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CAR-T Cell Therapy Receives FDA Breakthrough Designation – Pharmaceutical Processing

Novartis CAR-T cell therapy CTL019 receives FDA Breakthrough Therapy designation for treatment of adult patients with r/r DLBCL.

Novartis announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to CTL019, an investigational chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of adult patients with relapsed and refractory (r/r) diffuse large B-cell lymphoma (DLBCL), who have failed two or more prior therapies.

This is the second indication for which CTL019 has received this designation; the first being for the treatment of r/r B-cell acute lymphoblastic leukemia (ALL) in pediatric and young adult patients.

“At Novartis, we are eager to unlock the full potential of CTL019, including the potential to help patients with r/r DLBCL,” said Vas Narasimhan, Global Head of Drug Development and Chief Medical Officer, Novartis. “We look forward to working closely with the FDA to help bring this potential new treatment option to patients as soon as possible.”

CAR-T cell therapy is different from typical small molecule or biologic therapies currently on the market because it is manufactured for each individual patient. During the treatment process, T cells are drawn from a patient’s blood and reprogrammed in the laboratory to create T cells that are genetically coded to hunt the patient’s cancer cells and other B-cells expressing a particular antigen.

CTL019 was first developed by the University of Pennsylvania (Penn). In 2012, Novartis and Penn entered into a global collaboration to further research, develop and then commercialize CAR-T cell therapies, including CTL019, for the investigational treatment of cancers. Through the collaboration, Novartis holds the worldwide rights to CARs developed with Penn for all cancer indications.

In March 2017, Novartis announced that the FDA accepted the company’s Biologics License Application filing and granted priority review for CTL019 in the treatment of r/r pediatric and young adult patients with B-cell ALL.

The Breakthrough Therapy designation is based on data from the multi-center phase II JULIET study (NCT02445248), which is evaluating the efficacy and safety of CTL019 in adult patients with r/r DLBCL. JULIET is the second global CAR-T trial, following the Novartis ELIANA study (NCT02435849) investigating CTL019 in r/r B-cell ALL. Findings from JULIET are expected to be presented at an upcoming medical congress.

“We are encouraged by the FDA’s recognition in the potential of CTL019 for this indication, which follows our promising studies of this therapy for ALL and the FDA filing by Novartis in pediatric and young adult ALL that received priority review,” said the Penn team’s leader, Carl June, M.D., director of the Center for Cellular Immunotherapies in the Perelman School of Medicine at the University of Pennsylvania. “Work with our collaborators at trial sites across the world is paving a path to bring personalized cell therapies to more patients with these devastating blood cancers.”

According to FDA guidelines, treatments that receive Breakthrough Therapy designation are those that treat a serious or life threatening disease or condition and demonstrate a substantial improvement over existing therapies on one or more clinically significant endpoints based on preliminary clinical evidence. The designation also indicates that the agency will expedite the development and review of CTL019 in adults with r/r DLBCL.

This marks the 14thBreakthrough Therapy designation for Novartis since the FDA initiated the program in 2013, underscoring an emphasis to develop innovative treatments in disease areas with significant unmet need.

DLBCL is the most common form of lymphoma and accounts for approximately 30 percent of all non-Hodgkin lymphoma cases1. Ten to 15 percent of DLBCL patients fail to respond to initial therapy or relapse within three months of treatment, and an additional 20 to 25 percent relapse after initial response to therapy2.

Because CTL019 is an investigational therapy, the safety and efficacy profile has not yet been established. Access to investigational therapies is available only through carefully controlled and monitored clinical trials. These trials are designed to better understand the potential benefits and risks of the therapy. Because of the uncertainty of clinical trials, there is no guarantee that CTL019 will ever be commercially available anywhere in the world. ____________________________________________________

References:

1 American Society of Clinical Oncology. Lymphoma – Non-Hodgkin: Subtypes (Dec. 2016 revision).http://www.cancer.net/cancer-types/lymphoma-non-hodgkin/subtypes. Accessed March 2017.

