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I am glad you know more than the retina specialists I have spoken to.
Novartis Fails to Win U.K. Backing for Lucentis in Diabetic Eye Condition
By Andrea Gerlin - Jul 14, 2011 Novartis AG (NOVN), Europe’s second-largest drugmaker, failed to win approval from the U.K.’s health-cost agency for its drug Lucentis in diabetics who suffer an eye condition.
Lucentis wasn’t cost effective in patients with macular edema, the National Institute for Health and Clinical Excellence said today in a statement. The medicine costs 742 pounds ($1,200) for a monthly injection, NICE said. The agency advises the U.K.’s state-run National Health Service on which treatments represent value for money.
Novartis’s estimates for the cost of the treatment were based on “implausible assumptions” and if more realistic estimates had been made, the cost would have exceeded limits that NICE sets, the agency said. It was unable to recommend the medicine over an existing treatment for diabetics with macular edema, laser therapy.
“Laser therapy has provided stabilization of vision in many patients, but generally does not improve vision, and despite treatment many patients continue to lose vision,” the Basel, Switzerland-based company said in a statement. “Lucentis has been shown to double the likelihood of gaining vision and reduce the chance of losing vision by up to three-fold compared to laser” therapy.
One study found that patients required a median of eight to nine injections of Lucentis in the first year of treatment and two to three in the second year, according to the company.
Novartis said it plans to appeal NICE’s draft recommendations. The agency said it expects to publish a final decision next month.
Diabetic macular edema is caused by leaky blood vessels in the eye. Laser therapy cauterizes the vessels and seals them off, minimizing the leakage. NICE already recommends Lucentis for a separate eye condition, wet age-related macular degeneration.
To contact the reporter on this story: Andrea Gerlin in London at agerlin@bloomberg.net
To contact the editor responsible for this story: Phil Serafino at pserafino@bloomberg.net
Buy Price Target: $26.00 SGEN: ODAC Recommends Accelerated ADCETRIS Approval; Reiterate Buy And $26 PT
THINK ACTION:
We believe unanimous ODAC Panel votes in favor of accelerated ADCETRIS approval will likely result in very near-term FDA approval. In our view, the ODAC Panel appears unsatisfied with the Phase 3 AETHERA serving as a confirmatory study for final, full approval. We continue to believe: 1) the drug is incrementally less risky; 2) near term, SGEN shares should trade up, and; 3) we recommend that investors take advantage of potential and likely temporary share price weakness. We reiterate our Buy rating and our $26 PT.
KEY POINTS:
ADCETRIS ODAC Panel: Unanimous ODAC Panel votes in favor of accelerated ADCETRIS approval for RR Hodgkin's Lymphoma (HL) and Anaplastic Large Cell Lymphoma (ALCL) post-autologous stem cell transplant (ASCT) are very important, risk-reducing events leading toward near-term approval, in our view. We believe the ODAC Panel's suggestion that the Phase 3 AETHERA trial may be unsatisfactory to serve as a confirmatory trial for final and full approval was a surprise that will likely be mitigated, if necessary. Fundamentally, we believe ADCETRIS's profound efficacy and attractive current risk-benefit ratio would not be altered even if new confirmatory trials are warranted beyond the Phase 3 AETHERA study. In our view, the FDA appears interested in negotiating potential, new protocol designs prior to the current August 30 PDUFA. We note that, if agreement is not reached by this junction, it could be by the tentative ODAC meetings scheduled for September 14-15, or December 7-8, or at a new ad hoc meeting. As a reminder, the Phase 3 AETHERA study was initiated in April 2010 and should read out primary, end-point data in mid-2013. If alternative confirmatory trials are needed that are potentially larger and include more patients, we believe results might not become available at least until the 2015-16 timeframe. Whatever the outcome of negotiations with the FDA, we believe: 1) near-term approval is likely; 2) a potential need for new confirmatory trials would not affect our long-term ADCETRIS peak sales estimates ($507M in 2016), and; 3) we recommend investors take advantage of any potential, near-term share weakness. This is our view pending any additional information that could be provided on an SGEN conference call later today.
July 15 8:30AM EST SGEN Conference Call (Dial in 800.762.8779/code 4456034): We expect this conference call to provide SGEN management an opportunity to present its view of the ODAC Panel and possibly guide to potential strategies aimed at further reducing risk in the ADCETRIS story.
If Approved by Aug. 30 PDUFA, We Estimate SGEN Shares Should Trade to $26: We believe SGEN shares are likely to spike up to our $26 PT on potential approval by the Aug-30 PDUFA. We believe ADCETRIS would experience a relatively strong launch given: 1) relatively large number of HL and ALCL patients ready for ADCETRIS and 2) the strength of the SGEN commercial team that is headed by Bruce Seeley, who previously came to SGEN from Genentech where he was responsible for launching blockbuster drugs such as Herceptin.
We Believe High Short Interest Could be Wild-Card: We believe SGEN's high short interest could have incrementally positive implications for our thesis and our $26 PT. Currently, there are nearly 20.8M shares short, or 14.9% of float, that could impact our estimates. Should significant short covering occur during the regulatory approval process, or after, we believe this tailwind may provide a further source of potential upside.
Chardan: Seattle Genetics Inc. (SGEN): SGEN: FDA Panel Recommends Approval for Adcetris; Maintain Sell and $15 PT
FDA Advisory Panel Recommends Accelerated Approval for Adcetris
Yesterday, as universally expected, the FDA's Oncologic Drugs Advisory Committee (ODAC) Advisory Panel recommended accelerated approval for Adcetris to treat patients with relapsed or refractory Hodgkin lymphoma (HL), and relapsed or refractory systemic anaplastic large cell lymphoma (ALCL).
While the positive recommendations remove another hurdle for the drug, we believe that there were two key concerns highlighted during the meeting.
First, it is clear to us that a broader labeling for HL is not even up for discussion. The FDA repeatedly made the point during the morning session that the patients evaluated in the single, single-arm Phase II HL study, were patients with confirmed disease post-transplant with autologous stem cell therapy. Hence, we believe this will be the initial approved labeling for HL. We estimate this is an addressable patient population in the US of only slightly less than 1,000 patients per year.
Second, the accelerated approval will require the Company to conduct additional randomized controlled confirmatory studies, post approval. The discussion during the meeting makes it clear that the FDA has issues with the Company's Phase III AETHERA study. The FDA had previously raised these concerns with the Company at the time that the study was being designed, within the context that the Agency foresaw Acetris receiving accelerated approval, but the Company chose not to resolve them prior to its commencement. One of the FDA issues is the primary endpoint of the study, which is progression-free survival (PFS). The FDA believes that an endpoint of overall survival (OS) conveys more information about safety and efficacy, which it believes is important in the case of accelerated approvals given the limited experience regarding the longer term safety of the drug. In any case, the Company will now need to negotiate a plan for its confirmatory studies with the FDA before the accelerated approvals for both indications are granted. Clearly, we believe that the leverage in these negotiations now shifts to the FDA. Finally, we believe that investors should not ignore the closing remarks by Dr. Pazdur, the Director of the FDA's Office of Oncology Drug Products, in which he stated that the plan for the confirmatory studies is an important part of the accelerated approval process, and failure to reach agreement with the Company regarding the plan could result in the failure to grant approval by the PDUFA date of August 30, in which case the possible need to hold another meeting with the ODAC Advisory Panel to provide guidance on the issue.
