Table 1 Development stage assumptions used for the Rett syndrome compound valuation
From: Financing repurposed drugs for rare diseases: a case study of Unravel Biosciences
(a)Â Probabilities of success, PoS | Lower bound | Base case | Upper bound | Prob. of arriving to phase |
|---|---|---|---|---|
Discovery, preclinical development, and phase 1 | 62% | 67% | 72% | 100% |
Phase 2 | 43% | 53% | 63% | 67% |
Phase 3 | 59% | 69% | 79% | 36% |
FDA submission-to-launch | 94% | 94% | 94% | 25% |
Total cumulative probability of success (PoS) | 15% | 23% | 34% | – |
(b)Â Time for development (years) | Lower bound | Base case | Upper bound | |
|---|---|---|---|---|
Discovery, preclinical development, and phase 1 | 1 | 1 | 1 | |
Phase 2 | 1 | 2 | 3 | |
Phase 3 | 1 | 2 | 3 | |
FDA submission-to-launch | 1 | 1 | 1 | |
Total | 4 | 6 | 8 | |
(c)Â Cost of development ($, in millions) | Lower bound | Base case | Upper bound | |
|---|---|---|---|---|
Discovery, preclinical development, and phase 1 | 1 | 4 | 7 | |
Phase 2 | 9 | 14 | 40 | |
Phase 3 | 22 | 35 | 99 | |
FDA submission-to-launch | 37 | 37 | 37 | |
Total | 69 | 90 | 183 | |