105 patients treated with BPX-501 in BP-004
trial; 85 with more than 100 days of follow-up
Company reports encouraging results with
BPX-501 in high-risk pediatric leukemia patients
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 a review of results to date from its
multicenter BP-004 clinical trial of BPX-501 in the pediatric
setting, and provided an update on the regulatory pathway for
product registration in Europe at an investor and analyst event
held today. Clinical results on BPX-501 in children with
hematological malignancies were also presented in a poster
presentation at the American Society of Hematology (ASH) annual
meeting.
“We have made significant progress with our BPX-501 program
since initiating the BP-004 clinical trial in children two years
ago,” commented Tom Farrell, President and CEO of Bellicum
Pharmaceuticals. “To date, we have treated more than 100 patients
at leading pediatric transplant centers in Europe and the U.S.
Results thus far have been impressive—including GvHD- and
disease-free outcomes in children with a range of blood cancers and
genetic diseases, as reported this weekend in several presentations
at ASH. We’re also pleased with the progress we’ve made in
formalizing an expedient pathway to regulatory approval in Europe
with BPX-501 to treat both malignant and nonmalignant diseases. We
look forward to providing an update on our U.S. regulatory path in
the first half of 2017.”
Overall Summary of Results of BP-004 Study with BPX-501
(n=85)
The Company presented a review of results to date of the BP-004
study in pediatric patients with malignant and nonmalignant
diseases who underwent a haploidentical hematopoietic stem cell
transplant (HSCT) followed by an add-back of genetically modified
BPX-501 T cells. Eighty-five patients have been treated with
BPX-501 at multiple U.S. and European sites and followed for at
least 100 days (out of a total of 105 patients treated to date).
Only one case of transplant-related mortality has been reported,
unrelated to BPX-501 cells, and none out of 51 patients with
nonmalignant disorders. Compared to T-depleted haplo-transplants
alone, results have also shown significantly faster immune
recoveries, as well as reduced viral infections and reactivation,
and reductions in time to hospital discharge and
re-hospitalizations due to infection. In five cases where
uncontrolled acute GvHD was attributable to BPX-501 cells,
rimiducid was administered and symptoms resolved.
Results have been consistent across a range of diseases and
disorders where allogeneic HSCT is recognized as curative,
including hemoglobinopathies such as Beta Thalassemia Major (β0β0),
Sickle Cell Disease and Diamond-Blackfan Anemia; Primary Immune
Deficiencies such as Severe Combined Immune Deficiency (“Bubble
boy” disease) and Wiskott-Aldrich syndrome; leukemias and
lymphomas; and bone marrow failure syndromes. The Company also
reported that of six refractory AML patients treated under
compassionate use because of their active disease and not included
in the BP-004 summary data, 4 remain alive and in remission,
including two who are now 11 and 20 months post-transplant.
Regulatory Update for BPX-501 in the European
Union
The Company also announced today that the protocol assistance
provided by the European Medicines Agency (EMA) for the
registration study of BPX-501 in Europe is complete, and the
Company is finalizing plans for the BP-004 trial extension. The
Company will continue enrolling up to 40 additional patients with
malignant and nonmalignant diseases in the trial. Concurrent with
this study, the Company will initiate a comparator trial of
malignant and nonmalignant patients receiving a matched unrelated
donor (MUD) HSCT. This trial will include both retrospective
patients and up to 40 prospective patients. The primary endpoint
will be event-free survival (death, GvHD and infection) at 6
months.
Bellicum anticipates that it could pursue approval in the EU
under the “exceptional circumstances” provision. Exceptional
circumstances may be granted for medicines that treat very rare
diseases, or where controlled studies are impractical or not
consistent with accepted principles of medical ethics. The
Company continues to discuss the regulatory path to
approval in the U.S. with FDA and expects to provide an update in
the first half of 2017.
Also today at the ASH 2016 annual meeting, updated results were
presented in patients with acute myeloid leukemia (AML).
AML Highlights (Abstract #4683)
“T-cell depleted HLA-haploidentical allogeneic hematopoietic
stem cell transplantation (haplo-HSCT) followed by donor lymphocyte
infusion with T cells transduced with the inducible caspase 9 (iC9)
suicide gene in children with hematological malignancies”
In a poster session, investigators presented data in 33 children
with AML who received an α/β TCR-depleted haplo-HSCT and BPX-501
cells. The data indicate that infusion of BPX-501 results in low
non-relapse mortality, and low rates of acute and chronic Graft
versus Host Disease. Median follow-up was 8 months (range: 1–19
months). All 33 patients were high-risk CR1 (6/33) or CR2 (27/33).
Study outcomes include:
- All 33 patients engrafted with no
secondary graft failure
- One patient with steroid-resistant
Grade II skin acute GvHD received rimiducid with complete
resolution of disease in 24 hours
- One treatment-related mortality from
chronic GvHD was determined to be allograft-related and not from
BPX-501 T cells
- 3 of 33 patients have relapsed; the
probability of disease-free survival at 15 months is 83.6%
A replay of the investor and analyst meeting held today can be
accessed from the News & Events section of the Bellicum
website. An archived version of the webcast will be available for
at least two weeks following the event.
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
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.
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version on businesswire.com: http://www.businesswire.com/news/home/20161205006242/en/
Investors:Bellicum PharmaceuticalsAlan Musso,
832-384-1116CFOamusso@bellicum.comorMedia:BMC
CommunicationsBrad Miles,
917-570-7340bmiles@bmccommunications.comorBMC CommunicationsAmy
Bonanno, 914-450-0349abonanno@bmccommunications.com
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