Rockwell Announces FDA Advisory Committee Review Of Triferic; Horzion Pharma Presents Results of ACTIMMUNE(R) Phase 2 Data Print E-mail
By William Kent   
Thursday, 09 October 2014 19:24
Below is a look at some of the headlines for companies that made news in the healthcare sector on October 9, 2014.
Rockwell Medical, Inc. (Nasdaq: RMTI), a fully integrated biopharmaceutical company targeting end-stage renal disease (ESRD) and chronic kidney disease (CKD) with innovative products and services for the treatment of iron replacement, secondary hyperparathyroidism and hemodialysis, announced the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food & Drug Administration (FDA) is scheduled to review the Company's New Drug Application (NDA) for Triferic on November 6, 2014 in an afternoon session.

Triferic is Rockwell's investigational drug for the treatment of iron replacement and maintenance of hemoglobin in hemodialysis patients. Triferic is under review by the Office of Hematology and Oncology Products (OHOP) Division of Hematology products. The proposed indication for Triferic is for the treatment of iron loss or iron deficiency to maintain hemoglobin in patients with hemodialysis dependent chronic kidney disease and to reduce the prescribed dose of erythropoiesis stimulating agents (ESAs) required to maintain desired hemoglobin levels.

The FDA accepted the Triferic NDA for review on May 28, 2014. The PDUFA date for the FDA to complete its review of the Triferic NDA is January 24, 2015.

"We are pleased to discuss the potential efficacy benefit and safety profile of Triferic with the Advisory Committee, and we will continue to work closely with the FDA during the review of the NDA," said Mr. Robert L. Chioini, Founder, Chairman and CEO of Rockwell. "Triferic has a novel mechanism of action and mode of administration which addresses the unmet need of maintenance iron therapy for the hemodialysis population."



 
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Horizon Pharma plc (NASDAQ: HZNP)
announced the presentation of data from a Phase 2 clinical study of ACTIMMUNE (interferon gamma-1b) treatment in children with Friedreich's ataxia (FA). An abstract of the data has been published in a supplement to the Annals of Neurology and is being presented as a poster during the 139th Annual Meeting of the American Neurological Association in Baltimore, MD on Monday, October 13.

This single center, Phase 2 open-label study of 12 children ages five to 17 years with genetically confirmed FA with treatment of up to 12 weeks, evaluated the safety, tolerability and efficacy of ACTIMMUNE. The study measured frataxin levels, a biomarker of disease and clinical measures (Friedreich's Ataxia Rating Scale [FARS] score and other neurological evaluations) as efficacy markers. Additional details on the study design may be found at www.clinicaltrials.gov.

The results, as noted in the published abstract, showed ACTIMMUNE was well tolerated with no serious adverse events and two subjects reporting severe events and subsequent dose reductions. The safety findings generally reflected the labeled safety profile for ACTIMMUNE. Changes in frataxin protein levels, the primary study endpoint, were statistically significant in red blood cells, white blood cells and platelets. The magnitude of change observed was small and varied between tissues. Mean improvement in the FARS score, a clinically validated measurement of patient performance and secondary endpoint, was statistically significant (p=0.008) and was equivalent to two years of extension of disease progression. No other changes were observed.

"The highly significant improvement in the FARS score, a key measure of improvement in disease progression, supports further study of ACTIMMUNE in Friedreich's ataxia," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "We plan to work with the Collaborative Clinical Research Network in Friedreich's Ataxia (CCRN in FA) and the Friedreich's Ataxia Research Alliance (FARA) to rapidly move into a registration program for ACTIMMUNE in FA. We plan to meet with the FDA this quarter regarding the clinical development and regulatory pathway for ACTIMMUNE in FA."

The study was sponsored by FARA and conducted by David Lynch, M.D., Ph.D., professor of neurology at Children's Hospital of Philadelphia (CHOP) and principal investigator of the CCRN in FA. Investigational product was provided by Vidara Therapeutics Research Limited, now part of Horizon Pharma plc.

