| A | B | C | |
|---|---|---|---|
| 1 | Main | ||
| 2 | Brand | TAK-999, ARO-AAT | |
| 3 | Generic | fazirsiran | |
| 4 | Indication | alpha-1 antitrypsin deficiency (AATD) | |
| 5 | Competition | NTLA IND in 2H23 | |
| 6 | Economics | Takeda licensed for $300m upfront, US 50-50 and 20-25% Ex-US royalties | |
| 7 | Clinical Trials | ||
| 8 | |||
| 9 | |||
| 10 | Phase III TAK-999-3001 n=160 AATD liver | ||
| 11 | PE: METAVIR stage at Week 106 | ||
| 12 | |||
| 13 | Phase II "SEQUOIA" | ||
| 14 | Week 48 reductions of serum Z-AAT of 74%, 89% and 94% 25mg, 100mg, 200mg vs 0% for placebo | ||
| 15 | 50% achieved 1 point in METAVIR, but 38% also improved in placebo?? |