On December 5, 2018 Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), reported that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) and granted Priority Review for Tecentriq (atezolizumab), in combination with carboplatin and etoposide (chemotherapy), for the initial (first-line) treatment of people with extensive-stage small cell lung cancer (ES-SCLC) (Press release, Genentech, DEC 5, 2018, View Source [SID1234531891]). The FDA is expected to make a decision on approval by March 18, 2019. A Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a serious disease.

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"It’s been more than 20 years since there has been a new initial treatment option for extensive-stage small cell lung cancer that delivers a clinically meaningful survival benefit," said Sandra Horning, M.D., chief medical officer and head of Global Product Development. "We are working closely with the FDA to bring this Tecentriq-based regimen to people with this difficult-to-treat type of lung cancer as soon as possible."

This sBLA is based on results from the Phase III IMpower133 study, which met its co-primary endpoints of overall survival (OS) and progression-free survival (PFS) in the initial treatment of people with ES-SCLC. The safety profile of the combination was consistent with the safety profiles of the individual medicines, and no new safety signals were identified.

Tecentriq is currently approved by the FDA to treat people with metastatic non-small cell lung cancer (NSCLC) who have disease progression during or following platinum-containing chemotherapy, and have progressed on an appropriate FDA-approved targeted therapy if their tumor has ALK or EGFR gene abnormalities.

About the IMpower133 study

IMpower133 is a Phase III, multicenter, double-blinded, randomized placebo-controlled study evaluating the efficacy and safety of Tecentriq in combination with chemotherapy (carboplatin and etoposide) versus chemotherapy (carboplatin and etoposide) alone in chemotherapy-naïve people with ES-SCLC. The study enrolled 403 people who were randomized equally (1:1) to receive:

Tecentriq in combination with carboplatin and etoposide (Arm A), or
Placebo in combination with carboplatin and etoposide (Arm B, control arm)
During the treatment-induction phase, people received treatment on 21-day cycles for four cycles, followed by maintenance with Tecentriq or placebo until progressive disease (PD) as assessed by the investigator using Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1). Treatment could be continued until persistent radiographic PD or symptomatic deterioration was observed.

The co-primary endpoints were:

PFS as determined by the investigator using RECIST v1.1 in the intention-to-treat (ITT) population
OS in the ITT population
IMpower133 met its OS and PFS co-primary endpoints as per the study protocol. The analysis showed that Tecentriq and chemotherapy helped people live significantly longer compared to chemotherapy alone (OS=12.3 versus 10.3 months; hazard ratio [HR] = 0.70, 95 percent CI: 0.54-0.91; p=0.0069) in the ITT population. The one-year OS rate for people who received the Tecentriq-based combination was 51.7 percent compared to 38.2 percent for people who received chemotherapy alone. The Tecentriq-based combination also significantly reduced the risk of disease worsening or death compared to chemotherapy alone (PFS=5.2 versus 4.3 months; HR=0.77; 95 percent CI: 0.62-0.96; p=0.017). The one-year PFS rate for people who received the Tecentriq-based combination was 12.6 percent compared to 5.4 percent for people who received chemotherapy alone. Safety for the Tecentriq and chemotherapy combination appeared consistent with the known safety profile of the individual medicines, and no new safety signals were identified with the combination. Grade 3-4 treatment-related adverse events (AEs) were reported in 56.6 percent of people receiving Tecentriq plus chemotherapy compared to 56.1 percent of people receiving chemotherapy alone.

About lung cancer

According to the American Cancer Society, it is estimated that more than 234,000 Americans will be diagnosed with lung cancer in 2018. Lung cancer can be broadly divided into two major types: non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC). NSCLC is the most prevalent type, accounting for around 85 percent of all lung cancer cases, and SCLC accounting for approximately 15 percent of all cases. The majority (approximately 70 percent) of people with SCLC are diagnosed with ES-SCLC.

About Tecentriq (atezolizumab)

Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

Tecentriq U.S. Indication (pronounced ‘tē-SEN-trik’)

Tecentriq is a prescription medicine used to treat:

A type of bladder and urinary tract cancer called urothelial carcinoma.

