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.”

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.