Paroxysmal Nocturnal Hemoglobinuria News and Research

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USPTO grants Taligen patent for TT30 lead product candidate

Taligen Therapeutics, a biotechnology company developing therapies that regulate the complement system to treat inflammatory and immune diseases, announced today that the U.S. Patent and Trademark Office has issued U.S. Patent No. 7,759,304, entitled "Targeting Complement Factor H For Treatment of Diseases", covering the composition of matter for its lead product candidate, TT30, a novel fusion protein that selectively binds to complement activated cells to locally regulate the complement system.

24 Aug 2010

Taligen Therapeutics announces closing on $10M tranche of Series B financing

Taligen Therapeutics, a biotechnology company developing therapies that regulate the complement system to treat inflammatory and immune diseases, today announced that it has closed on a $10 million tranche of a Series B financing, based on substantial progress with the company's product pipeline including advancing the lead product candidate, TT30, toward clinical development.

23 Aug 2010

FDA approves Alexion's Rhode Island manufacturing facility as commercial supply source for Soliris

Alexion Pharmaceuticals, Inc. today announced that the US Food and Drug Administration (FDA) has approved Alexion's Rhode Island manufacturing facility (ARIMF) in Smithfield, Rhode Island as a second source of commercial supply for Soliris® (eculizumab).

23 Aug 2010

Taligen Therapeutics to highlight three pipeline candidates at XXIII ICW

Taligen Therapeutics, a biotechnology company developing therapies that regulate the complement system to treat inflammatory and immune diseases, today announced that preclinical data on three of its pipeline candidates will be highlighted at the XXIII International Complement Workshop taking place August 1-5, 2010, in New York City.

2 Aug 2010

Alexion second-quarter non-GAAP net income increases 56% to $36.9 million

Alexion Pharmaceuticals, Inc. today announced financial results for the three and six months ended June 30, 2010.

22 Jul 2010

Alexion's Soliris may benefit patients with thrombotic microangiopathy from uncontrolled complement activation

Researchers reported today that Soliris® (eculizumab), a first-in-class terminal complement inhibitor developed by Alexion Pharmaceuticals, Inc., may provide clinical benefits to patients with thrombotic microangiopathy (TMA) resulting from uncontrolled complement activation. TMA, the formation of blood clots in capillaries and small arteries, can lead to life-threatening damage in multiple organs including kidney failure, thrombocytopenia (abnormally low platelet count) and anemia.

15 Jun 2010

New research evaluating substantial disease burden of PNH presented at European Hematology Association

Alexion Pharmaceuticals, Inc. today announced new research evaluating the substantial disease burden of paroxysmal nocturnal hemoglobinuria, an ultra-rare blood disorder, in patients worldwide. The International PNH Registry, involving 580 patients from 99 sites in 14 countries as of May 2010, found that the debilitating symptoms and life-threatening complications of PNH are similar across patient populations around the world.

14 Jun 2010

Alexion Pharmaceuticals reports total revenues of $117.6M from net product sales of Soliris

Alexion Pharmaceuticals, Inc. today announced financial results for the quarter ended March 31, 2010.

22 Apr 2010

Alexion Pharmaceuticals' Soliris NDA for PNH receives approval in Japan

Alexion Pharmaceuticals, Inc. and Alexion Pharma International Sàrl today announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has approved the Company's New Drug Application (NDA) for the use of Soliris® (eculizumab) as a treatment for patients in Japan with paroxysmal nocturnal hemoglobinuria (PNH).

16 Apr 2010

Rare Disease Day 2010: Alexion Pharmaceuticals joins NORD and EURORDIS to raise awareness

Alexion Pharmaceuticals, Inc., has joined the National Organization for Rare Disorders (NORD) and the European Organization for Rare Diseases (EURORDIS) in supporting the goals of the third annual Rare Disease Day, February 28, 2010.

