Alnylam Pharmaceuticals ($ALNY) heralded positive preclinical data for its RNAi drug that targets a specific protein to treat a range of life-threatening diseases.
The study showed that the RNAi drug ALN-CC5 was able to regulate the complement component 5 (C5), a gene that encodes a protein responsible for host defense and immunity. When the gene is unmitigated, it can result in serious complications such as paroxysmal nocturnal hemoglobinuria, atypical hemolytic-uremic syndrome, myasthenia gravis, neuromyelitis optica and others, according to the company. ALN-CC5, when introduced beneath the skin, achieved about 90% inhibition of the component in animal tests.
RNAi is Alnylam's specialty, and the genetic delivery platform could represent an important breakthrough in personalized medicine once researchers figure out how to deliver it safely and effectively to act on a patient's DNA. Essentially, RNAi allows the use of natural processes to "silence" particular genes--like C5--that lead to complications. The discovery of the technique in 2006 was awarded the Nobel Prize in physiology or medicine.
In 2011, Alnylam launched its "5x15" program, which calls for the company to have 5 different RNAi candidates in clinical development by the end of the year 2015. ALN-CC5 is one such drug.
"We are excited about these pre-clinical data showing potent, dose-dependent and durable knockdown of serum C5 with about 90% inhibition of hemolysis activity using a subcutaneously administered RNAi therapeutic," said Alnylam Executive VP and Chief Medical Officer Akshay Vaishnaw in a statement. "We believe that if these results can extend in the clinical setting, they could represent a very promising therapeutic strategy and new treatment option for patients with complement-mediated diseases."
