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

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

Credit: Shutterstock

How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

James Peyer, Cambrian CEO

Brent Saun­ders joins $100M Se­ries C for a com­pa­ny out to be the Bridge­Bio of ag­ing

About a year ago, James Peyer, a CEO and co-founder of the little known longevity biotech Cambrian Biopharma, was trying to find some R&D talent last year when he met with more than a bit of experience in that department: David Nicholson, the former R&D chief of the erstwhile pharma giant Allergan.

It turned out Nicholson already had an interest in Peyer’s field. In their Allergan days, he and COO Brent Saunders held weekly meetups where they tried to figure out how to take the company’s dominance in aesthetics — which, until recently, was often what people meant by anti-aging science — and expertise with more traditional drug development, and use it to make drugs that extend people’s lifespan.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

Ugur Sahin, AP Images

As pres­sure to share tech­nol­o­gy mounts, BioN­Tech se­lects Rwan­da for lat­est vac­cine site

BioNTech’s first mRNA-based vaccine site in Africa will call Rwanda home, and construction is set to start in mid-2022, the company announced Tuesday at a public health forum.

The German company signed a memorandum of understanding, after a meeting between Rwanda’s Minister of Health, Daniel Ngamije, Senegal’s Minister of Foreign Affairs Aïssata Tall Sall, and senior BioNTech officials. Construction plans have been finalized, and assets have been ordered. The agreement will help bring end-to-end manufacturing to Africa, and as many as several hundred million doses of vaccines per year, though initial production will be more modest.

No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

Katie Fanning, Mozart Therapeutics CEO

Mozart Ther­a­peu­tics makes its of­fi­cial de­but, jump­ing in­to the hot Treg R&D field with some big-name in­vestors back­ing it

Treg cells have been getting more and more attention recently among autoimmune specialists. There’s been Jeff Bluestone’s Sonoma, the $157 million launch of GentiBio this summer and Egle Therapeutics — which launched just last week — to name a few.

Now, there’s a new Treg player jumping in that wants to distinguish itself in the market: Mozart Therapeutics. Today, the biotech is emerging from stealth in its official debut with a $55 million Series A — with a bunch of A-list Big Pharma names on board a syndicate led by ARCH.

Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

With San­doz con­tin­u­ing to drag on No­var­tis, Vas Narasimhan says he may fi­nal­ly be ready for a sale or spin­off

After years of rehab work aimed at getting Sandoz in fighting trim to compete in a market overshadowed by declining prices, CEO Vas Narasimhan took a big step toward possibly selling or spinning off the giant generic drug player.

The pharma giant flagged plans to launch a strategic review of the business in its Q3 update, noting that “options range from retaining the business to separation.”

Analysts have been poking and prodding Novartis execs for years now as Narasimhan attempted to remodel a business that has been a drag on its performance during most of his reign in the CEO suite. The former R&D chief has made it well known that he’s devoted to the innovative meds side of the business, where they see the greatest potential for growth.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.

FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 120,700+ biopharma pros reading Endpoints daily — and it's free.