The immune response to infections is a delicate balance. We need just enough action to clear away the offending bacteria or viruses, but not so much that our own bodies suffer collateral damage.

Macrophages are immune cells at the front line, detecting pathogens and kicking off an inflammatory response when needed. Understanding how macrophages determine when to go all-out and when to keep calm is key to finding new ways to strike the right balance particularly in cases where inflammation goes too far, such as in sepsis, colitis and other autoimmune disorders.

In a study published October 14, 2020 in the Proceedings of the National Academy of Sciences , researchers at University of California San Diego School of Medicine discovered that a molecule called Girdin, or GIV, acts as a brake on macrophages.

When the team deleted the GIV gene from mouse macrophages, the immune cells rapidly overacted to even small amounts of live bacteria or a bacterial toxin. Mice with colitis and sepsis fared worse when lacking the GIV gene in their macrophages.

The researchers also created peptides that mimic GIV, allowing them to shut down mouse macrophages on command. When treated with the GIV-mimic peptide, the mices inflammatory response was tempered.

When a patient dies of sepsis, he or she does not die due to the invading bacteria themselves, but from an overreaction of their immune system to the bacteria, said senior author Pradipta Ghosh, MD, professor at UC San Diego School of Medicine and Moores Cancer Center. Its similar to what were seeing now with dangerous cytokine storms that can result from infection with the novel coronavirus SARS-CoV-2. Macrophages, and the cytokines they produce, are the bodys own immune-stimulating agents and when produced in excessive amounts, they do more harm than good.

Digging deeper into the mechanism at play, Ghosh and team discovered that the GIV protein normally cozies up to a molecule called Toll-like receptor 4 (TLR4). TLR4 is stuck right through the cell membrane, with bits poking inside and outside the cell. Outside of the cell, TLR4 is like an antenna, searching for signs of invading pathogens. Inside the cell, GIV is nestled between the receptors two feet. When in place, GIV keeps the feet apart, and nothing happens. When GIV is removed, the TLR4 feet touch and kick off a cascade of immune-stimulating signals.

Ghoshs GIV-mimicking peptides can take the place of the protein when its missing, keeping the feet apart and calming macrophages down.

We were surprised at just how fluid the immune system is when it encounters a pathogen, said Ghosh, who is also director of the Institute for Network Medicine and executive director of the HUMANOID Center of Research Excellence at UC San Diego School of Medicine. Macrophages dont need to waste time and energy producing more or less GIV protein, they can rapidly dial their response up or down simply by moving it around, and it appears that such regulation happens at the level of gene transcription.

Ghosh and team plan to investigate the factors that determine how the GIV brake remains in place when macrophages are resting or is removed to mount a response to a credible threat. To enable these studies, the Institute for Network Medicine at UC San Diego School of Medicine recently received a new $5 million grant from the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health. Ghosh shares this award with her colleagues Debashis Sahoo, PhD, assistant professor at UC San Diego School of Medicine and Jacobs School of Engineering, and Soumita Das, PhD, associate professor of pathology at UC San Diego School of Medicine.

Reference: Swanson L, Katkar GD, Tam J, et al.TLR4 signaling and macrophage inflammatory responses are dampened by GIV/Girdin. PNAS. 2020.doi:10.1073/pnas.2011667117

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Protein That Prevents Immune System Overreaction Could Have Therapeutic Applications - Technology Networks

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Tools developed to probe the virome could aid in variety of research

Researchers at Washington University School of Medicine in St. Louis have received an $8.5 million grant to study the role of gut viruses in inflammatory bowel disease. Tools developed in the course of the project could accelerate research into other roles of the virome in health and disease.

The communities of bacteria that live in our digestive tracts help digest food and produce vitamins, protect against pathogens, and promote the healthy functioning of our immune system. But alongside gut bacteria thrives a vast community of viruses, and we know little about their impact on health and disease.

Efforts to study the gut viral community known as the virome have been hindered by a lack of tools to analyze viral diversity. Researchers at Washington University School of Medicine in St. Louis have received an $8.5 million grant from the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health (NIH) to develop the tools needed to study the role of the virome in inflammatory bowel disease. Once developed, such tools could be applied widely, opening up new avenues of research into the role of the virome in normal physiology and development, as well as diseases such as diabetes, AIDS and cancer.

The virome has been linked to a number of conditions inflammatory bowel disease, malnutrition, graft-versus-host disease and there is also some evidence that the virome supports human health in some ways, said principal investigator David Wang, PhD, a professor of molecular microbiology, and of pathology and immunology. But the problem that plagues virome studies is that people find an association, and then they cant pursue it. Once you find an association, the next step is to see what happens when you introduce the virus to an animal. Does it cause the disease? Make it worse? But there are no tools to carry this out.

Tools to analyze the viral community are relatively scarce partly because viruses are more diverse than bacteria. All bacteria carry certain basic housekeeping genes necessary for life, notably the 16S ribosomal RNA gene. Scientists use this universal gene to screen mixed communities of unidentified bacteria by pulling out all the 16S ribosomal RNA genes and using the sequences to classify the bacteria into families. There is no equivalent universal gene among viruses.

Wang and colleagues previously have discovered differences between the viromes of people with inflammatory bowel disease and healthy people. Inflammatory bowel disease is caused by chronic inflammation in the digestive tract and characterized by persistent diarrhea and abdominal pain. The researchers found that people with the condition carry more Caudovirales, a group of viruses that infects bacteria, and anelloviruses, a family of viruses that infects human cells, in their intestines. But they do not yet know what, if anything, the presence of these viruses means.

The new grant will allow the researchers to follow a group of people with inflammatory bowel disease over time, along with healthy members of their households for comparison. Inflammatory bowel disease tends to be cyclical, flaring up and then resolving again and again. By taking repeated stool samples and analyzing the genetic material of the viruses in such samples, the researchers will be able to see how the makeup of the gut viral community changes over the course of the disease, and gauge whether any particular groups of viruses become more abundant during flare-ups or resolutions. They also will assess what effect treatment has on the virome.

Such analysis will require the development of computational tools to identify the viruses by their genetic sequences, classify them into family groups, identify potential genes within viral sequences, and propose functions of the genes.

