X-Chem Signs Drug Discovery Collaboration with Alexion
Dec 15 14
X-Chem has signed drug discovery collaboration with Alexion. The contract has been signed for new therapies to treat patients with severe and ultra-rare disorders. According to the agreement, X-Chem will deploy its drug discovery engine, which is based on an ultra-large, high-diversity library in excess of 100 billion molecules, to identify new drug candidates against targets chosen by Alexion. The contract will see X-Chem receive an upfront payment and receive additional payments upon the achievement of specified research, development and regulatory milestones. X-Chem will also receive royalty payments on the sale of products resulting from the collaboration.
Alexion Pharmaceuticals, Inc. Announces Favorable Data from Clinical Trials of Soliris, or Eculizumab, in Patients with Atypical Hemolytic Uremic Syndrome
Nov 24 14
Alexion Pharmaceuticals, Inc. has announced favorable data from clinical trials of Soliris, or eculizumab, in patients with atypical hemolytic uremic syndrome, or aHUS. These data, from a total of seven presentations, continue to enhance the understanding of aHUS and underscore the effectiveness of sustained Soliris treatment in children and adults with aHUS. Data includes: New results from a 1-year update of the largest prospective trial of Soliris in adult patients with aHUS, in which TMA inhibition and improved renal outcomes were sustained and increased numbers of patients achieved renal improvement with ongoing treatment with Soliris; A reported 97% reduction in risk of progression to end-stage renal disease (ESRD) in patients with aHUS receiving Soliris compared to patients receiving supportive care only, based on an analysis from the two Soliris registration clinical trials; Observed improvements in hematologic and renal outcomes in patients with aHUS treated with Soliris regardless of dialysis or transplant history, based on three post-hoc sub-analyses from two prospective open-label, single-arm trials of Soliris in adult and pediatric patients; Baseline demographics from the Global aHUS Registry, confirming the devastating nature of aHUS and increasing the understanding of the disease; and Biomarker data in which markers of alternative complement pathway activation and endothelial cell activation were reduced with sustained Soliris treatment but remained elevated without clinical consequences, suggesting that patients with aHUS have ongoing dysregulation of complement and supporting the need for sustained terminal complement blockade with Soliris. In this open-label, single-arm trial, the primary endpoint of complete TMA response-defined as platelet count normalization, LDH normalization and preservation of renal function-was achieved by 30 patients receiving Soliris (73%) at 26 weeks and by 33 patients (80%) at 1 year. Patients also had continued improvement in renal function with sustained Soliris treatment, with 22 patients (54%) achieving an improvement in estimated glomerular filtration rate (eGFR) from baseline of greater than or equal to15 mL/min/1.73 m2 at 26 weeks and 25 patients (61%) achieving this endpoint at 1 year. There were no unexpected safety signals in the one-year study period. The most common drug-related adverse events (AEs) at 1 year were alopecia (7%), asthenia (5%), arthralgia (5%) and pain in extremity (5%). Two patients in the C10-004 study had meningococcal infections, both during the 26-week study period. One patient discontinued from the study and later recovered; the other continued treatment with no interruption and recovered without sequelae. No additional meningococcal infections were reported between 26 weeks and 1 year. I Researchers also presented an analysis that retrospectively evaluated the decline in renal function and progression to ESRD in patients in the pretreatment period of two clinical trials and compared this to the time to progression to ESRD in the same patients after beginning treatment with Soliris. Patients in the pretreatment period received supportive care, defined as plasma exchange/plasma infusion (PE/PI), dialysis and/or kidney transplant. In the analysis, patients receiving Soliris (N=33) had a 97% reduction in the risk of progression to ESRD over 3 years compared with the risk of progression to ESRD in patients receiving supportive care only (N=32). Additionally, over 3 years, no patient who initiated Soliris in chronic kidney disease (CKD) stage 2 or 3 progressed to ESRD, and the risk of progression for patients who initiated Soliris treatment in CKD stage 4 was reduced by 92% compared to the risk of progression in patients receiving supportive care only . These results are consistent with outcomes reported in prior clinical trials, in which patients with aHUS had improvements in renal outcomes and elimination of dialysis during ongoing treatment with Soliris. The most common AEs reported by subgroup were: for patients with baseline dialysis, headache (33.3%), diarrhea (29.2%), and hypotension (20.8%); for patients without baseline dialysis, headache (41.2%), diarrhea (35.3%), and asthenia (29.4%). Chantal Loirat, M.D., of the Hopital Robert Debre, Paris, presented a post-hoc sub-analysis from the C10-004 study that evaluated the safety and efficacy of Soliris in adult patients with aHUS (N=41) with and without a history of renal transplant. In the study, complete TMA response was achieved in 78% (25/32) of non-transplant patients and in 56% (5/9) of transplant patients. In addition, non-transplant patients had a mean eGFR improvement of +31.5 mL/min/1.73 m2 from baseline, while transplant patients had a mean improvement of +19.0 mL/min/1.73 m2.
Alexion Pharmaceuticals, Inc. Announces Board Changes, Effective from January 1, 2015
Nov 10 14
Alexion Pharmaceuticals, Inc. announced the planned retirement of company co-founder Stephen Squinto, Ph.D., effective January 1, 2015. Dr. Squinto currently serves as Executive Vice President, Chief Global Operations Officer. He co-founded Alexion in 1992 with Leonard Bell, M.D., the company's principal founder and current Chairman and Chief Executive Officer. Dr. Squinto has held numerous leadership positions at Alexion prior to his current role, including Executive Vice President, Head of Research and Development. Upon Dr. Squinto's retirement from Alexion on January 1, 2015, he will be the Chair of a newly formed Scientific Advisory Board that will provide strategic input into Alexion's discovery science and pipeline. Dr. Squinto and the Scientific Advisory Board will work directly with Dr. Martin Mackay, Executive Vice President and Global Head of Research and Development. Alexion's Scientific Advisory Board will be comprised of world-class scientists with expertise in key areas of focus at Alexion. With Dr. Squinto's retirement, and as part of Alexion's established succession planning, Alexion announced the promotion of Ms. Julie O'Neill to the position of Executive Vice President, Global Operations, effective January 1, 2015. Ms. O'Neill will report directly to the Chairman and Chief Executive Officer, Dr. Leonard Bell. She joined Alexion in February 2014 as Senior Vice President, Global Manufacturing Operations, serving simultaneously as Alexion's General Manager for Ireland. Ms. O'Neill has been responsible for all aspects of the company's global supply chain operations, including the ongoing expansion into Ireland and improvements to Alexion's manufacturing plant in Smithfield, Rhode Island. Prior to joining Alexion, Ms. O'Neill held leadership positions in pharmaceutical manufacturing and quality for more than 20 years. Most recently, Ms. O'Neill was Vice President of Operations and General Manager for Ireland at Gilead Sciences. Previously, Ms. O'Neill held leadership positions in operations, manufacturing and quality functions at Burnil Pharmacies and Helsinn Birex Pharmaceuticals.