The company's original tertiary target for the SCiStar study, after observing the safety and activity of AST - OPC1, was that 45 - 50 % of study subjects would recover at least 2 motor levels
on at least one side at 12 months, which is approximately double the rate of
spontaneous recovery expected based
on a similarly matched population and published literature.
The improvement of visual acuity in sham - treated eyes was unexpected based
on the natural history of LHON, for which partial
spontaneous recovery is reported in only 8 to 22 % of patients with the G11778 ND4 mutation (Lam et al. 2014, Riordan - Eva et al. 1995).