Tecentriq may be used when your bladder cancer:
has spread or cannot be removed by surgery, and if you have any one of the following conditions:
you are not able to take chemotherapy that contains a medicine called cisplatin, and your doctor has tested your cancer and found high levels of a specific protein on your cancer called programmed death-ligand 1 (PD-L1), as determined by an FDA-approved test, or
you are not able to take chemotherapy that contains any platinum regardless of PD-L1 status on your cancer, or
you have tried chemotherapy that contains platinum, and it did not work or is no longer working
The approval of Tecentriq in these patients is based on a study that measured response rate and duration of response. There is an ongoing study to confirm clinical benefit.

A type of lung cancer called non-small cell lung cancer (NSCLC).

Tecentriq may be used when your lung cancer:
has spread or grown, and
you have tried chemotherapy that contains platinum, and it did not work or is no longer working
If your tumor has an abnormal EGFR or ALK gene, you should have also tried an FDA-approved therapy for tumors with these abnormal genes, and it did not work or is no longer working.

It is not known if Tecentriq is safe and effective in children.

Important Safety Information

What is the most important information about Tecentriq?

Tecentriq can cause the immune system to attack normal organs and tissues and can affect the way they work. These problems can sometimes become serious or life threatening and can lead to death.

Patients should call or see their healthcare provider right away if they get any symptoms of the following problems or these symptoms get worse.

Tecentriq can cause serious side effects, including:

Lung problems (pneumonitis)–signs and symptoms may include new or worsening cough, shortness of breath, and chest pain
Liver problems (hepatitis)–signs and symptoms of hepatitis may include yellowing of the skin or the whites of the eyes, severe nausea or vomiting, pain on the right side of the stomach area (abdomen), drowsiness, dark urine (tea colored), bleeding or bruising more easily than normal, and feeling less hungry than usual
Intestinal problems (colitis)–signs and symptoms of colitis may include diarrhea (loose stools) or more bowel movements than usual, blood or mucous in the stools or dark, tarry, sticky stools, and severe stomach area (abdomen) pain or tenderness
Hormone gland problems (especially the thyroid, adrenal glands, pancreas, and pituitary)–signs and symptoms that the hormone glands are not working properly may include headaches that will not go away or unusual headaches, extreme tiredness, weight gain or weight loss, dizziness or fainting, feeling more hungry or thirsty than usual, hair loss, changes in mood or behavior (such as decreased sex drive, irritability, or forgetfulness), feeling cold, constipation, the voice gets deeper, urinating more often than usual, nausea or vomiting, and stomach area (abdomen) pain
Problems in other organs–signs and symptoms may include severe muscle weakness, numbness or tingling in hands or feet, confusion, blurry vision, double vision, or other vision problems, changes in mood or behavior, extreme sensitivity to light, neck stiffness, eye pain or redness, skin blisters or peeling, chest pain, irregular heartbeat, shortness of breath, or swelling of the ankles
Severe infections–signs and symptoms of infection may include fever, cough, flu-like symptoms, pain when urinating, and frequent urination or back pain
Severe infusion reactions–signs and symptoms of infusion reactions may include chills or shaking, itching or rash, flushing, shortness of breath or wheezing, swelling of the face or lips, dizziness, fever, feeling like passing out, and back or neck pain
Getting medical treatment right away may help keep these problems from becoming more serious. A healthcare provider may treat patients with corticosteroid or hormone replacement medicines. A healthcare provider may delay or completely stop treatment with Tecentriq if patients have severe side effects.

