Wounds and Healing

ROXRO PHARMA, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved SPRIX™ (ketorolac tromethamine) Nasal Spray, for the short-term (up to 5 days) management of acute moderate to moderately severe pain that requires analgesia at the opioid level. SPRIX is a prescription intranasal formulation of the analgesic ketorolac, a non-steroidal anti-inflammatory drug (NSAID), designed to provide ambulatory patients with a convenient, potent, and fast-acting option for acute moderate to moderately severe pain relief. Ketorolac tromethamine is a frequently administered non-narcotic injectable analgesic for moderately severe pain in U.S. hospitals today. The approval of SPRIX provides acute pain outpatients with a non-narcotic and easy-to-administer alternative to commonly prescribed opioids.

“The FDA approval of SPRIX provides an important new tool for physicians treating patients with moderate to moderately severe pain. This is particularly significant at a time when patients and physicians have become increasingly concerned with use of narcotic pain relievers,” said Roberto Rosenkranz, Chief Executive Officer of ROXRO. “Currently approved injectable forms of ketorolac are already well accepted by the physician community for in-hospital use, so we believe uptake of this more convenient form will be rapid.”

“SPRIX fills the need for a new non-opioid, non-injectable option for ambulatory pain control, because it minimizes the potential for abuse as well as the negative side effects associated with narcotic pain relievers while providing potent control of moderate to moderately severe pain at the opioid level,” said Askomur Buvanendran, Director Orthopedic Anesthesia, Rush University Medical Centers, Chicago, IL. “The convenient nasal spray formulation will also provide pain relief outside of the hospital setting.”

About SPRIX

SPRIX is a novel intranasal formulation of the potent non-steroidal anti-inflammatory drug (NSAID) ketorolac. Currently, ketorolac is most often administered in the hospital setting as an injection for the short-term treatment of moderately severe pain. Formulated as an easy-to-use spray, SPRIX is rapidly absorbed through the nasal mucosa, achieving peak blood levels as fast as an intramuscular injection of ketorolac. SPRIX has been studied in moderate to moderately severe pain, both alone and in combination with morphine. The New Drug Application package for SPRIX included data from more than 1,000 subjects and 14 clinical trials. SPRIX has been tested in four controlled efficacy studies, and met the primary efficacy endpoints in each trial. Phase 3 studies of adults who underwent elective abdominal or orthopedic surgery (n=300 and n=321) indicated that SPRIX provided a statistically significant greater reduction in the summed pain intensity difference, a commonly accepted measure of pain, over 48 hours as compared to those using placebo. SPRIX has also demonstrated a 26-36 percent reduction in morphine use by patients over a 48-hour period as compared with placebo.

Important Safety Information About SPRIX

WARNING: LIMITATIONS OF USE, GASTROINTESTINAL, BLEEDING, CARDIOVASCULAR, and RENAL RISK

– Limitations of Use The total duration of use of SPRIX and other ketorolac formulations should not exceed 5 days.

– Gastrointestinal (GI) Risk Ketorolac can cause peptic ulcers, GI bleeding, and/or perforation of the stomach or intestines, which can be fatal. SPRIX is CONTRAINDICATED in patients with peptic ulcer disease or history of GI bleeding.

– Bleeding Risk SPRIX inhibits platelet function and is CONTRAINDICATED in patients with suspected or confirmed cerebrovascular bleeding, hemorrhagic diathesis, incomplete hemostasis, or high risk of bleeding.

– Cardiovascular (CV) Risk NSAIDs may cause an increased risk of serious CV thrombotic events, myocardial infarction, and stroke, which can be fatal. This risk may increase with duration of use. Patients with CV disease or risk factors for CV disease may be at greater risk. SPRIX is CONTRAINDICATED for treatment of peri-operative pain in the setting of coronary artery bypass graft (CABG) surgery.

– Renal risk SPRIX is CONTRAINDICATED in patients with advanced renal impairment and in patients at risk for renal failure due to volume depletion.

Mild, transient nasal discomfort was the most frequently reported side-effect of SPRIX use. SPRIX is CONTRAINDICATED in patients with known hypersensitivity or a history of allergic reactions to aspirin, ketorolac, other NSAIDs or EDTA; in patients at risk for GI bleeding; prior to major surgery or during the peri-operative period in CABG surgery; in patients with advanced renal disease or volume depletion; patients with certain bleeding risks and during labor and delivery. SPRIX should not be used concurrently with probenecid or pentoxifylline. Treat patients for the shortest duration possible, and do not exceed 5 days of therapy with SPRIX. See the full prescribing information for more details.

About ROXRO

ROXRO PHARMA, Inc. is a late-stage specialty pharmaceutical company developing hospital-strength acute-pain products for convenient use by patients in the home setting. The company is led by a seasoned management team that has discovered, developed and commercialized several pain and cardiovascular medicines that are widely prescribed today.

Source: ROXRO PHARMA, Inc

View drug information on Pentoxifylline; Sprix.

