Mallinckrodt Diversifies Hospital Growth Portfolio, Acquiring Three Commercial-Stage, Global Specialty Hemostasis Brands From The Medicines Company

CHESTERFIELD, United Kingdom, Dec. 18, 2015 /PRNewswire/ -- Mallinckrodt plc (NYSE: MNK), a leading global specialty biopharmaceutical company, today announced that it has entered into a purchase agreement with The Medicines Company (NASDAQ: MDCO) under which subsidiaries of Mallinckrodt plc will acquire a global portfolio of three commercial-stage topical hemostasis drugs – RECOTHROM® Thrombin topical (Recombinant), PreveLeak™ Surgical Sealant, and RAPLIXA™ (Fibrin Sealant) – for an initial payment of approximately $175 million, inclusive of existing inventory. The transaction also includes potential added future considerations in the form of payments for achieving certain milestones, and is expected to be financed from cash on hand.

"Addition of this innovative portfolio of branded hemostats to our hospital platform is another demonstration of Mallinckrodt's 'acquire to invest' business strategy. Products that help control bleeding are essential to the vast majority of surgical practices. Yet this category – a market that has been estimated at $750 million in the U.S. and at least $1 billion globally – is an area in hospital products that has seen relatively little innovation and investment in recent years," said Mark Trudeau, Chief Executive Officer and President of Mallinckrodt.

Trudeau continued, "Acquiring and promoting leading hemostasis brand RECOTHROM alongside OFIRMEV® (acetaminophen) injection broadens Mallinckrodt's impact in the surgical suite for patients and physicians, and creates a strong framework to broadly commercialize innovative, highly durable new agents PreveLeak and RAPLIXA, which carry intellectual property protection to 2028 and 2031, respectively. As we continue to further diversify our portfolio, we're excited about the volume growth opportunity these products will add to our hospital offering for years to come."

On a reported U.S. GAAP1 basis, Mallinckrodt expects the transaction to be dilutive to its fiscal 2016 earnings per share (EPS); however, on an adjusted basis, the company expects the transaction to be neutral to EPS in fiscal 2016. On both a U.S. GAAP and adjusted basis, the company expects the transaction to be accretive to EPS beginning in fiscal 2017. Assuming a closing in the second quarter of fiscal 2016, the company expects the new products will add between $40 million and $45 million in incremental revenue in fiscal 2016, and provide low double-digit organic growth starting in fiscal 2017.

Through this acquisition, the company is expected to capitalize on synergies with Mallinckrodt's current U.S. commercial team focused on pain management in hospital surgical practice with OFIRMEV, the company's intravenous acetaminophen product indicated for the management of mild to moderate pain, and management of moderate to severe pain with adjunctive opioid analgesics. With expanded product offerings including OFIRMEV, RECOTHROM, PreveLeak and RAPLIXA, and with focused promotion, the company's commercial organization – supported by Mallinckrodt's strong sales, market access, national accounts and medical affairs teams – is expected to broaden access for patients who would benefit from these analgesic and hemostasis drugs.

Hemostasis Products

Each of the three drugs being acquired is approved by the U.S. Food and Drug Administration (FDA) and also in a number of other countries.

Introduced in 2009, RECOTHROM is widely used in the U.S., and is the first and only recombinant topical thrombin approved for use in adult and pediatric patients. It is FDA-approved as a topical thrombin indicated to aid hemostasis whenever oozing blood and minor bleeding from capillaries and small venules is accessible and control of bleeding by standard surgical techniques (such as suture, ligature, or cautery) is ineffective or impractical in adults and pediatric populations greater than or equal to one month of age. RECOTHROM is also marketed internationally through distributors.

Upon closing, Mallinckrodt will invest in the commercial launch and ongoing market development of both PreveLeak and RAPLIXA in fiscal year 2016. Both have shorter preparation times and are easier to use and store than similar products in their categories. PreveLeak is more flexible than hemostasis glue products, is a surgical sealant and is indicated for use in vascular reconstructions to achieve adjunctive hemostasis by mechanically sealing areas of leakage. PreveLeak is currently marketed in Europe through distributors. 

RAPLIXA is the first and only powder fibrin sealant in ready-to-use room temperature form, with the RAPLIXASpray™ device allowing for even application over a diffuse bleeding site and greater versatility. RAPLIXA is comprised of human-plasma derived fibrinogen and thrombin and FDA-approved as an adjunct to hemostasis for mild to moderate bleeding in adults undergoing surgery when control of bleeding by standard surgical techniques (such as suture, ligature and cautery) is ineffective or impractical. RAPLIXA is used in conjunction with an absorbable gelatin sponge (USP) and is applied directly or by using the RAPLIXASpray device.

FDA Accepts Biologic License Application for Fibrocaps Hemostatic Agent

The Medicines Company has announced that the U.S. Food and Drug Administration (FDA) has accepted the filing of a biologic license application (BLA) for the investigational hemostatic agent Fibrocaps, a dry powder formulation of fibrinogen and thrombin being developed to aid in hemostasis during surgery, where control of mild or moderate bleeding by conventional means is ineffective or impractical. The FDA action date (PDUFA date) for Fibrocaps is January 31, 2015. 

In August 2013, The Medicines Company announced that the Phase III trial, FINISH-3, which studied a total of 719 patients across 54 sites in the U.S. and Western Europe, met all of its primary and secondary hemostasis efficacy endpoints in each of four distinct populations: (1) spinal surgery; (2) hepatic resection; (3) soft tissue dissection; and (4) vascular surgery. 

"We believe Fibrocaps has the potential to become an important hemostatic product -- complementary to Recothrom® Thrombin, topical (Recombinant) -- which will allow us to continue to serve leading US hospitals, leveraging our existing operations. Our previously announced EMA filing also suggests that Fibrocaps can be our first hemostat product in the European market," said Adam Sharkawy, PhD, Senior Vice President, and Surgery and Perioperative Care Global Innovation Group Leader at The Medicines Company. 

"With the acceptance of the Fibrocaps BLA we now have 6 new molecular entity regulatory submissions under review at the FDA and the EMA," said Clive Meanwell, MD, PhD, Chairman and Chief Executive Officer of The Medicines Company. "These applications span our three areas of focus in leading hospitals, namely: acute cardiovascular care, surgery and perioperative care, and serious infectious disease care. Each is designed to contribute to our purpose which is to save lives, alleviate suffering, and contribute to the economics of healthcare by focusing on the needs of leading hospitals worldwide."

