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The "imminent" comment ticks me off more everyday that passes without an announcement. The stock down .16 and trading at $4.91 doesn't help either. The next scheduled clinical dev. is the pre-NDA meeting with the FDA Nov, 8th. I hope we don't have to wait till Nov to hear any updates.
I can forgive management for their error in judgement if they announce the UK approval tomorrow along with a EU marketing partner, $50 million up front payment, and royalties. If they do as I ask then I swear to not say another negative thing about management this year.
Well, make it a 3 - 1 split which would give you a $3.80 share price. In a few months the stock and the market cap would double.
RNAi Firms Find a New Rival (and Partner) in an Old Technology
Malorye Allison 10/4/07
What? Can antisense possibly be hot again? Many people wondered if the decades-old RNA-targeting approach to drug development was even still alive. But look again. Recent events show antisense isn’t just hanging in there; its products might actually beat their much-ballyhooed RNAi-based rivals (from the likes of firms like Cambridge’s Alnylam Pharmaceuticals and Worcester’s RXi Pharmaceuticals) to market.
Carlsbad, CA’s Isis Pharmaceuticals (NASDAQ: ISIS) has had a recent string of deals that suggests second-generation antisense really does have legs. What’s more, because of the antisense pioneer’s huge patent estate, companies will likely need to cut a deal with Isis to work in RNA targeting of any kind–including in the budding new field of microRNA-based drug development. (Alnylam, for instance, has a longstanding strategic alliance with Isis, and last month the two announced a joint venture focused on microRNA.) Meanwhile, Alnylam (NASDAQ: ALNY) is weathering controversy over safety concerns. In the wake of all this, RNAi companies could end up watching underdog Isis, with its “passé” antisense drugs, finish ahead in one of the pharmaceutical industry’s most exciting races.
Antisense and RNAi companies are all trying to do the same thing—create a new class of drugs that whack disease-causing RNA. Messenger RNA (mRNA) is the middle man in the genes-to-protein process. Most drugs attack the bad proteins themselves. Instead, antisense and RNAi companies try to stop “bad” proteins from even being made. The drugs aimed at doing so are all novel biologicals. Antisense uses single stranded DNA while the RNAi folks typically use some kind of small double stranded or looped RNA. The differences used to be clearer, but nowadays you hear about all kinds of approaches, including hybrids—hence the patent minefield. The big difference is that RNAi is a natural process that the cell itself uses to turn genes off.
Oh, and RNAi is very new while antisense has been around forever. A number of high-profile antisense compounds have already tanked gloriously on the way to market. Only one has been approved after 20 years of trying, and that lone drug is a niche ocular therapy from Isis—it’s more of an aside than a breakthrough. So, when RNAi came along a few years ago it was a classic Cinderella story with antisense relegated to the part of ugly stepsister. Suddenly, here was RNAi—a simple, very powerful way to knock out mRNA that seemed like it would lead especially quickly to marketable drugs. Antisense companies, including Isis and Cambridge, MA-based Hybridon (now Idera Pharmaceuticals), were shoved aside as yesterday’s technology.
But things are changing.
For one thing, the tough questions are now popping up for RNAi. Last year, a bunch of mice died in a key study by Stanford researcher Mark Kay. In the resulting Nature paper, Kay suggested that when you harness the cell’s innate RNAi pathway, natural production of microRNAs might inadvertently be curbed. That’s troubling. Although microRNAs have only recently emerged as big players in cellular biology, it is increasingly clear that they are very important. Alnylam responded last week with its own Nature paper and a press release. David Burncroft, one of that study’s authors, said via e-mail that it shows “Our approach to RNAi using siRNA molecules does not interfere with microRNA synthesis and function in the liver.”
Kay is not sure the latest paper settles the safety question, but he applauds it for “adding to the promise of RNAi therapeutics.” John Rossi from Southern California’s City of Hope concurs. “They have a good claim that they aren’t triggering toxicity,” he says. “But they haven’t proved it doesn’t happen [with these types of molecules].”
