A Cardiovascular Device Update

Biotech start-up Myosix SA (Paris) and the Paris Public Hospital Authority have signed agreements with Genzyme Biosurgery, a subsidiary of Genzyme (Cambridge, Massachusetts), to conduct a large-scale Phase II trial of a cell therapy for heart failure developed by the French researchers.The multicenter trial is to be carried out on 300 patients in France, other European countries, the U.S. and possibly Japan, starting this fall, and will be by far the largest cell therapy clinical trial ever conducted. The therapy is designed to improve a patient's heart function after heart attack by introducing muscle cells taken from another part of the body and introduced to damaged areas of the heart.

The procedure entails the removal of autologous skeletal myoblasts from the patient's leg, culturing them to increase their numbers (to between 400 million and 1 billion) and then injecting them into the heart during a coronary artery bypass operation. Myoblasts are dormant cells found in muscle tissue that only begin dividing when muscle injury occurs.

The technique was developed by Jean-Pierre Marolleau and Jean-Thomas Vilquin and was validated in a Phase I trial carried out on 10 patients at Saint Louis Hospital (Paris) between June 2000 and November 2001. This will aim to establish not only the safety and efficacy of the therapy but also its overall clinical benefit, said Duke Collier, president of Genzyme Biosurgery.

The studies in France will be co-financed by Assistance Publique-Hopitaux de Paris, which has budgeted about $1.3 million for the purpose, and Genzyme, which also will finance the trials elsewhere. That will add up to an investment of "several million dollars" for the company, Collier said. In addition, Genzyme will handle much of the cell-culture operations in cooperation with Saint Louis Hospital. Between them, they are developing a standardized procedure based on the one used by Myosix but also drawing on the industrial-scale cell manipulation expertise of Genzyme.

Collier said that the clinical development of the therapy as far as the regulatory approval stage would cost at least $50 million to $100 million, adding that "the cost could be a lot higher than that depending on the regulatory environment." He also said that Genzyme would be prepared to invest in manufacturing facilities in France if the product reached the market, pointing out that the company had "options to acquire commercialization rights for the rest of the world." Genzyme has had a presence in France for more than 10 years and currently employs around 100 people there and 1,000 in Europe as a whole.

Divesting cardiology drug effort

MediGene AG (Martinsried, Germany) has decided to spin off its cardiological drug discovery program, saying the move would allow it to concentrate on its core business and increase the value of the program by expansion and alternative financing within an external unit. The company said it would concentrate on its research and development projects in tumor diseases and free itself up for acquisition and individual licensing of other advanced products.

MediGene said it has not yet decided on the exact disposition of the cardiological program. Management would prefer for it to become an independent firm, which MediGene would manage along with strategic partners and investors.