Aquinox Offers Added Exploratory Analyses from LEADERSHIP Phase 2 Trial; FDA Approves Octapharma’s NUWIQ® Print
By David Fowler   
Friday, 18 September 2015 19:30
Below is a look at some of the headlines for companies that made news in the healthcare sector on September 18, 2015.
Aquinox Pharmaceuticals, Inc. (NASDAQ: AQXP), a clinical-stage pharmaceutical company discovering and developing targeted therapeutics in disease areas of inflammation and immuno-oncology, today presented additional exploratory analyses from its LEADERSHIP Phase 2 clinical trial in patients with bladder pain syndrome/interstitial cystitis (BPS/IC) at the 2015 International Society for the Study of BPS (ESSIC) Conference taking place in Rome, Italy. Pre-specified primary and secondary endpoint results from the LEADERSHIP trial were reported on June 25, 2015 and August 6, 2015, respectively.
   
Additional LEADERSHIP data presented today included exploratory analyses of responder rates for average daily pain and maximum daily pain. Evaluating patients that achieved at least a 30% and 50% reduction in their pain demonstrated that AQX-1125 approximately doubled the response rate compared to those patients that received placebo for both average and maximum daily pain. Further analyses also revealed that the two components of the O'Leary-Sant Interstitial Cystitis Symptom Index (ICSI) and the Problem Index (ICPI) each individually demonstrated a statistically significant improvement in favor of AQX-1125 compared to placebo.
   
"It is reassuring to see that these additional analyses consistently support the benefit of AQX-1125 observed in the primary and secondary endpoint results already reported," said Dr. Stephen Shrewsbury, CMO and Senior VP of Clinical Development. "We are pleased to present these encouraging results to the BPS/IC community attending ESSIC and we look forward to discussing these results with regulators, including the FDA, and the wider urology community as we advance towards pivotal trials."
   
Data presented at ESSIC also included baseline demographic information demonstrating the trial to be well balanced between AQX-1125 and placebo groups in terms of age, duration of diagnosis, concomitant medications and baseline scores for both pain (11-point NRS) and urological symptoms (O'Leary-Sant ICSI/PI & BPIC-SS). On the 11-point NRS scale, mean baseline scores for average daily pain on entry were 6.4 for patients randomized to AQX-1125 and 6.7 for placebo recipients. Mean baseline scores for maximum daily pain on entry were 7.6 for patients randomized to AQX-1125 and 7.9 for patients given placebo. On the combined 36-point O'Leary-Sant indices, mean baseline scores were 27.3 for patients randomized to AQX-1125 and 30.2 for patients given placebo and on the 38-point BPIC-SS mean baseline scores on entry were 29.6 for AQX-1125 and 31.6 for placebo. A majority of patients in both arms (~64%) were on background medications, including approximately 30% on Elmiron, for treatment of their BPS/IC. The results from LEADERSHIP are particularly compelling given that the baseline demographics represent a predominantly severe BPS/IC patient population, a majority of which were on background medications and yet still experiencing moderate to severe symptoms at study entry. The benefit from AQX-1125 compared to placebo is notable as patients in both groups were allowed to remain on most forms of background medications throughout the LEADERSHIP trial.
  
  
   
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Octapharma USA today announced the U.S. Food and Drug Administration 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.
    
NUWIQ® is the first B-domain deleted recombinant Factor VIII (FVIII) derived from a human cell-line, not chemically modified or fused with another protein, designed for the treatment of patients with Hemophilia A, congenital FVIII deficiency. Hemophilia A impacts the lives of up to 16,000 individuals in the U.S. and their caregivers. Although present therapies for Hemophilia A treatment exist in the U.S., significant challenges still remain, including development of inhibitors and the need for multiple infusions on a prophylactic basis. Octapharma USA is a subsidiary of global human protein products manufacturer Octapharma AG, which develops and manufactures high-purity recombinant and plasma-derived coagulation factor concentrates for patients with bleeding disorders.
    
“Octapharma has been committed to the bleeding disorders community for many years and its decade-long drive to find solutions for Hemophilia A challenges has never wavered,” said Octapharma USA President Flemming Nielsen. “Early development strategies were integral in the development of NUWIQ® and these initial goals have been realized with the FDA approval. NUWIQ® has demonstrated safety and efficacy in global clinical trials and has the potential to positively impact patients’ quality of life in the years ahead. Octapharma is dedicated to providing life enhancing and saving therapies for Hemophilia A and looks forward to bringing NUWIQ® to the U.S. marketplace.”
    
The European Commission first approved the therapy in August 2014. NUWIQ® is currently approved in many countries, including the United Kingdom, Australia, Canada, Germany, Italy and Sweden.
    
