) company, and partner
Isis Pharmaceuticals Inc.
) recently announced that their new drug application (NDA) for
pipeline candidate Kynamro has been accepted by the US Food and
Drug Administration (FDA) for standard review.
The companies are looking to get Kynamro approved for the
treatment of patients with homozygous familial hypercholesterolemia
(HoFH). Genzyme had submitted the NDA in March 2012.
Kynamro is the lead pipeline candidate at Isis Pharma. Isis
Pharma and Genzyme filed for European approval of Kynamro in July
2011 for use in patients with homozygous familial
hypercholesterolemia (hoFH) and severe heterozygous FH (heFH). On
its first quarter conference call, Isis Pharma had said that
Genzyme is preparing for the EU launch of Kynamro later this
With the acceptance of the NDA filing, Isis Pharma stands to
receive $25 million from Genzyme. US approval would trigger another
milestone payment of $25 million from Genzyme. The $25 million
milestone payment for the approval of Kynamro will be due in 2013
given the January 29, 2013 FDA action date, provided Kynamro is
approved at that time.
Meanwhile, Isis Pharma and Genzyme are conducting a 12-month
study (FOCUS FH - saFety and atherOgeniC lipoprotein redUction of
mipomerSen in FH) with Kynamro. The study is being conducted to
support the expansion of the initial label and support an
alternative dosing regimen (three times a week). The study is being
conducted under the FDA's Special Protocol Assessment (SPA)
Neutral on Isis Pharma
We currently have a Neutral recommendation on Isis Pharma, which
carries a Zacks #3 Rank (short-term Hold rating). We are positive
on Isis' agreements with
) which not only validate its antisense technology but also provide
Isis Pharma with funds in the form of upfront, milestone and other
payments. We expect investor focus to remain on the regulatory
progress of Kynamro.
BIOGEN IDEC INC (BIIB): Free Stock Analysis
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