On December 4, 2018 Cofactor Genomics, a clinical RNA sequencing and translational assay developer, reported a pilot study to evaluate use of Cofactor’s ImmunoPrismTM assay in Genocea Biosciences’ Phase 1/2a clinical trial testing the safety and efficacy of its lead personalized cancer vaccine candidate, GEN-009, in adult cancer patients with a variety of solid tumors (Press release, Cofactor Genomics, DEC 4, 2018, View Source [SID1234531892]).

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The pilot study is designed to enable Genocea to comprehensively characterize the immune responses that patients enrolled in the clinical trial generate in response to vaccination. The RNA-based assay developed by Cofactor should enable Genocea to compare the immune cell composition for 8 major immune types within and between patients, including expression reporting for key immune escape genes.

"We are working to develop truly effective personalized neoantigen vaccines with which to treat cancer patients. Through exploration of advanced technologies like Cofactor’s ImmunoPrism assay, we aim to better understand the intratumoral immune responses we are eliciting in response to our vaccine," said Jessica Baker Flechtner, Ph.D., Chief Scientific Officer at Genocea.

"Pilot studies such as this one, where our technology is implemented in the field to empower drug developers to find the most robust markers of therapeutic success, are extremely important in validating our cutting-edge technology," noted David Messina, Chief Operating Officer at Cofactor Genomics. "Demonstrating the utility of a new approach to immune profiling is best accomplished with partners like Genocea, who are eager to gain access to the most innovative and advanced assays."

Cofactor Genomics recently announced the release of their ImmunoPrism Immune Profiling Kit, which enables access to their proprietary molecular and machine-learning informatics for RNA analysis of the tumor microenvironment. The ImmunoPrism kit offers laboratories the ability to validate the assay, as Cofactor has done through their CAP-accredited laboratory.

Cofactor will present the results of this clinical validation work during an upcoming webinar titled, "Clinical Validation of a Multidimensional Pan-Cancer Immune Assay" on Thursday, December 13 at 11 AM EST: View Source

On December 4, 2018 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported the publication of data from the previously reported, multi-center Phase 1/2 clinical study evaluating the safety and efficacy of NiCord as a stand-alone, hematopoietic stem cell (bone marrow) transplant in the Journal of Clinical Oncology1,2 (Press release, Gamida Cell, DEC 4, 2018, View Source [SID1234531889]). NiCord is an investigational product candidate in Phase 3 development as a universal bone marrow transplant solution for patients with high-risk hematologic malignancies, or blood cancers.

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Results from the Phase 1/2 study showed that patients transplanted with NiCord had rapid and durable engraftment of neutrophils and platelets, as well as prompt immune reconstitution. The median time to neutrophil recovery was shortened by nearly 50 percent for patients who received NiCord compared to a retrospective cohort of patients who received standard umbilical cord blood. NiCord also demonstrated an acceptable safety profile for patients undergoing bone marrow transplant.

"In this study, patients who received NiCord had a clinically meaningful reduction in their time to neutrophil and platelet recovery compared to a retrospective cohort of patients who received a standard umbilical cord blood transplant. The neutrophil recovery observed with NiCord also resulted in fewer days spent in the hospital compared to the comparator cohort," said Mitchell Horwitz, M.D., principal investigator and professor of medicine at the Duke Cancer Institute. "These data suggest a potential step toward making stem cell transplantation safer and more accessible to patients with lethal blood cancers."

Despite the curative potential of bone marrow transplants, it is estimated that more than 40 percent of eligible patients do not receive one for various reasons, including finding a matched donor.3 While umbilical cord blood provides a source of stem cells for patients who do not have a matched related donor, it provides a smaller number of stem cells, which can delay engraftment and put patients at a greater risk for prolonged hospitalizations and life-threatening infections. NiCord is designed to address these limitations by offering a therapeutic dose of expanded cells while preserving the functional characteristics of stem cells.

