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That's the essence of PR ;)
This work was performed on a very small sample, in order to validate the test they will need a much bigger (hundreds) sample and this will take more than two years.
I don't think MRK will give up easily but finding the therapeutic dose could be difficult for this drug.
TEVA already declared that it is more serious about Germany especially after they has won a significant increase in the number of molecules for which it can contract to supply during 2008-2009 to AOK. They must increase their sales force there and buying a local company is the best way but there are only 2 suitable candidates.
Enrollment in ph.I and II studies of VX-680/MK-457 (an aurora kinase inhibitor) has been suspended.
http://clinicaltrials.gov/ct/search;jsessionid=B30A1E7C320CA9FEEEDF8B114C3A8AB4?term=VX-680&submit=Search
New phase 2 study shows that liraglutide leads to significant weight loss in obese people (20 Nov 2007)
http://www.novonordisk.com/press/sea/sea.asp?sShowNewsItemGUID=edb9506b-dd4a-474b-97a3-cb3c921636b8&sShowLanguageCode=en-GB
Novo Nordisk today announced clinical results from a double-blind, placebo-controlled phase 2 study comparing liraglutide, the once-daily human GLP-1 analogue, with orlistat, a lipase inhibitor, for treatment of obesity in people who do not have diabetes.
The study demonstrated that liraglutide given once daily over 20 weeks at the highest dose led to a weight loss from baseline of just above 7 kg in comparison to a weight loss of just below 3 kg in the placebo group and a weight loss of just above 4 kg in the orlistat-treated group. All doses of liraglutide reduced body weight. More than 75% of the people treated with the highest dose experienced a weight loss larger than 5%, and more than 25% experienced a weight loss larger than 10% relative to their body weight at randomisation. Finally, the study revealed a beneficial effect on systolic blood pressure after treatment with liraglutide.
Approximately 30% of the 564 participants in the study showed signs of prediabetes at randomisation. Following 20 weeks of treatment with any dose of liraglutide, between 80% and 90% of these participants no longer showed any sign of prediabetes, as opposed to around 40% in the placebo- and orlistat-treated groups.
Liraglutide was generally well tolerated. The overall withdrawal rate across the study was around 20%, and no more than 10% of the people who were treated with liraglutide withdrew from the trial due to adverse events. Consistent with all previous trials, the most common adverse events were related to the gastrointestinal systems and mainly rated as mild to moderate. The most frequently reported individual adverse event was nausea. The frequency of events was dose dependent and in the range of 20% to 50%. Nausea was most frequently observed at the beginning of the study.
In order to study the long-term weight reduction of liraglutide treatment, around 85% of all participants in the study volunteered to continue into an open label extension phase of the study.
Mads Krogsgaard Thomsen, chief science officer, said: "We are very encouraged by these new results. They give us reason to believe that liraglutide has the potential to become a new and important treatment option in the fight against serious obesity."
The results of the phase 2 trial do not change Novo Nordisk's expectations for the company's financial results for 2007, which were provided on 31 October in connection with the release of the financial results for the first nine months of 2007.
Conference call At 15.00 CET today, corresponding to 9.00 am New York time
Snip
Bought my first Procognia shares today.
Are you still holding?
Biocell is up more than 6% at TASE on this.
By email.
No link yet.
SO=sector outperformer (means CIBC think it's a "buy").
CIBC, as of 11/20, are initiating coverage of PLX with an SO rating and a $7, 12 to 18 month price target.
Looks like Ian Wilmut thinks iPS is the way to go.
Quark Pharmaceuticals Inc. (QURK) Announces First Systemic siRNA Dosing in Humans Commences Phase I Clinical Trial of Proprietary siRNA Molecule in Acute Renal Failure
http://www.clinicaspace.com/news_story.aspx?NewsEntityId=77923
FREMONT, Calif., Nov. 19 /PRNewswire/ -- Quark Pharmaceuticals, Inc., a development-stage pharmaceutical company focused on discovering and developing novel RNA interference-based therapeutics, announced today that it has commenced systemic dosing in humans of its proprietary product candidate, AKIi-5, a siRNA compound discovered and developed by Quark for the treatment of Acute Renal Failure (ARF), also called Acute Kidney Injury (AKI). Based on publicly available information, Quark believes that this is the first human clinical trial involving the systemic delivery of siRNA.
