Pediatric patients with beta thalassemia, SCID, and Wiskott-Aldrich
Syndrome are disease-free following haplo transplant with BPX-501 T cells
Additional details of ongoing BPX-501 trial and adoptive T cell
programs to be presented at ASH 2015
HOUSTON--(BUSINESS WIRE)--Nov. 9, 2015--
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 reported financial results for the third quarter of
2015 and provided an update on recent developments.
“During the third quarter we continued to make good progress in the
advancement of our stem cell transplant, CAR-T and TCR programs,” said
Tom Farrell, President and Chief Executive Officer of Bellicum. “We are
particularly excited about the interim data that will be presented at
ASH 2015 that underscore the potential of our lead product candidate
BPX-501 to improve outcomes for patients with blood cancers and
inherited blood diseases undergoing haplo transplantation. BPX-501 could
be used in the treatment of over 60 rare diseases, enabling a potential
curative transplant for many of those disorders, including sickle cell
disease, where treatment-related mortality has severely limited the
adoption of allogeneic transplantation."
Added Mr. Farrell, “We are also looking forward to multiple
presentations at ASH highlighting our CAR-T and TCR adoptive cell
therapy programs and the power of our cellular control technologies. We
continue to anticipate that three new product candidates, BPX-601,
BPX-701, and BPX-401 will be initiating clinical development in the
first half of 2016.”
Program Updates:
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BPX-501 at ASH 2015: Company to present interim data from the
BP-004 ongoing Phase 1/2 clinical trial. Bellicum will present
initial data in malignant and non-malignant blood diseases at the
upcoming 26th Annual Meeting of the American Society of
Hematology in early December in Orlando, Florida. The open label dose
escalation trial in pediatric patients is evaluating whether BPX-501 T
cells from a haploidentical donor, administered following a T-depleted
hematopoietic stem cell transplant (HSCT), are safe and can enhance
immune reconstitution.
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Enrollment in the BP-004 trial continues at a strong pace at sites in
Europe and in the U.S., with 53 pediatric patients enrolled as of
October 31st, including one patient with sickle cell
disease.
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Among non-malignant patients in the trial treated to date are four
children with beta thalassemia major (in its most severe form, the
β0/β0 type) who have successfully undergone the HSCT transplant
procedure with the add-back of BPX-501 gene-modified T cells. Within
two weeks of the transplant procedure all patients became blood
transfusion-independent. All four patients are alive, disease-free and
remain transfusion-independent.
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At a medical symposium in Parma, Italy in early September, principal
investigator Dr. Franco Locatelli shared initial outcomes from this
ongoing trial. His presentation included the first 15 children
enrolled in the clinical study with non-malignant inherited disorders
(four with severe combined immunodeficiency, or SCID; three with
Wiskott-Aldrich Syndrome; four with Fanconi anemia; three with beta
thalassemia, and one with hemophagocytic lymphohistiocytosis) who
received the BPX-501 T cell add-back following HLA-partially matched
family donor HSCT. In all cases, the BPX-501 T cells engrafted and
expanded with no secondary graft failures. Grade II skin-only graft
versus host disease (GvHD) was observed in one patient, and promptly
resolved with topical steroids. None of the patients so far have
developed chronic GvHD, and all are alive and disease-free.
-
BPX-601 preclinical data to be presented at ASH 2015. Bellicum
continues to advance its first GoCAR-T™ product candidate, containing
our proprietary iMC (inducible MyD88/CD40) activation switch, designed
to treat solid tumors expressing prostate stem cell antigen (PSCA).
The Company expects to file an IND for the initial indication of
pancreatic cancer by the end of 2015. In addition to pancreatic
cancer, PSCA is also expressed in prostate, ovarian, bladder,
esophageal and gastric cancers. BPX-601 is differentiated from
traditional CAR-T therapies with an MC co-stimulatory domain that is
activated by administration of rimiducid.
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BPX-401 CIDeCAR™ preclinical data to be highlighted in an oral
presentation at ASH 2015. BPX-401, a CIDeCAR™ product candidate
incorporating Bellicum’s proprietary MC co-stimulatory domain and the
CaspaCIDe® safety switch, is designed to target
blood cancers expressing CD19. BPX-401 is expected to enter the clinic
in the first half of 2016.
-
BPX-701 progressing toward the clinic. Bellicum continues to
advance its proprietary T cell receptor (TCR) product candidate
designed to target solid tumors expressing the
preferentially-expressed antigen in melanoma, or PRAME. The Company
has identified clinical sites for its BPX-701 CaspaCIDe®-enabled
TCR product candidate and expects to file an IND by the end of 2015,
initially for the indications of PRAME-expressing sarcomas and uveal
melanoma.
Third Quarter and Nine Months Ended September 30, 2015 Financial
Results:
Bellicum reported a net loss of $13.4 million for the third quarter of
2015 and $31.7 million for the nine months ended September 30, 2015,
compared to a net loss of $4.1 million and $9.7 million for the
comparable periods in 2014. The results included non-cash, share-based
compensation charges of $2.3 million and $5.9 million for the third
quarter and nine months ended September 30, 2015, respectively, and $0.1
million and $0.2 million for the comparable periods in 2014. As of
September 30, 2015, cash and investments totaled $163.2 million.
