Re: eteplirsen COGS = $75k/year
I just listened to the jefferies call, and Chris said large scale production for treatment price could be in the range of $300-$500k/year with COGS at 15%-25%, making the cost of producing the drugs to be $75k/year (do the math of 25% at $300k and 15% at $500k and you get $75k. Chris also stated that this was before they even started to focus on improvements in mfg cost reductions.
USA market size for Eteplirsen he called out at 2k patients. TAM for USA is then $600M - $1B; if they reach 70% of TAM would see $420m - $700m revenue, with margins at 75%-85% they are maintaining the typical high margins of biotech industry. He said that once the other 3 exons come online they would double to tripe that figure. With IND's projected for 2014, would be 2016 at the earliest for this added kick depending upon how many phases they are required to go thru... am not anticipating FDA would grant platform approval without at least 1 more exon going thru the traditional phases of testing, but one never knows.
Once the above revenue stream is established, the market cap will be $4B-$14B - with $14B using the $500k treatment price and ALXN like valuation where there is rest of world sales upside (assume that they haven't addressed the GSK patent issue by then - if so, will only be higher).
Given the relative low cost of moving this to market, it's hard for me to imagine Chris sell the company for a few billion - maybe $11B like VRUS, but given the platform they have and the current pipeline and what their market cap can be within 2 years, they can become a $20-$50B company within the next 5-10 years (if not sooner, like 2015-16 if they are able to sell Etep world wide starting in 2014.
I've waited these past 7 years mostly underwater... finally back above, and with line of sight to revenue is a good feeling.