Thumbs Up/Down

Thumbs Up/Thumbs Down: Major insurer delivers a bad blow to Duchenne families

Endpoints assesses the big biopharma R&D stories of the week, with a little added commentary on what they mean for the industry.

thumb down  Anthem’s ruling on Duchenne drug is a bad blow to families

The big US insurer Anthem has determined that Sarepta’s controversial Duchenne muscular dystrophy drug Exondys 51 is “investigational and not medically necessary,” a blow for families looking to gain coverage for this drug. The determination cites a long and troubled history for this drug, with failed studies and a host of questionable moves Sarepta $SRPT made in advancing this drug on the back of a tiny study. “Exondys 51 failed to show it improves health outcomes, and therefore it is not a covered benefit for our members,” Anthem spokeswoman Leslie Porras told Reuters, confirming the obvious. The discussion over this drug turned into a battle inside the FDA, with CDER director Janet Woodcock winning the argument in favor of an accelerated approval over the vehement objections of senior-level officials. As insurers refuse to cover this drug, which will be sold for $300,000, Woodcock has set up a scenario where families will be expected to fork over huge sums to treat children with a drug they have vowed works just fine. That would be a tragic outcome. The drug is experimental, of course. But families shouldn’t be left on the hook for the cost of a rare disease treatment like this, which may have no affect on the disease at all. And therein lies the tragedy behind this painful situation. Insurers can afford it and should step up and do the right thing, but we doubt they will. After all, insurers have investors as well. Sarepta’s shares were down 8% today on the news.

thumb Thumbs up for the fastest, most determined deal-maker in biopharma

Brent Saunders proved again that he’s the most intrepid buyer in the industry today. In Tobira’s blow-by-blow account of the auction that took place for the company in SEC documents, Saunders got the ball rolling last summer at dinner with Tobira execs in NYC, and didn’t let up until he had bagged the company and its (yes, failed) NASH drug for a big premium. Amid all the discussion about biotech valuations these days, Saunders’ Allergan has cash for new deals, and it’s not waiting around to see how the numbers change. We’ll see eventually if the strategy on Tobira pays off, but you have to admire the do-it-now attitude at Allergan.

thumb Hubs win, again

Our most popular story to ring the web traffic bell this week is definitely our scoop on Novartis‘ latest reshuffling of its global R&D ops. The company has some clear ideas about bringing together researchers into its main hubs, which once again underscores the industry trend toward intermingling with more biotechs and pharma R&D groups. Unfortunately, the trend has a heavy impact on employees, as we saw with Novartis’ recent decision to shutter its standalone cell therapy operations and integrate with the broader oncology group. But the irresistible momentum has to be recognized as an overall positive for the industry, which will see the continued expansion of industry R&D hubs at the expense of outliers like Singapore.

thumb down  RNAi suffers another damaging blow

Alnylam $ALNY has seen it all when it comes to RNAi development. They were there when enthusiasm for gene silencing hit a peak and Big Pharma stepped in. Then they watched Big Pharma step out. And they were ready when Sanofi decided to partner up in a big way. With two drugs in late-stage development, they were also hoping to do some pioneering commercial work in the not-too-distant future. Sadly, one of its two Phase III drugs imploded this week. Alnylam pinned it on an imbalance of deaths, meaning more patients were dying in the drug arm. But frankly, we don’t have a whole lot of hard insight on what went wrong. You can be sure that every bit of data coming out in the future will be given a hard look by analysts. This field still tends to advance and retreat as a group based on the work of the leaders. Right now, RNAi is back in the dog house. It’s up to Alnylam to get back out.

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John Carroll, Editor and Co-Founder

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