Hiding as usual.
Who is this jerk. Somebody hit him.
For years, almost all attention surrounding Sarepta Therapeutics has been on its drug for the most common gene mutation that causes Duchenne muscular dystrophy, called eteplirsen. This week, the company quietly disclosed plans to start late-stage trials of two further drugs for other genetic causes of DMD.
Together, the three experimental drugs would treat about 30 percent of the total worldwide patients with the rare disease. That’s more than double the number that would be treated by eteplirsen, which is now being considered for approval in the U.S.
“We’re really filling out our pipeline,” said Ian Estepan, a spokesman for Cambridge-based Sarepta (Nasdaq: SRPT).
The company’s goal in the long run has always been to develop drugs to treat as many different mutations as possible that cause the disease, which degenerates muscles to the extent that patients are forced to use wheelchairs by the age of 12. Eteplirsen is aimed at boys with a mutation in a part of the DNA called exon 51. That’s the most common one, responsible for about 13-15 percent of patients with the disease, and is also the target population of a competing drug developed by California-based BioMarin Pharmaceuticals (Nasdaq: BMRN). Both drugs will likely be reviewed by an FDA advisory committee later this year.
I am excited. It is within 10% of the 52-week high and will double before the end of the year. That is exciting.
It will also be a new all time high.
That is old news, is that really all you've got?
The FDA would have told them that up front if that was an issue (in fact, they did, Ana well as how to fix it with the additional studies that are already underway). Plus, the 12, as a percentage of the target patient population, is still within the zone, as a meaningful percentage of affected patients. The number of patients is small, so the number of trial subjects is small.
Still, since you can't attack the safety profile or efficacy, I guess the numbers game is your only hope.
And that isn't much, since it already passed the FDA.
When the FDA accepts the NDA SRPT will pop over $50 and upon final approval it will be over $100/share (next year by this time).
So go buy BMRN. Why even be here? Are you also posting on the PFE board about how great BMRN is?
If it is an acquisition, contingent on the approval of Eteplirsen, what value would the buyout be? The valuation would need to include the potential full valuation of Etwplirsen as well as the conditional value of the drugs in the pipeline, coupled with a reasonable P/E based in projected sales. That really drives to a net present value (risk adjusted) formula that lands us anywhere from $150/share to $300/share based on the probability of approval for each drug, reasonable market penetration, and total available market. Considering that Eteplirsen likely will only target the 13% of DMD patients with the specific mutation, Eteplirsen could easily exceed $500M/year in sales, which drives to a market capitalization valuation of about $5B without even considering the other drugs.
Also, why the buyout once they have cleared all the regulatory hurdles? That's like setting up a deal to sell my winning lottery ticket conditional on it winning, for a discount, before it has won. Once I've won, I don't need anybody to buy it, I've already won.
Just like we can sense it when a major storm is about to hit, I feel that something major (good) is about to happen with SRPT. We will see if I'm right or not, but I can feel it. It is going to be big and very soon (this month - so during July).
I have absolutely NOTHING to back this up with, no links, no web pages, no analyst comments, nothing. Just a pure gut feel.
These kinds of deals show the potential value of Biotechs, marjket buyout potential, and what a lucrative investment these can be. Just 2-weeks ago JUNO was at $60, got crushed down to $45 and then in 1-day was back over $60 again.
Many shorts piled on during the downturn, and they will get wiped out today.
At first I thought it was just that he had full faith in ISIS, just was tired of saying the same thing over and over and didn't want to be hyping a stock - even though he knew the future was outstanding and would be for a very long time.
Now I'm starting to wonder if his handlers told him it was going to be out of favor for a bit and they would take it down 20-30% so stop being so strong on it. Now, in view of the drop (for no apparent reason) it looks fairly suspicious.
$40/share on a buyout, surely you jest. Try $80-$120/share. We hit $55/share on the efficacy of Eteplirsen without a buyout and long before NDA or FDA Approval. Buyout would be much higher because the amount of uncertainty is greatly reduced.
Not sure what is about to come, but I feel it like a storm about to hit. I believe it will be all good news too.
No, actually,mist doesn't have to be announced until the board has voted on it.
It does seem to have made you clench up fast. LOL.
Romuluss - nobody likes you. You aren't going to convince anybody of anything because you are such a jerk. You really should leave the board.
I what Sarepta's springboard plans are after Eteplirsen is approved and to market. Once they have proven their exon skipping approach, they can expand it to additional/adjacent markets.
Please vote thumbs up / down as you think for AA.