The Phase I clinical trial is a multi-center, double-blind, placebo controlled, dose-escalation trial assessing the safety and pharmacokinetics of AKIi-5 administered intravenously as a single dose to patients undergoing major cardiac surgery. Patients will be enrolled in the trial in a number of centers in the United States, Europe and Israel. Quark expects to complete the trial in early 2008. Depending on the results of this trial, Quark expects to initiate a dose-ranging Phase II clinical trial measuring AKIi-5 clinical activity.
Daniel Zurr, Chief Executive Officer, commented, "The initiation of human dosing in our Phase I trial in ARF signifies a very important step in Quark's clinical program and marks an important milestone in the RNAi industry. For Quark, the trial serves to further validate the strength of our pipeline and our overall expertise in the RNAi arena. With AKIi-5 now in the clinic, RTP801i-14, which we licensed to Pfizer, in a Phase I/IIa clinical trial for the treatment of wet age-related macular degeneration, AHLi-11 in IND-enabling studies and additional RNAi-based candidate drugs in pre-clinical testing, we believe Quark has one of the most robust RNAi product portfolios in the industry.
"For the RNAi industry, the trial may be even more noteworthy as it represents the first documented systemic dosing of siRNA in humans. While the science of RNAi has been well-established, a key step in the acceptance of the technology as a promising therapeutic is the ability to deliver RNAi-based compounds systemically. We look forward to leading this important advance for the industry and, at the same time, continuing the development of this and other siRNA products in our pipeline."
Quark was granted an IND by the Food and Drug Administration (FDA) for AKIi-5 for the prevention of Acute Renal Failure in high-risk patients undergoing major cardiovascular surgery. AKIi-5 is a synthetic, chemically modified siRNA molecule discovered and patented by Quark that has an AtuRNAi technology-based structure licensed from Silence Therapeutics. Quark has also licensed certain intellectual property from Alnylam.
Quark has conducted pre-clinical studies of AKIi-5 for the prevention of acute renal failure in rats and monkeys. Rats treated with a single bolus injection of AKIi-5 were significantly protected from ischemia/reperfusion- induced acute kidney injury. In the rat studies, AKIi-5 effectively prevented the development of acute renal failure. Quark's pharmacokinetic, distribution, and toxicity studies in rats and monkeys indicate that AKIi-5 appears to have a favorable safety profile and has a relatively short residence time in the kidney.
About AKIi-5
AKIi-5 is a synthetic, chemically modified siRNA molecule designed to temporarily inhibit the expression of p53, a gene which plays a significant role in ARF by inducing tubular cell death (apoptosis) in response to injury. AKIi-5 is based on Quark's proprietary, patented concept of temporary and reversible inhibition, for therapeutic purposes, of the expression of the transcription factor human p53, which is associated with DNA repair and apoptosis. The concept was first published by Quark with the University of Illinois in a breakthrough paper in Science magazine (Science. 1999 Sep 10;285). Using RNA interference technology to temporarily inhibit p53 in acute settings such as acute kidney injury, apoptosis is delayed thereby allowing natural repair mechanisms to restore normal DNA and cellular integrity.
About Acute Renal Failure (ARF) / Acute Kidney Injury (AKI)
ARF is a syndrome characterized by a rapid decline of kidney function leading to death in a high percentage of cases. Major cardiac surgery is one of the many causes of ARF. During cardiac bypass surgery, lack of oxygen caused by reduced local blood flow to the kidneys, followed by rapid reintroduction of oxygen, or reperfusion, to the kidneys upon removal of the patient from cardiopulmonary bypass, initiates a chain of events that can lead to ARF. Currently, there are no approved drug therapies that effectively prevent or treat ARF.
About Quark Pharmaceuticals, Inc.
