Synageva Completes Target Enrollment in Phase 3 ARISE; Biogen Receives Notification from FDA of PDUFA Date Extension for ALPROLIX™ Print E-mail
By Staff and Wire Reports   
Monday, 02 December 2013 19:59
Below is a look at some of the headlines for companies that made news in the healthcare sector on December 2, 2013.

Synageva BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, announced it met the enrollment target of 50 patients in ARISE (Acid Lipase Replacement Investigating Safety and Efficacy), a global Phase 3 trial with sebelipase alfa in children and adults with lysosomal acid lipase deficiency (LAL Deficiency). To accommodate individuals still in the screening process, total enrollment will exceed the planned number of 50 patients, with the final patient expected to be randomized by the end of this year. As a result of today's enrollment update, the company expects to report top-line results from the ARISE trial during the second half of 2014.

Phase 3 ARISE trial with sebelipase alfa in LAL Deficiency - The ARISE trial is a randomized, double-blind, placebo-controlled study of sebelipase alfa in children and adults with LAL Deficiency and is designed to assess the effects of sebelipase alfa on a broad range of abnormalities associated with the disease. Patients enrolled in the trial are randomized to infusions of sebelipase alfa (1 mg/kg, every other week) or placebo for the 20-week, double-blind treatment period and then allowed to enter into a long-term, open-label extension period. The efficacy and safety results from the double-blind treatment period are expected to support global submissions for product registration.

Sebelipase alfa for LAL Deficiency - LAL Deficiency is a rare autosomal recessive lysosomal storage disease (LSD) caused by a marked decrease in LAL enzyme activity. LAL Deficiency presenting in children and adults, historically called Cholesteryl Ester Storage Disease (CESD), is an underappreciated cause of cirrhosis and accelerated atherosclerosis. These complications are due to the buildup of fatty material in the liver, blood vessel walls and other tissues as a result of the decreased LAL enzyme activity. Infants presenting with LAL Deficiency, historically called Wolman disease, show very rapid progression with death, usually in the first six months of life. Affected infants develop severe malabsorption, growth failure and liver complications.

Sebelipase alfa (SBC-102) is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL Deficiency. Synageva is evaluating sebelipase alfa in global Phase 3 clinical trials in infants, children and adults with LAL Deficiency. Sebelipase alfa has been granted orphan designation by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Japanese Ministry of Health, Labour and Welfare. Additionally, sebelipase alfa received fast track designation by the FDA, and Breakthrough Therapy designation by the FDA for LAL Deficiency presenting in infants.

SBC-103 for MPS IIIB - The mucopolysaccharidoses (MPS) consist of a group of rare LSDs caused by a deficiency of enzymes needed to break down complex sugars called glycosaminoglycans. The MPS III syndromes (also known as Sanfilippo syndromes) share complications with other MPS diseases but represent a clinically distinct subset with marked central nervous system degeneration. Mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome) is caused by a marked decrease in alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity which leads to the buildup of abnormal amounts of heparan sulfates (HS) in the brain and other organs. The accumulation of abnormal HS, particularly in the central nervous system, leads to severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility, and premature death.

SBC-103 is a recombinant form of the human NAGLU enzyme being developed by Synageva as an enzyme replacement therapy for MPS IIIB. Using various dosing approaches, SBC-103 reduced HS substrate storage in the brain, liver and kidney in an MPS IIIB animal model. SBC-103 has been granted orphan designation by the FDA and the EMA.

Synageva's additional pipeline programs and manufacturing platform - Synageva's additional pipeline programs include other proteins targeting rare diseases at various stages of preclinical development. These diseases are characterized by significant morbidity and mortality and these programs are selected based on scientific rationale, high unmet medical need, potential to impact disease course and strategic alignment with the company's corporate focus. In addition to these novel pipeline programs, Synageva is leveraging its manufacturing platform to develop improved biologic therapies for diseases with high unmet medical need.

Synageva's proprietary manufacturing platform utilizes technology to produce drug product with consistent characteristics that enable robustness and flexibility during scale-up. In addition, the platform can provide favorable structural properties for bio-distribution and cell targeting in comparison to glycoproteins produced from other sources.

Synageva routinely posts information that may be important to investors in the “Investor Relations” section of the company's web site at www.synageva.com. Synageva encourages investors and potential investors to consult this web site regularly for important information about the company.



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Biogen Idec (NASDAQ: BIIB)
announced the U.S. FDA has extended the initial Prescription Drug User Fee Act (PDUFA) date for its review of the Biologics License Application (BLA) for ALPROLIX™, the company's investigational long-lasting recombinant factor IX Fc fusion protein candidate for hemophilia B. The PDUFA date has been extended by three months, which is the standard extension period.

