By Mary Vanac

Clinical-stage regenerative medicine company Juventas Therapeutics Inc. — a Cleveland Clinic spin-out initially named AcelleRX Therapeutics Inc. — started enrolling patients in a Phase 1 clinical trial to evaluate the safety and efficacy of its leading stem cell factor for treating heart failure.

In preclinical studies of heart failure in pigs, JVS-100, as the factor is known, significantly increased cardiac function by promoting cell survival and increasing blood vessel formation in damaged hearts. JVS-100 works by encoding Stromal Cell-derived Factor-1, a growth factor that in adults recruits stem cells from the bone marrow to create new blood vessels. The JVS-100-treated pigs showed significant improvements in cardiac function, including reductions in left ventricular end systolic volume, according to a press release.

Dr. Douglas Losordo, director of the Feinberg Cardiovascular Research Institute at Northwestern University, is the principal investigator for the 16-person study. Also investigators on the study: Dr. Farrell Mendelsohn, a cardiologist at Baptist Medical Center-Princeton in Birmingham, Ala., director of the Center for Therapeutic Angiogenesis Interventional & Regenerative Cardiovascular Medicine; and Dr. Warren Sherman, an interventional cardiologist who directs Stem Cell Research and Regenerative Medicine for the Center for Interventional Vascular Therapies at Columbia University Medical Center.

“Juventas is excited to have initiated this Phase 1 heart failure trial and honored to be working with a top-tier group of interventional cardiologists,” Juventas president and CEO Rahul Aras said. “The trial is an important milestone and represents the first of several clinical opportunities for our company.”

Juventas was formed in 2007 to develop and commercialize the stromal cell-derived factor research of Dr. Marc Penn, a Cleveland Clinic cardiologist who also is senior medical director for the Clinic’s emerging businesses unit. Penn is chief scientific officer for Juventas.

“SDF-1 was discovered by Marc as a homing factor. It serves as a beacon for circulating stem cells,” Aras said. “We’ve gone on to show that it works through multiple mechanisms,” including preventing cells in damaged tissue from dying.

Coupling that mechanism with the new blood vessel-forming ability of stem cells creates a two-step process: New blood vessels are created and existing cells don’t die, Aras said.

“If you look at that from a competitor’s perspective, it has the potential to simplify what a lot of regenerative medicine companies are working on, through the extraction and redelivery of cells, by optimizing our own stem cells,” he said.

Since 2007, “we’ve transitioned the technology from a preclinical concept to the first clinical trial,” he said. “So we’re on target to be able to complete this trial by the end of the year. That will be a big step for the company.”

What’s next?

“We’ve led with heart failure because that’s where our preliminary data was and it’s a great clinical opportunity,” Aras said. “We also have strong data in the area of peripheral vascular disease and cosmetic wound healing.”

The factor can increase blood flow for patients who have PVD and accelerate wound closure and prevent scarring for patients who have had cosmetic surgery. “So we’re looking to move both those toward clinic in the near future,” Aras said.

Juventas has closed on about $7.7 million in venture funding since its start, he said. In July 2007, the company raised $1.5 million from North Coast Angel Fund and venture developer JumpStart Inc., both in Cleveland, as well as from Blue Chip Venture Co. of Cincinnati and from individual investors. The firm raised another $6.9 million from a group of venture capital firms led by Triathlon Medical Ventures in Cincinnati during its Series A fundraising round in October 2008.

Juventas also received Ohio Third Frontier grants worth $2 million from the Cleveland Clinic-led Global Cardiovascular Innovation Center to further its heart failure platform and from the Center for Stem Cell and Regenerative Medicine in Cleveland to help further its peripheral vascular disease platform, Aras said.

“We have four full-time employees, two part-time employees. We’re still relatively small. As our clinical opportunities expand, so will the company,” he said.

“We have rights to a pipeline of factors that were discovered by [Penn],” Aras said. “JVS-100 is the first in a line of downstream opportunities that exist. With the capital we have and what we’re focused on right now, we are looking to drive JVS-100 forward, but we do have other factors that we’ll look to commercialize as the company matures. ”

Other upcoming milestones?

“We’re engaging with the Food & Drug Administration on future clinical trials in the area of peripheral vascular disease,” he said. “The big milestone for us is the completion of this Phase 1 clinical trial. But we are acting aggressively to further our other clinical platforms as well.”