NiCord Phase 1/2 Study Design and Results
The publication, "Phase I/II study of stem cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide," described results from the completed multicenter, Phase 1/2 clinical trial of NiCord in 36 patients with high-risk hematologic malignancies and no readily available matched sibling or matched unrelated adult donor. The key primary endpoint was the cumulative incidence of neutrophil engraftment at 42 days. Additionally, the NiCord patient cohort was compared to a retrospective cohort of patients who received standard cord blood transplant using data from the Center for International Blood and Marrow Transplant Research (CIBMTR).

Key findings included the following:

Patients transplanted with NiCord had rapid and durable engraftment of neutrophils and platelets, as well as prompt immune reconstitution. The age-adjusted cumulative incidence of neutrophil engraftment at 42 days following transplantation was 94 percent for NiCord recipients compared to 85 percent for the CIBMTR cohort.
Among patients who engrafted, the median time to neutrophil recovery was 11.5 days (95% CI: 9-14 days) for NiCord recipients compared to 21 days (95% CI: 20-23 days) for the CIBMTR cohort (p<0.001).
For patients achieving platelet recovery, the median time to platelet recovery was 34 days (95% CI:32-42 days) and 46 days (95% CI:42-50 days) for the NiCord and CIBMTR cohorts, respectively (p<0.001).
NiCord demonstrated an acceptable safety profile, with hypertension reported as the most common adverse event attributable to NiCord infusion, and moderate to severe chronic graft vs. host disease reported in 9.8 percent of patients at one year following transplantation.
Primary hospital discharge occurred at a median of 20 days following transplantation. NiCord recipients spent a median of 73 days alive and out of hospital during the first 100 days following UCB transplantation.
"These data demonstrate the potential of NiCord to give patients with high-risk blood cancers an opportunity for a cure, particularly patients who would otherwise not be able to receive a bone marrow transplant using a matched donor source," stated Ronit Simantov, M.D., chief medical officer at Gamida Cell. "We are actively enrolling patients in our Phase 3 study, which is designed to confirm the potential of NiCord to be an effective transplantation solution. We look forward to completing patient enrollment expected in the second half of 2019."

About NiCord
NiCord, the company’s lead clinical program, is under development as a universal bone marrow transplant solution for patients with high-risk hematologic malignancies. NiCord has been granted breakthrough status by the U.S. Food and Drug Administration, making it the first bone marrow transplant alternative to receive this designation. It has also received U.S. and EU orphan drug designation. A Phase 3 study evaluating NiCord in patients with leukemia and lymphoma is ongoing in the United States, Europe and Asia.4 For more information on NiCord clinical trials, please visit www.clinicaltrials.gov.

On December 4, 2018 AVEO Oncology (NASDAQ:AVEO) reported a six-month extension to the interest only period under its existing amended and restated loan and security agreement with Hercules Capital, Inc. (Hercules) (Press release, AVEO, DEC 4, 2018, View Source [SID1234531888]). The extension was granted as a result of achieving certain predefined requirements under the agreement, including successfully meeting the primary endpoint of the Company’s Phase 3 TIVO-3 study of tivozanib in refractory advanced or metastatic renal cell carcinoma (RCC), by demonstrating a significant improvement in progression free survival.

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The Company will begin making principal payments on the $20.0 million facility starting on August 1, 2019. AVEO believes that its available cash, cash equivalents, and marketable securities, together with the extension of the interest only period under the Hercules loan agreement, which results in deferment of principal payments, will allow it to fund planned operations into Q3 2019. This estimate assumes no receipt of additional milestones from AVEO’s partners, no additional funding from new partnership agreements, no additional equity or debt financings, and no sales of equity through the exercise of outstanding warrants issued in connection with the 2016 private placement or outstanding warrants issued in connection with the settlement of the securities class action litigation.

"Extension of our cash runway takes us through several key anticipated milestones, with the presentation of our TIVO-3 data, including a planned update to the preliminary OS analysis which will contain additional patient data recovered in the ongoing OS sweep, and potential submission of a New Drug Application with the FDA for tivozanib in RCC. Both milestones are expected in the first half of 2019," said Michael Bailey, president and chief executive officer. "We also look forward to making important progress within this period on our immunotherapy combination strategy, the third pillar of our tivozanib strategy."

