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Company updates BPX-501 program
Enrollment progressing in early clinical trials of CAR T and TCR product candidates
Company to host conference call and webcast on
“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
PROGRAM HIGHLIGHTS AND CURRENT UPDATES
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
Congressof the European Hematology Association(EHA) 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, 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 millionProduct 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.
- 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).
- 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).
- 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 2017from 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 Cellectisand Servier.
- In April, Bellicum reported positive preclinical data at AACR 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
Bellicum reported a net loss of
Research and development expenses were
General and administrative expenses were
Conference Call and Webcast
Bellicum management will host a webcast and conference call at
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 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.
About Bellicum Pharmaceuticals
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.
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, BPX-701, BPX-601, 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 our BP-004 study and 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 timing of regulatory filings for BPX-501 and rimiducid; our research and development activities relating to our GoCAR-T and GoTCR technologies, and our collaborations with academic institutions and other companies. 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 Securities and Exchange Commission, including without limitation our annual report on Form 10-K for the year ended December 31, 2016 and our report on Form 10-Q for the quarter ended
|BELLICUM PHARMACEUTICALS, INC.|
|Unaudited Condensed Consolidated Balance Sheets|
|30, 2017||31, 2016|
|Cash and cash equivalents||$||75,148||$||33,140|
|Investment securities, available-for-sale, short-term||55,081||70,632|
|Prepaid expenses and other current assets||2,896||1,838|
|Investment securities, available-for-sale, long-term||4,405||—|
|Property and equipment, net||25,458||16,504|
|Accounts payable and other accrued liabilities||12,267||12,986|
|Current maturities of long-term debt||3,412||1,787|
|Other current liabilities||283||340|
|Other liabilities, net of current portion||1,939||1,914|
|Total Stockholders' Equity||122,707||96,574|
|Total liabilities and stockholders' equity||$||167,724||$||132,037|
|BELLICUM PHARMACEUTICALS, INC.|
|Unaudited Condensed Consolidated Statements of Operations|
|(in thousands, except share and per share amounts)|
|Three Months Ended||Six Months Ended|
|June 30,||June 30,|
|Research and development||17,959||12,031||33,254||22,889|
|General and administrative||5,486||4,179||11,413||8,463|
|Total operating expenses||23,788||16,360||45,365||31,632|
|Interest expense, net||(669||)||(250||)||(1,193||)||(145||)|
|Net loss attributable to common shareholders||$||(24,457||)||$||(16,509||)||$||(46,430||)||$||(31,584||)|
|Net loss per share attributable to common shareholders, basic and diluted||$||(0.74||)||$||(0.61||)||$||(1.54||)||$||(1.17||)|
|Weighted-average common shares outstanding, basic and diluted||33,074,463||26,910,284||30,201,116||26,896,405|
Bellicum Pharmaceuticals, Inc. Alan Musso, CFO 832-384-1116 firstname.lastname@example.org Media: BMC Communications Brad Miles646-513-3125 email@example.com Source: Bellicum Pharmaceuticals