Ax­el Bou­chon starts sign­ing play­ers for a new league of game-chang­ing biotechs

Ax­el Bou­chon

Ax­el Bou­chon is the point man at Bay­er who’s tak­ing a con­cept out­lined on a piece of pa­per back in De­cem­ber and turn­ing it in­to a $335 mil­lion biotech with more than 100 de­vel­op­ment staffers in key lo­ca­tions in the U.S. and Eu­rope. That’s a big task, but it’s on­ly one facet in a much more am­bi­tious life sci­ence game plan he’s quar­ter­back­ing for Bay­er, ramp­ing up 3 to 5 star­tups with fi­nanc­ing rounds stretch­ing from $100 mil­lion to $300 mil­lion or more.

Step 1 is Case­bia, a joint ven­ture Bay­er part­nered with CRISPR Ther­a­peu­tics that will look to carve out a lead­er­ship po­si­tion in three key ar­eas us­ing CRISPR/Cas 9 gene edit­ing tech. Swiss-based, CRISPR Ther­a­peu­tics has or­ga­nized its R&D group in Cam­bridge, MA, where the sci­en­tif­ic tal­ent is, and they’re plan­ning to move the start­up in­to the beat­ing heart of the re­gion’s boom­ing biotech hub on Kendall Square, as we re­port­ed on Fri­day.

In a fol­low-up in­ter­view, Bou­chon out­lined Case­bia’s fu­ture, as well as Bay­er’s plans to use this as one mod­el for a line­up of big biotech launch­es that are be­gin­ning to take shape now.

“Es­sen­tial­ly, over the last 4 months we gave the ba­by [Case­bia] a home,” Bou­chon tells me. “It’s a huge ben­e­fit, hav­ing two ful­ly fledged com­pa­nies as the moth­er.”

The Case­bia fam­i­ly in­volves about 30 em­ploy­ees right now, with on­ly a hand­ful ac­tu­al­ly signed up to work at the biotech. The rest, like Bay­er’s pro­tein en­gi­neer­ing staffers in Cologne, are con­tribut­ing to the work. But as in­ter­im CEO, Bou­chon has start­ed sign­ing up what will be a ded­i­cat­ed team of 80 in Cam­bridge, an­oth­er 10-15 in San Fran­cis­co, where it has a cam­pus at Mis­sion Bay, and an­oth­er 10-15 in Ger­many.

That may sound big com­pared to most ven­ture-backed biotech star­tups, where the num­ber is usu­al­ly lim­it­ed to 20-30 staffers, “but the joint ven­ture is carv­ing out en­tire ther­a­peu­tic ar­eas,” notes Bou­chon. “If we find 50 tar­gets, we work on 50 tar­gets.”

Bay­er is con­tribut­ing $300 mil­lion to fund­ing the re­search in the first 5 years. And Bou­chon isn’t the least bit in­tim­i­dat­ed by the num­bers. When I sug­gest­ed that a big Se­ries A for his oth­er star­tups could start at $50 mil­lion, he quick­ly ruled that out as “too small,” pre­fer­ring to start out with a big­ger num­ber like $100 mil­lion. He al­so likes the way Case­bia brings in a fran­chise play­er like CRISPR Ther­a­peu­tics to get things start­ed at a more ad­vanced lev­el.

“It’s a true team,” he adds, in a 50/50 part­ner­ship where he’ll be the in­ter­im CEO and CRISPR Ther­a­peu­tics’ Rodger No­vak, who we last saw cap­ping off a $140 mil­lion ven­ture round, is tak­ing the chair­man’s post. (No­vak has gone qui­et for now, as the biotech gives every ap­pear­ance of set­ting the stage for an IPO. Com­pa­nies based in Switzer­land are gen­er­al­ly more care­ful than most is fol­low­ing the rules.)

Bay­er has picked a slate of game-chang­ing fields to repli­cate Case­bia in. Bou­chon is a lit­tle re­luc­tant to call these new ven­tures moon shots, a pop­u­lar term in gov­ern­ment and the in­dus­try. But they’re all aimed at par­a­digm-shift­ing break­throughs in its three big R&D zones: hu­man, an­i­mal and plant tech.

Bay­er has picked five fields so far: DNA edit­ing, stem cells, RNA in­hi­bi­tion or ac­ti­va­tion, and the mi­cro­bio­me.

The mi­cro­bio­me has al­ready emerged as a next-gen tech at Bay­er’s ag di­vi­sion. Now they want to use that sci­en­tif­ic ex­per­tise to tack­le hu­mans, in a field that has drawn a group of new play­ers in Cam­bridge, MA and the Bay Area. Both of those hubs are like­ly to play a big role in Bay­er’s start­up plans, though the Mass­a­chu­setts clus­ter looms largest in the hunt for the best and the bright­est.

Says Bou­chon: “To be hon­est, the key hub with the key tal­ent is Boston and then San Fran­cis­co. Cam­bridge is num­ber one right now. That will at­tract al­so the next wave” of star­tups.

But Bay­er al­so likes com­bin­ing glob­al sites, and is mind­ful of the grow­ing role that Japan is play­ing in tar­get ar­eas like stem cell re­search.

None of this is go­ing to be easy or par­tic­u­lar­ly quick, notes Bou­chon. When you pick out next-gen fields, you can hope that this is like bet­ting on mon­o­clon­al an­ti­bod­ies in the ear­ly ‘80s. But there are some big sci­en­tif­ic and med­ical chal­lenges to face off, in­clud­ing the de­vel­op­ment of bet­ter de­liv­ery sys­tems to do this in vi­vo.

“The pit­fall is that it’s sci­ence and it’s very ear­ly.” Trans­lat­ing that to the pub­lic, when the tech­nol­o­gy is com­plex and dif­fi­cult to ex­plain, al­so makes it hard to con­vey the dis­rup­tive think­ing that Bay­er is drawn to with these biotech up­starts.

But that won’t stop him from try­ing.

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 help@endpointsnews.com 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.