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International Blood/Plasma News© Is Protected by Copyright Law EDITOR: KEITH BERMAN PUBLISHER: PATRICK ROBERT ISSN 0742-7719 Published by: The Marketing Research Bureau, Inc. 284 Racebrook Rd. Orange, CT 06477 VOLUME 35 ISSUE 12 JULY 2018 BUSINESS BRIEFS 166 BLOOD & BIOTECHNOLOGY 171 RESEARCH & DEVELOPMENT 173 PRODUCT SAFETY UPDATE 174 PEOPLE 175 NEW PRODUCTS 176 RECENT U.S. PATENTS 177 MEETINGS / SUBSCRIPTION FORM 180 ____________________COMPANIES IN THIS ISSUE__________________ ACCELERON PHARMA GRIFOLS ADMA BIOLOGICS HAEMONETICS ALKAHEST HARPOON THERAPEUTICS AMAG PHARMACEUTICALS HEMARUS THERAPEUTICS ARSANIS BIOSCIENCES GmbH ICER ARTHREX INSERM BAYER HEALTHCARE JEFFREY MODELL FOUNDATION BIO PRODUCTS LABORATORY NOVO NORDISK BIOPROTEIN TECHNOLOGIES OCTAPHARMA BIOVERATIV PHARMING CCRE HOLDINGS PLATOD CELGENE REGEN LABS SA CERUS ROCHE CHINA BIOLOGICS RUBIUS THERAPEUTICS CITIC CAPITAL HOLDINGS SANGAMO THERAPEUTICS CORD BLOOD REGISTRY SANQUIN CORD:USE CORD BLOOD BANK SHIRE CRISPR THERAPEUTICS SHIRE SINGAPORE CRTS des Armées SK CHEMICALS CRYO-CELL INTERNATIONAL TERUMO BCT CSL BEHRING VERTEX PHARMACEUTICALS DOVA PHARMACEUTICALS VFMCRP GI PARTNERS VIFOR PHARMA Notice: International Blood/Plasma News© is Protected by Copyright Law. Reproduction or Photocopy of Any Part Without The Publisher‘s Permission is Prohibited by Law. Page 166 JULY 2018 international blood/plasma news BUSINESS BRIEFS * Reflecting strong global demand for human polyvalent immunoglobulin products, HAEMONETICS reported 10.6% growth in fourth quarter revenues for its Plasma busi- ness unit, to $111.6 million from $100.9 million in the same period in 2017. Plasma business revenues for the full fiscal year ended March 31, 2018 increased by 6.7%, or by 7.1% on a con- stant currency basis. Specifically for the North American market, Plasma business revenue growth was 14.2% in the fourth quarter and 8.5% for fiscal year 2018. Fourth quarter fiscal 2018 operating income was $14.3 million (with an operating margin of 6.1%), compared with an operating loss of $57.5 million in the prior year quarter (and an operating margin of -25.2%). “In fiscal 2018, we focused on reorganizing and transforming our company. We launched our Complexity Reduction Initiative, strengthened our talent base and received key regulatory clear- ances in our Plasma business,” said Haemonetics’ CEO Chris Simon. “We enter fiscal 2019 with confidence in our planned product launches and company-wide productivity efforts, and we are poised for accelerated growth.” * ADMA BIOLOGICS announced the closing of a public offering of 8,368,200 shares of its com- mon stock at a public offering price of $4.78 per share. The gross proceeds from the offering are approximately $40 million before deducting underwriters’ discounts and commissions and other offering expenses. Included among planned uses of net proceeds are (1) continued remediation and ongoing improvement and enhancements at its plasma fractionation facility located in Boca Raton, Florida, (2) to submit a Prior Approval Supplement for, and re-launch of, Bivigam, (3) to resubmit the Biologics License Application (BLA) for its intravenous immunoglobulin product candidate, RI-002. * The U.S. FDA announced that an emergency use authorization (EUA) has been granted to the U.S. Department of Defense (DoD) to enable the emergency use of Pathogen-Reduced Leukocyte-Depleted Freeze-Dried Plasma (“French FDP”) manufactured by the Centre de Transfusion Sanguine des Armées. Under this EUA, the use of French FDP is authorized for the treatment of hemorrhage or coagulopathy of U.S. military personnel during an emergency involving agents of military combat (e.g., firearms, projectiles, and explosive devices) when plasma is not available for use or when the use of plasma is not practical. Hemorrhage, sometimes accompanied by coagulopathy, is the leading cause of preventable deaths among combat trauma casualties. French FDP can be used following reconstitution in settings where refrigeration is not available, thus enabling the rapid availability of plasma for use at the point of injury. Prior to freeze-drying, the product is pathogen reduced using CERUS’ INTERCEPT System for Plasma. It can be stored for up to two years from the date of manufacture at temperatures between 2°C (36°F) and 25°C (77°F). “Through our collaborative program with the DoD, they’ve made clear the importance of access to freeze-dried plasma in initial efforts to control hemorrhage from battlefield trauma. Granting this authorization will support access to this important product in the event it’s needed,” said U.S. FDA Commissioner Scott Gottlieb, MD. Page 167 JULY 2018 international blood/plasma news * CHINA BIOLOGICS’ board of directors has received an unsolicited, preliminary, non- binding letter from CCRE HOLDINGS, a