|Catalyst Pharma Announces a Clinical Trial for Pediatric Patients; AcelRx Announces Initiation of Clinical Study with ARX-04|
|By William Kent|
|Tuesday, 06 October 2015 19:17|
Below is a look at some of the headlines for companies that made news in the healthcare sector on October 6, 2015.
Catalyst Pharmaceuticals, Inc. (NASDAQ: CPRX), announced a clinical trial with Firdapse® (amifampridine phosphate) in pediatric patients with congenital myasthenic syndromes (CMS). Firdapse has received Breakthrough Therapy Designation from the FDA for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS), as well as orphan drug designations for LEMS and CMS.
Patrick J. McEnany, Catalyst's Chief Executive Officer said, "We continue to work towards the completion of our rolling submission of the NDA for Firdapse, which, as previously announced, we expect to complete during the fourth quarter of 2015. Our initial NDA submission will include data and information on the benefits of Firdapse for certain types of CMS and will request that CMS be included in our label for Firdapse. Our initial NDA submission will also include the positive results seen to date in children with CMS who are currently being treated with Firdapse under an investigator treatment IND."
Dr. Steven Miller, Catalyst's Chief Operating Officer and Chief Scientific Officer said, "With regard to our development plan for CMS, in addition to an investigator sponsored IND, we have initiated a small blinded clinical trial in the pediatric CMS population, ages 2 to 17. When the data from this new study is available, we intend to amend our NDA filing with these data. The design of this new study is based on the guidance that the FDA provided to us during our pre-NDA meeting in early 2015. Several academic institutions have been recruited for this new trial. We expect to complete this study by April of next year and at that time participants in this study may continue to receive Firdapse through our Expanded Access Program."
Additional information about this trial (NCT02562066) can be found on www.clinicaltrials.gov.
About Congenital Myasthenic Syndromes -- Congenital myasthenic syndromes, or CMS, is a rare neuromuscular disease comprising a spectrum of genetic defects and is characterized by fatigable weakness of skeletal muscles with onset at or shortly after birth or early childhood; in rare cases symptoms may not manifest themselves until later in childhood. The severity and course of the disease are variable, ranging from minor symptoms to progressive disabling weakness; symptoms may be mild, but sudden severe exacerbations of weakness or even sudden episodes of respiratory insufficiency also occur.
Congenital myasthenic syndrome(s) is rare, estimated at one-tenth that of myasthenia gravis, which in itself is rare. Based on currently available information, we estimate that there are between 1,000 and 1,500 CMS patients in the United States.
AcelRx Pharmaceuticals, Inc. (NASDAQ: ACRX), a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of moderate-to-severe acute and breakthrough pain, today announced the initiation of an open-label Phase 3 study (SAP302) of ARX-04 for the treatment of adult patients who present in the emergency room with moderate-to-severe acute pain associated with trauma or injury. The primary efficacy endpoint is the summed pain intensity difference (SPID) over 1-hour (SPID1). Safety endpoints, such as adverse events and vital signs will also be assessed, as will the patients' and healthcare providers' satisfaction with the method of pain control. The study is expected to be completed in early 2016.
"In our recently concluded SAP301 study, ARX-04 was shown to provide a rapid onset of action within 15 minutes of administration, making it well-suited for use in an emergency room setting, where speed is critical and where access to intravenous morphine may not be immediately available," stated Dr. Pamela Palmer, co-founder and chief medical officer of AcelRx Pharmaceuticals. "The SAP302 study is intended to provide us with valuable insights into the optimal use of ARX-04 in the emergency room setting, one of our initial target markets. It is also intended to complete the safety database requirements previously agreed to with the FDA." Howie Rosen, interim CEO of AcelRx, added, "As part of our development program, we expect to meet with the U.S. Food and Drug Administration to review plans for a New Drug Application for ARX-04 and will provide more guidance on regulatory timing following that meeting."
