Wyeth Pharmaceuticals, a division of Wyeth (NYSE WYE), announces findings from a placebocontrolled Phase 3 study of bazedoxifene 20 mg extended to five years, which indicated a significant reduction versus placebo in new vertebral fractures in postmenopausal women with osteoporosis. These and other data were presented at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting in Denver, Colo. Bazedoxifene, a selective estrogen receptor modulator (SERM), is under clinical investigation for the prevention and treatment of postmenopausal osteoporosis.

“These new data are important in that they suggest the reduction in vertebral fracture risk with bazedoxifene seen at five years is comparable to that seen at three years,” says Stuart Silverman, M.D., Clinical Professor of Medicine at the University of California, Los Angeles and CedarsSinai Medical Center, and the studys lead investigator.

About Study 301

The results being presented are from a twoyear extension of a threeyear Phase 3 trial, which enrolled 7,492 generally healthy postmenopausal women aged 55 to 85 years with osteoporosis. The primary endpoint of the threeyear Phase 3 study was the incidence of new vertebral fractures. Eligible subjects were randomized to daily treatment with bazedoxifene 20 mg or 40 mg, raloxifene (RLX) 60 mg, or placebo. At the conclusion of the pivotal threeyear study, a total of 4,216 subjects were enrolled in the extension to five years. Patients receiving bazedoxifene 20 mg continued on that dosage, while the RLX 60 mg treatment arm was discontinued (as prespecified in the protocol) after the threeyear database was finalized, and subjects receiving bazedoxifene 40 mg were transitioned to bazedoxifene 20 mg after all subjects completed four years of treatment. At five years, the study showed that the incidence of new vertebral fractures was significantly reduced in the bazedoxifene 20 mg group (4.5%) and in the group transitioned from bazedoxifene 40 mg to 20 mg (3.9%) compared with placebo (6.8%). This corresponds to relative risk reductions of 35% (P=0.014) and 40% (P=0.005), respectively. The incidence of venous thromboembolic events in this Phase 3 clinical trial was higher in bazedoxifenetreated subjects when compared with placebo. This finding is consistent with what was seen at three years.

Additional New Bazedoxifene Data Presented at ASBMR

Assessment of the Effect of Bazedoxifene on NonVertebral Fracture Risk (McCloskey EV, et al)

Data were also presented from a posthoc analysis performed using the Fracture Risk Assessment Tool (FRAX(R)), which was developed by the World Health Organization to calculate a womans 10year risk of experiencing an osteoporotic fracture. This posthoc analysis indicated that the higher a womans risk of a fracture, the greater the reduction in nonvertebral fractures when receiving bazedoxifene therapy based on her FRAX score.

Safety and Tolerability of Bazedoxifene in Postmenopausal Women with Osteoporosis Results of a 5Year, Randomized, Placebocontrolled Phase 3 Trial (de Villiers TJ, et al)

Bazedoxifene fiveyear safety and tolerability data in postmenopausal women with osteoporosis were also presented. The incidence of adverse events (AEs), serious AEs and discontinuations due to AEs was similar to that seen in the placebo group. The incidence of cardiac or cerebrovascular events, including myocardial infarction and stroke, in the bazedoxifene treatment groups was similar to that seen in the placebo group. Subjects treated with bazedoxifene had a higher incidence of deep venous thrombosis, hot flushes and leg cramps compared with placebotreated subjects. The effect of bazedoxifene on the breast and endometrium was comparable to placebo.

Costeffectiveness of Bazedoxifene in the US Incorporating the FRAX(R) Algorithm (Strom, et al)

Data from an analysis performed using a Marcov cohort model and published U.S. cost and epidemiological data evaluated the potential costeffectiveness and intervention thresholds of bazedoxifene treatment (20 mg and 40 mg doses combined) compared to placebo. Costeffective scenarios were projected for women with strong risk factors and with a Tscore above the threshold for osteoporosis. When effect was modeled for nonvertebral fractures, the data suggested that potential cost effectiveness improved further in women from the age of 60 years with prior fracture and at the threshold of osteoporosis (Tscore=2.5 SD).

About Bazedoxifene

Wyeth is pursuing regulatory approval of bazedoxifene for the prevention and treatment of postmenopausal osteoporosis in the United States and other countries worldwide. In April 2009, the European Commission granted marketing authorization for CONBRIZA(TM) (bazedoxifene) for the treatment of postmenopausal osteoporosis in women at increased risk of fracture. Wyeth intends to introduce CONBRIZA in certain European markets following receipt of necessary reimbursement authorizations in those markets.

Bazedoxifene paired with conjugated estrogens (BZA/CE) is also being studied by Wyeth for the treatment of moderatetosevere menopausal vasomotor symptoms such as hot flushes, night sweats and vulvar and vaginal atrophy, and for the prevention of postmenopausal osteoporosis.

