|Baxter Announces Primary Efficacy Endpoint for Phase 3 Trial of BAX 11; Cleveland Biolabs Successfuly Close of Phase 1 Study of CBL0102|
|By Staff and Wire Reports|
|Wednesday, 16 April 2014 18:19|
Baxter International Inc. (NYSE: BAX) announced topline results from a Phase 3 clinical trial evaluating the safety, efficacy and pharmacokinetics (PK) of BAX 111. BAX 111 is a recombinant von Willebrand factor (rVWF) under investigation for the treatment of bleeding episodes in patients with von Willebrand disease, the most common type of inherited bleeding disorder.
The study of BAX 111, the first recombinant treatment in clinical development for this condition, met its primary efficacy endpoint, as all patients achieved pre-specified success in the on-demand treatment of bleeding events (100%, 22 of 22 patients who experienced bleeds in the trial).
''As the first recombinant, stand-alone treatment in development, BAX 111 has the potential to offer people with von Willebrand disease a new therapeutic option that may allow for greater precision and flexibility in managing the disease,'' said Bruce Ewenstein, M.D., Ph.D., vice president of clinical affairs, in Baxter BioScience. ''With these findings, we have taken another significant step forward as we continue to expand on our increasingly broad pipeline of potential treatments to improve outcomes for patients with a range of bleeding disorders.''
The Phase 3 multicenter, open-label clinical trial assessed the safety, efficacy and pharmacokinetics of BAX 111 administered together with ADVATE or as a stand-alone therapeutic agent in the on-demand treatment of 37 patients with severe von Willebrand disease at trial sites in the United States, Europe, Australia, Japan, Russia and India. The primary endpoint was the number of patients experiencing successful treatment for bleeding episodes. Secondary endpoints included additional efficacy and safety measures, pharmacokinetics and health-related quality of life (HRQoL).
There were no reports of inhibitor development or thrombotic events in the study participants. The most common adverse events in the study were headache, vomiting/nausea and anemia (iron deficiency anemia), which were not considered to be related to treatment. There was one serious adverse event related to treatment, characterized by chest discomfort and increased heart rate during infusion, which rapidly resolved without further complication. The investigational treatment was developed using a plasma- and albumin-free manufacturing method.
Full data from the trial, including efficacy and safety outcomes, will be presented later in 2014. Both the European Commission and the U.S. Food and Drug Administration granted orphan-drug designation for BAX 111 in November 2010. Baxter intends to file for approval in the United States before the end of 2014 and, based on these results, intends to pursue a study of BAX 111 in a prophylaxis treatment setting before the end of the year.
Cleveland BioLabs, Inc. (NASDAQ: CBLI) announced the achievement of all objectives in a Phase 1 clinical trial of CBL0102, or quinacrine, an orally administered small molecule. The study was performed in patients with advanced cancers for which no standard care exists or which had become resistant to conventional therapies. All patients had tumors involving the liver.
CBL0102 is the first of a group of compounds identified by CBLI scientists that act by blocking activity of the chromatin remodeling complex, FACT (FAcilitates Chromatin Transcription), resulting in simultaneous modulation of several signal transduction pathways (p53, PI3K/AKT/mTOR, NF-kappaB and heat shock response) that are commonly deregulated in cancer (Guo et al., 2009 and Gurova et al., 2005). CBL0102 is being developed by Incuron, LLC, a joint venture between Bioprocess Capital Ventures and Cleveland BioLabs.
The primary objective of the study was to evaluate for a maximum tolerated dose and dose-limiting toxicities of CBL0102 in patients with advanced cancers. Secondary objectives were to characterize the drug's safety and to profile its pharmacokinetics. The study also assessed for preliminary evidence of CBL0102 antitumor activity. In particular, the study was designed to explore potential effects related to CBL0102's high relative biodistribution into the liver and therefore included only patients with primary or metastatic liver cancers. Patients were enrolled to receive sequentially higher starting doses of CBL0102 in seven cohorts. Study participants were treated with CBL0102 given orally daily. Patients could continue therapy for eight weeks (or longer if they appeared to be benefiting from therapy).
A total of 32 patients enrolled. Participants had cancers of breast, gastric, hepatic, pancreatic, and colorectal origin. The study successfully achieved both the primary and secondary objectives. CBL0102 was generally well-tolerated and a recommended Phase 2 dose of 400 mg/day was established. The most common adverse events were skin discoloration due to drug accumulation in skin, fatigue, upper abdominal pain, mild to moderate gastrointestinal disorders, and hepatic transaminase elevations, with most events being low grade. The analysis of pharmacokinetics showed that plasma exposures rose with increasing dose and that steady state had been achieved by 15 days of therapy. Liver biopsies were performed in two patients after four weeks of therapy and confirmed much higher liver concentrations of CBL0102 than were present in plasma.
