Company updates BPX-501 program
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 second quarter ended June 30, 2017, and
provided an update on recent developments.
“Since I joined the Company six months ago, we have conducted a
thorough review of our strategy and operations, and are very
optimistic about the opportunities before us,” said Rick Fair,
Bellicum’s President & Chief Executive Officer. “We continue to
be encouraged by the results from our ongoing BPX-501 pediatric
studies and our progress toward a filing in Europe. We have
adjusted our plans for U.S. registrational trials to enable an
efficient path to seeking approvals for the greatest areas of unmet
need. Lastly, we continue to be excited about the clinical progress
of our CAR T and TCR product candidates, and the application of our
molecular switch platform for future pipeline
expansion.” PROGRAM HIGHLIGHTS AND CURRENT
UPDATES
BPX-501Adjunct T-cell therapy incorporating the
CaspaCIDe® safety switch, administered after a haploidentical
hematopoietic stem cell transplant (haplo-HSCT), to improve
outcomes and reduce mortality
- Data Update Suggests BPX-501 Improves Outcomes of
Haploidentical Stem Cell Transplants During the
Presidential Symposium of the 22nd Congress of the European
Hematology Association (EHA) in June, Bellicum reported data from
98 pediatric patients within the BP-004 trial which showed rapid
immune recovery, a low incidence of transplant-related mortality, a
reduction in viral infections and a low rate of Graft versus Host
Disease (GvHD) that was manageable with either standard treatments
or rimiducid. The data suggest BPX-501 could improve outcomes of
haploidentical stem cell transplants, providing an option for the
many patients who could benefit from a life-saving transplant but
lack a matched donor.
- Positive Clinical Results of BPX-501 in Pediatric
LeukemiasAlso at EHA, Bellicum reported data from the
BP-004 trial in a cohort of 47 pediatric patients with acute
leukemias who lack a matched donor. The data showed rapid immune
reconstitution and low rates of relapse and mortality, suggesting
that BPX-501 may offer benefits in combination with HSCT in acute
leukemia patients.
- European BP-004 Pivotal Clinical Trial
ProgressingEnrollment in the pivotal EU BP-004 trial
remains on track for completion by the end of 2017. Bellicum
expects to initiate an observational trial in pediatric patients
receiving transplants from matched unrelated donors (MUD) without
BPX-501 in the third quarter. Outcomes from these trials are
expected to be the basis for filings of European Marketing
Authorization Applications for BPX-501 and rimiducid. The Company
expects to report top-line results of these studies in the second
half of 2018, with MAA filings planned for 2019.
- Company Clarifies U.S. Clinical Development
StrategyBellicum is finalizing plans for the design of
registrational trials of BPX-501 in the U.S. The Company’s current
plans include conducting a controlled clinical trial in adult
patients with acute myeloid leukemia (AML), which it expects to
fund in part through its $16.9 million Product Development Award
from the Cancer Prevention and Research Institute of Texas
("CPRIT"). In the pediatric non-malignant setting, Bellicum is
designing a registrational trial to evaluate BPX-501 in a distinct
subset of orphan inherited blood disorders.
BPX-601
- Phase 1 BPX-601 Clinical Trial
ContinuesBPX-601 is Bellicum’s novel GoCAR-T product
candidate, which is designed with its proprietary iMC activation
switch to allow control over the level of stimulation and
proliferation of the modified T cells. Enrollment and treatment is
ongoing in Bellicum’s Phase 1 trial in patients with nonresectable
pancreatic cancer who test positive for prostate stem cell antigen
(PSCA).
BPX-701
- Phase 1 BPX-701 Clinical Trial Continues
BPX-701 is a high affinity TCR product candidate designed with the
CaspaCIDe safety switch, enabling the elimination or reduction of
the engineered cells in the event of severe toxicities. Dosing has
been initiated in the Company’s Phase 1 clinical trial in patients
with refractory or relapsed AML and myelodysplastic syndromes (MDS)
who test positive for preferentially-expressed antigen in melanoma
(PRAME).