About Osteoporosis

Osteoporosis affects an estimated 75 million people in the United States, Europe and Japan. In the United States, the condition is a major public health threat for an estimated 44 million Americans, or 55 percent of the people 50 years of age and older. Osteoporosis is characterized by low bone mass and structural deterioration of bone tissue, leading to bone fragility and an increased risk of fractures. Up to 20 percent of a womans expected lifetime bone loss can occur in the years immediately following menopause. The treatment of postmenopausal osteoporosis could lead to significant improvement in the overall health for millions of women worldwide as well as reduce costs associated with postmenopausal osteoporosisrelated fractures.

About Wyeth Pharmaceuticals

Wyeth Pharmaceuticals, a division of Wyeth, has leading products in the areas of womens health care, infectious disease, gastrointestinal health, central nervous system, inflammation, transplantation, hemophilia, oncology, vaccines and nutritional products.

Wyeth is one of the worlds largest researchdriven pharmaceutical and health care products companies. It is a leader in the discovery, development, manufacturing and marketing of pharmaceuticals, vaccines, biotechnology products, nutritionals and nonprescription medicines that improve the quality of life for people worldwide. The Companys major divisions include Wyeth Pharmaceuticals, Wyeth Consumer Healthcare and Fort Dodge Animal Health.

The statements in this press release that are not historical facts are forwardlooking statements that are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. In particular, clinical trial data are subject to differing interpretations, and the views of regulatory agencies, medical and scientific experts and others may differ from ours. In addition, there can be no assurance that bazedoxifene will be commercially successful in the markets where approved or that bazedoxifene will be approved in the future in other formulations or indications and/or in other countries, including the United States. Other risks and uncertainties that could cause actual results to differ materially from those expressed or implied by forward looking statements include, among others, risks related to our proposed merger with Pfizer, including satisfaction of the conditions of the proposed merger on the proposed timeframe or at all, contractual restrictions on the conduct of our business included in the merger agreement, and the potential for loss of key personnel, disruption in key business activities or any impact on our relationships with third parties as a result of the announcement of the proposed merger; the inherent uncertainty of the timing and success of, and expense associated with, research, development, regulatory approval and commercialization of our products and pipeline products; government costcontainment initiatives; restrictions on thirdparty payments for our products; substantial competition in our industry, including from branded and generic products; emerging data on our products and pipeline products; the importance of strong performance from our principal products and our anticipated new product introductions; the highly regulated nature of our business; product liability, intellectual property and other litigation risks and environmental liabilities; the outcome of government investigations; uncertainty regarding our intellectual property rights and those of others; difficulties associated with, and regulatory compliance with respect to, manufacturing of our products; risks associated with our strategic relationships; global economic conditions; interest and currency exchange rate fluctuations and volatility in the credit and financial markets; changes in generally accepted accounting principles; trade buying patterns; the impact of legislation and regulatory compliance; risks and uncertainties associated with global operations and sales; and other risks and uncertainties, including those detailed from time to time in our periodic reports filed with the Securities and Exchange Commission, including our current reports on Form 8K, quarterly reports on Form 10Q and annual report on Form 10K, particularly the discussion under the caption “Item 1A, Risk Factors” in our Annual Report on Form 10K for the year ended December 31, 2008, which was filed with the Securities and Exchange Commission on February 27, 2009. The forwardlooking statements in this press release are qualified by these risk factors. We assume no obligation to publicly update any forwardlooking statements, whether as a result of new information, future developments or otherwise.

Source Wyeth Pharmaceuticals

Treating seconddegree burns with a nanoemulsion lotion sharply curbs bacterial growth and reduces inflammation that otherwise can jeopardize recovery, University of Michigan scientists have shown in initial laboratory studies.

UM burn surgeon Mark R. Hemmila, M.D., reports today at the Interscience Conference for Antimicrobial Agents and Chemotherapy on results achieved with a nanoemulsion developed at UM and licensed by UM to Ann Arborbased NanoBio Corporation.

The nanoemulsion shows promise in overcoming the limitations of current creams used in burn treatment, which arent able to penetrate skin to kill subsurface bacteria and dont have a strong effect on inflammation, says Hemmila, associate professor of surgery at the UM Medical School.

In a collaborative effort between the UM Department of Surgery and NanoBio Corporation, Hemmila led experiments at the UM Medical School in which a nanoemulsion lotion was able to reduce bacterial growth onethousandfold compared to control animals receiving no treatment or a placebo. The nanoemulsion showed a similar reduction when compared to a topical antimicrobial agent commonly used in people with burns.

The nanoemulsion is made of soybean oil, alcohol, water and detergents emulsified into droplets less than 400 nanometers in diameter. It has proved effective at killing a variety of bacteria, fungi and viruses in previous research.

The scientists used the nanoemulsion to treat partial thickness burns, better known as second degree burns, over 20 percent of the body, to test its effectiveness in the type of injuries doctors commonly see in people brought to tertiary hospital trauma and burn centers. Such burn victims typically require aggressive treatment in intensive care both to rein in infection and to try to prevent vital fluids from leaking from blood vessels into the damaged skin, a dangerous situation caused in part by excessive inflammation within the body.

