On June 12, 2018 Asterias Biotherapeutics, Inc. (NYSE MKT:AST), a biotechnology company dedicated to developing cellular immunotherapies to treat cancer and cell-based therapeutics to treat neurological conditions associated with demyelination, reported enrollment and dosing of the first subject in the first-in-human Phase 1 clinical trial of AST-VAC2 in the United Kingdom (Press release, Asterias Biotherapeutics, JUN 12, 2018, View Source;date=June+12%2C+2018&title=Asterias+Biotherapeutics+Announces+First+Patient+Dosed+in+First-in-Human+Clinical+Study+of+Immunotherapy+AST-VAC2+in+Non-Small+Cell+Lung+Cancer [SID1234527282]). This initial clinical trial, which is being sponsored, managed and funded by Cancer Research UK, will examine the safety and tolerability of AST-VAC2 in non-small cell lung cancer (NSCLC) as the study’s primary endpoints. Secondary and tertiary endpoints of the study include evaluations of the immunogenicity of AST-VAC2 in NSCLC.

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"We are excited to have launched this clinical trial using AST-VAC2 for NSCLC," said the trial’s chief investigator Professor Christian Ottensmeier MD, PhD, FRCP, Professor of Experimental Medicine within Medicine at the University of Southampton. "The study will allow us to demonstrate the safety and immunogenicity of what I believe is a groundbreaking dendritic cell technology. The approach has the potential to redefine the way we use dendritic cell vaccines in the clinic and may be applicable to many cancer indications."

"We are thankful for Cancer Research UK’s sponsoring the clinical trial and look forward to advancing our immunotherapy strategy around this trial and the earlier Phase 2 trial of AST-VAC1 in Acute Myeloid Leukemia (AML)," said Michael Mulroy, President and Chief Executive Officer of Asterias. "AST-VAC2 is an allogeneic approach that has the potential to avoid many of the issues that autologous therapies face today. We are evaluating further development of AST-VAC2 as a monotherapy or in combination with other therapies in various cancer indications that may benefit from this therapy."

"We’re thrilled to be working with Asterias to bring this novel immunotherapy to patients with lung cancer. This cell therapy has massive potential," said Dr Nigel Blackburn, Cancer Research UK’s director of drug development. "Through our innovative Clinical Development Partnership scheme we have been able to offer our expertise in drug development and clinical trials management to drive this experimental treatment into the clinic."

AST-VAC2 is a "first-in-class" allogeneic cancer immunotherapy that is composed of mature dendritic cells which are designed to kill tumor cells by stimulating immune responses to telomerase, a tumor antigen expressed by over 85% of malignant tumor cells, but not by most normal healthy cells. AST-VAC2 is intended to be available for "on demand" patient use because it is produced from allogeneic pluripotent stem cells that can be manufactured in scale and then cryopreserved.

As currently designed, the clinical study will administer AST-VAC2 in up to 24 subjects with a specific immunological marker called HLA-A2, in one of two cohorts depending on the stage of each subject’s NSCLC. In the first cohort, up to 12 subjects with advanced disease will receive AST-VAC2, and will be followed for safety, immune responses to telomerase, and overall clinical survival. The second cohort will evaluate AST-VAC2 in up to 12 early-stage subjects who have had successful resection of their tumor with no evidence of metastasis, and each patient will be followed for safety, immune responses to telomerase, overall clinical survival and time to relapse. Both cohorts will also have a control group consisting of patients that meet all inclusion/exclusion criteria for the study but who do not have the HLA-A2 marker. The supply of AST-VAC2 to be used in this trial is being manufactured by Cancer Research UK’s Biotherapeutics Development Unit.

The partnership between Asterias and Cancer Research UK is being conducted under Cancer Research UK’s Clinical Development Partnerships (CDP) scheme, which allows the first clinical trial of AST-VAC2 to be initiated without significant Asterias resources being allocated to the trial and the manufacturing of the product. On completion of the clinical trial, Asterias will have an exclusive first option to acquire the data from the trial.

