On November 1, 2017 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported that 14 abstracts have been selected for presentation, including three oral presentations, at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) 2017 annual meeting being held December 9-12, 2017 in Atlanta (Press release, Karyopharm, NOV 1, 2017, View Source [SID1234521401]). Four key abstracts being presented at the meeting will feature clinical data from Karyopharm’s ongoing Phase 1b/2 STOMP study evaluating selinexor, the Company’s lead, novel, oral SINE compound, in combination with backbone therapies for the treatment of patients with heavily pretreated multiple myeloma (MM). The four STOMP presentations will include updated data from the arms evaluating selinexor in combination with Velcade (bortezomib) and low-dose dexamethasone (SVd), selinexor in combination with Pomalyst (pomalidomide) and low-dose dexamethasone (SPd), and selinexor in combination with Revlimid (lenalidomide) and low-dose dexamethasone (SRd), and preliminary data from the arm evaluating selinexor in combination with Darzalex (daratumumab) and low-dose dexamethasone (SDd).

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“Despite several treatment advances for myeloma patients, there is a need for treatments with novel mechanisms, and many patients favor orally administered medicines,” said Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm. “Previously reported data from the ongoing Phase 1b/2 STOMP study showed promising response rates in patients with heavily pretreated myeloma when oral selinexor is combined with Velcade (“SVd”) or Pomalyst (“SPd”). We are very pleased to provide updated data for selinexor in combination with these agents, new data for selinexor in combination with Revlimid (“SRd”), and early results from the new Darzalex (“SDd”) combination arm at ASH (Free ASH Whitepaper) this year. We believe these data support the potential of selinexor as a backbone therapy with commonly used agents for multiple myeloma. Moreover, we believe the new data continue to support our ongoing Phase 3 BOSTON study of SVd in myeloma.”

Details for the ASH (Free ASH Whitepaper) 2017 presentations are as follows:

Phase 1b/2 STOMP Study Data Presentations

Title: Selinexor in combination with weekly low dose bortezomib and dexamethasone (SVd) induces a high response rate with durable responses in patients with refractory multiple myeloma (MM)
Presenter: Nizar Bahlis, Southern Alberta Cancer Research Institute, Calgary, Alberta
Abstract Number/Publication ID: 3135
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster II
Date and Time: Sunday, December 10, 2017; 6:00-8:00 PM ET
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

Title: Selinexor in Combination with Pomalidomide and Low Dose Dexamethasone in a Relapsed / Refractory Multiple Myeloma Patient Population with Prior Proteasome Inhibitor and Lenalidomide Exposure
Presenter: Christine Chen, Princess Margaret Cancer Center, Toronto, Ontario
Abstract Number/Publication ID: 3136
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster II
Date and Time: Sunday, December 10, 2017; 6:00-8:00 PM ET
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

Title: A Phase Ib/II Trial of Selinexor Combined with Lenalidomide and Low Dose Dexamethasone in Patients with Relapsed / Refractory Multiple Myeloma
Presenter: Darrell White, Dalhousie University and QEII Health Sciences Center, Halifax; Nova Scotia
Abstract Number/Publication ID: 1861
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster I
Date and Time: Saturday, December 9, 2017; 5:30-7:30 PM ET
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

Title: A Phase 1b Study to Assess the Combination of Selinexor and Daratumumab in Patients with Relapsed/Refractory Multiple Myeloma Previously Exposed to Proteasome Inhibitors (PI) and Immunomodulatory Drugs (IMiDs)
Presenter: Cristina Gasparetto, Duke University Cancer Center, Durham, North Carolina
Abstract Number/Publication ID: 3100
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster II
Date and Time: Sunday, December 10, 2017; 6:00-8:00 PM ET
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

Investigator-sponsored Study Oral Presentations

Title: Selinexor in Combination with Cladribine, Cytarabine and G-CSF for Relapsed or Refractory AML
Presenter: Geoffrey Uy, Washington University School of Medicine in St. Louis
Abstract Number/Publication ID: 816
Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Novel Targeted and Immune-based Approaches in the Treatment of AML; Monday, December 11, 2017 from 4:30-6:00 PM ET
Date and Time: Monday, December 11, 2017 at 5:45 PM ET
Location: Georgia World Congress Center, Building B, Level 5, Murphy BR 1-2

