|Delcath Receives Orphan Drug Designation From FDA For Melphalan; Exelixis Up 50% After Meteroric METEOR Results|
|By Mary Davila|
|Monday, 20 July 2015 19:20|
Below is a look at some of the headlines for companies that made news in the healthcare sector on July 20, 2015.
Delcath Systems, Inc. (NASDAQ: DCTH), a specialty pharmaceutical and medical device company focused on oncology with an emphasis on the treatment of primary and metastatic liver cancers, announced today that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has granted Orphan Drug Designation for melphalan for the treatment of cholangiocarcinoma. The OOPD is tasked with evaluating the scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products.
Orphan drug designation provides certain exclusivity benefits, tax credits for certain research and a waiver of the New Drug Application user fee. Cholangiocarcinoma is recognized by the FDA as an orphan disease, usually defined as a condition that affects fewer than 200,000 people nationwide.
Intrahepatic cholangiocarcinoma (ICC), a sub-category of cholangiocarcinoma, is a tumor in the bile duct that arises within the liver. It is the second most common primary liver tumor and represents approximately 15% of new HCC cases diagnosed annually. Surgical resection, the standard of care, is not possible for an estimated 80% to 90% of patients diagnosed with ICC.
The Company recently announced the expansion of its global Phase 2 clinical study in primary liver cancer (HCC) to include an ICC cohort, which is investigating the safety and efficacy of Melphalan/HDS treatment in patients with unresectable ICC confined to the liver. The study is being conducted at the same hospitals in Europe participating in the Company's Phase 2 HCC trial, and is expected to enroll 11 patients. The ICC cohort will evaluate tumor response (objective response rate) as measured by modified Response Evaluation Criteria in Solid Tumor (mRECIST), and will assess progression-free survival and safety. Additional analyses will be conducted to characterize the systemic exposure of melphalan administered by Melphalan/HDS, as well as to assess patient-reported clinical outcomes, or quality-of-life.
"We are pleased with the receipt of orphan drug designation for melphalan in the treatment of patients with cholangiocarcinoma as it is a key milestone that supports our broader regulatory and development strategy for our Melphalan/Hepatic Delivery System (Melphalan/HDS) as a therapy for primary and metastatic liver cancers," said Jennifer Simpson, Ph.D., M.S.N., C.R.N.P., President and Chief Executive Officer of Delcath. "ICC is a disease of significant unmet medical need and our Melphalan/HDS treatment may offer clinical benefit for ICC patients who face limited treatment options."
Shares of Exelixis, Inc. (NASDAQ: EXEL) were surging by more than 50 percent Monday after the company announced positive top-line results from METEOR in which the study met the primary endpoint of significantly improving progress-free survival.
Exelixis's positive top-line results stem from a phase 3 pivotal trial comparing cabozantinib to everolimus in 658 patients with metastatic renal cell carcinoma (RCC) who have experienced disease progression following treatment with a VEGF receptor tyrosine kinase inhibitor (TKI).
The trial met its primary endpoint of showing a statistically significant increase in progress-free survival in the first 375 randomized patients. Detailed results of the trial will be presented at an upcoming medical conference.
Cabozantinib has already received a Fast Track designation by the U.S. Food and Drug Administration FDA, while Exlixis plants to complete regulatory filings in the U.S. and European Union in early 2016 following the successful outcome of its study.
"The positive top-line results from METEOR represent strong progress for the kidney cancer community and for Exelixis, bringing us one step closer to our shared goal of delivering a new and meaningfully differentiated therapeutic option for the many metastatic RCC patients in need," said Michael M. Morrissey, Ph.D., the company's president and chief executive officer.
Adaptimmune Therapeutics plc (Nasdaq:ADAP), a clinical stage biopharmaceutical company focused on the use of T-cell therapy to treat cancer, today announced that data from its Phase I/II study of its affinity enhanced T-cell receptor (TCR) therapeutic targeting the NY-ESO-1 cancer antigen in patients with multiple myeloma has been published in Nature Medicine.
Amarantus BioScience Holdings, Inc. (OTCQX:AMBS), a biotechnology company developing therapeutic and diagnostic product candidates in orphan indications and neurology, announced that the first patient has been dosed in the Phase 2b clinical study of the Company's lead neurology therapeutic candidate eltoprazine for the treatment of Parkinson's disease levodopa-induced dyskinesia (PD-LID).
Amyris, Inc. (Nasdaq:AMRS), the industrial bioscience company, today announced the official launch of its Muck Daddy™ High Performance Hand Cleaner by teaming up with former Chicago Bears linebacker Brian Urlacher to celebrate the hard-working professionals who keep America's motors running.
Avinger, Inc., (NASDAQ:AVGR) a developer and manufacturer of image-guided, catheter-based systems for the treatment of peripheral arterial disease (PAD) and pioneer of the lumivascular approach to treating vascular disease, today announced that it will release its financial results for the second quarter of 2015 after the close of trading on Tuesday, July 28, 2015.