Baxter submits application to FDA for pediatric indication of Rixubis to treat Hemophilia B

Baxter International has filed an application to the US Food and Drug Administration (FDA) for a pediatric indication for Rixubis [Coagulation Factor IX (Recombinant)] to treat hemophilia B.

The submission was based on a Phase II/III clinical trial, designed to assess the efficacy and safety of Rixubis in 23 previously-treated male patients less than 12 years of age with severe or moderately severe hemophilia B.

In 2013, the company had secured FDA approval for Rixubis in the US for adults with hemophilia B and it had filed for marketing approval in Europe in November.

During the trial, patients were treated with a twice-weekly Rixubis prophylaxis regimen (median dose 56 IU/kg) over six months or for a minimum of 50 exposure days (EDs).

The company said that the median annualized bleeding rate (ABR) was 2.0 (0.0 for spontaneous bleeds and joint bleeds).

In the trial nine patients (39.1%) experienced no bleeds and 23 patients (88.5%) were treated with 1-2 infusions.

The company said that out of the 26 bleeds seen in the trial, only two (in two patients) were spontaneous and no reports of inhibitor development, no allergic reactions, and no thrombotic or treatment-related adverse events were observed among the study participants.

Baxter BioScience vice president of global research and development Anders Ullman said the positive results among a pediatric patient population are consistent with those observed in the Rixubis pivotal study among adult patients with hemophilia B.

"We submitted these data as part of our application for a pediatric indication for RIXUBIS to advance effective therapeutic solutions for children with hemophilia B," Ullman said.

European Medicines Agency Accepts Marketing Authorization Application for The Medicines Company's Fibrocaps

PARSIPPANY, NJ--(Nov 21, 2013) - The Medicines Company (MDCO) today announced that the European Medicines Agency (EMA) has accepted for review a marketing authorization application (MAA) for the investigational hemostatic agent Fibrocaps (human plasma-derived fibrinogen and thrombin). Fibrocaps was studied in the 719-patient Phase III FINISH-3 clinical trial as an adjunct to hemostasis in patients undergoing surgical procedures when control of mild or moderate bleeding by conventional surgical techniques is ineffective or impractical. 
The acceptance of the MAA marks the beginning of the review process in the European Union for Fibrocaps. The Company anticipates submitting a biologics license application (BLA) with the United States Food and Drug Administration in the first quarter of 2014. The Company also plans to submit a 510(k) application with the FDA for the complementary spray delivery device to assist surgeons in the accurate application of the dry powder Fibrocaps. The device was recently granted a European CE mark. 
"We believe Fibrocaps can become an important hemostatic solution within our surgery and perioperative care portfolio upon regulatory approval," said Jan Ohrstrom, MD, Senior Vice President Global Launch Leader, Hemostasis Solutions of The Medicines Company. "We are expanding our activities in surgery in pursuit of our purpose which is to save lives, alleviate suffering, and contribute to the economics of healthcare."
About The Medicines Company
The Medicines Company's purpose is to save lives, alleviate suffering, and contribute to the economics of healthcare by focusing on 3000 leading acute/intensive care hospitals worldwide. Its vision is to be a leading provider of solutions in three areas: acute cardiovascular care, surgery and perioperative care, and serious infectious disease care. The company operates in the Americas, Europe and the Middle East, and Asia Pacific regions with global centers today in Parsippany, NJ, USA and Zurich, Switzerland.
Forward-looking Statements
Statements contained in this press release about The Medicines Company that are not purely historical, and all other statements that are not purely historical, may be deemed to be forward-looking statements for purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, the words "believes," "plans, "anticipates" and "expects" and similar expressions, including the Company's preliminary revenue results, are intended to identify forward-looking statements. These forward-looking statements involve known and unknown risks and uncertainties that may cause the Company's actual results, levels of activity, performance or achievements to be materially different from those expressed or implied by these forward-looking statements. Important factors that may cause or contribute to such differences include whether the Company will make regulatory submissions for product candidates on a timely basis, whether its regulatory submissions will receive approvals from regulatory agencies on a timely basis or at all, whether physicians, patients and other key decision makers will accept clinical trial results, and such other factors as are set forth in the risk factors detailed from time to time in the Company's periodic reports and registration statements filed with the Securities and Exchange Commission including, without limitation, the risk factors detailed in the Company's Registration Statement on Form 10-Q filed on November 5, 2013, which are incorporated herein by reference. The Company specifically disclaims any obligation to update these forward-looking statements.

Baxter completes patient enrollment in phase III trial of BAX 855, extended half-life rFVIII to treat haemophilia A

Baxter International Inc. has completed enrollment in its phase III clinical trial of BAX 855, an investigational extended half-life, recombinant factor VIII (rFVIII) treatment for haemophilia A. The ongoing trial is aimed at assessing the efficacy of the compound in reducing annualized bleed rates (ABR) in both prophylaxis and on-demand treatment schedules, and will also evaluate its safety and pharmacokinetic profile.

BAX 855 was designed based on the full-length ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method] molecule, a product with 10 years of real-world experience. The BAX 855 molecule was modified with PEGylation technology designed to extend its duration of activity in the body.

"The BAX 855 development programme is a priority for Baxter as we evaluate the potential to provide an efficacious and safe treatment with an extended half-life for patients with hemophilia," said Anders Ullman, MD, Ph.D., vice president of global research and development in Baxter's BioScience business. "We are focused first and foremost on strategies to address optimal efficacy and minimize patients' bleeding episodes, while at the same time delivering on the convenience of less frequent dosing for this population with severe disease."

The phase II/III multi-centre, open-label study called PROLONG-ATE is evaluating BAX 855 among 146 adult patients with previously-treated severe haemophilia A. Patients participating in PROLONG-ATE receive treatment twice weekly (45 IU/kg) and are followed for six months. The primary endpoint of the study is the annualized bleed rate (ABR) during the treatment period. The study is also evaluating the safety and immunogenicity of the compound when administered on either prophylaxis and on-demand treatment regimens. Other outcome measures include number of infusions needed to treat bleeding episodes, time intervals between these episodes, pharmacokinetics and patient reported outcomes. To date, no inhibitors or safety issues have been reported in the study.

Based upon the results of the study, the company expects to complete the trial and file for regulatory approval late in 2014. Baxter is also initiating a continuation study for all patients who complete the pivotal phase II/III study, and expects to initiate a study of BAX 855 among pediatric patients in 2014.