Once the drug is approved then the Company's quarterly results will begin to matter. The Company is already incurring significantly higher expense associated with the build-out of its sales and marketing organization. The market opportunity for HL and ALCL is not large to begin with. Hence, if you layer on a more restrictive labeling for only post-transplant patients, and perhaps a longer time to generate additional data to expand the label, we believe that the initial addressable market for Adcetris will be far less than incorporated in current consensus estimates.
IntelGenx: A Fourth Quarter Diamond in the OBB Rough
http://seekingalpha.com/instablog/890923-spencer-knight/191933-intelgenx-a-fourth-quarter-diamond-in-the-obb-rough
Many times companies on the OBB market are forgotten due to the small size. However one company that is poised to have a great fourth quarter of this year is IntelGenx Technologies Corporation (IGXT.OB). IntelGenx focuses on improving existing drugs by adding IntelGenx's novel advanced controlled release technologies. IntelGenx is currently developing drugs that treat depression, osteoarthritis, hypertension, erectile dysfunction, and pain management. As the company continues to expand, investors should not be surprised to see IntelGenx pop up on a major healthcare companies buy list.
In December of 2010, President and CEO of IntelGenx Horst Zerbe spoke about the company and his objectives. When asked how he got started with new drug delivery systems; Zerbe discussed how pharmaceutical films are a much more efficient way for drug delivery. He also mentioned how he developed the Listerine film strips for fresh breath. Furthermore Zerbe stated how applying IntelGenx's novel delivery platforms to pre existing drugs allows the company to bypass phase two and phase three trials most of the time; which saves money.
Zerbe was also asked about the current market of pharmaceutical films and how these films work. Zerbe stated the market for pharmaceutical films is still very new as less than a handful of companies are working on pharmaceutical films. Also, two recently developed films for cough and cold in children have been approved for Novartis (NVS). He also mentioned how films make the drug more bioavailable and uptake is quicker as the medicine dissolves faster.
Zerbe gave the most speculative answer of the interview when asked what films the company currently has in development. Zerbe stated IntelGenx portfolio of products has a "large blockbuster potential and targets a combined market in the tens of billions of dollars." I wont say the combination of erectile dysfunction, migraine, insomnia, bipolar disorder, and pain management isn't a $10 billion market; but I think the numbers are a bit speculative at this point. I may be wrong 5-10 years from now, but currently over $10 billion potential is speculative rabble.
As I previously mentioned, IntelGenx will make a run in the fourth quarter. This is because the company has a PDUFA set for November 13, 2011. The NDA for CPI-300 was submitted in May of 2010. Briefly, CPI-300 is an anti-depressant to treat major depressive disorder (MDD) and contains a high strength form of Bupropion HCl. CPI-300 was rejected in February of 2010 due to some food affects and manufacturing issues. The manufacturing issue was an easy fix as the company simply had to fix the problems at a specific facility. In terms of the food affect issues, IntelGenx is confident a label specification and restrictions will lead to an approval.
As the PDUFA date approaches expect a long drawn out run that could see the share price stay around the 0.90-1.10 range. The current 52 week high is the lower end of this scale and it should be eclipsed as November gets closer. We may see the all time high eclipsed on approval as well. IntelGenx's all time high is about 1.58 from August of 2007. Also, as November becomes more near, we may see investment firms initiate coverage; which would cause an instant share price jump. IntelGenx is a stock to watch in the fourth quarter, and I recommend doing research now to see if this company is right for your portfolio.
Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.
Stocks: IGXT.OB
from memory I think it is 2014
kei
who cares about arena's volume. what does the company have that you find any value in?
confirms the value or aryx's drug but the management screwed that up royally
from cytr, the Company’s Bcr-Abl, Lyn and Fyn kinase inhibitor
the drugs for cml targeet bcr-abl, it must be the lyn and fyn kinase target that works for cll
their drug treated a few of the cml subgroups that standard of care didn't treat except the t315i mutation.
If looks like ponatinib works in all the mutations so why develop it for cml.
Today is Day Two of the FDA's hearings on Avastin for metastatic breast cancer. (Note: if you want to follow things in near real time, I'd suggest a Twitter search for #Avastin. I can particularly recommend Len Lichtenfeld's feed.) This has been a very contentious issue; as most of you know, Avastin was provisionally approved for these patients, then pulled when more trial data came in showing no benefit. Roche (RHHBY.PK)/Genentech's team is now appealing that decision, and the questions are:
Should Avastin be approved for metastatic breast cancer patients? The answer to this one is "depends on the evidence for it." So ...
Is there enough evidence to decide one way or another? Both the FDA and Roche seem to think that there is. The problem's that they come to opposite conclusions. So ...
What's the risk/benefit ratio for Avastin in these patients? Now the serious arguing starts. Avastin is not without its serious side effects -- but metastatic breast cancer is a terrible disease. The initial reports were promising, but none of the larger follow-up trials have really confirmed those results. Genentech is proposing still another confirmatory trial, with the drug to stay approved during that period, but the FDA seems to be arguing that leaving the drug approved for this indication will hurt more people than it helps.
And all of this is being done against a backdrop of emotional cancer patient testimony. The problem with that is summed up by one of the most fervent advocates, Patricia Howard, who told the FDA: "I’m not just a statistic; it is in your hands to ensure that I don’t become one."
It pains me to say this, but she's wrong. If we're ever going to get anywhere with cancer (or any other disease), we're going to need all the statistics we can get our hands on, and no amount of passionate testimony should be allowed to move one number in them. I've had family members with cancer; I've seen good friends and plenty of good people die from cancer. But cancer cells do not care about how strong your feelings are. The growth factor receptors, the checkpoint kinases, the apoptosis regulators, the metabolic enzymes and cell adhesion proteins: They don't give a damn; they have no damn to give. We have to fight them on those terms, on that battlefield, because that's the only one that matters and the only one where they can be defeated.
As it stands, I agree with the FDA's position: I don't think that Avastin has been shown to offer enough benefit. The 2008 provisional approval was already arguable -- the agency went against its own advisory committee just to do that much -- and the subsequent data have made it even less tenable. If we're going to have provisional approvals, then they have to be able to be taken back. And if we're going to evaluate drugs by their risks versus their benefits, then Avastin -- for this indication, in these patients -- doesn't (to my eyes) seem to make the cut.
If, on the other hand, you disagree with the provisional approval process, fine. Propose something more useful. If you disagree with the risk/benefit analysis in this case, then you should bring some new numbers or some new arguments (which is what Genentech is trying to do right now, as I write this; I hope that it doesn't slip over the line while doing it). If you disagree with the whole idea of risk/benefit analysis, then ... well, you'd better have something more useful to offer. And you'd better be sure that it doesn't end with the decisions going to whoever is the most passionate and tearful in making their case. That won't end well.
One more side issue: You'll note that I've done this whole blog post without talking about the price of Avastin. That's because I don't think that the price is the issue at all here. This is not a healthcare-rationing issue, no matter how much some people would like for it to be. Roche gets to charge what it thinks Avastin can bring; it and Genentech have put the time, effort, and money into the drug. But for metastatic breast cancer, as I said here, Avastin doesn't seem like a good idea even if it were free.
