|Pluristem Submits FDA Orphan Drug Application for Preeclampsia; Oncothyreon and Array Announce Initiation of Phase 1b Trial of ONT-380|
|By Mary Davila|
|Thursday, 20 February 2014 20:33|
Pluristem Therapeutics, Inc. (NasdaqCM: PSTI; TASE: PLTR), a leading developer of placenta-based cell therapies, announced it is submitting its application to the United Stated Food and Drug Administration requesting the Company to be granted Orphan Drug Designation for its PLancental eXpanded (PLX-PAD) cells in the treatment of severe preeclampsia.
Orphan Drug Designation may qualify a company for several benefits under the Orphan Drug Act of 1983 (ODA), as amended. These benefits may include a 7-year period of orphan drug exclusivity upon product approval, a tax credit for certain clinical testing expenses for the orphan drug, written guidance on the non-clinical and clinical studies needed to obtain marketing approval of an orphan drug, and orphan drug grants.
“Having just recently established our Preeclampsia Steering Committee comprised of key medical opinion leaders, this Orphan Drug application submission is an important step in advancing our preeclampsia development program,” stated Pluristem Chairman and CEO Zami Aberman. “The tremendous unmet medical need makes this indication one of our top priorities. We look forward to advancing into human trials and we are hopeful that the very strong preclinical data for PLX cells will translate into similar results at the clinical level.”
Pluristem has successfully received Orphan Drug Designation from the FDA for its PLX cells in two other indications: the treatment of aplastic anemia; and the treatment of Buerger's disease.
About Preeclampsia -- Preeclampsia is one of the most common medical complications of pregnancy, and one of the leading known causes of premature births, stillbirths and early neonatal and maternal deaths. If left untreated it can progress to eclampsia, the life-threatening occurrence of seizures during pregnancy. The only definitive treatment for preeclampsia is abortion or delivery. The disease occurs after the 20th week of pregnancy, and is characterized by high blood pressure and significant amounts of protein in the urine or end-organ dysfunction. According to the World Health Organization, preeclampsia occurs in approximately 6%–8% of pregnancies worldwide. It is estimated that preeclampsia costs the global health care system $3 billion annually.
Oncothyreon Inc. (NASDAQ: ONTY) and Array BioPharma Inc. (NASDAQ: ARRY) announced the initiation of a Phase 1b trial of ONT-380 (ARRY-380) in combination with Kadcyla (ado-trastuzumab emtansine or TDM-1) in patients with metastatic HER2+ breast cancer. ONT-380 is an orally active, reversible and selective small-molecule HER2 inhibitor invented by Array and being developed by Oncothyreon in collaboration with Array.
The trial (ClinicalTrials.gov Identifier NCT01983501) is a dose-escalation study in up to 48 patients who have been previously treated with Herceptin (trastuzumab) and a taxane for metastatic breast cancer. The primary objective is to determine the maximum-tolerated and/or recommended Phase 2 dose (MTD/RP2D) of ONT-380 in combination with the approved dose of Kadcyla. Secondary objectives include an evaluation of the safety and preliminary anti-tumor activity of the combination. Following determination of the MTD/RP2D, the study includes an expansion arm at the MTD/RP2D as well as an optional second expansion arm in patients with central nervous system metastases.
"ONT-380 and Kadcyla are both directed at HER2, but with differing mechanisms of action," said Diana Hausman, M.D., Chief Medical Officer of Oncothyreon. "The strategy of combining two agents targeting HER2 has previously proven useful in HER2+ breast cancer, and we believe the combination of ONT-380 and Kadcyla is particularly relevant to evaluate as the treatment of metastatic HER2+ breast cancer continues to evolve."
About ONT-380 -- ONT-380 is an orally active, reversible and selective HER2 inhibitor. In multiple preclinical tumor models, ONT-380 was well tolerated and demonstrated significant dose-related tumor growth inhibition that was superior to Herceptin and Tykerb (lapatinib). Additionally, in these models, ONT-380 demonstrated synergistic or additive tumor growth inhibition when dosed in combination with the standard-of-care therapeutics Herceptin or Taxotere (docetaxel). ONT-380 has also demonstrated superior activity, based on overall survival, compared to Tykerb and to the investigational drug, neratinib, in an intracranial HER2+ breast cancer xenograft model.
