Category Archives: Immunology

Immunology

To vax or not to vax: Pitt scientist discusses importance of vaccines – The Times

A recent poll shows that half of Americans say theyll get a COVID-19 vaccination when it becomes available.

Only about half of Americans in a recent poll say theyll get a COVID-19 vaccination when it becomes available, according to a recent study. A Pittsburgh-based immunology expert said he hopes they change their minds.

The Associated Press-NORC Center for Public Affairs Research found that if a vaccine against coronavirus becomes available to the public, only about half of those polled said they plan to get vaccinated; 20 percent said they will not; and about a third of those polled are not sure.

I advocate vaccines. They clearly have been shown to be safe in multiple studies, said Dr. William Klimstra, a professor of immunology at the University of Pittsburgh, during a Facebook Live discussion with Auditor General Eugene DePasquale. You just have to basically accept when you have essentially universal consensus among people who should know about something, in this case that vaccines are safe.

The top reason folks said they would be less inclined to get the vaccine is because of safety, particularly fear of potential side effects of the vaccine.

Seven out of 10 people polled who do not plan to get the vaccine said they are concerned about the side effects with nearly half being concerned with contracting the coronavirus. About one in three said they are not concerned about getting seriously ill from the virus.

Dr. William Klimstra, who works in the Department of Immunology and the Microbiology and Molecular Genetics Department at the University of Pittsburgh, is a member of the Center for Virus Research. An advocate of vaccines, he has been involved in several previous vaccine designs.

Klimstra spoke with Pennsylvania Auditor General Eugene DePasquale during a Facebook Live session on Wednesday and said the anti-vaccine rhetoric on social media and parts of the internet should not be accepted by anybody who thinks about this critically.

Were in an environment right now where long-standing excepted truths are being challenged through social media and other things, he said. Ive read a lot of the anti-vaccine literature and things that are on the internet, and people seem to feel Big Pharma is promoting these things and then scientists because they get grants. They have been compromised because of Big Pharma.

Klimstra said that vaccines are advocated by doctors, because theyre proven to be safe and effective.

I advocate vaccines. They clearly have been shown to be safe in multiple studies, he said. You just have to basically accept when you have essentially universal consensus among people who should know about something, in this case that vaccines are safe.

One argument against vaccines is that it causes autism, which Klimstra says, has been debunked completely.

Safety is scientists foremost priority when it comes to vaccine creation, Klimstra said. This is why its taking months to create a COVID-19 vaccine, he said.

The reason that its taking longer a frustratingly long time to get a coronavirus vaccine in the marketplace is because of the safety testing, he said.

Klimstra said people can have adverse reactions to vaccines. But its just vanishingly small with vaccines. And the links to autism are just not held up by science, he said.

Hundreds of millions of lives have been saved because of vaccines, Klimstra stressed.

Looking back, historically vaccines are one of the most productive and important medical technologies that have ever been used, he said.

Similarly to using masks, Klimstra said its not just about keeping yourself safe vaccines help prevent transmission of the virus.

The Associated Press-NORC Center for Public Affairs Research poll showed the top reasons for the nearly half of folks who said they would get a coronavirus vaccine are about protecting them, their family and their communities from the virus.

Among those polled, four out of five said they believe a vaccine is "an important criteria" for reopening, while nearly half say it's essential. About one-third say a vaccine is not essential for reopening.

Klimstra said vaccines are proven to be effective, calling them standard medical treatments.

Weve eliminated smallpox. Polio is almost eliminated, measles was nearly eliminated until people stopped taking the vaccine, he said. You wouldnt go to the doctor and not have a treatment for a medical condition where the treatment was nearly completely safe and worked a high percentage of the time. You wouldnt do that.

And thats what a vaccine is a standard medical treatment that saves lives. You just have to listen to people who should know about the issue.

For anti-vaxxers and those who arent convinced to get the coronavirus vaccine when it eventually becomes available, Klimstra said he hopes they will change their minds.

Its a critical issue both for themselves and for other people, he said.

Klimstra called it having a protective attitude and a positive community attitude.

These vaccines will be safe. I can guarantee that, he said. Given the technologies that will be utilized, they will be safe.

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To vax or not to vax: Pitt scientist discusses importance of vaccines - The Times

Immunology

CBD treatment improves arthritis symptoms in dogs, study finds – News-Medical.Net

Reviewed by Emily Henderson, B.Sc.May 28 2020

A team led by researchers at Baylor College of Medicine in collaboration with Medterra CBD conducted the first scientific studies to assess the potential therapeutic effects of cannabidiol (CBD) for arthritic pain in dogs, and the results could lead the way to studying its effect in humans. Researchers focused first on these animals because their condition closely mimics the characteristics of human arthritis, the leading cause of pain and disability in the U.S. for which there is no effective treatment.

