….there have been no Treatment Emergent Serious Adverse Events...

….there have been no Treatment Emergent Serious Adverse Events in any patient who has received VP-001 to date including those patients who have now received 3 doses of the highest dose of VP-001 administered

The first patient in this Phase 1/2 trial was dosed 16 months ago.

By now, by my calculations, there have been 12 patients dosed at various levels in the Single Ascending Dose (SAD) part of the trial and 3 patients dosed at 30mcg in the Multiple Ascending Dose (MAD) part of the trial. Two patients have already had 3 x 75mcg doses administered and 3 patients have received 2 x 30mcg doses. Each dose is administered by intravitreal injection.

I thought I’d provide some comparison by way of of safety data from three other successful Phase 1/2 trials in inherited retinal diseases, two of which were in gene therapies and the other testing an antisense therapy.

In the Beacon Therapeutics Phase 1/2 gene therapy trial in X-linked RP, at the 12 month point, of the 14 patients dosed at various levels, Serious Adverse Events had been reported in two patients – one was glaucoma related to the corticosteroid required by the gene therapy protocol and the other was visual impairment, caused by the subretinal injection procedure used in gene therapy administration. In addition, there were three cases of vitritis, one of eye pain, one of metamorphosia, one of photophosia and one of reduced visual acuity.

In the MeiraGTx Phase 1/2 gene therapy trial, also in X-linked RP, of 32 patients dosed, three had severe inflammation, seven had moderate inflammation and 24 mild. There was one case of retinal detachment, one of panuveitis and one of increased intraocular pressure.

ProQR conducted a Phase 1/2 trial of sepofarsen in leber congenital amaurosis (LCA). Like VP-001, sepofarsen was an antisense therapy administered by intravitreal injection. At the 12 month mark, of the 11 patients treated, there had been eight cases of cataract (6 requiring surgery) and two cases of retinal thinning.

Notably, in all three of these Phase 1/2 trials, treatment was considered to be “well tolerated”, with the risk/benefit profile considered to be sufficiently favourable to continue to a registrational trial.

In comparison, VP-001's safety profile to date is impeccable!