Sinapis Pharma is pleased to announce that it has received notice from the FDA that its Investigational New Drug application (IND) for the use of IV methamphetamine for acute stroke was approved on April 9, 2010. “We have accomplished a great deal of work for a small company on a very tight budget. We have GMP drug ready for the Phase I clinical trial and the Phase I site is prepared to begin this protocol,” said Dr. Donald Picker , the company CEO. Following completion of the Phase I trial the company will agree with the FDA on a protocol to treat stroke victims and commence its proof of principle clinical trial with the world’s leading stroke clinicians designing and running the program. The company also continues to produce encouraging data in animal models of traumatic brain injury, an even larger clinical indication and potential market for this drug treatment.

Sinapis Pharma, Inc. is an early stage biotechnology company whose lead compound methamphetamine, as a low-dose intravenous infusion, has shown remarkable pre-clinical efficacy in acute stroke and brain injury models.

Source: Sinapis Pharma, Inc

Genzyme Corp. has signed a consent decree agreeing to correct manufacturing quality violations at its Allston, Mass., manufacturing facility and will turn over to the federal government $175 million in unlawful profits from the sale of products that were made at the plant, the U.S. Food and Drug Administration announced today.

Under the consent decree of permanent injunction, the Cambridge, Mass.-based company agreed to adhere to a strict timetable to bring the plant in line with the regulatory requirements of the FDA.

The payment is known as a disgorgement in which a company must give up profits obtained by improper or illegal acts.

The decree was agreed to by Genzyme, its Chief Executive Officer Henri A. Termeer; its Senior Vice President for Manufacturing Sciences and Technical Operations, W. Blair Okita; and its Senior Vice President for Global Product Quality, Ronald Branning. It was filed in the U.S. District Court for the District of Massachusetts on May 24, 2010, and is subject to court approval.

Genzyme is a sole supplier of several enzyme replacement drugs for injection that are used to treat rare genetic disorders. During an inspection of the Allston plant from Oct. 8, 2009, until Nov.13, 2009, FDA inspectors found that the company’s systems for ensuring manufacturing quality were inadequate resulting in production delays, critical shortages of medically necessary products to consumers and drugs contaminated with metal, fiber, rubber and glass particles. These manufacturing problems violated the FDA’s regulations for manufacturing practice. Genzyme also temporarily suspended manufacturing of some products due to a viral contamination in a bioreactor that makes bulk amounts of its drugs. This resulted in additional drug shortages.

“It is critical for the safety of the drug supply that companies comply with basic manufacturing standards. FDA takes these obligations very seriously and expects manufacturers to do the same,” said Joshua Sharfstein, M.D., FDA principal deputy commissioner.

Cerezyme, Fabrazyme, Myozyme, and Thyrogen are drug products that undergo all or some stages of manufacture at the Allston plant. Cerezyme treats Gaucher’s disease, which causes fatty substances to accumulate in the liver, spleen and other organs. Fabrazyme treats Fabry disease, which prevents the body from breaking down oils and fats that build up in the eyes and the kidneys. Myozyme treats Pompe disease, a muscular disorder, and Thyrogen is used to diagnose thyroid cancer.

Genzyme has agreed to a work plan for making facility improvements. It begins with selecting, within 10 days of entry of the decree by the court, an independent expert who will inspect the plant and issue recommendations. Genzyme will use the expert’s recommendations to create a work plan, subject to FDA approval, that requires specific steps for bringing its Allston plant into compliance within given dates. If Genzyme fails to complete any step specified in the work plan, the company will have to pay a substantial fine. In addition, the consent decree provides a deadline for Genzyme to transfer its operations for filling drug vials from its Allston facility to other manufacturing sites or else it will have to disgorge further profits from the sales of drugs filled at Allston after that specific date.

FDA is working with Genzyme during the company’s remediation to ensure availability of the company’s medically necessary drugs.

For more information:
- CDER statement on availability of Thyrogen

Source
U.S. Food and Drug Administration

View drug information on Fabrazyme; Myozyme.

Kate Reid, general manager of Atlantis Healthcare UK, outlines the benefits of an individualised approach to non-compliance. “Current research suggests that 50 per cent of patients are non-adherent,” says Reid. “More worrisome is the fact that 60 to 70 percent of these patients are deliberately non-adherent. That means they’re not forgetful or financially strained, but sceptical of the treatment they’ve been prescribed.”

Reid says the reason for this scepticism stems from deep misperceptions of their own conditions (many like to believe they’re not as sick as the doctor says) and of their medications (many believe half the dosage will suffice on days when they’re feeling better). To address this intentional non-adherence, Reid believes patient support programmes must move beyond reminders and leaflets in favour of a more personalised outreach that addresses each patient’s logic for avoiding his or her medication.

