IV program - Length of stay of HE patients important more than 60% in for > 5 days, hospitals make money by day 4. - Hyperion Phase 2 ammonia scavenger (Ravicti - glycerol phenylbutyrate) stat sig at reducing future episodes, hospitalizations and severity. Good harbinger since contains same active ingredient. (Horizon decided to terminate program because of use in urea cycle disorder - much higher price) - 80% power to see difference at study end that was seen at planned interim. Primary is time to meaningful clinical improvement in encephalopathy symptoms (Present as K-M curve with log rank analysis). - If slide 18 accurately represents the powering then it looks like benefit is close to 40 hours! - stratifying enrolled patients for West Haven Scale at entry, most are 2 or 3 expect very few 4's - Standard of care may vary a little bit but not restricted in what is used.
Oral program - Now deciding if moving forward with 1 or 2 (that look most promising of the 3) of the oral candidates to optimize and move forward with. - Feel confidant have a BID formulation and possibly QD going forward - Optimize formulation and repeat (phase 1) middle of next year.
- See IV as $500-600M opportunity, Oral as $800-900M opportunity (US only) - COM patent 2030
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