With the tools we have now, more than half the sequences cant be classified because they are not similar enough to known sequences, Wang said. We frequently cant even tell whether weve found a virus that infects bacteria or human cells.

Wang and colleagues also will develop ways to cultivate viruses so they can study them. As nonliving things, viruses require a living cell to multiply, which makes cultivation in the lab tricky. To grow viruses that infect human cells, researchers must first culture human cells and then infect them with viruses. But the majority of the viruses in the intestinal tract are likely to infect bacteria, not human cells. Such viruses known as bacteriophages, Latin for bacteria eaters are even more complicated: Researchers must first identify the correct bacterial species from among the thousands in our intestines, culture that species, and then attempt to grow the virus within the bacterial culture.

In previous work, we established the first culture system for a gut virus, Wang said. Were relying on our experience there to try to culture more of these novel viruses. Some of these might actually grow in a quite straightforward way, its just that no one has tried yet. And once we have the viruses, then we can use them to start doing experiments in animal models of inflammatory bowel disease.

The impact of the gut bacterial community on human health is a hot topic of study, with a possible role in health conditions ranging from autoimmunity to heart disease to psychiatric illnesses. The virome may prove to be equally consequential if only we can find a way to investigate it.

This isnt a typical grant, because part of its goal is to build resources that will then be available to the scientific community, Wang said. Creating tools is unsexy and usually unfundable. But we have to build these tools before we can answer the exciting questions.

Along with Wang, the research team includes Michael S. Diamond, MD, PhD, the Herbert S. Gasser Professor of Medicine, and Scott Handley, PhD, an associate professor of pathology and immunology, both at Washington University; Thaddeus Stappenbeck, MD, PhD, of the Cleveland Clinic; and collaborators at Cambridge University in the United Kingdom, San Diego State University in California, and Flinders University in Australia.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Role of gut viruses in inflammatory bowel disease is focus of $8.5 million grant - Washington University School of Medicine in St. Louis

Its standard for drug companies to withhold details of clinical trials until after they are completed, tenaciously guarding their intellectual property and competitive edge. But these are extraordinary times, and now there is a growing outcry among independent scientists and public health experts who are pushing the companies to be far more open with the public in the midst of a pandemic that has already killed more than 193,000 people in the United States.

These experts sayAmerican taxpayers are entitled to know moresince the federal government has committed billions of dollars to vaccine research and to buying the vaccines once theyre approved. And greater transparency could also help bolster faltering public confidence in vaccines.

[September 8],nine pharmaceutical companies, including AstraZeneca and Pfizer,pledged to stand with science and rigorously vet any vaccine for the coronavirus an unusual pact among competitors. But the researchers said that missing from the joint statement was a promise to share more critical details about their research with the public and the scientific community.

Weve never had such an important clinical trial or series of clinical trials in recent history, said Dr. Eric Topol, a professor of molecular medicine at Scripps Research in La Jolla, Calif., and a longtime expert on clinical trials. Everything should be transparent.

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Are vaccines now in development safe? The companies making them uniformly won't release data - Genetic Literacy Project

Oct. 13, 2020 05:00 UTC

BEIJING & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- EdiGene, Inc., which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the successful completion of a RMB 450 million (approximately USD 67 million) Series B financing. 3H Health Investment led the round and other new investors included Sequoia Capital China, Alwin Capital and Kunlun Capital, along with continued support by previous investors, including IDG Capital, Lilly Asia Venture, Huagai Capital and Green Pine Capital Partners. Proceeds from the financing will be used to advance the companys pipeline into clinics and to expand the team.

Minchuan Wang, Ph.D., Partner of 3H Health Investment commented: As one of the most disruptive biomedical technologies, gene editing is rapidly moving toward clinics globally. EdiGene is leading the wave in China, as they have established outstanding research and development capabilities, and more importantly, they have developed an impressive portfolio of proprietary gene editing tools and product candidates. We are pleased to lead the Series B financing, and are excited to join force with EdiGene and other investors to bring life-changing innovative gene-editing therapies to patients in China and globally. We look forward to contributing our clinical, business, and policy resources to further strengthen the companys capabilities, and are confident that EdiGene is well-positioned to be a leader in gene-editing therapeutics.

We have raised approximately RMB 700 million (USD 100 million) in the past two years. We are delighted to add these top tier investors in closing of our Series B financing, and are grateful for the continuous support from the current investors, said Dong Wei, Ph.D.CEO of EdiGene, The round enables us to further scale up and transform our pipeline into clinical-stage, which is also a big step forward in building a globally competitive gene editing company. More importantly, we are closer to realizing our mission of bringing innovative and high-quality gene-editing therapies to patients in need.

We are very pleased to have the support and partnership from our investors, which propels the company to an exciting new stage, said Wensheng Wei, Scientific Founder of EdiGene, Together with the investors, we look forward to translating cutting-edge gene editing technologies into innovative therapies, bringing hope and health to patients and their families.

Founded in 2015, EdiGene has established four gene editing based platforms and is advancing its early stage programs into clinical development for patients with genetic diseases and cancer. The four platforms are ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. In addition, EdiGene has launched GMP manufacturing facility in 2018 in Guangdong Province.

About EdiGene, Inc

EdiGene is a biotechnology company focused on leveraging the cutting-edge genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of genetic diseases and cancer. The company has established its proprietary ex vivo genome-editing platforms for hematopoietic stem cells and T cells, in vivo therapeutic platform based on RNA base editing, and high-throughput genome-editing screening to discover novel targeted therapies. Founded in 2015, EdiGene is headquartered in Beijing, with subsidiaries in Guangzhou, China and Cambridge, Massachusetts, USA. More information can be found at http://www.edigene.com.

About 3H Health Investment

3H Health Investment is a dedicated healthcare venture firm. We focus on unmet medical needs and invest in emerging fields of science and medicine to deliver breakthroughs to patients. Leveraging our extensive resources with clinical institutions, industry partners and policy institutes, we build leading innovative healthcare companies together with our partners. Please visit http://www.3hhinvestment.com to learn more about us.