Also, having started so much later, (the first RNAi therapeutic firms were founded around 2002 and 2003) RNAi companies are way behind antisense firms in building their pipelines, and they don’t seem to be catching up that quickly. Only one of Alnylam’s drugs is in clinical trials yet—a respiratory syncytial virus treatment in Phase II, which the company says it will report on in the second half of this year.
Meanwhile, antisense suddenly has newfound momentum. Just a couple years ago, Isis CEO Stanley Crooke was a tortured man. The company had gotten that one little approval and then experienced a brutal string of failures. Isis’s new thing was second-generation antisense based on a novel chemistry. Unfortunately, all those new drugs were still creeping along the near end of the product pipeline.
Crooke was adamant that they were still in the game. “We’ve got the patents, we’ve got the patents!” he kept saying during one conversation. Just hearing the word “RNAi” launched him into a long winded, but well reasoned, explanation about how people who differentiated RNAi from antisense were really just muddying the waters: Isis’s years of research and huge patent portfolio, he said, put them in the ideal position to capitalize on RNA interference or any other form of RNA-targeting, and the newcomers were just trying to jump on their bandwagon.
At the time, it was difficult to know whether, like Tolkien’s Gollum, he was indeed cradling a treasure much more valuable than most people realized, or if he was just trying to keep Isis alive by any means. Now, the answer seems clear.
“The sea change,” as Crooke calls it, started in early May, when Isis netted a collaboration with Bristol-Myers Squibb around a new anti-cholesterol target. A few months later, a string of deals ensued starting with an announcement on September 7 that Isis and Alnylam were launching Regulus Therapeutics, a joint venture around microRNA-based drugs.
This is one of the most exciting and burgeoning new fields in medicine. No less a luminary than David Baltimore joined Regulus’ board of directors and became chair of the new company’s scientific advisory board. Regulus uses IP from both companies, but, “The only real way to target a microRNA is antisense,” says Crooke.
Just a few days after Regulus’s founding was announced, Isis inked a metabolic-disease deal with Ortho-McNeil worth potentially up to $230 million. That deal covers several compounds, two of which were held by Symphony GenIsis up until last week, when Isis re-acquired the firm, which it has spun off in 2006 expressly to keep its metabolic program alive.
Isis will be reporting Phase II data on one of those four drug candidates next Monday. Crooke won’t be fully vindicated until one of those second-generation molecules finally crosses the finish line. And good news for antisense could ultimately be good news for RNAi companies. “Any progress in the field of RNA-based therapeutics would be progress for the industry,” says Alnylam COO Barry Greene.
During that conversation two years ago, a weary Crooke said “The two most difficult things are raising children and drug discovery.” Hopefully his children have handed him fewer challenges than the antisense has. Meanwhile, the question for RNAi-based companies is: How quickly can a toddler turn into a teen? How’s that for a really scary question?
http://www.xconomy.com/2007/10/04/rnai-firms-find-a-new-rival-and-partner-in-an-old-technology
How in the heck did we close in the red today after the announcement? Hopefully we'll get some buyers tomorrow in anticipation of the CC. 11 million short shares are trying to hold it back.
I guarantee if RPRX did a 4 for 1 forward split then the market cap would double < 3 months. Lets see if we can get a shareholder vote.
Dude, thanks for the post, but I can't believe the top secret stuff.
Miracles can happen. We actually closed in the green. Hopefully someone leaked positive EMEA news.
I think its ISIS 301012.
ISIS 301012 — ISIS 301012 reduces the production of apolipoprotein B-100, or apoB-100, which is the protein that carries certain forms of cholesterol and triglyceride particles in the bloodstream. Cholesterol can be carried in the bloodstream in a variety of forms, with high-density lipoprotein, or HDL, being the good form, and low-density lipoproteins or LDL, and very low-density lipoproteins, or VLDL, being the bad or atherogenic forms, which are directly involved in heart disease. ApoB-100 is a target that the pharmaceutical industry has long recognized as attractive for intervention but that has proved to be undruggable using traditional small molecule approaches.
I would be happier if JP would quit talking about partnering and just emphasized the market potential & clinical progress for both drugs. He needs to tell investors that a number of companies have been in contact, but the longer the company holds out on selling or partnering then the larger the buy-out.
