bluebird bio to Present Immuno-Oncology and Gene Therapy Data at the ASGCT 19th Annual Meeting

On April 18, 2016 bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases and T cell-based immunotherapies for cancer, reported that data from clinical, preclinical, and research and manufacturing programs will be highlighted in ten presentations at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 19th Annual Meeting, taking place May 4-7, 2016 in Washington, D.C (Press release, bluebird bio, APR 18, 2016, View Source;p=RssLanding&cat=news&id=2158031 [SID:1234510988]).

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Two oral presentations given by bluebird’s academic collaborators will highlight previously presented data from bluebird bio’s ongoing gene therapy clinical trials. David Williams, M.D., chief of hematology/oncology at Boston Children’s Hospital will present interim data from the Starbeam Study of Lenti-D in cerebral adrenoleukodystrophy, and Marina Cavazzana, M.D., Ph.D., of Hospital Necker, University Paris Descartes, will present interim data from the HGB-205 study of LentiGlobin in severe sickle cell disease and transfusion-dependent β-thalassemia.

Eight additional presentations will be featured at the meeting, highlighting progress across the company’s preclinical, research and process development activities.

"As bluebird continues to build a differentiated T cell oncology franchise, we are excited to present three oncology abstracts that highlight our work on the next generation of technology for T cell-based immunotherapy – including methods of generating T cells with sustained anti-tumor activity, small-molecule regulated chimeric antigen receptors (CARs) and genome editing to generate improved CAR T cells," said Philip Gregory, D.Phil., chief scientific officer, bluebird bio. "From our hematopoietic stem cell programs, we will also share updates in five presentations covering improvements in scalable manufacturing, transduction efficiency and assay development – critical areas for making gene therapy available to more patients."

The abstracts are now available online on the ASGCT (Free ASGCT Whitepaper) Annual Meeting website.

Details of bluebird bio’s oral presentations are as follows:

Title: A Phase 2/3 Study of the Efficacy and Safety of Ex Vivo Gene Therapy With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy
Abstract Number: 250
Session: Clinical Trials Spotlight Symposium
Date: Thursday, May 5, 2016
Time: 9:00 – 9:20 a.m.
Location: Thurgood Marshall North/East
Note: Data previously presented at the 2016 American Academy of Neurology Annual Meeting

Title: Small Molecule-regulated Antigen Recognition System for Inducible T Cell Targeting of Cancer Cells
Abstract Number: 277
Session: Cancer-Immunotherapy, Cancer Vaccines I
Date: Thursday, May 5, 2016
Time: 5:15 – 5:30 p.m.
Location: Washington 4

Title: Clinical Outcomes of Gene Therapy with BB305 Lentiviral Vector for Sickle Cell Disease and β-Thalassemia
Abstract Number: 279
Session: Hematologic & Immunologic Diseases I
Date: Thursday, May 5, 2016
Time: 4:00 – 4:15 p.m.
Location: Washington 5-6
Note: Data previously presented at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting

Title: Towards the Clinical Application of BCMA CAR T cells: The Importance of Reduced Tonic Signaling and Methods to Enhance Memory T Cells
Abstract Number: 747
Session: Cancer-Immunotherapy, Cancer Vaccines III
Date: Saturday, May 7, 2016
Time: 10:45 – 11:00 a.m.
Location: Thurgood Marshall North

Details of bluebird bio’s poster presentations are as follows:

Title: PGE2 Increases Lentiviral Vector Transduction Efficiency of Human HSC
Abstract Number: 229
Session: Hematologic & Immunologic Diseases I
Date: Wednesday, May 4, 2016
Time: 5:30 p.m. – 7:30 p.m.
Location: Exhibit Hall C & B South

Title: Staurosporine Increases Lentiviral Transduction of Human CD34+ Cells
Abstract Number: 221
Session: Hematologic & Immunologic Diseases I
Date: Wednesday, May 4, 2016
Time: 5:30 p.m. – 7:30 p.m.
Location: Exhibit Hall C & B South

