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.

“(T)here is cur­rent­ly no ap­proved ther­a­py for pa­tients who progress on CAR-Ts,” not­ed Re­gen­eron’s Is­rael Lowy. “Our po­ten­tial­ly reg­is­tra­tional Phase 2 pro­gram is ini­ti­at­ing this month and will proac­tive­ly eval­u­ate ac­tive REGN1979 dos­es in in­do­lent and ag­gres­sive non-Hodgkin lym­phoma.”

Not­ed Baird’s Bri­an Sko­r­ney:

The fol­lic­u­lar lym­phoma da­ta don’t look as stel­lar as the pri­or da­ta cut but the DL­B­CL da­ta im­prove. We think this bis­pe­cif­ic pro­gram looks like a strong next-gen B-cell lym­phoma ther­a­py that could yield a fair­ly sig­nif­i­cant mar­ket op­por­tu­ni­ty.

Ar­Qule rights the ship with more proof-of-con­cept da­ta

Af­ter a less than flat­ter­ing show­ing at the last EHA, Ar­Qule is re­turn­ing tri­umphant.

Hom­ing in on the BTK-C481AS mu­ta­tion, Ar­Qule high­light­ed da­ta from a co­hort of six chron­ic lym­pho­cyt­ic leukemia pa­tients who have ei­ther not re­spond­ed to treat­ment or re­lapsed. Hav­ing re­ceived the 65 mg dose, four of them ex­pe­ri­enced a par­tial re­sponse — a sig­nif­i­cant feat giv­en they have al­ready ex­haust­ed avail­able ther­a­pies, the com­pa­ny said. Shares $AR­QL surged 47% pre-mar­ket to $9.26.

Bri­an Schwartz Ar­Qule

“We are now fo­cused on fi­nal­iz­ing the rec­om­mend­ed phase 2 dose and plan­ning for the ex­pan­sion of our clin­i­cal ef­forts with ARQ 531 in­to lat­er stage tri­als across mul­ti­ple in­di­ca­tions as a sin­gle agent and as a com­bi­na­tion ther­a­py,” CMO Bri­an Schwartz said in a pre­pared state­ment.

In ad­di­tion, in­ves­ti­ga­tors al­so not­ed a par­tial re­sponse for a pa­tient with Richter’s Trans­for­ma­tion who had pro­gressed on ibru­ti­nib and R-CHOP.

Lit­tle Ory­zon shows it’s do­ing al­right with AML drug on its own

Ory­zon has seen the first glimpse of ef­fi­ca­cy da­ta for its LSD1 in­hibitor, iadadem­stat when com­bined with chemother­a­py in el­der­ly pa­tients with acute myeloid leukemia. Clin­i­cal re­spons­es were ob­served rapid­ly with a me­di­an time of 1.5 months, and of the 5 evalu­able pa­tients, 3 achieved com­plete re­mis­sions (al­beit with in­com­plete hema­to­log­ic re­cov­ery) while 1 had a par­tial re­mis­sion.

The tri­al is be­ing con­duct­ed in Spain, where Ory­zon was found­ed. The com­pa­ny plans to re­cruit up to 36 pa­tients to set the stage for the broad­er ap­pli­ca­tion of iadadem­stat —  once part­nered but lat­er dumped by Roche — in oth­er leukemias.

BerGen­Bio spot­lights AML drug’s po­ten­tial in pa­tients un­fit for in­tense chemo

Nor­way’s BerGen­Bio is spot­light­ing 6 re­spons­es out of 14 evalu­able pa­tients on its drug, which is de­signed to pair with low-in­ten­si­ty chemother­a­py for acute myeloid leukemia pa­tients who can­not stand in­tense chemother­a­py. Their AXL in­hibitor be­m­cen­tinib helped push 4 pa­tients in­to com­plete re­mis­sion (with or with­out in­com­plete hema­to­log­ic re­cov­ery) and 2 oth­ers in­to par­tial re­mis­sion.

“Be­m­cen­tinib in com­bi­na­tion with LDAC re­sult­ed in a sub­stan­tial­ly high­er ORR than ex­pect­ed for sin­gle-agent cy­tara­bine and clear­ly war­rant fur­ther in­ves­ti­ga­tion of be­m­cen­tinib in an ex­pan­sion co­hort of AML pa­tients un­fit for in­ten­sive chemother­a­py,” pro­fes­sor Bjørn Tore Gjert­sen of Hauke­land Uni­ver­si­ty Hos­pi­tal and the Uni­ver­si­ty of Bergen said. “Our sig­nal cell-lev­el bio­mark­er analy­sis of AML blasts from pa­tients in­di­cates a sub­stan­tial ef­fect on cell sig­nal­ing and rep­re­sents a po­ten­tial new bio­mark­er strat­e­gy.”

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.


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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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.

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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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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.

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'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.

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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.