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 business highlights and financial results for the third quarter ended September 30, 2017 (Press release, bluebird bio, NOV 1, 2017, View Source [SID1234521381]).

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"2017 has been a year focused on execution. In January, we outlined a set of goals intended to bring us closer to our 2022 vision of becoming the gene therapy products company," said Nick Leschly, chief bluebird. "I’m pleased to say that we have accomplished many of these goals. We presented compelling data at ASCO (Free ASCO Whitepaper), EHA (Free EHA Whitepaper) and in the New England Journal of Medicineacross all of our clinical programs, treated our first patient in the expansion cohort of our bb2121 Phase 1 multiple myeloma study, and moved our second generation anti-BCMA CAR T program, bb21217, into the clinic. Coming into ASH (Free ASH Whitepaper), we have an early, but promising, indication that the changes we made in the HGB-206 study have improved engraftment and that mobilization and collection of stem cells using plerixafor may be a safe and viable option for patients with severe sickle cell disease. At ASH (Free ASH Whitepaper), we look forward to sharing additional data across our clinical programs as well as preclinical data that supports our future pipeline."

Recent Highlights

ASH PRESENTATIONS – Today, bluebird bio announced that the company will present clinical and pre-clinical data in 11 abstracts spanning the company’s research and development portfolio. Included among the presentations will be updated clinical data for the company’s clinical studies of LentiGlobin: Northstar (HGB-204) in patients with transfusion-dependent β-thalassemia (TDT), HGB-205 in patients with TDT or severe sickle cell disease (SCD), Northstar-2 (HGB-207) in patients with TDT and non-β0/β0 genotypes, and HGB-206 in patients with SCD. Updated data from the CRB-401 study of bb2121 anti-BCMA CAR T in patients with relapsed/refractory multiple myeloma will also be presented. The company will also present several preclinical and research posters.

HGB-206 UPDATED CLINICAL DATA – In the ASH (Free ASH Whitepaper) abstracts announced today, early results from 2 patients treated in the HGB-206 study using a modified protocol with LentiGlobin drug product (DP) manufactured under a refined manufacturing process demonstrate both higher DP vector copy number (VCN) and higher peripheral VCN after transplant. The toxicity profile observed was consistent with myeloablative conditioning with single-agent busulfan.

HGB-206 PLERIXAFOR SAFETY DATA – Safety data exploring the use of plerixafor mobilization in 3 patients showed an acceptable safety profile and a larger cell dose yield is highlighted in an ASH (Free ASH Whitepaper) abstract. HGB-206 continues to enroll, and patients will be treated under the amended study protocol with DP made from cells obtained through apheresis following plerixafor mobilization.

NORTHSTAR (HGB-204) UPDATED CLINICAL DATA – ASH (Free ASH Whitepaper) abstracts include updated results from the Phase 1/2 Northstar (HGB-204) study in patients with TDT using the original manufacturing process. The updated efficacy outcomes suggested that treatment with LentiGlobin drug product can potentially have a durable effect on eliminating or substantially reducing blood transfusions. Data also indicated that less favorable outcomes were seen in patients who had a low vector copy number (VCN). The safety profile continues to be consistent with autologous transplantation. No drug-product related adverse events (AEs) have been observed, and there is no evidence of clonal dominance.

RMAT FOR LENTIGLOBIN IN SCD – In October, the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy Designation for LentiGlobin for the treatment of patients with severe SCD. Under this designation, the FDA will work closely with bluebird bio to provide guidance on the future development of LentiGlobin, including providing advice on generating the evidence needed to support potential approval of the product candidate.