The nanoemulsion appears to reduce the action of two inflammatory agents or cytokines that play a role in cell signaling during this critical postburn period. Slowing this action may prevent initial burn damage from becoming worse, and thus reduce the severity of the burn and extent of skin grafting needed, says Hemmila.

The findings add one more possible use to a growing list of promising applications for the patented nanoemulsion technology developed by James R. Baker, Jr., M.D., director of the Michigan Nanotechnology Institute for Medicine and Biological Sciences at UM. Baker, a member of the research team, is the Ruth Dow Doan Professor of Nanotechnology and allergy division chief at the UM Medical School. He is founder and CEO of NanoBio Corporation.

Uses for nanoemulsions include treatments for cold sores, now in phase 3 clinical trials, and for toenail fungus and cystic fibrosis infections, as well as vaccines against influenza and bioterrorism agents.

Before the burn treatment can be tested in people, further laboratory studies are needed to examine the nanoemulsions effects on the overall healing process.

Patents/Disclosures The patented nanoemulsion technology is licensed by UM to NanoBio Corporation. Dr. Baker holds an equity interest in the company.

Others involved in the research Stewart C. Wang, M.D., Ph.D., director of the UM Burn Center and professor, UM Department of Surgery; Aladdein Mattar, M.D., UM Department of Surgery; Michael A. Taddonio, B.S., UM Department of Surgery; Joyce A. Sutcliffe, Ph.D., NanoBio Corporation.

Funding National Institutes of Health, American Association for the Surgery of Trauma, American College of Surgeons, and UM Department of Surgery.

Source
Anne Rueter

It is a long, slow slog to treat major depression. Many antidepressant medications are available, but no single biomarker or diagnostic test exists to predict which one is right for an individual. As a result, for more than half of all patients, the first drug prescribed doesnt work, and it can take months to figure out what does.

Now, based on the final results of a nationwide study led by UCLA, clinicians may be able to accurately predict within a week whether a particular drug will be effective by using a noninvasive test that takes less than 15 minutes to administer. The test will allow physicians to quickly switch patients to a more effective treatment, if necessary.

The study, called the Biomarkers for Rapid Identification of Treatment Effectiveness in Major Depression, or BRITEMD, measured changes in brainwave patterns using quantitative electroencephalography (QEEG), a noninvasive, computerized measurement that recognizes specific alterations in brainwave activity. These changes precede improvement in mood by many weeks and appear to serve as a biomarker that accurately predicts how effective a given medication will be. The study results appear in two articles published in the September issue of the journal Psychiatry Research.

Nine sites around the country collaborated on the study, which enrolled a total of 375 people who had been diagnosed with major depressive disorder (MDD). Each individual was given a baseline QEEG at the beginning of the trial and then prescribed the antidepressant escitalopram, commonly known as Lexapro, one of a class of drugs known as selective serotonin reuptake inhibitors that are commonly prescribed for depression. After one week, a second QEEG was taken.

The researchers examined a biomarker called the antidepressant treatment response (ATR) index a specific change in brainwave patterns from the baseline QEEG.

Subjects were then randomly assigned to continue with escitalopram or were given a different drug. A total of 73 patients who remained on escitalopram were tracked for 49 days to see if their results matched the prediction of the ATR biomarker. The ATR predicted both response and remission with an accuracy rate of 74 percent, much higher than any other method available. The researchers also found that they could predict whether subjects were more likely to respond to a different antidepressant, bupropion, also known as Wellbutrin XL.

“Until now, other than waiting, there has been no reliable method for predicting whether a medication would lead to a good response or remission,” said Dr. Andrew Leuchter, professor of psychiatry at the Semel Institute for Neuroscience and Human Behavior at UCLA and lead author of the study. “And that wait can be as long as 14 weeks. So these are very exciting findings for the patient suffering from depression. The BRITE results are a milestone in our efforts to develop clinically useful biomarkers for predicting treatment response in MDD.”

Major depressive disorder is a leading cause of disability, costing society in excess of $80 billion annually; approximately twothirds of these costs reflect the enormous disability associated with the disorder. An estimated 15 million people in the United States experience a depressive episode each year, and nearly 17 percent of adults will experience major depression in their lifetime.

“BRITE study results suggest that the ATR biomarker could potentially provide the greatest clinical benefit for those patients who might be receiving a medication that is unlikely to help them,” Leuchter said. “Our results suggest that it may be possible to switch these patients to a more effective treatment quickly. This would help patients and their physicians avoid the frustration, risk and expense of long and ineffective medication trials.”

Leuchter noted that research has shown that depression patients who do not get better with a first treatment experience prolonged suffering, are more likely to abandon treatment altogether and may become more resistant to treatment over time.

“So the benefits to the individual and to society are enormous,” he said.