On December 4, 2018 Celltrion Healthcare reported new data at the American Society of Hematology (ASH) (Free ASH Whitepaper) Meeting (ASH) (Free ASH Whitepaper) 2018, which shows that the efficacy and safety of CT-P10 is comparable to reference rituximab in patients with advanced follicular lymphoma (AFL) over two years (Press release, Celltrion, DEC 4, 2018, View Source [SID1234531887]).1

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The efficacy and safety of CT-P10 was evaluated in a randomized, double-blind, Phase III study which evaluated the overall survival (OS), progression-free survival (PFS), and sustained response in comparison to the reference biologic rituxan. The median follow-up duration was 23 months.i

140 patients were enrolled in a 1:1 ratio and 124 patients completed eight cycles of chemotherapy (R-CVP induction). 122 patients (62 patients in the CT-P10 group and 60 patients in the reference rituximab group) who showed response during the induction period, entered the maintenance period where a total of 12 cycles of rituximab monotherapy was to be administered every two months.i

The results showed that at the median follow-up duration of 23 months, the updated efficacy data in AFL patients demonstrated comparable OS (2-year OS of 93.2% and 95.3% for CT-P10 and reference rituximab, respectively) and PFS (2-year PFS was 75.2% and 73.5% for CT-P10 and reference rituximab, respectively). In terms of sustained response, the proportion of patients who showed relapse or disease progression after achieving overall response was 19.4% (13/67) in the CT-P10 group and 21.3% (13/61) in the reference rituximab group. CT-P10 was also well-tolerated and its safety profile including immunogenicity of CT-P10 was comparable to that of reference rituximab over 23 months of treatment.i

Christian Buske, Professor, Medical Director at the Comprehensive Cancer Center Ulm, Germany, Institute of Experimental Cancer Research and Attending Physician and Professor of Medicine at the Medical Department for Internal Medicine III, Hematology/Oncology, University Hospital Ulm said, "CT-P10 has already demonstrated non-inferiority of efficacy compared with reference rituximab combined with CVP in previously untreated AFL. The study results presented today have further strengthened these findings, showing no statistically meaningful difference between CT-P10 and reference rituximab for over two years and demonstrating a proven safety profile. I believe that these results will help to further increase physician confidence in using rituximab biosimilars and will broaden AFL patient access to efficacious and affordable therapies."

Mr HoUng Kim, Head of Strategy and Operations Division, Celltrion Healthcare, said: "The two-year study marks another significant milestone for Celltrion Healthcare, showing that CT-P10 is comparable to reference rituximab in terms of overall survival and progression-free survival. Now that CT-P10 is approved in the US, the availability of the first rituximab biosimilar has the potential to significantly improve access to rituximab for patients with non-Hodgkin’s lymphoma indications. As a cost-effective alternative to the reference product, CT-P10 will reduce the burden on healthcare systems resulting in better patient outcomes."

Also presented at ASH (Free ASH Whitepaper) and published in The Lancet Haematology, is data from the CT-P10 3.4 LTBFL trial which aimed to demonstrate therapeutic equivalence of CT-P10 to reference rituximab (as monotherapy) in patients with previously untreated Low Tumor Burden Follicular Lymphoma (LTBFL).2

The results showed that CT-P10 was well-tolerated and the safety profile was comparable to that of reference rituximab over seven months. 108 (83%) of 130 patients assigned to CT-P10 and 104 (81%) of 128 assigned to rituximab achieved an overall response by month seven (treatment difference estimate 1·8%; 90% CI –6·43 to 10·20) and therapeutic equivalence was shown (90% CIs were within the prespecified margin of 17%).ii

CT-P10 is the first rituximab biosimilar to be approved in the United States.3

— Ends—

Notes to editors:

