Backed by a decade of research in hemoglobin disorders, start-up firm HemaQuest Pharmaceuticals, though it was established only a couple of weeks ago, is hitting the ground running, with $20 million in Series A funding and plans for filing an investigational new drug application on its first compound by the end of the year.

The Newton, Mass.-based company aims to develop disease-modifying drugs to treat hemoglobin diseases, starting with sickle cell anemia and beta thalassemia, both of which are inherited disorders with few treatment options. HemaQuest's technology stems from a 10-year collaboration between Susan Perrine and Douglas Faller, from Boston University, and George Stamatoyannoupolous and his wife, Thalia Papayannopoulou, from the University of Washington in Seattle. That work centered on mechanisms to stimulate hemoglobin production and ultimately led to the discovery of "whole family of molecules that were active in this regard," said Ronald Berenson, president and CEO.

Those molecules have shown favorable pharmacokinetics, are orally available and, perhaps most important, have demonstrated a good safety profile, "which has been one of the major limitations" of existing therapies, especially for patients requiring chronic treatment, Berenson told BioWorld Today. He said he contacted Stamatoyannoupolous about a year ago, interested in taking those discoveries commercial. "We began putting a business plan together," he said, "and recently closed on the Series A financing."

The round included investments from Palo Alto, Calif.-based De Novo Ventures, San Diego-based Forward Ventures and Lilly Ventures, the venture capital arm of Indianapolis-based Eli Lilly & Co.

The funding could carry the company for two or three years, depending on the speed of its activities. HemaQuest anticipates submitting an IND this quarter for a lead candidate and might begin looking at other pipeline opportunities for broader types of anemia, neutropenia and other hematological disorders, possibly even hematological cancers.

Members of the firm's executive team include Berenson, Perrine as chief scientific officer, William Welch as vice president of development and George Hillman as chief operating officer. The company likely will "look to hire a few more people," but Berenson said the intention is to operate HemaQuest as a virtual model.

When asked whether the company has aspirations to create its own commercial and sales staff in the future, Berenson said, "the jury is still out." Since indications such as sickle cell anemia is a "fairly focused market," it might be possible for a small company to reach it with a concentrated sales force. But, he added, "the strength of the company lies" in its research and development expertise in blood-related disorders.