Quark Pharmaceuticals, Inc. is a development-stage pharmaceutical company focused on discovering and developing novel therapeutics based on its proprietary gene discovery science and technology, with an initial focus on drug candidates that work through the natural mechanism in the cell known as RNA interference, or RNAi, for the treatment of diseases associated with oxidative stress. Quark believes that its proprietary target gene discovery platform, BiFARTM, combined with its ability to design and successfully deliver synthetic molecules of the new class of RNAi therapeutics known as small-interfering RNA, or siRNA, to specific organs in the body, enables the Company to rapidly develop drug candidates. Quark has two internally discovered and developed clinical stage lead product candidates: RTP801i-14 in phase I/IIa clinical trial for the treatment of wet age-related macular degeneration, and AKIi-5 in phase I for the prevention of acute renal failure both of which have an AtuRNAi technology-based structure licensed from Silence Therapeutics, as well as a license from Alnylam. The Company has licensed RTP801i-14 to Pfizer on an exclusive worldwide basis. Quark has, in addition, a product candidate portfolio of RNAi therapeutics based on novel targets and therapeutic concepts discovered using BiFAR(TM) and designed for the treatment of oxidative stress associated diseases of the inner ear, lungs and additional organs of the body.
Quark is headquartered in Fremont, California and operates research and development facilities in Boulder, Colorado and Ness-Ziona, Israel. Additional information is available at www.quarkpharma.com
Thanks for posting that.
I guess Teva was also surprised regarding the immunogenicity concern. They were probably expecting "sameness" to Lovenox issue as reflected in their response to Aventis’ citizen petition from 2004 (MNTA is mentioned for finding substantial variation between batches of commercially available Lovenox).
http://www.fda.gov/ohrms/dockets/dailys/04/sep04/091504/03P-0064-emc00001-01.pdf
Merck beats Pfizer in this race:
Merck to start Israeli R&D
http://www.haaretz.com/hasen/spages/925063.html
By Ora Coren
Pharmaceutical and biotechnology giant Merck is starting drug development and commercialization operations in Israel. The company will sign an agreement this week and join the chief scientist's program for international cooperation between small Israeli firms and multinational companies.
Under the agreement, Merck Serono, the company's new division for innovative small molecules and biopharmaceuticals, will cooperate with Israeli startups in developing pharamceuticals, and will later be able to buy the rights to the drugs or help market them around the world.
The Industry and Trade Ministry will finance half of the research and development costs of these companies, through its Chief Scientist's Office.
This is a dramatic step forward for the Israeli biotech industry, which has had a hard time taking off. Even though Israeli biotechnology research is considered among the world's most advanced, the industry is finding it difficult to produce successful firms. Studies of this failure have noted that one of the ways to open this bottleneck would be for a major international giant to enter Israeli biotech research and development.
Merck will enter Israel before Pfizer, another international pharmaceutical giant, which is also considering opening development and commercialization operations here. Pfizer is negotiating to buy an Israeli biotech hothouse, but has still not signed any agreements.
Teva will now find itself with competition on its home turf. Merck, which bought Serono at the beginning of 2007 for $14 billion, now is a direct competitor of Teva in the muscular sclerosis market, with its product Rebif.
Teva has had almost exclusive access to Israeli biotechnology and pharmaceutical research, since international firms have kept away. Now, with Merck and Pfizer on the way, Teva will lose its exclusivity and face real competition here for the first time.
As for Serono, this is a real U-turn. Rebif is based on technology from the Weizmann Institute, and was produced by Interpharm in Rehovot. But Serono almost completely closed down Interpharm and moved production to Europe. Ironically, it will now find itself back in Israel.
In vitro kinetic studies show that the activity of prGCD is comparable with that of Cerezyme. Furthermore, the uptake and cellular activity of prGCD was higher than that of Cerezyme at higher concentrations. Similar results were obtained using cells isolated from patients with Gaucher’s disease. It is reasonable to postulate that prGCD will have improved therapeutic activity.
Do note that PLX are testing their drug in comparison to Cerezyme (at the same and at lower concentration) but according to their SPA they don't need to show superiority. If their drug will indeed be more efficient, it may change dosing.