An added benefit of the biomarker test, according to Leuchter, is that it is noninvasive, painless and fast about 15 minutes and only involves the placement of six electrodes around the forehead and on the earlobes.

Aspect Medical Systems, which developed the ATR biomarker, provided financial support for the study. Aspect also participated in the design and conduct of the study; the collection, management, analysis and interpretation of the data; and the preparation and review of the manuscript. Final approval of the form and content of the manuscript rested with the authors.

Other UCLA authors included Dr. Ian Cook, Dr. Karl S. Burgoyne and Dr. James T. McCracken. Leuchter is chair of Aspects neuroscience advisory board and has provided scientific consultation to them.

Source

The U.S. Food and Drug Administration (FDA) cleared the OVA1(TM) Test, the first blood test that, prior to surgery, can help physicians determine if a woman is at risk for a malignant pelvic mass. OVA1 is the first FDAcleared laboratory test that can indicate the likelihood of ovarian cancer with high sensitivity prior to biopsy or exploratory surgery, even if radiological test results fail to indicate malignancy. The test was developed by Vermillion, Inc. (OTC VRMLQ.PK), a molecular diagnostics company, in cooperation with Quest Diagnostics (NYSE DGX), the worlds leading provider of cancer diagnostics. Quest Diagnostics, which is a longtime investor in research and development of the OVA1 technology, has exclusive rights to offer the test to the clinical reference laboratory market in the U.S. for three years.

“When combined with other clinical information, the OVA1 biomarker panel can help assess the likelihood of malignancy of an ovarian tumor before surgery and facilitate decisions about referral to a gynecologic oncologist,” said Frederick R. Ueland, M.D., principal investigator of the prospective, multicenter OVA1 clinical trial. Dr. Ueland is an associate professor gynecologic oncology at the University of Kentuckys Markey Cancer Center.

The OVA1 Test is an in vitro diagnostic multivariate index (IVDMIA) test that combines the results of five immunoassays using a proprietary unique algorithm to produce a single numerical score indicating a womens likelihood of malignancy. The OVA1 Test provides a new option in the preoperative evaluation to help physicians assess if a pelvic mass is benign or malignant in order to help determine whether to refer a woman to a gynecologic oncologist for surgery. Numerous clinical practice guidelines recommend that women with ovarian cancer be under the care of a gynecologic oncologist. However, only an estimated one third of women who undergo surgery for possible ovarian cancer are referred to these specialist surgeons for their surgery.(1)

Vermillion received the Society for Gynecologic Oncologists (SGO) Basic Science Poster Award for an abstract on the performance of its OVA1 Test presented at SGOs 38th Annual Meeting on Womens Cancer in 2007. In reviewing the test application, the FDA evaluated results of a prospective, doubleblind clinical trial which included 27 demographically mixed sites representative of institutions where ovarian tumor subjects may undergo a gynecological examination.

“Surgery in the hands of a gynecologic oncologist is usually associated with more favorable patient outcomes,” said Jon R. Cohen, M.D., chief medical officer and senior vice president, Quest Diagnostics. “Physicians often do not know if a womans pelvic mass is malignant or benign until she undergoes surgery. The OVA1 Test is the first FDAcleared blood test to help clinicians determine whether to refer a woman to a gynecologic oncologist or have a gynecologic oncologist present at the time of surgery. We believe this test will help drive more favorable patient outcomes.”

“Unfortunately, advances in ovarian cancer diagnosis and treatment are few and far between. It is fitting that September, Ovarian Cancer Awareness Month, marks FDAs clearance of OVA1, a test that represents an important step forward toward improved outcomes,” said Gail S. Page, executive chairperson of the board of directors of Vermillion. “Quest Diagnostics had the foresight to recognize the potential value of this novel multivariate assay and supported its development. We look forward to collaborating to bring this new diagnostic option to the many women who will benefit from specialist care.”

The FDA clearance of OVA1 makes Quest Diagnostics the only diagnostic testing company to offer FDA cleared tests for ovarian cancer in the pre and postsurgical settings. In addition to offering the OVA1 Test, Quest Diagnostics was the first laboratory company to provide a new lab test that the FDA cleared in the third quarter of 2008 as an aid for monitoring for recurrence of epithelial ovarian cancer.

The OVA1 Test will be available for physician use in the fourth quarter of this year.

Ovarian cancer is the leading cause of death from gynecologic cancers in the United States and the fifthleading cause of cancer deaths in women.(2) Approximately 21,600 new cases of ovarian cancer will be diagnosed in the U.S. in 2009, and approximately 14,600 women will die of the disease.(3)

About the OVA1 Test

The OVA1 Test is a qualitative serum test that combines the results of five immunoassays into a single numerical score. It is indicated for women who meet the following criteria over age 18, ovarian adnexal mass present for which surgery is planned, and not yet referred to an oncologist. The test utilizes five wellestablished biomarkers Transthyretin (TT or prealbumin), Apolipoprotein A1 (Apo A1), Beta2Microglobulin (Beta2M), Transferrin (Tfr) and Cancer Antigen 125 (CA 125 II) and a proprietary algorithm to determine the likelihood of malignancy in women with pelvic mass for whom surgery is planned.

