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Pieris Pharmaceuticals to Present at Investor Conferences in November

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.

Novartis presents data at ASH for patients with serious blood disorders like lymphoma, leukemia and sickle cell disease

On November 1, 2017 Novartis reported that it will present new data from across its hematology portfolio at the upcoming 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, Atlanta, December 9-12 (Press release, Novartis, NOV 1, 2017, View Source [SID1234521393]). More than 75 abstracts will be presented, highlighting the robust Novartis development program for serious blood diseases.

“This is an exceptionally productive time in hematology, and the breadth of our Novartis Oncology data and presence at ASH (Free ASH Whitepaper) underscore our commitment to this space,” said Vas Narasimhan, Global Head Drug Development and Chief Medical Officer, Novartis. “Following the launch of Kymriah, the first FDA-approved CAR-T therapy, we are particularly excited about presenting additional data on this new approach to cancer treatment, as well as a new analysis for crizanlizumab, an investigational treatment for patients with sickle cell disease.”

KymriahTM* (tisagenlecleucel) suspension for intravenous infusion is a CD19-directed genetically modified autologous T cell immunotherapy, indicated for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. Additional results evaluating Kymriah in pediatric ALL and in relapsed/refractory diffuse large B-cell lymphoma (DLBCL) will be presented.

Data for Kymriah include results from the primary analysis of the JULIET study in adult patients with relapsed or refractory DLBCL, demonstrating sustained complete response rates based on extended follow up, and efficacy and safety findings from additional treated patients compared to a previously presented interim analysis. Additionally, results of a cost-effectiveness analysis of Kymriah for the treatment of relapsed or refractory ALL in the United States will be presented in an oral presentation.

Primary Analysis of JULIET: A Global, Pivotal, Phase 2 Trial of CTL019 in Adult Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma [Abstract #577; Monday, December 11, 7:00 AM EST]
Cost-Effectiveness Analysis of CTL019 for the Treatment of Pediatric and Young Adult Patients with Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia in the United States [Abstract #609; Monday, December 11, 7:30 AM EST]
Patient-Reported Quality of Life (QoL) Following CTL019 Infusion in Adult Patients with Relapsed/Refractory (r/r) Diffuse Large B-cell Lymphoma (DLBCL) [Abstract #5215; publication only]
Expert Elicitation of Long-Term Survival for Pediatric Acute Lymphoblastic Leukemia Patients Receiving CTL019 in ELIANA Phase II Study [Abstract #3377; Sunday, December 10, 6:00 PM EST]
Outcomes for chimeric antigen receptor T cell (CAR-T) pipeline therapies in other malignant blood cancers will also be shared at ASH (Free ASH Whitepaper):

Updated Safety and Efficacy of B-cell Maturation Antigen (BCMA)-specific Chimeric Antigen Receptor T Cells (CART-BCMA) for Refractory Multiple Myeloma (MM) [Abstract #505; Sunday, December 10, 4:30 PM EST]
Durable Remissions with Humanized CD19-Targeted Chimeric Antigen Receptor (CAR)-Modified T Cells in Children and Young Adults with Relapsed/Refractory Acute Lymphoblastic Leukemia, Including After Prior CAR Therapy [Abstract #1319; Saturday, December 9, 5:30 PM EST]
Data from a post-hoc sub-group analysis of the Phase II SUSTAIN investigational trial of crizanlizumab for time to first on-treatment sickle cell pain crisis will be featured:

Crizanlizumab 5.0 mg/kg Increased the Time to First On-Treatment Sickle Cell Pain Crisis: A Subgroup Analysis of the Phase II SUSTAIN Study [Abstract #613; Monday, December 11, 10:30 AM EST]
A matched comparison of Molecular Recurrence-free Survival (MRecFS) following treatment discontinuation in chronic myeloid leukemia (CML) patients on Tasigna (nilotinib) in ENESTfreedom versus patients on imatinib in the EURO-SKI trials will be presented in addition to updates from ENESTfreedom and ENESTop on Treatment-free Remission (TFR) outcomes:

