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

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP)

Can a smart­phone app de­tect Covid? Pfiz­er throws down $116M to find out

What can a cough say about a patient’s illness? Quite a bit, according to ResApp Health — and Pfizer’s listening.

The pharma giant is shelling out about $116 million ($179 million AUD) to scoop up the University of Queensland spinout and its smartphone technology that promises to diagnose Covid and other respiratory illnesses based on cough and breathing sounds, the university announced last week.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Severin Schwan, outgoing Roche CEO (via Getty Images)

Roche hires new di­ag­nos­tics chief from with­in, ahead of C-suite shake-up

More than two months after Severin Schwan announced he’s leaving Roche and handing the reins to diagnostics chief Thomas Schinecker, the pharma giant has revealed who’s taking Schinecker’s place.

Matt Sause, who currently leads Roche’s North American diagnostics business, is popping the cork on the big global promotion to take effect on March 15. The 20-year Roche veteran has served a handful of roles across the company’s diagnostics and pharma units, including a stint at Genentech where he was lifecycle leader for blockbuster Tecentriq’s head and neck cancer programs.

Rob Etherington, Clene CEO

Star­tup's gold nanocrys­tal ALS drug flops a PhII tri­al, a re­minder of the dis­ease's ob­sta­cles de­spite Amy­lyx OK

Despite the FDA approving an ALS drug for the first time in five years last week, the disease continues to fluster researchers, and another biotech is feeling the pain of a mid-stage failure.

Clene Nanomedicine reported early Monday that its ALS program, which uses gold nanocrystals to try to catalyze intracellular reactions, did not achieve its Phase II primary or secondary endpoints. And in a press release, the company noted for the first time that it’s speaking with “potential strategic partners” about the program — language that typically indicates a biotech is preparing to sell off an asset.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.