Prome­dior makes a 'break­through' with PhI­II IPF drug; NASH fo­cused NGM Bio­phar­ma sets terms for IPO; blue­bird bio says Lenti­Glo­bin is still un­der EU re­view, de­spite con­flict­ing re­port

→ Clos­ing in on 3 years since Bris­tol-My­ers $BMY struck an op­tion deal to buy Prome­dior for up to $1.25 bil­lion, the biotech says that it’s gained break­through ther­a­py sta­tus for its star fi­bro­sis drug PRM-151, now in a Phase III pro­gram for IPF. Prome­dior says that reg­u­la­tors based their de­ci­sion on pos­i­tive Phase II re­sults, which set up the new­ly launched late-stage pro­gram. Re­searchers will be us­ing forced vi­tal ca­pac­i­ty as a pri­ma­ry end­point and six-minute walk dis­tance as the key sec­ondary end­point.

Mer­ck-part­nered NASH hope­ful NGM Bio­phar­ma on Mon­day broke out the terms of its IPO. The com­pa­ny, which plans to list on the Nas­daq un­der the sym­bol NGM, is of­fer­ing about 6.7 mil­lion shares priced be­tween $14 to $16, ac­cord­ing to the fil­ing. At the mid­point of the range, the com­pa­ny would com­mand a mar­ket val­ue of more than $1 bil­lion. Net pro­ceeds from the of­fer­ing — ex­clud­ing the $62 mil­lion Mer­ck $MRK is fork­ing over in a con­cur­rent pri­vate place­ment — are ex­pect­ed to be rough­ly $89.5 mil­lion. Fel­low NASH drug de­vel­op­er Gen­fit is al­so ready­ing plans for its own IPO.

→ Looks like some­body got their wires crossed. In a strange turn of events, blue­bird bio $BLUE is­sued a state­ment that the EMA’s CHMP has not is­sued its rec­om­men­da­tion on the com­pa­ny’s gene-re­place­ment ther­a­py Lenti­Glo­bin, af­ter two char­i­ties — As­so­ci­azione Vene­ta Lot­ta al­la Ta­lassemia and the UK Tha­las­saemia So­ci­etyan­nounced on Mon­day that the keen­ly an­tic­i­pat­ed treat­ment (ex­pect­ed to car­ry a sev­en-fig­ure price tag) for be­ta tha­lassemia — a rare, in­her­it­ed blood dis­or­der — had se­cured con­di­tion­al EU ap­proval.

→ About a year fol­low­ing its US ap­proval for Crysvi­ta for pa­tients with X-linked hy­pophos­phatemia (XLH) — an in­her­it­ed form of rick­ets — in adult and pe­di­atric pa­tients, Ul­tragenyx $RARE has se­cured the first Latin Amer­i­can nod for the pricey rare dis­ease drug that has in­spired an­a­lysts to pre­dict block­buster peak sales. On Tues­day, the drug­mak­er said it had won ap­proval for the drug in Brazil. Crysvi­ta is al­so ap­proved for use in Eu­rope and Cana­da.

Eiger Bio­Phar­ma‘s $EIGR ex­per­i­men­tal GLP-1 an­tag­o­nist, avex­i­tide, has cleared a Phase II study by im­prov­ing glu­cose lev­els and sup­pressed in­sulin lev­els rel­a­tive to place­bo in post-bariatric surgery pa­tients, the com­pa­ny said on Mon­day. “With avex­i­tide in PBH (post-bariatric hy­po­glycemia) rep­re­sent­ing on­ly $0.36 of our $47 EIGR price tar­get, we re­it­er­ate that this pro­gram rep­re­sents es­sen­tial­ly a free call op­tion for in­vestors,” Baird an­a­lysts wrote in a note.

