Bayer Provides Update on Phase II/III Trial of BAY 86-6150

Berlin, May 3, 2013 – Bayer HealthCare today announced that a Phase II/III  trial evaluating the efficacy and safety of BAY 86-6150 in people with  hemophilia A and hemophilia B with inhibitors has been discontinued. The hope  that BAY 86-6150 might help patients with inhibitors to achieve better control  of their disease could not be fulfilled due to the detection of a neutralizing  antibody in the trial.

“Patient safety is our primary concern when designing clinical trials and  evaluating BAY 86-6150,” said Kemal Malik, MD, member of the Bayer HealthCare  Executive Committee and Head of Global Development. “Due to safety concerns, we  are discontinuing the BAY 86-6150 trial as a precautionary measure.”

About the Phase II/III Trial
The TRUST (TReatment with Unique recombinant rFVIIa STudy) trial is a Phase  II/III, multicenter, open-label clinical study designed to assess the safety  and efficacy of BAY 86-6150, a recombinant factor VIIa (rFVIIa) protein, in  patients with hemophilia A or B with inhibitors.

About Hemophilia A, Hemophilia B, and Inhibitors
Hemophilia A, also known as factor VIII deficiency or classic hemophilia, is  largely an inherited bleeding disorder in which one of the proteins needed to  form blood clots in the body is missing or reduced. Hemophilia A, the most  common type of hemophilia, is caused by a deficient or defective blood  coagulation protein, known as factor VIII. Hemophilia A is characterized by  prolonged or spontaneous bleeding, especially into the muscles, joints, or  internal organs.

Hemophilia B, also known as Christmas disease, is a less common type of  hemophilia.  People with hemophilia B have little or no factor IX.

Inhibitor development is a serious medical problem that can occur when a person  with hemophilia has an immune response to treatment with clotting factor  concentrates. About a third of hemophilia A patients develop inhibitors, with
the development of inhibitors being more common in hemophilia A.

About Bayer HealthCare
The Bayer Group is a global enterprise with core competencies in the fields of  health care, agriculture and high-tech materials. Bayer HealthCare, a subgroup  of Bayer AG with annual sales of EUR 18.6 billion (2012), is one of the world’s  leading, innovative companies in the healthcare and medical products industry  and is based in Leverkusen, Germany. The company combines the global activities  of the Animal Health, Consumer Care, Medical Care and Pharmaceuticals  divisions. Bayer HealthCare’s aim is to discover, develop, manufacture and  market products that will improve human and animal health worldwide. Bayer  HealthCare has a global workforce of 55,300 employees (Dec 31, 2012) and is  represented in more than 100 countries. More information at  www.healthcare.bayer.com.

Forward-looking statements
This release may contain forward-looking statements based on current  assumptions and forecasts made by Bayer Group or subgroup management. Various  known and unknown risks, uncertainties and other factors could lead to material  differences between the actual future results, financial situation, development  or performance of the company and the estimates given here. These factors  include those discussed in Bayer’s public reports which are available on the  Bayer website at www.bayer.com. The company assumes no liability whatsoever to  update these forward-looking statements or to conform them to future events or  developments.

For original Post click here.

FDA Accepts Biogen Idec’s Biologics License Application for First Long-Lasting Factor IX Therapy for Hemophilia B

WESTON, Mass.–(BUSINESS WIRE)–Mar. 4, 2013– Biogen Idec (NASDAQ: BIIB) announced today that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for the marketing approval of recombinant factor IX Fc fusion protein (rFIXFc) for the treatment of hemophilia B and granted the company a standard review timeline. Recombinant FIXFc is the first product candidate in a new class of long-lasting clotting factor therapies being developed with the goal of reducing the burden of treatment for hemophilia B.

“We are encouraged by the FDA’s acceptance of our application, as we believe rFIXFc has the potential to transform the care of hemophilia B by allowing for less frequent injections and helping patients to maintain low annualized bleeding rates,” said Glenn Pierce, M.D., Ph.D., senior vice president of Global Medical Affairs and chief medical officer of Biogen Idec’s hemophilia therapeutic area. “We are working with the FDA to bring the first major treatment advance for the hemophilia B community in 15 years.”

The regulatory submission was based on results from B-LONG, the largest registrational phase 3 clinical study in hemophilia B to date. The study showed that rFIXFc provides long-lasting protection from bleeding with fewer injections than are required with the current standard of care. The company’s BLA submission for rFVIIIFc for use in patients with hemophilia A is on track for filing during the first half of 2013.

About the Fc Fusion Technology Platform

rFIXFc is a clotting factor developed using Biogen Idec’s novel and proprietary monomeric Fc fusion technology, which makes use of a naturally occurring pathway that delays the destruction of factor and cycles it back into the bloodstream, resulting in a longer circulating half-life.

With this technology, rFIXFc is designed to provide long-lasting protection from bleeding and reduce the treatment burden associated with hemophilia B, which currently can require more than 100 injections annually for prophylaxis with commercially-available factor IX products. Fc fusion technology is used in seven FDA-approved products for the long-term treatment of chronic diseases including rheumatoid arthritis, psoriasis and platelet disorders.

About Hemophilia B

Hemophilia B is a rare, inherited disorder in which the ability of a person’s blood to clot is impaired. Hemophilia B occurs in about one in 25,000 male births annually and is caused by having substantially reduced or no factor IX activity, which is needed for normal blood clotting. People with hemophilia B therefore need injections of factor IX to restore the coagulation process and prevent frequent bleeds that could otherwise lead to pain, irreversible joint damage and life-threatening hemorrhages. The Medical and Scientific Advisory Council of the National Hemophilia Foundation recommends prophylaxis as the optimal therapy for people with severe hemophilia B. Currently, prophylaxis in hemophilia B typically requires injections up to three times per week to maintain a sufficient circulating level of clotting factor.

About the Biogen Idec and Sobi Collaboration

Biogen Idec and Swedish Orphan Biovitrum (Sobi) are partners in the development and commercialization of rFIXFc in hemophilia B and rFVIIIFc in hemophilia A. Biogen Idec leads development, has manufacturing rights, and has commercialization rights in North Americaand 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 www.biogenidec.com.

Safe Harbor

This press release contains forward-looking statements, including statements about the commercialization and impact of long-lasting hemophilia therapies and regulatory filings. 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 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.

Source: Biogen Idec press release

Biogen Idec Media Contact:
Banks Willis, +1-781-464-3260
Public Affairs
or
Biogen Idec Investor Relations Contact:
Kia Khaleghpour, +1-781-464-2442
Director, Investor Relations

Heart Health and Hemophilia

Hemophilia not protective against heart disease

By Sarah Aldridge | 02.06.2013

In the past decade, research has shown that people with hemophilia are just as prone to heart conditions as the general public.

