Four years af­ter it im­plod­ed at Sanofi, John Hood is res­ur­rect­ing the myelofi­bro­sis drug fe­dra­tinib

Fe­dra­tinib was one of the biggest clin­i­cal drug dis­as­ters that oc­curred at Sanofi un­der the tu­mul­tuous reign of CEO Chris Viehbach­er. But af­ter im­plod­ing with spec­tac­u­lar ef­fect right at the thresh­old of a new drug ap­pli­ca­tion four years ago, one of the drug’s co-in­ven­tors — a sci­en­tist named John Hood — has pa­tient­ly picked up all the shat­tered pieces and put it to­geth­er in­to a new com­pa­ny that just scored a $22.5 mil­lion ven­ture round.

Now Hood’s lit­tle biotech is go­ing af­ter that FDA/EMA ap­proval that elud­ed the phar­ma gi­ant.

If he’s right, Hood has scored one of the biggest biotech coups of the year. But let’s start this sto­ry with a lit­tle back­ground.

Sev­en years ago, Sanofi scooped up a biotech called Targe­Gen in a $635 mil­lion deal, bag­ging a mid-stage JAK2 myelofi­bro­sis drug — then dubbed TG 101348 — that had gath­ered promis­ing ear­ly-stage hu­man da­ta. Hood, the for­mer re­search chief at Targe­Gen and a co-in­ven­tor of the drug, moved on to co-found a biotech uni­corn com­pa­ny called Sa­mumed, fo­cused on re­gen­er­a­tive med­i­cine with Hood tak­ing the CSO job.

In 2013, the FDA dropped a clin­i­cal hold on fe­dra­tinib af­ter a few pa­tients be­gan to de­vel­op Wer­nicke’s en­cephalopa­thy — a dan­ger­ous neu­ro­log­i­cal con­di­tion tied to vi­t­a­min B de­fi­cien­cy. And Sanofi’s top team quick­ly put a bul­let in it, scrap­ping a pro­gram that in­clud­ed sev­en on­go­ing clin­i­cal tri­als. The phar­ma gi­ant shoved the mor­tal­ly wound­ed pro­gram on the shelf, and that’s where it sat un­til ear­ly 2016, when Hood de­cid­ed to start his own com­pa­ny, get the rights back and pur­sue transat­lantic ap­provals.

To hear Hood tell it, af­ter the clin­i­cal tri­als end­ed, the pa­tients in­volved were shift­ed to stan­dard of care and be­gan to re­lapse.

“That’s when physi­cians and pa­tients be­gan to reach out to me,” Hood says, look­ing for some way to get the drug un­der com­pas­sion­ate use rules. That in­spired him to go for it.

I hap­pened to pick up word of what was hap­pen­ing at the time, and Hood and I start­ed ex­chang­ing oc­ca­sion­al mes­sages around his progress. Wait for it, he would write, it was grad­u­al­ly com­ing to­geth­er.

Sanofi gave up the drug for eq­ui­ty in Hood’s com­pa­ny, Im­pact Bio­med­i­cines in San Diego, which now has a lean-and-mean team of six. The pack­age in­cludes pos­i­tive Phase III re­sults (with a p-val­ue of 0.0001 says a proud Hood) da­ta from 877 pa­tients in 18 stud­ies.

“There was no out-of-pock­et,” Hood tells me. Sanofi took an eq­ui­ty stake in the biotech for its share.

The FDA came through in Au­gust, drop­ping the clin­i­cal hold af­ter Hood made the case that the side ef­fects that so alarmed in­ves­ti­ga­tors and reg­u­la­tors in 2013 could be at­trib­uted to a small num­ber of cas­es of thi­amine de­ple­tion linked to the high meta­bol­ic rate of can­cer pa­tients and mal­nour­ish­ment.

Says Hood: “It was easy enough to mit­i­gate.”

Kevin John­son

Hood’s backed by a globe-trot­ting team at Medicxi, where Kevin John­son — a buoy­ant Cam­bridge grad and sci­en­tist who had been head of re­search at the leg­endary Cam­bridge An­ti­body Tech­nol­o­gy — put to­geth­er the fi­nanc­ing round to fund what comes next.

