Sangamo BioSciences Announces Target Date For First IND For Sangamo’s In Vivo ZFN Genome-Editing Platform For Monogenic Diseases

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Timeline for Hemophilia B IND Application Filing Targeting Second Quarter 2015

RICHMOND, Calif., Sept. 10, 2014 /PRNewswire/ — Sangamo BioSciences, Inc.  announced plans to submit an Investigational New Drug (IND) application for the treatment of hemophilia B in the second quarter of 2015.  This program, partnered with Shire, is the first therapeutic application of Sangamo’s zinc finger nuclease (ZFN)-mediated In Vivo Protein Replacement Platform (IVPRP). Sangamo is developing the IVPRP as a generally applicable strategy to provide a one-time, permanent genetic cure for monogenic diseases that are currently managed by protein replacement therapy, which involves costly repeat infusions over the lifetime of the patient.

“Shire has been impressed by the rapid progress of the novel hemophilia B program developed by Sangamo,” said Albert Seymour, Ph.D., Senior Vice President and Head of Global Research and Nonclinical Development at Shire.  “The Shire team continues to work very closely with Sangamo to submit the IND package to support this program.”

“Sangamo’s research and development team has succeeded in rapidly advancing the hemophilia B program through small and large animal proof of concept experiments, as well as pioneering manufacturing and regulatory pathways,” said Edward Lanphier, Sangamo’s president and CEO.  “I am very pleased with the progress that we and the new Shire team have made as we prepare to file an IND application for this first IVPRP program. Our experience with hemophilia B informs and supports our follow-on programs that leverage our IVPRP strategy, including our Shire-partnered program for the treatment of  hemophilia A and Sangamo’s proprietary programs in lysosomal storage disorders (LSDs).  With the success of these programs, going forward, it is our goal to develop and file two to four new INDs per year for the foreseeable future.”

“With the recent acceptance of an IND for mRNA delivery of ZFNs in Sangamo’s SB-728-T program, upcoming initiation of Phase 1 clinical testing of our hematopoietic stem-cell program for HIV (SB-728-HSC), and the unanimous approval of our Phase 1 ZFN Bcl11a program yesterday, at the NIH Recombinant Advisory Committee (RAC) meeting, keeping us on track to file an IND application for our beta-thalassemia program by the end of the year, we are realizing our goal of developing a new class of  human therapeutics that have the potential to provide genetic cures for a range of monogenic diseases and unmet medical needs,” added Mr. Lanphier.

“We are successfully forging a path to the clinic for Sangamo’s ZFN-based IVPRP,” said Geoff Nichol, M.B., Ch.B., Sangamo’s executive vice president of research and development.  “We have gained valuable expertise in clinical scale AAV manufacturing and the regulatory requirements for IND submissions of the hemophilia B and other IVPRP programs.  We are pleased to move forward with an IND package for our hemophilia B program which supports Shire’s IND requirements and, building on preclinical proof-of-concept data in our hemophilia A program, will implement a similar strategy as we bring that program to IND.  The proprietary LSD programs that Sangamo is developing based on this platform are also progressing well and on schedule.  Together, these assets are moving us rapidly towards the establishment of a ZFN-mediated platform for permanently treating protein and enzyme deficiency diseases and our long-term goal of initiating two to four new clinical programs per year.”

Sangamo is targeting an IND application filing for hemophilia A by year end 2015 and anticipates providing updated guidance of the IND timeline for this program by the end of the first quarter of 2015.  The two hemophilia programs are the first in a platform of IVPRP assets that use intravenous AAV-delivered ZFNs to target the albumin locus and drive permanent expression of therapeutic proteins for a wide range of deficiency diseases, including the hemophilias, LSDs and other metabolic diseases.

Background on Hemophilia A and B and Sangamo’s IVPRP Approach
Patients with hemophilia have a genetic mutation in the gene that encodes a blood-clotting factor (factor VIII in the case of hemophilia A, and factor IX in the case of hemophilia B), and, as a result, cannot make sufficient active factor VIII or IX protein to ensure efficient blood clotting. Sangamo’s IVPRP is designed to provide a one-time, potentially curative treatment for patients with monogenic diseases, such as hemophilia and LSDs, who currently require repeated infusions of protein replacement therapies throughout their lives.  The IVPRP approach enables the patient’s liver to permanently produce therapeutic levels of a corrective protein product such as factor VIII or IX to treat hemophilia, or replacement enzymes to treat LSDs.

The IVPRP approach makes use of the albumin gene locus, a highly expressing and liver-specific genomic “safe-harbor site”, that can be edited with zinc finger nucleases (ZFNs) to accept and express any therapeutic gene. With such a large capacity for protein production (approximately 15g/day of albumin), which is in excess of the body’s requirements, targeting and co-opting only a very small percentage of the albumin gene’s capacity is sufficient to produce the needed replacement protein at therapeutically relevant levels with no significant effect on albumin production.

Pending acceptance of the IND application, a single intravenous infusion will be used to deliver adeno-associated viral (AAV) vectors encoding the ZFNs and a copy of a replacement gene used to correct the genetic defect. The zinc finger nucleases drive targeted, permanent insertion of a normal factor IX gene at the albumin locus in the liver cells of patients with hemophilia B.  This results in ongoing production of factor IX protein by the gene-edited liver cells under the control of the highly active albumin promoter.