In a six-state study of more than 3,400 men with hemophilia, investigators working with the Centers for Disease Control and Prevention in Atlanta uncovered some hints about heart health and adults with hemophilia. “After HIV and intracranial hemorrhage, the third most common cause of death was heart disease,” says Roshni Kulkarni, MD, director of pediatric and adolescent hematology/oncology and professor in the Department of Pediatrics at Michigan State University in East Lansing. She was lead author of the study, published in the American Journal of Hematology in 2005. Using data from hospital records, Kulkarni and associates found the incidence of ischemic heart disease (reduced blood supply to the heart usually from coronary artery disease) was not significantly different when compared to nonhemophilic men. “They were at risk for heart disease just like the rest of the population, so hemophilia was not protective.”

In a 2009 research study published in Haemophilia, Barbara Konkle, MD, and colleagues identified cardiovascular disease as a co-morbidity of older men with hemophilia. A 2010 study in the Journal of Thrombosis and Haemostasis then showed that men with hemophilia had equivalent incidences of atherosclerosis (narrowing of the arteries) as men in the general population. A study of 185 men with hemophilia at the Indiana Hemophilia & Thrombosis Center in Indianapolis, published in Haemophilia in 2011, further showed they were twice as likely to develop coronary artery disease, stroke or heart attack as non-Hispanic white males. High blood pressure and smoking were contributing risk factors.

Further, exposure to high factor levels can trigger increased clot formation. This is a greater concern for patients with hemophilia B or inhibitors who use activated prothrombin complex concentrates (APCCs). “Taking an APCC is clearly a risk factor for heart disease,” Kulkarni says. “If you have bad blood vessels in your heart, they can form a clot there.”

Routine Heart Screenings

Primary care providers (PCPs) who perform routine screenings for cholesterol and triglycerides, can help identify and treat conditions that lead to heart disease.

So if you’re anxious about the angina that runs in your family, schedule an appointment with your PCP today. You’ll do your heart a favor.

Learn More

• Foley CJ, et al. Coronary atherosclerosis and cardiovascular mortality in hemophilia. Journal of Thrombosis and Haemostasis2010; 8:208–211.
• Konkle BA, et al. Emerging clinical concerns in the ageing haemophilia patient. Haemophilia 2009; 15(6):1197–1209.
• Kulkarni R, et al. Prevalence and risk factors for heart disease among males with hemophilia. American Journal of Hematology 2005; 70:36–42.
• Sharathkumar AA, et al. Prevalence and risk factors of cardiovascular disease (CVD) events among patients with haemophilia: Experience of a single haemophilia treatment centre in the United States (US). Haemophilia 2011; 17(4):597–604.

Inspiration Biopharmaceuticals and Ipsen Complete Sale Process for All Hemophilia Assets

Cambridge, MA February 6, 2013 – Inspiration Biopharmaceuticals (Inspiration) and Ipsen
today announced the sale of its proprietary hemophilia B product, IB1001 (recombinant FIX), to
Cangene Corporation (TSX: CNJ) (Cangene). Ipsen (Euronext: IPN; ADR: IPSEY) sold its
commercialization rights to IB1001 as part of the transaction. Cangene will acquire worldwide
rights to IB1001, a recombinant factor IX currently under regulatory review in the United States
and Europe. This transaction follows the announcement last month that Inspiration and Ipsen
had agreed to sell OBI-1 (recombinant porcine FVIII) to Baxter International. The announcement
of today’s transaction completes the sale process of Inspiration’s hemophilia programs.
Inspiration and Ipsen have signed an asset purchase agreement pursuant to which Cangene
would acquire worldwide rights to IB1001, as well as Inspiration’s rights to two product
candidates in pre-clinical development: IB1007 (recombinant FVIIa) and IB1008 (recombinant
FVIII). The total aggregate consideration for these rights may exceed $300 million; including the
upfront payment of $5.9 million, sales milestones totaling $50 million and annual net sales
payments tiered up to a double-digit percentage of global net sales.
Inspiration and Ipsen previously announced the sale of OBI-1 to Baxter International for a total
aggregate consideration that may exceed $700 million, including $135 million in upfront and
milestone payments. The United States Bankruptcy Court in Boston approved that transaction
on January 24, 2012. With the signing of the transaction for IB1001, total aggregate
consideration for Inspiration’s assets may exceed $1 billion.
John P. Butler, Inspiration’s Chief Executive Officer commented; “OBI-1 and IB1001 are
important products for people living with hemophilia, bringing innovation and expanding
treatment options. We are very pleased that Baxter and Cangene will bring these products to
market.”
IB1001 is currently under regulatory review by both the FDA in the US and the EMA in Europe.
The product was placed on clinical hold by the FDA in July 2012. Inspiration has been working
to resolve the issues that led to the clinical hold and has discussed its plans to address the
clinical hold with relevant regulatory authorities. “IB1001 was the first product Inspiration
developed, with a vision to expand global access to safe and effective recombinant therapies for people living with hemophilia. We are very pleased that Cangene will take IB1001 forward to
make this vision a reality,” commented John R. Taylor, Inspiration’s Chairman and a founder of
the company.
The asset purchase agreement was signed on February 6, 2013, and filed with the United
States Bankruptcy Court in Boston. The sale is a result of a joint sale process pursued by
Inspiration and Ipsen shortly after Inspiration filed for protection under Chapter 11 of the U.S.
Bankruptcy Code on October 30, 2012. Ipsen has been providing Inspiration with Debtor-inPossession (DIP) financing to fund Inspiration’s operations during the sale process.
The sale is subject to certain closing conditions, including Bankruptcy Court approval. Evercore
Partners served as exclusive financial advisor to Inspiration and Ipsen on the transaction.
Ropes & Gray served as legal advisor to Inspiration on the transaction. Murphy & King is
Inspiration’s bankruptcy counsel and FTI Consulting, Inc. is Chief Restructuring Officer for
Inspiration.

About Inspiration Biopharmaceuticals

As the only biopharmaceutical company dedicated solely to hemophilia, Inspiration is committed
to improving the care of people with this condition by broadening treatment choices, expanding
global access to care and advancing innovative therapies. Founded by two families whose sons
have hemophilia, Inspiration is inspired to make a difference in the lives of people impacted by
hemophilia around the world. Inspiration’s lead product candidates are IB1001, an
investigational intravenous recombinant factor IX being developed for the treatment of
hemophilia B, and OBI-1, an investigational recombinant porcine factor VIII being developed for
the treatment of serious bleeds in patients with congenital hemophilia A with inhibitors or
acquired hemophilia A.
For more information about Inspiration Biopharmaceuticals, please visit http://www.inspirationbio.com.

Contact

Michel Dahan
VP Commercial Development & Strategic Planning
Tel.: +1-617-588-1807
Email: mdahan@inspirationbio.com

For original press release, click here.

NEW PHASE 3 DATA REINFORCE LONG-LASTING PROTECTION FROM BLEEDING FOR PATIENTS WITH HEMOPHILIA A AND B

– Recombinant Fc fusion proteins show potential to transform care by providing long-lasting protection from bleeding with fewer injections than the current standard of care –

WARSAW, Poland–(BUSINESS WIRE)–Feb. 8, 2013– Biogen Idec (NASDAQ: BIIB) andSwedish Orphan Biovitrum (Sobi) (STO: SOBI) released data that confirmed the ability of investigational recombinant factors VIII Fc fusion protein (rFVIIIFc) and IX Fc fusion protein (rFIXFc) to provide long-lasting protection from bleeding with fewer injections than are required with the current standard of care for people with hemophilia. The data, from the largest phase 3 registrational studies conducted in hemophilia to date, were presented this week at the 6th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD).