The OVA1 Test is an aid to further assess the likelihood that malignancy is present when the physicians independent clinical and radiological evaluation does not indicate malignancy. The test should not be used without an independent clinical/radiological evaluation and is not intended to be a screening test or to determine whether a patient should proceed to surgery. Incorrect use of the OVA1 Test carries the risk of unnecessary testing, surgery, and/or delayed diagnosis.

About Vermillion

Vermillion, Inc. is dedicated to the discovery, development and commercialization of novel highvalue diagnostic tests that help physicians diagnose, treat and improve outcomes for patients. Vermillion, along with its prestigious scientific collaborators, has diagnostic programs in oncology, hematology, cardiology and womens health. Vermillion is based in Fremont, California.

About Quest Diagnostics

Quest Diagnostics is the worlds leading provider of diagnostic testing, information and services that patients and doctors need to make better healthcare decisions. The company offers the broadest access to diagnostic testing services through its network of laboratories and patient service centers, and provides interpretive consultation through its extensive medical and scientific staff. Quest Diagnostics is a pioneer in developing innovative diagnostic tests and advanced healthcare information technology solutions that help improve patient care.

(1) Journal of the National Cancer Institute, Vol. 98, No. 3, February 1, 2006

(2) Greenlee RT, Murray T, Bolden S, Wingo PA. Cancer statistics, 2000. CA Cancer J Clin. 2000;50(1)733

(3) 2009 American Cancer Society

OVA1(TM) Test is a trademark of Vermillion Inc.

Quest, Quest Diagnostics, the associated logo, and all associated Quest Diagnostics marks are the registered trademarks of Quest Diagnostics. All third party marks (R) and (TM) are the property of their respective owners

PTC Therapeutics, Inc. (PTC) announced the initiation of a Phase 3 trial of ataluren (formerly PTC124(R)), an investigational protein restoration therapy in patients with nonsense mutation cystic fibrosis (nmCF). Patients with CF lack adequate levels of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, a chloride channel necessary for normal function of the lung, pancreas, liver, and other organs. In nmCF, an interruption in the genetic code known as a nonsense mutation prematurely halts the synthesis of CFTR, causing the protein to be short and nonfunctioning. Nonsense mutations are categorized as Class I mutations that result in little or no production of the CFTR protein. CF patients with Class I mutations typically experience more severe disease symptoms than those with lowrisk genotypes, including a greater than twofold increased risk of death,(1) a higher probability of endstage lung disease,(2) and a higher prevalence of pancreatic insufficiency.(2) Ataluren is designed to promote restoration of the missing CFTR. Through advances in genetic analysis, a simple test can now determine if a patients disease is caused by a nonsense mutation.

The primary objective of the registrationdirected doubleblind, placebocontrolled study is to evaluate whether ataluren can improve lung function, as measured by forced expiratory volume in one second (FEV1), in patients with nmCF. Other outcome measures will evaluate whether ataluren can reduce symptoms associated with nmCF, decrease lung infections, reduce the frequency of cough, and improve patientreported quality of life. The 48week trial is now enrolling patients at multiple research centers in North America, Europe, and Israel. Study candidates include patients who are at least six years of age and have CF due to a nonsense mutation.

“Promising Phase 2 clinical trial data show that patients treated with ataluren can produce functional CFTR protein, resulting in improvements in chloride channel activity,” said Frank Accurso, M.D., Professor of Pediatrics and Section Head of Pulmonary Medicine of the University of Colorado, Denver and a leading ataluren investigator. “With the newly initiated Phase 3 study we hope to determine if the effects of ataluren on the underlying cause of the disease can result in clinical benefit for patients with nmCF.”

“As an oral therapy that may address the underlying cause of the disease, ataluren has the potential to improve the management of nmCF for patients and their physicians,” said Christiane DeBoeck, M.D., Ph.D., Principal Investigator for University Hospital Leuven. “It is our hope that this longterm clinical trial of ataluren will advance our knowledge of the disease and the standard of care for nmCF patients.”

“The initiation of this Phase 3 trial represents an important step forward in our efforts to develop treatments for the underlying cause of cystic fibrosis,” said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. “Phase 2 studies of ataluren showed encouraging potential to restore CFTR protein production and we are pleased to be supporting this study.” In July 2008, Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit affiliate of the Cystic Fibrosis Foundation, expanded its existing collaboration with PTC Therapeutics. PTC will receive up to $25 million from CFFT in support of the development of ataluren.

An orally administered therapy, ataluren is the first investigational new drug designed to enable the formation of a functioning protein in patients with genetic disorders due to a nonsense mutation. Restoring functional CFTR protein may enable ataluren to address the underlying cause of nmCF and change the course of the disease. Currently available therapies for CF focus only on the alleviation of symptoms.