To listen to the full interview as a podcast – click here

Reid will be providing a workshop focusing on patient adherence solutions at eyeforpharma’s 7th Annual Patient Adherence Summit (16-17 June, Zurich). Professor John Weinman, Head of Department, Health Psychology, King’s College London will lead the discussion with a team of health psychologists to answer your questions and review in detail adherence solutions that have been proven to work. To find out more about the conference, visit the Patient Adherence & Relationship Marketing Summit website.

Source:
Camilla Ohlsson
VP – Europe
eyeforpharma
T: +44 (0) 207 375 7223
E: cohlssoneyeforpharma
Join me on LinkedIn

Achievement of a milestone in creating synthetic life in the form of a self-replicating cell from synthesized DNA has focused attention on the field of synthetic biology. Researchers, academics and business leaders will discuss the evolution of the technology and the ethics, regulation and the potential commercial uses of synthetic biology during the seventh annual World Congress on Industrial Biotechnology and Bioprocessing, June 27-30, 2010 in Washington, D.C.

Brent Erickson, executive vice president for BIO’s Industrial and Environmental Section, stated, “Recent breakthroughs in the field of synthetic biology are focusing increased attention not only on the commercial possibilities but also on the development of ethical guidelines and regulatory policies for the science. Successful early applications for biofuels and bioremediation of pollutants show the potential of synthetic biology as a tool of industrial biotechnology that can produce environmental benefits. The annual World Congress promises significant discussions and presentations on the practical aspects of synthetic biology research.”

Sessions from the track on Synthetic Biology and Metabolic Engineering include:

Monday June 28

Synthetic Biology: The Difficult We Do Immediately, The Impossible Takes a Little Longer
8:45 a.m. – 10:15 a.m.

- Kevin Jarrell, Modular Genetics, Inc.
- Marcel Wubbolts, DSM, NV
- Lori Giver, Codexis, Inc

Creating the Super Strain: How Metabolic Engineering Will Drive the Future of Advanced Fuels
2:30 p.m. – 4:00 p.m.

- Dan Robertson, Joule Unlimited, Inc.
- Stephen del CardayrГ©, LS9, Inc.
- Balu Sarma, Praj Matrix – A Division of Praj Industries Ltd.

Tuesday, June 29

Ethical, Social, and Policy Issues of Synthetic Biology
9:00 a.m. – 10:30 a.m.

- Paula Olsiewski, The Alfred P. Sloan Foundation
- Thomas Murray, The Hastings Center, Inc.
- David Rejeski, The Woodrow Wilson Center
- Michele Garfinkel, J. Craig Venter Institute

Wednesday, June 30

Bioremediation
9:00 a.m. – 10:30 a.m.

- Thomas Videbaek, Novozymes
- Muhammad Asghar, University of Agriculture, Faisalabad
- Sudhir Meshram, Rajiv Gandhi Biotechnology Centre, R. T. M. Nagpur University
- James Burns, Bionavitas, Inc

The annual World Congress on Industrial Biotechnology and Bioprocessing – co-organized by BIO, BIOTECanada, and the U.S. Department of Energy – is the original and only conference dedicated solely to industrial biotechnology and the most recent advancements in the field. Visit www.bio/worldcongress.

Registration for the World Congress is now open and is complimentary for credentialed members of the news media. All programs at the World Congress on Industrial Biotechnology and Bioprocessing are open to attendance by members of the media. Reporters and editors working full-time for print, broadcast, and online news organizations may register onsite with valid media credentials. All freelancers, college and online publications are strongly encouraged to register in advance. For more information, please visit here.

Upcoming BIO Events

World Congress on Industrial Biotechnology and Bioprocessing
June 27-30, 2010
Washington, DC

BIO India
September 21 – 22, 2010
Hyderabad, India

BIO’s Livestock Biotechnology Summit
September 28-30, 2010,
Sioux Falls, SD

Source
BIO

A collaboration between the University of Leeds, Durham University and GlaxoSmithKline (GSK) is looking at ‘printing’ pills to order, to create safer and faster-acting medicines.

It should also bring new drugs to market faster, so patients can benefit more quickly from medical advances.

The research, led by Dr Nik Kapur from the University’s Faculty of Engineering, is set to revolutionise a process which has remained unchanged for over a thousand years.

GSK has developed a way of printing active pharmaceutical ingredients onto tablets – but the process can only currently be applied to just 0.5 per cent of all medicines used in tablet form. The researchers hope the new project will see this increase this to 40 per cent.

“Some active ingredients can be dissolved in a liquid, which then behaves like normal ink, so then the process is fairly straightforward,” explains Dr Kapur. “However, when you’re working with active ingredients that don’t dissolve, the particles of the drug are suspended in the liquid, which creates very different properties and challenges for use within a printing system.

“For some tablets, you may also need higher concentrations of active ingredients to create the right dose, and this will affect how the liquid behaves.”

A medicine droplet is 20 times larger than an ink droplet in a standard ink-jet system, so the challenges facing the researchers include the numbers of drops that each tablet can hold, and how to increase the level of active ingredient in each drop. The research will also look at the properties and behaviour of the suspension, the shape and size of the printing nozzle and ways to pump the suspension through the printing equipment.

