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June 11th, 2020

Regeneron Begins First Clinical Trials of Anti-Viral Antibody Cocktail REGN-COV2 for the Treatment and Prevention of COVID-19; Two-Antibody “Cocktail” Designed to Help Protect Against Viral Escape

On June 11, 2020, Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) announced initiation of the first clinical trial of REGN-COV2, its investigational dual antibody cocktail for the prevention and treatment of COVID-19. The REGN-COV2 clinical program will consist of four separate study populations: hospitalized COVID-19 patients, non-hospitalized symptomatic COVID-19 patients, uninfected people in groups that are at high-risk of exposure (such as healthcare workers or first responders), and uninfected people with close exposure to a COVID-19 patient (such as the patient's housemate). The placebo-controlled trials will be conducted at multiple sites. "We have created a unique anti-viral antibody cocktail with the potential both to prevent and treat infection, and also to preempt viral “escape,” a critical precaution in the midst of an ongoing global pandemic," said George D. Yancopoulos, MD, PhD,, Co-Founder, President and Chief Scientific Officer of Regeneron. "REGN-COV2 could have a major impact on public health by slowing spread of the virus and providing a needed treatment for those already sick – and could be available much sooner than a vaccine. The antibody cocktail approach may also have long-term utility for elderly and immuno-compromised patients, who often do not respond well to vaccines. Ultimately, the world needs multiple solutions for COVID-19, and the innovative biopharma industry is collectively working hard to help as many people as possible with a variety of complementary approaches." Regeneron scientists evaluated thousands of fully-human antibodies produced by the company's proprietary VelocImmune® mice, which have been genetically-modified to have a human immune system, as well as antibodies isolated from humans who have recovered from COVID-19.

June 10th

Reprogramming of Immune System Cures Child with Rare, But Often Fatal, Progression of Valley Fever to Disseminated Coccidioidomycosis

In the June 11, 2020 issue of the New England Journal of Medicine, a team of UCLA physicians and scientists describes the first case of immune modulation being used to cure a severe and often fatal fungal infection. The article is titled “Disseminated Coccidioidomycosis Treated with Interferon-γ and Dupilumab.” The UCLA team "retuned" a 4-year-old's immune system so that it could fight off disseminated coccidioidomycosis. The case, originally reported by UCLA in 2019, could pave the way for a new treatment for the infection, which affects hundreds of Americans each year, primarily in the Southwest, and kills approximately 40% of the people who contract it. The technique described in the study could also suggest a new paradigm for treating other severe fungal infections, bacterial infections such as tuberculosis, and severe viral infections such as influenza and COVID-19. "Immune modulation isn't currently part of the strategy with any of these severe infections," said Manish Butte, MD, PhD, the report's senior author, who holds the E. Richard Stiehm Endowed Chair in Pediatric Allergy, Immunology, and Rheumatology at the David Geffen School of Medicine at UCLA (photo shows Dr. Butte with young boy who was cured). "Our case suggests that, rather than hoping to get the upper hand with more and more antibiotics or antifungals, we can have some success by combining these established approaches with the new idea of programming the patient's immune response to better fight the infection." Each year, more than 100,000 people are infected with Coccidioides fungi, which reside in the soils of California, Arizona, and West Texas. Most people who are infected are asymptomatic, and about 20,000 experience the minor respiratory illness commonly known as Valley fever.

CytoDyn CEO Dr. Nadir Pourhassan and CMO Dr. Scott Kelly to Hold Conference Call (Thursday 4 pm EDT) to Provide Updates on CytoDyn’s COVID-19 Phase 2 and 3 Trials, NASH, BLA for HIV, and Manufacturing of Leronlimab in 2020-2021