Molecular Recurrence-Free Survival (MRecFS) Following Imatinib vs Nilotinib in Patients with Chronic Myeloid Leukemia in Chronic Phase (CML-CP): Matched Analysis of Patients in EURO-SKI and ENESTfreedom [Abstract #1601; Saturday, December 9, 5:30 PM EST]
Impact of Treatment Cessation on Overall Disease Outcomes in Patients with Chronic Myeloid Leukemia in Chronic Phase (CML-CP) Attempting Treatment-Free Remission (TFR): Findings from ENESTfreedom and ENESTop [Abstract #1598; Saturday, December 9, 5:30 PM EST]
Treatment-Free Remission (TFR) Among Patients with Chronic Myeloid Leukemia in Chronic Phase (CML-CP) Not Initially Eligible for Treatment Discontinuation Due to Unstable Deep Molecular Response (DMR): ENESTfreedom and ENESTop [Abstract #2878; Sunday, December 10, 6:00 PM EST]
Additionally, new insights will be presented from the pivotal, Phase III RATIFY trial of Rydapt (midostaurin) in adults with FLT3+ acute myeloid leukemia (AML):

An Analysis of the Maintenance and Post Completion Effect of Midostaurin Therapy in the International Prospective Randomized Placebo-Controlled, Double-Blind Trial (CALGB 10603/RATIFY [Alliance]) for Newly Diagnosed Acute Myeloid Leukemia (AML) Patients with FLT3 Mutations [Abstract #145; Saturday, December 9, 12:00 PM EST]
The Addition of Midostaurin to Standard Chemotherapy Decreases Cumulative Incidence of Relapse (CIR) in the International Prospective Randomized, Placebo-Controlled, Double-Blind Trial (CALGB 10603/RATIFY [Alliance]) for Newly Diagnosed Acute Myeloid Leukemia (AML) Patients with FLT3 Mutations [Abstract #2580; Sunday, December 10, 6:00 PM EST]
Prognostic Impact of NPM1/FLT3-ITD Genotypes from Randomized Patients with Acute Myeloid Leukemia (AML) Treated Within the International RATIFY Study [Abstract #467; Sunday, December 10, 5:30 PM EST]
Sandoz, a Novartis division, the pioneer and global leader in biosimilars, will present two studies examining the impact of granulocyte colony-stimulating factor (G-CSF) on patient outcomes, cost savings and expanded access for biosimilars including Zarxio (filgrastim-sndz).

Expanded Access to Obinutuzumab from Cost-Savings Generated by Biosimilar Filgrastim (BIOSIM-FIL) in the Prophylaxis of Chemotherapy-Induced (Febrile) Neutropenia: A Simulation Study [Abstract #3380; Sunday, December 10, 6:00 PM EST]
A Systemic Literature Review of Overall Survival and Delivered Dose Intensity in Cancer Patient Receiving Chemotherapy and G-CSF in Randomized Control Trials [Abstract #3424; Sunday, December 10, 6:00 PM EST]
Additional abstracts of note from the meeting are as follows.

Exjade/Jadenu (deferasirox)

Predicting Serum Ferritin Levels in Patients with Iron Overload Treated with the Film-Coated Tablet of Deferasirox During the ECLIPSE Study [Abstract #3508; Monday, December 11, 6:00 PM EST]
Jakavi (ruxolitinib)**

Primary Analysis of JUMP, a Phase 3b, Expanded-Access Study Evaluating the Safety and Efficacy of Ruxolitinib in Patients with Myelofibrosis (N = 2233) [Abstract #4204; Monday, December 11, 6:00 PM EST]
Results from the 208-Week (4-Year) Follow-Up of Response Trial, a Phase 3 Study Comparing Ruxolitinib (Rux) with Best Available Therapy (BAT) for the Treatment of Polycythemia Vera (PV) [Abstract #322; Sunday, December 10, 7:30 AM EST]
Role of Symptom Burden in Disability Leave Among Patients with Myeloproliferative Neoplasms (MPNs): Findings from the Living with MPN Patient Survey [Abstract #1637; Saturday, December 9, 5:30 PM EST]
Revolade/Promacta (eltrombopag)***