How small- to mid-sized biotechs can adopt pa­tient cen­tric­i­ty in their on­col­o­gy tri­als

By Lucy Clos­sick Thom­son, Se­nior Di­rec­tor of On­col­o­gy Pro­ject Man­age­ment, Icon

Clin­i­cal tri­als in on­col­o­gy can be cost­ly and chal­leng­ing to man­age. One fac­tor that could re­duce costs and re­duce bar­ri­ers is har­ness­ing the pa­tient voice in tri­al de­sign to help ac­cel­er­ate pa­tient en­roll­ment. Now is the time to adopt pa­tient-cen­tric strate­gies that not on­ly fo­cus on pa­tient needs, but al­so can main­tain cost ef­fi­cien­cy.

Ken Frazier appears before the Senate Committee on Finance for a hearing on prescription drug pricing on Capitol Hill in Washington, DC, February 26, 2019. Chris Kleponis for CNP via AP Images

Who’s next in line to suc­ceed Ken Fra­zier as CEO of the Keytru­da-blessed Mer­ck?

When Merck waved off a looming forced retirement for Ken Frazier last September, the board cited flexibility in CEO transition as a key factor in the decision. Having Frazier — who’s also chairman of the company — around beyond his 65th birthday in 2019 would ensure they install the best person at the best time, they said.

The board has evidently begun that process with a clear preference for internal candidates, sources told Bloomberg. CFO Robert Davis, chief marketing officer Michael Nally, and chief commercial officer Frank Clyburn are all in the running, according to an insider.

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In starved an­tibi­ot­ic field, Melin­ta soars as FDA grants speedy drug re­view

Such is the state of af­fairs in an­tibi­ot­ic land that the FDA agree­ing to pri­or­i­ty re­view an ap­pli­ca­tion to ex­pand the use of an an­tibi­ot­ic can rock­et up a stock more than two-fold.

On Wednes­day, Melin­ta Ther­a­peu­tics said its ap­proved an­tibi­ot­ic Baxdela had been grant­ed pri­or­i­ty re­view for use in com­mu­ni­ty-ac­quired bac­te­r­i­al pneu­mo­nia (CAPB). The FDA is ex­pect­ed to make its de­ci­sion by Oc­to­ber 24. Shares of the Con­necti­cut drug­mak­er $ML­NT cat­a­pult­ed, clos­ing up near­ly 224% at $6.41.

Brent Saunders at an Endpoints News event in 2017 — File photo

An­a­lyst call with Al­ler­gan ex­ecs stokes an­tic­i­pa­tion of a plan to split the com­pa­ny in ‘a month or two’

So what’s up at Al­ler­gan?

Ear­li­er this week the ubiq­ui­tous Ever­core ISI an­a­lyst Umer Raf­fat was on the line with com­pa­ny ex­ec­u­tives to probe in­to the lat­est on the num­bers as well as CEO Brent Saun­ders’ re­cent de­c­la­ra­tion that he’d be do­ing some­thing de­fin­i­tive to help long-suf­fer­ing in­vestors who have watched their shares dwin­dle in val­ue.

He came away with the im­pres­sion that a sig­nif­i­cant com­pa­ny split is on the way. And not on some dis­tant time hori­zon.

John Reed at JPM 2019. Jeff Rumans for Endpoints News

Sanofi's John Reed con­tin­ues to re­or­ga­nize R&D, cut­ting 466 jobs while boost­ing can­cer, gene ther­a­py re­search

The R&D reorganization inside Sanofi is continuing, more than a year after the pharma giant brought in John Reed to head the research arm of the Paris-based company.
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Arc­turus ex­pands col­lab­o­ra­tion, adding $30M cash; Ku­ra shoots for $100M raise

→  Rare dis­ease play­er Ul­tragenyx $RARE is ex­pand­ing its al­liance with Arc­turus $ARCT, pay­ing $24 mil­lion for eq­ui­ty and an­oth­er $6 mil­lion in an up­front as the two part­ners ex­pand their col­lab­o­ra­tion to in­clude up to 12 tar­gets. “This ex­pand­ed col­lab­o­ra­tion fur­ther so­lid­i­fies our mR­NA plat­form by adding ad­di­tion­al tar­gets and ex­pand­ing our abil­i­ty to po­ten­tial­ly treat more dis­eases,” said Emil Kakkis, the CEO at Ul­tragenyx. “We are pleased with the progress of our on­go­ing col­lab­o­ra­tion. Our most ad­vanced mR­NA pro­gram, UX053 for the treat­ment of Glyco­gen Stor­age Dis­ease Type III, is ex­pect­ed to move in­to the clin­ic next year, and we look for­ward to fur­ther build­ing up­on the ini­tial suc­cess of this part­ner­ship.”

