Alnylam Pharmaceuticals Inc. ( ALNY ) provided an update on its pipeline and its goals for the coming years. The company has progressed well with its 'Alnylam 5x15' program so far, and expects six to seven genetic medicine programs in the clinic by 2015 instead of five genetic medicine programs.

One of the important candidates under Alnylam's 'Alnylam 5x15' program is patisiran (ALN-TTR02), which is being developed for the treatment of transthyretin-mediated amyloidosis (ATTR).

The candidate is currently in the phase III APOLLO study in ATTR patients suffering from familial amyloidotic polyneuropathy (FAP). Patisiran is also in a phase II open-label extension study for the treatment of patients suffering from FAP. Alnylam intends to report data from the open-label extension study once a year with initial data expected later this year.

ALN-TTRsc, another important candidate under Alnylam's 'Alnylam 5x15' program is currently in a phase II study in ATTR patients suffering from familial amyloidotic cardiomyopathy (FAC) or senile systemic amyloidosis (SSA). Results from the phase II study are expected late in the year.

Patients successfully completing the phase II study will be eligible for an open-label extension study which is expected to be initiated in mid-2014. Moreover, Alnylam has plans to initiate a phase III study on ALN-TTRsc in patients suffering from TTR cardiac amyloidosis by year end.

Apart from these candidates, Alnylam also has plans to initiate a phase I study on ALN-AT3 (hemophilia and other rare bleeding disorders) soon with initial results expected by year end. Additionally, the company will file three Investigational New Drug (IND) applications by 2015 for ALN-CC5 (complement-mediated diseases), ALN-AS1 (hepatic porphyrias) and ALN-PCSsc for (hypercholesterolemia). We expect investor focus remain on the Alnylam's pipeline going forward.

Alnylam also said that it will acquire Merck & Co. Inc. 's ( MRK ) wholly owned subsidiary Sirna Therapeutics, Inc. for $175 million in cash and equity. Merck is also expected to receive up to $105 million as developmental and sales milestone payments per product along with single-digit royalties related to certain pre-clinical candidates discovered by Merck. Alnylam will also pay $10 million as milestone payments and single-digit royalties for products covered by Sirna's patent estate.

Moreover, Alnylam has expanded its strategic agreement with Sanofi ( SNY ) for the development and commercialization of candidates for the treatment of rare genetic diseases. As per the new agreement, Alnylam will retain most of the product rights in North America and Western Europe whereas Sanofi will become a major Alnylam shareholder with a stake of approximately 12% for an investment of $700 million.

Alnylam's collaborations with big companies like Merck and Sanofi are encouraging. The deals will not help Alnylam to generate revenues from royalties but it will also take its RNAi technology outside its core focus area.

Alnylam presently carries a Zacks Rank #4 (Sell). Some better-ranked stocks include Actelion Ltd. ( ALIOF ) with a Zacks Rank #1 (Strong Buy).

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Pipeline Update at Alnylam - Analyst Blog

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