Access Pharma Announces European License Agreement With Norgine; Alnylam Receives Orphan Drug Designations for ALN-AT3 Print E-mail
By Staff and Wire Reports   
Thursday, 07 August 2014 19:03
Below is a look at some of the headlines for companies that made news in the healthcare sector on August 7, 2014.
, an emerging biopharmaceutical company, announced it had entered into an exclusive license agreement with Norgine B.V., a leading independent European specialty pharmaceutical company,  for the commercialization of MuGard in Europe.  Under the terms of the license agreement, Access will receive up to $10 million in milestone payments and an escalating double digit royalty on the net sales of the oral mucositis product, MuGard, in the licensed territories.  Norgine will develop, manufacture, and commercialize MuGard in the European Union, Switzerland, Norway, Iceland and Lichtenstein. Norgine anticipates launching MuGard in 2015.

Commenting on the news, Jeffrey Davis, CEO of Access Pharmaceuticals, Inc stated, "Access is excited to announce this partnership with Norgine, a leading European specialty company, as it further validates our global MuGard commercial strategy." He added, "With product offerings in complementary therapeutic areas and an established presence in the cancer supportive care space, Norgine is an ideal partner for us and we believe this partnership will drive commercial adoption in Europe and facilitate access of a proven treatment option to help protect patients from oral mucositis, a debilitating side effect."

"This new partnership with Access Pharmaceuticals Inc. represents an excellent opportunity to ensure that cancer patients affected by oral mucositis can access MuGard, to help manage this condition for which there are limited effective treatment options," said Peter Stein, CEO of Norgine.  He continued, "This collaboration is further evidence of Norgine's commitment to acquire specialty pharmaceutical products to strengthen its portfolio."


Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY)
, a leading RNAi therapeutics company, announced today that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted Orphan Drug Designations for ALN-AT3 as an orphan medicinal product for the treatment of hemophilia A and hemophilia B. Alnylam is developing ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT), for the treatment of hemophilia and other Rare Bleeding Disorders (RBD).

“We are very pleased to have received Orphan Drug Designations from the EMA COMP for ALN-AT3, a key program in our ‘Alnylam 5x15’ product development and commercialization strategy. We believe that our subcutaneously delivered RNAi therapeutic represents an innovative approach for the management of hemophilia and has great potential to make a meaningful impact in the treatment of this often debilitating bleeding disorder,” said Saraswathy (Sara) Nochur, Ph.D., Senior Vice President, Regulatory Affairs and Quality Assurance at Alnylam. “Having announced positive top-line results in adult healthy volunteers earlier this year, we look forward to presenting initial Phase 1 results, including data in hemophilia subjects, later in the year.”

ALN-AT3 is currently being investigated in a multinational Phase 1 trial. At the World Federation of Hemophilia 2014 World Congress held in May, Alnylam presented positive top-line data from Part A of the study performed in adult healthy volunteers. Initial results from the sole dose cohort enrolled (n=4; 3:1, drug:placebo) showed that a single, low subcutaneous ALN-AT3 dose of 0.03 mg/kg resulted in an up to 28-32% knockdown of AT at nadir (p < 0.01 by ANOVA, relative to placebo). This resulted in a statistically significant (p < 0.01) increase in peak thrombin generation, that was temporally associated and consistent with the degree of AT knockdown. ALN-AT3 was found to be well tolerated with no significant adverse events reported. With these data and in light of a dose escalation stopping rule at a 40% level of AT knockdown, the company has transitioned to Part B of the study – an open-label, multi-dose, dose-escalation study enrolling up to 18 people with moderate to severe hemophilia A or B. The primary objective of this part of the study is to evaluate the safety and tolerability of multiple doses, specifically three weekly doses, of subcutaneously administered ALN-AT3 in hemophilia subjects. Secondary objectives include assessment of clinical activity as determined by knockdown of circulating AT levels and increase in thrombin generation at pharmacologic doses of ALN-AT3. In this part of the study, dose escalation will be allowed to proceed beyond the 40% AT knockdown level. The company plans to present initial clinical results, including results in hemophilia subjects from Part B of the study, by the end of the year.

Orphan Drug Designation by the European Commission provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, Orphan Drug Designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to centralized marketing authorization.

Also Thursday:

Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN)
today reported financial results for the three and six months ended June 30, 2014 and provided an update on the Company's portfolio of clinical stage compounds being developed for the treatment of the hepatitis C virus (HCV).

Accuray Incorporated (Nasdaq:ARAY)
announced today that it will report results for its fourth quarter and fiscal year ended June 30, 2014 on Thursday, August 21, 2014 after the market closes.

Amicus Therapeutics (Nasdaq:FOLD)
, a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, today announced financial results for the second quarter ended June 30, 2014.

Arrhythmia Research Technology, Inc. (NYSE:HRT)
, operating through its wholly-owned subsidiary, Micron Products, Inc., is a diversified manufacturer of proprietary, highly-engineered technologies, such as machined orthopedic implants, injection molded medical disposables and sensors for bio-monitoring electrodes.

Auspex Pharmaceuticals, Inc. (Nasdaq:ASPX), a late clinical stage biopharmaceutical company focused on developing and commercializing novel medicines for the treatment of orphan diseases, today announced its financial results for the three and six months ended June 30, 2014.

Cara Therapeutics, Inc. (Nasdaq:CARA)
, a biopharmaceutical company focused on developing and commercializing new chemical entities designed to alleviate pain and pruritus by selectively targeting kappa opioid receptors, today announced financial results for the second quarter ended June 30, 2014.

