Neil Ku­mar's Bridge­Bio lines up sub­sidiary No. 9 with a $27M shot at TTR amy­loi­do­sis

Neil Ku­mar

Neil Ku­mar — ex-McK­in­sey, ex-Third Rock, ex-BD guy at MyoKar­dia — set out to build a dif­fer­ent kind of biotech when he start­ed Bridge­Bio a cou­ple of years ago.

Look­ing at the mon­ey that was al­ready avail­able for plat­form plays and late-stage drug work, he was in­ter­est­ed in find­ing new mon­ey to go af­ter ear­ly-stage as­sets. So he adopt­ed an oil com­pa­ny strat­e­gy, lay­ing out the prospects for a biotech out­fit that would fo­cus on what we’ve learned from ge­net­ics dis­cov­ery over the past decade, then pluck­ing sin­gle as­sets that could be com­part­men­tal­ized in sub­sidiaries.

Rather than one wild­cat­ter op­er­a­tion look­ing to drill one well, Ku­mar want­ed to build a vir­tu­al team that could drill a num­ber of wells — look­ing for the few that could be a gush­er.

It was a mat­ter of more “shots on goal,” says Ku­mar, who got some help from MIT’s An­drew Lo in defin­ing the com­pa­ny. “Peo­ple get more in­ter­est­ed in that kind of di­ver­si­fi­ca­tion.”

Frank Mc­Cormick

Lo al­so in­vest­ed in the com­pa­ny, which Ku­mar co-found­ed with Frank Mc­Cormick, the for­mer co-founder of Onyx Phar­ma­ceu­ti­cals.

Rather than chase down and in­cu­bate drugs for in­dus­try buy­ers, much like In­cep­tion from Ver­sant or the oth­er ven­ture um­brel­la groups out there, he want­ed to build a fo­cused com­pa­ny that has the po­ten­tial to go on like Ul­tragenyx or say Bio­Marin and build a com­mer­cial or­ga­ni­za­tion.

‘There are a lot of ear­ly-stage, sin­gle as­set plays that sit on a decade of ge­nom­ic dis­cov­ery,” Ku­mar tells me. And that strat­e­gy brought Per­cep­tive Life Sci­ences and KKR in­to the fold with their fi­nan­cial sup­port.

Ku­mar and his team, which in­cludes some part-timers like Third Rock’s Charles Hom­cy, have been fly­ing un­der the radar most­ly. But to­day he’s un­veil­ing his ninth sub­sidiary op­er­a­tion, which neat­ly char­ac­ter­izes the kind of deals he’s been set­ting up.

This new cre­ation has been dubbed Ei­dos Ther­a­peu­tics, and its sin­gle as­set is AG10, a small mol­e­cule ther­a­py for transthyretin (TTR) amy­loi­do­sis, a field that Al­ny­lam and Io­n­is have both tack­led, run­ning in­to some se­ri­ous safe­ty is­sues along the way.

Is­abel­la Graef

The drug comes out of the work of Is­abel­la Graef at Stan­ford and Mamoun Al­hamad­sheh, the com­pa­ny sci­en­tif­ic co-founders, who have been nail­ing down pre­clin­i­cal ev­i­dence that the drug can sta­bi­lize TTR and pre­vent the cas­cade of events that caus­es the dis­ease — a dis­ease mod­i­fy­ing ap­proach that will now head to the clin­ic. Bridge­Bio has ear­marked $27 mil­lion to pay for the first two stages of clin­i­cal de­vel­op­ment.

“It gets us through Phase II work,” says Ku­mar. In­ves­ti­ga­tors will start by test­ing it in healthy vol­un­teers, look­ing to clar­i­fy the way the drug works.

“Then you go right in­to pa­tients, he adds. “Phase II reads out late next year.”

You can ex­pect to see more deals at Bridge­Bio, he adds. This is just the be­gin­ning.

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.