That next part still has to be worked out, the CEO tells me. I sug­gest­ed that if any­thing, reg­u­la­tors have em­braced speed­ed up de­vel­op­ment pro­grams in on­col­o­gy, which could put him right back at the FDA’s door, knock­ing for mar­ket en­try.

Af­ter spend­ing more than 18 months putting this all to­geth­er, though, Hood is still tak­ing it one step at a time. He’ll know more about how fast he can move af­ter meet­ing with reg­u­la­tors to test their ap­petite for speed.

I asked John­son what his first thoughts were when he and Hood talked it over.

My ini­tial re­ac­tion to the sto­ry was that I couldn’t grasp the thought process that led to it be­ing put on clin­i­cal hold and there­fore not reach­ing the mar­ket. We pieced that all to­geth­er at a lat­er stage, and while it looks like a strange de­ci­sion in ret­ro­spect, at the time, with those spe­cif­ic mar­ket con­di­tions, it would have been a brave de­ci­sion to keep go­ing. Of course it’s all dif­fer­ent now.
On John him­self, he re­al­ly does think like that and I love it! It’s my ex­pe­ri­ence that the top biotech and phar­ma en­tre­pre­neurs are dri­ven by a need to do good, rather than just make mon­ey. In the end I guess it’s a be­lief sys­tem- do the right things for the right rea­sons and the mon­ey will fol­low as a con­se­quence. It does tend to work out that way. He’s al­so what I call a hand­shake guy; you shake hands on some­thing and that’s it. He could have played us but he didn’t. That too is con­sis­tent with his phe­no­type.

In most cas­es like this, Hood and his tiny band of staffers would be set­ting him­self up to do a deal on the drug. But Hood isn’t jump­ing that far ahead ei­ther. Right now, he says, he’s work­ing on the kind of fi­nan­cial pack­age that would “al­low us to take it the whole way.”

That may sound a bit far-fetched, but the whole sto­ry up to this point was im­prob­a­ble.

Hood’s just get­ting start­ed again.

Brent Saunders [Getty Photos]

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Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Image: Chris Varma. Frontier

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Two years af­ter MPM Cap­i­tal en­list­ed Chris Var­ma on its busy on­col­o­gy team, the for­mer en­tre­pre­neur-in-res­i­dence is un­veil­ing his first ven­ture project out of his new stomp­ing grounds in the Bay Area: Fron­tier Med­i­cines.

For Var­ma, who’s al­so co-found­ed Blue­print Med­i­cines and built com­pa­nies at Third Rock and Flag­ship, this marks an­oth­er op­por­tu­ni­ty to ap­ply some cut­ting-edge sci­ence to “sev­er­al of the most im­por­tant and dif­fi­cult tar­gets in can­cer” — tar­gets that oth­ers have tried to tack­le with more clas­si­cal meth­ods and failed. The launch round comes in at $67 mil­lion, which should go some way in scaf­fold­ing a pre­clin­i­cal pipeline and push one or more as­sets in­to the clin­ic three years from now, he tells me.

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An­a­lysts have been watch­ing this race for awhile now, as Sanofi/Re­gen­eron won a snap pri­or­i­ty re­view for what is now their third dis­ease in­di­ca­tion for this treat­ment. And they’re not near­ly done, build­ing up hopes for a ma­jor fran­chise.

Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

No­var­tis holds back the copy­cat brigade's at­tack on its top drug fran­chise — for now

A fed­er­al judge has put a gener­ic chal­lenge to No­var­tis’ block­buster mul­ti­ple scle­ro­sis drug Gilenya on hold while a patent fight plays out in court.

Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

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Top an­a­lyst finds a sil­ver lin­ing in Ab­b­Vie’s $63B Al­ler­gan buy­out — but there’s a catch

Af­ter get­ting beat up on all sides from mar­ket ob­servers who don’t much care for the lat­est mega-deal to ar­rive in bio­phar­ma, at least one promi­nent an­a­lyst now is start­ing to like what he sees in the num­bers for Ab­b­Vie/Al­ler­gan.

But it’s go­ing to take some en­cour­age­ment if Ab­b­Vie ex­ecs want it to last.

Ab­b­Vie’s mar­ket cap de­clined $20 bil­lion on Tues­day as the stock took a 17% hit dur­ing the day. And SVB Leerink’s Ge­of­frey Porges can see a dis­tinct out­line of an up­side af­ter re­view­ing the fun­da­men­tals of the deal.

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