PLX's drug might work better than Cerezyme: it showed a longer plasma half-life and higher activity than Cerezyme in a phase I study (this may mean smaller and less frequent dosing).
But, PLX didn't do phase II, so efficacy in patients is yet to be proven. Moreover, the drug addresses a chronic disease and it is not yet known how will humans tolerate a plant driven drug over long period of time i.e immunogenicity.
[Till now we believed that both alleles are expressed, except for the x-chromosome and imprinted genes. Now we have to understand why mother nature introduce another level of complexity and do not express both alleles in about 10% of the genes. Making this preference may have a dramatic impact on the phenotype, if for example one allele carry a susceptibility to disease and the other do not. It introduces a variability we did not think about and now will have to take into account in population genetics.]
When only mum or dad matters
Nature online 15 November 2007 By Ewen Callaway
http://www.nature.com/news/2007/071115/full/news.2007.250.html
Hundreds of our genes turn on just one copy, rather than both.
The textbook rule that says activated human genes almost always express both of their copies — the one inherited from mum and that inherited from dad — seems not to be true. Instead, a good chunk of our genome could prefer the 'single life', according to new research.
Whether the maternal or paternal copy gets switched on in such cases seems to be random. But the result could have a big impact on disease susceptibility and other biological traits.
It had been thought that there are only a handful of situations in which just one of a pair of gene copies is used. But a new screen of 4,000 human genes has uncovered 371 that sometimes play favourites, suggesting that this phenomenon is far more pervasive than had been thought.
“What we’ve shown violates general rules about what we think about genes: if a gene is turned on you express both the maternal and paternal copy,” says Andrew Chess, a biologist at Harvard University in Cambridge, Massachusetts, who led the study along with colleague Alexander Gimelbrant.
This kind of selective gene expression could create an extra source of variation between people, even when some of their genes are identical.
“I like the idea that we're all mosaics, and this might contribute to differences,” says Steve Henikoff, a biologist at the Fred Hutchinson Cancer Research Center in Seattle, Washington.
Coalition of the expressed
Researchers have long known that some cells express just one version of certain genes, but this is for a specific purpose. Females have two X chromosomes but always shut down one copy, for example, to prevent making a double dose of some gene products. Immune cells designed to recognize the fingerprint of a specific disease turn off one set of immunoglobulin genes to optimize their specialist behaviour. And during development, embryos chemically flag one parent’s version of a set of known genes for silencing. This handful of instances accounts for hundreds of genes known to turn off a copy in a controlled way.
To hunt for more genes that behave similarly, Chess and Gimelbrant planted single human immune cells in dishes, and let the cells multiply. They analysed the millions of cell copies using a gene chip to distinguish between the maternal and paternal version of messenger RNA (mRNA) produced by 3,939 genes. They found that about 9% of the genes sometimes turned on just one copy. They publish their findings today in Science 1.
Even though the cells were all genetically identical, some turned on maternal versions, some paternal, and others activated both. "Even identical twins would make different choices," says Gimelbrant.
The team noticed a similar pattern in a much smaller subset of genes that they examined from specimens of donated human placenta and white blood cells. “This is not something weird that happens only in cell culture,” says Chess. Nor does it happen only in immune cells, although the researchers' further work on other cell types has not yet been published.
Sloppy genome
These differences could potentially help an organism to regulate gene expression and affect disease risk. Higher expression of a gene called APP is linked to early onset of Alzheimer’s disease, for example. And Chess’s team found that cells in which just one copy of APP was turned on made about half as much mRNA as the cells with both copies turned on. At this point, the connection to Alzheimer’s remains speculative, he says.
The random gene inactivation that Chess's team has found could be a spillover from more controlled forms of silencing, such as shutting down an X chromosome, says Huntington Willard, a geneticist at Duke University in Durham, North Carolina. "I tend not to think of it as some grand evolutionary plan, but simply as the sloppiness of gene regulation."
Chess doesn’t know just when cells switch off a gene copy, but he suspects it happens during development. Unanswered, too, is how the cells turn off one copy while keeping the second copy active. Chess’s lab is now digging into this question.