Active Biotech (NASDAQ:ACTI) today announces that the collaboration partner Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) will present data on laquinimod, an investigational therapy being evaluated in relapsing and progressive forms of MS, at the 31st European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress in Barcelona, October 7-10, 2015. For further information, please see www.tevapharm.com.
Advaxis, Inc. (NASDAQ:ADXS), a clinical-stage biotechnology company developing cancer immunotherapies, today announced that last Thursday the company received verbal notice from the U.S. Food and Drug Administration (FDA) that its Investigational New Drug (IND) application for axalimogene filolisbac (formerly ADXS-HPV) has been placed on clinical hold, affecting four clinical trials. The clinical hold, which pertains only to axalimogene filolisbac, was issued in response to Advaxis’s recent submission of a safety report to the FDA. The report involved a single event of one patient with end-stage cervical cancer who last received axalimogene filolisbac in early 2013 in an investigator-initiated trial. In late July 2015, the patient was hospitalized for end-stage cervical cancer symptoms. During hospitalization, routine blood cultures were positive for Listeria monocytogenes (Lm). Subsequent analysis determined that it was the highly attenuated strain of Lm used in axalimogene filolisbac which was incapable of causing infection and was highly sensitive to antibiotics. The patient received a course of intravenous antibiotics and was discharged. The patient returned to the hospital in mid-August, approximately two weeks later, with respiratory distress caused by her metastatic disease. The patient passed away later that day. The investigator ruled that the cause of death was due to progression of her cervical cancer.
Akers Biosciences, Inc. (NASDAQ:AKER) (AIM:AKR.L), a medical device company focused on reducing the cost of healthcare through faster, easier diagnostics, announces that all development specifications have been finalized and production has commenced for its two transformational breath tests designed for the health and wellness industry and consumers. Akers Bio is launching the BreathScan OxiChek™ and BreathScan KetoChek™ ("KetoChek") tests this week at the Supply Side West conference in Las Vegas, Nevada. The conference focuses on dietary supplements and sports nutrition, two key markets for these new products. OxiChek is the first disposable breath test to rapidly determine levels of oxidative stress (free radicals) in the body. OxiChek measures the most abundant free radicals including superoxides, hydrogen peroxide and aldehydes – unlike some pre-existing devices which measure only one. Frequent use of OxiChek may help consumers and professional clients manage and adhere to their regimen of nutritional supplementation for management of oxidative stress, which is implicated in many diseases, including cardiac, cancer and arthritic diseases.
Allscripts Healthcare Solutions, Inc. (Nasdaq:MDRX) will report its financial results for the three months ended September 30, 2015, after the close of regular stock market hours on Thursday, November 5, 2015. Allscripts management plans to host a conference call and webcast to discuss the company's earnings and other information at 4:30 p.m. Eastern Time that same day. The Allscripts earnings announcement will be distributed immediately after the close of regular stock market hours on November 5, 2015. The announcement will also be available at Allscripts investor relations website: http://investor.allscripts.com.
Arbutus Biopharma Corporation (Nasdaq:ABUS), an industry-leading therapeutic solutions company focused on developing a cure for chronic hepatitis B virus infection (HBV), today announced three presentations at the 2015 International Meeting on Molecular Biology of Hepatitis B Viruses being held on October 4 – 8, 2015, at Dolce Bad Nauheim, Germany. "We are excited to present these data supporting our HBV research and development efforts, in particular, related to TKM-HBV, our lead HBV RNAi clinical candidate, as well as our novel cccDNA formation inhibitors," said Dr. Michael J. Sofia, Arbutus's Chief Scientific Officer. "Our preclinical results validate that the unique three RNAi trigger design of TKM-HBV leads to reductions in hepatitis B surface antigen (HBsAg) and the other viral antigens but also results in a reduction of cccDNA levels, as well as unique synergistic effects of our novel cccDNA inhibitors in combination with nucleot(s)ide analogs."