The results from the Phase 1 clinical trial sponsored and managed by Cancer Research UK could be used to support advanced clinical studies in one or more of the following areas:

Non-small cell lung cancer
Acute Myeloid Leukemia, leveraging the results of the previous AST-VAC1 trial in AML
Other indications showing high levels of telomerase activity and susceptibility to immunotherapy
In combination with check point or immune pathway inhibitors
In combination with additional antigens, including those arising from the exciting new field of tumor neoantigens
About AST-VAC2

AST-VAC2 is an innovative immunotherapy product that contains mature dendritic cells derived from pluripotent stem cells. These non-patient specific (allogeneic) AST-VAC2 cells are engineered to express a modified form of telomerase, a protein widely expressed in tumor cells, but rarely found in normal cells. The modified form of telomerase invokes enhanced stimulation of immune responses to the protein. Similar to an earlier, Asterias-sponsored, hematological cancer program which provided proof-of-concept data, the AST-VAC2 dendritic cells instruct the immune system to generate responses against telomerase and, through this mechanism, target tumor cells. AST-VAC2 is based on a specific mode of action that is complementary to and potentially synergistic with other immune therapies such as checkpoint inhibitors or other immune pathway inhibitors.

About Non-Small Cell Lung Cancer and the AST-VAC2 Trial

Lung cancer (both small cell and non-small cell) is the leading cause of cancer-related death, accounting for about one-quarter of all cancer deaths and more than colorectal, breast, and prostate cancers combined. Non-small cell lung cancer (NSCLC) accounts for about 80% to 85% of lung cancers, according to the American Cancer Society. The three main types of NSCLC are adenocarcinoma, squamous cell carcinoma, and large cell carcinoma. The American Cancer Society’s estimates for lung cancer in the United States for 2017 are: about 222,500 new cases of lung cancer, and about 155,870 deaths from lung cancer. Despite the large number of people afflicted by non-small cell lung cancer, patients remain vastly underserved due to a scarcity of effective treatments. According to statistics published by Cancer Research UK, the five year survival rate for lung cancer patients in England and Wales is less than 10%.

As currently designed, the AST-VAC2 Phase 1 clinical trial will enroll up to twenty-four subjects into one of two cohorts, depending on the stage of their non-small cell lung cancer. The first cohort will evaluate AST-VAC2 in up to 12 subjects with advanced non-small cell lung cancer. Subjects in this cohort, who carry the major histocompatibility gene, HLA-A2, will receive six weekly injections of AST-VAC2 and will be followed for safety, immune responses to telomerase and overall clinical survival. These survival results will be compared directly to a control group who meet all of the other inclusion/exclusion criteria but do not possess the HLA-A2 gene. Assuming safety is demonstrated in the first cohort, enrollment will advance to a second cohort. In the second cohort, early stage subjects who have had successful resection of their tumor with no evidence of metastasis will be enrolled. Up to 12 subjects in this second cohort who carry the major histocompatibility allele HLA-A2 will receive six, weekly injections of AST-VAC2 and will be followed for safety, immune responses to telomerase, overall clinical survival and time to relapse. These survival results will again be compared directly to a control group who meet all of the inclusion/exclusion criteria of cohort 2 but are not HLA-A2+. Subjects will be followed for one year for immune response to telomerase and for 2 years for the survival endpoints. Asterias and Cancer Research UK are exploring the combination of AST-VAC2 with an immune pathway inhibitor.

On June 12, 2018 Agilent Technologies Inc. (NYSE: A) reported that the U.S. Food and Drug Administration (FDA) has approved its Dako PD-L1 IHC 22C3 pharmDx assay for expanded use (Press release, Agilent, JUN 12, 2018, http://www.agilent.com/about/newsroom/presrel/2018/12jun-ca18057.html [SID1234527279]).