The studies compared the pharmacokinetic activity of rFVIIIFc for hemophilia A and rFIXFc for hemophilia B to currently available treatments. In the studies, the long-lasting candidates stayed active in the body longer, enabling study participants to prevent bleeding with less frequent injections than are required with the current standard of care. In the A-LONG study, patients with hemophilia A were able to use once to twice weekly prophylactic (preventative dosing) injections of rFVIIIFc while maintaining low bleeding rates. In the B-LONG study, rFIXFc allowed patients with hemophilia B to use prophylactic injections every one to two weeks with low bleeding rates.

“Data from these phase 3 trials demonstrate a potential to transform the treatment of hemophilia by offering long-lasting protection from bleeding while meaningfully reducing treatment burden associated with this rare disease,” said Glenn Pierce, M.D., Ph.D., senior vice president of Global Medical Affairs and chief medical officer of Biogen Idec’s hemophilia therapeutic area. “Less frequent injections may help more people with hemophilia adhere to a preventative treatment schedule, which can help prevent the long-term health consequences associated with treating a bleed after it occurs.”

The current standard of care for hemophilia A and B requires frequent injections, which are a burden for patients. Prophylactic treatment for hemophilia A typically requires injections three times per week or every other day, and injections 2-3 times per week for the treatment of hemophilia B, according to the National Hemophilia Foundation’s Medical and Scientific Advisory Council guidelines. People with severe hemophilia who do not follow a prophylactic injection schedule remain vulnerable to bleeding that can cause irreversible joint damage and life-threatening hemorrhages.

Recombinant FVIIIFc and recombinant FIXFc were developed using Fc fusion technology, which has safely been used in FDA-approved medicines for more than a decade. Biogen Idecand Swedish Orphan Biovitrum applied Fc fusion technology in hemophilia for the first time with the goal of making clotting factors last longer and reduce the burden of injections for patients and their families.

About the A-LONG (rFVIIIFc) Data at EAHAD
The A-LONG results confirm the long-lasting characteristics of rFVIIIFc; specifically, the data show that rFVIIIFc stays in the body for 50 percent longer than Advate^® [antihemophilic factor (recombinant), plasma/albumin-free method], the most frequently used factor VIII therapy. In the trial, the terminal half-life for rFVIIIFc was 19 hours compared to 12 hours for Advate. Other measures of rFVIIIFc’s activity in the body reinforce its long-lasting characteristics: the mean time for maintaining a clotting factor activity level associated with less bleeding (time to 1 percent) was approximately 5 days for rFVIIIFc compared to 3.5 days for Advate and the average rate at which rFVIIIFc was cleared from the body was 2.0 mL/hr/kg compared with 3.0 mL/hr/kg for Advate. In the study’s individualized prophylaxis arm, patients received rFVIIIFc at a median dosing interval of 3.5 days and a median weekly dose of 78 IU per kg to prevent bleeding, which compares favorably to the recommended dose for the standard of care. Nearly one-third of patients were able to achieve every 5 day dosing in this arm. Overall, the A-LONG data indicate that rFVIIIFc has the potential to become the first product to offer hemophilia A patients long-lasting protection from bleeding with less frequent dosing than the current standard of care.

The A-LONG data were presented in the late-breaking oral abstract session and in poster 104, “Phase 3 clinical study of recombinant FC fusion factor FVIII (rFVIIIFc) demonstrated safety, efficacy, and improved pharmacokinetics (A-LONG).”

About the B-LONG (rFIXFc) Data at EAHAD
The B-LONG results confirm the long-lasting characteristics of rFIXFc; specifically, the data show that rFIXFc stays in the body for more than twice as long as BeneFIX^® [Coagulation Factor IX (Recombinant)], the only recombinant factor IX therapy currently approved for prophylactic use. The terminal half-life for rFIXFc was 82 hours compared to 34 hours for BeneFIX. Other measures of rFIXFc’s activity in the body reinforce its long-lasting characteristics: the mean time for maintaining a normal clotting factor activity level (time to 1 percent) was 11 days for rFIXFc compared to 5 days for BeneFIX and the average rate at which rFIXFc was cleared from the body was 3.2 mL/hr/kg compared with 6.3 mL/hr/kg for BeneFIX. All patients in the individualized interval prophylaxis arm of the study were able to go at least one week between rFIXFc injections and 50 percent were able to go 14 days or longer before needing another dose to prevent bleeding. The median weekly dose was 45 IU per kg, comparable to the recommended dose for the current standard of care. Overall, the B-LONG data support the potential for rFIXFc to become the first product to offer hemophilia B patients long-lasting protection from bleeding with a more convenient injection schedule than the current standard of care.

The B-LONG data were presented in poster 115, “Safety, efficacy, and improved pharmacokinetics (PK) demonstrated in a phase 3 clinical trial of extended half-life recombinant FC fusion factor IX (B-LONG).”

Importantly, the difference in the duration of activity of rFVIIIFc and rFIXFc was expected and consistent with the differences between the natural clotting factors that these products augment. Fc Fusion technology extends FVIII and FIX differently based on the biological differences in hemophilia A and B. The length of time that FVIII stays active is dictated by its own duration as well as that of the blood protein that it binds to, known as von Willebrand factor. The activity of FIX is not restricted in this way.

“These new data support the application of Fc fusion technology in hemophilia, using a naturally occurring pathway to delay the breakdown of factor in the body and cycle it back into the bloodstream,” said Birgitte Volck, M.D., Ph.D., senior vice president & chief medical officer of Sobi. “While Fc fusion has been used in medicines for more than a decade, rFVIIIFc and rFIXFc are the first investigational therapies to use the technology to successfully extend the half-lives of clotting factors, which could offer protection from bleeding while reducing the burden of injections for people with hemophilia.”

About the A-LONG Study and the rFVIIIFc Program
A-LONG was a global, open-label, multi-center phase 3 study that evaluated the efficacy, safety and pharmacokinetics of intravenously-injected rFVIIIFc in 165 male patients aged 12 years and older. The study results, first announced in October 2012, showed that rFVIIIFc was effective in the control and prevention of bleeding, routine prophylaxis and perioperative management, with low single-digit median annualized bleeding rates using individualized and weekly prophylactic regimens. Overall, 98 percent of bleeding episodes were controlled by one or two injections of rFVIIIFc. Recombinant FVIIIFc was generally well-tolerated and no inhibitors to rFVIIIFc were detected. The most common adverse events (incidence of ≥5 percent) occurring outside of the perioperative management period were nasopharyngitis, arthralgia, headache and upper respiratory tract infection. No serious adverse events were assessed to be related to the therapy by the investigators.

Ongoing clinical studies of rFVIIIFc include Kids A-LONG, for previously-treated children with hemophilia A under age 12, and ASPIRE, for patients who completed the A-LONG study or who complete the Kids A-LONG study.

