National Brain Tumor Society and the Brain Science Foundation Partner to Fund Research for Most Common Brain Tumor Type

On February 19, 2019 National Brain Tumor Society (NBTS), a leading nonprofit dedicated to the brain tumor community in the United States, reported a new partnership with the Brain Science Foundation (BSF) (Press release, National Brain Tumor Society, FEB 19, 2019, View Source [SID1234553933]). Under terms of the partnership, the organizations have established the Meningioma Research Fund to raise and grant funds specifically for meningioma research poised to identify and advance treatments aimed at dramatically improving survival and quality of life.

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Meningioma is the most common primary brain tumor, accounting for 37 percent of all brain tumor diagnoses and approximately 30,000 new cases every year in the United States. There are fifteen types of meningiomas presenting a wide range of physical, emotional, and occupational challenges – and can ultimately be life-threatening.

"Given the legacy of the Brain Science Foundation and its association with meningioma, and our shared goals with the National Brain Tumor Society to fund research aimed to advance the development of treatments, it quickly became evident that the community of patients, advocates, and healthcare providers could benefit from a formal collaboration between our respective organizations," says Steven Haley, founder of the Brain Science Foundation. "We believe NBTS’s depth and breadth of assistance, combined with the continuity of the BSF mission on a larger platform will be compelling and welcomed in the community we serve."

The Meningioma Research Fund creates an opportunity for donors specifically interested in supporting research on this tumor type to join together to scale up the impact of their philanthropy.

"Medical research directed toward meningioma is disproportionately small compared to other brain tumor types," says David Arons, Chief Executive Officer, National Brain Tumor Society. "Yet, meningiomas can be life-altering and cause lasting deficits to patients, as the only treatment option for many is invasive brain surgery. This partnership with the Brain Science Foundation and the creation of the Meningioma Research Fund will allow us to invest in best-in-class science and accelerate research that is aimed at advancing better treatment options for these patients."

The Brain Science Foundation was established in 2002 and has gone on to fund nearly $1 million annually in support of outstanding physician-scientists in brain tumor research, with a particular focus on meningioma. To date, BSF-funded researchers have impacted lives through a variety of research programs that have included the creation of advanced diagnostic tools, surgical instruments and techniques, post-operative care protocols and therapies that are less invasive but more effective.

About Meningioma

Meningioma is the most common primary brain tumor, accounting for 37.1 percent of all brain tumor diagnoses. It is estimated that 31,990 individuals will be diagnosed with a meningioma in the United States in 2019. Meningioma is most commonly diagnosed in adults, with an average age at diagnosis of 66, and is more common in women than men. African Americans are significantly more likely than Caucasians to develop meningioma. The 10-year relative survival rate for all meningioma patients is 81.5 percent, but only 53.5 percent for those with a malignant meningioma.

Autolus Therapeutics Announces Updated Results from Ongoing CARPALL Trial of Pediatric Acute Lymphoblastic Leukemia Presented at the EHA 1st European CAR T Cell Meeting in Paris

On February 19, 2019 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer, reported that Professor Persis Amrolia, Consultant in Bone Marrow Transplant at Great Ormond Street Hospital (GOSH) and NIHR Research Professor of Transplantation Immunology at University College London (UCL) Great Ormond Street Institute of Child Health, presented updated data from the ongoing Phase 1 CARPALL trial of AUTO1 in a poster presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 1st European CAR T Cell Meeting held in Paris, France, February 14-16, 2019 (Press release, Autolus, FEB 19, 2019, View Source [SID1234550832]).

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Enrolled patients had a median age of 9 years with a median of 4 lines of prior treatment. Seventeen patients were enrolled, and 14 patients received an infusion of CAR T cells. Ten of 14 patients had relapsed post allogeneic stem cell transplant. Eight patients were treated in second relapse, 5 in >second relapse and 3 had relapsed after prior blinatumomab or inotuzumab therapy. Two patients had ongoing CNS disease at enrollment.

Updated safety results
This data set confirms that AUTO 1 induces no severe cytokine release syndrome (CRS) (Grade 3-5). Nine patients experienced Grade 1 CRS, and 4 patients experienced Grade 2 CRS. No patients required tociluzumab or steroids. As previously reported, one patient experienced Grade 4 neurotoxicity; there were no other reports of severe neurotoxicity (Grade 3-5). The mean cumulative exposure to AUTO1 CAR T cells in the first 28 days as assessed by AUC was 1,721,355 copies/µg DNA. Eleven patients experienced cytopenia that was not resolved by day 28 or recurring after day 28: 3 patients Grades 1-3 and 8 patients Grade 4. Two patients developed significant infections, and 1 patient died from sepsis while in molecular complete response (CR).

Updated efficacy results
With a single dose of CAR T cells at 1 million cells/kg dose, 12/14 (86%) achieved molecular CR. Five patients relapsed with CD19 negative disease. Event free survival (EFS) based on morphological relapse was 67% (CI 34-86%) and 46% (CI 16-72%) and overall survival (OS) was 84% (CI 50-96%) and 63% (CI 27-85%) at 6 and 12 months, respectively.

CAR T cell expansion was observed in all responding patients (N=12), with CAR T cells comprising up to 84% of circulating T cells at the point of maximal expansion. The median persistence of CAR T was 215 days.

The median duration of remission in responding patients was 7.3 months with a median follow-up of 14 months. Five of 14 patients (37%) remain in CR with ongoing persistence of CAR T cells and associated B cell aplasia.

"AUTO1 combines a high molecular CR rate with excellent persistence and a good safety profile in pediatric acute B cell leukemia patients," said Professor Amrolia.

