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

Jean-Paul Clozel, Idorsia CEO (Patrick Straub/Keystone via AP Images)

Idor­si­a's brain bleed drug flunks PhI­II tri­al, a decade af­ter pre­vi­ous flop

Idorsia’s long journey with clazosentan came to an abrupt “unexpected result” Monday morning with a Phase III flop.

The Swiss biopharma said the drug did not meet the main goal of the late-stage REACT study, conducted in the US, Canada and Europe since early 2019.

The 409-patient trial tested the intravenous drug’s ability to prevent complications due to delayed cerebral ischemia following aneurysmal subarachnoid hemorrhage (aSAH), in which blood vessels in the brain narrow and blood accumulates around the brain’s surface, which then dials up the pressure on the brain.

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Kenji Yasukawa, Astellas CEO (Photographer: Akio Kon/Bloomberg via Getty Images)

Astel­las taps chief strat­e­gy of­fi­cer as next CEO to 'go on the ag­gres­sive'

Five years into its big R&D revamp, Astellas says it’s time for a changing of the guard.

Kenji Yasukawa, who took over as president and CEO in 2018, will step down to become chairman of the board in April, making room for Naoki Okamura to take over. Okamura joined the company in 1986 and has served in a variety of finance, business and strategy roles, including most recently as chief strategy officer.

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Clin­i­cal tri­al di­ver­si­ty da­ta show mis­match be­tween en­roll­ment and dis­ease preva­lence, GSK says

A lack of diversity in clinical trials has persisted despite decades of initiatives to try to turn the tide.

In a recent review of 17 years of clinical trials, drugmaker GSK found that there were some mismatches between the demographics of its US-based trials and how prevalent diseases were in those populations.

The results, the company says, will help GSK and others design studies that better represent epidemiological rates within races and ethnicities.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

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After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

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