Catalyst Biosciences Given Permission to Change Trial Length for CB 2679d
Catalyst Biosciences has amended its protocol of a Phase 1/2 clinical trial investigating the factor IX variant CB2679d in previously treated hemophilia B patients. The amendment moved one of the trial groups directly into dosing. The trial is being conducted in three sites in South Korea. See full article at hemophilianewstoday.com.
NY Times Shares Patient Voices: Hemophilia
As part of a series of audio and visual presentations designed to bring awareness and personify medical disorders, the New York Times has a section they have dedicated to several members of the bleeding disorder community. Each has pictures and audio recorded by the individual talking about the impact bleeding disorders have played in their lives. See full article at nytimes.com.
Alnylam and Sanofi Partner to Treat Rare Disorders
Alnylam, an RNAi therapeutics company, and Sanofi announced a strategic restructuring of their alliance to streamline development and commercialization of some products for rare disorders. Specifically Sanofi will obtain rights to develop fitusiran to treat hemophilia A and B. See full article at hemophiliafed.org.
Chris Bombardier Completes the Seven Summits by Climbing Mt. Vinson
Chris Bombardier, a member of our local hemophilia community, has completed the Seven Summits by climbing Mt. Vinson in Antarctica on January 6th. Chris has now climbed the highest peak on all seven continents, something that only 400 people have ever done, becoming the first person with severe hemophilia to accomplish the feat. See his post at his Facebook page here.
BioMarin Shares New Data on Gene Therapy for Hemophilia A
BioMarin Pharmaceuticals announced an update to a Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270) at the 59th American Society of Hematology (ASH) meeting in Atlanta. The results showed sustained normal or near-normal factor levels in hemophilia A patients over a period of 19 months. A Phase 3 trial will begin enrolling in December 2017. See full article at streetinsider.com.
Gene Therapy Shows Promise for Hemophilia B
A new study conducted at the Children's Hospital of Philadelphia reported positive results in a Phase 1/2 clinical trail for hemophilia B patients. The treatment allowed ten adult patients to safely produce sustained levels of clotting factor for more than a year. The gene therapy is scheduled to begin a Phase 3 trial to continue study of the treatment. See full article at news-medical.net.
Shire to Develop New Way to Administer Hemophilia Medications
Pharmaceutical company Shire announced that it had partnered with California-based Rani Therapeutics to develop a new way to administer medication to hemophilia A patients. The pill allows oral delivery of factor by transferring the medicine once it reaches the intestines. The method is expected to be pain free. See full article at reuters.com.
2017 NHF/Novo Nordisk Career Development Award Goes to Dr. Chris Ng
Dr. Christopher Ng, of our Colorado HTC, is the recipient of the 2017 NHF/Novo Nordisk Career Development Award. "The award funds basic, pre-clinical or clinical research approaches to yielding scientific information or answers contributing to better treatments for inheritable bleeding disorders." Dr. Ng's project is centered around on von Willebrand factor regulation. See full article at hemophilia.org.
Startup Could Bring Back Vioxx for Hemophilia Patients
Small pharmaceutical company Tremeau is working to bring Vioxx, a popular pain medication that Merck voluntarily pulled in 2004 amid evidence that it double chances of having a heart attack or stroke. The company believes that it could be approved to treat the severe joint pain caused by hemophilia, but would still come with risks. See full article at chicoer.com.
FDA Approves Hemlibra (Emicizumab) for Hemophilia A with Inhibitors
The FDA has approved Hemlibra (Emicizumab-kxwh) to treat patient with hemophilia A with inhibitors. The approval marks the first new medicine in nearly 20 years to treat such patients. Studies showed that Hemlibra substantially reduced bleeds in both adults and children and was administered once weekly. See full article at gene.com.
Alnylam Announces Successful Outcome Following FDA Meeting on Fitusiran
Alnylam announced a successful meeting with the FDA agreeing with the safety measures and a risk mitigation strategy to enable resumption of clinical studies with fitusiran. The study is targeting patients with hemophilia A and B and the measures follow the death of a patient in the study where the FDA put a hold on the research. See full article at seekingalpha.com.
Hemophiliac Readies for Climb of Mt. Vinson, Final in 7 Summits
Chris Bombardier, the first person with severe hemophilia to climb Mount Everest, is preparing to climb Mount Vinson in Antarctica this December. The climb will complete his goal of becoming the first person with hemophilia to climb the 7 Summits-the highest peak on each continent. See full article at hemophilianewstoday.com.
Study Shows Positive Outcomes for Hemophilia A Patients
Interim study results recently published in the journal Haemophilia, showed that prophylactic use of Eloctate led to joint health improvements in a group of hemophilia A patients. The results showed that regardless of joint health prior to treatment, those receiving prophylactic infusions of the therapy for nearly three years saw regular improvements. See full article at hemophilia.org.
FDA Accepts Beyer's Biologics License Application for BAY 94-9027
The FDA has accepted Bayer's Biologics License Application for BAY94-9027, an extended half life recombinant Factor VIII compound to treat hemophilia A in adults and adolescents. The formula is engineered to potentially prolong FVIII activity and may result in reduced frequency of infusions. See full article at prnewswire.com.
FDA Grants BioMarin Investigational New Drug Status to BMN 270
BioMarin's Investigational New Drug application for its gene therapy candidate BMN 270 to treat hemophilia A has been approved by the FDA. The decision was supported by clinical data of a Phase 1/2 trial showing five of six patients reaching low range of normal amounts of factor. See full article at hemophilianewstoday.com.
FDA Grants Orphan Drug Designation to Shire's SHP654 To Treat Hemophilia A
The US FDA has granted Shire with orphan drug designation for its gene therapy candidate SHP654 to treat hemophilia A. The therapy hopes to use a virus stereotype vector that is delivered to the liver that will help the body produce factor, reducing bleeds and the need to infuse factor. See full article at raredr.com.
UniQure Announces New Study for Hemophilia B Gene Therapy Program
UniQure announced their plans to advance AMT-061, which combines an AAV5 vector with the FIX Padua mutant, into a pivotal study in 2018 for patients with severe and moderately severe hemophilia B. Ongoing trials of similar AMT-061 already have shown promise. See full article at inpublic.globenewswire.com.
Hemophilia A Study Shows Potential to Reduce Inhibitors
A study on mice that was published in the journal Blood outlines findings that show potential in targeting ways to reduce inhibitors in Hemophilia A patients. The MZ B-cells found in the spleen were part of the focus in finding ways to prevent inhibitors from developing in patients. See full article at hemophilianewstoday.com.
BloodStream Podcast Presents Factor Revolution: 60 Years of Hemophilia
BloodStream Stories presents a new 3 part audio series detailing the last sixty years in hemophilia treatment. With over forty interviews the project gathers source material, news reports, and other original audio content to share the history of hemophilia treatment. Hear the podcast at bloodstream.libsyn.com.