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

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