“We are pleased that the treatment options for adults and children with Hemophilia A continue to advance with ever more innovative therapies being approved for the U.S.,” said Val Bias, chief executive officer of the National Hemophilia Foundation (NHF). “The continued commitment to develop life-enhancing products for the bleeding disorders community is absolutely vital. Empowering patients and providers with treatment options, as well as education and support programs, is extremely important to people living with Hemophilia A.”
     
The initial global clinical study program for NUWIQ® commenced with a pharmacokinetic (PK) evaluation in an open-label, multi-center clinical trial of 22 (20 adults, 2 adolescents) previously treated patients (PTPs). In this study, NUWIQ® demonstrated a mean half-life of 17.1 hours using a one-stage clotting assay in adults.  NUWIQ® was also evaluated in children using a one-stage clotting assay with a mean half-life of 11.9 hours for ages 2 to 5; and a mean half-life of 13.1 hours for ages 6 to 12. These PK results for mean half-life were longer than earlier generations of recombinant FVIII products currently available in the U.S.
    
The second set of global clinical studies for NUWIQ® also evaluated overall efficacy and tolerability in three prospective, open-label clinical studies in PTPs with severe Hemophilia A. Across all clinical studies, a total of 135 patients with Hemophilia A were treated with NUWIQ®, including 74 adults, 3 adolescents between ages 12 and 17, and 58 pediatric patients between ages 2 and 11. These 135 patients were treated with a total of 16,134 infusions over 15,950 exposure days using NUWIQ®.
    
In a study of 32 adults, overall prophylactic efficacy of NUWIQ® for spontaneous bleeds was rated as excellent or good in 92% of patients. In a study of 59 children, prophylactic efficacy for spontaneous bleeds was rated as excellent or good in 97% of patients. The mean annualized bleeding rates (ABR) for spontaneous bleeds during prophylaxis were approximately 1.5 in children and 1.2 in adults. For Hemophilia A patients receiving NUWIQ® prophylaxis compared to on-demand treatment, the ABR was reduced 96% for adults and 93% for children. Treatment of breakthrough bleeds during NUWIQ® prophylaxis was rated as excellent or good in 30 of 30 (100%) bleeds in adults and for 89 of 108 (82%) bleeds in children. For on-demand treatment with NUWIQ® in 20 adults and 2 adolescents, efficacy for the treatment of bleeds was excellent or good in 931 of 986 (94%) bleeds. Overall efficacy in surgical prophylaxis was rated excellent or good in 32 of 33 (97%) procedures using NUWIQ®.1
     
In all clinical studies, NUWIQ® had a total of 7 reported adverse events. Each of these adverse events occurred one time with a rate of 0.7% across all 135 patients. These events were parathesia, headache, injection site inflammation, injection site pain, back pain, vertigo, and dry mouth.
     
As part of its continuing commitment to the bleeding disorders community, Octapharma USA will offer Hemophilia A patients educational and support services in connection with the introduction of NUWIQ®. Octapharma USA aims to have NUWIQ® available in the U.S. marketplace by early 2016.   
    
    
Also Friday:
  
  
Balchem Corporation (NASDAQ:BCPC) announces the addition of Matthew D. Wineinger as director, expanding its Board of Directors to eight.   Mr. Wineinger is the President of United Sugars Corporation, a leading marketer of sugar. Before joining United Sugars in 2015, Wineinger served as President of Bulk Ingredients for Tate & Lyle PLC where he was responsible for global production and sales of $3.8 billion of commodity sweeteners, industrial starches, ethanol and acidulants.
   
BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), today announced that it has entered into a settlement agreement with Dr. Reddy's Laboratories, Inc. (Dr. Reddy's) that resolves patent litigation in the United States (U.S.) related to BioMarin's Kuvan® (sapropterin dihydrochloride) 100mg oral tablets.   Under the terms of the settlement, BioMarin will grant Dr. Reddy's a non-exclusive license to its patents related to Kuvan to allow Dr. Reddy to market a generic version of sapropterin dihydrochloride 100mg tablets in the U.S. for the indications approved for Kuvan beginning at a confidential date in the future, but which is more than five years from today, or earlier under certain circumstances. Additional details of the agreement remain confidential.
   