“We are excited to expand the ataluren registration program to include nmCF, an area of significant unmet medical need,” said Langdon Miller, M.D., Chief Medical Officer of PTC Therapeutics. “We are hopeful that this Phase 3 trial together with our ongoing studies in nmDMD/BMD, will add to a growing body of data demonstrating atalurens potential for treatment of patients with nonsense mutation genetic disorders.”

About Cystic Fibrosis (CF)

CF is a lifethreatening genetic disease that causes serious lung infections and digestive complications. According to the Cystic Fibrosis Foundation, CF affects approximately 30,000 adults and children in the United States and nearly 70,000 people worldwide. Genetic testing is required to confirm a complete diagnosis and to determine if a patients disease is caused by a nonsense mutation. It is estimated that nonsense mutations are the cause of CF in 10 percent of patients in the United States and Europe and over 50 percent of patients in Israel. Available treatments for CF are designed to alleviate symptoms rather than correct the underlying cause of the disease. These treatments include chest physical therapy to clear thick mucus from the lungs, antibiotics to treat lung infections, and a mucusthinning drug designed to reduce the number of lung infections and improve lung function. In addition, the majority of cystic fibrosis patients take pancreatic enzyme supplements to assist with food absorption in digestion.

About Ataluren (PTC124(R))

Ataluren is the first investigational new drug designed to restore the formation of a functioning protein in patients with genetic disorders due to a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. Ataluren is currently being investigated for use in patients with nmCF and nmDMD/BMD.

Ataluren has been granted orphan drug status for the treatment of nmCF and nmDMD/BMD by the U.S. Food and Drug Administration (FDA) and the European Commission. The FDA has also granted ataluren Subpart E designation for expedited development, evaluation, and marketing. The development of ataluren has been supported by the Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation), the FDA Office of Orphan Products Development, the Muscular Dystrophy Association, Parent Project Muscular Dystrophy, and the National Center for Research Resources.

Completed Ataluren Clinical Trials

Data from Phase 2a clinical trials of ataluren in pediatric and adult patients with nmCF show that administration of ataluren results in production of functional CFTR and statistically significant improvements in CFTR chloride channel function in the airways. Ataluren treatment is also associated with reductions in cough frequency and improvements in pulmonary function tests.

Across all ataluren clinical trials to date, including Phase 1 healthyvolunteer trials, ataluren has been generally well tolerated. In Phase 2a trials in nmCF and nmDMD/BMD, adverse events have been largely consistent with background symptoms and have usually been mild. No concerning adverse findings have been identified based on physical examinations, vital sign measurements, electrocardiograms, or laboratory studies. The mean compliance with ataluren therapy has been greater than 90 percent in all trials.

Collaboration with Genzyme

PTC Therapeutics has an exclusive collaboration with Genzyme Corporation for the development and commercialization of ataluren. PTC Therapeutics will market ataluren in the United States and Canada, while Genzyme will commercialize the product in other regions of the world.

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation, the leading organization focused on curing and controlling cystic fibrosis (CF), has invested nearly $300 million in drug research with biotech companies since 1998 to develop therapies to fight CF. As a result, the Foundation has built a drug pipeline with more than 30 promising therapies in development. Virtually all the approved CF therapies available today were made possible because of the support of the Foundation. Based in Bethesda, MD, the Foundation has 70 chapters and branch offices, and supports and accredits a nationwide network of more than 110 CF care centers that provide treatment and vital resources to patients and families.

About PTC Therapeutics

PTC is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary, smallmolecule drugs that target posttranscriptional control processes. Posttranscriptional control processes regulate the rate and timing of protein production and are of central importance to proper cellular function. PTCs internally discovered pipeline addresses multiple therapeutic areas, including genetic disorders, oncology, and infectious diseases. PTC has extensive knowledge of posttranscriptional control processes and has developed proprietary technologies that it applies in its drug discovery activities. PTCs expertise has been the basis for collaborations with leading biopharmaceutical companies such as Genzyme, Pfizer, Celgene, Gilead, Roche and ScheringPlough.

References

(1) McKone EF, Goss CH, Aitken ML. CFTR genotype as a predictor of prognosis in cystic fibrosis. Chest 2006 Nov;130(5)14417.

(2) de Gracia J., Mata F, Alvarez A, Casals T, Gatner S, Vendrell M, de la RD, Guarner L, Hermosilla E. Genotypephenotype correlation for pulmonary function in cystic fibrosis. Thorax 2005 Jul;60(7)55863.

Source PTC Therapeutics, Inc

UroToday.com Fragmentation of a prostate biopsy core can potentially impact the pathologic interpretation. A report in online version of the British Journal of Urology International by Dr. Jonathan Epsteins group at Johns Hopkins University assesses this variable.

