Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data from its RNAi therapeutic program for the treatment of hemoglobinopathies at the 54 th American Society of Hematology Annual Meeting being held December 8-11, 2012 in Atlanta. Alnylam scientists presented data showing that ALN-TMP, an RNAi therapeutic targeting Tmprss6, leads to disease modifying effects, including a correction in globin gene expression, in a model of β-thalassemia. These studies were conducted in collaboration with Boston Children’s Hospital. Alnylam’s program in hemoglobinopathies comprises part of its ‘Alnylam 5x15™’ product strategy, by which the company aims to advance five programs in clinical development, including programs in advanced stages, by the end of 2015.
“Our ‘Alnylam 5x15’ strategy is aimed at bringing innovative medicines to patients, with a focus on RNAi therapeutics toward genetically defined targets for diseases with very high unmet medical need. ALN-TMP exemplifies this strategy where we are advancing RNAi therapeutics for the treatment of hemoglobinopathies,” said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam. “We are very excited by these new data with ALN-TMP where we have demonstrated disease modifying effects in models of β-thalassemia, including amelioration of anemia, iron overload, extra-medullary hematopoiesis, and ineffective erythropoiesis, in addition to correction of globin gene expression.”
“Mouse genetic studies have shown that knockdown of Tmprss6 and up-regulation of the hepcidin pathway can result in disease modifying effects in models of β-thalassemia, a disease of enormous worldwide burden with limited treatment options for certain groups of patients. Our studies with ALN-TMP, an RNAi therapeutic targeting Tmprss6, recapitulate these genetic findings with an innovative pharmacologic strategy. This work is an example of how rare disease research that brings in RNA silencing technologies to ‘target the untargetable’ could rapidly lead to therapies for more common disorders,” said Mark Fleming, M.D., D.Phil., Pathologist-in-Chief at Boston Children’s Hospital and S. Burt Wolbach Professor of Pathology at Harvard Medical School, who collaborated on the work. “Specifically, we have demonstrated that ALN-TMP administration results in disease modifying effects in a model of β-thalassemia. Indeed, our studies show that ALN-TMP administration results in reduced iron overload, decreased extra-medullary hematopoiesis, increased hemoglobin levels, and a reduction in ineffective erythropoiesis. Further, we have found that these effects are accompanied by a decrease in membrane-associated hemoglobin, one of the primary causes of the decreased red blood cell survival in β-thalassemia. Clearly, these results could be of significance for the treatment of patients with β-thalassemia and possibly other hemoglobinopathies.”