Occurrence and Management of Cataracts in Patients with Chronic Immune Thrombocytopenia (cITP) During Long-Term Treatment with Eltrombopag (EPAG): Results from the EXTEND Study [Abstract #1053; Saturday, December 9, 5:30 PM EST]
Eltrombopag (EPAG) Treatment Improved Platelet Counts in Patients with Persistent or Chronic Immune Thrombocytopenia During a 2-Year, Phase IV, Open-Label Study [Abstract #3628; Monday, December 11, 6:00 PM EST]
A Retrospective Chart Review to Assess Burden of Illness Among Patients with Severe Aplastic Anemia with Insufficient Response to Immunosuppressive Therapy [Abstract #678; Monday, December 11, 10:30 AM EST]
Product Information
Approved indications for products vary by country and not all indications are available in every country. The product safety and efficacy profiles have not yet been established outside the approved indications. Because of the uncertainty of clinical trials, there is no guarantee that compounds will become commercially available with additional indications.

For full prescribing information, including approved indications and important safety information about marketed products, please visit View Source (link is external).

Crizanlizumab, CART-BCMA and CTL119 are investigational compounds. Efficacy and safety have not been established. There is no guarantee these compounds will become commercially available.

Disclaimer
This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as “potential,” “can,” “will,” “plan,” “expect,” “anticipate,” “look forward,” “believe,” “committed,” “investigational,” “pipeline,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures; general economic and industry conditions, including the effects of the persistently weak economic and financial environment in many countries; safety, quality or manufacturing issues, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

Moleculin Announces MD Anderson has Filed an IND with the FDA on its Drug WP1066 for the Treatment of Brain Tumors

On November 1, 2017 Moleculin Biotech, Inc., (NASDAQ: MBRX) (“Moleculin” or the “Company”), a clinical stage pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, reported that responses to U.S. Food and Drug Administration (“FDA”) requests for additional information relating to the physician-sponsored Investigational New Drug (“IND”) application to study WP1066 as a potential treatment for brain tumors have been submitted (Press release, Moleculin, NOV 1, 2017, View Source [SID1234521391]).

“We have been working closely with MD Anderson to help them respond to questions from the FDA,” commented Walter Klemp, Chairman and CEO of Moleculin. “A favorable response from the FDA on this request for IND would mean we will have two distinctly different potential cancer drugs in clinic, both Annamycin and WP1066.”
As the Company has disclosed previously, the physician-sponsored IND had been placed on clinical hold pending satisfactory responses to questions provided by the FDA. An MD Anderson physician is planning to conduct a Phase 1 clinical trial to study WP1066 in patients with glioblastoma or melanoma that has metastasized to the brain. Standard FDA procedure is to respond to such IND submissions within 30 days.

Inovio Pharmaceuticals Initiates Immuno-Oncology Clinical Study for Glioblastoma in Combination with Regeneron’s PD-1 Inhibitor

On November 1, 2017 Inovio Pharmaceuticals, Inc. (NASDAQ:INO) reported initiated a phase 1b/2a immuno-oncology trial in patients with newly diagnosed glioblastoma (GBM) designed to evaluate cemiplimab (also known as REGN2810), a PD-1 inhibitor developed by Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN), in combination with Inovio’s INO-5401 T cell activating immunotherapy encoding multiple antigens and INO-9012, an immune activator encoding IL-12 (Press release, Inovio, NOV 1, 2017, View Source [SID1234521388]).

The open-label trial of 50 patients will be conducted at approximately 30 U.S. sites, and the primary endpoints are safety and tolerability. The study will also evaluate immunological impact, progression-free survival and overall survival.

GBM is a devastating disease for both patients and caregivers. It is the most aggressive brain cancer and its prognosis is extremely poor, despite a limited number of new therapies approved over the last 10 years. The median overall survival for patients receiving standard of care therapy is approximately 15 months and the average five-year survival rate is less than three percent.