A Phase 1 trial of ONT-380, with both dose-escalation and expansion components, has been completed in 50 patients, 43 of whom had HER2+ metastatic breast cancer. All HER2+ breast cancer patients had progressed on a Herceptin-containing regimen. In addition, over 80% had been treated with Tykerb, with many having progressed on therapy. In this study, ONT-380 demonstrated an acceptable safety profile; treatment-related adverse events were primarily Grade 1. Because ONT-380 is selective for HER2 and does not inhibit EGFR, there was a low incidence and severity of treatment-related diarrhea, rash and fatigue. Additionally, there were no treatment-related cardiac events or Grade 4 treatment-related adverse events reported. The maximum tolerated dose of ONT-380 established in this Phase 1 trial was 600 mg twice daily (BID). Twenty-two HER2+ breast cancer patients with measurable disease were treated with ONT-380 at doses greater than or equal to 600 mg BID. In this heavily pretreated patient population, there was a clinical benefit rate (partial response [n = 3] plus stable disease for at least 6 months [n = 3]) of 27%. Notably, two of the patients with partial responses during treatment with ONT-380 had confirmed progressions while on prior Tykerb- and Herceptin-containing regimens.
In addition to the Phase 1b trial of ONT-380 in combination with Kadcyla described above, Oncothyreon recently initiated a second Phase 1b trial of ONT-380 in combination with Herceptin and/or Xeloda (capecitabine) in patients with metastatic HER2+ breast cancer. The Dana-Farber Cancer Institute, Boston, Massachusetts, is also currently conducting an investigator-sponsored trial of ONT-380 in combination with Herceptin in patients with brain metastases from HER2+ breast cancer.
Air Methods Corporation (Nasdaq:AIRM), the global leader in air medical transportation, announced today that its United Rotorcraft Division has been awarded contracts totaling up to $5.5 million with the Ecuadorian Navy, AgustaWestland, and an unnamed customer in the Middle East to design, manufacture, certify and complete several airframes for a variety of missions.
Alphatec Holdings, Inc. (Nasdaq:ATEC), the parent company of Alphatec Spine, Inc., a medical device company that provides physician-inspired solutions for patients with spinal disorders, announced today that it is postponing the release of its financial results for the fourth quarter and fiscal year 2013 and the conference call to discuss the results, both of which were originally scheduled for February 27th, 2014.
Applied Optoelectronics, Inc. (Nasdaq:AAOI), a leading provider of fiber-optic access network products for the cable broadband, internet data center, and fiber-to-the-home markets, today announced that management will present at four investor conferences in the month of March.
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for the company's Marketing Authorization Application (MAA) for VIMIZIM™ (elosulfase alfa) for the treatment of Morquio A syndrome, also called Mucopolysaccharidosis Type IVA (MPS IVA).
Cancer Genetics, Inc. (Nasdaq:CGIX), an emerging leader in DNA-based diagnostics, initiated two additional multi-year clinical trial contracts with biopharmaceutical customers.
Cempra, Inc. (Nasdaq:CEMP) today announced the dosing of the first patient in its Phase 1 study of solithromycin capsules in children.
When Cerner Corp. (Nasdaq:CERN) debuts its 13,000 square feet booth (6965) at the Healthcare Information and Management Systems Society annual conference in Orlando, Fla., visitors will hear first-hand experiences from three Cerner clients who have achieved HIMSS Stage 7 – the industry's top health care IT designation.
Chembio Diagnostics, Inc. (Nasdaq:CEMI), which develops, manufactures, markets and licenses point-of-care diagnostic tests, announced today that the Company will release financial results for the year of 2013, before the open of the market on Thursday, March 6, 2014.
Conatus Pharmaceuticals Inc. (Nasdaq:CNAT), a biotechnology company focused on the development and commercialization of novel medicines to treat liver disease, today announced its scheduled participation in the following investor events in February: Conatus is scheduled to participate in meetings with invited institutional investors at the Canaccord Genuity Orphan Drug One-on-One Day on Monday, February 24, 2014, in New York, Conatus is scheduled to discuss its approach to the development of treatments for nonalcoholic steatohepatitis (NASH) as part of a panel at the JMP Securities NASH Workshop on Thursday, February 27, 2014, in New York.
Curis, Inc. (Nasdaq:CRIS), an oncology-focused company developing novel, targeted drug candidates for the treatment of human cancers, today reported its financial results for the fourth quarter and year ended December 31, 2013.
Digirad Corporation (Nasdaq:DRAD) will release financial results for its fourth quarter and year ended December 31, 2013, before the market opens on Wednesday, February 26, 2014.
Informatica Corporation (Nasdaq:INFA), the world's number one independent provider of data integration software, today announced that it is exhibiting at booth # 6645 at the 2014 Healthcare and Information Management Systems Society (HIMSS) Annual Conference & Exhibition, which is scheduled for Feb. 23-27 at the Orange County Convention Center in Orlando, Fla.