On June 9, 2020, CytoDyn Inc. (OTC.QB: CYDY), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced that Nader Pourhassan, PhD, President and Chief Executive Officer, and Scott Kelly, MD, Chairman, Chief Medical Officer, and Head of Business Development, will host an investment community conference call on Thursday, June 11, 2020, to provide a comprehensive update on several recent clinical and regulatory developments, potential uplisting and its timing, potential licensing, and/or distribution agreements. Management will dedicate approximately 30-40 minutes to address questions from analysts and investors. The conference call will take place on Thursday, June 11, 2020, beginning at 1:00 p.m. PDT / 4:00 p.m. EDT. Dial-In access is available at 877-407-2986 US / 201-378-4916 International. A live audio webcast may also be accessed via CytoDyn’s corporate website at www.cytodyn.com under the Investors section/IR Calendar and will be archived for 30 days. Web participants are encouraged to go to the website 15 minutes prior to the start of the call to register, and to download and install any necessary software. The webcast can also be accessed via the following link: https://78449.themediaframe.com/dataconf/productusers/cydy/mediaframe/38.... A replay of the conference call will be available until July 11, 2020. To access the replay, interested parties may dial 877-660-6853 (US) / 201-612-7415 (international) and enter conference identification number 13705221.

CHOP Researchers Identify Targets for COVID-19 Vaccine Using Cancer Immunotherapy Tools; 65 Peptide Sequences That, When Targeted, Offer Greatest Probability of Providing Population-Scale Immunity Have Been Identified

Cancer researchers at Children's Hospital of Philadelphia (CHOP) have harnessed tools normally used for the development of cancer immunotherapies and adapted them to identify regions of the SARS-CoV-2 virus to target with a vaccine, employing the same approach used to elicit an immune response against cancer cells to stimulate an immune response against the virus. Using this strategy, the researchers believe a resulting vaccine would provide protection across the human population and drive a long-term immune response. The strategy is described in an article published online on June 5, 2020 in Cell Reports Medicine. The open-access article is titled “Identification of SARS-CoV-2 Vaccine Epitopes Predicted to Induce Long-Term Population-Scale Immunity.” "In many ways, cancer behaves like a virus, so our team decided to use the tools we developed to identify unique aspects of childhood cancers that can be targeted with immunotherapies and apply those same tools to identify the right protein sequences to target in SARS-CoV-2," said senior author John M. Maris, MD, a pediatric oncologist in CHOP's Cancer Center and the Giulio D'Angio Professor of Pediatric Oncology at the Perelman School of Medicine at the University of Pennsylvania. "By adapting the computational tools developed and now refined by lead author Mark Yarmarkovich, PhD in the Maris Lab, we can now prioritize viral targets based on their ability to stimulate a lasting immune response, predicted to be in the vast majority of the human population. We think our approach provides a roadmap for a vaccine that would be both safe and effective and could be produced at scale." The COVID-19 pandemic has led to an urgent need for the development of a safe and effective vaccine against SARS-CoV-2, the virus that causes the COVID-19 disease.

June 8th

CytoDyn Receives BLA Acknowledgment Letter from FDA; PDUFA Date Could Be Set by FDA on July 10

On June 8, 2020, CytoDyn, Inc., a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced that the U.S. Food and Drug Administration (FDA) has advised the Company, subject to its ongoing review, that it could receive its “PDUFA date” on July 10, 2020. Under the Prescription Drug User Fee Act (PDUFA), the FDA sets a goal date for the agency to complete its review of a new drug application; this date is commonly referred to as the “PDUFA date.” The FDA’s standard review time for new drug applications is 10 months. CytoDyn’s drug candidate, leronlimab, was previously granted Fast Track designation by the FDA and the Biologics License Application (BLA) was granted rolling review. The Company recently filed for Priority Review designation with the intent to reduce the FDA’s review time to six months. The FDA informs the applicant of a Priority Review designation within 60 days. Nader Pourhassan, PhD, President and Chief Executive Officer of CytoDyn, commented, “We are very encouraged by our recent correspondence with the FDA and remain hopeful of a PDUFA date to be announced by the agency on July 10. The review of our BLA could not be more timely, as our other trials for COVID-19 are approaching two important milestones and should the outcomes be successful, we believe the information in this BLA could be very useful in expediting approval of leronlimab to treat COVID-19, as well as HIV.” CytoDyn is currently enrolling patients in two clinical trials for COVID-19--a Phase 2 randomized clinical trial for mild-to-moderate COVID-19 population in the U.S., and a Phase 2b/3 randomized clinical trial for severe and critically ill COVID-19 population in several hospitals throughout the country.