Dr. J. Joseph Kim, Inovio’s President and Chief Executive Officer, said, “Inovio is successfully executing on its immuno-oncology strategy through both combination and in monotherapy trials. Our clinical partnerships and collaborations with MedImmune, Genentech and Regeneron each provide for clinical evaluation of Inovio immunotherapies combined with checkpoint inhibitors, given a strong scientific rationale to combine an immunotherapy, which generates antigen-specific killer T cells, with a checkpoint inhibitor which augments T cell activity. I believe that INO-5401, a three antigen product targeting WT-1, PSMA and hTERT, offers great potential to address multiple cancers. Our INO-5401 combination study in GBM, as well as its sister study in advanced bladder cancer, represents an important opportunity for Inovio and its collaborators to address significant unmet medical need.”

Under a May 2017 agreement between Inovio and Regeneron, the combination trial will be solely conducted and funded by Inovio, based upon a mutually agreed upon trial design, and Regeneron will supply cemiplimab. Inovio and Regeneron will jointly conduct immunological analyses in support of the study. Regeneron, as part of their immuno-oncology collaboration with Sanofi, is developing cemiplimab both as a monotherapy and in combination with other therapies for the treatment of various cancers.

Fate Therapeutics Announces Six Presentations at the 2017 ASH Annual Meeting

On November 1, 2017 Fate Therapeutics, Inc. (NASDAQ:FATE), a biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that two oral and four poster presentations detailing clinical and preclinical results will be featured at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition(Press release, Fate Therapeutics, NOV 1, 2017, View Source [SID1234521387]). The meeting will be held December 9-12, 2017 in Atlanta, Georgia.

An oral presentation will describe the generation of CD8αβ+ T cells from an induced pluripotent stem cell (iPSC) line engineered to express a chimeric antigen receptor (CAR). This breakthrough was led by Dr. Michel Sadelain, MD, PhD, Director, Center for Cell Engineering, Memorial Sloan Kettering Cancer Center (MSK), under the Company’s multi-year sponsored research collaboration with MSK. As part of the collaboration, Fate Therapeutics has created clonal iPSC master cell lines engineered to express CARs and other functional elements and are also modified to attenuate alloreactivity and enhance persistence for off-the-shelf CAR T-cell immunotherapy. The Company’s first iPSC-derived CAR T-cell product candidate FT819, which is derived from a clonal iPSC master cell line engineered to express a CAR targeting CD19 and edited to remove T-cell receptor (TCR) expression, is undergoing preclinical development.

A second oral presentation will describe the production under current good manufacturing practice (cGMP) conditions of FT500, the Company’s first-of-kind natural killer (NK) cell product candidate derived from a clonal iPSC master cell line. The transformative manufacturing paradigm, which will be described by Jeffrey S. Miller, MD, Deputy Director of the Masonic Cancer Center, University of Minnesota, enables the efficient production of a large clonal population of NK cells in a single production run and is capable of yielding thousands of doses of homogeneous drug product for off-the-shelf delivery to patients. Fate Therapeutics plans to file a landmark Investigational New Drug (IND) application with the U.S. Food & Drug Administration (FDA) in the first quarter of 2018 to initiate first-in-human clinical investigation of FT500 in combination with FDA-approved checkpoint inhibitors for the treatment of advanced solid tumors.

In addition, Fate Therapeutics will present clinical data from the PROTECT study of ProTmune, a next-generation hematopoietic cell graft for patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation (HCT). Key clinical outcomes, including incidence rates of acute graft-versus-host disease, cancer relapse and survival at 100 days following HCT, for the seven subjects administered ProTmune in the Phase 1 stage of PROTECT will be released. Three other poster presentations will highlight additional iPSC-derived immuno-oncology product candidates that the Company is developing.

