South San Francisco, CA, January 5, 2012 - Catalyst Biosciences, Inc., The Leading company in the discovery and development of engineered proteases, today announced that Pfizer Inc. has initiated a Phase 1 clinical trial for PF-05280602, an investigational proprietary, engineered variant of recombinant human Factor VIIa developed by Catalyst Biosciences. PF-05280602 has been engineered to provide improved acute and prophylactic treatment for hemophilia A & B patients with inhibitors. The IND filing and initiation of the Phase 1 clinical trial triggered additional milestone payments of $7.0 million payable to Catalyst by Pfizer under the terms of their research and license agreement.

“We are very excited to see the lead candidate from Catalyst’s hemostasis franchise advance into human clinical trials,” said Nassim Usman, Ph.D., Catalyst’s CEO. “In parallel, Catalyst is independently achieving significant progress in engineering nextgeneration recombinant human Factor IX and Xa variants with highly differentiated advantages for the treatment of acute and prophylactic bleeding disorders, including hemophilia and non-hemophilia indications. We are diligently executing on our goal of expanding Catalyst’s high-value pipeline of novel engineered proteases.”

In June 2009 Catalyst and Wyeth LLC, now a wholly owned subsidiary of Pfizer Inc., formed an exclusive, worldwide collaboration for the discovery, development and commercialization of improved, second-generation Factor VIIa products. Total payments under the collaboration, including an upfront payment of $21 million, ongoing research funding and milestone payments, could exceed $500 million, exclusive of double-digit royalty payments.

About Catalyst Biosciences

Catalyst Biosciences is developing the next generation of biopharmaceuticals by harnessing the catalytic power of engineered proteases that target proteins underlying diseases. Catalyst’s discovery platform rapidly creates and optimizes tailor-made protease drug candidates that cleave a wide variety of disease targets, either by improving existing protease drugs or by creating new protease drugs, known as Alterase™ therapeutics. Catalyst is focusing its product development efforts on drug candidates for hemophilia, non-hemophilic bleeding, and complement-driven diseases, including the prevention of delayed graft function and inflammation. Catalyst has ongoing research, development, and license agreements with Wyeth LLC, a wholly owned subsidiary of Pfizer Inc., and with MedImmune, LLC, the global biologics unit of AstraZeneca plc. Catalyst is privately held with backing by leading venture firms, including Burrill & Company, Essex Woodlands Health Ventures, HealthCare Ventures, Johnson & Johnson Development Corporation, Morgenthaler Ventures, Novartis BioVentures, RCT BioVentures, and Sofinnova Ventures. For more information, please visit www.catbio.com

# # #
Catalyst Biosciences, Inc.
William J. Dawson
Chief Financial Officer
+1.650.871.0761
info@catbio.com
BCC Partners for Catalyst Biosciences
Karen L. Bergman
+1.650.575.1509

Vienna, Austria — 12 January 2012

CSL Behring has announced that the first site has been initiated in its global phase II/III, multi-center study to evaluate the safety, efficacy and pharmacokinetics of recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP). The site is located in Vienna, Austria. The prospective, open-label study will enroll adolescents and adults (12 – 65 years) who have hemophilia B. CSL Behring, in collaboration with its parent company, CSL Limited (ASX:CSL), is developing rIX-FP for the prophylaxis and treatment of bleeding episodes in patients with congenital Factor IX (FIX) deficiency as part of the PROLONG-9FP clinical study program.

“The unmet medical need is great for a factor IX product with an extended half-life for use in treating people with hemophilia B, a life-long, debilitating clotting disorder,” said Russell Basser, MD, Senior Vice President, Global Clinical R&D at CSL Behring. “Such a therapy can mean fewer injections for patients, and may enable or enhance prophylactic treatment. This would be an improved convenience that may result in a better quality of life for patients.”

“We have entered a very exciting and promising era for patients with hemophilia,” said Elena Santagostino, MD, Principal Investigator for the study. “A recombinant factor IX product with a longer half-life, such as the product that CSL Behring is developing, will have the potential to prevent bleeds in people who have hemophilia B. Today, these individuals generally must undergo frequent infusions of factor product to effectively manage their condition. Such a routine tends to present challenges; maintaining adherence to it can be difficult and one’s quality of life can therefore be impacted. The product in development today aims to reduce the number of infusions a patient with hemophilia must undergo. As treating physicians and clinical researchers, we are proud to be a part of this effort and look forward with great anticipation to the results of our research.”

To date, the PROLONG-9FP program has established study sites in Austria, Bulgaria, France, Germany, Italy, Spain and in Israel. It is anticipated that in coming months additional trial sites will be established in the United States, Japan, and Russia.

