Prosensa to Provide Corporate Update to Key Stakeholders; Merck Initiates Rolling Submission for MK-3475 Print E-mail
By Staff and Wire Reports   
Monday, 13 January 2014 21:04
Below is a look at some of the headlines for companies that made news in the healthcare sector on January 13, 2014.

Prosensa Holding N.V. (NASDAQ: RNA)
, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet medical need, will be providing updates to both the financial and patient communities regarding drisapersen.

As previously announced, the company will be providing a full corporate update, which will now also include additional analyses of the drisapersen dataset, at the 32nd Annual J.P. Morgan Healthcare Conference on Thursday, January 16, 2014 at 10:00 AM Pacific Time. Furthermore, the company will be providing a separate update to the patient community on Tuesday, January 21, 2014 at 8:00 AM Eastern Time.  A live webcast of these presentations can be accessed through the Investors & Media section of the Prosensa corporate website at and will be archived for 90 days.

Drisapersen, (previously GSK2402968/PRO051), an antisense oligonucleotide which induces exon skipping of exon 51, is currently in-late stage development for DMD. Drisapersen has orphan drug status in the EU, US, Japan and Australia. In June 2013, drisapersen was granted Breakthrough Therapy designation by the US Food and Drug Administration.

The overall drisapersen clinical registration program comprises three double-blind, placebo-controlled studies (DMD114117, DMD114876 and DMD114044) and two long term open-label extension studies (DMD114673 and DMD114349).


Merck (NYSE: MRK)
, known as MSD outside the United States and Canada, said today it has started a rolling submission to the U.S. Food and Drug Administration (FDA) of a Biologics License Application for MK-3475, the company's investigational anti-PD-1 immunotherapy, for patients with advanced melanoma who have been previously treated with ipilimumab. A rolling submission allows completed portions of the application to be submitted and reviewed by the FDA on an ongoing basis. The company expects to complete the application in the first half of 2014.

“Our MK-3475 is a novel immunomodulatory molecule that holds promise for patients with advanced malignancy who now have limited treatment options,” said Roger M. Perlmutter, M.D., Ph.D., president, Merck Research Laboratories. “Initiation of this rolling submission represents an important milestone in the MK-3475 clinical development program for patients suffering from malignant melanoma.”

MK-3475 is currently being studied in three clinical trials for advanced melanoma including a Phase III trial of MK-3475 versus ipilimumab in ipilimumab-naïve advanced melanoma patients (PN 006). Enrollment is complete in the advanced melanoma cohorts in the company's Phase IB trial (PN 001) and the Phase II trial (PN 002) comparing two doses of MK-3475 versus chemotherapy in patients with advanced melanoma who have progressed after prior therapy. In April 2013, Merck announced that MK-3475 received a Breakthrough Therapy designation for advanced melanoma from the FDA.

Also Monday:

Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN)
today reported progress on the Company's portfolio of proprietary compounds for the treatment of chronic hepatitis C (HCV) and outlined its 2014 milestones.

Ahead of its presentation at the JP Morgan Healthcare Conference in San Francisco, Oxford Immunotec Global PLC (Nasdaq:OXFD) this morning announced that based on preliminary unaudited financial data, the Company expects total revenue for the fourth quarter of 2013 will be between $10.1 and $10.5 million.

Align Technology, Inc. (NASDAQ: ALGN)
today announced the promotion of Raphael S. Pascaud to vice president international, reporting directly to Thomas M. Prescott, Align Technology president and CEO. Mr. Pascaud assumes the position vacated by Richard Twomey, who left the Company at the end of 2013 to become CEO of a leading global healthcare products and services company.

Alcobra Ltd. (Nasdaq:ADHD)
, an emerging biopharmaceutical company primarily focused on the development and commercialization of its proprietary drug candidate, MG01CI (Sustained-Release Metadoxine), to treat cognitive dysfunction, announced today that it has appointed David C. Baker as Chief Commercial Officer.

Alkame Holdings, Inc. (OTCBB: PINSD) (OTCQB: PINSD)
, a health and technology holding company with a focus on patentable, innovative, and eco-friendly consumer products, is pleased to announce that the Company has entered into an agreement with Avanzar Sales and Distribution, LLC., a California-based beverage focused distribution and consulting company.

