Biotech Values

[biomaven0]

Couple of comparisons with the FGEN data:

Looks like AKBA slightly under-dosed here, with only 55% of the subjects hitting their primary endpoint. Their trial started with average HGB of around 10. vs around 9. in most of the Fibrogen trials, which typically reached their (slightly different) endpoints in around 90% of patients for the doses they are now using. The HGB rise per week was clearly somewhat higher in the Fibrogen trials as well. (Basically you can increase pretty much as quickly as you reasonably want depending on dosing, so overall efficacy for either drug is not really at issue here).

So the AKBA drug (with average dose of 450mg/day) is clearly less potent (by around 13X) than roxa (with doses around 80mg [1.7 mg/kg] two three times a week), and yet they still underdosed some compared with roxa. That level of chronic dosing raises the tox risk (hepatic, renal) in a large trial. The daily dosing vs 2 or 3 per week also increases the tox risk. The actual SAEs they experienced could absolutely have been random fluctuations, but still scary for potential partners.

Note AKBA had some SAE's considered related aside from the deaths:

Three drug-related SAEs were abnormal liver function test, myocardial ischemia, and angioedema

In the Phase 1/IIs' to date roxa has been in some 1,400 patients, so they obviously have a much bigger safety database. (Plus whatever one can infer from their ongoing trials getting a continued "proceed" signal).

Peter