Replies to post #192465 on Biotech Values

[mcbio]

FGEN cc notes:

Thanks for the nice summary. You saved me some time. ; )

1) Bundle or not: CEO said they would be focusing on things to make it clear it is a different drug- the inflammatory population (for which EPO works poorly). (My comment is that I am puzzled by why this is even an issue since oral only drugs are excluded from the bundle until 2024 "Payment for Oral-only Drugs under the ESRD PPS: Section 217(a)(1) of PAMA amended section 632(b)(1) of the American Taxpayer Relief Act of 2012, which now provides that the Secretary “may not implement the policy under section 413.174(f)(6) of title 42, Code of Federal Regulations (relating to oral-only ESRD-related drugs in the ESRD prospective payment system), prior to January 1, 2024.” Accordingly, CMS finalized that payment for ESRD-related oral-only drugs will not be made under the ESRD PPS prior to January 1, 2024.". See www.cms.gov/Newsroom/MediaReleaseDatabase/Fact-sheets/2014-Fact-sheets-items/2014-10-31-3.html )

Interesting. Perhaps a lot of the discussion has just been with regards to what will happen after 2024, since that's just 5 years or so after they expect roxa on the market (if all goes well).

2) CEO noted that the expected cost in the bundle (if they have to go that route - see above) for their drug would be at least $3k to $4k per patient per year.

Any thoughts on what this would translate into in terms of revenue for roxa if this is how it plays out?

3) Other indications that they plan to pursue are inflammatory anemias (e.g. Lupus Nephritis, Inflammatory Bowel Disease, Rheumatoid Arthritis, Myelodisplastic syndrome)

I wonder why they aren't already running trials in these specific additional indications. And I don't think a specific timeline was mentioned for start of these specific additional trials. They did mention that few LN patients get on dialysis because of inflammation. I think they also said they think they can address 30-40% of pre-diabetic patients not currently served.

4) They said the DSMB for the anemia trials (my comment: from previous digging is there is one DSMB for all the anemia trials) reviews data every 3 months (my comment: somewhat more often than typical), and meets face to face about every 6 months. Last meeting was April 22.

FWIW, I actually had in my notes that last meeting was 4/26, not 4/22. Will double-check that. Important because I may time my next add until after this next planned meeting. I'm assuming if there was a negative issue, we would likely hear about that in a PR the following morning or after the close.

5) As has been mentioned on this board a few days ago, he explained that they chose to do dosing only every few days because: A) they believe that the initial turn-on of HIF is "cytoprotective" (and noted that there are papers on this topic), B) they have seen tachyphalaxis in this class of drug, C) They believe that having HIF on too long generates toxicity. (My comment - other than the tachyphlaxis data this mostly sounded theoretical/speculative).

I took this whole discussion as quite a negative on the AKBA drug FWIW.

7) First filing in DMD is expected to be this month. First trial will be in wheelchair kids. Second will be ambulatory.

Interesting that they actually started their DMD work back in 2007 so have been looking at for some time. They noted they are open to looking at a variety of endpoints in the initial P2 trial.

[dewophile]

FGEN

Thanks for the notes! I've been holding off on a full position waiting for a bit more on safety checks, but the valuation is awfully tempting right now to add. Given your stats background and knowledge can you comment on how passing quarterly DSMB data reviews derisks the program. I assume the threshold for stopping if MACE and other adverse events trend in the wrong direction is far lower than the opposite (i.e stop for safety in favor of roxa) - but i really can't quantify this. If you had a good sense of this, as well as how far they are in enrollment, i think you can start to really semi quantify how liklihood of success changes over time in a setting like this were efficacy is a given.
Honestly a stoppage for safety and the stock totally tanking is the one thing stopping me from diving in whole heartedly, but i see that possibility shrinking over time
I also think now that i am enlightened by Peter as to the high event rate in incident dialysis there is a higher risk of this type of a trial being stopped in favor of roxa if it is indeed safer/more effective, nad it suck not to have a full position if this were to occur, however low thsi might be
TIA
PS: next DSMB review should be end of the month if it is quarterly since at the last call march 26 it had recently just taken place per the call