famzeretcel (human retinal progenitor cell therapy) / jCyte - LARVOL DELTA

Safety of a Single, Intravitreal Injection of 8.8M jCell (Famzeretcel) in Retinitis Pigmentosa (RP) (clinicaltrials.gov) - P2 | N=60 | Recruiting | Sponsor: jCyte, Inc

New P2 trial • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Immune Responses to Sequential Binocular Transplantation of Allogeneic Retinal Progenitor Cells to the Vitreous Cavity in Mice. (PubMed, Int J Mol Sci) - "Intravitreal transplantation of allogeneic human retinal progenitor cells (hRPCs) holds promise as a treatment for blinding retinal degenerations...Animals treated with repeat doses of gmRPCs did not show evidence of sensitization, nor was there immune-mediated destruction of the grafts. Despite the absence of immunosuppressive treatments, allogeneic gmRPC grafts survived following repeat dosing, thus providing support for the preliminary observation that repeated injection of allogeneic RPCs to the vitreous cavity is tolerated in patients with retinitis pigmentosa."

Journal • Preclinical • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • Transplant Rejection • Transplantation

Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (clinicaltrials.gov) - P2 | N=30 | Completed | Sponsor: jCyte, Inc | Active, not recruiting ➔ Completed

Trial completion • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (clinicaltrials.gov) - P2; N=30; Active, not recruiting; Sponsor: jCyte, Inc; Not yet recruiting ➔ Active, not recruiting

Clinical • Enrollment closed • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa (clinicaltrials.gov) - P2; N=84; Completed; Sponsor: jCyte, Inc; Active, not recruiting ➔ Completed; Trial completion date: Mar 2021 ➔ Nov 2020

Clinical • Trial completion • Trial completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (clinicaltrials.gov) - P2; N=30; Not yet recruiting; Sponsor: jCyte, Inc

Clinical • New P2 trial • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa (clinicaltrials.gov) - P2; N=84; Active, not recruiting; Sponsor: jCyte, Inc; Trial primary completion date: Sep 2020 ➔ Dec 2020

Clinical • Trial primary completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Intravitreal Injection of Allogeneic Human Retinal Progenitor Cells for the Treatment of Retinitis Pigmentosa: A Prospective Randomized Controlled Phase 2b Trial (RS-LONDON 2020) - "Intravitreal injection of hRPC is a novel approach for treatment of RP, and is independent of the genetic subtype. This phase 2b study demonstrates encouraging biological activity and a good safety profile, warranting progression to phase 3 trial."

"jCyte Inc. Announces Promising Phase 2b Results of jCell Therapy in Retinitis Pigmentosa https://t.co/P6bBNKbNi7" (@NewsFromBW)

Gene Therapies • Genetic Disorders • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

ReNeuron progressing blindness candidate through phase I/II trial (London South East) - "Initial short-term safety and tolerability data from the Phase I part of the study...are expected in early 2017...efficacy read-outs from the Phase II part of the study in a further six patients, are expected in the second half of 2017...it expects to file an application in late 2017 or early 2018 to commence a Phase II/III trial."

Anticipated new P3 trial • Anticipated P1 data • Anticipated P2 data • Age-related Macular Degeneration • Ophthalmology

Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa (clinicaltrials.gov) - P2; N=84; Active, not recruiting; Sponsor: jCyte, Inc; Trial primary completion date: Aug 2019 ➔ Sep 2020

Clinical • Trial primary completion date

ReNeuron presents positive clinical data at the American Academy of Ophthalmology Annual Meeting (Business Insider) - P1/2, N=21; NCT02464436; Sponsor: ReNeuron Limited; "From a safety perspective, according to Dr. Dugel, the clinical trial data continue to show a good overall safety profile for the hRPC therapy, with no immune or cell-related adverse events reported. The isolated episodes of surgically related adverse events are consistent with those expected for sub-retinal injection procedures, some of which may be mitigated in the future with technique refinements and injection site selection."

Media quote • P1/2 data

ReNeuron primed for further progress (Proactiveinvestors) - P2, N=NA; "The company has announced positive Phase II data from patients treated with its neural stem cells, with a Phase IIb trial imminent...A Phase I/II assessment of a very small group of sufferers of a blindness-causing disease called retinitis pigmentosa saw a significant improvement in vision after treatment...We remain extremely encouraged by the positive efficacy data we have seen thus far..."

P2 data

Preliminary results for the year ended 31 March 2019 (ReNeuron Press Release) - "ReNeuron Group plc...announce its preliminary results for the year ended 31 March 2019...hRPC stem cell therapy candidate for retinal disease: Top line results from all treated Phase 2a patients to be presented at the American Academy of Ophthalmology Annual Meeting in October 2019...Discussions ongoing with other commercial third parties regarding potential out-licence deals"

Licensing / partnership • P2a data

ReNeuron provides business update and results notification (PRNewswire) - "...the Company reported positive preliminary data in the first cohort of the Phase 2a part of its study of hRPC in RP...The Company expects to treat the final six patients of the study this summer and to report data from all 12 of the Phase 2a subjects in the second half of this calendar year. These results will form the basis of the Company's interactions with the European and US regulatory authorities regarding the remaining clinical development path of hRPC for the treatment of RP."

P2a data • Trial status

ReNeuron announces presentation of updated efficacy and safety data from retinitis pigmentosa study at the Sixth Annual Retinal Cell and Gene Therapy Innovation Summit (PRNewswire) - "ReNeuron Group plc...announced updated positive preliminary results 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). All three subjects in the first cohort of the Phase 2a part of the trial have demonstrated a sustained and further improvement in vision compared with their pre-treatment baseline."

P1/2 data

ReNeuron shares up 30% after it strikes deal worth up to £80mln with Chinese Pharma (Proactiveinvestors) - "Shares in ReNeuron Group...rose 30% after it inked a deal potentially worth up to £80mln in success-based payments with an arm of one of China's leading drug companies...Fosun Pharma is licensing the rights to the company’s CTX and hRPC cell therapy programmes in the People’s Republic for an initial £6mln."

Licensing / partnership • Stock price

ReNeuron partners with Fosun Pharma in China (ReNeuron Press Release) - "ReNeuron...is pleased to announce that it has signed an exclusive licence agreement...with Shanghai Fosun Pharmaceutical Industrial Development Co., Ltd., a subsidiary of Shanghai Fosun Pharmaceutical...for the development, manufacture and commercialisation of ReNeuron’s CTX and hRPC cell therapy programmes in the People’s Republic of China..."

Licensing / partnership

Clinical update and conference presentation - "ReNeuron...is pleased to announce a further update on progress with the ongoing Phase 1/2 clinical trial in the US of its hRPC cell therapy candidate in the blindness-causing disease, retinitis pigmentosa...The Company is pleased to confirm that the positive efficacy seen and previously announced has, to date, been sustained in the first patient cohort in the Phase 2 part of the study...Further read-outs from the patients treated in the Phase 1/2 study will be presented at the 6th Annual Retinal Cell and Gene Therapy Innovation Summit, taking place in Vancouver, Canada, on 26 April 2019."

P1/2 data • Trial status