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.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.