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Isis Pharmaceuticals, Inc. (ISIS - Analyst Report) 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 requires hospitalization.

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 Novartis' (NVS - Snapshot Report) 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 Actelion Ltd. (ALIOF) and Alexion Pharmaceuticals (ALXN - Analyst Report) look well-positioned. Actelion is a Zacks Rank #1 (Strong Buy) stock and Alexion a Zacks Rank #2 (Buy) stock.
 

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