Drugs produced in this way would be faster acting, as with the active ingredient on the pill’s surface, the pill would no longer need to be broken down by the digestive system before the drug can enter the bloodstream. Ultimately it would also be possible to print several drugs onto one pill, reducing the number of tablets to be swallowed by patients on multiple medicines.

Printing active ingredients onto pre-formed tablets speeds up and improves quality control, as each tablet contains exactly the correct dose. With some of the current quality assurance procedures rendered unnecessary, new drugs would reach patients much faster.

The first documented manufacture of pills goes back to Egyptian times, when active medicinal ingredients were rolled in bread or clay, but the earliest reference to a tablet – a compressed pill – is found in tenth century Arabic medical literature. The process had little changed when the first patent for tablets was applied for in 1843. First produced in small doses by pharmacists, mass production still uses the same process, but with much advanced technology and quality assurance.

Because most drugs only need very small doses, the pill or tablet acts as a carrier to make the medicine big enough to pick up and swallow. The active ingredient is usually just one thousandth of a pill, so has to be mixed with other ingredients to bulk it out to pill size. This is then split into the amount needed for each pill and compressed to create a tablet.

One of the major challenges is ensuring that each tablet contains the correct dose. This is currently done by statistically checking samples from each batch of pills post-production, but a printed system would enable quality control of each pill as it is produced. The new system would therefore both speed up production and provide a greater quality assurance and consistency of dosage than are currently possible under even the highest pharmaceutical standards.

The research is jointly funded by GSK and the Technology Strategy Board and will run for two years. Working with Dr Kapur on the project are colleagues from Leeds’ Schools of Mechanical Engineering, Maths and Chemistry and from the Durham University’s Department of Chemistry.

Source:
Abigail Chard
University of Leeds

For three September days, Lisbon, Portugal will become the capital city of pharma quality professionals. Senior level executives from Europe and World leading companies will meet to discuss the best practices on how to achieve QC laboratory excellence, how and where to deploy and develop measurable quality systems, Quality Risk Management, QbD & PAT and much more…

Dasa Laslopova, event producer, explains why this is a must-attend for Pharma QA professionals. “We prepared the agenda with a panel of industry experts. The quality of presentations guarantees the speakers panel. Novartis, Pfizer, Roche, Genzyme, UCB, Boehringer Ingelheim, Merck Sharp & Dohme, Teva, Eli Lilly, they all are present.”

One of advisor panel was Stefano Carella, Master Black Belt – EMEAC Regional Director, Supply Chain Solutions & Program Realization Office, Merck Sharp & Dohme, Italy. We asked him few questions regarding the future of Pharma QA:

What are the new challenges in the process of applying quality & lean principles, that Pharma manufacturers are facing in cGMP and ICH guidance environment?

(SC): “I can more focus on the lean aspect: 4 challenges: 1) making sure that lean is not perceived as the tool for headcount reduction, in other words avoiding the wrong acronym LEAN = Less Employees Are Needed. If a good lean project deployment plan is NOT built and drilled down in the organization properly people will not understand and won’t be onboard. 2) Companies are embracing lean not because it is the latest fashion but mostly because it is the only way to become more efficient and effective in operations. Top management normally thinks that it will be a short journey not understanding that it is a real journey that has the real objective to change the culture from not perceived waste to continuous improvement. It will take many years, so the second challenge is this misunderstanding about the duration of the project. 3) Other 2 important challenges are making sure that lean is deployed by real experts, not people who red a book, and the consequence of this is the 4) lack of sustainability, last typical problem, because of the wrong perception that lean is a set of tools rather then a behavioral change (TPS Senseis(=senior trainers) use to say that lean is 85 % behaviors and 15% tools. Perfect link with Quality is the result of bad lean implementation: quality is jeopardized.”

Where do you see the significant opportunities for improvements to achieve compliance based process excellence?

(SC): “First step is involvement: Involving quality at the very beginning of any continuous improvement journey, not as extended team members but as core team members. Second step is changing the idea of the role: In Toyota the role of quality is improving efficiency! A great cultural change. 3) Involve shopfloor people in kaizen / continuous improvement activities together with quality: quality will provide clarity around WHAT are the regulatory / GMP requirements and operations will develop HOW to perform the actions.”

How do you see the future of Quality Organization?

(SC): “More and more at the shopfloor level, working together with operations for the common good, for instance at the end of a production line to release on time in the optics of one piece flow, this is for sure the end of the journey, or “True North” in the Toyota way. This is very far from where we are now, so I think the nearer future is to move quality to go-see operations, to involve them in continuous improvement activities (Kaizen) to make sure that they support solution development to shopfloor problems and that understand complexity and try to solve it. So moving from Quality perceived as Police to Quality helping in problem solving and solutions development.”

