Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in
developing novel, controllable cellular immunotherapies for cancers
and orphan inherited blood disorders, announced that results will
be presented today from its global BP-004 clinical trial of BPX-501
in the Presidential Symposium at the 22nd Congress of the European
Hematology Association (EHA) being held in Madrid, Spain. Clinical
results reported in pediatric patients with a wide range of genetic
blood diseases and hematologic cancers suggest that BPX-501 lowers
the risks associated with haploidentical stem cell transplants,
increasing their acceptability for more patients who could benefit
but lack a matched donor. The EHA’s Presidential Symposium
showcases the top abstracts selected by the Scientific Program
Committee each year.
“The vast majority of patients in this study experienced
successful engraftment, no or low grade GvHD, rapid immune recovery
and early hospital discharge,” said Dr. Franco Locatelli, MD, PhD,
Director of the Department of Pediatric Hematology and
Oncology, Ospedale Pediatrico Bambino Gesù (OPBG), Rome. “All
cases of uncontrolled acute GvHD were resolved by administration of
rimiducid to activate the CaspaCIDe safety switch incorporated in
BPX-501. With low incidence of transplant-related mortality and
manageable GvHD, these data suggest that the addition of BPX-501 T
cells could represent a meaningful advance for patients in need of
a transplant but who lack a matched related donor.”
Updated Results of BP-004 Study in Global and EU
Populations Investigators reported on pediatric patients
with hematologic cancers and genetic blood diseases who underwent
treatment with BPX-501 after an alpha/beta T-cell depleted
haploidentical hematopoietic stem cell transplant (alpha/beta
T-cell depleted haplo-HSCT) and had at least six months of
follow-up. Investigators reviewed overall data on 98 patients from
12 centers in the U.S. and Europe, as well as on the subset of 61
patients at European sites.
At the 180-day measurement time point, cumulative incidence of
transplant-related mortality (TRM) was 5% in the overall patient
population. A significant reduction in viral infections and
reinfections was observed with BPX-501 compared to results from a
study in Rome (OPBG) in patients undergoing an alpha/beta T-cell
depleted haplo-HSCT without the addition of BPX-501 (n=107).
Additionally, there were no reported adverse events associated with
the use of BPX-501 or rimiducid. At six months of follow-up, the
cumulative incidence of Grade 2-4 acute Graft versus Host Disease
(GvHD) was 13 percent. The cumulative incidence of chronic GvHD was
three percent at one year of follow-up.
The administration of rimiducid was required in 11 patients with
acute GvHD who did not respond to standard treatments. In all 11
cases, rimiducid rapidly resolved the GvHD. In addition, the
non-reactive T cells recovered, with no recurrence of GvHD.
Data from the subset of 61 patients from European sites were
also reviewed in the presentation. At six months of follow-up there
was no transplant-related mortality. Cumulative GvHD incidence
remains low, with incidence rates of 10 percent for Grade 2-4 acute
GvHD, and three percent for Grade 3-4 acute GvHD. There was no
persistent chronic GvHD.
“Data from our global BP-004 study continue to show consistent
and strong overall outcomes across a wide range of diseases,
suggesting the potential to enable curative stem cell transplants
from partially matched donors for more children,” said Rick Fair,
President and CEO of Bellicum Pharmaceuticals. “Data from this
ongoing BP-004 study in Europe, along with comparative data from an
observational trial of pediatric patients undergoing a stem cell
transplant from matched unrelated donors, are expected to support
the filing of a European Marketing Authorization Application in
mid-2018.”
Patient Populations Included in Study, By Disease
Type
Genetic Blood Diseases (n=59) |
Hematologic Cancers (n=39) |
Primary immune deficiencies (n=26) |
Acute lymphoblastic leukemia (ALL) (n=21) |
Thalassemia major (n=8) |
Acute myeloid leukemia (AML) (n=14) |
Hemophagocytic lymphohistiocytosis (HLH) (n=6) |
Other (n=4) |
Sickle cell disease (n=5) |
|
Other (n=14) |
|
|
|
Hemoglobinopathies and Erythroid Disorders Poster
Presentation Also today at the 22nd Congress of EHA,
updated results will be presented in patients with
hemoglobinopathies and erythroid disorders, including five patients
with sickle cell disease, in a poster presentation titled: “The Use
of BPX-501 Donor T-Cell Infusion (with Inducible Caspase 9 Suicide
Gene) Together with HLA-Haploidentical Stem Cell Transplant to
Treat Children with Hemoglobinopathies and Erythroid
Disorders.”
All 15 patients in the cohort showed normal hemoglobin levels
and remained transfusion-free at 180 days, with no
transplant-related mortality.
The Presidential Symposium oral presentation (abstract S146)
will take place today, June 23 at 3:45 – 4:00 PM CEST
(9:45-10:00 AM EDT) and the poster (abstract P381) will be reviewed
today at 5:15 – 6:45 PM CEST (11:15 AM – 12:45 PM EDT). Both
presentations will be made available in the Events and
Presentations section of the Bellicum website shortly after
the time of the respective presentation.
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 administering BPX-501 engineered T cells to speed
immune reconstitution, provide control over viral infections and
enhance Graft-versus-leukemia effect, 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 Bellicum PharmaceuticalsBellicum 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 our BPX-501,
CaspaCIDe, CID, CAR T and TCR programs; the effectiveness of
BPX-501 and rimiducid; their possible range of application and
potential curative effects and safety in the treatment of diseases;
and, the timing and success of our clinical trials. 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, 2016 and our report on Form
10-Q for the quarter ended March 31, 2017. 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, Inc.
Alan Musso, CFO
832-384-1116
amusso@bellicum.com
Media:
BMC Communications
Brad Miles
646-513-3125
bmiles@bmccommunications.com
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