No­var­tis backs $30M round for can­cer biotech; Bay­er drug makes a ‘break­through’ at FDA

Ay­ala Ther­a­peu­tics — which fo­cus­es on rare can­cers that oc­cur due to dys­reg­u­la­tion of the Notch-ac­ti­vat­ing path­way — has raised $30 mil­lion in a round of fi­nanc­ing led by part­ner No­var­tis.

The funds, which al­so came from in­vestors in­clud­ing SBI JI In­no­va­tion Fund, Is­rael Biotech Fund, aMoon and Harel In­sur­ance & Fi­nance Group, will be used to fu­el the de­vel­op­ment of its lead drug, AL101. The pan-Notch in­hibitor is cur­rent­ly be­ing eval­u­at­ed for ade­noid cys­tic car­ci­no­ma (ACC) in a Phase II tri­al, and the com­pa­ny is al­so plan­ning to in­ves­ti­gate its use in a mid-stage study in triple neg­a­tive breast can­cer.

In 2017, Ay­ala inked a deal with Bris­tol-My­ers Squibb $BMY to de­vel­op treat­ments for can­cers with al­tered Notch genes. The fol­low­ing year, it joined forces with No­var­tis to de­vel­op and mar­ket an­oth­er drug, AL102, in com­bi­na­tion with BC­MA-tar­get­ing agents for mul­ti­ple myelo­ma pa­tients.

The Is­raeli biotech raised $17 mil­lion in its first round of fi­nanc­ing in April 2018.

→ The FDA grant­ed Bay­er’s Aliqopa (co­pan­lis­ib) — used for the treat­ment of adult pa­tients with re­lapsed mar­gin­al zone lym­phoma (MZL) who have re­ceived at least two oth­er ther­a­pies pri­or — break­through ther­a­py des­ig­na­tion based on over­all re­sponse rate (ORR) from its piv­otal Phase II CHRONOS-1 study. The com­pa­ny says that it “is con­duct­ing two ad­di­tion­al Phase III stud­ies to eval­u­ate the ef­fi­ca­cy and safe­ty of Aliqopa in com­bi­na­tion with oth­er ther­a­pies with iNHL (in­clud­ing MZL) who have re­lapsed fol­low­ing one or more pri­or ther­a­pies.”

→ Ger­man-based Bay­er $BAYRY and Foun­da­tion Med­i­cine $FMI are en­ter­ing a glob­al col­lab­o­ra­tion for the de­vel­op­ment and com­mer­cial­iza­tion of NGS-based com­pan­ion di­ag­nos­tic tests, which help find a pos­si­ble can­cer treat­ment ther­a­py via mol­e­c­u­lar in­for­ma­tion from a pa­tient’s tu­mor genome. Bay­er will al­so have ac­cess to Foun­da­tionOne CDx — Foun­da­tion Med­i­cine’s se­quenc­ing based in vit­ro di­ag­nos­tic de­vice.

The first project that will be un­der­tak­en is de­vel­op­ing a com­pan­ion di­ag­nos­tic for Vi­t­rakvi (larotrec­tinib), the first and on­ly TRK in­hibitor ap­proved in the US for adult and pe­di­atric pa­tients with “sol­id tu­mors that have a neu­rotroph­ic tropomyosin re­cep­tor ki­nase (NTRK) gene fu­sion with­out a known ac­quired re­sis­tance mu­ta­tion that are ei­ther metasta­t­ic or where sur­gi­cal re­sec­tion will like­ly re­sult in se­vere mor­bid­i­ty, and have no sat­is­fac­to­ry al­ter­na­tive treat­ments or that have pro­gressed fol­low­ing treat­ment.”

As­traZeneca spin­out Viela Bio has notched a com­mer­cial deal with sea­soned Chi­nese phar­ma Han­soh cen­tered around inebi­lizum­ab, the lead drug among six as­sets from the phar­ma gi­ant. The an­ti-CD19 drug treats neu­romyelitis op­ti­ca spec­trum dis­or­der, a rare dis­ease that af­fects the eye and spine. All told, the deal is worth up to $220 mil­lion in­clud­ing up­front and mile­stones.

The Park­er In­sti­tute is em­bark­ing on a new re­search jour­ney to find out why some pa­tients de­vel­op au­toim­mune dis­or­ders fol­low­ing can­cer im­munother­a­py. To­geth­er with two oth­er non­prof­its (ded­i­cat­ed to di­a­betes and health­care re­spec­tive­ly), it’s pledg­ing $10 mil­lion over three years to sup­port re­searchers at Yale, UCSF, Vir­ginia Ma­son and Dana-Far­ber. The hope is to both ad­vance can­cer treat­ment and bet­ter un­der­stand how type 1 di­a­betes de­vel­ops in gen­er­al.

→ In 2015, In­ovio joined forces with As­traZeneca’s $AZN Med­Im­mune unit for $27.5 mil­lion up­front, as well as an­oth­er po­ten­tial $700 mil­lion in mile­stone pay­ments — for the ex­clu­sive rights to In­ovio’s im­munother­a­py, de­signed to tar­get can­cers caused by hu­man pa­pil­lo­mavirus (HPV), in ad­di­tion to two ad­di­tion­al DNA-based can­cer vac­cine prod­ucts. On Wednes­day, In­ovio in a fil­ing dis­closed that Med­Im­mune was re­tain­ing its rights to the HPV im­munother­a­py, ME­DI0457 (pre­vi­ous­ly called INO-3112), but walk­ing away from the rest.

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.


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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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.

Fol­low­ing CAR-T pi­o­neer­s' foot­steps, Tes­sa launch­es Chi­na JV in $120M deal

These days just about every biotech se­ri­ous about glob­al de­vel­op­ment — and not just com­mer­cial­iza­tion — has a Chi­na strat­e­gy. Tes­sa Ther­a­peu­tics, a Bay­lor as­so­ci­at­ed out­fit based out of Sin­ga­pore, is no ex­cep­tion.

Tak­ing a page out of the CAR-T pi­o­neers’ play­book, Tes­sa is es­tab­lish­ing a joint ven­ture with Chi­na-Sin­ga­pore Guangzhou Knowl­edge City, which is ini­tial­ly putting down $40 mil­lion for a 13% stake with $40 mil­lion more to come in a sec­ond stage. The biotech, which now re­tains an 87% con­trol, is al­so rolling out its own con­tri­bu­tions in two phas­es, start­ing with $20 mil­lion and all its tech­nol­o­gy li­cense rights for Chi­na.

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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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.