CSL Behring and CSL have engineered rIX-FP to extend the half life of Factor IX while minimizing any tolerability issues. In the process, recombinant albumin—a protein with an inherently long half-life—is used as a fusion partner. A specifically designed linker connects the recombinant factor IX and recombinant albumin as a means of optimizing the efficacy of rIX-FP.

The Phase II/III study consists of a screening period, a pharmacokinetic (PK) evaluation period, followed by an approximate 12-month safety and efficacy evaluation period with rIX-FP. A surgical prophylaxis sub-trial is included. More information can be found at http://clinicaltrials.gov/ct2/results?term=rIX-FP+.

About Hemophilia
Hemophilia is an inherited bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. In nearly all cases, it affects only males. The disease is caused by deficient or defective blood coagulation proteins known as factor VIII or IX. The most common form of the disease is hemophilia A, or classic hemophilia, in which the clotting factor VIII is either deficient or defective. Hemophilia B is characterized by deficient or defective factor IX. Hemophilia A affects approximately 1 in 5,000 to 10,000 people. Hemophilia B affects approximately 1 in 25,000 to 50,000 people. The recommended treatment for patients who are factor deficient is to treat by replacement factor therapy.

About CSL Behring
CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic diseases in newborns. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of
CSL Limited (ASX: CSL), a biopharmaceutical company headquartered in Melbourne, Australia. For more information, visit www.cslbehring.com.

Contact:
Sheila A. Burke, Director, Communications & Public Relations
Worldwide Commercial Operations
CSL Behring
610-878-4209 (o)
484-919-2618 (c)
Sheila.Burke@cslbehring.com

For complete New Release click here.

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jan. 8, 2012– Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today its key “Alnylam 5×15” and partner program goals for 2012.

• Advance ALN-APC Program for Hemophilia into Clinical Studies. ALN-APC is an RNAi therapeutic targeting protein C, a genetically defined target, for the treatment of hemophilia. By reducing levels of protein C, a natural anticoagulant protein, ALN-APC is intended to act by increasing thrombin generation and reducing the frequency of bleeding in hemophilia patients, including those more severe patients with inhibitors against their replacement factors. Alnylam is exploring both systemically delivered LNP and subcutaneously delivered GalNAc-conjugate approaches for ALN-APC with the goal of selecting the clinical candidate in the first half of 2012. Alnylam plans to advance its ALN-APC program toward the clinic with a goal of initiating a Phase I clinical trial in the first half of 2013 with data expected in the second half of 2013.

“We believe that ALN-TTR, our lead ‘Alnylam 5×15’ program, has the potential to become the leading innovative medicine for the treatment of transthyretin-mediated amyloidosis. In 2012, we aim to advance ALN-TTR02 through an initial Phase I study and then into the start of a Phase II study in ATTR patients, positioning our program for the start of pivotal trials in 2013. Further, we also intend to advance our subcutaneous TTR program, ALN-TTRsc, with an IND filing this year, as we view this program as a differentiated opportunity for additional applications in the ATTR disease setting,” said Barry Greene, President and Chief Operating Officer of Alnylam. “We also view our ALN-APC hemophilia program as an exciting opportunity to fundamentally change the management of this genetic disease where new approaches for patients are greatly needed. Clinical data showing a markedly reduced bleeding phenotype in hemophilia patients who have co-inherited pro-thrombotic gene mutations greatly support our enthusiasm for this program.¹ Accordingly, we aim to advance both lipid nanoparticle and conjugate versions of ALN-APC toward final candidate selection, with an IND filing targeted in 2013. Thereafter, we expect this program to advance rapidly toward pivotal trials.”

“While we focus Alnylam resources on our ALN-TTR and ALN-APC programs, we will also advance additional ‘Alnylam 5×15’ and partner programs through existing alliances and new partnerships that we aim to form in 2012 and beyond. In this regard, we believe our recent clinical successes greatly strengthen the attractiveness of many of our programs for partnership,” saidLaurence Reid, Ph.D., Senior Vice President and Chief Business Officer of Alnylam. “We are also pleased to announce today the designation of our fifth ‘Alnylam 5×15’ program, ALN-TMP, an RNAi therapeutic targeting Tmprss6 for the treatment of hemoglobinopathies. Based on its performance in pre-clinical models, we believe that ALN-TMP could become a disease modifying therapy for both beta-thalassemia and sickle cell anemia, areas of enormous unmet medical need. Along with other programs, we will look to advance this new program with a new partnership.”

The Phase I trial is a prospective, open-label study that will assess the safety, tolerability and pharmacokinetics of BAX 855 in previously-treated patients aged 12 years or older with severe hemophilia A. When used for prophylaxis, Baxter s ADVATE requires patients to infuse every two to three days to reduce the occurrence of bleeding episodes. This Phase I trial is the first step in assessing whether BAX 855 can be infused less frequently.