Investigator-sponsored Study Poster Presentations

Title: Selinexor maintenance is feasible and tolerable after allogeneic stem cell transplant (allo-SCT) for patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)
Presenter: Hongtao Liu, University of Chicago Medical Center
Abstract Number/Publication ID: 3312
Session: 732. Clinical Allogeneic Transplantation: Results: Poster II
Date and Time: Sunday, December 10, 2017 from 6:00-8:00 PM ET
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

Title: A Phase I/II study of Selinexor (SEL) with Sorafenib in Patients (pts) with Relapsed and/or Refractory (R/R) FLT3 mutated Acute Myeloid Leukemia (AML)
Presenter: Naval Daver, University of Texas MD Anderson Cancer Center
Abstract Number/Publication ID: 1344
Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster I
Date and Time: Saturday, December 9, 2017 from 5:30-7:30 PM ET
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

Title: Phase I/II Study of Liposomal Doxorubicin (DOX) in Combination with Selinexor (SEL) and Dexamethasone (Dex) for Relapsed and Refractory Multiple Myeloma (RRMM)
Presenter: Rachid Baz, H. Lee Moffitt Cancer Center and Research Institute
Abstract Number/Publication ID: 3095
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster II
Date and Time: Sunday, December 10, 2017 from 6:00-8:00 PM ET
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

Preclinical Oral Presentations

Title: Non-cytotoxic Low Doses of Selinexor Promote the Differentiation of AML Cells Harboring Mutant-NPM1 into Monocytes
Presenter: Saunthararajah Yogen, Cleveland Clinic
Abstract Number/Publication ID: 105742
Session: 603. Oncogenes and Tumor Suppressors: Nuclear Export and Metabolic Regulation; Monday, December 11, 2017, 6:15-7:45 PM ET
Date and Time: Monday, December 11, 2017 at 6:30 PM ET
Location: Georgia World Congress Center, Building C, Level 1, C101 Auditorium

Title: PAK4 Inhibition Impacts Growth and Survival, and Increases Sensitivity to DNA-Damaging Agents in Waldenstrom Macroglobulinemia
Presenter: Li Na, Dana Farber Cancer Institute
Abstract Number/Publication ID: 102879
Session: 622. Lymphoma Biology—Non-Genetic Studies: Novel Mechanisms Implicated in Lymphoma Biology; Monday, December 11, 2017, 10:30AM — 12:00 PM ET
Date and Time: Monday, December 11, 2017 at 11:45 AM ET
Location: Georgia World Congress Center, Building C, Level 1, C101 Auditorium

Preclinical Poster Presentations

Title: XPO1 Inhibitor Selinexor Overcomes Ibrutinib Resistance in Mantle Cell Lymphoma (MCL) via Nuclear Retention of IκB
Presenter: Mei Ming, University of Chicago
Abstract Number/Publication ID: 104320
Session: 605. Molecular Pharmacology, Drug Resistance-Lymphoid and Other Diseases
Date and Time: Monday, December 11, 2017; 6:00-8:00 PM ET
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

Title: XPO1 Inhibition Synergizes with BCR Inhibition, Blocks Tumor Growth and Prolongs Survival in a Bioluminescent Animal Model of Primary Central Nervous System Lymphoma
Presenter: Marta Crespo, Hall d’Hebron, Barcelona
Abstract Number/Publication ID: 107008
Session: 625. Lymphoma: Pre-Clinical-Chemotherapy and Biologic Agents
Date and Time: Sunday, December 10, 2017; 6:00-8:00 PM ET
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

Title: Eltanexor (KPT-8602), a Second-Generation Selective Inhibitor of Nuclear Export (SINE) Compound, in Patients with Refractory Multiple Myeloma
Presenter: Robert Frank Cornell, Vanderbilt University Medical Center
Abstract Number/Publication ID: 107422
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster II
Date and Time: Sunday, December 10, 2017; 6:00-8:00 PM ET
Location: Georgia World Congress Center, Building A, Level 1, Hall A2

About Selinexor

Selinexor (KPT-330) is a first-in-class, oral Selective Inhibitor of Nuclear Export / SINE compound. Selinexor functions by binding with and inhibiting the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells. To date, over 2,200 patients have been treated with selinexor, and it is currently being evaluated in several mid- and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in a pivotal, randomized Phase 3 study in combination with Velcade (bortezomib) and low-dose dexamethasone (BOSTON), in combination with low-dose dexamethasone (STORM) and backbone therapies (STOMP), and in diffuse large B-cell lymphoma (SADAL), and liposarcoma (SEAL), among others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with one or more approved therapies in a variety of tumor types to further inform Karyopharm’s clinical development priorities for selinexor. Additional clinical trial information for selinexor is available at www.clinicaltrials.gov.

