Bol­stered by promis­ing PhI­Ib snap­shot, uniQure plays catch-up with Spark in the race to a he­mo­phil­ia B gene ther­a­py

The piv­otal lead switch that uniQure ex­e­cut­ed late last year ap­pears to have paid off, as the gene ther­a­py pi­o­neer of­fers some pos­i­tive, if ear­ly, num­bers for its tweaked he­mo­phil­ia B treat­ment.

Robert Gut

Among three pa­tients treat­ed with AMT-061 in the dose-con­fir­ma­tion study, re­searchers re­port­ed a mean fac­tor IX lev­el that rep­re­sent­ed 31% of nor­mal lev­els at six weeks, ex­ceed­ing the 12% com­mon­ly re­gard­ed as suf­fi­cient to sub­stan­tial­ly re­duce spon­ta­neous bleeds. A quick break­down by in­di­vid­ual showed FIX ac­tiv­i­ty in the first pa­tient was 37% of nor­mal 10 weeks af­ter ad­min­is­tra­tion; in the sec­ond pa­tient, 23% at week 8; and the third, 30% at week 6.

Hap­py to hear that none of the pa­tients re­ceived fac­tor in­fu­sions, ex­pe­ri­enced re­port­ed bleed­ing events or re­quired im­muno­sup­pres­sion, in­vestors sent the stock up $QURE 39%.

Ed­ward Nash

It’s al­so mu­sic to the ears of some sup­port­ive an­a­lysts, who are call­ing the re­sults “very good” and “a clear pos­i­tive” and large­ly agree­ing with ex­ecs that a “mild, asymp­to­matic and tran­sient in­crease in liv­er en­zyme lev­els” ob­served in the tri­al was not a cause for con­cern.

The re­sults af­firm uniQure’s com­pet­i­tive po­si­tion with Spark Ther­a­peu­tics, which is al­so de­vel­op­ing a he­mo­phil­ia B ther­a­py to fol­low up its his­toric reti­na eye dis­ease treat­ment.

Ed­ward Nash of Sun­Trust notes:

As a ref­er­ence, SPK-9001 from Spark showed in a Phase I/II study that the range of FIX ac­tiv­i­ty lev­els was be­tween 14% and 68% at Week 6 and be­tween 16% and 78% at Week 8.

Joseph Schwartz

An­oth­er de­tail that may sep­a­rate uniQure from Spark, Leerink’s Joseph Schwartz points out, is that AMT-061 may be able to tar­get a broad­er spec­trum of pa­tients. Two of the three pa­tients in uniQure’s Phase IIb tri­al were en­rolled af­ter pre­vi­ous­ly screen-fail­ing an­oth­er gene ther­a­py study due to pre-ex­ist­ing neu­tral­iz­ing an­ti­bod­ies to a dif­fer­ent AAV vec­tor. In fact, ex­ecs were so con­fi­dent about the AAV5 vec­tor they have vowed not to ex­clude any pa­tients on the pa­tients of pre-ex­ist­ing neu­tral­iz­ing an­ti­bod­ies.

Am­s­ter­dam- and Lex­ing­ton, MA-based uniQure re­placed its lead pro­gram, AMT-060, with AMT-061 last Oc­to­ber, claim­ing that a new gene vari­ant called FIX-Pad­ua in­tro­duced in the tweaked as­set will boost FIX ac­tiv­i­ty while keep­ing the same de­liv­ery tech and man­u­fac­tur­ing process.

“Based on the long-term FIX da­ta from our Phase I/II tri­al of AMT-060, where we saw FIX ac­tiv­i­ty lev­els con­tin­ue to in­crease be­yond the lev­els achieved at six to ten weeks, we are hope­ful that we will con­tin­ue to see sim­i­lar trends in these pa­tients,” said CMO Robert Gut in a state­ment.

Catch­ing up with Spark and its Big Phar­ma al­lies at Pfiz­er, uniQure plans to be­gin dos­ing for a Phase III study in Q1 2019.

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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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.

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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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.

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.