BioRestorative Therapies, Inc. (OTCQB:BRTX), a life sciences company focused on stem cell-based therapies, today announced that the Company has begun to evaluate encapsulation technology for potential use as a cell delivery system for the Company's metabolic program.
Connecture, Inc. (NASDAQ:CNXR), a provider of web-based information systems used to create health insurance marketplaces, announced that Christopher Neuharth, vice president of product design and user experience, will be a speaker at the 2015 Private Healthcare Exchanges Conference, held July 23-24 in Chicago, Ill.
Emergent BioSolutions (NYSE:EBS) today announced that it has been awarded a contract by the Biomedical Advanced Research and Development Authority (BARDA) valued at $19.7 million to develop and manufacture cGMP lots of three Ebola monoclonal antibodies in CHO cell lines at a 2000 Liter scale.
Fibrocell Science, Inc., (NASDAQ:FCSC), an autologous cell and gene therapy company focused on developing first-in-class treatments for rare and serious skin and connective tissue diseases with high unmet needs, and Intrexon Corporation (NYSE:XON), a leader in synthetic biology, today announced the submission of an Investigational New Drug (IND) Application with the U.S. Food and Drug Administration for FCX-007, Fibrocell's lead orphan gene-therapy drug candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
ICU Medical, Inc., (Nasdaq:ICUI), a leader in the development, manufacture and sale of innovative medical devices used in infusion therapy, oncology and critical care applications, today announced the time of its second quarter 2015 earnings release conference call.
Integra LifeSciences Holdings Corporation (NASDAQ:IART), today announced that it has completed the acquisition of all of the outstanding shares of TEI Biosciences Inc. and TEI Medical Inc. ("TEI").
Lombard Medical, Inc. (Nasdaq:EVAR), a medical device company focused on Endovascular Aortic Repair (EVAR) of abdominal aortic aneurysms (AAAs), today announced that it will release its 2015 second quarter and six-month financial results after the market closes on Monday, July 27, 2015.
Medigus Ltd. (Nasdaq:MDGS) (TASE:MDGS), a medical device company developing minimally invasive endosurgical tools and a leader in direct visualization technologies, announced today that the National Aeronautics and Space Administration (NASA) has successfully used Medigus' micro ScoutCam™ 1.2, the world's smallest camera, for the first time in space as part of NASA's Visual Inspection Poseable Invertebrate Robot (VIPIR) tool.
OXiGENE, Inc. (Nasdaq:OXGN), a biopharmaceutical company developing novel therapies for treatment of cancer, today announced that Matthew M. Loar has been appointed Chief Financial Officer.
PRA Health Sciences (NASDAQ:PRAH) is looking to hire new talent. To help attract potential employees from around the world, PRA, a leading global contract research organization (CRO), is launching a new Candidate Experience initiative.
Radius Health, Inc. (NASDAQ:RDUS), a science-driven biopharmaceutical company developing new therapeutics for patients with advanced osteoporosis as well as other serious endocrine-mediated diseases including hormone responsive metastatic breast cancer, today announced that Debasish Roychowdhury, M.D., who previously served as Seragon's Acting Chief Medical Officer, has been elected to the company's Board of Directors.
SANUWAVE Health, Inc. (OTCQB:SNWV), a leading shock wave technology company, today announced that Barry Jenkins, Chief Financial Officer, has resigned from the Company to accept a top management position at a leading medical services company.
Sharps Compliance Corp. (NASDAQ:SMED), a leading full-service national provider of comprehensive waste management solutions including medical, pharmaceutical and hazardous, today announced the acquisition of Alpha Bio/Med Services LLC, a route-based pick-up service located in Pennsylvania.
StemCells, Inc. (Nasdaq:STEM), a world leader in the research and development of cell-based therapeutics for the treatment of disorders of the brain, eye and spine, announced today that it has completed transplantation of the first subject in its Radiant Study at the Retina Foundation of the Southwest.
TetraLogic Pharmaceuticals Corporation (NASDAQ:TLOG) a clinical-stage biopharmaceutical company focuses on discovering and developing novel small molecule therapeutics in oncology and infectious diseases, today provided an update on the status of its hepatitis B virus (HBV) clinical program.
Tekmira Pharmaceuticals Corporation (Nasdaq:TKMR) today announced plans to change its corporate name to Arbutus Biopharma Corporation ("Arbutus", ticker symbol "ABUS"), an industry-leading therapeutic solutions company focused on developing a cure for chronic hepatitis B virus infection (HBV), to be effective on or before August 3, 2015.
TearLab Corporation (Nasdaq:TEAR) (TSX:TLB) expects to announce its Q2 2015 financial results after market closes on Thursday, August 6, 2015.
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced it has appointed John Pinion as Chief Quality Operations Officer and Senior Vice President, Analytical Sciences and Research, effective July 13, 2015. In this newly created role,