About Sequoia Capital China

The Sequoia Capital team helps daring founders build legendary companies. In partnering with Sequoia Capital, companies benefit from our unmatched community and the lessons weve learned over 48 years. As The Entrepreneurs behind the Entrepreneurs, Sequoia Capital China focuses on four sectors: TMT, healthcare, consumer/service, and industrial technology. Over the past 15 years weve had the privilege of working with approximately 600 companies in China.

About Alwin Capital

Alwin Capital is a leading venture capital in China specializing in early stage life science investments. Driven by theme-focused research in life science, Alwin Capital has developed investment strategies that capture outstanding opportunities for innovative diagnostic and therapeutic developments. We are committed to the long-term investment in life science with the pursuit of the excess investment return based on the scientific insights. Founded in 2015, our management team has decades of experience in healthcare research and medical markets. We have invested in dozens of companies in life science and cultivated a bunch of leading companies in respective segments.

About Kunlun Capital

Kunlun Capital is a venture firm specializes on technological innovative companies investment. Founded in 2015 by Mr. Yahui Zhou, founder of Shenzhen-listed Kunlun Wanwei and NASDAQ-listed Opera, we now manage an investment fund of more than RMB 10 billion.

Our core is consumer value and we focus on technology, product or data-driven innovative business model. We identify the leading companies of different segments and look for founding teams with global potential and local capabilities. Our investment cases include Bellen, Obio Technology, Keya Medical, musical.ly, Dada Group, KK World, pony ai, Dreame, Leyan Technologies, etc.

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EdiGene Raises Approximately USD 67 Million in Series B Financing to Advance Gene Editing Based Programs into Clinical Stage - BioSpace

Commenting on the Groups sales in the first nine months, Roche CEO Severin Schwan said: Roche is at the forefront of the fight against COVID-19 with a growing portfolio of diagnostics solutions, the development of new medicines and a number of partnerships across the industry. With the recent launch of the rapid antigen test, we further strengthened our position as a leading supplier of COVID-19 tests. At the same time, we continue to deliver solutions for patients suffering from other severe diseases. I am particularly pleased about the FDA approvals in the third quarter for three new medicines: Enspryng and Evrysdi for rare diseases, and the cancer medicine Gavreto. After the pandemic-related decline in the second quarter, sales stabilised in the third quarter due to continued strong demand for our new medicines and COVID-19 tests. Based on our current assessment, we confirm the outlook for the full-year.

Roches contributions to the fight against the COVID-19 pandemic in the third quarter:

Outlook confirmed for 2020Based on the current assessment of the COVID-19 impact, sales are expected to grow in the low- to mid-single digit range, at constant exchange rates. Core earnings per share are targeted to grow broadly in line with sales, at constant exchange rates. Roche expects to increase its dividend in Swiss francs further.

Group salesIn the first nine months of 2020, Group sales increased 1% to CHF 44.0 billion.

Sales in the Pharmaceuticals Division decreased 1% to CHF 34.3 billion. Sales grew strongly in the first quarter (+7%). As a result of COVID-19, they decreased in the second quarter (-6%) and since summer first signals of recovery are seen (-4% in the third quarter). Key growth drivers were the cancer medicine Tecentriq, the multiple sclerosis medicine Ocrevus, the haemophilia medicine Hemlibra, Actemra/RoActemra in immunology and Perjeta in breast cancer.

With a strong growth of 35% the new medicines generated sales of CHF 13.7 billion and grew by CHF 3.7 billion at constant exchange rates over 2019, more than offsetting the impact of the competition from biosimilars (sales reduction CHF 3.5 billion at constant exchange rates).

In the US, overall sales decreased 4%. While sales of Ocrevus, Hemlibra, Tecentriq, Actemra/RoActemra and Kadcyla increased, the competition from biosimilars for Herceptin, MabThera/Rituxan and Avastin affected total growth as expected. Ocrevus sales increased by 23% and were driven by both new and returning patient demand but partly impacted by COVID-19 effects. Hemlibra sales increased 68%, resulting from the ongoing rollout in the US. Tecentriq sales increased by 46%, driven by the launch in unresectable hepatocellular carcinoma (HCC) as well as the growth in the new indications extensive stage small cell lung cancer (ES-SCLC) and metastatic triple-negative breast cancer.

In Europe, sales increased 4% as the strong demand for Tecentriq, Ocrevus, Hemlibra, Kadcyla and Perjeta was able to offset the impact of lower sales of Herceptin (-32%), Avastin (-16%) and MabThera/Rituxan (-32%). The first biosimilar versions of Avastin were introduced in Europe in the third quarter of 2020.

In the International region (+6%), growth was mostly driven by Perjeta, Actemra/RoActemra, Alecensa, Tecentriq and Ocrevus, partially offset by the impact of the National Reimbursement Drug List update in China and COVID-19.

Sales decreased in Japan (-6%) as a result of the considerable competition from biosimilars, generics, COVID-19 and government price cuts. This decline was partially compensated by recently launched products including Tecentriq and Hemlibra.

The Diagnostics Division recorded very strong sales growth of 9% to CHF 9.7 billion, with particularly strong growth of 18% in the third quarter. After a 5% increase in the first quarter, momentum slowed to 2% growth in the second quarter as a result of the pandemic. The overall very strong sales growth is primarily due to the industry-leading portfolio of new COVID-19 tests. The Molecular Diagnostics business made the largest contribution (+77%) with PCR tests for COVID-19. Sales of diagnostic solutions for SARS-CoV-2 developed only this year clearly exceeded COVID-19 related declines in routine diagnostics sales. Additional product launches in the third quarter, including the SARS-CoV-2 antigen rapid test, further strengthened Roche's position as the world's leading supplier of COVID-19 tests.

Growth was reported in North America (+22%), EMEA3 (+9%), Latin America (+12%) and Japan (+5%). In the Asia-Pacific region (-4%), sales were heavily impacted by the pandemic, especially in China. Overall, demand was impacted by COVID-19 in all regions since the second quarter. Routine testing decreased significantly due to a decline in regular health checks while emergency and SARS-CoV-2 testing increased significantly.

