Alexion Pharmaceuticals, Inc. Announces Executive Appointments
Sep 17 14
Alexion Pharmaceuticals, Inc. announced key measures to strengthen and broaden its executive leadership team in line with the company's expanding global mission to develop and commercialize life-transforming therapies for patients with severe and rare disorders. David Hallal is being promoted to the newly created position of Chief Operating Officer and has been appointed to Alexion's Board of Directors. Mr. Hallal had previously served as Executive Vice President and Chief Commercial Officer, and will continue to lead all commercial operations globally with responsibility for country operations in each of Alexion's affiliates in EMEA, Japan, Australasia, and Latin America. As Chief Operating Officer, Mr. Hallal will additionally now lead key enterprise-wide initiatives and he will expand his role with external stakeholders. Clare Carmichael is being promoted to Executive Vice President and Chief Human Resources Officer (CHRO) from her previous position of Senior Vice President and CHRO. Ms. Carmichael will continue to lead and significantly expand the Company's global talent initiatives including talent management, talent acquisition, training and development, and compensation and benefits. John Moriarty, J.D. is being promoted to Executive Vice President and General Counsel from his previous position of Senior Vice President and General Counsel. Mr. Moriarty will continue to lead all of the Company's legal, government affairs and corporate communications activities, further expanding each of these functions to support the Company's ongoing global growth. Additionally, the company is also announced that Edward Miller, J.D. has joined Alexion in the newly created position of Senior Vice President and Chief Compliance Officer. Mr. Miller will lead all corporate compliance functions globally for Alexion. Mr. Miller will lead all compliance functions globally for Alexion. From 2000 to 2013, Mr. Miller held numerous compliance and legal leadership positions at Boehringer Ingelheim Pharmaceuticals/Boehringer Ingelheim GmbH, including Vice President, Associate General Counsel, Global Litigation & Government Investigations, Vice President/Acting Global Compliance Officer, Vice President, Chief Compliance Officer and Head of Litigation, and Chief Compliance Officer, Head of Litigation. All four executives will report directly to Dr. Bell. The promotions of Mr. Hallal, Ms. Carmichael, and Mr. Moriarty, and the addition of Mr. Miller, are designed to facilitate the further growth of Alexion to serve more patients with severe and devastating disorders around the world.
Alexion Pharmaceuticals, Inc. Presents New Data from an Integrated Analysis of Asfotase Alfa in Pediatric Patients with Severe Hypophosphatasia
Sep 14 14
Alexion Pharmaceuticals, Inc. announced that researchers presented new data from an integrated analysis of survival from two open-label, Phase 2 studies of asfotase alfa in pediatric patients (ages <=5 years at enrollment) with hypophosphatasia (HPP) compared with data from a retrospective natural history study of untreated historical control patients matched for age and disease severity. In this analysis, survival in patients with HPP at high risk of death who were treated with asfotase alfa for up to five years was significantly improved (89% vs. 27%, p<0.0001) compared with untreated historical control patients. These late-breaking results were presented at the American Society for Bone and Mineral Research (ASBMR) 2014 Annual Meeting in Houston, where researchers also presented new data from the ongoing open-label extension phases of two Phase 2 clinical studies in which sustained gains in physical function and reductions in disability and pain were observed in pediatric patients receiving asfotase alfa treatment for up to three years. HPP is a genetic, chronic and progressive ultra-rare metabolic disease that can lead to progressive damage to multiple vital organs, destruction and deformity of bones and premature death.(4-8) Asfotase alfa is an investigational enzyme replacement therapy for the treatment of HPP. Survival Data from Studies of Asfotase Alfa in Severely Affected Pediatric Patients Compared With Historical Controls (Abstract 1097): In an oral session, researchers reported that treatment with asfotase alfa significantly improved survival in pediatric patients (ages <=5 years at enrollment) with severe HPP.(1) Over the five-year analysis period, 89% (33/37) of patients treated with asfotase alfa survived compared with 27% (13/48) of untreated historical control patients. Invasive ventilator-free survival was also significantly improved in treated patients (p<0.0001); 83% (21/25) of treated patients required no invasive ventilation and survived, compared with 25% (12/48) of historical control patients. These results were from an integrated analysis of survival from two multicenter, open-label, ongoing Phase 2 studies of patients with HPP who were treated with asfotase alfa, compared with data from a retrospective natural history study of untreated historical controls, matched for age and disease severity. Findings from the retrospective natural history study were previously reported at the Pediatric Academic Societies (PAS) meeting in May 2014.