ABC
1Main
2BrandTAK-999, ARO-AAT
3Genericfazirsiran
4Indicationalpha-1 antitrypsin deficiency (AATD)
5CompetitionNTLA IND in 2H23
6EconomicsTakeda licensed for $300m upfront, US 50-50 and 20-25% Ex-US royalties
7Clinical Trials
8
9
10Phase III TAK-999-3001 n=160 AATD liver
11PE: METAVIR stage at Week 106
12
13Phase II "SEQUOIA"
14Week 48 reductions of serum Z-AAT of 74%, 89% and 94% 25mg, 100mg, 200mg vs 0% for placebo
1550% achieved 1 point in METAVIR, but 38% also improved in placebo??