The team next plans to survey the rest of humanity's 20,000 genes for signs of similar deactivation. If his team’s sample is representative of the whole genome, more than 1,000 genes should sometimes go it alone.
*
References
1. Gimelbrant, A. et al. Science 318, 1136-1149 (2007)
CAMH,
I was a bit surprised from that bounce and the fact it held through the day.
OT Football - I follow but not out of patriotic reasons (the Israeli team has very little to show). Are you a fan of England?
More on personalized medicine:
Personalized medicine slowly taking shape
Thu Nov 15, 2007 By Ben Hirschler
http://www.reuters.com/articlePrint?articleId=USDIS55581820071115
NEW YORK (Reuters) - Personalized medicine -- tailoring treatments to an individual's genetic profile -- has been one of the main dreams of the gene revolution, but putting it into practice is proving tough.
While advances are being made in a few areas, so-called pharmacogenetics will not change the commercial landscape for the bulk of pharmaceuticals for several years, drugmakers told the Reuters Health Summit in New York this week.
"Pharmacogenetics is not going to transform this market any time soon," said Jean-Pierre Garnier, chief executive of GlaxoSmithKline Plc.
"Let's be clear -- it's going to take 20 years plus. Does that mean you are going to have zero happening? No. It's already happening. But it is going to be very specific examples."
The decoding of the human genome in 2000 sparked hopes that a new era of tailored medicine was just around the corner.
In fact, uncovering the genetic differences that determine how a person responds to a drug, and developing tests, or biomarkers, for those differences, is proving more challenging than initially hoped.
As a result, people with complex diseases like depression are still being prescribed medicines on a trial-and-error basis, and adverse drug reactions remain a major cause of injury and hospitalizations.
That is not stopping companies from investing in the field.
One pioneer is Roche Holding AG, which already relies on genetic tests to determine if women with breast cancer will respond to its drug Herceptin. Roche has offered $3 billion to acquire Ventana Medical Systems Inc to build its presence in this area.
Glaxo, too, has work in progress, with a large clinical trial now under way to see if an extended-release version of diabetes drug Avandia -- recently tainted by safety worries -- can help a sub-set of people with Alzheimer's disease.
SHOTGUN TREATMENT
Pharmacy benefit manager Medco Health Solutions Inc CEO David Snow believes genetic profiling will be key to future patient care, as well as costs.
"Right now, medicine is practiced in a shotgun way. It is very inefficient and very wasteful," he said.
Medco is starting to address the issue product by product, starting with Bristol-Myers Squibb Co's Coumadin, also known generically as warfarin, a notoriously difficult to use blood thinner that can cause serious bleeding in some patients.
The Food and Drug Administration announced in August that Coumadin would come with new instructions explaining that people with certain genes may need a lower dose.
Janet Woodcock, FDA deputy commissioner, said such genetic markers were promising but had to be backed up by clinical evidence.
"Real progress is where you have a label that says you must do this test before you use this drug and you know that is going to enhance either safety or efficacy or dosing," she said.
That is something Snow plans to address with a large clinical trial Medco is working on in collaboration with the Mayo Clinic.
Roche CEO Franz Humer said personalized medicine would become increasingly important over the next 5 to 10 years -- particularly in cancer -- from both a regulatory point of view and to justify high prices for modern drugs.
"That's where development of modern medicine is heading ... (but) it is as complex to find a biomarker as it is to find a new drug," he said. "Don't expect these things to happen overnight."
CAMH
Up 70% as I type on this
http://biz.yahoo.com/bw/071115/20071115005843.html?.v=1
I was thinking that mortality data in the adjuvant setting takes more time, therefore it was not likely to get efficacy yet.
These products are mentioned under the Technology tab at their home page.
http://www.inspirationbio.com/page.php?mode=privateview&pageID=9
Still there's a good chance the trial will be stopped after the next interim analysis in 6 months.
Couple of more Avastin events to look for in the near future.