On November 1, 2017 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that new data on its approved and investigational medicines for blood diseases will be presented at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting from 9 – 12 December, in Atlanta (Press release, Hoffmann-La Roche, NOV 1, 2017, View Source [SID1234521400]). Ten Roche medicines will be featured in over 75 abstracts, including 26 oral presentations, across eight blood diseases.

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“At ASH (Free ASH Whitepaper) this year, we look forward to presenting a wealth of data highlighting potential advances across the spectrum of blood diseases, from rare conditions like haemophilia A to common blood cancers like lymphoma,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “Our ongoing development programme in haematology is one of the largest in this area, underscoring our commitment to developing practice-changing medicines and improving outcomes for people with diseases of the blood.”

Among Roche’s clinical data to be featured at ASH (Free ASH Whitepaper) are results from the ongoing trials for the investigational medicine emicizumab. Updated data with an additional six months of follow-up from the phase III HAVEN 1 and HAVEN 2 studies evaluating the safety and efficacy of emicizumab in adults, adolescents and children with haemophilia A with inhibitors will be presented. The HAVEN 2 study will be highlighted as part of ASH (Free ASH Whitepaper)’s official press program on 9 December at 07:30am EST. Additional results from the emicizumab clinical development programme will be presented during the meeting, including preliminary data from the phase III HAVEN 4 study exploring emicizumab prophylaxis administered every four weeks in people with haemophilia A with and without inhibitors, as well as real-world data from a non-interventional study in children under 12 years of age with haemophilia A with inhibitors.

Roche will also be sharing data for medicines in late-stage development for a range of blood cancers. Highlights include results from a randomised phase II study evaluating polatuzumab vedotin, an investigational anti-CD79b antibody drug conjugate, in combination with MabThera/Rituxan (rituximab) and bendamustine versus MabThera/Rituxan and bendamustine for the treatment of people with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). Based on data from this study, polatuzumab vedotin was recently granted Breakthrough Therapy Designation by the US Food and Drug Administration (FDA) and had previously received the PRIME (PRIority MEdicines) designation in Europe.

Additionally, results from studies of Gazyva/Gazyvaro (obinutuzumab), including new data from the
phase III GALLIUM study in previously untreated follicular lymphoma, and data from the phase III PrefMab study evaluating patient preference for the subcutaneous (SC) formulation of MabThera/Rituxan Hycela (rituximab/rituximab and hyaluronidase human) as a treatment for DLBCL and follicular lymphoma will also be shared. Finally, results from multiple studies assessing the safety and efficacy of Venclexta/Venclyxto (venetoclax) across chronic lymphocytic leukaemia (CLL), multiple myeloma (MM) and acute myeloid leukaemia (AML) will be presented. Venclexta/Venclyxto is being developed by AbbVie and Roche.

Key abstracts featuring Roche medicines that will be presented at ASH (Free ASH Whitepaper) can be found in the table below.

Follow Roche on Twitter via @Roche and keep up to date with ASH (Free ASH Whitepaper) Annual Meeting news and updates by using the hashtag #ASH17.

On November 1, 2017 bluebird bio, Inc. (Nasdaq:BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, reported that four oral and seven poster presentations will feature data from bluebird programs during the 59th Annual Meeting of the American Society for Hematology (ASH) (Free ASH Whitepaper) (Press release, bluebird bio, NOV 1, 2017, View Source [SID1234521399]). The data will highlight bluebird’s advancement of its LentiGlobin product candidate in patients with transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD), and its bb2121 product candidate in patients with relapsed/refractory multiple myeloma. Preliminary data from these abstracts will be available on the ASH (Free ASH Whitepaper) conference website at 9:00 am ET today.