Inogen, Inc. (Nasdaq:INGN) today announced that it has closed its previously announced initial public offering of 4,411,763 shares of its common stock at a price to the public of $16.00 per share.
Innovus Pharmaceuticals, Inc., (OTC BB:INNV) today announced that it will exhibit Zestra®, its topical over the counter ("OTC") product for female sexual arousal and desire at the 2014 Annual International Society for the Study of Women's Sexual Health Meeting in San Diego from February 20-22, 2014.
IPC The Hospitalist Company, Inc. (Nasdaq:IPCM), a leading national hospitalist physician group practice, today announced financial results for the fourth quarter and full year ended December 31, 2013.
Kratos Defense & Security Solutions, Inc. (Nasdaq:KTOS), a leading National Security Solutions provider, and Norse, the leading provider of live threat intelligence-based cybersecurity solutions, announced today that Kratos' SecureInfo business group and Norse will jointly offer services to deliver increased cybersecurity awareness for healthcare providers.
LeMaitre Vascular, Inc. (Nasdaq:LMAT), announced today that it will release its fourth quarter 2013 financial results on February 25, 2014, after the market close.
LHC Group Inc. (Nasdaq:LHCG), a national provider of post-acute care services, announced today details for the release of its results for the fourth quarter and year ended December 31, 2013.
Medgenics, Inc. (NYSE MKT: MDGN) (LSE:MEDU) (LSE:MEDG), the developer of a novel platform technology for the sustained production and delivery of therapeutic proteins in patients using ex-vivo gene therapy and their own tissue for the treatment of rare and orphan diseases, today announced financial results for the fiscal year ended December 31, 2013 and the filing with the U.S. Securities and Exchange Commission ("SEC") of the Company's Annual Report on Form 10-K.
Merck (NYSE: MRK), known as MSD outside the United States and Canada, announced today that the Biologics License Application for V503, its investigational 9-valent human papillomavirus (HPV) vaccine, has been accepted for standard review by the U.S. Food and Drug Administration.
Merrimack Pharmaceuticals, Inc. (Nasdaq:MACK) today announced that the company will host its Fourth Quarter 2013 Investor Conference Call and webcast at 4:30 p.m., Eastern time, on Thursday, February 27.
Merge Healthcare Incorporated (Nasdaq:MRGE), a leading provider of innovative enterprise imaging, interoperability and clinical systems that seek to advance healthcare, today announced its financial and business results for the fourth quarter of 2013 and provided 2014 guidance.
Nuvilex, Inc. (OTCQB:NVLX), an international biotechnology company providing cell and gene therapy solutions for the treatment of deadly diseases, announced today that it has entered into a stock purchase agreement with Lincoln Park Capital Fund, LLC (Lincoln Park), a Chicago-based institutional investor.
OncoMed Pharmaceuticals, Inc. (Nasdaq:OMED), a clinical-stage company developing novel therapeutics that target cancer stem cells (CSCs), or tumor-initiating cells, today announced the initiation of patient treatment for its third multi-center Phase 1b clinical trial of OMP-54F28 (Fzd8-Fc) with carboplatin and paclitaxel in patients with platinum-sensitive ovarian cancer.
OraSure Technologies, Inc. (Nasdaq:OSUR) today announced that Douglas A. Michels, President and CEO, will speak to the investment community at the 2014 Citi Global Healthcare Conference in New York City.
RCM Technologies, Inc. (Nasdaq:RCMT) will hold a conference call on Thursday, February 27, 2014 at 10:30 a.m. Eastern time to discuss the financial results for the thirteen week and fifty-two week periods ended December 28, 2013.
Stemline Therapeutics, Inc. (Nasdaq:STML) announced today that it will present at the Citi 2014 Global Healthcare and RBC Capital Markets Global Healthcare conferences in February.
Symmetry Medical Inc. (NYSE:SMA), a leading global source of innovative medical device solutions, including surgical instruments, orthopedic implants, and sterilization cases and trays, announced fourth quarter and full year 2013 financial results for the period ended December 28, 2013.
Tekmira Pharmaceuticals Corporation (Nasdaq:TKMR) (TSX:TKM), a leading developer of RNA interference (RNAi) therapeutics, today announced that Dr. Mark Murray, President and CEO of Tekmira, will provide a corporate overview at the 2014 RBC Capital Markets' Global Healthcare Conference on Tuesday, February 25, 2014 at 3:05 pm ET at the New York Palace Hotel.
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 fourth quarter and fiscal year ended December 29, 2013.
XOMA Corporation (Nasdaq:XOMA), a leader in the discovery and development of therapeutic antibodies, announced today members of its management team are scheduled to present at three upcoming investor conferences.