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Novato, California-based BioMarin Pharmaceutical said that the U.S. Food and Drug Administration has granted marketing approval for Kuvan, the first specific drug therapy approved for the treatment of phenylketonuria, or PKU. PKU is a genetic disorder affecting approximately 50,000 diagnosed patients in the developed world and is caused by a deficiency of the enzyme PAH. PAH is needed to metabolize the enzyme phenylalanine, an essential amino acid found in most protein-containing foods. BioMarin said it has begun shipments of Kuvan to its distribution channel and will begin promotion of the drug immediately.

Drug giant GlaxoSmithKline and Redwood City, California-based OncoMed Pharmaceuticals said they entered into a worldwide strategic alliance to discover, develop, and market novel antibody therapeutics to target cancer stem cells. Cancer stem cells are believed to play a key role in the establishment, metastasis, and recurrence of cancer. The alliance with GSK will be conducted through its Center of Excellence for External Drug Discovery, or CEEDD. The companies said the alliance leverages OncoMed's expertise in the discovery and development of cancer stem cell antibody therapeutics and provides GSK with an option to license four product candidates directed at multiple cancer stem cell targets from OncoMed's library of monoclonal antibodies. OncoMed will receive an undisclosed initial payment comprised of cash as well as an equity investment. In addition, OncoMed is eligible to earn milestone payments up to $1.4 billion from GSK based on the achievement of specified discovery, development, regulatory, and commercial milestones. OncoMed will also receive double-digit royalties on all collaboration product sales. 

Amgen announced results from a second randomized, pivotal late-stage clinical trial for a drug to treat chronic Immune Thrombocytopenic Purpura, or ITP. It said its drug Nplate had the desired effect of increasing and sustaining platelet counts in ITP patients who have not had their spleen removed. ITP is a condition in which the body produces antibodies against the platelets in the blood. The Thousand Oaks, California-based biotech giant said Nplate-treated patients taking concurrent ITP medications such as corticosteroids were able to reduce or discontinue these medications. Nplate works similarly to thrombopoietin (TPO), a natural protein in the body. The active peptide component stimulates the TPO receptor, which is necessary for growth and maturation of bone marrow cells and plays a central role in increasing platelet counts.

 

Researchers at the Stanford University School of Medicine have isolated a human blood cell that represents the great-grandparent of all blood cells, a finding that could lead to new treatments for blood cancers and other blood diseases. This cell, called the multipotent progenitor, is the first offspring of the much-studied blood-forming stem cell that resides in the bone marrow and gives rise to all cells of the blood. It's also the cell that is thought to give rise to acute myelogenous leukemia when mutated. Though such cells have been isolated in mice, the research, published in the journal Cell Stem Cell, was the first time the cells had been isolated in human blood.

RHEUMATOID ARTHRITIS: RIGEL REPORTS POSITIVE MID-STAGE RESULTS

South San Francisco, California-based Rigel Pharmaceuticals said its oral syk kinase inhibitor, R788, demonstrated statistically significant results in treating Rheumatoid Arthritis patients in a recently completed mid-stage clinical trial. Groups treated with the drug at both 100mg and 150mg twice a day orally showed higher response rates than a placebo group. The efficacy for both dose groups were “fairly comparable,” according to the company. The onset of the effect in these dose groups occurred as early as one week after initiation of therapy. RA is a progressive, painful and potentially debilitating disease that affects more than 2 million people in the United States. It is a chronic inflammatory disease that puts the body's immune system into overdrive where it ultimately causes inflammation in the joints and destroys soft tissues, cartilage and bone. Rigel's drug stops the progression of the disease by interrupting the cellular signaling at the trigger point of inflammation.

Calrsbad, California-based Isis Pharmaceuticals said that the U.S. Food and Drug Administration has granted orphan drug status to ISIS 333611 for the treatment of an inherited form of amyotrophic lateral sclerosis, or ALS. ALS, also known as Lou Gehrig's disease, is a progressive neurodegenerative disease resulting in the loss of muscle control that can lead to death. ISIS 333611 is Isis' first drug treating neurodegenerative diseases to enter development and is administered directly into the cerebral spinal fluid. ISIS 333611 is a second-generation antisense drug in development for the treatment of ALS through the inhibition of Cu/Zn superoxide dismutase (SOD1), a molecule that is associated with an inherited, aggressive form of ALS. In preclinical rodent studies, inhibition of SOD1 extended survival of rats that showed many features of ALS.

