Novel cellular immunotherapies for cancers and orphan inherited blood disorders (Seite 4)
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Quarterly earnings reports matter a lot more for some companies than others. According to Wall Street analysts, investors should look beyond Bellicum Pharmaceuticals Inc
BLCM 2.44%
’s first-quarter earnings miss and focus on its drug pipeline, which includes leading cancer treatment candidate BPX-501.
Jefferies' Analysis
For Jefferies analyst Biren Amin, the biggest takeaway from Bellicum’s earnings report was that the pipeline is on-track.
“BLCM continues discussions with [the] FDA to determine U.S. regulatory pathway for BPX-501,” Amin noted in a new report.
“BLCM also maintained that BP-004 is on track with enrollment, which will drive potential filing in EU in mid-2018.”
Amin expects an update on BPX-501 by mid-2017.
Cantor Fitzgerald's Take
Cantor Fitzgerald analyst Elemer Piros expects that update to come at the European Hematology Association meeting in June.
“In our view, the focus remains on emerging data from the Phase ½ study with BXP-501 in pediatric haplo-stem cell transplant patients,” Piros explained.
In addition, Piros is looking for protocol for pivotal trials of BPX-501 in the U.S. to be finalized by the end of 2017. Cantor expects Bellicum’s reported cash balance of $165 million to be plenty to get the company through the end of 2018.
Piros believes Bellicum’s modified T-cell cancer treatments could eventually open the door for blood stem cell transplantation for more than 13,000 U.S. patients.
Both Cantor Fitzgerald and Jefferies remain bullish on Belicum’s shares. Jefferies maintains a Buy rating and has lowered its price target from $19 to $18. Cantor Fitzgerald is even more optimistic, maintaining an Overweight rating and a $32 price target.
https://www.benzinga.com/analyst-ratings/analyst-color/17/05…
BLCM 2.44%
’s first-quarter earnings miss and focus on its drug pipeline, which includes leading cancer treatment candidate BPX-501.
Jefferies' Analysis
For Jefferies analyst Biren Amin, the biggest takeaway from Bellicum’s earnings report was that the pipeline is on-track.
“BLCM continues discussions with [the] FDA to determine U.S. regulatory pathway for BPX-501,” Amin noted in a new report.
“BLCM also maintained that BP-004 is on track with enrollment, which will drive potential filing in EU in mid-2018.”
Amin expects an update on BPX-501 by mid-2017.
Cantor Fitzgerald's Take
Cantor Fitzgerald analyst Elemer Piros expects that update to come at the European Hematology Association meeting in June.
“In our view, the focus remains on emerging data from the Phase ½ study with BXP-501 in pediatric haplo-stem cell transplant patients,” Piros explained.
In addition, Piros is looking for protocol for pivotal trials of BPX-501 in the U.S. to be finalized by the end of 2017. Cantor expects Bellicum’s reported cash balance of $165 million to be plenty to get the company through the end of 2018.
Piros believes Bellicum’s modified T-cell cancer treatments could eventually open the door for blood stem cell transplantation for more than 13,000 U.S. patients.
Both Cantor Fitzgerald and Jefferies remain bullish on Belicum’s shares. Jefferies maintains a Buy rating and has lowered its price target from $19 to $18. Cantor Fitzgerald is even more optimistic, maintaining an Overweight rating and a $32 price target.
https://www.benzinga.com/analyst-ratings/analyst-color/17/05…
Bellicum is a small-cap biotech developing a host of genetically modified cell-based therapies for blood disorders and various cancers. While cell-based immunotherapies are now a common feature of many pharma pipelines, Bellicum stands apart from the crowd because of its proprietary "chemical induction of dimerization" (CID) technology that's designed to enhance the safety and efficacy profiles of these novel cancer-fighting cell therapies.
Specifically, the biotech's cellular therapies incorporate a molecular switching mechanism that can be triggered by a small molecule known as rimiducid to either induce programmed cell death (apoptosis) in the event of a safety issue, or cause the infused cells to proliferate to enhance potency.
Using its CID platform, Bellicum designed its lead T-cell therapy product candidate, BPX-501, to improve patient outcomes during a half-matched T-depleted, hematopoietic stem-cell transplantation (HSCT) -- a process that involves the intravenous infusion of stem cells as a way to restart the production of blood cells in patients with bone-marrow or immune-system disorders.
