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FDA Grants Orphan Drug Designation to Bellicum’s BPX-501 for the Treatment of Immunodeficiency and GvHD Following Stem Cell Transplant
Following an allogeneic transplant, a lack of sufficient mature T cells constitutes immune deficiency that can contribute to infections, viral reactivation, and relapse. The ability to correct this immune deficiency through the add-back of mature donor T cells, without raising the risk of uncontrollable GvHD, has the potential to fundamentally change the risk profile of allogeneic transplantation.
“Expanding the availability of hematopoietic stem cell transplants to
more patients with rare genetic diseases and blood cancers is a primary
goal of our BPX-501 program,” commented
BPX-501 is currently being evaluated in multiple Phase 1/2 clinical
trials in adults and pediatric patients with leukemias, lymphomas, and
genetic blood diseases in the U.S. and
About Orphan Drug Designation
BPX-501 is an adjunct T cell therapy of genetically modified donor T cells incorporating Bellicum’s proprietary CaspaCIDe safety switch. The product candidate is designed to provide a safety net to eliminate the BPX-501 alloreactive T cells should severe GvHD occur, enabling physicians to more safely perform haploidentical stem cell transplants by adding back the BPX-501 genetically engineered T cells to speed immune reconstitution and provide control over viral infections.
Bellicum is a clinical stage biopharmaceutical company focused on discovering and developing cellular immunotherapies for cancers and orphan inherited blood disorders. Bellicum is using its proprietary Chemical Induction of Dimerization (CID) technology platform to engineer and control components of the immune system. Bellicum is developing next-generation product candidates in some of the most important areas of cellular immunotherapy, including hematopoietic stem cell transplantation (HSCT), and CAR-T and TCR cell therapies. More information can be found at www.bellicum.com.
This press release contains forward-looking statements for purposes
of the safe harbor provisions of the Private Securities Litigation
Reform Act of 1995. We may, in some cases, use terms such as "predicts,"
"believes," "potential," "proposed," "continue," "estimates,"
"anticipates," "expects," "plans," "intends," "may," "could," "might,"
"will," "should" or other words that convey uncertainty of future events
or outcomes to identify these forward-looking statements.
Forward-looking statements include statements regarding our intentions,
beliefs, projections, outlook, analyses or current expectations
concerning, among other things: the timing of our clinical trials and of
our research and development activities relating to BPX-501; the
effectiveness and success of our research and development activities
relating to our CaspaCIDe platform; and the effectiveness of BPX-501 and
its possible range of applications and potential curative effects and
safety profile. Various factors may cause differences between Bellicum’s
expectations and actual results as discussed in greater detail in
Bellicum’s filings with the