About CT-P10 (biosimilar rituximab)
CT-P10 is a mAb that targets CD20, a transmembrane protein found on the surface of most B cells. By binding specifically to CD20, CT-P10 depletes B cells by three main mechanisms: Induction of apoptosis, stimulation of CDC (complement-dependent cytotoxicity) and stimulation of ADCC (antibody-dependent cell-mediated cytotoxicity). CT-P10 is approved in the EU for the treatment of patients with non-Hodgkin lymphoma (NHL), chronic lymphocytic leukaemia, rheumatoid arthritis, granulomatosis with polyangiitis and microscopic polyangiitis. Further details of the approved indications and safety information for CT-P10 are available in the summary of product characteristics (SmPC).4 Celltrion and Teva Pharmaceutical Industries Ltd. entered into an exclusive partnership in October 2016 to commercialize CT-P10 (Truxima) in the U.S. and Canada.

About Advanced FL
Follicular lymphomas are the second most frequent subtype of nodal lymphoid malignancies in Western Europe5 and are a subtype of NHL.6 It is a slow-growing lymphoma that develops from B lymphocytes (B cells). It is characterised by painless swelling of the lymph nodes, fever for no apparent reason, drenching night sweats, fatigue, infections and bleeding. Most cases are advanced at the time of diagnosis but since the advent of rituximab, overall survival has increased to in excess of 20 years. It is called ‘follicular’ lymphoma because the abnormal lymphocytes often collect in lymph nodes in clumps that are known as ‘follicles’. Follicular lymphoma is more common in people aged over 65, but it can occur in people of any age.

On December 4, 2018 Triumvira Immunologics Inc. (Triumvira), reported the presentation of key preclinical data at the American Society of Hematology (ASH) (Free ASH Whitepaper) 60th Annual Meeting and Exposition, held December 1-4, 2018, in San Diego, CA (Press release, Triumvira Immunologics, DEC 4, 2018, View Source [SID1234531886]). The data show that TAC-engineered T cells provide long-lasting tumor rejection in mouse models of BCMA-positive and CD19-positive hematological malignancies and suggest the presence of persisting TAC-T cells that protect against tumor regrowth upon tumor rechallenge.

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The results build on data recently published in Nature Communications (Helsen et al., 2018) describing the proprietary T cell Antigen Coupler (TAC) technology, a chimeric receptor that co-opts the endogenous T cell receptor (TCR) for T cell activation and tumor cell killing. The new data, presented during a podium presentation and a poster presentation, now describe additional preclinical pharmacology of the company’s therapeutic programs in multiple myeloma (BCMA) and B-cell lymphoma (CD19). A CD19-directed TAC-T cell product is expected to enter clinical testing in H1 2019.

The podium presentation, presented by Ksenia Bezverbnaya, BSc., McMaster University, (Publication 3267: T cell engineered with a novel chimeric receptor demonstrate durable in vivo efficacy against disseminated multiple myeloma) focused on two TAC constructs each of which was fused to a different BCMA binder. A single dose of TAC-T cells (106) engineered with either construct cured mice with disseminated KMS-11 multiple myeloma cells and protected mice, in remission for 3 months after treatment, against tumor regrowth upon a fresh inoculation of tumor cells.

The poster presentation, presented by Christopher Helsen, Ph.D., Director of Research & Development, and Head of Platform Development at Triumvira (Publication 962: T cells engineered with T cell antigen coupler (TAC) receptors for haematological malignancies) described the preclinical pharmacology of TAC-T cells directed against the CD19 and BCMA antigens in multiple models of CD19-positive leukemia and lymphoma and the BCMA-positive KMS-11 multiple myeloma model. TAC-T cells specific for either antigen produced long-lasting tumor rejection and effectively protected mice in tumor rechallenge experiments indicating the presence of persisting TAC-T cells.

"These data provide further evidence that TAC furnishes T cells with properties that are highly desirable attributes for future T cell therapies," states Dr. Paul Lammers, President & CEO of Triumvira, "TAC-T cells demonstrate superiority over 2nd generation CAR-T cells, and effectively rids animals from cancer in the absence of notable toxicities and protects these animals for a prolonged period of time. These new data further strengthens the argument for bringing the TAC technology to patients."