|Vanda Announces FDA Acceptance for Review of NDA for Maintenance Treatment of Schizophrenia; XenoPort Announces Positive Phase 2 Study Results for XP23829|
|By William Kent|
|Tuesday, 15 September 2015 19:03|
Below is a look at some of the headlines for companies that made news in the healthcare sector on September 15, 2015.
Vanda Pharmaceuticals Inc., (NASDAQ: VNDA), announced the U.S. FDA has accepted for review Vanda's filing of a supplemental New Drug Application (sNDA) for Fanapt® seeking approval as a maintenance treatment of schizophrenia in adults.
Vanda is seeking approval based on the results of the REPRIEVE clinical study, which evaluated the long-term maintenance of efficacy and safety of Fanapt®.
The FDA has set a user fee goal date under the Prescription Drug User Fee Act of May 27, 2016, which is the target date for the FDA to complete its review of the sNDA.
XenoPort, Inc. (NASDAQ: XNPT) announced positive preliminary top-line results from its Phase 2 clinical trial of XP23829 as a potential treatment for moderate-to-severe chronic plaque-type psoriasis. XP23829 met its primary endpoint in both 800 mg once daily and 400 mg twice daily doses, demonstrating statistically significant improvements in percent change from baseline to week 12 in Psoriasis Area and Severity Index (PASI) score. XP23829 is a patented prodrug of monomethyl fumarate (MMF) in a novel oral formulation that was designed to potentially offer physicians and patients an effective, better tolerated and easier to use therapeutic option to currently available fumarate products.
Richard Kim, M.D., chief medical officer of XenoPort, stated, "We believe these clinical data demonstrate for the first time that a MMF prodrug other than dimethyl fumarate (DMF) can be effective in reducing lesions in psoriatic patients. The magnitude of XP23829's effect on the primary efficacy endpoint met our expectations for this relatively short duration trial and we are particularly encouraged by the results with 800 mg once-daily dosing. Based on what is known about fumarates, we believe that the efficacy of XP23829 is likely to improve with a more extended duration of treatment beyond 12 weeks. We are also pleased with the safety and tolerability profile of XP23829 emerging from this study. We believe that this demonstration of efficacy, safety and tolerability of XP23829 could lead to a differentiated product in psoriasis. We also believe that there is potential for the observations from this study to read through to other potential indications such as multiple sclerosis (MS)."
Description of the Trial -- This randomized, double-blind, placebo-controlled Phase 2 clinical trial of XP23829 was conducted in 33 sites in the United States in subjects with moderate-to-severe chronic plaque-type psoriasis. Two hundred eligible subjects were randomized to placebo or one of three treatment arms of XP23829: 400 mg or 800 mg once daily (QD) or 400 mg twice daily (BID). The 12-week treatment period included a three-week titration period followed by nine weeks of treatment at the targeted dose. There was a washout phase of up to four weeks prior to randomization for subjects who were previously taking systemic agents for the treatment of psoriasis. Treatment assignment was stratified based on prior biologic use and approximately 35% of randomized subjects had previous experience with biological treatments for their psoriasis.
The primary endpoint of the study was the percent change from baseline to week 12 in PASI score. Least squares mean percent reductions in PASI score at 12 weeks were as follows:
400 mg QD 800 mg QD 400 mg BID Placebo
Least squares mean -38.1 -48.2 -50.7 -25.0
p-value 0.066 0.001 <0.001
XP23829 was safe and generally well tolerated. There were no deaths or life-threatening adverse events. No subjects met the safety discontinuation criteria and the majority of treatment emergent adverse events were non-serious and mild or moderate in severity. Diarrhea adverse event rates were consistent with other drugs in the fumaric acid ester class ranging from 22% to 40% in the XP23829 treatment groups compared with 15% for placebo. Other treatment emergent adverse events occurring at an incident rate of greater than or equal to 10% were nausea, abdominal pain, vomiting and headache. The incidence of flushing in the XP23829 dose groups was similar to placebo. Gastrointestinal events were the most frequent adverse event leading to withdrawal during XP23829 treatment. There were two treatment emergent serious adverse events assessed as possibly related to treatment with XP23829: acute cholecystitis and enterocolitis. Both subjects recovered.
No subjects experienced Grade 3 or Grade 4 lymphopenia. Less than 5% of subjects in any XP23829 treatment group reached Grade 2 lymphopenia and less than 15% reached Grade 1 at any visit. Lymphocyte levels in all subjects experiencing lymphopenia returned to within normal limits after treatment.
