BeiGene lines up a next-gen CT­LA-4 to com­ple­ment its PD-1 pil­lar in new deal worth up to $270M

BeiGene is jump­ing on the band­wag­on for PD-(L)1/CT­LA-4 com­bos — with a twist.

Through a de­vel­op­ment and com­mer­cial­iza­tion pact with San Diego-based BioAt­la, BeiGene is pair­ing its PD-1 drug tislelizum­ab with BA3071, a CT­LA-4 block­ing “con­di­tion­al­ly ac­tive bi­o­log­ic” that gets turned on or off based on whether it’s in­side the tu­mor mi­croen­vi­ron­ment.

Scott Smith

San Diego-based BioAt­la is re­spon­si­ble for ear­ly clin­i­cal work, while BeiGene will lead the joint ef­forts there­after to de­vel­op the com­bo and deal with reg­u­la­tors around the world. The Chi­nese biotech, which is com­mit­ting $20 mil­lion up­front and $249 mil­lion in biobucks as well as a cost-shar­ing scheme, is al­so pick­ing up all costs in Asia (ex­cept Japan), Aus­tralia and New Zealand from here on out. It has a full li­cense to com­mer­cial­iza­tion rights.

CT­LA-4 was the first check­point to hit the mar­ket, with Bris­tol-My­ers snag­ging boast­ing rights with Yer­voy. But while ef­fec­tive in un­leash­ing a T cell re­sponse and im­prov­ing sur­vival rates, their sig­nif­i­cant tox­i­c­i­ty has al­so made their use prob­lem­at­ic and thus thwart­ed their promi­nence. In re­cent years, though, a crop of biotechs has risen up on the promise to tar­get CT­LA-4 with high­er speci­fici­ty as CT­LA-4 re­mains the hottest add-on in PD-(L) com­bo stud­ies.

It is this back­drop that BioAt­la has en­tered with its plat­form, which de­signs drugs that on­ly bind to the tar­get when they sense the unique phys­i­ol­o­gy and me­tab­o­lism of a tu­mor. In ad­di­tion to an­ti­bod­ies like BA3071, the plat­form can al­so give birth to an­ti­body drug con­ju­gates, bis­pecifics and CAR-Ts.

BioAt­la pres­i­dent Scott Smith (yes, the for­mer Cel­gene COO) says the deal fits with its strat­e­gy for ad­vanc­ing CABs be­yond clin­i­cal proof-of-con­cept, as BeiGene is “a rec­og­nized leader in Chi­na-in­clu­sive glob­al clin­i­cal de­vel­op­ment.”

The com­pa­ny is prep­ping an im­mi­nent first IND fil­ing for both the BA3071 monother­a­py and the com­bo with tislelizum­ab, with a Phase I/II ex­pect­ed to start in the sec­ond half of 2019.

For BeiGene, the deal marks an­oth­er ad­di­tion to the broad pipeline around tislelizum­ab, which spans a dozen so­lo in­di­ca­tions as well as ear­ly com­bo stud­ies with its own PARP and BTK in­hibitors.


Im­age: BeiGene

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.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 52,900+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 52,900+ biopharma pros reading Endpoints daily — and it's free.

News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

J&J gains an en­thu­si­as­tic en­dorse­ment from Pres­i­dent Don­ald Trump for their big new drug Spra­va­to

Pres­i­dent Don­ald Trump has lit­tle love for Big Phar­ma, but there’s at least one new drug that just hit the mar­ket which he is en­am­ored with.

Trump, ev­i­dent­ly, has been read­ing up on J&J’s new an­ti-de­pres­sion drug, Spra­va­to. And the pres­i­dent — who of­ten likes to break out in­to a full-throat­ed at­tack on greedy drug­mak­ers — ap­par­ent­ly en­thused about the ther­a­py in a meet­ing with of­fi­cials of Vet­er­ans Af­fairs, which has long grap­pled with de­pres­sion among vet­er­ans.

Savara shares are crushed as PhI­II tri­al flunks pri­ma­ry, key sec­on­daries — but they can’t stop be­liev­ing

In­vestors are in no mood to hear biotechs tout the suc­cess of a “key” sec­ondary end­point when the piv­otal Phase III flunks the pri­ma­ry goal. Just ask Savara. 

The Texas biotech $SVRA went look­ing for a sil­ver lin­ing as com­pa­ny ex­ecs blunt­ly con­ced­ed that Mol­gradex, an in­haled for­mu­la­tion of re­com­bi­nant hu­man gran­u­lo­cyte-macrophage colony-stim­u­lat­ing fac­tor (GM-CSF), failed to spur sig­nif­i­cant­ly im­proved treat­ment out­comes for pa­tients with a rare res­pi­ra­to­ry dis­ease called au­toim­mune pul­monary alve­o­lar pro­teinosis, or aPAP.

As an­oth­er an­tibi­otics biotech sinks in­to a cri­sis, warn­ings of a sec­tor ‘col­lapse’

Another antibiotics company is scrambling to survive today, forcing the company’s founding CEO to exit in a reorganization that eliminates its research capabilities as the survivors look to improve on minuscule sales of their newly approved treatment. And the news — on top of an alarming series of failures — spurred at least one figure in the field to warn of a looming collapse of the antimicrobial resistance research field.

Endpoints News

Basic subscription required

Unlock this story instantly and join 52,900+ biopharma pros reading Endpoints daily — and it's free.

Neil Woodford, Woodford Investment Management via YouTube

Un­der siege, in­vest­ment man­ag­er Wood­ford faces an­oth­er in­vest­ment shock

Em­bat­tled UK fund man­ag­er Neil Wood­ford — who has con­tro­ver­sial­ly blocked in­vestors from pulling out from his flag­ship fund to stem the blood­let­ting, af­ter a slew of dis­ap­point­ed in­vestors fled fol­low­ing a se­ries of sour bets — is now pay­ing the price for his ac­tions via an in­vestor ex­o­dus on an­oth­er fund.

Har­g­reaves Lans­down, which has in the past sold and pro­mot­ed the Wood­ford funds via its re­tail in­vest­ment plat­form, has re­port­ed­ly with­drawn £45 mil­lion — its en­tire po­si­tion — from the in­vest­ment man­ag­er’s In­come Fo­cus Fund.

Fol­low­ing CAR-T pi­o­neer­s' foot­steps, Tes­sa launch­es Chi­na JV in $120M deal

These days just about every biotech se­ri­ous about glob­al de­vel­op­ment — and not just com­mer­cial­iza­tion — has a Chi­na strat­e­gy. Tes­sa Ther­a­peu­tics, a Bay­lor as­so­ci­at­ed out­fit based out of Sin­ga­pore, is no ex­cep­tion.

Tak­ing a page out of the CAR-T pi­o­neers’ play­book, Tes­sa is es­tab­lish­ing a joint ven­ture with Chi­na-Sin­ga­pore Guangzhou Knowl­edge City, which is ini­tial­ly putting down $40 mil­lion for a 13% stake with $40 mil­lion more to come in a sec­ond stage. The biotech, which now re­tains an 87% con­trol, is al­so rolling out its own con­tri­bu­tions in two phas­es, start­ing with $20 mil­lion and all its tech­nol­o­gy li­cense rights for Chi­na.