Published in the journal PAIN, the study first showed both in laboratory tests and mouse models that CBD, a non-addictive product derived from hemp (cannabis), can significantly reduce the production of inflammatory molecules and immune cells associated with arthritis. Subsequently, the study showed that in dogs diagnosed with the condition, CBD treatment significantly improved quality of life as documented by both owner and veterinarian assessments. This work supports future scientific evaluation of CBD for human arthritis.

CBD is rapidly increasing in popularity due to its anecdotal health benefits for a variety of conditions, from reducing anxiety to helping with movement disorders. In 2019, Medterra CBD approached Baylor to conduct independent scientific studies to determine the biological capabilities of several of its products."

Dr. Matthew Halpert, corresponding author, research faculty in the Department of Pathology and Immunology at Baylor

In the current study, Halpert and his colleagues first measured the effect of CBD on immune responses associated with arthritis, both in human and murine cells grown in the lab and in mouse models. Using Medterra tinctures, they found that CBD treatment resulted in reduced production of both inflammatory molecules and immune cells linked to arthritis.

The researchers also determined that the effect was quicker and more effective when CBD was delivered encapsulated in liposomes than when it was administered 'naked.' Liposomes are artificially formed tiny spherical sacs that are used to deliver drugs and other substances into tissues at higher rates of absorption.

Halpert and colleagues next assessed the effect of naked and liposome-encapsulated CBD on the quality of life of dogs diagnosed with arthritis.

"We studied dogs because experimental evidence shows that spontaneous models of arthritis, particularly in domesticated canine models, are more appropriate for assessing human arthritis pain treatments than other animal models. The biological characteristics of arthritis in dogs closely resemble those of the human condition," Halpert said.

Arthritis is a common condition in dogs. According to the American Kennel Club, it affects one out of five dogs in the United States.

The 20 client-owned dogs enrolled in the study were seen at Sunset Animal Hospital in Houston. The dog owners were randomly provided with identical unidentified medication bottles that contained CBD, liposomal CBD, or a placebo. Neither the owners nor the veterinarian knew which treatment each dog received.

After four weeks of daily treatment, owners and veterinarians reported on the condition of the dogs, whether they observed changes in the animals' level of pain, such as changes related to running or gait. The dogs' cell blood count and blood indicators of liver and kidney function also were evaluated before and after the four weeks of treatment.

"We found encouraging results," Halpert said. "Nine of the 10 dogs on CBD showed benefits, which remained for two weeks after the treatment stopped. We did not detect alterations in the blood markers we measured, suggesting that, under the conditions of our study, the treatment seems to be safe."

The findings support conducting studies to evaluate CBD for the treatment of human arthritis.

Source:

Journal reference:

Verrico, C., et al. (2020) A randomized, double-blind, placebo-controlled study of daily cannabidiol for the treatment of canine osteoarthritis pain. Pain. doi.org/10.1097/j.pain.0000000000001896.

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CBD treatment improves arthritis symptoms in dogs, study finds - News-Medical.Net

Immunology

Early Immunological Studies Show Universally Effective Antibodies Against SARS-CoV-2 in Recovered Humans – Technology Networks

The first round of results from an immunological study of 149 people who have recovered from COVID-19 show that although the amount of antibodies they generated varies widely, most individuals had generated at least some that were intrinsically capable of neutralizing the SARS-CoV-2 virus.

Antibodies vary widely in their efficacy. While many may latch on to the virus, only some are truly neutralizing, meaning that they actually block the virus from entering the cells.

Since April 1, a team of immunologists, medical scientists, and virologists, has been collecting blood samples from volunteers who have recovered from COVID-19. The majority of the samples they have studied showed poor to modest neutralizing activity, indicating a weak antibody response. However, a closer look revealed everyones immune system is capable of generating effective antibodiesjust not necessarily enough of them. Even when neutralizing antibodies were not present in an individuals serum in large quantities, researchers could find some rare immune cells that make them.

This suggests just about everybody can do this, which is very good news for vaccines, says Michel C. Nussenzweig, head of the Laboratory of Molecular Immunology at Rockefeller. It means if you were able to create a vaccine that elicits these particular antibodies, then the vaccine is likely to be effective and work for a lot of people.

Moreover, the researchers identified three distinct antibodies that were shown to be the most potent of the bunch in neutralizing the virus. They are working to develop them further into therapeutic and preventive drugs.

The findings are shared on BioRxiv ahead of submission to peer-reviewed scientific journals. Nussenzweigs collaborators include, Davide F. Robbiani, Marina Caskey, Paul Bieniasz, Theodora Hatziioannou, and Charles M. Rice.

From the beginning of April and over 5 weeks, 149 people who had recovered from COVID-19 visited The Rockefeller Hospital to donate plasma, the portion of the blood that contains the antibodies, and the immune B-cells that produce them. The participants had experienced symptoms for an average of 12 days, and had their first symptoms on average 39 days before plasma donation.

