Take­da has stum­bled again in its quest to build up the block­buster po­ten­tial of its mul­ti­ple myelo­ma drug Nin­laro. The phar­ma gi­ant says the drug com­bined with dex­am­etha­sone failed to pro­vide sig­nif­i­cant help for pa­tients with rare cas­es of sys­temic light-chain amy­loi­do­sis, so now they’re scrap­ping the en­tire Phase III pro­gram.

Philip Row­lands LinkedIn

The drug failed on the first pri­ma­ry end­point on hema­to­log­i­cal re­spons­es, and re­searchers are throw­ing in the tow­el on or­gan de­te­ri­o­ra­tion and death, the sec­ond pri­ma­ry end­point, with lit­tle ex­pec­ta­tion of suc­cess. The drug com­bo with dex was com­pared against a physi­cian’s choice se­lec­tion of dex­am­etha­sone plus ei­ther mel­pha­lan, cy­clophos­phamide, thalido­mide, or lenalido­mide; or dex­am­etha­sone alone.

We won’t have the num­bers un­til a lat­er sci­en­tif­ic con­fer­ence, but Take­da on­col­o­gy chief Phil Row­lands said the tri­al was “one of the largest” mount­ed in this field. Pa­tients suf­fer­ing from amy­loi­do­sis ex­pe­ri­ence an ac­cu­mu­la­tion of amy­loid in or­gans, which can trig­ger even­tu­al or­gan fail­ure. 

Take­da has had high hopes for Nin­laro since its ap­proval in 2015. But it al­so faces sig­nif­i­cant com­pe­ti­tion with a grow­ing va­ri­ety of drugs in the same mar­ket.

This lat­est set­back on Nin­laro comes fast on the heels of an­oth­er stum­ble ear­li­er in the year, when the com­pa­ny said that it was with­draw­ing their ap­pli­ca­tion to sell the drug as a main­te­nance ther­a­py for myelo­ma pa­tients af­ter stem cell trans­plants. Take­da had pur­sued that  in­di­ca­tion with pos­i­tive pro­gres­sion-free sur­vival stats, but the agency is hold­ing out for over­all sur­vival da­ta be­fore they con­sid­er a green light.

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.

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.

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.

→ 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.

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.