|Pfizer To Submit New Application For Breast Cancer Drug; Ataluren Phase 3 Trial Results Published in The Lancet Respiratory Medicine|
|By Staff and Wire Reports|
|Friday, 16 May 2014 19:56|
Pfizer Inc. (NYSE: PFE) announced that it will submit a New Drug Application (NDA) with the United States Food and Drug Administration (FDA) for palbociclib, combined with letrozole, as first-line systemic treatment of post-menopausal women with estrogen receptor positive (ER+), human epidermal growth factor receptor 2 negative (HER2-) locally advanced or metastatic breast cancer. Pfizer expects to submit the NDA early in the third quarter of this year. This decision was based on discussions with the FDA regarding the final results of PALOMA-1, a randomized, Phase 2 trial comparing palbociclib plus letrozole versus letrozole alone in this population of patients.
Palbociclib received Breakthrough Therapy designation from the FDA in April 2013, for the first-line systemic treatment of women with advanced or metastatic ER+, HER2- breast cancer. This designation was based on interim data from the PALOMA-1 trial.
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced the results of a Phase 3 study of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) were published in Lancet Respiratory Medicine. The results demonstrated positive trends in both the primary endpoint, lung function as measured by relative change in % predicted FEV1 (forced expiratory volume in one second) and in the secondary outcome measure, rate of pulmonary exacerbations. The collective data from this trial, including retrospective and subgroup analyses support the conclusion that ataluren was active and showed clinically meaningful improvements over placebo in these trials.
"The overall data from this trial are promising. Patients on ataluren experienced fewer pulmonary exacerbations and showed a stabilization in their FEV1 results, particularly in the subgroup of patients that did not use chronic inhaled aminoglycosides. Such stabilization of disease is an important clinical endpoint, particularly for this patient population that has one of the most severe forms of CF. CF patients with nonsense mutations do not produce any functional CFTR protein and therefore generally have a more severe form of cystic fibrosis. Current treatments for nonsense mutation cystic fibrosis focus on alleviating symptoms and reducing infections, whereas ataluren targets the underlying cause of disease," stated Michael Konstan, M.D., lead study investigator from University Hospitals Rainbow Babies and Children's Hospital in Cleveland, Ohio.
The Phase 3 double-blind, placebo-controlled study, which was conducted across 11 countries, compared ataluren (n=116) to placebo (n=116) in nmCF patients. The primary endpoint, the relative change from baseline in %-predicted FEV1 at 48 weeks, showed a positive trend favoring ataluren versus placebo, and a larger effect in patients not receiving chronic inhaled tobramycin. In the intent-to-treat population, there was a 3% difference in the relative change from baseline in %-predicted FEV1 between the ataluren and placebo groups at Week 48 (-2.5% change on ataluren vs. -5.5% change on placebo; p=0.12) which was not statistically significant. An analysis of the relative change from baseline in %-predicted FEV1 across all post-baseline study visits demonstrated an average difference between ataluren and placebo of 2.5% (-1.8% average change on ataluren vs. -4.3% average change on placebo; p= 0.048). There were 23% fewer pulmonary exacerbations in the ataluren group compared to placebo (p=0.0992). Further results from a post hoc analysis of the subgroup of patients not receiving chronic inhaled tobramycin showed a 5.7% difference in relative change from baseline in % predicted FEV1 favoring ataluren, with a mean change from baseline of -0.7% in the ataluren arm, and – 6.4% in the placebo arm (nominal p=0.0082). In addition, there were 40% fewer exacerbations in ataluren-treated patients in this subgroup. The outcomes observed in multiple endpoints between the subgroup of patients who were not prescribed chronic inhaled tobramycin and the subgroup of patients who were prescribed chronic inhaled tobramycin as well as post-hoc in vitro testing showing the interference of aminoglycoside antibiotics with ataluren activity support the hypothesis that inhaled tobramycin may interfere with ataluren's mechanism of action.
Safety results indicate that ataluren was generally well tolerated. The overall incidence of adverse events through Week 48 was similar in the ataluren and placebo groups, except for the occurrence of creatinine elevations that occurred more frequently in the ataluren group in connection with concomitant treatment with systemic aminoglycosides. Most treatment emergent adverse events were of mild (Grade 1) or moderate (Grade 2) severity, and no life-threatening adverse events were reported. Most serious adverse events reported in this study were CF pulmonary exacerbations and were considered unrelated to ataluren treatment. Eight patients in the ataluren arm and three patients in the placebo arm discontinued treatment due to an adverse event.
