|Isis Pharma Initiates Clinical Study of ISIS-HTT Rx; Bristol-Myers Announces Additional Phase IIa for BMS-955176|
|By William Kent|
|Tuesday, 21 July 2015 19:51|
Below is a look at some of the headlines for companies that made news in the healthcare sector on July 21, 2015.
Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced it has initiated a Phase 1/2a clinical study of ISIS-HTTRx in patients with Huntington's disease (HD). ISIS-HTTRx is the first therapy to enter clinical development that is designed to directly target the cause of the disease by reducing the production of the protein responsible for HD. HD is a rare genetic neurological disease in which patients experience deterioration of both mental abilities and physical control. Presently, there are no disease-modifying treatments for HD, with current therapies focused only on treating disease symptoms. ISIS-HTTRx has been granted orphan drug designation by the European Medicines Agency for the treatment of patients with HD. Orphan drug designation is granted to products designed to diagnose, prevent or treat life-threatening or very serious conditions that affect not more than five in 10,000 persons in the European Union.
Isis Pharmaceuticals, Inc. "Although the toxic protein produced from the huntingtin (HTT) gene in HD patients has been a target of interest for many years, no therapies have advanced to clinical trials to treat the underlying cause of the disease. Our antisense technology has enabled us to discover and develop ISIS-HTTRx, the first therapeutic approach designed to treat the genetic cause of HD. Together with Roche, we are committed to investigating this approach to treat patients with HD, a devastating disease that typically affects generations of families," said C. Frank Bennett, Ph.D., senior vice president of research at Isis Pharmaceuticals.
"Initiating the clinical study of ISIS-HTTRx in patients with HD is the first step in developing a treatment that could significantly impact a patient's disease. It is also an important milestone in our collaboration with Roche. As we advance this program, we will continue to benefit from Roche's scientific expertise in developing therapeutics for neurodegenerative conditions," said B. Lynne Parshall, chief operating officer of Isis Pharmaceuticals.
The randomized, placebo-controlled, dose escalation Phase 1/2a clinical study will evaluate the safety and activity of ISIS-HTTRx in patients with early stage HD. In this study, ISIS-HTTRx will be administered intrathecally as an injection directly into the cerebral spinal fluid. Intrathecal administration of antisense drugs has been shown to be well tolerated in multiple clinical studies in patients.
"The initial development of this antisense drug for Huntington's disease came out of a longstanding productive partnership between Isis and CHDI, and its advancement now to clinical trial is testament to Isis' perseverance and scientific expertise," said Robi Blumenstein, president of CHDI Management, which oversees the activities of CHDI Foundation, a nonprofit research organization exclusively dedicated to the development of therapies that will slow the progression of HD. "It's exciting that therapeutic candidates grounded in the biology of Huntington's disease are finally making their way to clinical trial."
Bristol-Myers Squibb Company (NYSE: BMY) announced additional Phase IIa proof-of-concept data for BMS-955176, a novel investigational agent designed to prevent the maturation of HIV-1. The study findings, which are being presented in a late-breaking oral presentation at the 8th IAS Conference on HIV Pathogenesis, Treatment and Prevention in Vancouver, confirmed the antiretroviral activity of BMS-955176 when administered with atazanavir (± ritonavir) and support further development of the second-generation HIV-1 maturation inhibitor.
BMS-955176 is designed to inhibit one of the last steps of the HIV-1 viral lifecycle, resulting in the release of immature non-infectious HIV-1 particles. As part of a multi-part proof-of-concept study, a two-drug combination of BMS-955176 (80 mg) plus atazanavir (unboosted) had a maximum median change in HIV-1 RNA of -2.23 log10 c/mL from baseline through study discharge (Day 42). The standard of care (SOC) control of atazanavir 300 mg and ritonavir 100 mg plus tenofovir disoproxyl fumarate 300 mg plus emtricitabine 200 mg in a fixed dose combination had a maximum median change in HIV-1 RNA of -2.39 log10 c/mL from baseline through study discharge (Day 42). In addition, a lower dose of BMS-955176 (40 mg) plus atazanavir and ritonavir had a similar maximum median change in HIV-1 RNA of -2.20 log10 c/mL. Length of therapy for all treatment groups was 28 days. Study endpoints included change in HIV-1 RNA from baseline to Day 28 and from baseline to the end of the study (Day 42) and safety.
"The BMS-955176 data provide further compelling evidence of its potential as a second-generation maturation inhibitor that suppresses HIV-1 in a novel way," said Douglas Manion, M.D., Head of Specialty Development, Bristol-Myers Squibb. "The 25 years we have spent fighting this disease have given us the expertise to help address the unmet needs in HIV treatment and we are committed to developing improved solutions for treatment-experienced patients."
Achaogen, Inc. (NASDAQ:AKAO), a clinical-stage biopharmaceutical company developing novel antibacterials to treat multi-drug resistant (MDR) gram-negative infections, today announced that on July 17, 2015, the compensation committee of the Company's board of directors granted 10 new employees options to purchase an aggregate of 60,650 shares of the Company's common stock and 40,650 restricted stock units.