Disclosure: None
I used the neptune product for 3 months. it reduced my triglycerides, but they were already normal, it did not reduce my ldl which was supposed to be their claim to fame
I have had better results with long acting niacin
Still don't get how they get SNGX out of Soligenix. A bit of a nitpick from me.
they requested the symbol sgnx but nasdaq screwed up the symbol.
Doesn't it follow that the commercial upside is limited given this practice as well?
the product compounded in the formularies is the immediate release pills. They also have an extended release pill that extends the treatment to the area lower down in the gut. That treats the entire gi system.
Genentech Scoops Up Tumor-Starving Drug Program From Forma Therapeutics In Rare Deal
http://www.xconomy.com/boston/2011/06/27/genentech-scoops-up-tumor-starving-drug-program-from-forma-therapeutics-in-rare-deal/
Genentech doesn’t acquire many drugs from other people, partly because it has a prolific internal R&D operation. But the South San Francisco-based unit of Roche, the world’s biggest maker of cancer treatments, is announcing an unusual deal today in which it is paying to get an early-stage cancer drug program from Cambridge, MA-based Forma Therapeutics that could enable Forma to generate returns for its investors without going public or getting acquired.
Here’s the deal: Forma is handing over the exclusive worldwide rights to a small-molecule drug program—still in early-stage animal testing—that’s designed to starve tumors by blocking a molecular target involved in cancer cell metabolism. In return, Genentech is making an upfront payment, providing support for research, covering all the development costs, and agreeing to make milestone payments if the drug hits certain development goals. That’s not unusual, but what comes next is: If the Forma drug reaches its development goals, Genentech has the option to acquire the full rights to the asset, leaving Forma with no royalty stream. If Genentech exercises that option, it would make an asset buyout payment that would be distributed to Forma’s investors, plus further milestone payments to Forma if certain sales goals are met.
What it means is that if this drug pans out, Forma essentially would be able to deliver returns to its venture backers by selling off just one of 8-10 cancer metabolism targets it has discovered so far, and remain an independent company free to discover and develop as many other drugs as it can. What’s more, in this scenario, investors will get a return on their investment from Genentech without having to hand over their equity stakes. So it’s conceivable the VCs could get a return on one of Forma’s drugs, and then potentially an even bigger payday if the startup goes public or gets acquired. The agreement is also another sign of emerging interest in the cancer metabolism field, which many Big Pharma companies are pursuing, and which sparked a big deal last August when Cambridge, MA-based Agios Pharmaceuticals secured a $130 million upfront payment from Summit, NJ-based Celgene (NASDAQ: CELG).
“This deal gives us the best of both worlds,” says Forma CEO Steven Tregay. “It gives us near-term funding to continue to build our capabilities up for our pipeline, while at the same time giving us a well-defined exit for our investors down the road.” Tregay wouldn’t divulge any specific financial terms, but he said Forma was burning about $10 million a year in cash prior to the Genentech deal, and that “we now have a pretty nice cash horde going forward.”
Steven Tregay
James Sabry, the vice president of Genentech Partnering, said in a statement that Genentech “is very pleased to enter into this structurally innovative agreement with Forma. The program represents a promising addition to our portfolio.”
This deal is the latest sign that Forma was onto something when it staked out a contrarian drug discovery strategy when it was founded about three years ago, at a time other companies were cutting research in the depths of the recession. Tregay, a former managing director at the Novartis Option Fund, teamed up with scientists from the Broad Institute to put together a startup with specialized skills needed for a soup-to-nuts drug discovery operation. The company has secured about $33 million in equity financing from the likes of Novartis Option Fund, Lilly Ventures, and Bio*One Capital of Singapore, plus another $45 million in non-dilutive cash from Cubist Pharmaceuticals, Eisai Pharmaceuticals, the Leukemia & Lymphoma Society, and the Experimental Therapeutics Centre of Singapore, Tregay says.
The money has given Forma enough of a cushion to hire about 90 people who do things like X-ray crystallography work to characterize 3-D structures of protein targets; high-throughput screening of drug candidates; and synthesize new experimental drugs—as well as form a computational group and pharmacology team. By getting all those people together in one company, Forma has bucked the industry trend, in which many Big Pharma and biotech companies have turned to low-cost outsourcing firms. By putting together its own discovery team, Forma been able to develop drug programs against 8-10 new and different cancer metabolism targets, and identify new targets that work against protein-protein interactions, Tregay says.
By forming an integrated discovery team—at a moment when Big Pharma has been hungry for truly innovative new drug candidates—Forma put itself in a strong bargaining position. In this case, Forma juggled multiple offers for its cancer drug metabolism program and got the asset-buyout structure it wanted, with a partner that has a strong track record in developing cancer drugs, Tregay says.
Certainly, this deal like anything else, has its trade-offs. If Genentech goes on to develop this compound into a multi-billion product like bevacizumab (Avastin), then Forma won’t have any piece of the action in royalties, and the asset buyout terms will probably look small, Tregay acknowledged. Essentially, the deal caps Forma’s upside potential with this drug. But that concession was worthwhile when considering the returns it can earn, while retaining flexibility to discover and develop other innovative drugs, Tregay says.
The structure of this deal, which Tregay worked on with Sabry, is something you can bet other entrepreneurs will be inquiring about how to duplicate. I’ve written here about some of the different models biotech companies are experimenting with, as a way to keep innovation humming along in a period when the IPO market is lackluster and acquisitions are rare, making it extremely tough for investors to get any realistic shot at a decent return.
The fact that a deal got done on a drug that’s just been tested in mice—and still doesn’t have a target date to enter clinical trials—suggests that biotech companies like Forma are suddenly holding a better set of cards at the negotiating table than they did a year or two ago. Tregay offered up an amusing riff on how the dealmaking environment has heated up in the past few months. Essentially, Big Pharma doesn’t care how much money a biotech company needs to get to provide a return to investors—but it does want innovative drugs to fill up its pipeline, and it will pay for them.
“They won’t look at you and say, ‘Yeah, I feel your pain, I’ll pay up.’” Tregay says. “You have to be in a situation where you have multiple parties who are highly interested in a highly differentiated asset. It has to be one where they feel like if they don’t get it from you, they can’t just get it from the next guy. Or, they can’t already have something like it in-house, and just want to see if yours is better. You really have to bring true innovation and value to them. If you bring that value proposition to them, you can let market forces set the price.”
Here is a 2009 paper in a peer-reviewed ophthalmic journal showing that an eye drop administration of nipradilol gets to the choroid and retina in the back of the eye by diffusion from the front of the eye, in a manner very similar to the squalamine formulation :
http://www.iovs.org/content/50/6/2839.long
oxgn's IV data in AMD or cancer was not as good as squalamine, so I wouldn't expect it to work through the eye.
If you remember squalamine's data was better than visudyne but not as good as lucentis.
are there other small molecules that inhibit vegf and pdgf that have been delivered as an eyedrop?
The data shows that squalamine had an effect in an iv formulation, they just couldn't get enough into the back of the eye.
