PR Newswire - December 6, 1995

Dr. Tricot reported that the clinical site was able to mobilize the necessary cells from 10 of the initial 12 patients, from which SyStemix then isolated a sufficient quantity of pure HSCs for two transplants. Target levels of engraftment (recovery) were reached for 6 of the 7 patients for whom first transplant data was reported, as early as 12 days for neutrophils (median 16) and as early as 18 days for platelets (median 21). Bone marrow biopsies showed normal cellularity in all lineages as early as 15 days after transplant. The remaining patient had a viral infection which delayed blood cell recovery although the bone marrow biopsy showed engraftment, and required a back-up infusion of mobilized peripheral blood on Day 31. Results from the entire series of first transplants from the 26 patients planned for the trial are expected to be announced by mid-1996.

In a separate presentation, SyStemix' scientists presented data from its HIV gene therapy collaboration with Sandoz which showed that white blood cells can be protected against the HIV virus by transducing human stem/progenitor cells with a REVM10 gene. Gene-modified human stem/progenitor cells were transplanted into the SyStemix SCID-hu mouse, a preclinical model with a human immune system, to demonstrate in vivo that when these cells differentiate the "daughter" T-cells still retain the gene, expressed at therapeutic levels, resulting in protection from HIV-1 infection.

This data represents an important advance in the use of gene-modified stem/progenitor cells in the cellular therapy of AIDS. Unlike T-cells, the stem cells are self-renewing and pluripotent, creating the potential for long-lasting, targeted therapies which are cost-effective.

"The results presented at ASH show clinically that the HSC will engraft in a timely, complete and sustained manner and preclinically that the stem cell can be gene-modified, with progeny continuing to express the gene and be protected," said Christopher A. Juttner, M.D., Vice President Clinical/Medical Affairs at SyStemix. "It is our hope that HSC transplants in the cancer setting will ultimately improve disease-free survival rates for patients, compared to alternative transplants which may either be tumor contaminated or lack sufficient self-renewing HSCs."

SyStemix scientists and collaborators had a total of 14 presentations and posters at the meeting. One of these was on hematopoietic gene discovery, in which SyStemix researchers showed that CD34+Thy+Lin- stem cells removed from peripheral blood express a broad range of genes, including those encoding for cytokine receptors, such as an interferon receptor, as well as for signal transducers and transcription factors. Prior to these findings, efforts at characterizing the genes relevant to stem cells have been limited, largely due to the inability of obtaining large quantities of cells. SyStemix was able to use its clinical scale, high speed cell sorting system to amass sufficient stem cells. Several clones corresponding to novel cytokine receptors and other markers have been identified and are being evaluated at SyStemix.

SyStemix scientists also separately reported that thrombopoietin (Tpo) can stimulate the proliferation of HSCs, direct differentiation of the HSC toward megakaryocyte development, and enhance the number of CD34+ progenitors generated from HSCs. These findings provide further scientific rationale for SyStemix' development of novel cellular transfusion products using disease-free HSCs.

"The presentations at this meeting demonstrate the breadth of our potential product portfolio and the strength of our technology platform," commented John J. Schwartz, Ph.D., President and CEO of SyStemix. "This work expands the foundation for clinical application of isolated, ex vivo expanded and gene-modified HSCs."

Last month, the Company announced that it plans to file an Investigational New Drug (IND) application with the Food & Drug Administration (FDA) for transplantation (in utero) for genetic disease by the end of this year. Additional cell and gene therapy trials are slated for 1996, in addition to the ongoing Phase I/II trial using HSCs for support of multiple myeloma patients after chemotherapy.

SyStemix, Inc. is pioneering new methods and standards for creating living therapeutics. SyStemix is focused on advanced cell and gene therapies and gene discovery for the treatment of cancer, genetic and infectious diseases, based on diverse applications of human hemapoietic stem cells.

CONTACT: Wendy R. Hitchcock, Chief Financial Officer of SyStemix, Inc., 415-813-4108; or Reagan Codner, 212-505-1919, or Justin Jackson, 415-352-6262, both of Burns McClellan, for Systemix/ 08:35 EST

Copyright (c) 1995/PR NewsWire. Reproduced with permission. Reproduction of this article (other than one copy for personal reference) must be cleared through the Permissions Desk, PR Newswire, 810 Seventh Avenue, New York, NY 10019.
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