About Sangamo
Sangamo BioSciences, Inc. is focused on Engineering Genetic Cures™ for monogenic and infectious diseases by deploying its novel DNA-binding protein technology platform in therapeutic gene regulation and genome editing. The Company has ongoing Phase 2 clinical trials to evaluate the safety and efficacy of a novel ZFP Therapeutic® for the treatment of HIV/AIDS (SB-728-T) and NGF-AAV for Alzheimer’s disease (CERE-110). Sangamo’s other therapeutic programs are focused on monogenic and rare diseases.  The company has formed a strategic collaboration with Shire International GmbH to develop therapeutics for hemophilia, Huntington’s disease and other monogenic diseases, and with Biogen Idec for hemoglobinopathies, such as sickle cell disease and beta-thalassemia. It has also established strategic partnerships with companies in non-therapeutic applications of its technology, including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the Company’s website at

ZFP Therapeutic® is a registered trademark of Sangamo BioSciences, Inc.

This press release may contain forward-looking statements based on Sangamo’s current expectations. These forward-looking statements include, without limitation, references relating to research and development of novel ZFP TFs and ZFNs and therapeutic applications of Sangamo’s ZFP technology platform for the treatment of hemophilia B and other monogenic diseases, the timing of submissions of INDs for the treatment of hemophilia B, hemophilia A and lysosomal storage diseases, and the timing of submissions of INDs for other partnered programs and Sangamo proprietary programs. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the tolerability and efficacy of ZFNs, technological challenges, Sangamo’s ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo’s public filings with the Securities and Exchange Commission, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo assumes no obligation to update the forward-looking information contained in this press release.

SOURCE Sangamo BioSciences, Inc.

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Pfizer Announces Positive Top-Line Results From Phase 3 Study Of NONACOG ALFA (BeneFIX®) Once-Weekly Prophylaxis For Hemophilia B

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Once-Weekly Prophylaxis Treatment with BeneFIX® Significantly Reduced Annualized Bleeding Rate Compared to On-Demand Treatment

Wednesday, July 16, 2014 – 7:00am EDT

Pfizer Inc. (NYSE:PFE) today announced the positive results of a Phase 3 study comparing a prophylaxis regimen of BeneFIX® Coagulation Factor IX (Recombinant) 100 IU/kg once-weekly to on-demand treatment in people with moderately severe to severe hemophilia B. The top-line results of the study showed that the primary study endpoint was met and hemophilia B patients taking once-weekly BeneFIX (100 IU/Kg) showed a statistically significant reduction in the annualized bleeding rate (ABR) (P < 0.0001) relative to on-demand treatment with BeneFIX.

In the study, the median ABR value, a commonly used measure of efficacy for prophylaxis regimens in hemophilia, was 2.0 for the prophylaxis regimen, compared to 33.6 for the on-demand regimen, representing a 94% decrease in bleeding rates. The mean ABR value was 3.6 for the prophylaxis period, compared to 32.9 for the on-demand treatment, which represents a reduction of 89% (P < 0.0001).

“These results are important because they add to the growing body of clinical evidence showing that prophylaxis treatment has the potential to reduce the number of bleeds in a year, the most critical factor in hemophilia management,” said Steven J. Romano, M.D., senior vice president and Medicines Development Group Head, Global Innovative Pharmaceuticals, Pfizer Inc. “Pfizer remains committed to the research and development of new and innovative products for the hemophilia community.”

Study results also showed that prophylaxis treatment significantly reduced both spontaneous and traumatic ABR compared to on-demand treatment with BeneFIX. The median spontaneous ABR value was 1.0 for the prophylaxis regimen, compared to 22.4 for the on-demand regimen, and the median traumatic ABR value was 1.0 for the prophylaxis period, compared to 4.1 for the on-demand treatment. In addition to meeting the primary endpoint, the secondary study endpoints showed that none of the 1,254 prophylaxis infusions administered during the study were associated with a less than expected therapeutic effect (LETE) occurrence, which was defined as a spontaneous bleed occurring within 48 hours of a prophylaxis infusion. The majority (82.1%) of bleeding episodes in the prophylaxis arm were resolved after one infusion.

Adverse events observed in the study, for both the prophylaxis and on-demand periods, were consistent with the known adverse event profile of BeneFIX. The most common adverse events reported during the prophylaxis treatment period were arthralgia (20%), upper respiratory infection (20%), toothache (20%), pyrexia (16%), headache (16%), pharyngitis (12%), back pain (12%) and local swelling (12%). No inhibitor development, thrombotic events or allergic reactions related to this product were observed in this study.

These results are preliminary, top-line data and are subject to additional analyses. Complete results from this study will be submitted for presentation at upcoming medical congresses and submitted for publication in a peer-reviewed journal.

Study Background

This study was a Phase 3, open-label, non-randomized two-period study consisting of six months of on-demand therapy only, followed by 12 months of routine prophylaxis with BeneFIX 100 IU/kg once-weekly. Participants included in this study were males with a mean age of 31.3 years (N=25) and moderately severe to severe hemophilia B (FIX:C ≤2%). Of the 25 participants enrolled in the study, all received at least one dose of study drug, and no participants discontinued treatment early. The mean duration of treatment was 550 days.