Title: Qualification of a p24 ELISA Assay for Quantitation of Total Lentiviral Vector Concentration
Abstract Number: 473
Session: Pharmacology/Toxicology Studies or Assay Development
Date: Thursday, May 5, 2016
Time: 6:00 p.m. – 8:00 p.m.
Location: Exhibit Hall C & B South

Title: Efficient Generation of CART Cells by Homology Directed Transgene Integration into the TCR-Alpha Locus
Abstract Number: 323
Session: Targeted Genome Editing II
Date: Thursday, May 5, 2016
Time: 6:00 p.m. – 8:00 p.m.
Location: Exhibit Hall C & B South

Title: Development of a Stable Producer Cell Line for Scalable Lentiviral Vector Production for Gene Therapy of Hemoglobinopathies
Abstract Number: 458
Session: Vector and Cell Engineering/Manufacturing I
Date: Thursday, May 5, 2016
Time: 6:00 p.m. – 8:00 p.m.
Location: Exhibit Hall C & B South

Title: Characterization of Nanoparticles in Lentiviral Vector Preparations
Abstract Number: 709
Session: Vector and Cell Engineering/Manufacturing II
Date: Friday, May 6, 2016
Time: 6:00 p.m. – 8:00 p.m.
Location: Exhibit Hall C & B South

Arrowhead Pharmaceuticals Presents Preclinical Data on Renal Cell Carcinoma Program at AACR 2016

On April 18, 2016 Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) reported that they have presented a poster on ARC-HIF2, its preclinical development program targeting HIF2-α for the treatment of renal cell carcinoma (RCC), at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2016 (AACR16), in New Orleans (Press release, Arrowhead Pharmaceuticals, APR 18, 2016, View Source [SID:1234510987]). ARC-HIF2 is Arrowhead’s first RNAi therapeutic program to target tissues outside the liver.

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The poster titled, "Novel HIF-2α targeted RNAi therapeutic for renal cell carcinoma" (abstract 2064), describes data from various stages of development of ARC-HIF2, including RNAi trigger selection, HIF2-α target validation, delivery and targeting ligand validation, and multiple RCC tumor models. These data show that important advancements are being made in this program and for Arrowhead’s Dynamic PolyconjugateTM (DPCTM) delivery platform generally, including the following key findings:

Proof-of-concept ligand dependent, functional delivery was demonstrated using the DPC targeted delivery platform
Silencing HIF2-α expression by RNA interference resulted in reduction of HIF-2α regulated genes
In two different RCC tumor bearing mouse models, ARC-HIF2 inhibited tumor growth and promoted tumor cell death and structural degeneration

Phase I clinical trial of lenalidomide in combination with bevacizumab in patients with advanced cancer.

Lenalidomide and bevacizumab have antitumor activity in various tumor types. We conducted a phase I study of this combination in patients with advanced cancer.
A "3 + 3" study design was used. Lenalidomide 10 or 20 mg (orally, days 1-21) and bevacizumab 5, 7.5, or 10 mg/kg, (intravenously, every 2 weeks) were given at four escalating dose levels, followed by an expansion phase at the highest maximum tolerated dose (MTD) (1 cycle = 4 weeks). Dose-limiting toxicity (DLT), MTD, adverse events, and clinical outcomes were assessed.
Thirty-one patients were enrolled (median age, 60 years; men, 52 %). The most common tumor types were colorectal carcinoma (n = 11) and melanoma (n = 5). Overall, 105 cycles (median, 2) were administered. No DLTs were observed. The maximum tested dose (level 4) was used in the expansion phase. The most common toxicities were fatigue (n = 7, 23 %) and skin rash (n = 4, 13 %). One patient developed a transient ischemic attack (3.2 %); prophylactic anticoagulation became mandatory in the subsequent 17 treated patients. Of 31 patients, 27 were evaluable for response. Stable disease (SD) was noted in 10 (37 %) patients, including five patients with SD for ≥6 months (tumor types: clear cell sarcoma, germ cell tumor, colorectal carcinoma, and melanoma). The median progression-free survival and overall survival were 2.8 and 5.5 months, respectively.
The combination of lenalidomide with bevacizumab in patients with advanced solid tumors was safe. Prolonged stable disease was noted in selected tumor types, warranting further clinical evaluation.