Bieniasz and Hatziioannous team used an essay they had developed to test the neutralizing activity of the plasma samples. This involved mixing the plasma with a pseudo SARS-CoV-2 virus and measuring how well this mixture could still infect human cells in a dish.

In 33 percent of donors, the neutralizing activity of plasma was below detectable levels. Its possible that for many in this group, their immune systems first line of defense had resolved the infection quickly, before the antibody-producing cells were called in.

The majority of the plasma samples showed poor to modest neutralizing activity. And for 1 percent of donors it was remarkably high. Like in other diseases, everyone responds differently, says Robbiani, research associate professor at Laboratory of Molecular Immunology. Some people have poor response, some average. And then there is a fraction of people that are exceptional responders.

Those elite responders are crucial to the teams plans. The high numbers of neutralizing antibodies in their serum makes it possible for researcher to catch the rare B cells that make them. They can then clone the antibodies from those cells, and use them to emulate the same strong defense in other people.

Out of the numerous antibodies generated by elite responders who had the best performing plasma, the team identified 40 that neutralized the virus, and zeroed in on three that could do so even at very low concentrations. The team has cloned these most potent antibodies and is now working to develop them for clinical use.

Neutralizing antibodies found in this study bind to at least three distinct sites on the receptor-binding domain (RBD) subunit of the spike protein, which is what SARS-CoV-2 uses to gain entry to host cells. A second look at the low-performing plasma samples revealed they also contained these RBD-binding antibodies, albeit in small quantities.

We now know what an effective antibody looks like and we have found similar ones in more than one person, Robbiani says. This is important information for people who are designing and testing vaccines. If they see their vaccine can elicit these antibodies, they know they are on the right track.

ReferenceConvergent Antibody Responses to SARS-CoV-2 Infection in Convalescent Individuals. Davide F. Robbiani et al. bioRxiv, doi: https://doi.org/10.1101/2020.05.13.092619.

This article is based on research findings that are yet to be peer-reviewed. Results are therefore regarded as preliminary and should be interpreted as such. Find out about the role of the peer review process in research here. For further information, please contact the cited source.

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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Early Immunological Studies Show Universally Effective Antibodies Against SARS-CoV-2 in Recovered Humans - Technology Networks

Immunology

IHW organises web summit on vasculitis care during COVID-19 organised – Express Healthcare

Rheumatologists, immunologists highlighted that rheumatoid diseases caused by malfunctioning of immune system need continuous care, voluntary discontinuation of prescribed medicine will cause more harm than good

At a time when fear of coronavirus infection is adding to worries of patients living with chronic diseases, rheumatologists and immunologists at the IHW Councils web summit on vasculitis care during COVID-19 highlighted that rheumatoid diseases caused by malfunctioning of the immune system need continuous care and voluntary discontinuation of prescribed medicine will cause more harm than good.

Vasculitis is a combination of diseases. Though it is not very common in India but the impact of the disease can be very serious. Though the medicines prescribed for vasculitis are immunosuppressant in nature, they do not make the patient more vulnerable to COVID-19 infection. It will be highly recommended that vasculitis patients should not stop their medicines until they have confirmed COVID-19 infection and are advised by doctors to do so as the mortality of vasculitis is far higher than COVID-19, saidDr Debasish Danda, Professor & Founder of Clinical Immunology & Rheumatology, CMC Vellore; President-Elect, Asia Pacific League of Associations for Rheumatology (APLAR).

While highlighting a greater need for awareness about diseases like vasculitis, which has nearly 20 different types of conditions, Dr Danda said, Unfortunately, we do not know the right prevalence of the disease in India as there is no population-based study. According to facility-based studies conducted at the Christian Medical College, Vellore, vasculitis variant like Takayasu has seen 600 patients in 15 years and this is among the largest facility-based studies conducted anywhere in the world. Diseases like Takayasu, a form of vasculitis that harms the medium blood vessels, are often misdiagnosed as TB due to thesimilarity in symptoms such as fever and rapid weight loss.

Discussing the types of vasculitis,Dr Ramnath Misra, Head, Clinical Immunology & Rheumatology, KIMS, Bhubaneswar & Ex- Dean and Head Clinical Immunology, SGPGIMS, Lucknow,said, There are two types of vasculitis: firstly, primary vasculitis where the name of the vasculitis type depends on the size of the affected vessels. The most common types of vasculitis in this segment are Takayasu, Kawasaki, etc. The other type is known as secondary vasculitis which happens due to any existing disease in the patient. The number of patients affected by secondary vasculitis outnumbers the patients suffering from primary vasculitis.

Globally, May is observed as Vasculitis Awareness Month. Recently, a number of COVID-19-infected children from Europe and North America have shown multi-system the inflammatory condition with some features similar to those of Kawasaki disease and toxic shock syndrome, prompting intensive care arrangements.

