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Archive - Oct 1, 2015

Capricor Therapeutics to Present on October 7 at Partnering Forum of Stem Cell Meeting; Company Developing Novel Therapeutics for Post-MI, Heart Failure, and DMD Cardiomyopathy; CDC-Derived Exosomes Also Being Investigated for Such Purposes

Capricor Therapeutics, Inc. (NASDAQ:CAPR) a biotechnology company focused on the discovery, development, and commercialization of first-in-class therapeutics announced on September 30, 2015 that Linda Marbán (photo), Ph.D., Capricor’s President, CEO, and Director, will present at the annual Partnering Forum of the Stem Cell Meeting on the Mesa to be held October 7-9 in La Jolla, California (http://stemcellmeetingonthemesa.com). Dr. Marban’s presentation is scheduled for Wednesday, October 7, beginning at 11:45 am Pacific Daylight Time. A live video webcast will be available at http://stemcellmeetingonthemesa.com/webcast/ with a replay of the webcast to be published on http://capricor.com/news/events/. Please visit http://stemcellmeetingonthemesa.com/ for full information including registration. Complimentary attendance at this event is available for credentialed investors and members of the media only. This conference brings together senior executives and top decision makers in the industry within the scientific community with the shared goal of advancing and translating cutting-edge research into revolutionary treatments and cures. Capricor’s lead programs target post myocardial infarction (heart attack), heart failure, and Duchenne muscular dystrophy. The company has two leading product candidates under investigation: CAP-1002, a cardiac cell therapy, and Cenderitide, a natriuretic peptide receptor agonist. CAP-1002 is in development for the treatment of post myocardial infarction (heart attack), advanced heart failure, and Duchenne muscular dystrophy-associated cardiomyopathy. CAP-1002 is unique in cardiac cell therapy in that it is the only allogeneic, intracoronary-delivered, cardiosphere-derived cell (CDC) treatment under development.

Dormant Ancient Genes May Awaken to Cause Deadly Disease--Human Endogenous Retrovirus-K (HERV-K) Genes May Be Involved in Amyotrophic Lateral Sclerosis (ALS) (Lou Gehrig’s Disease)

Scientists at the National Institutes of Health have discovered that reactivation of ancient viral genes embedded in the human genome may cause the destruction of neurons in some forms of amyotrophic lateral sclerosis (ALS). The results, published in the September 30, 2015 issue of Science Translational Medicine, suggest a link between human endogenous retroviral genes (HERVs) and ALS. The article is titled “Human Endogenous Retrovirus-K Induces Motor Neuron Disease.” The findings also raise the question of whether antiretroviral drugs, similar to those used for suppressing HIV, may help some ALS patients. For generations, humans have been passing on genetic remnants of HERV infections that may have happened millions of years ago. Although nearly eight percent of the normal human genome is made up of these genes, very little is known about their role in health and disease. “People call the genes for these viruses junk DNA. Our results suggest they may become activated during ALS,” said Avindra Nath, M.D., Clinical Director at the NIH’s National Institute of Neurological Disorders and Stroke (NINDS) and a senior author of the study. “Ultimately we hope the results will lead to effective treatments for a heartbreaking disorder.” Currently, there is no effective treatment for the more than 12,000 Americans who live with ALS. This fatal disorder destroys neurons that control movements, including speaking, walking, breathing, and swallowing. On rare occasions, HIV-infected, AIDS patients develop ALS-like symptoms. In many of these patients, the symptoms can be reversed by treatment with antiretroviral drugs. Previous studies found reverse transcriptase, a protein encoded by retroviral genes, in the blood of some ALS patients, but its role in the disorder is unknown. These observations prompted Dr.

Cancer Risk Increases with Height, Large-Scale Swedish Study Finds; 5.5 Million Men & Women Included in Largest-Ever Study of This Association

Cancer risk has been found to increase with height in both Swedish men and women, according to research presented at the 54th Annual European Society for Paediatric Endocrinology (ESPE) Meeting (October 1-3, 2015) in Barcelona Spain. The presentation was titled “Positive Association Between Height and Cancer in the Swedish Population.” This long-term study is the largest ever carried out on the association between height and cancer in both genders. Researchers from the Karolinska Institutet and the University of Stockholm examined 5.5 million men and women in Sweden, born between 1938 and 1991 and with adult heights ranging between 100 cm (3.3 feet) and 225 cm (7.4 feet). The scientists followed the group of individuals from 1958 or from the age of 20 until the end of 2011, and found that for every additional 10 cm (3.9 inches) of height, the risk of developing cancer increased by 18% in women and 11% in men. Additionally, taller women had a 20% greater risk of developing breast cancer, whilst the risk of developing melanoma increased by approximately 30% per additional 10 cm (3.9 inches) of height in both men and women. Previous studies have also shown the same association between height and cancer. That is to say, taller individuals have a higher risk of developing different types of cancer, including breast cancer and melanoma. However, this association has never been studied in men and women on such a large scale before. "To our knowledge, this is the largest study performed on linkage between height and cancer including both women and men," said medical doctor Emelie Benyi, also a Ph.D. student at Karolinska Institutet, who led the study and presented the report at the ESPE meeting.

