Bellicum Presents Updated Clinical Results at 2017 BMT Tandem Meetings on BPX-501 in Orphan Inherited Blood Disorders and Hem...
February 22 2017 - 4:15PM
Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in
developing novel, controllable cellular immunotherapies for cancers
and orphan inherited blood disorders, today announced the
presentation of updated clinical results from its multicenter
BP-004 clinical trial of BPX-501 and rimiducid at the 2017 BMT
Tandem Meetings. Results on 73 patients with more than six months
of follow-up demonstrated that the use of BPX-501 following an
alpha/beta-depleted, haploidentical hematopoietic stem cell
transplant (haplo-HSCT) resulted in rapid immune recovery in
patients with inherited blood disorders and hematological cancers.
“The results from our BPX-501 clinical program continue to
demonstrate positive and consistent results across multiple centers
and diseases, underscoring its potential to address the needs of a
wide range of pediatric patients who lack access to a suitable
matched donor,” commented Rick Fair, President and CEO
of Bellicum Pharmaceuticals. “The data reported today,
including incidences of GvHD- and disease-free outcomes in children
with blood cancers and genetic diseases, compare favorably to
historical outcomes in patients receiving a matched unrelated donor
(MUD) transplant, the comparative population for our ongoing
registration trial in Europe.”
Updated Results of BP-004 StudyTo date, 122
pediatric patients have undergone treatment with BPX-501.
Investigators reported on 91 patients with 100 days of follow-up,
73 patients with six months, and 45 patients with more than one
year of follow-up. Genetic blood diseases (n=54) include SCID
(Severe Combined Immune Deficiency) (n=11), thalassemia major
(n=8), Wiskott-Aldrich syndrome (n=6), sickle cell disease (n=3),
and several others. Hematological cancers (n=37) include ALL (acute
lymphoblastic leukemia) (n=22), AML (acute myeloid leukemia)
(n=13), and others.
Patients receiving BPX-501 following a haplo-HSCT demonstrated
rapid immune reconstitution by month six, including full recovery
and normalization of T-cells, B-cells and immunoglobulins.
Cumulative incidence of treatment-related mortality remains very
low in the BP-004 study, with six-month and one-year survival rates
of 98.4 percent and 97.2 percent, respectively, and no mortality
associated with use of BPX-501 or rimiducid. Of 73 patients with
more than six months of follow-up, 22 percent had acute Grade 1-2
GvHD, 2.7 percent had Grade 3, and there were no cases of Grade 4
GvHD. In the BP-004 study, rimiducid was used on six patients
experiencing GvHD that was not controlled by the use of standard
treatments. In all five cases of uncontrolled acute GvHD, the
administration of rimiducid rapidly resolved the symptoms. As
previously reported, there was one case of severe chronic GvHD
attributed to cells from the donor graft, and unrelated to BPX-501,
in a malignant patient that did not resolve with administration of
rimiducid.
The poster presentation can be accessed in the Events and
Presentations section of the Bellicum website.
About BPX-501BPX-501 is an adjunct T-cell
therapy administered after allogeneic HSCT, comprising genetically
modified donor T cells incorporating Bellicum’s
CaspaCIDe® safety switch. It is designed to provide a safety
net to eliminate alloreactive BPX-501 T cells (via administration
of activator agent rimiducid) should uncontrollable GvHD occur.
This enables physicians to more safely perform stem cell
transplants by adding back BPX-501 engineered T cells to speed
immune reconstitution and provide control over viral infections,
without unacceptable GvHD risk. The ongoing BP-004 clinical study
of BPX-501 is being conducted at transplant centers in the U.S.
and Europe.
About the BMT Tandem MeetingsThe BMT
Tandem Meetings are the combined annual meetings of the Center
for International Blood & Marrow Transplant Research (CIBMTR)
and the American Society for Blood and Marrow Transplantation
(ASBMT). Attended by investigators, clinicians, laboratory
technicians, clinical research professionals, nurses, pharmacists,
administrators, and allied health professional attendees, the
scientific program addresses the most timely issues in
hematopoietic cell transplantation.
About Bellicum PharmaceuticalsBellicum is
a leader in developing novel, controllable cellular immunotherapies
for cancers and for 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.
Forward-Looking StatementThis press release
contains forward-looking statements for purposes of the safe harbor
provisions of the Private Securities Litigation Reform Act of 1995.
Bellicum may, in some cases, use terms such as "predicts,"
"believes," "potential," "proposed," "continue," “designed,”
"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:
our research and development activities relating to BPX-501,
rimiducid and CaspaCIDe; the effectiveness of rimiducid and of
CaspaCIDe; the effectiveness of BPX-501 and its possible range of
application and potential curative effects and safety in the
treatment of diseases including as compared to other treatment
options and competitive therapies; the timing and success of our
BP-004 clinical trial, including the rate and progress of
enrollment; and, the timing of regulatory filings for BPX-501 and
for rimiducid. Various factors may cause differences between
Bellicum’s expectations and actual results as discussed in greater
detail under the heading “Risk Factors” in Bellicum’s filings with
the Securities and Exchange Commission, including without
limitation our annual report on Form 10-K for the year
ended December 31, 2015. Any forward-looking statements that
Bellicum makes in this press release speak only as of the date of
this press release. Bellicum assumes 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.
Investors:
Bellicum Pharmaceuticals
Alan Musso, CFO
832-384-1116
amusso@bellicum.com
Media:
BMC Communications
Brad Miles, 646-513-3125
bmiles@bmccommunications.com
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