Startups, Venture

Third Rock’s Ambys hauls $140M for liver disease, inking Takeda deal ahead of launch

It won’t shock anyone that a new company has rolled off Third Rock’s startup conveyor belt (this VC firm is a machine at creating new companies), but their latest venture is leaping off the start line with a distinct advantage: $140 million in launch money and a rare deal with Big Pharma partner Takeda.

The new company is called Ambys Medicines, christened after the Mexican salamander famed for regenerating limbs: Ambystoma mexicanum. You guessed it, the company will be working in regenerative medicine.

Jeff Tong

I spoke with Third Rock’s venture partner Jeff Tong, who’s serving as interim CEO for the time being, about the company’s tech. He said they’ve licensed a smattering of research from the labs of Ambys’ scientific founders, and are building an in-house R&D unit led by a couple of interim Third Rock vets sitting in as execs.

Martin Burke

The company is tackling three different avenues in liver disease: a cell therapy platform, a gene therapy, and gain-of-function small molecules. Tong said it was imperative that Ambys pursue all three areas at once. This is part of the reason they decided to partner with Takeda — to get a big chunk of cash that would support its ambitious R&D plans.

“It will allow us to pursue the three areas simultaneously,” Tong said. “They’re all ambitious, and running them in parallel is very important.”

Markus Grompe

He says many young biotechs make the fatal mistake of funneling all their cash into the first program that shows big promise. “It sucks all the resources out of the company, and then the other programs — even though they’re also promising — die on the line,” he said.

So Ambys and Third Rock designed a deal with Takeda that brought an infusion of capital to the company’s launch. Here’s the details: Takeda chipped in $100 million upfront (including participating in the company’s $60 million Series A round), bringing Ambys’ launch cash to $140 million total. In return, Takeda will get an option for ex-US rights for the first four products — whatever they may be — that reach IND at Ambys. If Takeda chooses to exercise those options, then the pharma giant also coughs up 50% of the development costs and some milestone payments come into play.

Juan Carlos Izpisua Belmonte

A key win in the deal, though, is that Ambys has a tight grip on US rights.

“We see many exciting deals being struck with significant upfronts, but the challenge for many is they give up worldwide rights on at least the first program if not more. To control the company’s destiny, you must have US rights,” Tong said.

Holger Willenbring

Scientific founders at Ambys include Martin Burke of University of Illinois, Markus Grompe at Oregon Health & Science University, Juan Carlos Izpisua Belmonte at the Salk, and Holger Willenbring from UCSF. The company’s executive team includes Third Rock’s Jeffrey Finer (CTO) and Glenn Pierce (CMO), alongside Michael Holmes as CSO and Stanley Hollenback as SVP of pharmacology.


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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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