The treatment protocol is based on the results of a phase I trial of BAX 855, assessing its safety, tolerability and pharmacokinetics. That trial found that the half-life (measuring the duration of activity of the drug in the body) of the investigational compound was approximately 1.5-fold higher compared to ADVATE. An extended half-life was achieved in all patients in the study using BAX 855, no patients developed inhibitors to either the base molecule, BAX 855 or to PEG, and no patients had allergic reactions. No treatment-related or serious adverse events were reported, and no patients withdrew from the study due to adverse events.

BAX 855 is built from the same native FVIII protein used in the production of ADVATE, and employs proprietary PEGylation technology from Nektar Therapeutics designed to extend the duration of activity of proteins. PEGylation technology has been widely used in various approved treatments.

ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method] is indicated for the control and prevention of bleeding episodes in adults and children (0-16 years) with haemophilia A. ADVATE is also indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children (0-16 years) with haemophilia A. ADVATE is not indicated for the treatment of von Willebrand disease.

ADVATE has a demonstrated efficacy profile and a low rate of inhibitor development. ADVATE is a full-length (derived from the complete FVIII gene) recombinant FVIII product that is processed without any blood-based additives. Because no blood-derived components are added at any stage of the manufacturing process, the potential risk of transmitting pathogens that may be carried in blood-based additives is eliminated. There have been no confirmed reports of transmission of HIV, HBV or HCV with rFVIII treatments.

ADVATE is approved in 60 countries worldwide including the United States, Canada, 27 countries in the European Union, Argentina, Australia, Brazil, Chile, China, Colombia, Croatia, Ecuador, Hong Kong, Iceland, Iraq, Japan, Kuwait, Macau, Malaysia, Mexico, New Zealand, Norway, Panama, Puerto Rico, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Tunisia, Turkey, Ukraine, Uruguay, and Venezuela.

Medicines Co. wraps $240M deal to buy ProFibrix following PhIII success

The Medicines Company has swooped in to buy out the Dutch biotech ProFibrix, chipping in $90 million in cash on top of a $10 million option anted up in June as Phase III data on a lead surgical therapy loomed. And the buyer has agreed to pay up to $140 million more for a set of unspecified milestones.

The Medicines Company ($MDCO) is landing Fibrocaps in the deal, an easy-to-use dry-powder formulation of fibrinogen and thrombin that can be used to stop bleeding during surgery, a process referred to as hemostasis. In their release today the companies noted that the lead biologic hit all primary and secondary endpoints on four surgical indications: spinal surgery, hepatic resection, soft tissue dissection and vascular surgery. Investigators recruited 719 patients for the Phase III study.

The plan now is to file for EU approval in the fourth quarter of this year, with an FDA application following in the first quarter of 2014, commented ProFibrix CEO Jan Ohrstrom. And The Medicines Company believes it can earn more than $300 million a year on the product, provided it wins key approvals.

"The company was founded in 2007 with an A round from Index Ventures," adds Ohrstrom. Phase I was run in 2008 and investigators are now wrapping a 5-year clinical program, with more work scheduled on laparoscopic surgery as well as a pediatric program. The CEO says more indications are also being "toyed with," but he declined to elaborate. The company's lab in Leiden will stay operational, says Ohrstrom, the former chief medical officer of ZymoGenetics. ProFibrix currently has 25 employees in Leiden and Seattle, where it has a clinical development site.

"Subject to regulatory approval, we believe Fibrocaps can become an important hemostatic product--complementary to Recothrom (Thrombin, topical [Recombinant])," says Clive Meanwell, the CEO at The Medicines Co. "We anticipate leverage of our work with U.S. surgery centers and entry into the European market. … ProFibrix also has a proprietary recombinant fibrinogen development program which potentially allows us to create the world's first recombinant thrombin and recombinant fibrinogen combination products. We plan to integrate the ProFibrix team with our existing Recothrom team--expanding our activities in surgery in pursuit of our purpose which is to save lives, alleviate suffering and improve the economic efficiency of leading hospitals worldwide."

The market for hemostatic products has been busy with new developments for years. Just a few weeks ago Omrix Biopharmaceuticals won a recommendation from the CHMP in Europe for the sealant matrix Evarrest, which also combines fibrinogen and thrombin. But The Medicines Company is hoping that ProFibrix's off-the-shelf technology will quickly win converts.

MDCO, Bristol-Myers Squibb begin global alliance for Recothrom

The global license and two-year collaboration signed between the Medicines Company (MDCO) and Bristol-Myers Squibb (BMS) for Recothrom, a recombinant thrombin approved by the US Food and Drug Administration (FDA) for use as a topical hemostat to control non-arterial bleeding during surgical procedures has become effective. The agreement was first announced in December 2012. The companies have satisfied all required regulatory and closing conditions. Glenn Sblendorio, president and chief financial officer of The Medicines Company, said, "We look forward to the start of our global license and collaboration for Recothrom. We believe that completing this deal is another step to establish The Medicines Company as a leader in acute and intensive care medicine globally." Recothrom is a surgical hemostat that is applied topically to stop bleeding during surgery. Its active ingredient is recombinant human thrombin (rThrombin) and the product is indicated as an aid to hemostasis whenever control of bleeding by standard surgical techniques (such as suture, ligature, or cautery) is ineffective or impractical. Recothrom is part of a class of surgical hemostats commonly referred to as "active" hemostats. Other classes of surgical hemostats include mechanical hemostats, flowable hemostats and fibrin sealants. Recothrom was approved in the United States in January 2008 and in Canada in December 2010. The Medicines Company provides medical solutions to improve health outcomes for patients in acute and intensive care hospitals worldwide.

The Medicines Company and Bristol-Myers Squibb Agree to Global Alliance For Recothrom

The Medicines Company builds on portfolio and capabilities in acute and intensive care hospital medicine with marketed perioperative biologic

Parsippany, NJ, and Princeton, NJ, USA I December 12, 2012 I The Medicines Company (NASDAQ:MDCO) and Bristol-Myers Squibb Company (NYSE: BMY) announced today that the companies have signed a global license and two year collaboration for Recothrom®, a recombinant thrombin approved by the U.S. Food and Drug Administration for use as a topical hemostat to control non-arterial bleeding during surgical procedures.

Clive Meanwell, M.D., Ph.D., Chairman and CEO of The Medicines Company said, “As a marketed, growing product to control blood loss in the hospital, we believe Recothrom is a strategic fit that enables operating leverage with The Medicines Company’s emerging perioperative care portfolio. The financial structures and net revenues fit well with our strategy for aggressive and sustainable growth in acute and intensive care medicine. Of course, thrombin is also a hemodynamic target we know well based on our experience with Angiomax® (bivalirudin), a thrombin inhibitor.”

