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.

Albert Bourla appears before the Senate Committee on Finance for a hearing on prescription drug pricing on Capitol Hill in Washington, DC, February 26, 2019. Chris Kleponis for CNP via AP Images

UP­DAT­ED: Pfiz­er CEO Al­bert Bourla is back in the M&A game, but why is he pay­ing $11.4B for Ar­ray?

Pfiz­er $PFE has cut short its time on the side­lines of bio­phar­ma M&A.

Mon­day morn­ing the phar­ma gi­ant un­veiled an $11.4 bil­lion deal to ac­quire Ar­ray Bio­Phar­ma, beef­ing up its on­col­o­gy work and adding a new re­search hub in Boul­der, Col­orado to its glob­al op­er­a­tions.

At $48 a share, Ar­ray $AR­RY in­vestors will be get­ting a 62% pre­mi­um off the Fri­day close of $29.59.

Pfiz­er, which has strug­gled to gain all the up­side promised in past buy­outs like Medi­va­tion, high­light­ed the ac­qui­si­tion of 2 ap­proved drugs in the deal — Braftovi (en­co­rafenib) and Mek­tovi (binime­tinib).

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Gene ther­a­py R&D deals turn red hot as Big Phar­ma steps up to play

This September will mark the 20th anniversary of the death of Jesse Gelsinger, a young man suffering from X-linked genetic disease of the liver. He was killed in a gene therapy study conducted by Penn’s James Wilson, and the entire field endured a lengthy deep freeze as the field grappled with the safety issues inherent in the work.

Some thought gene therapy R&D would never survive. But it did. And this year marked a landmark approval for Zolgensma, a new gene therapy for spinal muscular atrophy Novartis priced at $2.1 million.

“Gene therapy is the hottest item on the block now. But there was a time when we first got into this trial, where there wasn’t a person in the world who believed that gene therapy would work. We have to remember that,” noted gene therapy investigator Jerry Mendell told SMA News Today.

We’re still right on the pioneering frontier when it comes to getting approvals for gene therapies and launching marketing campaigns with the European green light for bluebird's leading program last Friday underscoring the nascent nature of the field. But gene therapy R&D is booming, and has been for several years now.

The rapid growth of gene therapy clinical development is well known, but we decided to put some numbers on it, to quantify what’s going on. DealForma chief Chris Dokomajilar took a lot over the past 10 years, as the number of deals, R&D partnerships and buyouts steadily gained steam, spiking last year and on track to maintain the surge in 2019.

The upfronts and totals for the dollars on deals so far in 2019 is already close to the 2018 mark, underscoring a new phase of negotiations as the major players step up to gain a piece of the late-stage and commercial action.

Once again, we’re looking at an “overnight” biotech success story, decades in the making.

At some point, that may start to brake the numbers we’re seeing. But for now, as rivals line up to compete for frontline prominence across a range of diseases, the arrows are all pointed north.

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A uni­corn stalks Wall Street in search of IPO cash; CASI Phar­ma in-li­cens­es CD19 ther­a­py from Chi­na’s Ju­ven­tas

→ A herd of up­start biotechs will look to Wall Street for some ma­jor wind­falls this week as a burst of new of­fer­ings con­tin­ues to feed cash in­to the R&D sys­tem. To­day we learned that Bridge­Bio will look to raise in the neigh­bor­hood of $225 mil­lion by of­fer­ing 15 mil­lion shares for $14 to $16 each. And they have a string of joint bookrun­ners: J.P. Mor­gan, Gold­man Sachs, Jef­feries, SVB Leerink, KKR, Piper Jaf­fray, Mizuho Se­cu­ri­ties, BMO Cap­i­tal Mar­kets and Ray­mond James. If suc­cess­ful, Bridge­Bio will emerge with a mar­ket cap of around $1.7 bil­lion. There are 5 biotechs look­ing to IPO this week, in­clud­ing Akero and Pre­vail.

