35 children with genetic immune disorders and
hemoglobinopathies are disease- and GvHD-free following
haploidentical stem cell transplant with BPX-501
Webcast of ASH investor/analyst event scheduled
for Monday, December 5 at 12:15 p.m. PST
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, today announced results from the BP-004
multicenter clinical trial which includes children with Primary
Immune Deficiencies (PIDs) and hemoglobinopathies who received an
add-back of BPX-501 modified T cells following a haploidentical, T
cell-depleted hematopoietic stem cell transplant (haplo-HSCT). The
data were presented in oral and poster sessions today during the
58th Annual Meeting of the American Society of Hematology (ASH).
Results show that all 35 children treated are alive, free from GvHD
and cured of their underlying disease.
According to Neena Kapoor, MD, Director of the Blood and Marrow
Transplantation Program at Children’s Hospital of Los Angeles and
who reviewed study results of patients with PIDs in an oral
presentation, “These results show that the inclusion of BPX-501 T
cells may make haplo-HSCT a first-line option for children with
PIDs who lack a suitable, HLA-matched donor.” Dr. Kapoor added,
“Delayed immune reconstitution leading to severe infectious
complications is the primary cause of morbidity and mortality in
PID patients who undergo a T-depleted haplo-transplant. The data
reflect that the add-back of BPX-501 donor T cells provides faster
immune recovery and lower rates of re-hospitalizations due to
infection. The CaspaCIDe safety switch, engineered into BPX-501 T
cells, provides a critical safety net to address the risk of
uncontrolled acute GvHD.”
PID Highlights (Abstract #72)
“Outcome of Children with Primary Immune-Deficiencies (PIDs)
Enrolled in a Phase I-II Trial Based on the Infusion of BPX-501
Donor T Cells Genetically Modified with a Novel Suicide Gene
(Inducible Caspase 9, iC9) After T-Cell Depleted HLA-Haploidentical
Allogeneic Stem Cell Transplantation (Haplo-HSCT)”
PIDs in the study include Severe Combined Immune Deficiency
(“Bubble boy” disease) (n=11), Wiskott-Aldrich syndrome (n=6),
Chronic Granulomatous Disease (n=2) and other rare Immune
Deficiencies (n=4). Study outcomes for these patients included:
- All 23 PID patients engrafted with no
secondary graft failure.
- All patients are alive and free of
disease with a median follow-up of 404 days (range: 118-728
days).
- Median time to neutrophil and platelet
recovery was 16 days and 10 days, respectively.
- Five children experienced Grade I or
Grade II acute GvHD. Three of the cases resolved with either
topical or systemic steroids. The other two GvHD cases resolved
following infusion of rimiducid and activation of the CaspaCIDe®
safety switch. There was one mild case of chronic GvHD, which also
resolved. No one experienced severe Grade 3 or Grade 4 GvHD.
- There was a low rate of hospital
re-admission to treat infections. No reported adverse events were
associated with administration of BPX-501.
Hemoglobinopathy Highlights (Abstract #2286)
“Clinical Outcome and Immune Recovery after Adoptive Infusion of
BPX-501 Cells (Donor T Cells Transduced with iC9 Suicide Gene) in
Children with Hemoglobinopathies and Diamond-Blackfan Anemia Given
a/b T-Cell Depleted HLA-Haploidentical Stem Cell Transplantation
(HSCT)”
Updated BP-004 study results were also reviewed in a poster
session of 12 pediatric patients with Thalassemia Major β0/β0
(n=9), Sickle Cell Disease (n=1) and Diamond-Blackfan Anemia (n=2).
HSCT is a potentially curative treatment for these patients, who
otherwise typically require a lifetime of blood transfusions. Study
results demonstrated that the BPX-501 cells expanded and persisted
in the patients over time, contributing to overall immune
reconstitution and successful transplant outcomes. Results reported
in the poster included:
- All 12 patients attained full donor
chimerism. One patient with secondary graft failure was
re-transplanted from the same donor and then attained full donor
chimerism.
- All patients are alive, GvHD-free and
free of disease.
- The median initial hospital discharge
occurred at 21 days (range: 14-55) and there were no
re-hospitalizations.
- Grade I/II skin acute GvHD occurred in
2 patients, and was successfully treated with steroids. No chronic
GvHD occurred.
- Median time to last red blood cell
transfusion was 6.5 days (range: 4-33 days) post-transplant.
“Our BPX-501 program continues to gain momentum as additional
centers recognize its potential to improve haplo-HSCTs in both
nonmalignant and malignant diseases,” commented Tom Farrell,
President and CEO of Bellicum Pharmaceuticals. “The reliable and
expedient performance of our molecular switch technology, combined
with an advanced method of T-depleted stem cell transplantation,
have the potential to make curative haplo-transplants available to
many more patients suffering from genetic diseases and blood
cancers.”
Investor/Analyst Luncheon
Bellicum will host an investor and analyst luncheon on Monday,
December 5, 2016 from 12:15 – 1:15 p.m. PST at the San Diego
Marriott Gaslamp Quarter Hotel. Management and investigators Dr.
Franco Locatelli (Ospedale Pediatrico Bambino Gesù), Dr. Neena
Kapoor (Children’s Hospital of Los Angeles), and Dr. Kris Mahadeo
(Montefiore Medical Center) will discuss BPX-501 Phase 2 clinical
data in the nonmalignant and malignant settings. The luncheon will
be webcast live and may be accessed from the News &
Events section of the Bellicum website. An archived version of
the webcast will be available for replay for at least two weeks
following the event.
About PIDs
Primary Immune Deficiencies (PIDs) are caused by genetic
abnormalities that prevent the development of normal immune
responses, which lead to an increased susceptibility to infections.
A hematopoietic stem cell transplant (HSCT) is the current mainstay
of treatment for severe forms of PIDs, including Severe Combined
Immune Deficiency (SCID), Wiskott-Aldrich syndrome, Chronic
Granulomatous Disease (CGD) and several others.
About Hemoglobinopathies
Hemoglobinopathies are a diverse group of genetic disorders
caused by the abnormal structure or production of hemoglobin, a
protein molecule in red blood cells that carries oxygen throughout
the body. Depending on disease severity, standard treatments
include blood transfusions and drugs to control symptoms, however a
stem cell transplant is often preferred for severe cases.
About BPX-501
BPX-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 risk of uncontrollable GvHD. The
ongoing BP-004 Phase 1/2 clinical study of BPX-501 is being
conducted at transplant centers in the U.S. and Europe.
About Bellicum Pharmaceuticals
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.
Forward-Looking Statement
This 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
CaspaCIDe; the effectiveness of BPX-501, 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
clinical trials, including the rate and progress of enrollment in
our clinical trials; 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20161203005044/en/
Investors:Bellicum PharmaceuticalsAlan Musso, CFO,
832-384-1116amusso@bellicum.comorMedia:BMC
CommunicationsBrad Miles,
917-570-7340bmiles@bmccommunications.comorBMC
CommunicationsAmy Bonanno,
914-450-0349abonanno@bmccommunications.com
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