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Rocket Pharmaceuticals to Present Preliminary Phase 1 Data of RP-L102 “Process B” for Fanconi Anemia at the 61st American Society of Hematology Annual Meeting

On November 6, 2019 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform clinical-stage gene therapy company, reported presentations at the upcoming 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held December 7-10, 2019 in Orlando, Florida (Press release, Rocket Pharmaceuticals, NOV 6, 2019, View Source [SID1234550533]). The two poster presentations will highlight clinical data from the Phase 1 study of RP-L102 utilizing "Process B" for the treatment of Fanconi Anemia (FA), as well as long-term follow-up data from the Phase 1/2 EUROFANCOLEN trial.

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Details for Rocket’s poster presentations are as follows:
Title: Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells
Session Title: Gene Therapy and Transfer: Poster II
Presenter: Sandeep Soni, M.D.
Session Date: Sunday, December 8, 2019
Session Time: 6:00 p.m. – 8:00 p.m. EST
Location: Orange County Convention Center, Hall B

Title: Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy
Session Title: Gene Therapy and Transfer: Poster III
Presenter: Paula Río, Ph.D.
Session Date: Monday, December 9, 2019
Session Time: 6:00 p.m. – 8:00 p.m. EST
Location: Orange County Convention Center, Hall B

The Sunday poster session will be followed by a breakout session to give investors and analysts the opportunity to ask questions and discuss the data. The breakout session, hosted by Rocket management, will be held on Sunday, December 8th at 8:30 p.m. EST, directly after Dr. Soni’s presentation. At the event, Dr. Soni, Clinical Associate Professor of Stem Cell Transplantation and Regenerative Medicine at the Stanford University School of Medicine and principal investigator of the U.S. Phase 1 trial of RP-L102 and Paula Río, Ph.D., Senior Scientist, División de Terapias Innovadoras en el Sistema Hematopoyético, CIEMAT/CIBERER Unidad Mixta de Terapias Avanzadas CIEMAT/IIS Fundación Jiménez Díaz will be participating in a Q&A panel. For further information, please contact [email protected].

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a FANC-A gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the ‘gold standard’ test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patient’s blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as ‘nature’s gene therapy’ provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

Xencor to Present Initial Data from the Phase 1 Study of XmAb®13676 in B-Cell Malignancies at the American Society of Hematology Annual Meeting

On November 6, 2019 Xencor, Inc. (NASDAQ: XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of cancer, autoimmune diseases, asthma and allergic diseases, reported that initial data from its ongoing Phase 1 dose-escalation study of XmAb13676, a CD20 x CD3 bispecific antibody, in patients with B-cell malignancies will be presented in a poster session during the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Orlando, Florida on Monday, December 9, 2019 (Press release, Xencor, NOV 6, 2019, View Source [SID1234550532]).

"Initial results from dose-escalation cohorts demonstrate encouraging clinical activity in heavily pretreated patients with several subtypes of non-Hodgkin lymphoma, as well as chronic lymphocytic leukemia. The most common treatment-related adverse event has been fever. Cytokine release syndrome, the second most common adverse event, has been generally manageable with premedication," said Bassil Dahiyat, Ph.D., president and chief executive officer at Xencor.

Key Highlights from the Abstract

The abstract with accepted data from the study are available through the ASH (Free ASH Whitepaper) website. Updated results will be shared at the ASH (Free ASH Whitepaper) Annual Meeting.

At data cut off on June 28, 2019, 36 patients with relapsed/refractory non-Hodgkin’s lymphoma (r/r NHL) and 8 patients with relapsed/refractory chronic lymphocytic leukemia (r/r CLL) had received XmAb13676. The study was designed in two parts: Part A to establish an initial priming dose and Part B to escalate dosing on subsequent administration. Prophylactic treatment for cytokine release syndrome (CRS) was mandated prior to each administration of XmAb13676.
Patients with r/r NHL (n=36) had a median age of 61.5 years, a median of 3.5 prior therapies and had been diagnosed a median of 24.6 months prior to treatment. In the efficacy evaluable population and at doses ranging from 80 to 125 mcg/kg, objective responses were observed in 33% of patients (n=6/18), including 42% of DLBCL patients (n=5/12). CRS occurred in 42% of patients (n=15/36), and one patient receiving an initial dose of 125 mcg/kg experienced Grade 4 CRS. A priming dose of 45 mcg/kg was chosen for Part B.
Patients with r/r CLL (n=8) had a median age of 76 years, a median of 4.5 prior therapies and had been diagnosed a median of 76.1 months prior to treatment. There was one complete response (Richter transformation) in five patients treated at the 20 mcg/kg dose level, the highest dose administered in the ongoing Part A of the study. CRS occurred in 25% of patients (n=2/8), and one patient experienced Grade 3 CRS.
Presentation Details