Not surprising:
Roche says Avastin study misses endpoint
http://www.reuters.com/article/health-SP/idUSZAT00715220071113
Tue Nov 13, 2007 2:04am EST
ZURICH, Nov 13 (Reuters) - Roche (ROG.VX: Quote, Profile, Research) on Tuesday said a late-stage study of its cancer drug Avastin in combination with gemcitabine chemotherapy and Tarceva for pancreatic cancer did not meet the primary endpoint for survival.
The phase III trial comparing gemcitabine chemotherapy and Tarceva with or without Avastin as first-line treatment for advanced pancreatic cancer showed some evidence of clinical activity on secondary endpoints but the trial did not meet the primary endpoint of overall survival, Roche said in a statement.
Data from the AVITA trial are now being further analysed to determine the extent of benefit of adding Avastin to the Tarceva/gemcitabine combination therapy, Roche said.
Avastin is made by U.S. biotech firm Genentech, which is majority owned by Swiss drugmaker Roche Holding AG.
(Reporting by Thomas Atkins)
Before you ever try to light another cig, give me a call. I'll tell you some things to make you change your mind.
If you're looking for confusion, see #msg-24073573 and its reply.
Here are Dr. Sanjeev Arora, Executive Vice Chair at the Univ. of New Mexico Health Sciences Center and an HCV investigator comments (CIBC hosted a CC with him):
Dr. Arora believes PROVE 1 and 2 demonstrate telaprevir has clear clinically meaningful benefit vs. standard of care, which he believed performed within the expected range. He said the different relapse rates in the 12+12 arms of PROVE 1 and 2 likely relate to selection of pts with RVR in PROVE 1.
He felt that higher SVR rates may be observed in ph.III, as docs have increased experience managing rash and a higher threshold for stopping therapy. He believes that current data supports moving telaprevir into ph.III studies shortly, and that the FDA will require SVR24 as the endpoint.
He emphasized that safety of the earlier-stage PIs remains an open question.
Lots of perhaps in the current situation, but the fact is MNTA will have to supply additional data regarding immunogenicity via more animal or clinical trials. Either way, that means a delay of months to years.
While the market's reaction shows it doubts MNTA's characterization platform to ever satisfy the FDA, I find MNTA's platform very strong and accurate. I believe that MNTA will provide the required data and that M-enoxaparin will be approved eventually. This together with the value in their pipeline, made me buy some more on yesterday's additional downside.
The N370S mutation is most common (if memory serves about 70%)in the Ashkenazi Jewish population and the L444P mutation in non-Jewish patients.
And do they test patients for the N370S mutation?
"For Gaucher...they think about 90% would respond to Plicera"
Makes sense, they probably test patients for the common N370S mutation in GS.
"if the FDA would accept some sort of surrogate end-point and perhaps a shorter trial"
They would probably need to produce similar improvements in
hepatosplenomegaly and hematologic parameters as with Cerezyme after 6 months treatment in Type 1 Gaucher patients.
Maybe for Types 2 and 3 patients they could have other parameters and a short cut but they are not testing these patients are they?
Each therapeutic strategy has significant limitations, and currently none can effectively treat all of the associated pathologies. Disadvantages of ERT include regular IV treatments, little direct effect on the CNS-affected variants of GD (types 2 and 3)1, and high cost.
Pharmacological Chaperones strategy (which I like for being a paradoxes of molecular medicine because an enzyme inhibitors can actually increase the amount of enzyme activity),is attractive because: oral administration, crossing the BBB and low cost.
Some potential problems I can think of are: will if work for all missense mutations? potential rash, dosing (as the chaperone molecule may need to dissociate from the enzyme once it reaches the lysosome in order to be active), long term tolerability and potential effect against closely related hydrolases.
Will be interesting to watch (efficacy results from the 6 month study are expected in 3Q08).
I tend to take Genzyme's side as stated in #msg-24221129 nevertheless, this is a positive step for Amicus.
