WASHINGTON (dpa-AFX) - Alnylam Pharmaceuticals, Inc. (ALNY) Sunday announced the fifth component of its 'Alnylam 5x15' program, which was launched in January 2011 to have five RNAi therapeutic programs in clinical development by the end of 2015.
RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon.
The company increased its guidance to end 2011 with approximately $260 million in cash, and expects to provide financial guidance for 2012 in connection with its 2011 financial results announcement in February.
As part of its 'Alnylam 5x15' strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in clinical development, including programs in advanced stages, on its own or with a partner by the end of 2015.
The 'Alnylam 5x15' programs include ALN-TTR for the treatment of transthyretin-mediated amyloidosis, ALN-APC for the treatment of hemophilia, ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, and ALN-TMP for the treatment of hemoglobinopathies.
John Maraganore, chief executive officer, said,' As we effect our transformation from a platform company to a product company, now is the time to focus our near-term efforts on what we believe to be our highest value opportunities with accelerated clinical development plans. Specifically, we aim to maximally apply Alnylam resources to our programs in transthyretin-mediated amyloidosis and hemophilia, while advancing our other multiple pipeline programs through existing alliances and new partnerships.'
Alnylam expects ALN-TTR02 to be the 'go-to-market' drug candidate for the company's transthyretin-mediated amyloidosis or ATTR program. The company recently filed a Clinical Trial Application for ALN-TTR02 to conduct a Phase I trial in the U.K. to evaluate the safety and tolerability of a single dose of ALN-TTR02, and assessment of clinical activity of ALN-TTR02 as measured by serum TTR levels. In addition, Alnylam plans to start a Phase II multi-dose study of ALN-TTR02 in ATTR patients in the second half of 2012.
Alnylam also plans to advance ALN-TTRsc, which utilizes a GalNAc-conjugate delivery approach and subcutaneous dose administration. Alnylam believes that ALN-TTRsc represents an attractive opportunity for product differentiation in the ATTR clinical setting.
Alnylam plans to advance its ALN-APC program toward the clinic with a goal of initiating a Phase I clinical trial in the first half of 2013. ALN-APC is an RNAi therapeutic targeting protein C, a genetically defined target, for the treatment of hemophilia.
ALN-PCS for the treatment of severe hypercholesterolemia is currently in Phase I study. Alnylam plans to partner its ALN-PCS program prior to initiating a Phase II study.
ALN-HPN for the treatment of refractory anemia is also planned to be developed with a partner, prior to initiating a Phase I study.
The fifth 'Alnylam 5x15' program, the company announced today, is ALN-TMP for the treatment of hemoglobinopathies, including beta-thalassemia and sickle cell anemia.
Alnylam also expects to seek partners to advance additional 'Alnylam 5x15' candidate programs for development. These include programs focused on erythropoiesis, alpha-1-antitrypsin deficiency-associated liver disease, severe hypertriglyceridemia, and acute intermittent porphyria.
Alnylam expects to form additional partnerships in 2012 related to its 'Alnylam 5x15' and partner-based programs. Further, the company aims to form additional partnerships related to non-therapeutic applications of RNAi technology including Alnylam Biotherapeutics and its VaxiRNATM platform.
Cambridge, Massachusetts-headquartered Alnylam continues to maintain a significant equity position in Regulus Therapeutics, Inc., a leading microRNA therapeutics company formed in 2007 by Alnylam and Isis Pharmaceuticals, Inc(ISIS).
ALNY closed Friday's regular trade at $8.67, translating into a market capitalisation of $370.4 million. Over the past year, the stock traded in a range of $5.88 - $12.34.
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