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Does anyone have any thoughts on RPTP, positive or negative? I am trying to decide if I want to park some money here.
I found this interesting re: Huntington's...
And we've got some pretty exciting stuff coming right around the corner. I mean we've got -- we've been working on primarily two diseases in the clinic, Huntington’s Disease which is on the pipeline chart and then our NAFLD, nonalcoholic fatty liver disease and these are both indications, big indications, different indications in the sense of the conduit patient populations were dealing with. Huntington's being a large orphan disease which most people probably in the audience know someone or are someone who has been disappointed with results in Huntington -- before. It's a difficult condition neurodegenerative condition. But we have some excellent pre-clinical data from our collaborators also there is some clinical small amount of clinical data out there that points to the fact that this maybe a very effective drug for Huntington's disease. So, we're going to be talking more and more about this as the time goes on this fall.
It really hasn't been a topic of conversation. Most of our analysts don't really ask us much questions about it because of course everyone is focused on cystinosis and how the launch is going, but this could be a really transforming event for the company and we at the company are very enthusiastic about it.
We think there is a good chance if you are willing to follow science that this could actually have an affect. So, what I want to do is talk a little about Huntington's disease and but there is a longer timeline there and we're still enrolling in our Phase 2b study. But Huntington's disease is disease. It effects unfortunately folks in their mid life in their 20s and 30s, it's slowly progressing disease, often takes 10, 15, 20 years to fully succumb from the condition. And as I said it's a big orphan, it's 40,000 to 60,000 people with this condition in U.S. and Europe. So, there is a lot of opportunity here. There are no therapies available really to slow the progression of the disease.
So if you diagnose a Huntington's disease, you need to make plan and that's been the tragedy of Huntington's disease for a long time as people actually because it's a generic disease they know they can be screened for, they know they're ultimately probably going to get it. So, what you got is, you got a ticking time bomb in terms of you know that ultimately you're going to end up with some of the symptoms with this disease. So, there is a lot of interest in getting these patients effective therapy that will slow the progression of the disease.
In our program, we've been working with a number of groups on the use of cystamine to increase levels of BDNF in these patients. Patients with Huntington's disease have lower than average levels of BDNF in their -- and in animal studies that were using transgenics which are basically a knock out similar knock out to what the Huntington's disease would be in humans have low levels BDNF in their brain. So most people will say that if you can increase levels of BDNF from the brain you potentially could change the progression of the diseases and that's really our target. Sustaining number of labs has been show in animal models, non human primate models to increase levels of brain BDNF and so that's really the basic target.
So our trial which started a number of years ago is paid for by the French government actually it was started out by the folks of the Cherry Institute who works with cysteamine and -- really in Huntington's disease models and determined in those models and there is published data we can share with you if you'd like that again cysteamine could increase levels of BDNF and actually additional data out of the to folks that we work with this well has shown at a therapy on some of these transgenic animals will slow down the basically the muscles spasticity is lessened with the use of cystamine. So, there is a these are animal models and not human studies, but the same time I think they are both essentially independent groups pointed the same effect. So we have been enrolled 96 patients in this study.
This is done through a collaboration with the Canadian, with the French clinical sites. It's set-up in two phases. So there is an 18 month phase where it's a placebo controlled blinded phase. So, we have a one group gets cystamine, the other group gets the placebo and that phase lasts for 18 months. And that's a key point because one of the problems with the generic disease is that often patients who have these conditions once they get into a clinical setting and they get to socialize with other patients they start feeling better. So you get what they call a placebo effect. And the placebo effect can last for some period of time. It can really make it difficult to differentiate a modest drug effect from no effect at all.
So 18 months is we consider the clinicians consider to be long enough so that we would see probably that placebo effect could be minimized. So there is an 18 month period that's blinded. Now that's the phase that we will unblind in the first quarter of next year. So, the last patient coming off of this drug 18 months and they are all going to active drug after that. But the last patient coming off that blinded study comes off in November.