Kawasaki is a medium vessel vasculitis that is almost exclusively seen in very young children, especially infants and those below five. Though there is no known cause of the disease, Kawasaki disease may have an association with coronavirus, as Northwestern Universitys Kawasaki disease expert Dr Anne Rowley suggests that serious illness and shock in children may have been caused by the mutation of the virus. Kawasaki is not a rare disease Japan sees 13,000 new cases annually, while at PGI Chandigarh, we get about 80-100 children every year with symptoms of Kawasaki disease, saidDr Surjit Singh, Head, Department of Paediatrics and Chief, Allergy Immunology Unit, Advanced Paediatrics Centre, PGIMER, Chandigarh & Principal Investigator, ICMR Centre for Advanced Research in Primary Immunodeficiency Diseases.

The common symptoms of Kawasaki disease are redness in eyes, lips, tongue as well as swelling in neck, hands and feet. Early detection of the disease is the single-most effective way to prevent any further damage it is advisable to diagnose the disease within 5-6 days, otherwise it may cause permanent damage to coronary arteries. In many cases adult heart attack patients were found to have Kawasaki disease as children that remained undiagnosed, Dr Singh added.

Talking about Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV),Dr Aman Sharma, Professor, Clinical Immunology and Rheumatology, Internal Medicine, PGIMER, Chandigarh, said, ANCA-associated vasculitis causes inflammation in small vessels and can even destroy them, damaging organs. We receive about 350-400 patients every year in PGIMER Chandigarh vasculitis is not as rare as we think it to be.

The virtual summit on managing vasculitis and coronavirus, organised by theIntegrated Health and Wellbeing (IHW) Council, was also attended by Dr Vishad Viswanath, Director, Institute of Rheumatology & Immunology Research, Thiruvananthapuram, Kerala and Dr Rohini Samant, Professor, Department of Rheumatology, Hinduja National Hospital, Mumbai.

Kamal Narayan, CEO, IHW Council, who moderated the session, said, Though vasculitis diseases are rare, there is a high possibility that they have substantial prevalence in India, given our large pool of population. Clinicians are almost unanimous in their views that it is not possible to prevent such diseases as there is no scientific knowledge on what causes these diseases. In such a condition, early and timely diagnosis of vasculitis diseases itself is a big prevention as it can help in effective treatment and management. Following our philosophy of enabling people to live a healthy life, we found it important to make more people aware about these diseases which can help in early reporting and diagnosis.

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IHW organises web summit on vasculitis care during COVID-19 organised - Express Healthcare

Immunology

Third-year UND medical student among volunteers on state penitentiary immunology teams – Grand Forks Herald

Espelien was one person on three four-person immunology teams to conduct mass testing at the North Dakota State Penitentiary two weeks ago. About half of those teams are made up of medical student volunteers, said John Hagan, a UND School of Medicine professor who also oversees medical care in Department of Corrections and Rehabilitation facilities. Espelien had worked in the prison one other time, on a mental health rotation as part of her education, but her latest task at the penitentiary - donning full-body personal protective equipment and administering COVID-19 tests to state prisoners in their pods - was unlike any she'd been asked to do before as a medical student.

"I was really excited to be able to talk to patients and do hands-on patient care for the first time in months," she said. "It was good to get back in the swing of things, and I really enjoyed talking to the residents there. A lot of them were joking and in fairly good spirits, with the whole thing going on."

It's widely acknowledged that prison and jail populations are particularly vulnerable to COVID-19. Due to the confined space, prisoners' inability to practice social distancing, and staff's limited ability to isolate sick inmates, once the virus is introduced to a prison population, it has been found to spread efficiently.

DOCR took quick steps early in the pandemic to prevent the spread of the virus in their facilities. They closed their doors to new intakes on March 13, and Hagan said North Dakota's prisons have reduced their population by about 15% - but mass testing is still essential for protecting those populations, he said.

Two weeks ago, Hagan put out a call to his medical students and residents looking for volunteers for the immunology teams in the prisons.

"We've actually had more requests to come out than we have slots," Hagan said. "I love the fact that these are adults who are in advanced learning, and they're eager to get out and take care of sick people. I'm pretty proud of them."

Espelien, an Albuquerque native who completed her undergraduate studies in San Diego, was drawn to North Dakota by the quality of UND's medical school and family ties in Park River, and, going into her fourth year of medical school, she aims to apply for an OB/GYN residency. Her work in the penitentiary was part of Hagan's internal medicine rotation. She said she's not aware of any other medical school that offers rotations in state prisons.

Her work with the immunology team began Wednesday, May 13, and lasted three days, the time it took to complete the mass tests in the North Dakota State Penitentiary. Mass tests also were completed in four other DOCR facilities, but only the testing event in the NDSP yielded any positive test results - four people in custody reportedly tested positive for the virus, according to the DOCR website.

"That'll be good information for them to know, and they'll be able to isolate those residents and, hopefully, keep all the fellow residents and staff members safe," Espelien said. "I think it's important to keep doing this epidemiologic testing at the penitentiaries to make sure that we can keep any outbreaks controlled and make sure that this vulnerable patient population is protected."

