FDA

Merck Issues Update on Phase III Trial for Preladenant

Paul Quintaro — Thu, 23 May 2013 21:00:42 +0000

Merck (NYSE: MRK), known as MSD outside the United States and Canada, today provided an update on the clinical program for preladenant, Merck's investigational adenosine A2[A ]receptor antagonist for the treatment of Parkinson's disease (PD). An initial review of data from three separate Phase III trials did not provide evidence of efficacy for preladenant compared with placebo.

Based on these results, Merck is taking steps to discontinue the extension phases of these studies and no longer plans to pursue regulatory filings for preladenant. The decision to discontinue ...

Celgene Announces FDA Grants Priority Review for ABRAXANE sNDA in Advanced Pancreatic Cancer

Paul Quintaro — Thu, 23 May 2013 11:34:08 +0000

Celgene International Sàrl, a subsidiary of Celgene Corporation (Celgene) (NASDAQ: CELG) today announced that the U.S. Food and Drug Administration (FDA) has assigned a Priority Review designation to the supplemental New Drug Application (sNDA) for the use of ABRAXANE^® (paclitaxel protein-bound particles for injectable suspension) (albumin-bound) in combination with gemcitabine for the first–line treatment of patients with advanced pancreatic cancer.

The FDA grants Priority Review to medicines that, if approved, have the potential to offer significant improvement compared to ...

Omeros Announces INDA for OMS824 in Huntington's Disease Cleared by FDA

Paul Quintaro — Thu, 23 May 2013 11:23:07 +0000

Omeros Corporation (NASDAQ: OMER) today announced that its Investigational New Drug Application (IND) to evaluate OMS824 in Huntington's disease has been cleared by the U.S. Food and Drug Administration (FDA). OMS824 selectively inhibits phosphodiesterase 10 (PDE10), an enzyme expressed in areas of the brain linked to a wide range of diseases that affect cognition, including Huntington's disease and schizophrenia. OMS824 has shown promising results in animal models directly relevant to Huntington's disease and, as previously announced, OMS824 was well tolerated and exhibited favorable pharmacokinetic properties in a Phase 1 clinical program. Omeros plans to advance OMS824 into Phase 2 clinical trials for Huntington's disease next quarter and for schizophrenia later ...

Boston Scientific Announces Results from Promus Premier, Synergy Drug-Eluting Stent

Paul Quintaro — Wed, 22 May 2013 13:18:35 +0000

Boston Scientific Corporation (NYSE: BSX) reports positive results from two trials evaluating new, innovative drug-eluting stent (DES) technologies, which are emerging treatment options for coronary heart disease. Data from the first human use NG PROMUS Clinical Trial evaluating the safety and effectiveness of the Promus PREMIER™ Everolimus-Eluting Platinum Chromium Coronary Stent System, and two-year follow-up data from the EVOLVE Trial comparing the safety and effectiveness of the SYNERGY Everolimus-Eluting Bioabsorbable Polymer-Coated Platinum Chromium Coronary Stent System to the PROMUS Element™ Stent System were presented today at the annual EuroPCR Scientific Program in Paris.

The NG PROMUS Clinical Trial evaluated the clinical and angiographic outcomes for the Promus PREMIER Stent System at 30 days. John Ormiston, M.D., of Mercy Angiography, Auckland City, New Zealand is the primary investigator for the trial and presented data at the conference.

"The Promus PREMIER Stent demonstrated excellent safety and effectiveness with zero percent target lesion revascularization and stent thrombosis," said Dr. Ormiston. "In addition, the rate of technical success, the primary endpoint of the trial, was very high at 99.2 percent. The Promus PREMIER Stent System truly is a major step forward in stent technology."

The Promus PREMIER Stent System features the only customized stent architecture of its ...

Covidien Says Trial Data for Endoscopic Ablation Therapy Shows Elimination of Precancerous Esophageal Tissue, Significantly Cut Disease Progression

Paul Quintaro — Wed, 22 May 2013 11:29:54 +0000

Covidien (NYSE: COV), a leading global provider of healthcare products, today announced that results from a prospective, multicenter, randomized, controlled clinical trial show that endoscopic ablation therapy using the Barrx™ RF Ablation System is effective at eliminating Barrett's esophagus, a pre-cancerous condition of the esophagus, and preventing disease progression.

