dm+d

Unassigned

New Medicines

Retinitis pigmentosa

Information

New molecular entity
ReNeuron
ReNeuron

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Phase II Clinical Trials
Yes
Yes
Oct 21ReNeuron plans a PIII trial for Retinitis pigmentosa by end of 2022 [4].
May 15FDA grants fast track status [4].
Aug 13Has orphan drug status in the EU and US for the treatment of retinitis pigmentosa [4].

Category

A human retinal progenitor cell (hRPC) stem cell therapy. The hRPC when transplanted into the retina has the potential to preserve existing photoreceptors, by reducing or halting further deterioration of vision. The progenitor cells mature into functional photoreceptors that engraft into the photoreceptor layer, bringing the possibility of restored vision.
The prevalence of RP is estimated at ~1 in 5,000 individuals. XLRP accounts for 6-10% of cases [1,2]. This means ~ 1000 people in England are likely to affected at on time but not all will be eligible for this.
Retinitis pigmentosa
Subconjunctival
Parenteral

Trial or other data

May 22In up to 12 month follow up data from the PIIa part of the PI/IIa (NCT02464436) trial, administration of ReN 003 in patients with retinitis pigmentosa continued to demonstrate meaningful clinical effect at all time points. The mean improvements from baseline in visual acuity in the treated eye ranged from +11.4 letters (n = 8), +10.8 letters (n = 8), +14 letters (n = 8), +15.7 letters (n = 6), 16.5 letters (n = 4) and +14.3 letters (n = 3) over the period of 1, 2, 3, 6,9 and 12 months post-treatment. In comparison, the mean change in the visual acuity in untreated eye ranged from +0.3 letters (n = 8; difference from treated = +11.1 letter), +1.6 letters (n = 8; difference from treated = +9.2 letter), +5.1 letters (n = 8; difference from treated = +8.9 letter), +6.5 letters (n = 6; difference from treated = +9.2 letter), +6 letters (n = 4; difference from treated = +10.5 letter) and +7 letters (n = 3; difference from treated = +7.3 letter) over the period of 1, 2, 3, 6,9 and 12 months [4].
Jan 22PI/IIa trial (NCT02464436) is no longer recruiting, having enrolled 29 adults. Collection of primary outcome safety data is expected to complete Jun 22 [3].
Dec 15PI/IIa trial to investigate the safety and tolerability of subretinally transplanted ReN 003, in patients with retinitis pigmentosa starts (RN03-CP-0001; NCT02464436). The open-label, prospective trial intends to enrol 33 patients in the US, the UK (at the Oxford Eye Hospital) and Spain. Participants will undergo vitrectomy surgery and subretinal implantation of hRPC in the study eye [3].