Bellicum Announces Updated Clinical Data from BPX-501 Study in Children with Primary Immune Deficiencies and Hemoglobinopathies

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

SAN DIEGO–(BUSINESS WIRE)–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.

Contacts

Investors:
Bellicum Pharmaceuticals
Alan Musso, CFO,
832-384-1116
amusso@bellicum.com
or
Media:
BMC
Communications
Brad Miles, 917-570-7340
bmiles@bmccommunications.com
or
BMC
Communications
Amy Bonanno, 914-450-0349
abonanno@bmccommunications.com