We recently reiterated our Neutral recommendation on
), which carries a Zacks #3 Rank (short-term Hold rating).
Isis Pharma's lead pipeline candidate, Kynamro (mipomersen), is
being developed for the treatment of familial hypercholesterolemia
(FH). Isis Pharma has a collaboration agreement with Genzyme, a
) for Kynamro.
In late March 2012, Sanofi submitted a new drug application
seeking US Food and Drug Administration (FDA) approval for the use
of Kynamro for the treatment of patients with homozygous (Ho) FH
(HoFH). A response from the FDA should be out by January 29, 2013.
Approval would trigger a $25 million milestone payment from
Meanwhile, the EU filing for HoFH and severe heterozygous FH
(heFH) was submitted in July 2011 - the companies are expecting to
launch Kynamro later this year. Isis Pharma and Genzyme are
conducting a 12-month study (FOCUS FH - saFety and atherOgeniC
lipoprotein redUction of mipomerSen in FH).
This study is being conducted to support the expansion of the FH
patient population to include severe heterozygous FH in the US,
support an alternative dosing regimen (three times a week), and
broaden the FH indication beyond severe in Europe. Successful
development could allow Kynamro's label expansion in the 2015-2016
We are also positive on Isis Pharma's 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.
However, we are concerned about Isis Pharma's dependence on
Kynamro for future growth. With all eyes focused on the successful
development and commercialization of this candidate, any
development or regulatory hurdle would be a major setback for the
company. A delay in gaining approval would weigh heavily on the
In fact, although Isis Pharma has consistently presented
encouraging results on Kynamro, concerns regarding the candidate's
safety profile remain with the company reporting elevations in
liver transaminases, which is a sign of possible liver damage. We
remain worried that concerns regarding the safety profile of the
drug could limit its commercial potential once it is launched.
Moreover, Kynamro is targeting the highly competitive and
crowded cardiovascular disease market. In addition to competing
with existing therapies, the company will also face competition
from companies that are pursuing the development of drugs based on
) is not far behind with the company having filed for US and EU
approval of its candidate, lomitapide, in early March 2012 - a
response in the US could be out in January 2013.
We expect investor focus to remain on the regulatory progress of
AEGERION PHARMA (AEGR): Free Stock Analysis
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SANOFI-AVENTIS (SNY): Free Stock Analysis
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