Advaxis, Inc. (NASDAQ:ADXS), a clinical-stage biotechnology company developing cancer immunotherapies, today announced that the United States Patent and Trademark Office (USPTO) has granted U.S. Patent No. 9,084,747 with composition of matter claims covering ADXS-HER2.
Argos Therapeutics Inc. (Nasdaq:ARGS), an immuno-oncology company focused on the development and commercialization of fully individualized immunotherapies for the treatment of cancer based on the Arcelis® technology platform, today announced the formation of the company's first Scientific Advisory Board (SAB).
athenahealth, Inc. (NASDAQ:ATHN), a leading provider of cloud-based services and mobile applications for medical groups and health systems, today announced athenaText®, a secure text messaging service that is fully integrated with athenahealth's cloud-based EHR (electronic health record) platform, and accessible through the standalone athenaText and Epocrates mobile apps.
CombiMatrix Corporation (NASDAQ:CBMX) announces that President and CEO Mark McDonough will present at the July 2015 RedChip Online Global CEO Conference on Thursday, July 23, at 1:00 p.m. Eastern time (10:00 a.m. Pacific time).
EDAP TMS SA (Nasdaq:EDAP), the global leader in therapeutic ultrasound, today announced that it has received a letter from the U.S. Food and Drug Administration ("FDA") related to its Direct De Novo 510(k) petition for Ablatherm HIFU in the United States.
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, today announced that it has commenced an underwritten public offering of its common stock pursuant to its existing shelf registration statement.
GW Pharmaceuticals plc (Nasdaq:GWPH) (AIM:GWP), a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform, announced that the Company has appointed Julian Gangolli to the Board of Directors effective immediately.
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.
Intuitive Surgical, Inc. (NASDAQ:ISRG), the industry leader in robotic-assisted surgery, today announced financial results for the quarter ended June 30, 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.
MediciNova, Inc.,(NASDAQ:MNOV) and the Tokyo Stock Exchange (Code Number: 4875), today announced it is currently in late-stage discussions with investigators at Penn State University (principal investigator Dr. Rebecca Bascom) to conduct a Phase 2 study of MN-001 (tipelukast) for the treatment of moderate to severe IPF (idiopathic pulmonary fibrosis).
Momenta Pharmaceuticals, Inc. (NASDAQ:MNTA), a biotechnology company specializing in the characterization and engineering of complex drugs, will release its financial results for the second quarter ended June 30, 2015 before the U.S. financial markets open on Tuesday, August 4, 2015.
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 second quarter 2015 financial results after the close of trading on Tuesday, August 4, 2015.
Novavax, Inc. (Nasdaq:NVAX), a clinical-stage vaccine company focused on the discovery, development and commercialization of recombinant nanoparticle vaccines and adjuvants, today announced positive top-line data from a Phase 1 clinical trial of its Ebola virus glycoprotein (GP) recombinant nanoparticle vaccine candidate adjuvanted with Matrix-M™ as part of the World Health Organization’s (WHO) Fifth Teleconference on Ebola Vaccine Clinical Trials.
NeuroDerm Ltd. (NASDAQ:NDRM), a clinical stage pharmaceutical company developing drugs for central nervous system (CNS) diseases, today announced the closing of its public offering of 4,524,698 ordinary shares at a price to the public of $17.00 per share, which includes 590,178 shares purchased pursuant to the full exercise of the underwriters' option to purchase additional shares.
Oxford Immunotec Global PLC (Nasdaq:OXFD) today announced that it plans to release second quarter 2015 financial results prior to market open on Tuesday, August 4, 2015. Dr. Peter Wrighton-Smith, Chief Executive Officer and Rick Altieri, Chief Financial Officer will host a conference call to review the Company's results at 8:00 a.m. Eastern Time the same day.
Paratek Pharmaceuticals, Inc. (Nasdaq:PRTK), a biopharmaceutical company focused on the development and commercialization of innovative therapies based upon tetracycline chemistry, today opened its new corporate headquarters in Boston at 75 Park Plaza.
Royal Philips (NYSE:PHG) (AEX:PHIA) and Profound Medical Corp. (TSXV:PRN), a Toronto-based medical device company, today announced that they have signed a joint development agreement to support Profound Medical's proprietary TULSA (Transurethral Ultrasound Ablation) technology designed to treat patients with prostate cancer on Philips' Ingenia and Achieva 3T MRI systems.
T2 Biosystems, Inc. (NASDAQ:TTOO) today announced that it will release its 2015 second quarter and six-month financial results after the market closes on Tuesday, August 4, 2015.
TriVascular Technologies, Inc. (Nasdaq:TRIV), manufacturer of the Ovation Prime® Abdominal Stent Graft System, today announced that the Company will release 2015 second quarter financial results after market close on Tuesday, August 4, 2015.