Roches contributions to the fight against the COVID-19 pandemicIn September, the Elecsys Anti-SARS-CoV-2 S antibody test was launched for markets accepting the CE Mark. Roche has filed for Emergency Use Authorisation (EUA) from the FDA. The Elecsys Anti-SARS-CoV-2 S immunology test, which targets antibodies against the spike protein, can be used to quantitatively measure antibodies in people who have been exposed to SARS-CoV-2 and can play an important part in characterising a vaccine-induced immune response. The majority of current candidate vaccines aim to induce an antibody response against the spike protein of the virus.

In the same month, Roche received an EUA from the FDA for its cobas SARS-CoV-2 & Influenza A/B test for use on the cobas 6800/8800 Systems. This test is intended for the simultaneous qualitative detection and differentiation of SARS-CoV-2, influenza A and influenza B in patients suspected by their healthcare provider of having a respiratory viral infection; it is also available in markets accepting the CE Mark.

For urgent and emergency care settings, Roche also received EUA from the FDA in September for the cobas SARS CoV-2 & Influenza A/B test on the cobas Liat System, which provides results in 20 minutes.

Roche also launched the SARS-CoV-2 rapid antigen test in markets accepting the CE Mark and plans to submit files for an EUA with the FDA. The SARS-CoV-2 rapid antigen test is for use in point of care settings for symptomatic people. This can help healthcare professionals identify a SARS-CoV-2 infection in people suspected of carrying the virus with results typically ready in 15 minutes. In addition, it serves as a valuable initial screening test for individuals who have been exposed to SARS-CoV-2-infected patients or in a high-risk environment.

Roche also announced that it intends to launch a high-volume SARS-CoV-2 Antigen test as an aid in the diagnosis of SARS-CoV-2 infection. The test is planned to be made available at the end of 2020 for markets accepting the CE Mark. Roche also intends to file for EUA from the FDA. The test is performed by healthcare professionals and uses swab samples from patients with signs and symptoms suggestive of COVID-19, or people with either known or suspected exposure to SARS-CoV-2.

The portfolio of our recently developed SARS-CoV-2 tests as well as our existing diagnostics menu for critical care have become a significant factor in supporting patient management during the COVID-19 pandemic. Roche has already increased its overall production of tests fourfold over the usual volumes and has committed significant funds to continue expanding productions capacity for PCR tests over the coming year. Our investments are expected to result in more than 1,000 new jobs in the US and Europe.

The phase III Empacta study met its primary endpoint, showing that patients with COVID-19 associated pneumonia who received Actemra/RoActemra plus standard of care were 44% less likely to progress to mechanical ventilation or death compared to patients who received placebo plus standard of care. The cumulative proportion of patients who progressed to mechanical ventilation or death by day 28 was 12.2% in the Actemra/RoActemra arm versus 19.3% in the placebo arm. There was no statistical difference in mortality between patients who received Actemra/RoActemra or placebo by day 28. Approximately 85% of the 389 patients were from minority racial and ethnic groups. The trial was conducted in Brazil, Kenya, Mexico, Peru, South Africa and the USA.

In August, Roche and Regeneron joined forces in the fight against COVID-19 to develop, manufacture and distribute REGN-COV2, Regenerons investigational antiviral antibody cocktail. Regeneron has submitted a request to the FDA for an Emergency Use Authorization (EUA) for REGN-COV2. Initial data from the REGN-COV2 phase II portion of an ongoing study showed a reduction in viral load, an acceleration of symptom alleviation and a decrease in medical visits in non-hospitalised patients with COVID-19. Additional data from this study are expected by the end of 2020. We will be working with health authorities and global health institutions in a concerted, collective response, with the aim of achieving broad approvals.

Drug launches, filings, pivotal phase III trial readouts and pivotal trial starts planned by the Roche Group are largely on track.

The Covacta study of Actemra/RoActemra did not meet its primary endpoint of improved clinical status in hospitalised adult patients with severe COVID-19-associated pneumonia. In addition, the key secondary endpoints, which included the difference in patient mortality at week four, were not met; however, there was a positive trend in time to hospital discharge in patients treated with Actemra/RoActemra. This study generated robust information which will help physicians make decisions about the treatment of patients with this disease.

Overview of Roche Diagnostics COVID-19 diagnostic solutions developed in the first nine months of 2020

Regulatory achievements in the third quarterRegulators around the globe granted approvals for new Roche medicines, line extensions of existing medicines and new tests.

The FDA approved Evrysdi (risdiplam), an oral medication for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. Evrysdi showed clinically meaningful improvements in motor function across two clinical trials in people with varying ages and levels of disease severity, including types 1, 2, and 3 SMA. Evrysdi also improved survival without permanent ventilation at 12 and 23 months of treatment, compared to natural history.

FDA approval was granted for Enspryng (satralizumab-mwge) as the first and only subcutaneous treatment for adults living with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a rare, lifelong and debilitating autoimmune disorder of the central nervous system, often misdiagnosed as multiple sclerosis, that primarily damages the optic nerve(s) and spinal cord, causing blindness, muscle weakness and paralysis. Enspryng demonstrated significant reduction in the risk of relapse compared with placebo as a monotherapy and when used concurrently with baseline immunosuppressant therapy.

The FDA also approved Gavreto (pralsetinib) for the treatment of adults with metastatic RET fusion-positive non-small cell lung cancer as detected by an FDA approved test. This indication was approved under the FDAs Accelerated Approval programme, based on data from the phase I/II ARROW study. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

The European Commission granted conditional marketing authorisation for Rozlytrek for the treatment of adult and paediatric patients 12 years of age and older with solid tumours expressing a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and who have not received a prior NTRK inhibitor and who have no satisfactory treatment options. The European Commission has also approved Rozlytrek for the treatment of adults with ROS1-positive, advanced non-small cell lung cancer (NSCLC) not previously treated with ROS1 inhibitors.

The FDA also granted approval for Tecentriq plus Cotellic and Zelboraf for the treatment of BRAF V600 mutation-positive advanced melanoma patients.

In Japan, Tecentriq in combination with Avastin, was approved by the Ministry of Health, Labour and Welfare for the treatment of unresectable hepatocellular carcinoma.

