→ FDA com­mish Scott Got­tlieb was spread­ing the love Mon­day morn­ing, wel­com­ing back FDA staff and look­ing to get the world’s fastest drug reg­u­la­to­ry agency back up to full swing AS­AP. Of course, it could all come crash­ing to a halt again Feb­ru­ary 15, un­less law­mak­ers ei­ther out­law gov­ern­ment shut­downs or come to a com­pro­mise of sorts. In the mean­time, the FDA is open for busi­ness.

I look for­ward to be­ing out­side Build­ing 1 to­day to greet ALL my col­leagues as we re­turn to our posts TO­GETH­ER. I know there will be ad­di­tion­al chal­lenges ahead as we catch up on missed work and dead­lines. I’ll sup­port you in all of these ef­forts as we forge ahead as ONE #FDA pic.twit­ter.com/0zJSm­NXU9o

— Scott Got­tlieb, M.D. (@SGot­tliebF­DA) Jan­u­ary 28, 2019

→ Not every­thing was shut­tered at the FDA. The agency has ap­proved Al­lo­gene’s IND for a clin­i­cal tri­al us­ing their off-the-shelf CAR-T AL­LO-501 for non-Hodgkin lym­phoma. The drug will be in­fused in­to pa­tients fol­low­ing fly/cy lym­phode­ple­tion and AL­LO-647, their CD52 an­ti­body.

→ The FDA has giv­en Xenon’s $XENE XEN007 rare pe­di­atric dis­ease sta­tus to treat Al­ter­nat­ing Hemi­ple­gia of Child­hood (AHC) — a rare, se­vere and de­bil­i­tat­ing neu­ro­log­i­cal move­ment dis­or­der that presents in in­fan­cy and has no ap­proved treat­ments. The agency has al­so al­lowed the CNS drug de­vel­op­er to test its XEN1101 in hu­mans for epilep­sy and oth­er neu­ro­log­i­cal dis­or­ders.

→ Fib­ri­ot­ic dis­ease drug de­vel­op­er In­da­lo Ther­a­peu­tics has ap­point­ed Robert Jacks as its pres­i­dent and CEO, who comes  from Sym­bio­mix Ther­a­peu­tics where he served as co-founder, pres­i­dent and CFO. The com­pa­ny is ex­pect­ed to take its lead drug, IDL-2965, in­to the clin­ic in ear­ly 2019.

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.