The SURF Trial* (SUrveillance vs. RadioFrequency ablation) included 136 patients with Barrett's esophagus containing confirmed low-grade dysplasia. Upon enrollment, patients were randomly assigned to receive either endoscopic ablation therapy and ...

Salix Pharma Says Results for Budesonide Foam Statistically Significant

Paul Quintaro — Wed, 22 May 2013 11:26:29 +0000

Salix Pharmaceuticals, Ltd. (NASDAQ: SLXP) today announced the successful completion and outcome of two pivotal Phase 3 studies to evaluate the efficacy and safety of budesonide foam in the treatment of active mild to moderate ulcerative proctitis (UP) or ulcerative proctosigmoiditis (UPS). In each of the two pivotal Phase 3 studies, a statistically significant proportion of subjects treated with budesonide foam achieved clinical remission compared to subjects treated with placebo foam.

“We are pleased with the outcome of our pivotal studies of budesonide foam in the treatment of UP and UPS,” stated Bill Forbes, Executive Vice President, Medical, Research and Development and Chief Development Officer, Salix. “Patients with UP and UPS can be challenging to treat. Oral treatments may be limited by their ability to deliver drug to the rectum and sigmoid colon, and enema treatments may be limited by patients' ability to retain the liquid. Knowing this, our partner, Dr. Falk Pharma GmbH, developed budesonide foam which is approved and sold in Europe for the treatment of UP and UPS. We believe foam formulations provide important advantages over enema formulations principally with respect to drug retention and patient acceptance. We are working to submit a New Drug Application for budesonide foam as a treatment for UP and UPS by the end of September 2013. If approved, budesonide foam would be the first foam product approved in the United States for patients with UP (ulcerative colitis up to 10 cm from the rectum) as well as for patients suffering from UPS (ulcerative colitis up to 40 cm from the rectum). We believe this combination of ...

Results from Phase III Studies of Naloxegol for Treatment of Opioid-Induced Constipation Presented at Digestive Disease Week 2013

Eddie Staley — Tue, 21 May 2013 13:26:00 +0000

AstraZeneca (NYSE: AZN) today presented the results of two pivotal Phase III studies of naloxegol showing the 25 mg dose of the investigational drug met its primary and secondary endpoints for efficacy and showed a safety profile consistent with previous studies. Data was presented at the Digestive Disease Week (DDW) meeting in Orlando, Florida. Naloxegol is a peripherally-acting mu-opioid receptor antagonist, which has been specifically designed for the treatment of opioid-induced constipation (OIC), a common and often debilitating side effect of prescription opioid pain medicines.

The Phase III studies, KODIAC-04 and -05, were 12-week, multicenter, randomized, double blind, placebo-controlled pivotal trials that evaluated 12.5 mg and 25 mg doses of naloxegol, administered once-daily. The primary endpoint in both trials ...

CytRx Offers Initial Results from Trial with Aldoxorubicin in Solid Tumors

Paul Quintaro — Tue, 21 May 2013 12:34:49 +0000

CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, announced initial data from a Phase 1b clinical trial evaluating the pharmacokinetics and safety of aldoxorubicin, its novel conjugate of the widely used chemotherapeutic agent doxorubicin, in patients with metastatic solid tumors who have either relapsed or not responded to treatment with standard therapies. The data indicate that aldoxorubicin has a distribution half-life of 20-24 hours following infusion, which is significantly longer than doxorubicin's initial half-life of five minutes. The data also demonstrate that aldoxorubicin demonstrated a distribution ...

New data reinforces strength of Novartis once-daily COPD portfolio in improving lung function, shortness of breath and reducing rate of exacerbations

Eddie Staley — Tue, 21 May 2013 12:22:52 +0000

New data from the Novartis chronic obstructive pulmonary disease (COPD) portfolio were presented today at the American Thoracic Society (ATS) International Conference May 17-22, 2013 in Philadelphia, PA, USA. In total, Novartis presented 34 respiratory abstracts, featuring latest findings from the IGNITE clinical trial program including BLAZE[1] and SPARK[2],[3],[6],[7] studies, plus data from pooled GLOW1 and 2 studies[4],[8],[9],[10].

The late-breaking BLAZE study included patient self-reported data that demonstrated improvements in shortness of breath with investigational once-daily QVA149 (indacaterol maleate 110 mcg / glycopyrronium 50 mcg) when compared to placebo and blinded tiotropium 18 mcg[1]. The SPARK study showed that QVA149 significantly reduced the rate of all COPD exacerbations versus glycopyrronium 50 mcg and open-label (OL) tiotropium 18 mcg[2],[3].

