Bellicum Pharmaceuticals Reports Second Quarter 2016 Financial Results and Provides Corporate Update
Two INDs cleared by
Conference call and webcast to be held Monday, August 8, 2016 at 5 p.m. Eastern
“In the last six months, we have achieved important milestones across
our stem cell transplant, TCR and CAR T programs,” said
Program and Regulatory Updates
Received US and EU orphan drug designations for BPX-501 and
rimiducid, and announced strategy to pursue EMA approval under
exceptional circumstances based on expanded BP-004 trial. Bellicum
has met with regulatory authorities in
Europeto discuss the potential approval pathway for BPX-501 and for rimiducid for the treatment of immunodeficiency and GvHD following a haploidentical HSCT in pediatric patients with leukemias, lymphomas and rare inherited blood diseases who do not have a matched donor. Based on these regulatory discussions, Bellicum believes that data from the European arm of its BP-004 trial, with a six-month follow-up time and expanded to enroll additional patients, could form the basis of Marketing Authorization Applications for BPX-501 and rimiducid. In place of a randomized trial, the Company intends to collect data from a concurrent observational study of allogeneic HSCT outcomes in the pediatric setting. Details will be further refined in a formal protocol assistance process.
- Reported new interim data from BP-004 trial in an oral presentation at the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation in April. Results demonstrated disease-free outcomes, reduced treatment-related mortality, reduced infection rates, faster immune reconstitution, and significant reductions in time-to-hospital discharge and re-hospitalizations, compared to historical controls. The Company expects to provide updated data by the end of 2016.
- Initiated BP-008, a Phase 1 study of BPX-501 to treat post-transplant relapse in adults and children with blood cancers. The safety study, which includes matched as well as haploidentical transplant recipients, will also evaluate the potential for a titrated dose of rimiducid to resolve uncontrolled GvHD while preserving a greater proportion of BPX-501 cells.
Following allowance by the
FDAof its Investigational New Drug (IND) application, Bellicum is completing preparations for the start of BP-012, a Phase 1 BPX-601 GoCAR-T™ trial in an initial indication of non-resectable pancreatic cancer. GoCAR-T contains Bellicum’s proprietary iMC activation switch and is designed to treat solid tumors expressing prostate stem cell antigen. The clinical trial (NCT02744287), which is expected to enroll up to 30 patients in a 3+3 dose escalation/de-escalation design, will be conducted at Baylor Sammons Cancer Centerin Dallas, Texas.
With its BPX-701 IND allowed by the
FDA, the Company is preparing for initiation of BP-011, a Phase 1 clinical trial with its high-affinity T cell receptor (TCR) product candidate. BPX-701 incorporates the CaspaCIDe safety switch and is designed to target malignant cells expressing the preferentially-expressed antigen in melanoma, or PRAME. Initial planned indications include Refractory or Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndromes, with an additional study planned for metastatic uveal melanoma. BP-011 (NCT02743611), which is expected to enroll up to 36 AML/MDS patients in a 3+3 dose escalation/de-escalation design, will be conducted at Oregon Health and Science Universityand Leiden University Medical Center(LUMC).
CD19 CAR T Program:
July 2016, the Company decided to support CD19 programs designed to establish clinical proof-of-concept for CaspaCIDe in the CD19 setting being advanced by two of our academic collaborators, in place of advancing BPX-401. The Company believes that this strategy allows a cost-effective and differentiated approach to the highly competitive landscape of CD19-targeted therapies in development.
Expanded research collaboration with
Leiden University Medical Centerfor 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.
U.S. patent issued that strengthens the IP around Bellicum’s
CaspaCIDe cell therapy safety platform. U.S. patent 9,393,292 was
Baylor College of Medicinefor a method of cell therapy that enables the selective elimination of administered cells that have been modified to express an inducible caspase-9 protein (iCasp9). Bellicum holds exclusive worldwide rights to the invention.
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 5:00 p.m. EDT today to discuss the financial results. To access the call, participants should dial (855) 779-9069 (U.S. domestic) and (631) 485-4863 (international) at least 10 minutes prior to the start of the call, using Conference ID number 53430483. The event will be webcast live and can also be accessed in the Events & Presentations section of bellicum.com. An archived version of the webcast will be available for replay in the Investor and Media section of the Bellicum website for at least two weeks following the call.
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 rimiducid, CaspaCIDe,
GoCAR-T, or iMC; the effectiveness of rimiducid, CaspaCIDe,
GoCAR-T, or iMC, their possible range of application and
potential curative effects and safety in the treatment of diseases; the
timing and success of our clinical trials, including the rate and
progress of enrollment in our BP-004 clinical trial or in any
observational studies and in clinical trials for BPX-601 and BPX-701;
the timing of regulatory filings for BPX-501 and for rimiducid; our
research and development activities relating to BPX-501, BPX-601 and
BPX-701; and the potential applications and effectiveness of our product
candidates BPX-501, BPX-601 and BPX-701, including as compared to other
treatment options and competitive therapies. 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|
|Cash and cash equivalents||$||49,549||$||70,241|
|Investment securities, available-for-sale - short-term||56,025||23,820|
|Receivables and other current assets||2,605||2,829|
|Investment securities, available-for-sale, long-term||30,999||56,304|
|Property and equipment, net||10,512||6,882|
|Other assets, net||240||330|
|Accounts payable and other accrued liabilities||6,347||7,186|
|Other current liabilities||264||259|
|Other liabilities, net of current portion||881||944|
|Total Stockholders' Equity||127,487||152,017|
|Total liabilities and stockholders' equity||$||149,930||$||160,406|
|BELLICUM PHARMACEUTICALS, INC.|
|Unaudited Condensed Statements of Operations|
|(in thousands, except share and per share amounts)|
|Three Months Ended||Six Months Ended|
|June 30,||June 30,|
|Research and development||12,181||8,012||23,169||13,730|
|General and administrative||4,179||2,777||8,463||4,974|
|Total operating expenses||16,360||10,789||31,632||18,704|
|Interest income (expense), net||(250||)||171||(145||)||221|
|Net loss attributable to common shareholders||$||(16,509||)||$||(10,534||)||$||(31,584||)||$||(18,292||)|
|Net loss per share attributable to common shareholders, basic and diluted||$||(0.61||)||$||(0.40||)||$||(1.17||)||$||(0.70||)|
|Weighted-average common shares outstanding, basic and diluted||26,910,284||26,268,610||26,896,405||26,264,025|