ReNeuron Group plc (RNUGF)

[georgejjl]

NEWS!!!

hRPC for retinal disease
During the period under review, and subsequent to it, we have made significant
progress advancing the clinical development of our human retinal progenitor cell
(hRPC) therapy candidate in the blindness-causing disease, retinitis pigmentosa
(RP). A Phase 1/2a open-label clinical trial is ongoing to evaluate the safety,
tolerability and preliminary efficacy of our hRPC stem cell therapy candidate in
patients with advanced RP. The Phase 2a element of the study, which uses a
cryopreserved hRPC formulation, enrols subjects with some remaining retinal
function and is being conducted at two clinical sites in the US: Massachusetts Eye
and Ear in Boston and Retinal Research Institute in Phoenix, Arizona.
In February 2019, we reported positive preliminary data in the first cohort of three
patients in the Phase 2a element of the study, with all three subjects in the cohort
demonstrating a rapid improvement in vision compared with their pre-treatment
baseline.
In April 2019, further data from the first patient cohort in the study were presented
at the sixth annual Retinal Cell and Gene Therapy Innovation Summit in Vancouver,
Canada, which preceded the 2019 annual meeting of the Association for Research
in Vision and Ophthalmology. In the presentation, it was reported that the first
cohort of patients in the Phase 2a element of the study had demonstrated a
sustained and further improvement in vision compared with baseline, with a mean
improvement from baseline in visual acuity of + 23 letters on the ETDRS eye chart
in the treated eye (the untreated control eyes did not show meaningful
improvement). An improvement of + 23 letters is equivalent to reading an
additional four lines of letters on the ETDRS eye chart, the standardised eye chart
used to measure visual acuity in clinical trials. An improvement of at least + 15
letters from baseline is considered to be clinically meaningful by the US Food and
Drug Administration (FDA), as stated in their recent guidance on gene therapy for
retinal disorders. In addition to these objective measurements, all three subjects
had also noted a subjective improvement in vision in their treated eye.
Dosing of the second cohort of three subjects in the Phase 2a element of the study
is complete and dosing of the remaining two cohorts is in progress. These later
cohorts comprise patients with a greater baseline level of visual acuity than those
patients earlier in the study, as we seek to assess preliminary efficacy in patient
groups with differing levels of remaining vision. The clinical protocol for the study
allows for up to 12 patients (four cohorts of three patients each) to be treated in
the Phase 2a element of the study.
We expect to treat the remaining patients in the study shortly and to report
preliminary data from all treated Phase 2a subjects in October at the American
Academy of Ophthalmology 2019 Annual Meeting in San Francisco. These results
will form the basis of our future interactions with the European and US regulatory
authorities regarding the future clinical development path of hRPC for the
treatment of RP. Our clinical programme in RP benefits from Orphan Drug
Designation in both Europe and the US, as well as Fast Track designation from the
US Food and Drug Administration (FDA).

http://4965zs3ha2l125fk78zkozo3.wpengine.netdna-cdn.com/wp-content/uploads/RENE-2019-preliminary-results-FINAL.pdf

Good luck and GOD bless,