BioMarin Pharmaceutical : Positive Results for Phase I/II Trial for BMN 110 for MPS IVA

April 29, 2010 - GALNS Demonstrates Benefits in Endurance and Pulmonary Function Endpoints - BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced positive results for the Phase I/II trial for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. The company expects to initiate a pivotal Phase III trial in the fourth quarter of 2010.

Highlights from the Phase I/II study:

- Endurance improvements with GALNS were consistent with, and in some cases, better than those observed in pivotal studies of approved enzyme replacement therapies.
- Clinically meaningful improvements in two measures of endurance (6-minute walk distance and 3-minute stair climb) were achieved at both 24 weeks and 36 weeks as compared to baseline. (See Table 1 below).
- Clinically meaningful improvements in two measures of pulmonary function (forced vital capacity and maximum voluntary ventilation) were achieved at 36 weeks as compared to baseline... BioMarin Pharmaceutical's Press Release -

deCODE genetics "deCODEing Smoking: New Genetic Factors Behind Nicotine Dependence and Lung Cancer"

April 25, 2010 – Cigarette smoking is a major cause of illness and death worldwide. But it is a complex behavior, and how much people smoke, how hard they find it to quit, and the impact of long-term smoking on health varies greatly among individuals. A substantial portion of this variability is genetic. Two years ago, deCODE discovered the first common, single-letter variation (SNP) in the sequence of the human genome, on chromosome 15q25, associated with nicotine addiction and risk of lung cancer. Today, deCODE scientists and academic colleagues from 23 institutions in a dozen countries build on this work with the discovery of common SNPs on chromosomes 8p11 and 19q13 that among smokers increase the number of cigarettes smoked per day (CPD), a measure of nicotine addiction, and increase risk of lung cancer... deCODE genetics' Press Release -

Biodesix : VeriStrat® Outcomes Data in Lung Cancer to be Presented at IASLC/ESMO 2nd European Lung Cancer Conference

April 19, 2010 – Biodesix announced that results from the VeriStrat biomarker analysis of a Phase III clinical trial will be presented by David Carbone, M.D., Ph.D., Professor of Medicine and Cancer Biology, Vanderbilt University School of Medicine, on Friday, April 30th at the 2nd European Lung Cancer Conference to be held in Geneva. The purpose of the study was to evaluate the ability of VeriStrat to predict patient outcomes to epidermal growth factor receptor tyrosine kinase inhibitor (EGFR-TKI) therapy in previously treated non-small cell lung cancer (NSCLC) patients.

VeriStrat, a blood-based proteomic test, is currently used by physicians to help them guide treatment for patients with previously treated advanced NSCLC... Biodesix's Press Release -

Johnson & Johnson Pharmaceutical Research and Development : FDA Approves New Drug Application for PANCREAZE

April 13,2010 - Pancreatic Enzyme Therapy Approved by FDA to Treat Exocrine Pancreatic Insufficiency - Johnson & Johnson Pharmaceutical Research and Development (J&JPRD) announced that the U.S. Food and Drug Administration (FDA) has approved a new drug application (NDA) for PANCREAZE™ (pancrelipase) delayed-release capsules for the treatment of exocrine pancreatic insufficiency due to cystic fibrosis or other conditions. Exocrine pancreatic insufficiency is the inability to properly digest and absorb food due to a lack of enzymes made by the pancreas that are important in the digestion of fats, proteins, and sugars... Johnson & Johnson's Press Release -

GeNO LLC : Investigational New Drug Application for Nitric Oxide Delivery System

March 9, 2010 – GeNO LLC a privately held, advanced development-stage technology company, announced that the U.S. Food and Drug Administration (FDA) has granted clearance of its Investigational New Drug (IND) application for its stand-alone gas cylinder Nitrosyl TM Delivery Platform (NDP). The initial indication to be studied for nitric oxide delivered via the GeNO Nitrosyl delivery system is as a diagnostic agent for administration as an adjunct to right heart catheterization in patients with Pulmonary Arterial Hypertension (WHO Group 1) to add information to improve clinical decision making. No agents are currently approved for this indication... [PDF] GeNO's Press Release -

Spectrum Pharmaceuticals : Belinostat Data Presentation at 101st Annual Meeting of the American Association for Cancer Research

Apr 21, 2010 - Data Suggest HDAC Inhibitors, Such as Belinostat, May Be Efficacious in the Treatment of Small Cell Lung Cancer - Results Indicate Synergy With Conventional Chemotherapy Agents - Spectrum Pharmaceuticals, Inc. (NasdaqGM:SPPI), a commercial-stage biotechnology company with a primary focus in oncology, announced results of a pre-clinical study conducted by the National Cancer Institute of belinostat in the treatment of small-cell lung cancer that was presented in a poster session on Wednesday, April 21, 2010 at the 101st Annual Meeting of the American Association for Cancer Research (AACR), being held at the Walter E. Washington Convention Center in Washington, DC.