Brainsway (TASE:BRIN), the leader in the development of advanced noninvasive treatments for brain disorders, today announced its partnership with the United States Navy to implement Deep Transcranial Magnetic Stimulation (Deep TMS). The devices purchased by the Navy will be used to help treat sailors suffering from clinical depression, with the first system to be deployed in San Diego, California. “The Navy’s purchase of our Deep TMS devices is indicative of its ongoing efforts to bring the most advanced medical treatments to the men and women who serve our country,” said Dr. Guy Ezekiel, chief executive officer of Brainsway. “It is our honor to partner with the Navy in its commitment to ensuring the mental health and safety of U.S. sailors.”
Community Health Systems, Inc. (NYSE:CYH) today announced that it will provide an online Web simulcast and rebroadcast of its third quarter 2015 conference call. The Company will issue a press release announcing its results on Monday, November 2, 2015, after the regular close of trading. The conference call is scheduled to begin at 10:00 a.m. Central time, 11:00 a.m. Eastern time, on Tuesday, November 3, 2015. A live broadcast of the conference call will be available online at www.chs.net. To listen to the live call, please go to the web site at least 15 minutes early to register, download, and install any necessary audio software. The online replay will follow shortly after the call and continue through December 3, 2015. During this call, Community Health Systems will review the Company's financial and operating results for the third quarter ended September 30, 2015.
ConforMIS, Inc. (NASDAQ:CFMS) today announced that it has substantially completed its investigation of excess moisture and ethylene glycol residue on specific serial numbers of patient-specific instrumentation for its iUni, iDuo, iTotal CR and iTotal PS knee replacement product systems, which was previously announced by the company on August 31, 2015. As part of the investigation, an independent testing laboratory determined that the ethylene glycol residue on the affected instrumentation posed no significant health hazard to patients and also found that no residue was present on any implants tested. Based on the results of the investigation, ConforMIS believes that no monitoring of patients is necessary. The results of ConforMIS' investigation suggest that the affected instrumentation underwent the commonly used ethylene oxide sterilization process in the presence of excess water and, as a result, contained small amounts of ethylene glycol residue. ConforMIS is in the process of completing final testing and implementing corrective actions, and expects to resume full production in October. ConforMIS reaffirms its financial guidance provided on August 31, 2015, for total revenue for the full year 2015 in a range of $64 million to $66 million, representing year-over-year growth of 33% to 37% on a reported basis and 39% to 43% on a constant currency basis. This guidance range reflects an $8 million impact from the recalled products that were shipped and not used, the lower production capacity over the period of investigation and resolution and the potential commercial disruption.
MedAssets (NASDAQ:MDAS) today announced that it plans to release its financial results for the third quarter and nine-month period ended September 30, 2015 after 4:00 p.m. ET on Monday, November 2, 2015. The conference call to discuss these results and the company's business and financial highlights is scheduled for the same afternoon, Monday, November 2nd, at 5:00 p.m. ET. You will be able to access a live webcast (audio with slides) from the "Events & Presentations" page at http://ir.medassets.com. To access the conference call, dial 888-517-2464 (or 847-413-3615 for international/local callers), and provide PIN code 9160777. For those unable to listen to the live broadcast, a Webcast replay will be archived on MedAssets' Website for at least 60 days. A conference call replay will be available for one month by calling 888-843-7419 or 630-652-3042, and entering PIN code 9160777.
Merge Healthcare Incorporated (NASDAQ:MRGE), a leading provider of clinical systems and innovations that seek to transform healthcare, entered into an agreement with Rush-Copley Medical Center to provide Merge PACS™, iConnect® Access and iConnect® Enterprise Archive. Rush-Copley Medical Center is a 210-bed hospital providing state-of-the-art health and wellness services to the greater Fox Valley region of Illinois, including the state’s second largest city, Aurora. Rush-Copley Medical Center was seeking to overhaul its suite of imaging technology to provide solutions that would streamline the process for their more than 170,000 radiology procedures per year. By leveraging Merge PACS and iConnect solutions, Rush-Copley will be able to access a consolidated view of a patient’s health record, reducing the amount of duplicate tests ordered allowing for more efficient patient care. “We are continually searching for workflow improvement solutions that provide radiologists with the ability to customize their personal work lists and improve inefficiencies,” said Thomas Markuszewski, director imaging services at Rush-Copley Medical Center. “We chose Merge Healthcare as our business partner to support us as we move to Stage 2 of Meaningful Use.”