Before receiving Tecentriq, patients should tell their healthcare provider about all of their medical conditions, including if they:

have immune system problems (such as Crohn’s disease, ulcerative colitis, or lupus); have had an organ transplant; have lung or breathing problems; have liver problems; have a condition that affects the nervous system (such as myasthenia gravis or Guillain-Barre syndrome); or are being treated for an infection
are pregnant or plan to become pregnant. Tecentriq can harm an unborn baby. Patients should tell their healthcare provider right away if they become pregnant or think they may be pregnant during treatment with Tecentriq. If patients are able to become pregnant:
A healthcare provider should do a pregnancy test before they start treatment with Tecentriq.
They should use an effective method of birth control during their treatment and for at least 5 months after the last dose of Tecentriq.
are breastfeeding or plan to breastfeed. It is not known if Tecentriq passes into the breast milk. Do not breastfeed during treatment and for at least 5 months after the last dose of Tecentriq
Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Tecentriq in people with urothelial carcinoma include:

feeling tired
decreased appetite
nausea
constipation
urinary tract infection
diarrhea
fever
The most common side effects of Tecentriq in people with non-small cell lung cancer include:

feeling tired
decreased appetite
muscle pain
cough
shortness of breath
Tecentriq may cause fertility problems in females, which may affect the ability to have children. Patients should talk to their healthcare provider if they have concerns about fertility.

These are not all the possible side effects of Tecentriq. Patients should ask their healthcare provider or pharmacist for more information. Patients should call their doctor for medical advice about side effects.

On December 5, 2018 Tetraphase Pharmaceuticals, Inc. (NASDAQ:TTPH), a biopharmaceutical company focused on developing and commercializing novel antibiotics to treat life-threatening multidrug-resistant (MDR) infections, reported that President and Chief Executive Officer Guy Macdonald will present a corporate overview at the BMO Prescriptions for Success Healthcare Conference on Wednesday, December 12, 2018 at 9:40 a.m. ET at the Mandarin Oriental Hotel in New York City (Press release, Tetraphase, DEC 5, 2018, View Source [SID1234531909]).

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A live audio webcast of the BMO Prescriptions for Success Healthcare Conference presentation will be available on the Company’s website at View Source The archived presentation will be available for 30 days.

On December 4, 2018 Amgen (NASDAQ:AMGN) reported the first clinical results from studies evaluating investigational novel bispecific T cell engager (BiTE) immunotherapies AMG 420 and AMG 330 (Press release, Amgen, DEC 4, 2018, View Source;p=RssLanding&cat=news&id=2379192 [SID1234531859]). In two separate Phase 1 dose escalation studies, AMG 420, which targets B-cell maturation antigen (BCMA), and AMG 330, which targets CD33, provided early evidence of tolerability and anti-tumor activity in patients with relapsed and/or refractory multiple myeloma and relapsed or refractory acute myeloid leukemia (AML), respectively. These data were highlighted during oral presentations at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition in San Diego.

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BiTE antibody construct technology, pioneered by Amgen, is an innovative treatment approach that helps the body’s immune system attack cancer cells without the removal of immune cells from the patient. Amgen is studying a number of "off-the-shelf" investigational BiTE immunotherapies, with distinct targets, across a range of hematologic and solid tumors.

"Building on our success with the only approved BiTE immunotherapy available for patients, Amgen is emphasizing our commitment to the potential of this platform by advancing the development of approximately a dozen novel molecules across hematologic and solid tumor targets in hopes of continuing to offer meaningful advances to patients in need," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "We’re encouraged by the early results of investigational BiTE immunotherapies AMG 420 and AMG 330, especially when considered in the context of these heavily pre-treated patients, many of whom have run out of available options. We look forward to sharing more results from our BiTE pipeline at future medical meetings."

ASH Abstract #1010: Treatment with AMG 420, an anti-B-Cell Maturation Antigen (BCMA) Bispecific T-cell Engager (BiTE) Antibody Construct, Induces Minimal Residual Disease (MRD) Negative Complete Responses in Relapsed and/or Refractory (R/R) Multiple Myeloma (MM) Patients: Results of a First-in-Human (FIH) Phase 1 Dose Escalation Study

The data shared at ASH (Free ASH Whitepaper) were the first presentation of all endpoints from this Phase 1 dose-escalation trial of AMG 420 in patients with relapsed and/or refractory multiple myeloma. The objectives of the study included assessment of safety, tolerability and anti-tumor activity of AMG 420 per International Myeloma Working Group 2006 Uniform Response Criteria for Multiple Myeloma.