"Based on the data presented today at AACR, we believe belinostat may be an effective treatment option for small-cell lung cancer,"... Spectrum Pharmaceuticals' Press Release -

Ariad : Preclinical Data on Its Investigational ALK Inhibitor, AP26113, Demonstrating That It Can Overcome Mutation-Based Drug Resistance in Cancer...

Apr 20, 2010 - ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) announced results of preclinical studies on its investigational anaplastic lymphoma kinase (ALK) inhibitor - AP26113 - showing potent inhibition of the target protein and of mutant forms that are resistant to the first-generation ALK inhibitor, which currently is in clinical trials in patients with cancer. ARIAD scientists presented these data today at the annual meeting of the American Association for Cancer Research (AACR) in Washington, D.C.

Genetic studies indicate that abnormal expression of ALK is a key driver of certain types of non-small cell lung cancer (NSCLC) and neuroblastomas, as well as anaplastic large cell lymphoma. Since ALK is generally not expressed in normal adult tissues, it represents a highly promising molecular target for cancer therapy... ARIAD Pharmaceuticals' Press Release -

Synta Pharmaceuticals : Results at AACR Supporting Correlative Markers for Elesclomol Activity

Apr 20, 2010 - Link demonstrated between elesclomol anti-cancer activity and levels of HIF-1a and LDH - Additional results presented demonstrating activity of elesclomol in cisplatin-resistant lung cancer models - Synta Pharmaceuticals Corp. (NASDAQ: SNTA), a biopharmaceutical company focused on discovering, developing, and commercializing small molecule drugs to treat severe medical conditions, today announced preclinical results presented at the American Association for Cancer Research (AACR) 101st Annual Meeting that support correlative markers for elesclomol anti-cancer activity. In addition, results were presented by Synta collaborators showing that elesclomol demonstrates activity in cisplatin-resistant lung cancer models... Synta Pharmaceuticals' Press Release -

Graceway Pharmaceuticals : Maxair Autohaler Remains Available Through December 31, 2013

04/19/2010 - Graceway Pharmaceuticals, LLC announced that the Food and Drug Administration (“FDA”) published a long-awaited Final Rule regarding the continued availability of Maxair Autohaler (pirbuterol acetate inhalation aerosol) and other pressurized metered-dose inhalers containing chlorofluorocarbons (CFCs). Under the rule, Maxair Autohaler will remain available for physicians and patients through December 31, 2013.

In support of its decision, FDA noted that it received a large number of comments from physicians and patients addressing the public health benefits of pirbuterol – the active ingredient in Maxair Autohaler. FDA likewise observed that the need for patient education was an important factor in extending the effective date of the rule, and the Agency committed to actively monitor the patient transition to CFC-free alternatives... Graceway Pharmaceuticals' Press Release -

Patient Enrollment Complete in Tragara Pharmaceuticals’ Phase II Trial of Capoxigem® in Non-Small Cell Lung Cancer

April 6, 2010 – Tragara Pharmaceuticals, Inc. announced the completion of patient enrollment to its APRiCOT-L study, a phase II clinical trial of its oral, once-daily anti-cancer agent, Capoxigem® (apricoxib, TG01), in non-small cell lung cancer (NSCLC). The APRiCOT-L study is a randomized, double-blind, multi-center, placebo-controlled trial designed to evaluate Capoxigem in combination with erlotinib in second and third line NSCLC patients that have failed a platinum-containing regimen. Tragara is utilizing a biomarker in order to identify the patient population that will benefit most from Capoxigem therapy; biomarker response is an enrollment criterion for the study. The trial enrolled 128 patients and data from the study is expected to be available in Q3 2010.
“The completion of patient enrollment in this study is a significant milestone for Tragara and for the development of Capoxigem in several respects,”... [PDF] Tragara Pharmaceuticals' Press Release -