Metabolix, Inc. (NASDAQ:MBLX), an advanced biomaterials company focused on sustainable solutions for the plastics industry, today announced that it will present its latest findings on the multifunctional benefits of bio-based PHA copolymers in a range of applications for polyvinyl chloride (PVC) and wood polymer composites. The new findings will be a key part of a technical presentation, entitled “Bio-based PHA Copolymers as Multifunctional Additives for Poly Vinyl Chloride Compound,” to be delivered by Mansoor Akhthar, Metabolix application development manager - PVC, at the upcoming Vinyltec 2015 conference Oct.13-15 in Akron, Ohio. New research is leading to the development and commercialization of PHA biopolymer technology that improves processing and performance characteristics in a range of PVC applications including flexible, semi-rigid and wood polymer composites, particularly when high levels of fillers and PVC recyclate are incorporated. Among the findings, Metabolix has shown that its PHA biopolymers – highly miscible in PVC – can be used as process aids that act as both a lubricant and fusion aid with a resulting reduction in machine torque to increase ease of processing. The use of PHA also allows the increased use of mineral fillers, wood flour or PVC recyclate with improved properties of the final parts. Another key finding is that all of these performance and processing advantages can deliver significant cost improvements.
Neothetics, Inc. (NASDAQ:NEOT), a clinical-stage specialty pharmaceutical company developing therapeutics for the aesthetic market, today announced the issuance of U.S. Patent No. 9,132,084 by the United States Patent and Trademark Office (USPTO). This marks issuance of the company's third United States patent in its portfolio that is directed to Neothetics' lead asset, LIPO-202, the first non-invasive injectable drug candidate for reduction of central abdominal bulging due to subcutaneous fat in non-obese subjects. This particular patent is directed to formulations and methods for reducing adipose tissue using LIPO-202. George Mahaffey, Neothetics' president and chief executive officer, stated: "We are pleased to have received issuance of this most recent patent from the USPTO, which further strengthens our robust intellectual property portfolio protecting our lead body contouring product candidate, LIPO-202, which is currently in pivotal Phase 3 trials."
RestorGenex Corporation (OTCQX:RESX), a specialty biopharmaceutical company focused on developing products for oncology, ophthalmology and dermatology, today announced that Stephen M. Simes, RestorGenex's CEO, will provide a company overview at two upcoming growth conferences featuring biotechnology and pharmaceutical companies. The first presentation will take place at the 2015 Aegis Growth Conference on October 9, 2015, at 9:00 am PT in The Encore at Wynn Las Vegas, in Las Vegas, Nevada. The second presentation will take place at the Dawson James Securities Growth Stock Conference on October 15, 2015. The presentation will take place at 11:15 am ET in the Wyndham Grand Hotel in Jupiter, Florida.
Rockwell Medical, Inc. (NASDAQ:RMTI), a fully-integrated biopharmaceutical company targeting end-stage renal disease (ESRD) and chronic kidney disease (CKD) with innovative products and services for the treatment of iron replacement, secondary hyperparathyroidism and hemodialysis, announced today that four individual Triferic clinical abstracts were selected by ASN's Program Committee for poster presentation at the ASN Kidney Week 2015 Annual Meeting, November 3-8 at the San Diego Convention Center in San Diego, CA. Triferic is the only FDA approved iron product indicated to replace iron and maintain hemoglobin in hemodialysis patients in the United States.