In the study, 42 patients with relapsed and/or refractory multiple myeloma who had progression after at least two prior lines of treatment (including a proteasome inhibitor and an immunomodulatory imide drug) received AMG 420 at varying doses [0.2 to 800 µg/day (d)]. AMG 420 induced clinical responses in 13 patients, including complete responses (CR) in seven patients. Four patients treated at the 400 µg/d dose achieved minimal residual disease (MRD) negative complete responses, meaning that no cancer cells were detectable in the bone marrow. The objective response rate at 400 µg/d was 70 percent (seven of 10 patients), with six patients still responding up to 7.5 months. One dose-limiting toxicity was observed up to the 400 µg/d dose (peripheral polyneuropathy, which improved to baseline after intravenous immunoglobulin and corticosteroid treatment).

Of those patients with serious adverse events (AEs) (n=20, 48 percent), 17 required hospitalization and four had prolonged hospitalization. Serious AEs included infections (n=12), peripheral polyneuropathy (n=2), and one each of liver failure, cardiac failure, edema, biliary obstruction, spinal cord compression, renal failure and weight loss. Treatment-related serious AEs included polyneuropathy (n=2, both grade 3) and edema (n=1, grade 3). Cytokine release syndrome (CRS) was seen in 16 patients (grade 1, n=13; grade 2, n=2; grade 3, n=1). In this study, 800 µg/d was determined to not be tolerable, as two out of the three patients treated at this dose experienced dose-limiting toxicities.

Two patients died during the course of the study from AEs not considered treatment-related. One patient died after the first cycle of treatment from acute respiratory distress due to concurrent flu and aspergillosis. The second patient died from liver failure secondary to a viral infection during the course of treatment.

"These first-in-human data of a BCMA-targeting BiTE immunotherapy showed encouraging evidence of AMG 420 activity, with no major toxicities up to the 400 µg/d dose in patients with relapsed and/or refractory multiple myeloma who received a median of four prior therapies," said Max S. Topp, M.D., professor, Hospital of Wuerzburg, Germany and AMG 420 clinical study investigator. "Despite recent treatment advances, multiple myeloma continues to be a disease characterized by cycles of relapse and recurrence requiring additional therapies to help control the disease."

Additionally, AMG 420 was granted Fast Track Designation by the U.S. Food and Drug Administration (FDA). Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions.

ASH Abstract #25: A Phase 1 First-in-Human Study of AMG 330, an Anti-CD33 Bispecific T-cell Engager (BiTE) Antibody Construct, in Relapsed/Refractory Acute Myeloid Leukemia (R/R AML)

In a separate first-in-human Phase 1 dose escalation study, 40 patients with relapsed or refractory AML were enrolled to receive AMG 330 in 12 dose cohorts with a target dose range of 0.5 to 480 µg/d. The study objectives were to evaluate the safety, pharmacokinetics and pharmacodynamics of AMG 330 and to estimate the maximum tolerated dose. Results showed that two patients in the trial achieved a CR at the 240 µg/d dose and two patients achieved a CR with incomplete blood count recovery, one at the 240 µg/d dose and one at the 120 μg/d dose. The CRs were not sustained beyond one cycle of treatment.

Patients in the trial received a median of one (range: 1-6) cycle with AMG 330; the majority of patients discontinued treatment for disease progression. Other reasons for study discontinuation included AEs (n=6, 2 treatment-related) and patient request (n=2).

Serious AEs were seen in 73 percent of patients (treatment-related in 17 patients). The most common serious AEs seen in more than one patient included CRS (n=11), febrile neutropenia (n=7), pneumonia (n=4), leukopenia (n=4), pyrexia (n=3), thrombocytopenia (n=3) and subdural hematoma (n=2). One patient died on study due to AML progression and one due to intracranial hemorrhage (neither treatment-related). There were dose-limiting toxicities of grade 2 CRS and grade 4 ventricular fibrillation with a target dose of 480 μg/d administered as a single-step regimen.