Event information: 6th Annual Achieving Quality & Process Excellence in Pharma Industry will be held between 15th – 17th September 2010 in Lisbon, Portugal. Read more

Source:
Peter Novak
eventsjacobfleming

Shire plc (LSE: SHP, Nasdaq: SHPGY), the global specialty biopharmaceutical company, announced the results of a study evaluating the safety and efficacy of Vyvanse® (lisdexamfetamine dimesylate) Capsules CII for the treatment of Attention Deficit Hyperactivity Disorder (ADHD) in adolescents aged 13 to 17 years. The data, presented in an oral session at a national scientific meeting of psychiatrists in New Orleans, LA, demonstrated that treatment with Vyvanse (30, 50, and 70 mg/d) significantly improved ADHD symptoms compared to placebo in adolescents with ADHD.

Vyvanse is currently indicated for the treatment of ADHD in children aged 6 to 12 years and in adults. Recently, Shire submitted a supplemental New Drug Application (sNDA) for use of Vyvanse in the treatment of adolescents aged 13 to 17 years with ADHD.

ADHD is one of the most common psychiatric disorders in children and adolescents. Sixty to 85 percent of children with ADHD may continue to meet criteria for the disorder during their teenage years. In the United States, it is estimated that approximately 9.7 percent of adolescents aged 13 to 17 years have been diagnosed with ADHD at some point in their lives based on results from the 2003 National Survey of Children’s Health (NSCH), a telephone survey of households with at least one child aged 17 years or younger.(+)

“In this study, Vyvanse improved ADHD symptoms of inattention, hyperactivity and impulsivity in adolescents compared to placebo,” said Ann C. Childress, MD, president of the Center for Psychiatry and Behavioral Medicine, Inc. in Las Vegas, Nevada and lead investigator for this study. “These results are important as we look for additional ways to effectively manage ADHD symptoms in adolescent patients.”

As with other stimulant medications, Vyvanse is classified as a controlled substance (CII) because of its potential for abuse.

About the Study

In this double-blind, placebo-controlled, four-week, forced-dose phase 3 trial, 309 adolescents were randomized into either one of three Vyvanse dosage groups, 30, 50 and 70 mg/day, or into a placebo group. The 232 adolescents who received Vyvanse, regardless of their dosage group, had significant improvements in their symptoms, compared to the 77 adolescents receiving placebo, as measured by the average changes in the participants’ scores on the clinician administered ADHD Rating Scale IV (ADHD-RS-IV) from baseline to end point, the study’s primary end point. The ADHD-RS-IV, which contains 18 items, is based on the ADHD diagnostic criteria as defined in the Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition, Text Revision®, a publication of the American Psychiatric Association.

Least squares (LS) mean (standard error) change from baseline at end point in ADHD-RS-IV total scores (primary efficacy result) were -18.3 (1.25), -21.1 (1.28), -20.7 (1.25) for 30, 50, and 70 mg/d LDX, respectively; -12.8 (1.25) for placebo (P less than or equal to .0056 vs placebo for each) with negative differences in LS mean values indicating improvement in ADHD symptoms.

The safety of Vyvanse also was evaluated during the study. The most frequently occurring treatment-emergent adverse events (greater than or equal to 5 percent) in all of the groups treated with Vyvanse were decreased appetite, headache, insomnia, weight decrease and irritability. Safety results were generally consistent with the known safety profile associated with Vyvanse treatment.

Shire Development Inc. supported this study. Dr Ann Childress is a consultant and speaker for and has received grant/research support from Shire.

About Vyvanse

Vyvanse, which was introduced in the United States in July 2007 for the treatment of ADHD in children aged 6 to 12 years and approved in April 2008 to treat ADHD in adults, is currently available in six once-daily dosage strengths of 20 mg, 30 mg, 40 mg, 50 mg, 60 mg and 70 mg. To date, more than 9 million Vyvanse prescriptions have been filled.

Vyvanse is a therapeutically inactive prodrug stimulant, in which d-amphetamine is covalently bonded to l-lysine, and after oral ingestion it is converted to pharmacologically active d-amphetamine.

INDICATION AND IMPORTANT SAFETY INFORMATION

Vyvanse is indicated for the treatment of ADHD. Efficacy based on two controlled trials in children aged 6 to 12 and two controlled trials in adults. Vyvanse is indicated as an integral part of a comprehensive treatment program that may include other measures (psychological, educational, social).

Amphetamines have a high potential for abuse; prolonged administration may lead to dependence. Misuse of amphetamine may cause sudden death and serious cardiovascular adverse events. See Full Prescribing Information for complete Boxed WARNING.

Vyvanse should not be taken by patients who have advanced arteriosclerosis; symptomatic cardiovascular disease; moderate to severe hypertension; hyperthyroidism; known hypersensitivity or idiosyncrasy to sympathomimetic amines; agitated states; glaucoma; a history of drug abuse; or during or within 14 days after treatment with monoamine oxidase inhibitors (MAOIs).