Invida Group : Actelion Extends Partnership with Invida to Market Tracleer in Key Asian Markets

March 26, 2010 – Invida Group, the leading provider of healthcare brands and services to the Asia Pacific region, today announced that its contract with Swiss biotechnology company, Actelion, has been extended through 2015 to continue commercialization of Tracleer® for the treatment of pulmonary arterial hypertension (PAH) in crucial markets throughout Asia. Actelion signed with Invida initially in 2005, and has extended the contract to market the company’s lead product, Tracleer, in Thailand, Malaysia, Philippines, Vietnam, and Hong Kong.



By extending the contract, Invida will continue to leverage its experience in regulatory and medical affairs, specialized marketing, and its well-established network of key opinion leaders throughout the region. This partnership will allow Invida to continue working with a market-leading biotech firm and leverage its capabilities to commercialize an orphan drug that has proven ability to positively impact disease progression, as well as reduce severity and symptoms in,patients with PAH... [PDF] Invida' Press Release -

Phadia : point of care in vitro specific IgE allergy test into the physician’s office with final FDA clearance of the ImmunoCAP Rapid System

January 25, 2010 – Phadia, the world leader in allergy blood testing diagnostics, today announced that the United States Food and Drug Administration (FDA) has cleared the ImmunoCAP® Rapid Reader II, a part of the ImmunoCAP Rapid System, used to aid in the diagnosis of allergy in the physician’s office.
This clearance now allows Phadia to begin marketing the ImmunoCAP Rapid System to physicians throughout the United States having laboratories that meet CLIA standards for Moderately Complex testing. The test requires a simple capillary blood draw and test results are available to the physician in 20 minutes.

Up to 90% of all paediatric asthma patients have allergies and up to 60% of adult asthma patients have allergies that trigger their asthma attacks. Reducing exposure to these allergens is a key recommendation of the NIH-funded National Asthma Education and Prevention Program and is an important step in the effective management of allergic asthma... Phadia's Press Release -

Elekta : Next Generation SBRT for Lung Cancer with Ground-Breaking 4D Image Guidance to Manage Tumor Motion and Enhance Safety

April 08, 2010 - New technologies enable accurate, non-invasive lung tumor treatment without additional intra-fraction radiation imaging -
Elekta has introduced an advanced solution for treating lung tumors that enables doctors to visually confirm the tumor’s position during the breathing cycle. This new technology treats the lesion with a continuous radiation beam, increasing therapy accuracy while using less imaging radiation during treatment delivery.

Lung tumors have been among the most challenging radiation therapy targets because the patient’s breathing causes tumors to move. New research has shown that the lung tumor shifts position from day to day during the course of treatment (baseline shift).1 Doctors often have had to use external skin surface markers or implanted markers to estimate lung tumor position during the breathing cycle and then apply the beam only during certain points in the patient’s respiration. These strategies require complex, time-consuming planning and delivery, and prolong treatment with an inefficient stop-start (i.e., gated) beam delivery.

“Elekta’s XVI Symmetry ™ and XVI Intuity™ completely change the approach when it comes to the treatment of lung tumors,”... Elekta's Press Release -

NeoPharm : Orphan Drug Application Filing for IL13-PE38QQR for the Treatment of Idiopathic Pulmonary Fibrosis

Mar 10, 2010 - NeoPharm, Inc. (Other OTC: NEOL.PK), announced that it has filed an orphan drug application with the Office of Orphan Products Development of the United States Food and Drug Administration (FDA) to have Orphan Drug Designation granted to IL13-PE38QQR (IL13-PE) for the treatment of Idiopathic Pulmonary Fibrosis (IPF). The US Orphan Drug Act is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. IPF is the most deadly disease of the lungs in humans with very high morbidity. It is estimated that at least 55,000 patients are diagnosed with IPF each year in America and about 45,000 patients die each year with the disease. There is currently no proven effective treatment for the cure of this lethal disease...NeoPharm's Press Release -

Merrimack Pharmaceuticals : Enrollment in a Phase 1/2 Combination Study of MM-121 and Tarceva® in Patients with Non-Small Cell Lung Cancer

February 22, 2010 – Merrimack Pharmaceuticals, Inc. announced that the first patient has received an initial dose in a Phase 1/2 clinical study combining MM-121 with Tarceva® (erlotinib) in patients with nonsmall cell lung cancer (NSCLC). This is the first of multiple trials that Merrimack and sanofi-aventis expect to initiate in 2010 as part of a broad Phase 2 clinical development program for MM-121.