"I'm excited to have participated in this phase 2 study with positive efficacy data that we believe justifies further development of XP23829 into moderate-to-severe chronic plaque-type psoriasis," stated Alice Bendix Gottlieb, M.D., Ph.D., Dermatologist-in-Chief; Harvey B. Ansell Professor of Dermatology, Tufts University School of Medicine and Lead Investigator for the XP23829 trial. "Despite the wide availability of biologics, there still remains a significant unmet medical need for a more effective, safe, well-tolerated, and convenient oral treatment for patients with psoriasis. Fumarates have been the leading treatment for psoriasis in Germany for more than 2 decades. With this long-term real-world fumarate experience and these data in consideration, I look forward to the potential of seeing XP23829 in Phase 3 development for moderate-to-severe chronic plaque-type psoriasis."
"We are extremely pleased by the preliminary top-line results from this Phase 2 study. I want to thank the clinical investigators and the psoriasis sufferers who participated in the study. We look forward to getting the complete data set for this trial later this month and to presenting more comprehensive results at future medical conferences and in publications," stated Ronald W. Barrett, Ph.D., chief executive officer of XenoPort.
Dr. Barrett continued, "In the near future, we intend to share these data with psoriasis and multiple sclerosis experts, speak with regulatory authorities regarding next steps and explore potential partnerships that could accelerate the development of XP23829 globally. We recently completed non-clinical development studies and manufacturing activities necessary to support Phase 3 development and we believe we will be ready to potentially initiate Phase 3 studies in 2016."
3D Systems (NYSE:DDD) announced today that it will be hosting a customer webinar on how 3D design software and 3D printing from 3DS can deliver affordable, custom prosthetics and prosthetic fairings that are personalized and individualized.
Amedica Corporation (Nasdaq:AMDA), a company that develops and commercializes silicon nitride ceramics as a biomaterial platform, today announced its expected participation in the following investor conference: 2015 Ladenburg Thalmann Healthcare Conference, Tuesday, September 29, 2015, 11:30 AM Eastern Time, New York, NY, Dr. Sonny Bal, Chairman and CEO, will conduct a formal presentation and participate in one-on-one meetings.
Amgen (NASDAQ: AMGN) will participate in the Bank of America Merrill Lynch Global Healthcare Conference on Friday, Sept. 18, 2015, in London, beginning at 9 a.m. Greenwich Mean Time. David W. Meline, executive vice president and chief financial officer at Amgen, will present at the conference. Live audio of the presentation can be accessed from the Events Calendar on Amgen's website, www.amgen.com, under Investors. A replay of the webcast will also be available on Amgen's website for at least 90 days following the event.
Amicus Therapeutics (Nasdaq:FOLD), a biotechnology company at the forefront of therapies for rare and orphan diseases, today announced that a Pre-NDA meeting was held with the U.S. Food and Drug Administration (FDA) to discuss the oral small molecule pharmacological chaperone migalastat for the treatment of Fabry disease.
Bellerophon Therapeutics, Inc. (Nasdaq:BLPH), a clinical stage biotherapeutics company, today announced changes to its leadership team to align with the Company's focus of progressing its nitric oxide therapy program for patients suffering from Pulmonary Hypertension (PH), using Bellerophon's proprietary INOpulse® delivery system.
BG Medicine, Inc. (Nasdaq:BGMD), the developer of the BGM Galectin-3(R) Test, today announced that on September 14, 2015, the Company was notified by The NASDAQ Stock Market LLC ("NASDAQ") that trading in the Company's common stock will be suspended on NASDAQ effective with the open of business on Wednesday, September 16, 2015, due to the Company's continuing non-compliance with the stockholders' equity requirement set forth in NASDAQ Listing Rule 5550(b) as of September 10, 2015.
BioRestorative Therapies, Inc. (OTCQB:BRTX), a life sciences company focused on stem cell-based therapies, today announced that the Company has entered into a one-year research collaboration agreement with the University of Pennsylvania.
Care Advantage, McKesson Specialty Health’s national specialty pharmacy, received full Specialty Pharmacy Accreditation from URAC, a Washington, D.C.-based healthcare accrediting organization that assesses quality standards for the healthcare industry. URAC’s Specialty Pharmacy Accreditation provides a symbol of excellence for organizations to showcase their validated commitment to quality and accountability.
Cesca Therapeutics Inc. (NASDAQ:KOOL), an autologous cell-based regenerative medicine company, today announced that it has undertaken a restructuring initiative geared towards reducing costs associated with its traditional cord blood banking business and further aligning available resources behind the Company's high impact clinical programs, including the Phase III pivotal clinical trial for no option critical limb ischemia (CLI) patients, recently approved by the FDA.