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"This year at ASH (Free ASH Whitepaper), we have the opportunity to share updated data across our clinical studies in severe genetic diseases and cancer, and to provide a look at some of the preclinical work that will inform the next phase of clinical development at bluebird," said Dave Davidson, chief medical officer. "The new data in sickle cell disease suggest that the changes made to the HGB-206 protocol and to our manufacturing process are having a favorable impact on the engraftment of the gene-modified stem cells. The two patients treated in Group B have consistently higher DP VCN and in vivo VCN than Group A patients, and patient 1313 has the highest Month 3 HbAT87Q level seen to date in the study. We plan to share updated clinical data on these patients at ASH (Free ASH Whitepaper). Additionally, we are very encouraged by the emerging profile of plerixafor mobilization in patients with sickle cell disease. Early data show a more favorable safety profile and substantial improvement in the collection of CD34+ cells compared to bone marrow harvest, suggesting that plerixafor may offer a more effective and less burdensome means to collect stem cells in patients with sickle cell disease."

Clinical Presentations Summary

Interim Results from a Phase 1/2 Clinical Study of LentiGlobin Gene Therapy for Severe Sickle Cell Disease (Oral Abstract #527)

Presenter: Julie Kanter, M.D., Medical University of South Carolina, Charleston, SC

Date & Time: Sunday, December 10 at 5:30 pm

Location: Bldg C, Lvl 1, C101 Auditorium

Abstract Results as of July 21, 2017:

9 patients with severe SCD have received LentiGlobin drug product (DP). All successfully underwent bone marrow harvest (median 2 harvests, range 1-3) to collect the stem cells used to produce LentiGlobin drug product.
Patients in this study are divided into three cohorts: A, B and C. Patients in Group A were treated under the original study protocol. Patients in Group B were treated under an amended study protocol that included changes intended to address drug product vector copy number (VCN) and engraftment challenges seen in Group A. Patients in both Group A and B had drug product made from stem cells collected using bone marrow harvest. Group C will be composed of patients treated under the amended study protocol and with drug product made from stem cells collected using apheresis with plerixafor rather than via bone marrow harvest.
7 patients were treated in Group A; initial results in these patients were presented at ASH (Free ASH Whitepaper) 2016. The median cell dose was 2.1 (1.8-5.1) x 106 CD34+ cells/kg, median DP VCN was 0.6 (0.3-1.3) copies/diploid genome, and 8%-42% CD34+ cells were transduced. As of the data cutoff:
Median follow-up was 18.3 (14.9-23.8) months since LentiGlobin infusion
Median VCN in peripheral blood was 0.1 (0.1-0.2) copies/genome and median HbAT87Qlevel was 0.9 (0.4-2.4) g/dL at last measurement.
Patients experiencing multiple vaso-occlusive crises (VOCs) prior to study entry (n=6; median annualized frequency 4, range 2-28 VOCs annually) have had numerically fewer VOCs since LentiGlobin DP infusion (median annualized frequency 1, range 0-24 annually, 14%-100% reduction).
2 patients were treated in Group B. As of the data cutoff:
Patient 1313, whose DP VCNs were reported at ASH (Free ASH Whitepaper) 2016, was treated with two DP lots, one of which was manufactured using the original process, and the other using the refined process.
The total DP cell dose was 2.2 x 106 CD34+ cells/kg. DP VCNs were 1.4 (old process) and 3.3 (refined process) copies/genome. 46% (old process) and 83% (refined process) of CD34+ cells were transduced.
VCN in peripheral blood was 0.5 copies/genome and HbAT87Q level was 1.5 g/dL at 3 months after LentiGlobin infusion
Patient 1312 was treated with two DP lots manufactured using the refined process.
The total DP cell dose was 3.2 x 106 CD34+ cells/kg. DP VCNs were 5.0 and 2.9 copies/genome. 95% and 90% of CD34+ cells were transduced.
VCN in peripheral blood was 2.6 copies/genome at 1 month after LentiGlobin infusion.
HbAT87Q was not yet available at time of data cut for abstract
Additional patients have been enrolled in HGB-206, and data including mobilization results and DP characteristics for these patients will also be presented at ASH (Free ASH Whitepaper).
The toxicity profile observed from start of conditioning to latest follow-up was consistent with myeloablative conditioning with single-agent busulfan.
Successful Plerixafor-Mediated Mobilization, Apheresis, and Lentiviral Vector Transduction of Hematopoietic Stem Cells in Patients with Severe Sickle Cell Disease (Poster Abstract #990)

