On June 15, 2016 BTG plc (LSE: BTG), the specialist healthcare company, reported that it has completed the acquisition of Galil Medical, a leading provider of cryoablation products for the treatment of kidney and other cancers (Press release, BTG, JUN 15, 2016, View Source [SID:1234513373]).

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BTG announced on 6 May 2016 that it had entered into an agreement to acquire Galil.

On June 15, 2016 Exemplar Genetics reported its President, John Swart, Ph.D., will present today at the 15th Annual World Preclinical Congress during the Preclinical Models in Oncology session (Press release, Exemplar Genetics, JUN 15, 2016, View Source [SID:1234513374]). Dr. Swart’s presentation titled ‘Genetically Engineered Miniswine Models of Cancer’ will provide an overview of Exemplar’s novel ExeGen TP53R167H and ExeGen KRASG12D/+/TP53R167H/+ miniswine research models developed for oncology to help bridge the pre-clinical gap from small animal models to humans.

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Dr. Swart commented, "We believe the development of these new genetically engineered miniature swine models will resolve inadequacies with traditional rodent models of cancer, which while valuable in certain aspects of research, have been inefficient at translating to human clinical trials."

As described in the Journal of Clinical Investigation publication, "Development and translational imaging of a TP53 porcine tumorigenesis model", current cancer research models are informative yet fail to accurately recapitulate human disease, and pre-clinical results utilizing these models often have poor translational value to the clinic. In particular, the lack of a large-animal model that accurately replicates human cancer pathologies has been a significant barrier to the development of effective diagnostics, as well as surgical and therapeutic interventions.

Exemplar’s genetically engineered ExeGen TP53R167H model expresses a mutation in the gene that encodes p53 (TP53), which is orthologous to one commonly found in humans. It is estimated p53 function is compromised in the vast majority of human tumors, through either TP53 gene mutation or alterations targeting the numerous regulators of p53 signaling. Studies with the TP53R167H model demonstrated tumor formation and characteristic chromosomal instability similar to what is seen in humans with mutant p53 alleles. Additionally, Exemplar has developed the ExeGen KRASG12D/+/TP53R167H/+ model which contains a conditional KRAS mutation on the background of TP53-targeted pigs that should allow for the inducement of human-like tumors in a tissue specific manner.

In April 2016, the U.S. Food & Drug Administration (FDA) exercised enforcement discretion in regard to Exemplar’s ExeGen low-density lipoprotein receptor (LDLR) miniswine clearing it for commercial use as a research model. As the first genetically engineered miniswine model reviewed and cleared by the FDA, this powerful investigational platform is available to researchers and drug developers helping forge a more reliable, consistent path from pre-clinical testing through human studies. Exemplar continues to work closely with the FDA to make each of the miniswine research models in its broad, extensive pipeline for use in the evaluation of several human health conditions including rare diseases, cancer, cystic fibrosis, neuromuscular/neurodegenerative disorders, and cardiovascular disease, available to researchers working on solutions for these devastating diseases.

On June 15, 2016 Rgenix, a cancer therapeutics company developing first-in-class drugs targeting novel cancer pathways, reported a $33 million Series B financing led by Novo A/S and Sofinnova Partners, with participation from existing investors including Partnership Fund for New York City, Alexandria Venture Investments, and Conegliano Ventures LP (Press release, Rgenix, JUN 15, 2016, View Source [SID1234523088]). The financing will support clinical development of Rgenix’s lead drug candidates, RGX-104 and RGX-202, as well as further development of its therapeutics pipeline.

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"We are thrilled to have attracted top-tier investors to advance development of our novel cancer therapeutics"

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"We are thrilled to have attracted top-tier investors to advance development of our novel cancer therapeutics," said Masoud Tavazoie, M.D. Ph.D., Chief Executive Officer and co-founder of Rgenix. "This financing validates the potential of our lead immunotherapy RGX-104, which will be entering clinical trials this fall, and also demonstrates the strength of our discovery platform, developed in the laboratory of Rgenix co-founder Dr. Sohail Tavazoie at The Rockefeller University. The funding will enable our team to deliver an innovative therapy to cancer patients while simultaneously pushing forward our pipeline of other novel drug candidates."

Antoine Papiernik, Managing Partner at Sofinnova Partners, commented: "We are very excited to back such a high quality team at Rgenix. We also believe that RGX-104 could revolutionize treatment to cancer patients that today lack effective therapies."

Despite recent advances in cancer therapy, most patients will eventually succumb to their disease due to drug resistance and immune evasion. RGX-104 is a small molecule that reverses immune evasion and drug resistance by targeting immunosuppressive cells in the tumor microenvironment via a novel pathway, resulting in strong anti-tumor activity in several drug-resistant cancer types in pre-clinical models, both as a single agent and in combination with approved immunotherapies such as PD-1 inhibitors. The target of RGX-104 was discovered using Rgenix’s miRNA platform that has yielded several new cancer targets across multiple prevalent cancer types.

"We are excited to support Rgenix’s novel approach to treating cancers of high unmet need," said Nilesh Kumar, Senior Principal of Novo Ventures*. "Rgenix has an exciting platform founded on strong science from The Rockefeller University; the lead program is a first in class opportunity addressing a key mechanism in tumor immunosuppression in various cancer types."

In connection with the financing, Nilesh Kumar of Novo Ventures and Antoine Papiernik of Sofinnova Partners will join the Rgenix Board of Directors together with existing members, including Executive Chairman Eric Rowinsky, M.D., Masoud Tavazoie, M.D. Ph.D., Nancy Chang, Ph.D., and Saeed Tavazoie, Ph.D.

