Vertex Pharmaceutical's (VRTX) Telaprivir is effective, but path to market needs clarification

The results look impressive as they show a rapid response in Telaprivir treated patients vs. plaebo in the first 4 weeks. However, the interpretation of results at 12 weeks becomes questinable due to lack of statistical significance. Analysts were focusing on a small subset of 20 patients who were to receive the Vertex drug for just 12 weeks and then be checked 20 weeks later for signs of relapse. Here is the quoted text from the press release regarding those 20 patients: "Analysis of PROVE 1 Patients who Finished All Treatment at 12 Weeks. Seventeen of 175 patients received at least one dose of telaprevir in “Arm D” of the PROVE 1 study (telaprevir + peg-IFN + RBV). According to the study protocol, patients in Arm D were eligible to stop all treatment at week 12 if they met on-treatment...

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Altus Pharmaceuticals (ALTU) is showing clinical and financial progress

Altus Pharmaceuticals (ALTU) is a small cap (~$400M) market cap biopharmaceutical company with a unique crystallization technology that amongst other things allows for better purity and longer half life for other wise rapidly broken down proteins and enzymes. This technology is very difficult to master and creates a natural barrier to entry even beyond patent expiration. I have previously written about ALTU when the stock was trading around $18 back in November 2006 (link). Since then Altus has signed a development and commercialization agreement with Genentech (DNA) (link) for ALTU-238 a longer acting version of human growth hormone currently in phase II clinical trials with a $2 billion market. On May 10th, Altus announced initiation of a phase III clinical trial for ALTU-135, an enzyme...

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February Performance Update and reflections on past picks

Looking back, I am still happy with the picks I have made so far. ALTU, ARNA and VRTX are obvious laggards but I think 2007 will be a good year for all of these good small and mid sized biotechs as clinical trial results become available. What stands out was the good decision to get out of CELG and VRTX based on valuations. I still don't like CELG at $54 but at $32 I like VRTX a lot and I am waiting for a better buying opportunity around $30. Meanwhile, I am selling more April $20 puts to take advantage of time decay of option values since there is not going to be much news announced in the next two months. I believe VRTX stock is being punished because the management has decided to release already available clinical data in April at medical conferences. Investors, being a paranoid bunch,...

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Onyx Pharmaceutical's (ONXX) success may pave the way for competition

Onyx Pharmaceutical (ONXX) is enjoying the rewards of its potential blockbuster, Nexavar. On Monday, November 19th, FDA approved Nexavar, already approved for Kidney Cancer, for treatment of liver cancer. The product did so well in clinical trials that the company and its partner Bayer had to halt the trials for ethical reasons to give Nexavar to the control group as well. Onyx's stock has been doing tremendously well. In 2007, it has raised more than 500% from about $10 to its current price of $53, but off of its 52 week high of $...

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Gene's mutations found to cause life-threatening aortic disease

Scientists have identified the first genetic mutations that cause the aorta – the body's main artery – to widen, tear and rupture. Finding biological markers that flag aneurysm, a bulging of the aorta that leads to dissection, a lengthwise separation of tissues in the artery wall, is critically important for early diagnosis. Aneurysms can be managed initially with medication and then successfully repaired to prevent catastrophic dissection and rupture, Milewicz said. Many patients never have a chance at treatment because they go undiagnosed, even when they go to emergency rooms with severe chest pain because diagnostic tests for heart attack do not uncover aortic defects. Actor John Ritter, for example, died in September 2003 from an undiagnosed dissection that ruptured....

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New Poll Feature on Biotechnology Stock Blog

In an attempt to have a more interactive blog and to hear more feedback from subscribers I have decided to add a Polling feature. You can find this tool on the right hand side of this page. I am hoping this tool will allow all of us to see the majority's response to a particular investment related topic and compare it to our own personal views. With 116 subscribers the results should be statistically significant. Also, you can suggest Poll questions by emailing me at arsaadat1@gmail.com The first Poll question is a very general question regarding your sentiment on the Biotechnology sector in 2008. I happen to be very bullish (greater than 25% return). I am interested to see how the majority feels. Going foreword, I am thinking about asking questions regarding small cap or large cap biotech...

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Drug Patent Expirations in October 2010

Drug Patent Expirations in October 2010 *Drugs may be covered by multiple patents Tradename Applicant Generic Name Patent Number Patent Expiration ORGARAN Organon Usa Inc danaparoid sodium 5,164,377 Oct 3, 2010 MIRAPEX ER Boehringer Ingelheim pramipexole dihydrochloride 4,886,812 Oct 8, 2010 MIRAPEX Boehringer Ingelheim pramipexole dihydrochloride 4,886,812 Oct 8, 2010 CLIMARA PRO Bayer Hlthcare estradi...

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The Next Ambien?

Early Monday morning, a decision from the U.S. Food and Drug Administration is expected for yet a whole new type of sleeping pill. It's called Rozerem, from Japanese drug giant Takeda. Rozerem targets the same receptors as the over-the-counter aid melatonin, but with potentially clearer efficacy. (Some have even dubbed it "super-melatonin.") The idea is that by targeting two melatonin receptors in the brain, called M1 and M2, the drug can avoid many of the side effects that are troublesome in other sleeping pills....

