Alnylam Pharmaceuticals, Inc.
) inked a collaboration deal with The Alpha-1 Project (TAP), the
venture philanthropy subsidiary of the Alpha-1 Foundation, for the
continued development of ALN-AAT. ALN-AAT is a subcutaneously
administrated ribo nucleic acid interference (RNAi) therapeutic
being developed for the treatment of alpha-1 antitrypsin (AAT)
deficiency-associated liver disease.
TAP works with patients, academia, pharmaceutical and biotech
companies and other public health organizations for cures and
therapies for chronic obstructive pulmonary disease (COPD) and
liver disease caused by AAT deficiency.
The research and development activities for ALN-AAT will be
partially funded by TAP and the rest by Alnylam. The company
intends to file an investigational new drug (IND) application for
ALN-AAT in mid 2015. Results from pre-clinical studies have shown
that treatment with ALN-AAT resulted in approximately 90% knockdown
of serum AAT.
As per company sources, there are around 10,000 to 20,000 people in
the U.S. and EU respectively suffering from AAT deficiency with
associated liver pathology.
Alnylam has made significant progress with its pipeline so far.
Patisiran and ALN-TTRsc are two of the most advanced candidates at
Alnylam. The company is evaluating patisiran in the phase III
APOLLO study in transthyretin-mediated amyloidosis patients
suffering from familial amyloidotic polyneuropathy. Alnylam intends
to file for regulatory applications for the candidate later in the
Meanwhile, ALN-TTRsc is in a phase II study in patients suffering
from familial amyloidotic cardiomyopathy. ALN-TTRsc is also an RNAi
therapeutic targeting the TTR gene being developed for the
treatment of ATTR. The company has multiple pipeline related
activities in the coming quarters. We expect investor focus to
remain on the development of the pipeline.
Alnylam, a biopharmaceutical company, presently carries a Zacks
Rank #3 (Hold). Some better-ranked stocks in the sector include
Regeneron Pharmaceuticals, Inc.
Gilead Sciences Inc.
Alexion Pharmaceuticals, Inc.
). While Gilead and Regeneron are Zacks Rank #1 (Strong Buy)
stocks, Alexion holds a Zacks Rank #2 (Buy).
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