News and Comments

VERTEX: First Drugs ever to target the root-cause of cystic fibrosis are promising.

  Monday, June 13, 2011

Like many other chronic progressive intractable diseases, Cystic Fibrosis (CF) pathology is about misfolding of the transmembrane conductance regulator (CFTR) protein as a result of CFTR gene mutations. The malfunctioning CFTR protein loses its trafficking capability and fails to reach the cell membrane, resulting in the accumulation of thick, sticky mucus that plugs the airways and chokes the pancreas. The symptoms are chronic coughing, difficulty breathing, malabsorption of food and nutrients from the intestine, inability to gain weight, diarrhea, or bulky, foul-smelling, greasy stools, or constipation. The disease is debilitating and affects about 100,000 babies, children and adults worldwide. The presence of salty skin confirms the diagnosis of the disease. More...

VERTEX: What About Now?

  Wednesday, May 25, 2011

Yesterday, the FDA approved Incivek (telaprevir), Vertex’ (VRTX) HCV protease inhibitor, which demonstrated in clinical trials it cures more treatment naïve patients and more patients who did not benefit from conventional treatments in much less time than Merck’s Victrelis (boceprevir). Less than a month ago, the FDA Antiviral Drugs Advisory Committee voted 18-0 to recommend approval Incivek for HCV patients with genotype 1 chronic hepatitis. The recommendation was based on clinical trial data demonstrating that combining Incivek with pegylated interferon/ribavirin produced a higher cure rate in less time than standard therapy alone. This improvement is most notable for hard-to-treat patients including those with HCV genotype 1, people with liver cirrhosis, and those who did not respond to a prior course of conventional. Merck's drug Victrelis (boceprevir) also an HCV protease inhibitor, was given a positive recommendation and was eventually approved. More...

SEQUENOM Announces the Date of Launch of the Non-Invasive Down Syndrome

  Saturday, May 07, 2011

When Sequenom (SQNM) was welcoming analysts and shareholders who are “interested in discussing the results for the first quarter of 2011,” the firm’s CEO was quite aware that most attendees were, in fact, much more interested in the progress of the firm’s plans for the non-invasive prenatal tests, especially the Down Syndrome test. Shareholders, he knew, are interested in reading the future of Sequenom Center for Molecular Medicine (CMM). As a matter of fact, that’s what investors in development-stage biotech firms and small firms generating poor revenues firms on the road to realizing their dreams should look for i.e., the firm’s technologies and programs. In the case of Sequenom, its success in bringing non-invasive predictive prenatal tests to the market, especially, the promised Down Syndrome test, is a game changer that could either make this firm or break it. Meeting or exceeding expectations and promises of controlling expenses even if they lead to 25% reduction in losses, would be of minimal or no impact at all when the fate of the test is officially declared. More...

REGENERON: A GREAT LESSON TO LEARN

  Thursday, May 05, 2011

First, we congratulate our subscribers who did not follow the skeptics and believed in the rationality of science and kept their Vertex’ (VRTX) and Regeneron’s (REGN) shares despite years of negative campaigns against the two firms. Following the rally in VRTX, another rally started in REGN for rational reasons. Both firms are among our TOP TEN selection. More...

Elan's Choices

  Saturday, April 16, 2011

Biogen Idec (BIIB) announced positive results of a process aimed at Identifying high-risk patients for a life-threatening side effect of prolonged use of the multiple sclerosis drug Tysabri. This test is a necessity because it detects an antibody that points to the presence of the Polyomavirus JC (JC virus), which causes the life-threatening progressive multifocal leukoencephalopathy (PML). This side effect in one per thousand patients has deprived Elan from billions of dollars in expected sales of Tysabri.  More...

VERTEX: Time For Celebration

  Wednesday, April 13, 2011

From no treatment at all, to treatments that do not work well, new therapies are reaching the market, probably this month or next, moving hepatitis C virus (HCV) victims from the dark zone of desperation into the light of amazing anticipation – probably of a cure. We don’t know if the opening statement is one of those run-on sentences, from which you might have lost your breath trying to read it to your spouse, but we know with certainty that the news coming from Vertex’ (VRTX) drug is breathtaking. The road has been long, very long, yet, this is always the case when new molecules are designed to become drugs for life-threatening diseases that have yet to find treatments. The fact is that it is a real miracle just finding a road towards conquering a disabling virus that disrupts the functioning of one of the major vital organs. HCV could cause fibrosis (cirrhosis) of the liver, which could, sometimes, necessitate liver transplant or cause cancer. More...

ImmunoGen: No Exaggeration.

  Thursday, April 07, 2011

NO. It was not exaggerated, nor sophisticated what we wrote time and time again about the unparalleled safety and efficacy of the ImmunoGen’s Targeted Antibody Payload (TAP). It is no exaggeration announcing our conviction that cancer products based on Immunogen’s TAP   technology will generate billions of dollars in revenues. What we talked about has, indeed, become today’s great news.  More...

ImmunoGen: Steadfast Determination to Conquer Cancer

  Wednesday, April 06, 2011

The Program Committee of the 102nd Annual Meeting of the American Association for Cancer Research (AACR) taking place in Orlando, FL designated ImmunoGen’s (IMGN) cancer drug IMGN853 poster presentation “Highly Rated.” More...

PROLOR: The Added Value Of Reversible PEGYlation

  Wednesday, March 30, 2011

When Professor Irving Boime and his team at Washington University in St. Louis observed that human chorionic gonadotropin (hCG) has a longer half life than other natural hormones, researchers’ curiosity led them to discover that a carboxyl terminal peptide (CTP) linked to the hormone molecule increases its half life. Experimenting with CTP on other proteins left no doubt in the minds of the researchers that linking Nature’s CTP to protein therapeutics could be safe and effective in extending their half-life. When it comes to reproduction, Mother Nature does not err and does not cause harm. It selected CTP to link to the hormone, which without it, pregnancy would have never been possible. Read also The Value of CTP Technology  More...

SANGAMO: THE NEXT PINNACLE IN HIV TREATMENT?

  Monday, March 07, 2011

When the human immunodeficiency virus (HIV) was identified as the cause of the mysterious immune system depletion characterized by horrific vulnerability to infection, multiple dreadful symptoms and ultimately death, the world panicked. The medical and research communities were taken by surprise. Nothing could be said or done to help the agonizing victims or prevent their imminent death. It took a few years to introduce the first HIV drug, a nucleoside reverse transcriptase inhibitor (NRTI). This milestone was followed by the development and marketing of non-nucleoside reverse transcriptase inhibitors (NNRTI), then a third class antiretroviral protease inhibitors (PIs). As HIV continued to reproduce and resistant variants of the virus emerge, It became necessary to combine the antiretroviral drugs in what has become known as highly active antiretroviral therapy (HAART). Indeed, the HAART combination resulted in profound reductions in AIDS morbidity and mortality.  More...


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