Cytori Therapeutics(NASDAQ: CYTX), today announced that the presentation and corporate update given on September 10 by its President and CEO Marc Hedrick, M.D., is now available for on-demand viewing at VirtualInvestorConferences.com.
DENTSPLY International Inc. (NASDAQ:XRAY) and Sirona Dental Systems Inc. (NASDAQ:SIRO) today announced that the Boards of Directors of both companies have unanimously approved a definitive merger agreement under which the companies will combine in an all-stock merger of equals.
The Female Health Company (NASDAQ:FHCO), manufacturer of the world's leading female condom, the FC2 Female Condom® (FC2), announces the achievement of a significant milestone: the Company has now sold more than 500 million female condoms in more than 144 countries since FHC's first generation product, FC1, was introduced in 1995.
Gordagen Pharmaceuticals, an Australian private company commercializing evidence-based nutraceuticals and pharmaceuticals based on tocotrienols, today announced the appointment of Natural Alternatives International, Inc. (NAI) in San Marcos, California to manufacture its nutraceutical products for the US market.
Halfpenny Technologies Inc. (“HTI”) announced the latest feature expansion of their Intelligent Clinical Data Exchange Platform to provide mobile messaging notifications to health plans. The HTI - Hospital Utilization Notification Solution provides rapid notice of, and information regarding, health plan member’s hospital events including emergency room or inpatient admissions, changes in level of care, transfer to another facility, and discharge.
Hemispherx Biopharma (NYSE MKT:HEB) announced today the publication in the current issue of the peer reviewed Journal of Clinical and Cellular Immunology (Strayer D, et al, J Clin Cell Immunol 2015;6:4 http://doi.org/10.4172/2155-9899.1000348) an article entitled "Low NK Cell Activity in Chronic Fatigue Syndrome (CFS) and Relationship to Symptom Severity" in which 17 studies were reviewed that evaluated NK cell cytotoxicity (NKCC) data and the relationship to different CFS case definitions and CFS disease severity.
INC Research Holdings, Inc. (Nasdaq:INCR), a leading global Phase I to Phase IV contract research organization, today announced the time of its presentation at the Morgan Stanley Global Healthcare Conference in New York has changed. The presentation is now scheduled for 12:15 PM EDT on Friday, Sept. 18.
Integra LifeSciences Holdings Corporation (Nasdaq:IART) today announced that Integra filed a lawsuit in the U.S. District Court for the District of Delaware against HyperBranch Medical Technology, Inc. The lawsuit alleges that HyperBranch's Adherus Autospray Dural Sealant, Adherus Dural Sealant, and Adherus Spinal Sealant infringe patents that Integra licenses from Incept LLC.
Marathon Pharmaceuticals, LLC, a biopharmaceutical company focused on developing treatments for rare neurological and movement disorders, today announced that Dr. Jordan Dubow has joined the company as chief medical officer and vice president of clinical and medical affairs. In his new role, Dr. Dubow will oversee clinical development, medical affairs activities and drug safety for Marathon’s rare disease treatments.
MRI Interventions, Inc. (OTCQB:MRIC) today announced that its ClearPoint Neuro Navigation System will be on exhibit at the Congress of Neurological Surgeons (CNS) 2015 Annual Meeting September 26 - 30, 2015, in New Orleans, LA and will also be featured in the Innovations Showcase.
Natus Medical Incorporated (NASDAQ:BABY) today announced that Jonathan Kennedy, Senior Vice President and Chief Financial Officer, will present at the Morgan Stanley Global Healthcare Conference in New York City.
Novavax, Inc., (Nasdaq:NVAX) a clinical-stage vaccine company focused on the discovery, development and commercialization of recombinant nanoparticle vaccines and adjuvants, today announced its RSV F-protein recombinant nanoparticle vaccine candidate (RSV F Vaccine) was shown to be highly immunogenic in a guinea pig model of maternal immunization. The preclinical study was published in the journal Vaccine.
Octapharma USA today announced the U.S. Food and Drug Administration (FDA) has approved NUWIQ®, Antihemophilic Factor (Recombinant), an intravenous therapy for adults and children living with Hemophilia A. The NUWIQ® approval includes on-demand treatment and control of bleeding episodes; routine prophylaxis to reduce the frequency of bleeding episodes; and perioperative management of bleeding.
Optivia Biotechnology Inc., a leading global provider of high-quality transporter assay services and innovative transporter research solutions, announces the launch of its Opti-Target™ Transporter Screening Panel. The new panel will assist in the discovery and development of new classes of transporter-targeted therapeutics; including CNS disorders, oncology, and metabolic diseases. It also aids researchers in adopting a transporter-conscious strategy to improve drug response and safety.
