Ambys Medicines has raised $60m in a series A round backed by Takeda and signed a strategic partnership agreement with the corporate worth $80m.
Ambys Medicines, a US-based regenerative medicine developer focused on serious liver diseases, may not be a typical spinout but it is the latest company emerging from venture capital firm Third Rock Ventures’ unique “discover, launch, build” model.
The approach has the firm collaborate with researchers to identify high-potential projects before helping them launch their company with significant series A funding and assisting their growth in the marketplace through a network of pharmaceutical partners.
Third Rock will be familiar to Global University Venturing readers – its portfolio includes companies such as Celsius Therapeutics, a developer of treatments for cancer and autoimmune diseases, cancer therapy developer Relay Therapeutics and cancer-focused biopharmaceutical company Constellation Pharmaceuticals.
Ambys Medicines is the latest addition, having been launched last week with $140m in total financing, made up of $60m in series A capital provided by Third Rock and pharmaceutical company Takeda, with an additional $80m committed by the corporate as part of a strategic partnership.
The line-up behind the company is impressive – the scientific co-founders hail from four institutions, a level of cooperation seldom seen in technology transfer, illustrating just how compelling Third Rock’s approach is.
The researchers include Martin Burke, professor of chemistry at University of Illinois at Urbana-Champaign, and Markus Grompe, Ray Hickey professor and director of the Papé Family Paediatric Research Institute as well as director of the Oregon Stem Cell Centre at Oregon Health and Science University.
The rest of the team are Juan Carlos Izpisua Belmonte, professor and Roger Guillemin chair of the Gene Expression Laboratories at research institute Salk Institute for Biological Studies, and Holger Willenbring, professor of surgery at University of California San Francisco.
They have been joined by Jeffrey Tong, interim chief executive, Michael Holmes, chief scientific officer, Jeffrey Finer, interim chief technology officer, Glenn Pierce, interim chief medical officer, and Stanley Hollenbach, senior vice-president of pharmacology.
Tong and Finer are venture partners at Third Rock, while Pierce is an entrepreneur-in-residence.
Ambys Medicines will develop treatments for chronic liver diseases, including cell therapy for hepatocyte transplantation, gene therapy for liver regeneration and drug therapy to replace lost protein function.
Of these approaches, the first is a particularly intriguing technique. It involves transplanting hepatocytes, cells that make up between 70% and 85% of the liver and are responsible for its core functions – metabolism, storage, digestion and bile production. The necessity for such alternative transplants has arisen from a shortage of donor organs.
Tong said: “With the launch of Ambys, we are seizing the opportunity to radically reshape the way in which serious liver diseases are treated.
“Major advances in the fields of gene and cell therapy and gain-of-function drug therapies, coupled with deeper understanding of liver biology, create the potential to develop drug therapies that can now restore or replace natural liver function.”
There is another unusual aspect about Ambys – the involvement of Takeda. The Japan-based drug developer may not be a stranger to corporate venturing activities, but it is rarely involved in larger rounds.
GCV Analytics, the data analytics platform operated by our sister site Global Corporate Venturing, shows that since the beginning of 2011, out of more than 30 deals in which it has participated, Takeda has taken part in only four rounds of $60m or more. The following table shows the 10 biggest rounds backed by Takeda since January 2011.
The fact that Takeda has put its weight behind not only Ambys’s $60m series A round but also a collaboration deal worth $80m signals how convinced the corporate must be of the startup’s potential. In fact, Takeda’s total commitment is $100m, meaning it supplied a third of that series A round.
Asit Parikh, head of the gastroenterology therapeutic area unit at Takeda and on the board of Ambys, said: “This is a unique confluence in time to bring together multiple regenerative approaches to tackle very serious unmet needs remaining in chronic liver disease today. We are excited to be joining Ambys at the launch of this exciting company.”
Takeda stands to gain big if its bet pays off – the corporate has secured an option to the international commercialisation rights of the first four drug candidates that receive an investigational new drug status from US regulator the Food and Drug Administration under the partnership. If it exercises an option, Takeda will be responsible for 50% of development costs and will be required to make development and regulatory milestone payments. Ambys, meanwhile, will retain the full rights to commercialising any drug in the US.
Tong added: “We are thrilled that Takeda has joined us at the outset to develop our vision to build a transformative approach to liver diseases. 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.”
Ambys is off to a promising start – the money is expected to give the company a four to five-year runway and the goal will be to push several drug candidates into human clinical trials during that period. With dozens of diseases affecting the liver, Ambys’s broad approach could prove a game-changer.
