Replies to post #154671 on Biotech Values

[mcbio]

I don't think of much of Martin's company. I'd only consider them if I see data. There is a lot I consider risky at this stage so I guess one could call it boom or bust I think its pretty speculative. I interacted a bit on twitter with Martin just playing devils advocate on their lead indication. I did a pubmed search didn't find much besides rat data (but I may not have searched well). Also saw that he helped design their MD molecule. I thought he was a finance guy makes me wonder more.

I'm just intrigued off the bat that we're talking about a $30M market cap for a company involved in the orphan space. Presumably once you see the data, provided it's good, the stock should already be a lot higher given that paltry valuation. Of course, we have no clinical data on this drug (there is data for other drugs with similar mechanism as it relates to proteinuria reduction) so who knows if it will work.

I caught the Twitter exchange as a bunch of people, not just you, had direct questions for him but I thought he was able to answer the concerns pretty well. Not sure if you caught the presentation on Retrophin's Web site at http://www.retrophin.com/ . There's a link in the middle of the page entitled "Corporate Presentation" that you can click on that goes through the rationale for the lead drug. It talks about the basis for how proteinuria reduction can help against FSGS (I know this seems to be a controversy and obviously still needs to be proven in the clinic). Also shows on slide 13 how ARBs themselves reduce proteinuria by ~45% in FSGS. ERAs (sitaxsentan specifically) reduce proteinuria by ~35% but there are apparently safety issues with the ERAs because they're not selective for ET-A only (hit ET-B also, which is an issue). So, the rationale for RTRX's RE-021 is that it's both an ARB and ERA but it's selective for ET-A so in theory it will combine the ~45% proteinuria reduction capability of an ARB with the ~35% proteinuria reduction capability of an ERA (but without the safety issues) for a much greater proteinuria reduction than either type agent alone. The key, as someone noted on Twitter, will presumably be if this combined mechanism will provide sufficiently greater benefit than that achieved by generic ARBs alone. At this paltry market cap, I'm willing to take the gamble given that, as you note, there are other drugs in RTRX's pipeline. Obviously there is dilution-risk here given the tiny valuation and the fact they may need to raise funds soon.