Bellicum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company. The Company is focused on discovering and developing cellular immunotherapies for various forms of cancer, including hematological cancers and solid tumors, as well as orphan inherited blood disorders. The Company uses its chemical induction of dimerization (CID) technology platform to engineer and then control components of the immune system. The Company is developing next-generation product candidates in the areas of cellular immunotherapy, including hematopoietic stem cell transplantation (HSCT), chimeric antigen receptors (CAR) T cells therapy and T-cell receptor (TCR) cell therapies. The Company's product candidates include BPX-501, BPX-401, BPX-601 and BPX-701. BPX-501 is an adjunct T cell therapy for allogeneic hematopoietic stem cell transplantation. The Company's CID-based technologies include CaspaCIDe, CIDeCAR and GoCAR-T.
Some analysts have focused on their Control Swith approach as differentiated.
Here is company description of tech:
Our Proprietary CID Technology Platform We are developing next-generation product candidates in some of the most important areas of cellular immunotherapy, including hematopoietic stem cell transplantation (HSCT), CAR T cell therapy, and TCR cell therapy.
Our proprietary CID technology platform was designed to address the challenges of current cellular immunotherapies by enabling control over cellular activities and functions, such as growth, activation, proliferation and cell death. Our CID platform consists of molecular switches—modified forms of signaling proteins— which are triggered inside the patient by infusion of small molecule rimiducid, instead of by natural upstream signals. Our current product candidates are based on either a “safety switch,” or an “activation switch.” After rimiducid is administered, the “safety switch” is designed to lead to programmed cell death, or apoptosis, and the “activation switch” is designed to lead to proliferation and/or activation and/or persistence of immune cells.
Rimiducid has been designed to bind to a specifically designed domain of CID switch proteins. Once introduced, rimiducid couples, or dimerizes, CID switch proteins together to create a cluster that triggers the signaling cascade. Aside from its impact on CID-modified immune cells bearing switch proteins, rimiducid has no other known effect on the body.
Post by Gene Editing on Sept 19, 2016 5:22:44 GMT -6
Positioned Well In Genetic Blood Disorders — Highlights Strength of Technology What's Incremental To Our View After meeting with management, we believe Bellicum’s BPX-501 could become an important player in the β -thalassemia market. We believe Bellcium is well positioned initially in a subset of β-thalassemia — and perhaps the broader population. Data suggests that BPX-501 appears more effective than Bluebird's solution in β0/β0 patients. We believe BPX-501 is an elegantly simple yet potentially effective treatment. We believe BPX-501 could be first to market both in the EU and the U.S. Next catalysts for the stock include YE data and regulatory updates. Reiterate our BUY rating and $30 PT. Bellicum is an under the radar player in the thalassemia market. Management has started to highlight the potential of BPX-501 not only in broader market of bone marrow transplant patients, but also in smaller “high profile” subsets of patients. BPX-501 is a therapy to improve the broader bone marrow transplant market – which also includes blood disorders such as β-thalassemia. Discussions about new therapies for β-thalassemia are often been dominated by more complex gene-therapies, such as those by bluebird bio (BLUE, $71.47, Buy, Edward Nash). We believe high profile genetic solutions for β-thalassemia, while powerful and potentially have broader utility, could face stiff competition from Bellicum’s BPX-501 in β-thalassemia. We believe Bellcium is an under-the-radar player in the β-thalassemia market. Bellicum’s BPX-501 has shown robust response in β0/β0 thalassemia patients, better than gene therapy. Bellicum has shown robust reconstitution of Hb levels and no non-malignant patient death. With continued remission and barring any uncontrolled graft versus host disease, BPX-501 could become the standard of care for bone marrow transplant based therapies. We believe BPX-501 could successfully lead a subset of the thalassemia market – about one third of thalassemia patients who harbor two mutated alleles, termed β0/β0 thalassemia with almost no hemoglobulin. We expect BPX-501 EU approval in 2018 and a U.S. launch strategy to take shape in 4Q16. Catalysts that could drive stock include: (1) Additional FDA and EMA color around BPX-501 in non-malignant setting by YE16, (2) data updates in 4Q16 (ASH) for BPX-501 — we expect to see no transplant-related mortality in nonmalignant cases, and (3) initial data readouts from BPX-601, potentially by mid-2017. Valuation and risks: Our DCF/NPV valuation for BLCM yields a $30 PT — $23 from non-malignant stem cell transplant, $1 from malignant stem cell transplant, and $6 from cash/technology. The greatest risk, in our view, is efficacy and safety failure in stem cell transplants, and Bellicum’s approach being made obsolete by their numerous and larger CAR T cell peers.
