Washington Editor

Gamida Cell Ltd. now has the green light to proceed with a pivotal study of its allogeneic stem cell product StemEx in hematological malignancies, leukemia and lymphoma.

The company, of Jerusalem, reached an agreement under a special protocol assessment with the FDA on the design of a global, historical-controlled trial to support approval. In short, that means the Phase III study is adequately designed for an eventual marketing application, pending positive data, and it's set to begin early next year.

"The field of cell therapy is very new," President and CEO Yael Margolin told BioWorld Today, "and there aren't yet very clear regulations." But the SPA process lays out a roadmap for StemEx's registration, especially given the "quite innovative" inclusion of the historical control arm. Margolin declined to specify the nature of that comparator group, though she noted that the FDA agreed that its use represents "a true control" and would not lead to bias in the trial's final analysis. After the study has gotten under way, Gamida Cell would publish findings on the historical cohort in a peer-reviewed journal, she said.

The trial will take place in the U.S., Europe and Israel over the course of about two years, ending late in 2008. Margolin expects StemEx would reach the market in 2009, as planned.

She declined to comment on the study's size or endpoints, but past findings show the product's potential. A Phase I/II study conducted at the M.D. Anderson Cancer Center in Houston demonstrated efficacy trends, as StemEx treatment improved leukemia patients' short-term survival and was engrafted in all who were evaluable, in a median of 52 days. In terms of safety, its use led to graft-vs.-host disease in just 20 percent of patients, a complication common to traditional bone marrow transplants.

StemEx - made of cord blood highly enriched with ex vivo-expanded stem cells - has orphan drug status from the FDA for use as hematopoietic support in relapsed or refractory blood cancer patients who are receiving high-dose therapy. Currently, such patients become candidates for bone marrow transplants, but about 70 percent of them can't have the procedure.

According to Margolin, about 25,000 leukemia and lymphoma patients are annually diagnosed in the U.S. Of them, about 20 percent find family marrow matches, and another 10 percent get matched up with non-family marrow, but the remainder doesn't have a transplant option.

"The unmet clinical need is tremendous," Margolin said, and StemEx, which is transplanted in combination with non-expanded cells from the same cord blood unit, could fill that void.

Its pivotal program also is designed to support approval in Europe and Israel. The product's development is managed through a joint venture with Teva Pharmaceutical Industries Ltd., also of Jerusalem. Per terms of that arrangement, for which Teva paid $25 million, Gamida Cell granted the joint venture an exclusive license to develop and market StemEx specifically for hematological diseases. Both Gamida Cell and Teva would equally split revenues.

Gamida Cell plans to move ahead on its own to test StemEx's use more broadly in areas such as solid tumors, autoimmune diseases and storage disorders, all of which can be treated with bone marrow transplants to induce remission. A study in one of those three areas is expected to begin next year, funded largely by a recent $16 million round of financing for privately held Gamida Cell. (See BioWorld Today, Sept. 28, 2006.)

Elsewhere in the regenerative medicine company's portfolio is a cardiac regeneration product expected to move into the clinic next year, as well as preclinical programs for neurological disorders and peripheral vascular disease.