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Physicians in the United States can now gain valuable information to help them identify cervical cancer patients who are most likely to benefit from treatment with KEYTRUDA, an anti-PD1 immunotherapy manufactured by Merck (known as MSD outside the United States and Canada).

PD-L1 IHC 22C3 pharmDx is now the first FDA-approved IHC test for determining PD-L1 expression in cervical cancer and is the first FDA-approved companion diagnostic to identify patients with cervical cancer for treatment with KEYTRUDA. This follows an initial FDA approval for PD-L1 IHC 22C3 pharmDx in non-small cell lung cancer and a subsequent expanded approval to include gastric or gastroesophageal junction adenocarcinoma.

"PD-L1 is a critical biomarker for identifying patients who are likely to derive benefit from anti-PD-1 immunotherapy, and with an increasingly complex marketplace, pathologists look to Agilent as the clear leader to provide accurate and reliable PD-L1 testing," said Sam Raha, president of Agilent’s Diagnostics and Genomics Group. "This expansion of use for the Dako PD-L1 IHC 22C3 pharmDx assay gives patients with cervical cancer the possibility of having their tumor sample tested for PD-L1 expression, and determining eligibility for treatment with KEYTRUDA."

Cervical cancer is the third most common gynecologic cancer in the United States, with 13,000 cases expected to be diagnosed this year alone. The overall survival rate has not improved in the past 40 years, despite advancements in prevention and early detection. Cervical cancer patients previously had few treatment options other than highly toxic chemotherapy.

KEYTRUDA is a humanized monoclonal antibody that increases the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes, which may affect both tumor cells and healthy cells. KEYTRUDA and other targeted immunotherapies are revolutionizing cancer treatment, with their therapeutic value being demonstrated across a growing list of cancer types.

Agilent is a worldwide leader in partnering with pharmaceutical companies to develop immunohistochemical-based diagnostics for cancer therapy. Agilent developed PD-L1 IHC 22C3 pharmDx in partnership with Merck & Co.

On June 12, 2018 Galectin Therapeutics Inc. (NASDAQ:GALT), the leading developer of therapeutics that target galectin proteins, reported that Harold H. Shlevin, Ph.D., currently the Company’s Chief Operating Officer (COO), has been appointed Chief Executive Officer (CEO) and President to succeed Peter G. Traber, M.D., who has tendered his resignation as President, CEO and Chief Medical Officer (Press release, Galectin Therapeutics, JUN 12, 2018, View Source [SID1234527278]). The transition will be effective July 6, 2018.

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Board Chairman, Richard Uihlein commented "As the Company enters this next strategic phase, we are extremely pleased that Dr. Harold Shlevin, who has been Galectin’s COO since 2012, and who has extensive healthcare leadership experience, has agreed to take on the broader role of CEO at this critical juncture. Harold has significant business development experience as an executive and will be responsible for directing and overseeing the potential partnering of our NASH Phase 3 compound. Dr. Shlevin will also oversee future NASH cirrhosis trials and is responsible for the Company’s clinical trials in cancer immunotherapy and any other potential new clinical trials in other indications."

"In conjunction with this transition, the Company has also engaged Back Bay Life Science Advisors, a Boston-based, internationally focused integrated strategy and transaction advisory organization to support the Company, and the management team in its exploration of strategic alternatives. After meeting Dr. Jonathan Gertler and his team, I look forward to my continued involvement in this process. Back Bay, management and the Board have earned and deserve my personal attention and full confidence.", added Mr. Uihlein.

Regarding Dr. Traber, Board Chair Richard Uihlein stated, "Peter has been tireless in his efforts in guiding Galectin over the past seven years. As a distinguished scientist and hepatologist, Peter has a keen interest in conquering NASH cirrhosis, and we believe our compound, GR-MD-02, has shown tremendous potential in this regard. Peter’s clinical and scientific vision was critical to the company in reaching its current Phase 3 ready development trajectory. His recent leadership in meeting with the FDA and being allowed to proceed to clinical phase 3 has been indispensable and positions the Company for its next stage of growth and development. We thank him for his many contributions to the Company."

