The newly published study where the KD on factor 7 reaching 2log using .5mg/KG is a bigger deal than many realizes.
I don't think ARWR will ever reach $100 billion, but $10 billion is possible. I think we will see a MC reverse between ARWR and ALNY in the next 2-3 years if all goes well.
Interesting you are bringing attention to a different ticker yourself. SRPT is a case study in biotech investing. They are about to file NDA based on their P2 study of 12 patients by Q2. If this happens, SRPT should trade up to $30 in short order. The Adcom in Oct will be the AdCom of 2015.
You must be new to biotech investing. You win some and lose some. As I posted yesterday, I had free 35/40 call spreads and small number of $5 puts. I closed out my $5 puts for loss today, no biggie. The key to biotech investing is never bet what you can't lose and you always live to trade another day.
I don't spam. But CLDN opportunity is too great to pass up. When I posted on 3/29, ARWR has no immediate binary event. 3/4mg will not be known until late May. Remember, we just passed 85-day and it will be a month before data is know. So CLDN's binary is way ahead of ARWR. Not sure why this post offended you so much. It's one post I made that mentioned another ticker. Not sure what you mean by "flooded this board".
CLDN just announced that CUPID-2 failed miserably. I'm not sure if anyone looked into CLDN due to my post. If they did, I hope they made out ok as the reason I mentioned CLDN is due to it's high-impact binary, it's zero or hero. For myself, I have about 1 dozen of free call spreads 35/40 and a very small number of short $5 puts. I will take a small hit on the short puts tomorrow. No biggie.
If my post offends you so much, please ignore me.
Replicor showed multi-log HBV KD 2 years ago, and yet have not release any tangible clinical data to back it up. It's the biggest joke in the HBV space today. Think about it: an obscure Canadian private company with 2-3 employees and conducting clinical trials strictly in poor Southeast Asian countries. Said another way, if they truly achieved what they said they have, they would have been bought out in the past 2 years and with a buyout price of $5-$10 billions at the minimum.
Replicor, put it in bluntly, is a scam. Nobody in the medical circle believes their claims including Dirk. He is being facetious when he said ARWR, ISIS should just pack up and move on to other diseases. ARWR is fully aware of Replicor and I have discussed with them on several occasions. For starters, Replicor has not even gone thru the standard GLP toxicity testings. That puts them at least 3 years behind everyone else. That's why they have to run their clinical trials in poor SE Asian countries with criminally lax standards.
If they start a trial in any first world countries, then it's sign that they are legit.
AAV1 is actually AAV2/1. All the AAV in use today is based on AAV2 packaging vector, therefore, people are just using the cap to denote the actual AAV hybrid. So AAV1 is AAV2/1, AAV5 is AAV2/5, so on and so forth.
This is the ideal situation for a bear raid where price will flash crash to below $10 one of these days. If you are long, don't set stop loss. You will be taken if you do.
This is old old news. Why are we rehashing this over and over again???
There are 2 primary short thesis and everyone needs to understand them thoroughly:
1. CUPID-1's success is strictly due to the severe patient population imbalance between placebo and high-dose. Rebuttal: if you remove the sickest 4 patients from the placebo group, the analysis still confirms the treatment effect in favor of high-dose group, albeit at a higher p-value, which is totally expected due to reduced the total subject count.
2. Mydicar's dosage is too small to make any difference. Low-dose and mid-dose in CUPID-1 didn't show any transgene expression, while high-dose did. Rebuttal: There is a significant threshold effect in gene therapy. Hi-dose crossed that threshold and exhibited lasting treatment effects in patients. The fact that transgene is found in hi-dose indicates the dosage is not too small, while low and mid-dose are. A very small SERCA gene expression will dramatically improve heart contractibility.
If you are a serious trader/investor in CLDN, you need to understand the above 2 issues thoroughly. Now you have at most 2 weeks before you need to complete your last leg of due diligence and place your bet.
Good perspective. If CUPID-2 is successful, sideliners will do well buying in immediately. I think your $350 price target is very do-able.
However, the key is CUPID-2 being successful. If the price doesn't go back up to $20 next week, then the odds of failure dramatically goes up. We can chalk up the recent price decrease to OPEX manipulation. But if we don't recover soon to fair value, then I'm afraid bad result has been leaked.
Thanks for the kind words.
You might be right about me missing the big gain. I may do some adjustment close to OPEX. Right now, I suspect the results will not be out until after OPEX as the 85-day for 4mg will be the week of 4/20. They will need a month to get data all squared away, so that puts them in 5/20, IMHO.
For the first time since Oct 8, I feel we have reached a good R/R. I just started a sizable short position in May $7 puts this morning. If I get assigned, I will be happy with $6.2 cost basis going into 3/4mg data. If not, then I will make 30%+ in a little over 4 weeks.
Sure, RNA is a big deal, so is CART, so is gene therapy, or another dozen or so innovative ways of combating diseases. I'm not saying what ARWR has no value. I'm simply saying they have not proven themselves yet, just like another dozen or so biotechs I can name. All of them work in a lucrative target market, but there is no clear indication that any of them will make it to commercialization. ARWR is not unique in that regard and they have proven to be mediocre at best so far. It doesn't look like "a diamond in the rough". They have a long way to go to prove themselves. If you have strong convictions in the science, then you need to be patient and let things work out. And as an investor, you need to prepare yourself not to lose a lot of money if they fail to do so.
The truth is most investors today will be happy with a double, and that will be around $15 for people who bought in after Oct 8th. Don't kid yourself about $40-$50 buyout, not gonna happen.
This board is going crazy and I see a lot of impatient grasshoppers screaming for buyout. And a lot of people also complaining about conspiracies. This is a great indication of investor frustration. Jesse Livermore said famously that it's his sitting not his trading that made the bulk of his profits. So sit we must.
Despite some incremental progress in the past 6 months, ARWR has not lived up to its potential. Far from it, it has experienced delay after delay. It is not exhibiting behaviors like a potential blockbuster biotech. Far from it. Your comparison of ARWR to GILD is as ridiculous as it comes.
While the target HepB market size is large, it's unclear if ARWR will ever get a piece of it based on the way it's operating. This board reads just like AMRN, which arguably has a even bigger promise but languishes in sub-$3 range. They arguably suffered more injustice from FDA and short-sellers than ARWR. AMRN bulls always dreams about buyout and it's been 3 years and still none came.
I know you and many longs are extremely frustrated, and trust me, I know how you feel. I have been in ARNA, HGSI and DNDN, among many others. I know how hard it is to watch your dream biotech going nowhere while you know deep in your heart that it should be worth 10x or even 100x times more. My trading experience has taught me to stay flexible and take every opportunity to reduce your holding cost. But most importantly, don't get emotionally attached to your investment. You are not a medical scientist and your vested interest in ARWR is only money. When I read you posts, I got the impression that ARWR is your only investment and you even dream of ARWR at night. Relax, ARWR is just another typical biotech. At this very moment, I can count at least a dozen just like it.
Who is your broker? It will not surprise me if this happens at non-direct brokers such as Scottrade. Go with Interactive Brokers and this will never happen to you again, pre-market or not.
Thank you! That's my point. Just because FDA allowed 3 dose of 1 mg for 3 months doesn't mean FDA tied the multi-dose to the highest single dose trial. It makes no sense.