Ambys Medicines Debuts in Unusual Deal with Takeda Pharmaceutical

Jeff Tong_Ambys CEO

Jeff Tong, Ambys CEO / Tri Nguyen / Ambys Medicines 

Ambys Medicines, based in Redwood City, California, has launched in an unusual deal with Osaka, Japan’s Takeda Pharmaceutical. First off, Ambys closed on a Series A round worth $60 million from Third Rock Ventures and Takeda. That’s fairly typical.

What is a bit unusual is Takeda is also investing another $80 million in capital to help Ambys fund its research. As part of that deal, Takeda holds an option to commercialization rights outside the U.S. for the first four products that hit the level of an investigational new drug (IND) application developed under the partnership. And, if Takeda chooses to exercise an option for a product, it will share in 50 percent of the development costs and make development and regulatory milestone payments. Ambys holds all U.S. rights.

Ambys is named after the Mexican salamander, Ambystoma mexicanum, that can regenerate its limbs. The company’s focus is, appropriately enough, on regenerative medicine. It has three different programs in liver disease: cell therapy, gene therapy, and gain-of-function small molecules.

This three-shots-on-goal approach is partly why the company went with the big relationship with Takeda, because it allows Ambys to run three programs simultaneously. The company’s chief executive officer and Third Rock venture partner Jeff Tong told Xconomy this gives the company enough cash to operate for a “minimum of four or five years. As long as the programs are productive, this is a company that doesn’t need to worry about its finances.”

All programs are in early development, but Tong hopes the company will be ready to move at least one of the programs into the clinic in the next couple years.

The company’s focus on liver diseases aligns with Takeda’s focus on gastroenterology. “This partnership underscores the exciting potential we see to deliver on the promise of regenerative medicine for people with liver disease,” said Asit Parikh, head, Gastroenterology Therapeutic Area Unit at Takeda, in a statement.

Scientific founders of Ambys include Martin Burke of the University of Illinois, Markus Grompe of Oregon Health & Science University, Juan Carlos Izpisua Belmonte at the Salk Institute for Biological Studies, and Holger Willenbring from the University of California, San Francisco.

Tong told Xconomy that what was unusual about the deal was that Ambys received a large amount of cash up front, but “always retains its independence.”

The deal did not include buyout clauses, for example, meaning if the drugs were promising, Takeda would have an option to buy all or part of the company. In this case, Takeda is investing in the programs with commercialization and development clauses, but no obligation or option to buy the company outright.

“We are thrilled that Takeda has joined us at the outset to develop our vision to build a transformative approach to liver diseases,” Tong said in a statement. “Ambys will undertake an intense and sustained effort to advance fundamental science and technology, while developing multiple programs aimed at diverse liver disease targets. We are uniting a broad range of scientific innovators to help lead a new era of discovery and clinical translation for people with severe liver diseases, and we are delighted to join forces with Takeda in this important effort.”

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