Pfiz­er biotech spin­out nabs $125M megaround as Bain-backed syn­di­cate bets on its late-stage game plan

Spring­Works Ther­a­peu­tics launched in the fall of 2017 with a whop­ping $103 mil­lion round and some well-de­vel­oped Pfiz­er as­sets that the com­pa­ny had high hopes for. And now Bain, Pfiz­er and the rest of the found­ing in­vestors have joined hands with an even big­ger group of in­vestors led by Per­cep­tive to bankroll a pair of late-stage stud­ies ready to be­gin in a mat­ter of weeks.

The Se­ries B weighs in at a heavy­weight $125 mil­lion, bring­ing their to­tal raise to $228 mil­lion, with ac­cess to more if need­ed.

The fo­cus of this lat­est megaround is cen­tered on two drugs that are be­ing re­pur­posed for rare dis­eases: nirogace­s­tat, a gam­ma sec­re­tase in­hibitor for the treat­ment of desmoid tu­mors (rare soft tis­sue tu­mors), and PD-0325901, a MEK in­hibitor for the treat­ment of neu­rofi­bro­mato­sis type 1-as­so­ci­at­ed plex­i­form neu­rofi­bro­mas. 

Both of these tar­gets are well de­fined, with mul­ti­ple tri­als for re­lat­ed drugs over the past decade or more. The gam­ma sec­re­tase in­hibitor was born in the Alzheimer’s field, where Eli Lil­ly ex­pe­ri­enced a colos­sal dis­as­ter years ago. That MEK in­hibitor, Spring­Works ex­ecs say, al­so has po­ten­tial to make good as a back­bone com­bi­na­tion drug in on­col­o­gy, fol­low­ing oth­er ap­proved ther­a­pies.

Im­age: Saqib Is­lam. CHECK­RARE via YOUTUBE

The big fi­nanc­ing takes the com­pa­ny all the way through the two late-stage stud­ies, says CEO Saqib Is­lam, with a clas­sic cross­roads that could ar­rive in H2 of next year if they do an in­ter­im analy­sis and if it’s pos­i­tive, set­ting up a pos­si­ble FDA ap­pli­ca­tion. The gam­ma sec­re­tase study should read out in ear­ly 2021.

The com­pa­ny, which now has 41 staffers head­ed to about 70 at the end of the year, says the CEO, has the ca­pac­i­ty to go the dis­tance by it­self in rare dis­eases. And as a vet of Alex­ion, Is­lam — who more re­cent­ly did deals for Mod­er­na — says they are ready to do what’s nec­es­sary to launch com­mer­cial op­er­a­tions. Larg­er in­di­ca­tions could be cov­ered with part­ner­ships, and he point­ed to a tie-up they have with BeiGene, com­bin­ing their RAF dimer in­hibitor li­fi­rafenib (BGB-283) with the MEK.

More deals are clear­ly in the works, he adds, which could trig­ger a faster ex­pan­sion of the staff and ca­pa­bil­i­ties at Spring­Works, which has the re­search team in RTP and the cor­po­rate staff now head­quar­tered in Stam­ford, CT.

I asked Is­lam the ob­vi­ous ques­tion: With a syn­di­cate this size with these play­ers, and plans to push through piv­otal tri­als, an IPO would seem to be in the cards as long as the mar­ket holds up. He laughed a lit­tle and lim­it­ed him­self to the stan­dard re­frain: All fi­nanc­ing op­por­tu­ni­ties are un­der re­view.

Pfiz­er has helped craft sev­er­al new com­pa­nies like this re­cent­ly. There’s Bain-backed Cerev­el as well as Al­lo­gene from Arie Bellde­grun and David Chang. In every case, Pfiz­er ex­ecs of­floaded as­sets they no longer want­ed in the pipeline, but which they felt had re­al com­mer­cial prospects in the right hands. Is­lam al­so not­ed that they are in­clud­ed in Pfiz­er’s port­fo­lio re­view process, which could al­so trig­ger more deals down the road as Pfiz­er con­tin­ues to shed projects.

Per­cep­tive Ad­vi­sors led the show for the Se­ries B. New in­vestors par­tic­i­pat­ing in this fi­nanc­ing in­clude Box­er Cap­i­tal of Tavi­s­tock Group, HBM Health­care In­vest­ments, BVF Part­ners, Sur­vey­or Cap­i­tal, Sam­sara Bio­Cap­i­tal, Ar­row­Mark Part­ners, Glax­o­SmithK­line, and Lau­ri­on Cap­i­tal Man­age­ment, as well as “sev­er­al oth­er long-term in­sti­tu­tion­al in­vestors.” All of the com­pa­ny’s ex­ist­ing in­vestors – Or­biMed, Bain Cap­i­tal, Pfiz­er, via Pfiz­er Ven­tures, and LifeArc – al­so par­tic­i­pat­ed.

It’s cu­ri­ous to see GSK in­clud­ed here as a di­rect in­vestor. In re­cent months its cor­po­rate ven­ture arm SR One has been ne­go­ti­at­ing to spin out from un­der the phar­ma gi­ant, which is more in­ter­est­ed in us­ing all of its cash in di­rect line with the R&D game plan set by Hal Bar­ron.

Is­lam said he could of­fer on­ly lim­it­ed in­sight in­to GSK’s cor­po­rate in­volve­ment, but added that “they could be a fab­u­lous part­ner for a cou­ple of things down the road.”

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.

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.

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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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.

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.

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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