2017 ASH (Free ASH Whitepaper) Oral Presentations

FT819 iPSC-derived CAR19 T-Cell Cancer Immunotherapy
Title: Generation of Clonal Antigen Specific CD8αβ+ Cytotoxic T Lymphocytes from Renewable Pluripotent Stem Cells for Off-the-Shelf T-Cell Therapeutics
Last Author: Michel Sadelain, MD, PhD, Director, Center for Cell Engineering, Memorial Sloan Kettering Cancer Center
Publication Number: 163
Session: 703. Adoptive Immunotherapy: Immune Therapeutics for Hematologic Cancers
Date and Time: Saturday, December 9, 2017, 12:00 PM
Location: Building B, Level 2, B206

FT500 iPSC-derived NK Cell Cancer Immunotherapy
Title: Clinical Translation of Pluripotent Cell-Derived Off-the-Shelf Natural Killer Cell Cancer Immunotherapy
Last Author: Jeffrey S. Miller, MD, Deputy Director of the Masonic Cancer Center, University of Minnesota
Publication Number: 656
Session: 711. Cell Collection and Processing
Date and Time: Monday, December 11, 2017, 10:45 AM
Location: Building B, Level 2, B216-B217
2017 ASH (Free ASH Whitepaper) Poster Presentations

iPSC-derived CAR NK Cell Cancer Immunotherapy
Title: Engineering Human Induced Pluripotent Stem Cells with Novel Chimeric Antigen Receptors to Generate Natural Killer Cell Cancer Immunotherapies with Targeted Anti-Tumor Activity
Last Author: Dan S. Kaufman, MD, PhD, Director of Cell Therapy, UCSD
Publication Number: 1905
Session: 703. Adoptive Immunotherapy: Poster I
Date and Time: Saturday, December 9, 2017, 5:30 PM – 7:30 PM
Location: Building A, Level 1, Hall A2

iPSC-derived Cancer Immunotherapy Product Platform
Title: Multi-Functional Genetic Engineering of Pluripotent Cell Lines for Universal Off-the-Shelf Natural Killer Cell Cancer Immunotherapy
Last Author: Bahram Valamehr, PhD, VP Cancer Immunotherapy, Fate Therapeutics
Publication Number: 3187
Session: 703. Adoptive Immunotherapy: Poster II
Date and Time: Sunday, December 10, 2017, 6:00 PM – 8:00 PM
Location: Building A, Level 1, Hall A2

FT516 iPSC-derived hnCD16 NK Cell Cancer Immunotherapy
Title: Genetically Engineered Pluripotent Cell-Derived Natural Killer Cell Therapy Provides Enhanced Antibody Dependent Cellular Cytotoxicity Against Hematologic Malignancies and Solid Tumors in Combination with Monoclonal Antibody Therapy
Last Author: Dan S. Kaufman, MD, PhD, Director of Cell Therapy, UCSD
Session: 703. Adoptive Immunotherapy: Poster III
Publication Number: 4452
Date and Time: Monday, December 11, 2017, 6:00 PM – 8:00 PM
Location: Building A, Level 1, Hall A2

ProTmune
Title: ProTmune, the Next Generation Graft for Allogeneic Hematopoietic Cell Transplantation: Phase 1 Safety and Efficacy Data
First Author: Richard Maziarz, MD, Principal Investigator, Oregon Health Sciences University
Session: 722. Clinical Allogeneic Transplantation: Acute and Chronic GVHD, Immune Reconstitution
Publication Number: 4498
Date and Time: Monday, December 11, 2017, 6:00 PM – 8:00 PM
Location: Building A, Level 1, Hall A2
About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables genetic engineering, high-throughput single-cell isolation and clonal selection of human iPSCs and supports long-term maintenance of human iPSCs as master pluripotent cell lines. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. Similar to master cell lines used for the manufacture of monoclonal antibodies, clonal iPSC master cell lines can serve as a renewable cell source for the consistent and repeated manufacture of homogeneous cell products with the potential to treat many different diseases and many thousands of patients in an off-the-shelf manner. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 90 issued patents and 100 pending patent applications.