2 Sehn, L. Paramount prognostic factors that guide therapeutic strategies in diffuse large B-cell lymphoma. Hematology, December 2012; 1; 402-409.

(Source: GlobeNewswire)

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PolarityTE(TM), Inc. Enters Formal Agreement with Cell Therapy and … – Yahoo Finance

SALT LAKE CITY, UT–(Marketwired – Apr 20, 2017) – PolarityTE, Inc. (“Polarity”) ( NASDAQ : COOL ) announces the signing of a manufacturing agreement with Cell Therapy and Regenerative Medicine (“CTRM”) at the University of Utah School of Medicine. CTRM is the established manufacturer of hematopoietic stem cell transplants for renowned institutions of the Salt Lake region such as the Huntsman Cancer Institute and Primary Children’s Hospital. In addition, CTRM manufactures a variety of regenerative medicine products, is FACT accredited, and has technical expertise in current Good Tissue Practice (cGTP) and current Good Manufacturing Practice (cGMP).

Denver Lough, MD, PhD, Chairman and CEO, stated, “This agreement with CTRM should provide PolarityTE with rapid clinical translation of the promising products we are developing, and takes us one step closer to achieving our goal of clinical application of our launch product, SkinTE. As we prepare for market entry in 2018, our established relationship with CTRM creates a springboard for the anticipated scale-up to address the large burn and chronic wound markets, with the pursuit of our own independent manufacturing facility. Plans are solidifying for a unique solution to both commercial and emergent relief manufacturing of SkinTE and future Polarity products. Our goal is not only to meet demand, but also to be able to deliver promptly around the globe when urgent response is needed.”

About Cell Therapy and Regenerative Medicine (CTRM) CTRM at the University of Utah is engaged in delivering some of the world’s most advanced cellular therapies to patients.CTRM provides unique resources and expertise that feworganizations can internally create to achieve large scale manufacturing of cell- and tissue-based products for clinical trials.Highly experienced CTRM staff support the Blood and Marrow Transplant (BMT) Programs at Huntsman Cancer Institute and Primary Children’s Hospital to deliver high quality stem cell products for the treatment of adult and pediatric patients diagnosed with leukemia, lymphoma, aplastic anemia and other types of blood diseases and disorders.CTRM is also developing and providing novel clinical grade cellular and tissue engineered products via comprehensive “bench” to “bedside” services that coordinate efforts of clinicians, entrepreneurs, researchers and bioengineers. CTRM with its industry partners is translating some of the newest cellular and tissue based discoveries into clinical applications to extend and improve the quality of life for individuals suffering from debilitating diseases and injuries.For more information go to http://www.medicine.utah.edu/cell.

About PolarityTE, Inc. PolarityTE, Inc. is the owner of a novel regenerative medicine and tissue engineering platform developed and patented by Denver Lough, MD, PhD. This radical and proprietary technology employs a patient’s own cells for the healing of full-thickness functionally-polarized tissues. If clinically successful, the PolarityTE platform will be able to provide medical professionals with a truly new paradigm in wound healing and reconstructive surgery by utilizing a patient’s own tissue substrates for the regeneration of skin, bone, muscle, cartilage, fat, blood vessels and nerves. It is because PolarityTE uses a natural and biologically sound platform technology, which is readily adaptable to a wide spectrum of organ and tissue systems, that the company and its world-renowned clinical advisory board, are poised to drastically change the field and future of translational regenerative medicine. More information can be found online at http://www.polarityte.com.

