Red blood cells need iron to carry oxygen from the lungs to the rest of the body, and having too little or too much can lead to serious health problems.
When iron levels go awry, the number of healthy red blood cells circulating in the bloodstream shrinks, causing a condition called anemia. Disc Medicine, a new biotech that aims to treat anemia caused by a variety of blood disorders, launched out of Cambridge, MA venture firm Atlas Venture on Tuesday with $50 million. The company is developing drugs to impact hepcidin, a liver hormone that helps the body regulate iron absorption.
Atlas teamed up with Brian MacDonald, Disc’s founder and interim CEO, to start the company and make new treatments for anemia. Disc, which is also based in Cambridge, plans to use the Series A financing round to advance two treatments for a range of disorders into human testing sometime in 2021.
For MacDonald, Disc represents a second crack at turning discoveries made in the field of hepcidin biology and iron metabolism into new medicines. Previously he founded and ran Merganser Biotech, which licensed a class of peptides that act like hepcidin from the lab of Tomas Ganz and Elizabeta Nemeth at UCLA, where the molecules were discovered. But when Merganser began Phase 1 testing of the drug it had developed as a treatment for the genetic blood disorder beta thalassemia and other diseases, it didn’t make it through that initial study.
“There was an unanticipated safety issue related to administration of the drug,” which was being administered subcutaneously, MacDonald says.
Around the time that company was winding down, however, he found out that the Atlas group was also looking at hepcidin as a potential target for new drugs.
“It was kind of a happy meeting, where I was able to reuse all the learnings I’d gained at the first company at the same target using a very different approach,” he says. “Sometimes the first idea is not necessarily the one that works, and I’m hoping that that’s the case for me, that we have more success the second time.”
Disc has been working to develop an oral small-molecule drug that would increase hepcidin levels by targeting a protein, hemojuvelin, that plays a role in production of the hormone. The idea is that by doing so, it could treat anemia caused by disorders that result in a dangerous build-up of iron in the body, MacDonald says. (Levels of hepcidin and iron are inversely related.)
Some patients with damaged red blood cells, such as those with beta thalassemia, end up with anemia and too much iron, because of the regular blood transfusions they require. Consequently, patients typically require additional treatment to remove the excess iron.
Injectable biologic drugs for anemia, like Amgen’s epoetin alfa (Epogen) and darbapoeitin alfa (Aranesp), have been around for years. But other biotechs, such as FibroGen (NASDAQ: FGEN) and Akebia Therapeutics (NASDAQ: AKBA), are racing to replace those blockbuster treatments with an anemia pill.
With its $50 million financing, Disc has also hedged its bet on developing an anemia pill. On Tuesday it announced it had licensed a series of AbbVie (NYSE: ABBV) antibodies designed to decrease hepcidin—thereby raising patients’ iron levels—to treat anemia related to a range of chronic inflammatory and hematologic diseases, such as kidney disease and rheumatoid arthritis. Terms of the deal weren’t disclosed, but it means the startup is advancing a small-molecule and an antibody treatment in parallel.
“We felt that it was important, particularly knowing my experience at Meganser, to have a second project,” MacDonald says.
A prospective drug developed from the AbbVie antibodies is slated to reach human testing first, in early 2021; Disc anticipates its anemia pill will enter clinical trials later that year, MacDonald says.
The venture arm of European diabetes drug giant Novo Nordisk (NYSE: NVO) led Disc’s Series A financing. Atlas and Access Biotechnology, the life sciences investment arm of US industrial group Access Industries, also participated.