NantKwest Announces Durable Complete Responses in Phase I … – Business Wire (press release)

CULVER CITY, Calif.--(BUSINESS WIRE)--NantKwest Inc. (Nasdaq:NK), a pioneering, next-generation, clinical-stage immunotherapy company focused on harnessing the unique power of the immune system using natural killer (NK) cells to treat cancer, infectious diseases, and inflammatory diseases, today announcedthe final results of a phase Iclinical trial of the companys aNK cell therapyplatforminrelapsed hematological malignancies.

The datapublished in thejournalOncotargetbyinvestigatorsat Princess Margaret Cancer Centre in Toronto, Canada,demonstratedcontinuing evidence of safety and efficacy, with an overall response rate of 42% and no evidence of grade 3 or 4 adverse events from the infusions. Of note 2 out of the 12 patients in the safety study with relapsed Hodgkin's Lymphoma and Multiple Myeloma, demonstrated durable complete response with single agent aNK therapy, and remain free of disease to date, 10 years and 2 years respectively.

Our study was designed to assess safety andpreliminary evidence ofefficacy in patients with relapsed, refractory hematological malignancies whose disease recurred afterautologoushematopoieticcell transplantation (AHCT).In this heavilypretreated patient population that hasa particularly poor prognosis, wedemonstrated safetywithminimal toxicityand showed preliminary evidence of efficacy,takingadvantage of the unique properties of natural killer (NK) cells as an immunotherapeuticagent,said Armand Keating, MD, FRCP(C),Director, Cell Therapy Program,Princess Margaret Cancer Centreand University Health Network.

Dr. Keating continued, In this clinical study completed in 2015 of 12 patients with lymphoma and multiple myeloma who had relapsed after AHCT for refractory/relapsed disease, we report encouraging results with four patients (33%) currently alive and two complete responses, one for over two years and the second for over 10 years and conclude that aNK cell therapy warrants further clinical investigation.

Patrick Soon-Shiong, Chairman and CEO of NantKwestcommented, Consistent with previous studies,Dr. Keatingsclinical trial results, reporting a 42% overall response rate, provide additional clinical validation of the unique potentialtodeliverlong-term remissions withlimitedtoxicityusing the companys novelNK cell therapy.OuraNK cell therapy iscurrently in an ongoing PhaseII clinical study in Merkel cell carcinoma andrepresenta critical,foundational component in the companys recently launched NANT Cancer Vaccine clinical trialprogram.

Dr. Soon-Shiong continued, We believe ourproduct portfolio ofnoveloff-the-shelfNK cell therapiesis unique inofferinga more uniform, consistentand optimized product potency with minimaltoxicityand we remainfocused ontranslating these unique advantages to patient careas rapidly as possible.

aNKCell Therapy Platform

NantKwests aNK cell therapy platformis being developed asan allogeneic, off-the-shelf therapy,offering a potent, standardized, uniform and consistent product, further optimized to enhance the key capability of natural killer cells to directly target and kill cancer cells.This novel cell therapy iscurrentlyinan ongoing Phase II clinical trial in Merkel cell carcinomaand is a critical, foundationalcomponent in the companys recently launch NANT Cancer Vaccine clinical trialprogram.

About NANT Cancer Vaccine

The NANT Cancer Vaccine is the first combination immunotherapy protocol to orchestrate the delivery of metronomic low dose radiation and chemotherapy with molecularly informed tumor associated antigen vaccines and natural killer cells, to activate the innate and adaptive immune system and to induce immunogenic cell death. By inducing immunogenic celldeath and protecting as well as enhancing the innate and adaptive immune system, the NANT Cancer Vaccine seeks to attain long-term sustainable remission of multiple tumor types with lower toxicity and higher efficacy than current standards of care.

About NantKwest

NantKwest(Nasdaq:NK) is a pioneering, next generation, clinical-stage immunotherapy company focused on harnessing the unique power of our immune system using natural killer (NK) cells to treat cancer, infectious diseases and inflammatory diseases. NK cells are the bodys first line of defense due to the innate ability of NK cells to rapidly identify and destroy cells under stress, such as cancer or virally-infected cells.