wholly-owned subsidiary of CITIC CAPITAL HOLDINGS, proposing to acquire all of the outstanding shares of the company not already owned by CITIC for $110 in cash per share, or about $3.7 billion. In a news release, China Biologics’ board said it is “reviewing and evaluating CITIC’s proposal and no decisions have been made with respect to the Company’s response to the proposal,” and that “there can be no assurance that any definitive offer will be made, that any agreement will executive or that this or any other transaction will be approved or consummated.” * The U.S. FDA has approved BIO PRODUCTS LABORATORY’S Biologics License Application (BLA) for new 5% and 25% human albumin products, to be marketed under the name ALBUMINEX. The 5% solution is approved in 250 mL and 500 mL glass vials, and the 25% solution is approved in 50 mL and 100 mL glass vials. Both products are indicated for hypovolemia, ascites, hypoalbuminemia including from burns, acute nephrosis, acute respiratory distress syndrome and cardiopulmonary bypass. * Cambridge, Massachusetts-based RUBIUS THERAPEUTICS announced pricing of its initial public offering of 10,483,000 shares of common stock at a public offering price of $23.00 per share. In addition, Rubius has granted its underwriters a 30-day option to purchase up to an ad- ditional 1,572,450 shares at the IPO price, less underwriting discounts and commissions. Gross proceeds from the offering are expected to be $241.1 million. The company’s shares will trade on the NASDAQ Global Select Market under the ticker symbol “RUBY.” Rubius plans to use $95 million of the IPO proceeds to purchase and renovate a facility to manu- facture Red Cell Therapeutics (RCTs), applying its proprietary Rubius Erythrocyte Design (RED) platform. The RED platform genetically engineers and cultures CD34+ hematopoietic precursor cells from O-negative blood donors to express biotherapeutic proteins within the RCT cell or on the cell surface. About $42 million will reportedly be used to finance a Phase 1/2a clinical trial of its lead product, RTX-134, intended as a treatment for phenylketonuria (PKU); the company plans to file an Investigational New Drug application (IND) for RTX-134 during the first quarter of 2019. Remaining proceeds will fund additional R & D activities, including preclinical development of RCTs to treat certain rare diseases, cancers and autoimmune diseases. Prior to this IPO, Rubius had raised approximately $240 million, most recently including a $100 million crossover financing in early 2018 (see the March 2018 issue ofInternational Blood/Plasma News). * GI PARTNERS, a private equity investment firm, announced that it will acquire CORD BLOOD REGISTRY (CBR) from AMAG PHARMACEUTICALS for $530 million in an all-cash sale. The parties expect to close the transaction in the third quarter of 2018. Reportedly the world’s largest cord blood collection and storage firm, CBR currently stores cord blood stem cell samples from more than 600,000 children. Subject to customary closing conditions and regulatory approvals, the transaction is expected to close in the third quarter of 2018. Page 168 JULY 2018 international blood/plasma news * Separately, privately-held CRYO-CELL INTERNATIONAL announced that it has entered into an agreement to acquire Florida-based CORD:USE CORD BLOOD BANK for $14 million in cash and the issuance of common shares. * The U.S. Institute for Clinical and Economic Review (ICER) is currently assessing the comparative clinical effectiveness and economic outcomes of three approved C1 esterase inhibitor therapies and one investigational agent for the prevention of hereditary angio- edema attacks. This review will focus on long-term prophylaxis of patients with hereditary angioedema types 1 and 2. The approved products include HAEGARDA (CSL BEHRING), CINRYZE (SHIRE) and RUCONEST (PHARMING) and the investigational product is Shire’s lanadelumab, an investigational monoclonal antibody targeting plasma kallikrein that is currently under review by the U.S. FDA; an approval decision for lanadelumab is expected by September 21, 2018. A Draft Evidence Report is scheduled to be released on October 11, 2018, followed by a meeting for Evidence Presentation two weeks later, and publication of the Final Evidence Report and Meeting Summary on November 15, 2018. * Separately, a newly published analysis by the U.S. Institute for Clinical and Economic Review (ICER) reached several conclusions favoring the use of ROCHE’S Hemlibra (emi- cizumab) over bypassing agents (BPAs) as prophylaxis in persons with hemophilia A with inhibitors for whom immune tolerance induction (ITI) has been unsuccessful. In particular, prophylaxis with emicizumab: • Substantially reduces bleeding events compared to prior prophylaxis with BPAs; • Significantly
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