J&J’s IL-23 star guselkum­ab grabs the spot­light in PhI­II pso­ri­a­sis show­down

J&J has long had high hopes for its an­ti-in­flam­ma­to­ry IL-23 drug guselkum­ab. And to­day in Vi­en­na they de­tailed the first round of Phase III da­ta for se­vere plaque pso­ri­a­sis that ex­plains why.

Philippe Sza­pary, Janssen

In­ves­ti­ga­tors say this drug not on­ly hand­i­ly beat a place­bo, it al­so out­stripped the megablock­buster Hu­mi­ra in key mea­sures of ef­fi­ca­cy. And that will help po­si­tion the phar­ma gi­ant as it starts to line up reg­u­la­to­ry ap­provals for a new ther­a­py that’s like­ly head­ed in­to a heav­i­ly com­pet­i­tive mar­ket.

In the VOY­AGE 1 study, J&J says that the co-pri­ma­ry end­points were met at week 16, with 85.1% of pa­tients re­ceiv­ing guselkum­ab 100 mg at weeks 0 (the start of ther­a­py) and 4 and then every eight weeks achiev­ing cleared (IGA 0) or min­i­mal dis­ease (IGA 1) com­pared with 6.9% of pa­tients re­ceiv­ing place­bo.  Near­ly three-quar­ters of pa­tients re­ceiv­ing guselkum­ab (73.3%) achieved a PASI 90 re­sponse, or near com­plete skin clear­ance, com­pared with 2.9% of pa­tients re­ceiv­ing place­bo.

That’s al­so good news for Mor­phoSys, which part­nered with J&J on the drug.

All ma­jor sec­ondary end­points in VOY­AGE 1 achieved sta­tis­ti­cal sig­nif­i­cance in com­par­isons of guselkum­ab with Hu­mi­ra (adal­i­mum­ab). Here’s the sum­ma­ry:

At week 16, fol­low­ing three in­jec­tions of guselkum­ab and ten in­jec­tions of adal­i­mum­ab, sig­nif­i­cant­ly high­er pro­por­tions of pa­tients re­ceiv­ing guselkum­ab achieved IGA 0/1 and PASI 90 (85.1 per­cent and 73.3 per­cent, re­spec­tive­ly) com­pared with pa­tients re­ceiv­ing adal­i­mum­ab (65.9 per­cent and 49.7 per­cent, re­spec­tive­ly).  At week 24, the pro­por­tion of pa­tients who achieved a PASI 90 re­sponse was sig­nif­i­cant­ly high­er in the guselkum­ab group com­pared with the adal­i­mum­ab group (80.2 per­cent vs. 53.0 per­cent, re­spec­tive­ly).  High­er lev­els of skin clear­ance among the guselkum­ab group con­tin­ued through weeks 24 and 48, with sig­nif­i­cant­ly more pa­tients re­ceiv­ing guselkum­ab achiev­ing IGA 0/1 and PASI 90, as well as mea­sures of full skin clear­ance, as in­di­cat­ed by a 100 per­cent im­prove­ment in PASI score (PASI 100) or an IGA score of 0, com­pared with adal­i­mum­ab.

J&J is look­ing to in­crease its pres­ence in a mar­ket where it’s been build­ing sales for Ste­lara while main­tain­ing an old main­stay, Rem­i­cade, an an­ti-TNF drug like Hu­mi­ra. This new drug is slat­ed to ar­rive as biosim­i­lars for both of the old drugs are an­gling to hit the mar­ket, though Ab­b­Vie has vowed to fight to the bit­ter end over Hu­mi­ra’s patent pro­tec­tion.

“I think (guselkum­ab) ac­tu­al­ly works bet­ter than what we had seen from the da­ta in Phase II,” says Philippe Sza­pary, the VP of im­munol­o­gy clin­i­cal de­vel­op­ment at Janssen, who says both the PASI 90 and clear­ance rates are high­er than the mid-stage re­sults, which may be ex­plained by an ex­tra load­ing dose used in Phase III.

That dif­fer­ence has helped es­tab­lish “good main­te­nance over time” with a rel­a­tive­ly quick on­set for many that could help demon­strate to pa­tients that they’re on the right track.

Even with da­ta from two more late-stage tri­als on track for a lat­er re­lease, Sza­pary says that J&J is on track for reg­u­la­to­ry sub­mis­sions by the end of this year.

“There’s a lot more to come out,” Sza­pary adds, as J&J lays out its case that this drug is bi­o­log­i­cal­ly avail­able to a wide range of pa­tients, from front­line use on to oth­ers.

This fast-mov­ing field has seen plen­ty of dra­ma over the past year. At one point As­traZeneca and Am­gen thought they were on track with bro­dalum­ab, then in­ci­dents of sui­ci­dal ideation drove Am­gen out and per­suad­ed As­traZeneca to sell rights to the drug to a trou­bled Valeant, which won a re­cent pan­el vote.

In the mean­time, Eli Lil­ly made it on­to the mar­ket with Taltz (ix­ek­izum­ab) with No­var­tis out with the first new con­tender, Cosen­tyx. Mer­ck is still in the clin­ic with MK-3222.

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.


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.