Four positive results were initially reported in the Missouri River Correctional Center in Bismarck as well, but the case count in MRCC was updated to zero after the North Dakota Department of Health reported that malfunctioning testing equipment yielded 82 false positives statewide last week.

Espelien said tasks were split among the team members. One person screened patients' temperatures and asked questions about symptoms, and another team member ensured samples were labeled properly and the correct paperwork was filled out. Espelien was one of the team members who administered the swab tests.

Residents of the penitentiary had the option to either take the test or decline the test and isolate for 14 days. Espelien said that, though a few people weren't happy about the tests, which can be uncomfortable to take, she said everyone was pleasant and had generally positive attitudes.

"I think that there are just some questions about how things are going outside of the penitentiary, since they only get to see what's on the news or what they hear from family members and staff," Espelien said. "So some of them were asking me questions about when antibody tests will be available, or how the throat swab is different from the nasal swab, and if one is better than the other. So I guess there was an air of curiosity, more than fear or worry or things like that."

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Third-year UND medical student among volunteers on state penitentiary immunology teams - Grand Forks Herald

Immunology

argenx Announces Positive Topline Results from Phase 3 ADAPT Trial of Efgartigimod in Patients with Generalized Myasthenia Gravis – GlobeNewswire

Regulated information Inside information

May 26, 2020Breda, the Netherlands / Ghent, Belgium argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer, today announced positive topline data from the pivotal ADAPT trial of efgartigimod. ADAPT met its primary endpoint defined as percentage of responders on the Myasthenia Gravis Activities of Daily Living (MG-ADL) score among acetylcholine receptor-antibody positive (AChR-Ab+) generalized myasthenia gravis (gMG) patients. Responders are defined as having at least a two-point improvement on the MG-ADL score for at least four consecutive weeks. Based on these results, argenx plans to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) by the end of 2020.

Highlights of topline ADAPT data

The efgartigimod data showed rapid and robust responses in people with gMG, as well as a favorable tolerability profile, said James F. Howard Jr., M.D., Professor of Neurology (Neuromuscular Disease), Medicine and Allied Health, Department of Neurology, The University of North Carolina at Chapel Hill School of Medicine and principal investigator for the ADAPT trial. Patients with this devastating disease can experience chronic and potentially life-threatening muscle weakness that has a major impact on their quality of life, and more treatment options are needed. These data are very encouraging as they show efgartigimod has potential to make a meaningful impact on daily living activities, and we are hopeful they will lead to a new treatment being available for the gMG community.

With the ADAPT trial, we set out to evaluate efgartigimods ability to redefine the treatment paradigm for people living with gMG. The data showed that efgartigimod drove fast and deep responses, including in a proportion of patients who achieved minimal or no symptoms after treatment. In addition, we saw responses that lasted beyond eight or 12 weeks, supporting our plans to offer individualized dosing schedules that are purpose-fit to the variability in disease course that gMG patients experience, commented Wim Parys, M.D., Chief Medical Officer of argenx. Based on these data, we intend to submit a BLA for efgartigimod to the FDA before the end of the year, taking us one step closer to potentially making efgartigimod available to patients in 2021. All of us at argenx want to thank the patients and healthcare providers who participated in the ADAPT trial. ADAPT is the first pivotal trial of efgartigimod and these data further our confidence in its broad opportunity in other severe, IgG-mediated autoimmune diseases.

Additional ADAPT results, including secondary endpoints and prespecified analyses

Detailed data from the ADAPT trial will be submitted for presentation at a future medical meeting.

Phase 3 ADAPT Trial DesignThe Phase 3 ADAPT trial was a randomized, double-blind, placebo-controlled, multi-center, global trial evaluating the safety and efficacy of efgartigimod in patients with gMG. A total of 167 adult patients with gMG in North America, Europe and Japan enrolled in the trial and were treated. Enrolled patients had a confirmed gMG diagnosis and an MG-ADL total score of five or greater. Patients were on a stable dose of at least one gMG treatment prior to randomization, including acetylcholinesterase inhibitors, corticosteroids or nonsteroidal immunosuppressive drugs, and were required to remain on that stable dose throughout the primary trial. Patients were eligible to enroll in ADAPT regardless of antibody status, including patients with AChR antibodies (AChR-Ab+) and patients where AChR antibodies were not detected.

Patients were randomized in a 1:1 ratio to receive efgartigimod or placebo for a total of 26 weeks as part of the primary trial. ADAPT was designed to enable an individualized treatment approach with an initial treatment cycle followed by a variable number of subsequent treatment cycles. Treatment cycles consist of four infusions of efgartigimod (10mg/kg IV) or placebo at weekly intervals. Retreatment with additional treatment cycles was initiated according to clinical response. The primary endpoint was the number of AChR-Ab+ patients who achieved a response on the MG-ADL score defined by at least a two-point improvement for four or more consecutive weeks.