CORPORATE UPDATE
- Addition of Chief Business Officer to Expand
Partnership Opportunities Greg Naeve, Ph.D., an
accomplished product strategy and business development executive,
is joining Bellicum’s leadership team in August 2017 from Pfizer,
where he led efforts to identify and implement multiple strategic
partnerships and translational science collaborations across Pfizer
Worldwide R&D, including CAR T alliances with Cellectis and
Servier.
PRECLINICAL RESEARCH
- In April, Bellicum reported positive preclinical data at AACR
on its novel dual-switch technology incorporated into CAR T and TCR
constructs, an approach offering the possibility of both activating
cells to enhance efficacy and eliminating them to manage toxicity.
Bellicum is working to incorporate its dual-switch technology into
future CAR T and TCR product candidates.
- The Company continues to work with academic collaborators to
explore the applicability of CaspaCIDe in CD19 CARs, the first of
which is expected to enter the clinic in the second half of this
year in patients with B-cell malignancies.
SECOND QUARTER AND SIX MONTHS ENDED JUNE 30, 2017
FINANCIAL RESULTS
Bellicum reported a net loss of $24.5 million for the second
quarter of 2017 and $46.4 million for the six months ended June 30,
2017, compared to a net loss of $16.5 million and $31.6 million for
the comparable periods of 2016. The results included non-cash,
share-based compensation charges of $3.2 million and $6.6 million
for the second quarter and six months ended June 30, 2017 and $3.1
million and $6.2 million for the comparable periods in 2016.
As of June 30, 2017, cash, restricted cash and investments
totaled $139.0 million. Based on current operating plans, Bellicum
continues to expect to end 2017 with approximately $85 to $95
million in cash and investments, and that current cash resources
will be sufficient to meet operating requirements through 2018.
Research and development expenses were $18.0 million and $33.3
million, for the three and six months ended June 30, 2017,
respectively, compared to $12.0 million and $22.9 million during
the comparable periods in 2016. The higher expenses in the 2017
periods were primarily due to an increase in clinical development
and manufacturing costs due to increased enrollment in trials,
principally BPX-501, and increased personnel expenses, overhead
charges and manufacturing facility start-up costs.
General and administrative expenses were $5.5 million and $11.4
million for the three and six months ended June 30, 2017,
respectively, compared to $4.2 million and $8.5 million during the
comparable periods in 2016. The higher expenses in the 2017 periods
were primarily due to the Company’s overall growth, including an
increase in personnel related costs, principally due to hiring
additional employees and severance costs, higher facility costs and
increased legal, accounting and travel expenses.
At June 30, 2017, Bellicum had 33,193,229 shares of common stock
outstanding.
Conference Call and WebcastBellicum management
will host a webcast and conference call at 5:00 p.m. Eastern today
to discuss the financial results. To access the call, participants
should dial 877-407-3103 (U.S. domestic) and 201-493-6791
(international) at least 10 minutes prior to the start of the call.
The event will be webcast live and can also be accessed in
the Events & Presentations section
of bellicum.com. An archived version of the webcast will be
available for replay in the Investors & Media section
of the Bellicum website for at least two weeks following the
call.
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 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,
BPX-701, BPX-601, rimiducid, CaspaCIDe, dual switch, CAR T and TCR
programs; 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 our BP-004 study and comparator trials;
the rate and progress of enrollment in our clinical trials for
BPX-501, BPX-701 and BPX-601, including our planned registration
trials for BPX-501 and rimiducid; the timing of regulatory filings
for BPX-501 and rimiducid; our research and development activities
relating to our GoCAR-T and GoTCR technologies, and our
collaborations with academic institutions and other companies.
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 June 30, 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.