Celator Pharmaceuticals, Inc. (Nasdaq:CPXX)
, a pharmaceutical company developing new and more effective therapies to treat cancer, today reported business highlights and financial results for the second quarter ended June 30, 2014.

Ekso Bionics Holdings, Inc. (OTCQB:EKSO)
, a robotic exoskeleton company, today reported financial results for the quarter ended June 30, 2014, as well as recent highlights.

The Ensign Group, Inc. (Nasdaq:ENSG)
, the parent company of the Ensign™ group of skilled nursing, rehabilitative care services, assisted and independent living, home health, hospice care and urgent care companies, today reported operating results for the second quarter of 2014.

Endocyte, Inc. (Nasdaq:ECYT)
, a leader in developing targeted small molecule drug conjugates (SMDCs) and companion imaging agents for personalized therapy, today announced that three posters will be presented by Endocyte scientists at the 248th American Chemical Society (ACS) National Meeting & Exposition being held in San Francisco, August 10-14, 2014.

Flexion Therapeutics, Inc. (Nasdaq:FLXN)
today announced key operational highlights and financial results for the second quarter ended June 30, 2014, as well as more recent accomplishments.

Galectin Therapeutics Inc. (Nasdaq:GALT)
, the leading developer of therapeutics that target galectin proteins to treat fibrosis and cancer, today reported its financial results for the three and six months ended June 30, 2014.

Hyperion Therapeutics, Inc. (Nasdaq:HPTX)
today reported net revenue of $37.1 million during the second quarter of 2014 from the sales of its two products for the treatment of urea cycle disorders, RAVICTI® (glycerol phenylbutyrate) Oral Liquid and BUPHENYL® (sodium phenylbutyrate) Tablets and Powder.

KYTHERA Biopharmaceuticals, Inc.(Nasdaq:KYTH)
today reported financial results for its second quarter 2014 and provided an update on recent accomplishments.

Minerva Neurosciences, Inc. (Nasdaq:NERV)
, a clinical stage biopharmaceutical company focused on the development and commercialization of a portfolio of product candidates to treat patients suffering from neuropsychiatric diseases, today reported business highlights and financial results for the second quarter ended June 30, 2014.

MRI Interventions, Inc. (OTCQB:MRIC)
, a commercial stage medical device company focused on creating innovative platforms for performing the next generation of minimally invasive surgical procedures in the brain and heart, announced today its financial results for the quarter ended June 30, 2014.

NeoStem, Inc. (Nasdaq:NBS)
, a leader in the emerging cellular therapy industry, today provided an update on the progress of its business and reported second quarter 2014 financial results.

Novavax, Inc. (Nasdaq:NVAX)
, a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of recombinant nanoparticle vaccines and adjuvants, announced today that Barclay "Buck" Phillips, SVP, Chief Financial Officer and Treasurer, will present at the Wedbush Securities Life Sciences Management Access Conference to be held on Wednesday, August 13, 2014 at 3:40 pm ET in New York City at the Le Parker Meridien hotel.

Oculus Innovative Sciences, Inc. (Nasdaq:OCLS)
today announced financial results for the first quarter of fiscal year 2015, ended June 30, 2014.

Receptos, Inc. (Nasdaq:RCPT)
, a biopharmaceutical company developing therapeutic candidates for the treatment of immune and metabolic diseases, will announce financial results for the second quarter ended June 30, 2014 on Tuesday, August 12 before the markets open.

Relypsa, Inc. (Nasdaq:RLYP)
, a biopharmaceutical company, today announced that John Orwin, Chief Executive Officer, will present a corporate overview at the Wedbush PacGrow Lifesciences Conference on August 13, 2014 at 9:10 a.m. ET.

RXi Pharmaceutical Corporation (Nasdaq:RXII)
, a biotechnology company focused on discovering, developing and commercializing innovative therapies addressing major unmet medical needs using RNA-targeted technologies, will visit the NASDAQ MarketSite in Times Square.

TearLab Corporation (Nasdaq:TEAR) (TSX:TLB)
today reported its consolidated financial results for the second quarter ended June 30, 2014. All dollar amounts are expressed in U.S. currency and results are reported in accordance with United States generally accepted accounting principles.

Tornier N.V. (Nasdaq:TRNX)
, a global medical device company focused on providing surgical solutions to orthopaedic extremity specialists, reported today its financial results for the second quarter ended June 29, 2014.

Tekmira Pharmaceuticals Corporation (Nasdaq:TKMR) (TSX:TKM)
, a leading developer of RNA interference (RNAi) therapeutics, today announced that the U.S. Food & Drug Administration (FDA) has verbally confirmed they have modified the full clinical hold placed on the TKM-Ebola Investigational New Drug Application (IND) to a partial clinical hold.

XOMA Corporation (Nasdaq:XOMA)
, a leader in the discovery and development of therapeutic antibodies, today reported its operational highlights and financial results for the quarter ended June 30, 2014.

ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP)
today announced financial results for the second quarter ended June 30, 2014, and provided an update on the company's product development activities.

"Featured Content" profiles are meant to provide awareness of these companies to investors in the small-cap and growth equity community and should not in any way come across as a recommendation to buy, sell or hold these securities. BiomedReports is not paid or compensated by newswires to disseminate or report news and developments about publicly traded companies, but may from time to time receive compensation for advertising, data, analytics and investor relation services from various entities and firms. Full disclosures should be read in the 'About Us Section'.

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