About BeneFIX

BeneFIX is a recombinant coagulation factor IX product indicated for the control, prevention and perioperative management of bleeding episodes in adult and pediatric patients with hemophilia B. BeneFIX received FDA approval in the U.S. on February 11, 1997, and was approved in the European Union later that year. It has been studied in clinical trials in both previously treated and untreated patients, and established in both on-demand and preventive care, additionally shown to help control bleeds in major and minor surgeries. BeneFIX is not approved for prophylaxis use in the United States.

BeneFIX Indications and Usage

BeneFIX is an injectable medicine that is used to help control and prevent bleeding in people with hemophilia B. Hemophilia B is also called congenital factor IX deficiency or Christmas disease.

BeneFIX is NOT used to treat hemophilia A.

Important Safety Information for BeneFIX

  • BeneFIX is contraindicated in patients who have manifested life-threatening, immediate hypersensitivity reactions, including anaphylaxis, to the product or its components, including hamster protein.
  • Call your health care provider right away if your bleeding is not controlled after using BeneFIX.
  • Allergic reactions may occur with BeneFIX. Call your health care provider or get emergency treatment right away if you have any of the following symptoms: wheezing, difficulty breathing, chest tightness, your lips and gums turning blue, fast heartbeat, facial swelling, faintness, rash or hives.
  • Your body can make antibodies, called “inhibitors,” which may interfere with the effectiveness of BeneFIX.
  • If you have risk factors for developing blood clots, such as a venous catheter through which BeneFIX is given by continuous infusion, BeneFIX may increase the risk of abnormal blood clots. The safety and efficacy of BeneFIX administration by continuous infusion have not been established.
  • Some common side effects of BeneFIX are nausea, injection site reaction, injection site pain, headache, dizziness and rash.

Please see full Prescribing Information for BeneFIX available at

About Hemophilia

Hemophilia is a type of bleeding disorder that causes the blood to take a long time to clot as a result of a deficiency in one of several blood clotting factors, and occurs almost exclusively in males. People with hemophilia B have a deficiency in clotting factor IX, a specific protein in the blood. Hemophilia B is also called congenital factor IX deficiency or Christmas disease. People with hemophilia face specific risks and need to be careful not to cause injury to their bodies, as injuries can prompt a bleed, which have the potential to be life threatening.

Pfizer Inc.: Working together for a healthier world®

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products. Our global portfolio includes medicines and vaccines as well as many of the world’s best-known consumer health care products. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, Pfizer has worked to make a difference for all who rely on us. To learn more, please visit us at


Media: Jennifer Kokell, (212) 733-2596
Investor: Ryan Crowe, (212) 733-8160

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LFB announces First patient has begun treatment with Factor VIIa (Recombinant) in Phase III Clinical Trial in Hemophilia A and B with inhibitors

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Posted on 15 May 2014 by ER

FRAMINGHAM, MA, May 14, 2014 – LFB SA, through its U.S. subsidiary, announced today that the first patient has begun treatment with LR769 in a Phase 3 clinical trial of this novel recombinant form of human Factor VIIa in patients with congenital hemophilia A or B with inhibitors.

This Phase 3 clinical trial is a global open-label, multicenter study designed to evaluate the efficacy, safety and pharmacokinetics of LR769 in adolescent and adult hemophilia A and B patients with inhibitors. The study will evaluate two dosing regimens for the treatment of bleeding episodes and should be completed in 2015. More details can be found on, study identifier NCT02020369.

“The start of patient treatment is an important milestone in the development of LR769 for hemophilia A and B patients with inhibitors to Factor VIII or IX. LFB is committed to working with the hemophilia community to find new treatment options for this condition and to improve the lives of people with hemophilia,” commented Christian Béchon, Chairman and Chief Executive Officer, LFB SA. “Based on promising data from earlier studies, we believe that LR769 has the potential to be a significant advance for people with hemophilia, and we will fully explore its potential as the first new therapeutic option in more than 15 years.”

Additional Phase 3 studies will assess the efficacy of LR769 for the treatment of bleeding episodes in pediatric hemophilia patients with inhibitors and will evaluate the prevention of bleeding complications in patients undergoing surgery. These studies are expected to start in early 2015.

About LFB group

LFB ( is a biopharmaceutical group that develops, manufactures and markets medicinal products for the treatment of serious and often rare diseases in major therapeutic fields, namely hemostasis, immunology and intensive care. The LFB Group is the leading supplier of plasma-derived medicinal products in France and sixth worldwide, and is also among the leading European companies for the development of new-generation medicinal products or treatments derived from biotechnologies. The LFB Group is pursuing a growth strategy that seeks to extend its activities at the international level and develop innovative therapies. The LFB Group currently markets its products in 30 countries around the world with a global turnover of €477 million in 2013.



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BioMarin is developing AAV-factor VIII vector, BMN 270, for the treatment of hemophilia A

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BMN 270: AAV-factor VIII vector for Hemophilia A

Current Clinical Trials

BioMarin expects to initiate clinical studies with BMN 270 in early 2015.

What is Hemophilia A?

Hemophilia usually is inherited. “Inherited” means that the disorder is passed from parents to children through genes. People born with hemophilia have little or no clotting factor. Clotting factor is a protein needed for normal blood clotting. There are several types of clotting factors. These proteins work with platelets (PLATE-lets) to help the blood clot.