BELLICUM PHARMACEUTICALS, INC. |
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Unaudited Condensed Consolidated Balance
Sheets |
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(in thousands) |
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|
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June |
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December |
|
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|
|
|
|
30, 2017 |
|
31, 2016 |
Current Assets: |
|
|
|
|
|
|
|
|
Cash and cash equivalents |
|
|
|
|
|
$ |
75,148 |
|
|
$ |
33,140 |
|
Investment securities, available-for-sale, short-term |
|
|
|
|
|
|
|
|
55,081 |
|
|
|
70,632 |
|
Prepaid expenses and other current assets |
|
|
|
|
|
|
2,896 |
|
|
|
1,838 |
|
|
|
|
|
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|
|
|
|
Non-Current Assets: |
|
|
|
|
|
|
|
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Investment securities, available-for-sale, long-term |
|
|
|
|
|
|
|
|
4,405 |
|
|
|
— |
|
Property and equipment, net |
|
|
|
|
|
|
25,458 |
|
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|
16,504 |
|
Restricted cash |
|
|
|
|
|
|
4,383 |
|
|
|
9,640 |
|
Other assets |
|
|
|
|
|
|
353 |
|
|
|
283 |
|
Total assets |
|
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|
|
|
$ |
167,724 |
|
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$ |
132,037 |
|
|
|
|
|
|
|
|
|
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Current Liabilities: |
|
|
|
|
|
|
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Accounts payable and other accrued liabilities |
|
|
|
|
|
|
12,267 |
|
|
|
12,986 |
|
Current maturities of long-term debt |
|
|
|
|
|
|
3,412 |
|
|
|
1,787 |
|
Other current liabilities |
|
|
|
|
|
|
283 |
|
|
|
340 |
|
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|
|
|
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Long-Term Liabilities: |
|
|
|
|
|
|
|
|
Long-term debt |
|
|
|
|
|
|
27,116 |
|
|
|
18,436 |
|
Other liabilities, net of current portion |
|
|
|
|
|
|
1,939 |
|
|
|
1,914 |
|
|
|
|
|
|
|
|
|
|
Total Stockholders' Equity |
|
|
|
|
|
|
122,707 |
|
|
|
96,574 |
|
Total liabilities and stockholders' equity |
|
|
|
|
|
$ |
167,724 |
|
|
$ |
132,037 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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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 |
|
Six Months Ended |
|
|
June 30, |
|
June 30, |
|
|
|
2017 |
|
|
|
2016 |
|
|
|
2017 |
|
|
|
2016 |
|
|
|
|
|
|
|
|
|
|
Grant Revenues |
|
$ |
- |
|
|
$ |
101 |
|
|
$ |
128 |
|
|
$ |
193 |
|
|
|
|
|
|
|
|
|
|
Operating Expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
|
17,959 |
|
|
|
12,031 |
|
|
|
33,254 |
|
|
|
22,889 |
|
License fees |
|
|
343 |
|
|
|
150 |
|
|
|
698 |
|
|
|
280 |
|
General and administrative |
|
|
5,486 |
|
|
|
4,179 |
|
|
|
11,413 |
|
|
|
8,463 |
|
Total operating expenses |
|
|
23,788 |
|
|
|
16,360 |
|
|
|
45,365 |
|
|
|
31,632 |
|
Operating loss |
|
|
(23,788 |
) |
|
|
(16,259 |
) |
|
|
(45,237 |
) |
|
|
(31,439 |
) |
|
|
|
|
|
|
|
|
|
Interest expense, net |
|
|
(669 |
) |
|
|
(250 |
) |
|
|
(1,193 |
) |
|
|
(145 |
) |
Net loss attributable to common shareholders |
|
$ |
(24,457 |
) |
|
$ |
(16,509 |
) |
|
$ |
(46,430 |
) |
|
$ |
(31,584 |
) |
|
|
|
|
|
|
|
|
|
Net loss per share attributable to common shareholders, basic and
diluted |
|
$ |
(0.74 |
) |
|
$ |
(0.61 |
) |
|
$ |
(1.54 |
) |
|
$ |
(1.17 |
) |
|
|
|
|
|
|
|
|
|
Weighted-average common shares outstanding, basic and diluted |
|
|
33,074,463 |
|
|
|
26,910,284 |
|
|
|
30,201,116 |
|
|
|
26,896,405 |
|
|
Investors:
Bellicum Pharmaceuticals, Inc.
Alan Musso, CFO
832-384-1116
amusso@bellicum.com
Media:
BMC Communications
Brad Miles
646-513-3125
bmiles@bmccommunications.com
Source: Bellicum Pharmaceuticals
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