Cardio3 Biosciences SA has broadened its existing relationship with the Mayo Clinic to the point where the company is now its preferred partner for its entire portfolio of regenerative medicine technologies.

No cash is changing hands in this new agreement, but Cardio3, of Mont-Saint-Guibert, Belgium, will have first sight of inventions from the Mayo Clinic Center for Regenerative Medicine in return for providing up-front guidance on their development, from initial invention through to the point where they are ready for a commercial alliance. "The main point is Mayo gets industry and commercial experience from us . . . and we get access to a very broad portfolio," Cardio3 CEO Christian Homsy told BioWorld Today.

The idea is to build industrial robustness early into the Mayo innovation process, so that endgame issues, such as manufacturability, receive early consideration. Cardio3 scientists will make their expertise in areas such as cell therapy, GMP production, culturing and regulation available to Mayo researchers, in order to ensure that the resulting technologies are pharma-ready. "One of industry's great failures is to take programs too early from academia," Homsy said.

He said it is too early at this point for Cardio3 to discuss what other technologies it may take on, but the scope of the deal is broad.

Regenerative medicine is one of three main focus areas for the Mayo clinic across its three campuses, alongside personalized medicine and the science of health care delivery. "We have an exhaustive list of everything that is under development," Homsy said. As well as regenerative medicine, he expects Cardio3 to move into areas such as immunology and oncology on the strength of the alliance.

The relationship builds on Cardio3's development of C-Cure, a cell therapy treatment for heart failure, based on the development of a "cardiopoiesis platform" by Andre Terzic, director of the Mayo Clinic Center for Regenerative Medicine, in Rochester, Minn. That enables scientists to reprogram bone marrow-derived mesenchymal stem cells into cardiopoietic (cardiac progenitor) cells, using a "cardiogenic" cocktail containing transforming growth factor-b, bone morphogenetic protein 4, activin A, fibroblast growth factor 2, cardiotrophin and a-thrombin.

The therapy is undergoing a phase III trial in Europe and Israel at present. It passed its first test recently, when the trial's data safety and monitoring board recommended continuation of the study based on the safety data observed to date. The result of a sterner test, a futility analysis, is due in the first quarter of next year.

Its progress to date has built trust between the two organizations.

"Mayo is a conservative institution – in the good sense of the word," Homsy said. Although a small European biotechnology firm may not be the most obvious partner for one of the leading academic medical centers in the U.S., Cardio3 is one of the few companies that has managed to stay the course with the first wave of cell therapies.

Although big pharma firms such as Novartis AG and Pfizer Inc. are showing a renewed interest in the area, through the progress of chimeric antigen receptor T-cell therapies, those approaches remain immature for now. Genzyme stayed in the area longer than most other big firms, but Paris-based Sanofi Group sold its regenerative medicine business to Ann Arbor, Mich.-based Aastrom Biosciences Inc., following its acquisition of the big biotech firm.

But commercial nous is just as significant as the ability to navigate the technical and regulatory complexity of bringing a cell therapy to market. Five years on from being the first company to gain a European cell therapy approval, another Belgian firm, Leuven-based Tigenix NV, has struggled to build a franchise out of its Chondrocelect autologous cell therapy for regenerating cartilage in the knee. Struggles with obtaining broad reimbursement caused it to turn over marketing and distribution rights to Stockholm-based Swedish Orphan Biovitrum AB recently. (See BioWorld Today, April 4, 2014.)

In a cost-constrained era, using costly cell therapy for cartilage replacement is a hard sell. "We got learnings from the Dendreon story as well," Homsy said, referring to the difficulties Seattle-based Dendreon Corp. had with building sales momentum for its prostate cancer therapy Provenge (sipuleucel-T), which comes with a heavy logistical and cost burden.

The Mayo alliance is a first step in a recently unveiled business development strategy based on Cardio3's technological capabilities in cell therapy and on its clinical expertise in cardiovascular disease.

"It's just part of it. It's certainly not the end of it," Homsy said.

The company is exploring several other opportunities, some of them at a later stage of development than the Mayo research portfolio.