INTERIM LENTI-D DATA IN NEJM – In October, bluebird bio announced the publication in theNew England Journal of Medicineof interim clinical data on the initial 17 patients treated in the Starbeam study of Lenti-D drug product in cerebral adrenoleukodystrophy (CALD). These data were also presented at the Child Neurology Society (CNS) Annual Meeting. As of August 25, 2017, 15/17 patients (88%) in the initial study cohort remained free of major functional disabilities (MFDs) at 24 months, the primary endpoint of the trial. The safety profile of Lenti-D was consistent with myeloablative conditioning. No patients treated with Lenti-D had graft versus host disease (GvHD), and there was no graft rejection or clonal dominance. An expansion cohort of the Starbeam study is enrolling additional patients to enable the manufacture of Lenti-D in Europe and subsequent treatment of subjects in Europe. Findings from this study will, and to add to the overall clinical data package for potential future regulatory filings in the United States and Europe.

FIRST PATIENT TREATED IN BB21217 STUDY – In September, bluebird bio announced that the first patient was treated in CRB-402, the company’s Phase 1 study of bb21217 in patients with relapsed/refractory multiple myeloma. bb21217 is an anti-BCMA CAR T product candidate manufactured in the presence of a PI3 kinase inhibitor, designed to enrich for a more potent, longer-living T cell subtype that in preclinical in vivo studies showed improved anti-tumor activity. Subsequent to study initiation, in September Celgene exercised its option to exclusively license bb21217, resulting in a $15 million option exercise payment from Celgene. Also in September, the U.S. Food and Drug Administration (FDA) granted orphan drug designation for bb21217.

CRB-401 ADVANCES TO EXPANSION COHORT – In September, bluebird bio announced that the first patient was treated in the expansion cohort of CRB-401, the company’s Phase 1 study of bb2121 anti-BCMA CAR T therapy in patients with relapsed/refractory multiple myeloma. Patients in the expansion cohort will be treated at a dose range of 150 to 450 x 106 CAR+ T cells and will be required to have prior exposure to a proteasome inhibitor, an immunomodulatory agent and daratumumab.

NEW BOARD APPOINTMENT – In September, Mary Lynne Hedley, Ph.D., was appointed to the Board of Directors. bluebird bio also announced that with Dr. Hedley’s appointment, John Maraganore, Ph.D., transitioned off the Board of Directors.
Third Quarter 2017 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2017 were $1.1 billion, compared to $884.8 million as of December 31, 2016, an increase of $257.8 million.
Revenues: Total revenue was $7.7 million for the third quarter of 2017 compared to $1.6 million for third quarter of 2016. The increase is attributable to the commencement of revenue recognition for the bb2121 license and manufacturing services under the company’s agreement with Celgene and revenue recognized under bluebird bio’s out-licensing agreement with Novartis Pharma AG (Novartis). In August, Novartis received FDA approval of KYMRIAH and as a result, the company expects to recognize royalty revenue beginning in the fourth quarter of 2017.
R&D Expenses: Research and development expenses were $61.5 million for the third quarter of 2017, compared to $64.0 million for the third quarter of 2016. The decrease in research and development expenses was attributable to decreased platform related expenses as a result of a one-time $15.0 million upfront license payment expensed in the third quarter of 2016, partially offset by increased employee-related costs due to increased headcount to support overall growth, increased clinical trial costs, and increased facility related costs.
G&A Expenses: General and administrative expenses were $23.0 million for the third quarter of 2017, compared to $14.6 million for the third quarter of 2016. The increase in general and administrative expenses was attributable to increased employee-related costs due to increased headcount to support overall growth, increased commercial-related costs attributable to market research costs, increased facility-related expenses, and increased professional and consulting fees.
Cost of License Revenue: Cost of license revenue was $1.1 million for the third quarter of 2017. Cost of license revenue is composed of amounts payable to third party licensors in connection with amounts received under our out-license arrangement with Novartis.
Net Loss: Net loss was $78.8 million for the third quarter of 2017 compared to $77.0 million for the third quarter of 2016.
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.

On November 1, 2017 vTv Therapeutics Inc. (vTv Therapeutics) (Nasdaq:VTVT) reported a corporate update and reported financial and operational results for the third quarter ended September 30, 2017 (Press release, vTv Therapeutics, NOV 1, 2017, View Source [SID1234521445]).

