Isis Pharmaceuticals, Inc.
) has seen significant gains in its share price ever since the
company reported encouraging results on its pipeline candidates.
Shares are up 21% over the last few days following the release of
data on ISIS-SMNRx and ISIS-APOCIIIRx.
The first set of encouraging news came with the company
announcing follow-up preliminary data on ISIS-SMNRx from a single
dose, open-label phase I study conducted in children with spinal
muscular atrophy (SMA). Results showed that children receiving
the two highest doses of the candidate (6 mg and 9 mg) continued
to show improvements in muscle function tests up to 14 months
after being administered a single injection.
The Hammersmith Functional Motor Scale-Expanded (HFMSE) was
used to analyze motor function in the children belonging to the
9-14 months age group. Isis Pharma said that improvement in HFMSE
scores were dose-dependent with higher improvements being
observed in the highest dose arm (9 mg). Most of the children in
this arm continued to show improvement during follow up with no
children declining. The safety profile also looked favorable.
ISIS-SMNRx is currently in phase II development - while a
phase II study is evaluating the safety, pharmacokinetic and
pharmacodynamic profile of ISIS-SMNRx in infants with type I SMA,
a phase Ib/IIa multiple-dose study is being conducted in children
with type II and type III SMA. Results from these studies should
be out later this year/early next year and Isis Pharma could move
ISIS-SMNRx into phase III development next year.
A couple of days after reporting data on ISIS-SMNRx, Isis
Pharma presented phase II data on its cholesterol management
candidate, ISIS-APOCIIIRx. Results were presented from a phase II
study evaluating ISIS-APOCIIIRx as a monotherapy in patients with
familial chylomicronemia syndrome (FCS).
FCS, a rare orphan disease which is estimated to affect about
3,000 to 5,000 people across the world, is characterized by
severely high triglyceride levels. Due to the high triglyceride
levels, FCS patients often suffer from other health issues like
abdominal pain, eruptive fatty skin lesions, enlargement of the
liver and spleen, and recurrent acute pancreatitis that often
Isis Pharma said that three patients on ISIS-APOCIIIRx
experienced a significant reduction in triglycerides (average
reduction of 69%) - the triglyceride level of all three patients
went below 500 mg/dL thereby leading to a significant decline in
the risk of an acute pancreatic event. Substantial reduction in
apoC-III and apoCIII-associated very low-density
lipoprotein-cholesterol (VLDL-C) particles was also achieved.
ISIS-APOCIIIRx' safety profile was favorable and the candidate
was well tolerated. Isis Pharma said that there were no
flu-like symptoms, treatment-related elevations of liver enzymes
greater than three times upper limit of normal, abnormalities in
renal function, clinically meaningful changes in other laboratory
values and treatment related serious adverse events.
Isis Pharma is working on moving ISIS-APOCIIIRx into phase III
development early next year in patients with FCS as well as
patients with severely high triglycerides. Although Isis Pharma
is yet to enter into discussions with regulatory authorities, it
believes a single phase III study with less than 50 patients,
along with the expanding phase II and phase III safety database
in other high-triglyceride patient populations will be enough to
support a rapid filing for FCS. At present, Isis Pharma expects
to file for the FCS and the severely high triglycerides
indications in 2016 and 2017, respectively.
While the data on ISIS-APOCIIIRx is encouraging, we note that
) oral LCQ908 is also being developed for the FCS indication and
is already in phase III development.
Isis Pharma currently carries a Zacks Rank #3 (Hold). At
present, companies like
) look well-positioned. Actelion is a Zacks Rank #1 (Strong Buy)
stock and Alexion a Zacks Rank #2 (Buy) stock.
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