Although half-matched HSCT can be life-saving in many instances, this procedure does have serious life-threatening drawbacks, such as graft-versus-host-disease (GvHD) or an increased risk of infection from the eradication of T-cells before infusion. BPX-501's built-in safety switch, however, should solve this problem by lowering the risk of uncontrolled bouts of GvHD, while still allowing patients to benefit from a higher T-cell count.
The good news is that the therapy's early-stage results across a range of rare blood disorders are proving to be a game changer for many HSCT patients. As such, Bellicum is hoping to file for BPX-501's first regulatory approval in the EU by mid-2018 and nail down an acceptable regulatory pathway for the therapy in the U.S. by the middle of this year.
The downside, though, is that the biotech's cash runway probably isn't sufficient to see it all the way through to BPX-501's worldwide commercialization. Bellicum, after all, has around $150 million remaining in cash following its latest secondary offering, but it also has a quarterly burn rate of around $20 million that's bound to grow as its clinical activities expand into late-stage development.
https://www.fool.com/investing/2017/03/29/better-buy-bellicu…
Specifically, the biotech's cellular therapies incorporate a molecular switching mechanism that can be triggered by a small molecule known as rimiducid to either induce programmed cell death (apoptosis) in the event of a safety issue, or cause the infused cells to proliferate to enhance potency.
Using its CID platform, Bellicum designed its lead T-cell therapy product candidate, BPX-501, to improve patient outcomes during a half-matched T-depleted, hematopoietic stem-cell transplantation (HSCT) -- a process that involves the intravenous infusion of stem cells as a way to restart the production of blood cells in patients with bone-marrow or immune-system disorders.
Although half-matched HSCT can be life-saving in many instances, this procedure does have serious life-threatening drawbacks, such as graft-versus-host-disease (GvHD) or an increased risk of infection from the eradication of T-cells before infusion. BPX-501's built-in safety switch, however, should solve this problem by lowering the risk of uncontrolled bouts of GvHD, while still allowing patients to benefit from a higher T-cell count.
The good news is that the therapy's early-stage results across a range of rare blood disorders are proving to be a game changer for many HSCT patients. As such, Bellicum is hoping to file for BPX-501's first regulatory approval in the EU by mid-2018 and nail down an acceptable regulatory pathway for the therapy in the U.S. by the middle of this year.
The downside, though, is that the biotech's cash runway probably isn't sufficient to see it all the way through to BPX-501's worldwide commercialization. Bellicum, after all, has around $150 million remaining in cash following its latest secondary offering, but it also has a quarterly burn rate of around $20 million that's bound to grow as its clinical activities expand into late-stage development.
https://www.fool.com/investing/2017/03/29/better-buy-bellicu…
Annemarie Moseley plans step down as the company’s COO and executive vice president of clinical development, effective July 31. She will remain a consultant to Bellicum through January.
“I would like to thank Annemarie for her many contributions to the success of Bellicum, particularly in establishing the clinical program for BPX-501 in Europe and the U.S., and developing the company’s cell therapy manufacturing capabilities,” Rick Fair, Bellicum’s president and CEO, said in a press release. “Annemarie’s tireless efforts over the last six years leave us well positioned as we evaluate our controlled CAR T and TCR candidates in the clinic and prepare for the commercialization of BPX-501.”
Bellicum has begun a search for a chief medical officer and promoted Alan Smith to executive vice president of technical operations. He will be responsible for Bellicum’s manufacturing, process and systems development, and supply chain groups. Smith previously served as senior vice president of manufacturing since 2015.
“Alan’s expertise and strong leadership in this expanded role will continue to serve us well as we prepare for the expected commercialization of BPX-501 and expansion of the clinical programs for our controllable CAR T and TCR therapies,” Fair said in the release.
Fair also is a new addition to Bellicum, having joined the company as president and CEO in January.