After the 26-week primary ADAPT trial, patients were eligible to roll-over into an open-label extension, ADAPT Plus.

About Efgartigimod

Efgartigimod is a first-in-class antibody fragment designed to reduce disease-causing immunoglobulin G (IgG) antibodies and block the IgG recycling process. Efgartigimod binds to the neonatal Fc receptor (FcRn), which is widely expressed throughout the body and plays a central role in rescuing IgG antibodies from degradation. Blocking FcRn reduces IgG antibody levels representing a logical potential therapeutic approach for several autoimmune diseases known to be driven by disease-causing IgG antibodies, including: myasthenia gravis (MG), a chronic disease that causes muscle weakness; pemphigus vulgaris (PV), a chronic disease characterized by severe blistering of the skin; immune thrombocytopenia (ITP), a chronic bruising and bleeding disease; and chronic inflammatory demyelinating polyneuropathy (CIDP), a neurological disease leading to impaired motor function.

About Myasthenia Gravis (MG)

MG is a rare and chronic autoimmune disease where IgG antibodies disrupt communication between nerves and muscles, causing debilitating and potentially life-threatening muscle weakness. More than 85% of people with MG progress to generalized MG (gMG) within 18 months, where muscles throughout the body may be affected, resulting in extreme fatigue and difficulties with facial expression, speech, swallowing and mobility. In more life-threatening cases, MG can affect the muscles responsible for breathing. Patients with confirmed AChR antibodies account for 80-90% of the total gMG population. There are approximately 65,000 people in the United States and 20,000 people in Japan living with the disease.

Conference Call DetailsManagement will host a conference call and webcast presentation today at 2:30 p.m. Central European Summer Time (CEST) / 8:30 a.m. Eastern Daylight Time (EDT). To participate in the conference call, please select your phone number below and use the confirmation code 6295982. The webcast may be accessed on the Investors page of the argenx website at http://www.argenx.com or by clicking here.

Dial-in numbers:Please dial in 510 minutes prior to 2:30 p.m. CEST / 8:30 a.m. EDT using the number and conference ID below.

Confirmation Code: 6295982

Belgium: +32 (0)2 793 3847Belgium: 0800 484 71France: +33 (0)1 7070 0781France: 0805 101 465Netherlands: +31 (0)2 0795 6614Netherlands: 0800 023 5015UK: +44 (0) 844 481 9752UK: 0800 279 6619US: +1646 741 3167US: 1877 870 9135

About argenxargenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx is translating immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx is evaluating efgartigimod in multiple serious autoimmune diseases, and cusatuzumab in hematological cancers in collaboration with Janssen. argenx is also advancing several earlier stage experimental medicines within its therapeutic franchises. argenx has offices in Belgium, the United States and Japan. For more information, visit http://www.argenx.com and follow us on LinkedIn at https://www.linkedin.com/company/argenx/.

Contacts:

Beth DelGiacco, Vice President, Investor Relations (US)+1 518 424 4980bdelgiacco@argenx.com

Joke Comijn, Director Corporate Communications & Investor Relations (EU)+32 (0)477 77 29 44+32 (0)9 310 34 19JComijn@argenx.com

Forward-looking Statements

The contents of this announcement include statements that are, or may be deemed to be, forward-looking statements. These forward-looking statements can be identified by the use of forward-looking terminology, including the terms believes, estimates, anticipates, expects, intends, may, will, or should and include statements argenx makes concerning the safety, tolerability and efficacy of efgartigimod and the results of the ADAPT trial; the timing of planned regulatory submissions with the FDA and, if approved, launch in the U.S.; the therapeutic and commercial potential of efgartigimod; the opportunity of efgartigimod in other severe, IgG-mediated autoimmune diseases; and the intended results of its strategy.By their nature, forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. argenxs actual results may differ materially from those predicted by the forward-looking statements as a result of various important factors, including argenxs expectations regarding its the inherent uncertainties associated with competitive developments, preclinical and clinical trial and product development activities and regulatory approval requirements; failure to demonstrate the safety, tolerability and efficacy of argenxs product candidates; final and quality controlled verification of data and the related analyses; the expense and uncertainty of obtaining regulatory approval, including from the U.S. Food and Drug Administration and European Medicines Agency; the possibility of having to conduct additional clinical trials; argenxs reliance on collaborations with third parties; estimating the commercial potential of argenxs product candidates; argenxs ability to obtain and maintain protection of intellectual property for its technologies and drugs; argenxs limited operating history; and argenxs ability to obtain additional funding for operations and to complete the development and commercialization of its product candidates. A further list and description of these risks, uncertainties and other risks can be found in argenxs U.S. Securities and Exchange Commission (SEC) filings and reports, including in argenxs most recent annual report on Form 20-F filed with the SEC as well as subsequent filings and reports filed by argenx with the SEC. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. argenx undertakes no obligation to publicly update or revise the information in this press release, including any forward-looking statements, except as may be required by law.

