Bellicum Pharmaceuticals Reports Third Quarter 2015 Financial Results and Recent Program Updates
Pediatric patients with beta thalassemia, SCID, and Wiskott-Aldrich Syndrome are disease-free following haplo transplant with BPX-501 T cells
Additional details of ongoing BPX-501 trial and adoptive T cell programs to be presented at ASH 2015
“During the third quarter we continued to make good progress in the
advancement of our stem cell transplant, CAR-T and TCR programs,” said
Added Mr. Farrell, “We are also looking forward to multiple presentations at ASH highlighting our CAR-T and TCR adoptive cell therapy programs and the power of our cellular control technologies. We continue to anticipate that three new product candidates, BPX-601, BPX-701, and BPX-401 will be initiating clinical development in the first half of 2016.”
BPX-501 at ASH 2015: Company to present interim data from the
BP-004 ongoing Phase 1/2 clinical trial. Bellicum will present
initial data in malignant and non-malignant blood diseases at the
upcoming 26th Annual Meeting of the
American Society of Hematologyin early December in Orlando, Florida. The open label dose escalation trial in pediatric patients is evaluating whether BPX-501 T cells from a haploidentical donor, administered following a T-depleted hematopoietic stem cell transplant (HSCT), are safe and can enhance immune reconstitution.
Enrollment in the BP-004 trial continues at a strong pace at sites in
Europeand in the U.S., with 53 pediatric patients enrolled as of October 31st, including one patient with sickle cell disease.
- Among non-malignant patients in the trial treated to date are four children with beta thalassemia major (in its most severe form, the β0/β0 type) who have successfully undergone the HSCT transplant procedure with the add-back of BPX-501 gene-modified T cells. Within two weeks of the transplant procedure all patients became blood transfusion-independent. All four patients are alive, disease-free and remain transfusion-independent.
At a medical symposium in Parma,
Italyin early September, principal investigator Dr. Franco Locatellishared initial outcomes from this ongoing trial. His presentation included the first 15 children enrolled in the clinical study with non-malignant inherited disorders (four with severe combined immunodeficiency, or SCID; three with Wiskott-Aldrich Syndrome; four with Fanconi anemia; three with beta thalassemia, and one with hemophagocytic lymphohistiocytosis) who received the BPX-501 T cell add-back following HLA-partially matched family donor HSCT. In all cases, the BPX-501 T cells engrafted and expanded with no secondary graft failures. Grade II skin-only graft versus host disease (GvHD) was observed in one patient, and promptly resolved with topical steroids. None of the patients so far have developed chronic GvHD, and all are alive and disease-free.
- BPX-601 preclinical data to be presented at ASH 2015. Bellicum continues to advance its first GoCAR-T™ product candidate, containing our proprietary iMC (inducible MyD88/CD40) activation switch, designed to treat solid tumors expressing prostate stem cell antigen (PSCA). The Company expects to file an IND for the initial indication of pancreatic cancer by the end of 2015. In addition to pancreatic cancer, PSCA is also expressed in prostate, ovarian, bladder, esophageal and gastric cancers. BPX-601 is differentiated from traditional CAR-T therapies with an MC co-stimulatory domain that is activated by administration of rimiducid.
- BPX-401 CIDeCAR™ preclinical data to be highlighted in an oral presentation at ASH 2015. BPX-401, a CIDeCAR™ product candidate incorporating Bellicum’s proprietary MC co-stimulatory domain and the CaspaCIDe® safety switch, is designed to target blood cancers expressing CD19. BPX-401 is expected to enter the clinic in the first half of 2016.
- BPX-701 progressing toward the clinic. Bellicum continues to advance its proprietary T cell receptor (TCR) product candidate designed to target solid tumors expressing the preferentially-expressed antigen in melanoma, or PRAME. The Company has identified clinical sites for its BPX-701 CaspaCIDe®-enabled TCR product candidate and expects to file an IND by the end of 2015, initially for the indications of PRAME-expressing sarcomas and uveal melanoma.
Third Quarter and Nine Months Ended
Bellicum reported a net loss of
Grant revenues were
Research and development expenses were
General and administrative expenses were
Bellicum is a clinical stage biopharmaceutical company focused on discovering and developing cellular immunotherapies for cancers and 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, and CAR-T and TCR cell therapies. 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 and success of our clinical trials, including the rate and
progress of enrollment in such trials; the timing of an IND filing for
BPX-601 and BPX-701; our research and development activities and
expenses relating to BPX-501, BPX-401, BPX-601, BPX-701, CaspaCIDe, and
GoCAR-T; the effectiveness of BPX-501, its possible range of application
and its potential curative effects and safety in the treatment of blood
cancers and inherited blood diseases; and the potential applications and
effectiveness of our product candidates, 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.|
|Unaudited Condensed Balance Sheets|
|September 30,||December 31,|
|Cash and cash equivalents||$92,487||$191,602|
|Investment securities, available-for-sale - short-term||20,744||-|
|Receivables and other current assets||2,090||1,620|
|Investment securities, available-for-sale, long-term||50,018||-|
|Property and equipment, net||6,323||2,427|
|Other assets, net||446||145|
|Accounts payable and other accrued liabilities||$4,849||$3,372|
|Other current liabilities||170||264|
|Other liabilities, net of current portion||984||522|
|Total Stockholders' Equity||166,105||191,636|
|Total liabilities and stockholders' equity||$172,108||$195,794|
|BELLICUM PHARMACEUTICALS, INC.|
|Unaudited Condensed Statements of Operations|
|(in thousands, except share and per share amounts)|
|Three Months Ended||Nine Months Ended|
|September 30,||September 30,|
|Research and development||9,792||2,257||23,522||7,881|
|General and administrative||3,882||1,300||8,856||2,332|
|Total operating expenses||13,674||3,557||32,378||10,213|
|Interest income (expense), net||209||(1,199)||430||(1,220)|
|Preferred stock dividends||-||(328)||-||(1,432)|
|Net loss attributable to common shareholders||$(13,408)||$(4,424)||$(31,700)||$(11,099)|
|Net loss per share attributable to common|
|shareholders, basic and diluted||$(0.51)||$(2.08)||$(1.21)||$(5.45)|
|Weighted average common shares outstanding, basic and diluted||26,376,456||2,124,247||26,301,914||2,036,691|
|Other comprehensive loss:|
|Unrealized loss on investment securities||-||-||(204)||-|