Amarin $AMRN has staked its claim to the next big blockbuster in the cardio space, heralding a clear statistical win on the primary endpoint for its 7-year clinical quest to prove that its industrial strength purified fish oil Vascepa can significantly reduce major cardio threats for a broad swath of patients.
An early preview of the top-line data rolled out at a bleary-eyed time in the morning on Monday pointed a direct arrow at a relative 25% drop in risk for major cardio events — such as death, stroke and hospitalization due to angina — hitting a highly statistical p=<0.001 for the patients in the years-long REDUCE-IT study. And researchers also high-fived each other for a clean safety profile in the same headline data to make the statement.
The data supported “robust” evidence of its effect across multiple secondary endpoints, according to the company. We won’t see the particulars until later, but the company is on track to file for approvals in early 2019.
Analysts at Jefferies have said that a success here could transform Vascepa into a $2 billion-plus drug. And that number resonated this morning, as Amarin’s share prize zoomed up 288%. The stunning advance followed fresh remarks from Jefferies about the relative value that Amarin underscored this morning relative to other drugs that have tried — and failed — to make much of an impression.
The magnitude of MACE reduction is so far the greatest among all therapies on top of statins, far exceeding the clinically meaningful CV benefit expectation. Recall two PCSK9 inhibitors (Repatha and Praluent) and Ilaris all reduced MACE risks by 15% in recently completed CVOTs (FOURIER, ODYSSEY, and CANTOS). The FDA has expanded Repatha’s label to include certain CV risk reduction and is currently reviewing sBLA of Praluent in MACE reduction (PDUFA on 4/28/19). Our doc survey suggested 10-15%+ risk reduction in MACE is considered clinically meaningful.
“We are delighted with these topline study results,” said John Thero, president and CEO of Amarin, in a statement. “Given Vascepa is affordably priced, orally administered and has a favorable safety profile, REDUCE-IT results could lead to a new paradigm in treatment to further reduce the significant cardiovascular risk that remains in millions of patients with LDL-C controlled by statin therapy, as studied in REDUCE-IT.”
None of this is going to make it easy for the company, though, with a host of angry short investors likely to try whatever it takes to foment a backlash against the data for the last big catalyst of Q3. Further complicating their success, AstraZeneca has its own outcomes study for the rival Epanova ready to read out next year, while GSK’s Lovaza went generic several years ago.
But as the morning wore on, it became apparent that Amarin was holding on to its big gains and supporters are swooning.
Patients enrolled in REDUCE-IT had registered an LDL-C level between 41-100 mg/dL (median baseline LDL-C 75 mg/dL) controlled by statin therapy with a slate of cardiovascular risk factors including: persistent elevated triglycerides between 150-499 mg/dL (median baseline 216 mg/dL) and either established cardiovascular disease or diabetes mellitus and at least one other CV risk factor.
That’s a big market.
This Phase III has been endlessly debated as the time drew near for the readout. Amarin’s Vascepa — approved more than 4 years ago for hypertriglyceridemia — demonstrated its ability to significantly reduce triglycerides in those patients while influencing biomarkers on inflammation. But as we know from past big cardio studies, that certainly doesn’t necessarily translate into the kind of hard and demonstrable health benefits such that payers are willing to cover much larger mass populations of at-risk members.
REDUCE-IT set out to answer that question, with researchers enrolling more than 8,000 patients so they could compare it as an add-on to statins in reducing instances of major adverse cardiovascular events (MACE) as compared to statin-treated patients plus placebo.
We already know from the recent ASCEND study that popular fish oil products sold in every pharmacy have no clear health benefits, and Amarin has been built on the notion that its highly-purified, omega-3 fatty acid product could distinguish itself from the supplement field. Company execs said that fish oil’s weakness would simply make it easier to prove Vascepa’s value, once the outcomes data were in.
Is it good enough?
Even positive cardio data don’t always translate into commercial opportunity, and analysts now will see whether Amarin has come up with data that are good enough to qualify as the practice-changing event that the company’s backers have insisted would now be in reach.
The difference amounts to a market with millions of potential patients.
Supportive Jefferies analysts who helped build support for the drug’s success recently noted:
Positive REDUCE-IT will be practice changing as it will provide POC that using a highly purified Omega-3 drug (vascepa) in MD patients on stable statins and relatively well-controlled LDL-C (<100mg/dL) contributes to a CV benefit. While LDL-C has decreased over decades, TGs continue to rise. The market needs this type of drug.
But for every advocate, you could count two more who bet against the drug and any upbeat rise in the share price. The die hards won’t retreat without putting up a fight, but their numbers were definitely dwindling Monday.
Image: John Thero. AMARIN
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