Por­to­la gets EU clear­ance to mar­ket An­dexxa un­der new name; Four biotechs set terms for Nas­daq de­but

Por­to­la $PT­LA has made in­roads for its an­ti­co­ag­u­lant an­ti­dote in Eu­rope, win­ning a con­di­tion­al OK to mar­ket an­dex­anet al­fa as On­dexxya. Sold in the US as An­dexxa, the drug is de­signed to re­verse the blood thin­ning ef­fects of Fac­tor Xa in­hibitors like apix­a­ban and ri­varox­a­ban in acute sit­u­a­tions. Por­to­la’s fourth quar­ter rev­enue for 2018 was $15.3 mil­lion, $14 mil­lion of which was net prod­uct rev­enue from An­dexxa sales.

→ French-based Cal­ixar an­nounced to­day that it has en­tered in­to an ex­clu­sive li­cens­ing agree­ment with Re­gen­eron $REGN. Un­der the agree­ment, Re­gen­eron has ex­clu­sive rights to ac­cess its tech­nol­o­gy and ex­per­tise to con­duct re­search and dis­cov­ery of an­ti­bod­ies against an undis­closed tar­get in var­i­ous ther­a­peu­tic fields. This agree­ment in­cludes up­front mile­stone pay­ments to Cal­ixar; how­ev­er, fi­nan­cial terms were not dis­closed.

Cor­texyme has set terms for its IPO, which is down­sized from its orig­i­nal plan of $86 mil­lion. The South San Fran­cis­co-based com­pa­ny is now plan­ning to raise $75 mil­lion by sell­ing 4.4 mil­lion shares be­tween $16 and $18, based on a pitch around its the­o­ry that a bac­te­r­i­al pathogen plays a ma­jor role in Alzheimer’s. At the mid­point range, mar­ket val­ue would hit $476 mil­lion. Re­nais­sance Cap­i­tal says that “in­sid­ers in­tend to pur­chase $35 mil­lion worth of shares in the of­fer­ing.” The com­pa­ny will list un­der the tick­er CRTX.

Mile­stone Phar­ma­ceu­ti­cals, a Mon­tre­al, Cana­da-based biotech de­vel­op­ing ther­a­pies for heart con­di­tions, laid plans to raise $75 mil­lion in an IPO. The com­pa­ny, which in­tends to list un­der the tick­er $MIST, is of­fer­ing 5 mil­lion shares priced be­tween $14 to $16. At the mid­point of that range, its mar­ket val­ue would hit $378 mil­lion. The an­nounce­ment comes less than a year af­ter the com­pa­ny raised $80 mil­lion in a round of fi­nanc­ing to de­vel­op its lead drug, etri­pamil, a cal­ci­um chan­nel block­er that Mile­stone has for­mu­lat­ed in­to a nasal spray to treat tachy­car­dia.

→ Weeks af­ter blue­print­ing the sci­en­tif­ic foun­da­tion for the im­muno-on­col­o­gy com­pa­ny in a pre­clin­i­cal study pub­lished in Na­ture Med­i­cine, Lil­ly-part­nered NextCure set terms to raise $75 mil­lion in an IPO on Mon­day. The Beltsville, Mary­land-based com­pa­ny, which has al­ready raised $180 mil­lion in ven­ture cap­i­tal to go af­ter PD-L1 neg­a­tive tu­mors where ex­ist­ing check­point in­hibitors are im­po­tent, counts Lieping Chen — a not­ed I/O re­searcher who was be­hind Am­plim­mune, which was swal­lowed by As­traZeneca $AZN — as its sci­en­tif­ic founder. NextCure plans to list un­der the sym­bol $NXTC and is of­fer­ing 5 mil­lion shares at a price range of $14 to $16 — at the mid­point of that range it will car­ry a mar­ket val­ue of $350 mil­lion.

→ New York, NY-based Ap­plied Ther­a­peu­tics, de­vel­op­ing ther­a­pies for di­a­bet­ic car­diomy­opa­thy, an­nounced terms for its IPO to raise $60 mil­lion by of­fer­ing 4 mil­lion shares at a price range of $14 to $16. At the mid­point range, mar­ket val­ue would hit $292 mil­lion. The com­pa­ny will list un­der the tick­er $APLT.

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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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.