Results of Clinical Study Show Promise of First-in-Class, Exosome-Based, Predictive Liquid Biopsy Test to Address Unnecessary Surgical Biopsies, Over-Diagnosis, and Over-Treatment of Indolent Prostate Cancer

Exosome Diagnostics, Inc., a developer of revolutionary, biofluid-based molecular diagnostics, presented new positive data from a clinical study for the company’s novel, urine-based prostate cancer liquid biopsy, ExoIntelliScore™ Prostate (formally referred to as Exo106). The data demonstrated that ExoIntelliScore Prostate accurately predicted, in pre-radical prostatectomy (RP) urine samples of patients with prostate cancer, objective clinical features present in RP specimens and also provided initial improved discrimination of RP Gleason Score 4+3, a subset of patients at elevated risk for aggressive disease. These results add to a growing body of clinical validation evidence that show ExoIntelliScore Prostate’s unique ability to accurately, and completely non-invasively, identify high-grade prostate cancer. The results also suggest the potential for the first-in-class genetic-based assay to be utilized throughout the diagnostic paradigm, including prior to initial biopsy, as well as for sequential monitoring of disease progression in patients enrolled in active surveillance. The data were presented at a poster session entitled, “A Non-Invasive Urine Exosome Gene Expression Assay (ExoIntelliScore™ Prostate) Accurately Predicts Pathologic Stage and Grade in the Prostatectomy Specimen,” at the 18th European Cancer Congress (ECCO) – 40th European Society for Medical Oncology (ESMO) Annual Meeting in Vienna, Austria (September 25-29). Note that a link to a PDF of the complete poster is provided at the end of this article. “The current diagnostic landscape for prostate cancer is imprecise, setting off a cascade of events that starts with unnecessary and inaccurate biopsies, and leads to over-diagnosis and over-treatment of the disease with radical treatment choices, including prostatectomies,” said co-author James A.

1000 Genomes Project Establishes World’s Largest Catalog of Human Genomic Variation; Nature Publishes Results, Together with Powerful Related Stories, All Open-Access, in Priceless Issue for Scientists and the World

An international team of scientists from the 1000 Genomes Project Consortium has created the world’s largest catalog of genomic differences among humans, providing researchers with powerful clues to help them establish why some people are susceptible to various diseases. While most differences in peoples’ genomes — called variants — are harmless, some are beneficial, while others contribute to diseases and conditions, ranging from cognitive disabilities to susceptibilities to cancer, obesity, diabetes, heart disease, and other disorders. Understanding how genomic variants contribute to disease may help clinicians develop improved diagnostics and treatments, in addition to new methods of prevention. The National Human Genome Research Institute (NHGRI), part of the National Institutes of Health, helped fund and direct this international public-private consortium of researchers in the United States, the United Kingdom, China, Germany, and Canada. In two studies published online on Sept. 30, 2015, in Nature, and then in the October 1, 2015 Nature print issue, investigators examined the genomes of 2,504 people from 26 populations across Africa, East and South Asia, Europe and the Americas. Nature features the project completion on the cover of its October 1, 2015 print issue. This collector’s-item Nature issue also provides a number of other notable related articles. These include “Human Genome Project: 25 Years of Big Biology,” by Eric D. Green, James D. Watson, and Francis S. Collins; a Nature editorial titled “Variety of Life” on the 1000 Genomes Project; and an article by the UK10K Consoritium titled “The UK10K Project Identifies Rare Variants in Health and Disease.” All these articles, as well as the two 1000 Genomes Project studies, are open-access.

Solution to 50-Year-Old Riddle of Why Colliding Connective Tissue Fibroblasts Repel Each; “Molecular Bumper” Mechanism May Play Role in Metastasis

When fibroblast cells from the connective tissue collide, they repel one another – this phenomenon was discovered more than 50 years ago. It is only now, however, that researchers at the University of Basel in Switzerland have discovered the molecular basis for this process, as they report online on October 1, 2015 in Developmental Cell. The article is titled “SrGAP2-Dependent Integration of Membrane Geometry and Slit-Robo-Repulsive Cues Regulate Fibroblast Contact Inhibition of Locomotion.” Their findings could have important implications for cancer research. Fibroblasts are motile constituents of the connective tissue and also regulate its stiffness. Moreover, fibroblasts play an important role in malignant skin diseases such as melanoma. In research, they serve as a model system for studying cell migration. In the early 1950s, the English researcher Michael Abercrombie discovered that colliding fibroblasts repel one another and, in the process, change their direction of motion. He called this phenomenon “contact inhibition of locomotion.” Although individual proteins were identified as key factors in this process, the molecular basis of this reaction remained something of a mystery. In particular, it was unclear which repulsion signals were involved in the process, how these signals entered the cells from the outside, and how they influenced the cytoskeleton, which in turn regulates the cell’s movement.Professor Olivier Pertz’s research group at the University of Basel has now precisely answered these questions. The group identified a coherent signaling axis consisting of three proteins called Slit2, Robo4, and srGAP2 which operates as follows. The repulsion factor Slit2 binds to the receptor Robo4, whereupon the signal enters the cell’s interior and activates srGAP2.