Cannabis Science, Inc. (OTCQB: CBIS), a U.S. company specializing in cannabis formulation-based drug development, today announced that accomplished scholar, epidemiologist, and public health physician Allen A. Herman, M.B., Ch.B., Ph.D. has been named as the Chief Medical Officer (CMO) of CBIS. Dr. Herman already serves on the Scientific Advisory Board of CBIS.   Dr. Herman served as a project officer and program director at the National Institute of Health (NIH). For his work in maternal and child health Dr. Herman received the National Institute's Award of Merit. Dr. Herman designed the basis for a nationwide $160 million infant mortality reduction initiative and was the co-director of joint projects between the NIH and the Centers for Disease Control and Prevention (CDC). His work as the scientific director of a $25 million NIH Infant Mortality Reduction research program defined the basis for much of the current community-based maternal and child health work designed to provide a healthy start for families. In addition to his work on maternal and child health, Dr. Herman was a member of the World Mental Health Survey team and helped design, execute, analyze, and publish peer-reviewed scientific papers from the South African Stress and Health Study, the largest mental health survey in Africa.
   
Celsus Therapeutics Plc (NASDAQ:CLTX) and Volution Immuno Pharmaceuticals SA today announced that, following shareholder approval obtained at a general meeting of the shareholders of Celsus held on September 16, 2015, the previously announced acquisition by Celsus of Volution has closed. The combined company has changed its name to Akari Therapeutics, Plc and will trade on the NASDAQ Capital Market under the symbol "AKTX" beginning on September 21, 2015. Akari will focus on development and commercialization of life-transforming treatments for a range of rare and orphan autoimmune and inflammatory diseases caused by dysregulation of complement C5, including paroxysmal nocturnal hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (aHUS). 
   
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 that the Phase 1a/1b clinical trial combining FPA008 with OPDIVO® (nivolumab) in multiple tumor types was featured today in a trial-in-progress poster at the CRI-CIMT-EATI-AACR Inaugural International Cancer Immunotherapy Conference in New York City.  FPA008 is Five Prime's monoclonal antibody that inhibits colony stimulating factor-1 receptor (CSF1R). OPDIVO® is Bristol-Myers Squibb's PD-1 immune checkpoint inhibitor. The poster entitled "A Phase 1a/1b Study of FPA008 in Combination with Nivolumab in Patients with Selected Advanced Cancers" was presented by F. Stephen Hodi, Jr., M.D., of Dana-Farber Cancer Institute.
   
High Performance Beverage Co. (OTC PINK: TBEV) is pleased to announce they have officially initiated the sales launch of their first to market High Performance Sports Drink™.   The first phase of sales and marketing for their one of a kind and first to market sports drink will find their new beverage for sale on their corporate website www.hpbev.net and www.amazon.com.   High Performance Beverage is a leading edge sports beverage manufacturer catering to a wide range of athletes of all types and ages.
   
Matinas BioPharma Holdings, Inc. (OTCQB:MTNB), a clinical-stage biopharmaceutical company focused on identifying and developing safe and effective antifungal and anti-bacterial therapeutics for the treatment of serious and life-threatening infections, today presented data from a preclinical study demonstrating tissue targeting and penetration potential of MAT2203, an orally-administered, encochleated formulation of the broad spectrum antifungal medication amphotericin B (AmB).
   
Medytox Solutions, Inc. (OTCQB: MMMS) announces its partnership with "Moments of Change," an Innovations in Integrated Treatment conference by Foundations Events, which highlights developments in behavioral health for professionals and their patients. The holding company has signed on as an event partner and will highlight its new and innovative approaches to treatment and lab analysis.   "We listen to the needs of the industry to guide the development of our testing solutions, so participating in 'Moments of Change' is of paramount importance," said Seamus Lagan, CEO of Medytox Solutions, Inc. "By networking with other companies creating solutions for treatment professionals of all kinds -- as well as the professionals themselves -- we are able to anticipate changes in testing and treatment nationwide."
   
Momenta Pharmaceuticals, Inc. (Nasdaq:MNTA), a biotechnology company specializing in the characterization and engineering of complex drugs, today announced the appointment of Georges Gemayel, Ph.D. to its Board of Directors effective January 1, 2016.  Dr. Gemayel has over 25 years of experience in the global biopharmaceutical industry including management and executive positions at biopharmaceutical companies such as Roche Labs, Genzyme and Altus Pharmaceuticals.   “I am pleased to welcome Georges to Momenta’s Board of Directors during this exciting stage in our Company’s growth,” said Craig Wheeler, President and CEO, Momenta Pharmaceuticals. “We believe his extensive experience in biopharmaceutical product development and commercialization will benefit the Company as we continue to advance our robust product pipeline and work toward our goal of bringing multiple products to market in the coming years.”
   
Orgenesis Inc. (OTCQB: ORGS), a cell therapy and regenerative medicine company with a novel therapeutic technology dedicated to converting a patient's own cells into functioning insulin-producing cells as a treatment for diabetes, today announced it will present a poster session at the European Society of Gene and Cell Therapy (ESGCT) Congress held in Helsinki, Finland from September 17-20, 2015.   The poster, which Orgenesis is presenting with its collaborator, Pall Life Sciences, is titled: "Industrialization of a Cell-based Autologous Therapy Targeting Diabetes: Industrialization of a Liver Cell Proliferation Process from Petri dish to the Xpansion™ ® Multiple Bioreactor." The poster will outline and discuss new technological innovations that allow a 1,000-fold expansion of liver-derived cells.
   
