On March 13, 2018 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the fourth quarter and full year ended December 31, 2017 (Press release, Bellicum Pharmaceuticals, MAR 13, 2018, View Source [SID1234524726]).
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"In the past year, we made substantial progress toward our vision of delivering cures through controllable cell therapy," said Bellicum’s President & CEO Rick Fair. "We completed enrollment in our first registrational trial of BPX-501 and remain on track for our first filing for product approval in Europe in 2019. We moved three new projects featuring our industry-leading cellular control technology into clinical trials, including the first-ever GoCAR-T with our iMC activation switch. We also made substantial preclinical progress on our next generation ‘dual-switch’ platform containing both activation and safety switches in the same CAR-T cell, and initiated plans to move two dual-switch CAR-T projects into clinical trials in 2019."
2017 HIGHLIGHTS AND CURRENT UPDATES
Bellicum Submits Response to FDA Clinical Hold on BPX-501 Trials
Last week, the Company submitted a full response to the FDA clinical hold notification, including requested changes to study protocols to provide guidelines for comprehensive monitoring and management of neurologic adverse events associated with hematopoietic stem cell transplants. The Company expects the response to satisfy the conditions for removal of the clinical hold, which applies to BPX-501 clinical trials in the U.S.
Recruitment Complete in BPX-501 E.U. Registration Trial
The Company completed enrollment in the treatment arm of its BP-004 E.U. registration trial in pediatric patients undergoing haploidentical hematopoietic stem cell transplant (haplo-HSCT) and expects to report updated data from this trial at upcoming medical meetings. The Company remains on track to file European Marketing Authorization Applications for BPX-501 and rimiducid in 2019.
Positive BPX-501 Interim Results Reported in AML and Primary Immunodeficiencies
Earlier today, the Company announced interim survival results in pediatric patients with acute myeloid leukemia (AML) suggesting that the addition of BPX-501 T cells to a haplo-HSCT may improve the anti-leukemic effect of stem cell transplantation. The Company also reported interim data in pediatric patients with primary immunodeficiencies (PIDs) undergoing a curative haplo-HSCT with BPX-501 demonstrating favorable disease-free and overall survival rates at one year. These interim results have been submitted for presentation at an upcoming medical meeting.
Robust BPX-601 GoCAR-TCell Expansion Observed Following Rimiducid Administration
The Phase 1 study of BPX-601—the first product featuring the Company’s iMC activation switch—is enrolling patients with nonresectable pancreatic cancer who test positive for prostate stem cell antigen (PSCA). The first patient dosed with rimiducid—to activate iMC following infusion of BPX-601 cells—showed a robust expansion of circulating BPX-601 cells following a single dose of rimiducid, providing the first clinical proof of concept of iMC. The patient continues to be evaluated for safety and efficacy, and the clinical site is enrolling additional patients. Bellicum expects to report findings from the initial cohorts of pancreatic cancer patients at an upcoming medical meeting and to expand the trial to other PSCA-expressing cancers later this year.
Collaborator CD19 CAR-T Trial Initiated
The first patients have been treated in a Phase 1 pediatric ALL clinical trial of a CD19 CAR-T incorporating the CaspaCIDe safety switch, which is being conducted in collaboration with Ospedale Pediatrico Bambino Gesù (OPBG), a leading European pediatric research center and hospital. The trial is designed to assess the impact of CaspaCIDe in managing the acute toxicities of CAR-T therapy.
Completed Buildout of In-House Manufacturing and Vector Production Facility
The Company recently completed the buildout and initial launch of a 30,400 square foot state-of-the-art cell manufacturing and vector production facility at its headquarters in Houston, Texas. This facility is designed and constructed to satisfy both U.S. and European regulatory standards, and the Company expects the facility will meet U.S. clinical trial and early commercialization requirements.
Bellicum Continues to Strengthen its Management Team and Board of Directors
Since August 2017, the Company has added Gregory Naeve, Ph.D. (Chief Business Officer), William Grossman, M.D., Ph.D. (Chief Medical Officer), and several key leadership appointments to strengthen its clinical and quality functions. Additionally, Edmund P. Harrigan, M.D. was recently appointed to Bellicum’s Board of Directors, bringing 28 years of cross-functional pharmaceutical industry experience, most recently as Senior Vice President, Worldwide Safety and Regulatory at Pfizer.