On June 15, 2016 Apogenix, a biopharmaceutical company developing next-generation immuno-oncology therapeutics, reported final topline results from a Phase I trial evaluating APG101 in low to intermediate-1 risk transfusion-dependent patients with myelodysplastic syndromes (MDS) (Press release, Apogenix, JUN 15, 2016, View Source [SID1234524579]). The study showed that APG101 was well tolerated. In addition, the trial showed that APG101 efficiently stimulates erythropoiesis in these patients.

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The single arm Phase I trial enrolled twenty patients with low to intermediate-1 risk MDS who were transfusion dependent. Patients had to be refractory to erythropoietin-stimulating agents (ESAs). The patients were treated with APG101 over a period of three months and followed for an additional six months. An extension of treatment was not intended. The primary objectives of the study were safety and tolerability. Secondary objectives included changes in transfusion frequency and changes in parameters involved in erythropoiesis.

In the study, treatment with APG101 led to a significant decrease in transfusion frequency for more than six months (end of follow up period) in 44% of the patients. In addition, measurements of parameters involved in erythropoiesis (i.e., number and function of progenitor cells) further supported the activity of APG101 in this patient population. This evidence of in vivo activity of APG101 confirms in vitro data recently published (Oncotarget Vol. 7 No. 12, 2016). More details from the final results of the study are being submitted for presentation at a major medical meeting later this year.

"The topline data from this Phase I trial continue to support the activity of APG101 in MDS patients," said Harald Fricke, M.D., Chief Medical Officer. "We were particularly excited to see that APG101 appeared to decrease the number of transfusions required by this very sick patient population. Our next step will be to initiate a Phase II trial in MDS to evaluate APG101 in various doses in combination with an erythropoietin-stimulating agent, and we are currently soliciting input from key opinion leaders on the design of that trial."

About Myelodysplastic Syndromes (MDS)
MDS is a bone marrow disorder that is characterized by ineffective hematopoiesis and can lead to severe anemia. In most cases, the anemia is treated with blood transfusions that eventually result in an iron overload, which can damage the liver and other organs. At the same time, the number of thrombocytes that are responsible for coagulation and the number of leucocytes that are responsible for immune defense significantly decrease in patients with this disorder. As a result, MDS patients frequently suffer from sudden bleeding and life-threatening infections. In addition, they are at risk of developing acute myeloid leukemia, a type of blood cancer.

About APG101
Apogenix’s lead immuno-oncology candidate APG101 is a fully human fusion protein that consists of the extracellular domain of the CD95 receptor and the Fc domain of an IgG antibody. APG101 is being developed for the treatment of solid tumors and malignant hematological diseases. By blocking the CD95 ligand, which negatively regulates erythrocyte production in MDS patients, APG101 directly addresses the cause of the disorder and could thus potentially provide a cure for MDS.

On June 14, 2016 Transgenomic, Inc. (TBIO), (NASDAQ: TBIO), reported plans to expand its ICE COLD-PCR (ICP)-powered cancer assays ten-fold over the next 18 months, targeting a portfolio of more than 200 exons/mutations expected to be available to partners, cancer researchers, drug developers and clinicians by 2018 (Press release, Transgenomic, JUN 14, 2016, View Source [SID:1234513324]). Multiplexed ICP technology’s ultra-high sensitivity down to 0.01% makes it possible to run ICP assays using either tissue, blood or serum samples on any sequencing platform. Recently-released studies indicate high concordance between ICP detection tests and assays produced using conventional technology, with the added benefit that the ICP tests detected mutations in both plasma and tissue samples that were missed by conventional methods.

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The expanded ICP cancer test menu will be released over the course of the next 18 months, with about 10 new exons/mutations expected to be issued every 6-8 weeks. The new tests will available in several formats–for use by TBIO’s partners and licensees, through TBIO’s Biomarker Discovery Services group and Oncology CLIA laboratory, as well as through its ICEme kits, which customers can run on Sanger, NGS and other platforms in their own laboratories. The focus, as with TBIO’s current menu of detection tests and panels, will be on actionable mutations relevant to cancer treatment decisions, with priority given to mutations requested by customers along with those newly validated by cancer researchers. The company also intends to incorporate some of the added mutations into panels for use with specific cancers and tumor types.

TBIO President and CEO Paul Kinnon commented, "The rapidly growing use of genomic testing as an essential part of cancer treatment makes this a good time to build on the foundation we have created over the past year and to exponentially expand the menu of tumor exons/mutations that can be tested using our ICP technology. We have refined and industrialized the development of new tests, as we demonstrated with the recent rapid development and release of our EGFR C797S ICP assay. We therefore believe that our ambitious goal of offering more than 200 exons/mutations by 2018 is achievable. We are optimistic that the accuracy, versatility, ease of use and rapid turnaround of ICP cancer testing, along with its availability in multiple formats and on a variety of platforms, will generate strong demand as we expand the test menu. We look forward to working with a range of customers and diagnostic laboratory partners to apply the power of TBIO’s expanded ICP product line to accelerate cancer research and improve patient outcomes."

ICE COLD-PCR achieves its ultra-high sensitivity through selective amplification of mutant DNA. The result is up to a 500-fold increase in sensitivity in identifying mutations with the most precise sequence alteration detection rates available. ICP was originally developed by the laboratory of Dr. Mike Makrigiorgos at the Dana-Farber Cancer Institute, which has exclusively licensed rights to the technology to Transgenomic.