BPX-501 showed robust efficacy and no patient death in non-malignant trial. Bellicum’s BPX-501 has shown robust response in non-malignant stem cell transplant patients. The data update (EBMT, April 2016) from the trial with non-malignant patients showed all 24 pediatric patients were alive and disease-free. The treatment cohort included patients with thalassemia major β0/β0, severe combined immunodeficiency, Fanconi anemia, Wiskott-Aldrich syndrome and sickle cell disease. Patients showed successful immune reconstitution/ protection, low incidence of skin-only acute GvHD (n=4) and mild chronic GvHD (n=1), and significantly reduced post-transplant expenses — an approx. 50% reduction in time to discharge and an approx. 35% reduction in re-hospitalization rate. BPX-501 showed better efficacy in thalassemia major β0/β0 patients than gene therapy. Early data with BPX-501 has shown sustained blood-transfusion free survival. Thalassemia major β0/β0 is harder to treat compared to non-β0/β0 patients (i.e., β+/β0, β0/βE, βX/β0). Bellicum’ treatment regimen in β0/β0 patients showed reconstitution of hemoglobin to 10g/dl levels in all patients with hemoglobinopathies within three months post-BPX-501 treatment. This is in stark contrast to the efficacy observed in β0/β0 patients treated with bluebird bio’s (BLUE, $71.47, Buy, Edward Nash) gene therapy drug, LentiGlobin (ASH, December 2015) — hemoglobin levels at 5g/dl at six months after treatment. However, it is notable BLUE's data represents only the protein produced by the transgene, though endogenous protein levels probably would not be very large. Additionally BLUE has identified two compounds for process optimization and in vitro results showed vector copy numbers and the percentage of transduced cells were increased with the addition of the compounds. Most β0/β0 patients treated with LentiGlobin continue to receive blood transfusion, albeit at a reduced volume. Target patient groups — β0/β0 for Bellicum and non-β0/β0 for Bluebird Bio. The BPX-501 trial for non-malignant patients has enrolled only β0/β0 patients, but we expect similar robust efficacy in non-β0/β0. Bluebird’s LentiGlobin has also shown robust efficacy in non-β0/β0 patients — an ongoing sustained transfusion free survival of 7-23+ months, and Hb level of 10g/dl at six months post treatment. Given the fact that LentiGlobin treatment involves autologous vs haploidentical transplantation with BPX-501, we believe the rate of adoption will be higher for LentiGlobin due to GvHD related concerns associated with BPX-501. We estimate ~30% of thalassemia patients are β0/β0 and ~70% are non- β0/β0. Bellicum’s BPX-501 could potentially enter the market two years before Bluebird’s LentiGlobin. Bellicum has certain advantages — (1) compared to Bluebird’s LentiGlobin gene therapy and autologous transplant, Bellicum’s treatment involves haploidentical transplant (patient’s parents are the donors), sparing the patients to go through bone marrow donation process; (2) Bellicum engineers only the T cells (like other T cell therapy peers) compared to Bluebird’s genetic engineering of the stem cells with the modified Hb gene, which could raise regulatory concerns; and (3) approval of BPX-501 will make it eligible for treatment for all non-malignant/malignant indications that requires bone marrow transplant, as opposed to Bluebird’s indication specific approval of gene therapy. Both Bellicum and Bluebird are pursuing the EU approval before the U.S. approval. BPX-501 could receive the EU approval in 2018, approx. two years ahead of Bluebird’s conditional approval, giving an additional advantage to Bellicum. The key concern with BPX-501 is the risk of GvHD. In the trial with malignant patients, we have seen one patient death from chronic GvHD caused by allograft T cells, and is not considered BPX-501 or rimiducid related. In the upcoming Phase 2 trial with non-malignant patients, risk GvHD could play the major role in the success of the drug
Post by Gene Editing on Sept 23, 2016 9:14:37 GMT -6
suntrust Positive Data Update In Non- Malignant Setting — Encouraging For Filing Reiterate Buy and $30 PT What's Incremental To Our View Encouraging data update, positive for approval — disease free in the nonmalignant setting at 13 months (n=18) versus the prior update of 7 months (n=11). BPX-501 showed zero treatment related deaths in primary immune deficiency patients and no disease at 13 month median follow up. The next catalysts will be further BPX-501 data in December. This data helps add conviction around our view that BPX-501 is well positioned for filing and approval. BPX-501 shows ongoing encouraging results from the non-malignant setting at 13 month median follow up. Dr. Franco Locatelli gave a presentation at the 2016 European society for immunodeficiencies meeting showing preliminary results for 18 pediatric primary immune-deficiency patients. The data showed that with a median follow up time of 13 months all patients remain disease free with no treatment related mortality after a T-depleted haploidentical hematopoietic stem cell transplant and BPX-501 infusion. Previously we had seen initial results for 11 of these patients in April at the European Society for Blood and Marrow Transplantation in April 2016 which showed patients were disease free and had no treatment related mortality at 7 months median follow up. Results from both malignant and non-malignant settings expected at ASH. Bellicum plans to present an update on BP-004 at ASH on December 3-6, where we also expect a further BPX-501 update. We expect a European filing in the non-malignant setting in late 2017. Catalysts that could drive stock include: (1) Additional FDA and EMA color around BPX-501 in non-malignant setting by YE16, (2) data updates in 4Q16 (ASH) for BPX-501 — we expect to see no transplant-related mortality in nonmalignant cases, and (3) initial data readouts from BPX-601, potentially by mid-2017. Valuation and risks: Our DCF/NPV valuation for BLCM yields a $30 PT— $23 from non-malignant stem cell transplant, $1 from malignant stem cell transplant, and $6 from cash/technology. The greatest risk, in our view, is efficacy and safety failure in stem cell transplants, and Bellicum’s approach being made obsolete by their numerous and larger CAR T cell peers.
