Bellicum Pharmaceuticals Reports First Quarter 2016 Financial Results
“We continue to make good progress advancing our stem cell transplant,
CAR T and TCR programs,” said
Continued Mr. Farrell, “We are also preparing to advance three of our next-generation CAR T and TCR product candidates into the clinic in 2016. We believe the inclusion of our proprietary cellular control switches and our novel MC co-stimulatory domains may improve the function of T-cell therapies for attacking both solid and hematologic cancers.”
PROGRAM HIGHLIGHTS
BPX-501
-
Reported new interim data from BP-004 trial, showing disease-free
outcomes in pediatric patients, including those with blood cancers
who had undergone T-depleted, haploidentical hematopoietic stem cell
transplantation (HSCT) followed by BPX-501 donor T-cell replacement.
At the 42nd Annual Meeting of the European Society for Blood and
Marrow Transplantation (EBMT), preliminary outcomes of 17 pediatric
leukemia patients were reviewed in an oral presentation, showing that
BPX-501 cells expand in vivo and persist over time,
contributing to adaptive immunity. Additionally, the relapse rate
compared favorably with that of historical controls, with 16 of 17
patients in the trial showing disease-free outcomes. The median
follow-up period for these patients was approximately seven months.
Initial outcomes for nonmalignant patients at the same site were also
reviewed, which showed that all 24 children treated remain
disease-free (median follow-up period of approximately seven months),
consistent with earlier results presented at the 57th Annual Meeting
of the
American Society of Hematology (ASH) inDecember 2015 . Transplant-related mortality (TRM) was 0% (0 of 49) across all patients reported. -
Received orphan drug designation from
FDA for the combination of BPX-501 genetically modified T cells and activator agent rimiducid as “replacement T-cell therapy for the treatment of immunodeficiency and Graft versus Host Disease after allogeneic hematopoietic stem cell transplant.” -
Preparing to meet with the
European Medicines Agency and U.S. FDA, with the goal of defining the path to regulatory filing and approval.
BPX-601: Preparing to initiate a Phase 1 clinical trial with BPX-601 GoCAR-T™ product candidate in mid-2016 in the initial indication of non-resectable pancreatic cancer. GoCAR-T contains Bellicum’s proprietary iMC (inducible MyD88/CD40) activation switch and is designed to treat solid tumors expressing prostate stem cell antigen (PSCA).
BPX-701: Preparing to initiate a Phase 1 clinical trial with BPX-701 high affinity T cell receptor (TCR) product candidate in mid-2016. BPX-701 incorporates the CaspaCIDe® safety switch and is designed to target malignant cells expressing the preferentially-expressed antigen in melanoma, or PRAME. Initial planned indications include Refractory or Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndromes, with an additional clinical trial planned for metastatic uveal melanoma.
BPX-401: Continued to advance CIDeCAR CAR T therapy, with plans to initiate clinical development in the second half of 2016.
First Quarter 2016 Financial Results:
Bellicum reported a net loss of
Research and development expenses were
General and administrative expenses were
About
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 rimiducid, CaspaCIDe,
CIDeCAR, GoCAR-T, MC, or iMC; the effectiveness of rimiducid, CaspaCIDe,
CIDeCAR, GoCAR-T, MC, or iMC, their possible range of application and
potential curative effects and safety in the treatment of diseases; the
timing and success of our clinical trials, including the rate and
progress of enrollment in our BP-004 clinical trial and clinical trials
for BPX-601, BPX-701 and BPX-401; the timing of regulatory filings for
BPX-501 and for rimiducid; our research and development activities
relating to BPX-501, BPX-601, BPX-701 and BPX-401; and the potential
applications and effectiveness of our product candidates BPX-501,
BPX-601, BPX-701 and BPX-401, 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
| BELLICUM PHARMACEUTICALS, INC. | ||||||||
| Condensed Balance Sheets | ||||||||
| (in thousands) | ||||||||
| March 31, 2016 | December 31, 2015 | |||||||
| Current Assets: | ||||||||
| Cash and cash equivalents | $ | 61,790 | $ | 70,241 | ||||
| Investment securities, available-for-sale - short-term | 46,482 | 23,820 | ||||||
| Receivables and other current assets | 2,629 | 2,829 | ||||||
| Non-Current Assets: | ||||||||
| Investment securities, available-for-sale, long-term | 43,536 | 56,304 | ||||||
| Property and equipment, net | 8,731 | 6,882 | ||||||
| Other assets, net | 346 | 330 | ||||||
| Total assets | $ | 163,514 | $ | 160,406 | ||||
| Current Liabilities: | ||||||||
| Accounts payable and other accrued liabilities | 7,143 | 7,186 | ||||||
| Other current liabilities | 261 | 259 | ||||||
| Long-Term Liabilities: | ||||||||
| Long-term debt | 14,829 | — | ||||||
| Other liabilities, net of current portion | 915 | 944 | ||||||
| Total Stockholders' Equity | 140,366 | 152,017 | ||||||
| Total liabilities and stockholders' equity | $ | 163,514 | $ | 160,406 | ||||
| BELLICUM PHARMACEUTICALS, INC. | ||||||||
| Condensed Statements of Operations | ||||||||
| (in thousands, except share and per share amounts) | ||||||||
|
Three Months Ended March 31, |
||||||||
| 2016 | 2015 | |||||||
| Grant Revenues | $ | 92 | $ | 107 | ||||
| Operating Expenses: | ||||||||
| Research and development | 10,988 | 5,718 | ||||||
| General and administrative | 4,284 | 2,197 | ||||||
| Total operating expenses | 15,272 | 7,915 | ||||||
| Operating loss | (15,180 | ) | (7,808 | ) | ||||
| Interest income, net | 105 | 50 | ||||||
| NET LOSS | $ | (15,075 | ) | $ | (7,758 | ) | ||
| Net loss attributable to common shareholders | $ | (15,075 | ) | $ | (7,758 | ) | ||
| Net loss per share attributable to common shareholders, basic and diluted | $ | (0.56 | ) | $ | (0.30 | ) | ||
| Weighted-average common shares outstanding, basic and diluted | 26,882,526 | 26,259,392 | ||||||
View source version on businesswire.com: http://www.businesswire.com/news/home/20160509006023/en/
Source:
Investors:
Bellicum Pharmaceuticals, Inc.
Alan Musso,
832-384-1116
CFO
amusso@bellicum.com
or
Media:
BMC
Communications
Brad Miles, 646-513-3125
bmiles@bmccommunications.com