We'll see said the blind man
the reason I sent you the reply was because I came across an old post that you made saying that you would like more detail about acuity's drug but you doubted you would get anywhere because the company was private.
Magainin changed their name to Genaera. This biotech values board was originally the Genaera board. It was one of my biggest financial bonanzas.
They had good data in wet amd treating both eyes through an iv infusion. The data was not as good as Lucentis so there was no way it was going to get approved. The drug was given as a once weekly infusion for 4 weeks, then a person received an infusion once a month. It would not have been practical to come into the office once a week for the infusion. The problem was that the drug didn't stay in the back of the eye for more than 4 or 5 days so vision improvement only lasted for the first month.
Even with this suboptimal dosing there was stabilization of vision at the longer period of time but you didn't get the improvement seen in Lucentis.
This company bought the drug out of bankrupcy and they were able to get it made as an eyedrop because squalamine is a small molecule. The patient can now be treated on a daily basis and get the drug into the back of the eye in sufficient amounts. I guess we will see if it is a case of Zebra's Law.
Ohr Pharmaceutical Announces Clinical Squalamine Eye Drop Program for Wet-AMD
Topical Treatment of Wet-AMD Offers Several Advantages Over Current Standard-of-Care
:Ohr Pharmaceutical, Inc. Related Quotes
Symbol Price Change
OHRP.OB 0.42 0.00
Press Release Source: Ohr Pharmaceutical Inc. On Tuesday June 21, 2011, 8:15 am
NEW YORK, NY--(Marketwire - 06/21/11) - Ohr Pharmaceutical Inc. (OTC.BB:OHRP - News) announced today that it is advancing its clinical Wet Age-related Macular Degeneration ("Wet-AMD") program with a novel topical formulation. Using its proprietary technology, Ohr reformulated Squalamine for ophthalmic indications from an intravenous infusion ("IV") to a topical eye drop. The topical formulation is designed for enhanced uptake to the back of the eye and decreased potential for side effects. The previous IV formulation had been awarded fast track status and a Special Protocol Assessment ("SPA") by the U.S. Food and Drug Administration for a Phase III registration study in patients with Wet-AMD.
"The Squalamine eye drop program has the potential to create a monumental shift in the way patients are treated for Wet-AMD," commented Dr. Shalom Z. Hirschman, M.D., Ohr's Chief Scientific Advisor.
Squalamine eye drops may offer several potential competitive advantages over Lucentis®, the current standard-of-care ("SOC"), a product with global revenues in excess of $1.5 billion annually.
SOC requires monthly injections directly into the eye; Squalamine delivered topically can be conveniently self-administered by the patient on a daily basis
SOC has the propensity for side effects and potential inherent complications of an intravitreal injection; Squalamine has shown a good safety profile even when administered systemically in significantly higher doses
Broad-spectrum inhibition of multiple angiogenic growth factors in addition to VEGF including PDGF
Squalamine exhibited efficacy in more advanced Wet-AMD ("fellow eye") in previous clinical trials
"We are delighted to be moving ahead with Ohr's Wet-AMD clinical program with a very patient-friendly method of treatment," stated Irach B. Taraporewala, Ph.D., Chief Executive Officer of Ohr Pharmaceutical. "There is an immense potential market for such a product given the incidence of Wet-AMD and the rapidly aging population in the United States and throughout the world."
About Squalamine
Squalamine is a small molecule anti-angiogenic with a novel intracellular mechanism of action, that counteracts not only Vascular Endothelial Growth Factor ("VEGF") but also other angiogenic growth factors such as Platelet Derived Growth Factor ("PDGF"). Recent clinical evidence has shown PDGF to be an additional key target for the treatment of Wet-AMD. Using the intravenous formulation in over 250 patients in Phase 1 and Phase 2 trials for the treatment of Wet-AMD, Squalamine showed good safety and efficacy in both early and advanced Wet-AMD.
About Wet Age-Related Macular Degeneration
Wet-AMD is a medical condition which usually affects older adults and generally results in a loss of vision. AMD occurs in "dry" and "wet" forms. The wet form (Wet-AMD) accounts for approximately 15 percent of all AMD, yet is responsible for 90 percent of severe vision loss associated with AMD. According to the National Eye Institute (NEI), the prevalence of Wet-AMD among adults 40 years or older in the U.S. alone is estimated at 1.75 million people. In addition, more than 200,000 new cases are diagnosed yearly in the U.S.
About Ohr Pharmaceutical Inc
Ohr Pharmaceutical Inc. (OTC.BB:OHRP - News) (www.ohrpharmaceutical.com) is a public pharmaceutical development company dedicated to the clinical development of new drugs for underserved therapeutic needs in large and growing markets. The company is focused on two lead compounds: Topical Squalamine eye drops for the treatment of the wet form of age-related macular degeneration, and OHR/AVR118 for the treatment of cancer cachexia, currently being investigated in a Phase II trial.
Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995:
This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are made only as the date thereof, and Ohr Pharmaceutical undertakes no obligation to update or revise the forward-looking statement whether as a result of new information, future events or otherwise. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties, including the future success of our scientific studies, our ability to successfully develop products, rapid technological change in our markets, changes in demand for our future products, legislative, regulatory and competitive developments, the financial resources available to us, and general economic conditions. For example, there can be no assurance that Ohr will be able to sustain operations for expected periods. Shareholders and prospective investors are cautioned that no assurance of the efficacy of pharmaceutical products can be claimed or assured until final testing; and no assurance or warranty can be made that the FDA or Health Canada will approve final testing or marketing of any pharmaceutical product. Ohr's most recent Annual Report and subsequent Quarterly Reports discuss some of the important risk factors that may affect our business, results of operations and financial condition. We disclaim any intent to revise or update publicly any forward-looking statements for any reason.
Contact:
Contact:Ohr Pharmaceutical Inc.Sam BackenrothVice President, Business Development212-682-8452Email Contact
I only posted the article because it was picked up by feurstein before the companies put out a release.
I do not think the drug will be a big seller but the stock did well today.
I think that was in cancer
Pluristem and United Therapeutics Enter Into an Exclusive License Agreement to Develop and Commercialize PLX Cells for the Treatment of Pulmonary Hypertension
PSTI 2.9750 0.00
Press Release Source: Pluristem Therapeutics Inc. On Monday June 20, 2011, 4:00 am
HAIFA, Israel, June 20, 2011 (GLOBE NEWSWIRE) -- Pluristem Therapeutics Inc. (Nasdaq:PSTI - News) (TASE:PLTR - News) today announced that its wholly owned subsidiary, Pluristem Ltd., has entered into an exclusive out-license agreement with United Therapeutics Corporation (Nasdaq:UTHR - News) for the use of Pluristem's PLacental eXpanded (PLX) cells to develop and commercialize a cell-based product for the treatment of Pulmonary Hypertension (PH).
Under the terms of the agreement, United Therapeutics will receive exclusive worldwide licensing rights for the development and commercialization of the future product for treating PH patients. Pluristem will retain all manufacturing rights; participate in the pre-clinical and clinical trial activities, as well as provide the commercial grade product.
