The recent announcement of Amarin's (AMRN) upcoming FDA Advisory Panel, scheduled to take place on October 16, 2013, has generated its fair share of negative publicity from certain bloggers, who point to this as a potential roadblock for the company's Vascepa drug getting approved for an expanded patient population. However, at the same time, other bloggers are pointing positively to this same advisory panel announcement and saying that this is business as usual for the FDA, and claiming that this is just the next step along the way towards a sNDA approval. These alternative perspectives cannot both be correct, and thus, lead to two questions for biotechnology investors. Investors need to know who is correct amongst the naysayers and the lauders, and more importantly, why are they correct?
The answer to the first question is pretty clear to me, and it is that the FDA Advisory Panel is indeed business as usual. The naysayers are wrong, and I predict that Amarin and Vascepa will have no problems getting a positive recommendation from the panel on October 16th with no recommended delays. Going a step further, I expect FDA approval for the ANCHOR indication thereafter, and again with no delays. Having answered the first question, now let me address the reasons why this outcome is the case.
My reasons for the aforementioned positive statement for Vascepa's success are based on what an advisory panel does and what it does not do. The FDA's own website states that:
"Advisory committees provide FDA with independent opinions and recommendations from outside experts on applications to market new drugs, and on FDA policies. The marketing applications include data to show the safety and effectiveness of human drugs. The outside experts receive summary information about the applications and copies of FDA's review of the application documents. Based on this information, advisory committees may recommend approval or disapproval of a drug's marketing application."
A careful reader will note that advisory committees provide opinions and recommendations on applications to market new drugs, and that these marketing applications include data to show the safety and effectiveness of new drugs. Therefore, the real questions to be asked around this are not those posed by Adam Feurerstein and others (whether a combination statin-therapy is next on the market or what are the true cardiovascular benefits of any fish oil), but instead are a) is Vascepa safe for the patient population with triglyceride levels ranging from 200 to 499 mg/dL (and mixed dyslipidemia), including those on statins and b) is Vascepa effective at lowering triglycerides? Those are the only two questions the panel will truly be considering, and they have-- in reality-- already been answered.
The answer to any questions regarding whether Vascepa is safe for the patient population for which Amarin is seeking a new indication is a resounding yes. Beyond the fact that Vascepa has a safety profile akin to taking a placebo, more importantly, the drug has also been shown to lower LDL-cholesterol in addition to triglycerides, thus avoiding the problems that have been a thorn in the already approved Lovaza. Lovaza has only limited use to reduce triglyceride levels in adult patients with severe (≥500 mg/dL) hypertriglyceridemia. This is due to the fact that it may increase levels of LDL-cholesterol, and thus has not been approved for use in the patient population that the ANCHOR indication would seek to address. It stands to reason that if Lovaza has been approved, then Vascepa will have no issues in terms of safety since it performs better than Lovaza and does not have the same risk of raising LDL-cholesterol that Lovaza does. Further, safety data from both the MARINE study and the ANCHOR study was considered in the original FDA approval of Vascepa, so the FDA has in fact already given its answer regarding the safety of Amarin's drug.
The second question for the advisory panel is whether Vascepa is effective at lowering triglycerides in the patient population, and again the answer to this question is a resounding yes. A study published in the American Journal of Cardiology based on results from the ANCHOR study indicated that Vascepa is effective. The study, entitled 'Efficacy and Safety of Eicosapentaenoic Acid Ethyl Ester (AMR101) Therapy in Statin-Treated Patients With Persistent High Triglycerides' had a conclusion that left nothing to the imagination - "AMR101 4 g/day significantly decreased median placebo-adjusted TG, non-HDL cholesterol, LDL cholesterol, apolipoprotein B, total cholesterol, very-low-density lipoprotein cholesterol, lipoprotein-associated phospholipase A2, and high-sensitivity C-reactive protein in statin-treated patients with residual TG elevations."
For the less scientifically inclined, the basics are that Vascepa at the four gram a day dosage decreased triglyceride levels by 21.5% and non-HDL cholesterol by 13.6%. It was also shown that this dosage produced even better numbers on patients with higher-efficiency statin regimens and patients with higher baseline triglyceride levels. Of note for comparisons to Lovaza, Vascepa reduced LDL cholesterol by 6.2% as opposed to raising it, which again is a risk with the use of Lovaza. In summary, Vascepa is effective.
The questions that the advisory panel must ask have in effect already been answered, and Vascepa is both safe and effective at reducing triglyceride levels in the patient population that Amarin is seeking to obtain an indication for with the ANCHOR data. Amarin President John Thero indicated that the company expected the advisory panel at the Jefferies 2013 Global Healthcare Conference on June 6 (the webcast is available here). When the actual announcement regarding the advisory panel came on June 19th, I doubt anyone at Amarin was surprised, and I believe that there is nothing out of the ordinary about this under a special protocol assessment.
I believe that Amarin's naysayers have confused the purposes of the committee for the indication sought, and convoluted the actual indication being sought. In fact, at this time Amarin is not seeking an indication for the reduction of cardiovascular events, and will not be doing so until the completion of the REDUCE-IT study. Therefore, questions about this are not relevant. The truth is simple - Vascepa works at reducing triglycerides and is safe - and that is all the FDA Advisory Panel will be concerned with in October. Investors should expect a green light from the panel, and a large majority of the panel (possibly unanimous) to give positive recommendations for approval of the ANCHOR indication on October 16th.
Dr. Eliot Briton, the President of American Board of Clinical Lipidology summed it up best:
"For key first-in-class indications, an FDA advisory committee meeting is expected, and this public forum will be an important opportunity to discuss the ANCHOR data, which demonstrated Vascepa's unique potential as an adjunct to diet in the treatment of adult patients with high triglycerides (TG 200-499 mg/dL) and mixed dyslipidemia… Currently, many of these patients are receiving another prescription omega-3 which is not indicated for this disorder. Having instead an omega-3 product which lowers LDL-cholesterol in addition to triglycerides, has tolerability comparable to placebo, and is FDA-approved for use on top of statin therapy would be a welcome addition to the physician's armamentarium for comprehensive lipid management."
Amarin will have no problems moving forward, and investors should look upon the panel as an opportunity rather than a hurdle to overcome. The current price of the stock, hovering near its 52-week low, makes for a prime buying opportunity ($6.29 p/s as of market close on Friday, June 21, 2013). Additionally, the news released on June 21st regarding the positive results of the 'Phase 1 Clinical Trial Measuring the Bioavailability of a Fixed-Dose Combination of Vascepa(R) and a Statin Compared to Concomitant Administration' should have investors excited about new potential avenues of sales and profit for the company.