“The Medicines Company’s expertise in advancing the treatment of critical care patients in hospital settings worldwide makes it the natural partner to bring Recothrom to patients and physicians,” said Giovanni Caforio, President, U.S. Pharmaceuticals of Bristol-Myers Squibb. “This agreement is part of Bristol-Myers Squibb’s ongoing efforts to simplify operations, improve our efficiency and better position ourselves to focus on our important work in areas of significant unmet medical need that are critical to our long-term success."

Recothrom net revenues in 2011 were $65 million. The product is currently commercially available in the United States and Canada. The intellectual property license agreement is global and The Medicines Company anticipates pursuing approvals in additional countries.

The transaction is expected to be accretive to earnings per share (EPS) for The Medicines Company in 2013.

Under terms of the agreement, The Medicines Company will pay Bristol-Myers Squibb an upfront collaboration payment of $105 million and an upfront option fee of $10 million. The Medicines Company has also agreed to pay Bristol-Myers Squibb a tiered royalty on annual net revenues of Recothrom during the two-year collaboration term. Bristol-Myers Squibb will retain responsibility for the manufacturing of Recothrom and will be The Medicine Company’s exclusive supplier of Recothrom during the term of the agreement. The option enables The Medicines Company to acquire the Recothrom assets for a purchase price based on average net sales during the two-year collaboration term.

The transaction is expected to be minimally accretive to EPS for Bristol-Myers Squibb in 2013 and 2014.

The transaction is subject to the satisfaction or waiver of closing conditions, including the expiration or termination of applicable waiting periods under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, and the delivery by Bristol-Myers Squibb of certain audited financial information relating to the business.

Baxter Submits Application for FDA Approval of Recombinant Factor IX for the Treatment of Hemophilia B

DEERFIELD, Ill. - Baxter International Inc. (NYSE:BAX) today announced that the company has submitted a biologics license application (BLA) to the United States (U.S.) Food and Drug Administration (FDA) for approval of BAX 326, a recombinant factor IX (rFIX) protein being investigated for the treatment and prophylaxis of bleeding episodes for patients over 12 years of age with hemophilia B.

Hemophilia B, also known as Christmas disease, is the second most common type of hemophilia and results from insufficient amounts of clotting factor IX, a naturally occurring protein in blood that helps to control bleeding. 1 Approximately 25,000 people worldwide, including more than 4,000 in the U.S., have been diagnosed with hemophilia B. 2

The BLA filing is based on results from a global Phase III study conducted in 10 countries around the world. The prospective, controlled, multicenter study evaluated the pharmacokinetics, efficacy, safety and immunogenicity of BAX 326 in 73 patients with severe or moderately severe hemophilia B previously treated with other factor IX therapy. The study met its primary objectives and the company plans to present the complete data from the study in late 2012. Baxter expects to file its application for BAX 326 in Europe in 2013.

"Hemophilia B patients have relatively limited options for their treatment today, with only one commercially available recombinant (genetically engineered) protein. As part of our long-standing commitment to the hemophilia community, we continue to pursue new potential treatment options like BAX 326 to support patients with this debilitating disease," said Prof. Hartmut J. Ehrlich, M.D., vice president of global research and development in Baxter's BioScience business.

In select countries, Baxter currently offers a plasma-derived factor IX treatment, Immunine [Factor IX Concentrate (Human)], for patients with hemophilia B, which has more than 16 years of patient experience in Europe and Latin America. In addition, Baxter recently announced a partnership with Chatham Therapeutics, LLC to develop a gene therapy based treatment for hemophilia B. Gene therapy could represent another important first for the community as an innovative potential therapy for hemophilia B treatment.

Symphogen Publishes Final Rozrolimupab (SYM001) Phase 2 Trial Results in ITP in BLOOD Journal

COPENHAGEN, DENMARK — The hematology journal BLOOD published today a full-length article describing final Phase 2 data for Symphogen’s rozrolimupab, a novel human recombinant mixture of 25 antibodies which all are manufactured simultaneously from a single batch. The data demonstrates rozrolimupab’s favorable safety profile and its induction of a rapid increase in platelet counts in patients with Primary Immune Thrombocytopenia Purpura (ITP). Professor Tadeusz Robak, MD, University of Lodz, Poland, is the first author of the article entitled “Rozrolimupab, A Mixture of 25 Recombinant Human Monoclonal RhD Antibodies, in the treatment of Primary Immune Thrombocytopenia” which has been prepublished in First Edition of Blood and can be viewed on Blood Online at http://bloodjournal.hematologylibrary.org/content/early/recent.

The Phase 2 study was an open-label, multi-center clinical trial evaluating the efficacy, safety, and tolerability of rozrolimupab (SYM001) in adult, RhD positive, non-splenectomized ITP patients. A total of 61 patients were treated with single doses from 75µg/kg to 300µg/kg as single i.v. infusions of 15-20 minutes’ duration. The trial demonstrated that at 300µg/kg, 8 of 13 (62%) of patients responded at day 7. Already within 5 to 8 hours after rozrolimupab administration, 23% of the patients achieved platelet responses (≥ 30×10⁹/L and increase in platelet count by > 20×10⁹/L from baseline). Median time to response was 59 hours (approximately 2.5 days) and the median duration of response was 14 days.

The most common adverse events observed, were headache (20%), mostly mild or moderate, pyrexia (13%), chills (10%), and fatigue (8%). Four serious adverse events considered related to study drug were reported: decreased hemoglobin, extravascular hemolysis/dizziness and two cases of transient rise in D-dimer values without clinical symptoms.

According to Professor Robak, “These Phase 2 results suggest an efficacy and safety profile similar to that seen with plasma derived immunoglobulin products. It seems promising that rozrolimupab rapidly yields platelet responses. This unique recombinant human monoclonal antibody mixture, rozrolimupab can be produced indefinitely and may represent a novel and convenient replacement for blood-derived immunoglobulins with more limited supply.”

Kirsten Drejer, Symphogen chief executive officer, added, “Symphogen has reached an important milestone by generating clinical proof of concept for a product consisting of a mixture of 25 monoclonal antibodies. The multicenter study included involvement of the regulatory authorities of the USA, Europe and Asia, and we are confident that antibody mixtures represent a viable new class of antibody therapeutics offering well-characterized and potentially more efficacious alternatives to existing treatments.”

