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Sarepta Therapeutics, Inc. Message Board

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  • belco_mc belco_mc Oct 19, 2012 7:05 PM Flag

    The New Investing Reality

    Here are some facts for you:

    1. we don't know if eteplirsen works longer term. we know it provides an initial clinical benefit, but do not yet know if it will sustain over time.

    2. we don't know if it will get AA. if not, we don't know the size or cost of a phase III. By the way, SRPT doesn't have the money to fund a phase III at this time.

    3. We don't know what we can sell the drug for- there are a lot of estimates on revenue but they vary widely.

    4. We don't know the manufacturing costs. Since we don't know the sales price either, we don't know the profitability of this drug.

    5. Eteplirsen helps the symptoms of DMD, but doesn't cure it. If we are entering the golden age of muscular dystrophy advancement, then there is a chance some other company finds a cure leaving eteplirsen worthless.

    I could go on and on. The point is that there is substantial risk in the drug. I've never shorted it and only played it from the long side. I've made a lot of money in it. I have followed it a long time and have a family member with DMD, so I know how brutal this disease really is. Longer term, I think SRPT goes a lot higher. But risks remain and it will remain a volatile stock. I think the CEO has done an excellent job and am a fan for this company.

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    • 1. we don't know if eteplirsen works longer term. we know it provides an initial clinical benefit, but do not yet know if it will sustain over time.

      Evidence thus far shows that the benefit is increasing over time. While we don't know for sure if this trend will continue, how in the hell would the possibility of a future drop off in benefit, which is a risk with all drugs, cause a rapid drop off in the share price right now? That makes no sense. Why would that concern all of a sudden become a major driving factor in the value of this company?

      2. we don't know if it will get AA. if not, we don't know the size or cost of a phase III. By the way, SRPT doesn't have the money to fund a phase III at this time.

      Just the possibility of accelerated approval should have this much higher. If no AA, it would seem that the company could land a partner to pay for a Phase III if it's needed. We have seen hundreds of companies, including Prosensa, land partners with much lass compelling data, and a much higher risk profile from questionable safety. Hard to believe anyone is betting their hard-earned money that neither of these things will happen: AA or partnership with big upfront payment.

      3. We don't know what we can sell the drug for- there are a lot of estimates on revenue but they vary widely.

      Estimates vary widely, but none of them are below what I would consider a great chance to make a good profit.

      4. We don't know the manufacturing costs. Since we don't know the sales price either, we don't know the profitability of this drug.

      The company will price this accordingly. Rest assured, if approved they won't be losing money on this drug. There is no alternative on the horizon.

      5. Eteplirsen helps the symptoms of DMD, but doesn't cure it. If we are entering the golden age of muscular dystrophy advancement, then there is a chance some other company finds a cure leaving eteplirsen worthless.

      It is thought that this drug, if given early on in life, can prevent symptoms from manifesting for many years. When they do show, they would likely be much more mild. Current evidence shows the drug stopping the disease progression.
      Any new therapy will be held to a much higher standard. They would have to beat Sarepta on safety (nearly impossible, almost every drug, unlike Eteplirsen, has safety issues), efficacy (possible, but by how much? If it's only a small amount does this outweigh potential harmful side-effects?), and price (by then, our manufacturing will be much cheaper, we will be able to lower price if needed to compete with newer drugs). In a hypothetical, if there is a new "cure" 5-10 years from now and it produces kidney damage or raises cancer risks, FDA will be faced with a tradeoff of eliminating a much more mild muscular dystrophy or possibly causing serious side effects in the boys. Any new company entering this space would have a much higher bar. Even if they passed the FDA with their cure, families would have to decide whether potential risks outweighed the potential benefits.

      Yes, there are risks and I appreciate your input, I just don't see any of the above-mentioned possibilities as being a reason someone would bet against this company...

      Sentiment: Strong Buy

    • 1. We don't know if it works long term, true. But we DO know that it works better than anything else that's ever existed before. Nothing has ever made dystrophin, with no toxicity over the first year of administration, and caused statistically improvement in multiple aspects of the disease. So, we may not know it works long term. Heck, we don't know if it will make the boys funnier either. But, we DO know that it is showing unprecedented positive effects with no harm noted after 48 weeks, and there is nothing else even close in 2012.

