FDA Approves Baxter's RIXUBIS; TG Therapeutics Announces Special Protocol Assessment Agreement With The FDA for TG-1101 Print
By David Fowler   
Monday, 15 September 2014 19:29
Below is a look at some of the headlines for companies that made news in the healthcare sector on September 15, 2014.
Baxter International Inc. (NYSE: BAX) announced FDA has approved RIXUBIS [Coagulation Factor IX (Recombinant)] for routine prophylactic treatment, control and prevention of bleeding episodes, and perioperative management in children with hemophilia B. RIXUBIS was the first recombinant factor IX (rFIX) approved for routine prophylaxis and control of bleeding episodes in the U.S. for adults living with this chronic condition.
''In addition to the positive reception we've received from adult RIXUBIS patients, the approval for pediatric patients offers a valuable new option, particularly as our clinical data demonstrated a significant reduction in bleeding episodes for patients who were treated prophylactically, an important factor for this young patient population,'' said John Orloff, M.D., vice president of global research and development at Baxter BioScience.
The approval is based on the results of a clinical trial investigating the efficacy and safety of RIXUBIS among 23 previously-treated male patients less than 12 years of age with severe or moderately severe hemophilia B. The patients were treated with a twice-weekly RIXUBIS prophylaxis regimen (mean dose 56 IU/kg) for a mean treatment duration of six months and a mean of 54 exposure days (EDs). The median annualized bleeding rate (ABR) was 2.0 (0.0 for spontaneous bleeds and joint bleeds). Nine patients in the study (39.1%) experienced no bleeds and 23 bleeding episodes (88.5%) were treated with 1-2 infusions. There were no reports of inhibitor development, no severe allergic reactions, and no thrombotic or treatment-related adverse events among the study participants. Common adverse reactions observed in >1% of subjects in clinical studies were dysgeusia, pain in extremity, and positive test for furin antibody. These data were presented during the 55th Annual Meeting of the American Society of Hematology (ASH) in New Orleans, LA.
Baxter's application for marketing approval for RIXUBIS for adults and pediatric patients is currently under review in the European Union, with a regulatory decision expected later this year. The treatment also recently gained regulatory approval in Australia.
TG Therapeutics, Inc. (Nasdaq: TGTX) announced it has reached an agreement with the FDA regarding a Special Protocol Assessment (SPA) on the design, endpoints and statistical analysis approach of a Phase 3 clinical trial for TG-1101 (ublituximab), its glycoengineered anti-CD20 monoclonal antibody, in combination with Imbruvica® (ibrutinib) for the treatment of Chronic Lymphocytic Leukemia (CLL) in patients with high risk cytogenetics. The SPA provides agreement that the Phase 3 trial design adequately addresses objectives that would support the regulatory submission for drug approval.
Full details of the Phase 3 clinical trial will be released at the launch of the study, which is expected to occur before the end of the year. In this randomized controlled trial, patients will receive either TG-1101 plus ibrutinib or ibrutinib alone. The trial will enroll approximately 330 patients, with approximately the first two-thirds of the patients included in the ORR assessment. As per the SPA, the Company plans to use the ORR data from the trial as the basis for submission of a Biologics License Application (BLA) for accelerated approval for TG-1101. All patients will then be followed for PFS assessment, which is designed to support full approval.
Additionally, the Company reported that enrollment into the CLL cohort of its ongoing combination Phase 2 study of TG-1101 plus ibrutinib is now closed. The Company expects it will have over 30 CLL patients evaluable for safety and efficacy available for presentation at the Annual Meeting of the American Society of Hematology (ASH) this December.
Dr. Jeff Sharman, Medical Director of Hematology Research for the US Oncology Network, and currently the lead investigator on the Company's Phase 2 combination trial of TG-1101 plus ibrutinib, will be the Study Chair for this Phase 3 trial. Dr. Sharman commented, "We have been very pleased with the safety and activity profile of the combination seen thus far in our Phase 1/2 trial, and are excited to lead this important clinical trial. Combining a glycoengineered anti-CD20 monoclonal antibody with ibrutinib has the potential to materially increase the number of patients benefitting from treatment with ibrutinib therapy. The Phase 2 trial has enrolled very quickly throughout our clinical research network with patients, physicians, and nurses recognizing the benefits of this study."
