Ver­tex shut­ters a PhI­II cys­tic fi­bro­sis com­bo study, shares slip

Shares of Ver­tex slid Mon­day af­ter­noon af­ter the com­pa­ny said it will scrap one of its late-stage stud­ies for a com­bi­na­tion ther­a­py for cys­tic fi­bro­sis af­ter it failed to pass muster in a fu­til­i­ty analy­sis.

Ver­tex $VRTX is halt­ing its study of VX-661 and Ka­ly­de­co (iva­caftor) in peo­ple with one copy of the F508del mu­ta­tion and one copy of a mu­ta­tion that re­sults in min­i­mal CFTR pro­tein func­tion (F508del het/min). The study stum­bled in its first phase, with­out an im­prove­ment in lung func­tion.

Shares of Ver­tex slipped 3.4% on the news.

That het/min tar­get rep­re­sent­ed the largest mar­ket op­por­tu­ni­ty for Ver­tex with VX-661, ac­cord­ing to a note from Leerink’s Ge­of­frey Porges this morn­ing. But Porges al­so called it the least like­ly of the four tri­als to suc­ceed. Even so, the re­main­ing late-stage stud­ies in the VX-661 stud­ies of­fer a sub­stan­tial mar­ket op­por­tu­ni­ty. Notes Porges:

Our analy­sis and con­ver­sa­tions with MEDA­Corp spe­cial­ists sug­gest that Vx-661/Ka­ly­de­co could gen­er­ate an im­prove­ment in ppFEV1 in the range of 4-5% in the ho­mozy­gous delF508 pa­tients, com­pared to Orkam­bi’s 2-3%, with greater ef­fects on hos­pi­tal­iza­tions and ex­ac­er­ba­tions as well; Vx-661 should be eas­i­er to take (less drug-drug in­ter­ac­tions, less tol­er­a­bil­i­ty and meta­bol­ic ef­fects) and have more ef­fect than Orkam­bi; this suc­cess should trans­late in­to in­cre­men­tal rev­enue po­ten­tial that we es­ti­mate to be in the range of ~$485mm, as­sum­ing com­pa­ra­ble pric­ing to Orkam­bi in all mar­kets.

Ver­tex Chief Med­ical Of­fi­cer Jef­frey Chodake­witz

“While we rec­og­nize that peo­ple with CF with min­i­mal func­tion mu­ta­tions have a form of the dis­ease that is par­tic­u­lar­ly dif­fi­cult to treat, we be­lieved it was im­por­tant to eval­u­ate whether a dual com­bi­na­tion of VX-661 and iva­caftor could pro­vide some ben­e­fit to these pa­tients giv­en they do not to­day have a med­i­cine to treat the cause of their dis­ease,” said Ver­tex Chief Med­ical Of­fi­cer Jef­frey Chodake­witz. “These re­sults sug­gest that a triple com­bi­na­tion reg­i­men may pro­vide this group of peo­ple with CF the best chance at ob­tain­ing a mean­ing­ful ben­e­fit and we look for­ward to be­gin­ning the first study of a next-gen­er­a­tion cor­rec­tor to­geth­er with VX-661 and iva­caftor in this group of pa­tients lat­er this year, pend­ing da­ta from our on­go­ing Phase 1 stud­ies in healthy vol­un­teers.”

VX-661 is Ver­tex’s sec­ond-gen cor­rec­tor, a suc­ces­sor to the first-gen cor­rec­tor lumacaftor, in­clud­ed in the drug com­bi­na­tion for Orkam­bi. Two third-gen cor­rec­tors, VX-152 and VX-440, have been in ear­ly-stage stud­ies as Ver­tex plumbs new ap­proach­es that can treat CF bet­ter than Orkam­bi, which has suf­fered a high dropout rate. The treat­ment tar­gets the un­der­ly­ing ge­net­ic cause of the dis­ease.

The VX-661 pro­gram in­cludes four Phase III stud­ies in mul­ti­ple dif­fer­ent groups of peo­ple with CF who have at least one copy of the F508del mu­ta­tion.

Ver­tex has a $25.4 bil­lion mar­ket cap.

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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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.

Image: Shutterstock

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.)

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.