Cleveland Clinic Unveils 'Top 10' Medical Innovations For 2008
10.03.07, 11:32 AM ET
CLEVELAND, Oct. 3 /PRNewswire/ -- Cleveland Clinic today announced its Top 10 Medical Innovations list, highlighting technologies which will likely have a big impact on healthcare in 2008.
The list of breakthrough devices and therapies was selected by a panel of Cleveland Clinic physicians and scientists and was unveiled during Cleveland Clinic's 2007 Medical Innovation Summit, which is currently underway. They include therapies for valvular heart disease, unusually high cholesterol, and severe neurologic disorders.
"These innovations represent exciting technologies which may have a major impact in clinical practice in the near future," said Tomislav Mihaljevic, M.D., who chaired the Top 10 Medical Innovations List. "We believe this list represents the best, innovative approaches in health care today affecting some of the most prevalent diseases affecting millions of patients world-wide."
The Top 10 Medical Innovations for 2008 are:
10. Dual energy source computed tomography (CT) imaging: The dual source CT features two X-ray sources and two radiation detectors which allow for imaging of patients more quickly and with less radiation. The speed at which the dual-source scanner operates allows physicians image patients with high or irregular heart rates, which used to be significant limitation of this technology.
9. Engineered cartilage products for joint repair: The use of biologic and engineering principles to design natural biomaterials that are used to replace joint cartilage tissue that is damaged from injury or arthritis. The materials are surgically implanted into the joint with the intent to restore the damaged cartilage and avoid artificial joint replacement.
8. Implanted device allowing neural control of objects by the severely disabled: Novel communication interfaces are being developed to provide the ability to control devices and to potentially restore limb movements to individuals with spinal cord injuries, stroke, ALS, as well as other central nervous system injuries. This interface system is designed to restore functionality for severely motor-impaired individuals.
7. Image fusion for diagnostic and therapeutic use: Used to diagnose medical problems, both anatomic and physiologic in nature, as well as to assist minimally invasive procedures - such as stent placement or tumor ablation.
6. Live attenuated influenza vaccine for children as young as six months: Nasal drops containing live attenuated flu can be used as a vaccine in lieu of needles, and provide effective protection from influenza for this high-risk population.
5. New oral anticoagulant drugs for treating and prevent thrombosis: Newer anticoagulant treatments, which include low molecular weight heparins, are being introduced with the goal of curbing complications such as bleeding and thrombosis.
4. Convergence of advances in genome scanning and informatics to support clinical applications: Genetic testing that can be used to develop personalized risk assessments and disease management plans for variety of genetically caused diseases.
3. RNA-based therapeutics: This particular innovation uses RNA antisense technology to treat patients who are unable to reach their targeted cholesterol levels with statins alone or who are statin intolerant. The therapy is intended to reduce the production of ApoB-100, a protein that carries certain forms of cholesterol and triglycerides in the bloodstream.
2. Percutaneous aortic heart valves: For high risk patients, a technique has been developed which involves inserting a new balloon expandable wire mesh valve with internal valve leaflets. The valve is inserted through a groin or small chest wall incision and then fed up through a catheter into position with X-ray screening and the balloon inflated to secure the new valve.
1. Flexible intralumenal robotics: A novel catheter-based technology that allows precise remote manipulations within the intra-luminal space with precision and reproducibility, which would surpass human capabilities. This technology is likely to have applications in urology, cardiology, cardiac surgery and other specialties.
"Cleveland Clinic scientists and clinicians are engaged in a continuous dialogue about how to provide patients with the best possible care. Their constant interaction, combined with the Clinic's role as a site of basic science and as an end user of medical products, provides a unique perspective for evaluating what will be the novel and next generation therapeutics and products," said Christopher Coburn, Executive Director, CCF Innovations, Cleveland Clinic's technology commercialization arm. "The second annual Top 10 list is a result of a survey of dozens of Cleveland Clinic thought leaders and we hope it stimulates widespread discussions on innovation and new technology."
Four major criteria served as the basis for qualifying and selecting the Top 10 Medical Innovations. Nominated innovations were required to:
- Have significant potential for short-term clinical impact (either a major improvement in patient benefit or an improved function that enhances healthcare delivery).