NantKwests unique NK cell-based platform, with the capacity to grow active killer cells as a biological cancer therapy, has been designed to induce cell death against cancer or infected cells by three different modes of action: (1) Direct killing using activated NK cells (aNK) that release toxic granules directly into the cell through cell to cell contact, (2) Antibody-mediated killing usinghaNKs, which are NK cells engineered to incorporate a high affinity receptor that binds to an administered antibody, enhancing the cancer cell killing effect of that antibody, and (3) Targeted activated killing usingtaNKs, which are NK cells engineered to incorporate chimeric antigen receptors (CARs) to target tumor-specific antigens found on the surface of cancer cells.

Our aNK, haNK and taNK platform addresses certain limitations of T cell therapies including the reduction of risk of serious "cytokine storms" reported after T cell therapy. As an off-the-shelf therapy, NantKwest's NK cells do not rely on a patients own often compromised immune system. In Phase 1 clinical trials in patients with late stage cancer, NantKwest's NK cells have been successfully administered as an outpatient infusion therapy without any reported severe side effects, even at doses of 10 billion cells.

By leveraging an integrated and extensive genomics and transcriptomics discovery and development engine, together with a pipeline of multiple, clinical-stage, immuno-oncology programs that include a Phase 2 trial for a rare form of melanoma and the planned initiation of a clinical trial of NK cells targeted to breast cancer, we believe NantKwest is uniquely positioned to be the premier immunotherapy company and transform medicine by delivering living drugs in a bag and bringing novel NK cell-based therapies to routine clinical care. For more information please visitwww.nantkwest.com.

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NantKwest Announces Durable Complete Responses in Phase I ... - Business Wire (press release)

Gamida Cell Receives $3.5M Grant from Israeli Government to Help Develop CordIn Therapy – Sickle Cell Anemia News

The Israeli government has awardedGamida Cella $3.5 million grant to advance the development of clinical trials forCordIn, a therapy totreatsickle cell disease (SCD) and thalassemia.

The money,granted by the Ministry of Economy and Industrys Israel Innovation Authority (IIA), will also support further development and salesof NiCord, a breakthrough product for blood cancers such as leukemia and lymphoma.

The grant follows the recent closing of a $40 million financing round and the Jerusalem-based companysrecent expansion to theUnited States, after appointing Dr. Ronit Simantovas the companys new U.S.-headquartered chief medical officer.

CordIn is Gamidas candidate for the treatment of SCD and thalassemia, as well as bone marrow failure syndromes and genetic metabolic diseases.Itsa variation of NiCord, developed as an alternative to bone marrow transplants to treat patients with blood cancers who cannot find a donor with fully matched tissue. The product comes from expandingcells from the umbilical cord.

A bone marrow transplant is the only established cure for SCD. Due to the difficulties in finding a suitable match, only a few hundred patients canbe treated this way.

Clinical trial data has been encouraging so far, showing successful and swift engraftment as well as the curing of disease symptoms in these hard to-engraft patients.

Particularly, interim results from a Phase 1/2 clinical trial (NCT02504619) appear to suggest that CordIn could effectively overcome the engraftment barriers of umbilical cord blood and has the potential to increase access to curative transplants for SCD.

We are very pleased with the Israeli governments ongoing contributions and support in helping to bring Gamida Cells very important products to market, Gamida President and CEO Yael Margolin said in a press release. The grant provides additional funding for the development and commercialization of NiCord, as well as our additional pipeline programs.

The Phase 1/2 trial, launchedin January 2015, marked the first time that a patient with SCD was transplanted with CordIn. The transplant took place at UCSF Benioff Childrens Hospital in Oakland, California. The trial is still recruiting patients in the United States.

For more information, please contact Iddo Peleg in Israel at +972-2-659-5666 orclinicaltrials@gamida-cell.com.Those interested can also call Jean-Hugues Dalle in France at +33 1 40 03 53 88 or jhugues.dalle@gmail.com.

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Cell Design Labs Appoints Roger Sidhu, MD as Chief Medical Officer – GlobeNewswire (press release)

August 01, 2017 08:00 ET | Source: Cell Design Labs

EMERYVILLE, Calif., Aug. 01, 2017 (GLOBE NEWSWIRE) -- Cell Design Labs, Inc. today announced the appointment of Roger Sidhu, M.D., to the newly created position of Chief Medical Officer. Dr. Sidhu will be responsible for the design and development of Cell Design Labs clinical programs as well as providing insight into therapeutic applications for its proprietary CAR-T and adoptive T cell therapies (TCR-T) for the treatment of cancer and other complex diseases. In this new role, Dr. Sidhu will report to Brian Atwood, Chief Executive Officer of Cell Design Labs.