About the B-LONG Study and the rFIXFc Program
B-LONG was a global, open-label, multi-center phase 3 study that evaluated the efficacy, safety and pharmacokinetics of intravenously-injected rFIXFc in 123 male patients aged 12 years and older. The study results, first announced in September 2012, showed that rFIXFc was effective in the control and prevention of bleeding, routine prophylaxis, and perioperative management, with low single-digit median annualized bleeding rates using individualized prophylactic regimens at a median dosing interval of 14 days. More than 90 percent of bleeding episodes were controlled by a single injection of rFIXFc. Recombinant FIXFc was generally well-tolerated and no inhibitors to rFIXFc were detected. The most common adverse events (incidence of ≥5 percent) occurring outside of the perioperative management arm (i.e., Arms 1, 2 and 3, but not Arm 4) were nasopharyngitis, influenza, arthralgia (joint pain), upper respiratory infection, hypertension and headache. One serious adverse event, obstructive uropathy in the setting of hematuria, was assessed to be possibly related to therapy by the investigator. The patient continued rFIXFc treatment and the event resolved with medical management.

Ongoing clinical studies of rFIXFc include Kids B-LONG, for previously treated children with hemophilia B under age 12, and B-YOND, for patients who completed the B-LONG study or who complete the Kids B-LONG study.

About the Fc Fusion Technology Platform
Recombinant FVIIIFc and recombinant FIXFc are clotting factors developed using Biogen Idec’s novel and proprietary monomeric Fc fusion technology, which makes use of a naturally occurring pathway that delays the breakdown of factor in the body and cycles it back into the bloodstream, enabling it to remain in the body longer following an injection. Fc fusion technology is used in seven FDA-approved products for the long-term treatment of chronic diseases including rheumatoid arthritis, psoriasis and platelet disorders.

About Hemophilia
Hemophilia is a rare, inherited disorder in which the ability of a person’s blood to clot is impaired. Hemophilia A is caused by reduced or no Factor VIII protein, whereas hemophilia B is caused by reduced or no Factor IX protein. Both proteins are needed for normal blood clotting. Hemophilia A and hemophilia B occur in about one in 5,000 and one in 25,000 male births, respectively. People with hemophilia need injections of clotting factors to restore the coagulation process and prevent frequent bleeds that could otherwise lead to pain, irreversible joint damage and life-threatening hemorrhages. The Medical and Scientific Advisory Council of the National Hemophilia Foundation recommends prophylaxis as the optimal therapy for people with severe hemophilia A and severe hemophilia B. Currently, prophylaxis for hemophilia A typically requires injections three times per week or every other day to maintain a sufficient circulating level of clotting factor, while prophylaxis in hemophilia B typically requires injections two to three times per week.

About the Biogen Idec and Sobi Collaboration
Biogen Idec and Sobi are partners in the development and commercialization of rFVIIIFc and rFIXFc. 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, Russia, the Middle Eastand 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 visitwww.biogenidec.com.

About Sobi
Sobi is an international healthcare company dedicated to bringing innovative therapies and services to improve the lives of rare disease patients. The product portfolio is primarily focused on inflammation and genetic diseases, with three late stage biological development projects within hemophilia and neonatology. Sobi also markets more than 40 products for companies in the specialty and rare disease space. In 2011, Sobi had revenues of SEK 1.9 billion and around 500 employees. The share (STO: SOBI) is listed on NASDAQ OMX Stockholm. More information is available at www.sobi.com.

Safe Harbor
This press release contains forward-looking statements, including statements about the commercialization and impact of long-lasting hemophilia therapies. 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 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 Biogen Idec’s drug development and commercialization activities, please review the Risk Factors section of Biogen Idec’s 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.

Source: Biogen Idec

BIOGEN IDEC CONTACT:
Media Contact:
Jeff Boyle, +1-781-464-3260
or
Investor Relations Contact:
Kia Khaleghpour, +1-781-464-2442
or
SWEDISH ORPHAN BIOVITRUM CONTACT:
Media Contact:
Birgitte Volck, +45 4 012 2310/+46 8 697 2188
or
Analyst/Investor Contact:
Jorgen Winroth, +46 8 697 2000/+1-212-579-0506

Click here for original Press Release.

Baxter Phase III of FEIBA NF for Prophylaxis Treatment

Baxter Announces Phase III Data Evaluating Prophylaxis Treatment of FEIBA NF for Hemophilia Patients with Inhibitors

Pivotal study to form basis for upcoming biologic license application to the U.S. FDA

DEERFIELD, Ill., JANUARY 8, 2013 – Baxter International Inc. (NYSE:BAX) today announced pivotal Phase III study results evaluating the efficacy and safety of routine prophylaxis compared to on-demand treatment of FEIBA NF [Anti-Inhibitor Coagulant Complex], Nanofiltered and Vapor Heated, in patients with hemophilia A or B and inhibitors. Top-line results from the study showed a reduced median annual bleed rate (ABR) from 28.7 during FEIBA NF on-demand treatment to 7.9 during FEIBA NF prophylactic treatment (a 72.5% reduction). The Phase III study will form the basis of a biologics license application (BLA) to be filed with the U.S. Food and Drug Administration (FDA) in the first quarter of 2013.

As many as one-third of people with hemophilia develop an inhibitor to a product used to treat or prevent bleeding episodes. 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.

“Treatment with FEIBA NF resulted in a significant reduction in annual bleed rate (ABR) of all types of bleeds in the prophylaxis arm as compared to the on-demand arm,” said lead investigator, Dr. Sandra Antunes MD, UNIFESP, Sao Paulo, Brazil. “Three of the 17 intent to treat patients (17.6 %) in the prophylaxis arm did not experience any bleeding episodes during the study, and this is very significant for hemophilia patients with inhibitors.”

The Phase III prospective, open label, randomized, multi-center, parallel study investigated the efficacy, safety and health-related quality of life benefits of FEIBA NF prophylactic treatment compared to on-demand treatment in 36 patients with hemophilia A or B and inhibitors over a 12-month period. The most commonly reported adverse reactions in the study were hypersensitivity, dizziness, headache, rash, hypotension and hepatitis B surface antibody positive laboratory test result. The occurrence of a transitory increase in hepatitis B surface antibodies has been seen in certain plasma-derived products and could be attributed to the passive transfer of antibodies following FEIBA NF treatment. None of the subjects showed any signs or symptoms of hepatitis B infection.

This latest study adds to the clinical evidence supporting the prophylactic use of FEIBA, building on an investigator initiated study showing that FEIBA can reduce bleeding events in patients with severe hemophilia A and inhibitors when compared to on-demand treatment (results published in The New England Journal of Medicine in November 2011).

“One of the greatest remaining challenges in the management of hemophilia is the development of inhibitors, which can lead to more difficult-to-control and sometimes life-threatening bleeding. The FEIBA NF prophylaxis study demonstrates Baxter’s dedication to providing treatment options to the hemophilia community, including effective inhibitor management,” said Prof. Hartmut J. Ehrlich, M.D., vice president of global research and development in Baxter’s BioScience business.