This trial is designed to provide new insights about our investigational longer-acting FVIII molecule, BAX 855, with the ultimate goal of improving care for patients living with hemophilia A, said Prof. Hartmut J. Ehrlich, M.D., vice president of global research and development in Baxter s BioScience business. The Phase I results will serve as the foundation for advancing this important program through clinical development and determining whether BAX 855 can offer a treatment regimen requiring fewer infusions than ADVATE.

 BAX 855 employs Baxter s proprietary full-length plasma/albumin-free recombinant protein platform that does not contain any human or animal-derived additives. BAX 855 leverages Nektar Therapeutics (NASDAQ: NKTR) proprietary PEGylation technology, which is designed to extend the duration of activity of proteins and larger molecules. Baxter and Nektar have a collaboration to develop PEGylated products designed to provide new long-acting therapies for hemophilia patients.

ADVATE was recently approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A. With the inclusion of prophylaxis in the adult patient population, ADVATE became the only antihemophilic factor approved in the United States for prophylactic use in both children and adults. This approval was supported by a Phase IV prophylaxis study sponsored by Baxter demonstrating that ADVATE for routine prophylaxis significantly reduced median annual bleed rates (ABR) in hemophilia A patients. In the study, patients experienced 44 bleeds (per patient per year) during on-demand treatment compared to one bleed (per patient per year) while on either of the prophylactic regimens evaluated, a 98 percent reduction in annual bleed rate (p<0.0001). Nearly half (42 percent) of patients experienced zero bleeding episodes during one year on prophylactic therapy. Evaluable patients were those with at least 90 percent adherence to their prescribed prophylactic regimen.

 About ADVATE

ADVATE was initially approved by the FDA in July 2003 for control and prevention of bleeding episodes in adults and children with hemophilia A.

ADVATE (derived from the complete FVIII gene) is a recombinant FVIII therapy 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 53 countries worldwide including the United States, Canada, 27 countries in the European Union, Argentina, Australia, Brazil, Chile, 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. Since the initial approval of ADVATE, more than 10 billion international units have been distributed, and ADVATE is the number one chosen rFVIII worldwide.

ADVATE is an Antihemophilic Factor (Recombinant) indicated for:

• Control and prevention of bleeding episodes in adults and children
  (0-16 years) with hemophilia A
• Routine prophylaxis to prevent or reduce the frequency of bleeding
  episodes in adults and children (0-16 years) with hemophilia A
• Perioperative management in adults and children (0-16 years) with
  hemophilia A
• ADVATE is not indicated for the treatment of von Willebrand disease

Important Risk Information for ADVATE

 ADVATE is contraindicated in patients with known anaphylaxis to mouse or
hamster protein or other constituents of the product. Allergic-type hypersensitivity reactions, including anaphylaxis, are possible and have been reported with ADVATE. Symptoms have manifested as dizziness, paresthesia, rash, flushing, face swelling, urticaria, dyspnea, and pruritis. Discontinue use if hypersensitivity symptoms occur and administer appropriate emergency treatment.

Carefully monitor patients treated with AHF products for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests. Inhibitors have been reported following administration of ADVATE predominantly in previously-untreated patients (PUPs) and previously minimally-treated patients (MTPs).

If expected plasma FVIII levels are not attained, or if bleeding is not controlled with an expected dose, perform an assay that measures factor VIII inhibitor concentration.

The serious adverse drug reactions (ADRs) seen with ADVATE are hypersensitivity reactions and the development of high-titer inhibitors necessitating alternative treatments to Factor VIII. The most common ADRs observed in clinical trials (frequency = 10 percent of patients) were pyrexia, headache, cough, nasopharyngitis, vomiting, arthralgia, and limb injury.

Please see full prescribing information at www.advate.com.

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.

This release includes forward-looking statements concerning a Phase I trial of the Company s investigational compound BAX 855, including with respect to expectations related to clinical outcomes. The statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those in the forward-looking statements: timely submission and approval of anticipated regulatory filings; clinical results validating the use of BAX 855 to treat patients with severe hemophilia A; satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; and other risks identified in Baxter’s most recent filing on Form 10-K and other SEC filings, all of which are available on the company’s website. Baxter does not undertake to update its forward-looking statements.

1. What is Hemophilia? World Federation of Hemophilia. Accessed on: 29 June 2011. Available at: http://www.wfh.org/2/1/1_1_1_What_Is_Hemophilia.htm

2. Frequently Asked Questions About Hemophilia. World Federation of Hemophilia. Accessed on: 24 August 2011. Available at: http://www.wfh.org/index.asp?lang=EN

3.  Hemophilia A. National Hemophilia Foundation. Accessed on: 29 June 2011. Available at http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=180&contentid=45&rptname=bleeding.