"The FA community has been working toward a viable treatment option and we were proud to be able to sponsor this initial pilot study. We are encouraged that ACTIMMUNE was well tolerated and there was a significant improvement observed in a clinical outcome measure," said Ronald Bartek, president, director and co-founder of FARA. "We are very pleased to partner with Horizon Pharma and look forward to working together to explore the potential for ACTIMMUNE in FA.



Also Thursday:


Aastrom Biosciences, Inc. (Nasdaq:ASTM)
, a leading developer of patient-specific expanded cellular therapies for the treatment of severe diseases and conditions, today announced plans to change its corporate name to Vericel Corporation and move its corporate headquarters to Cambridge, Massachusetts.

 Abiomed, Inc. (Nasdaq:ABMD)
announced today that on Thursday, October 30, 2014, the Company will release financial results for the second quarter of fiscal 2015.

ActiveCare Inc. (OTCBB:ACAR)
, a leader in diabetes monitoring and wellness services for self-insured employers nationwide, added 2,438 new members between July 1, 2014 and September 30, 2014.

Amarantus Bioscience Holdings, Inc. (OTCQB:AMBS)
, a biotechnology company focused on developing diagnostics and therapeutics for Alzheimer's disease, Parkinson's disease and ophthalmological disorders, reported positive data from its LP-002 study of the Lymphocyte Proliferation Test (LymPro Test®) blood diagnostic for Alzheimer's disease (AD).

AngioDynamics (Nasdaq:ANGO)
, a leading provider of innovative, minimally invasive medical devices for vascular access, surgery, peripheral vascular disease and oncology, today reported financial results for the first quarter ended August 31, 2014.

Applied Genetic Technologies Corporation (Nasdaq:AGTC)
, a clinical stage biotechnology company developing adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, today announced that Sue Washer, AGTC President and CEO, will present at two upcoming conferences.

Avalanche Biotechnologies, Inc. (Nasdaq:AAVL),
a clinical-stage biotechnology company focused on discovering and developing novel gene therapies to transform the lives of patients with sight-threatening ophthalmic diseases, today announced that Thomas W. Chalberg, Jr., Ph.D., Founder and Chief Executive Officer, will present at the Ophthalmology Innovation Summit at the American Academy of Ophthalmology 2014 Annual Meeting on Thursday, October 16, 2014 at 8:49 am CDT in Chicago, IL.

BioCorRx, Inc. (BICX)
, a leader in addiction treatment, will host its first shareholder conference call on Thursday, October 16, 2014, at 11:00 am Pacific Standard Time to discuss its operations through the end of the second quarter 2014 as well as outlook for the remainder of this year and 2015. 

Capricor Therapeutics, Inc. (OTCBB:CAPR)
, a biotechnology company focused on developing novel therapeutics for the treatment of cardiovascular diseases, today announced that it plans to develop a clinical program using Cenderitide for the treatment of post-acute heart failure using Insulet's drug delivery system based on the OmniPod® insulin management system.

Cyclacel Pharmaceuticals, Inc. (Nasdaq:CYCC) (Nasdaq:CYCCP)
, a biopharmaceutical company developing oral therapies that target the various phases of cell cycle control for the treatment of cancer and other serious disorders today announced that the independent Data and Safety Monitoring Board (DSMB) for the Company's Phase 3 SEAMLESS study in acute myeloid leukemia (AML) has completed its fourth planned safety review and recommended that the study should continue as planned without any modifications.

Ekso Bionics Holdings, Inc. (OTCQB:EKSO)
, a leading robotic exoskeleton company, announced today the promotion of Thomas Looby to President and Chief Commercial Officer, effective immediately.

Endologix, Inc. (Nasdaq:ELGX)
, developer and marketer of minimally invasive treatments for aortic disorders, announced today details relating to the release of its third quarter 2014 financial results, which will take place on Thursday October 30, 2014 after the close of the market.