Platelets are small blood cell fragments that form in the bone marrow—a sponge-like tissue in the bones. Platelets play a major role in blood clotting. When blood vessels are injured, clotting factors help platelets stick together to plug cuts and breaks on the vessels and stop bleeding.

The two main types of hemophilia are A and B. If you have hemophilia A, you’re missing or have low levels of clotting factor VIII (8). About 8 out of 10 people who have hemophilia have type A. If you have hemophilia B, you’re missing or have low levels of clotting factor IX (9).

Source: National Heart, Lung, and Blood Institute

Program Overview

BioMarin is developing AAV-factor VIII vector, BMN 270, for the treatment of hemophilia A. In BioMarin’s hemophiliac factor VIII deficient mouse models, BMN 270 restored factor VIII plasma concentrations to levels projected to be adequate for normal clotting in humans. The standard of care for the 60 percent of hemophilia A patients who are severely affected is a prophylactic regimen of IV infusions three times per week. Even with prophylactic regimens, many patients still experience spontaneous bleeding events that result in progressive and debilitating joint damage due to result of chronically low factor levels.

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New Phase 3 Data Confirm Long-Lasting Characteristics Of ALPROLIX™ and ELOCTATE™ Across Multiple Hemophilia Populations

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Monday, December 9, 2013 11:30 am EST

– Interim Analyses from Phase 3 Pediatric Studies Suggest Prolonged Half-Life of Investigational Long-Lasting Therapies in Children Under Age 12 –

CAMBRIDGE, Mass. & STOCKHOLM–()–Today Biogen Idec (NASDAQ:BIIB) and Swedish Orphan Biovitrum AB (publ) (Sobi) (STO:SOBI) announced new results from Phase 3 studies of their investigational long-lasting recombinant factor IX and VIII Fc fusion protein candidates for hemophilia B and A, ALPROLIXTM and ELOCTATETM, including an interim analysis of pediatric pharmacokinetics (PK) data. These interim data are the first to demonstrate that ALPROLIX and ELOCTATE have consistently prolonged half-lives (a measure of the time therapy circulates in the bloodstream) in children, compared to study participants’ prior therapies. Investigators presented these results, which were consistent with PK results in adults and adolescents, at the 55th Annual Meeting of the American Society of Hematology (ASH) in New Orleans, held on December 7 to 10.

ALPROLIX and ELOCTATE use a technology called Fc fusion and demonstrate prolonged circulation in the body, which has been shown in studies of adults with hemophilia to extend the time between prophylactic infusions.

“As seen in adult studies, interim data demonstrated that in children,  the use of Fc fusion technology prolongs the half-lives of these experimental therapies,” said Guy Young, M.D., director, Hemostasis and Thrombosis Center, Children’s Hospital Los Angeles. “We are investigating whether prophylactic dosing intervals longer than the currently available therapy are possible with these treatments. The frequency of traditional prophylactic regimens is a significant challenge for parents of children with hemophilia, many of whom require infusions multiple times per week.”

Kids B-LONG and Kids A-LONG are ongoing, multi-center Phase 3 studies of ALPROLIX and ELOCTATE for previously treated children under age 12 with hemophilia B and A, respectively. Kids B-LONG and Kids A-LONG are designed to investigate the safety, efficacy and PK of ALPROLIX and ELOCTATE. In Kids B-LONG, the mean half-life of ALPROLIX was more than three times longer than currently available factor IX therapies. The Kids A-LONG interim analysis showed the mean half-life of ELOCTATE was approximately one and a half times that of existing factor VIII therapies. For both ALPROLIX and ELOCTATE, prolonged half-lives were seen in each age group analyzed – under six and six to under 12 years old.

In 43 children treated with ELOCTATE as of February 8, 2013, and 23 children receiving ALPROLIX as of April 23, 2013, no inhibitors (antibodies that may interfere with the activity of the therapy) were detected. Overall, the pattern of treatment-emergent adverse events reported was typical of the populations studied, with no unique safety issues identified.

Final results of the pediatric studies will evaluate the safety and efficacy of ALPROLIX and ELOCTATE, as well as provide further PK information. Investigators plan to report these results at a future medical meeting. The primary endpoint for both studies is the occurrence of inhibitor development over the study period. Secondary outcome measures include the annualized bleeding rate (ABR), or projected number of yearly bleeding episodes, and assessments of response to treatment.

At the meeting, new analyses of data from B-LONG and A-LONG, the Phase 3 studies of ALPROLIX and ELOCTATE in adolescents and adults, were presented. These data provided additional insight into the prophylactic dosing regimens that may be possible with these therapies, if approved. Prophylaxis regimens involve regularly scheduled infusions designed to prevent or reduce bleeding episodes. In these studies, a prolonged half-life helped study participants achieve effective prevention or reduction of bleeding episodes with fewer prophylactic infusions.

“These data underscore our commitment to advancing scientific understanding and innovation in hemophilia, and our goal of introducing the first long-lasting therapies to those with hemophilia,” said Glenn Pierce, M.D., Ph.D., senior vice president of Global Medical Affairs and chief medical officer at Biogen Idec’s hemophilia therapeutic area. “These analyses add to the growing body of data supporting the clinical benefits of ALPROLIX and ELOCTATE in people of different ages with hemophilia B and A, and their potential to reduce bleeding episodes with fewer prophylactic infusions, if approved.”

