On August 8, 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 second quarter ended June 30, 2017, and provided an update on recent developments (Press release, Bellicum Pharmaceuticals, AUG 8, 2017, View Source;p=RssLanding&cat=news&id=2292700 [SID1234520145]).

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"Since I joined the Company six months ago, we have conducted a thorough review of our strategy and operations, and are very optimistic about the opportunities before us," said Rick Fair, Bellicum’s President & Chief Executive Officer. "We continue to be encouraged by the results from our ongoing BPX-501 pediatric studies and our progress toward a filing in Europe. We have adjusted our plans for U.S. registrational trials to enable an efficient path to seeking approvals for the greatest areas of unmet need. Lastly, we continue to be excited about the clinical progress of our CAR T and TCR product candidates, and the application of our molecular switch platform for future pipeline expansion."

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

Data Update Suggests BPX-501 Improves Outcomes of Haploidentical Stem Cell Transplants
During the Presidential Symposium of the 22nd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in June, Bellicum reported data from 98 pediatric patients within the BP-004 trial which showed rapid immune recovery, a low incidence of transplant-related mortality, a reduction in viral infections and a low rate of Graft versus Host Disease (GvHD) that was manageable with either standard treatments or rimiducid. The data suggest BPX-501 could improve outcomes of haploidentical stem cell transplants, providing an option for the many patients who could benefit from a life-saving transplant but lack a matched donor.

Positive Clinical Results of BPX-501 in Pediatric Leukemias
Also at EHA (Free EHA Whitepaper), Bellicum reported data from the BP-004 trial in a cohort of 47 pediatric patients with acute leukemias who lack a matched donor. The data showed rapid immune reconstitution and low rates of relapse and mortality, suggesting that BPX-501 may offer benefits in combination with HSCT in acute leukemia patients.

European BP-004 Pivotal Clinical Trial Progressing
Enrollment in the pivotal EU BP-004 trial remains on track for completion by the end of 2017. Bellicum expects to initiate an observational trial in pediatric patients receiving transplants from matched unrelated donors (MUD) without BPX-501 in the third quarter. Outcomes from these trials are expected to be the basis for filings of European Marketing Authorization Applications for BPX-501 and rimiducid. The Company expects to report top-line results of these studies in the second half of 2018, with MAA filings planned for 2019.

Company Clarifies U.S. Clinical Development Strategy
Bellicum is finalizing plans for the design of registrational trials of BPX-501 in the U.S. The Company’s current plans include conducting a controlled clinical trial in adult patients with acute myeloid leukemia (AML), which it expects to fund in part through its $16.9 million Product Development Award from the Cancer Prevention and Research Institute of Texas ("CPRIT"). In the pediatric non-malignant setting, Bellicum is designing a registrational trial to evaluate BPX-501 in a distinct subset of orphan inherited blood disorders.
BPX-601

Phase 1 BPX-601 Clinical Trial Continues
BPX-601 is Bellicum’s novel GoCAR-T product candidate, which is designed with its proprietary iMC activation switch to allow control over the level of stimulation and proliferation of the modified T cells. Enrollment and treatment is ongoing in Bellicum’s Phase 1 trial in patients with nonresectable pancreatic cancer who test positive for prostate stem cell antigen (PSCA).
BPX-701

Phase 1 BPX-701 Clinical Trial Continues
BPX-701 is a high affinity TCR product candidate designed with the CaspaCIDe safety switch, enabling the elimination or reduction of the engineered cells in the event of severe toxicities. Dosing has been initiated 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).
CORPORATE UPDATE

Addition of Chief Business Officer to Expand Partnership Opportunities
Greg Naeve, Ph.D., an accomplished product strategy and business development executive, is joining Bellicum’s leadership team in August 2017 from Pfizer, where he led efforts to identify and implement multiple strategic partnerships and translational science collaborations across Pfizer Worldwide R&D, including CAR T alliances with Cellectis and Servier.
PRECLINICAL RESEARCH

In April, Bellicum reported positive preclinical data at AACR (Free AACR Whitepaper) on its novel dual-switch technology incorporated into CAR T and TCR constructs, an approach offering the possibility of both activating cells to enhance efficacy and eliminating them to manage toxicity. Bellicum is working to incorporate its dual-switch technology into future CAR T and TCR product candidates.

The Company continues to work with academic collaborators to explore the applicability of CaspaCIDe in CD19 CARs, the first of which is expected to enter the clinic in the second half of this year in patients with B-cell malignancies.
SECOND QUARTER AND SIX MONTHS ENDED JUNE 30, 2017 FINANCIAL RESULTS

Bellicum reported a net loss of $24.5 million for the second quarter of 2017 and $46.4 million for the six months ended June 30, 2017, compared to a net loss of $16.5 million and $31.6 million for the comparable periods of 2016. The results included non-cash, share-based compensation charges of $3.2 million and $6.6 million for the second quarter and six months ended June 30, 2017 and $3.1 million and $6.2 million for the comparable periods in 2016.

As of June 30, 2017, cash, restricted cash and investments totaled $139.0 million. Based on current operating plans, Bellicum continues to expect to end 2017 with approximately $85 to $95 million in cash and investments, and that current cash resources will be sufficient to meet operating requirements through 2018.

Research and development expenses were $18.0 million and $33.3 million, for the three and six months ended June 30, 2017, respectively, compared to $12.0 million and $22.9 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 trials, principally BPX-501, and increased personnel expenses, overhead charges and manufacturing facility start-up costs.

General and administrative expenses were $5.5 million and $11.4 million for the three and six months ended June 30, 2017, respectively, compared to $4.2 million and $8.5 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.

At June 30, 2017, Bellicum had 33,193,229 shares of common stock outstanding.