The sand­box: Park­er In­sti­tute throws its re­search mus­cle be­hind gene ther­a­py tech for de­stroy­ing can­cer cells

John Bea­dle

John Bea­dle and the big re­search team at PsiOxus have thought a lot about in­fil­trat­ing the ranks of can­cer cells, to go in­side cells to cause their self-de­struc­tion with one of the in­dus­try’s lead­ing “un­armed” on­colytc virus pro­grams. But they’re al­so go­ing one big step fur­ther, us­ing their gene ther­a­py tech to pen­e­trate these cells to de­liv­er weapons for their mass de­struc­tion. And they’re lin­ing up some pow­er­house al­lies at the Park­er In­sti­tute for Can­cer Im­munother­a­py to speed the work.

What’s the big idea?

PsiOxus — with 95 most­ly re­search staffers in Ox­ford and Philadel­phia — gained con­sid­er­able at­ten­tion for its sys­temic ap­proach to de­liv­er­ing on­colyt­ic virus­es, in­fect­ing cells that would then burst, at­tract­ing T cells in­to the tu­mor. But there are al­so scores of on­colyt­ic virus­es in the pipeline. In this case they’re work­ing a re­verse strat­e­gy to CAR-T. In­stead of mod­i­fy­ing the T cell to go af­ter can­cer cells, they’re mod­i­fy­ing the can­cer cells to get them to en­gage with T cells — en­gi­neer­ing the can­cer cell to ex­press T cell en­gag­ing lig­ands.

The ques­tion has been what genes should be added to do the best job. And they be­lieve the IV ap­proach — steer­ing clear of in­tra­tu­moral in­jec­tions — should help sim­pli­fy things con­sid­er­ably, up­ping their chances of suc­cess in hit­ting the tar­get.

PsiOxus al­ready gained the sup­port of Bris­tol-My­ers Squibb, which part­nered on the biotech’s NG-348 in a $915 mil­lion deal in late 2016. That drug en­codes two im­munomod­u­la­to­ry MiTe pro­teins in its genome: a hu­man CD80 and an an­ti­body frag­ment spe­cif­ic for the T-cell re­cep­tor CD3 pro­tein, both de­signed to muster T cells to at­tack spe­cif­ic can­cer cells on­ly.

Saman­tha Buck­trout

“They’re such a great group,” says Park­er In­sti­tute Di­rec­tor of Re­search Saman­tha Buck­trout about PsiOxus. “When we go to have dis­cus­sions, it’s a ‘no-idea-is-stu­pid’ zone, but al­so an ego-free zone. They’ve done a lot of rig­or­ous work to move their pipeline for­ward, both pre-clin­i­cal and in the clin­ic, and aren’t afraid to take risks. We see a lot of blue sky in terms of where we can go with them, sci­en­tif­i­cal­ly.”

Those blue skies are what spurred tech mogul Sean Park­er to set up his epony­mous in­sti­tute, or­ga­niz­ing a net­work of more than 300 promi­nent sci­en­tists with fi­nan­cial sup­port and cre­at­ing net­works of ex­perts to as­sist the com­pa­nies they work with to dig deep­er and go be­yond the fron­tiers of com­mer­cial I/O. But they’re al­so goal ori­ent­ed, pur­su­ing what PI­CI chief Jeff Blue­stone calls a “sand­box” strat­e­gy: a con­tained, or­ga­nized ap­proach to their work dri­ven by a set of clear ob­jec­tives.

Ex­tra fund­ing is al­ways good, says Bea­dle, but it’s the peo­ple Park­er brings to the ta­ble that make the big dif­fer­ence.

“The key is their net­work of in­sti­tu­tions and aca­d­e­mics,” notes the CEO, who ex­pects to even­tu­al­ly put to­geth­er an­oth­er round for the biotech be­fore even­tu­al­ly set­ting their sights on an IPO.

The plan at PsiOxus is to move two pro­grams in­to the clin­ic, with an IND com­ing in Q1 of next year. The first is a CD40 ag­o­nist, and the sec­ond will in­clude a pack­age of 4 dif­fer­ent genes cov­er­ing a bis­pe­cif­ic with chemokines to at­tract T cells and an­oth­er to ex­press in­ter­fer­on al­pha to ac­ti­vate den­drit­ic cells.

Robert Forrester, Verastem

Ve­rastem CEO For­rester steps to the ex­it as the board hunts com­mer­cial-savvy ex­ec for the be­lea­guered biotech

Robert For­rester is step­ping down as CEO of Ve­rastem On­col­o­gy $VSTM just 8 months af­ter the com­pa­ny nabbed an ap­proval for du­velis­ib, a PI3K drug with a sto­ried past — and what ap­pears as not much of a fu­ture.

The biotech put out word this morn­ing that For­rester will take an ad­vi­so­ry role with Ve­rastem while COO Dan Pa­ter­son steps up to take charge of the lead­er­ship team and the board looks around for a new CEO.

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.

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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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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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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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.

In­vestor day prep at Mer­ck in­cludes a new strat­e­gy to pick up the pace on M&A — re­port

Mer­ck’s re­cent deals to buy up two bolt-on biotechs — Ti­los and Pelo­ton — weren’t an aber­ra­tion. In­stead, both ac­qui­si­tions mark a new strat­e­gy to beef up its dom­i­nant can­cer drug op­er­a­tions cen­tered on Keytru­da while look­ing to ad­dress grow­ing con­cerns that too many of its eggs are in the one I/O bas­ket for their PD-1 pro­gram. And Mer­ck is go­ing af­ter more small- and mid-sized buy­outs to calm those fears.

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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