In­ozyme brings $67M to the bank to build up its cal­ci­fi­ca­tion-fo­cused rare dis­ease pipeline

In­ozyme founder Ax­el Bolte is in­ten­si­fy­ing his faith in his rare-dis­ease up­start’s lead ex­per­i­men­tal drug by test­ing it for a sec­ond in­di­ca­tion fo­cused on dis­or­ders char­ac­ter­ized by ab­nor­mal cal­ci­fi­ca­tion. An ex­pand­ed clin­i­cal pro­gram for the drug — which is be­ing read­ied for hu­man test­ing — needs more cap­i­tal, and the biotech has raised some in a fresh $67 mil­lion round, led by Piv­otal bioVen­ture Part­ners and Sofinno­va In­vest­ments.

Bolte launched the com­pa­ny with a $49 mil­lion round and armed with slate of no­table bio­phar­ma ex­ec­u­tives, af­ter run­ning in­to Yale sci­en­tist Demetrios Brad­dock and his work on cal­ci­fi­ca­tion in his ca­pac­i­ty as an in­vest­ment ad­vi­sor for HBM Part­ners. He spent most of 2016 rais­ing funds, and in 2017 In­ozyme was of­fi­cial­ly born.

Sarah Bha­gat

The Boston, Mass­a­chu­setts-based com­pa­ny —  which has raised $116 mil­lion in ven­ture cap­i­tal to date — has bet on the role of py­rophos­phate, or PPi, in rare, life-threat­en­ing dis­or­ders that are man­i­fest­ed by the over-cal­ci­fi­ca­tion of soft tis­sues and un­der-min­er­al­iza­tion of bone. Armed with this sci­en­tif­ic ba­sis In­ozyme is de­vel­op­ing en­zyme re­place­ment ther­a­pies us­ing in­or­gan­ic PPi to treat cal­ci­fi­ca­tion dis­or­ders.

This new in­fu­sion will help shep­herd its lead en­zyme re­place­ment ther­a­py — INZ-701 — in­to hu­man stud­ies for use in for se­vere dis­or­ders of cal­ci­fi­ca­tion as­so­ci­at­ed with de­fi­cien­cies in the en­zyme ENPP1, in­clud­ing Gen­er­al­ized Ar­te­r­i­al Cal­ci­fi­ca­tion of In­fan­cy and Au­to­so­mal Re­ces­sive Hy­pophos­phatemic Rick­ets Type 2. In­ozyme is cur­rent­ly com­plet­ing IND‑en­abling stud­ies for the drug and ex­pects to ini­ti­ate clin­i­cal tri­als in 2020.

The funds will al­so be used to test the drug in pa­tients with mu­ta­tions in the ABCC6 gene, which can lead to a con­di­tion known as pseu­dox­an­thoma elas­ticum.

In­ozyme has al­so added to its pipeline by adding a sec­ond re­search pro­gram tar­get­ing an undis­closed rare bone dis­ease.

The Se­ries A2 fi­nanc­ing al­so in­clud­ed the par­tic­i­pa­tion of RA Cap­i­tal Man­age­ment, Cowen Health­care In­vest­ments, Rock Springs Cap­i­tal, Lon­gi­tude Cap­i­tal, NEA, No­vo Hold­ings, and Sanofi Ven­tures.

Rob Hopfn­er, man­ag­ing part­ner of Piv­otal bioVen­ture Part­ners, and Sarah Bha­gat, prin­ci­pal at Sofinno­va In­vest­ments, al­so joined In­ozyme’s board.


Im­age: Ax­el Bolte. TUR­OS CAP­I­TAL

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.

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.

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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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.

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.

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.

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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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.