Michael S. Weiss, Executive Chairman and Interim Chief Executive Officer of TG Therapeutics, stated, "The TG-1101 SPA agreement is a major milestone for us as it represents the first clearly defined development and regulatory pathway for the approval of TG-1101 for the treatment of CLL. As we've mentioned previously, reaching agreement with the FDA on this combination trial with ORR as a primary endpoint for accelerated approval was our number one priority. We are thrilled to get this first SPA in place earlier than anticipated, which puts us in an excellent position to build out the remainder of our registration program for both TG-1101 and TGR-1202. We would like to thank the FDA for its invaluable guidance throughout this process. The speed at which the FDA reviewed the submission demonstrates a real commitment to drive novel medicines to the patients who need them as rapidly as possible." Mr. Weiss continued, "We look forward to launching this trial by year end and are excited to continue to work with Dr. Sharman, the entire US Oncology team, and all investigators, who have been instrumental in our development of this important combination."
Also Monday:
Amgen (NASDAQ: AMGN) today announced results from a new exploratory analysis of the open-label extension study of the pivotal Phase 3 fracture trial, which found that treatment with Prolia®(denosumab) for eight years enabled a substantial proportion of women with osteoporosis to achieve non-osteoporotic T-scores (>-2.5) at the lumbar spine and total hip from baseline. Additional Prolia data of interest are detailed below. 
Analogic Corporation (Nasdaq:ALOG), enabling the world's medical imaging and aviation security technology, today announced results for its fourth quarter ended July 31, 2014.
Auris Medical Holding AG (NASDAQ: EARS) today provided an update on the Company's business and announced financial results for the second quarter ended June 30, 2014.
Amgen (NASDAQ: AMGN) and UCB (Euronext Brussels:UCB) today announced results from several exploratory analyses of the Phase 2 study evaluating romosozumab in postmenopausal women with low bone mineral density (BMD). 
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that CenterWatch, a leading source for global clinical trial information, has named it one of the fastest drug developers in the industry. 
Celator Pharmaceuticals, Inc. (Nasdaq:CPXX), a pharmaceutical company developing new and more effective therapies to treat cancer, today announced the publication of preclinical data in Pediatric Blood & Cancer that further characterize the therapeutic potential of lead compound CPX-351 in hematological malignancies and support its ongoing clinical testing in pediatric patients with ALL. 
Dig-it Underground, Inc. (PINKSHEETS: DIGX) -- Robert Wodarczyk, President of Dig-it Underground, Inc., stated today, "With the legalization of adult use marijuana in the States of Colorado and Washington, the cannabis industry has seen continued growth and expansion. 
Five Prime Therapeutics, Inc. (Nasdaq:FPRX), a clinical-stage biotechnology company focused on discovering and developing novel protein therapeutics for cancer and inflammatory diseases, announced today that GlaxoSmithKline (GSK) has exercised its option to obtain an exclusive, worldwide license to products containing or directed to an undisclosed muscle disease target that Five Prime discovered using its proprietary library of human extracellular proteins and target screening and discovery capabilities.
Fonar Corporation (NASDAQ: FONR), The Inventor of MR Scanning™, today announced its 2014 fiscal year-end results. The Company's two industry segments are: development, manufacturing and servicing of the UPRIGHT® Multi-Position™ MRI and management of Stand-Up® MRI (UPRIGHT® MRI) centers. 
Galectin Therapeutics Inc. (Nasdaq:GALT), the leading developer of therapeutics that target galectin proteins to treat fibrosis and cancer, today announced that the U.S. Patent and Trademark Office has issued a patent for the Company's application titled "Galactose-Pronged Carbohydrate Compounds for the Treatment of Diabetic Nephropathy and Associated Disorders." 
The Board of Directors of GreeneStone Healthcare Corporation (OTCBB:GRST), a provider of healthcare services including Addiction and Mental Health, has approved a plan to become more specialized with a greater focus on the company's addiction treatment businesses.
Hemispherx Biopharma, Inc. (NYSE MKT:HEB), announced today a peer-reviewed publication entitled "Intranasal Seasonal Influenza Vaccine and a TLR-3 Agonist, Rintatolimod, Induced Cross-Reactive IgA Antibody Formation Against Avian H5N1 and H7N9 Influenza HA in Humans" (Vaccine (2014), http://dx.doi.org/10.1016/j.vaccine.2014.07.078).