Less Frequent Prophylactic Dosing Observed with ELOCTATE

A post-hoc analysis of A-LONG participants who were receiving a prophylactic (preventive) regimen with currently available factor VIII therapies prior to the study, evaluated self-reported dose, dose interval and bleeding rates over the preceding 12 months while on currently approved factor VIII therapies. These data points were also collected for on-study ELOCTATE therapy.

The majority of participants reported infusions three times a week with  their previous factor VIII therapy. During the study, 98.8 percent of  participants had ELOCTATE prophylactic dosing regimens with less frequent infusions than they reported using before the study. Their last on-study prophylactic dosing intervals were every three days (36.3 percent), twice weekly (28.8 percent), every four days (5.0 percent) and  every five days (30.0 percent). Overall, these participants reported a median of 6.0 bleeding episodes in the prior year with their existing therapy; in the last three months of on-study ELOCTATE therapy, they had a median annualized bleeding rate of zero. Weekly factor consumption for prophylaxis remained consistent with ELOCTATE and prior therapy for the majority of participants.

“Prophylactic dosing schedules have the potential to be less frequent and reduce bleeding episodes with the emergence of long-lasting therapy,” said Birgitte Volck, M.D., Ph.D., senior vice president development and chief medical officer of Sobi. “These data may help physicians understand how prophylactic dosing schedules could differ between investigational, long-lasting ELOCTATE therapy and currently available factor VIII treatments. The post-hoc analysis showed that ELOCTATE enabled bleeding control for study participants with less frequent prophylactic infusions than their previous therapy.”

Study investigators reported that ELOCTATE was well tolerated, and no inhibitors were detected during the A-LONG study. Safety events were representative of those occurring in the general hemophilia population. Outside of the perioperative (surgical) period, the most common adverse events (incidence of greater than or equal to five percent) included nasopharyngitis (common cold), arthralgia (joint pain), headache and upper respiratory infection.

Hemophilia Quality of Life Measure Validated

This study evaluated the reliability, validity and sensitivity of the Hemophilia-Specific Quality of Life Index (Haem-A-QoL), a 46-item questionnaire used to assess health-related quality of life specifically in people with hemophilia. The analysis used EuroQoL-5 Dimension (EQ-5D) data to validate Haem-A-QoL data collected from participants in the A-LONG and B-LONG studies. EQ-5D is a quality-of-life health assessment tool that is commonly used in late-stage studies for other diseases. These data further validate the usefulness of the Haem-A-QoL measurement in adults with hemophilia.

“Without adequate control over bleeding episodes, hemophilia not only leads to physical complications, but also may impact a person’s ability to attend work or school, enjoy leisure activities or maintain positive mental health,” said Aoife Brennan, M.D., senior medical director, Clinical Development of Biogen Idec’s hemophilia therapeutic area. “To understand these effects, we are committed to examining the impact of our investigational therapies ALPROLIX and ELOCTATE on a broad range of measurements, including quality-of-life measurement tools. A preliminary analysis of Haem-A-QoL scores in A-LONG and B-LONG showed numerical improvements in total scores from baseline, and we are conducting further analyses to understand the potential clinical implications of these results.”


ALPROLIX and ELOCTATE are investigational fully recombinant, long-lasting clotting factor therapies being developed for hemophilia B and A, respectively. They use Fc fusion technology, which takes advantage of a naturally occurring pathway that delays the breakdown of Immunoglobulin G Subclass 1, or IgG1 (protein commonly found in the body), and cycles it back into the bloodstream. The Fc portion of IgG1 is fused to factors IX and VIII in ALPROLIX and ELOCTATE, respectively.  This technology is thought to be responsible for the prolonged time ALPROLIX and ELOCTATE circulate in the body. While Fc fusion is an established technology that has been used for more than 15 years, Biogen Idec is the only company to apply it in hemophilia.

Based on Phase 3 study results, prophylactic therapy with ALPROLIX and ELOCTATE may help prevent or reduce bleeding episodes in people with severe hemophilia B and A, respectively. Regulatory applications for both therapies are currently under review in several countries including the United States, Canada and Australia.

About the Biogen Idec and Sobi Collaboration

Biogen Idec and Swedish Orphan Biovitrum (Sobi) are partners in the development and commercialization of ALPROLIX for hemophilia B and ELOCTATE for hemophilia A. Biogen Idec leads development, has manufacturing rights, and has commercialization rights in North America and all other regions excluding the Sobi territory. Sobi has the right to opt in to assume final development and commercialization in Europe (including Russia), the Middle East and Northern Africa.

About Biogen Idec

Through cutting-edge science and medicine, Biogen Idec discovers, develops and delivers to patients worldwide innovative therapies for the treatment of neurodegenerative diseases, hemophilia and autoimmune disorders. Founded in 1978, Biogen Idec is the world’s oldest independent biotechnology company. Patients worldwide benefit from its leading multiple sclerosis therapies, and the company generates more than $5 billion in annual revenues. For product labeling, press releases and additional information about the company, please visit

About Sobi

Sobi is an international specialty healthcare company dedicated to rare diseases. Our mission is to develop and deliver innovative therapies and services to improve the lives of patients. The product portfolio is primarily focused on inflammation and genetic diseases, with three late stage biological development projects within hemophilia and neonatology. We also market a portfolio of specialty and rare disease products for partner companies. Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2012, Sobi had total revenues of SEK 1.9 billion (€ 215 M) and about 500 employees. The share (STO: SOBI) is listed on NASDAQ OMX Stockholm. More information is available at

