On November 7, 2017 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 third quarter ended September 30, 2017, and provided an update on recent developments (Press release, Bellicum Pharmaceuticals, NOV 7, 2017, View Source;p=RssLanding&cat=news&id=2315120 [SID1234521670]).
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"We made good progress advancing our pipeline in the third quarter. Enrollment in our clinical program for BPX-501 remains on track and we progressed our plans for future trials in adult AML and a pediatric orphan blood disorder," said Rick Fair, Bellicum’s President & Chief Executive Officer. "On BPX-601, we modified our Phase 1 trial to accelerate evaluation of our first clinical GoCAR-T candidate, and we look forward to reporting preliminary results next year. Finally, we continued to advance several exciting preclinical programs, leveraging our dual-switch controllable cell therapy platform."
PROGRAM HIGHLIGHTS AND CURRENT UPDATES
BPX-501
Adjunct T-cell therapy incorporating the CaspaCIDe safety switch, administered after a haploidentical hematopoietic stem cell transplant (haplo-HSCT), to improve outcomes and reduce mortality
Bellicum Continues to Advance its BPX-501 Program
Enrollment in the EU BP-004 clinical trial remains on track to be complete by the end of 2017. Bellicum has also initiated C-004, an observational trial in pediatric patients receiving transplants from matched unrelated donors (MUD) without BPX-501. The outcomes of both trials could form the basis for filings of European Marketing Authorization Applications for BPX-501 and rimiducid. A BPX-501 abstract, highlighting data on immune reconstitution from the EU BP-004 clinical trial, has been accepted for an oral presentation at the upcoming 59th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December.
Company Prepares for Additional BPX-501 Trials in U.S.
Planning is ongoing for two additional trials of BPX-501 to expand the eligible patient population and support potential U.S. registration. These trials are being developed in adult patients with acute myeloid leukemia (AML) and in a distinct orphan inherited blood disorder patient population.
BPX-601
Novel GoCAR-T product candidate designed with the proprietary iMC activation switch to improve efficacy
Phase 1 BPX-601 Clinical Trial Progressing
Enrollment and treatment is currently ongoing in the Company’s Phase 1 trial in nonresectable pancreatic cancer patients who test positive for prostate stem cell antigen (PSCA). Based on data from the initial three treated patients, the Company—with support from the principal investigator and the FDA—amended the study protocol to allow activation of the iMC switch seven days following the administration of BPX-601 versus the previous 30-day schedule. This will enable an earlier evaluation of the first clinical experience with GoCAR-T, the Company’s platform to enhance and control CAR-T cell activation, proliferation, and survival.
BPX-701
High affinity T-cell receptor (TCR) product candidate designed with the CaspaCIDe safety switch
Phase 1 BPX-701 Clinical Trial Progressing
Enrollment and treatment is currently ongoing in the Company’s Phase 1 clinical trial in patients with refractory or relapsed AML and myelodysplastic syndromes (MDS) who test positive for preferentially-expressed antigen in melanoma (PRAME).
CD19 Program
Bellicum is working with academic collaborators to evaluate the benefit of CaspaCIDe in managing serious toxicities associated with CD19 CAR-T cells
In collaboration with Ospedale Pediatrico Bambino Gesù (OPBG), a leading European pediatric research center and hospital, a Phase 1 clinical trial of a CaspaCIDe-enabled CD19 CAR-T is expected to begin in the fourth quarter.
Preclinical Programs
At ASH (Free ASH Whitepaper) 2017, the Company will present preclinical results of its controllable CAR-T technology in three poster presentations, including a study evaluating Bellicum’s dual-switch CAR-T cells targeting CD123. The data will further support the potential of the Company’s technology platform to control cells in vivo and improve the benefit / risk profile of adoptive cell therapies.
Third Quarter and Nine Months Ended September 30, 2017 Financial Results
Bellicum reported a net loss of $23.4 million for the third quarter of 2017 and $69.9 million for the nine months ended September 30, 2017, compared to a net loss of $17.7 million and $49.3 million for the comparable periods in 2016. The results included non-cash, stock-based compensation charges of $3.7 million and $10.2 million for the third quarter and nine months ended September 30, 2017, and $3.1 million and $9.2 million for the comparable periods in 2016.
As of September 30, 2017, cash and investments totaled $118.6 million. Bellicum expects that it will end 2017 with approximately $90 to $95 million in cash, cash equivalents and investments, and continues to expect that current cash resources will be sufficient to meet operating requirements through 2018.
Research and development expenses were $18.1 million and $51.4 million for the three and nine months ended September 30, 2017, respectively, compared to $13.3 million and $36.2 million during the comparable periods in 2016. The higher expenses in the 2017 periods were primarily due to an increase in clinical development and manufacturing costs due to increased enrollment in clinical trials, principally BP-004, and increased personnel expenses, overhead charges and manufacturing facility start-up costs.
General and administrative expenses were $4.6 million and $16.0 million for the three and nine months ended September 30, 2017, respectively, compared to $4.3 million and $12.7 million during the comparable periods in 2016. The higher expenses in the 2017 periods were primarily due to the Company’s overall growth, including an increase in personnel related costs, principally due to hiring additional employees and severance costs, higher facility costs and increased legal, accounting and travel expenses.
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 occur. This could enable physicians to more safely perform allogeneic stem cell transplants by administering BPX-501 engineered T cells to speed immune reconstitution, provide control over viral infections and enhance Graft-versus-leukemia 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.