Acquisition of Four Key Gut Bacteria (FLVR) by 3 Months of Age May Reduce Risk of Childhood Asthma

New research by scientists at the University of British Columbia (UBC) and British Columbia Children's Hospital finds that infants may be protected from developing asthma if they acquire four types of gut bacteria by three months of age. The research was published in the September 30, 2015 issue of Science Translational Medicine and the article is titled “Early Infancy Microbial and Metabolic Alterations Affect Risk of Childhood Asthma.” More than 300 families from across Canada participated in this research through the Canadian Healthy Infant Longitudinal Development (CHILD) Study. "This research supports the hygiene hypothesis that we're making our environment too clean. It shows that gut bacteria play a role in asthma, but it is early in life when the baby's immune system is being established," said the study's co-lead researcher Dr. B. Brett Finlay, Peter Wall Distinguished Professor in the Michael Smith Laboratories and the Departments of Microbiology & Immunology and Biochemistry and Molecular Biology at UBC. Asthma rates have increased dramatically since the 1950s and now affect up to 20 per cent of children in western countries. This discovery opens the door to developing probiotic treatments for infants that might prevent asthma. The finding could also be used to develop a test for predicting which children are at risk of developing asthma. In the study, the researchers analyzed fecal samples from 319 children involved in the CHILD Study. Analysis of the gut bacteria from the 319 samples revealed lower levels of four specific gut bacteria in three-month-old infants who were at an increased risk for asthma.

IL-17 Receptor Antagonist (Brodalumab) Highly Effective Against Moderate to Severe Plaque Psoriasis in Phase III Clinical Studies; NEJM Article Says Results Superior to Those Achieved with Stelara, Already in Clinical Use

An experimental, biologic treatment, brodalumab, achieved 100 percent reduction in psoriasis symptoms in twice as many patients as a second, commonly used treatment, according to the results of a multi-center clinical trial led by Mount Sinai researchers and published online on October 1, 2015 in the New England Journal of Medicine. The article is titled “Phase 3 Studies Comparing Brodalumab with Ustekinumab in Psoriasis.” The study drug, brodalumab, is a monoclonal antibody, akin to proteins built by the human immune system to recognize and block specific target molecules. A therapeutic antibody, brodalumab was designed to block the function of the immune signaling protein interleukin 17 (IL-17) (image). If not blocked, IL-17 docks into specifically shaped proteins, IL-17 receptors, to pass on signals that contribute to abnormal, psoriatic inflammation. "Brodalumab is the only IL-17 receptor antagonist in clinical development," said Mark Lebwohl, M.D., Sol and Clara Kest Professor and Chairman of the Kimberly and Eric J. Waldman Department of Dermatology, Icahn School of Medicine at Mount Sinai in New York City. "Studies demonstrate that brodalumab binds to the IL-17 receptor, thus preventing IL-17 and its partner molecules or ligands from doing so, to counter inflammatory diseases. When it comes to complete clearing, our results are better than any previously published and confirm that targeting the IL-17 receptor is highly effective in the treatment of moderate to severe plaque psoriasis. Treatment was so effective that many patients did not have a dot of psoriasis left on their bodies." Plaque psoriasis is a non-contagious chronic disease in which the immune system causes skin cells to grow at an accelerated rate.

Novel Approach Uses mRNA for MafA Transfer Factor to Reprogram Pancreatic Duct-Derived Cells to Produce and Secrete Insulin in Response to Glucose; May Speed Development of Transplantable Cells to Treat Diabetes

A new technique to produce cells with insulin-secretion capabilities has been developed, according to research presented at the 54th Annual European Society for Paediatric Endocrinology Meeting in Barcelona, Spain (October 1-3, 2015). The technique could be further developed to be used in the transplantation for patients with type 1 diabetes. It is well established that, typically, in type 1 diabetes, the body's immune system mistakenly attacks and destroys beta cells in the pancreas. These unique cells are responsible for producing, storing, and secreting insulin - the hormone that regulates levels of glucose in the blood. Currently one of the most promising therapies in the fight against diabetes is the replacement of beta cells. In the replacement therapy for type 1 diabetes, researchers from Université Catholique de Louvain in Belgium have previously shown that human pancreatic duct-derived cells (HDDCs) are an attractive source of cells that may potentially be reprogrammed to behave like beta cells. The HDDCs are found in the adult pancreas and are progenitor cells - cells that have a tendency and the capacity to differentiate into specific types of other cells. In this study, the research group reprogrammed HDDCs to behave like beta cells and secrete insulin within the pancreas, whilst responding to glucose. The researchers used messenger RNA (mRNA) for a specific transcription factor - a protein that controls which genes are turned off or on in the genome - called MafA (image). The mRNA codes for the production of the MafA protein that binds to nuclear DNA in order to orchestrate the changes in cellular functions. This technique allowed the researchers to avoid any potential genetic modification of the target cells.