"The majority of adult AML patients will not be cured with standard chemotherapy, underscoring the need for innovative treatment options for those who have relapsed or are refractory to currently available treatments1," said Farhad Ravandi, M.D., Janiece and Stephen A. Lasher professor of medicine and chief of section of Developmental Therapeutics in the Department of Leukemia at the University of Texas – MD Anderson Cancer Center and AMG 330 clinical study investigator. "These early data are encouraging as they indicate AMG 330 may have anti-leukemic activity in heavily pretreated patients with relapsed or refractory AML, validating the need for continued evaluation of the BiTE platform in targeting CD33."

About BiTE Technology
Bispecific T cell engager (BiTE) antibody construct is an innovative technology that can be engineered to target any tumor antigen expressed by any type of cancer. The protein molecules are designed to kill malignant cells using the patient’s own immune system by bridging T cells to tumor cells. BiTE antibody construct helps connect the T cells to the targeted cell, with the intent of causing T cells to inject toxins which trigger cancer cell death (apoptosis). Amgen is developing BiTE antibody constructs to uniquely (or specifically) target numerous hematologic malignancies and solid tumors.

On December 4, 2018 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported new preclinical data on the Company’s induced pluripotent stem cell (iPSC) product platform and its iPSC-derived, off-the-shelf cell-based cancer immunotherapy pipeline at the 60thAmerican Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (Press release, Fate Therapeutics, DEC 4, 2018, View Source [SID1234531876]). Last week, the Company announced that the U.S. Food and Drug Administration (FDA) allowed its Investigational New Drug (IND) Application for FT500, the Company’s universal, off-the-shelf natural killer (NK) cell product candidate derived from a clonal master iPSC line. The clinical trial of FT500 is expected to be the first-ever clinical investigation in the U.S. of an iPSC-derived cell product.

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"Fate Therapeutics is at the forefront in developing off-the-shelf, cell-based cancer immunotherapies, and is rapidly progressing multiple iPSC-derived CAR T- and NK cell product candidates that have the potential to disrupt autologous and allogeneic approaches to cell therapy," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "The clearance of our FT500 IND by the FDA is a landmark achievement and serves as a roadmap for advancement of our iPSC-derived cell product pipeline into clinical development. We believe the use of clonal master iPSC lines uniquely enables the production of multi-functional, cell-based cancer immunotherapies that are uniformly engineered, are available for off-the-shelf administration to patients and can be effectively combined in multi-dose, multi-cycle regimens with well-established immune-oncology agents, such as checkpoint inhibitor blockade and monoclonal antibody therapy, including in earlier lines of therapy."

Several of the Company’s iPSC-derived cell product candidates undergoing development were discussed during an investor event at ASH (Free ASH Whitepaper) by its collaborating experts in the field of cell-based cancer immunotherapy including:

Michel Sadelain, M.D., Ph.D., Director, Center for Cell Engineering & Gene Transfer and Gene Expression Laboratory, Memorial Sloan Kettering Cancer Center;

Jeffrey S. Miller, M.D., Deputy Director, Masonic Cancer Center, University of Minnesota; and

Dan S. Kaufman, M.D., Ph.D., Professor of Medicine, Division of Regenerative Medicine, Director of Cell Therapy, University of California – San Diego.
FT500
The clinical trial of FT500 is intended to evaluate the safety and tolerability of multiple doses of FT500, in multiple dosing cycles with nivolumab, pembrolizumab or atezolizumab, in subjects that have progressed or failed on immune checkpoint blockade (CPB) therapy. Although CPB therapy can lead to prolonged responses, refractory disease and progression after initial response remain major causes of mortality. In patients receiving CPB therapy, mutations in beta-2-microglobulin (B2M), an essential component for the stable presentation of antigens by tumor cells, have been identified in approximately 30% of patients with progressing disease and are associated with poor overall survival. Investigators have demonstrated in various tumor model systems that NK cells have the potential to rescue CPB therapy by recognizing and killing B2M-deficient target cells. An oral presentation at ASH (Free ASH Whitepaper) by Dr. Miller showed that FT500 synergizes with T cells and anti-PD1 antibody to produce pro-inflammatory cytokines and completely clear target cancer cells in an in vitro three-dimensional tumor spheroid model.