Grant revenues were $0.1 million and $0.2 million for the three and nine
months ended September 30, 2015, respectively, and $0.7 million and $1.8
million during the comparable periods in 2014. The decrease in grant
revenues was primarily due to the June 2014 expiration of the Company’s
grant award from the Cancer Prevention and Research Institute of Texas.
Research and development expenses were $9.8 million and $23.5 million,
respectively, for the three and nine months ended September 30, 2015,
compared to $2.3 million and $7.9 million during the comparable periods
in 2014. The higher expenses in the 2015 periods were primarily due to
an increase in manufacturing and clinical expenses as a result of
increased patient enrollment in our BPX-501 clinical trials, increased
expenses for the IND-enabling activities on the Company’s CAR-T and TCR
product candidates and increased personnel and infrastructure costs.
General and administrative expenses were $3.9 million and $8.9 million,
respectively, for the three and nine months ended September 30, 2015,
compared to $1.3 million and $2.3 million during the comparable periods
in 2014. The increased G&A expenses in 2015 were due to the growth of
the organization and the costs associated with operating as a public
company.
About Bellicum Pharmaceuticals
Bellicum is a clinical stage biopharmaceutical company focused on
discovering and developing cellular immunotherapies for cancers and
orphan inherited blood disorders. The Company is using its proprietary
Chemical Induction of Dimerization, or CID, technology platform to
engineer and control components of the immune system in real time.
Bellicum is developing next-generation product candidates in some of the
most important areas of cellular immunotherapy, including hematopoietic
stem cell transplantation, or HSCT, and CAR-T and TCR cell therapies.
More information can be found at www.bellicum.com.
*CaspaCIDe® and DeCIDe® are
trademarks registered with the U.S. Patent and Trademark Office.
CIDeCAR™ and GoCAR-T™ are trademarks of Bellicum Pharmaceuticals.
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: the
timing and success of our clinical trials, including the rate and
progress of enrollment in such trials; the timing of an IND filing for
BPX-601 and BPX-701; our research and development activities and
expenses relating to BPX-501, BPX-401, BPX-601, BPX-701, CaspaCIDe, and
GoCAR-T; the effectiveness of BPX-501, its possible range of application
and its potential curative effects and safety in the treatment of blood
cancers and inherited blood diseases; and the potential applications and
effectiveness of our product candidates, including as compared to other
treatment options and competitive therapies. 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, 2014 and our Report on Form 10-Q for the quarter
ended June 30, 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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BELLICUM PHARMACEUTICALS, INC.
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Unaudited Condensed Balance Sheets
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(in thousands)
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September 30,
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December 31,
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2015
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2014
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Current Assets:
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Cash and cash equivalents
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$92,487
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$191,602
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Investment securities, available-for-sale - short-term
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20,744
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-
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Receivables and other current assets
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2,090
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1,620
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Non-Current Assets:
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Investment securities, available-for-sale, long-term
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50,018
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-
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Property and equipment, net
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6,323
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2,427
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Other assets, net
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446
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145
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Total assets
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$172,108
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$195,794
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Current Liabilities:
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Accounts payable and other accrued liabilities
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$4,849
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$3,372
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Other current liabilities
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170
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264
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Long-Term Liabilities:
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Other liabilities, net of current portion
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984
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522
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Total Stockholders' Equity
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166,105
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191,636
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Total liabilities and stockholders' equity
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$172,108
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$195,794
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BELLICUM PHARMACEUTICALS, INC.
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Unaudited Condensed Statements of Operations
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(in thousands, except share and per share amounts)
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Three Months Ended
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Nine Months Ended
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September 30,
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September 30,
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2015
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2014
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2015
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2014
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Grant Revenues
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$57
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$660
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$248
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$1,766
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Operating Expenses:
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Research and development
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9,792
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2,257
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23,522
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7,881
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General and administrative
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3,882
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1,300
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8,856
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2,332
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Total operating expenses
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13,674
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3,557
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32,378
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10,213
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Operating loss
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(13,617)
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(2,897)
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(32,130)
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(8,447)
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Interest income (expense), net
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209
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(1,199)
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430
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(1,220)
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Net Loss
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$(13,408)
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$(4,096)
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$(31,700)
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$(9,667)
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Preferred stock dividends
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-
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(328)
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-
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(1,432)
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Net loss attributable to common shareholders
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$(13,408)
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$(4,424)
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$(31,700)
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$(11,099)
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Net loss per share attributable to common
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shareholders, basic and diluted
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$(0.51)
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$(2.08)
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$(1.21)
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$(5.45)
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Weighted average common shares outstanding, basic and diluted
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26,376,456
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2,124,247
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26,301,914
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2,036,691
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Net loss
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$(13,408)
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$(4,096)
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$(31,700)
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$(9,667)
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Other comprehensive loss:
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Unrealized loss on investment securities
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-
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-
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(204)
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-
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Comprehensive loss
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$(13,408)
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$(4,096)
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$(31,904)
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$(9,667)
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View source version on businesswire.com: http://www.businesswire.com/news/home/20151109006666/en/
Source: Bellicum Pharmaceuticals, Inc.
Investors:
Bellicum Pharmaceuticals
Alan Musso, CFO
832-384-1116
amusso@bellicum.com
or
Media:
BMC
Communications
Brad Miles
646-513-3125
bmiles@bmccommunications.com
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