Vir chief Scan­gos picks Her­bert ‘Skip’ Vir­gin to run R&D; Arde­lyx forges $160M deal with Ky­owa Hakko Kirin

Her­bert “Skip” Vir­gin

A month ago George Scan­gos blazed through a biotech buy­out, two bil­lion-dol­lar drug de­vel­op­ment deals and more than $500 mil­lion in fi­nanc­ing to build a pipeline of new ther­a­pies for in­fec­tious dis­eases at his start­up biotech Vir. Now he’s se­lect­ed Her­bert “Skip” Vir­gin as R&D chief and CSO. Vir­gin is join­ing Vir from Wash­ing­ton Uni­ver­si­ty School of Med­i­cine in St. Louis, Mis­souri, where he has served as the Ed­ward Mallinck­rodt Pro­fes­sor and Chair of the De­part­ment of Pathol­o­gy & Im­munol­o­gy since 2006.

Arde­lyx $ARDX is pick­ing up a $30 mil­lion up­front in a pact with Ky­owa Hakko Kirin cov­er­ing the Japan­ese rights to its late-stage drug tena­panor for car­diore­nal dis­eases. The li­cens­ing deal al­so in­cludes $130 mil­lion in mile­stones and high-teens for the roy­al­ties.

PNC Bank is ac­quir­ing The Trout Group, the in­vestor re­la­tions and strate­gic ad­vi­so­ry firm that boasts a glob­al list of clients in­clud­ing Toca­gen, Gen­fit and Zai Lab. Un­der the de­fin­i­tive agree­ment, an­nounced Mon­day, Trout will be com­bined with PNC sub­sidiary Sole­bury Com­mu­ni­ca­tions — a sim­i­lar but not in­dus­try-spe­cif­ic firm — to be­come Sole­bury Trout Com­mu­ni­ca­tions. Trout founder/CEO Jonathan Fass­berg will be re­tained as one of three co-CEOs, serv­ing along­side Sole­bury’s Jeff Gross­man and Lisa Wol­ford. The new com­pa­ny will be dou­ble the size of 30-strong Trout, and will like­ly ex­pand ac­cess to biotech bank­ing busi­ness for PNC.

→ Lim­it­ed sup­ply of cus­tomized virus­es cru­cial to the de­liv­ery of gene ther­a­py is hold­ing up biotechs de­vel­op­ing such nov­el treat­ment, The New York Times re­ports. With an ex­pen­sive and ar­du­ous pro­ce­dure, the few firms ca­pa­ble of man­u­fac­tur­ing these dis­abled virus­es for clin­i­cal tri­als are “swamped with or­ders and re­quests.” A GEN col­umn pub­lished ear­li­er this month de­scribes the is­sue as a “ca­pac­i­ty crunch,” with Bio­Plan As­so­ci­ates es­ti­mat­ing that the CMO short­fall in world­wide cell/gene ther­a­py is 5 times cur­rent ca­pac­i­ty. A ready source of virus is be­com­ing para­mount; so much so that Bio­Marin has de­cid­ed to build its own man­u­fac­tur­ing plant. As CMO Mil­li­pore­Sig­ma’s Udit Ba­tra tells the NYT, “It’s a re­al is­sue.”

Scot­land’s Sym­promics is col­lab­o­rat­ing with UCL on a gene ther­a­py for Parkin­son’s dis­ease. Their first tar­get look­ing to con­trol gene ex­pres­sion will be Young-On­set Parkin­son’s dis­ease.

Gilead’s Solval­di is fi­nal­ly sell­ing in Chi­na, and the biotech gi­ant $GILD is charg­ing just shy of $9,000 for a 12-week reg­i­men — a frac­tion of the US price of $84,000. The launch, first re­port­ed by Chi­nese out­lets, comes two months af­ter the he­pati­tis C drug nailed an ap­proval from the CF­DA. Chi­na ac­counts for 10 out of 130 mil­lion hep C pa­tients world­wide, Caix­in ob­serves. Solval­di is the first di­rect an­ti-vi­ral agent in Chi­na, but it won’t be the last. Bris­tol-My­ers Squibb $BMY and John­son & John­son $JNJ are al­ready rac­ing to mar­ket their own prod­ucts af­ter nab­bing CF­DA ap­provals on Gilead’s heels.

→ The FDA has giv­en Cy­tomX $CT­MX the OK on its IND for the com­pa­ny’s CT­LA-4 pro­body ther­a­peu­tic, be­ing de­vel­oped in part­ner­ship with Bris­tol-My­ers Squibb. CT­LA-4 is the tar­get of BMS’ check­point in­hibitor Yer­voy, and is the first tar­get to get to the clin­ic un­der the com­pa­nies’ part­ner­ship formed in 2014. The IND ac­cep­tance gets Cy­tomX a $10 mil­lion mile­stone pay­ment, bring­ing the to­tal pay­ments from the deal to over $275 mil­lion — with $4.8 bil­lion still avail­able in po­ten­tial mile­stones.

→ Cincin­nati biotech Cin­Rx just closed $10 mil­lion in a Se­ries B to ad­vance sev­er­al drug can­di­dates in di­verse in­di­ca­tions, rang­ing from large mar­kets to or­phan in­di­ca­tions. The com­pa­ny plans to file an IND in the first half of 2018 for its most ad­vanced pro­gram, CIN-102, for the treat­ment of gas­tro­pare­sis. This lat­est round, led by undis­closed new and ex­ist­ing in­vestors, fol­lows a $26 mil­lion Se­ries A closed in 2015. The com­pa­ny’s pipeline in­cludes treat­ments for gas­troin­testi­nal dis­or­ders in­clud­ing IBS, along with car­dio­vas­cu­lar dis­ease and der­ma­tol­ogy.


Brit­tany Meil­ing and John Car­roll con­tributed to this re­port.

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II 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.

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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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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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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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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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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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.