The FDA is get­ting its hands on a ‘hu­man em­u­la­tion sys­tem,’ and Em­u­late hopes they nev­er let go

The fu­ture for the or­gans-on-chips in­dus­try will de­pend to a large ex­tent on the FDA’s abil­i­ty to un­der­stand how it works as well as its prac­ti­cal, near-term po­ten­tial for re­plac­ing an­i­mals in pre­clin­i­cal re­search as the sys­tem evolves. And now reg­u­la­tors are about to get some hands-on ex­pe­ri­ence with a col­lab­o­ra­tive ef­fort to build a hu­man em­u­la­tion sys­tem in­side the agency.

The FDA has agreed to work with Em­u­late, a spin­off of the Wyss In­sti­tute which has been de­vel­op­ing and im­prov­ing on the in­dus­try stan­dard in the field, snag­ging part­ner­ships with the likes of Mer­ck and J&J. The work will be done un­der a Co­op­er­a­tive Re­search and De­vel­op­ment Agree­ment (CRA­DA), cre­at­ing a plat­form for tox test­ing for prod­ucts like foods, di­etary test­ing and cos­met­ics.

“The fo­cus is to as­sess the sys­tem’s ca­pa­bil­i­ty,” says Geral­dine Hamil­ton, pres­i­dent and CSO, “putting the chips, soft­ware and in­stru­men­ta­tion in the hands of FDA re­searchers, so they can bet­ter un­der­stand its po­ten­tial in sur­pass­ing an­i­mals for this kind of work.”

The agree­ment calls for a full-time com­mit­ment of five staffers at the FDA for three years, with a sim­i­lar com­mit­ment com­ing from Em­u­late.

This part­ner­ship isn’t about re­plac­ing an­i­mals for tox­i­col­o­gy stud­ies in pre­clin­i­cal drug re­search, but when I sug­gest­ed that would be the ul­ti­mate goal for where the col­lab­o­ra­tion could be head­ed, giv­ing the FDA more con­fi­dence in its ap­pli­ca­tions, she quick­ly con­curred.

“That is ex­act­ly the pur­pose,” says Hamil­ton.

The tech al­so isn’t ex­act­ly brand new to the FDA.

“Suzanne Fitz­patrick, se­nior ad­vi­sor for tox­i­col­o­gy at the FDA,” adds the CSO, “has been in­volved in col­lab­o­ra­tive re­search ef­forts since the time the tech­nol­o­gy was un­der de­vel­op­ment at the Wyss In­sti­tute at Har­vard.” But it is a big step for­ward.

“This is the start­ing point,” says Hamil­ton. “We are al­ready do­ing sim­i­lar ef­forts with in­dus­try part­ners, in the food in­dus­try, on very sim­i­lar col­lab­o­ra­tions.”

Fitz­patrick had this to say in a blog post to­day:

In some ways, sci­ence is like a recipe in that both can go through a num­ber of in­car­na­tions be­fore they work. There’s a lot of ex­per­i­ment­ing and tweak­ing, col­lab­o­rat­ing and com­par­ing. And that’s what we’ll be do­ing at FDA with the or­gans-on-chips re­search. Sci­ence is the foun­da­tion of FDA’s de­ci­sions but many peo­ple don’t re­al­ize how much sci­en­tif­ic re­search is con­duct­ed by the agency. We’re ex­cit­ed to be at the fore­front of this ground-break­ing re­search, which may one day be rou­tine­ly used to safe­guard pub­lic health.

Birthing a new tech­nol­o­gy is no quick or easy prospect. But Em­u­late is mak­ing mea­sur­able progress and plans on more high-pro­file part­ner­ships lat­er in the year.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Sanofi/Re­gen­eron mus­cle ahead of a ri­val No­var­tis/Roche team, win first ap­proval in key rhi­nos­i­nusi­tis field

Re­gen­eron and their part­ners at Sanofi have beat the No­var­tis/Roche team to the punch on an­oth­er key in­di­ca­tion for their block­buster an­ti-in­flam­ma­to­ry drug Dupix­ent. The drug team scored an ac­cel­er­at­ed FDA ap­proval for chron­ic rhi­nos­i­nusi­tis with nasal polyps, mak­ing this the first such NDA for the field.

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. 

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.

FDA re­jects Ac­er's rare dis­ease drug, asks for new tri­al — shares crater

Ac­er Ther­a­peu­tics’ bid to re­pur­pose celipro­lol — a be­ta-block­er on the mar­ket for hy­per­ten­sion — as a treat­ment for a rare, in­her­it­ed con­nec­tive tis­sue dis­or­der has hit a se­vere set­back. The New­ton, Mass­a­chu­setts-based com­pa­ny on Tues­day said the FDA re­ject­ed the drug and has asked for an­oth­er clin­i­cal tri­al.

The com­pa­ny’s shares $AC­ER cratered near­ly 77% to $4.47 in Tues­day morn­ing trad­ing.

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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