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Last $117.84 USD
Change Today +0.27 / 0.23%
Volume 460.6K
ALNY On Other Exchanges
As of 8:10 PM 07/2/15 All times are local (Market data is delayed by at least 15 minutes).

alnylam pharmaceuticals inc (ALNY) Key Developments

Alnylam Pharmaceuticals, Inc. - Special Call

To discuss on new interim Phase 1 results with ALN-AT3i

Alnylam Reports New Positive Clinical Data for ALN-AT3, a Subcutaneously Administered, Investigational RNAi Therapeutic Targeting Antithrombin (AT) for the Treatment of Hemophilia and Rare Bleeding Disorders

Alnylam Pharmaceuticals, Inc. announced that new positive data from its ongoing Phase 1 clinical trial with ALN-AT3 – an investigational RNAi therapeutic for the treatment of hemophilia and rare bleeding disorders (RBD) – were reported in an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) 2015 Congress being held June 20 – 25, 2015 in Toronto, Ontario, Canada. Additional study results from 12 hemophilia A and B subjects demonstrated that subcutaneous administration of ALN-AT3 achieved potent and dose-dependent knockdown of AT of up to 86%. AT knockdown was highly durable, with effects lasting over two months after the last dose, supporting further evaluation of a once-monthly subcutaneous dose regimen. In addition, AT knockdown was associated with statistically significant increases in thrombin generation with a mean increase of up to 350% and marked improvements in whole blood clotting; these results demonstrate a re-balancing of hemostasis in severe hemophilia subjects. In an exploratory post-hoc analysis, a reduced frequency of bleeding was observed at higher AT knockdown levels including a maximum bleed-free interval of 114 days. Importantly, ALN-AT3 was found to be generally well tolerated, including no clinically significant increases in D-dimer, a biomarker of pathologic clot formation. Based on these data, the company expects to accelerate the advancement of ALN-AT3 with pivotal studies planned to begin in mid-2016. The ongoing Phase 1 trial of ALN-AT3 is being conducted in Bulgaria, Russia, Switzerland, and the U.K. as a single- and multi-dose, dose-escalation study comprised of three parts. Part A – which is complete – was a randomized, single-blind, placebo-controlled, single-dose, dose-escalation study (n=4 per cohort; 3:1 randomization of ALN-AT3: placebo) in healthy volunteer subjects. This part of the study was completed after the first dose cohort received a single subcutaneous dose of ALN-AT3 at 30 micrograms per kilogram (mcg/kg). Part B of the study – which is now complete – was an open-label, multi-dose, dose-escalation study that enrolled 12 subjects with severe hemophilia A or B. Subjects in Part B received 3 weekly subcutaneous doses of ALN-AT3 at doses of 15, 45, or 75 mcg/kg with a volume per injection ranging from 0.2 to 0.7 mL. Part C of the study – which is ongoing – is an open-label, multi-dose, dose escalation study of up to 12 subjects with moderate or severe hemophilia A or B in which subjects are receiving 3 monthly subcutaneous doses of ALN-AT3. The initial dose cohort of Part C has received ALN-AT3 at a dose of 225 mcg/kg (volume less than 1mL), which is equivalent to the cumulative dose level from 3 weekly doses of 75 mcg/kg that was found to be generally well tolerated in Part B. The primary objective of Parts B and C of the study is to evaluate the safety and tolerability of multiple doses of subcutaneously administered ALN-AT3 in hemophilia subjects. Secondary objectives include assessment of clinical activity as determined by knockdown of circulating AT levels and increase in thrombin generation at pharmacologic doses of ALN-AT3. For subjects enrolled at select sites in the U.K. and Switzerland, the effects of ALN-AT3 are also being monitored by ROTEM® thromboelastometry, which measures clotting time and clot strength in whole blood following a physiologic coagulation stimulus. In addition, exploratory analyses of bleeding are being performed. In the U.K., enrollment has been aided by the Southern Academic Coagulation Consortium (SACC). New results were presented from 12 hemophilia subjects in Part B of the ongoing Phase 1 study in an oral presentation at ISTH and include all available data as of the data cut-off date of June 2, 2015. Subcutaneous doses of ALN-AT3 resulted in potent, dose-dependent and statistically significant knockdown of plasma AT of up to 86%. At the top dose of 75 mcg/kg (n=3), the mean maximum AT knockdown was 59 ± 7% (p less than 0.05), with nadir levels achieved between days 28 and 42. AT knockdown was found to be highly durable, with effects lasting over two months after the last dose. For example, at the 45 mcg/kg dose, mean AT knockdown was 36 ± 11% at day 70. AT knockdown with ALN-AT3 was associated with statistically significant increases in thrombin generation and improvements in whole blood clot formation, providing continued evidence for a re-balancing of hemostasis and potential correction of the hemophilia phenotype in severe hemophilia subjects. The association between levels of AT knockdown and thrombin generation was assessed in a post hoc exploratory analysis in which AT knockdown was categorized into tertiles. In the high tertile (greater than 66% AT knockdown), ALN-AT3 administration resulted in mean increases in thrombin generation of 350 ± 239% (p less than 0.05). In this same tertile, the observed level of thrombin generation (120 ± 81 nM peak thrombin) was comparable to levels observed in healthy volunteers (120 ± 33 nM peak thrombin) from Part A of the Phase 1 study, demonstrating an apparent normalization of thrombin generation. Thrombin generation is known to be a biomarker for bleeding frequency and severity in people with hemophilia (Dargaud, et al., Thromb Haemost; 93, 475-480 (2005)). Additional evaluation of the effects of ALN-AT3 employed the use of ROTEM® thromboelastometry in 3 severe hemophilia subjects in which ALN-AT3 administration resulted in statistically significant, AT knockdown-dependent improvements in whole blood clot formation, including a shortening of clot formation time (CFT). At day 35, the CFT for all 3 subjects was significantly shorter than on day 1, with an over three-fold shortening of CFT from 1,166 ± 614 sec to 323 ± 46 sec (p less than 0.05).