Presenter:John Tisdale, M.D., National Heart, Lung and Blood Institute (NHLBI), Bethesda, MD

Date & Time:Saturday, December 9 at 5:30 pm

Location: Bldg A, Lvl 1, Hall A2

Abstract Results:

Three patients with severe sickle cell disease (SCD) have undergone plerixafor mobilization.
A total of 15.3, 5.6, and 9.0 x 106 CD34+ cells/kg were collected in a single day of apheresis. In contrast, bone marrow harvest collections for all prior patients in the study yielded a mean of 5.0 (range 0.3—10.8) x 106 CD34+ cells/kg per harvest (N=21).
Ex vivo cultured CD34+ cells isolated from bone marrow harvests consisted of an average of 41.0% (17.3%—50.7%) CD34dim cells. In contrast, ex vivo cultured CD34+ cells isolated from plerixafor mobilized peripheral blood (PB) contained an average of 8.2% (1.5—19.5%) CD34dimcells. CD34dim cells, which express low levels of CD34, are generally less primitive/less likely to be true primitive stem cells than other CD34+ positive cells.
Similar drug product vector copy numbers were observed after research-scale transduction of CD34+ cells collected from a bone marrow harvest and from plerixafor mobilized cells from the same patient.
The mobilization and apheresis procedures had an acceptable toxicity profile. No dose-limiting toxicities were observed after plerixafor dosing.

Clinical Outcomes up to 3 Years Following LentiGlobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar HGB-204 Study (Oral Abstract #360)

Presenter:Janet Kwiatowski, M.D., MSCE, Children’s Hospital of Philadelphia, Philadelphia, PA

Date & Time:Sunday, December 10 at 10:45 am

Location: Building B, Lvl 2, B213-B214

Abstract Results, as of June 2, 2017:

As previously reported, the study has completed its treatment phase and eighteen patients with TDT (8 with β0/β0 and 10 with non-β0/β0 genotypes) received LentiGlobin drug product (DP).
The median drug product vector copy number (VCN) was 0.7 (range: 0.3-1.5) copies/diploid genome, the median cell dose was 8.1 (range: 5.2-18.1) x 106 CD34+ cells/kg, and the proportion of transduced CD34+ cells was 17-58%.
Of the 10 patients with non- β0/β0 genotypes, 8 have been free of transfusions for a median of 27.1 (range 12.5-35.2) months.
The 2 patients with non-β0/β0 genotypes who still require intermittent transfusions had annual transfusion volumes reduced by 30% and 94%; both received DP with a VCN in the lower range (DP VCNs: 0.3 and 0.4 copies/diploid genome).
Two patients with β0/β0 genotypes have not received a transfusion in more than a year. At the patients’ last study visit (Month 24/Month 12), total Hb levels were 9.0 and 10.2 g/dL, HbAT87Qlevels were 8.2 and 6.8 g/dL, and peripheral VCNs were 0.9 and 0.6, respectively.
Six patients with β0/β0 genotypes have continued transfusions. Their annual transfusion volumes have decreased by a median of 63% (range: 19% to 81%).
The safety profile continues to be consistent with myeloablative conditioning with single-agent busulfan. No drug-product related serious adverse events (AEs) have been observed, and there is no evidence of clonal dominance.
Additional Clinical Presentations

Results from the HGB-207 (Northstar-2) Trial: A Phase 3 Study to Evaluate Safety and Efficacy of LentiGlobin Gene Therapy for Transfusion-Dependent β-thalassemia (TDT) in Patients with non-β0/β0Genotypes (Oral Abstract #526)

Presenter: Mark C. Walters, M.D., UCSF Benioff Children’s Hospital, Oakland, Calif

Date & Time:Sunday, December 10 at 5:15 pm

Location: Bldg C, Lvl 1, C101 Auditorium

Abstract contains data presented at European Hematology Association (EHA) (Free EHA Whitepaper) 2017 annual meeting. Updated data to be included in ASH (Free ASH Whitepaper) presentation.

