Bellicum Pharmaceuticals Provides Operational Update and Reports Financial Results for the Fourth Quarter and Year Ended December 31, 2016
On track to complete enrollment in E.U. registration trials for BPX-501 and rimiducid this year
Reported updated clinical data with BPX-501 demonstrating rapid immune recovery and low rates of GvHD in children with blood cancers and orphan inherited blood disorders
Initiated clinical trials of controllable CAR T and TCR product candidates
Management to host conference call and webcast today at 5 p.m. Eastern
“Bellicum had a productive 2016 marked by significant clinical and regulatory progress with our diverse pipeline of controllable cell therapies,” said
2016 PROGRAM HIGHLIGHTS AND CURRENT UPDATES
Adjunct T-cell therapy, administered after allogeneic hematopoietic stem cell transplantation (HSCT) to support faster immune recovery, improved infection control, and reduced mortality and GvHD, being evaluated in blood cancers and orphan inherited blood disorders
- The Company advanced discussions with the U.S.
FDAon BPX-501’s path for product registration in the U.S. Bellicum expects to conduct separate clinical trials for nonmalignant and malignant pediatric patients in the haploidentical stem cell transplant setting, including a non-randomized clinical trial in patients with orphan inherited blood disorders and a controlled trial in patients with blood cancers. Further details remain under discussion, and the Company expects to finalize discussions with the FDAon both protocols in the second quarter of 2017.
- In the fourth quarter, Bellicum completed protocol assistance with the
European Medicines Agency(EMA), and continues to enroll patients in the BP-004 clinical trial to support E.U. product registration of BPX-501 and rimiducid. The Company will also conduct a comparator trial to evaluate outcomes of pediatric patients with malignant and nonmalignant diseases receiving a matched unrelated donor (MUD) HSCT. The primary endpoint is event-free survival at six months (with events defined as transplant-related or non-relapse mortality, severe GvHD, and serious infection). The Company expects both trials to be fully enrolled later this year and to submit Marketing Authorization Applications (MAAs) to the European Medicines Agency(EMA) by mid-2018.
- Bellicum reported updated clinical data from the BP-004 clinical trial that are supportive of E.U. regulatory submission. Data presented at the BMT (Bone Marrow Transplant) Tandem Meetings last month showed that cumulative incidence of treatment-related mortality remained very low, with six-month and one-year survival rates of 98.4 percent and 97.2 percent, respectively. There were no serious adverse events associated with use of BPX-501 or rimiducid. For the subset of 73 patients with six months of follow-up, the patients had rapid immune reconstitution, including full recovery and normalization of T cells, B cells and immunoglobulins.
- Bellicum was awarded
$16.9 millionto help fund a global clinical program with BPX-501 in adults with high- and intermediate-risk AML (acute myeloid leukemia) from the Cancer Prevention and Research Institute of Texas(CPRIT). The Company expects to initiate a clinical trial later this year that evaluates outcomes of patients who receive a haploidentical HSCT with the post-transplantation cyclophosphamide (PT/Cy) regimen with or without BPX-501.
- BPX-501 and rimiducid received Orphan Drug Designations in the E.U. and U.S.
Novel GoCAR-T product candidate designed with the proprietary iMC activation switch to improve efficacy
- Bellicum has begun patient dosing in an initial Phase 1 clinical trial with BPX-601 in patients with nonresectable pancreatic cancer who test positive for prostate stem cell antigen (PSCA). BPX-601 is believed to be the first CAR T-cell product candidate to enter the clinic that is designed to enable control over the expansion and stimulation of the cells.
High affinity T-cell receptor (TCR) product candidate designed with the CaspaCIDe® switch to improve safety
- The Company initiated a Phase 1 clinical trial with BPX-701 in the U.S., initially in HLA-A2 positive patients with refractory or relapsed acute myeloid lymphoma (AML) and myelodysplastic syndromes (MDS) who test positive for preferentially-expressed antigen in melanoma (PRAME).
- Bellicum reported preclinical results with its GoCAR-T and GoTCR technologies at ASH 2016, demonstrating unique control of cell proliferation and persistence, which may improve outcomes in solid tumors.
- The Company continues to advance a novel dual-switch technology, with both activation and safety switches, designed to manage the persistence and safety of tumor antigen-specific CAR T cells, with new preclinical data planned to be reported at the
American Association of Clinical Research(AACR) annual meeting in April.
2016 AND RECENT CORPORATE UPDATES
- Bellicum welcomed new Chief Executive Officer
Rick Fairin preparation for the next phase of the Company’s growth. Mr. Fair joined Bellicum from Genentech/ Roche, where he most recently served as Senior Vice President, Head of Oncology Global Product Strategy, and was responsible for the successful development and commercialization of multiple novel cancer therapies.