Source: Baxter International Inc.

Baxter International Inc.
Media Contacts
Deb Spak, (847) 948-2349
Brian Kyhos, (847) 948-4210
or
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Clare Trachtman, (847) 948-3085

ADVATE for routine prophylaxis reduced annual bleed events in hemophilia A patients from forty-four to one as compared to an on demand regimen in a clinical study

Once every third day pharmacokinetic dosing option offers some patients the opportunity for fewer infusions annually

DEERFIELD, Ill., December 16, 2011 – Baxter International Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin Free Method] for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A.  ADVATE is the only antihemophilic factor approved in the United States for prophylactic use in both adults and children. 

The approval is based on a Phase IV prophylaxis study sponsored by Baxter demonstrating that ADVATE for routine prophylaxis significantly reduced median annual bleed rates (ABR) in hemophilia A patients from 44 to one as compared to an on-demand regimen.  Forty-two percent of study patients experienced zero bleeds during one year on prophylaxis.  Of the two prophylactic regimens approved for use, the dosing schedule of every three days, a pharmacokinetic-driven regimen based on patient’s clinical response, offered some patients the option of fewer infusions over one year of treatment. 

“Emerging data provide important information to help physicians optimize care for hemophilia patients of all ages by preventing unexpected bleeding events that can have a detrimental impact on the lives of patients,” said Leonard Valentino, M.D., Director, Rush Hemophilia and Thrombophilia Center and Section of Pediatric Hematology/Oncology, Rush University Medical Center, Chicago, and lead investigator of this study. “These data confirm the important clinical benefits of ADVATE when used as a prophylactic therapy to reduce bleeding episodes.”

The study findings demonstrated a statistically significant reduction in the median annual bleeding rate, with patients experiencing 44 bleeds (per patient per year) during on-demand treatment compared to one bleed (per patient per year) while on either of the prophylactic regimens evaluated, a 98 percent reduction in annual bleed rate (p<0.0001).  Nearly half (42 percent) of patients experienced no bleeding episodes while on one year of prophylactic treatment.  Evaluable patients had greater than or equal to 90 percent adherence to the prescribed prophylactic regimen.  While the trial was not powered to demonstrate equivalence in bleeding rate between the two prophylaxis arms, there was no statistically significant difference in bleeding frequency observed between the two prophylaxis regimens studied.

“This latest clinical milestone for ADVATE is an important step forward for people living with hemophilia A as we continue to research ways to advance care for this patient population,” said Bruce Ewenstein, M.D., Ph.D, vice president, clinical affairs, Baxter’s BioScience business. “This rigorous clinical study demonstrated that the number of bleeding episodes experienced each year could be reduced to as low as one event with prophylactic treatment.  Further, the pharmacokinetic-driven dosing regimen based on patient’s clinical response every third day, offers some patients the option of fewer infusions over one year of treatment than the current standard prophylaxis regimen.”

For the prophylaxis regimen to prevent or reduce frequency of bleeding episodes, ADVATE dosing of three to four times weekly (between 20 to 40 international units of factor VIII per kg body weight every other day) may be used.  Alternatively, an every third day dosing regimen targeted to maintain FVIII trough levels greater than or equal to one percent may be employed.  The serious adverse drug reactions (ADRs) seen with ADVATE are hypersensitivity reactions and the development of high-titer inhibitors necessitating alternative treatments to Factor VIII.  The most common ADRs observed in clinical trials (frequency ≥ 10% of patients) were pyrexia, headache, cough, nasopharyngitis, vomiting, arthralgia, limb injury.

Study Design
The approval is based on the ADVATE Phase IV study comparing two prophylactic regimens to on demand treatment of bleeding episodes in previously treated patients with severe or moderately severe hemophilia A.  The multi-center, open-label, prospective, randomized, controlled clinical trial evaluated the relative efficacy of ADVATE use in two prophylactic regimens (standard prophylaxis [20-40 international units/kg every 48 hours] and pharmacokinetic-driven prophylaxis [20-80 international units/kg every 72 hours, targeted to maintain FVIII trough levels at least 1 percent or higher]) compared to that of on-demand treatment in 53 previously treated patients with severe to moderately severe hemophilia A.  Initial treatment with six months of on-demand therapy was followed by 12 months of either prophylactic regimen.

All patients had a history of at least eight joint hemorrhages per year prior to entering the study.  Additionally, each patient evaluated was adherent to greater than 90 percent of the prescribed number of prophylactic infusions, and no patient in the study surpassed the upper boundary of 110 percent of the prescribed number of prophylactic infusions.