About FEIBA NF
FEIBA NF is not indicated for prophylaxis use in the United States. Canada, The Netherlands, Israel, Australia/New Zealand, Japan and South Korea also do not have a prophylaxis indication.

Indications for FEIBA NF
In the U.S., FEIBA NF [Anti-Inhibitor Coagulant Complex] is indicated for the control of spontaneous bleeding episodes or to cover surgical interventions in hemophilia A and hemophilia B patients with inhibitors.

Clinical experience suggests that patients with a Factor VIII inhibitor titer of less than five Bethesda Units (B.U.) may be successfully treated with Antihemophilic Factor.

Patients with titers ranging between 5 and 10 B.U. may either be treated with Antihemophilic Factor or FEIBA NF. Cases with Factor VIII inhibitor titers greater than 10 B.U. have generally been refractory to treatment with Antihemophilic Factor.

Inadequate response to treatment may result from an abnormal platelet count or impaired platelet function that were present before treatment with FEIBA NF, Nanofiltered and Vapor Heated.

Detailed Important Risk Information for FEIBA NF
Thrombotic and thromboembolic events have been reported during postmarketing surveillance following infusion of FEIBA VH or FEIBA NF, particularly following the administration of high doses and/or in patients with thrombotic risk factors.

The use of FEIBA NF is contraindicated:

• In patients who have known anaphylactic or severe hypersensitivity reactions to the product.
• In patients who are known to have a normal coagulation mechanism.
• For the treatment of bleeding episodes resulting from coagulation factor. deficiencies in the absence of inhibitors to coagulation factor VIII or coagulation factor IX.
• In patients with significant signs of disseminated intravascular coagulation (DIC).
• In patients with acute thrombosis or embolism (including myocardial infarction).

At first sign or symptoms of an infusion/hypersensitivity reaction or a thrombotic/thromboembolic event, FEIBA NF administration should be stopped immediately and diagnostic and therapeutic measures initiated as appropriate.

Allergic-type hypersensitivity reactions, including severe anaphylactoid reactions, have been reported following the infusion of FEIBA. The symptoms include urticaria, angioedema, gastrointestinal manifestations, bronchospasm, and hypotension; these reactions can be severe and can be systemic.

Many of the reported cases of thromboembolic events occurred with doses above 200 units/kg/day or in patients with other risk factors.

Infusion of FEIBA NF should not exceed single dosage of 100 U/kg and daily doses of 200 U/kg of body weight. Patients receiving more than 100 U/kg of FEIBA NF must be monitored for the development of DIC and/or symptoms of acute coronary ischemia. High doses of FEIBA NF should be given only as long as absolutely necessary to stop bleeding.

FEIBA VH or FEIBA NF should be used with particular caution and only if there are no therapeutic alternatives in patients at risk of DIC, arterial or venous thrombosis.

If clinical signs of intravascular coagulation occur, which include changes in blood pressure, changes in pulse rate, respiratory distress, chest pain and/or cough, infusion of FEIBA NF should be stopped promptly.

Non-hemophilic patients with acquired inhibitors against factors VIII, IX or XII may have both a bleeding tendency and an increased risk of thrombosis at the same time.

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

Adverse reactions reported in clinical studies with FEIBA were anamnestic response, somnolence, dizziness, dysgeusia, dyspnea, hypoesthesia, nausea, chills, pyrexia, chest pain and chest discomfort.

Please see full prescribing information for FEIBA NF at:
http://www.baxter.com/downloads/healthcare_professionals/products/feiba-nf-pi.pdf.

Licenses and licensing conditions may vary from country to country; therefore please always consult your local full prescribing information. Please check FEIBA NF website for information on indications approved in other countries.

About Hemophilia A
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  According to the World Federation of Hemophilia, more than 400,000 people in the world have hemophilia.²  All races and economic groups are affected equally.¹

About Hemophilia B
Hemophilia B is the second most common type of hemophilia (also known as Christmas disease) and is the result of insufficient amounts of clotting factor IX, a naturally occurring protein in blood that controls bleeding.³  Approximately 25,000 people worldwide, including more than 4,000 in the U.S., have been diagnosed with hemophilia B.⁴  Hemophilia B is often a debilitating, chronic disease with complications that include bleeding episodes, hemophilic arthropathy (bleeding into a joint) and hospitalization.⁵

About Inhibitors
As many as one-third of 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 therapy. Inhibitors cause the body to work against the factor replacement therapy, neutralizing its effect and preventing an individual’s blood from appropriate clotting.⁶  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, lower life expectancy and poor health-related quality of life.^6,7

About Baxter in Hemophilia
Baxter has more than 60 years experience in hemophilia and has introduced a number of therapeutic firsts for hemophilia patients. Baxter has the broadest portfolio of hemophilia treatments in the industry and is able to meet individual therapy choices, providing a range of options at each treatment stage. The company’s work is focused on optimizing hemophilia care and improving the lives of people living with hemophilia A and B worldwide.

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 the company’s Phase III study evaluating the efficacy and safety of routine prophylaxis compared to on-demand treatment of FEIBA NF in hemophilia patients with inhibitors, including expectations regarding related regulatory filings. 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: satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; changes in laws and regulations; product quality or patient safety issues; and other risks identified in Baxter’s most recent filing on Form 10-K and other SEC filings, all of which are available on Baxter’s website. Baxter does not undertake to update its forward-looking statements.

¹What is Hemophilia? World Federation of Hemophilia. Accessed on: May 24, 2012. Available at: http://www.wfh.org/en/page.aspx?pid=646 

²Frequently Asked Questions About Hemophilia. World Federation of Hemophilia. Accessed on: May 24, 2012. Available at:http://www.wfh.org/en/page.aspx?pid=637 

³Frequently Asked Questions About Hemophilia. World Federation of Hemophilia. Accessed on April 20, 2012 Available at:http://www.wfh.org/en/page.aspx?pid=637 

⁴World Federation of Hemophilia Report on the Annual Global Survey 2010. World Federation of Hemophilia. Accessed on April 20, 2012. Available at:http://www1.wfh.org/publications/files/pdf-1427.pdf

⁵Lee, C. A. (2011) Hemophilia Care in the Modern World, in Current and Future Issues in Hemophilia Care (eds E.-C. Rodríguez-Merchán and L. A. Valentino), Wiley-Blackwell, Oxford, UK. Accessed on April 20, 2012. Screen shot of page available here

⁶What are Inhibitors (section)? World Federation of Hemophilia. Accessed on December 14, 2012. Available at: http://www.wfh.org/en/page.aspx?pid=651 

⁷Leissinger, Cindy A. Prevention of Bleeds in Hemophilia Patients With Inhibitors: Emerging Data and Clinical Direction. American Journal of Hematology. 2004; 77:187-193.

 

For Original Press release, click here.