Baxter Announces ADVATE Approval in China for the Treatment of Hemophilia A

Baxter International Inc. announced the approval of ADVATE[Recombinant Human Coagulation Factor VIII for injection] for the control and prophylaxis of bleeding episodes in individuals with hemophilia A (congenital factor VIII deficiency) in China by the State Food and Drug Administration (SFDA). It is estimated that more than 50,000 people in China are living with hemophilia A.

''The introduction of recombinant FVIII therapies in China offers new treatment options for hemophilia patients. The launch of ADVATE is another step in advancing hemophilia care in China,'' said Professor Yang Renchi, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, the leading professional hematological institution providing basic medical research with clinical services in China.

''Great strides have been made in managing hemophilia, allowing people with this serious condition to live longer, more active and fulfilling lives than ever before,'' said Guan Tao, Secretary General of Hemophilia Home, the hemophilia patient organization in China. ''The availability of ADVATE will be an important milestone for people with hemophilia in China.''

ADVATE is infused directly into the bloodstream and works by temporarily raising the level of factor VIII in the bloodstream, allowing the body's blood clotting process to properly function. Extensive global use and multiple clinical trials demonstrate clinical evidence for ADVATE. With SFDA's action, ADVATE is now approved in 54 countries worldwide.

''The approval of ADVATE in China marks an important milestone for Baxter and supports our ongoing commitment to treating individuals living with hemophilia,'' said Ludwig Hantson, Ph.D., president of Baxter's BioScience business.

Baxter continues to work closely with the Chinese hemophilia community, including both patients and treaters, to provide access to care for this life-saving, life-sustaining therapy. In 2010, Baxter cooperated with the Ministry of Health to set up a ''Hemophilia Disease Management System,'' China's first nationwide hemophilia patient registration and management system integrating diagnosis and treatment information. In recent years, Baxter has donated more than five million IUs of hemophilia products to Chinese patients and has provided a number of resources to raise awareness of the disease.

About ADVATE

ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method] was initially approved by the FDA in July 2003 for control and prevention of bleeding episodes in adults and children (0-16 years) with hemophilia A. ADVATE is a full-length (derived from the complete FVIII gene) recombinant FVIII product that is processed without any blood-based additives. Because no blood derived components are added at any stage of the manufacturing process, the potential risk of transmitting pathogens that may be carried in blood-based additives is eliminated. There have been no confirmed reports of transmission of HIV, HBV or HCV with rFVIII therapies.

ADVATE is approved in the United States, Canada, 27 countries in the European Union, Argentina, Australia, Brazil, Chile, China, Colombia, Croatia, Hong Kong, Iceland, Iraq, Japan, Macau, Malaysia, New Zealand, Norway, Panama, Puerto Rico, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Uruguay and Venezuela.

In the United States, ADVATE [Antihemophilic Factor (Recombinant) Plasma/AlbuminFree Method] is also indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children (0-16 years) with hemophilia A. ADVATE is the only antihemophilic factor approved in the United States for prophylactic use in both adults and children. ADVATE is not indicated for the treatment of von Willebrand disease.

ProFibrix Phase II with FibrocapsTM Meets Primary Endpoint – On Track for Phase III in 2012

LEIDEN, The Netherlands & SEATTLE--(BUSINESS WIRE)-- ProFibrix B.V., a leader in the development of innovative products for hemostasis, today announced that its multicenter Phase II clinical trial with Fibrocaps in liver resection surgery resulted in a highly statistically significant 50% reduction in mean time to hemostasis (TTH) compared to active control.
A total of 56 patients were enrolled in the company’s Dutch prospective, randomized, controlled, multi-center Phase II study with lead product Fibrocaps for mild to moderate surgical bleeding. The study results show that Fibrocaps has a very good safety profile along with rapid hemostatic activity that succeeds in significantly reducing mean time to hemostasis, the primary end point of the Phase II trial.
Professor dr. R.J. Porte from the University Medical Center Groningen, the Lead Investigator of the study, said: “We’re very pleased with the excellent performance of Fibrocaps in this Phase II study. I have no doubt that the substantial reduction in mean time to hemostasis we report for Fibrocaps is clinically very meaningful, and demonstrates the strength of this unique dry powder formulation of fibrinogen and thrombin.”
Jan Öhrström, CEO of ProFibrix said: “Our clinical data constitute the strongest result ever reported for fibrin sealants in a Phase II study in this indication. These positive results ensure that we remain on track to initiate a pivotal Phase III trial in H1 2012, and target a BLA filing in 2013. In the coming months we expect to report on the results of the Phase II trial we are running in parallel in the U.S. Altogether we believe that the positive outcome of the European and U.S studies should allow us to generate strong support from investors as we continue developing Fibrocaps and its line extensions.”

Recombinant Human Albumin for Use in Unique Sealant Technology

Novozymes Biopharma is pleased to announce that its recombinant human albumin, albucult®, is being used by Neomend in its ProGEL® PLATINUM surgical sealant.
This product will be commercially available in Europe beginning in the second quarter of 2011. Neomend is an innovator in sealant and adhesion prevention products for the surgical marketplace. The company utilises albucult as a key component in ProGEL PLATINUM, the only sealant of its kind developed to treat pleural air leaks following lung resection surgery. Neomend’s ProGEL is a hydrogel polymer consisting of Novozymes’ albucult and a cross-linking component of polyethylene glycol (PEG). When they are combined, a rapid cross-linking reaction creates a hydrogel matrix that results in the formation of a strong, adherent and flexible seal. The technology, which has received a European CE Mark, enables surgeons to more effectively treat lung air leaks during surgery and has already been used in more than 8,000 procedures in the US since April of last year.
David Renzi, Neomend’s President and CEO, commented: “Novozymes’ extensive experience in manufacturing, regulatory and global supply, combined with its commitment to innovation, makes the company the ideal partner for Neomend to work with on its unique sealant technology. We will launch ProGEL PLATINUM in Europe in the second quarter of this year, while also continuing the development of this platform technology into other clinical areas such as spine, general surgery and gynecology.”
“Novozymes is delighted that Neomend has selected albucult for use in its unique sealant technology”, said Dermot Pearson, Marketing Director, Novozymes Biopharma. “The partnership demonstrates Novozymes’ commitment to providing quality materials for use in the healthcare, pharmaceutical and biotechnology industries and allows the company to position itself as a reliable partner in the wound sealant and medical device markets.” The use of albucult in ProGEL PLATINUM and other medical devices confers a range of unique advantages not possible with animal-derived ingredients such as sustainability of supply, GMP compliance and improved biocompatibility. Albucult also delivers unprecedented performance and quality benefits to medical applications including sealants, device coating and cell therapy applications while ensuring batch-to-batch consistency, which can significantly reduce lot testing burdens for customers.