Amicus Therapeutics (Nasdaq:FOLD)
, a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today provided its full-year 2014 strategic outlook and financial guidance. John F. Crowley, Chairman and CEO of Amicus, will discuss Amicus' corporate objectives and key milestones in a presentation at the 32nd Annual J.P. Morgan Healthcare Conference on Thursday, January 16, 2014 at 11:30 a.m. PT (2:30 p.m. ET).

Anavex Life Sciences Corp. (OTCQB:AVXL)
today announced that they have established an agreement with The Roskamp Institute for the clinical development of ANAVEX PLUS, a new combination drug of ANAVEX 2-73 and donepezil (Aricept®) for the treatment of Alzheimer's disease.

Arno Therapeutics, Inc. (OTCQB:ARNI)
, a clinical stage biopharmaceutical company focused on the development of oncology therapeutics, today announced that it has expanded its management team with the appointments of Joseph Bisaha, PhD as Vice President, Clinical Operations and Project Management and David M. Jackson, PhD as Vice President, Diagnostics.

Baxano Surgical, Inc. (Nasdaq:BAXS)
, a medical device company focused on designing, developing and marketing minimally invasive products to treat degenerative conditions of the spine affecting the lumbar region, today announced preliminary unaudited revenue for fourth quarter and full fiscal year 2013 in anticipation of investor meetings in San Francisco during the week of January 13th.

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN)
announced today that new practice guidelines issued by the American College of Medical Genetics and Genomics (ACMG) support the need for lifelong management of PHE levels in patients with phenylketonuria or PKU.

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN)
announced today that it has selected an AAV-factor VIII vector, BMN 270, to develop for the treatment of hemophilia A and has initiated IND-enabling studies.

Caliber Imaging & Diagnostics (OTCQB:LCDX)
, formerly Lucid, Inc., announces the appointment of Daniel Mark Siegel, M.D. to its Board of Directors, effective immediately. This increases the number of Board members to eight.

Celsius Holdings, Inc., (Other OTC:CELH)
the creator and marketer of Celsius®, the world's first and only negative calorie beverage backed by clinical science, has commenced manufacturing of its Celsius ready to drink product in Dusseldorf, Germany.

Conatus Pharmaceuticals Inc. (Nasdaq:CNAT)
, a biotechnology company focused on the development and commercialization of novel medicines to treat liver disease, today announced plans to initiate a placebo-controlled (open to sponsor) Phase 2b clinical trial in the second half of 2014 evaluating its lead drug candidate emricasan in Post Orthotopic Liver Transplant (POLT) recipients.

Cyclacel Pharmaceuticals, Inc. (Nasdaq:CYCC) (Nasdaq:CYCCP)
today reviewed 2013 achievements and provided an outline of the Company's key clinical development objectives for 2014, which will be highlighted at the Company's presentation during the Biotech Showcase™ 2014 Conference at 9:30 a.m. PST, Monday, Jan. 13, 2014, at the Parc 55 Wyndham Hotel – Union Square at 55 Cyril Magnin Street in San Francisco.

Enzymotec Ltd. (Nasdaq:ENZY)
, a developer, manufacturer and marketer of innovative bio-active lipid ingredients, today announced the completion of its Migdal Ha'Emeq manufacturing facility expansion and a successful operating run of the new krill oil extraction process.

Galena Biopharma (Nasdaq:GALE),
a biopharmaceutical company developing and commercializing innovative, targeted oncology treatments that address major unmet medical needs to advance cancer care, today announced it has acquired Mills Pharmaceuticals, which has the worldwide rights to GALE-401 (Anagrelide CR), a patented, controlled release formulation of anagrelide.

Galectin Therapeutics Inc. (Nasdaq:GALT)
, the leading developer of therapeutics that target galectin proteins to treat fibrosis and cancer, today announced that patient enrollment in the first cohort of a Phase 1 trial of GR-MD-02 is now complete.

Hemispherx Biopharma (NYSE MKT:HEB)
, today announced it will make a presentation at the Biotech Showcase held in conjunction with the 32nd JP Morgan Healthcare Conference in San Francisco, California.

Idenix Pharmaceuticals, Inc. (Nasdaq:IDIX)
, a biopharmaceutical company engaged in the discovery and development of drugs for the treatment of human viral diseases, today announced interim data from the Company's ongoing phase II 12-week HELIX-1 clinical trial evaluating an all-oral, direct-acting antiviral (DAA) HCV combination regimen of samatasvir (IDX719), Idenix's once-daily pan-genotypic NS5A inhibitor, and simeprevir (TMC435), a once-daily protease inhibitor jointly developed by Janssen R&D Ireland and Medivir AB, plus ribavirin.