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argenx Announces Positive Topline Results from Phase 3 ADAPT Trial of Efgartigimod in Patients with Generalized Myasthenia Gravis - GlobeNewswire

Immunology

Q32 Bio Launches to Develop Novel Therapeutics Targeting Regulators of Innate and Adaptive Immunity – P&T Community

CAMBRIDGE, Mass., May 27, 2020 /PRNewswire/ --Q32 Bio, a biotechnology company developing biologics to restore healthy immune regulation, today announced the company's pipeline and plans to enter the clinic this year with its lead candidate. Enabled by a $46M Series A financing led by Atlas Venture, and a world class team and Scientific Advisory Board, Q32 Bio is advancing a portfolio of biologics targeting the body's innate and adaptive immune systems. The company has a robust product pipeline including a monoclonal antibody antagonist of the interleukin-7 receptor (IL-7R), and a complement therapeutics platform that has generated fusion proteins that downregulate complement activity specifically in disease-affected tissues. The company is led by industry veterans Michael Broxson as Chief Executive Officer, and co-founder, Shelia Violette, Ph.D., as Chief Scientific Officer and President of Research.

Q32 Bio was seeded and incubated by Atlas Venture with foundational science from renowned researchers in immunology Michael Holers, M.D. and Joshua Thurman, M.D., both from the University of Colorado, and Steven Tomlinson, Ph.D. from the Medical University of South Carolina. Dr. Holers serves as chairman of the Scientific Advisory Board, which is comprised of distinguished global experts in autoimmune and inflammatory disease.

The subsequent Series A financing included Atlas Venture, OrbiMed Advisors, Abingworth, Sanofi Ventures, University of Colorado and Children's Hospital Colorado Center for Innovation.

"Autoimmune and inflammatory diseases are driven by dysregulation of the immune response," said Mr. Broxson. "Q32 Bio has a preeminent team with expertise in both innate and adaptive immunity, a board of directors and scientific advisory board made up of leaders in our field, and a syndicate of blue-chip investors. This gives us a running start in developing therapies that may improve and save lives. I am thrilled to join Q32 Bio and look forward to advancing our first two immune-regulating therapies into first-in-human studies planned for 2020 and 2021."

Integrated Approach to Restoring Healthy Immune Regulation Q32 Bio's most advanced program, ADX-914, is a fully human anti-IL-7R antibody licensed from Bristol Myers Squibb (NYSE: BMY) that re-regulates adaptive immune function. The cytokine IL-7 has demonstrated the ability to drive several T-cell mediated pathological processes. It stimulates T-effector and T-memory cells, lowering the threshold-response to disease related antigens; inhibits the immunosuppressive function of T-regulatory cells; and promotes pathogenic autoantibody production. Inhibition of IL-7R signaling has the potential to durably and safely restore healthy immune regulation in numerous autoimmune and inflammatory diseases. Q32 Bio is advancing plans for the first-in-human trial of ADX-914 while continuing to monitor the potential impact of COVID-19 on clinical operations. Pending confirmation that trial sites are able to operate and enroll patients safely, a biomarker-guided ADX-914 Phase 1 trial is planned to start in late 2020.

Q32 Bio is developing ADX-914 (formerly known as BMS-986265) as part of an exclusive worldwide licensing agreement with Bristol Myers Squibb. Under the terms of the agreement, Bristol Myers Squibb received an upfront payment and became a minority shareholder of Q32 Bio. Bristol Myers Squibb has the potential to earn additional milestone payments subject to the achievement of certain development, regulatory and sales-based milestones, as well as tiered annual net sales royalties. Q32 Bio is solely responsible for all future development and commercialization costs of ADX-914.

Q32 Bio's lead program in innate immunity, ADX-097, is based on a groundbreaking platform providing tissue-targeted regulation of the complement system. Complement is an integral component of the innate immune system that provides a first line of defense for clearing pathogens and removing damaged cells. In a wide range of autoimmune and inflammatory diseases the complement system becomes hyperactivated, causing the immune system to attack and damage otherwise healthy tissue. Q32 Bio's proprietary and unique platform has yielded a pipeline of novel protein therapeutics that provide potent inhibition of complement in diseased tissues without long-term systemic blockade a key differentiator versus current complement therapeutics. Q32 Bio has initiated IND-enabling activities for ADX-097 and first-in-human dosing is planned for 2H 2021.

"Given their broad roles in mediating immunity, the complement system and IL-7 signaling pathways are high potential arenas for drug development," said Dave Grayzel, MD, Partner, Atlas Venture and chairman of the Q32 Bio Board of Directors. "With a best-in-class IL-7R antibody and fusion proteins designed to target the complement system at sites of activation in a unique and differentiated manner, Q32 Bio is well positioned to make a meaningful impact for patients with serious inflammatory and autoimmune diseases."