The FoundationOne Liquid companion diagnostic test (F1L CDx) received FDA approval. Furthermore, the comprehensive liquid biopsy service received the CE Mark in May. Both regulatory milestones allow for the new test to be commercialised in all markets that recognise the CE Mark and/or the FDA approval. F1L CDx is the most comprehensive pan-tumour liquid biopsy test for all solid tumours, incorporating multiple companion diagnostics. This test supports efforts to improve treatment results by helping oncologists optimise and personalise treatment for their patients with advanced cancer during all lines of therapy, and particularly for those where tissue-based testing is not possible.

Additional regulatory achievements in the third quarter of 2020:

Diagnostics key launches in the third quarter In addition to the new COVID-19 portfolio, Roche received FDA approval for the Ventana HER2 Dual ISH DNA Probe Cocktail assay for the detection of the HER2 biomarker in breast cancer and as a companion diagnostic for Herceptin therapy. HER2 - human epidermal growth factor receptor 2 - is an important biomarker sometimes found in breast cancers. Its detection and inhibition can help healthcare professionals manage this aggressive cancer more effectively. This new assay is designed to be completed within the same day, enabling clinicians to get results back faster than with other common methods of confirmatory testing for HER2. Results can be read using light microscopy, eliminating the need for a specialised fluorescence microscope.

The FDA also authorised the cobas EBV test, the first quantitative in vitro diagnostic test for Epstein-Barr virus (EBV) DNA in the US. The test meets World Health Organization standards for consistent result reporting among laboratories across the US, allowing for results to be easily comparable across hospitals and laboratories. Monitoring of Epstein-Barr virus DNA can help prevent the progression of life-threatening diseases such as cancer in transplant patients.

Roche also received FDA clearance for the BK virus quantitative test on cobas 6800/8800 Systems to support better care for transplant patients. The test provides standardised, high-quality results that can help healthcare professionals better assess the risk of complications caused by the BK virus in transplant patients and identify effective treatment options.

In August, the FDA approved the cobas HIV-1/HIV-2 qualitative test for use on the fully automated cobas 6800/8800 Systems in the US. The test provides healthcare professionals with a single result to confirm HIV diagnosis and differentiate HIV-1 and HIV-2, an important distinction needed to identify appropriate treatment options.

In September, Roche launched the Elecsys HIV Duo immunoassay in the US, following FDA approval in April 2020. Through separate measurement of the HIV p24 antigen (the virus) and anti-HIV antibodies (caused by immune reaction), this test can detect an acute HIV infection earlier than current methods. This approval enables a robust, comprehensive infectious diseases menu on the cobas e 801 system and a significant step towards bringing holistic value to the US market within the area of infectious diseases.

Roche announced FDA approval in September for the expanded use of CINtec PLUS Cytology, the first triage test based on biomarker technology for women whose cervical cancer screening results are positive for high-risk types of human papillomavirus (HPV). Additional information from this test supports clinical decisions on which women will benefit most from immediate follow-up. Laboratories can now use CINtec PLUS Cytology to triage positive results from the cobas HPV Test run on the fully integrated, automated and high-throughput cobas 6800/8800 Systems.

Key development milestones in the third quarter of 2020Regulatory filings and product launches for 2020 as well as pivotal trial read-outs and pivotal starts in 2020 are largely on track. We are making significant efforts to protect all studies with continued support from health authorities, but the ultimate outcome will depend on the length and severity of the pandemic.

Results from the phase III IMpassion031 study, evaluating Tecentriq in combination with chemotherapy (Abraxane, albumin-bound paclitaxel; nab-paclitaxel; followed by doxorubicin and cyclophosphamide) in comparison with placebo plus chemotherapy (including nab-paclitaxel), demonstrated a statistically significant and clinically meaningful improvement in pathological complete response (pCR) for the treatment of people with early TNBC, regardless of PD-L1 expression. The IMpassion031 study is the second positive phase III study from Roche to demonstrate the benefit of Tecentriq in TNBC and the first Tecentriq study to demonstrate a benefit in early TNBC.

The final overall survival (OS) analysis of the phase III IMpassion130 study, evaluating Tecentriq in combination with nab-paclitaxel, compared with placebo plus nab-paclitaxel, as a first-line treatment for patients with metastatic TNBC, was consistent with the first and second interim analyses. There was no significant difference in OS between the treatment groups in the ITT population. Clinically meaningful improvements of 7.5 months in median OS were seen with Tecentriq plus nab-paclitaxel in PD-L1-positive patients.

Detailed results from the phase III Archway study showed that 98.4% of Port Delivery Systems (PDS) patients were able to go six months without needing additional treatment and achieved vision outcomes equivalent to patients receiving monthly ranibizumab eye injections, a current standard of care. This study evaluates the investigational Port Delivery System with ranibizumab for the treatment of neovascular or wet age-related macular degeneration (nAMD), a leading cause of blindness globally.

New two-year data from part 1 of the pivotal Firefish study of Evrysdi in infants aged two to seven months with symptomatic Type 1 SMA showed that infants treated with the therapeutic dose of Evrysdi (17/21) continued to improve and achieve motor milestones.

Pharmaceuticals Division

Key pharmaceutical productsAvastin (-22%). For advanced colorectal, breast, lung, kidney, cervical and ovarian cancer, and relapsed glioblastoma (a type of brain tumour). Sales were impacted by the biosimilar competition in the US, Europe and Japan.

MabThera/Rituxan (-27%). For forms of blood cancer, rheumatoid arthritis and certain types of vasculitis. The sales decline was driven by all regions, due to the launch of biosimilars in the US and most EU markets and in Japan.

Herceptin (-31%). For HER2-positive breast cancer and HER2-positive metastatic gastric cancer. Sales were impacted by biosimilars in the US, Europe and Japan. In the US, the switch to Kadcyla in the adjuvant setting also impacted sales.

Actemra/RoActemra (+33%). For rheumatoid arthritis, forms of juvenile idiopathic arthritis and giant cell arteritis as well as CAR T cell-induced severe or life-threatening cytokine release syndrome. A number of countries included Actemra/RoActemra in their treatment guidelines for severe COVID-19 pneumonia. Actemra/RoActemra is not currently approved for this use; Roche is conducting several phase III clinical studies. The US and the International region were the major contributors to the sales increase.

