Ac­tive Biotech shares crater af­ter Teva's MS drug laquin­i­mod flops (again); FDA ap­proves Foun­da­tion Med­i­cine's ge­nom­ic tu­mor di­ag­nos­tic test

→ We al­ready knew from a Phase III read­out last spring that Te­va’s big mul­ti­ple scle­ro­sis drug hope laquin­i­mod didn’t work in treat­ing re­laps­ing re­mit­ting MS. This morn­ing we find out that it’s no bet­ter in the pri­ma­ry pro­gres­sive form of the dis­ease. Te­va’s part­ner Ac­tive Biotech re­port­ed that the Phase II in PPMS — dubbed ARPEG­GIO — flunked the pri­ma­ry on brain at­ro­phy as well as a sec­ondary on time to con­firmed dis­abil­i­ty pro­gres­sion. Ac­tive’s shares $AC­TI cratered on the news, drop­ping more than 40%. Te­va has spent years in pur­suit of a laquiniq­mod ap­proval, de­spite mount­ing ev­i­dence that it couldn’t com­pete. Re­cent­ly the strug­gling out­fit launched a re­struc­tur­ing that led to the de­par­ture of R&D chief Michael Hay­den.

→  5AM Ven­tures and New En­ter­prise As­so­ci­ates led a $7.5 mil­lion seed round for Ak­ou­os, a start­up gene ther­a­py com­pa­ny fo­cused on in­ner ear ther­a­pies. “Our mis­sion is to make healthy hear­ing avail­able to all,” said Man­ny Si­mons, founder and CEO of Ak­ou­os. “Our cor­ner­stone part­ner­ship with Mass­a­chu­setts Eye and Ear, the world’s largest hear­ing re­search cen­ter, and with Lon­za, a glob­al leader in vi­ral gene and cell ther­a­py man­u­fac­tur­ing, sets a strong foun­da­tion for the im­por­tant work to be done in this emer­gent field.”

→ Al­ny­lam CEO John Maraganore is get­ting ready to start mar­ket­ing the biotech’s first RNAi drug, and he be­lieves that the com­pa­ny will have so many new drugs fol­low­ing the path to the mar­ket that Al­ny­lam won’t need reg­u­lar steep in­creas­es in prices to keep rev­enue grow­ing. As a re­sult, he says he will lim­it an­nu­al price hikes to the in­fla­tion rate, which is cur­rent­ly much, much low­er than what the rest of the in­dus­try does.

Foun­da­tion Med­i­cine $FMI has scored a key FDA OK for its com­pre­hen­sive com­pan­ion di­ag­nos­tic test for sol­id tu­mors, along with a pre­lim­i­nary de­ter­mi­na­tion that the di­ag­nos­tic will be cov­ered by the Cen­ters for Medicare and Med­ic­aid Ser­vices. De­signed to help health care pro­fes­sion­als make can­cer treat­ment de­ci­sions, Foun­da­tionOne CDx is a pro­fil­ing test that as­sess­es ge­nom­ic al­ter­ations in 324 genes known to dri­ve can­cer growth. The test al­so re­ports ge­nom­ic bio­mark­ers that can in­form the use of im­munother­a­pies. The com­pa­ny es­ti­mates that 1 in 3 pa­tients across 5 com­mon ad­vanced can­cers would match with an FDA-ap­proved ther­a­py. “To­day’s his­toric par­al­lel re­view de­ci­sion from the FDA and CMS rep­re­sents a ma­jor ad­vance­ment in per­son­al­ized can­cer care,” said CEO Troy Cox in a state­ment.

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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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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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.

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. 

Adding mar­quee in­vestors, Black­Thorn bags $76M to back an AI-dri­ven strat­e­gy for pre­ci­sion neu­ro med­i­cine

As ar­ti­fi­cial in­tel­li­gence and ma­chine learn­ing loom ever larg­er in drug dis­cov­ery and de­vel­op­ment, a biotech op­er­at­ing at the “nexus” of tech­nol­o­gy and neu­ro­sciences has cashed in with $76 mil­lion in fresh fi­nanc­ing.

The big idea at Black­Thorn Ther­a­peu­tics is to do for neu­robe­hav­ioral dis­or­ders what ge­net­i­cal­ly tar­get­ed ther­a­py has done for on­col­o­gy: Re­de­fine pa­tient pop­u­la­tions by the un­der­ly­ing bi­ol­o­gy — dys­reg­u­lat­ed brain cir­cuits, or neu­rotypes — in­stead of symp­toms, there­by find­ing the pa­tients who are most like­ly to ben­e­fit at en­roll­ment phase.

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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