Sudden death has been reported in association with CNS stimulant treatment at usual doses in children and adolescents with structural cardiac abnormalities or other serious heart problems. Sudden death, stroke, and myocardial infarction have been reported in adults taking stimulant drugs at usual doses in ADHD. Physicians should take a careful patient history, including family history, and physical exam, to assess the presence of cardiac disease. Patients who report symptoms of cardiac disease such as exertional chest pain and unexplained syncope should be promptly evaluated. Use with caution in patients whose underlying medical condition might be affected by increases in blood pressure or heart rate.

New psychosis, mania, aggression, growth suppression, visual disturbances and exacerbation of tics and Tourette’s syndrome have been associated with the use of stimulants. Use with caution in patients with a history of psychosis, seizures or EEG abnormalities, bipolar disorder, or depression. Growth should be monitored in children during treatment with stimulants, and patients who are not growing (gaining height or weight) as expected may need to have their treatment interrupted.

The most common adverse reactions (greater than or equal to 5 percent and at least twice the rate of placebo) reported in the pivotal clinical trials were pediatric decreased appetite, insomnia, upper abdominal pain, irritability, decreased weight, vomiting, nausea, dizziness and dry mouth; adult decreased appetite, insomnia, dry mouth, nausea, diarrhea, anxiety and anorexia.

About ADHD

ADHD is one of the most common psychiatric disorders in children and adolescents. Worldwide prevalence of ADHD is estimated at 5.3 percent (with large variability), according to a comprehensive systematic review of this topic published in 2007 in the American Journal of Psychiatry. In the United States, approximately 7.8 percent of all school-aged children, or about 4.4 million children aged 4 to 17 years, have been diagnosed with ADHD at some point in their lives, according to the Centers for Disease Control and Prevention (CDC). The disorder is also estimated to affect 4.4 percent of US adults aged 18 to 44 based on results from the National Comorbidity Survey Replication. When this percentage is extrapolated to the full US population aged 18 and over, almost 10 million adults are believed to have ADHD.

ADHD is a psychiatric behavioral disorder that manifests as a persistent pattern of inattention and/or hyperactivity-impulsivity that is more frequent and severe than is typically observed in individuals at a comparable level of development. The specific etiology of ADHD is unknown and there is no single diagnostic test for this disorder. Adequate diagnosis requires the use of medical and special psychological, educational and social resources, utilizing diagnostic criteria such as Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition, Text Revision (DSM-IV-TR®) or International Classification of Diseases 10 (ICD-10).

Although there is no cure for ADHD, there are accepted treatments that specifically target its symptoms. Standard treatments include educational approaches, psychological or behavioral modification, and/or medication.

SHIRE PLC

Shire’s strategic goal is to become the leading specialty biopharmaceutical company that focuses on meeting the needs of the specialist physician. Shire focuses its business on attention deficit hyperactivity disorder (ADHD), human genetic therapies (HGT) and gastrointestinal (GI) diseases as well as opportunities in other therapeutic areas to the extent they arise through acquisitions. Shire’s in-licensing, merger and acquisition efforts are focused on products in specialist markets with strong intellectual property protection and global rights. Shire believes that a carefully selected and balanced portfolio of products with strategically aligned and relatively small-scale sales forces will deliver strong results.

“SAFE HARBOR” STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM ACT OF 1995

Statements included herein that are not historical facts, including statements preceded by, followed by, or that include the words “expects”, “estimates” or similar statements, are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, the Company’s results could be materially adversely affected. The risks and uncertainties include, but are not limited to, risks associated with: the inherent uncertainty of research, development, approval, reimbursement, manufacturing and commercialization of the Company’s Specialty Pharmaceutical and Human Genetic Therapies products (including, without limitation, velaglucerase alfa), as well as the ability to secure and integrate new products for commercialization and/or development; government regulation of the Company’s products; the Company’s ability to manufacture its products in sufficient quantities to meet demand; the impact of competitive therapies on the Company’s products; the Company’s ability to register, maintain and enforce patents and other intellectual property rights relating to its products; the Company’s ability to obtain and maintain government and other third-party reimbursement for its products; and other risks and uncertainties detailed from time to time in the Company’s filings with the Securities and Exchange Commission.

(+) For this survey, parents or guardians responded to survey items on behalf of 102,353 sample children (completion rate: 68.8 percent). NSCH data were weighted to estimate national rates of ADHD diagnosis. As a proxy for ADHD diagnosis, respondents were asked, “Has a doctor or health professional ever told you that [child] has ADD or ADHD?”

Source: Shire plc

View drug information on Vyvanse.

CoDa Therapeutics, Inc., a biopharmaceutical company focused on the development and commercialization of therapeutics for wound care and tissue repair, announced positive results from its Phase 2 NOVEL Study of NEXAGON® in patients with chronic venous leg ulcers. NEXAGON® is a topically applied, novel therapeutic candidate, with the potential to revolutionize the wound healing treatment paradigm by leveraging a new mechanism and target involved in the healing process.