Xolair (+2%, US only). For chronic idiopathic urticaria and allergic asthma. The sales increase was driven by the demand in both indications. Xolair remains the market leader in the larger allergic asthma indication.

Lucentis (-14%, US only). For eye conditions, including neovascular (wet) age-related macular degeneration, macular oedema following retinal vein occlusion, diabetic macular oedema, and diabetic retinopathy. Sales decreased in all approved indications and were especially affected by the COVID-19 pandemic due to disruptions in hospitals and ophthalmology practices and many patients delaying treatment during restrictions.

Highlights for medicines launched since 2012Ocrevus (first approved in 2017; CHF 3.3 billion, +29%). For the treatment of both the relapsing (RMS) and primary progressive (PPMS) forms of multiple sclerosis (MS). The strong demand for this treatment in both indications has continued, while the COVID-19 pandemic has had a certain negative impact. In the US, growth was driven both by new and returning patients, with a higher proportion of sales coming from returning patients. In Europe and the International region, Ocrevus continues to show strong initial uptake where launched.

Perjeta (first approved in 2012; CHF 2.9 billion, +17%). As therapy for HER2-positive breast cancer. Sales grew strongly in the International region. The increased patient demand for Perjeta for adjuvant early breast cancer therapy supports its continued strong growth.

Tecentriq (first approved in 2016; CHF 2.0 billion, +64%). Approved either alone or in combination with targeted therapies and/or chemotherapies in various forms of NSCLC, in small cell lung cancer (SCLC), certain types of metastatic urothelial cancer, and in PD-L1-positive metastatic TNBC. In the US and several other countries, Tecentriq in combination with Avastin is approved for people with unresectable or metastatic HCC and in the US and two other countries Tecentriq is approved in combination with Cotellic and Zelboraf for the treatment of people with BRAF V600 mutation-positive advanced melanoma. Strong sales growth was reported by all regions, driven mainly by the indications in ES-SCLC and TNBC. Sales in Japan increased due to robust uptake in first-line NSCLC and first-line ES-SCLC.

Hemlibra (first approved in 2017; CHF 1.6 billion, +79%). For treating people with haemophilia A with factor VIII inhibitors. It is also approved to treat people with haemophilia A without factor VIII inhibitors. Hemlibra is the only prophylactic treatment that can be administered subcutaneously and with multiple dosing options (once weekly, once every two weeks or once every four weeks). Sales continued to show a strong uptake in all regions, despite COVID-19 restrictions having some impact on potential new patients.

Kadcyla (first approved in 2013; CHF 1.3 billion, +37%). For treating HER2-positive breast cancer. The increased demand for Kadcyla was driven by its usage in the early breast cancer setting. Sales benefited from the positive read-out from the Katherine study and patients switching to the new standard of treatment.

Esbriet (first approved in 2014; CHF 844 million, +9%). For idiopathic pulmonary fibrosis. Sales continued to expand, driven by growth in the US and Europe.

Alecensa (first approved in 2015; CHF 841 million, +35%). To treat ALK-positive lung cancer. Alecensa showed continued sales growth across all regions.

Gazyva/Gazyvaro (first approved in 2013; CHF 472 million, +27%). For chronic lymphocytic leukaemia (CLL), rituximab-refractory follicular lymphoma and previously untreated advanced follicular lymphoma. Sales increased in all regions.

Polivy (first approved in 2019; CHF 126 million, +>500%). Part of combination therapy for the treatment of adults with relapsed or refractory diffuse large B-cell lymphoma.

Xofluza (first approved in 2018; CHF 28 million, +286%). For the treatment of acute, uncomplicated influenza, or flu, in people 12 years of age and older and people with high risk of developing flu-related complications.

Rozlytrek (first approved in 2019; CHF 15 million, +319%). For a specific form of NSCLC and for solid tumours expressing a specific gene fusion. In Japan, Rozlytrek was approved for treatment of ROS1 fusion- positive NSCLC.

Evrysdi (risdiplam, first approved in 2020; CHF 8 million*4). For the treatment of spinal muscular atrophy (SMA) in adults and children two months of age and older.

Phesgo (fixed-dose combination of Perjeta and Herceptin with hyaluronidase, first approved in 2020; CHF 7 million*). For the treatment of early and metastatic HER2-positive breast cancer by subcutaneous injection (SC) administered in combination with intravenous chemotherapy.

Enspryng (satralizumab, first approved in 2020; CHF 7 million*). For a rare neurodegenerative disease (neuromyelitis optica spectrum disorder).

Diagnostics Division

In the first nine months 2020, COVID-19 and emergency testing strongly increased while routine testing decreased as a result of continued declining or delayed regular health checks and medical appointments. Nevertheless, Roches broad, diversified test portfolio and its large number of instruments installed worldwide provided for a strong sales growth.

During 2020, Roche has increased its production capacity (reagents and consumables) for COVID-19 testing massively. This includes all our products used in fighting COVID-19 infections.

Centralised and Point of Care Solutions sales declined by 7%, its immunodiagnostics business (-8%) was strongly impacted by the COVID-19 impact on routine testing worldwide. COVID-19 related products such as the Elecsys Anti-SARS-CoV-2 test, Custom Biotech, Elecsys IL-6 test and the SARS-CoV-2 rapid antigen test partly offset the COVID-19 impact.

Sales in Molecular Diagnostics increased 77%, with 88% growth in the underlying molecular business. Growth was driven by virology (predominantly SARS-CoV-2), Quantitative PCR (to detect molecular/genetic targets) and Nucleic Acid Purification (to isolate and purify genetic material), Molecular Diagnostics systems, Molecular Point-of-Care (influenza viruses).

Diabetes Care sales decreased 2%, with the continued contraction of the Blood Glucose Monitoring (BGM) market due to patients switching to Continuous Glucose Monitoring (CGM) systems. The COVID-19 pandemic also had an impact. The decrease was reflected mainly in the EMEA region, notably in Germany, UK and Italy. The positive uptake of digital diabetes management solutions continued: AccuChek SugarView, RocheDiabetes Care Platform and mySugr.

