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: Roche bags 'break­through' an­ti-fi­bro­sis drug in $1.4B biotech buy­out deal

Roche is snapping up a “breakthrough” anti-fibrotic drug in a $1.4 billion buyout.

The pharma giant announced Friday that it is acquiring Promedior, primarily to get its hands on PRM-151, a recombinant form of human pentraxin-2 (PTX-2) protein that has nailed down mid-stage clinical data on idiopathic pulmonary fibrosis and demonstrating its potential for a range of fibrotic conditions.

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Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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No­var­tis spin­out’s first an­ti-ag­ing PhI­II is a flop, so now they’ll turn to Parkin­son’s chal­lenge as shares wilt

Novartis spinout resTORbio is grappling with the collapse of its lead clinical program this morning — an anti-aging R&D failure that will badly damage their rep in the field.

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No­var­tis scores its lat­est FDA OK — this time for a new sick­le cell dis­ease drug picked up in a $665M deal

Novartis’ decision to buy Oklahoma-based biotech Selexys 3 years ago for up to $665 million has paid off with an FDA approval today.

Blessed with the FDA’s breakthrough drug designation for a speedy review, the pharma giant has pinned down an approval for crizanlizumab, a new therapy designed to reduce the frequency of painful incidents of vaso-occlusive crises among sickle cell disease patients 16 or older.

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As­traZeneca gains EU nod for di­a­betes triple; Am­gen and Duke launch re­al-world PC­SK9 ob­ser­va­tion­al study

→ Weeks after winning EU approval to start marketing dapagliflozin as Forxiga, AstraZeneca has racked up another OK for a triplet combo involving the SGLT2 diabetes drug. Named Qtrilmet, the pill combines Forxiga with the DPP-4 inhibitor Onglyza (saxagliptin) and the bedrock drug metformin in a modified-release format. That 3-in-1 approach proved superior in reducing average blood glucose levels to a number of other dual combinations across 5 Phase III trials, including Forxiga plus metformin, Onglyza with metformin, or glimepiride with metformin.

Five drugs, in­clud­ing two No­var­tis ther­a­pies, win EMA en­dorse­ment

As is custom, an EMA panel on Friday issued its weekly recommendations on marketing applications submitted by drug developers. This week, the agency backed the use of five new therapies — including two Novartis drugs — but issued no negative reviews.

Novartis’ S1P drug for relapsing forms of multiple sclerosis (MS) drug, Mayzent (known chemically as siponimod), which was approved by the FDA in March — has been given the nod by the EMA. The Swiss drugmaker already sells its other MS drug, Gilenya, in both regions.

Atom­wise's X-37 spin­out gets $14.5 mil­lion to launch AI dis­cov­ery ef­forts

The folks behind Atomwise’s spinout X-37 like to think in cosmological metaphors, and you can think of their AI drug development model as probes sent into space from a central station. That station just got $14.5 million in Series A funding from DCVC Bio, Alpha Intelligence Capital and Hemi Ventures to back those missions.

X-37 uses Atomwise’s AI platform to identify drug targets and – unlike the parent company, which largely sticks to computers  – bring those into a wet lab and preclinical testing.  In addition to AI professionals, it’s led in by part by drug developers from Velocity Pharmaceutical Development.

Ab­bott Lab­o­ra­to­ries CEO Miles White pass­es ba­ton down to suc­ces­sor; Lon­za CEO Marc Funk hits the ex­it

→ Abbott Laboratories has named a successor to CEO Miles White after he announced that he was stepping down in March after 21 years of service. Robert Ford, the company’s COO and president, will take the helm. Ford is known for his work in the $25 billion merger between St. Jude Medical into Abbott in January 2017. White will remain with the company as executive chairman of the board. 

→ After snapping up Novartis’ Swiss facility, Novartis Center of Excellence, in July, Lonza has announced that their CEO, Marc Funk, is hitting the exit for “personal reasons.” Funk has been the CEO of the company for less than a year — brought onto the company back in March. In the meantime, chairman Albert Baehny will serve as interim CEO. 

UCB adds on more pos­i­tive PhI­II da­ta for IL-17A/17F in­hibitor bimek­izum­ab, clear­ing a path to the FDA

A month after posting positive top-line data from their first Phase III trial of the IL-17A/17F inhibitor bimekizumab, Belgium’s UCB says they’ve added more upbeat results from their second late-stage test in moderate-to-severe plaque psoriasis.

That leaves the company on track for regulatory submissions in the middle of next year, says CMO Iris Loew-Friedrich.
Their drug beat out a placebo on the co-primaries — a 90% improvement in PASI 90 (the Psoriasis Area and Severity Index) and Investigator Global Assessment (IGA) response of clear or almost clear (IGA 0/1) at week 16, compared to placebo. Investigators also boasted of hitting some key secondaries.
UCB is angling to enter an increasingly crowded market space.
In their first of 3 Phase III studies for bimekizumab, researchers touted top-line wins on statistically significant results on clearing plaque psoriasis, including a victory over J&J’s IL-23 contender Stelara on key endpoints. The drug targets both IL-17A and IL-17F, a modification on the IL-17A strategy laid out for Taltz (Eli Lilly) and Cosentyx (Novartis). And the new group also includes J&J’s Tremfya and AbbVie’s Skyrizi.

Social image: UCB