Under the terms of the agreement, United Therapeutics will make an upfront payment of $7 million to Pluristem. Pluristem is eligible to receive regulatory milestone payments and other payments accumulating together with the upfront payment to a total of approximately $55 million and reimbursement of costs of its development and clinical activities. United Therapeutics will bear all the costs of conducting the clinical trials for this indication. Following commercialization, United Therapeutics shall purchase commercial supplies from Pluristem at a specified margin over Pluristem's cost. In addition, United Therapeutics will pay Pluristem specified royalties as a percentage from its gross profits generated from the developed product.
"This is an important milestone for our company, as it exemplifies our belief that Pluristem's PLX cells are a platform technology that can be used for the treatment of numerous diseases. This agreement is in line with our strategy of being a state of the art cell manufacturer while maintaining all the production and Intellectual Property rights for future product candidates," said Zami Aberman, Chairman and CEO of Pluristem. "We are very pleased to partner with United Therapeutics, an expert and a leader in the area of PH, and to cooperate with their talented team to quickly bring this product to market and improve the quality of life of Pulmonary Hypertension patients."
"Our mission to help patients suffering from Pulmonary Hypertension has led us to seek companies exhibiting innovative approaches and cutting edge technology, with whom we could partner for developing new therapies. Pluristem's impressive results of their current clinical trials, their strong intellectual property and unique manufacturing capabilities, convinced us to select Pluristem as our partner in developing an important cell therapy for treating PH," said Roger Jeffs, President and Chief Operating Officer of United Therapeutics. "We are excited to enter into this partnership and to work alongside Pluristem in advancing this exciting platform."
The signing ceremony will be held tomorrow, June 21st at 09:30 Israel time at the Tel Aviv Stock Exchange in the presence of Dr. Roger Jeffs, President and Chief Operating Officer of United Therapeutics, and Mr. Zami Aberman, Chairman and CEO of Pluristem Therapeutics.
Closing of the agreement is subject to certain closing conditions and is expected by the end of August 2011.
About Pulmonary Hypertension (PH)
Pulmonary Hypertension is the damage that occurs to the pulmonary vessels (leading from the heart to the lungs) when the blood pressure in those vessels is abnormally high. The disease can be secondary to other conditions or unrelated to any identifiable disorder. Approximately 1,000 new cases of this catastrophic disorder are diagnosed annually in the US*.
*National Library of Medicine
About United Therapeutics Corporation
United Therapeutics Corporation (Nasdaq:UTHR - News) is a biotechnology company focused on the development and commercialization of unique products to address the unmet medical needs of patients with chronic and life-threatening conditions, including cardiovascular, cancer, and infectious diseases.
About Pluristem Therapeutics
Pluristem Therapeutics Inc. (Nasdaq:PSTI - News) (TASE:PLTR - News) is a leading developer of placenta-based cell therapies. The company's patented PLX (PLacental eXpanded) cells drug delivery platform releases a cocktail of therapeutic proteins in response to a host of local and systemic inflammatory diseases. PLX cells are grown using the company's proprietary 3D micro-environmental technology and are an off-the-shelf product that requires no tissue matching prior to administration. Data from two phase I studies indicate that Pluristem's first PLX product candidate, PLX-PAD, is safe and potentially effective for the treatment of end stage peripheral artery disease. Pluristem's pre-clinical animal models have demonstrated PLX cells are also potentially effective in nerve pain and muscle damage, when administered locally, and in inflammatory bowel disease, MS and stroke, when administered systemically.
Pluristem has a strong patent portfolio, GMP certified manufacturing and research facilities, strategic relationships with major research institutions and a seasoned management team.
For more information visit www.pluristem.com, or follow us on Twitter @Pluristem, the contents of which are not part of this press release.
The Pluristem Therapeutics Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=6882
Safe Harbor Statement
This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995 and federal securities laws. For example, we are using forward looking statements when we discuss the belief that our PLX cells are a platform technology that can be used for the treatment of numerous diseases, when we discuss the timing to bring this product to market and its ability to improve the quality of life of Pulmonary Hypertension patients, when we say that closing of the agreement with United Therapeutics is expected by the end of August 2011 and imply that we may receive future payments if our products are commercialized and generate gross profits or when we say that data from two Phase I clinical trials indicate that Pluristem's first PLX product, PLX-PAD, is safe and potentially effective for the treatment of end stage PAD or that Pluristem's pre-clinical animal models have demonstrated PLX cells are also potentially effective in nerve pain and muscle damage when administered locally and in inflammatory bowel disease, MS and stroke when administered systemically. These forward-looking statements are based on the current expectations of the management of Pluristem only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; we may encounter delays or obstacles in launching our clinical trials; our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; our products may wind up being more expensive than we anticipate; results in the laboratory may not translate to equally good results in real surgical settings; our patents may not be sufficient; our products may harm recipients; changes in legislation; inability to timely develop and introduce or commercialize new technologies, products and applications; loss of market share and pressure on pricing resulting from competition, which could cause the actual results or performance of Pluristem to differ materially from those contemplated in such forward-looking statements. Except as otherwise required by law, Pluristem undertakes no obligation to publicly release any revisions to these forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. For a more detailed description of the risks and uncertainties affecting Pluristem, reference is made to Pluristem's reports filed from time to time with the Securities and Exchange Commission.
Contact:
Investor Relations Contacts:Pluristem:William Prather R.Ph., M.D.Sr. VP Corporate Development1-303-883-4954William.PratherMD@pluristem.comDaya LettvinInvestor & Media Relations Director+972-54-647-5580daya@pluristem.comMedia Contact:Pluristem:Matthew KriegerRuder Finn - for Pluristem+972-54-467-6950matthew@ruderfinn.co.il
Pfizer, Acura Painkiller Approved by FDA
Adam Feuerstein
06/19/11 - 04:47 PM EDT
TOWN, STATE (TheStreet) -- U.S. regulators approved a new tamper-resistant painkiller from Pfizer(PFE) and Acura Pharmaceuticals(ACUR) Friday night, but you wouldn't have known it unless you were logged on to Twitter.
Oxecta is a short-acting painkiller consisting of the narcotic oxycodone mixed with ingredients developed by Acura that make it hard for drug addicts to crush or dissolve the tablet to get a quick high. Pfizer will sell Oxecta -- formerly known as Acurox-- in the U.S. for which Acura receives milestone payments and royalties based on sales.
The U.S. Food and Drug Administration approved Oxecta late Friday, confirmed spokeswoman Karen Riley, but the agency chose not to make a public statement. Pfizer and Acura didn't say anything publicly about Oxecta's approval on Friday either, presumably deciding to wait for Monday morning to issue a press release.
With none of the involved parties willing to speak about Oxecta, the public announcement of the drug's approval was made by Geoff Chambers, an economist and biotech stock blogger who tweets under the handle @Gekkowire.
Chambers tweeted "$ACUR approved" at 8:46 pm ET after getting an email confirmation from FDA spokeswoman Riley.
Acura shares closed Friday's regular session down 5% to $3.87. The stock fell another 6% to $3.23 in after-hour trading when it seemed as if there would be no announcement about Oxceta's approval.
Acura's stock price is sure to rebound and move higher when trading opens Monday. How much so likely depends on the data and language FDA allowed in the Oxecta label describing the drug's tamper-resistant qualities.