Profound Medical Corp. (TSXV:PRN), a medical device company developing and commercializing a unique, minimally invasive treatment to ablate the prostate gland in prostate cancer patients, announced today that it has granted 839,000 share options to employees of the Company, including 525,000 share options granted to certain officers. Each such share option entitles the holder thereof to acquire one common share in the capital of the Company at an exercise price of $1.50, being the equivalent of the issue price per subscription receipt issued in connection with the private placement that closed on April 30, 2015. The exercise price represented a premium to the market price of the Company's common shares on the TSX Venture Exchange on the date the options were granted. The options were issued with a term of 10 years.
   
Sientra, Inc. (NASDAQ:SIEN), a medical aesthetics company, today announced the pricing of its follow-on public offering of 3,000,000 shares of its common stock at a price to the public of $22.00 per share. In addition, Sientra has granted the underwriters a 30-day option to purchase up to 450,000 additional shares of common stock on the same terms and conditions.   Piper Jaffray and Stifel are acting as joint book-running managers for the offering. Leerink Partners and William Blair are acting as co-managers.
   
Summit (NASDAQ:SMMT) (AIM:SUMM), the drug discovery and development company advancing therapies for Duchenne Muscular Dystrophy and C. difficile infection ('CDI'), announces new preclinical data on SMT19969, a novel and selective oral antibiotic for the treatment of CDI, will be presented at the 55th Interscience Conference on Antimicrobial Agents and Chemotherapy ('ICAAC 2015') being held in San Diego, USA from September 17-21, 2015.   The data being presented are from preclinical in vitro studies that provide further evidence to support SMT19969's profile as a potential treatment for CDI and ability to reduce the high rates of recurrence currently associated with the disease. SMT19969 was shown to have high potency against 107 clinical isolates of C. difficile selected to maximise the diversity of their resistance to common classes of antibiotics, and SMT19969 also continues to display a low resistance development profile.
   
TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer, wet age-related macular degeneration and fibrotic diseases, today announced results from two oral presentations, one by Dr. Kevin Elias and one by Dr. Neil Horowitz, both of the Dana-Farber Cancer Institute and Harvard Medical School, at the 18th World Congress on Gestational Trophoblastic Diseases in Densapar, Bali, Indonesia. The single patient compassionate use Phase 2 clinical trial enrolled a 37-year old woman with persistent and unresectable metastatic choriocarcinoma, an aggressive form of gestational trophoblastic neoplasia (GTN). Prior to entry into the trial, the patient had received multiple treatments, including laparoscopic hysterectomy, five prior chemotherapy regimens and stem cell transplant.
  
Ubiquitech Software Corp. (OTC PINK: UBQU), an International Technology and Services company that also owns the division HempLife Today™, is announcing the Company's name change to CannaMark Brands™, Inc., a name that better reflects the Company's current business plan and future opportunities that are in development.   "We are very pleased to adopt the name CannaMark Brands™, Inc. We believe it better reflects our objective to nurture innovative solutions that enhance the quality of life for all through investments and operations in various strategic growth areas," said CEO, James Ballas. With CannaMark Brands™, Inc., the Company and all of its growing divisions will now be aligned under one parent company in support of rapidly growing alternative trends and technologies. "Now that we are becoming a company with more defined brands, merchant sites, customer bases and advertising specialties, the new name of CannaMark Brands™ is a much better fit, and better reflects the direction of our company," added Mr. Ballas.
   
Versartis, Inc. (NASDAQ:VSAR), an endocrine-focused biopharmaceutical company that is developing somavaratan (VRS-317), a novel, long-acting form of recombinant human growth hormone (rhGH) for growth hormone deficiency (GHD), today announced additional data from its ongoing Extension Study of somavaratan in pre-pubertal GHD children.   The complete 18-month data will be presented at a late-breaker poster presentation at the upcoming 54th Annual Meeting of the European Society for Paediatric Endocrinology (ESPE) taking place in Barcelona, Spain on October 3, 2015.
  
ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on the development and commercialization of new cancer immunotherapies, today announced the presentation of clinical and preclinical data from the Company's Ad-RTS-IL-12 program in various malignancies at the CRI-CIMT-EATI-AACR Inaugural International Cancer Immunotherapy Conference taking place September 16-19, 2015 in New York City. Ad-RTS-IL-12 is a novel gene therapy candidate for the controlled expression of IL-12, a critical protein for stimulating an anti-cancer T cell immune response.



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