A retrospective review was performed from 2007 to 2008 for prostate biopsy cases that contained prostate cancer (CaP) in fragmented cores. A total of 463 cases were identified. A control set of 200 cases with CaP but no fragmented cores served as the control group. The comparison included the number of parts per case, the number of cores per specimen part, and number of parts containing cancer. The highest overall Gleason score in the cases with unfragmented cores was compared with the highest Gleason score within fragmented cores. They found the mean number of parts per case in the fragmented group was 7.5 with no significant difference in the number of parts per case in the fragmented and unfragmented groups.

The mean number of cores per specimen was higher in the fragmented group (2.6) compared to the unfragmented group (2.1). Analysis confirmed an association between fragmentation and the number of cores per part. There were a greater number of parts with CaP in the fragmented group (2.8) compared to the unfragmented group (1.6). Fragmentation was associated with the number of parts containing cancer. Fragmented cores also were associated with a higher Gleason score mean (6.6) compared to 6.2 in the unfragmented group.

The differences cited in this analysis may impact models that assess patient risk and outcomes. For example, the percentage or number of cores involved with cancer is used to estimate outcomes or determine if a patient is a candidate for active surveillance. The accurate percentage of a core involved with CaP cannot be determined when cores are fragmented. The investigators point out that a fragmented core involved with 50% CaP might actually only be involved with 10% of the entire core prior to fragmentation. Similarly, the Gleason score can be affected if cancer is contained in two fragments and the most predominant pattern may be altered by the fragmentation. They felt that Gleason grade 4 tissue is more likely to fragment as it contains less stroma to hold it together.

Fajardo DA, Epstein JI
BJU Int. 2009 Jul 6. Epub ahead of print.
doi10.1111/j.1464410X.2009.08737.x

Written by UroToday.com Contributing Editor Christopher P. Evans, MD, FACS UroToday the only urology website with original content written by global urology key opinion leaders actively engaged in clinical practice. To access the latest urology news releases from UroToday, go tourotoday.com

Marketing exclusivity periods for firstinclass drugs have fallen dramatically in recent decadesfrom a median of 10.2 years in the 1970s to 2.5 years in the early part of this decadeunderscoring the competitive nature of drug development, a new study recently completed by the Tufts Center for the Study of Drug Development has found.

According to the study, the average time between first and second followon drugs fell even more rapidly from a median of 16.1 years in the 1960s to 1.1 years in the 200003 period.

“Since the early 1990s, nearly one in three followon drugs entered clinical testing earlier than did the firstinclass drug,” said Tufts CSDD Director of Economic Analysis and study author Joseph A. DiMasi, Ph.D., noting that “distinctions about innovativeness drawn between firstinclass and followon drugs may not be meaningful.”

He added that, contrary to widespread belief that development of followon drugssometimes called “metoo” drugsbegins after the first new drug in a therapeutic category receives marketing approval, development of nearly all followon drugs begins well before the firstinclass drug receives approval.

“New drug development remains a competitive race, and the first candidate to receive marketing approval to address a specific indication belongs to the sponsor that completes the development gauntlet first,” DiMasi said.

The study, reported in the September/October Tufts CSDD Impact Report, released today, also found that

Approximately onehalf of what turned out to be followon drugs had a U.S. or worldwide patent filed before the firstinclass drug had filed any such patents.

Since the early 1990s, 90% of followon drugs had initial pharmacologic testing and 87% were in clinical studies somewhere in the world prior to the firstinclass drug approval.

The average time for market entry between second and third followon drugs also dropped significantly, from a median of 5.1 years in the 1960s to 1.3 years in the 1990s.

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Nearly 600,000 West Africans Affected By Flooding, U.N. Says

The U.N. on Monday “sharply increased its toll of the number of people affected by floods in West Africa, putting the number at more than 592,000 in no less than 10 countries,” Agence FrancePresse reports. Yvon Edoumou, a spokesperson for the U.N. Office for the Coordination of Humanitarian Aid (OCHA), said the situation is the result of more rain, a lack of infrastructure and minimal preparation for the rainyseason by governments in the region (9/7). Almost 70 people in the area have died and hundreds of thousands still face health risks, said Herve Ludovic de Lys, OCHA region head, Reuters reports (Matthews, 9/6). “Sanitation and hygiene deteriorate quickly when West Africa floods. Sewage mixes with drinking water. Electricity fails. Health centers are damaged. Hectares of standing water become breeding grounds for mosquitoes that spread malaria,” VOA News (Stearns, 9/7).

Sri Lankan Government Orders UNICEF Official To Leave Country

James Elder, UNICEFs communications head in Sri Lanka, was ordered to leave the country within two weeks and his visa canceled, after he expressed “concerns about the plight of Tamils in the governmentrun welfare camps,” the Guardian reports (Ramesh, 9/6). UNICEF issued a statement on Monday, defending Elder “Through Mr. Elder, UNICEF has consistently spoken out against the suffering of children on both sides of the intense hostilities earlier this year and called for their protection … [we] will continue to uphold its mandate in Sri Lanka, and elsewhere, to advocate and speak out on behalf of vulnerable children and women” (9/7).