FDA does not allow claims of abuse deterrence without data from long-term epidemiological studies that can demonstrate reductions in drug abuse. What FDA will allow, however, is data and language describing the tamper-resistant qualities of the drug and results from studies that simulate abuse.
Oxecta is the first immediate-release opioid approved for the relief of moderate-to-severe pain that is also designed to deter common methods of misuse and abuse. The "typical" patient for such a drug would be someone who just had root canal or some other type of surgery and needs a painkiller for a short period of time -- a few days or up to one month.
Relatively cheap generic drugs currently dominate the market for short-acting opioid pain relievers, so Pfizer and Acura will need the drug's label to adequately describe Oxecta's tamper resistance in the drug's label in order to justify premium pricing.
Extended release, or long-acting, opioids that also aim to deter abuse are already on the market for patients who suffer from chronic pain. Purdue Pharma sells a tamper-resistant form of its popular Oxycontin. Pfizer and Pain Therapeutics(PTIE) expect an FDA approval decision on June 23 for Remoxy, another long-acting abuse-resistant opioid.
Acura has been trying to get Oxecta, then known as Acurox, approved since January 2009. FDA first rejected the drug in June 2009. In April 2010, an FDA advisory panel voted against recommending the drug's approval. Acura and King Pharmaceuticals reformulated Oxecta/Acurox and resubmitted for approval in December 2010. Later, Pfizer acquired King and took over marketing responsibilities for the Acura partnership.
Pfizer will pay royalties ranging from 5% to 25% to Acura, based on levels of sales of Oxecta and other drugs included in the partnership.
--Written by Adam Feuerstein in Boston.
the trials didn't work but it was only one ingredient and it needed many ingredients
If anyone actually believes he was in this for the good of humanity, all he had to do was find any biotech with some resources to get the ball going.]
If you watched the movie he tried to do a deal with Elan, they backed out saying that he couldn't patent the one of the ingrediants and then they patented the ingredient and ran trials without him
Has anyone ever heard of this
http://articles.mercola.com/sites/articles/archive/2011/06/11/burzynski-the-movie.aspx
Bill Clinton: universal health coverage saves money
I left the conference before he spoke. It is a pretty well attended conference
Former U.S. President Bill Clinton speaks at the 2011 Fiscal Summit on Solutions for America's future in Washington, May 25, 2011. REUTERS/Jason Reed
On Tuesday June 7, 2011, 5:42 pm EDT
By Bill Berkrot
NEW YORK (Reuters) - Former President Bill Clinton said the United States could save more than $1 trillion a year by adopting any other advanced nation's healthcare system.
He also said there are important advances included in President Obama's healthcare reforms and urged that it be improved upon rather than repealed.
"Our healthcare system has gotten all out of whack," Clinton said in a speech on Tuesday at the Jefferies Global Healthcare Conference, stressing the need to bring inflation in healthcare costs back in line with economic inflation.
Clinton said Canada and the European countries that have universal health coverage for their citizens spend a smaller percentage of their gross domestic product on healthcare than the United States does.
"Germany and France, with what is considered the most effective systems in the world in terms of universal coverage and quality of treatment, they spend 10 percent. Canada spends 10.5 percent," Clinton said.
"The United States spends 17.2 percent without having universal coverage," Clinton said.
"That means if we just scrapped our system and adopted any other wealthy country's system, at a minimum we would have a trillion dollars more a year for pay raises, for investment in new technology, to create new jobs or whatever."
He said resistance to change, in addition to political maneuvering, is part of what is keeping the government Medicare program from any meaningful reforms.
"The beneficiaries in the present would rather hold onto the present than make sure they've done what's necessary to preserve it for the future, and that's part of the problem with Medicare," Clinton said.
He said Social Security, which many people fear could go bankrupt if changes are not adopted before the bulk of the giant baby generation retires, would eventually fix itself.
"Social Security is primarily just a demographics problem," Clinton said, noting that not enough people will be working to pay for all the members of his generation collecting Social Security payments.
"Although if the economy stays this anemic we'll all be working too and that's not all bad, you'll live longer if you do," he said.
"But when we all die off that will all be fixed," he quipped.
(Reporting by Bill Berkrot; Editing by Gary Hill)
acuity's drug is in opko health
http://www.opko.com/research/?doc=ophthalmics
thank you for this,
I never knew about this company.
I will ask some analysts that I know to see if they have ever seen this data
This looks like a real warning
Kids are putting Drano, tin foil, and a little water in soda bottles and capping it up - leaving it on lawns. When you go to pick up the trash, and the bottle is shaken just a little - in about 30 seconds or less it builds up a gas and explodes with enough force to remove some of your extremities. The liquid that comes out is boiling hot as well.
Don't pick up any plastic bottles that may be lying in your yards or in the gutter, etc. Pay attention to this. A plastic bottle with a cap. A little Drano. A little water. A small piece of foil. Disturb it by moving it; and BOOM!! No fingers left and other serious effects to your face, eyes, etc.
People are finding these "bombs" in mailboxes and in their yards, just waiting for you to pick it up intending to put it in the trash. But, you'll never make it!!! It takes about 30 seconds to blow after you move the thing.
See "SNOPES" below -- it's true -- the video at SNOPES shows the Indiana State Police Bomb Squad detonating one -- it's truly horrifying! .... .. I checked "Truth or Fiction" and "they" agree this is TRUTH!!! So be warned and beware.
http://www.snopes.com/crime/warnings/bottlebomb.asp
What if the fda now says that they will not approve any cv drug without an outcomes study.
amrn would have a huge problem
I am sending an email to my friends asking why I am taking slow release Niacin
NIH pulls plug on AIM-HIGH trial with niacin
May 26, 2011 | Lisa Nainggolan
Bethesda, MD - A trial of extended-release niacin (Niaspan, Abbott) given in addition to statin therapy in patients with a history of cardiovascular disease, high triglycerides, and low levels of HDL cholesterol has been halted prematurely, 18 months ahead of schedule, because niacin offered no additional benefits in this patient population [1].
There was also a small, unexplained increase in ischemic stroke in the high-dose, extended-release niacin group, in the Atherothrombosis Intervention in Metabolic Syndrome with Low HDL Cholesterol/High Triglyceride and Impact on Global Health Outcomes (AIM-HIGH) study, according to a statement from the National Heart Lung and Blood Institute (NHLBI), which sponsored it.
Despite treatment with statin therapy for elevated LDL-cholesterol levels, those with low levels of HDL cholesterol remain at significant risk for cardiovascular events, and AIM-HIGH was designed to examine whether raising HDL using extended-release niacin would be beneficial in such patients. AIM-HIGH was a five-year study of almost 3500 patients, and results were originally expected in September 2012.
The decision to stop the trial was made at a regularly scheduled meeting of the study's independent data and safety monitoring board (DSMB) on April 25, 2011. The DSMB concluded that "high-dose, extended-release niacin offered no benefits beyond statin therapy alone in reducing cardiovascular-related complications in this trial. The rate of clinical events was the same in both treatment groups, and there was no evidence that this would change by continuing the trial."
