Japan­ese bil­lion­aire Hi­roshi Mik­i­tani bankrolls As­pyr­i­an with a $150M mega-round, back­ing a glob­al roll­out plan for PhI­II can­cer ther­a­py

Among the world’s bil­lion­aires, Hi­roshi Mik­i­tani is one of the most promi­nent en­thu­si­asts. Start­ing with 6 staffers and a small sum of cash, he turned Rakuten in­to an on­line pow­er­house in Japan that is ranked among the world’s top e-tail­ers. An icon­o­clast in an econ­o­my dom­i­nat­ed by tra­di­tion­al­ists, he’s man­dat­ed that every­one in his com­pa­ny learn and speak Eng­lish as their first lan­guage. His re­cent ven­tures in­clude a part­ner­ship with Wal­mart for an on­line su­per­mar­ket in Japan. 

And he’s worth an es­ti­mat­ed $7 bil­lion.

Now, Mik­i­tani is putting in the li­on’s share of a $150 mil­lion mega-round in­to a small, pre­vi­ous­ly low-pro­file San Diego biotech he di­rects as chair­man of the board, adding to the eq­ui­ty he had al­ready bought up. And he’s putting some of that trade­mark gung-ho en­thu­si­asm be­hind a new ven­ture that is jump­ing from a small, sin­gle-arm Phase I/II tri­al in ad­vanced, treat­ment-re­sis­tant cas­es straight in­to a Phase III pro­gram.

A chunk of that new mon­ey is be­ing re­served for build­ing the foun­da­tion of their com­mer­cial pro­gram for ASP-1929, ahead of any mid-stage demon­stra­tion of proof-of-con­cept suc­cess. And Rakuten As­pyr­i­an is go­ing all out to build a ful­ly in­te­grat­ed out­fit, with plans to de­vel­op a pipeline of ther­a­peu­tic pro­grams that it plans to mar­ket on its own, with of­fices in the US, Japan and Eu­rope.

Miguel Gar­cia-Guz­man

CEO Miguel Gar­cia-Guz­man tells me that the mid-stage por­tion of ef­fi­ca­cy and safe­ty da­ta from their 42-pa­tient study of their pho­toim­munother­a­py is weeks away from a pub­lic un­veil­ing. But it’s all for­ward mo­tion now that the com­pa­ny has raised a to­tal of $238 mil­lion.

“We have enough con­fi­dence to move in­to a Phase III straight on,” says Gar­cia-Guz­man, point­ing to their lead ef­fort on an EGFR-tar­get­ing ther­a­py for head and neck squa­mous cell car­ci­no­mas. New mid-stage stud­ies are al­so be­ing set up for oth­er can­cers with an EGFR tar­get, which is quite com­mon.

Why the ear­ly con­fi­dence?

The Japan­ese bil­lion­aire is back­ing a new tech­nol­o­gy that was de­vel­oped at the Na­tion­al Can­cer In­sti­tute in the lab of Hisa­ta­ka Kobayashi, an imag­ing ex­pert who made a some­what serendip­i­tous dis­cov­ery that con­ju­gat­ing an an­ti­body with a dye called IRDye700DX (IR700), in­fus­ing it in­to pa­tients and then hit­ting it with a near in­frared light would cre­mate can­cer cells with­out off-tar­get tox­i­c­i­ty. Kobayashi out-li­censed it to As­pyr­i­an Ther­a­peu­tics, which now goes by the name of Rakuten As­pyr­i­an.

Mik­i­tani be­came fa­mil­iar with the work at the NCI as he was hunt­ing down a bet­ter ther­a­py for his fa­ther, who was dy­ing of pan­cre­at­ic can­cer. And while it was too late to save his fa­ther, he seized on it as the next big thing in can­cer, back­ing As­pyr­i­an from the be­gin­ning.

There’s been a lot more work on the tech­nol­o­gy since those ear­ly days at the NCI, says the CEO. The com­pa­ny has been work­ing on laser tech and dif­fer­ent fiberop­tics “to il­lu­mi­nate large in­ter­nal tu­mors, which can be eas­i­ly im­plant­ed in the tu­mor.” Once the fiberop­tics are in­side the tu­mor, you “switch on the light for 5 min­utes (a day af­ter the in­fu­sion), and treat­ment is com­plete.”

If the an­ti­body con­ju­gate is bound to can­cer cells, says Gar­cia-Guz­man, cell mem­brane in­tegri­ty is de­stroyed and necro­sis is trig­gered.

“This is not a phar­ma­co­log­i­cal ef­fect,” he adds, “it’s more bio­phys­i­cal, this al­lows a very tar­get­ed ap­proach.”

Mik­i­tani is the on­ly named in­vestor in this round, but the CEO tells me that he’s the lead play­er, with a ma­jor­i­ty in­ter­est in the com­pa­ny. Oth­er pri­vate in­vestors have stepped in, but Gar­cia-Guz­man says the biotech has steered clear of the VC set, de­ter­mined to con­trol their own fu­ture and main­tain their in­de­pen­dence.

With Mik­i­tani’s sup­port, the com­pa­ny has swelled from a start­up crew of 10 a cou­ple of years ago to 85 now. By the end of this year, you can ex­pect 100 to 110 on the pay­roll. And they plan to keep on grow­ing ag­gres­sive­ly, look­ing to build out all the func­tions of a biotech up­start with glob­al as­pi­ra­tions.

That’s a tall or­der. But Mik­i­tani seems to spe­cial­ize in tall or­ders. And the staff at As­pyr­i­an is all in.

Im­age: Hi­roshi Mik­i­tani at a con­fer­ence in 2018. AP IM­AGES

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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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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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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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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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

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.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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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.