(9) Patients in the asfotase alfa trials were five years of age or younger at enrollment and had been diagnosed with HPP prior to six months of age. Included in the survival analysis were patients who had one or more of the following signs of severe HPP: rachitic chest, history of respiratory distress, or Vitamin B(6) -responsive seizures. Median duration of treatment was two years, with patients treated for up to five years. Asfotase Alfa in Infants and Young Children with Life-Threatening HPP: New Extension Study Results (Abstract FR0435): In an oral poster presented on September 12, Dr. Whyte and colleagues reported that severely affected infants and young children with HPP (age <=3 years at study entry, N=11) treated with asfotase alfa for up to three years experienced sustained improvement in growth and physical function. Findings were from the extension phase of a multinational, open-label Phase 2 study of asfotase alfa treatment in severely affected infants with HPP. Data from this study were previously presented by investigators at the PAS meeting in May 2014. Patients (N=11) had early (3 months, p=0.03) and sustained (3 years, p=0.008) bone healing as measured by the radiographic global impression of change (RGI-C) scale and the rickets severity scale (RSS). While 10 patients required respiratory support at or soon after entry into the study, only one patient continued to require respiratory support (supplemental oxygen) at the last assessment. Researchers reported a three-year survival rate of approximately 90%. New data presented at ASBMR included the following: For patients treated with asfotase alfa, improvement in growth as measured by height/length Z-score was observed. Median height/length Z-score was -3.7 at baseline, indicating marked delay relative to peers; over the course of treatment, median change from baseline in Z-score steadily increased from -0.3 at three months to +2.3 at three years. In asfotase alfa treated patients, improvement in functional development was observed as measured by the Bailey's Scales of Infant and Toddler Development 3rd Edition (BSID-3), with all evaluable patients (N=9) demonstrating increases in age-equivalent scores, indicating acquisition of new gross motor, fine motor and cognitive skills during treatment. New data presented at ASBMR showed the following: In patients treated with asfotase alfa, a significant and clinically meaningful decrease in disability was observed, as measured by the Child Health Assessment Questionnaire (CHAQ), from a median of 1 at baseline to 0.25 at six months to 0 at 24 months and last assessment (p<=0.007). A significant reduction in pain was observed in treated patients, as measured by the Pediatric Outcomes Data Collection Instrument (PODCI), from a median baseline score of 78 to a median score of 100 at last assessment (p=0.0389). The PODCI is a comfort/pain rating scale, with 100 representing best possible outcome or best health. Improvements in strength and agility were observed for treated patients as measured by the shuttle run, one-legged hop test and standing long jump, three components of the BOT-2. Median time to complete a 50 foot shuttle run improved from 22 seconds at baseline to 9 seconds at last assessment (p<0.0001). The median number of one-legged stationary hops completed in 15 seconds improved from 0 at baseline to 21 hops at last assessment (p=0.0001). The median distance jumped via a standing long jump improved from 11 inches at baseline to 46 inches at 36 months (p<0.0001). Improvement in growth, as measured by height Z-score, was observed in treated patients, from a median of -1.26 at baseline to a median of -0.72 at last assessment (p=0.0027). HPP is a genetic, chronic and progressive ultra-rare metabolic disease characterized by defective bone mineralization that can lead to destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death. Asfotase alfa is an investigational, highly innovative, first-in-class targeted enzyme replacement therapy. Asfotase alfa is designed to address the underlying cause of HPP by aiming to restore the genetically defective metabolic process, thereby preventing or reversing the severe and potentially life-threatening complications of life-long dysregulated mineral metabolism.
Alexion Pharmaceuticals, Inc. Presents at 2014 Morgan Stanley Global Healthcare Conference, Sep-08-2014 01:25 PM
Aug 28 14
Alexion Pharmaceuticals, Inc. Presents at 2014 Morgan Stanley Global Healthcare Conference, Sep-08-2014 01:25 PM. Venue: The Grand Hyatt Hotel, New York, New York, United States. Speakers: Leonard Bell, Co-Founder, Chief Executive Officer, Treasurer and Director.