Tissue Diagnostics sales increased 5%, supported by advanced staining, instrument sales and companion diagnostics business. However, overall sales were impacted by the COVID-19 pandemic.

About RocheRoche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve peoples lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the worlds largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the eleventh consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2019 employed about 98,000 people worldwide. In 2019, Roche invested CHF 11.7 billion in R&D and posted sales of CHF 61.5 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit http://www.roche.com.

All trademarks used or mentioned in this release are protected by law.

References[1] Unless otherwise stated, all growth rates in this document are at constant exchange rates (CER: average 2019).[2] Launched since 2012: Erivedge, Perjeta, Kadcyla, Gazyva, Esbriet, Cotellic, Alecensa, Tecentriq, Ocrevus, Hemlibra, Xofluza, Polivy, Rozlytrek, Phesgo, Enspryng, Evrysdi[3] EMEA = Europe, Middle East and Africa[4] recently launched, no growth figures available

Cautionary statement regarding forward-looking statementsThis Annual Report contains certain forward-looking statements. These forward-looking statements may be identified by words such as believes, expects, anticipates, projects, intends, should, seeks, estimates, future or similar expressions or by discussion of, among other things, strategy, goals, plans or intentions. Various factors may cause actual results to differ materially in the future from those reflected in forward-looking statements contained in this Annual Report, such as: (1) pricing and product initiatives of competitors; (2) legislative and regulatory developments and economic conditions; (3) delay or inability in obtaining regulatory approvals or bringing products to market; (4) fluctuations in currency exchange rates and general financial market conditions; (5) uncertainties in the discovery, development or marketing of new products or new uses of existing products, including without limitation negative results of clinical trials or research projects, unexpected side effects of pipeline or marketed products; (6) increased government pricing pressures; (7) interruptions in production; (8) loss of or inability to obtain adequate protection for intellectual property rights; (9) litigation; (10) loss of key executives or other employees; and (11) adverse publicity and news coverage.

The statement regarding earnings per share growth is not a profit forecast and should not be interpreted to mean that Roches earnings or earnings per share for 2020 or any subsequent period will necessarily match or exceed the historical published earnings or earnings per share of Roche.Roche Group Media RelationsPhone: +41 61 688 8888 / e-mail: media.relations@roche.com

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First nine months of the year with 1% sales growth at constant exchange rates, significant impact of COVID-19 pandemic - GlobeNewswire

Study May Lead to Biomarkers for Tailoring COVID-19 Vaccines and Therapeutics

Potential Development of Human Monoclonal Antibody Therapeutics to SARS-CoV-2

Results from the PRECISION Study Expected in First Half of 2021

CHATHAM, N.J., Oct. 15, 2020 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced that the first participant was enrolled in the observational PRECISION study (TNX-C002), to examine the immune responses to COVID-19 in healthy volunteers who have recovered from COVID-19 or were asymptomatic. The research is part of an ongoing collaboration between Columbia University and Tonix that focuses on T cell and antibody responses to SARS-CoV-2 (CoV-2), the virus that causes COVID-19. The research encompasses two projects. The study led by Dr. Ilya Trakht, Ph.D., Associate Research Scientist at Columbia University Vagelos College of Physicians and Surgeons, has the potential to lead to the isolation and characterization of therapeutically relevant fully human monoclonal antibodies to SARS-CoV-2. The study led by Dr. Sergei Rudchenko, Ph.D., Assistant Professor of Medical Sciences at Columbia University Vagelos College of Physicians and Surgeons, is designed to generate DNA aptamer-based anti-idiotypes to selected monoclonal antibodies identified in Dr. Ilya Trakhts study. Such aptamers have the potential to identify biomarkers for protective CoV-2 immunity and may lead to accelerated precision medicine-driven vaccines designed to protect against COVID-19.

Data from the PRECISION study may help guide research to determine which vaccine or therapeutic is appropriate on an individual basis, said Seth Lederman, M.D., President and Chief Executive Officer of Tonix Pharmaceuticals. This work may also guide the selection of appropriate participants for clinical trials of COVID-19 vaccines, including future human trials using Tonixs TNX-1800, a live replicating, attenuated COVID-19 vaccine candidate designed to confer durable T cell immunity.

About TNX-1800

TNX-1800 is a live modified horsepox virus vaccine for percutaneous administration that is designed to express the Spike protein of the SARS-CoV-2 virus that causes COVID-19 and to elicit a predominant T cell response. Horsepox and vaccinia are closely related orthopoxviruses that are believed to share a common ancestor. Live replicating orthopoxviruses, like vaccinia or horsepox, can be engineered to express foreign genes and have been explored as platforms for vaccine development because they possess; (1) large packaging capacity for exogenous DNA inserts, (2) precise virus-specific control of exogenous gene insert expression, (3) lack of persistence or genomic integration in the host, (4) strong immunogenicity as a vaccine, (5) ability to rapidly generate vector/insert constructs, (6) readily manufacturable at scale, and (7) ability to provide direct antigen presentation. Relative to vaccinia, horsepox has substantially decreased virulence in mice1. Horsepox-based vaccines are designed to be single dose, vial-sparing vaccines, which can be manufactured on conventional cell culturing systems, with the potential for mass scale production.

1Noyce RS, et al. (2018) PLoS One. 13(1):e0188453

About Tonix Pharmaceuticals Holding Corp.