- Have a high probability of success. - Be on the market or close to being introduced. - Have sufficient data available to support its nomination. The Top 10 Medical Innovations for 2008 were announced Oct. 3 at the annual Cleveland Clinic Medical Innovation Summit. A panel of eight Cleveland Clinic thought leaders and a moderator discussed each technology and its reason for inclusion during a panel session.
In developing the Top 10, Cleveland Clinic enlisted the expertise of AlixPartners, LLP, an independent international management consulting firm. AlixPartners led the process to probe the opinions of Cleveland Clinic physicians and researchers, create a field of nominated innovative technologies for consideration, and develop a consensus perspective on the Top 10 Medical Innovations for 2008.
For more information about this year's Medical Innovation Summit and the conference agenda, visit http://www.clevelandclinic.org/innovations/summit/default.htm.
About CCF Innovations
CCF Innovations, the technology commercialization arm of Cleveland Clinic, organizes the Medical Innovation Summit and stewards the Clinic's technology innovation strategy. It enhances product-oriented innovation throughout Cleveland Clinic and transforms promising therapies, devices and diagnostics into beneficial medical products, via spin-off companies, licensees and equity partnerships.
About Cleveland Clinic
Cleveland Clinic, located in Cleveland, Ohio, is a not-for-profit multispecialty academic medical center that integrates clinical and hospital care with research and education. Cleveland Clinic was founded in 1921 by four renowned physicians with a vision of providing outstanding patient care based upon the principles of cooperation, compassion and innovation. U.S. News & World Report consistently names Cleveland Clinic as one of the nation's best hospitals in its annual "America's Best Hospitals" survey. Approximately 1,800 full-time salaried physicians and researchers at Cleveland Clinic and Cleveland Clinic Florida represent more than 100 medical specialties and subspecialties. In 2006, there were 3.1 million outpatient visits to Cleveland Clinic. Patients came for treatment from every state and from more than 80 countries. There were more than 53,000 hospital admissions to Cleveland Clinic in 2006. Cleveland Clinic's Web site address is www.clevelandclinic.org.
SOURCE Cleveland Clinic
http://www.forbes.com/prnewswire/feeds/prnewswire/2007/10/03/prnewswire200710031131PR_NEWS_USPR_____...
Did you see that trade at $4.44 of 1100 shares this morning? How can they let it drop .44 on 2,000 shares traded on the day? I don't know if it was an attempt to take out some stop losses or specialist games. Javelin management needs to talk to this guy since he's scaring off the retail investor.
We don't know what we're waiting for and thats the confusion investors are having. JAV has been a little less then forthcoming.
Doesn't the UK/EMEA have deadlines for making their decision? This is whats confusing me. It seems like Javelin has no idea when the decision will be made. At least the FDA gives you a date you can pretty much rely on.
Javelin Pharmaceuticals Still On Target With Dyloject
posted on: October 01, 2007 | about stocks: JAV
Javelin Pharmaceuticals (AMEX: JAV) provided investors with an update on the status of Dyloject and other pipeline drug candidates last Thursday at the UBS Global Life Sciences Conference. The company’s CEO was absent at the conference because he was in Europe preparing for the UK launch of Dyloject, pending EMEA approval. Javelin expects approval imminently and plans to launch Dyloject in the UK during the fourth quarter and they expect to record revenues from the sale of this product in the UK during 2007.
Dyloject is subject to a mutual recognition process for its initial European launch, first in the UK during 4Q07 and then following in Germany after six to nine months. The Company estimates peak sales of Dyloject, an injectable form of the anti-inflammatory & pain medication diclofenac, in Europe after 12 months of $75 million once formulary issues are resolved over the first two to three months of marketing. A launch in the US market is expected by late 2008 or early 2009 with a domestic sales potential of over $175 million. Dyloject will be targeted to hospitals for use in acute care settings as a non-opiate option for pain relief that has fewer side effects and no addiction potential. Javelin expects to report results in late 2007 or early 2008 from the first of two studies of Dyloject in the evaluation of abdominal and orthopedic pain trials in the US.