Longer Term Follow-up on the First Patients with Severe Hemoglobinopathies Treated with LentiGlobin Gene Therapy (Poster Abstract #4609)

Presenter: Marina Cavazzana, M.D., Necker-Enfants Malades Hospital, Paris, France

Date & Time:Monday, December 11 at 6:00 pm

Location: Bldg A, Lvl 1, Hall A2

Abstract contains data presented at European Hematology Association (EHA) (Free EHA Whitepaper) 2017 annual meeting. Updated data to be included in ASH (Free ASH Whitepaper) presentation.

Durable clinical responses in heavily pretreated patients with relapsed/refractory multiple myeloma: Updated results from a multicenter study of bb2121 anti-BCMA CAR T cell therapy (Oral Abstract #740)

Presenter: Jesus Berdeja, M.D., Sarah Cannon Research Institute and Tennessee Oncology, Nashville, TN

Date & Time:Monday, December 11 at 3:00 pm

Location: Bldg C, Lvl 1, Hall C1

Abstract contains data presented at American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2017 annual meeting. Updated data to be included in ASH (Free ASH Whitepaper) presentation.

Preclinical Presentations

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown γ-Globin Induced and Simultaneous Repression of β-Globin for the Potential Treatment of Sickle Cell Disease (Poster Abstract #3557)

Presenter:Olivier Negre, Ph.D., bluebird bio

Date & Time:Monday, December 11 at 6:00 pm

Location: Bldg A, Lvl 1, Hall A2

A novel TGF-β/interleukin receptor signal conversion platform that protects CAR/TCR T cells from TGF-β-mediated immune suppression and induces T cell supportive signaling networks (Poster Abstract #1911)

Presenter: Benjamin Boyerinas, Ph.D., bluebird bio

Date & Time:Saturday, December 9 at 5:30 pm

Location: Bldg A, Lvl 1, Hall A2

A Drug-Regulated CAR Platform (DARIC) Induces Effective and Reversible Tumor Control In Vivo Using Non-Immunosuppressive Rapamycin Dosing (Poster Abstract #1910)

Presenter:Unja Martin, Ph.D., bluebird bio

Date & Time:Saturday, December 9 at 5:30 pm

Location: Bldg A, Lvl 1, Hall A2

Gene Editing of TRAC Locus Utilizing megaTAL Nucleases Increases Expression of Transgenic TCRs Delivered via Lentiviral Vector-Mediated Gene Transfer (Poster Abstract #1906)

Presenter: Michael Magee, Ph.D., bluebird bio

Date & Time:Saturday, December 9 at 5:30 pm

Location: Bldg A, Lvl 1, Hall A2

ROR1-directed chimeric antigen receptor T cell recognition of self-antigen is associated with acute toxicity, T cell dysfunction, and poor tumor control (Poster Abstract #4450)

Presenter:James Rottman, Ph.D., bluebird bio

Date & Time:Monday, December 11 at 6:00 pm

Location: Bldg A, Lvl 1, Hall A2

Webcast Information

bluebird bio will host a live webcast at 8:30 a.m. ET on Wednesday, November 1, 2017. The live webcast can be accessed under "Calendar of Events" in the Investors and Media section of the company’s website at www.bluebirdbio.com. Alternatively, investors may listen to the call by dialing (844) 825-4408 from locations in the United States or (315) 625-3227 from outside the United States. Please refer to conference ID number 3795968.

About TDT

Transfusion-dependent β-thalassemia (TDT), also called Cooley’s anemia, is a rare and severe genetic blood disease.

Despite the availability of lifelong supportive care with blood transfusions and chelation treatments, many people with TDT experience serious complications and organ damage due to underlying disease and iron overload.

Allogeneic hematopoietic stem cell transplant (HSCT) is currently the only available option to address the underlying cause of TDT, though it carries significant risks. Complications of allogeneic HSCT include a significant risk of treatment-related mortality, graft failure, graft vs. host disease (GvHD) and opportunistic infections, particularly in patients who undergo non-sibling-matched allogeneic HSCT.

About SCD

Sickle cell disease (SCD) is an inherited disease caused by a mutation in the beta-globin gene that results in sickle-shaped red blood cells. Common complications include anemia, vaso-occlusive crisis, infections, stroke, overall poor quality of life and sometimes, early death.