- Bellicum expanded its research collaboration with Ospedale Pediatrico Bambino Gesù (OPBG) with its first partnered product candidate, an OPBG-designed, CaspaCIDe-enabled CD19 CAR, expected to enter the clinic in the second half of 2017 in patients with B-cell malignancies.
- Bellicum and Adaptimmune entered a staged collaboration to develop next-generation T-cell therapies. The companies will evaluate the potential of Bellicum's GoTCR technology and Adaptimmune's affinity-optimized SPEAR® T cells to create enhanced TCR product candidates, with the option of progressing to a two-target co-development and co-commercialization phase.
- The Company expanded its research collaboration with Leiden University Medical Center for discovery of natural high-affinity TCRs for several cancers. Bellicum will provide financial support to LUMC over a three-year term in exchange for the right to exclusively license any high-affinity TCRs discovered under the new agreement.
- Bellicum made substantial progress toward completion of its U.S. cGMP cellular therapy and viral vector manufacturing facility in
Houston, and has recently begun manufacturing BPX-501 clinical trial drug for U.S. centers. Bellicum expects to complete the remaining work in mid-2017.
ANTICIPATED 2017 MILESTONES
Bellicum expects to:
FDAdiscussions on the U.S. regulatory path for BPX-501 and rimiducid in the second quarter, and begin enrollment of U.S. registration trials during the second half of 2017
- Complete enrollment into the European BP-004 and MUD comparator clinical trials this year, and prepare for the anticipated MAA filings for BPX-501 and rimiducid to the EMA by mid-2018
- Update BP-004 clinical data at medical conferences in 2017, including the annual meetings of the
European Hematology Association(EHA) and the American Society of Hematology(ASH)
- Initiate the CPRIT-supported clinical trial of BPX-501 in adults with AML by year-end
- Present preclinical data on Bellicum’s novel dual-switch technology at AACR in April
In addition, as part of its Bellicum partnership, OPBG is expected to initiate a Phase 1 clinical trial with a CaspaCIDe-enabled CD19 CAR in the second half of 2017 and report initial data in 2018.
Fourth Quarter and Full Year 2016 Financial Results
Cash Position and Guidance: Bellicum ended the year on
R&D Expenses: Research and development expenses were
License fees were
G&A Expenses: General and administrative expenses were
Net Loss: Bellicum reported a net loss of
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 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.
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 adding back BPX-501 engineered T cells to speed immune reconstitution and provide control over viral infections, without unacceptable risk of uncontrollable GvHD. The ongoing BP-004 Phase 1/2 clinical trial of BPX-501 is being conducted at transplant centers in the U.S. and
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, 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 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 presentation of our data at scientific meetings; the timing of regulatory filings for BPX-501 and rimiducid; our research and development activities relating to our GoCAR-T and GoTCR technologies; the timing and success of our collaborations, including with OPBG, Leiden and Adaptimmune; our expectations regarding our cash position and receipt of the CPRIT grant funds; and the success of our manufacturing expansion. 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|
|December 31,||December 31,|
|Cash and cash equivalents||$||33,140||$||70,241|
|Investment securities, available-for-sale - short-term||70,632||23,820|
|Receivables and other current assets||1,838||2,829|
|Investment securities, available-for-sale, long-term||-||56,304|
|Property and equipment, net||16,504||6,882|
|Other assets, net||283||330|
|Accounts payable and other accrued liabilities||$||12,986||$||7,186|
|Current maturity of long term debt||1,787||-|
|Other current liabilities||340||259|
|Other liabilities, net of current portion||20,350||944|
|Total Stockholders' Equity||96,574||152,017|
|Total liabilities and stockholders' equity||$||132,037||$||160,406|
|BELLICUM PHARMACEUTICALS, INC.|
|Unaudited Condensed Statements of Operations|
|(in thousands, except share and per share amounts)|
|Three Months Ended||Year Ended|
|December 31,||December 31,|
|Research and development||15,084||10,223||51,263||33,561|
|General and administrative||4,210||3,816||16,925||12,672|
|Total operating expenses||19,594||17,039||68,768||49,417|
|Interest and other income (expense), net||(425||)||157||(861||)||587|
|Net loss attributable to common shareholders||$||(19,938||)||$||(16,848||)||$||(69,241||)||$||(48,548||)|
|Net loss per share attributable to common|
|shareholders, basic and diluted||$||(0.74||)||$||(0.63||)||$||(2.57||)||$||(1.84||)|
|Weighted average common shares outstanding, basic and diluted||27,043,002||26,770,194||26,950,906||26,346,603|