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“We’ve made significant progress in the third quarter of 2017 across our strategic initiatives, and we attribute that to our ongoing development of innovative therapies, particularly in the areas of Alzheimer’s and diabetes,” said Steve Holcombe, president and CEO of vTv Therapeutics. “We look forward to building on this momentum as we approach several important milestones next year, including the Part A readout out of our Phase 3 STEADFAST Study of azeliragon in patients with mild Alzheimer’s disease.”

Third Quarter 2017 Highlights

Issuance of U.S. Patent Covering Methods of Treatment Using Azeliragon

The U.S. Patent and Trademark Office issued patent number 9,717,710 (‘710 Patent) with claims protecting methods of treatment using azeliragon, vTv Therapeutics’ oral antagonist of the Receptor for Advanced Glycation Endproducts (RAGE) for treatment of mild Alzheimer’s disease. The ‘710 Patent, expiring in October 2034, includes claims covering methods of treating patients with mild Alzheimer’s disease by administering about 5 mg per day of azeliragon.
2017 Alzheimer’s Association International Conference

In two poster presentations titled, “Assessment of Azeliragon QTc Liability through Integrated, Model-Based Concentration QTc Analysis” and “Effect of Food on the Pharmacokinetics of Azeliragon in Healthy Adult Subjects,” vTv Therapeutics’ researchers reviewed study data that indicated no deleterious effect of azeliragon on QT at therapeutic and supra-therapeutic doses and that azeliragon may be given without regard to meals.
vTv Therapeutics and JDRF Enter Into Industry Partnership

JDRF, the leading global organization funding type 1 diabetes (T1D) research, committed $3 million in funding, matched by vTv Therapeutics, to support a Phase 2 Proof of Concept study to explore the effect of vTv Therapeutics’ liver-selective glucokinase activator TTP399 as an oral drug for the treatment of T1D. The study will evaluate whether TTP399 is well tolerated when administered as an add-on to insulin therapy for people with T1D and whether TTP399 has the potential to significantly improve daily glucose profiles and HbA1c.
53rd European Association for the Study of Diabetes Annual Meeting

In a poster presentation titled “Beyond topline results for the oral (non-peptide) GLP-1R agonist TTP273 in type 2 diabetes: How much and when?” vTv Therapeutics’ researchers reviewed results from a concentration/effect analysis on the LOGRA study showing that lower doses of TTP273 may show more pronounced effects for key efficacy endpoints, including a reduction in HbA1c, weight, and fasting plasma glucose.
Upcoming Anticipated Milestones

STEADFAST Study (azeliragon in patients with mild Alzheimer’s disease): Expected to report top-line results from each of the Part A and Part B studies in early 2018 and late 2018, respectively.

Third Quarter 2017 Financial Results

Cash Position: Cash and cash equivalents as of September 30, 2017 were $20.5 million compared to $32.5 million as of June 30, 2017.
R&D Expenses: Research and development expenses were $9.0 million in the third quarter of 2017, compared to $9.6 million in the second quarter of 2017. The decrease in research and development expenses was primarily driven by decreases in spending for the STEADFAST Study due to the relative costs of patient visits.
G&A Expenses: General and administrative expenses were $2.6 million and $3.0 million, for the third and second quarters of 2017, respectively. The decrease in general and administrative costs was primarily due to the reduction in professional service fees between the periods.
Net Loss Before Non-Controlling Interest: Net loss before non-controlling interest was $12.4 million for the third quarter of 2017 compared to net loss before non-controlling interest of $13.4 million for the second quarter of 2017.
Net Loss per Share: GAAP net loss per share was $0.38 and $0.41 for the three months ended September 30, 2017 and June 30, 2017, respectively, based on weighted-average shares of 9.7 million in each period. Non-GAAP net loss per fully exchanged share was $0.38 and $0.41 for the three months ended September 30, 2017 and June 30, 2017, respectively, based on non-GAAP fully exchanged weighted-average shares of 32.8 million in each period.

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 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 [SID1234521436]). 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.

vTv Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, vTv Therapeutics, 2017, NOV 1, 2017, View Source [SID1234521415]).

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