Dr. Shlevin said, "I am extremely grateful for the Board’s confidence in our experienced team, many of whom I recruited to Galectin Therapeutics and worked with for many years prior to joining Galectin. It has been a personal and professional pleasure to work with Peter over the last six years, and the Company and I wish him well in his future endeavors."

"We believe our NASH-CX Phase 2 trial was the first large, randomized clinical trial of any drug to demonstrate a clinically meaningful improvement in HVPG in NASH cirrhosis patients. With our management team and other core team members and the Board leadership under Dick Uihlein, I am confident in our ability to build value for our shareholders and advance GR-MD-02 to provide patients a treatment option for dealing with their NASH cirrhosis," concluded Dr. Shlevin.

About Dr. Shlevin:

Dr. Shlevin is a 25-year bioscience-industry executive with broad senior management experience in development and commercialization of medical devices, pharmaceuticals, diagnostics and vaccines. As COO of Galectin Therapeutics, he was responsible for all operational aspects of the company, including regulatory affairs & quality assurance, manufacturing, clinical development, business development and commercial development.

Prior to his work at Galectin, Dr. Shlevin served Georgia Institute of Technology’s Advanced Technology Development Center (ATDC) as manager of bioscience partnering and commercialization efforts. He was also previously President and CEO of Solvay Pharmaceuticals US and a member of Solvay’s global pharmaceutical management team. He has also held senior executive positions at Bausch & Lomb Pharmaceuticals, CIBA Vision Ophthalmics (which he co-founded), and CIBA-Geigy Pharmaceuticals, amongst others. Dr. Shlevin earned a B.A. degree from Boston University and M.S. and Ph.D. degrees in physiology from the University of Rochester Medical School and postdoctoral fellowship at Mayo Foundation and Mayo Medical School.

About NASH Cirrhosis

NASH cirrhosis is the final stage in the progression of non-alcoholic steatohepatitis (NASH), a disease of the liver that affects millions of people in the U.S. and worldwide. The liver cell death and inflammation seen in NASH eventually causes progressive scarring of the liver, which eventually can result in liver cirrhosis. While the early stages of NASH can be treated by changes in lifestyle, such as losing weight and exercising, once the disease progresses to NASH cirrhosis there is no treatment available short of a liver transplant. Of the total number of individuals in the world believed to presently have NASH, it is predicted that NASH cirrhosis will eventually kill 20 million of those people.

One of the results of NASH cirrhosis is an increase in blood pressure in the portal vein that brings blood and nutrients from the digestive tract through the liver and then out to the rest of the body. As the scarring effect of cirrhosis on the liver progresses, blood flow through the liver becomes more difficult, increasing the blood pressure in the portal vein, creating varying degrees of portal hypertension. Eventually, this increase in blood pressure causes the veins connected to the liver to dilate and form esophageal varices, which are dilated veins that divert blood through the esophagus, bypassing flow through the liver. These dilated veins in the esophagus are prone to bleeding, which is a major cause of morbidity and mortality in patients with NASH cirrhosis. About half of the patients with well compensated NASH cirrhosis do not have varices, and identification of these patients is determined by endoscopy, which is included in the standard of care for all patients with cirrhosis.

About GR-MD-02

GR-MD-02 is a complex carbohydrate drug that targets galectin-3, a critical protein in the pathogenesis of fatty liver disease and fibrosis. Galectin-3 plays a major role in diseases that involve scarring of organs including fibrotic disorders of the liver, lung, kidney, heart and vascular system. The drug binds to galectin-3 proteins and disrupts its function. Preclinical data in animals have shown that GR-MD-02 has robust treatment effects in reversing liver fibrosis and cirrhosis.