The top 10 block­buster drugs in the late-stage pipeline — Eval­u­ate adds 6 new ther­a­pies to heavy-hit­ter list

Vertex comes in for a substantial amount of criticism for its no-holds-barred tactical approach toward wresting the price it wants for its commercial drugs in Europe. But the flip side of that coin is a highly admired R&D and commercial operation that regularly wins kudos from analysts for their ability to engineer greater cash flow from the breakthrough drugs they create.

Both aspects needed for success in this business are on display in the program backing Vertex’s triple for cystic fibrosis. VX-659/VX-445 + Tezacaftor + Ivacaftor — it’s been whittled down to 445 now — was singled out by Evaluate Pharma as the late-stage therapy most likely to win the crown for drug sales in 5 years, with a projected peak revenue forecast of $4.3 billion.

The latest annual list, which you can see here in their latest world preview, includes a roster of some of the most closely watched development programs in biopharma. And Evaluate has added 6 must-watch experimental drugs to the top 10 as drugs fail or go on to a first approval. With apologies to the list maker, I revamped this to rank the top 10 by projected 2024 sales, instead of Evaluate's net present value rankings.

It's how we roll at Endpoints News.

Here is a quick summary of the rest of the top 10:

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John Chiminski, Catalent CEO - File Photo

'It's a growth play': Catal­ent ac­quires Bris­tol-My­er­s' Eu­ro­pean launch pad, ex­pand­ing glob­al CD­MO ops

Catalent is staying on the growth track.

Just two months after committing $1.2 billion to pick up Paragon and take a deep dive into the sizzling hot gene therapy manufacturing sector, the CDMO is bouncing right back with a deal to buy out Bristol-Myers’ central launchpad for new therapies in Europe, acquiring a complex in Anagni, Italy, southwest of Rome, that will significantly expand its capacity on the continent.

There are no terms being offered, but this is no small deal. The Anagni campus employs some 700 staffers, and Catalent is planning to go right in — once the deal closes late this year — with a blueprint to build up the operations further as they expand on oral solid, biologics, and sterile product manufacturing and packaging.

This is an uncommon deal, Catalent CEO John Chiminski tells me. But it offers a shortcut for rapid growth that cuts years out of developing a green fields project. That’s time Catalent doesn’t have as the industry undergoes unprecedented expansion around the world.

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UP­DAT­ED: Chica­go biotech ar­gues blue­bird, Third Rock 'killed' its ri­val, pi­o­neer­ing tha­lassemia gene ther­a­py in law­suit

Blue­bird bio $BLUE chief Nick Leschly court­ed con­tro­ver­sy last week when he re­vealed the com­pa­ny’s be­ta tha­lassemia treat­ment will car­ry a jaw-drop­ping $1.8 mil­lion price tag over a 5-year pe­ri­od in Eu­rope — mak­ing it the plan­et’s sec­ond most ex­pen­sive ther­a­py be­hind No­var­tis’ $NVS fresh­ly ap­proved spinal mus­cu­lar at­ro­phy ther­a­py, Zol­gens­ma, at $2.1 mil­lion. A Chica­go biotech, mean­while, has been fum­ing at the side­lines. In a law­suit filed ear­li­er this month, Er­rant Gene Ther­a­peu­tics al­leged that blue­bird and ven­ture cap­i­tal group Third Rock un­law­ful­ly prised a vi­ral vec­tor, de­vel­oped in part­ner­ship with the Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter (MSK), from its grasp, and thwart­ed the de­vel­op­ment of its sem­i­nal gene ther­a­py.