#ASH17: Ve­rastem's Ab­b­Vie castoff is head­ed for the FDA — even af­ter miss­ing over­all sur­vival goal

When Ve­rastem $VSTM CEO Robert For­rester high­light­ed the pos­i­tive end­point from the piv­otal study of du­velis­ib a few months ago, he didn’t want to talk about the sec­ondary da­ta points. That, he said, was be­ing saved for a fu­ture sci­en­tif­ic con­fer­ence.

And to­day at ASH you can see the added da­ta isn’t some­thing you’d want to dis­cuss much.

Robert For­rester

The drug — which hit sta­tis­ti­cal sig­nif­i­cance on pro­gres­sion-free sur­vival — flunked the key over­all sur­vival sec­ondary, fail­ing to do any bet­ter than Arz­er­ra (ofa­tu­mum­ab) in ex­tend­ing the lives of pa­tients with re­lapsed or re­frac­to­ry chron­ic lym­pho­cyt­ic leukemia/small lym­pho­cyt­ic lym­phoma.

The pos­i­tive da­ta that Ve­rastem is stick­ing with is for the me­di­an PFS: 13.3 months ver­sus 9.9 months for the con­trol. By the in­ves­ti­ga­tor as­sess­ment, there was an even bet­ter 17.6-month me­di­an PFS ver­sus 9.7 months for Arz­er­ra.

This is the da­ta that Ve­rastem is now hus­tling to the FDA, look­ing for an ap­proval to treat sec­ond-line cas­es. The p val­ue for the OS da­ta, p=0.4807, demon­strat­ed the drug group was no worse off, some­thing that was “like­ly due to oth­er avail­able ther­a­pies fol­low­ing pro­gres­sion,” ac­cord­ing to the com­pa­ny.

This was the drug that Ab­b­Vie $AB­BV paid $275 mil­lion up­front to part­ner on with In­fin­i­ty $IN­FI, but then walked away as the drug proved suc­cess­ful — but ex­treme­ly dis­ap­point­ing — in a Phase II study. For­rester picked up the rights to the drug for ex­act­ly noth­ing down as In­fin­i­ty re­struc­tured and re­fo­cused on some­thing else.

For­rester — and ex-In­fin­i­ty R&D chief Ju­lian Adams, for that mat­ter — be­lieve the drug can def­i­nite­ly win an ap­proval. And Ve­rastem be­lieves it can mar­ket the drug look­ing for peak sales of sev­er­al hun­dred mil­lion dol­lars a year.

Ve­rastem’s PR to­day out­lines the sales mod­el. Diep Le, Ve­rastem’s CMO, said:

CLL/SLL most­ly af­fects el­der­ly pa­tients and many are un­able or un­will­ing to be hos­pi­tal­ized or come in­to the clin­ic for fre­quent IV in­fu­sions. The CLL/SLL treat­ment land­scape there­fore is mov­ing away from chemother­a­pies and to­ward more tar­get­ed, prefer­ably oral reg­i­mens. While pa­tients are liv­ing longer many will be in­tol­er­ant to, or re­lapse fol­low­ing, their ini­tial ther­a­py em­pha­siz­ing the need for new op­tions. Oral du­velis­ib is the first PI3K in­hibitor to show ef­fi­ca­cy as an oral monother­a­py in a ran­dom­ized Phase 3 study in pa­tients with re­lapsed or re­frac­to­ry CLL/SLL and may of­fer an ap­peal­ing al­ter­na­tive for pa­tients who have pro­gressed or re­lapsed.

The NDA will be de­liv­ered in Q1.

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 53,000+ 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.

Search­ing for the next block­buster to fol­low Darza­lex, J&J finds a $150M an­ti-CD38 drug from part­ner Gen­mab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.


Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.


Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ 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 53,000+ biopharma pros reading Endpoints daily — and it's free.

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 53,000+ biopharma pros reading Endpoints daily — and it's free.

'We kept at it': Jef­frey Blue­stone plots late-stage come­back af­ter teplizum­ab shown to de­lay type 1 di­a­betes

Late-stage da­ta pre­sent­ed at the Amer­i­can Di­a­betes As­so­ci­a­tion an­nu­al meet­ing in 2010 pushed Eli Lil­ly to put a crimp on teplizum­ab as the phar­ma gi­ant found it un­able to re­set the clock on new­ly di­ag­nosed type 1 di­a­betes. At the same con­fer­ence but in dif­fer­ent hands nine years lat­er, the drug is mak­ing a crit­i­cal come­back by scor­ing suc­cess in an­oth­er niche: de­lay­ing the on­set of the dis­ease.

In a Phase II tri­al with 76 high-risk in­di­vid­u­als — rel­a­tives of pa­tients with type 1 di­a­betes who have di­a­betes-re­lat­ed au­toan­ti­bod­ies in their bod­ies — teplizum­ab al­most dou­bled the me­di­an time of di­ag­no­sis com­pared to place­bo (48.4 months ver­sus 24.4 months). The haz­ard ra­tio for di­ag­no­sis was 0.41 (p=0.006).

Bain’s biotech team has cre­at­ed a $1B-plus fund — with an eye to more Big Phar­ma spin­outs

One of the biggest investors to burst onto the biotech scene in recent years has re-upped with more than a billion dollars flowing into its second fund. And this next wave of bets will likely include more of the Big Pharma spinouts that highlighted their first 3 years in action.

Adam Koppel and Jeff Schwartz got the new life sciences fund at Bain Capital into gear in the spring of 2016, as they were putting together a $720 million fund with $600 million flowing in from external investors and the rest drawn from the Bain side of the equation. This time the external investors chipped in $900 million, with Bain coming in for roughly $180 million more.

They’re not done with Fund I, with plans to add a couple more deals to the 15 they’ve already posted. And once again, they’re estimating another 15 to 20 investments over a 3- to 5-year time horizon for Fund II.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,000+ biopharma pros reading Endpoints daily — and it's free.