Stanford's ideas generate $65.1 million in revenues

A new report card for one of the nation's most powerful innovation engines shows that Stanford-based inventions generated $65.1 million in income for the university in 2009 despite the recession -- up from $62.5 million the previous year.
Stanford's total earnings from inventions were $1.1 billion during the past four decades, with more than half coming from just two inventions: the hypertext searching used by Google and groundbreaking DNA-splicing technology, according to an annual survey by the Association of University Technology Managers.
No longer isolated "ivory towers," schools like Stanford harvest great ideas and then try to send them out in the world. There they can be turned into commercial products -- and reward the campus with royalty income from licensing rights.
The two most lucrative inventions are decades old and are no longer producing revenue. Recombinant DNA, a 1974 invention, creates artificial DNA through gene splicing and brought in $225 million; Google's "PageRank" tool, patented in 1996, brought in $336 million.
Every school dreams of the next Gatorade -- a simple mixture of water, sugar, lemon juice, sodium, potassium, and phosphate that has yielded the University of Florida more than $80 million since 1973.
But instead of bringing home the bacon, many universities throw money into the void with little hope of return.
There's a chasm between a good idea and a product, termed "the valley of death."
Patents are expensive, with fees and legal costs involved in obtaining a single patent range from $20,000 to $25,000. Because it takes so long to bring a product to market, the royalties are based on license deals done 10 to 15 years ago.
Stanford ranks 10th in annual earnings among the nation's campuses, according to the new survey. By comparison, the University of California system had 47 startup companies and $103 million in royalties.
Nationally, scientific research from about 150 universities created 555 startup companies and resulted in more than 4,500 patent optioning and licensing deals last year, earning $1.8 billion in payouts.
Stanford's royalty revenue in 2009 came from 517 different technologies, generating from $3 million to $38 million. Its current big money-maker is an antibody invention, which led to the development of many valuable drugs.
But the statistics are sobering: After Google's PageRank and recombinant DNA inventions, "the rest of it is a bunch of technologies that generated much less income," said Katharine Ku, Stanford's Director for Technology Licensing, in a rare public presentation to the school's faculty senate last year. Only 19 inventions brought in more than $5 million. About 58 earned about $1 million, she said.
Fewer than half of the 300 research universities actively seeking patents have managed to break even from technology transfer efforts. Instead, two-thirds of the revenue tracked by the association has gone to only 13 institutions, including Stanford and UC.
Stanford's other best ideas, among the 7,400 inventions total, are FM Sound Synthesis, which led to the ringing of cell phones; recombinant DNA, used to make vast amounts of proteins like insulin, among other products; MINOS, an optimization software program; functional antibodies and DSL, which provides digital data transmission over the phone wires.
Ku recalled when Stanford engineering students Larry Page and Sergey Brin took their research project, based on a new search technology, to her office in hopes of finding a company interested in licensing their invention. Stanford marketed it to every potential licensee it could think of -- without success. So Page and Brin created their own company, and licensed the PageRank tool from Stanford. In exchange, Stanford was given Google stock.
Further reading

