Key pos­i­tive da­ta on month­ly HIV reg­i­men of­fer hope for pa­tients sad­dled with stan­dard dai­ly pills

HIV pa­tients have much to cheer about this week. Af­ter re­ports that a sec­ond HIV pa­tient has ex­pe­ri­enced sus­tained re­mis­sion fol­low­ing a bone mar­row trans­plant, a pair of drug­mak­ers on Thurs­day post­ed piv­otal da­ta that showed their month­ly in­jectable treat­ments worked as well as the stan­dard dai­ly three-drug cock­tail in sup­press­ing the virus that caus­es AIDS.

The in­jectable reg­i­men com­pris­ing two drugs — John­son & John­son’s $JNJ rilpivirine (sold as Edu­rant) and Vi­iV Health­care’s ex­per­i­men­tal treat­ment cabote­gravir — was test­ed in a two late-stage stud­ies in more than 1180 pa­tients al­to­geth­er. The to­tal dataset will be used to sub­mit ap­pli­ca­tions to mar­ket the in­jectable for­mu­la­tion to reg­u­la­tors lat­er this year, Vi­iV said.

The best way to thwart the spread of HIV is to use con­doms. But for those al­ready af­flict­ed with the virus, an­ti­retro­vi­ral ther­a­py must be tak­en every sin­gle day to sup­press the virus or un­bri­dled repli­ca­tion can over­whelm the im­mune sys­tem and even­tu­al­ly cause AIDS.

Apart from dras­ti­cal­ly re­duc­ing the num­ber of times treat­ment must be ad­min­is­tered to 12 from 365, the in­jectable reg­i­men — if ap­proved — could in cer­tain parts of the world re­duce the stig­ma of pick­ing up HIV pre­scrip­tions at phar­ma­cies.

Dai­ly pills al­so rack up ex­pen­sive bills in the Unit­ed States, with month­ly ex­pen­di­ture on treat­ment hit­ting thou­sands per month — de­pend­ing on the reg­i­men, in­sur­ance provider and re­bates/dis­counts. End­points News has asked the man­u­fac­tur­ers about their pric­ing plans for the in­jectable month­ly reg­i­men, and is await­ing com­ment.

Ac­cord­ing to the WHO es­ti­mates, 36.9 mil­lion peo­ple were liv­ing with HIV glob­al­ly in 2017.

In­ject­ed every four weeks, 48-week da­ta on the two-drug for­mu­la­tion from both stud­ies — FLAIR and AT­LAS — were pos­i­tive, with about 1% of pa­tients from ei­ther study de­vel­op­ing drug re­sis­tance, sim­i­lar to those who take stan­dard dai­ly ther­a­py, Vi­iV said.

Pos­i­tive AT­LAS da­ta was pub­lished last Au­gust. On Thurs­day, the two drug­mak­ers broke out the de­tailed num­bers for both AT­LAS and FLAIR.

The AT­LAS study found vi­ro­log­ic sup­pres­sion rates at week 48 were sim­i­lar be­tween treat­ment arms — (cabote­gravir + rilpivirine: 285/308 [92.5%]) ver­sus cur­rent an­ti­retro­vi­ral ther­a­py  (294/308 [95.5%]). In the FLAIR study vi­ro­log­ic sup­pres­sion rates at week 48 were al­so sim­i­lar be­tween treat­ment arms — (cabote­gravir + rilpivirine: 265/283 [93.6%]) ver­sus the stan­dard Tri­umeq reg­i­men: (264/283 [93.3%]).

Vi­iV — which was es­tab­lished in 2009 by British drug­mak­er GSK $GSK in part­ner­ship with Pfiz­er $PFE — is al­so test­ing the two-drug in­jectable ver­sion ad­min­is­tered every two months in the AT­LAS-2M study. It al­ready has an ar­se­nal of 13 ap­proved an­ti­retro­vi­ral med­i­cines.

GSK’s ap­proach to grab­bing mar­ket share from Gilead $GILD  — which cur­rent­ly dom­i­nates the HIV mar­ket that is es­ti­mat­ed to hit $22.5 bil­lion by 2025 — is to con­vince reg­u­la­tors, doc­tors and pa­tients to adopt its two-drug reg­i­mens, ver­sus Gilead’s triple-drug cock­tails, that could po­ten­tial­ly re­sult in few­er tox­ic side-ef­fects, re­duce dos­ing fre­quen­cy, and be more cost-ef­fec­tive — al­though crit­ics sug­gest this may not be the most ef­fec­tive strat­e­gy be­cause the virus will on­ly have to fight against two drugs which could cul­mi­nate in drug re­sis­tance. But if the in­jectable month­ly reg­i­men makes it to the mar­ket, Gilead’s crown may be more vul­ner­a­ble than ever.

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.

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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.

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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