Ambys Medicines Completes $47 Million Series A Extension

SOUTH SAN FRANCISCO — Ambys Medicines, a company pioneering cell-based therapies for severe liver diseases, has completed a $47 million extension of its Series A financing. This brings the total Series A funding raised by Ambys to $107 million.

Ambys is developing an entirely new approach to treating severe liver disease. The company’s liver-cell production processes can produce, at scale, high-quality liver cells that function in vivo as normal healthy liver cells. The company is advancing multiple preclinical programs. The proceeds from this financing will enable the continued expansion of Ambys’s proprietary liver cell therapy production capabilities and the advancement of the company’s lead program, AMI-918, through pre-IND activities. Third Rock Ventures led the financing with participation from Takeda, Schroders Capital, Laurion Capital, Smilegate Investment, and Alexandria Venture Investments, among others.

“Serious liver diseases affect tens of millions of people worldwide, with most patients experiencing severe, irreversible outcomes and a median overall survival of just two years,” said Ronald Park, M.D., Chief Executive Officer of Ambys Medicines. “This takes a tremendous toll on both patients and their family members. Beyond the human toll, liver diseases drive a huge portion of societal health spending. It’s one of the biggest problems in healthcare but has received much less focus than it deserves. At Ambys, we’re working to redefine how severe liver diseases are treated, and we’ve made important progress over the last three years towards our goal. Importantly, we’ve presented data validating our ability to expand mature, fully functioning liver cells that maintain biological function and are core to our therapeutic approach. And we’ve opened our state-of-the-art manufacturing facility to enable industrial-scale cGMP production of our therapeutic liver cells. Our focus now is on advancing our lead program to the clinic in 2023 and progressing our follow-on programs.”

The liver is one of the most regenerative organs in the body. When it loses regenerative capacity, patients start on the irreversible path of disease. Decades of clinical experience have shown that when patients receive adequate quantities of high-quality liver cells, those cells can support or even replace liver function. Until now, however, it hasn’t been possible to produce mature liver cells in sufficient quantities needed for therapeutic purposes. Amby’s liver-cell production processes overcome this challenge. The company has already demonstrated the ability to produce at scale high-quality liver cells that function in vivo as normal healthy liver cells.

“Ambys’s technology is a breakthrough in cell production with the potential to solve an enormous medical problem,” said Erwin Boos, PharmD, Head of Biotech Venture Investments at Schroders Capital. “There is still significant work to validate this approach in the clinic, but we believe strongly in the underlying science, which may enable a true breakthrough in medicine, and we believe just as strongly in the passion of the Ambys team to deliver on the promise of their technology.”

Amby’s first program, AMI-918, is an allogeneic liver-cell therapy to manage acute liver disease. This therapy would provide a potential life-extending solution to the 100,000 patients in the U.S. alone who, each year, experience liver function crises by improving outcomes, reducing re-hospitalization rates, and extending the time to liver transplantation.

Amby’s second program is designed to bring the promise of its liver-cell platform to many more patients by providing extended durability of replacement cells and easier dosing and administration without the need for immunosuppressive therapy, which is intended to enable access to the broader population of patients with chronic liver disease. This follow-up program aims to slow disease progression and ultimately reverse disease in patients with decompensated liver disease. This advanced technology has the potential to benefit more than a half-million liver disease patients in the U.S. alone each year.

“When we started this effort, it was a moonshot, given the tremendous challenge of producing the types of cells needed at scale. With the opening of our production facility earlier this year, we are well on our way to overcoming that hurdle and will be able to produce high-quality, mature liver cells to support clinical development and early commercialization activities,” said Markus Grompe, M.D., Chief Scientific Officer of Ambys Medicines. “With that gate lifted, we are in the process now of working through pre-IND activities as we prepare to move AMI-918 into clinical development in 2023.”

Liver disease has many causes, including viral infections, cancer or other tumors, inherited diseases, or exposure to toxins (including poisons, drugs, or alcohol). In most instances, liver cells lose their ability to function normally. And, without a properly functioning liver, a host of serious medical issues arise from the inability to metabolize foods and medicines, process fats, and store energy. As liver function declines, patients become critically ill.

Worldwide, more than 50 million people live with chronic liver disease, and liver diseases account for more than 2 million deaths per year worldwide. And while liver transplantation is the second most common solid organ transplantation, less than 10% of patients in need of a transplant will receive one. Liver disease is severely compromising to those affected, resulting in high social and economic costs.