Biogen Idec Safe Harbor

This press release contains forward-looking statements, including statements about the potential impact and therapeutic effect of ALPROLIX and ELOCTATE, our investigational long-lasting recombinant factor product candidates. These statements may be identified by words such as “believe,” “expect,” “may,” “plan,” “potential,” “will” and similar expressions, and are based on our current beliefs and expectations. Drug development and commercialization involve a high degree of risk. Factors which could cause actual results to differ materially from our current expectations include the risk that unexpected concerns may arise from additional data or analysis, regulatory authorities may require additional data or information or further studies, or may fail to approve or may delay approval of our drug candidates, or we may encounter other unexpected hurdles. For more detailed information on the risks and uncertainties associated with our drug development and commercialization activities, please review the Risk Factors section of our most recent annual or quarterly report filed with the Securities and Exchange Commission. Any forward-looking statements speak only as of the date of this press release and we assume no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.

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Hybrid Clotting Factor Means Fewer Injections, Better Disease Control for Hemophilia Patients

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Sacramento, Calif. (PRWEB) December 12, 2013

A Phase III clinical trial led by UC Davis researchers has confirmed that a new coagulation factor (rFIXFc) dramatically reduces the number of injections needed to maintain effective clotting for hemophilia B patients.

The recombinant protein fuses clotting factor IX with an immunoglobulin (antibody) molecule, which prevents the body from rapidly metabolizing the hybrid protein. As a result, rFIXFc can be administered once a week, or even every two weeks, rather than every other (or every third) day. This extended half-life could have an enormous impact on hemophilia treatment. The findings appeared in today’s New England Journal of Medicine.

“The major challenge is spontaneous bleeding, either in weight-bearing joints or internally,” said lead investigator Jerry Powell, who directs the UC Davis Hemophilia and Thrombosis Center. “These events can be painful, life-threatening or both. To compound matters, teenage boys often drop off infusing, increasing their risk. Fewer injections should improve compliance.”

Hemophilia B is a congenital disorder caused by reduced levels of clotting factor IX, a protein that helps blood coagulate. By contrast, hemophilia A, the most common form of the disease, is caused by a loss of factor VIII. These conditions almost always affect men.

The trial enrolled 123 patients, 12 or older, who had been treated previously for hemophilia B and was divided into four groups: the first received weekly injections; the second received higher doses every 10 days; the third was only treated for specific bleeding episodes; and the fourth focused on how patients fared while undergoing surgery. An additional subgroup compared rFIXFc’s half-life in the body to that of recombinant factor IX.

The median treatment time was 51.6 weeks, during which compliance was excellent: 95.1 percent in group 1 and 100 percent in group 2. But most importantly, the annualized bleeding rate was reduced by 83 percent in group 1 and 87 percent in group 2, compared to patients receiving episodic treatment in group 3.

In addition, 23 percent of patients in group 1 and 42.3 percent in group 2 experienced no bleeding at all. In group 4, there were 14 surgeries, during which bleeding was well-controlled.

Before the study, more than 80 percent of participants in the first two groups injected at least twice a week. However, with rFIXFc, they only needed to infuse once a week, or every two weeks, to effectively control their bleeding.

While recombinant clotting factors have dramatically extended lifespans for hemophiliacs, the proteins’ short half-lives have been a limiting factor. The problem stemmed from lysosomes, cellular “chop shops” that break down proteins and other molecules. However, fusing an immunoglobulin molecule to factor IX allows the hybrid protein to temporarily escape the lysosomes, providing coagulation protection for a much longer period.

“This is a major innovation,” said Powell. “The body handles rFIXFc like an immunoglobulin molecule, but the factor retains its ability to stop bleeding.”

Some issues still need to be resolved. For example, as many as 3 percent of patients have an immune reaction to factor IX. Because the study was narrowly focused on whether the combined factor would effectively control bleeding, and all participants had successfully used factor IX, researchers did not address whether previously untreated hemophiliacs would develop an immune response to rFIXFc. This question will be addressed in future research. However, none of the study participants had an immune reaction to rFIXFc.

In addition, although the new factor is designed to treat only hemophilia B, the trial’s success outlines an effective strategy to develop long-lasting factors for other forms of the disease.

“This is the first modification to coagulation factors to increase the half-life,” Powell said. “My hope is that other pharmaceutical companies will focus on developing additional long-lasting factors to treat other types of hemophilia.”

The recombinant protein rFIXFc was created by Biogen-Idec. The company paid for extra staff to coordinate the study and lab tests and provided rFIXFc for the trial. In addition, some patients were reimbursed for travel expenses. Powell did not receive any compensation from Biogen-Idec to conduct the research.

For original Article, click here.

Baxter Receives FDA Approval of FEIBA [Anti-Inhibitor Coagulant Complex] for Prophylactic Treatment of Hemophilia A&B Patients with Inhibitors

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Pivotal Phase III data showed 72% reduction in median annual bleed rate with FEIBA prophylactic treatment compared to an on-demand regimen

For Original Press release, click here.

DEERFIELD, Ill., December 19, 2013 – Baxter International Inc. (NYSE:BAX) today announced that the United States Food and Drug Administration (FDA) granted approval of Baxter’s FEIBA [Anti-Inhibitor Coagulant Complex], the first and only FDA-approved treatment for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A or B who have developed inhibitors.