South Africa Launches Two Week Child Health Campaign

South Africas Health Ministry “launched a major campaign Monday to get vaccinations and immunityboosting vitamins to 3 million children across the country over the next two weeks,” the SAPA/AP/Independent Online write. For the campaign, temporary clinics have been set up and health workers are going doortodoor to offer interventions aimed at reducing deaths from diarrhea, pneumonia and measles (9/7).

Christian Science Monitor Profiles Development Worker In Rwanda

The Christian Science Monitor profiles Josh Ruxin, who runs “several publichealth projects in Rwanda,” including Rwanda Works. The organization “draws on Ruxins experience as founder of the Millennium Village in Rwanda, one of 80 experimental villages across Africa using communitybased strategies to end extreme poverty. The villages success malaria rates have dropped; healthcare has improved; and where for years there had been chronic hunger, farmers are now reaping bumper harvests partly inspired the Rwandan government to roll out a similar grassroots antipoverty plan across the country” (Moore, 9/6).

High Risk Of Epidemics In Southern Sudan Area Attacked By LRA, WHO Says

Ezo County in southern Sudan “largely inaccessible to humanitarian workers” is at “high risk of epidemicprone diseases,” because repeated attacks by the Ugandan rebel Lords Resistance Army have significantly damaged health facilities in the area, the WHO says, VOA News reports. “By damaging health facilities you firstly damage a neutral target, something that should always be protected and then you prevent people from accessing health services by destroying and stopping that health facility from operating,” Paul Garwood, a WHO spokesperson, said (Schlein, 9/6).

U.S. Funding Helps To Train Health Professionals On How To Control The Spread Of VectorBorne Diseases

The International News examines a U.S.supported program in Pakistan that is training health professionals to help control the spread of vectorborne diseases. According to the newspaper, the project is “part of the over $3.4 billion aid that the U.S. government is providing to Pakistan over five years to improve economic growth, education, health and governance, and to assist with earthquake reconstruction” (9/4).

This information was reprinted from globalhealth.kff.org with kind permission from the Henry J. Kaiser Family Foundation. You can view the entire Kaiser Daily Global Health Policy Report, search the archives and sign up for email delivery at globalhealth.kff.org.

© Henry J. Kaiser Family Foundation. All rights reserved.

The NPA has called for all registered pharmacies to be exempt from the Welsh Assembly Governments proposals for there to be a charge to single use carrier bags.

Margaret Peycke, NPA Information Manager, said “Medical products or surgical appliances must be suitably and securely packaged so that the contents cannot be identified during transport from the dispensary, or in the case of medicines purchased for selfcare from the pharmacy counter. For larger items or a large number of smaller items this will involve the use of single use carrier bags. The reuse of patients own bags may present not only hygiene considerations but practical problems for pharmacies, especially when the pharmacy collects the prescription from the surgery, orders the items, dispenses them and has them waiting for the patient or their carer to collect. To ensure confidentiality and security the majority of pharmacies bag and seal the items on a prescription as soon as it has been dispensed.”

“To exempt part of the business from charging for the bags and expect the charge to be levied when the bags are used for other products for example baby nappies and milk will be virtually impossible to interpret, implement and maintain accurate records. We therefore propose that all supply of single use carry bags by community pharmacy should be exempt from the levy. This is a pragmatic solution to what otherwise would be a situation which is not only near impossible to manage but also risks damaging the relationship between patients and healthcare professionals in the community.”

An extensive review has failed to find good evidence which convincingly demonstrates reflexology (a practice involving applying pressure to, or, massaging feet) is an effective treatment for any medical conditions.

Details of the review, conducted by Dr Edzard Ernst, Director of Complementary Medicine at the Peninsula Medical School in the United Kingdom, were published in the Medical Journal of Australia.

“There is little doubt that a foot massage is pleasantly relaxing, however specific medical claims should always be supported by sound evidence,” Dr Ernst said.

“In the case of reflexology, this unfortunately does not appear to be the case.”

Dr Ernst searched six electronic databases and identified 217 trials related to reflexology.

After the methodological quality of the trials was assessed by two independent reviewers, all but 18 randomised controlled trials were excluded from the review.

Of these 18 remaining studies, 12 failed to show convincingly that reflexology is an effective treatment, five suggested positive effects and the direction of one result was unclear.

“The methodological quality was often poor, and sample sizes were generally low,” Dr Ernst said.

“Most higherquality trials did not generate positive findings.”

Dr Ernst warned patients against using reflexology as a diagnostic tool.

“Most proponents of reflexology would argue that this method is free of risks,” he said.

“However, if used as a diagnostic tool, it will generate falsepositive and falsenegative diagnoses. Moreover, if employed as an alternative therapy to treat serious conditions, reflexology can be lifethreatening.”