"The expanding Novartis COPD portfolio brings us another step closer to meeting the unmet needs of millions of patients worldwide," said Tim Wright, Head of Development, Novartis Pharmaceuticals. "These important results for both QVA149 and Seebri^® Breezhaler^® add further weight to our COPD portfolio, providing the right treatment for the right patient at the right time."

COPD affects an estimated 210 million people worldwide[11] and is projected to be the third leading cause of death by 2020[5]. New treatments which effectively manage COPD are very important to patients and physicians, as COPD can impose a significant burden on patients and reduce quality of life[12],[13].

The BLAZE study showed that after six weeks of treatment, QVA149 significantly improved patient self-reported shortness of breath during daily activities versus both placebo (p<0.001) and tiotropium 18 mcg (p=0.021)[1]. BLAZE was the first study to evaluate direct electronic patient self-reported shortness of breath and showed that QVA149 significantly improved lung function versus placebo and tiotropium 18 mcg (as demonstrated by mean FEV[1])at all time points (45 minutes pre-dose to four hours post-dose) after six weeks of treatment (p<0.001)[1].

The SPARK study, recently published in Lancet Respiratory Medicine[14], demonstrated that QVA149 significantly reduced the rate of all COPD exacerbations (mild, moderate and severe) by 15% versus glycopyrronium 50 mcg (p=0.0012) and 14% versus OL tiotropium 18 mcg (p=0.0017)[2],[3]. The primary endpoint of the study was also met since QVA149 demonstrated a significantly reduced rate of moderate or severe COPD exacerbations by 12% versus glycopyrronium (p=0.038)[2],[3]. The rate of moderate or severe exacerbations was numerically lower (p=0.096) in patients on QVA149 compared to OL tiotropium 18 mcg[2],[3]. SPARK also showed that patients receiving QVA149 had substantially improved lung function (measured by trough FEV[1])compared to glycopyrronium 50 mcg and OL tiotropium 18 mcg (both p<0.0001)[2],[6]. In addition, QVA149 showed significant differences in health-related quality of life as demonstrated by St George's Respiratory Questionnaire (SGRQ) total scores of QVA149 versus glycopyrronium 50 mcg (p<0.01) and OL tiotropium 18 mcg (p<0.05)[2],[6].

All treatments in the BLAZE and SPARK studies had an acceptable safety profile with no meaningful differences between the treatment groups in the incidence of adverse or serious adverse events[1],[2],[7].

In a pooled analysis of GLOW1 and GLOW2 data, once-daily glycopyrronium 50 mcg (Seebri^® Breezhaler^®) demonstrated significant improvements in lung function during first 4 hours following morning administration (measured by FEV[1 ]AUC[0-4h]) versus placebo and OL tiotropium 18 mcg, at Day 1, 12 weeks and 26 weeks[4]. Once-daily glycopyrronium 50 mcg also demonstrated sustained improvements in ...

UPDATE: Hologic Receives FDA Approval for a New Low-dose 3D Mammography (Breast Tomosynthesis) Solution for Breast Cancer Screening

Eddie Staley — Tue, 21 May 2013 12:19:25 +0000

Hologic (NASDAQ: HOLX), a leading developer, manufacturer and supplier of premium diagnostics, medical imaging systems and surgical products, with an emphasis on serving the healthcare needs of women, today announced that the U.S. Food and Drug Administration (FDA) approved the use of Hologic's new C-View 2D imaging software. C-View 2D images may now be used in place of the conventional 2D exposure previously required as part of a Hologic 3D mammography (breast tomosynthesis) screening exam.

To view the multimedia assets associated with this release, please click: http://www.multivu.com/mnr/60258-hologic-receives-fda-approval-c-view-software-3d-mammography-solution

C-View images are generated from the 3D tomosynthesis data acquired during the mammography exam, eliminating the need for additional 2D exposures. The combination of Hologic's 3D and C-View 2D images results in less time under compression, for greater patient comfort and a lower radiation dose, while still providing the 2D images required as part of Hologic's FDA approved 3D mammography screening exam. Clinical studies have shown that screening with Hologic's 3D mammography technology using C-View imaging results in clinical performance superior to that of a conventional 2D mammogram.

"Approval of our C-View software is an important evolution in Hologic's 3D mammography screening ...

