FDA fol­lows through with a ground­break­ing OK for Yescar­ta, Gilead­'s new CAR-T break­through

Just two weeks af­ter Gilead closed on its $12 bil­lion Kite buy­out, the FDA has fol­lowed through with a ground­break­ing ap­proval of Yescar­ta (axi-cel), putting its CAR-T drug neck-and-neck with No­var­tis’ pi­o­neer Kym­ri­ah as the two drugs are prepped for a launch.

In a vir­tu­al heart­beat, Gilead used its con­sid­er­able cash re­serves to reach a deal to buy out Kite and axi-cel 6 weeks ago, ac­quir­ing its new­ly ap­proved CAR-T and all the next-gen tech­nol­o­gy now in the works. The ac­qui­si­tion made Gilead an overnight leader in CAR-T. While beat­en to a his­toric first FDA ap­proval for a CAR-T by an ag­gres­sive group at No­var­tis — ini­tial­ly green-light­ed at the end of Au­gust for pe­di­atric and young adult pa­tients with a form of acute lym­phoblas­tic leukemia — Gilead is re­tain­ing most of the Kite gang and hon­ing its man­u­fac­tur­ing ef­fort, shav­ing off the time it takes to turn around these per­son­al­ized ther­a­pies.

In clas­sic ag­gres­sive form, Gilead came out gun­ning, of­fer­ing the ther­a­py at a price of $373,000 — $102,000 less than its ri­val at No­var­tis. On the oth­er hand, No­var­tis of­fered a val­ue-based deal to a num­ber of pay­ers in which they on­ly get re­im­burse­ment for pa­tients who re­spond to ther­a­py, evening the play­ing field.

Once again, FDA com­mis­sion­er Scott Got­tlieb took the hon­ors in con­grat­u­lat­ing the de­vel­op­ers, as he did when No­var­tis came out in front. And this time, he promised to lend the agency’s help for the rest of the bur­geon­ing cell ther­a­py field. Said Got­tlieb:

“To­day marks an­oth­er mile­stone in the de­vel­op­ment of a whole new sci­en­tif­ic par­a­digm for the treat­ment of se­ri­ous dis­eases. In just sev­er­al decades, gene ther­a­py has gone from be­ing a promis­ing con­cept to a prac­ti­cal so­lu­tion to dead­ly and large­ly un­treat­able forms of can­cer. This ap­proval demon­strates the con­tin­ued mo­men­tum of this promis­ing new area of med­i­cine and we’re com­mit­ted to sup­port­ing and help­ing ex­pe­dite the de­vel­op­ment of these prod­ucts. We will soon re­lease a com­pre­hen­sive pol­i­cy to ad­dress how we plan to sup­port the de­vel­op­ment of cell-based re­gen­er­a­tive med­i­cine. That pol­i­cy will al­so clar­i­fy how we will ap­ply our ex­pe­dit­ed pro­grams to break­through prod­ucts that use CAR-T cells and oth­er gene ther­a­pies. We re­main com­mit­ted to sup­port­ing the ef­fi­cient de­vel­op­ment of safe and ef­fec­tive treat­ments that lever­age these new sci­en­tif­ic plat­forms.”

The CD19 T cell im­munother­a­py — which reengi­neers pa­tient cells in­to a po­tent can­cer ther­a­py — has been ap­proved for adult pa­tients with re­lapsed or re­frac­to­ry large B-cell lym­phoma af­ter two or more lines of sys­temic ther­a­py. That la­bel in­cludes dif­fuse large B-cell lym­phoma (DL­B­CL), pri­ma­ry me­di­asti­nal large B-cell lym­phoma, high-grade B-cell lym­phoma, and DL­B­CL aris­ing from fol­lic­u­lar lym­phoma.

Alessan­dro Ri­va

The R&D work will now be led by Alessan­dro Ri­va, a for­mer No­var­tis sci­en­tist pro­mot­ed yes­ter­day to ex­ec­u­tive vice pres­i­dent in charge of on­col­o­gy R&D, with a seat on the lead­er­ship team.

Left be­hind in the race to CAR-T dom­i­nance, for now at least, is Juno Ther­a­peu­tics. Its ri­val drug was mired down by a lethal tox­i­c­i­ty is­sue that killed 5 pa­tients, forc­ing the biotech to switch over to its num­ber 2 pro­gram.

No­var­tis man­aged to sur­prise quite a few an­a­lysts by peg­ging Kym­ri­ah at $475,000, which is sig­nif­i­cant­ly low­er than the $600,000 max­i­mum pro­vid­ed in some of the spreads. In a sto­ry we pub­lished yes­ter­day, how­ev­er, a num­ber of ex­perts said the full cost of ther­a­py may well end up at $1 mil­lion to $1.5 mil­lion.

The da­ta, though, are jaw drop­ping.

The FDA’s ap­proval comes through on the da­ta for ZU­MA-1, which demon­strat­ed that 72% of the pa­tients in­volved had an ob­jec­tive re­sponse rate to the ther­a­py. And 51% demon­strat­ed a com­plete re­mis­sion, with no sign of the can­cer left.

In a re­cent farewell let­ter to the staff, Kite CEO Arie Bellde­grun summed it up by writ­ing:

In a span of just a few short years, we grew from few­er than 10 em­ploy­ees to al­most 700. The com­pa­ny’s val­ue in­creased 2300% from the time of our IPO to near­ly $12 bil­lion with the ac­qui­si­tion by Gilead Sci­ences. Our clos­ing $180 per share price rep­re­sents not just a 960% ap­pre­ci­a­tion from the IPO price of $17 per share, but the largest ever pre-com­mer­cial bio­phar­ma ac­qui­si­tion.

Now Gilead can see if it’s ag­gres­sive team can make the most of the block­buster peak sales es­ti­mates that have yet to be climbed.

To­day, Bellde­grun had this to say:

“The FDA ap­proval of Yescar­ta is a land­mark for pa­tients with re­lapsed or re­frac­to­ry large B-cell lym­phoma. This ap­proval would not have been pos­si­ble with­out the coura­geous com­mit­ment of pa­tients and clin­i­cians, as well as the on­go­ing ded­i­ca­tion of Kite’s em­ploy­ees. We must al­so rec­og­nize the FDA for their abil­i­ty to em­brace and sup­port trans­for­ma­tion­al new tech­nolo­gies that treat life-threat­en­ing ill­ness­es. We be­lieve this is on­ly the be­gin­ning for CAR T ther­a­pies.”

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.

Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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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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