Biogen Idec Submits Application to FDA for First Long-Lasting Factor IX Therapy for Hemophilia B

WESTON, Mass.–(BUSINESS WIRE)–Jan. 4, 2013– Today Biogen Idec (NASDAQ: BIIB) announced the company recently submitted a Biologics License Application (BLA) to theU.S. Food and Drug Administration (FDA) for the marketing approval of recombinant factor IX Fc fusion protein (rFIXFc) for the treatment of hemophilia B. rFIXFc is the first product candidate in a new class of long-lasting clotting factor therapies that are being developed with the goals of reducing the burden of treatment for this condition and enhancing protection from bleeding. The regulatory submission was based on results from B-LONG, the largest registrational phase 3 clinical study in hemophilia B to date.

“The submission to FDA of rFIXFc is an important milestone towards the introduction of a long-lasting factor IX therapy which is the first major treatment advance for the hemophilia B community in 15 years,” said Glenn Pierce, M.D., Ph.D., senior vice president of Global Medical Affairs and chief medical officer of Biogen Idec’s hemophilia therapeutic area. “Our Fc fusion technology uses a natural pathway in the body to keep factor in circulation longer. The clinical efficacy and safety data for rFIXFc were robust and showed that dosing every one to two weeks provided significant protection from bleeding. Based on these data, we believe rFIXFc has the potential to have a major impact on the global adoption of prophylactic treatment in hemophilia B, helping patients prevent bleeding episodes and potentially reduce their long-term consequences.”

Biogen Idec’s BLA submission to the U.S. FDA for rFVIIIFc for use in patients with hemophilia A is on track for filing in 1H 2013.

About the Fc Fusion Technology Platform

rFIXFc is a clotting factor developed using Biogen Idec’s novel and proprietary monomeric Fc fusion technology, which makes use of a naturally occurring pathway that delays the destruction of factor and cycles it back into the bloodstream, resulting in a longer circulating half-life.

With this technology, rFIXFc is designed to provide long-lasting protection from bleeding and reduce the treatment burden associated with hemophilia B, which currently can require more than 100 injections annually for prophylaxis with commercially-available factor IX products. Fc fusion technology is used in seven FDA-approved products for the long-term treatment of chronic diseases including rheumatoid arthritis, psoriasis and platelet disorders.

About Hemophilia B

Hemophilia B is a rare, inherited disorder in which the ability of a person’s blood to clot is impaired. Hemophilia B occurs in about one in 25,000 male births annually and is caused by having substantially reduced or no factor IX activity, which is needed for normal blood clotting. People with hemophilia B therefore need injections of factor IX to restore the coagulation process and prevent frequent bleeds that could otherwise lead to pain, irreversible joint damage and life-threatening hemorrhages. The Medical and Scientific Advisory Council of the National Hemophilia Foundation recommends prophylaxis as the optimal therapy for people with severe hemophilia B. Currently, prophylaxis in hemophilia B typically requires injections up to three times per week to maintain a sufficient circulating level of clotting factor.

About the Biogen Idec and Sobi Collaboration

Biogen Idec and Swedish Orphan Biovitrum (Sobi) are partners in the development and commercialization of rFIXFc in hemophilia B and rFVIIIFc in 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 www.biogenidec.com.

Safe Harbor

This press release contains forward-looking statements, including statements about the commercialization and impact of long-lasting hemophilia therapies and regulatory filings. 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 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.

Source: Biogen Idec

Biogen Idec Media Contact:
Jeff Boyle, 781-464-3260
Senior Director, Public Affairs
or
Biogen Idec Investor Relations Contact:
Kia Khaleghpour, 781-464-2442
Director, Investor Relations

For Original Press Release, Click here.

Baxter Presents Phase I/III Data Evaluating Safety and Efficacy of BAX 326, an Investigational Recombinant Factor IX for Hemophilia B

Study Shows More Than 40 Percent of Patients Experienced No Bleeds

BAX 326 Granted Orphan-drug Designation by U.S. Food and Drug Administration

DEERFIELD, Ill.–(BUSINESS WIRE)– Baxter International Inc. (BAX) today announced pivotal Phase I/III study results evaluating the safety and efficacy of BAX 326, an investigational recombinant factor IX (rFIX) protein, for the treatment and prophylaxis of bleeding episodes for patients with hemophilia B over 12 years of age. The data were presented at the 54^th Annual Meeting and Exposition of the American Society of Hematology (ASH) in Atlanta. BAX 326 was recently granted orphan-drug designation by the U.S. Food and Drug Administration (FDA), a special status given to a product that when approved, will address an unmet need for people with a rare disease or condition. Data supporting the prophylaxis indication, which is the basis for the orphan drug designation, was included in the biologics license application (BLA).

”With only one recombinant protein currently available to treat hemophilia B, it was important to focus research efforts on finding another option for patients with this debilitating disease,” said lead investigator Jerzy Windyga, MD, PhD, Institute of Hematology and Blood Transfusion, Warsaw, Poland. ”In this study, more than 40 percent of patients using BAX 326 as a prophylactic treatment experienced no bleeds, an important finding given the potentially harmful impact of bleeding episodes for patients.”

The Phase I/III prospective, controlled, multicenter study investigated the pharmacokinetics, efficacy and safety of BAX 326 in 73 previously-treated patients with severe or moderately severe hemophilia B. Results from the study showed that twice-weekly prophylactic treatment with BAX 326 achieved a median annualized bleed rate of 1.99 with 43 percent of patients experiencing no bleeds. The impact of prophylaxis with BAX 326 also translated into statistically significant improvements in physical health-related quality of life (HRQoL), as measured by improvements in the Bodily Pain and Role-Physical domains in the Short Form Health Survey (SF-36v2) tool. Statistically significant changes were not seen in the other physical health-related domains or the mental health domains.

No inhibitors were detected and no cases of anaphylaxis were reported in any patients. Three treatment-related adverse events were reported in 2 of the 73 (2.7%) patients, all of which were mild and transient: dysgeusia (distortion of the sense of taste) occurred twice in one patient and pain in extremity occurred once in another patient. More than 70 percent of subjects (56 of 73) had 50 or more exposure days to BAX 326 during the study.

”As a company committed to advancing care for people with hemophilia for more than 60 years, Baxter continues to deliver significant innovation in the field of hemophilia. If it is approved, BAX 326 will expand treatment options for patients,” said Prof. Harmut J. Ehrlich, M.D., vice president of global research and development in Baxter’s BioScience business.

To continue on its path of innovation, earlier this year Baxter announced a partnership with Chatham Therapeutics, LLC to develop a gene therapy-based treatment for hemophilia B. Gene therapy represents another important area of research that may become the future of hemophilia B treatment.

About Hemophilia B

Hemophilia B is the second most common type of hemophilia (also known as Christmas disease) and is the result of insufficient amounts of clotting factor IX, a naturally occurring protein in blood that controls bleeding.¹ Approximately 25,000 people worldwide, including more than 4,000 in the U.S., have been diagnosed with hemophilia B. ² Hemophilia B is often a debilitating, chronic disease with complications that include bleeding episodes, hemophilic arthropathy (bleeding into a joint) and hospitalization.³

About Baxter in Hemophilia

Baxter has more than 60 years experience in hemophilia and has introduced a number of therapeutic firsts for hemophilia patients. Baxter has the broadest portfolio of hemophilia treatments in the industry and is able to meet individual therapy choices, providing a range of options at each treatment stage. The company’s work is focused on optimizing hemophilia care and improving the lives of people living with hemophilia A and B worldwide.

