ASH Annual Meeting also featured poster presentations of GoCAR-T and
Supporting preclinical data for CIDeCAR product candidate BPX-401 and
GoCAR-T product candidate BPX-601 highlighted
ORLANDO, Fla.--(BUSINESS WIRE)--Dec. 7, 2015--
Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a clinical stage
biopharmaceutical company focused on discovering and developing novel
cellular immunotherapies for cancers and orphan inherited blood
disorders, presented preclinical data from studies of its CIDeCAR™ CAR
T-cell technology, including product candidate BPX-401, in an oral
presentation at the 57th American Society of Hematology (ASH)
Annual Meeting today. The preclinical in vivo data show that
tumors can be eliminated quickly and safely with the Company’s CIDeCAR
novel costimulatory domain and safety switch.
In the oral presentation, “Expression of MyD88/CD40 Drives In Vivo
Activation and Proliferation of Chimeric Antigen Receptor Modified T
Cells That Can Be Effectively Regulated by Inducible Caspase-9,”
Bellicum scientists compared CIDeCARs targeting CD19 or other tumor
antigens to CAR constructs based on 4-1BB and CD28. CIDeCAR combines
Bellicum’s novel costimulatory domain MC (MyD88/CD40) and the CaspaCIDe® safety
switch. Bellicum’s small molecule rimiducid was used to activate the
CaspaCIDe safety switch. The CaspaCIDe safety switch was also employed
as a “dimmer switch,” titrating the number of CIDeCAR cells in order to
remain within a therapeutic window that maximizes both safety and
efficacy, for improved clinical benefit.
The addition of the MC costimulatory domain led to a significant
increase in in vivo activation and persistence of the CIDeCAR
CAR T cells when compared to CARs constructed with conventional
costimulatory domains CD28 and 4-1BB.
Studies also showed that CAR T cells with the MC costimulatory domain
eliminated cancer cells faster in animal models than CARs using CD28
In CD19 and HER2 animal models, the majority of CAR T cells can be
eliminated if needed, leading to rapid recovery from cytokine
release-like syndrome, without tumor relapse.
Experiments also demonstrate that the CaspaCIDe safety switch can be
titrated. Lower concentrations of small molecule activator rimiducid
led to elimination of only a portion of circulating CAR T cells,
including possibly the most active cells, without losing the antitumor
effect of treatment.
GoCAR-T™ and GoTCR™ Poster Presentations
Two additional poster presentations1,2 at ASH featured the
Company’s GoCAR-T and GoTCR technologies. GoCAR-T technology uses
rimiducid to control persistence of CAR-T cells in vivo for
therapeutic effect. In animal experiments targeting CD19 and Prostate
Stem Cell Antigen (PSCA), Bellicum scientists demonstrated that a single
IV injection of GoCAR-T cells containing the proprietary iMC (inducible
MyD88/CD40) activation switch could eliminate large, established solid
tumors and lymphomas. Administration of rimiducid stimulated the
survival and proliferation of GoCAR-T cells, which may allow more
effective therapy of solid tumors in patients.
In an additional poster session, Bellicum scientists used the same iMC
activation switch to complement HLA-A2/Preferentially Expressed Antigen
in Melanoma (PRAME)-specific TCR-modified T cells to create “GoTCR”s
that also could be activated by rimiducid, similar to the GoCAR-T
platform. PRAME-targeted GoTCRs were active against PRAME-expressing
tumors in vitro and in vivo. Activation with weekly
administration of rimiducid led to more effective tumor control. In this
application of the CID technology, toxicity is controlled by the
cessation of rimiducid administration, acting like “lifting one’s foot
off of a gas pedal,” without the loss of tumor control.
“These preclinical data support the advancement of our product
candidates BPX-401 and BPX-601 into clinical trials. We are also excited
about the potential of our GoTCR platform to address the limitations of
TCR therapy by enabling control over the cells’ proliferation and
persistence,” commented Tom Farrell, Bellicum’s President and CEO.
1 Foster A, Mahendravada A, Shinners N, et al. “Inducible
MyD88/CD40 allows rimiducid-dependent activation to control
proliferation and survival of chimeric antigen receptor-modified T
cells.” 2015 Annual Meeting of the American Society of Hematology,
2 Hoang T, Foster A, Lu A, et al. “Inducible MyD88/CD40
enhances proliferation and survival of PRAME-specific TCR-engineered T
cells and increases anti-tumor effects in myeloma 2015.” Annual Meeting
of the American Society of Hematology, Orlando, FL
About Bellicum Pharmaceuticals
Bellicum is a clinical stage biopharmaceutical company focused on
discovering and developing novel cellular immunotherapies for various
forms of cancer, including hematological cancers and solid tumors, as
well as orphan inherited blood disorders. The Company is using its
proprietary Chemical Induction of Dimerization, or CID, technology
platform to engineer and control components of the immune system in real
time. The Company is developing next-generation product candidates in
some of the most important areas of cellular immunotherapy, including
hematopoietic stem cell transplantation, or HSCT, CAR T cell therapy,
and dendritic cell vaccines.
*CaspaCIDe® is a trademark registered with the U.S. Patent
and Trademark Office. CIDeCAR™, GoCAR-T™ and GoTCR™ are trademarks
of Bellicum Pharmaceuticals.
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-401 and BPX-601;
the effectiveness and success of our research and development activities
relating to our GoCAR-T and GoTCR platforms; and the effectiveness of
BPX-401 and BPX-601 and their possible range of applications and
potential curative effects and safety profiles. Various factors may
cause differences between Bellicum’s expectations and actual results as
discussed in greater detail in Bellicum’s filings with the Securities
and Exchange Commission, including without limitation, under the
heading “Risk Factors” in our annual report on Form 10-K for the year
ended December 31, 2014 and our Report on Form 10-Q for the quarter
ended June 30, 2015. Any forward-looking statements that we make in
this press release speak only as of the date of this press release. We
assume no obligation to update our forward-looking statements whether as
a result of new information, future events or otherwise, after the date
of this press release.
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Source: Bellicum Pharmaceuticals, Inc.
Bellicum Pharmaceuticals, Inc.
Chief Financial Officer
Bellicum Pharmaceuticals, Inc.
Brad Miles, 917-570-7340