MM-121, Merrimack’s lead oncology therapeutic candidate, is an antibody designed to block signaling of ErbB3. Erlotinib is a small molecule targeting the epidermal growth factor receptor (EGFR).
“Our Network Biology analysis of cancer cells indicates that tumors often become resistant to EGFR-targeted therapies by compensating for tumor growth restriction through the ErbB3 receptor,"... [PDF] Merrimack Pharmaceuticals' Press Release -

Clinical Data : Results of Phase I Studies of Stedivaze™ Demonstrating Safety and Tolerability in Patients with Asthma and COPD

March 30, 2010 - Enrollment continues in Phase III ASPECT trial of Stedivaze – Clinical Data, Inc. (NASDAQ: CLDA), announced results from two Phase I studies of Stedivaze™ (apadenoson), which demonstrated that Stedivaze was safe and well tolerated in patients with asthma and chronic obstructive pulmonary disease (COPD). Stedivaze is a potent and highly selective agonist of the adenosine A2A receptor subtype in development as a pharmacologic stress agent for myocardial perfusion imaging (MPI). Currently available adenosine agonists must be used with caution or are contraindicated in patients with asthma and COPD. The high selectivity of Stedivaze offers a potential advantage for the safe use in this population, accounting for approximately 10 percent of the 7.6M MPI tests performed annually.1 The Company is also actively enrolling patients in ASPECT 1, a Phase III trial designed to demonstrate the safety and effectiveness of Stedivaze.
“The positive results from our preliminary studies in asthmatics and COPD patients are encouraging and represent a milestone toward our goal of developing a coronary vasodilator that is both safe and well tolerated in these populations,”... [PDF] Clinical Data's Press Release -

Pearl Therapeutics : Positive Results from Phase 1 Trial of PT003, a Bronchodilator Combination Therapeutic for Chronic Obstructive Pulmonary Disease

March 29, 2010 – Pearl Therapeutics Inc., a company developing clinically differentiated combination therapies for the treatment of highly prevalent chronic respiratory diseases, announced positive results from a Phase 1 safety and pharmacokinetics study of PT003, its lead combination therapeutic for the treatment of chronic obstructive pulmonary disease (COPD). Based on these results, and previously announced positive results from Phase 2a studies of PT003’s individual components, the company has advanced PT003 into a Phase 2b trial.

PT003 is an inhaled combination bronchodilator product comprised of glycopyrrolate, a long-acting muscarinic antagonist (LAMA), and formoterol, a well-known, established, long-acting ß2-agonist (LABA), delivered via a hydrofluoroalkane metered dose inhaler (HFA-MDI). PT003 is the first and only dual long-acting rapid bronchodilator LAMA-LABA combination product in development in an HFA-MDI formulation, the most widely used inhalation drug delivery format.

“COPD is a progressive lung disease affecting millions of patients, representing an important unmet clinical need. Pearl’s technology has the potential to satisfy the medical community’s need for long-acting bronchodilator combination products that allow bronchodilators of different pharmacological classes to be combined, thereby improving patient outcomes, medication delivery and adherence,”... Pearl Therapeutics' Press Release -

Vectura to receive $9.5m payment and up to $25m loan facility

18 March 2010: Vectura Group plc (LSE: VEC), the specialty pharmaceutical company, announces that it has agreed with Sandoz AG to take full control of the US development and commercialisation of VR315, its combination product for asthma/COPD. Good progress continues to be made with the development of VR315. In Europe, there is no change to the agreement signed in April 2006, and Sandoz will continue to be fully responsible for the development, manufacture and commercialisation of the product.


Under the revised agreement, Vectura regains full rights and responsibilities for the development and commercialisation of VR315 in the US and is released from its profit share obligations. The reacquisition of full control of this important product in the US is part of Vectura’s strategy to become a cash-generative specialty pharmaceutical company, and VR315 will form the cornerstone of Vectura’s nascent US operations... Vectura's Press Release -