Intuitive Surgical, Inc. (Nasdaq:ISRG), the leader in operative surgical robotics, will hold its quarterly conference call to discuss Third Quarter 2014 results, Tuesday, October 21st, at 4:30 p.m. Eastern Time.
IRADIMED CORPORATION (Nasdaq:IRMD) today announced that it will host a conference call on Thursday, September 18, 2014 beginning at 11:00 a.m. Eastern Time to provide an update on the Company's September 2nd press release regarding the receipt of a warning letter from the FDA.
Kips Bay Medical, Inc. (OTCQB:KIPS), a medical device company focused on manufacturing and commercializing its external saphenous vein support technology, eSVS® Mesh, for use in coronary artery bypass grafting, or CABG, surgery, today announced that its common stock began trading on the OTCQB Marketplace on September 12, 2014.
MELA Sciences, Inc. (Nasdaq:MELA), developer of the MelaFind® system, a non-invasive optical diagnostic tool that assists dermatologists in the diagnosis of melanoma at its most curable and cost-effective stage and that has received both FDA Pre-Market Approval (PMA) for the U.S. and CE Marking certification for the European Union, today announced that MELA CEO Rose Crane will provide a corporate overview at the Craig-Hallum Capital Group's 2014 Alpha Select Conference in New York City.
RedHill Biopharma Ltd. (Nasdaq:RDHL) (TASE:RDHL), an Israeli biopharmaceutical company focused on late clinical-stage drugs for inflammatory and gastrointestinal diseases, including gastrointestinal cancers, today announced that Adi Frish, RedHill's Senior VP Business Development and Licensing, will be presenting at the 7th Annual BioPharm America International Partnering Conference, on Tuesday, September 23, 2014, at 2:45 p.m. EDT, in the Suffolk room of the Boston Marriott Copley Placein Boston, Massachusetts.
Relypsa, Inc. (Nasdaq:RLYP), a biopharmaceutical company, today announced that data in heart failure (HF) patients from its pivotal Phase 3 clinical trial of patiromer, the company's lead product candidate, were presented as a late-breaking oral presentation at the 18th Annual Scientific Meeting for the Heart Failure Society of America (HFSA) being held in Las Vegas. 
Response Genetics, Inc. (Nasdaq:RGDX), a company focused on the development and sale of molecular diagnostic tests for cancer, today announced it has launched a new and innovative Non-Small Cell Lung Cancer Profile that merges rapid turnaround on National Comprehensive Cancer Network (NCCN) guideline markers with next-generation sequencing (NGS). 
TearLab Corporation (Nasdaq:TEAR) (TSX:TLB) announced that preliminary results of the TearLab Cataract Study were presented today at the XXXIII Congress of the European Society of Cataract and Refractive Surgeons ("ESCRS").
TG Therapeutics, Inc. (Nasdaq:TGTX) announced today that it has reached an agreement with the U.S. Food and Drug Administration (FDA) regarding a Special Protocol Assessment (SPA) on the design, endpoints and statistical analysis approach of a Phase 3 clinical trial for TG-1101 (ublituximab), its glycoengineered anti-CD20 monoclonal antibody, in combination with Imbruvica® (ibrutinib) for the treatment of Chronic Lymphocytic Leukemia (CLL) in patients with high risk cytogenetics. 
Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the presentation of results from a long-term Phase 1/2 extension study, conducted by Kyowa Hakko Kirin Pharma, Inc., of the investigational fully human anti-FGF23 monoclonal antibody KRN23 (UX023) in adult patients with X-linked hypophosphatemia (XLH). 
VBI Vaccines, Inc. (Nasdaq:VBIV) presented new data last week that demonstrates continued support for the development of the company's prophylactic CMV vaccine candidate (VBI-1501A).
ZS Pharma (Nasdaq:ZSPH), a biopharmaceutical company developing novel treatments for kidney, cardiovascular, liver and metabolic disorders, today announced results of a secondary analysis of ZS003, a two week pivotal Phase 3 trial of ZS-9 (sodium zirconium cyclosilicate), its investigational treatment for hyperkalemia.

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