Inhibitor development is considered one of the most serious complications associated with hemophilia treatment today. As many as one-third of previously untreated patients with severe or moderately severe hemophilia A are at risk for developing inhibitors, which are antibodies produced by the body’s immune system in response to factor replacement treatment. The presence of an inhibitor makes response to treatment more challenging, and patients with inhibitors have an increased risk of developing complications.

The approval is based on data from a pivotal Phase III study, known as FEIBA PROOF, in which treatment with a FEIBA prophylactic regimen showed a 72 percent reduction in median annual bleed rate (ABR) compared to treatment with an on-demand regimen. In the intent-to-treat (ITT) analysis, three of the 17 (18%) adult patients in the prophylactic arm reported no bleeding episodes. The most frequently reported adverse reactions observed in >5% of subjects in the prophylaxis trial were anemia, diarrhea, hemarthrosis, hepatitis B surface antibody positive, nausea, and vomiting. The serious adverse reactions seen with FEIBA are hypersensitivity reactions and thromboembolic events, including stroke, pulmonary embolism and deep vein thrombosis.

“The PROOF study demonstrated that a prophylactic regimen with FEIBA can significantly reduce the rate of bleeding episodes, as compared to an on-demand regimen, in hemophilia patients with inhibitors. This is important among these patients who often have difficult-to-treat bleeds and are at risk of additional complications,” said Steven Pipe, M.D., Director, Division of Pediatric Hematology and Oncology, at the C.S. Mott Children’s Hospital at the University of Michigan. “This FDA approval of a prophylactic regimen should change the way physicians think about managing hemophilia with inhibitors, validating FEIBA prophylaxis as an effective new option to treat their patients.”

Pro-FEIBA, a prospective investigator-initiated, randomized, crossover study published in the New England Journal of Medicine in 2011 showed a 62 percent reduction in all bleeding episodes with FEIBA prophylaxis compared to an on-demand regimen. Together with the data from the FEIBA PROOF study, the results provide valuable evidence that prophylaxis with FEIBA can significantly reduce bleeding rates when compared to on-demand treatment.

“FEIBA has been an effective treatment for hemophilia patients with inhibitors for more than 35 years as an on-demand treatment. FEIBA was first licensed in the US as FEIBA VH and then as FEIBA NF. This additional indication for prophylactic treatment is aimed at reducing the number of bleeds among this patient population,” said Ludwig Hantson, Ph.D., president of Baxter’s BioScience business. “This latest approval reflects our legacy of offering new approaches to help reduce the disease burden of hemophilia, and supports Baxter’s ongoing commitment to pursuing a bleed-free world.”

FEIBA is approved in more than 60 countries worldwide and is indicated for prophylaxis in more than 40 countries.

FEIBA PROOF Study Details The prospective, open label, randomized, multi-center, two-arm parallel study investigated the efficacy and safety of FEIBA prophylactic treatment compared to on-demand treatment in 36 patients with hemophilia A or B with inhibitors over a 12-month period. The most frequently reported adverse reactions observed in >5% of subjects in the prophylaxis trial were anemia, diarrhea, hemarthrosis, hepatitis B surface antibody positive, nausea, and vomiting. The occurrence of hepatitis B surface antibodies has been seen in other plasma-derived products and could be due to the passive transfer of antibodies following FEIBA treatment. None of the subjects showed any signs of active hepatitis B infection.

Indications and Detailed Important Risk Information for FEIBA [Anti-Inhibitor Coagulant Complex] FEIBA is an Anti-Inhibitor Coagulant Complex indicated for use in hemophilia A and B patients with inhibitors for:

  • Control and prevention of bleeding episodes
  • Perioperative management
  • Routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

FEIBA is not indicated for the treatment of bleeding episodes resulting from coagulation factor deficiencies in the absence of inhibitors to coagulation Factor VIII or coagulation Factor IX.

Detailed Important Risk Information


  • Thromboembolic events have been reported during post-marketing surveillance, particularly following the administration of high doses and/or in patients with thrombotic risk factors.
  • Monitor patients receiving FEIBA for signs and symptoms of thromboembolic events.

The use of FEIBA is contraindicated in patients with:

  • Known anaphylactic or severe hypersensitivity reactions to FEIBA or any of its components, including factors of the kinin generating system
  • Disseminated intravascular coagulation (DIC)
  • Acute thrombosis or embolism (including myocardial infarction)

Thromboembolic events (including venous thrombosis, pulmonary embolism, myocardial infarction, and stroke) can occur with FEIBA, particularly following the administration of high doses (above 200 units per kg per day) and/or in patients with thrombotic risk factors.

Infusion of FEIBA should not exceed a dose of 100 units per kg body weight every 6 hours and daily doses of 200 units per kg body weight. Monitor patients receiving more than 100 units per kg of body weight of FEIBA for the development of DIC, acute coronary ischemia and signs and symptoms of other thromboembolic events. If clinical signs or symptoms occur, such as chest pain or pressure, shortness of breath, altered consciousness, vision, or speech, limb or abdomen swelling and/or pain, discontinue the infusion and initiate appropriate diagnostic and therapeutic measures.