ANTICIPATED 2018 MILESTONES
Report updated data from the BP-004 study of BPX-501
Initiate pivotal clinical trials of BPX-501 in adult AML and in either pediatric AML or PIDs, pending regulatory clearances
Report initial results from the BPX-601 clinical trial, and expand the trial to include additional PSCA-expressing cancers
Present initial findings from the BPX-701 clinical trial at upcoming medical meetings
Fourth Quarter and Full Year 2017 Financial Results
Cash Position and Guidance: Bellicum ended the year on December 31, 2017 with cash, restricted cash and investments totaling $106.5 million, compared to $113.4 million at December 31, 2016. In the fourth quarter of 2017, the Company paid off its Hercules Capital debt facility with a $35.0 million loan from Oxford Finance. The new loan provided approximately $2.1 million in additional liquidity, interest-only payments until February 1, 2020 and a lower interest rate. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements through the first quarter of 2019.
R&D Expenses: Research and development expenses were $14.3 million and $65.7 million for the fourth quarter and year ended December 31, 2017, respectively, compared to $15.1 million and $51.3 million during the comparable periods in 2016. The higher expenses in 2017 were primarily due to increased clinical trial costs, particularly for BPX-501, start-up costs related to Bellicum’s in-house manufacturing facility and contract manufacturers in Europe and increased personnel and consulting expenses. The higher R&D expenses in the fourth quarter of 2016 were attributable to costs associated with characterization studies of rimiducid.
G&A Expenses: General and administrative expenses were $5.1 million and $21.0 million for the fourth quarter and year ended December 31, 2017, respectively, compared to $4.2 million and $16.9 million during the comparable periods in 2016. The increased G&A expenses in 2017 were primarily due to Bellicum’s overall growth, including an increase in personnel-related costs, facility costs, and other administrative costs.
Net Loss: Bellicum reported a net loss of $21.9 million for the fourth quarter of 2017 and $91.8 million for the year ended December 31, 2017, compared to a net loss of $19.9 million and $69.2 million for the comparable periods in 2016. The results included non-cash, share-based compensation charges of $3.4 million and $13.6 million for the fourth quarter and year ended December 31, 2017, respectively, and $3.1 million and $12.3 million for the comparable periods in 2016.
Shares Outstanding:
At December 31, 2017, Bellicum had 33,285,177 shares of common stock outstanding.
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 877-407-3103 (domestic) and 201-493-6791 (international) at least 10 minutes prior to the start of the call. The event will be webcast live and can also be accessed in the Investors & Media section of bellicum.com. An archived version of the webcast will also be available for replay in the Investors & Media section of the Bellicum website for at least two weeks following the call.
About BPX-501
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 or other T-cell mediated complications occur. This enables physicians to more safely perform stem cell transplants by administering BPX-501 engineered T cells to speed immune reconstitution, provide control over viral infections and enhance Graft-versus-leukemic effect without unacceptable GvHD risk. The ongoing BP-004 clinical study of BPX-501 is being conducted at transplant centers in the U.S. and Europe.
About BPX-601
BPX-601 is a GoCAR-T product candidate containing Bellicum’s proprietary inducible MyD88/CD40, or iMC, activation switch, designed to treat solid tumors expressing prostate stem cell antigen, or PSCA. Preclinical data show enhanced T cell proliferation, persistence and in vivo anti-tumor activity compared to traditional CAR-T therapies. A Phase 1 clinical trial in patients with nonresectable pancreatic cancer is ongoing. In addition to pancreatic cancer, PSCA is expressed in several other solid tumor indications, including: gastric, esophageal, cholangiocarcinoma, glioblastoma, prostate and bladder cancers. The Company plans to expand the clinical development of BPX-601 to include additional PSCA expressing cancer types.
About BPX-701
BPX-701 is a high affinity T cell receptor product candidate designed with the CaspaCIDe safety switch. It is currently being tested in a Phase 1 study of patients with refractory or relapsed acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) who test positive for PRAME, or preferentially expressed antigen in melanoma. In preclinical studies, PRAME-specific clones showed high reactivity against a panel of PRAME positive tumor cell lines, metastatic melanoma, sarcomas and neuroblastoma tissues. In vitro study data showed that BPX-701 demonstrated strong affinity to panels of cancer cells presenting PRAME peptides and low affinity to non-tumor cells, as well as complete elimination of BPX-701 cells in response to rimiducid.