OX2 Therapeutics Receives FDA Approval for Phase I Clinical Trial to Treat High-Grade Glioblastoma with Novel Peptide Checkpoint Inhibitor (CD200AR-L); First-of-Its-Kind Peptide Also Being Developed As Platform for Possible Treatment of Other Solid Tumors

On Monday, June 8, 2020, OX2 Therapeutics, Inc., a privately held Minneapolis company, announced that it has received clearance from the U.S. Food and Drug Administration (FDA) to launch a phase I clinical trial with its new combination therapy for treatment of recurrent high-grade brain tumors for which no curative therapy is available. OX2 Therapeutics has developed the first-of-its-kind peptide platform that targets the activation receptor of the CD200 immune checkpoint. The peptide activates the immune system through a mechanism that modulates the suppressive effects of the CD200, PD-1/PD-L1, and CTLA4 immune checkpoints to allow a more robust anti-tumor response (see graphic image of mechanism below). “This single peptide has the potential to replace the toxic antibody therapies that are currently used to block these immune checkpoints,” said two of the company co-founders, Chief Medical Officer Christopher Moertel (photo at end), MD, Professor, and Chief Scientific Officer Michael Olin (photo here), PhD, Associate Professor, both from the Division of Hematology/Oncology, Department of Pediatrics, in the University of Minnesota School of Medicine. Dr. Moertel is the Kenneth and Betty Jayne Dahlberg Professor in the Pediatrics Department’s Division of Pediatric Hematology and Oncology of the University of Minnesota Medical School, within the University’s Masonic Cancer Center; Medical Director of the Pediatric Neuro-Oncology and Neurofibromatosis Programs; and Clinical Neuro-Oncology Leader of the Medical School’s Brain Tumor Program. Dr. Moertel has over 25 years of experience as a neuro-oncologist, directing numerous clinical trials, has served on numerous national and local professional committees, and is the author of a number of book chapters, articles and abstracts.

June 7th

New Test for Rare Inherited Metabolic Disease (CAD Deficiency) Identifies Children Who May Benefit from Simple Supplement of Uridine; Only 17 in World Known to Have Life-Threatening CAD Deficiency

Scientists at the Sanford Burnham Prebys Medical Discovery Institute in La Jolla, California, and the Centro de Biología Molecular Severo Ochoa in Madrid, Spain have created a test that determines which children with CAD deficiency—an extremely rare, inherited metabolic disease—are likely to benefit from receiving uridine (https://en.wikipedia.org/wiki/Uridine), a nutritional supplement that has dramatically improved the lives of other children with the condition. In the body, uridine is a key nucleoside that is one of the four nucleosides found in RNA molecules. The CAD (image) deficiency study was published online on May 28, 2020 in Genetics in Medicine (https://www.nature.com/articles/s41436-020-0833-2). The open-access article is titled “Cell-Based Analysis of CAD Variants Identifies Individuals Likely to Benefit from Uridine Therapy.” “The effect of uridine for some children with CAD deficiency is nothing short of amazing. These kids go from bedridden to interacting with people and moving around,” says Hudson Freeze (https://www.sbpdiscovery.org/our-scientists/hudson-freeze-phd), PhD, Director of the Human Genetics Program at Sanford Burnham Prebys and co-corresponding author of the study. “Our results identified 11 children who have mutations in both copies of the CAD gene and would likely benefit from uridine therapy. With this test, we can provide hope to some families, while sparing others from unrealistic expectations. That’s incredibly important.”

June 6th

DNA Oligos in Tumor-Penetrating Peptides Can Boost Effectiveness of Checkpoint Inhibitors in Cancer Immunotherapy

One promising strategy to treat cancer is stimulating the body’s own immune system to attack tumors. However, tumors are very good at suppressing the immune system, so these types of treatments don’t work for all patients. MIT engineers have now come up with a way to boost the effectiveness of one type of cancer immunotherapy. They showed that if they treated mice with existing drugs called checkpoint inhibitors, along with new nanoparticles that further stimulate the immune system, the therapy became more powerful than checkpoint inhibitors given alone. This approach could allow cancer immunotherapy to benefit a greater percentage of patients, the researchers say. “These therapies work really well in a small portion of patients, and in other patients they don’t work at all. It’s not entirely understood at this point why that discrepancy exists,” says Colin Buss PhD 2020, the lead author of the new study. The MIT team devised a way to package and deliver small pieces of DNA that crank up the immune response to tumors, creating a synergistic effect that makes the checkpoint inhibitors more effective. In studies in mice, the scientists showed that the dual treatment halted tumor growth, and in some cases, also stopped the growth of tumors elsewhere in the body. Sangeeta Bhatia (photo), MD, PhD, the John and Dorothy Wilson Professor of Health Sciences and Technology and Electrical Engineering and Computer Science, and a member of MIT’s Koch Institute for Integrative Cancer Research and the Institute for Medical Engineering and Science, is the senior author of the paper, which was published online on June 3, 2020 in PNAS.