In response to a request from FDA, Biogen Idec submitted additional information related to the validation of a manufacturing step for ALPROLIX. Due to the timing of this submission, the FDA extended the PDUFA date to allow additional time for review of the marketing application.



Also Monday:




22nd Century Group, Inc. (OTCBB: XXII)
today announced that Joseph Pandolfino, Chief Executive Officer, and Henry Sicignano III, President, will present at RetailInvestorConferences.com.

Advanced Medical Isotope Corporation (OTCBB:ADMD)
, a company engaged in the development, production and distribution of medical isotopes, today announced that its Board of Directors has approved a three year business strategy focused on transitioning to full operations in 2014, following the anticipated receipt of FDA clearance for its patented brachytherapy cancer product, Yttrium-90 RadioGel™.

AeroGrow International, Inc. (OTCQB: AERO)
, which sells the AeroGarden® line of high-output, soil-free indoor gardens, seed kits, plant nutrients and grow lights, announced that the all new Miracle-Gro AeroGarden Extra Indoor Garden will be launched exclusively on one of the world's leading television shopping networks on Cyber Monday, December 2, 2013.

Alcobra Ltd. (Nasdaq:ADHD)
, an emerging biopharmaceutical company primarily focused on the development and commercialization of its proprietary drug candidate, MG01CI (Metadoxine extended-release), to treat cognitive dysfunctions, such as ADHD and Fragile X Syndrome, announced today that an overview of the Company's business strategy will be given by Alcobra's Chief Executive Officer, Dr. Yaron Daniely, at the 25th Annual Piper Jaffray Healthcare Conference, being held at the New York Palace Hotel in New York City on December 3-4, 2013.

The Alkaline Water Company Inc. (OTCBB: WTER)
, developers of an innovative state of the art proprietary electrolysis beverage process, packaged and sold in 3 liter and 1 gallon sizes under the trade name Alkaline88, is very pleased to announce sales have commenced to Texas based Brookshire Grocery Company.

Amarin Corporation plc (Nasdaq:AMRN)
, a biopharmaceutical company focused on the commercialization and development of therapeutics to improve cardiovascular health, announced today that the United States Patent and Trademark Office (USPTO) has published notification of a Notices of Allowance for Amarin's U.S. Patent Applications Serial Numbers 13/685,281, and 13/685,291, each titled "Stable Pharmaceutical Composition and Methods of Using Same."

Astro-Med, Inc. (NASDAQ: ALOT)
, a manufacturer of specialty high tech printers and data acquisition systems, announces that its CEO, Everett V. Pizzuti, will retire effective January 31, 2014, the end of the Company's current fiscal year.

Bioheart, Inc. (OTCQB: BHRT)
, a biotechnology company focused on the discovery, development and commercialization of autologous cell therapies, announced that it has entered into an agreement with StemMD.

BIOLASE, Inc. (NASDAQ: BIOL)
, the world's leading manufacturer and distributor of dental lasers, and a pioneer in laser surgery in other medical specialties, today announced that it has introduced its GALAXY BioMill CAD/CAM System at the Greater New York Dental Meeting, the largest dental congress and exposition in the United States.

Celldex Therapeutics, Inc. (Nasdaq:CLDX)
today announced that it has launched a randomized study (METRIC) of Glembatumumab vedotin (CDX-011) in patients with metastatic triple negative breast cancers that over-express glycoprotein NMB (gpNMB).

Cerebain Biotech Corp.
, a subsidiary of Discount Dental Materials, Inc. (OTCBB: DDOO) announced today that it has engaged Superior Inc. to represent the Company in Poland for the purpose of Clinical Testing of the Company's Medical Device for the treatment of Alzheimer's Disease utilizing the Omentum.

Chembio Diagnostics, Inc. (Nasdaq:CEMI)
, a leader in point-of-care diagnostic tests for infectious diseases, has completed its Clinical Laboratory Improvement Amendment waiver studies for its DPP® HIV 1/2 Assay.

Conatus Pharmaceuticals Inc. (Nasdaq:CNAT)
, a biotechnology company focused on the development and commercialization of novel medicines to treat liver disease, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Conatus' drug candidate emricasan for the treatment of liver transplant recipients with reestablished fibrosis to delay the progression to cirrhosis and end-stage liver disease.

Guar Global Ltd. (OTCQB: GGBL)
, a company aiming to implement innovative strategies and methods to increase crop yields of guar, an organic gelling agent used extensively in hydraulic fracturing by the oil and gas sector, announced today that its Chief Technologist, Dr. Subodh K. Singh, will be presenting at the first ever Guar International Conference 2013 held under the auspices of the National Commodity & Derivatives Exchange (NCDEX) at the Marriot Hotel, Jaipur, India, December 6-7th, 2013.