Profibrix - Market Summary

The Business of Recombinant Protein Production

Since the development of recombinant technology in the late 1970s, the use of recombinant proteins in therapeutics has become an attractive strategy for altering the biology of disease progression. In the early 1980s, recombinant human insulin from Escherichia colibecame the first recombinant pharmaceutical to enter the market. Development of several growth hormones produced in bacteria followed, as did production of monoclonal antibodies such as infliximab and ritxumiab from mammalian cell lines. By January of 2009, the number of protein-based recombinant pharmaceuticals licensed by the U.S. Food and Drug Administration and European Medicines Agency reached 151.1
 Although recombinant protein technology provides a wealth of commercial opportunities, bringing a recombinant protein to the marketplace requires a substantial investment of time and resources. Because the synthesis and purification processes are technically complex and vary with each protein, estimating resources and timelines can be challenging at best. Yet for small companies, meeting these challenges is vital, says Vladimir Kostyukovsky, senior technical manager at Kymanox, Inc. “With start ups, you need to hit your milestones by a certain date, or you’re out of business.”
The most common expression systems for recombinant pharmaceuticals are derived from bacteria (usually E. coli), yeast (usually Saccharomyces cerevisiae), or mammalian cells. Key properties of individual expression systems affect convenience and quality of production. Characteristics inherent to a target protein also affect production capabilities. Making the transition from producing a laboratory stock of protein to commercial quantities also requires strategic planning and flexibility.
Protein synthesis and purification Microbial systems versus mammalian systems
Recombinant protein production in microbial systems (mainly bacteria or yeast) tends to be faster and cheaper than in mammalian systems. The robust cellular structure of bacteria and yeast make them amenable to culturing, but mammalian cells—derived from multicellular organisms—are not adapted to survive outside the body and are thus sensitive to external conditions like shear stress and osmotic shock. Bacteria and yeast replicate every 20 minutes to 2 hours, while mammalian cells do so every 24 hours to 2 days. Mammalian cells require complex—often proprietary—media with amino acids. Bacterial cell medium consists of simple carbon and nitrogen sources and inorganic salts. The cost for bacterial cell media is 90% lower than for mammalian Chinese hamster ovary (CHO) media. Fermentation or bioreactor runs take 24 to 72 hours in bacteria or yeast versus 14 days to 3 weeks in CHO cells.
Yeast and mammalian cell lines have inherent mechanisms for secreting properly folded, active proteins in culture medium. In E. coli, synthesized protein accumulates internally, and cell lysis is required to isolate the target protein. The process of lysis releases intracellular contents such as proteases or endotoxins, which can decrease yield and complicate purification. Recently, a strain of the gram-positive Corynebacterium glutamicum, which does not have endotoxins, has been engineered to express correctly folded, active recombinant proteins directly into the extracellular fermentation broth.
The following characteristics of target proteins influence their likelihood of being expressed and purified in a functionally active state:
Protein size. Eukaryotic cells, such as mammalian and yeast, have the advantage of being able to express proteins of 200 to 250 kDa, the size of most monoclonal antibodies. In some cases, expression of proteins as large as 400 kDa is possible. In E. coli, it is difficult to obtain proteins larger than 60 kDa in soluble forms.
Protein complexity. The number of protein subunits is inversely related to the likelihood of functional expression. Again, eukaryotic cells have a greater advantage in producing fully assembled, functional, multidomain proteins. Monoclonal antibodies, which consist of 4 subunits, can be secreted by eukaryotic cells as fully assembled complexes. E. coli can be used to express multidomain proteins, but they often lack activity.
Glycosylation. Posttranslational addition of sugar motifs to proteins profoundly affects biological activity, function, clearance from circulation, and antigenicity. Patterns of glycosylation are highly species-specific, and mammalian cell lines have glycosylation repertoires most similar to humans.
Common Challenges
Potential complications lie at every step of the process of recombinant protein expression and purification. The complications vary with each specific protein, but because proteins share common motifs, certain technical pitfalls are encountered frequently.
Protein aggregation. Suboptimal conditions during protein expression, purification, or storage can alter structure and result in aggregation or loss of activity. In bacterial expression systems, intracellular accumulation of protein can lead to formation of inclusion body aggregates.
Aggregation can sometimes be reversed by adjusting media composition, growth temperature, inducer concentration, promoter strength, or plasmid copy number. However, inclusion bodies formed by proteins native to eukaryotes but produced in bacteria tend to be resistant to solubilization. Even proteins that are successfully solubilized can become denatured and thus require potentially complicated in vitro refolding processes.
Refolding. The process of refolding is often preceded by a partial purification to remove host cell proteins that could aggregate with the target protein. Because refolding techniques vary and are often empirical, the online database REFOLD provides refolding protocols that have been successful for a wide range of proteins.2
Proteolytic degration. Host proteases, which can degrade the target protein, are commonly active during cell disruption, as intracellular contents are released. Genetic engineering can be used to remove a host’s more active proteases.
Disulfide bond generation. Disulfide bridges, which stabilize protein structure, are often abundant in secreted proteins. As with refolding, disulfide bond generation and maintenance are achieved through empirical processes. Possible strategies include targeting a specific extracellular excretion pathway or overexpression of chaperones or foldases.
Contamination. Contaminants can originate from host cells or from growth medium. Contaminants can interfere with protein function or cause adverse reactions in human consumers, such as through human pathogens harbored in mammalian expression systems or endotoxins in gram-negative bacteria. The process of removing contaminants can be extensive—often involving several rounds of chromatography—and each additional step diminishes yield.
Making the Transition to Clinical Production
The early steps in developing a recombinant pharmaceutical often require multiple iterations to optimize expression and purification of functionally active protein. As development moves closer to commercialization, schedules, pricing, and other aspects of managing a supple chain become paramount. This transition from focusing on technical concerns to the context of the clinic and broader marketplace can be challenging.
Shailesh Maingi, CEO and president of Kineticos Life Sciences, suggests there are key issues to consider throughout the process of production. First, are you going to make the protein yourself or outsource production? “From a business perspective, this decision is probably the most critical,” Maingi says. “If you want to produce it yourself, there is a huge investment required, not only in money, but in time and resources. If you’re a small, virtual company, you’re almost always going to outsource. If you’re a large company, you’re almost always going to insource because you have the capability and you have the technology.”
Smaller companies have the option of producing a lab supply of protein that can be used in preclinical toxicology studies. Outsourcing for larger-scale production can be done prior to initiating clinical trials.
If you decide to outsource, how do you choose a vendor? At the stages of protein expression and purification, working with a vendor that is flexible in its approach is important, because it is impossible to predict how complex the process will be for a given protein. Any vendor should be able to provide a clear proof of concept and performance history of the technology. Cost, quality, and convenience are important considerations as well. During the expression phase, proximity to the vendor can provide convenience for evaluating efficiency and yield of the process.

In summary, while the path to producing a functionally active and meaningful recombinant pharmaceutical can lead to great commercial opportunity, it can also be plagued with technical pitfalls and failures. Careful consideration of various strategies at every step of the process can optimize use of time and resources.

Source: Joel White, Business Manager, Biotechnology; Ajinomoto AminoScience, Raleigh, North Carolina

Drug Discovery & Development - October 07, 2010

Kraig Biocraft Laboratories Announces Application for Trademark of Spider Silk ...

LANSING, MI, Oct 05, 2010 (MARKETWIRE via COMTEX) -- Kraig Biocraft Laboratories, Inc.(KBLB 0.15, +0.03, +22.92%) announced today that that it has filed intent to use applications with the US Patent and Trademark office seeking registration of numerous marks for the purpose of branding recombinant silk fibers.
"The Company views branding of our recombinant silk to be just as important as it is with any other product line. It is anticipated that the proposed marks will enhance product marketability and distinguish our advanced technology from other fibers in the marketplace," said Kim Thompson, the Company's CEO and founder. "This is just the beginning of the Company's efforts to further distinguish itself in the market place as we move toward the commercialization of our technology."
The intent to use applications included seven separate proposed marks including Spiderpillar(TM) and Monster Silk(TM).
The Company recently announced the successful development of recombinant spider silk based fibers using genetic engineering. 
The Company will also hold a shareholder press conference on Friday, October 8, 2010 at 4:30 p.m. EDT and will issue a reminder press release the morning of the conference call, which will include the phone number and access code instructions.

Bristol-Myers Squibb to Acquire ZymoGenetics

BMS to Pick Up ZymoGenetics for $885M
NEW YORK & SEATTLE, Sep 07, 2010 (BUSINESS WIRE) -- --Gains Full Rights to Promising Phase II Hepatitis C Biologic, Pegylated-Interferon Lambda

--Obtains FDA-Approved Specialty Surgical Biologic, RECOTHROM(R)

--Attains Early Clinical and Pre-clinical Programs in Oncology and Immunoscience

Bristol-Myers Squibb Company (BMY 26.78, +0.17, +0.64%) and ZymoGenetics, Inc.(ZGEN 9.76, +4.46, +84.06%) announced today that the companies have signed a definitive agreement providing for the acquisition of ZymoGenetics by Bristol-Myers Squibb, for $9.75 per share in cash. The transaction, with an aggregate purchase price of approximately $885 million, or approximately $735 million net of cash acquired, has been unanimously approved by the boards of directors of both companies. The board of directors of ZymoGenetics intends to recommend that ZymoGenetics' shareholders tender their shares in the tender offer. In addition, shareholders holding approximately 37% of the outstanding shares of ZymoGenetics' common stock have entered into agreements with Bristol-Myers Squibb to support the transaction and to tender their shares in the offer.

