Replies to post #184194 on Biotech Values

[iwfal]

RNA / BMRN:

Did anyone else (besides me) find it interesting that BMRN acquired all of RNA rather then work out some sort of licensing deal? From the call it was pretty clear that BMRN's interest at this time was only in acquiring Drisapersen.

As I noted on twitter the size of the deal for a non-competitive technology (inherent toxicity) implies to me that:

1) this is as much a play on SRPT's drug not being approved near term as RNA's drug receiving approval - since SRPT would clearly get 90 percent of the market with lesser AE (and fact that patient parents don't care about uncertainty of proof)

2) doing some kind of deal with Sarepta is contemplated. Patents obviously. But also may involve data sharing etc.. Otherwise the RNA product has a significant risk of limited life, and it would be hard to make return on the $ spent on the acquisition in 3 or 4 years

[jq1234]

Not really. The net cash deal is about $600m excluding RNA cash position, plus two $80m CVRs for near term approval. The risk isn't as high as everyone else thinks. I still think most people under-estimated RNA IP value. Forget about US for a moment, Drisapersen is highly likely getting approved based on Ataluren precedence at EMA, thus leave EU market to Drisapersen for quite some time due to IP. You should know EU market is as large if not larger than US in these ultra orphan genetic disorder indications.

As of the deal is focused on Drisapersen only, that is what BMRN has to say, it is in short to intermediate terms, but not entirely. Remember, FDA requested two confirmatory trials, one is Drisapersen most likely against natural history, the other one is randomized controlled trial from a different exon, thus regardless of what they say about the deal is, they'd bring another exon into ph3 regardless of whether FDA grants AA.

Overall, this is just slightly higher than what I thought SRPT would need to pay for IP deal with RNA. By the way, the tox isn't really an issue once you get to see the very large dataset. You could say it is as well tolerated as ERTs if not better.

[DewDiligence]

BMRN—Did anyone else (besides me) find it interesting that BMRN acquired all of RNA rather then work out some sort of licensing deal?

Inasmuch as RNA is domiciled in the Netherlands, there may be tax benefits to owning RNA outright.