Since Amicus and PLX are competitors and you're long FOLD:
UPDATE 1-Amicus Therapeutics signs licensing deal with Shire
Thu Nov 8, 2007 8:13am EST By Toni Clarke
http://www.reuters.com/article/marketsNews/idUKN0835896320071108?rpc=44
BOSTON, Nov. 8 (Reuters) - Amicus Therapeutics Inc (FOLD.O: Quote, Profile, Research) said on Thursday it granted British drugmaker Shire Plc (SHP.L: Quote, Profile, Research) marketing rights outside the United States to three drugs Amicus is developing to treat rare genetic disorders.
Under an agreement with Shire, Amicus, a small biotechnology company based in Cranbury, New Jersey, will receive $50 million in cash and up to $150 million more if certain milestones are met. If the drugs are commercialized, Amicus will receive up to $240 million in sales milestone payments and double-digit royalties.
The companies will equally split the cost of developing and bringing the drugs to the market, and Amicus will retain rights to the lucrative U.S. market.
The agreement gives Shire an additional plank in its effort to build a stable of drugs for human genetic disorders. In 2005, it paid $1.6 billion for Transkaryotic Therapies Inc., a company that developed a drug called Replagal to treat the genetic disorder Fabry disease. Shire also sells Elaprase, a drug for Hunter Syndrome.
Shares of Amicus have risen 6.6 percent since its initial public offering at the end of May. If successful, the company would compete with Genzyme Corp. (GENZ.O: Quote, Profile, Research), one of the world's biggest biotechnology companies and a leader in the development of drugs for rare inherited disorders.
The agreement with Shire covers Amicus's experimental drug Amigal, which is currently in mid-stage trials to treat Fabry disease, a disorder in which deficient activity of a certain enzyme can cause kidney failure and increased risk of heart attack and stroke.
It also covers Plicera, a drug in mid-stage trials to treat Gaucher disease, a condition that can cause enlarged liver and spleen, low levels of red blood cells and central nervous system impairment.
A third drug that is part of the agreement with Shire, known as AT2220, is in early stage trials for the rare muscle disorder Pompe disease.
Current sales of drugs for these three diseases, known as lysosomal storage disorders, come to roughly $1.7 billion, with the vast majority going to Genzyme.
John Crowley, the chief executive of Amicus, said roughly 60 percent of the market is outside the United States, and that while he wouldn't rule out a separate partnership for the U.S. market, right now the company plans to build itself into a fully-fledged biotech company.
At a time when many biotechnology companies are seeking to wait as long as possible before signing partnerships, hoping that they will get a better deal the further their products are along in the pipeline, Crowley said the terms of the Shire deal were too good to pass up.
"For us, the economics of this deal were just so strong," he said. (Reporting by Toni Clarke, editing by Steve Orlofsky)
They check more autoimmune diseases as candidates for CF101 especially now after finding a marker in the blood. That might be the parachute they need to stop them from free falling, in case the RA phase II trial blows up.
It is critical to control BP precisely, how can this be done by vaccination is science fiction to me.
After yesterday's event this may have a new meaning if TEVA did use or Procognia's technology to strengthen their generic Lovenox ANDA. Can they still do it now? (Is it possible to add new data after submitting the ANDA?)
On a side note, Procognia's advantage over Momenta's is in biological systems where time to decision is crucial like when doing clone selection (MNTA's tech requires more time and preparations).
"Do you consider PARD a buy here?"
I think that the phase III study of picoplatin in 2nd line SCLC compared to best supportive care, can achieve benefit on survival because the primary endpoint is increased median overall survival and given the poor performance of the control arm (very aggressive cancer,terminally ill patients, hight rate of mortality), strong ph.II results in this setting plus they got a SPA. I believe the potential market is rather small for this indication.
I am more skeptic regarding the other settings in first line mCRC, compared picoplatin/5-FU/LV (FOLPI) vs. oxaliplatin, in first line HRPC and in advanced solid tumors (oral formulation).
I don't feel knowledgeable enough to buy or tell anyone to do so yet.
"What do you know of picoplatin?"
In 2001, AZN returned all picoplatin rights to AnorMED after several phase I and II trials in solid tumors. The drug should have better safety profile than other platinum agents and overcome resistance.