Pharmaceutical Product Development, LLC (PPD) today announced the company earned recognition for excellence in multimedia and digital communications that promote the people and technology vital to advancing the development of life-changing medicines. PPD earned five honors in the annual Telly Awards and three in the Digital Health Awards.
Perseon Corporation (Nasdaq:PRSN) today announced that data supporting the use of the company's cutting edge microwave ablation technology, MicroThermX, was presented at the 38th Congress of Italian Society of Oncological Surgery (SICO) in Naples, Italy from September 9 – 12, 2015.
Pfenex Inc. (NYSE MKT: PFNX), a clinical-stage biosimilars company, announced today that Dennis M. Fenton, Ph.D., has been appointed to the company's board of directors, deepening the manufacturing and product development expertise of the biosimilar company. Dr. Fenton, an industry pioneer, has over three decades of experience in the biotechnology industry.
Pfizer Inc. invites investors and the general public to view and listen to a webcast of a conference call with investment analysts at 10 a.m. EDT on Tuesday, October 27, 2015. The purpose of the call is to provide an update on Pfizer’s results, as reflected in the company’s Third Quarter 2015 Performance Report, to be issued that morning.
RXi Pharmaceuticals Corporation (NASDAQ: RXII), a biotechnology company focused on discovering and developing innovative therapies primarily in the areas of dermatology and ophthalmology, today announced that Dr. Geert Cauwenbergh, RXi's President and CEO, will be participating in the upcoming 15th Annual Biotech in Europe Forum for Global Partnering and Investment Conference, with an oral presentation and panel discussion. This Forum is recognized as the leading international stage for those interested in investing and partnering in the biotech and life sciences industry.
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, will present at the Singular Research 10th Annual Best of the Uncovereds Investor Conference, being held Thursday, September 17, 2015 at the Luxe Hotel in Los Angeles, CA.
Sigma-Aldrich Corporation (NASDAQ: SIAL) today announced that the Cerilliant® brand within its Applied Diagnostics and Testing business segment now offers the first-ever reference solutions for qualitative evaluation of drug interferences in a testing laboratory's analytical methods. These seven multi-component solution mixes contain more than 50 of the most routinely monitored over-the-counter (OTC) and prescription drugs. Cerilliant offers the seven mixes separately or as a kit for added convenience.
SWK Holdings Corporation (OTCQB:SWKH), a life science focused specialty finance company, announced that the previously disclosed 1-for-100 reverse stock split of its common stock, immediately followed by a 10-for-1 forward stock split of its common stock was executed effective as of 5:00 p.m. on September 15, 2015.
Stereotaxis, Inc. (NASDAQ:STXS), a global leader in innovative technologies for the treatment of cardiac arrhythmias, announced today that the NASDAQ Capital Market has established September 15, 2015 as the "ex-warrants" date for its previously-announced warrants offering.
T2 Biosystems, Inc. (Nasdaq:TTOO), a company developing innovative diagnostic products to improve patient health, today announced that new data on the company's T2Candida® Panel will be presented at the Interscience Conference on Antimicrobial Agents and Chemotherapy (ICAAC) and the International Society of Chemotherapy (ICC) joint meeting in San Diego, September 17-21, 2015.
TapImmune, Inc. (OTCMKTS: TPIV), congratulates Mayo Clinic for being awarded a grant of $13.3 million from the U.S. Department of Defense. This grant, commencing September 15, 2015, will cover the costs for a 280 patient Phase II Clinical Trial of Folate Receptor Alpha Vaccine in patients with Triple Negative Breast Cancer.
Technavio has published a new report on the global anti-aging products market, which is expected to grow at a CAGR of nearly 8% from 2015-2019.
TriVascular Technologies, Inc. (NASDAQ:TRIV) announced today that the first European patients have been treated successfully with the Ovation iX Abdominal Stent Graft System in Germany and England. Building off of the clinically proven Ovation® platform, the Ovation iX System provides physicians with options for greater procedural consistency and ease of use.
Veritas Genetics, a pioneer in improving the accessibility of genetic testing, is establishing an R&D center in the Hangzhou Economic & Technological Development Area (HEDA), Hangzhou, China, and expanding its China-based leadership team. The R&D center will be dedicated to developing products that address the unique genetic needs of Asian populations.
Vical Incorporated (Nasdaq:VICL), a biotechnology company developing vaccines and therapeutics for prevention and treatment of infectious diseases, today announced that the Company will present a poster summarizing results from preclinical studies of its antifungal compound, VL-2397 (formerly known as ASP2397), at the upcoming Interscience Conference of Antimicrobial Agents and Chemotherapy (ICAAC) and the International Congress of Chemotherapy joint meeting in San Diego, California.