Astellas Pharma Inc. / Ocata Therapeutics, Inc.

Stargardt's macular dystrophy and dry age-related macular degeneration (AMD)

Disease Overview

Stargardt's disease is an inherited juvenile-onset macular dystrophy caused largely by ABCA4 mutations, leading to progressive central vision loss in adolescence and young adulthood. Dry AMD is the leading cause of irreversible vision loss in older Americans, with no approved disease-modifying therapy at deal time - both conditions destroy retinal pigment epithelium, the cell type Ocata's lead programs were designed to replace.

Competitive Landscape

In late 2015 the regenerative ophthalmology field was thin and high-risk. Ocata (formerly Advanced Cell Technology) was widely viewed as the most clinically advanced human embryonic stem cell-derived RPE program globally, with Phase 1/2 data published in The Lancet (Schwartz et al., 2015) for both Stargardt and dry AMD. Direct competitors included the London Project to Cure Blindness (Pfizer-supported, hESC-RPE patch, later spun into Athersys/UK academic groups) and Cell Cure Neurosciences (BioTime/Lineage, OpRegen, suspension RPE). For dry AMD broadly, no approved therapy existed - Roche/Genentech's lampalizumab and Apellis's pegcetacoplan were still in development. Astellas paid a 79% premium ($8.50/share) to gain a leading position in cell therapy and an ophthalmology franchise to offset upcoming VESIcare and Tarceva LOE. What changed: Ocata's hESC platform gave Astellas a credible regenerative-medicine flagship at a time when Japanese pharma was being pushed by METI to lead in iPSC/hESC therapeutics. The deal struggled at first - only 36% of shares were tendered by the initial deadline, with shareholders arguing for a higher price - and ultimately closed February 2016 at $379M. The Stargardt and dry AMD RPE programs did not yield an approved Astellas product in the decade following.