SANUWAVE Health, Inc. (OTCQB:SNWV), a shockwave technology company, announced today that Kevin A. Richardson II, SANUWAVE's Chairman of the board and Chief Executive Officer, and Lisa Sundstrom, SANUWAVE's Interim Chief Financial Officer, will present at the 2015 Aegis Growth Conference, being held Wednesday, October 7, 2015 through Friday, October 9, 2015 at the Encore at Wynn Las Vegas, Las Vegas, Nevada. Event: 2015 Aegis Growth Conference; Date: Thursday, October 8, 2015; Time: 10:45 a.m. PT; Location: The Encore at Wynn Las Vegas, Las Vegas, Nevada. Company management will be available for one-on-one meetings with investors participating in the conference. For those investors who would like to schedule an appointment, please contact Todd Markey by phone, (818) 280-6800, or email [email protected]
TOMI™ Environmental Solutions, Inc. (OTCQB:TOMZ), a global bacteria decontamination and infection prevention company, announced it will exhibit at the American Biological Safety Association (ABSA) from October 9-14, 2015 at the Rhode Island Convention Center in Providence. The conference showcases the latest in training, products and services in biological safety. TOMI will be exhibiting at booths 110 and 112. On Wednesday, October 14th at 3:45 PM Eastern Time, Miguel Grimaldo, MEng, from Galveston National Lab and the University of Texas Medical Branch-Galveston, will be delivering a presentation titled, Effectiveness of Decontamination of Laboratory Room Surfaces with Low Concentrations of Hydrogen Peroxide Using Atmospheric Cold Plasma Activation. TOMI's Binary Ionization Technology® (BIT™) originated with the Defense Advanced Research Projects Agency (DARPA) and has recently received a grant from United States Agency for International Development (USAID), the Center for Disease Control (CDC), and the Office of the President to support the ongoing development of SteraMist™ Mobile Decontamination Chambers to combat and prevent the spread of the Ebola epidemic. The TOMI team will be demonstrating its award winning technology in a mock hospital room on the exhibit floor.
WaferGen Bio-systems (Nasdaq:WGBS) announced today the commercial launch and presentation of data from the ICELL8™ Single-Cell System at the American Society of Human Genetics (ASHG) annual meeting in Baltimore, MD. In the hands of world-class early access partners globally, WaferGen's ICELL8 Single-Cell system has consistently demonstrated the power to isolate thousands of single-cells, the control to identify and process individual cells using CellSelect™ automated imaging and software and the ability to provide critical insight derived from running multiple samples or experiments on a single chip. These revolutionary technology advancements in single-cell analysis have the potential to not only accelerate the drug discovery and development process, but also expand fundamental biological discovery and understanding. WaferGen will present a poster at ASHG highlighting data generated by the Company demonstrating the ICELL8 Single-Cell System's ability to identify rare cells from a cell population at a sensitivity of <1%. The poster, number 1906W, will be presented at the conference on Wednesday, October 7, from 6-7pm. Additional details will also available at WaferGen's ASHG booth (number 1027).
Xtant Medical Holdings, Inc. (OTCQX:BONE), a leader in the development of regenerative medicine products and medical devices, today announced that its wholly owned subsidiary, Bacterin International, Inc., has been awarded a grant from the Montana Board of Research and Commercialization Technology (MBRCT) in the amount of $100,000 for the development and commercialization of the next generation of demineralized bone matrix (DBM) allografts. Bacterin's research, supported in part by the funding from MBRCT, will focus on enhancing the regenerative capacity of DBM allografts. The new products are anticipated to produce faster and more complete bone healing compared to currently available therapies leading to improved clinical outcomes for Bacterin’s surgeon customers and their patients. Successful commercialization of the therapies developed as a result of the research will further strengthen Bacterin’s DBM product portfolio by providing a differentiated product within the $410 million US market for demineralized bone matrices. "We continue to be very grateful for the continued financial support provided by the State of Montana to advance our research initiatives in the field of regenerative medicine," stated Dan Goldberger, CEO of Xtant Medical. "Past funding from the MBRCT has been instrumental to Bacterin’s growth over the years and we appreciate the opportunity to turn this grant into another commercial success."