On June 11, 2018 BridgeBio Pharma reported that it has entered into an agreement with Alexion Pharmaceuticals, Inc. to acquire cyclic pyranopterin monophosphate (cPMP; ALXN1101), a synthetic enzyme co-factor therapy for patients with the ultra-rare disease caused by molybdenum cofactor deficiency (MoCD) Type A (Press release, BridgeBio, JUN 11, 2018, View Source [SID1234576280]). In addition, BridgeBio announced that it was launching a new subsidiary, Origin Biosciences, with sufficient capital to support clinical development of ALXN1101 through potential regulatory approval and commercialization.

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MoCD is an ultra-rare autosomal recessive inborn error of metabolism. The disease is caused by a mutation in the MOCS1 gene and leads to defective production of cPMP. Clinical signs of MoCD present shortly after birth and progress rapidly. Newborns with MoCD experience difficulty feeding and intractable seizures yet have no approved available therapies. Patients have a median survival of three years, and those who survive often have severe and irreversible injury to their central nervous system.

"Historically, replacing missing or defective proteins has proven highly efficacious for treating loss of function monogenic conditions – in the case of MoCD type A, we are replacing the missing or defective cPMP, providing children with much needed MoCD activity," said Michael Henderson, M.D., senior vice president of asset acquisition at BridgeBio. "BridgeBio’s team is committed to continuing the development of ALXN1101 for infants born with MoCD Type A deficiency, their families and caregivers."

ALXN1101 is a synthetic version of cPMP, the missing cofactor causing MoCD Type A. In previous work with a recombinant form of cPMP, 11 patients with MoCD Type A had normalization of biomarkers within two days, eight patients showed some suppression of seizures, and three patients had near-normal development. ALXN1101 has received Breakthrough Therapy designation from the US FDA.

"Patients born with MoCD face a bleak future, and we will do all we can to pursue the development of this exciting compound, which has the potential to replace the missing enzyme," said Neil Kumar, Ph.D., CEO of BridgeBio. "BridgeBio aims to sustainably pursue even the rarest of diseases, such as MoCD, especially where we can support drug programs that target well described genetic diseases at their source."

While specific terms of the deal have not been disclosed, BridgeBio has committed sufficient resources to Origin Biosciences to enable clinical development, regulatory approval and to support commercialization of ALXN1101. Alexion will receive additional payments upon the realization of development and sales milestones.

On June 11, 2018 Nordic Nanovector ASA (OSE: NANO) reported that a poster reporting the ability of Betalutin (177Lu-lilotomab satetraxetan) to reverse resistance to anti-CD20 treatment in non-Hodgkin’s lymphoma (NHL) cell lines will be presented at the inaugural AACR (Free AACR Whitepaper) International Meeting: Advances in Malignant Lymphoma (22-26 June, Boston, MA, USA) (Press release, Nordic Nanovector, JUN 11, 2018, View Source [SID1234553500]).

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The preclinical data, described in an abstract published online (link here), demonstrate that treatment of rituximab-resistant NHL cells with Betalutin significantly elevated the expression of the CD20 receptor on the surface of cells. The increase in CD20 receptors re-sensitizes the cells to the anti-CD20 NHL immunotherapies rituximab (Rituxan) and obinutuzumab (Gazyva/Gazyvaro), causing increased tumour cell death.

These results support previous preclinical studies that highlight the synergistic anti-tumour effects of combining Betalutin with rituximab immunotherapy. Nordic Nanovector is planning to investigate this novel combination therapy in patients with relapsed/refractory follicular lymphoma in the Archer-1 Phase 1b clinical study. This study is targeting dosing of the first patient in the second half of 2018.

Details for the poster presentation are as follows:

Poster Title: 177Lu-lilotomab satetraxetan has the potential to counteract resistance to rituximab and obinutuzumab in non-Hodgkin’s lymphoma Session

Date and Time: Saturday 23 June, 11:45 AM to 1:45 PM (Eastern Daylight Time)

Poster Session A: Basic and Translational Science 1

Location: Salon F, 4th floor, Boston Marriot Copley Plaza Permanent

Abstract Number: A28

The poster will be available on the company’s website at the time of the presentation: www.nordicnanovector.com.