Roger brings extensive experience as he was a leader in the development of Amgens deep hematology/oncology portfolio, said Brian Atwood, Co-Founder, President and Chief Executive Officer of Cell Design Labs. His broad clinical knowledge and established track record will be instrumental in guiding the clinical development of our innovative cell therapies, particularly as we look toward the application of our proprietary synNotch and THROTTLE Switch technology platforms in liquid and solid tumors.

The science and vision of Cell Design Labs management team is quite compelling, said Dr. Sidhu. The opportunity to create next-generation customized cell therapies represents a new era in medicine as this powerful technology platform may play a major role in developing more effective and safer therapeutics for patients with cancer, autoimmune and infectious diseases.

Dr. Sidhu comes to Cell Design Labs from Amgen, where he most recently held the position of Global Product General Manager. In that role, he was responsible for strategy, development and commercialization of Amgens portfolio of early-stage immuno-oncology molecules with a focus on solid tumors. He directed programs across multiple platform technologies including CAR-T cell therapies, antibodies, vaccines and bispecific T-cell engagers (BiTEs). Prior to serving as Global Product General Manager, he held escalating positions within Amgens hematology/oncology department where he provided therapeutic area leadership for a portfolio of hematology/oncology therapeutics including Vectibix, Imlygic, Blincyto, Kyprolis, rilotumumab, trebaninib, Neupogen/Neulasta, Aranesp, Nplate and XGEVA. He has extensive experience leading global product specific partnerships and collaborations. Importantly, he led successful regulatory filings for oncology therapeutics resulting in marketing approval from both the U.S. Food and Drug Administration and the European Medicines Agency. He received his B.S. in Biochemistry from the University of Alberta in Alberta, Canada, his M.D. from Queens University in Ontario, Canada and completed a medical oncology fellowship at the British Columbia Cancer Agency in British Columbia, Canada. He is an author on numerous publications and presentations.

About Cell Design Labs, Inc. Cell Design Labs is a biotherapeutics company pioneering breakthrough science to develop disruptive cell-based therapies. Based on innovative research from Dr. Wendell Lims lab at UC San Francisco, Cell Design Labs leverages the power of the bodys immune system to develop smart, living therapies with the capability to treat our most challenging diseases with unprecedented power, precision, safety and durability. Using its proprietary technology platform for custom cell engineering, Cell Design Labs will develop its own portfolio of anticancer therapies as well as create partnerships with leading oncology companies. Initially focused on cancer, including both hematologic and solid tumors, this broad technology may also have applications in other complex diseases such as autoimmune and degenerative disorders. To learn more about Cell Design Labs, please visit our web site at: http://www.celldesignlabs.com.

synNotch and THROTTLE Switch are trademarks of Cell Design Labs, Inc.

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Cell Design Labs Appoints Roger Sidhu, MD as Chief Medical Officer - GlobeNewswire (press release)

New cancer therapy in clinical trial at Nebraska Medical Center has … – Omaha World-Herald

By the time scans showed that his B-cell non-Hodgkin lymphoma was coming back last fall, James Olson had run through many of his treatment options.

In early January, his doctor recommended that the Kansas City, Missouri, man travel to the Nebraska Medical Center in Omaha to see whether he was a candidate for a new type of therapy still in clinical trials.

In May, Olson, 69, received an infusion of his own immune cells, which had been removed from his body and modified to recognize and attack the cancer. Known as CAR-T or chimeric antigen receptor T-cell therapy, it represents a new way of targeting some cancers beyond that of traditional treatments.

About 10 weeks later, his scans are good and hes got the stamina to mow his lawn and do some house painting. Hes enjoying the little pleasures of life, even seasonal chores like installing a window air conditioning unit at his home.

His doctors at the medical center, meanwhile, hope theyll soon be able to offer CAR-T to more patients.

The trial that Olson participated in still is underway, and the medical center continues to accept new patients. Another version of the treatment has shown so much promise that its been fast-tracked by the Food and Drug Administration and could receive conditional approval late this fall. The medical center is set to participate in an expanded trial for that treatment, possibly as soon as this month.

Recently, an expert panel unanimously endorsed a version of CAR-T for children and young adults with recurring acute lymphoblastic leukemia. If the FDA clears the drug, medical center doctors hope to offer the treatment to young adults up to age 25. Theyd also like to see new trials with that drug or others for older adults with recurring ALL, as the leukemia is known.