"The acquisition of ZymoGenetics brings us full ownership of a promising investigational biologic that strengthens our very diversified Hepatitis C portfolio. Building on our leadership in virology, we are developing a strong portfolio to help patients with Hepatitis C," said Lamberto Andreotti, chief executive officer, Bristol-Myers Squibb. "In addition, ZymoGenetics brings proven capabilities with therapeutic proteins and revenue from a marketed specialty surgical biologic. This acquisition is another example of our strategic, targeted approach to business development."

"By joining forces with Bristol-Myers Squibb, we believe we will enhance the long-term potential of ZymoGenetics' portfolio of assets, while providing a compelling valuation for our shareholders," said Douglas E. Williams, Ph.D., chief executive officer of ZymoGenetics. "Our collaboration with Bristol-Myers Squibb in the development of PEG-Interferon lambda has been extremely positive and it has given us an opportunity to fully appreciate their capabilities. We believe that this transaction will maximize the potential for our products and product candidates to make a meaningful difference for patients in need."

Bristol-Myers Squibb gains the following as a result of the acquisition:

-- Full ownership of pegylated-interferon lambda, a novel interferon in Phase IIb development for the treatment of Hepatitis C infection, which, if approved, could be an important contributor to Bristol-Myers Squibb's future growth. The companies have collaborated on the development of pegylated-interferon lambda since January 2009. Four-week and 12-week results from a Phase IIa study will be presented at the American Association for the Study of Liver Diseases meeting later this year.

-- RECOTHROM(R), a recombinant thrombin approved by the U.S. Food and Drug Administration for use as a topical hemostat to control non-arterial bleeding during surgical procedures.

-- IL-21 protein, a cytokine currently being tested in an open-label, Phase II clinical study as a potential immunotherapy treatment for metastatic melanoma.

-- An earlier-stage pipeline of six biologic drug candidates, including an anti-IL-31 antibody, currently in pre-clinical development for atopic dermatitis.

-- Potential milestone and royalty payments from six partnered programs in various stages of clinical development by EMD Serono, Inc., an affiliate of Merck KGaA, and Novo Nordisk.

"ZymoGenetics is a leader in advancing novel biologics, particularly genomics-based therapies," said Elliott Sigal, M.D., Ph.D., executive vice president and chief scientific officer, Bristol-Myers Squibb. "We expect ZymoGenetics' pipeline and biologics capabilities to complement and enhance our existing efforts in Hepatitis C, oncology and immunoscience."

Initially, the transaction is expected to be modestly dilutive to earnings per share (EPS) for Bristol-Myers Squibb. In 2010, the transaction is expected to be dilutive to EPS by approximately $0.03. In 2011, the transaction is expected to be dilutive to EPS by approximately $0.07.

Under the terms of the definitive agreement, Bristol-Myers Squibb will commence a cash tender offer on or about September 9, 2010 to purchase all of the outstanding shares of ZymoGenetics' common stock for $9.75 per share. The closing of the tender offer is subject to customary terms and conditions, including the tender of a number of shares which is equal to or greater than 48,282,192 shares (which represents approximately 56% of the outstanding shares as of August 31, 2010, which represent a majority of the shares on a fully-diluted basis, excluding certain shares underlying derivative securities that are significantly out-of-the-money), and the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. The agreement also provides for the parties to effect, subject to customary conditions, a merger to be completed following the completion of the tender offer which would result in all shares not tendered in the tender offer being converted into the right to receive $9.75 per share in cash. The merger agreement contains a provision under which ZymoGenetics has agreed not to solicit any competing offers for the company. Bristol-Myers Squibb will finance the acquisition from its existing cash resources. The companies expect the tender offer to close approximately thirty days after commencement of the tender offer.

Morgan Stanley & Co. Incorporated is serving as financial advisor to Bristol-Myers Squibb in connection with the acquisition, and Bristol-Myers Squibb is represented by Kirkland & Ellis LLP. Goldman, Sachs & Co. is serving as financial advisor to ZymoGenetics in connection with the acquisition, and ZymoGenetics is represented by Latham & Watkins LLP and Fenwick & West LLP.

Minnesotans Help Produce As Well As Benefit from Lifesaving Therapies

ANNAPOLIS, Md., Sept. 1 /PRNewswire/ -- This month, Minnesotans are recognized for their outstanding contributions to lifesaving therapies that treat critically ill individuals and for their donations of the blood component, plasma, that makes the creation of these unique therapies possible. Throughout September, Minnesota Gov. Tim Pawlenty is recognizing "Plasma Protein Therapies Month" in the North Star State, helping to raise awareness for the rare, genetic diseases treated with the therapies and valuing the contributions of voluntary plasma donors in the state.

Plasma protein therapies, which include plasma-derived therapies and recombinant blood clotting factors (a biotechnology product), are used every day to treat people with hemophilia, a blood clotting disorder that causes painful internal bleeding and debilitating joint damage; primary immunodeficiency diseases, which rob a person's ability to fight even common infections; and alpha-1 antitrypsin deficiency, also known as genetic chronic obstructive pulmonary disease (COPD). In addition, a plasma protein therapy, albumin, is used in critical care settings, when treating severe trauma, burns and during major surgery.

Plasma, a biological material that cannot be manufactured, is used to create lifesaving therapies that come solely from committed plasma donors. The state of Minnesota is home to 11 plasma collection centers. As Minnesota celebrates the generosity of its donors and raises awareness for the rare diseases that are treated with the therapies during Plasma Protein Therapies Month, we encourage everyone to donate and to help ensure a higher quality of life for patients across the United States and around the world.

"Always serious and often life-threatening, patients requiring plasma protein therapies suffer from serious diseases and disorders and need access to critical therapies manufactured from high-quality plasma from dedicated donors such as those in Minnesota," said Josh Penrod, Vice President, PPTA Source. "Donors continue to be recognized and valued as a critical component in the production of these therapies."

For more information about plasma protein therapies, the diseases they treat and donating plasma, please go to www.donatingplasma.org.

The Plasma Protein Therapeutics Association (PPTA) represents the world's leading manufacturers of plasma-derived and recombinant biological therapies, collectively known as plasma protein therapies and the collectors of source plasma. These critical therapies are infused or injected by more than 1 million people worldwide to treat a variety of rare, life threatening diseases and serious medical conditions. PPTA members produce in excess of 80 percent of the plasma protein therapies used in the United States today and more than 60 percent worldwide. PPTA is a global trade association that administers international, voluntary standards programs to help ensure the highest quality and safety of plasma protein therapies and the plasma collected to manufacture them. Additionally, PPTA works in partnership with the patient community and consumer advocates to help ensure continued access to lifesaving plasma protein therapies.

What is Recombinant - Molecular Engineering?