Wednesday October 24, 2012
by John McCamant, editor Medical Technology Stock Letter
Isis Pharmaceuticals (ISIS) has had a gut wrenching couple of days; the stock fell when the FDA released briefing documents for an advisory panel meeting for its drug, Kynamro, which showed that malignant growth developed in 3.1% of patients vs. 0.9% of patients on placebo.
Then, the company cleared a hurdle when it received a positive 9-6 FDA panel vote for Kynamro to treat Homozygous Familial Hypercholesterolemia (HoFH), a rare genetic lipid disorder that causes severe elevations in total cholesterol
Despite all the negativity from Wall Street regarding the cancer data, the issue was never a major concern for the panel. The committee did not appear to be greatly concerned about the possibility of a cancer signal brought up in the FDA review.
The bottom line was that most of the cancers observed in the clinical trials occurred in elderly patients who did not have homozygous FH and that a young homozygous FH population would be less susceptible to an elevated cancer risk.
While the committee did express concerns about both the efficacy and safety of the drug, ultimately the severity of homozygous FH led the panel to recommend approval. “We need a toolkit, we need as many options as possible for these patients,” said one panel member.
The biggest obstacle to Kynamro was the question over liver safety. Committee members wrestled with the issue without reaching a consensus.
The belief is that the FDA’s proposed Risk Evaluation and Mitigation Strategies (REMS) will work as intended. The REMS would require special certification for health care professionals and pharmacies that prescribe and dispense the drug.
While FDA approval is never a certainty despite a positive panel vote, as the FDA does not have to follow the panel’s advice, we believe that the drug will be approved by the PDUFA date of January 29th.
The relatively close 9-6 vote and the market sell-off combined to keep pressure on ISIS’ stock price. It may take some time for the stock to fully recover in the current market. That being said, there are some significant upcoming catalysts that should help the stock recover.
First, ISIS should receive European approval by year end. Remember this application addresses larger markets as they are seeking approval to treat both HoFN and HeFN, the latter being a much larger market opportunity.
Following European approval we expect FDA approval on or before the PDUFA data of January 29th. ISIS is a buy under $12.
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