Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in
developing novel, controllable cellular immunotherapies for cancers
and orphan inherited blood disorders, today reported financial
results for the third quarter ended September 30, 2018, and
provided an update on recent developments.
“We made significant progress during the quarter across our
programs. Rivo-cel remains on track for MAA filing in the E.U. in
2019 for pediatric patients with acute leukemias and nonmalignant
blood diseases,” said Bellicum’s President & CEO Rick Fair. “We
also received and incorporated health authority input on our
planned late-stage trial in adult AML and MDS and remain on track
to initiate the trial by year-end.”
Continued Mr. Fair: “In our GoCAR-T programs, we are nearing
completion of the dose escalation portion of our Phase 1/2 study of
BPX-601 in solid tumors, and expect to report preliminary results
from the lower dose cohorts in patients with advanced pancreatic
cancer in December. We also made substantial progress toward IND
applications for two new dual-switch GoCAR-T candidates in
2019.”
PROGRAM HIGHLIGHTS AND CURRENT UPDATES
On Track to Initiate Phase 2/3 Study of Rivo-cel in
Adult AML and MDS by Year-endBased on impressive clinical
trial results to date with rivo-celTM (rivogenlecleucel, formerly
called BPX-501) in pediatric leukemia patients, Bellicum is
finalizing its plans to initiate a global Phase 2/3 trial in adult
patients with intermediate/high-risk acute myeloid leukemia (AML)
and myelodysplastic syndromes (MDS) by the end of the year. The
Company completed review of the study protocol with the U.S. FDA in
the third quarter and has incorporated its input into the design of
the trial.
Rivo-cel Pediatric Registration Trials on Track for E.U.
Filing in 2019; Significant Data Update at ASH
2018Prospective enrollment was recently completed in the
BP-004 and C-004 E.U. registration trials of pediatric patients
with leukemias, lymphomas and inherited blood disorders. These
trials will serve as the basis for the Company’s planned 2019
European Marketing Authorization Application regulatory filing. In
December, Bellicum will present interim data at the American
Society of Hematology (ASH) Annual meeting, with final results
expected in early 2019. Among the highlights will be late interim
analyses of the overall results from the BP-004 trial in children
with acute leukemias and nonmalignant blood diseases, as well as
the comparator C-004 trial—a multicenter, observational study of
similar pediatric patients receiving a matched unrelated donor
(MUD) transplant. Disease outcomes from several patient subsets, as
well as the cumulative clinical experience of patients from BP-004
who received rimiducid to treat steroid refractory
Graft-versus-Host-Disease will also be presented. ASH 2018 is being
held in San Diego, California on December 1-4.
Commercial Planning Activities for Rivo-cel Continue to
Advance in Europe Under the recently appointed General
Manager of Europe, Thierry Darcis, M.D., M.B.A., Bellicum continues
to build out an E.U.-based team to prepare for the
commercialization of rivo-cel, if approved. Dr. Darcis and his
leadership team have extensive experience launching orphan products
in Europe, and Dr. Darcis has previously led successful product
introductions for ViroPharma and NPS Pharmaceuticals. He also held
leadership roles with Zogenix, Novartis and GlaxoSmithKline.
Initial Clinical Data on BPX-601 To Be Presented at ESMO
Immuno-Oncology ConferenceBPX-601 is Bellicum’s first
GoCAR-T® clinical candidate incorporating the co-activation domain,
iMC. The Company expects to report preliminary safety and
translational findings from the lower cell-dose cohorts of its
Phase 1 dose-escalation safety study in late-stage pancreatic
cancer patients at the European Society for Medical Oncology
Immuno-Oncology Conference in Geneva, Switzerland in December. The
Company is evaluating BPX-601 in adults with relapsed or refractory
pancreatic, gastric, and prostate cancers who test positive for
prostate stem cell antigen (PSCA).
Preclinical Dual-Switch Candidates On Track to Enter
Clinic in 2019 Bellicum’s research team continues to
advance its next-generation GoCAR-T projects, which have been
designed with both activation and safety switch technologies to
potentially enhance efficacy and safety. The Company expects to
submit IND applications for two new dual-switch GoCAR-T product
candidates in 2019.
Third Quarter 2018 Financial Results Bellicum
reported a net loss of $23.8 million for the third
quarter of 2018 and $70.8 million for the nine months
ended September 30, 2018, respectively, compared to a net loss
of $23.4 million and $69.9 million for the
comparable periods of 2017. The results included non-cash,
share-based compensation charges of $3.7
million and $10.9 million for the third quarter and
nine months ended September 30, 2018, and $3.7
million and $10.2 million for the comparable periods
in 2017.
As of September 30, 2018, cash, restricted cash and
investments totaled $118.4 million. Based on current operating
plans, Bellicum continues to expect that current cash resources
will be sufficient to meet operating requirements through 2019.
Research and development expenses were $16.4
million and $51.4 million, for the three and nine months
ended September 30, 2018, respectively, compared to $18.1
million and $51.4 million during the comparable
periods in 2017.
General and administrative expenses were $7.0
million and $18.0 million for the three and nine
months ended September 30, 2018, respectively, compared
to $4.6 million and $16.0 million during the
comparable periods in 2017.
At September 30, 2018, Bellicum had 43,351,159 shares of
common stock outstanding.
About Rivo-cel (BPX-501) Rivo-celTM
(rivogenlecleucel) is an allogeneic polyclonal T cell product
designed to reduce relapse of leukemia following a stem cell
transplant. The cell treatment contains a diverse repertoire of T
cells which may contribute to a robust graft vs. leukemia effect.