In November, the Cancer Prevention and Research Institute of Texas awarded Bellicum nearly $17 million to further development for its BPX-501 drug, which is being designed to fight acute myeloid leukemia.
http://www.bizjournals.com/houston/news/2017/05/16/houston-p…
“I would like to thank Annemarie for her many contributions to the success of Bellicum, particularly in establishing the clinical program for BPX-501 in Europe and the U.S., and developing the company’s cell therapy manufacturing capabilities,” Rick Fair, Bellicum’s president and CEO, said in a press release. “Annemarie’s tireless efforts over the last six years leave us well positioned as we evaluate our controlled CAR T and TCR candidates in the clinic and prepare for the commercialization of BPX-501.”
Bellicum has begun a search for a chief medical officer and promoted Alan Smith to executive vice president of technical operations. He will be responsible for Bellicum’s manufacturing, process and systems development, and supply chain groups. Smith previously served as senior vice president of manufacturing since 2015.
“Alan’s expertise and strong leadership in this expanded role will continue to serve us well as we prepare for the expected commercialization of BPX-501 and expansion of the clinical programs for our controllable CAR T and TCR therapies,” Fair said in the release.
Fair also is a new addition to Bellicum, having joined the company as president and CEO in January.
In November, the Cancer Prevention and Research Institute of Texas awarded Bellicum nearly $17 million to further development for its BPX-501 drug, which is being designed to fight acute myeloid leukemia.
http://www.bizjournals.com/houston/news/2017/05/16/houston-p…
Bellicum is working on a variety of genetically modified cell therapies that have the potential to be game-changers for their respective indications. But the company's value proposition appears to have been overshadowed by its chief competitors, Kite Pharma and Juno Therapeutics, in the modified cell therapy arena. Point blank: Kite and Juno both sport multibillion-dollar valuations that absolutely dwarf Bellicum's current market cap.
To be fair, both Kite and Juno are farther along in the drug development process. Kite is steadily marching toward a regulatory approval for its non-Hodgkin lymphoma treatment known as axicabtagene ciloleucel, and Juno is also racing toward a regulatory filing for its cell therapy, JCAR017, in diffuse large B-cell lymphoma. However, Bellicum is nipping at the heels of both of these better-known cell therapy companies with its lead product candidate BPX-501, which has the potential to be safer than its competitors' drugs.
Turning to the details, BPX-501 is a T-cell therapy product candidate designed to improve patient outcomes during a half-matched T-depleted, hematopoietic stem-cell transplantation. And if approved, it would probably generate well over $1 billion in sales within a few short years. The good news is that the early stage results have been extremely encouraging, prompting Bellicum to advance BPX-501 into registration studies both at home and abroad.
To be fair, both Kite and Juno are farther along in the drug development process. Kite is steadily marching toward a regulatory approval for its non-Hodgkin lymphoma treatment known as axicabtagene ciloleucel, and Juno is also racing toward a regulatory filing for its cell therapy, JCAR017, in diffuse large B-cell lymphoma. However, Bellicum is nipping at the heels of both of these better-known cell therapy companies with its lead product candidate BPX-501, which has the potential to be safer than its competitors' drugs.
Turning to the details, BPX-501 is a T-cell therapy product candidate designed to improve patient outcomes during a half-matched T-depleted, hematopoietic stem-cell transplantation. And if approved, it would probably generate well over $1 billion in sales within a few short years. The good news is that the early stage results have been extremely encouraging, prompting Bellicum to advance BPX-501 into registration studies both at home and abroad.
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Antwort auf Beitrag Nr.: 53.099.610 von Shanghai2006 am 20.08.16 01:51:40http://www.nytimes.com/2016/08/02/health/cancer-cell-therapy…
Bleibt einem ja nix anderes übrig, als einzusteigen!
Gruss,s.
Bleibt einem ja nix anderes übrig, als einzusteigen!
Gruss,s.
