Bellicum Pharmaceuticals Provides Operational Update and Reports Financial Results for Fourth Quarter and Year Ended December 31, 2014
Management to host webcast and conference call today at
“In 2014 Bellicum achieved significant milestones, including private and
public financings, the expansion of our lead, global clinical program,
and important additions to our leadership team and board,” said
Added Mr. Farrell, “Looking forward, we expect to expand our BPX-501 program with additional Phase 1/2 clinical trials in different transplant settings, and disclose initial top-line data from ongoing studies by year-end. We also anticipate advancing our first TCR product candidate, BPX-701, into the clinic by the end of 2015.”
- Completed enrollment of the initial phase of a Phase 1/2 clinical trial of BPX-501 in the HSCT setting: The Phase 1 cohorts of this trial enrolled an equal number of patients with blood cancers and non-malignant genetic diseases such as Fanconi’s syndrome or severe combined immunodeficiency (SCID). This open label dose escalation study in pediatric patients is evaluating whether genetically engineered BPX-501 T cells from haplo-identical donors administered following a T-depleted HSCT are safe and can enhance immune reconstitution. BPX-501 contains the CaspaCIDe® safety switch, giving doctors the ability to eliminate the T cells should they cause Graft-versus-host disease.
- Advanced our CAR-T and TCR cell therapy programs: Bellicum continued to advance its pipeline of next-generation CAR-T and TCR controllable cell therapies with a TCR product candidate expected to enter a Phase 1/2 clinical trial by the end of 2015. BPX-701, a TCR-based therapy that incorporates the CaspaCIDe® safety switch, is initially being developed to treat PRAME-expressing sarcomas and neuroblastomas.
- Began enrolling the third patient cohort of the Phase 1 clinical trial of BPX-201: BPX-201 is a dendritic cell vaccine using DeCIDe® technology for patients with metastatic castrate-resistant prostate cancer. Bellicum is currently preparing for further development of BPX-201 in combination with a checkpoint inhibitor.
- Presentation at the 2014 ASH meeting: Bellicum presented a poster with information demonstrating preclinical proof of principle that inducible MyD88/CD40 allows rimiducid-mediated co-stimulation to control activation and proliferation of CAR-T cells.
Rimiducid recognized as first in new drug class: The World
Health Organization’s (WHO)
International Nonproprietary Name (INN) Expert Grouphas published the recommended nonproprietary name “rimiducid” for the small molecule drug previously known as AP1903, establishing it as the first of a new drug class assigned the unique suffix “-ducid.” Rimiducid is the lead small molecule activator for Bellicum’s platform of Chemical Induction of Dimerization (CID)-controllable molecular switches.
$250 millionsince August 2014: The Company successfully completed its IPO in December 2014, raising gross proceeds of $160.6 millionbefore underwriting discounts, commissions and expenses, and inclusive of the full exercise of the underwriters’ over-allotment option. In August 2014, the Company raised $55.0 millionin gross proceeds in a private placement of Series C convertible preferred stock, with an additional $39.1 millionraised upon exercise of warrants in conjunction with the IPO.
Strengthened board of directors, executive team: In late 2014,
Reid Huber, Ph.D., Executive Vice President and Chief Scientific Officer at Incyte Corporation, and Jon P. Stonehouse, President and CEO of BioCryst Pharmaceuticals, to its board of directors. The Company also made two senior management appointments. Alan Musso, most recently CFO and Treasurer at Targacept, joined Bellicum in the role of Chief Financial Officer and Treasurer, and Peter Hoang, a former investment banker and most recently head of new venture formation and development at the University of Texas M.D. Anderson Cancer Center, joined as Senior Vice President, Business Development and Strategy.
Restructured license agreement with
ARIAD Pharmaceuticals: In the fourth quarter of 2014, Bellicum paid ARIAD $50 millionin return for a fully paid-up license to its cell-signaling technology and a return by ARIAD of its equity stake of 677,463 shares of Bellicum common stock. The scope of the license and the field of use were also expanded, giving Bellicum a worldwide exclusive license to ARIAD’s cell-signaling technology for broad use in human cell therapies for all diseases on a royalty-free and milestone-free basis.
Anticipated 2015 Program Milestones:
- BPX-501: Expect to disclose initial top-line data from ongoing studies in HSCT setting by year-end.
- BPX-201: Expect to initiate Phase 1/2 trial of checkpoint inhibitor combination in metastatic castrate-resistant prostate cancer by end of 2015.
- BPX-701: Expect to initiate Phase 1/2 trial of CaspaCIDe® TCR product candidate for the treatment of PRAME-expressing sarcomas and neuroblastomas by year-end.
- BPX-401 and BPX-601, respectively: Continue preclinical work for advancing our next-generation CAR-T product candidate for hematological cancers expressing the CD19 antigen into human clinical trials in the first half of 2016, and Bellicum’s GoCAR-T product candidate in solid tumors expressing PSCA in the second half of 2016.