26 Feb 2010

Alexion Pharmaceuticals announces financial results for quarter and year ended December 31, 2009

Alexion Pharmaceuticals, Inc. today announced financial results for the quarter and year ended December 31, 2009. For the three months ended December 31, 2009, Alexion Pharmaceuticals, Inc. ("Alexion" or, the "Company") reported net product sales of Soliris® (eculizumab) of $110.6 million, reflecting strong additions of new patients, compared to $77.4 million for the same period in 2008.

11 Feb 2010

New data shows patients with Type II PNH cells experience platelet consumption and increased thrombosis risk

Alexion Pharmaceuticals, Inc. today announced presentation of data showing that the presence of Type II paroxysmal nocturnal hemoglobinuria (PNH) cells is correlated with thrombocytopenia, potentially indicating that patients with Type II cells experience ongoing platelet consumption and increased thrombosis risk.

7 Dec 2009

Data on Alexion Pharmaceuticals' Soliris to be presented at the ASH meeting

Alexion Pharmaceuticals, Inc. today announced that researchers are scheduled to present data relating to Soliris® (eculizumab) as a treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH) during the 51st Annual Meeting of the American Society of Hematology (ASH), to be held December 5 – 8, 2009 at the Ernst N. Morial Convention Center in New Orleans.

11 Nov 2009

Alexion Pharmaceuticals reports increased revenues of 11% in third quarter 2009

Alexion Pharmaceuticals, Inc. today announced financial results for the quarter ended September 30, 2009.

22 Oct 2009

Real Americans share experiences, common need for patient-centric reform

Teva Pharmaceuticals USA, the world's leading generic pharmaceutical company, today announced the start of its "Patient First" project as part of the Year of Affordable Healthcare campaign. The initiative, available at: www.yearofaffordablehealth.com/patients, features a first-hand perspective of everyday Americans as they struggle with the enormous and sometimes insurmountable expense of paying for their prescription medicines.

21 Aug 2009

Soliris (Eculizumab) granted orphan drug designation in Europe and U.S. for atypical hemolytic uremic syndrome

Alexion Pharmaceuticals, Inc. has announced today that Soliris® (eculizumab), its first-in-class complement inhibitor, has been granted Orphan Medicinal Product Designation by the European Commission for the treatment of patients with atypical Hemolytic Uremic Syndrome (aHUS). AHUS is an ultra-rare, inherited, and life-threatening complement-inhibitor deficiency disease that often progresses to end-stage kidney disease or failure. The U.S. Food and Drug Administration granted orphan drug designation to Soliris for the same indication in May 2009. Soliris is not approved for the treatment of patients with aHUS.

6 Aug 2009

Scientists edit genes in human stem cells

Researchers at the Johns Hopkins School of Medicine have successfully edited the genome of human- induced pluripotent stem cells, making possible the future development of patient-specific stem cell therapies. Reporting this week in Cell Stem Cell, the team altered a gene responsible for causing the rare blood disease paroxysmal nocturnal hemoglobinuria, or PNH, establishing for the first time a useful system to learn more about the disease.

18 Jun 2009

PDL BioPharma and Alexion Pharmaceuticals resolve patent dispute

PDL BioPharma, Inc. and Alexion Pharmaceuticals, Inc. today jointly announced that the companies have entered into a definitive license agreement and settlement agreement that resolve the legal disputes between them relating to Alexion's humanized antibody, Soliris (eculizumab) and PDL's patents known as the Queen et al. patents.

5 Jan 2009

FDA approves Soliris (eculizumab), first treatment for paroxysmal nocturnal hemoglobinuria

The U.S. Food and Drug Administration (FDA) has approved Soliris (eculizumab), the first product for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare type of blood disorder that can lead to disability and premature death.

19 Mar 2007

Researchers have identified a new clinical syndrome

Researchers have identified a new clinical syndrome that produces serious and sometimes fatal symptoms in a variety of seemingly unrelated diseases. The syndrome, described in the April 6 issue of the Journal of the American Medical Association, is caused by hemolysis, the destruction of red blood cells.

5 Apr 2005