Integra LifeSciences Holdings Corporation (Nasdaq:IART)
will release its fourth quarter 2013 financial results on Tuesday, February 25, 2014. The company has scheduled a conference call for 8:30 AM ET the same day to discuss the results.

Intelligent Living Inc. (OTCQB: ILIV)
announced the immediate availability of its new intelligent wellness app, "Quit Smoking Now!™", which is now available for download to users of Android smartphones via Google Play Store at  

Intuitive Surgical, Inc. (Nasdaq:ISRG)
today reported that equity awards approved by the Compensation Committee of the Board of Directors, which consists entirely of Independent Directors, were made to 15 new employees. Pursuant to NASDAQ Marketplace Rule 5635(c)(4), the equity awards were granted under the Intuitive Surgical, Inc. 2009 Commencement Incentive Plan, which the Board of Directors of Intuitive Surgical, Inc. adopted for the granting of equity awards to new employees.

Merrimack Pharmaceuticals, Inc. (Nasdaq:MACK)
today announced that preclinical data on an analysis of HER2, HER3 and heregulin levels in gastric and gastroesophageal junction (GEJ) tumors will be presented at the American Society of Clinical Oncology 2014 Gastrointestinal Cancers Symposium (ASCO GI), January 16-18, 2014 at the Moscone West Building in San Francisco.

NanoString Technologies, Inc. (Nasdaq:NSTG)
, a provider of life science tools for translational research and molecular diagnostic products, announced today preliminary unaudited revenue for the fourth quarter and full year ended December 31, 2013.

Natural Health Trends Corp. (OTCQB:NHTC)
estimates revenue for the quarter ended December 31, 2013 to be $19.1 million.

NeoStem, Inc. (Nasdaq:NBS)
, a leader in the emerging cellular therapy industry, and its subsidiary, Progenitor Cell Therapy, LLC, an internationally recognized contract development and manufacturing organization, announced today that NeoStem and PCT will present at five sessions at the Phacilitate Cell & Gene Therapy Forum 2014 in Washington DC.

Novavax, Inc. (Nasdaq:NVAX)
announced a number of promotions within its clinical operations group in preparation for expanded clinical trial activities in 2014 and 2015.

Novavax, Inc. (Nasdaq:NVAX)
announced the continuation of their partnership with PATH to develop Novavax' RSV (respiratory syncytial virus) vaccine for maternal immunization.

Neuralstem, Inc. (NYSE MKT: CUR)
announced that President and CEO Richard Garr will present at the sixth annual Biotech Showcase in San Francisco on Tuesday, January 14, 3:30 p.m. PT, in Room Powell (Track D).

Nymox Pharmaceutical Corporation (Nasdaq:NYMX)
today announced that recruitment is being closed for the Company's Phase 2 study of NX-1207 for low risk localized prostate cancer (NX03-0040) and that enrollment will be formally completed shortly. Initial top line study results are expected to be available approximately 6-8 weeks after completion of enrollment.

OncoMed Pharmaceuticals, Inc. (Nasdaq:OMED)
, a clinical-stage company developing novel therapeutics that target cancer stem cells (CSCs), or tumor-initiating cells, today announced that it has started a multi-center Phase 1b clinical trial of its first-in-class Wnt-pathway-targeting decoy receptor OMP-54F28 (Fzd8-Fc) with nab-paclitaxel (Abraxane®) and gemcitabine in pancreatic cancer.

Palatin Technologies, Inc. (NYSE MKT: PTN)
announced today that in view of the unusual market activity in common stock of the Company on Monday, January 13, 2014, the NYSE MKT (the "Exchange") has contacted the Company in accordance with the Exchange's usual practice.

Prosensa Holding N.V. (NASDAQ: RNA)
, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet medical need, will be providing updates to both the financial and patient communities regarding drisapersen.

Protalix BioTherapeutics, Inc. (NYSE MKT:PLX) (TASE:PLX)
, announced today that Dr. David Aviezer, the Company's President and Chief Executive Officer, will discuss the Company's corporate objectives and key milestones in a presentation at the 32nd Annual J.P. Morgan Healthcare Conference on Thursday, January 16, 2014 at 12:00 PM, Pacific Time.

