Gamida Cell's StemEx gets FDA orphan drug designation

Dr. Yael Margolin, formerly of Denali Ventures and Teva, was named new president and CEO.

Stem cell expansion products developer Gamida Cell announced today that the FDA has granted orphan drug designation to the company’s flagship product StemEx for use as hematopoietic support in patients with relapsed or refractory hematologic malignancies (cancer of the blood system) who are receiving high dose therapy.

An orphan drug status is granted by The FDA Office of Orphan Products Development (OOPD) to promote the development of products that demonstrate promise for the treatment of rare diseases or conditions. Orphan drug designation provides for various regulatory and economic benefits, including seven years of market exclusivity in the U.S. to the first entity that obtains marketing approval in the designated indication.

Gamida Cell also announced today the appointment of Dr. Yael Margolin as the company’s new President and CEO. Dr. Margolin has been serving on the company's board of directors since 2000.

Prior to joining Gamida Cell, Margolin served as the Vice President of Denali Ventures LLC, where she was responsible for investments in pharmaceutical and biotechnology companies and served on several Boards of Directors. Dr. Margolin also spent seven years at Teva Pharmaceutical Industries Ltd. (Nasdaq: TEVA; TASE: TEVA), where she was responsible for new product initiatives, evaluation of investment opportunities for the R&D division, and multiple drug development programs.

Teva recently announced it will invest $25 million in a joint venture with Gamida Cell to develop and market StemEx. "We plan to begin a pivotal trial of StemEx during 2005,” said Dr. Margolin.

Europe Israel (MMS) chairman and president Mordechai (Motti) Zisser said, “We are pleased that Dr. Margolin has chosen to accept the appointment of CEO. Her scientific background coupled with her savvy business finesse are sure to serve Gamida Cell well”. Zisser is a major shareholder of Gamida Cell, and a member of the board of directors. Gamida Cell operates in the field of expansion of hematopoietic (blood) stem cell therapeutics in clinical development for cancer, as well as future regenerative cell-based medicines including cardiac, pancreatic and neurological repair and autoimmune diseases. Investors include Elscint (Europe-Israel), Biomedical Investments, Denali Ventures, Teva, Auriga Ventures, Pamot and Comsor.

The company's flagship product is StemEx for the treatment of hematological malignancies. Following the presentation of the results of a Phase I/II study of StemEx, Gamida Cell is now pushing for approval based on pivotal studies planned for 2005 with fast track designation. Plans are also underway to begin a Phase I/II clinical trial in a cardiac repair indication, in 2005. In addition, pre-clinical studies show that the company’s expanded population of stem cells reverses diabetes.

Gamida Cell states that the combined market potential for its stem cell expansion products is estimated at $40 billion worldwide. This includes products based on stem cells from umbilical cord blood as well as placenta, bone marrow, peripheral blood and adult organs like the liver, heart, skin, neural and pancreatic tissues.

Gamida Cell is currently pursuing partnerships, joint ventures and/or strategic alliances.

Published by Globes [online], Israel business news - www.globes.co.il - on Tuesday, March 15, 2005

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