Ab­b­Vie nabs pe­di­atric OK for hep C drug Mavyret; Glen­mark launch­es cheap­er SGLT2 in­hibitor in In­dia

→ Build­ing off a sur­prise suc­cess with Mavyret in adults, Ab­b­Vie has nabbed an FDA ap­proval to mar­ket the he­pati­tis C drug for chil­dren ages 12 to 17. The drug — a com­bi­na­tion of gle­capre­vir and pi­brentasvir — racked up $3.438 bil­lion in net rev­enue for 2018, far ex­ceed­ing peak sales ex­pec­ta­tions. The OK was based on a tri­al of 47 HCV pa­tients with geno­types 1-4 sug­gest­ing a 100% cure rate, though it cov­ers all six geno­types.

→ In­dia’s Glen­mark on Tues­day launched a new SGLT2 in­hibitor called Re­mogliflozin. It is the first-ever launch of the di­a­betes drug, which was part­ly de­vel­oped by GSK, and it will be sold in In­dia. SGLT2 in­hibitors, such as Forx­i­ga, In­vokana and Jar­diance, work by pre­vent­ing the kid­neys from re­ab­sorb­ing glu­cose back in­to the blood. Re­mogliflozin, which has shown to be com­pa­ra­bly ef­fi­ca­cious and safe as Forx­i­ga in a late-stage head-to-head study, will be sold at a price of 25 ru­pees (36 cents) per day, a dis­count of more than 50% to ex­ist­ing SGLT2 in­hibitors on the In­di­an mar­ket, a com­pa­ny spokesper­son told End­points News.

→ A day af­ter the FDA added boxed warn­ings to zolpi­dem and oth­er in­som­nia med­i­cines for seem­ing to cause dan­ger­ous be­hav­iors, Ei­sai is go­ing all in on a sleep-wake reg­u­la­tion agent that it says com­pares fa­vor­ably to zolpi­dem. The Japan­ese com­pa­ny was col­lab­o­rat­ing with Pur­due Phar­ma on lem­borex­ant, but now says it will buy out all world­wide de­vel­op­ment and com­mer­cial­iza­tion rights. “Pur­due’s busi­ness pri­or­i­ties have shift­ed since this col­lab­o­ra­tion was ini­ti­at­ed,” CEO Craig Lan­dau said, adding they will now fo­cus on on­col­o­gy, non-opi­oid pain and oth­er CNS pro­grams. Oth­er po­ten­tial in­di­ca­tions in­clude ir­reg­u­lar sleep-wake rhythm dis­or­der in pa­tients with Alzheimer’s Dis­ease.

→ We fi­nal­ly know ex­act­ly how many jobs Mer­ri­mack Phar­ma’s $MACK lat­est pro­gram ter­mi­na­tion cost. In an SEC fil­ing, the com­pa­ny said 13 staffers now re­main, around half of the 27 who sur­vived the last round of lay­offs in No­vem­ber. A few weeks ago Mer­ri­mack an­nounced it was aban­don­ing its ex­per­i­men­tal drug, MM-310, af­ter an ear­ly-stage study in­volv­ing pa­tients with sol­id tu­mors showed that treat­ment with the drug was cu­mu­la­tive­ly tox­ic to pa­tients, de­spite tri­al pro­to­col amend­ments. Its pipeline now con­sists of two pre­clin­i­cal pro­grams.

→ As Mo­tif Bio works to re­solve the FDA’s con­cerns about the safe­ty of iclaprim, it’s dou­bling down on the an­tibi­ot­ic with a new com­bo pro­gram that broad­ens its ap­pli­ca­tion be­yond skin in­fec­tions. The UK biotech is part­ner­ing up with Scot­land’s Lamel­lar Bio­med­ical to test iclaprim along­side LMS-611 for cys­tic fi­bro­sis lung in­fec­tions. Mo­tif Bio says the two drugs have been grant­ed or­phan des­ig­na­tion for Staphy­lo­coc­cus au­reus pneu­mo­nia in pa­tients with CF in the US and Eu­rope, re­spec­tive­ly.

Na­tal­ie Grover con­tributed re­port­ing.


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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Ab­b­Vie touts new da­ta for Hu­mi­ra suc­ces­sor; Gilead inks dis­cov­ery deal

→ Ab­b­Vie is tout­ing new pos­i­tive da­ta com­par­ing their ag­ing block­buster Hu­mi­ra with their hoped-for block­buster upadac­i­tinib. Over 48 weeks a larg­er pro­por­tion of pa­tients tak­ing the ex­per­i­men­tal drug ex­pe­ri­enced clin­i­cal re­mis­sion than in the con­trol arm with Hu­mi­ra. Their drug brought in $20 bil­lion last year, top­ping the scales in the num­ber 1 slot.

→ Gilead has turned to Van­cou­ver-based Ab­Cellera for its lat­est dis­cov­ery deal. Ab­Cellera will use its know-how in “sin­gle-cell screen­ing of nat­ur­al im­mune sources” to find an­ti­body can­di­dates for Gilead to pur­sue in the in­fec­tious dis­ease field. The deal in­cludes an up­front and mile­stones.

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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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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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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Turns out, Rudy Tanzi did­n't see much of a sto­ry about a hid­den link be­tween En­brel and Alzheimer's ei­ther

The Wash­ing­ton Post man­aged to whip up the quick­est in­dus­try con­sen­sus I’ve ever seen that one of its re­porters was pur­vey­ing overblown non­sense with a sto­ry that Pfiz­er was sit­ting on da­ta sug­gest­ing that En­brel could be an ef­fec­tive treat­ment for Alzheimer’s. 

In cov­er­ing that bit of an­ti-Big Phar­ma fan­ta­sy — there are lots of rea­sons to go af­ter phar­ma, but this piece was lu­di­crous — I not­ed com­ments in the sto­ry from some promi­nent peo­ple in the field crit­i­ciz­ing Pfiz­er for not pub­lish­ing the da­ta. I sin­gled out Rudy Tanzi at Har­vard and then ap­plied some added crit­i­cism for the things he’s done to hype — in my opin­ion — high­ly ques­tion­able as­sump­tions. You can see it in the link.