Post by Gene Editing on Sept 23, 2016 12:22:45 GMT -6
Alert: Data keep getting better Earlier this morning, BLCM reported data update from 18 patient study investigating BPX-501 in pediatric patients with primary immune deficiencies (PIDs). In line with our expectations in non-malignant patients, data continue to be encouraging – all patients are disease free and alive at a median 13 month follow-up. We believe this to be impressive, particularly in PID patients with no alternative to transplant. We expect a comprehensive update at ASH to solidify BPX-501 regulatory path in nonmalignant patients. These data follow a comprehensive update on 24 non-malignant patients provided at EBMT in April 2016 which reported all 24 patients to be disease free with no treatment related mortalities at a median 7 month follow-up. While data from PID patients alone has not been presented, co. indicated that PID patients were some of the early enrollers for the trial which started Dec 2014, indicating a higher median DFS in PID patients. The co. notes that these data are from a single site in Rome and that patients at other centers continue to enroll. We now have a clear path in EU and expect to see similar clarity from FDA by YE2016. We note that while 6 month data is sufficient for filing for both malignant and non-malignant pediatric patients in EU, 13 month data gets us closer to what doctors want to see (15-18 months) before they become absolutely comfortable with the agent, particularly in the malignant setting. We believe that updated, comprehensive BPX-501 data at ASH 2016 and clarity on FDA approval pathway by YE2016 are important near-term catalysts for the stock. Given that the path in non-malignant transplant is easier, we note that 100% credit to non-malignant only (everything else zero) gives a FV of $18/sh. Every 5% probability of success in malignant transplant adds ~$2/sh to DCF. At 100% probability to BPX-501, we see FV of company at $64/sh (>250% upside potential).
Under: Been on the sidelines for a bit holding (building) cash. Now that "BIGLEY" has rolled out the tax plan its time to jump in.
Dec 21, 2017 19:06:02 GMT -6
martyc: Looks like you are buying Msft again!
Dec 15, 2017 11:23:29 GMT -6
martyc: The news that Trump called Rupert to congratulate him sure seems to indicate that this is heading to approval
Dec 15, 2017 11:22:23 GMT -6
Under: DIS finally getting some traction.?
Dec 14, 2017 17:08:45 GMT -6
martyc: I took an entry level position in DIS. Will add eventually to overweight when it becomes clearer that the deal will go thru. Can't believe how well positioned they will be. 60% Hulu. 20% of content watched on NFLX they can pull. More in thread
Dec 14, 2017 11:05:16 GMT -6
Under: Great posts on $DIS
Dec 13, 2017 17:50:49 GMT -6
Under: $ROKU Citron on a war path.
Nov 28, 2017 15:11:20 GMT -6
Under: $HAS takeover bid for $MAT?
Nov 10, 2017 16:16:07 GMT -6
martyc: Not looking like the market will provide any discounted opp for SGMO. Call was just too professional and all signs indicate they are on a great path for commercialization. Happy with core but wish I had some trading shs
Nov 10, 2017 9:04:05 GMT -6
martyc: For anyone looking to find an entry point into SGMO, I'm almost hoping is sells off in next few days so I can add more. They are really clicking but the fact they haven't signed new deals might cause some to exit. Watching as I have room for trading shs
Nov 9, 2017 18:28:09 GMT -6
martyc: Been an interesting ride so far. I figured the Bears would be about this good but hoped the O wouldn't look so lame. Another building yr but still possible to get to 8-8 IMO
Nov 9, 2017 18:26:08 GMT -6
Under: whats up with your Bears this year Marty?
Nov 9, 2017 17:35:25 GMT -6
martyc: Hope you were long ROKU. I wanted to see Q first so missed out
Nov 9, 2017 7:08:53 GMT -6