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.


  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.


 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.


 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.


  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.

UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

GSK of­floads two vac­cines in $1.1B deal as it works to re­vive the pipeline

GlaxoSmithKline is leaving the deep dark woods and its viruses behind.

GSK has agreed to divest its vaccines for rabies, RabAvert, and tick-born encephalitis vaccine, Encepur, to Bavarian Nordic, part of the company’s broader efforts to narrow its pipeline and focus on oncology and immunology.

The deal is worth up to nearly $1.1 billion, with a $336 million upfront payment. GSK acquired the vaccines from Novartis as part of an exchange for their late-stage oncology programs in 2015 under former chief Sir Andrew Witty.

Pfiz­er gets some en­cour­ag­ing PhI­II news on a fran­chise sav­ior, but is a dos­ing ad­van­tage worth the $295M up­front?

Close to 3 years after Opko tried to defend itself as shares tumbled on the news that its long-acting growth hormone had failed to outperform a placebo, the Pfizer partner $PFE is back. And this time they’re pitching Phase III data that demonstrates their drug is non-inferior — or maybe a tad better — than their well-known but fading standard in the field.
The comparator drug here is Genotropin, which earned a marginal $142 million for Pfizer last year — down 9% from the year before. Approved 24 years ago, biosimilars are now in development that Pfizer would like to stay out in front of. The market leader here is Norditropin, a growth hormone from Novo Nordisk which uses the same basic ingredient as Genotropin which the pharma giant sells with a kid-friendly self-injectable pen. That would also present some big competition if the new therapy from Opko/Pfizer makes it to the market.
The new data, says researchers, underscore that a weekly injection of somatrogon performed as well or slightly better than Genotropin (somatropin) in young children with growth hormone deficiency. Investigators tracked height velocity at 10.12 cm/year, edging out the older drug’s 9.78 cm/year. That 0.33 difference may not prove compelling to payers, though, who have been known to overlook dosing advantages in favor of lower costs.
That message may have weighed on the stock reaction this morning, with a 30%-plus hike $OPK giving way to more marginal gains.
Back in late 2016, Opko had to defend itself against a devastating Phase III setback as their initial late-stage trial failed against a sugar pill. Opko later blamed that setback on outliers in the study, though it wasn’t able to expunge the failure.

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As­traZeneca's Farx­i­ga scores FDA nod to cut risk of hos­pi­tal­iza­tion for heart fail­ure in di­a­bet­ics

While the FDA recently spurned an application to allow AstraZeneca’s blockbuster drug Farxiga for type 1 diabetes that cannot be controlled by insulin, citing safety concerns — the US regulator has endorsed the use of the SGLT2 treatment to reduce the risk of hospitalisation for heart failure in patients with type-2 diabetes and established cardiovascular disease or multiple CV risk factors.

IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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Alex­ion clinch­es aHUS ap­proval for Ul­tomiris as the clock ticks on Soliris con­ver­sion

Alexion has racked up a second approval for Ultomiris, the successor therapy to Soliris, as its mainstay blockbuster therapy faces a patent review process that could drastically shorten its patent exclusivity.

The FDA OK for atypical hemolytic uremic syndrome (aHUS) on Friday was widely expected after Alexion posted a full slate of positive Phase III data in January. But regulators also flagged concerns about serious meningococcal infections, slapping a black box warning on the label and mandating a REMS.

FDA ap­proval lets Foamix set its maid­en ac­ne ther­a­py on course for US mar­ket launch

Months ago, Foamix leaned on its biggest shareholders — Perceptive Advisors and OrbiMed — to financially grease its wheels, ahead of the FDA decision date for its acne therapy. On Friday, that approval came in — and the topical formulation of the antibiotic minocycline is set for a January launch.

The therapy, Amzeeq (formerly known as FMX101), was approved to treat inflammatory lesions of non-nodular moderate-to-severe acne vulgaris in patients aged 9 and older.

Christine Bunt, Robert Langer. Verseau

Armed with Langer tech and $50M, Verseau hails new check­point drugs un­leash­ing macrophages against can­cer

The rising popularity of CD47 has propelled the “don’t-eat-me” signal to household name status in the immuno-oncology world: By blocking that protein, the theory goes, one can stop cancer cells from fooling macrophages. But just as PD-(L)1 merely represents the most fruitful of all checkpoints regulating T cells, Verseau Therapeutics is convinced that CD47 is one of many regulators one can modulate to stir up or tame the immune system.