$49M round; ex­pe­ri­enced team; A-list VCs — In­ozyme launch­es with rare dis­ease strat­e­gy and a yen for big­ger things to come

Ax­el Bolte

It was Ax­el Bolte’s work as a ven­ture part­ner for HBM that orig­i­nal­ly took him to the cam­pus at Yale, with a chance to start meet­ing the sci­en­tists there. And that in­tro­duc­tion even­tu­al­ly led him to Demetrios Brad­dock, who had been do­ing lab work on cal­ci­fi­ca­tion that he’s de­vout­ly hop­ing could have an im­pact on a long line of dis­eases.

“Ul­ti­mate­ly I al­ways want­ed to be di­rect­ly in­volved as an en­tre­pre­neur and op­er­a­tional in com­pa­nies,” says Bolte, “which is what I did be­fore in large or­ga­ni­za­tions.”

It was Brad­dock’s work that made that hap­pen. And to­day Bolte is of­fi­cial­ly open­ing the shop at In­ozyme, the lat­est in a long line­up of star­tups to set out to make their mark in Cam­bridge, MA.

The re­al re­search work at In­ozyme got start­ed ear­ly this year, Bolte tells me. That was af­ter he spent much of 2016 rais­ing cash, which led to to­day’s an­nounced $49 mil­lion A round with some VCs he knows well. Lon­gi­tude Cap­i­tal took the lead for a round that in­cludes New En­ter­prise As­so­ci­ates, No­vo Ven­tures and Sanofi Ven­tures.

Demetrios Brad­dock

They’re back­ing a very fa­mil­iar strat­e­gy that starts with rare dis­eases, a clas­sic strat­e­gy that al­lows for piv­otal da­ta on small pa­tient groups.

Their lead en­zyme re­place­ment ther­a­py is for the treat­ment of Gen­er­al­ized Ar­te­r­i­al Cal­ci­fi­ca­tion of In­fan­cy — GACI, which hits new­borns — and Au­to­so­mal Re­ces­sive Hy­pophos­phatemic Rick­ets Type 2.  GACI is trig­gered by a ge­net­ic mu­ta­tion that caus­es low lev­els of py­rophos­phate and cal­ci­fi­ca­tion of the ar­ter­ies and heart. But af­ter get­ting es­tab­lished in rare dis­eases, Bolte sees a big­ger fu­ture ahead in ma­jor dis­eases like chron­ic kid­ney dis­ease.

Right now the com­pa­ny has 8 staffers, right in line for where it’s at at this stage. It in­cludes some high­ly ex­pe­ri­enced biotechies with re­sumes that lean heav­i­ly on rare dis­eases:

  • COO Hen­ric Bjarke: for­mer vice pres­i­dent and ther­a­peu­tic area head for the meta­bol­ic busi­ness unit at Alex­ion, where he was re­spon­si­ble for as­fo­tase-al­fa for the treat­ment hy­pophos­phata­sia and oth­er de­vel­op­ment pro­grams.
  • Chief tech­ni­cal op­er­a­tions of­fi­cer Steven Jun­gles, a vet from Ul­tragenyx and Bio­Marin.
  • Chief med­ical of­fi­cer Er­ic Yuen, with stints at Mer­ck, John­son & John­son, Ul­tragenyx and Bio­Clin.
  • Reg­u­la­to­ry chief Ruhi Ahmed from Ul­tragenyx and Bio­Marin.
  • Fi­nance VP Stephen Bas­so out of Alex­ion.

Now Bolte plans to dou­ble that num­ber as the com­pa­ny heads to the clin­ic.

“We have some ex­pe­ri­ence in CMC and these kinds of things,” says the CEO. “We ex­pect an IND in 2019 and then clin­i­cal tri­als should start at the end of ’19, with ini­tial tri­al re­sults in 2020.”

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.


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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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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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.

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.

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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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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