Forward Looking Statements Certain statements contained in this release are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward looking statements contained in this release relate to, among other things, the Company’s ongoing compliance with the requirements of The NASDAQ Stock Market and the Company’s ability to maintain the closing bid price requirements of The NASDAQ Stock Market on a post reverse split basis. They are generally identified by words such as “believes,” “may,” “expects,” “anticipates,” “should'” and similar expressions. Readers should not place undue reliance on such forward-looking statements, which are based upon the Company’s beliefs and assumptions as of the date of this release. The Company’s actual results could differ materially due to risk factors and other items described in more detail in the “Risk Factors” section of the Company’s Annual Reports and other filings with the SEC (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. The Company specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

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HER2-Specific CAR T-Cell Therapy Active in Progressive … – Cancer Network

Administration of autologous HER2-specific chimeric antigen receptor (CAR)-modified virus specific T Cells (VSTs) was safe and had clinical benefit for some patients with progressive glioblastoma, a disease with limited effective therapeutic options.

Results of a small phase I study of this monotherapy were published in JAMA Oncology, by Nabil Ahmed, MD, MPH, of Baylor College of Medicine in Houston, and colleagues.

CAR T-cell therapies are an attractive strategy to improve the outcomes for patients with glioblastoma, they wrote. In our study, we infused HER2-CAR VSTs intravenously because T cells can travel to the brain after intravenous injections, as evidenced by clinical responses after the infusion of tumor-infiltrating lymphocytes for melanoma brain metastasis and by detection of CD19-CAR T cells in the cerebrospinal fluid of patients with B-precursor leukemia.

The study included 17 patients with progressive HER2-positive glioblastoma (10 patients aged 18 or older; 7 patients younger than 18). Patients were given one or more infusions of autologous VSTs specific for cytomegalovirus, Epstein-Barr virus, or adenovirus and genetically modified to express HER2-CARs. Six patients were given multiple infusions.

Infusions were well tolerated with no dose limiting toxicities presenting. Two patients had grade 2 seizures and/or headaches, which the researchers wrote were probably related to the T-cell infusion.

Although HER2-CAR VSTs did not expand, they were detected in the peripheral blood for up to 12 months after the infusion.

Although we did not observe an expansion of HER2-CAR VSTs in the peripheral blood, T cells could have expanded at glioblastoma sites. At 6 weeks after T-cell infusion, the MRI scans of patients 3, 7, 10, 16, and 17 showed an increase in peritumoral edema, the researchers wrote. Although these patients were classified as having a progressive disease, it is likely that the imaging changes for some of these patients were due to inflammatory responses, indicative of local T-cell expansion, especially since these patients survived for more than 6 months.

Only 16 of the 17 patients were evaluable for response. Patients underwent brain MRI 6 weeks after T-cell infusion. One patient had a partial response for longer than 9 months and seven patients had stable disease for between 8 weeks to 29 months. Three patients with stable disease are alive without any evidence of progression from 24 to 29 months of follow-up. Eight patients progressed after the infusion.

The median overall survival was 11.1 months from the first T-cell infusion and 24.5 months from diagnosis.

The researchers noted that the inclusion of children in the study, who have a better prognosis than adults, may have affected the results; however, there was no significant difference between the survival probability for children and that for adults in this clinical study.

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Henrietta Lacks’ Cells May Be Responsible For The Future Of Medicine – Huffington Post

When Henrietta Lacks, a 31-year-old black woman from Virginia,sought treatment for stomach pain at Baltimores Johns Hopkins Hospital in 1951, doctors discovered a fast-growing cancerous tumor on Lacks cervix.

Doctors harvested Lacks cells without her permission during surgery a clear ethical violation today in the hopes of using them for scientific research.Those same cells continued to replicate long after her death from cervical cancer, however, and they fueled some of the most noteworthy scientific advancements in modern medicine.

Now The Immortal Life of Henrietta Lacks,a movie staring television personality Oprah Winfrey, based on the 2010 book of the same name, seeks to cement Lacks place in medical history.

I am a student of the African American culture … I have never, in all of my readings, in all of my stories, heard of HeLa or Henrietta Lacks, Oprah said at press event in April. I could not believe that. How could I have been in this town all this time and never seen one thing about her?

