Alnylam welcomes you to Capella, the destination for updates on our progress in translating the science of RNAi toward innovative medicines.
July 28, 2015
We are hosting a series of online “RNAi Roundtables” during July, August and September, at which Alnylam scientists and key clinical collaborators will review recent progress in many of the company’s development-stage pipeline programs and discuss the related disease areas.
July 28, 2015
On July 28, 2015, we hosted an online RNAi Roundtable to review the progress with ALN-HBV in development for the treatment of Hepatitis B Virus (HBV) Infection.
July 23, 2015
On July 23, 2015, we hosted an online RNAi Roundtable to review the progress with ALN-CC5 in development for the treatment of Complement-Mediated Diseases.
July 23, 2015
On July 22, 2015, we hosted an online RNAi Roundtable to review the progress with ALN-AT3 in development for the treatment of Hemophilia and Rare Bleeding Disorders.
June 23, 2015
Positive Interim Clinical Results from Ongoing Phase 1 Trial of ALN-AT3 for the Treatment of Hemophilia and Rare Bleeding Disorders
We presented positive interim clinical data from our ongoing Phase 1 study for ALN-AT3, a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders. Data were presented at the International Society on Thrombosis and Haemostasis (ISTH) 2015 Congress held June 20 – 25, 2015. New clinical results from 12 subjects with severe hemophilia show 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. Furthermore, in an exploratory post-hoc analysis, a reduced frequency of bleeding was observed at higher AT knockdown levels with a maximum bleed-free interval of 114 days. Very importantly, ALN-AT3 continues to be generally well tolerated.
June 14, 2015
Positive Initial Phase 1/2 Clinical Results for ALN-CC5 for the Treatment of Complement-Mediated Diseases
We reported positive initial clinical results from our Phase 1/2 trial of ALN-CC5, an investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases. The new clinical data are being presented at the 20th Congress of the European Hematology Association (EHA) held June 11 – 14, 2015. Initial study results from 12 healthy volunteer subjects showed that subcutaneous administration of a single dose 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.
May 17, 2015
We presented pre-clinical data on ALN-AAT, an investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of AAT deficiency-associated liver disease. New data, presented at the Digestive Disease Week (DDW) meeting, held May 16 – 19, 2015, showed a robust knockdown of serum AAT of up to 93% in non-human primates (NHPs) with monthly subcutaneous dosing. This level of knockdown was highly durable, lasting for greater than 30 days following the final dose. Further, ALN-AAT was found to have a wide therapeutic index based on results from GLP toxicology studies. In addition, study results were reported from a transgenic mouse model of alpha-1 liver disease, where mice overexpress the human Z-AAT protein.
April 21, 2015
We presented initial 12-month clinical data from our ongoing Phase 2 open-label extension (OLE) study with patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin (TTR)-mediated amyloidosis in patients with familial amyloidotic polyneuropathy (FAP). Study results, presented at the 67th Annual Meeting of the American Academy of Neurology (AAN) being held April 18 – 25, showed a mean 2.5 point decrease in modified Neuropathy Impairment Score (mNIS+7) at 12 months in patients who had reached the 12-month endpoint (N=20) at the time of data cutoff. This decrease in neuropathy progression compares favorably to the 13 to 18 point increase in mNIS+7 at 12 months that can be estimated from the literature in untreated FAP patients with similar baseline characteristics.
April 13, 2015
We have published pre-clinical results with ALN-AT3, an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia, in Nature Medicine. The paper documents a broad set of pre-clinical data supporting the clinical advancement of ALN-AT3. Among the many findings reported, subcutaneous administration of ALN-AT3 led to potent, dose-dependent, and durable knockdown of AT in wild-type mice, hemophilia A mice, and non-human primates (NHPs). In addition, ALN-AT3 treatment improved hemostasis in hemophilia mice and normalized thrombin generation in a non-human primate “inhibitor” model of hemophilia A (HA). Furthermore, long-term ALN-AT3 administration – even at highly exaggerated doses – was shown to be well tolerated in hemophilia mice, supporting a wide therapeutic index in the disease setting.
March 15, 2015
Results from Retrospective Natural History Study of Patients with Familial Amyloidotic Cardiomyopathy (FAC)
We presented results from a retrospective natural history study evaluating disease progression in transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) patients with familial amyloidotic cardiomyopathy (FAC). These results were presented at a meeting with members of the Association of Black Cardiologists (ABC) at the American College of Cardiology (ACC) Annual Scientific Session, held March 14 – 16, 2015. Amongst other findings, study results showed a mean decline of 140 meters in 6-minute walk distance (6-MWD) over an 18-month period in FAC patients with mild-to-moderate heart failure. Also at the ACC meeting, we presented complete results from our Phase 2 clinical trial with revusiran, an investigational RNAi therapeutic for the treatment of FAC. Consistent with preliminary results presented last year, revusiran achieved an up to 98.2% knockdown of serum TTR – the disease-causing protein – and was found to be generally well tolerated.