Early Results Suggest Cannabinoids May Result in Decreased Production of HIV-1 in the CNS; Scientists Also Show Evidence of Decreased Incorporation of HIV-1 RNA into Exosomes Released from Infected Cells

On June 5, 2020, George Mason University in Virginia, announced that two of its scientists--Fatah Kashanchi, PhD, Director, Laboratory of Molecular Virology, School of Systems Biology, and Lance Liotta, MD, PhD, Co-Director and Co-Founder, Center for Applied Proteomics and Molecular Medicine--are working to mitigate virally driven HIV pathogenesis in the central nervous system (CNS). Preliminary data the researchers have gathered suggests that cannabinoids—specifically, cannabidiol (CBD) and Δ9-tetrahydrocannabinol (THC)--may be effective in reducing HIV-1 transcription of both short, non-coding RNA such as trans-activating response (TAR) RNA, and full-length genomic RNA, thereby resulting in decreased production of virus. The researchers have also found that the reduction in transcription results in a decreased incorporation of HIV-1 RNA into extracellular vesicles (EVs)/exosomes released from infected cells. This has been previously shown to contribute to dysfunction in recipient cells, including activation of the NF-kB pathway, a protein complex that controls transcription of DNA, cytokine production, and cell survival through toll-like receptor 3 (TLR3), and to increased susceptibility to infection. TLR3 is a member of the toll-like receptor family of pattern-recognition receptors of the innate immune system. The researchers hypothesize that cannabinoid treatment may affect host cell pathways, including autophagy and the endosomal sorting complexes required for transport (ESCRT) pathways, to alter EV/exosome release which can potentially mitigate EV/exosome-related dysfunction during viral infection of the CNS. The researchers have two aims for their work. First, they intend to define the mechanisms of cannabinoid-mediated decreased EV/exosome production and release in HIV-1 infected cells.

Sanger Institute’s Gosia Trynka Awarded 2020 Leena Peltonen Prize for Excellence in Human Genetics; Prize to Be Presented During European Society of Human Genetics (ESHG) 2020 Annual Meeting Being Held Virtually June 6-9

It has been announced that the winner of this year’s Leena Peltonen Prize for Excellence in Human Genetics, is Gosia Trynka, PhD, Immune Genomics Group Leader at the Wellcome Sanger Institute, Hinxton, Cambridge, UK. The prize is awarded to an outstanding young researcher in the field of human genetics, and honors the memory of Dr. Leena Peltonen (https://en.wikipedia.org/wiki/Leena_Peltonen-Palotie), a world-renowned human geneticist from Finland who died in 2010 and who contributed greatly to the identification of genes for human diseases. The prize is to be presented during the ESHG 2020 53rd annual meeting being held entirely online June 6-9 (https://2020.eshg.org/). The Leena Peltonen Prize is funded by the Leena Peltonen Memorial Fund in the Paulo Foundation. It includes an award of €10 000 (~$12,665). An international nomination committee consists of faculty members of the Wellcome Trust Leena Peltonen School of Human Genomics. The committee is chaired by the Director of the Institute for Molecular Medicine Finland (FIMM). After earning her MSc in Biotechnology, Biophysics, and Biochemistry from Jagiellonian University, Krakow, Poland, Gosia Trynka (https://www.sanger.ac.uk/person/trynka-gosia/) obtained a PhD cum laude from the University of Groningen, The Netherlands. In 2012, she moved to the US to take up a postdoctoral fellowship at Brigham and Women's Hospital, Harvard Medical School, and the Broad Institute. Dr. Trynka has led the Immune Genomics Group at the Wellcome Sanger Institute in the UK since 2014 and, this year, she took on an additional responsibility as the Experimental Science Director at Open Targets (https://www.opentargets.org/), a public/private partnership that uses genomics data to improve drug target identification and prioritization. Dr.