Rivo-cel’s antiviral benefits may also reduce morbidity and
mortality in patients susceptible to infection following a
transplant. The product’s CaspaCIDe® safety switch enables this
approach by allowing physicians to reduce the number of
alloreactive cells in the event of uncontrolled GvHD. Rivo-cel
addresses a major unmet need in adult and pediatric leukemia,
lymphoma and genetic blood disease patients following a
haploidentical stem cell transplant.
About BPX-601BPX-601, the Company’s first
GoCAR-T® product candidate, incorporates iMC, Bellicum’s
inducible co-activation domain. iMC (inducible MyD88/CD40) is
designed to provide a powerful boost to T cell proliferation and
persistence, and enable the CAR-T to override key immune inhibitory
mechanisms, including PD-1 and TGF-beta. BPX-601 is being evaluated
as a treatment for solid tumors expressing prostate stem cell
antigen (PSCA), including pancreatic, prostate and gastric
cancers.
About Bellicum Pharmaceuticals Bellicum is
a clinical stage biopharmaceutical company striving to deliver
cures through controllable cell therapies. The Company’s
next-generation product candidates are differentiated by powerful
cell signaling technologies designed to produce more effective
CAR-T, TCR and allogeneic T cell therapies. Its lead product
candidate, rivo-celTM, is an allogeneic polyclonal T cell therapy
that has shown promising clinical trial results in reducing
leukemia relapse after a stem cell transplant. Bellicum’s lead
GoCAR-T® candidate, BPX-601, is designed to be a more efficacious
CAR-T cell product capable of overriding key immune inhibitory
mechanisms. 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. 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 rivo-celTM,
BPX-601, rimiducid, CaspaCIDe®, iMC, dual switch, CAR-T and TCR
programs; the effectiveness of rivo-cel and BPX-601, their possible
ranges 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
current and planned clinical trials, including the timing of
receipt of data from such clinical trials and the timing of our
reports of such data; the timing and success of regulatory filings;
our ability to build a successful commercial organization in
Europe; our research and development activities relating to our
GoCAR-T technologies; the presentation of our preclinical and
clinical data at medical or scientific meetings and our cash uses
and cash runway. 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 quarterly report on Form 10-Q for the three months
ended September 30, 2018 and our annual report on Form 10-K for the
year ended December 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.
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BELLICUM
PHARMACEUTICALS, INC. |
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Unaudited Condensed Consolidated Balance
Sheets |
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(in thousands) |
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September 30, |
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December 31, |
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2018 |
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2017 |
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Current Assets: |
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Cash and cash
equivalents |
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$ |
53,027 |
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$ |
38,839 |
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Investment
securities, available-for-sale, short-term |
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59,720 |
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60,057 |
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Receivables and other
current assets |
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2,792 |
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|
2,754 |
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Non-Current
Assets: |
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Investment
securities, available-for-sale, long-term |
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- |
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1,368 |
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Property and equipment,
net |
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22,402 |
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25,942 |
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Restricted cash |
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|
5,635 |
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|
6,190 |
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Other assets |
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|
396 |
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|
378 |
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Total
Assets |
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$ |
143,972 |
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$ |
135,528 |
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Current
Liabilities: |
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Accounts payable and
other accrued liabilities |
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$ |
10,680 |
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$ |
9,679 |
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Other current
liabilities |
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|
3,743 |
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|
|
2,477 |
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Long-Term
Liabilities: |
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Long-term debt |
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35,609 |
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34,946 |
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Other liabilities, net
of current portion |
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|
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|
1,483 |
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|
3,778 |
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Total stockholders'
Equity |
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|
92,457 |
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|
84,648 |
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Total
Liabilities and Stockholders' Equity |
|
|
|
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$ |
143,972 |
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|
$ |
135,528 |
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BELLICUM
PHARMACEUTICALS, INC. |
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Unaudited Condensed Consolidated 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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2018 |
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2017 |
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2018 |
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2017 |
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Grant revenues |
$ |
292 |
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$ |
126 |
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$ |
808 |
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$ |
254 |
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Operating
Expenses: |
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Research
and development |
|
16,413 |
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|
18,101 |
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|
|
51,361 |
|
|
|
51,355 |
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License
fees |
|
139 |
|
|
|
151 |
|
|
|
319 |
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|
|
849 |
|
General
and administrative |
|
6,968 |
|
|
|
4,579 |
|
|
|
18,027 |
|
|
|
15,992 |
|
Total operating
expenses |
|
23,520 |
|
|
|
22,831 |
|
|
|
69,707 |
|
|
|
68,196 |
|
Operating
loss |
|
(23,228 |
) |
|
|
(22,705 |
) |
|
|
(68,899 |
) |
|
|
(67,942 |
) |
|
|
|
|
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Interest expense, net
of interest income |
|
(573 |
) |
|
|
(726 |
) |
|
|
(1,917 |
) |
|
|
(1,919 |
) |
Net loss
attributable to common shareholders |
$ |
(23,801 |
) |
|
$ |
(23,431 |
) |
|
$ |
(70,816 |
) |
|
$ |
(69,861 |
) |
Net loss
per share attributable to common |
|
|
|
|
|
|
|
shareholders, basic and diluted |
$ |
(0.55 |
) |
|
$ |
(0.71 |
) |
|
$ |
(1.81 |
) |
|
$ |
(2.24 |
) |
|
|
|
|
|
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|
Weighted average common
shares outstanding, basic and diluted |
|
43,334,727 |
|
|
|
33,178,611 |
|
|
|
39,168,559 |
|
|
|
31,204,521 |
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Source: Bellicum Pharmaceuticals
Investors:
Solebury Trout
Chad Rubin
646-378-2947
crubin@soleburytrout.com
Media:
Solebury Trout
Brad Miles
646-513-3125
bmiles@soleburytrout.com
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