      2. AA? We may not know whether it will happen, but that facts indicate that it is very likely. The news about tox issues with competitors surely did not hurt the growing body of evidence that the SRPT chemistry is far superior either. Eteplirsen from what I read about AA, is almost the perfect type of drug that people were thinking about when they came up with the idea.

      3.We don't know exactly what we can sell the drug for. True. But, we do know that it is expensive to make the drug and that SRPT will be allowed to receive a significant profit above and beyond manufacturing costs. Beyond that, someone (Micro/Dr. Myst.) posted a piece in which they suggested that 100,00/ year / boy treated as a potential amount for manufacturing costs. If so, 300 - 400k/boy seems completely possible. Regardless, SRPT will show a handsome profit, when able to sell the drug. Sure.........we don't just exactly how handsome.

      4. Manufacturing costs for similar molecules and drugs are known, and/or readily approximated.

      5. By the time some other company finds, AND advances something that shows a better effect than Eteplirsen or something that is significantly cheaper and shows an equal effect, it will be many years downs the road, if ever, regardless of how wonderful it may or may not be. So, sure, some other drug may supplant Eteplirsen SOMEDAY..................but not until SRPT has made a lot of money.

      I agree we don't know the cost of phase 3, or the size, or the design. I agree that there isn't enough $ to go it alone, unless there is a cash raise. I remain convinced that if CG had intentions along that line, he would have raised the cash when the share price was high. He did not. Either he's an idiot, or he had some other plan in mind to get Eteplirsen over the finish line - either the ability to be paid while doing phase 3 due to AA designation or some form of partner/buyout, or both.

      Risks? Sure they exist. The problem I have is that SRPT was seriously de-risked with A) better than hoped for results and B) nearest competitors chemistry woes where they themselves currently have NONE. When a stock is derisked in a big way, the point I was making, is that it should then, all things being equal, be worth more. That for some reason does not seem to be the case lately here. Your arguments don't explain this phenomenon, or hold water from where I sit. But thanks for throwing them out there.

    • LOL. Now you are bashing belco, Funny how you have now turned. I knew you were a lightweight. take care

      • 2 Replies to zwerp2000
      • pdbrown,

        Thanks for your reply. I appreciate your response and each of your points are valid. I have mentioned that I have a family member with DMD and have seen what this disease does to the body. It's ruthless. It's a tough, tough disease to combat. Given how bad this disease is, I would be very surprised if we don't get AA. Making this drug available to boys with exon 51 problems is the right thing to do. There is no downside.

        My point is that volatility is here to stay until things get resolved. There are still many risks and people will bet against it. The stock, SRPT, is simply a call option on eteplirsen. There is no underlying value.

        I hope it all works out. It's time for the DMD community to have some hope placed on a real drug.

      • I'm not bashing, zwerpy. I'm just giving the 'other side of the trade' to some of the longs here that can't see it. There are real risks here. That's a fact. In the end, I'm betting with SRPT. And since name calling is fun, you're still one of the biggest idiots here. You've passed Starfe. I actually like starfe because he doesn't take himself too seriously. You, on the other hand, actually think you have something insightful to offer. That's the funny part.

    • Your statements are not FACTS. You are speculating on each and every point. Moreover, much of what you write is completely incorrect and I will not spent time telling you why, because you're not worth the time. In addition, what you just wrote pertains to just about every biotech at this stage of development, so you are essentially preaching to the choir on your bogus concerns. There is no drug that comes close to eteplirsen, as it pertains to helping kids with DMD. You are clueless and your statements are reckless. I'd like to see you send your post to one of the parents of a trial patient; they will likely smack you upside the head for being so naive and misinformed. There is a post by riskon4life that I thought hit the nail on the head regarding AA. I suggest you read it.

      Lastly, your post is a typical soft bashing attempt. You are an idiot in my opinion -- it's as simple as that.

      Sentiment: Strong Buy

    • BEST REPLY EVER

      Somebody should print your reply out and mail it to every investor as part of an education campaign.

      All investors, please read belco's reply

      Sentiment: Strong Sell

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