NEXAGON® achieved the endpoints of safety, reduction in wound size and complete healing after four weeks in the randomized, vehicle-controlled, double-blind, Phase 2 NOVEL study. Based on these compelling results, CoDa plans to initiate additional NEXAGON® studies and has scheduled a near-term end-of-Phase 2 meeting with the FDA to discuss potential registration studies to support marketing approval.

The three-arm trial randomized 98 patients at multiple sites to receive low or high dose NEXAGON® treatment or vehicle, in addition to compression bandaging (standard-of-care). After only three applications over a four-week treatment period, high-dose NEXAGON® demonstrated a 69% reduction in the size of venous leg ulcers. In addition, complete healing of 31% of wounds seen in the high-dose treatment arm was five times higher than complete healing in the vehicle arm. Importantly, no drug-related adverse events were observed in either of the low or high dose NEXAGON® arms, confirming favorable safety results from previous preclinical and Phase 1 clinical studies.

Dr. Thomas Serena, a NOVEL Study Investigator, and Founder and Medical Director of the Penn North Centers for Advanced Wound Care, said, “The data from CoDa’s Phase 2 NEXAGON® trial are very impressive therapeutically, especially considering the accelerated rate of complete wound healing and excellent safety profile after only four weeks of treatment. By targeting the inhibition of Connexin43, which may be a ‘master switch’ in wound healing, NEXAGON® is designed to improve both the rate and quality of tissue repair while managing inflammation. If patients can experience better and faster treatment outcomes, NEXAGON® has the potential to address significant treatment limitations and economic burdens imposed by chronic wounds on patients and insurers.”

In the U.S., venous leg ulcers account for the loss of 2 million working days and nearly $3 billion in treatment costs each year. Duration of treatment may last over a year in certain cases, and frequently involves the use of significant healthcare resources, resulting in substantial costs for the U.S. healthcare system.

Dr. David Eisenbud, a vascular surgeon with expertise in wound evaluation and treatment, and former President of the American Academy of Wound Management, said, “The results from the Phase 2 NOVEL Study far exceed expected healing outcomes using today’s standard of care. As a physician focused on wound healing, my own venous ulcer patients would benefit tremendously from a treatment that closes their wounds by an average of nearly 70% in just four weeks. That is simply not achievable right now, and the NEXAGON® treatment results showing a 31% incidence of complete healing at four weeks are remarkable. The healing process tends to be much slower, and other chronic wound healing studies usually use a 12-16 week treatment period. Given the rapid and significant wound healing and safety results seen in the Phase 2 study, additional studies of NEXAGON® are highly warranted in patients with venous leg ulcers.”

In addition to Dr. Eisenbud, market research and interviews with practicing clinicians reveal that a product with the expected healing profile of NEXAGON® for chronic wounds would be seen as a major improvement over current standard of care treatments.

Bradford Duft, President and CEO of CoDa said, “After several years of intense work by a small and dedicated team, the results from this Phase 2 study represent a important milestone for CoDa. We are confident that the therapeutic potential of NEXAGON® will support our ongoing Series B financing and corporate partnering discussions. Chronic wounds represent one of the most significant unmet medical needs in the world today. The Phase 2 data support our conviction that CoDa’s Gap Junction Modulation technology is at the forefront of a potential paradigm shift in how we treat patients with venous leg ulcers and other chronic wounds. With our clinical development program mapped out, we are poised to contribute a major improvement in the quality and rate of wound healing for these patients as well as the U.S. healthcare system. We look forward to our upcoming end-of-Phase 2 meeting with the FDA, after which we will share the details of our future development plans.”

About the Phase 2 NOVEL Study

The NOVEL Study was a randomized, vehicle-controlled, double-blind Phase 2 clinical study to evaluate two doses of NEXAGON® in patients with venous leg ulcers. 98 patients (89 evaluable) were enrolled at sites in New Zealand and the United States, and randomized on a 1:1:1 basis to receive low or high dose NEXAGON® treatment or vehicle, in addition to compression bandaging (standard-of-care). Patients in all three treatment arms received three applications over a four-week treatment period. Study endpoints were reduction in wound size and complete healing after four weeks, as well as safety. Patients were evaluated at the end of treatment and are being followed for up to 12 weeks to further evaluate ulcer healing.

About Venous Leg Ulcers

For more than half a million patients in the U.S. suffering from venous leg ulcers each year, the wound healing process is often time-consuming and costly, and may gravely impact quality of life. Of all ulcer types, venous leg ulcers are the most common, resulting in the loss of 2 million working days and nearly $3 billion in treatment costs per year in the US. The substantial costs associated with venous leg ulcers are related to the prevalence of the indication as well as the lengthy duration of treatment. The healing process for venous leg ulcers may take over a year in some cases and frequently requires significant healthcare resources during the healing process. Although venous leg ulcers place a significant burden on patients and the U.S. healthcare system, existing treatment options are unable to fully address this unmet need.