For a snapshot of how influential Lacks cells, also called HeLa cells, have been on science, look no further than PubMed, the National Institutes of Healths online library for medical research. SearchingHeLanets more than 90,000 results. Indeed, HeLas influence is so widespread, involved and often, interconnected, thats its impossible to pinpoint its scope.

Heres how HeLa will influence the future of medicine:

Indeed, HeLa cells have been a cornerstone of research for Andrew Adey, assistant professor of molecular and medical genetics at Oregon Health and Science University, where he does cancer research.

Because HeLa cells are extremely well documented, Adey uses them as a control for the cancer-detecting technologies his group develops.

We use HeLa cells to calibrate and refine our technologies because we know exactly which mutations are present, he told The Huffington Post.Just about every single technology we work on developing, we use HeLa cells first.

Its not possible to quantify how many lives have been saved from research based on HeLa cells, but one measure, in combination with gains in preventative medicine, might point to the scope:Deaths from cervical cancer, the disease that took Lacks life,declined more than 60 percent between 1955 and 1992, according to the NIH.

HeLa cell research has also had an immense influence on health beyond cancer, contributing also to the development of the polio vaccine and helping to map the human genome.

The number of lives saved by research that utilized HeLa is most likely in the millions, Adey said.

HeLa-based research laid the groundwork for whats known as precision medicine: treatments tailored to an individuals environment, lifestyle and genes, rather than a one-size-fits-all prescription.

Precision medicine is regarded by some oncologists as the future of cancer treatment. One example is immunotherapy, in which scientists stimulate a patients immune system to treat his or her cancer. The stimulation can range from drugs to vaccines to cell transfers.

This type of research had been championed by former President Barack Obama, who allotted $215 million for a Precision Medicine Initiative in his 2016 budget.

HeLa cells continue to be a major tool in many laboratories focused on the development of cancer drugs, said Patricia Thompson-Carino,a pathology professor at Stony Brook School of Medicine.

HeLa cells have bolstered scientists knowledge about cancer treatment resistance and helped doctors to better match cancer drugs to patients. And although immunotherapy is multifaceted, and certainly doesnt work for everyone, former President Jimmy Carters immune system-boosting melanoma treatmentin 2015 is one notable success.

Today, HeLas not the only immortal cell line aiding researchers, but it arguably paved the way for those lines and the immeasurable medical innovation that followed.

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PolarityTE (COOL) Signs Manufacturing Agreement with Cell Therapy and Regenerative Medicine – StreetInsider.com

Get daily under-the-radar research with StreetInsider.com’s Stealth Growth Insider Get your 2-Wk Free Trial here.

PolarityTE, Inc. (NASDAQ: COOL) announces the signing of a manufacturing agreement with Cell Therapy and Regenerative Medicine at the University of Utah School of Medicine. CTRM is the established manufacturer of hematopoietic stem cell transplants for renowned institutions of the Salt Lake region such as the Huntsman Cancer Institute and Primary Children’s Hospital. In addition, CTRM manufactures a variety of regenerative medicine products, is FACT accredited, and has technical expertise in current Good Tissue Practice (cGTP) and current Good Manufacturing Practice (cGMP).

Denver Lough, MD, PhD, Chairman and CEO, stated, “This agreement with CTRM should provide PolarityTE with rapid clinical translation of the promising products we are developing, and takes us one step closer to achieving our goal of clinical application of our launch product, SkinTE. As we prepare for market entry in 2018, our established relationship with CTRM creates a springboard for the anticipated scale-up to address the large burn and chronic wound markets, with the pursuit of our own independent manufacturing facility. Plans are solidifying for a unique solution to both commercial and emergent relief manufacturing of SkinTE and future Polarity products. Our goal is not only to meet demand, but also to be able to deliver promptly around the globe when urgent response is needed.”

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PolarityTE (COOL) Signs Manufacturing Agreement with Cell Therapy and Regenerative Medicine – StreetInsider.com

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