Hemispherx Biopharma, Inc. (NYSE MKT:HEB)
today announced that it is supporting the University of Pittsburgh's National Institutes of Health funded study (grant 1PO1CA132714) currently underway as part of the University's Chemokine Modulation Research initiative which includes Ampligen® as an adjuvant.

Immunomedics, Inc. (Nasdaq:IMMU)
, a biopharmaceutical company primarily focused on the development of monoclonal antibody-based products for the targeted treatment of cancer, autoimmune and other serious diseases, today announced that five posters on its product candidates will be presented at the 55th American Society of Hematology Annual Meeting, scheduled for December 7 – 10, 2013 in New Orleans, LA.

Karyopharm Therapeutics Inc. (Nasdaq:KPTI)
, a clinical-stage pharmaceutical company focused on the discovery and development of novel first-in-class drugs directed against nuclear transport targets for the treatment of cancer and other major diseases, announced today that Karyopharm will host an investor reception and webcast at the 55th American Society of Hematology (ASH) Annual Meeting and Exposition, which is taking place December 7-10, 2013 in New Orleans.

Merge Healthcare Incorporated (Nasdaq:MRGE)
, a leading provider of clinical systems and innovations that seek to transform healthcare, today announced the release ofiConnect® Network, an advanced imaging network that allows hospitals, providers and imaging centers to exchange imaging information securely and electronically.

NeoStem, Inc. (Nasdaq:NBS)
, a leader in the emerging cellular therapy industry, announced today that the Company and its subsidiary, Progenitor Cell Therapy, LLC, will present at multiple upcoming conferences in December.

Organovo Holdings, Inc. (NYSE MKT: ONVO)
the San Diego-based regenerative medicine company focused on using its breakthrough NovoGen 3D human tissue printing technology to create tissue on demand for medical research and therapeutic applications, today announced that Keith Murphy, CEO, will present at RetailInvestorConferences.com.

Oxford Immunotec Global PLC (Nasdaq:OXFD)
announced today Herman ("Herm") Rosenman has been appointed as a member of the Company's Board of Directors and will serve as Chairman of the Board's Audit Committee. Mr. Rosenman served most recently as Senior Vice President, Finance and Chief Financial Officer of Gen-Probe Incorporated, a publicly traded molecular diagnostics company, which was acquired by Hologic, Inc. in 2012.

Oxford Immunotec Global PLC (Nasdaq:OXFD)
today announced the closing of its initial public offering of 6,164,000 ordinary shares at an initial public offering price of $12.00 per share, which includes the exercise in full by the underwriters of their option to purchase up to 804,000 additional ordinary shares.

Palatin Technologies, Inc. (NYSE MKT: PTN)
announced today that Dr. Carl Spana, the Company's chief executive officer, will participate in a "Fireside Chat" at the 25thAnnual Piper Jaffray Healthcare Conference at 4:00 p.m. ET on December 4, 2013.

PDL BioPharma, Inc. (NASDAQ: PDLI)
today announced that John P. McLaughlin, the company's president and chief executive officer, is scheduled to present at the 25th Annual Piper Jaffray Healthcare Conference in New York tomorrow, Tuesday, December 3, 2013 at 3:00 p.m. Eastern Time.

Rosetta Genomics Ltd. (NASDAQ: ROSG), a leading developer and provider of microRNA-based molecular diagnostics, announces the posting of the following Letter to Shareholders to the Investors section of the Company's website at www.rosettagenomics.com.

Stevia First Corp. (OTCQB: STVF)
, an early-stage agribusiness based in California's Central Valley growing region and focused on the industrial scale production of stevia, the all-natural zero-calorie sweetener that is rapidly transforming the food and beverage industry, announced today that Robert Brooke, President and Chief Executive Officer will present at the 6th Annual LD MICRO Conference at the Luxe Sunset Boulevard Hotel in Los Angeles on Tuesday, December 3rd, 2013, at 3:00 PM PST.

Where Food Comes From, Inc. (OTCQB: WFCF)
, the trusted resource for third party verification of food production practices, today announced that chairman and CEO John Saunders will present at the LD Micro Conference on Tuesday, December 3, 2013.

ZELTIQ®, (Nasdaq:ZLTQ)
a medical technology company focused on developing and commercializing products utilizing its proprietary controlled-cooling technology platform, today announced that Patrick F. Williams, Senior Vice President and Chief Financial Officer, is scheduled to present at the J.P. Morgan SMid Cap Conference at The Westin River North in Chicago.

Z Trim Holdings, Inc. (OTC: ZTHO)
, a bio-technology company providing value-added ingredients to a variety of industries, today announced the addition of Seth Cox as an independent consultant, to assist the technical sales staff in growing the company's successful food ingredient business.



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