Fourth Quarter and Full Year 2014 Financial Results:
Cash Position and Guidance: Bellicum ended the year on
December 31, 2014with cash and cash equivalents totaling $191.6 million, compared to $11.2 millionat December 31, 2013. The increased cash balance reflects the net proceeds of approximately $244 millionreceived from its IPO, sales of preferred stock and exercise of warrants during 2014, offset primarily by $50 millionthat was paid to ARIAD to restructure Bellicum’s license agreement and net cash used for Company operations. Based on current operating plans, Bellicum expects to have approximately $140 millionin cash, cash equivalents and investments as of December 31, 2015, and that current cash resources will be sufficient to meet its operating requirements through at least the first half of 2017.
R&D Expenses: Research and development expenses in the
fourth quarter of 2014 were
$3.9 millionand $11.0 millionfor the year ended December 31, 2014, compared to $2.5 millionand $7.0 millionduring the comparable periods in 2013. The higher expenses in the 2014 periods were primarily due to increases in manufacturing and clinical trial expenses.
G&A Expenses: General and administrative expenses in the
fourth quarter of 2014 were
$2.2 millionand $5.4 millionfor the year ended December 31, 2014, compared to $0.8 millionand $2.8 millionduring the comparable periods in 2013. The increased G&A expenses were due to the growth of the organization and establishing infrastructure to facilitate the needs of a public company and meet its business objectives.
Net Loss: Bellicum reported a net loss of
$74.3 millionfor the fourth quarter of 2014 and $84.0 millionfor the year ended December 31, 2014, compared to a net loss of $2.5 millionand $8.0 millionfor the comparable periods in 2013. The higher net loss amounts for the 2014 periods were due primarily to a charge of $43.2 millionincurred in the fourth quarter of 2014 in conjunction with the ARIAD license restructure transaction and a non-cash accounting charge of $24.4 millionrecorded for the change in fair value of warrants that were exercised in conjunction with Bellicum’s December IPO, of which $23.2 millionwas a fourth quarter expense.
Shares Outstanding: At
December 31, 2014, Bellicum had 26,372,592 shares of common stock outstanding.
Conference Call and Webcast:
Bellicum management will host a webcast and conference call at
Bellicum is a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for various forms of cancer, including hematological cancers and solid tumors, as well as 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. The Company is developing next-generation product candidates in some of the most important areas of cellular immunotherapy, including hematopoietic stem cell transplantation, or HSCT, CAR T cell therapy, and dendritic cell vaccines.
*CaspaCIDe® and DeCIDe® are trademarks registered with the U.S. Patent and Trademark Office.
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,"
"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 of our clinical trials and of our research and development
activities and expenses relating to BPX-501, BPX-401, BPX-601, BPX-701
and BPX-201 and our expectations regarding our cash resources. Various
factors may cause differences between Bellicum’s expectations and actual
results as discussed in greater detail in Bellicum’s filings with the
|BELLICUM PHARMACEUTICALS, INC.|
|Unaudited Condensed Balance Sheets|
|Cash and cash equivalents||$||191,602||$||11,168|
|Receivables and other current assets||1,620||1,000|
|Property and equipment, net||2,427||2,290|
|Other assets, net||145||484|
|Accounts payable and other accrued liabilities||$||3,371||$||1,685|
|Other current liabilities||265||120|
|Current portion of line of credit||-||400|
|Other liabilities, net of current portion||522||563|
|Line of credit, net of current portion||-||400|
|Total Stockholder's equity (deficit)||191,636||(28,152||)|
|Total liabilities and stockholder's equity (deficit)||$||195,794||$||14,942|
|BELLICUM PHARMACEUTICALS, INC.|
|Unaudited Condensed Statements of Operations|
|(in thousands, except share and per share amounts)|
|Three Months Ended||Year Ended|
|December 31,||December 31,|
|Research and development||3,930||2,485||11,008||7,050|
|ARIAD license restructuring||43,212||-||43,212||-|
|General and administrative||2,263||816||5,398||2,813|
|Total operating expenses||49,405||3,301||59,618||9,863|
|Change in fair value of warrant liability||(23,174||)||-||(24,371||)||-|
|Interest expense, net||(1,734||)||(11||)||(1,756||)||(47||)|
|Preferred stock dividends||-||(399||)||(1,432||)||(1,093||)|
|Net loss attributable to common shareholders||$||(74,298||)||$||(2,892||)||$||(85,397||)||$||(9,062||)|
|Net loss per share attributable to commonshareholders - basic and diluted||$||(18.99||)||$||(1.68||)||$||(34.04||)||$||(5.25||)|
|Weighted average common shares outstanding, basic and diluted||3,912,352||1,725,992||2,508,960||1,725,992|