Abstract: 4079
Title: Preliminary Safety and Anti-tumor Activity of XmAb13676, an Anti-CD20 x Anti-CD3 Bispecific Antibody in Patients with Relapsed/Refractory Non-Hodgkin’s Lymphoma and Chronic Lymphocytic Leukemia
Presenter: Krish Patel, M.D., Director of the Lymphoma Program at Swedish Cancer Institute
Session: 626. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Prospective Clinical Trials: Poster III
Date & Time: Monday, December 9, 2019, 6:00 p.m. – 8:00 p.m. EST
Location: Orange County Convention Center, Hall B
About XmAb13676

XmAb13676 is a tumor-targeted antibody that contains both a CD20 binding domain and a cytotoxic T-cell binding domain (CD3) in a Phase 1 clinical trial for the treatment of B-cell malignancies. An XmAb bispecific Fc domain serves as the scaffold for these two antigen binding domains and confers long circulating half-life, stability and ease of manufacture on XmAb13676. CD20 is highly expressed on B-cell tumors, including in chronic lymphocytic leukemia (CLL) and non-Hodgkin lymphoma (NHL). Engagement of CD3 by XmAb13676 activates T cells for highly potent and targeted killing of CD20-expressing tumor cells.

MaaT Pharma to Present Initial Results with its Lead Microbiota Biotherapeutic in Intestinal GvHD at 61st American Society of Hematology (ASH) Annual Meeting

On November 6, 2019 MaaT Pharma reported that a poster will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting with initial data on it’s lead microbiome restoration biotherapeutic, MaaT013, as a treatment in patients with gastrointestinal-predominant, Steroid-Refractory and Steroid-Dependent acute Graft-versus-Host-Disease (GI aGvHD) following allogeneic Hematopoietic Stem-Cell Transplantation (allo-HSCT) (Press release, MaaT Pharma, NOV 6, 2019, View Source [SID1234550531]). MaaT Pharma provided the product as a pharmaceutical preparation to hospitals as part of its compassionate use program for patients who had previously received and failed up to five previous systemic treatments for GvHD. MaaT013 is an enema formulation of a microbiota biotherapeutic characterized by a high diversity and consistent richness of microbial species derived from pooled healthy donors. The product is manufactured at the company’s centralized European cGMP production facility using its proprietary microbiome restoration biotherapeutic platform. The poster will be presented during the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Orlando, Florida from December 7 – 10, 2019. The poster abstract was published in the online meeting program on November 6, 2019 at 9:00 AM EDT and also in the online November supplemental issue of the journal Blood.

Poster Presentation details:

Title: Successful and Safe Treatment of Intestinal Graft-Versus-Host Disease (GvHD) with Pooled-Donor Full Ecosystem Microbiota Biotherapeutics
Abstract No: 1993
Session Name: 722. Clinical Allogeneic Transplantation: Acute and Chronic GvHD, Immune Reconstitution: Poster I
Date/Time: Saturday, December 7, 2019 / 5:30 PM – 7:30 PM EDT
Location: Orange County Convention Center, Hall B

MaaT Pharma will announce the results through a press release following the presentation and will further make the poster available on the company website under "News".

In addition to the compassionate use program, MaaT Pharma is currently investigating MaaT013 in a Phase II clinical trial in acute SR GI GvHD patients (NCT03359980). The study, named HERACLES, is a multi-center, single-arm, open-label study, enrolling 32 patients to evaluate the safety and efficacy of MaaT013 in steroid-resistant, gastrointestinal-predominant aGvHD (SR GI aGvHD) patients. Acute GvHD is a serious, often fatal syndrome typically involving the gut, skin and liver. MaaT Pharma has established the most complete approach to restoring patient-microbiome symbiosis in the gut to improve efficacy of cancer treatments and survival outcomes in life-threatening diseases. The company announced in June this year the completion of a third positive safety assessment of MaaT013 in the Phase II trial by an independent Data and Safety Monitoring Board (DSMB). MaaT013 has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

Celularity to Present New Data at the ASH Annual Meeting on Novel Allogeneic, Off the Shelf, Placental Derived CAR T and NK Cell Therapy Programs

On November 6, 2019 Celularity, Inc., a clinical-stage cell therapeutics company developing allogeneic cellular therapies harnessed from human placentas, reported it will present data supporting its allogeneic, off-the-shelf, placental derived cell therapy programs at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, taking place December 7-10 at the Orange County Convention Center in Orlando, Florida (Press release, Celularity, NOV 6, 2019, View Source [SID1234550530]).

"We believe the placenta is nature’s cell factory, and the data being presented at ASH (Free ASH Whitepaper) will illustrate the potential of our investigational placental derived NK- and T cell-based allogeneic cell therapy programs in oncology," said Robert J. Hariri, M.D., Ph.D., Founder, Chairman and CEO at Celularity. "We look forward to advancing Celularity’s leading-edge technologies that harness the placenta’s unique immunologic and pro-regenerative biology to produce therapeutic solutions targeting unmet healthcare needs globally."

