Bellicum Pharmaceuticals Provides Operational Update and Reports Financial Results for the Second Quarter Ended June 30, 2015
Conference call and webcast to be held
“Over the last six months, we made substantial progress across all of
our controlled immunotherapy programs,” said
Continued Mr. Farrell, “We also advanced our pipeline of CAR-T and TCR products and expect to file two INDs by the end of 2015, for BPX-701, our TCR product candidate for solid tumors, and BPX-601, our CAR-T product candidate for solid tumors overexpressing prostate stem cell antigen, or PSCA. We believe the broad utility of our proprietary cellular control technologies and their potential to provide greater levels of efficacy and safety will become increasingly important as the field evolves toward the treatment of solid tumors and additional antigen targets. We also continued to strengthen our leadership team and have identified and leased space for the build-out of facilities to enable in-house cell therapy manufacturing to supply clinical trials.”
BPX-501 on track for year-end read-out of initial data: The
Company continued to enroll its Phase 1/2 clinical trials with BPX-501
in the allogeneic hematopoietic stem cell transplant (HSCT) setting.
In the BP-004 trial, additional leading U.S. and European pediatric
transplant centers have been initiated and the protocol has been
amended to increase the study size to allow up to 90 patients. The
open label dose escalation trial is evaluating whether BPX-501 T cells
from a haploidentical donor, typically the child’s mother or father,
administered following a T-depleted HSCT, are safe and can enhance
immune reconstitution. To date, about half of the patients randomized
into the trial have non-malignant inherited diseases, such as severe
combined immunodeficiency (SCID), Wiskott-Aldrich Syndrome and beta
thalassemia, all chronic life-long disorders for which HSCT is
curative. The BPX-501 T cells contain the CaspaCIDe®
safety switch for use in the event of GvHD, allowing the add-back of T
cells in order to promote faster immune recovery and anti-viral
activity after a T-depleted transplant procedure. The Company expects
to report initial top-line data from ongoing clinical trials in the
HCST setting in
July 2015, the intellectual property for BPX-501 was strengthened with a U.S. method of use patent issued to Baylor College of Medicine. The patent, licensed exclusively to Bellicum, is scheduled to expire in 2031.
DOTTI trial data published in BLOOD highlight safety, effectiveness
of CaspaCIDe®-enabled T cell add-back: Clinical
results from an investigator-initiated 12-patient trial demonstrated
that infusing haploidentical donor T cells containing the CaspaCIDe®
safety switch, following a T-depleted haplo-HSCT, led to improved
immune reconstitution and infection control. The trial data also
showed that when GvHD occurs, it can be rapidly controlled and
eliminated by removing alloreactive cells in vivo, and that the
productive, anti-viral and anti-cancer cells remain, repopulate and
maintain immunity. The trial results were simultaneously published in
BLOOD and featured as a late-breaking oral presentation at the
American Society of Gene and Cell Therapy(ASGCT) annual meeting in May 2015.
- BPX-601 is progressing ahead of schedule: The Company’s first GoCAR-T™ product candidate, containing the proprietary inducible MC (MyD88/CD40) activation switch, designed to treat solid tumors overexpressing prostate stem cell antigen, or PSCA, is progressing ahead of schedule and we now expect to file an IND for the initial indication of pancreatic cancer by the end of 2015. Previous guidance was for initiation of clinical trials in the second half of 2016. In addition to pancreatic cancer, PSCA is also expressed in prostate, ovarian, bladder, esophageal and gastric cancers.
BPX-701 moves toward clinic: Bellicum continued to advance
development of its next-generation, proprietary T cell receptor (TCR)
product candidate designed to target solid tumors expressing the
preferentially-expressed antigen in melanoma, or PRAME. The Company
licensed the technology to develop, manufacture and commercialize
high-affinity TCR product candidates against PRAME and an additional
Leiden University Medical Centerin April. Bellicum has identified clinical sites for its BPX-701 CaspaCIDe®-enabled TCR product candidate and expects to file an IND by the end of 2015. The Company is planning to initially develop this product candidate for the indications of PRAME-expressing sarcomas and uveal melanoma.
- BPX-401 progressing on track: BPX-401, a CIDeCAR™ product candidate constructed with Bellicum’s proprietary MC co-stimulatory domain and the CaspaCIDe® safety switch, is designed to target blood cancers expressing CD19, including potential indications of acute lymphocytic leukemia, chronic lymphocytic leukemia, and certain non-Hodgkin’s lymphomas. BPX-401 is expected to enter the clinic in the first half of 2016.
