The res­ur­rec­tion of LUM001: Mike Grey gets his drug back from Shire — along with $120M to gam­ble on PhI­II tri­als

EX­CLU­SIVE: In drug de­vel­op­ment, fail­ure isn’t al­ways fa­tal. Just ask Mike Grey.

When Grey com­plet­ed his deal to sell Lu­me­na to Shire back in 2014 for $260 mil­lion-plus, Shire was left in full con­trol of the charge to what it hoped would be a near-term ap­proval for a drug de­signed to con­trol ex­cess bile acids and ex­treme itch­ing.

A year lat­er, though, the lead pro­gram for LUM001 was ap­par­ent­ly in tat­ters, with an ug­ly mid-stage fail­ure for rare cas­es of Alag­ille syn­drome (AL­GS) that ap­peared to wipe out the block­buster val­ue Shire CEO Flem­ming Orn­skov had once en­vi­sioned when he did the deal. Then 2 years ago there was an­oth­er crit­i­cal tri­al break­down for the drug, then known as SHP625, with no sig­nif­i­cant re­duc­tion from base­line in serum al­ka­line phos­phatase or oth­er liv­er pa­ra­me­ters in pa­tients with pri­ma­ry scle­ros­ing cholan­gi­tis (PSC).

But it wasn’t dead. 

Alex Doren­baum

To­day, 4 years af­ter he ced­ed con­trol of the oral in­hibitor of the api­cal sodi­um de­pen­dent bile acid trans­porter, Grey is step­ping up with a fresh batch of top-line da­ta that has helped res­ur­rect the failed ther­a­py, along with a $120 mil­lion mega-round to launch the new com­pa­ny he’s put to­geth­er to make that hap­pen. And Shire — now soon to be merged with Take­da — has hand­ed over the world­wide rights to the drug for an un­spec­i­fied up­front and eq­ui­ty in the start­up.

“Clear­ly the tri­als did not turn out as you hoped,” Grey tells me in a phone in­ter­view from Lon­don as he wrapped the new con­tract. “Some­times, it takes a lit­tle time to get it right.”

Su­san Dubé

LUM001/SHP625 is now dubbed mar­al­ix­i­bat, which Grey says had a suc­cess­ful per­for­mance in Shire’s Phase IIb study called ICON­IC in AL­GS. An im­proved tri­al de­sign helped pave the way to a suc­cess­ful in­ter­im read­out at 48 weeks with sig­nif­i­cant re­duc­tions in bile acids and pru­ri­tus com­pared to place­bo. He al­so ob­tained pos­i­tive re­sults from a sub­group analy­sis in PF­IC, land­ing the right kind of da­ta to gain a break­through drug des­ig­na­tion from the FDA.

Armed with the new mon­ey from a group of deep pock­et­ed in­vestors — who are al­so en­dors­ing the da­ta and the drug and Mike Grey — Mirum is prep­ping a pair of Phase III stud­ies slat­ed to be­gin as ear­ly as pos­si­ble in 2019. 

Grey is go­ing for it, and he has the mon­ey need­ed to get it done and on to the FDA. Af­ter that, he’s plan­ning to be ready to launch mar­ket­ing op­er­a­tions — though he isn’t rul­ing out a buy­out along the way.

Cia­ra Kennedy

How much did he pay to get the drug back, along with rights to an­oth­er drug called volix­i­bat, the oth­er AS­BT in­hibitor from Lu­me­na once known as LUM002?

“Some­what less than what it sold for,” is all he’s say­ing this week.

It’s un­like­ly that any­one knows more about this drug than Grey, who nev­er gave up on it. Get­ting a drug back from a buy­er isn’t easy un­der any cir­cum­stances. But these deals have re­al po­ten­tial. Just ask John Hood, who got his drug fe­dra­tinib back from Sanofi years af­ter a land­mark col­lapse and then al­most im­me­di­ate­ly flipped it to Cel­gene for $1.1 bil­lion in cash — plus bil­lions more in mile­stones — at the be­gin­ning of this year.

Grey’s al­so not alone. Al­so buy­ing in to the res­ur­rec­tion of LUM001 is his lean-and-mean crew of 10 drawn from the old Lu­me­na team along with some se­lect To­bi­ra vets, in­clud­ing:

  • Chris Peetz has been ap­point­ed as pres­i­dent of Mirum and a mem­ber of the com­pa­ny’s board of di­rec­tors
  • Pamela Vig joins as chief sci­en­tif­ic of­fi­cer
  • Lara Long­pre will serve as the com­pa­ny’s chief de­vel­op­ment of­fi­cer
  • Shelly Xiong has been ap­point­ed head of reg­u­la­to­ry
  • Co-founders and ad­vis­ers: Alex Doren­baum, Su­san Dubé, Cia­ra Kennedy and Niall O’Don­nell.
Niall O’Don­nell

New En­ter­prise As­so­ci­ates led the round with par­tic­i­pa­tion from Deer­field Man­age­ment, Fra­zier Health­care Part­ners, No­vo Hold­ings A/S, Pap­pas Cap­i­tal, River­Vest Ven­ture Part­ners and Rock Springs Cap­i­tal. And they’re pro­vid­ing the board to watch things play out: Ed Math­ers, part­ner, NEA; Patrick Heron, man­ag­ing gen­er­al part­ner, Fra­zier Health­care Part­ners; Jonathan Leff, part­ner, Deer­field; Ti­ba Aynechi, part­ner, No­vo Hold­ings; and Niall O’Don­nell, man­ag­ing di­rec­tor, River­Vest.

They’re all suit­ed up, and ready to tack­le late-stage tri­als.


Im­age: Mike Grey. PAP­PAS CAP­I­TAL

Norbert Bischofberger. Kronos

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With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Francesco De Rubertis

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“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

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Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

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Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
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After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.

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For drugmakers, FDA advisory panels are often an apprehended barometer of regulators’ final decisions. While the experts’ endorsement or criticism often translate directly to final outcomes, the FDA sometimes stun observers by diverging from recommendations.

A new paper out of Milbank Quarterly put a number on that trend by analyzing 376 voting meetings and subsequent actions from 2008 through 2015, confirming the general impression that regulators tend to agree with the adcomms most of the time — with discordances in only 22% of the cases.

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