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 BAX 326, an investigational recombinant factor IX (rFIX) protein, as well as Baxter’s partnership with Chatham Therapeutics, LLC to develop a gene therapy-based treatment for hemophilia B. 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: satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; additional clinical results; changes in laws and regulations; product quality or patient safety issues; and other risks identified in Baxter’s most recent filing on Form 10-K and other SEC filings, all of which are available on Baxter’s website. Baxter does not undertake to update its forward-looking statements.

¹ Frequently Asked Questions About Hemophilia. World Federation of Hemophilia. Accessed on November 28, 2012. Available at: http://www.wfh.org/en/page.aspx?pid=637

² World Federation of Hemophilia Report on the Annual Global Survey 2010. World Federation of Hemophilia. Accessed on November 28, 2012. Available at:http://www1.wfh.org/publications/files/pdf-1427.pdf

³ Lee, C. A. (2011) Hemophilia Care in the Modern World, in Current and Future Issues in Hemophilia Care (eds E.-C. Rodríguez-Merchán and L. A. Valentino), Wiley-Blackwell, Oxford, UK. Chapter 29. Accessed on November 28, 2012.

Contact:

Baxter International Inc.
Media Contacts:
Brian Kyhos, (224) 948-4210
Deborah Spak, (224) 948-2349
or
Investor Contacts:
Mary Kay Ladone, (224) 948-3371
Clare Trachtman, (224) 948-3085

For original article, click here.

Inspiration Biopharmaceuticals Highlights Positive Interim Data on its Two Lead Investigational Product Candidates

 Recombinant Porcine FVIII (OBI-1) and Recombinant Factor IX (IB1001)

Interim results suggest that OBI-1 was well tolerated and effective in the treatment of serious bleeding episodes in study patients with Acquired Hemophilia A.

The PK results with IB1001 represent the first data on the use of IB1001 in children less than 12 years of age with hemophilia B.

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Inspiration Biopharmaceuticals, Inc. (Inspiration) announced interim clinical results for the company’s two lead product candidates, an intravenous recombinant porcine FVIII (OBI-1) in the treatment of serious bleeds in acquired hemophilia A (AHA) and an intravenous recombinant FIX (IB1001) for the treatment and prevention of bleeding in children less than 12 years of age with hemophilia B. The data were presented in posters at the 54th American Society of Hematology Annual Meeting in Atlanta, Georgia.

Inspiration’s OBI-1 investigational drug met the primary endpoint of providing effective or partially effective treatment of serious bleeding in 16 subjects with acquired hemophilia A based on the investigator’s assessment of response to treatment.

The poster titled, “Recombinant B-domain deleted porcine FVIII (OBI-1) safety and efficacy in acquired hemophilia A: interim results”, described interim results from the prospective Phase 2/3 trial of the administration of OBI-1 in the treatment of serious bleeds. Sixteen AHA subjects and 1 compassionate use subject with AHA all had successful control (either effective or partially effective) of serious hemorrhage at 24 hours post the initial dose of OBI-1 and subsequent resolution of their bleed. Therapeutic FVIII levels were achieved with peak levels above 100% and maintained with intermittent OBI-1 administration determined by the peak FVIII and fall-off blood levels. In three subjects who developed anti-OBI-1 titers there was no correlation to the anti-hFVIII titers or clinical response as evaluated by FVIII levels and the resolution of bleeding. The mean immediate (20 minute) post-infusion factor VIII level was 230% and, given an initial dose of 200 U/kg, indicated an incremental recovery of about 1.15 IU/dL per IU/kg. There were 11 serious adverse events through September 2012 and 5 deaths were reported. None of the deaths were attributed by the investigator to OBI-1 treatment. One serious adverse event (pneumonia and atrial fibrillation) was deemed by the investigator to be probably not/remotely related to OBI-1. Antibodies to OBI-1 developed in 3 subjects, but all responded favorably to treatment. No specific treatment was given for the anti-OBI-1 antibodies. These interim results suggest that in this study OBI-1 was well-tolerated and effective in the treatment of serious bleeding episodes in AHA. Authors of the study included Jean St.-Louis MD, Rebecca Kruse-Jarres MD, Anne Greist MD, Amy Shapiro MD, Hedy Smith MD, Pratima Chowdary MD, Anja Drebes MD, Jay Lozier MD, and Edward Gomperts MD.

Inspiration intends to file a biologics license application (BLA) for OBI-1 in the first half of 2013. OBI-1 has received orphan drug designation in the US and EU, and was recently granted fast track designation by the United States Food and Drug Administration (FDA).

In addition, Inspiration’s IB1001 demonstrated an average in vivo recovery of 0.69±0.21 (mean±SD) in the 7 subjects < 12 years of age with hemophilia B versus that seen in adults (age 12 years and older) of 0.98±0.22 (mean±SD) previously reported by Martinowitz U, et al. (ref. Haemophilia, 18, 2012).

The poster titled, “IB1001, an investigational recombinant factor IX, pharmacokinetics in pediatric patients with hemophilia B”, reports preliminary pharmacokinetic (PK) data from 7 subjects (4 between 6 and 12 years of age and 3 less than 6 years of age) who underwent a 72-hour PK assessment followed by treatment with IB1001. Compared to the findings previously reported with IB1001 by Martinowitz U, et al. in adult subjects 12 years of age and older, these pediatric subjects demonstrated a more rapid metabolism of factor IX with a shorter terminal half-life (mean±SD of 19.3±7.8 h vs. 29.6±18.2 h in adults), a smaller AUC _0-∞ (mean±SD of 1059±264 vs. 1668±598 in adults), and a lower in vivo recovery (mean±SD 0.69±0.21 vs. 0.98±0.22 in adults). These results are similar to those reported by Berntorp, et al (Haemophilia, 7, 2001) with nonacog alfa. Authors of the study were Shashikant J. Apte, M.D.; Uptal Chowdari, MD; M Joseph John, MD; Ri Liesner, MD; Vijay Ramanan, M.D.; Amy Shapiro, MD; Bonnie Mills, PhD; Edward Gomperts, MD; and Martin Lee, PhD.

Inspiration filed a marketing authorization application in Europe for IB1001 late 2011 and the regulatory review is ongoing. Subsequently, Inspiration filed a biologics license application in the United States for IB1001 in the first half of 2012 and the regulatory review is ongoing. The FDA placed a clinical hold on IB1001 in July 2012 when a number of study subjects developed antibodies to the host cell protein, Chinese Hamster Ovary cell proteins (CHOP), of IB1001. No clinical sequelae were associated with these antibodies and they did not impact the clinical effectiveness of the investigational product. Inspiration has made significant progress in addressing the situation, and intends to submit data to the FDA in the first part of 2013 to respond to the clinical hold.

