Biotech Values

[iwfal]

FGEN cc notes:

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 )

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.


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

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.

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).

6) The hope to be fully enrolled in the fibrosis ph2 by the end of this year (but too early to know for sure).

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