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Decoupled moves in Biotech indices highlight opportunities and challenges

The chart below shows returns for the last 6 months of the two Biotech indices that I follow: BTK and NBI. After a significant and equivalent downside move in March, both indices have turned positive with an explosive move since middle of July. I speculate that the March downside move was a response to concerns over Obama's health care proposal and its impact on Medicare. However, upon further examination, the adjustments in Medicare rebates would translate to no more than a double digit change in revenues. Moreover, the biotech story is in large a product story where launch of a new product has a much higher impact on revenues than any adjustments...

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Multi-species genome comparison sheds new light on evolutionary processes, cancer mutations

In a study published in the July 22 issue of the journal Science, a team of 25 scientists from the United States, France and Singapore compared the organization of the chromosomes of eight mammalian species: human, mouse, rat, cow, pig, dog, cat and horse. Using sophisticated computer software to align and compare the mammals' genetic material, or genomes, the team determined that chromosomes tend to break in the same places as species evolve, resulting in rearrangements of their DNA. Prior to the discovery of these breakage hotspots, the prevailing view among scientists was that such rearrangements occurred at random locati...

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Genetic Regulator Of Lifespan Identified

The finding implies that lifespan is not simply dependent on accumulated wear and tear or metabolism, as some researchers have suggested, but is at least partly controlled by an active genetic program in cells--one that could theoretically be boosted. One of the immediate implications of the work is that it emphasizes the functional difference between nicotinamide and nicotinic acid. Nicotinic acid (niacin) is a known anticholesterol treatment, while nicotinamide (or niacinimide) is sometimes touted for anti-aging abilities and is in clinical trials as a therapy for diabetes and cancer. However, the two substances are sometimes sold interchangeably as supplements under the name vitamin B3. "Our study raises the concern of taking high doses of nicotinamide,"...

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Curing HIV in Germany?

Every virus-resistant person has two mutant copies of a gene called CCR5, and a new biotech tool called zinc finger nucleases givve anyone that mutation. Instead of transferring bone marrow from another person, doctors could take a few cells from a patient, modify them to be HIV-resistant and then put them back in." (technology available now from Sigma-Aldrich, CompoZr ZFN)...

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DCA "The Cure" for Cancer?

DCA is an odourless, colourless, inexpensive, relatively non-toxic, small molecule. And researchers at the University of Alberta believe it may soon be used as an effective treatment for many forms of canc...

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MicroRNAs may be key to HIV's ability to hide, evade drugs

The team showed that a cluster of miRNAs bind to a certain location on the viral RNA, which in turn, blocks the creation of important proteins, and HIV replication. Resting CD4 T cells, they found, are “enriched” with more than the normal amount of these miRNAs compared to the activated T cells. When the researchers used antisense technology to block miRNA-caused viral inhibition, they found that the HIV again was active and able to replicate – proving miRNA’s critical role in maintaining laten...

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Pill makes women pick bad mates

Major histocompatibility complex (MHC) genes are involved in immune response and other functions, and the best mates are those that have different MHC smells than you. The new study reveals, however, that when women are on the pill they prefer guys with matching MHC odors. MHC genes churn out substances that tell the body whether a cell is a native or an invader. When individuals with different MHC genes mate, their offspring's immune systems can recognize a broader range of foreign cells, making them more fit. Past studies have suggested couples with dissimilar MHC genes are more satisfied and more likely to be faithful to a mate. And the opposite is also true with matchng-MHC couples showing less satisfaction and more wandering eyes. "Not only could MHC-similarity in couples lead to fertility...

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Genetic Test Predicts Response to Warfarin

That information already is being put to medical use. Last August, the U.S. Food and Drug Administration changed the labeling to say that doctors should consider a genetic test when first prescribing warfarin, better known by its brand name, Coumadin. But warfarin is a notoriously difficult drug to manage, especially at the start. Too much can lead to hemorrhages; too little can allow clots to form. One individual may do well on 1.5 milligrams a day, while another may require 20 milligrams daily. Two genes are known to affect the response to warfarin. One, designated CYP2C9, governs the metabolism of the medication, or how fast it is eliminated from the body. The other, designated VKORC1, governs sensitivity, or how the body reacts to a given dose of warfarin. The new study of 297 people...

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Patient in Experimental Gene Therapy Study Dies, F.D.A. Says

The case could be another setback for gene therapy, a field with a troubled history and numerous treatment failures, including the death of a teenager in 1999 in an experiment. The new therapy being tested, made by Targeted Gemetics of Seattle, is a virus-based product injected directly in the joints in hope of relieving active inflammatory arthritis. That chronic condition can affect multiple joints and organs, and it is quite different from the wear-and-tear arthritis that commonly occurs with aging. The patient became ill soon after receiving a second injection, the drug agency said. The date is not exactly clear, but the illness was recognized as a “serious adverse event” last Friday, and the agency immediately suspended the study. That means no more injections can be given. The patient...