Alnylam Pharmaceuticals, Inc. - Special Call

To discuss new Phase 1/2 clinical results with ALN-CC5

Alnylam Pharmaceuticals, Inc. Reports Positive Initial Clinical Results for ALN-CC5

Alnylam Pharmaceuticals, Inc. announced initial positive results from its ongoing Phase 1/2 clinical trial with ALN-CC5, an investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases. These new clinical data are being presented at the 20th Congress of the European Hematology Association (EHA) held June 11 14 in Vienna, Austria. Initial study results from 12 healthy volunteer subjects showed that single subcutaneous dose administration of ALN-CC5 resulted in potent, dose-dependent, durable, and statistically significant knockdown of serum C5 of up to 96%. In addition, single dose administration of ALN-CC5 achieved inhibition of serum complement activity of up to 92%, including an up to 61% inhibition of serum hemolytic activity. Further, ALN-CC5 has been found to be generally well tolerated to date. The company is continuing to dose healthy volunteer subjects in both the single ascending dose (SAD) and multiple ascending dose (MAD) stages of the study, and expects to present additional data from the trial in late 2015. Consistent with previous guidance, the company also plans to begin enrolling patients with paroxysmal nocturnal hemoglobinuria (PNH) into the trial by the end of 2015. The Phase 1/2 trial of ALN-CC5 is being conducted in the U.K., in three parts. Parts A and B are randomized, double-blind, placebo-controlled, single-dose (Part A) and multi-dose (Part B), dose-escalation studies, designed to enroll up to a total of 60 healthy adult volunteers. To mitigate against the risk of Neisseria meningitides, subjects received meningococcal vaccination and oral ciprofloxacin. Subjects in Part A are receiving a single subcutaneous administration of ALN-CC5 at fixed doses of 50, 200, 400, or 600 mg. Subjects in Part B are receiving multiple ascending doses of ALN-CC5, at a fixed dose level of 100 mg in the first cohort, administered once weekly for up to five weeks; bi-weekly and monthly dosing schedules may also be evaluated. The primary objective of these first two parts of the study is to evaluate safety and tolerability of single and multiple subcutaneous doses of ALN-CC5. Additional objectives include evaluation of clinical activity for ALN-CC5 as measured by knockdown of serum C5 and inhibition of serum complement activity, including measurements of complement alternative pathway (CAP) and complement classical pathway (CCP) activity by ELISA and serum hemolytic activity toward sheep erythrocytes. Part C will be an open-label, multi-dose study in up to eight patients with PNH, and will assess safety, tolerability, and clinical activity of ALN-CC5, administered as multiple subcutaneous doses for up to 13 weeks. This part of the study will include an exploratory evaluation of the effects of ALN-CC5 on levels of lactate dehydrogenase (LDH), a measure of endogenous red blood cell hemolysis.

Alnylam Pharmaceuticals, Inc. Appoints David-Alexandre Gros as Senior Vice President, Chief Business Officer

Alnylam Pharmaceuticals, Inc. announced the appointment of David-Alexandre DA Gros, M.D. to the position of Senior Vice President, Chief Business Officer. Dr. Gros joins Alnylam with extensive experience in the pharmaceutical industry and in healthcare investment banking and consulting. At Alnylam, he will be responsible for the company's corporate and business development, finance, corporate communications, and investor relations functions. In addition, Dr. Gros will be joining the Alnylam Management Board. Most recently, Dr. Gros was Executive Vice President and Chief Strategy Officer at Sanofi, where he was a member of the Executive Committee and the Global Leadership Team. His responsibilities included leading corporate strategy, business development and licensing, mergers and acquisitions, alliance management, and structured investments including the corporate venture fund. During his tenure, he oversaw the execution of over 100 successful transactions and the launch of Sanofis corporate venture activities, and he was closely involved in the company's key alliances with Alnylam and Regeneron. Prior to Sanofi, he held management positions with a focus on the pharmaceutical industry in investment banking at Centerview Partners and Merrill Lynch & Co, and in consulting at McKinsey & Co.


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