American Society of Clinical OncologyAnnual Meeting ( ASCO) data highlights potent anti-tumor effects of MC co-stimulatory domain: Bellicum’s poster presentation at ASCOhighlighted results from two preclinical studies that evaluated the in vivo tumor-killing abilities of its CIDeCAR cells. The studies showed that MC co-stimulation increased T cell proliferation and enhanced efficacy in lymphoma and solid tumor models in vivo compared to CAR T cells that included the more commonly utilized co-stimulatory molecule CD28. Bellicum’s CD19-targeted CIDeCAR (BPX-401) elicited dose-dependent elevation of cytokines, analogous to cytokine release syndrome, but cytokine levels were rapidly normalized upon administration of rimiducid, safely and without loss of tumor control.
- BPX-201 program to discontinue after current study concludes: Based on the prioritization of our other pipeline opportunities, the Company no longer intends to progress its BPX-201 vaccine into additional studies after the conclusion of the ongoing Phase 1 trial.
- Expansion of facilities to bring manufacturing of U.S. clinical supply in-house: Bellicum leased an additional 27,000 square feet at its corporate headquarters for the manufacture of BPX-501 for clinical studies and to support the development of its expanding pipeline of TCR and CAR-T adoptive cell therapy product candidates.
Strengthened board of directors and management team: In
July 2015, Bellicum added Stephen R. Davis, currently the interim Chief Executive Officer of ACADIA Pharmaceuticals, to its Board of Directors. The Company has also recruited three experienced executives to its management team: Scott Cullisonjoined as Vice President, Commercial Planning and Program Management, Anne Freseas Vice President, Human Resources, and Steve Toleras Vice President, Pharmaceutical Development.
Second Quarter and Six Months Ended
Bellicum reported a net loss of
Grant revenues were
Research and development expenses in the second quarter of 2015 were
General and administrative expenses in the second quarter of 2015 were
Conference Call and Webcast:
Bellicum 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 (855) 779-9069 (U.S. domestic) and (631) 485-4863 (international) at least 10 minutes prior to the start of the call, using Conference ID number 92664473. 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 also be available for replay in the Investor and Media section of our website for at least two weeks following the call.
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. Bellicum 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. 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
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 of our clinical trials, including the rate and progress of
enrollment in such trials, our research and development activities and
expenses relating to BPX-501, BPX-401, BPX-601, BPX-701, BPX-201,
CaspaCIDe, DeCIDe and GoCAR-T and the potential applications and
effectiveness of our product candidates, including as compared to other
treatment options. 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.|
|Unaudited Condensed Balance Sheets|
|June 30,||December 31,|
|Cash and cash equivalents||$||101,646||$||191,602|
|Investment securities, available-for-sale||17,649||-|
|Receivables and other current assets||2,644||1,620|
|Investment securities, available-for-sale, long-term||53,270||-|
|Property and equipment, net||4,798||2,427|
|Other assets, net||230||145|
|Accounts payable and other accrued liabilities||$||2,568||$||3,372|
|Other current liabilities||406||264|
|Other liabilities, net of current portion||254||522|
|Total Stockholders' Equity||177,009||191,636|
|Total liabilities and stockholders' equity||$||180,237||$||195,794|
|BELLICUM PHARMACEUTICALS, INC.|
|Unaudited Condensed Statements of Operations|
|(in thousands, except share and per share amounts)|
|Three Months Ended||Six Months Ended|
|June 30,||June 30,|
|Research and development||8,012||3,235||13,730||5,624|
|General and administrative||2,777||592||4,974||1,032|
|Total operating expenses||10,789||3,827||18,704||6,656|
|Interest income (expense), net||171||(8||)||221||(21||)|
|Preferred stock dividends||-||(564||)||-||(1,104||)|
|Net loss attributable to common shareholders||$||(10,534||)||$||(3,845||)||$||(18,292||)||$||(6,675||)|
Net loss per share attributable to common shareholders, basic and diluted
|Weighted average common shares outstanding, basic and diluted||26,268,610||2,119,518||26,264,025||1,992,142|
|Other comprehensive loss:|
|Unrealized loss on investment securities||(204||)||-||(204||)||-|