The Company currently has roughly $50 million in cash, 48 million shares outstanding, and a cash burn rate of about $2 million per month with an expected increase upon Dyloject approval & launch for manufacturing and marketing costs. The Company's other products in development include Rylomine, an intranasal formulation of morphine that is in Phase 3 trials for the treatment of acute moderate-to-severe pain in the US & Europe; and PMI-150, which is a proprietary nasal formulation of ketamine that is under registration studies for the treatment of acute moderate-to-severe pain. I rate the stock a buy with an $8 ($384 million market cap) end of year target and a $12-plus ($576 million market cap) price target by the end of 2008 given European & US peak sales potential of at least $250 million for Dyloject alone, which exceeds the Company’s current market cap of just $244 million.
http://seekingalpha.com/article/48598-javelin-pharmaceuticals-still-on-target-with-dyloject?source=y...
BKCC - I think a fund liquidated in order to cover some redemptions. See below:
How to Avoid the Coming Hedge Fund Panic This Fall
Although the ongoing global credit crunch is applying severe pressure again this week on global equities, upcoming hedge fund redemptions scheduled for September 30 risk causing an even greater panic.
The risk of a crash is a distinct possibility as everyone heads for the exits at the same time, marking the point of maximum pessimism in this liquidity squeeze.
The big threat to financial markets, in addition to deteriorating sub-prime and mortgage-related casualties, is the massive unwinding of hedge fund assets ahead of September 30.
Most hedge funds open the liquidity window for redemptions every 90 days or quarterly. In order to facilitate orderly withdrawals, they also require investors to place their redemption notices at least 30 days' in advance prior to quarter-end.
That means the next liquidity window is quickly approaching on September 30. It also implies that a swath of selling lies ahead this month and in September as money-managers create liquidity to meet redemptions. And redemptions will likely be enormous.
Over the last six weeks a blizzard of closures, bailouts and collapses has affected more than two dozen hedge funds worldwide, including one of the biggest at Goldman Sachs Group.
Like a bad nightmare, investors in hedge funds are awakening to the sober realty that many of their hedge funds hold a mish-mash of complex securities in their portfolios - namely leveraged mortgage-backed securities, SWAPS, CDOs and other garbage most investors barely understand.
It's not over yet. My advice remains the same: Don't panic and sell into weakness. This market will find a bottom eventually. Corporate earnings, though moderating, will continue to expand next year amid low global inflation, lower interest rates and buoyant money-supply growth overseas.
This is a credit meltdown, not an earnings-related bear market typically squeezed by tightening credit conditions. Keep your powder dry.
ERIC ROSEMAN, Investment Director
RosemanBlog.SovereignSociety.com
I'm not worried about the stock going up, I just don't feel like riding out a slide to $4.00 on a delay. If we get the UK approval then I can't see the stock ever trade below $5.00.
PANC- In todays presentation the speaker stated that some trial participants use the liquid formulation to wash down their other drugs. He specifically addressed the taste issue and said their formulations will not encounter compliance issues due to taste. The drug does not need refrigeration and the volumes are small.
The UK approval & sales are still anticipated for the 4th qtr. So the PR must be "imminent".
PANC- I've always wanted to get a position in the company so I'm jumping in with you. I couldn't care less if bevirimat is in liquid or pill form. The drug clearly works and hasn't shown any safety risks.
SNUS
They have a presentation on thursday so they can't run and hide for much longer.
Can you reach the phone? You better call for a pizza delivery.
ANSV - since your prior post started out Ouch, Ouch, I thought it was a good time to mention Zingo. One of your prior posts stated that a friend described the injection as a punch in the arm. I contacted their IR and they described it more like a puff of air. Wouldn't you be surprised for a device approved for a 3 yr old to produce any pain? I'm invested because of "Adlea", but I thought you would be interested in the companies description for Zingo.
I can just imagine the commercials. All we need is a good looking girl to say "nothing turns me on like a nice set of balls". Then we could have a picture of two sets of balls with Androxal on the nice pair.
Docs will have guys beating down the doors for Androxal.
"Imminent" was a poor choice of words, but all is forgiven if they make the announcement before the open tomorrow.