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Agenus Presents Posters on Checkpoint Antibody Product Candidates at the American Association for Cancer Research (AACR) 2016 Annual Meeting

On April 18, 2016 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology company developing checkpoint modulator antibodies and cancer vaccines, reported that three abstracts on the Company’s checkpoint modulator product candidates were accepted for poster presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2016 Annual Meeting (Press release, Agenus, APR 18, 2016, View Source [SID:1234510982]). The conference is taking place in New Orleans from April 16-20, 2016.

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Poster Details:

OX40
Poster Title: INCAGN01949: A Novel Anti-OX40 Agonist Antibody with the Potential to Enhance Tumor Specific T-cell Responsiveness, while Selectively Depleting Intratumoral Regulatory T Cells
Poster Number: #3204
Session Date: Tuesday, April 19, 2016
Session Time: 8:00 AM -12:00 PM CDT

GITR
Poster Title: A Novel Agonist Antibody (INCAGN01876) that Targets the Costimulatory Receptor GITR
Poster Number: #3220
Session Date: Tuesday, April 19, 2016
Session Time: 8:00 AM -12:00 PM CDT

CTLA-4
Poster Title: AGEN1884 and AGEN2041: Two Functionally Distinct Anti-CTLA-4 Antagonist Antibodies
Poster Number: #5005
Session Date: Wednesday, April 20, 2016
Session Time: 7:30 AM -11:00 AM CDT

Agenus is partnered with Incyte Corporation for the development of INCAGN1949 and INCAGN1876, and is partnered with Recepta Biopharma SA for certain South American rights for AGEN1884 and AGEN2041. Abstracts and posters will become available on the Company’s website at View Source following the poster sessions.

TG Therapeutics, Inc. Announces Pre-Clinical Data Presentation on TGR-1202 Demonstrating Differentiated Effects on T-Cells

On April 18, 2016 TG Therapeutics, Inc. (Nasdaq:TGTX) reported the presentation of preclinical data describing the differential regulation of human T-cells by TGR-1202 in a poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2016, which is being held April 16 – 20, 2016 at the Ernest N. Morial Convention Center in New Orleans, Louisiana (Press release, TG Therapeutics, APR 18, 2016, View Source [SID:1234510981]).

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As part of an ongoing research collaboration with the H. Lee Moffitt Cancer Center and Research Institute in Tampa, FL, data was generated comparing TGR-1202 with the PI3K-delta inhibitors idelalisib and duvelisib in in-vitro models assessing the impact on T-cell subsets. Key conclusions from the poster are as follows:

TGR-1202 exhibits dose dependent cytotoxicity against human T cells beginning 25uM, comparable to the PI3K-delta inhibitors idelalisib and duvelisib,
TGR-1202 allows relative conservation of TH2 cytokine expression and GATA-3 mRNA compared to other PI3K-delta inhibitors idelalisib and duvelisib
Regulatory T cell (Treg) population number and function is reduced overall after treatment with all three agents but to a lesser degree in TGR-1202 treated samples
TGR-1202 does not robustly reduce the expression of PD-1 and CTLA-4 on Tregs, suggesting that a suppressive phenotype is maintained to a greater extent compared to idelalisib and duvelisib.
Michael S. Weiss, the Company’s Executive Chairman and Interim Chief Executive Officer, stated, "Many of the adverse events associated with existing PI3K-delta inhibitors such as colitis and hepatic toxicity, and most recently with opportunistic infections, are thought to be related to T-cell immune mediated mechanisms. This pre-clinical data now begins to elucidate key differences in the impact of these inhibitors on human T-cell subsets, which may explain the differentiated safety profile observed with TGR-1202 to date in the clinic. These data represent the first step in improving our understanding and we look forward to continuing our research collaboration with the team at Moffitt."

PRESENTATION DETAILS

A copy of the poster is available on the Company’s website at www.tgtherapeutics.com, located on the Publications Page, within the Pipeline section.