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The Readout

Last call: Tomorrow, STAT Plus subscribers can join us for tips on how to interpret clinical trials like a pro. More here

Amarin fish oil capsule shows dramatic benefit for cardiovascular patients

The biopharma company Amarin is making some heart-medicine history today with its proprietary, prescription formulation of fish oil, called Vascepa.

In stunning clinical trial results that upend years of skepticism about the long-term heart benefit of products containing omega-3 fatty acids, Vascepa significantly reduced the risk of deaths, heart attacks, strokes, and other serious cardiovascular events compared with a placebo.

Amarin designed its cardiovascular outcomes study, known as REDUCE-IT, hoping to show Vascepa could meet the primary endpoint with a 15 percent risk reduction.

The result announced this morning: a 25 percent risk reduction — highly statistically significant.

“This is absolutely the most significant study in the field of cardiovascular risk reduction since the statins were introduced,” said Dr. Matthew Budoff, a cardiologist at UCLA and a Vascepa study investigator.

Read more.

On Nightstar's ocular gene therapy


Nightstar Therapeutics, a U.K.-based biotech, just brought its experimental gene therapy for X-linked retinitis pigmentosa stateside — introducing new genes to a 23-year-old Puerto Rican man in Miami to help reverse his inherited blindness. Julio Adomo Nieves is the 16th person to join the worldwide trial. The company this weekend released proof-of-concept data showing dose-related improvements for the drug.

Retinitis pigmentosa is a popular candidate for gene therapy — Horama, a French biotech, is working on a similar approach to the disease. It makes sense: Spark Therapeutics’ Luxturna, the gene therapy for another inherited eye condition, was approved late last year — costing $425,000 per eye. The patient population there is less than 2,000. Retinitis pigmentosa offers up a larger market — with an estimated 17,000 patients in need of therapy in the U.S. and Europe. 

The risk with diagnostics like Cologuard


Direct-to-consumer marketing is fueling the adoption of diagnostics like Cologuard, a colon cancer detection test. The drug’s maker, Exact Sciences, recently formed a marketing partnership with Pfizer to ramp up distribution of this simple, stool-based DNA test. 

But some physicians worry that the convenience of these DNA diagnostics winds up eclipsing the flaws inherent in these tests. Colonoscopy remains the gold standard for cancer screens, one gastroenterologist opines — citing a 2014 New England Journal of Medicine study that found that Cologuard detects 93 percent of colon cancers. Another way to spin that, however, is that the diagnostic doesn’t detect the cancer in 1 in 13 test takers.

Read more. 

On modeling Alzheimer's better

When it comes to Alzheimer’s research, we keep coming up on dead ends. So maybe it’s time researchers get back to basics — and instead of testing drugs in humans and failing miserably, they instead work on developing better mouse models for the disease.

That’s exactly what Jackson Laboratory and Indiana University are doing right now — with aims to release a handful of new mouse models that produce more human-like amyloid and tau proteins. 

“The vast majority of those models don’t model the other pathologies of the disease,” John Dunlop, the vice president of neuroscience research at Amgen, told STAT. “As we move into therapeutics approaches that are not directly targeting amyloid, we’ll need to generate mouse models that are based on the mechanistic biology we’re looking at.”

Read more. 

More reads

  • uBiome jumps into therapeutics with $83 million Series C. (TechCrunch)
  • Novartis and Bayer lose a UK court fight over a switch to a cheaper eye drug. (STAT)

Thanks for reading! Until tomorrow,


Monday, September 24, 2018


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