Prosensa Holding N.V. (Nasdaq:RNA)
and GlaxoSmithKline (GSK) today announced that Prosensa has regained all rights from GSK to drisapersen and will retain rights to all other programs for the treatment of Duchenne Muscular Dystrophy (DMD).

Portola Pharmaceuticals (Nasdaq:PTLA)
today announced that it has entered into a second clinical collaboration agreement with Bristol-Myers Squibb Company (NYSE:BMY) and Pfizer Inc. (NYSE:PFE) to study Portola's investigational Factor Xa inhibitor reversal agent, andexanet alfa* (PRT4445), with the oral Factor Xa inhibitor Eliquis® (apixaban).

QIAGEN N.V. (Nasdaq:QGEN) (Frankfurt:QIA)
today reconfirmed plans to release its report on results for the fourth quarter and full-year of 2013 on Wednesday, January 29, at 22:00 Central European Time (CET) / 16:00 Eastern Time (ET).

Relypsa, Inc. (Nasdaq:RLYP)
, a clinical-stage biopharmaceutical company, today provided a corporate update including new data on the modification of renin-angiotensin-aldosterone system inhibiting (RAASi) therapies from Part B of its two-part Phase 3 trial evaluating patiromer as a treatment for hyperkalemia; a new agreement with Lanxess to ensure the commercial manufacture and supply of the active ingredient of patiromer; and an update with respect to its senior management team.

REVA Medical, Inc. (ASX:RVA)
is pleased to announce that it has completed enrollment in the clinical trial of the ReZolve2 drug-eluting bioresorbable scaffold. A total of 112 patients have been enrolled in the trial to provide the data needed to apply for European CE Marking.

Sabra Health Care REIT, Inc. (Nasdaq:SBRA)
announced today that Rick Matros, the company's Chairman and Chief Executive Officer, Harold Andrews, the Company's Chief Financial Officer, and Talya Nevo-Hacohen, the Company's Chief Investment Officer, will be participating in the 2014 Stifel Senior Housing and Healthcare Real Estate Conference Tuesday, January 28, 2014 in Miami, Florida.

StemCells, Inc. (Nasdaq:STEM)
announced today that its technology was featured January 10 in a television segment which aired at 10 PM on CTV News Channel and 11 PM on the CTV National News.

Tengion, Inc. (OTCQB:TNGN)
, a leader in regenerative medicine, today announced continued clinical progress in its two lead programs, the Neo-Kidney Augment and the Neo-Urinary Conduit.

Tekmira Pharmaceuticals Corporation (Nasdaq:TKMR) (TSX:TKM)
, a leading developer of RNA interference (RNAi) therapeutics, announced today that it has signed an Option Agreement with Monsanto, pursuant to which Monsanto may obtain a license to use Tekmira's proprietary delivery technology.

TG Therapeutics, Inc. (Nasdaq:TGTX)
today announced that Michael Weiss, the Company's Interim Chief Executive Officer, will be presenting at the 32nd Annual J.P. Morgan Healthcare Conference, being held at the Westin St. Francis Hotel in San Francisco.

Tornier N.V. (Nasdaq:TRNX)
, a global medical device company focused on providing surgical solutions to orthopaedic extremity specialists, announced today preliminary fourth quarter and full year 2013 revenue results.

Vascular Solutions, Inc. (Nasdaq:VASC)
announced today that the U.S. Court of Appeals for the Federal Circuit has refused to grant an interim stay during appeal of the injunction issued by the District Court for the District of Minnesota against Boston Scientific's Guidezilla guide extension catheter, thereby causing the injunction to take effect starting January 13, 2014., Inc. (Nasdaq:VITC)
, a leading online retailer of health and wellness products, announced today that it has been ranked #2 in customer satisfaction among Internet Retailer's top 100 U.S. websites during the 2013 holiday season, surpassed only by, according to the ForeSee Experience Index (FXI): 2013 U.S. Retail Edition.

ZELTIQ®, (Nasdaq:ZLTQ)
a medical technology company focused on developing and commercializing products utilizing its proprietary controlled-cooling technology platform, announced today preliminary unaudited revenue for the fourth quarter and full year 2013.

Zogenix, Inc. (Nasdaq:ZGNX)
, a pharmaceutical company developing and commercializing products for the treatment of central nervous system disorders and pain, announced today preliminary unaudited gross product sales on sales of SUMAVEL® DosePro® for the quarter ended December 31, 2013.

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