ASH abstracts are now available at View Source

Details for the 2019 ASH (Free ASH Whitepaper) poster presentations are as follows:

Title: Development of CD38 CAR Engineered Human Placental Hematopoietic Stem Cell Derived Natural Killer Cells (PNK-CAR38) As Allogeneic Cancer Immunotherapy
Date: Saturday, December 7, 2019

Title: Engineering High Affinity and Cleavage Resistant CD16 to Augment ADCC of Placental Hematopoietic Stem Cells-Derived Natural Killer Cells
Date: Saturday, December 7, 2019

Title: Preclinical Evaluation of Human Placental-Derived Allogeneic CD19 CAR-T Cells Against B Cell Malignancies
Date: Sunday, December 8, 2019

Title: Results of a Phase I Study of PNK-007, Allogeneic, Off the Shelf NK Cell, Post Autologous Transplant in Multiple Myeloma (NCT02955550)
Date: Monday, December 9, 2019

Title: Immune Monitoring of CD34+ Placental Cell Derived Natural Killer Cell Therapy (PNK-007) in Phase I Study of Multiple Myeloma
Date: Monday, December 9, 2019

About PNK-007
PNK‐007 is the only allogeneic, off-the-shelf NK cell therapy being developed from placental hematopoietic stem cells as a potential treatment option for various hematologic cancers and solid tumors. NK cells are a unique class of immune cells, innately capable of targeting cancer cells and interacting with adaptive immunity. When derived from the placenta, these cells offer intrinsic safety and versatility, allowing potential use across a range of organs and tissues. PNK cells are currently being investigated as a treatment for acute myeloid leukemia (AML) and multiple myeloma (MM).

About CYNK-001
CYNK-001, a cryopreserved formulation of PNK-007 cells, is the only cryopreserved, off-the-shelf NK cell therapy being developed from placental hematopoietic stem cells as a potential treatment option for various hematologic cancers and solid tumors.

Promega Enters Global Collaboration with Merck to Develop Microsatellite Instability (MSI) Companion Diagnostic for Use with KEYTRUDA®

On November 6, 2019 Promega Corporation reported it has entered into a global collaboration with Merck, known as MSD outside the United States and Canada, to develop Promega’s microsatellite instability (MSI) technology as an on-label, solid tumor companion diagnostic (CDx) for use with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) (Press release, Promega, NOV 6, 2019, View Source [SID1234550529]). The global collaboration will initially seek regulatory approval for the Promega MSI CDx in the United States and China. Plans to seek approvals in additional territories may follow.

"It is gratifying to see our MSI technology have such meaning within the oncology community," said Bill Linton, president and CEO, Promega Corporation. "Promega developed this technology well over a decade ago and our long-term commitment to R&D helped evolve its use."

Promega MSI technology has been validated in labs around the world to characterize solid tumor MSI status. MSI testing functionally measures the genomic accumulation of insertion or deletion (INDEL) errors caused by a deficient mismatch-repair system (dMMR) that occurs in certain types of solid tumors, and this screening may be used to better characterize tumors and guide therapeutic choices for MSI-High cancer types. Tumors with MSI-High status have been shown to respond to immune checkpoint inhibitor (ICI) therapies. This outcome may be explained by MSI-driven tumor expression of mutation-associated neoantigens (MANA) that are believed to cause immune cell infiltration into the tumor microenvironment. Tumor induced inhibition of immune cell activity can be overcome with ICI therapies, allowing for tumor cell destruction by the immune cells.

"Unlike other DNA-based molecular screening options, Promega MSI technology uses five monomorphic mononucleotides, which is recommended by the National Cancer Institute," said Jeff Bacher, Ph.D., senior research scientist, Promega Corporation. "Our test uses a sensitive and specific panel of markers for detection of MSI status and offers valuable insight to help inform physicians on how best to treat patients with cancer including those likely to benefit from immune checkpoint inhibitor treatment."

Promega MSI technology is one of the leading standard tests for MSI status detection in research laboratories and recently achieved innovation status and priority review by the National Medical Products Administration (NMPA) in China. It has been used extensively in clinical research for more than 15 years and is supported by more than 140 peer-reviewed publications. Promega continues to advance the promise of MSI technology globally. In addition to the Merck collaboration announcement, Promega intends to seek regulatory clearance for an MSI in vitro diagnostic (IVD) test in the United States, China and Europe. These products are intended to launch in the first half of 2020 in the United States, China and Europe.

To learn more about microsatellite instability, visit: www.promega.com/ExploreMSI.

To request more information regarding the details of the announcement, visit: www.promega.com/msiCDx.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

About Promega MSI Technology

The Promega technology is a PCR-based method for detecting MSI, a form of genomic instability caused by insertion or deletion of additional bases into DNA microsatellites, regions of repeating bases distributed throughout the human genome. During DNA replication, failure of the mismatch repair system to correct these errors causes MSI. MSI status is a measure of dMMR found in certain solid tumors, providing oncologists, pathologists and patients with information that characterizes tumors and can guide care and treatment. The current Promega Research Use Only MSI assay has been available and used in the market as part of Lab-Developed Tests since 2004.