San Diego, California-based Neurocrine Biosciences said it has received communication from the U.S. Food and Drug Administration indicating that it is prepared to approve marketing of the drug indiplon in 5 mg and 10 mg capsules for the treatment of insomnia pending additional clinical and pre-clinical data. The FDA told the company that the approvable letter did not address any of the issues raised previously in a 2006 Approvable Letter from the agency. Neurocrine said it was disappointed in the FDA’s action, but said it will speak to regulators to determine what next steps are required.

Nanogen said it has delivered to the Centers for Disease Control and Prevention the second phase milestone requirements, including the point-of-care prototype device to detect influenza viruses rapidly in clinical samples. The San Diego-based diagnostics maker, along with its business partner HX Diagnostics based in Emeryville, California, had been awarded a contract from the CDC to fund an accelerated multiphase development plan for a rapid diagnostic test for pandemic flu. The company said delivery of the prototype device is an important milestone demonstrating the feasibility of meeting the CDC’s specifications for the rapid test. The company is continuing product development and initiating clinical trials with further funding of the project pending approval by the CDC. Each year 5 percent to 20 percent of the population in the United States is infected with the influenza virus with approximately 36,000 deaths as a result.

University of California at Irvine researchers have identified the chemical switch that triggers the genetic mechanism regulating people’s internal body clock. The researchers said their finding, which uncovers the most specific information about the body’s circadian rhythms to date, identifies a precise target for new pharmaceuticals that can treat sleep disorders and a host of related ailments. The study, which appears in the journal Nature, found that a single amino acid, BMAL1 protein, activates the genes that regulate circadian rhythms.

 

Researchers at the University of California at San Diego have developed a technique using computer modeling to identify potential side effects of pharmaceuticals, and have used it to study a class of drugs that includes tamoxifen, the most prescribed drug in the treatment of breast cancer. Early identification of adverse effects of drugs before they are tested in humans is crucial in developing new therapeutics, as unexpected effects account for a third of all drug failures during the development process. The technique, described online in PLoS Computational Biology, uses the power of computational modeling to screen specific drug molecules using a worldwide repository, the Protein Data Bank, containing tens of thousands of three-dimensional protein structures. Drug molecules are designed to bind to targeted proteins in order to achieve a therapeutic affect, but if the small drug molecule that functions as a “key” attaches to an off-target protein that has a similar binding site, or “lock,” side effects can result. To identify which proteins might be unintended targets, the UCSD researchers take a single drug molecule and look for how it might bind to as many of the proteins encoded by the human proteome as possible. 

Bristol-Myers Squibb and Gilead Sciences said they agreed to commercialize in Europe Atripla, a once-a-day, single-dose combination of three antiviral drugs for the treatment of HIV. If approved by European regulators, Atripla would represent the first and only once-daily single tablet regimen for HIV infection in the European Union. The companies expect the European regulators to issue its decision by the end of the year. Under the agreement between Foster City, California-based Gilead and BMS, the two companies share responsibility for commercializing Atripla throughout the European Union and certain other European countries. Atripla consists of Gilead’s drugs Emtriva and Viread and BMS’s Truvada.

 

Scientists at the University of California at Davis have found that a common antibacterial chemical added to bath soaps can alter hormonal activity in rats and in human cells in the laboratory—and does so by a previously unreported mechanism. The findings come as an increasing number of studies—of both lab animals and humans—are revealing that some synthetic chemicals in household products can cause health problems by interfering with normal hormone action. Called endocrine disruptors, or endocrine-disrupting substances, such chemicals have been linked in animal studies to a variety of problems, including cancer, reproductive failure, and developmental anomalies. The study was the first to examine the hormone-disrupting effects of the antibacterial compound triclocarban, which is also known as TCC, or 3,4,4'-trichlorocarbanilide. It is widely used in household and personal care products, including bar soaps, body washes, cleansing lotions, wipes, and detergents. Triclocarban-containing products have been marketed broadly in the United States and Europe for more than 45 years, and an estimated 1 million pounds of triclocarban are imported annually for the U.S. market. The researchers said consumers should not take their study as guidance on whether to use tricloarban-containing products and that additional analyses of antibacterials and their endocrine effects are planned.