"We are very encouraged by the data from this trial. Given spirometry and pulmonary exacerbation results in the subgroup of patients not receiving chronic inhaled tobramycin, and a favorable safety profile, this study supports further clinical testing of ataluren as a potential first-in-class treatment for nmCF patients not receiving chronic inhaled tobramycin," stated Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics, Inc. "We look forward to initiating a confirmatory ataluren trial in nmCF patients later this year."
Akers Biosciences, Inc. (Nasdaq:AKER) (LSE:AKR), a leading designer and manufacturer of rapid diagnostic screening and testing products, entered into an agreement with Jai Capital LLC, a Nevada company specializing in market development, distribution, and joint ventures of medical devices in India, to market the Company's PIFA Heparin/Platelet Factor-4 Rapid Assay, PIFA Dengue Assay, Battlefield Blood Transfusion Card and Tri-Cholesterol rapid tests.
Analogic Corporation (Nasdaq:ALOG), enabling the world's medical imaging and aviation security technology, today announced availability of its new Flex Focus™ 300 Ultrasound System for Urology at the American Urological Association (AUA) Annual Meeting in Orlando, Fla.
Chimerix, Inc. (Nasdaq:CMRX), a biopharmaceutical company developing novel, oral antivirals in areas of high unmet medical need, today announced that it has regained the worldwide rights to CMX157, Chimerix's novel lipid acyclic nucleoside phosphonate, from Merck, known as MSD outside the United States and Canada.
EDAP TMS SA (Nasdaq:EDAP), the global leader in therapeutic ultrasound, announced that its HIFU and lithotripsy technologies will be featured at the 109th Annual American Urology Association (AUA) Meeting that will be held in Orlando, FL on May 16 - 24, 2014.
Medical Marijuana, Inc. (OTC Pink:MJNA) is pleased to announce that its portfolio company, HempMeds™, is exhibiting at the American Academy of Anti-Aging Medicine (A4M) Orlando World Congress May 16-17.
Nanosphere, Inc. (Nasdaq:NSPH), a company enhancing medicine through targeted molecular diagnostics, today announced that new performance and clinical outcome data for three Verigene® multiplex infectious disease tests will be presented at the 114th General Meeting of the American Society for Microbiology (asm2014), to be held in Boston, Massachusetts on May 17 – 20, 2014.
Neptune Technologies & Bioressources Inc. (Nasdaq:NEPT) (TSX:NTB), announced today that the Corporation will nominate as management's director nominees for the upcoming annual general meeting of Neptune shareholders
OraSure Technologies, Inc. (Nasdaq:OSUR), the market leader in point of care diagnostics, announced that it will webcast its 2014 Annual Meeting of Stockholders beginning at 10:00 a.m. Eastern Time (7:00 a.m. Pacific Time) on Thursday, May 22, 2014.
OraSure Technologies, Inc. (Nasdaq:OSUR), a leader in the development, manufacture and distribution of point of care diagnostic and collection devices and other technologies designed to detect or diagnose critical medical conditions, including HCV and HIV, will visit the NASDAQ MarketSite in Times Square in recognition of National Hepatitis Testing Day.
OXiGENE, Inc. (Nasdaq:OXGN), a clinical-stage biopharmaceutical company developing novel therapeutics to treat cancer, announced that David (Dai) Chaplin, Ph.D., has been appointed to the position of President and Chief Executive Officer, replacing Peter Langecker, M.D., Ph.D.
Hospitals and health systems participating in Premier, Inc.'s (Nasdaq:PINC) Hospital Engagement Network (HEN) have avoided more than 35,600 readmissions, contributing to a savings of at least $313 million over 2012 and 2013.
Propanc Health Group Corporation (OTCQB:PPCH), a developmental stage a healthcare company focused on new cancer treatments for patients with solid tumors, today announced its key strategic objectives for the development of its lead product, PRP.
TESARO, Inc. (Nasdaq:TSRO), an oncology-focused biopharmaceutical company, today announced that Earl M. (Duke) Collier, Jr. has been elected to the TESARO Board of Directors.