By eight weeks of therapy, a partial tumor regression was recorded in one breast cancer patient, who experienced a 46% reduction in target lesion maximum dimensions. Disease stabilization was observed in four other patients (patients with breast cancer, hepatocellular carcinoma, salivary gland cancer, and rectal cancer). In the patient with hepatocellular carcinoma, long-term stabilization was observed for a period of 7.5 months, during which the patient remained on continuous CBL0102 treatment.
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, will release its first quarter 2014 financial results on Wednesday, April 30th, 2014 after the close of the market.
Amyris, Inc. (Nasdaq:AMRS), the leading synthetic biology innovator and renewable products company, today announced its industrial fermentation plant, located in the town of Brotas in southeastern Brazil, has successfully resumed industrial production following a planned maintenance period.
Apricus Biosciences, Inc. (Nasdaq:APRI), a pharmaceutical company focusing on the development and commercialization of novel therapeutics for men's and women's health, today announced that Takeda Pharmaceuticals International GmbH, the Company's partner in the United Kingdom, has accepted the first shipment of Vitaros®, Apricus' novel topical on-demand treatment for erectile dysfunction ("ED").
Athersys, Inc. (Nasdaq:ATHX) will release its first quarter 2014 financial results at approximately 4:00 PM (Eastern Time) on Thursday, May 8, 2014, and host a conference call shortly thereafter at 4:30 PM (Eastern Time) to review the results.
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin, will host a conference call and webcast on Thursday, May 1, at 5:00 p.m. ET to discuss first quarter 2014 financial results and provide a general business update.
Bio-Reference Laboratories, Inc. (Nasdaq:BRLI) announced today that it has hired Robert Daber, Ph.D. to assume the dual role as Director of Research and Development and Director of Cancer Genetics at its Elmwood Park international headquarters.
Celldex Therapeutics, Inc. (Nasdaq:CLDX) announced today that final data from its Phase 1 study of CDX-1401 in solid tumors, including long-term patient follow-up, have been published in Science Translational Medicine (Vol 6 Issue 232).
CAS Medical Systems, Inc. (Nasdaq:CASM), a leader in medical devices for non-invasive patient monitoring, today announced that it has signed an agreement with Gamida to serve as the exclusive distributor of the Company's FORE-SIGHT® Cerebral Oximeters in France.
Cesca Therapeutics Inc. (Nasdaq:KOOL), an autologous cell-based regenerative medicine company, today announced the restructuring of its sales, marketing and technical support organization and the appointment of Tim Lee as Director of International Sales.
Dehaier Medical Systems Ltd. (Nasdaq:DHRM), an emerging leader in the development, assembly, marketing and sale of medical devices and sleep respiratory products in China, today announced that it will serve as the exclusive product-appointed distribution agent for some of the world's leading medical equipment companies in bidding for multiple government procurement projects in China, the procurement projects we are bidding for are designated by these companies.
iKang Healthcare Group, Inc.(Nasdaq:KANG) is pleased to announce the launch of its new website, www.ikanggroup.com.
Innovus Pharmaceuticals, Inc., (OTCBB: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 American Congress of Obstetricians and Gynecologists clinical meeting in Chicago.
Islet Sciences, Inc. (OTCQB:ISLT) and BHV Pharma today announced positive results from two 12-week phase 2b clinical studies of remogliflozin etabonate in type 2 diabetics.
NanoString Technologies, Inc. (Nasdaq:NSTG), a provider of life science tools for translational research and molecular diagnostic products, today announced that the company will release 2014 first quarter financial results after the close of trading on Wednesday, May 7, 2014.
Relypsa, Inc. (Nasdaq:RLYP), a biopharmaceutical company, today announced the closing of its public offering of 4,130,611 shares of its common stock at a price to the public of $24.50 per share for gross proceeds of approximately $101 million, which includes the exercise in full by the underwriters of their option to purchase up to 538,775 additional shares of common stock.
TearLab Corporation (Nasdaq:TEAR) (TSX:TLB) expects to announce its Q1 2014 financial results after market closes on Thursday May 8, 2014.
TESARO, Inc. (Nasdaq:TSRO) will announce its first quarter 2014 financial results on Wednesday, April 30, 2014, after the close of the U.S. financial markets.
Tornier N.V. (Nasdaq:TRNX), a global medical device company focused on providing surgical solutions to orthopaedic extremity specialists, announced today that it will report financial results for the first quarter ended March 30, 2014 after the close of the regular trading market on Tuesday, May 6, 2014.
Trinity Biotech plc (Nasdaq:TRIB), a leading developer and manufacturer of diagnostic products for the point-of-care and clinical laboratory markets, will report financial results for the first quarter fiscal year 2014 on Tuesday, April 29, 2014.
Xenetic BioSciences (OTCBB:XBIO), a biopharmaceutical company focused on developing next-generation biologic drugs and novel orphan oncology therapeutics, today reported financial results for the year-ended December 31, 2013.