So, that's pretty much a hard timeline. And then all the patients go into an open label study, they all go on to cystamine and then we look at the data after 36 months. So the dosing is less than cystinosis it's every 12 hour dosing. So we're using the same dosing schedule we'll use for process being cystinosis and endpoint is the universal rating 100 Huntington's disease rating scale so we're looking at changes from baseline from -- in that scale for muscle spasticity.
Relatively measuring levels of BDNF in the -- patients which has been again shown to be depressed in Huntington's patients, so we are looking for elevation of that...A clinical sites again two phases, if the 18 months data looks good we'll meet with FDA and EMA and decide what the next step is again with the unmet need in this group. I think if the data is strong I think we'll have a good argument for potentially getting -- using this study as a registration study with potentially some post approval commitments, that's speculation at this point.
So, upcoming catalyst as I mentioned we've now been approved and we are currently generating revenue with PROCYSBI and cystinosis. We have a positive opinion from EMA. If we get the approval in the third quarter we will be launching in Europe early next year we're not partnered with this project. We are going global ourselves.
And so we now have negotiated the approval path in Canada where there is probably a 100 of these patients. So it's a little bit of a around the globe in terms where you find these patient and we have identified where we think the hotspots are and we are going after those markets pretty aggressively. Coming up as I mentioned we are looking for our EMA approval in the third quarter we are also looking for a determination by the orphan exclusivity in Europe which is 10 years and of course we are optimistic about that.
We haven't heard from them at this point. And as I mentioned the Phase 2/3 data from Huntington's will be in the first quarter of next year, we'll be announcing that at that point and then the Huntington's study the of this study is still being enrolled. We hope to have enrolled as well at least the end of this year or early next year a lot of it will depend on the pediatric study so we are enrolling kids. So, hopefully enrollment will be full pick-up during the summer time.
It's difficult to get kids into the study and biopsy. Live enthusiasm from the clinical side is almost going a little slower than we expected, but it's a one year study. So we can get enrollment done first half of next year then hopefully we can get data out to you guys some time in 2015.
Interesting comments at JMP
First drug approval PROCYSBI for the treatment of nephropathic cystinosis.
Read here
8:37AM Raptor Pharma's PROCYSBI receives positive recommendation from CHMP (RPTP) 9.37 : Co announced that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization for PROCYSBI 25mg and 75mg gastro-resistant hard capsules, cysteamine (as mercaptamine bitartrate) for the treatment of proven nephropathic cystinosis. If approved, PROCYSBI will be indicated for the treatment of proven nephropathic cystinosis. A decision is expected from the European Commission within a few months of the CHMP recommendation.
7:01AM Raptor Pharma licenses intellectual property for Rett syndrome from French Research Institutes (RPTP) 6.21 : Co announced that it has licensed pending patents related to the use of cysteamine for the treatment of Rett syndrome and associated disorders of methyl-CpG binding protein 2 (MECP2). Under terms of the agreement, Raptor receives exclusive global rights to develop and commercialize cysteamine and related compounds to treat MECP2-associated disorders. Raptor's license agreement is with the Technology Transfer Accelerator of South Eastern France that represents the French medical research organizations where the technology was invented, including the Institut Curie, INSERM and Aix-Marseille Universite. Financial terms of the licensing agreement have not been disclosed. When issued, the initial patents covered by this agreement are expected to be valid until 2030.
The priceing on the new drug will come sometime in June. At that time you should see a nice price increase here. Until then we will probably go from todays price to maybe 8.
Any idea what a good price for this stock is in the next few months?
People need to be carefull on stop losses. The MM just went down and took out them all the way dow to 6.62.
3 month chart and 6 month chart are looking money.. Just look at that RSI, pullbacks on the money 9 and 14 DMA. MFI, i mean you can't find a better chart then this.. i will merry this chart its that perfect. we are looking at new records high.. Good luck to everyone.. i am going to flip the shit off this stock on weekly bases on its way up... but will hold a long position untill i think its time to sell. veeeeeeeeeeee Good luck to all
6 month weekly 9 and 14 ma
http://stockcharts.com/h-sc/ui?s=RPTP&p=W&yr=0&mn=6&dy=0&id=p97736741005
6 month weekly with 50 and 200 ma
http://stockcharts.com/h-sc/ui?s=RPTP&p=W&yr=0&mn=6&dy=0&id=p80001004649
Now that we have our drug approved,we now need to have it priced and this will move. We should get back to the 7s either today or tomorrow.