FT516
FT516 is a universal, off-the-shelf NK cell product candidate manufactured from a clonal master iPSC line engineered to uniformly express a high-affinity, non-cleavable CD16 (hnCD16) Fc receptor. Since CD16 binds the Fc region of tumor-bound antibodies, FT516 can be combined with FDA-approved monoclonal antibody therapies to target a broad spectrum of tumor-associated antigens. In preclinical studies, FT516 exhibits potent and persistent anti-tumor activity in vitro and in vivo against multiple tumor types, including in combination with monoclonal antibody therapies that target CD20, HER2 and EGFR. The Company plans to submit an IND to the FDA by the end of 2018 to evaluate the safety and tolerability of multiple doses of FT516 in multiple dosing cycles in combination with FDA-approved monoclonal antibody therapy.

FT596
FT596 is a universal, off-the-shelf chimeric antigen receptor (CAR) NK cell product candidate that expresses a proprietary CAR targeting CD19, a novel IL-15 receptor fusion for cytokine-independent persistence, and a hnCD16 Fc receptor for augmented antibody-dependent cellular-cytotoxicity (ADCC). A poster presentation at ASH (Free ASH Whitepaper) by scientists from the Company and the University of California – San Diego highlighted new in vivo data demonstrating that FT596 displays long-term persistence without systemic cytokine support. Additionally, FT596 prevents tumor progression and promotes sustained long-term survival in a B-cell leukemia xenograft model. Moreover, as proof-of-concept for the mitigation of antigen escape, FT596 in combination with rituximab completely eliminates CD19+ and CD19- B-cell tumor cells in a co-culture cytotoxicity assay.

FT538
FT538 is a universal, off-the-shelf NK cell product candidate that expresses both a novel IL-15 receptor fusion for cytokine-independent persistence and a hnCD16 Fc receptor for augmented ADCC, and additionally lacks CD38 expression to eliminate fratricide when combined with CD38-targeting monoclonal antibody therapy. A poster presentation at ASH (Free ASH Whitepaper) by scientists from the Company and the University of Minnesota highlighted new in vitro data showing that FT538 in combination with daratumumab is resistant to fratricide and eliminates multiple myeloma cancer cells in a serial re-challenge assay. The Company is investigating the additional therapeutic benefit of including a novel humanized CAR targeting B-cell Maturation Antigen (BCMA) as a dual-targeting mechanism to address multiple myeloma and other BCMA-positive malignancies.

FT819
FT819 is an off-the-shelf, TCR-less CD19 CAR T-cell product candidate manufactured from a clonal master iPSC line that is undergoing preclinical development under a collaboration with Memorial Sloan Kettering Cancer Center (MSK) led by Dr. Sadelain. FT819 is engineered to completely eliminate expression of the T-cell receptor and to insert a next-generation CAR receptor into the T-cell receptor alpha locus, a strategy which is intended to convey enhanced safety and efficacy while eliminating the possibility of graft-versus-host disease. A poster presentation at ASH (Free ASH Whitepaper) by scientists from the Company and MSK showcased new in vivo data demonstrating that FT819, when thawed and directly infused, effectively controls tumor progression comparable to peripheral blood CAR T cells in a preclinical mouse model of acute lymphoblastic leukemia.

A copy of the Company’s presentation from its ASH (Free ASH Whitepaper) investor event can be accessed under "Events & Presentations" in the Investors & Media section of the Company’s website at www.fatetherapeutics.com.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary iPSC product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered in repeat doses to mediate more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event, and selecting a single iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf to treat many patients. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 100 issued patents and 100 pending patent applications.

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