Where adequate medical care is available, common treatments for patients with SCD largely revolve around prevention of infection and management and prevention of acute sickling episodes. Chronic management may include hydroxyurea pharmacotherapy and, in certain cases, chronic transfusions. Given the limitations of these treatments, there is no effective long-term treatment. The only advanced treatment for SCD is allogeneic HSCT. Complications of allogeneic HSCT include a significant risk of treatment-related mortality, graft failure, GvHD and opportunistic infections, particularly in patients who undergo non-sibling-matched allogeneic HSCT.

On November 1, 2017 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported that four abstracts, including an oral presentation on the Company’s lead product candidate BPX-501, were accepted for presentation at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, Bellicum Pharmaceuticals, NOV 1, 2017, View Source [SID1234521398]).

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In the oral presentation, Pietro Merli of Ospedale Pediatrico Bambino Gesù in Rome, Italy will review data from the ongoing EU BP-004 study, highlighting rapid immune recovery in pediatric patients with blood cancers, immune deficiencies and other non-malignant disorders receiving treatment with BPX-501 following an alpha/beta-depleted haploidentical hematopoietic stem cell transplant. The Company will also present data highlighting preclinical results of its controllable CAR-T technology in three poster presentations, including a study evaluating Bellicum’s dual-switch CAR-T cells targeting CD123. ASH (Free ASH Whitepaper) 2017 is being held in Atlanta, Georgia on December 9-12.

ASH Presentations on Bellicum Programs

BPX-501:

Oral Presentation: “Donor T Cells Genetically Modified with a Novel Suicide Gene (Inducible Caspase 9, iC9) Expand and Persist Over Time After Post-Allograft Infusion in Patients Given αβ T-Cell and B-Cell Depleted HLA-Haploidentical Allogeneic Stem Cell Transplantation (αβ Haplo-HSCT) Contributing to Accelerate Immune Recovery”
Abstract Number: 211
Session Name: 722. Clinical Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution: Prevention
Session Date: Saturday, December 9, 2017
Session Time: 2:00 p.m. – 3:30 p.m. EST
Presentation Time: 2:00 p.m. EST

Controllable CAR-T Technology:

Poster Presentation: “Dual-Switch CAR-T Cells: Orthogonal Molecular Switches to Control Activation and Elimination of CAR-T Cells to Target CD123+ Cancer”
Abstract Number: 3184
Session Name: 703. Adoptive Immunotherapy: Poster II
Date: Sunday, December 10, 2017
Presentation Time: 6:00 p.m. – 8:00 p.m. EST

Poster Presentation: “Inducible MyD88/CD40 (iMC) Costimulation Enhances Polyclonal Epstein-Barr Virus-Specific Cytotoxic T Lymphocyte (EBV-CTL) Proliferation and Anti-Tumor Activity”
Abstract Number: 3337
Session Name: 801. Gene Therapy and Transfer: Poster II
Date: Sunday, December 10, 2017
Presentation Time: 6:00 p.m. – 8:00 p.m. EST

Poster Presentation: “MyD88/CD40 Enhanced CD19-Specific CAR-T Cells Maintain Therapeutic Efficacy Following Resolution of Cytokine-Related Toxicity Using Inducible Caspase-9”
Abstract Number: 4615
Session Name: 801. Gene Therapy and Transfer: Poster III
Date: Monday, December 11, 2017
Presentation Time: 6:00 p.m. – 8:00 p.m. EST

On November 1, 2017 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for cancer, respiratory and other diseases, reported that members of the management team will present at the following upcoming investor conferences (Press release, Pieris Pharmaceuticals, NOV 1, 2017, View Source [SID1234521395]):

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Stifel Healthcare Conference, Tuesday, November 14, 10:15 am EST at Lotte New York Palace Hotel, New York City. A webcast of the Company’s presentation will be available at this link.

Jefferies London Healthcare Conference, Thursday, November 16, 8:40 am GMT at the Waldorf Hilton, Aldwych, London.

Evercore ISI Biopharma Catalyst / Deep Dive Conference, Wednesday, November 29, 1:15 pm EST at the Boston Harbor Hotel, Boston.