About ADVATE
ADVATE was initially approved by the FDA in July 2003 for control and prevention of bleeding episodes in adults and children with hemophilia A.  ADVATE (derived from the complete FVIII gene) is a recombinant FVIII therapy 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 any rFVIII therapies.

ADVATE is approved in 52 countries worldwide including the United States, Canada, 27 countries in the European Union, Argentina, Australia, Brazil, Chile, Colombia, Croatia, Hong Kong, Iceland, Iraq, Japan, Macau, Malaysia, New Zealand, Norway, Puerto Rico, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Uruguay and Venezuela. Since the initial approval of ADVATE eight years ago, nearly 10 billion international units have been distributed, and ADVATE is the number one chosen rFVIII worldwide.

ADVATE is an Antihemophilic Factor (Recombinant) indicated for:

• Control and prevention of bleeding episodes in adults and children (0-16 years) with Hemophilia A
• Perioperative management in adults and children (0-16 years) with hemophilia A
• Routine prophylaxis to prevent or reduce frequency of  bleeding episodes in adults and children (0-16 years) with hemophilia A
• ADVATE is not indicated for the treatment of von Willebrand disease

Detailed Important Risk Information for ADVATE
ADVATE is contraindicated in patients with known anaphylaxis to mouse or hamster protein or other constituents of the product.

Allergic-type hypersensitivity reactions, including anaphylaxis, are possible and have been reported with ADVATE.  Symptoms have manifested as dizziness, paresthesia, rash, flushing, face swelling, urticaria, dyspnea, and pruritus.  Discontinue use if hypersensitivity symptoms occur and administer appropriate emergency treatment.

Patients treated with AHF products should be monitored for the development of FVIII inhibitors.  Inhibitors have been reported following administration of ADVATE predominantly in previously untreated patients (PUPs) and previously minimally treated patients (MTPs).

If expected plasma FVIII levels are not attained, or if bleeding is not controlled with an expected dose, test for the presence of inhibitors.

The most serious adverse reactions seen with ADVATE are hypersensitivity reactions and the development of high-titer inhibitors necessitating alternative treatments to FVIII.

The most common adverse reactions observed in clinical trials (frequency less than 10% of patients) were pyrexia, headache, cough, nasopharyngitis, vomiting, arthralgia, and limb injury.

Please see full prescribing information at www.baxter.com/downloads/healthcare_professionals/products/ADVATE_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 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, more than 400,000 people in the world have hemophilia.¹ All races and economic groups are affected equally.¹

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.
1. What is Hemophilia? World Federation of Hemophilia. Accessed on: 29 June 2011. Available at: http://www.wfh.org/2/1/1_1_Hemophilia.htm .
2. Frequently Asked Questions About Hemophilia. World Federation of Hemophilia. Accessed on: 29 June 2011.  Available at: http://www.wfh.org/2/1/1_1_1_FAQ.htm#difference .
3. Hemophilia A. National Hemophilia Foundation. Accessed on: 29 June 2011. Available at: http://www.hemophilia.org/NHFWeb/MainPgs/MainNHF.aspx?menuid=180&contentid=45&rptname=bleeding .

If you like Christmas music and want to support vWD research keep reading!!!!!!

Produced by Dan Pavelich, whose catchy confections with bands like the Bradburys and the Click Beetles have reaped rave reactions from here to eternity, Hi-Fi Christmas Party Volume 3 features a batch of the coolest holiday tunes you could hope for.

The idea for the series arrived when Dan learned his daughter, Mari, had Von Willebrand’s Disease, a bleeding disorder akin to hemophilia. He soon discovered research for the condition lacked serious funds, so he approached some of his colleagues and asked if they would submit their talents to a compilation album he was putting together where proceeds from all sales would go to research the ailment.

So not only does Hi-Fi Christmas Party (Vandalay Records) offer a great repertoire of songs, but it also benefits an awfully good cause.

As was the situation with the previous two installments of the collection, this set includes a dazzling cast of characters. Sixteen different acts total, and each one brings their own special herbs and spices to the festivities. 

For more of the reveiw, click here.

CAMBRIDGE, Mass., Dec 13, 2011 (BUSINESS WIRE) — Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it presented multiple posters and presentations at the 53rd American Society of Hematology (ASH) Annual Meeting and Exposition held in San Diego, CA between December 10 — 13, 2011. At the meeting, research was presented from programs in the company’s “Alnylam 5×15” product pipeline, including pre-clinical data from ALN-APC, an RNAi therapeutic targeting protein C for the treatment of hemophilia, and ALN-HPN, an RNAi therapeutic targeting the hepcidin pathway for the treatment of refractory anemia. In addition, pre-clinical research was also presented from candidate programs in beta-thalassemia and erythropoiesis.