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing small molecules and biologics to treat and prevent human disease and alleviate suffering. Tonixs portfolio is primarily composed of central nervous system (CNS) and immunology product candidates. The immunology portfolio includes vaccines to prevent infectious diseases and biologics to address immunosuppression, cancer and autoimmune diseases. The CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonixs lead vaccine candidate, TNX-1800*, is a live replicating vaccine based on the horsepox viral vector platform to protect against COVID-19, primarily by eliciting a T cell response. Tonix expects data from animal studies of TNX-1800 in the fourth quarter of this year. TNX-801*, live horsepox virus vaccine for percutaneous administration, is in development to protect against smallpox and monkeypox and serves as the vector platform on which TNX-1800 is based. Tonix is also developing TNX-2300* and TNX-2600*, live replicating vaccine candidates for the prevention of COVID-19, but using bovine parainfluenza as the vector. Tonixs lead CNS candidate, TNX-102 SL**, is in Phase 3 development for the management of fibromyalgia. The Company expects topline data in the Phase 3 RELIEF study in the fourth quarter of 2020. Tonix is also currently enrolling participants in the Phase 3 RALLY study for the management of fibromyalgia using TNX-102 SL, and the results are expected in second half of 2021. TNX-102 SL is also in development for agitation in Alzheimers disease and alcohol use disorder (AUD). Both programs are Phase 2 ready, and the AAD program has FDA Fast Track designation. Tonixs programs for treating addiction conditions also include TNX-1300* (T172R/G173Q double-mutant cocaine esterase 200 mg, i.v. solution), which is in Phase 2 development for the treatment of life-threatening cocaine intoxication and has FDA Breakthrough Therapy designation. TNX-601 CR** (tianeptine oxalate controlled-release tablets) is another CNS program, currently in Phase 1 development as a daytime treatment for depression while TNX-1900**, intranasal oxytocin, is in development as a non-addictive treatment for migraine and cranio-facial pain. Tonixs preclinical pipeline includes TNX-1600** (triple reuptake inhibitor), a new molecular entity being developed as a treatment for PTSD; TNX-1500* (anti-CD154), a monoclonal antibody being developed to prevent and treat organ transplant rejection and autoimmune conditions; and TNX-1700* (rTFF2), a biologic being developed to treat gastric and pancreatic cancers.

*TNX-1800, TNX-801, TNX-2300, TNX-2600, TNX-1300, TNX-1500 and TNX-1700 are investigational new biologics and have not been approved for any indication.

**TNX-102 SL, TNX-601 CR, TNX-1600 and TNX-1900 are investigational new drugs and have not been approved for any indication.

This press release and further information about Tonix can be found at http://www.tonixpharma.com.

Forward Looking Statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as anticipate, believe, forecast, estimate, expect, and intend, among others. These forward-looking statements are based on Tonix's current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2019, as filed with the Securities and Exchange Commission (the SEC) on March 24, 2020, and periodic reports filed with the SEC on or after the date thereof. All of Tonix's forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.

Contacts

Jessica Morris (corporate)Tonix Pharmaceuticalsinvestor.relations@tonixpharma.com(212) 688-9421

Travis Kruse (media)Russo Partnerstravis.kruse@russopartnersllc.com(212) 845-4272

Peter Vozzo (investors)Westwickepeter.vozzo@westwicke.com(443) 213-0505

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Tonix Pharmaceuticals Enrolls First Participant in the PRECISION Study, an Observational Study to Facilitate Development of Precision Medicine...

WALTHAM, Mass., Oct. 15, 2020 (GLOBE NEWSWIRE) -- OncXerna Therapeutics, Inc., a precision medicine company using an innovative RNA-based biomarker platform to predict patient responses for potentially first-in-class targeted oncology therapies, today announced that Laura Benjamin, Ph.D., Founder and CEO of OncXerna, will participate at the Jefferies Virtual London Healthcare Conference taking place November 17-19, 2020.

About OncXerna Therapeutics

OncXerna is aiming to deliver next-generation precision medicine for a larger group of cancer patients by leveraging the companys deep understanding of how to prospectively identify patients based on the dominant, RNA-based biology of their tumor microenvironments. This allows OncXerna to pair those patients with OncXernas clinical-stage therapies and known mechanism of action that directly address these biologies, to dramatically improve patient outcomes. For more information on OncXerna, please visit oncxerna.com/

About OncXernas RNA-based Biomarker Platform

Existing precision medicines target only approximately 10% of cancersthose with gene mutations or oncogenic drivers for a small number of genes. Using its proprietary biomarker platform, OncXerna is leveraging the companys deep understanding of tumor biology at the RNA level to identify the dominant biology underlying a patients cancer. OncXernas first biomarker panel is specific to the tumor microenvironment (TME Panel-1). Initial results from TME Panel-1 reveal 4 different dominant biologies, demonstrating the presence of specific patient subgroups and their predictive value in responding to treatment. OncXerna is further optimizing the biomarker platforms tumor microenvironment panel through multiple research collaborations, including a collaboration with Moffitt Cancer Center.

About Bavituximab

Bavituximab is an investigational antibody that reverses immune suppression by inhibiting phosphatidylserine (PS) signaling and is currently in Phase 2 clinical trials to treat a specific subset of patients with advanced gastric cancer to improve their response to anti-PD-1 treatment. The mechanism of action of bavituximab is to block tumor immune suppression signaling from PS to multiple immune cell receptor families (e.g., TIMs and TAMs). The dominant biology targeted by bavituximab may be relevant for patients with many types of solid tumors whose immune systems are too suppressed to benefit from currently available immune oncology therapies. OncXernas clinical trials currently combine bavituximab with KEYTRUDA to test the hypothesis that relieving immunosuppression can enhance responses to checkpoint inhibitors. Bavituximab is an investigational agent that has not been licensed or approved anywhere globally, and it has not been demonstrated to be safe or effective for any use, including for the treatment of advanced gastric cancer.

About Navicixizumab

Navicixizumab is an investigational anti-DLL4/VEGF bispecific antibody that has demonstrated antitumor activity in patients who have progressed on Avastin (bevacizumab) in a Phase 1a/b clinical trial. The U.S. Food and Drug Administration granted Fast Track designation to navicixizumab for the treatment of high-grade ovarian, primary peritoneal or fallopian tube cancer in patients who have received at least three prior therapies and/or prior treatment with Avastin. OncXerna is targeting patients whose dominant tumor biology is driven by angiogenesis with a focus beyond VEGF to include broader anti-angiogenic pathways. Navicixizumab is an investigational agent that has not been licensed or approved anywhere globally, and it has not been demonstrated to be safe or effective for any use, including for the treatment of advanced ovarian cancer.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

Investor and Media Contact:

Ashley R. RobinsonLifeSci Partners, LLCarr@lifesciadvisors.com

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OncXerna Therapeutics to Participate at the Jefferies Virtual London Healthcare Conference - GlobeNewswire