J&J's Janssen Announces Primary Efficacy, Safety Findings from Phase 3 Study of Simeprevir Once Daily

Paul Quintaro — Tue, 21 May 2013 12:18:29 +0000

Janssen R&D Ireland (Janssen) today announced primary efficacy and safety results from the global Phase 3 PROMISE study demonstrating that use of the investigational protease inhibitor simeprevir (TMC435) led to sustained virologic response 12 weeks after the end of treatment (SVR12) in 79 percent of treatment-experienced genotype 1 chronic hepatitis C adult patients with compensated liver disease, including all stages of liver fibrosis, when administered once daily with pegylated interferon and ribavirin.

In the study, 37 percent of patients receiving placebo plus pegylated interferon and ribavirin achieved SVR12. In the simeprevir arm, on-treatment failure rates were 3 percent and relapse rates were 19 percent, compared to 27 percent and 48 percent in the placebo arm. All patients had previously relapsed following pegylated interferon-based therapy. The data were presented today as a late breaker oral presentation at Digestive Disease Week 2013 in Orlando, Florida based on abstract number 869b, "Simeprevir (TMC435) With Peginterferon/Ribavirin for Treatment of Chronic HCV Genotype 1 Infection in Patients Who Relapsed After Previous Interferon-Based Therapy: Results From PROMISE, a Phase III Trial."

"Hepatitis C is a complex disease and physicians need multiple treatment options to provide their patients the best possible chance at successful therapy," said Eric Lawitz, ...

Abbott Announces CE Mark for World's Longest Coronary Drug Eluting Stent

Eddie Staley — Tue, 21 May 2013 12:03:50 +0000

Abbott (NYSE: ABT) today announced CE Mark in Europe for the XIENCE Xpedition™ 48 Everolimus Eluting Coronary Stent System, the first-of-its-kind treatment for very long blockages in the vessels that supply blood to the heart due to coronary artery disease (CAD). XIENCE Xpedition 48 leverages the proven design and clinical outcomes of the XIENCE family of drug eluting stents in a unique 48 mm length. Abbott continues to offer physicians more options for the treatment of patients with complex coronary artery disease and is the only major manufacturer to offer a coronary drug eluting stent greater than 38 mm in length. XIENCE Xpedition 48 is the latest in a long history of stent innovations pioneered by Abbott, which was the first company to offer physicians size-specific metallic stents for use in small and large vessels of the heart.

Studies indicate that physicians choose to use multiple shorter length stents to meet the challenges of treating long blockages – or lesions – in as many as 30 percent of interventional heart procedures, as conventional stent sizes may not always fully cover the lesion.[1] It has been shown, however, that the use of a single stent instead of multiple stents may result in several procedural benefits, including the use of fewer devices, less exposure to X-ray during the procedure, and reduced procedure time, which could lead to economic benefits.[2]

In patients with coronary artery disease, lesions are caused by the buildup of fat and cholesterol inside blood vessels; long lesions are most often seen in patients with diabetes, a significant and growing portion of the population due to changing diet and lifestyle habits.[3] When the blood vessels feeding the heart are blocked or partially blocked due to CAD, patients can experience symptoms such as chest pain ...

Peregrine Pharmaceuticals Reaches Agreement With FDA on a Phase III Trial Design for Bavituximab in Second-Line Non-Small Cell Lung Cancer

Eddie Staley — Mon, 20 May 2013 12:48:43 +0000

Peregrine Pharmaceuticals (NASDAQ: PPHM), a biopharmaceutical company developing first-in-class monoclonal antibodies focused on the treatment and diagnosis of cancer, today announced that it has reached agreement with the U.S. Food and Drug Administration (FDA) on a Phase III registration trial design of the company's lead clinical immunotherapeutic candidate bavituximab in second-line non-small cell lung cancer (NSCLC). The trial design was supported by promising data from a Phase IIb trial in patients treated with bavituximab plus docetaxel. Final data from the study will be presented at the upcoming ASCO Annual Meeting on Saturday, June 1, 2013.

"We are very pleased with the outcome from this highly collaborative effort with the FDA which allows us to proceed with our proposed Phase III clinical trial," said Robert Garnick, Ph.D, head of regulatory affairs at Peregrine. "We believe this trial, when combined with bavituximab's supporting data to date, could be sufficient to support a future BLA submission."