Second Quarter and Six Months Ended June 30, 2016 Financial Results Bellicum reported a net loss of $16.5 million for the second quarter of 2016 and $31.6 million for the six months ended June 30, 2016, compared to a net loss of $10.5 million and $18.3 million for the comparable periods in 2015. The results included non-cash, stock-based compensation charges of $3.1 million and $6.2 million for the second quarter and six months ended June 30, 2016 and $2.1 million and $3.6 million for the comparable periods in 2015. As of June 30, 2016, cash and investments totaled $136.6 million. Bellicum continues to expect to end 2016 with approximately $80 to $90 million in cash, cash equivalents and investments, and that current cash resources will be sufficient to meet operating requirements through 2017. This guidance includes planned spending in the second half of 2016 of approximately $15 million for capital projects to enable in-house U.S. manufacturing. Research and development expenses were $12.2 million and $23.2 million, for the three and six months ended June 30, 2016, respectively, compared to $8.0 million and $13.7 million during the comparable periods in 2015. The higher expenses in the 2016 periods were primarily due to an increase in manufacturing and clinical expenses as a result of increased patient enrollment in our BPX-501 clinical trials, increased expenses for the IND-enabling activities on our product candidates BPX-601, BPX-701 and increased personnel and infrastructure costs. General and administrative expenses were $4.2 million and $8.5 million for the three and six months ended June 30, 2016, respectively, compared to $2.8 million and $5.0 million during the comparable periods in 2015. The higher expenses in the 2016 periods were primarily due to the growth of the organization, including an increase in costs related to personnel, higher facility costs and increased legal, accounting and travel related expenses.
Bellicum Pharmaceuticals Reports Second Quarter 2016 Financial Results and Provides Corporate Update | wallstreet-online.de - Vollständiger Artikel unter:
http://www.wallstreet-online.de/nachricht/8830537-bellicum-p…
Bellicum Pharmaceuticals Reports Second Quarter 2016 Financial Results and Provides Corporate Update | wallstreet-online.de - Vollständiger Artikel unter:
http://www.wallstreet-online.de/nachricht/8830537-bellicum-p…
Products in the Pipeline:
BPX-501: 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 Europe to 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. BPX-601: Following allowance by the FDA of 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 Center in Dallas, Texas. BPX-701: 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 University and Leiden University Medical Center (LUMC). CD19 CAR T Program: In 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. Corporate Updates Expanded research collaboration with Leiden University Medical Center for 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 issued to Baylor College of Medicine for 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.
Bellicum Pharmaceuticals Reports Second Quarter 2016 Financial Results and Provides Corporate Update | wallstreet-online.de - Vollständiger Artikel unter:
http://www.wallstreet-online.de/nachricht/8830537-bellicum-p…
BPX-501: 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 Europe to 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. BPX-601: Following allowance by the FDA of 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 Center in Dallas, Texas. BPX-701: 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 University and Leiden University Medical Center (LUMC). CD19 CAR T Program: In 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. Corporate Updates Expanded research collaboration with Leiden University Medical Center for 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 issued to Baylor College of Medicine for 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.
Bellicum Pharmaceuticals Reports Second Quarter 2016 Financial Results and Provides Corporate Update | wallstreet-online.de - Vollständiger Artikel unter:
http://www.wallstreet-online.de/nachricht/8830537-bellicum-p…
Tom Farrell, President and Chief Executive Officer of Bellicum said:
" In the last six months, BLCM has achieved important milestones across stem cell transplant, TCR and CAR T programs. BLCM received orphan drug status from the EU and US for BPX-501 and rimiducid, and has reached initial agreement with EMA around a pathway to filing Marketing Authorization Applications for each based on the ongoing BP-004 clinical trial. In addition, BLCM is pleased to report that the investigational new drug applications for both BPX-701 TCR and BPX-601 GoCAR-T product candidates have been cleared by the FDA, and BLCM is preparing to start Phase 1 studies."
Vollständiger Artikel unter:
http://www.wallstreet-online.de/nachricht/8830537-bellicum-p…
" In the last six months, BLCM has achieved important milestones across stem cell transplant, TCR and CAR T programs. BLCM received orphan drug status from the EU and US for BPX-501 and rimiducid, and has reached initial agreement with EMA around a pathway to filing Marketing Authorization Applications for each based on the ongoing BP-004 clinical trial. In addition, BLCM is pleased to report that the investigational new drug applications for both BPX-701 TCR and BPX-601 GoCAR-T product candidates have been cleared by the FDA, and BLCM is preparing to start Phase 1 studies."
Vollständiger Artikel unter:
http://www.wallstreet-online.de/nachricht/8830537-bellicum-p…
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