HOUSTON, TX, USA I January 11, 2016 I Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for cancers and orphan inherited blood disorders, today announced regulatory milestones and program updates on its CAR T and TCR product candidates.
Bellicum is preparing for the initiation of clinical studies in 2016 of its three most advanced CAR T and TCR adoptive cell therapy product candidates, BPX-701, BPX-601, and BPX-401. Today the Company submitted required documentation, including clinical trial protocols, for BPX-701 and BPX-601 for review by the National Institutes of Health (NIH) Recombinant DNA Advisory Committee (RAC). If selected for public review, such review would be expected to take place at the next RAC meeting scheduled for March 8-10, 2016.
The Company further announced that it expects to begin enrolling patients in Phase 1 trials of BPX-701 and BPX-601 in mid-2016, and BPX-401 in the second half of 2016, with IND filings to follow RAC review in each case.
BPX-701 is a CaspaCIDe®-enabled natural high affinity T cell receptor (TCR) product candidate designed to target malignant cells expressing the preferentially-expressed antigen in melanoma (PRAME). Initial planned indications for BPX-701 development are Refractory or Relapsed Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS) with an additional study planned for metastatic uveal melanoma. Each of these are orphan indications where PRAME is highly expressed and for which current treatment options are limited. The Company expects to submit European regulatory filings to allow initiation of clinical development at a European site after the U.S. IND has been allowed.
BPX-601 is a GoCAR-T™ product candidate containing Bellicum’s proprietary iMC (inducible MyD88/CD40) activation switch, designed to treat solid tumors expressing prostate stem cell antigen (PSCA). As reported at ASH, preclinical data shows enhanced T-cell proliferation, persistence and in vivo anti-tumor activity compared to traditional CAR T therapies. The initial planned indication for BPX-601 development is non-resectable pancreatic cancer.
BPX-401 is a CIDeCAR™ product candidate incorporating Bellicum’s proprietary MC co-stimulatory domain and the CaspaCIDe safety switch, designed to target blood cancers expressing CD19.
“Throughout 2015, we made significant progress across all of our adoptive cell therapy programs,” said Tom Farrell, President and CEO of Bellicum Pharmaceuticals. “We’re now poised to bring three novel CAR T and TCR product candidates into the clinic in 2016, utilizing our molecular switch and proprietary co-stimulatory domain technologies. Importantly, we have in place the financial and management resources needed not only to bring these novel programs forward but also to continue discovering exciting new ways that our technologies can potentially improve the treatment of cancers and other diseases.”
Background on the NIH RAC Process
Clinical trial protocols and other required information for product candidates that involve gene transfer are reviewed by both the FDA and the NIH, through the Recombinant DNA Advisory Committee or RAC. The NIH’s Office of Biotechnology Activities (OBA) convenes quarterly RAC meetings to make recommendations and selectively elicit public discussion of scientific, safety or ethical issues. The OBA notifies the FDA of the outcome of RAC reviews and reports are posted to the OBA website.
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.
SOURCE: Bellicum Pharmaceuticals
Post Views: 323
HOUSTON, TX, USA I January 11, 2016 I Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for cancers and orphan inherited blood disorders, today announced regulatory milestones and program updates on its CAR T and TCR product candidates.
Bellicum is preparing for the initiation of clinical studies in 2016 of its three most advanced CAR T and TCR adoptive cell therapy product candidates, BPX-701, BPX-601, and BPX-401. Today the Company submitted required documentation, including clinical trial protocols, for BPX-701 and BPX-601 for review by the National Institutes of Health (NIH) Recombinant DNA Advisory Committee (RAC). If selected for public review, such review would be expected to take place at the next RAC meeting scheduled for March 8-10, 2016.
The Company further announced that it expects to begin enrolling patients in Phase 1 trials of BPX-701 and BPX-601 in mid-2016, and BPX-401 in the second half of 2016, with IND filings to follow RAC review in each case.
BPX-701 is a CaspaCIDe®-enabled natural high affinity T cell receptor (TCR) product candidate designed to target malignant cells expressing the preferentially-expressed antigen in melanoma (PRAME). Initial planned indications for BPX-701 development are Refractory or Relapsed Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS) with an additional study planned for metastatic uveal melanoma. Each of these are orphan indications where PRAME is highly expressed and for which current treatment options are limited. The Company expects to submit European regulatory filings to allow initiation of clinical development at a European site after the U.S. IND has been allowed.
BPX-601 is a GoCAR-T™ product candidate containing Bellicum’s proprietary iMC (inducible MyD88/CD40) activation switch, designed to treat solid tumors expressing prostate stem cell antigen (PSCA). As reported at ASH, preclinical data shows enhanced T-cell proliferation, persistence and in vivo anti-tumor activity compared to traditional CAR T therapies. The initial planned indication for BPX-601 development is non-resectable pancreatic cancer.
BPX-401 is a CIDeCAR™ product candidate incorporating Bellicum’s proprietary MC co-stimulatory domain and the CaspaCIDe safety switch, designed to target blood cancers expressing CD19.
“Throughout 2015, we made significant progress across all of our adoptive cell therapy programs,” said Tom Farrell, President and CEO of Bellicum Pharmaceuticals. “We’re now poised to bring three novel CAR T and TCR product candidates into the clinic in 2016, utilizing our molecular switch and proprietary co-stimulatory domain technologies. Importantly, we have in place the financial and management resources needed not only to bring these novel programs forward but also to continue discovering exciting new ways that our technologies can potentially improve the treatment of cancers and other diseases.”
Background on the NIH RAC Process
Clinical trial protocols and other required information for product candidates that involve gene transfer are reviewed by both the FDA and the NIH, through the Recombinant DNA Advisory Committee or RAC. The NIH’s Office of Biotechnology Activities (OBA) convenes quarterly RAC meetings to make recommendations and selectively elicit public discussion of scientific, safety or ethical issues. The OBA notifies the FDA of the outcome of RAC reviews and reports are posted to the OBA website.
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.
SOURCE: Bellicum Pharmaceuticals
Post Views: 323