SNUS - I'll be surprised if it ends the day positive unless SNUS reports superiority. Management has stated they were raising cash post Phase III release which is going to stop a lot of investors from jumping in on non-inferiority even if the toxicity profile if better.
I called him friday and his secretary was screening the calls. She gave me the story that he wasn't able to take my call at this time and I could leave a message. I'm still waiting for the return call.
If someone is trying to wear out the weak then their doing a good job. I'm getting tired of this.
Yes, this is turning into a nice ride. Only question is whats going to happen first a buy-out or a partnership for ISIS 301012? The stock is still under the radar due to the problems with first generation antisense.
I'm sure they would have released a PR if they had encountered some problem with the UK approval. Imminent can mean anything from days to months. I really don't see a credibility issue.
JAV is the only biotech I've ever invested in that is actually ahead of schedule in getting a drug to market. Who would have ever thought that Ketamine is this close to approval?
We'll we finally have someone that needs shares or a short doing a little covering. I think we were up .80 on less then 3K shares.
Why didn't they webcast their ThinkEquity presentation? It would have been interesting to see if the "imminent" time frame was used for UK approval.
Medicure mulling early data release for heart drug
Thu Sep 20, 2007 11:52am EDT
TORONTO, Sept 20 (Reuters) - Medicure Inc (MPH.TO: Quote, Profile, Research) said on Thursday it completed enrollment of patients for the Phase 3 trial of its heart treatment ahead of schedule, which could pave the way for early dissemination of the data.
The small biotech company said it enrolled more than 3,000 patients in the study for its MEND-CABG II trial, a treatment for reducing post-operative heart attacks in patients undergoing coronary artery bypass.
Medicure had targeted completion by November. The company is still sticking to its original schedule to release the data in early 2008, but a company spokesman said it could be sooner.
"This is probably one of, if not the biggest independent Phase 3 trials in Canadian biotech history. Three thousand patients is a big number to manage by one company and we've pulled it off so far," said Medicure spokesman Adam Peeler.
"This brings things into focus in terms of how close we actually are to data from the trial."
Peeler said the company is eyeing the summer of 2008 to submit a new drug application to the U.S. Food and Drug Administration.
"This is huge, positive news for the sector. The fact that it's the largest clinical trial for a biotech company in Canada, this is a major milestone that they have achieved here," said Paradigm Capital analyst Claude Camire.
"This really speaks to the true value of management that was able to complete this ahead of time."
Medicure shares were unchanged by late morning at C$1.10 after climbing as high as C$1.15 earlier in the day.
http://www.reuters.com/article/marketsNews/idUKN2039221120070920?rpc=44
BCRX: You have to be kidding. Blame the needle.
9:53am 09/20/2007
BCRX8.00, -3.78, -32.1%) was a huge early loser, tumbling 35% to $7.69. Late Wednesday, the company said preliminary findings from a Phase II study with intramuscular injection of peramivir, a drug candidate to treat seasonal and life-threatening influenza, show that while a single dose demonstrated improvement over placebo, the improvement was not statistically significant. The company also said that due to the introduction of a shorter injection needle in the Phase II trial, that only one-third of subjects received an adequate intramuscular injection. "Based on these results, we have a clear and concise plan to correct the issues identified in this study and continue our preparations to initiate our Phase III program by year end," said BioCryst Chief Executive Jon Stonehouse in a statement.
Do you feel better now? The trading sucks for a company that will release imminent news.
What we need is a partnership and option to buy once FDA approval for Proellex is granted or Phase III complete. The stock price would trend up and then your eventual buy-out would be at the premium management and shareholders anticipate. The buyer would also be protected in case things didn't quite pan out.
I'll be surprised if RPRX is not bought-out in the next six - nine months. It doesn't make sense to sit around and wait for Phase III data before you make an offer. The time to get Proellex on the market has shortened with the anemia indication, safety issues have been de-risked, block-buster drug, and potential suitors are sniffing around.
If I was interested in the pipeline then I'd be making an offer sooner rather then later. Why risk someone else buying the company?
I think most of the short position on biotechs are really hedges. Institutions are not worried about a quick gain as much as a big loss so they short for insurance.
I'm not expecting a big move on the Dyloject UK approval, but I think it will start a nice uptrend for the stock.