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CDC seeing more regular flu cases now

U.S. health officials are seeing a surprisingly high number of cases of ordinary, seasonal flu at a time when the flu season typically peters out. About half of people recently testing positive for the flu have the new swine flu virus, Dr. Daniel Jernigan of the Centers for Disease Control and Prevention in Atlanta said Friday. "The H1N1 virus is not going away," Jernigan said. The virus "appears to be expanding throughout the United States" and poses "an ongoing public health threat," he said. Swine flu continues to affect more younger people — those ages 5 to 24 — and CDC is still seeing relatively few cases in older people In the United States, there are now more than 4,700 probable and confirmed cases of swine flu, and 173 hospitalizations and four deaths, Jernigan said. The tally...

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Multi-species genome comparison sheds new light on evolutionary processes, cancer mutations

In a study published in the July 22 issue of the journal Science, a team of 25 scientists from the United States, France and Singapore compared the organization of the chromosomes of eight mammalian species: human, mouse, rat, cow, pig, dog, cat and horse. Using sophisticated computer software to align and compare the mammals' genetic material, or genomes, the team determined that chromosomes tend to break in the same places as species evolve, resulting in rearrangements of their DNA. Prior to the discovery of these breakage hotspots, the prevailing view among scientists was that such rearrangements occurred at random locati...

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California expands newborn test for genetic abnormalities

California now moves to the forefront as it will test for 75 conditions through the standard infant heel prick. That more than doubles the disorders the old test covered. "This is a wonderful day, a great day, where we're not leaving any babies behind," said David Swift, who credits expanded testing with saving his daughter's life. The mandatory screening program will apply to all the 500,000-plus babies born in the state each year, though parents can opt out for religious reasons. Since the state began piloting the program May 2, the tests have identified 15 babies with serious conditions that previously would have gone undetected...

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Statin report: Could degrade adults brains

Statins may promote oligodendrocyte lineage commitment by parenchymal glial progenitor cells; this might reduce the available progenitor pool, and hence degrade the long-term regenerative competence of the adult white matter. GEN News Highlights Statins affect glial progenitor cells, which are important to brain health as we age, according to a group of scientists. These cells are similar to stem cells and can become either astrocytes or oligodendrocytes. The researchers found, however, that statins caused the glial progenitor cells to turn into only oligodendrocytes....

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Researchers Create First Targeted Knockout Rats Using Zinc Finger Nuclease Technology

In a paper published in the July 24, 2009 issue of Science, researchers describe the novel application of ZFNs to generate rats with permanent, heritable gene mutations, paving the way for the development of novel genetically modified animal models of human disease. ZFN technology will make the generation of such animals faster and will create new opportunities in species other than mice. "Until now, rat geneticists lacked a viable technique for 'knocking out,' or mutating, specific genes to understand their function," said Howard Jacob, Ph.D. Director of the Human and Molecular Genetics Center at the Medical College of Wisconsin. "This study demonstrates that ZFN technology bypasses the current need to conduct cumbersome experiments involving nuclear transfer (cloning) or embryonic stem...

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The Biologic Revolution

The Biology Revolution BIOTECHNOLOGY is simply “technology applied on biology”, especially when used in agriculture, food science, and medicine. Indeed, the modern world went through the “physics revolution” that saw the birth of nuclear power and the “chemical revolution” that lead to the chemical and pharmaceutical industries. And now it is the biology revolution. Biotechnology now plays a central role in: >> developing medicine and other therapies >> understanding diseases >> forensics >> improving animal and plant production >> increasing industry effectiveness >> environmental monitoring >> developing alternatives to fossil fuels Some aspects of biotechnology are very contentious. Genetically modified food, use of animals in research,...

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Incentives for the Biotechnology Industry

A company undertaking biotechnology activity and has been approved with BioNexus Status by the Malaysian Biotechnology Corporation Sdn Bhd is eligible for the following incentives: a) 100% income tax exemption for 10 years commencing from the first year the company derives profit, or b) Investment Tax Allowance of 100% on the qualifying capital expenditure incurred within a period of 5 years and effective from 2 September 2006, a BioNexus status company will be given a concesionary tax rate of 20% on income from qualifying activities for 10 years upon the expiry of the tax emption period. This enhanced incentive from 2 September 2006. c) Tax exemption on dividens distributed by a BioNeus status company; d) Examption on import duty and sales tax on raw materials/components and machinery...

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Biotechnology, An Instrument of Wealth Creation

BUDGET 2008 offered good news for biotechnology. The government has again demonstrated its commitment to biotechnology as another engine growth. Malaysia has high hopes for biotechnology. The country wants to deploy biotechnology to add value to the nation’s resources, especially its largely untapped biodiversity. Nearly RM300 million has been set aside to fund various biotech activities. The will be money for research and development, funding to buy technology, and allocations to promote investment. The Malaysian Biotechnology Corporation (MBC) will anchor programmes to attract investment in biotech. Already, 36 companies have been given BioNexus status. With that, they enjoy benefits including tax incentives and access to grants for R&D. By the year’s end, the number of BioNexus companies...

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