Day's not over yet...nor the week for that matter. Lots of big-boys eyeing RPTP now that FDA approval is past. Let the day-traders create the little trends, keep your eye on the big one.
I am holding here long. first day shorts are all over it.. not surprising. its a hold..
Is it me or did anyone expect a little more pop and hold the price.
It has been Approved. Here is FDA link.
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm350091.htm
It must be some sort of leak of info. Strange.
Yea same here have not seen anything anywhere. I was thinking it would be halted, but now I think we may see it after market closes.
Yea same here have not seen anything anywhere. I was thinking it would be halted, but now I think we may see it after market closes.
This is all over the board. I still haven't seen anything about approval YET.
All of sudden volume poured in, looks like approval news has leaked? either way I'm glad my patience paid off.
These market makers are doing every thing to shake shares thisw morning.
This went up on heavy volume and down on low volume. ??????
Its actually gone red. Hopefully closes green. Not sure what to make of this.
~ come'on push!!! :)
We could see more volume in the first hr then we saw all yesterday.
This just starting to heat up going into tuesday.
Some panic. Still holding my shares
This hard to watch, other than the pop yesterday its just a slow downhill drop everyday.
Wow, what a tanking this is doing, on big red streak for weeks now. Breaking lower bollinger band as we speak,I would call it a falling knife if it wasn't like watching paint dry everyday.
Good One on http://EmergingGrowth.com
The press sometimes provides opportunities that would normally not occur in the equity markets, as they tend to exaggerate facts and news about publicly traded companies. Today’s media has the amazing ability to reach so many investors, as TV and internet have come a tremendously long way in the past decade. Potential investors are informed through multiple communication channels, as the speed of information is faster now than ever before. Investors looking to take advantage of the media and its ability to push stock prices both higher and lower can achieve great points of entry as well as good exit levels. Recent news developments regarding multi-level marketing firms and a public showdown between two billionaire hedge fund managers may provide a good opportunity in NATR.
Nature’s Sunshine Products (NASDAQ: NATR) operates in the personal products industry, a subset of the Consumer Staples sector. The company manufactures and distributes nutritional and personal care items, which include herbal products, vitamins, homeopathic products, oils and lotions, aloe vera gel, herbal shampoo, toothpaste, and skin cleanser. Nature’s Sunshine has a market capitalization of $240.51 million, and offers a dividend yield of 1.31%. In the company’s last earnings report that was announced on November 2nd of last year, net sales were $33.7 million, compared with $33.5 million in the same quarter a year ago, an increase of 0.5 percent. Operating income was $2.7 million, compared with $2.3 million in the same quarter a year ago, an increase of 15.0 percent. Overall the earnings report was slightly better than expectations, showing steady growth from the previous year. The stock reacted favorably post-earnings report until the worries about multi-level marketing firms in December began to ramp-up.
NATR is a smaller player in the MLM space, and uses this technique to sell health and wellness products. The stock got hammered as a by-blow of the Herbalife (NYSE: HLF) fight in December. On Dec. 18th the company’s stock price was at $15.44, and by Dec. 26th it was down more than 10% to $13.02. It’s since recovered but is going to be volatile as MLM firms get more media interest and bad press. The negative media exposure is expected to increase over the next few weeks as billionaire hedge fund manager Bill Ackerman publicly announced his short position on Herbalife. This investment was highlighted this past Friday on CNBC when Ackerman got into a heated exchange with hedge fund manager Carl Icahn.
Whether Icahn has a position in the stock is still unknown, but the two investors went toe-to-toe in one of the most entertaining moments in financial TV history. Ackerman has accused Herbalife of basically running a pyramid scheme, publicly stating that it will eventually be revealed and the company will go bankrupt. Whether this will occur is anyone’s guess, but the fight between the two hedge fund managers is expected to get a ton of press this week. This is expected to cause Herbalife and many of its similar users of MLM to drop, as folks will reconsider their investment in these companies. Some are expected to sell, as hedge fund titans such as Ackerman have the ability to move the stock. NATR is closely correlated with Herbalife, so the potential selloff may provide a good buying opportunity and a nice entry point in NATR.