About CoDa Therapeutics Inc

CoDa Therapeutics is a clinical stage biotechnology company focused on developing novel targeted therapies that address major unmet medical needs in inflammation, wound-healing and tissue repair. The company is pioneering a new field of science: gap junction modulation, using a new class of therapeutics that can modulate wound responses and reduce inflammation. CoDa has two open INDs and has completed one Phase 2 and two Phase 1 clinical trials in skin and eye, where NEXAGON® was shown to be safe and tolerable following administration to over 350 wounds on more than 125 subjects. CoDa’s technology, which can be conveniently applied topically, been shown to work across a wide variety of wound and inflammatory settings and conditions. CoDa presently has issued patents in the US, Europe and elsewhere, and pending applications in more than a dozen other patent families directed to methods and compositions for the treatment of acute wounds, chronic wounds, scarring, abnormal scarring, inflammation and pain, fibrosis, surgical adhesions, and orthopedic procedures, as well as combination therapies and improved medical devices.

About NEXAGON®

The active ingredient in NEXAGON®, which has been shown to work across a wide variety of tissues, is CODA001, a natural, unmodified antisense oligonucleotide that down-regulates the key gap junction protein Connexin43 to dampen inflammatory responses and enhance healing. Data shows that for optimal healing Connexin43 is normally dialed-down at the edges of acute wounds (i.e., wounds that will heal normally). Conversely, other data demonstrate that Connexin43 is wrongly up-regulated at the edge of chronic wounds (i.e., wounds that are difficult to heal such as venous and diabetic ulcers). CoDa believes that one can better target available medical options and design more effective wound-healing alternatives by devising a therapeutic approach based on biological mechanisms naturally at work or conversely, at fault, in a given situation. The answer is thought to lie in Connexin43, which can be seen as a “master switch” in wound healing that is temporarily turned “off” for superior healing of acute wounds, and when left “on” can lead to the unwanted inflammation and/or stalled healing characteristic of chronic wounds.

Source: CoDa Therapeutics, Inc

As many as one in four people in westernized countries experience pain or discomfort in their upper abdomen, and physicians have almost nothing to offer except anti-acid medicines, which usually don’t work. Now, in a small but novel study, researchers have found evidence that an abnormal amount of inflammatory cells populates the upper intestine of affected individuals, which suggests a fresh way of understanding the common complaint.

The study, published in the September issue of Clinical Gastroenterology and Hepatology and conducted by researchers in the U.S., Sweden, England, and Australia, may also point to innovative methods to treat the condition and eliminate discomfort.

“Newly-designed, targeted anti-inflammatory medicine aimed at blocking the function of these cells might be very useful, if our results are validated,” says the study’s lead researcher, Nicholas J. Talley, M.D., Chair of Internal Medicine at Mayo Clinic in Jacksonville.

“We are quite intrigued by what we have discovered, because it probably represents a new disease entity, one that might be capable of diagnosis and management,” Dr. Talley says.

The scientists don’t know why inflammatory cells are present in one particular region of the small intestine, the duodenum that connects to the stomach, but they theorize that it could result from an allergic reaction to certain foods. Patients examined did not have infections, celiac disease (an autoimmune reaction to gluten protein), or cancer.

“I believe food intolerance can lead to motor and sensory abnormalities that are perceived as pain and discomfort,” Dr. Talley says. “But we have no evidence yet that this is definitely the case.”

To conduct the study, researchers in Sweden offered endoscopic examinations to 51 Swedish participants who complained of “nonulcer dyspepsia” as well as 49 randomly selected participants who had no pain. Dyspepsia is chronic or recurrent pain, or a feeling of abdominal fullness after eating or nausea, and the nonulcer form means there is not any structural abnormality such as an ulcer. For reasons that are not clear, sensitivity to stomach acid occurs in some of these patients, but acid suppression therapy does not work in two-thirds of patients who try it. There are really very few effective therapies, Dr. Talley says.

During the endoscopy procedure, physicians removed biopsy tissue from several places in the small intestine of participants, and the samples were examined by pathologists who did not know who the samples belonged to.

The researchers found significantly more eosinophil cells in people with nonulcer dyspepsia, compared to the control group population, but these cells were found only in the duodenum, the place in the intestine where most chemical digestion takes place. Eosinophils are white blood cells, part of the immune system, which fight parasites.

The researchers cannot yet say whether duodenal esoinophilia is the cause of the pain or an effect of another factor causing the disorder, although Dr. Talley says “a casual link remains our hypothesis.

“The presence of these cells has been overlooked because no one has used rigorous quantification methods before, and because biopsy examinations of the duodenum are not routinely performed,” he says. “Now we have a new direction to go in.”

###

The study was funded in part by Swedish grants, the University of Sydney, and Astra Zeneca Inc.

Source: Kevin Punsky

Mayo Clinic