye chart used to
measure visual acuity in clinical trials).
Since those results were announced, further long-term data from
the study have been gathered from patients at six, nine, twelve and
now, for the first patient treated, eighteen months
follow-up. The Company is pleased to report that the latest
data continue to demonstrate the efficacy of the therapy, with a
clinically meaningful benefit being observed at all
time-points. These results are particularly encouraging as RP
is characterized by inexorable progression to blindness, with no
therapy currently available for the vast majority of patients.
The results announced on February 24,
2020 excluded two subjects who experienced sight loss as a
result of complications arising from the surgical procedure.
The Company is pleased to report that one of these two patients has
now recovered their vision and is back to at least baseline at one
year post treatment.
As previously reported, the degree of efficacy observed varies
between patients, with the latest mean results set out in the
tables below. The first table shows the latest data including
the above-mentioned patient who has recovered to at least the
baseline level of vision in the treated eye. The second table
shows the latest data excluding both of the patients who originally
experienced surgical complications, to provide a comparison against
the data previously announced on February
24, 2020.
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
|
+7.9
letters (n=9)
|
+0.2 (n=9)
|
+7.7
(n=9)
|
2
|
+8.0
letters (n=9)
|
+1.2 (n=9)
|
+6.8
(n=9)
|
3
|
+10.8 letters
(n=9)
|
+4.4 (n=9)
|
+6.4
(n=9)
|
6
|
+9.6
letters (n=9)
|
+3.4 (n=9)
|
+6.2
(n=9)
|
9
|
+7.1
letters (n=8)
|
+1.2 (n=8)
|
+5.9
(n=8)
|
12
|
+11.9 letters
(n=5)
|
+4.3 (n=5)
|
+7.6
(n=5)
|
18
|
+17.0 letters
(n=1)
|
+1.0 (n=1)
|
+16.0
(n=1)
|
* Excluding one patient who experienced surgical
complications
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 (n=8)
|
+11.1
(n=8)
|
2
|
+10.8
letters (n=8)
|
+1.6 (n=8)
|
+9.2
(n=8)
|
3
|
+14.0
letters (n=8)
|
+5.1 (n=8)
|
+8.9
(n=8)
|
6
|
+12.3
letters (n=8)
|
+3.4 (n=8)
|
+8.9
(n=8)
|
9
|
+11.8
letters (n=7)
|
+1.2 (n=7)
|
+10.6
(n=7)
|
12
|
+13.4
letters (n=4)
|
+4.6 (n=4)
|
+8.8
(n=4)
|
18
|
+17.0
letters (n=1)
|
+1.0 (n=1)
|
+16.0
(n=1)
|
* Excluding both patients who originally experienced surgical
complications
The equivalent data set announced by the Company on February 24, 2020 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
|
+11.4 letters
(n=8)
|
+0.3 (n=8)
|
+11.1
(n=8)
|
2
|
+10.8 letters
(n=8)
|
+1.6 (n=8)
|
+9.2
(n=8)
|
3
|
+14.0 letters
(n=8)
|
+5.1 (n=8)
|
+8.9
(n=8)
|
6
|
+15.7 letters
(n=6)
|
+6.5 (n=6)
|
+9.2
(n=6)
|
9
|
+16.5 letters
(n=4)
|
+6.0 (n=4)
|
+10.5
(n=4)
|
12
|
+14.3 letters
(n=3)
|
+7.0 (n=3)
|
+7.3
(n=3)
|
* Excluding both patients who originally experienced surgical
complications
On June 17, 2020, the Company
announced that it had received regulatory approval from both the
U.S. Food and Drug Administration (FDA) and Medicines and
Healthcare products Regulatory Agency (MHRA) to expand the ongoing
Phase 2a clinical study to treat patients with RP at a higher dose
level. These approvals will enable the treatment of up to a
further nine patients in the Phase 2a extension segment of the
study (beyond the ten Phase 2a patients already treated). The
Company expects to commence treating patients shortly in both the
U.S. and the U.K. under the revised approved study protocol,
subject to a continued easing of COVID-19 related restrictions at
the relevant clinical sites.
On this basis, and as announced on June
17, the Company expects to present further data from the
expanded Phase 2a clinical trial during the next twelve months and
expects to have sufficient data from the study to enable it to seek
approval in the second half of 2021 to commence a single pivotal
clinical study with its hRPC cell therapy candidate in RP.
"The latest longer-term follow-up data from our Phase 2a study
of our hRPC cell therapy candidate in patients with RP continue to
demonstrate a meaningful clinical benefit at all time-points,
including in the first patient who has reached 18 months
follow-up," commented Olav Hellebø, Chief Executive Officer of
ReNeuron. "The persistence of the observed clinical benefit is
particularly encouraging. We look forward to announcing
further data from the study in due course, as a prelude to taking
our hRPC cell therapy into a pivotal, randomized study."
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 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 treat
diseases of the brain. The Company also has the ability
through its conditionally immortalised induced pluripotent stem
cell (iPSC) platform to make any tissue cells of choice; in-house
programmes are focused on treatments for blood cancers and
diabetes. 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