These people need something else, and this may be it, said Dr. Matthew Lunning, a hematologist and oncologist with Nebraska Medicine.

The federal panels endorsement came at a time when a number of companies are racing to develop therapies based on the approach, which scientists first began to explore decades ago. If approved by the FDA, the Novartis product endorsed by the panel would become the first gene therapy approved in the United States. In addition to targeting relapsed lymphoma and leukemia, researchers also are beginning to study the therapy for solid tumors, the kind that start in organs or tissue. Leukemia and lymphoma are considered blood cancers.

Dr. Julie Vose, chief of hematology and oncology at Nebraska Medicine, said the therapy offers many bright spots in oncology.

We just have to learn how to use it in each specific disease and in each specific patient, she said, noting that the therapy is not a one-size-fits-all treatment as existing methods have been. We have to personalize that treatment.

But even with FDA approval, she said, the treatments potential side effects, which include fever and flu-like symptoms ranging from mild to extremely severe, mean it can only be done at specialized centers like the medical center. Indeed, one earlier trial was closed because some patients suffered serious complications.

Vose said trials in non-Hodgkin lymphoma all have produced good results, including the trial currently underway at the medical center and eight other sites nationwide.

Nationally, patients involved in the trial have had a complete remission rate of 60 percent after 30 days and 40 percent after 60 days. Officially, the condition is known as refractory diffuse large B-cell non-Hodgkin lymphoma, an aggressive disease that is among about 80 subtypes of non-Hodgkin lymphoma.

These were patients who had failed every other possible treatment, Vose said. And more than half had good response to the treatment.

Treatment for newly diagnosed non-Hodgkin lymphoma and acute lymphoblastic leukemia usually begins with chemotherapy. About 30 percent of non-Hodgkin lymphoma patients and roughly 40 percent of acute lymphoblastic leukemia patients relapse after chemo. The majority of those go on to have a blood or bone marrow transplant, from which about half will relapse.

Patients who relapse after the transplant, or those who arent candidates for a transplant, could be potential candidates for CAR-T therapy.

Unlike traditional gene therapy, however, CAR-T doesnt involve replacing disease-causing genes with healthy ones. Instead, technology is used to reprogram T cells, immune cells that normally help the body fight infection and cancer. In lymphoma patients, however, Vose said, T cells go haywire and dont properly fight cancer.

During treatment, the patients T cells are collected during an outpatient procedure and sent to a lab in California for processing. The patient then receives several days of intense chemotherapy. The modified T cells are returned to Omaha and put back into the patient, who is monitored at the hospital for seven to 10 days. The entire process takes about three weeks.

Olson, who drove to Omaha last week for a checkup, said he didnt really have any side effects from the procedure. I wondered if theyd given me a placebo, said Olson, who spent his working life in real estate and was diagnosed in 2010. He was well aware of the possible risks after reading and signing a 33-page consent form.

The next question, for patients and researchers alike, is how long the treatment will hold.

We dont know how long because this is such a new treatment, Vose said, but many of the patients (who) have been treated over the past year are doing well.

That question, like many in cancer treatment, comes with its own risks and benefits to balance. If half of the lymphoma is gone and it lasts years, Lunning said, thats better than a remission that lasts a month.

This report includes material from the Washington Post and Associated Press.

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New cancer therapy in clinical trial at Nebraska Medical Center has ... - Omaha World-Herald

ViaCyte Announces First Patients Implanted with PEC-Direct Islet Cell Replacement Therapy in International Clinical … – PR Newswire (press release)

The first cohort of type 1 diabetes patients is receiving multiple small-format cell-filled devices called sentinels in order to evaluate safety and implant viability. These sentinel units will be removed at specific time points and examined histologically to provide early insight into the progression of engraftment and maturation into pancreatic islet cells including insulin-producing beta cells. A second cohort of up to 40 patients is expected to begin enrolling later this year to evaluate both safety and efficacy. The primary efficacy measurement in the trial will be the clinically relevant production of insulin, as measured by the insulin biomarker C-peptide, in a patient population that has little to no ability to produce endogenous insulin at the time of enrollment. Other important endpoints will be evaluated including injectable insulin usage and the incidence of hypoglycemic events. ViaCyte's goal is to demonstrate early evidence of efficacy in the first half of 2018 and definitive efficacy 6 to 12 months later.