The Phase III clinical trial will be a randomized, double-blind, placebo-controlled trial evaluating bavituximab plus docetaxel versus docetaxel alone enrolling approximately 600 patients at sites worldwide. The trial will enroll Stage IIIB/IV non-squamous, NSCLC patients who have progressed after standard front-line treatment. The primary endpoint of the trial will be overall survival (OS).

"The promising survival and safety data from the Phase IIb clinical trial in second-line NSCLC combined with the safety profile from over 400 patients treated to date with bavituximab provide strong support for this Phase III clinical trial," said Joseph Shan, vice president of clinical and regulatory affairs of Peregrine. "We look forward to finalizing the clinical protocol and initiating the global Phase III trial by year-end."

Bavituximab is a novel investigational immunotherapy that activates the maturation of dendritic cells and cancer-fighting (M1) macrophages leading ...

Given Imaging Announces New Data Confirming SmartPill(R) Improves Diagnosis and Clinical Management of Patients With Motility Issues

Paul Quintaro — Mon, 20 May 2013 12:14:42 +0000

Given Imaging Ltd, (Nasdaq: GIVN), a world leader in GI medical devices and pioneer of capsule endoscopy, today announced several studies confirming an expanded role for SmartPill in diagnosing gastrointestinal motility disorders. All data were presented at Digestive Disease Week^® (DDW) taking place May 18 - 21, 2013 at the Orange County Convention Center, Orlando, FL where Given Imaging is exhibiting at booth #1059 throughout the conference.

"The SmartPill motility monitoring system gives us a complete GI profile that allows us to recognize multiregional transit irregularities in patients with possible upper or lower GI dysmotility," said Braden Kuo, M.D., Director, Gastrointestinal Motility Laboratory, Massachusetts General Hospital. "These new studies add to the growing body of data showing that SmartPill is an extremely valuable tool enabling us to diagnose and treat motility disorders for better patient care."

A key poster presentation, Wireless Motility Capsule Alters Diagnosis and Affects Clinical Management in Patients with Suspected Gastrointestinal Dysmotility, poster Mo2092, presented by Shreya Raja, M.D., Division of Gastroenterology and Hepatology, Johns Hopkins School of Medicine, and colleagues, retrospectively examined how SmartPill findings affect clinical care. The study reviewed numerous data points including pressure, pH and temperature data collected by SmartPill to determine the diagnostic yield of wireless motility capsule and assess changes in 51 consecutive patients with suspected dysmotility. Clinical management decisions after the SmartPill procedure, including medication changes, referrals for additional diagnostic tests, and outside referrals, were also analyzed. Results show that data collected by SmartPill impacts patient care by altering diagnosis and clinical management. Alteration in diagnosis was frequent and change in medication was made ...

Synageva's Sebelipase Alfa Receives Breakthrough Therapy Designation for Early Onset LAL Deficiency from the U.S. Food and Drug Administration

Eddie Staley — Mon, 20 May 2013 11:34:14 +0000

Synageva BioPharma (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to sebelipase alfa for the treatment of early onset lysosomal acid lipase deficiency (LAL Deficiency), also known as Wolman disease. The designation was based on data generated from clinical trials with sebelipase alfa in patients with early onset LAL Deficiency. The FDA also confirmed that late onset LAL Deficiency is "a serious and life threatening disease or condition" and that Breakthrough Therapy designation could be obtained for this aspect of the disease with additional clinical information. According to the FDA, Breakthrough Therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for Breakthrough Therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. A Breakthrough Therapy designation conveys all of the fast track program features, as well as more intensive FDA guidance on an efficient drug development program. "We are pleased that the FDA designated sebelipase alfa as Breakthrough Therapy for patients with early onset LAL Deficiency, or Wolman disease, " said Anthony Quinn, MBChB, PhD, FRCP, Senior Vice President and Chief Medical Officer of Synageva. "We are deeply aware of the devastating impact this disease has on infants who often die within the first six months of life because of this disease. Our ongoing Phase 2/3 trial delivers hope for these infants and their families. We continue to progress site activation and patient enrollment in both this trial and the global Phase 3 ARISE trial in children and adults, and look forward to working closely with the FDA to support approval of the drug in an efficient manner." About Synageva's lead programs sebelipase alfa for LAL Deficiency and SBC-103 for MPS IIIB LAL Deficiency is a rare autosomal recessive lysosomal storage disorder (LSD) caused by a marked decrease in LAL enzyme activity. Late onset LAL Deficiency, sometimes called Cholesteryl Ester Storage ...