Express Scripts Holding Company (Nasdaq:ESRX)
, the largest pharmacy benefit management company in North America, announced today its intention to release its third quarter earnings on Tuesday, October 28, 2014 after the market closes and will hold its quarterly conference call to discuss third quarter results on Wednesday, October 29, 2014, at 8:30 a.m. Eastern Time (7:30 a.m. Central Time).

India Globalization Capital, Inc. (NYSE MKT: IGC)
, announces that it has received land in Nagpur, India, valued at over $4 Million in exchange for IGC's 22% minority ownership in Sricon.

Integra LifeSciences Holdings Corporation (Nasdaq:IART)
will release its third quarter 2014 financial results on Monday, November 3, 2014.

IPC The Hospitalist Company, Inc. (Nasdaq:IPCM)
, a leading national hospital medicine and post-acute physician group practice company, will report its financial results for the third quarter ended September 30, 2014 on Wednesday, October 22, 2014, after the market close.

Lombard Medical, Inc. (Nasdaq:EVAR)
, a medical device company focused on endovascular aneurysm repair (EVAR) of abdominal aortic aneurysms (AAAs), today announced that its lead product, Aorfix™, is now being used by leading surgeons at 5 US Department of Veterans Affairs (VA) medical centers.

Materialise NV (Nasdaq:MTLS)
, a leading provider of additive manufacturing software and of sophisticated 3D printing solutions in the medical and industrial markets, has listed its 3D-printed cardiovascular HeartPrint® models as a medical device in the USA and EU markets. After years of 3D printing anatomical models for educational and research purposes, the Company addressed the need for models that can assist with diagnosing, planning and practicing complex cardiovascular procedures.

Momenta Pharmaceuticals, Inc. (Nasdaq:MNTA)
, a biotechnology company specializing in the characterization and engineering of complex drugs, announced top-line results from the dose-escalation component (Part A) of the Phase 1/2 trial evaluating necuparanib in combination with Abraxane® (ABX; nab-paclitaxel) and gemcitabine (GEM) in patients with advanced metastatic pancreatic cancer (ClinicalTrials.gov Identifier NCT01621243).

NanoString Technologies, Inc. (Nasdaq:NSTG)
, a provider of life science tools for translational research and molecular diagnostic products, today announced that the Company will release third quarter 2014 financial results after the close of trading on Tuesday, November 4, 2014.

Otonomy, Inc. (Nasdaq:OTIC)
, a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapeutics for diseases and disorders of the inner and middle ear, today announced the enrollment of the first patients in a multiple-dose safety study of OTO-104 initiated in the United Kingdom (U.K.) in patients with Ménière's disease.

The Spectranetics Corporation (Nasdaq:SPNC)
today announced that the Company will release 2014 third quarter financial results after market close on Thursday, October 23, 2014.

StoneMor Partners L.P. (NYSE:STON)
announced today that it will host an investor and analyst day on November 12, 2014 at New York City's Grand Hyatt Hotel.

Tekmira Pharmaceuticals Corporation (Nasdaq:TKMR) (TSX:TKM)
, a leading developer of RNA interference (RNAi) therapeutics, today announced that Dr. Ian MacLachlan, Tekmira's Executive Vice President and Chief Technical Officer, will present preclinical data for TKM-HBV, the Company's product candidate targeting hepatitis B virus, at the 10th Annual Meeting of the Oligonucleotide Therapeutics Society.

VeriTeQ Corporation (OTC Markets:VTEQ)
, a provider of implantable medical device identification and radiation dose measurement technologies, announced today that it has entered an agreement providing an exclusive license of multiple patents from North Carolina State University ("NCSU").

XOMA Corporation (Nasdaq:XOMA)
, a leader in the discovery and development of therapeutic antibodies, today announced it has initiated dosing in its Phase 1 study exploring the safety and tolerability of single intravenous doses (IV) of XOMA 358, the lead compound from the Company's XMetD program, in healthy volunteers.



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