"Islet transplants have been used to successfully treat patients with unstable, high-risk type 1 diabetes, but the procedure has limitations, including a very limited supply of donor organs and challenges in obtaining reliable and consistent islet preparations," said trial investigator James Shapiro, MD, PhD, FRCSC, Director of the Clinical Islet Transplant Program, University of Alberta. "An effective stem cell-derived islet replacement therapy would solve these issues and has the potential to help a greater number of people."

"Patients with high-risk type 1 diabetes complications, such as hypoglycemia unawareness, are at constant risk of life-threatening low blood glucose," said Jeremy Pettus, MD, investigator in the clinical trial and Assistant Professor of Medicine at UC San Diego. "The PEC-Direct islet cell replacement therapy is designed to help patients with the most urgent medical need."

The PEC-Direct product candidate is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or severe hypoglycemic episodes. It is estimated that about 140,000 people in Canada and the US have such high-risk type 1 diabetes. In addition to providing an unlimited supply of cells for implantation, the PEC-Direct approach has other potential advantages relative to cadaver islet transplants such as delivering a more consistent product preparation under quality-controlled cGMP conditions, and a more straightforward and safe mode of delivery.

The clinical trial is being supported in part by JDRF, the leading global organization funding type 1 diabetes research. "JDRF remains dedicated to accelerating the delivery of beta cell replacement therapies to the T1D community, and we commend ViaCyte in its announcement of the first patients to be implanted with the PEC-Direct islet cell replacement therapy," said Derek Rapp, JDRF President and Chief Executive Officer. "JDRF is excited to support this clinical development given its potential to help those people with type 1 diabetes that need it the most those at high risk of life-threatening acute complications. JDRF and ViaCyte share a continuing commitment to realizing the potential of beta cell replacement strategies to deliver insulin independence without immune suppression for people living with type 1 diabetes, and ultimately, at JDRF we hope this will move us forward in fulfilling our vision of a world without type 1 diabetes."

"There are limited treatment options for patients with high-risk type 1 diabetes to manage life-threatening hypoglycemic episodes," said Paul Laikind, PhD, President and Chief Executive Officer of ViaCyte. "We believe that the PEC-Direct product candidate has the potential to transform the lives of these patients and we are excited to move closer to that goal with the initiation of clinical evaluation announced today. This also represents a step towards a broader application of the technology. We remain fully committed to developing a functional cure for all patients with insulin-requiring diabetes. To that end, we are hard at work on next-generation approaches as well, and expect the work with PEC-Direct to further advance our knowledge and drive progress."

In addition to JDRF, the California Institute for Regenerative Medicine (CIRM)'s Alpha Clinic, the Sanford Stem Cell Clinical Center, the JDRF Canadian Clinical Trials Network (CCTN), the Stem Cell Network, and Alberta Innovates Health Solutions (AIHS) are all providing support for the trial.

About the PEC-Direct Product Candidate The PEC-Direct product candidate delivers stem cell-derived pancreatic progenitor cells, called PEC-01 cells, in a device designed to allow direct vascularization of the cells in the device. After implantation, these cells are expected to become mature human islet tissue including well-regulated beta cells producing insulin on demand. The direct vascularization of the implanted cells is expected to allow for robust and consistent engraftment but will necessitate the use of maintenance immune suppression therapy.

About ViaCyte ViaCyte is a privately-held regenerative medicine company developing novel cell replacement therapies as potential long-term diabetes treatments to reduce the risk of hypoglycemia and diabetes-related complications. ViaCyte's product candidates are based on the derivation of pancreatic progenitor cells from stem cells, which are then implanted in a durable and retrievable cell delivery device. Once implanted and matured, these cells are designed to secrete insulin and other pancreatic hormones in response to blood glucose levels. ViaCyte has two products in clinical development. The PEC-Direct product candidate delivers the pancreatic progenitor cells in a non-immunoprotective device and is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or recurrent severe hypoglycemic episodes. The PEC-Encap (also known as VC-01) product candidate delivers the same pancreatic progenitor cells in an immunoprotective device and is being developed for all patients with diabetes, type 1 and type 2, who use insulin. ViaCyte is headquartered in San Diego, California. The Company is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF. For more information on ViaCyte, please visit http://www.viacyte.com and connect with ViaCyte on Twitter and Facebook.

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ViaCyte Announces First Patients Implanted with PEC-Direct Islet Cell Replacement Therapy in International Clinical ... - PR Newswire (press release)