“The research we presented at this meeting highlights the significant progress we are making in translating RNAi therapeutics toward an innovative class of medicines for a broad range of human disease,” said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President and Chief Medical Officer of Alnylam. “Specifically, we were pleased to present additional updates from some of our ‘Alnylam 5×15′ programs, including ALN-APC for the treatment of hemophilia and ALN-HPN for the treatment of refractory anemia in addition to candidate programs in beta-thalassemia and erythropoiesis. Importantly, all four programs are focused on genetically defined targets expressed in the liver, where we have recently proven safe and effective delivery in man, with target and/or disease biomarkers measureable in early clinical studies. We remain on track to nominate our fifth ‘Alnylam 5×15′ program at or around year’s end from these and other candidate programs.”

Presentations and posters from Alnylam scientists at the ASH Meeting included a poster titled “RNAi-Mediated Inhibition of Activated Protein C. A New Approach for Hemophilia Treatment,” where Alnylam scientists presented pre-clinical data from its ALN-APC program. These data demonstrate dose-dependent silencing of protein C mRNA with silencing of greater than 90% and an ED50 of approximately 0.02 mg/kg. When administered as a single dose of 0.3 mg/kg, the lipid nanoparticle (LNP)-formulated siRNA achieved greater than 75% silencing of protein C mRNA with effects lasting for over two weeks. Based on Western blot analysis, the reduction of protein C mRNA led to virtually undetectable levels of circulating protein C protein levels in plasma. By reducing levels of protein C, ALN-APC is expected to increase thrombin generation in hemophilia.

A presentation titled “Targeting the Hepcidin Pathway with RNAi Therapeutics for the Treatment of Anemia,” showed new pre-clinical data from the company’s ALN-HPN program for the treatment of refractory anemia. New data was shown highlighting the ability of an RNAi therapeutic to silence the hepcidin pathway in pre-clinical models, resulting in associated dose-dependent increases in serum iron and transferrin saturation, a measure of effective iron mobilization in vivo. New data also revealed transferrin receptor type 2 (TFR2) as the optimal target in the hepcidin pathway, as TFR2 silencing was associated with marked decreases in hepcidin expression and greater levels of transferrin saturation as compared with direct targeting of the hepcidin mRNA itself. TFR2 silencing was also found to be more effective than direct silencing of hepcidin in animal models, including correction of hemoglobin levels in a model of inflammatory anemia. TFR2 is a genetically validated target as loss of function human mutations are associated with a hereditary form of hemochromatosis. Further efforts on ALN-HPN will focus on TFR2 as the molecular target.

In a poster titled “RNAi-Mediated Inhibition of Tmprss6 Elevates Hamp1 [Hepcidin] Expression and Reduces Serum Iron Levels in Mice,” Alnylam scientists and collaborators from Children’s Hospital Boston showed that the systemic administration of an LNP-formulated siRNA targeting Transmembrane protease, serine 6 (Tmprss6) represents a potential novel approach to treat congenital iron overload disorders. In a pre-clinical model of iron overload in beta-thalassemia, potent and dose dependent silencing of Tmprss6 was achieved, resulting in a concomitant induction of hepcidin mRNA. As a result of increased hepcidin levels, treatment with the Tmprss6 siRNA resulted in significant decreases in serum iron concentration and transferrin saturation. Moreover, Tmprss6 silencing with a single dose of the RNAi therapeutic was associated with a dramatic normalization of multiple hematological parameters including hemoglobin, normalization of splenic iron levels and tissue histology, and reductions in splenomegaly. These findings suggest that iron restriction via Tmprss6 silencing can correct the disease phenotype of beta-thalassemia intermedia in a pre-clinical model.

Lastly, in a poster titled “Liver Specific Delivery of siRNA Targeting EGLN Prolyl Hydroxylases Activates Hepatic Erythropoietin Production and Stimulates Erythropoiesis,” Alnylam scientists and collaborators at the Dana-Farber Cancer Institute and the Massachusetts Institute of Technology (MIT) highlighted the potential of RNAi therapeutics targeting liver-expressed egl nine homolog (EGLN) prolyl hydroxlase genes to promote effective and durable erythropoiesis. Pre-clinical data demonstrated that LNP-formulated EGLN siRNA induced hepatic erythropoietin (EPO) mRNA activation leading to increased serum EPO levels and stimulation of erythropoiesis. Following a single dose, increases in serum EPO and hematocrit were durable for approximately two weeks and one month, respectively. Treatment with EGLN siRNAs corrected anemia in both renal failure and inflammatory anemia pre-clinical disease models.

About RNA Interference (RNAi)

RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.