UP­DAT­ED: Sanofi Gen­zyme deserts gene ther­a­py de­vel­op­er Voy­ager Ther­a­peu­tics

While gene ther­a­py com­pa­nies re­joice as the sec­tor gains trac­tion with ap­provals and a flur­ry of M&A ac­tiv­i­ty, one play­er is feel­ing the heat.

Back in 2015, Voy­ager Ther­a­peu­tics joined forces with Sanofi Gen­zyme in a deal worth up to $845 mil­lion ($100 mil­lion up­front + a po­ten­tial $745 mil­lion in mile­stones) to co-de­vel­op gene ther­a­pies for se­vere cen­tral ner­vous sys­tem dis­or­ders. But two years lat­er, the French drug­mak­er re­treat­ed, elect­ing to not pick up the op­tion to work on Voy­ager’s Parkin­son’s dis­ease pro­gram. (Last year, the FDA dis­ap­point­ed Voy­ager, telling the com­pa­ny that it was not open to an ac­cel­er­at­ed fil­ing on the Parkin­son’s drug on the ba­sis of Phase II da­ta — in­stead of re­quir­ing an ad­di­tion­al piv­otal study.)

In­vestors fret as VBI's hep B vac­cine fails key sec­ondary PhI­II study goal

Sobered by mount­ing costs, Dy­navax $DVAX last month made the de­ci­sion to fo­cus all its re­sources on its 2017-ap­proved he­pati­tis B vac­cine Hep­lisav-B, which ri­vals and su­per­sedes the ef­fi­ca­cy and con­ve­nience pro­file of GSK’s $GSK es­tab­lished En­ger­ix-B. The Cal­i­for­nia-based com­pa­ny will be on the look­out for an­oth­er com­peti­tor — VBI Vac­cines, which on Mon­day un­veiled late-stage da­ta on its hep B vac­cine: Sci-B-Vac.

John Oyler, Founder & CEO of BeiGene, at the US-China Biopharma Innovation and Investment Summit in Shanghai on October 23, 2018; Credit: Endpoints News, PharmCube

UP­DAT­ED: As Bris­tol-My­ers/Cel­gene tie up loose ends, BeiGene pock­ets $150M from PD-1 breakup

As soon as Bristol-Myers Squibb announced its $74 billion buyout for Celgene, BeiGene emerged as a prominent example of a player whose pact with the big biotech could sour, as its PD-1 candidate seems to overlap with Opdivo. After six months of suspense, the partners say they are finally bringing the 2-year-old deal to an amicable end.

BeiGene $BGNE gets $150 million for the termination in addition to full global rights to tislelizumab. In 2017 Celgene had paid $263 million in upfront license fees to develop the PD-1 inhibitor for solid cancers in the US, Europe, Japan and the rest of the world outside Asia. It also threw in a $150 million equity investment in exchange for BeiGene handling its commercial operations — think Abraxane, Revlimid and Vidaza — in China.

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Exterior of the 1 million square foot Discovery Labs in Upper Merion, PA (PR Newswire)

Philadel­phia cham­pi­ons life sci­ences 'co-work­ing,' re­viv­ing for­mer GSK cam­pus in $500M makeover

In a boost to Philadel­phia’s thriv­ing life sci­ences scene, a for­mer Glax­o­SmithK­line cam­pus and a near­by site has been turned in­to what its de­vel­op­er calls “the largest cowork­ing ecosys­tem” for health­care com­pa­nies in the coun­try.

The Dis­cov­ery Labs, a com­pa­ny spawned by MLP Ven­tures, has se­lect­ed two lo­ca­tions in the King of Prus­sia area as the $500 mil­lion test case for its strat­e­gy of ac­quir­ing and con­vert­ing old phar­ma­ceu­ti­cal R&D fa­cil­i­ties world­wide. The sites add up to 1.64 mil­lion square feet.

Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.