About AUTO1
AUTO1 is a CD19 CAR T cell investigational therapy designed to overcome the limitations in safety – while maintaining similar levels of efficacy – compared to current CD19 CAR T cell therapies. Designed to have a fast target binding off-rate to minimize excessive activation of the programmed T cells, AUTO1 may reduce toxicity and be less prone to T cell exhaustion, which could enhance persistence and improve the T cells’ abilities to engage in serial killing of target cancer cells. In 2018, Autolus signed a license agreement under which Autolus acquired global rights from UCL Business plc (UCLB), the technology-transfer company of UCL, to develop and commercialize AUTO1 for the treatment of B cell malignancies. AUTO1 is currently being evaluated in two Phase 1 studies, one in pediatric ALL and one in adult ALL.

For information about the CARPALL trial, visit View Source

About Pediatric Acute Lymphoblastic Leukemia (ALL)
According to the American Cancer Society, ALL is the most common cancer diagnosed in children, with approximately 3,400 new cases diagnosed in the United States each year. Pediatric ALL occurs when the bone marrow makes too many immature lymphocytes, which are a type of white blood cell. The current standard of care for pediatric ALL patients is combination chemotherapy. Although pediatric patients typically respond well to first-line treatment, 10 to 20% of total patients relapse with chemotherapy-resistant disease, leading to a significant unmet need in pediatric patients with high-risk relapsed or refractory ALL.

Gamida Cell Announces Agreement with Editas Medicine to Evaluate Use of CRISPR Genome Editing Technology in NAM-NK Cells

On February 19, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported an agreement with Editas Medicine, Inc., a leading genome editing company, to evaluate the potential use of Editas Medicine’s CRISPR technology to edit NAM-NK cells, which are natural killer cells that have been expanded using Gamida Cell’s proprietary nicotinamide-based, or NAM, technology (Press release, Gamida Cell, FEB 19, 2019, View Source [SID1234533505]). Through this agreement, the companies aim to discover optimized NAM-NK cells that could be used to improve the treatment of hematologic malignancies (blood cancers) and solid tumors.

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"We are encouraged by the early data generated in the Phase 1 study of NAM-NK as an investigational therapy for patients with non-Hodgkin lymphoma and multiple myeloma, and we are pleased to have the opportunity to accelerate and broaden our NAM-NK research efforts through this agreement," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "By leveraging the collective expertise of the Gamida Cell and Editas Medicine teams, we hope to enhance the efficacy of NAM-NK cells through CRISPR editing and potentially bring life-changing immunotherapy treatments to patients."

"Natural killer cells are increasingly recognized as a potential breakthrough approach to treating various cancers. This agreement with Gamida Cell enables us to combine our industry-leading genome editing platform with Gamida Cell’s proprietary NAM-NK cells in an effort to develop best-in-class cellular medicines," said Charles Albright, Ph.D., chief scientific officer of Editas Medicine.

Under the terms of the agreement, Gamida Cell and Editas Medicine will engage in joint research to evaluate unnamed targets by combining Gamida Cell’s proprietary NAM-based cell expansion technology with Editas Medicine’s CRISPR technology. The research initiative is focused on exploring the potential to edit NAM-NK cells to further optimize their tumor-killing properties, and compare the function of the edited and unedited cells in inducing NK cell tumor clearance.

About NAM-NK
Gamida Cell applied the capabilities of its NAM-based cell expansion technology to highly functional NK cells to develop NAM-NK, an innate immunotherapy for the treatment of hematologic and solid tumors in combination with standard-of-care antibody therapies. NAM-NK addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. NAM-NK is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.1

NAM-NK is an investigational therapy, and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

Epizyme Announces Date of Fourth Quarter and Full Year 2018 Results and Upcoming Presentation at the 8th Annual SVB Leerink Global Health Care Conference

On February 19, 2019 Epizyme, Inc. (NASDAQ: EPZM), a clinical-stage biopharmaceutical company creating novel epigenetic therapies, reported that management will host a conference call in conjunction with the announcement of its fourth quarter and full-year 2018 financial results and present at the 8th Annual Leerink Partners Health Care Conference (Press release, Epizyme, FEB 19, 2019, View Source [SID1234533504]). Details of both events are as follows:

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Fourth Quarter and Full-Year 2018 Financial Results: Management will host a conference call and webcast to discuss its fourth quarter and full year 2018 financial results and other business highlights at 8:30 a.m. ET on Tuesday, February 26, 2019. To participate in the conference call, please dial (877) 844-6886 (domestic) or (970) 315-0315 (international) and refer to conference ID 1088195.
8th Annual SVB Leerink Global Health Care Conference Presentation: Management will present a company overview at the 8th Annual SVB Leerink Global Health Care Conference on Thursday, February 28, 2019 at 2:30 p.m. ET in New York City.
Live webcasts will be available in the investor section of the company’s website at www.epizyme.com. The webcasts also will be archived for 60 days following the call and presentation.

Synthetic Biologics to Report 2018 Year End Operational Highlights and Financial Results on February 27, 2019

On February 19, 2019 Synthetic Biologics, Inc. (NYSE American: SYN), a late-stage clinical company developing therapeutics designed to preserve the microbiome to protect and restore the health of patients reported operational highlights and financial results for the year ended December 31, 2018 on Wednesday, February 27, 2019, and will host a conference call the same day at 4:30 p.m. EST (Press release, Synthetic Biologics, FEB 19, 2019, View Source [SID1234533494]). The dial-in information for the call is as follows:

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U.S. (toll free): 1-888-347-5280
International: +1 412-902-4280

Participants are asked to dial in 15 minutes before the start of the call to register. The call will also be webcast over the Internet at View Source." target="_blank" title="View Source." rel="nofollow">View Source An archived replay of the call will be available for approximately ninety (90) days at the same URL, View Source beginning approximately one hour after the call’s conclusion.