About Alnylam Pharmaceuticals

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, and ALN-APC for the treatment of hemophilia. As part of its “Alnylam 5×15(TM)” strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in advanced stages of clinical development by the end of 2015. Alnylam has additional partner-based programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT for the treatment of Huntington’s disease. The company’s leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. In addition, Alnylam and Isis co-founded Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline and Sanofi. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam’s VaxiRNA(TM) platform applies RNAi technology to improve the manufacturing processes for vaccines; GlaxoSmithKline is a collaborator in this effort. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 100 peer-reviewed papers, including many in the world’s top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com .

Alnylam Forward-Looking Statements

Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including without limitation, statements regarding Alnylam’s views with respect to the potential for RNAi therapeutics, including ALN-APC and ALN-HPN, its expectations regarding the potential of identifying RNAi therapeutics for beta-thalassemia and erythropoiesis, its expectations with respect to the timing and success of its pre-clinical studies, the expected timing for nominating the fifth ‘Alnylam 5×15′ program, and Alnylam’s expectations regarding its “Alnylam 5×15″ product strategy, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Alnylam’s ability to discover and develop novel drug candidates, successfully demonstrate the efficacy and safety of its drug candidates, including ALN-APC, ALN-HPN, and potential candidates for beta-thalassemia and erythropoiesis , the pre-clinical and clinical results for its product candidates, which may not support further development of product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials, obtaining, maintaining and protecting intellectual property, obtaining regulatory approval for products, competition from others using technology similar to Alnylam’s and others developing products for similar uses, and Alnylam’s ability to establish and maintain strategic business alliances and new business initiatives, as well as those risks more fully discussed in the “Risk Factors” section of its most recent quarterly report on Form 10-Q on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam does not assume any obligation to update any forward-looking statements.

SOURCE: Alnylam Pharmaceuticals, Inc.

        Alnylam Pharmaceuticals, Inc.
        Cynthia Clayton, 617-551-8207
        Senior Director, Investor Relations and
        Corporate Communications
        or
        Spectrum
        (Media)
        Amanda Sellers, 202-955-6222

(Reuters) – A single treatment with gene therapy, an experimental technique for fixing faulty genes, has been shown for the first time to boost output of a vital blood clotting factor, possibly offering a long-term solution for people with hemophilia B.

Researchers said the same technology was also being studied as a treatment for hemophilia A, the far more common type of the inherited bleeding disorder.

“It is a technique for potentially permanently curing patients,” said Dr. Charles Abrams, American Society of Hematology secretary and associate chief of hematology/oncology at the University of Pennsylvania in Philadelphia.

The strategy involves replacing the defective gene that causes the bleeding disorder with a correct version delivered via a virus to the patient’s liver cells — the only cells in the body capable of producing certain clotting factors missing or deficient in people with hemophilia.

The factors are numbered using Roman numerals. The two main forms of the disease are hemophilia A, caused by a lack of clotting factor VIII, and hemophilia B, caused by a lack of clotting factor IX.

Researchers from the University College London Cancer Institute and St. Jude Children’s Research Hospital in Memphis, Tennessee, studied six men with severe hemophilia B who were producing clotting factor IX, also called FIX, at less than 1 percent of normal levels. The general goal of current treatment with manufactured FIX is to achieve factor levels greater than 1 percent of normal.

Four of the six trial participants have stopped routine treatment and remain free of spontaneous bleeding. The other two have increased the interval between FIX infusions to once every 10 days to two weeks from two to three times a week, said Dr. Andrew Davidoff, chairman of the department of surgery at St. Jude’s and co-author of the study.

HIGH COST FOR CURRENT TREATMENT

Frequent treatments with manufactured FIX can cost hundreds of thousands of dollars a year, making hemophilia a tempting target for gene therapy.

The trial “is truly a landmark study,” Dr. Katherine Ponder, hematology and oncology professor at Washington University in St. Louis, said in a New England Journal of Medicine editorial.

“If further studies determine that this approach is safe, it may replace the cumbersome and expensive protein therapy currently used for patients with hemophilia B,” she wrote.

The trial results were published in the NEJM and reported on Saturday at a meeting of the American Society of Hematology in San Diego.

The approach involved the use of a novel viral “vector,” designed to target the liver specifically.

The six trial subjects were broken into three groups with each group receiving a different concentration of new genes.

FIX levels in the first subject have remained at 2 percent for nearly two years, while the two patients treated with the highest dose have seen FIX levels rise to between 3 and 12 percent, researchers said.

One high-dose subject developed elevated levels of transaminases, an indicator of possible liver damage, and another had a slight increase in liver enzymes. Both cases were resolved with steroids, the researchers said.

Plans are to treat more patients with the highest dose used so far, and if research continues to succeed, the treatment could be widely available “in the next five years or so,” said Dr. Amit Nathwani, co-lead study author of the Department of Hematology at UCL Cancer Institute in London.