About Inspiration’s Recombinant Porcine FVIII (OBI-1)

In the fourth quarter of 2010, OBI-1 entered late-stage clinical testing in individuals with acquired hemophilia, a rare, potentially life-threatening bleeding disorder, which, unlike congenital hemophilia, typically affects older adults and occurs equally in both males and females. Further, Inspiration has initiated a second pivotal clinical trial in individuals with congenital hemophilia A who have developed inhibitors against human FVIII.

About Inspiration’s Recombinant Factor IX (IB1001)

Inspiration’s lead product candidate is an intravenous recombinant FIX product (IB1001) being developed for the treatment and prevention of bleeding in individuals with hemophilia B. IB1001 has completed pivotal Phase 3 clinical testing in the U.S., Europe, Israel and India. Pharmacokinetic results in the clinical study have demonstrated non-inferiority to the one approved recombinant FIX product currently marketed for the treatment of hemophilia B.

About Inspiration Biopharmaceuticals

Inspiration Biopharmaceuticals is exclusively dedicated to developing treatments for hemophilia, with a primary mission to broaden access to care, by providing safe and effective recombinant therapies and advancing innovation for people living with hemophilia. Inspiration has a broad portfolio of recombinant hemophilia products, which includes two products in late-stage clinical development and two preclinical programs. For further information on Inspiration, please visit http://www.inspirationbio.com.

Contacts

Inspiration Biopharmaceuticals
Michel Dahan, +1 617-588-1807
Vice President, Commercial Development
& Strategic Planning
mdahan@inspirationbio.com
or
Karen Tubridy, PharmD, +1 617-588-1804
Sr. Vice President
Clinical & Medical Affairs
ktubridy@inspirationbio.com

 

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Baxter Presents Data on BAX 326, an Investigational Recombinant Factor IX for Hemophilia B

Baxter Presents Phase I/III Data Evaluating Safety and Efficacy of BAX 326, an Investigational Recombinant Factor IX for Hemophilia B, Showing More Than 40 Percent of Patients Experienced No Bleeds

Date(s): 10-Dec-2012 9:01 AM

BAX 326 Granted Orphan-drug Designation by U.S. Food and Drug Administration

DEERFIELD, Ill.–(BUSINESS WIRE)–Dec. 10, 2012– Baxter International Inc.(NYSE:BAX) today announced pivotal Phase I/III study results evaluating the safety and efficacy of BAX 326, an investigational recombinant factor IX (rFIX) protein, for the treatment and prophylaxis of bleeding episodes for patients with hemophilia B over 12 years of age. The data were presented at the 54^th Annual Meeting and Exposition of the American Society of Hematology (ASH) inAtlanta. BAX 326 was recently granted orphan-drug designation by the U.S. Food and Drug Administration (FDA), a special status given to a product that when approved, will address an unmet need for people with a rare disease or condition. Data supporting the prophylaxis indication, which is the basis for the orphan drug designation, was included in the biologics license application (BLA).

”With only one recombinant protein currently available to treat hemophilia B, it was important to focus research efforts on finding another option for patients with this debilitating disease,” said lead investigator Jerzy Windyga, MD, PhD,Institute of Hematology and Blood Transfusion, Warsaw, Poland. ”In this study, more than 40 percent of patients using BAX 326 as a prophylactic treatment experienced no bleeds, an important finding given the potentially harmful impact of bleeding episodes for patients.”

The Phase I/III prospective, controlled, multicenter study investigated the pharmacokinetics, efficacy and safety of BAX 326 in 73 previously-treated patients with severe or moderately severe hemophilia B. Results from the study showed that twice-weekly prophylactic treatment with BAX 326 achieved a median annualized bleed rate of 1.99 with 43 percent of patients experiencing no bleeds. The impact of prophylaxis with BAX 326 also translated into statistically significant improvements in physical health-related quality of life (HRQoL), as measured by improvements in the Bodily Pain and Role-Physical domains in the Short Form Health Survey (SF-36v2) tool. Statistically significant changes were not seen in the other physical health-related domains or the mental health domains.

No inhibitors were detected and no cases of anaphylaxis were reported in any patients. Three treatment-related adverse events were reported in 2 of the 73 (2.7%) patients, all of which were mild and transient: dysgeusia (distortion of the sense of taste) occurred twice in one patient and pain in extremity occurred once in another patient. More than 70 percent of subjects (56 of 73) had 50 or more exposure days to BAX 326 during the study.

”As a company committed to advancing care for people with hemophilia for more than 60 years, Baxter continues to deliver significant innovation in the field of hemophilia. If it is approved, BAX 326 will expand treatment options for patients,” said Prof. Harmut J. Ehrlich, M.D., vice president of global research and development in Baxter’s BioScience business.

To continue on its path of innovation, earlier this year Baxter announced a partnership with Chatham Therapeutics, LLC to develop a gene therapy-based treatment for hemophilia B. Gene therapy represents another important area of research that may become the future of hemophilia B treatment.

About Hemophilia B

Hemophilia B is the second most common type of hemophilia (also known as Christmas disease) and is the result of insufficient amounts of clotting factor IX, a naturally occurring protein in blood that controls bleeding.¹ Approximately 25,000 people worldwide, including more than 4,000 in the U.S., have been diagnosed with hemophilia B. ² Hemophilia B is often a debilitating, chronic disease with complications that include bleeding episodes, hemophilic arthropathy (bleeding into a joint) and hospitalization.³

About Baxter in Hemophilia

Baxter has more than 60 years experience in hemophilia and has introduced a number of therapeutic firsts for hemophilia patients. Baxter has the broadest portfolio of hemophilia treatments in the industry and is able to meet individual therapy choices, providing a range of options at each treatment stage. The company’s work is focused on optimizing hemophilia care and improving the lives of people living with hemophilia A and B worldwide.

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 BAX 326, an investigational recombinant factor IX (rFIX) protein, as well as Baxter’s partnership with Chatham Therapeutics, LLC to develop a gene therapy-based treatment for hemophilia B. 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: satisfaction of regulatory and other requirements; actions of regulatory bodies and other governmental authorities; additional clinical results; changes in laws and regulations; product quality or patient safety issues; and other risks identified in Baxter’s most recent filing on Form 10-K and other SEC filings, all of which are available on Baxter’s website. Baxter does not undertake to update its forward-looking statements.

¹ Frequently Asked Questions About Hemophilia. World Federation of Hemophilia. Accessed on November 28, 2012. Available at:http://www.wfh.org/en/page.aspx?pid=637

² World Federation of Hemophilia Report on the Annual Global Survey 2010.World Federation of Hemophilia. Accessed on November 28, 2012. Available at: http://www1.wfh.org/publications/files/pdf-1427.pdf

³ Lee, C. A. (2011) Hemophilia Care in the Modern World, in Current and Future Issues in Hemophilia Care (eds E.-C. Rodríguez-Merchán and L. A. Valentino), Wiley-Blackwell, Oxford, UK. Chapter 29. Accessed on November 28, 2012.

Source: Baxter International Inc.

Baxter International Inc.
Media Contacts:
Brian Kyhos, (224) 948-4210
Deborah Spak, (224) 948-2349
or
Investor Contacts:
Mary Kay Ladone, (224) 948-3371
Clare Trachtman, (224) 948-3085

For press release, click here.