The stock currently trades at $14.46, in between its 52-week range of $13.02 and $17.73. The company does not have any huge competitors in the industry; however, there are other similar companies. The company’s main competitors in the sector include Xenoport (NASDAQ: XNPT), market cap $365m, Depomed (NASDAQ: DEPO), market cap $348m, GTX Inc (NASDAQ: GTXI), market cap $305m, and Raptor Pharmaceutical (NASDAQ: RPTP), market cap $282m. NATR is well positioned in the sector and is expected to exceed growth expectations in 2013. That said, a buying opportunity might be available within the next few weeks.
Add a few more. Average PT of $9.
It's like a big kids version of monopoly. I just bought park place.
Excellent move today...above $6 and moving to $7
You did a great job my friend.
sold my shares between $5.83-5.79 today. locking in some profits from a $4.94 average. have a gut feeling that i'll be able to buy back cheaper. was not impressed by todays action during the last hours. GLTA
maybe there is some short covering going on, because today is the last day in 2012 and some positions have to be closed. i was speculating on that to happen and it seems the i was right about that.
who are pumping this stock?even dough on negative news.what kind of game is this?
Agree. This creates a an excellente buying opportunity.
Bought today a few more at lower prices and preparing to add more.
Very interesting company at near tearm, of course we could see some short term weakness.
until now, i'm quite impressed by the price action. the two articles should have helped either. let's see if RPTP is able to close above the SMA200.
Raptor Pharma shares should be bought on any weakness, says JMP Securities
in, http://finance.yahoo.com/news/raptor-pharma-shares-bought-weakness-122856459.html
Oppenheimer Says Raptor Pharma (RPTP) Procysbi Delay Immaterial, Reiterates Buy
Oppenheimer has an Outperform rating on Raptor Pharmaceuticals (NASDAQ: RPTP) with a price target of $11.00.
in, http://www.streetinsider.com/Analyst+Comments/Oppenheimer+Says+Raptor+Pharma+(RPTP)+Procysbi+Delay+Immaterial,+Reiterates+Buy/7969012.html
They have money and orphan status. 3 months doesn't representa major problem
what have change is that now myself and other investors will have the opportunity to bUY shares at better prices and hold a Litle more.
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http://www.raptorpharma.com/index.htm
http://finance.yahoo.com/q/ks?s=RPTP
Raptor Pharmaceuticals Corp. operates as a development stage biotechnology company in the United States. The company engages in the discovery, research, and preclinical development of drug candidates and drug-targeting platforms for the treatment of various brain disorders and neurodegenerative diseases, genetic disorders, and cancer through the proprietary use of receptor-associated proteins and mesoderm development proteins. Its clinical-stage products include DR Cysteamine, which is in Phase IIb clinical trail for the treatment of nephropathic cystinosis or cystinosis; and in Phase IIa clinical trail for the treatment of non-alcoholic steatohepatitis. The company also develops Convivia, a Phase IIa clinical trail product for the treatment of aldehyde dehydrogenase or ALDH2 deficiency; DR Cysteamine, which is in Phase II clinical trail for the treatment of Huntington?s Disease; and Tezampanel and NGX 426 that is in Phase II clinical trail for the treatment of migraine, acute pain, and chronic pain. In addition, its preclinical development products comprise HepTide for the treatment of Hepatocellular Carcinoma and Hepatitis C; WntTide for the treatment of breast cancer; and NeuroTrans for the treatment of neurodegenerative diseases. The company has collaboration agreements with the University of California, San Diego for a clinical study of DR Cysteamine in juvenile patients with Non-Alcoholic Steatohepatitis and to study DR Cysteamine in patients with cystinosis. Raptor Pharmaceuticals Corp was founded in 2005 and is based in Novato, California.
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