Johns Hop­kins has $65M to spend on tak­ing their drugs right up to hu­man stud­ies

Re­searchers at Johns Hop­kins Uni­ver­si­ty now have a new way to take a promis­ing ther­a­py to the thresh­old of Phase I, bur­nish­ing the val­ue of what they have for the spin­outs and bio­phar­ma groups that will take a se­lect few ther­a­pies to the clin­ic.

Jim Fly­nn

Deer­field Man­age­ment is fund­ing the pro­gram with $65 mil­lion to start. But that mon­ey, of course, comes with strings at­tached.

The new in-house in­cu­ba­tor is called Blue­field In­no­va­tions, and its pri­ma­ry goal is to ad­vance Johns Hop­kins re­search that shows strong com­mer­cial promise. The pro­gram will take the uni­ver­si­ty’s re­search through pre-clin­i­cal de­vel­op­ment all the way to IND.

Ad­vanc­ing ther­a­peu­tic pro­grams in-house is an in­creas­ing­ly pop­u­lar path for uni­ver­si­ties, be­cause ad­vanced as­sets bring in more mon­ey on tech trans­fer deals. We saw this hap­pen in 2016 when Mer­ck paid Har­vard $20 mil­lion for ad­vanced as­sets de­vel­oped at the uni­ver­si­ty’s in-house ac­cel­er­a­tor. And again when ad­vanced re­search from Irv Weiss­man’s lab at Stan­ford was spun out to Forty Sev­en with a $75 mil­lion vote of con­fi­dence from Google’s ven­ture arm.

Dave Gib­bons

Dave Gib­bons, who han­dles com­mer­cial li­cens­ing at the biotech-heavy cam­pus at UC San Diego, tells me it’s un­usu­al for uni­ver­si­ties to get such big checks on tech trans­fer deals. Uni­ver­si­ties prac­ti­cal­ly give their tech away be­cause the re­search is so im­ma­ture.

“Our deal terms are about two or­ders of mag­ni­tude less than what com­pa­nies do with each oth­er,” Gib­bons said. “But that re­flects the very ear­ly stage of the work. There’s nor­mal­ly a lack of clin­i­cal da­ta be­yond very ba­sic dis­cov­ery.”

If, how­ev­er, a uni­ver­si­ty like Johns Hop­kins can de-risk their pro­grams and de­vel­op them up to IND, “you’d have a tremen­dous­ly more valu­able as­set for li­cens­ing,” Gib­bons said.

Deer­field’s bet

Deer­field’s Jim Fly­nn tells me the ex­cess of dis­cov­ery-stage re­search in acad­e­mia makes uni­ver­si­ties like Johns Hop­kins un­tapped gold mines.

“There’s an enor­mous amount of fer­tile dis­cov­ery sit­ting there ly­ing fal­low at our most promi­nent in­sti­tu­tions,” Fly­nn said.

That’s why Deer­field is chip­ping in the funds to back this new pro­gram at Johns Hop­kins. Un­der a pret­ty broad um­brel­la agree­ment, Deer­field will have con­trol of as­sets de­vel­oped through the pro­gram.

“We’ll ul­ti­mate­ly de­cide where it ends up,” Fly­nn said. “Whether that’s at the uni­ver­si­ty, a spin­out biotech, or per­haps in bet­ter hands with a phar­ma­ceu­ti­cal com­pa­ny.”

Fly­nn not­ed Johns Hop­kins had set out some terms that en­sure the uni­ver­si­ty can “ful­ly par­tic­i­pate” in the trans­fers. When asked what ex­act­ly that means, Fly­nn said, “we make sure they prof­it ad­e­quate­ly if a prod­uct is sold.”

In the­o­ry, the uni­ver­si­ty could prof­it in a num­ber of ways, from mon­e­tiz­ing fu­ture roy­al­ties to snag­ging a share of a spin­out’s eq­ui­ty.

Good or bad for biotech?

While it’s clear that more de­vel­oped as­sets (and big­ger tech trans­fer deals) could be good for uni­ver­si­ties like Johns Hop­kins, some ques­tions come to mind.

Gib­bons and I are both based in San Diego, where small biotechs do­ing pre-clin­i­cal re­search make up the bulk of the life sci­ence econ­o­my. These small­er com­pa­nies re­ly quite heav­i­ly on li­cens­ing tech from lo­cal acad­e­mia. Gib­bons tried to imag­ine UC San Diego part­ner­ing with an in­vest­ment group to launch a pro­gram like this new one at Johns Hop­kins.

“I can see a pro­gram like this di­min­ish­ing the num­ber of star­tups and small busi­ness­es that the uni­ver­si­ty works with,” Gib­bons said. “If we were to hang on­to an as­set, the like­ly buy­er will be Big Phar­ma. Yes, you’ve got a more valu­able as­set, but that means you price out small­er play­ers we tra­di­tion­al­ly work with. And what will that mean for the lo­cal econ­o­my?”

I asked Fly­nn what he thought of that no­tion.

“I hear what he’s say­ing, and we’re sen­si­tive to lo­cal biotechs and the Bal­ti­more econ­o­my,” Fly­nn said. “But the way I see it is that there’s too much fruit on the ta­ble. There’s more at Johns Hop­kins than we can pos­si­bly han­dle our­selves.”

Fly­nn al­so not­ed it’s im­por­tant to re­mem­ber the pa­tient, not just the in­dus­try.

“You want these pro­grams to take their best course for­ward — the one most like­ly to get a drug in a pa­tient’s hand,” Fly­nn said. “And some­times that’s not small biotech.”

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.

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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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.

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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