LOS ANGELES, Calif.—Capricor Therapeutics, Inc. today announced that it has executed a collaboration agreement and exclusive license option with Janssen Biotech, Inc. Capricor's stock immediately rose more than 20 percent. Under the terms of the agreement, Capricor and Janssen will collaborate on the development of Capricor's cell therapy program for cardiovascular applications, including its lead product, CAP-1002. CAP-1002 is an allogeneic cardiosphere-derived cell (CDC) therapeutic under evaluation in patients who have suffered a large myocardial infarction. Pursuant to the agreement, Capricor and Janssen will collaborate on elements of cell manufacturing development. Capricor will contribute to the costs of the manufacturing collaboration, and will receive an upfront payment of $12.5 million from Janssen.
Under the terms of the agreement, Janssen has the right to enter into an exclusive license agreement for CAP-1002 at any time until sixty days following delivery by Capricor of the six-month follow-up results from Phase 2 of Capricor's ALLSTAR clinical trial for CAP-1002. If Janssen exercises its option rights, Capricor will be eligible to receive up to $325 million in additional payments. In addition, a royalty would be paid on commercial sales of CAP-1002. According to Capricor CEO, Dr. Linda Marbán, the Phase 1 Caduceus study, which involved 14 patients, showed reductions of infarct size of 25 percent at six months and 40 percent at one year. The ALLSTAR trial will involve 300 patients.
"This collaboration with Janssen, one of the worlds largest and most respected healthcare companies with a strong presence in cardiovascular and metabolism, is a tremendous milestone for Capricor Therapeutics and an important validation of our lead product, CAP-1002, and the underlying science. We are proud to be working with Janssen to support the continued development of CAP-1002 and for the additional non-dilutive capital to further our research and development that will add to our pipeline," said Marbán, adding that, in her view, Janssen’s regenerative team is unrivaled. “Maybe the most important take-away is that this collaboration with such an outstanding team is an external validation of Capricor technology.”
Marbán explains that the advantage of an allogeneic cardiosphere-derived cell (CDC) is that its development can be closely controlled while, at the same time, being less costly than using a patient’s own cells. “A CDC is a cell derived from a donor heart,” she notes, “when it cannot be used for transplantation. We take the heart back to the lab, section it and culture it. During the process, we melt them like a snowball.” Otherwise, she notes, injecting the cells through a cardiac catheter would be like “flushing a bowling ball down the toilet. Our cells work by going in and acting as a drug. In effect, they tell the local environments to get in and fix the problem. Within a month, they’re gone from the recipient’s system.”
Capricor Therapeutics, Inc. is a publicly traded biotechnology company focused on the development of novel therapeutics to prevent and treat heart disease. The company has two leading product candidates: CAP-1002 and Cenderitide. The company was formed through the November 2013 merger between Capricor, Inc., a privately held company whose mission is to improve the treatment of heart disease by commercializing cardiac stem cell therapies for patients, and Nile Therapeutics, Inc., a clinical-stage biopharmaceutical company developing innovative products for the treatment of cardiovascular diseases.
CAP-1002, Capricor's lead product candidate, is a proprietary allogeneic adult stem cell therapy for the treatment of heart disease. The product is derived from donor heart tissue. The cells are expanded in the laboratory using a specialized process and then introduced directly into a patient's heart via infusion into a coronary artery using standard cardiac catheterization techniques.
CAP-1002 is currently not an approved product and is strictly for investigational purposes.