Blood­ied Cel­gene posts promis­ing ozan­i­mod PhI­II MS da­ta, but is it re­al­ly a $6 bil­lion drug?

Cel­gene pumped out its Phase III da­ta on its block­buster con­tender ozan­i­mod over the week­end, of­fer­ing some en­cour­ag­ing com­par­isons with Avonex in treat­ing pa­tients with re­laps­ing mul­ti­ple scle­ro­sis. But some of these num­bers will like­ly trig­ger some sec­ond guess­ing about the drug’s peak sales abil­i­ty af­ter Cel­gene paid $7.2 bil­lion to get its hands on the drug.

In­ves­ti­ga­tors re­cruit­ed 1,346 pa­tients for the SUN­BEAM tri­al, post­ing sta­tis­ti­cal­ly sig­nif­i­cant scores for the an­nu­al­ized re­lapse rate. But in a pooled analy­sis of SUN­BEAM and RA­DI­ANCE Part B stud­ies, their drug did not hit the goal for the time to 3-month con­firmed dis­abil­i­ty pro­gres­sion.

An­a­lysts have been do­ing some cross-tri­al com­par­isons with No­var­tis’ Gilenya, which is com­ing off patent pro­tec­tion at the end of 2019, and con­clud­ed that Cel­gene looks like it could leap out on­to the mar­ket with a sim­i­lar ef­fi­ca­cy pro­file but bet­ter safe­ty fea­tures. If so, Cel­gene could gain a sig­nif­i­cant edge in the ri­val­ry to come for this drug, which Cel­gene has said is worth $4 bil­lion to $6 bil­lion a year in peak sales.

Ge­of­frey Porges

Sev­er­al an­a­lysts were will­ing to give Cel­gene — long a dar­ling of the biotech in­vestor crowd — a thumb’s up for the da­ta, which the big biotech bad­ly needs af­ter its mis­steps over the last two weeks, cut­ting longterm fore­casts and trig­ger­ing an 18% drop in the share price $CELG. Just be­fore that, Cel­gene was forced to con­cede that its pro­gram for mon­gersen had im­plod­ed in Phase III.

“The posters and pre­sen­ta­tions sug­gest that ozan­i­mod in­deed has a dif­fer­en­ti­at­ed safe­ty and ef­fi­ca­cy pro­file to oth­er wide­ly used med­i­cines in the MS cat­e­go­ry,” not­ed Leerink’s Ge­of­frey Porges, who says an OK in MS along with in­flam­ma­to­ry bow­el dis­ease and more could cre­ate $2.9 bil­lion in sales by 2022. “This prod­uct, along with lus­pa­ter­cept, has be­come the stan­dard bear­er for Cel­gene’s late stage pipeline, and in our view the da­ta at the meet­ing jus­ti­fy some re­cov­ery in sen­ti­ment about the com­pa­ny’s port­fo­lio and out­look.”

Sun­Trust’s Yatin Sune­ja did some ba­sic math and came out up­beat about Cel­gene’s mar­ket prospects.

Yatin Sune­ja

Ef­fi­ca­cy high­lights (pri­mar­i­ly fo­cused on ozan­i­mod 1mg) in­clud­ed (1) 48% ARR re­duc­tion vs. Avonex (pri­ma­ry end­point; broad­ly in-line with Gilenya’s 52% re­duc­tion in TRANS­FORMS, in our view), (2) T1 GdE le­sion re­duc­tion at month 12 of 63% vs. Avonex (which we be­lieve is bet­ter than Gilenya’s 55% re­duc­tion in TRANS­FORMS) and (3) 33% re­duc­tion in whole brain vol­ume loss vs. Avonex (at least in-line with Gilenya’s 32% re­duc­tion in TRANS­FORMS). While the tri­al was not pow­ered for 3-month con­firmed dis­abil­i­ty pro­gres­sion (an ex­plorato­ry end­point), there was a 31% re­duc­tion vs. Avonex.

Baird’s Bri­an Sko­r­ney has been fol­low­ing the pro­gram, and I asked for his take on the re­sults. His re­sponse:

I think the ozan­i­mod da­ta had no sur­pris­es, which giv­en the last two weeks of Cel­gene sur­pris­es is a rel­a­tive­ly good thing. I think every­one ex­pect­ed an ef­fi­ca­cy pro­file that looks on par with Gilenya and a safe­ty pro­file that looks bet­ter than Gilenya and that is what we saw. It def­i­nite­ly ap­pears to have a bet­ter car­diac pro­file. We will see how the FDA la­bels around that but with­out ini­tial dose mon­i­tor­ing, it seems like a more com­pelling oral to start pa­tients on than Gilenya. Every­thing is a cross tri­al com­par­i­son but the per­spec­tive is that Avonex is re­al­ly safe and ozan­i­mod seemed to match it pret­ty nice­ly, so com­pared to Gilenya, it will be per­ceived as look­ing safe. Things that peo­ple are con­cerned about with Gilenya, like liv­er tox and in­fec­tion risk, all look bet­ter here. I don’t think this is a par­a­digm shift­ing drug though, the way Ocre­vus  ap­pears to be.

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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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.

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.

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).