He also said the team was working to use the technique for treating hemophilia A.

ISI Group analyst Mark Schoenebaum said the gene therapy could pose big competition for companies such as Biogen Idec that are producing recombinant factor concentrates.

“This clearly presents a curveball to our (and much of Wall Street’s) assumptions around the future of the hemophilia market,” he said in an email to investors.

The analyst said estimated sales of the hemophilia factors accounted for between $10 and $17 of his $125 price target for shares of Biogen, which closed at $112.95 on Friday.

People with hemophilia bleed more following trauma than people without the disease, and those with severe disease may bleed spontaneously.

Hemophilia B is much less common than hemophilia A. About one in five hemophilia patients has hemophilia B, according to the National Institutes of Health.

The global market for Factor VIII products is about $5 billion, while the market for Factor IX is worth about $1 billion.

Hemophilia is an inherited condition that affects men more frequently. Worldwide, about one in 5,000 men is born with hemophilia A and 1 in 25,000 men is born with hemophilia B each year.

(Reporting by Deena Beasley)

For nearly 30 years, the Hemostasis Laboratory, part of Diagnostic Laboratories at BloodCenter of Wisconsin, has built a reputation for detail and quality in its evaluations. Hemostasis Reference Laboratory at BloodCenter of Wisconsin is proud to offer the most comprehensive von Willebrand Disease testing in the United States. Now, BloodCenter is pleased to announce three new assays to add to the largest menu of assays: von Willebrand Factor (VWF) Quantitative Multimer, VWF D1472H Ristocetin-binding Polymorphism and VWF Full Gene Sequencing.

VWF Quantitative Multimer is the first offering in the market. By taking a historically subjective analysis and making it quantitative, BloodCenter has increased diagnostic confidence in the result. Doctors will have quantitative information on the severity of the multimer abnormality. Quantitation also acts as a new research tool for evaluation of patients with acquired defects of VWF such as people with heart disease being treated with a ventricular assist device.

VWF D1472H Ristocetin-binding Polymorphism affects the most commonly used test to evaluate VWF function — the VWF:RCo test. The D1472H variation of the VWF gene appears to interfere with this test, resulting in an inaccurate measure of VWF function. This genetic variation is carried by 63 percent of african Americans and 17 percent of caucasian Americans, and its significance was identified at BloodCenter’s Blood Research Institute by Veronica Flood, M.D. and Robert R. Montgomery, M.D. The D1472H test allows identification of these people promoting accurate patient diagnosis.

VWF Full Gene Sequencing adds to our current strength in genetic diagnosis of von Willebrand disease. Patients with a quantitative deficiency of VWF are at increased risk for bleeding. Many of these patients have low VWF levels due to defects of their VWF gene.

New Assays for von Willebrand Disease VWF gene sequence analysis expands the ability of physicians to diagnose their patients and improves the ability of patients to understand their disease and make family planning decisions. Many of the patient samples received at BloodCenter are for evaluation of inherited and acquired bleeding disorders. Correctly diagnosing von Willebrand Disease is complex but essential to providing optimal treatment.

BloodCenter of Wisconsin is actively involved in von Willebrand Disease research and one of the BloodCenter’s goals is to refine the diagnostic process by contributing sophisticated analytic methods and developing evidence-based treatment protocols.

The experience of our Diagnostic Laboratories team, and the desire to provide industry-leading service, enables BloodCenter to go beyond simply providing a test result. The Hemostasis Reference Laboratory’s Medical Director, Dr. Kenneth D. Friedman, M.D. stated, “These tests have the capacity to allow hematologists to take better care of their patients with von Willebrand Disease.”

Strong History of Innovation, Expertise and Results BloodCenter’s Hemostasis Reference Laboratory has an international reputation for its diagnostic services, scientific excellence, physician consultation and innovative approaches. Since its inception in 1981, the Hemostasis Reference Laboratory continues to provide comprehensive diagnostic testing for bleeding, thrombotic, fibrinolytic and platelet function disorders.

BloodCenter is unique as they have a large team of experts who are interested in advancing both the understanding and care of patients with von Willebrand Disease.  Team members include Thomas Abshire, MD; Veronica Flood, MD; Kenneth Friedman, MD; Joan Gill, MD; Sandy Haberichter, PhD and Robert Montgomery, MD.   “It is very rewarding to see a basic and clinical research finding translated into new clinically relevant testing that offers patients the opportunity for improved clinical care,” Dr. Montgomery indicated.

For more than 50 years, BloodCenter has helped clinicians serve patients with blood disorders. The Diagnostic Laboratories staff is dedicated to improving patient care through their leadership