Patients should not stop taking niacin
The NHLBI explains that the rationale for AIM-HIGH was based on data from observational studies and a few small clinical studies. "This study sought to confirm earlier and smaller studies," says Dr Susan B Shurin (acting director of the NHLBI) in the statement. "Although we did not see the expected clinical benefit, we have answered an important scientific question about treatment for cardiovascular disease."
Several other trials testing this hypothesis, including a large international trial of high-dose, extended-release niacin, the Heart Protection Study 2 Treatment of HDL to Reduce the Incidence of Vascular Events (HPS2-THRIVE), are still under way; results of HPS2-THRIVE are expected in 2013. Extended-release formulations of niacin are being used in these trials because the immediate-release form of niacin is associated with a high incidence of flushing.
Previous studies do not suggest that stroke is a potential complication of niacin, and it remains unclear whether this trend in AIM-HIGH arose by chance or was related to niacin administration or some other issue, says the NHLBI. "Patients who were not in the AIM-HIGH trial should not stop taking high-dose, extended-release niacin without talking to their doctor first," says Shurin.
All AIM-HIGH study participants have been informed of the results and will be scheduled for clinic visits within the next 2.5 months. Participants will be followed for an additional 12 to 18 months.
AIM-HIGH co-principal investigator Dr Jeffrey Probstfield (University of Washington, Seattle) said: "The lack of effect on cardiovascular events is unexpected and a striking contrast to the results of previous trials and observational studies. The AIM-HIGH findings do not support the trial's hypothesis that, in the population studied, adding extended-release niacin to simvastatin in participants with well-controlled LDL cholesterol can provide additional clinical benefit."
AIM-HIGH enrolled 3414 participants in the US and Canada with a history of cardiovascular disease, low HDL cholesterol, and high triglycerides, who were all prescribed simvastatin and who were also randomized to either high-dose, extended-release niacin in gradually increasing doses up to 2000 mg per day (n=1718) or placebo (n=1696). Of the participants, 515 were given a second LDL-cholesterol-lowering drug, ezetimibe (Zetia, Merck/Schering-Plough), in order to maintain LDL-cholesterol levels at the target range between 40 and 80 mg/dL.
Participants who took high-dose, extended-release niacin and statin treatment had increased HDL cholesterol and lowered triglyceride levels than participants who took a statin alone. However, the combination treatment did not reduce fatal or nonfatal MI, strokes, hospitalizations for acute coronary syndrome, or revascularization procedures.
During the 32-month follow-up period, there were 28 strokes (1.6%) reported among participants taking high-dose, extended-release niacin vs 12 strokes (0.7%) in the control group. Nine of the 28 strokes in the niacin group occurred in participants who had discontinued the drug at least two months and up to four years before their stroke.
Therapeutic use Owing to the highly corrosive action of the acid, only the salts of dichloroacetic acid are used therapeutically, including its sodium and potassium salts, sodium dichloroacetate and potassium dichloroacetate.
http://en.wikipedia.org/wiki/Dichloroacetic_acid
[edit] Lactic acidosisThe dichloroacetate ion stimulates the activity of the enzyme pyruvate dehydrogenase by inhibiting the enzyme pyruvate dehydrogenase kinase.[8] Thus, it decreases lactate production by shifting the metabolism of pyruvate from glycolysis towards oxidation in the mitochondria. This property has led to trials of DCA for the treatment of lactic acidosis in humans.[9][10][11][12]
A randomized controlled trial in children with congenital lactic acidosis found that while DCA was well tolerated, it was ineffective in improving clinical outcomes.[10] A separate trial of DCA in children with MELAS (a syndrome of inadequate mitochondrial function, leading to lactic acidosis) was halted early, as all 15 of the children receiving DCA experienced significant nerve toxicity without any evidence of benefit from the medication.[11] A randomized controlled trial of DCA in adults with lactic acidosis found that while DCA lowered blood lactate levels, it had no clinical benefit and did not improve hemodynamics or survival.[12]
Thus, while early case reports and pre-clinical data suggested that DCA might be effective for lactic acidosis, subsequent controlled trials have found no clinical benefit of DCA in this setting. In addition, clinical trial subjects were incapable of continuing on DCA as a study medication owing to progressive toxicities.
[edit] Potential cancer applicationsCancer cells generally use glycolysis rather than respiration (oxidative phosphorylation) for energy (the Warburg effect), as a result of hypoxia that exists in tumors and damaged mitochondria.[13] Usually dangerously damaged cells kill themselves via apoptosis, a mechanism of self-destruction that involves mitochondria, but this mechanism fails in cancer cells.
A phase one study published in January 2007 by researchers at the University of Alberta, who had tested DCA on cancer cells grown in mice, found that DCA restored mitochondrial function, thus restoring apoptosis, allowing cancer cells to self-destruct and shrink the tumor.[14]
These results received extensive media attention, beginning with an article in New Scientist titled "Cheap, ‘safe’ drug kills most cancers".[15] Subsequently, the American Cancer Society and other medical organizations have received a large volume of public interest and questions regarding DCA.[16] Clinical trials in humans with cancer have not been conducted in the USA and are not yet final in Canada, emphasizing the need for caution in interpreting the preliminary results.[16][17]
[edit] Results of phase II clinical trialsIn in vitro studies, Evangelos Michelakis of University of Alberta found that in tissue samples from 49 patients, DCA caused depolarization of mitochondria in GBM tissue but not in normal brain tissue.[18]
Five palliative patients with primary GBM were entered into a phase II trial. Three had not responded to several chemotherapies; two were newly diagnosed. After surgical removal of tumor mass, they were treated with DCA and chemotherapy.[18]
Of the five patients tested, one died after three months. The surviving four were followed for 15 months. Their Karnofsky scores were unchanged in two cases, and decreased by 10 points in two patients.[18]
DCA was associated with tumor regression and had a good safety profile. DCA side effects were minimal.[18]
Michelakis is proceeding with phase three human studies with private funding from philanthropic groups and individuals. DCA's legal status as a discovery is public domain because it was made or discovered as far back as 1864[19] and has been used in the treatment of canine and human lactic acidosis, some who presented at the beginning of treatment with cancer.
Planned and ongoing clinical trialsDCA is non-patentable as a compound, though a patent has been filed for its use in cancer treatment.[26] Research by Evangelos Michelakis has received no support from the pharmaceutical industry. Concerns have been raised that without strong intellectual property protection, the financial incentive for drug development is reduced, and therefore clinical trials of DCA may not be funded.[15][16][17][27] However, other sources of funding exist; previous studies of DCA have been funded by government organizations such as the National Institutes of Health, the Food and Drug Administration, the Canadian Institutes of Health Research and by private charities (e.g. the Muscular Dystrophy Association). Recognizing anticipated funding challenges, Michelakis's lab took the unorthodox step of directly soliciting online donations to fund the research.[28] After 6 months, his lab had raised over $800,000, enough to fund a small Clinical Phase 2 study. Michelakis and Archer have applied for a patent on the use of DCA in the treatment of cancer.[29][30]
On 24 September 2007, the Department of Medicine of Alberta University reported that after the trial funding was secured, both the Alberta local ethics committee and Health Canada approved the first DCA clinical trial for cancer.[31] This initial trial was relatively small with enrollment of up to 50 patients. The trial was completed in August 2009[32]. In May 2010 the team published a press release[33] stating no conclusions could be drawn as a result of the trial. A paper describing the results was published[34] but not linked from the press release. Only five patients had been treated with the drug during the trial.