Hypersensitivity and allergic reactions, including severe anaphylactoid reactions, can occur following the infusion of FEIBA. The symptoms include urticaria, angioedema, gastrointestinal manifestations, bronchospasm, and hypotension. These reactions can be severe and systemic (e.g., anaphylaxis with urticaria and angioedema, bronchospasm, and circulatory shock). Other infusion reactions, such as chills, pyrexia, and hypertension have also been reported. If signs and symptoms of severe allergic reactions occur, immediately discontinue administration of FEIBA and provide appropriate supportive care.

Because FEIBA is made from human plasma, it may carry a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent.

The most frequently reported adverse reactions observed in >5% of subjects in the prophylaxis trial were anemia, diarrhea, hemarthrosis, hepatitis B surface antibody positive, nausea, and vomiting.

The serious adverse reactions seen with FEIBA are hypersensitivity reactions and thromboembolic events, including stroke, pulmonary embolism and deep vein thrombosis.

Use of antifibrinolytics within approximately 6 to 12 hours after the administration of FEIBA is not recommended.

Please see the FEIBA full Prescribing Information at: products/feiba_us_pi.pdf.

About Hemophilia Hemophilia is a rare genetic blood clotting disorder that primarily affects males.1  People living with hemophilia do not have enough of, or are missing, one of the blood clotting proteins naturally found in blood.1  Two of the most common forms of hemophilia are A and B.2  In people with hemophilia A, clotting factor VIII (FVIII) is not present in sufficient amounts or is absent.2  Without enough FVIII, people with hemophilia can experience spontaneous, uncontrolled internal bleeding that is painful, debilitating, damaging to joints and potentially fatal.2  People with hemophilia B (also called Christmas disease) do not have sufficient amounts of clotting factor IX.2  In about 30% of cases, there is no family history of hemophilia and the condition is the result of a spontaneous gene mutation.3  According to the World Federation of Hemophilia, it is estimated that more than 400,000 people in the world have hemophilia.2 All races and economic groups are affected equally. It is estimated that approximately 17,000 people in the U.S. have been diagnosed with hemophilia A or B.4

About Inhibitors As many as one-third of previously untreated patients with severe or moderately severe hemophilia A are at risk for developing inhibitors,5,6,7 which are antibodies produced by the body’s immune system in response to factor replacement treatment.8,9  The presence of an inhibitor makes response to treatment more challenging, and patients with inhibitors have an increased risk of developing complications such as joint damage.10  Inhibitors cause the body to work against the factor replacement treatment, neutralizing its effect and preventing an individual’s blood from clotting.10,11  Individuals who have inhibitors have a form of hemophilia that is more difficult to control, with an increased risk of uncontrolled bleeding, compared to patients without inhibitors.  Inhibitor development is considered one of the most serious complications associated with hemophilia treatment, and may include other associated complications such as impaired movement, increased need for surgery and greater complexity or risk associated with surgery.12,13

About Baxter International Inc. Baxter International Inc., through its subsidiaries, develops, manufactures and markets products that save and sustain the lives of people with hemophilia, immune disorders, cancer, infectious diseases, kidney disease, trauma and other chronic and acute medical conditions. As a global, diversified healthcare company, Baxter applies a unique combination of expertise in medical devices, pharmaceuticals and biotechnology to create products that advance patient care worldwide.

1About Bleeding Disorders: Treatment. World Federation of Hemophilia. Accessed on: October 9, 2013. Available at:
2Frequently Asked Questions about Hemophilia. World Federation of Hemophilia. Accessed on: October 9, 2013. Available at: 
3Hemophilia A. National Hemophilia Foundation. Accessed on: October 9, 2013. Available at: .
4World Federation of Hemophilia Report on the Annual Global Survey 2011. World Federation of Hemophilia. Accessed on October 9, 2013. Available at:
5Ehrlich HJ, Bray GL, Gomperts ED.: Comparison of high responder inhibitor frequency in recent studies of previously untreated patients with hemophilia A. Thromb.Haemost. 1998;79: 242-243.
6Kreuz W, Escuriola-Ettingshausen C, Zyschka A eta!.: Inhibitor development in previously untreated patients with hemophilia A: a prospective long-term follow-up comparing plasma-derived and recombinant products. Semin.Thromb.Hemost.2002;28: 285-290.
7Hay CR: Factor VIII inhibitors in mild and moderate-severity haemophilia A. Haemophilia. 1998;4: 558-563.
8Santagostino E, Mancuso ME, Rocino A, et al. Environmental risk factors for inhibitor development in children with haemophilia A: a case-control study. Br J Haematol. 2005;130:422-427.
9Oldenburg J, Pavlova A. Genetic risk factors for inhibitors to factors VIII and IX. Haemophilia. 2006;12(Suppl. 6):15-22.
10Perry D, Berntorp E, Tait C, et al. FEIBA prophylaxis in haemophilia patients: a clinical update and treatment recommendations. Haemophilia. 2010;16:80-89.
11About Bleeding Disorders. What are Inhibitors?. World Federation of Hemophilia. Accessed on October 9, 2013. Available at: .
12Leissinger, Cindy A. Prevention of Bleeds in Hemophilia Patients with Inhibitors: Emerging Data and Clinical Direction. American Journal of Hematology. 2004; 77:187-193.
13Kulkarni R. Comprehensive care for the patient with haemophilia and inhibitors undergoing surgery: practical aspects. Haemophilia. 2013;19:2-10.

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