In May 2011, online reports[35] suggested that the Alberta group had released new data which the media "had not reported". However, this appeared to be caused by confusion between dates (the previous update was May 2010[36]) and cancer charities moved quickly to counter these rumours[37][38], which were subsequently covered in New Scientist magazine [39].
Thanks for BUTTing in, no pun intended on your toilet paper recommendation. I too like PG, which is why I have that end of my investment covered. I am looking to diversify. Thanks.
Wasn't it Marie Antoinette that said, let them use sand
good writeup...I am follower number 5
hgsi's successful phase 3 was all based on data mining their phase 2.
Even though I still think the drug is weak
I wondered why this happened
http://finance.yahoo.com/news/Diamyd-Medical-Diamyd-bw-1300743674.html?x=0&.v=1
I should have taken some action because of this
http://finance.yahoo.com/news/Diamyd-Medical-Diamyd-Reports-bw-2489919626.html?x=0&.v=1
Three Late-Stage Leukemia Company Previews for ASCO 2011
May 8, 2011By MD Becker Partners
At the upcoming ASCO Annual Meeting being held June 3-7, 2011, in Chicago, Illinois, Eisai Co., Ltd. (ESALF) is expected to report detailed results from its DACO-016 Phase 3 trial of Dacogen® [decitabine] as a frontline treatment for elderly patients [65+ years old] with acute myelogenous leukemia [AML]. As announced less than one year ago, Dacogen’s top-line results did not meet the primary endpoint of superiority over low-dose cytarabine in terms of overall survival in this study, although a trend was reported to be evident.
Shares of SuperGen, Inc. (SUPG), which climbed as high as $2.89 on expectations for positive trial results, reached a new 52-week low of $1.71 in July 2010 following the negative top-line news. SuperGen receives a 20-30% royalty on worldwide sales of Dacogen from its development and commercialization partners – Eisai in North America and Johnson & Johnson (JNJ) outside of North America.
Despite the negative top-line results, shares of SuperGen have since rebounded and reached a new 52-week high in April 2011. Optimism may stem from the fact that both Eisai and Johnson & Johnson are continuing to analyze the data and planning to move forward with North America and European regulatory filings in 2011 based on the primary analysis and secondary endpoints. Accordingly, investors will anxiously await the detailed Phase 3 results being presented on Monday, June 6, 2011 at ASCO to better gauge the likelihood of FDA approval in AML [Abstract #6504 “Results from a randomized phase III trial of decitabine versus supportive care or low-dose cytarabine for the treatment of older patients with newly diagnosed AML”].
Results from the Dacogen study may also be of interest to investors in Cyclacel Pharmaceuticals, Inc. (CYCC), which recently launched a multicenter, randomized, pivotal Phase 3 trial for the company’s sapacitabine oral capsules as a front-line treatment of elderly patients aged 70 years or older with newly diagnosed AML who are not candidates for intensive induction chemotherapy. Unique among drugs available to treat AML patients, sapacitabine is the only oral agent in late-stage clinical development. It is also the only candidate to progress into a pivotal study on the basis of survival data from a randomized Phase 2 study. Historically, sponsors advanced molecules to pivotal development in AML based on Phase 2 studies with primary endpoints of complete remission [CR].
The pivotal Phase 3 study is being conducted under a Special Protocol Assessment [SPA] agreement that Cyclacel reached with the FDA. The primary efficacy endpoint for the study is an improvement in overall survival from either of the two pairwise comparisons [Arm A versus Arm C, or Arm B versus Arm C] in the following three arms consisting of approximately 150 patients per arm:
•Arm A: sapacitabine administered in alternating cycles with Dacogen
•Arm B: sapacitabine administered alone
•Arm C: Dacogen administered alone
Cyclacel is testing the treatment regimen of sapacitabine administered in alternating cycles with Dacogen [Arm A] in an on-going pilot study, with data expected at ASCO 2011 [Abstract #6587 “Phase I/II study of sapacitabine and decitabine administered sequentially in elderly patients with newly diagnosed acute myeloid leukemia”]. Thirty-day and sixty-day mortality outcomes from this pilot study may be helpful in determining the odds of success in the Phase 3 pivotal study. To put this in perspective, thirty-day mortality in AML patients aged 70 years or older ranged from 17% to 21% in a recently published Phase 3 study [Harousseau JL, et al, Blood, 2009:114:1166]. Accordingly, results from the pilot study that demonstrate thirty-day mortality with sapacitabine is equal or less than 21% could be encouraging for Cyclacel.
The Phase 3 study builds on promising 1-year survival observed in elderly patients aged 70 years or older with newly diagnosed AML or AML in first relapse enrolled in a Phase 2 study of single agent sapacitabine. In a disease setting where patients are typically treated with chemotherapy agents like cytarabine for an average of 1 to 2 cycles, patients in Cyclacel’s Phase 2 study achieved a median of 12 cycles of treatment with sapacitabine.
In addition, approximately 45% of patients in the Phase 2 study had transformed into AML after being diagnosed with myelodysplastic syndromes [MDS] and were previously treated with Dacogen or Celgene Corporation’s (CELG) Vidaza® [azacitidine]. Only newly diagnosed AML patients are expected to be enrolled in the ongoing Phase 3 trial, none of whom had been previously treated with Dacogen or Vidaza and none of whom had relapsed, potentially increasing the odds for a successful trial.
Finally, Sunesis Pharmaceuticals, Inc. (SNSS) will also be presenting at ASCO [Abstract #TPS201, “Adaptive design of VALOR, a phase III trial of vosaroxin or placebo in combination with cytarabine for patients with first relapsed or refractory acute myeloid leukemia”]. Unlike the aforementioned frontline trials being conducted under SPA’s, Sunesis is studying vosaroxin in relapsed/refractory AML in an ongoing Phase 3 trial. Approximately 450 patients will be randomized to receive either vosaroxin or placebo in combination with cytarabine.
Cytarabine, a generic chemotherapy drug introduced several decades ago, is already a critical part of the treatment for younger patients with AML who are fit to withstand its toxicity. Unfortunately, several companies that make cytarabine have recently experienced production difficulties and others cannot make the drug fast enough to keep up with demand. This has resulted in a severe shortage of cytarabine that has reportedly affected leukemia clinical trials being run by the Cancer and Leukemia Group B [CALGB]. Accordingly, investors will be looking to Sunesis for an update on enrollment in the VALOR Phase 3 trial to determine whether or not the cytarabine shortage has been a factor.
“My personal opinion on AML affecting the elderly population is that the field is in need of a total revamp whereby certain chemotherapy agents need to be combined with targeted therapies to overcome drug resistance and provide meaningful survival data,” said Daruka Mahadevan, M.D. Ph.D., Director, Phase I Program, Arizona Cancer Center. “If you can increase the survival of a 70-year old patient by ten years, that would be a real achievement. Sapacitabine is interesting as it is an oral agent, while vosaroxin in combination of cytarabine may provide short term control – but is unlikely to provide a survival benefit.”