Experienced Leadership TeamMichael Broxson brings over 20 years of industry experience to Q32 Bio. He joined from Goldfinch Bio, where he was Chief Business and Operating officer. Prior to Goldfinch he served in leadership roles in business development, new product planning, strategy and finance at Takeda Pharmaceuticals. He holds BA and MSPH degrees in Toxicology from Tulane University and an MBA from the University of Chicago's Booth School of Business and is a CFA charterholder.

Shelia Violette, Ph.D., joined Q32 Bio from Atlas Venture, where she was Entrepreneur in Residence and initially co-led the formation of Q32 Bio. Previously she served in leadership roles in the Immunology Research group at Biogen, joining Stromedix as Vice President of Research and returning to Biogen as Vice President of Research, spearheading the tissue injury and fibrosis therapeutic area. She holds a Ph.D. in Pharmacology from Yale University.

About Q32 BioQ32 Bio is a biotechnology company developing therapies targeting powerful regulators of the innate and adaptive immune systems, to re-balance immunity in severe autoimmune and inflammatory diseases. Q32 Bio's lead programs, focused on the IL-7R pathway and complement system, address immune dysregulation to help patients take back control of their lives. For more information, please visit http://www.Q32bio.com.

About Atlas VentureAtlas Venture is a leading biotech venture capital firm. With the goal of doing well by doing good, the company has been building breakthrough biotech startups for over 25 years. Atlas works side by side with exceptional scientists and entrepreneurs to translate high impact science into medicines for patients. Our seed-led venture creation strategy rigorously selects and focuses investment on the most compelling opportunities to build scalable businesses and realize value. For more information, please visitwww.atlasventure.com.

About AbingworthAbingworth is a leading transatlantic life sciences investment firm. We help transform cutting-edge science into novel medicines by providing capital and expertise to top caliber management teams and building world-class companies. With offices in Menlo Park (California), Boston and London, Abingworth has invested in approximately 160 life science companies, leading to more than 40 M&A/exits and over 65 IPOs since 1973.

About OrbiMed OrbiMed is a leading healthcare investment firm, with $13 billion in assets under management. OrbiMed invests globally across the healthcare industry, from start-ups to large multinational corporations, utilizing a range of private equity funds, public equity funds, and royalty/credit funds. OrbiMed maintains offices in New York City, San Francisco, Shanghai, Hong Kong, Mumbai and Herzliya. OrbiMed seeks to be a capital provider of choice, providing tailored financing solutions and extensive global team resources to help build world-class healthcare companies.

About Sanofi VenturesSanofi Ventures is the corporate venture capital arm of Sanofi. Sanofi Ventures invests in early-stage biotech and digital health companies with innovative ideas and transformative new products and technologies of strategic interest to Sanofi. Among these areas are oncology, immunology, rare diseases, vaccines, potential cures in other core areas of Sanofi's business footprint, and digital health solutions. For more information, visitwww.sanofiventures.com.

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SOURCE Q32 Bio Inc.

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Q32 Bio Launches to Develop Novel Therapeutics Targeting Regulators of Innate and Adaptive Immunity - P&T Community

Immunology

argenx Announces Positive Topline Results from Phase 3 ADAPT Trial of Efgartigimod in Patients with Generalized Myasthenia Gravis | Antibodies | News…

DetailsCategory: AntibodiesPublished on Tuesday, 26 May 2020 10:23Hits: 154

BREDA, The Netherlands / GHENT, Belgium I May 26, 2020 I argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer, today announced positive topline data from the pivotal ADAPT trial of efgartigimod. ADAPT met its primary endpoint defined as percentage of responders on the Myasthenia Gravis Activities of Daily Living (MG-ADL) score among acetylcholine receptor-antibody positive (AChR-Ab+) generalized myasthenia gravis (gMG) patients. Responders are defined as having at least a two-point improvement on the MG-ADL score for at least four consecutive weeks. Based on these results, argenx plans to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) by the end of 2020.

Highlights of topline ADAPT data

The efgartigimod data showed rapid and robust responses in people with gMG, as well as a favorable tolerability profile, said James F. Howard Jr., M.D., Professor of Neurology (Neuromuscular Disease), Medicine and Allied Health, Department of Neurology, The University of North Carolina at Chapel Hill School of Medicine and principal investigator for the ADAPT trial. Patients with this devastating disease can experience chronic and potentially life-threatening muscle weakness that has a major impact on their quality of life, and more treatment options are needed. These data are very encouraging as they show efgartigimod has potential to make a meaningful impact on daily living activities, and we are hopeful they will lead to a new treatment being available for the gMG community.

With the ADAPT trial, we set out to evaluate efgartigimods ability to redefine the treatment paradigm for people living with gMG. The data showed that efgartigimod drove fast and deep responses, including in a proportion of patients who achieved minimal or no symptoms after treatment. In addition, we saw responses that lasted beyond eight or 12 weeks, supporting our plans to offer individualized dosing schedules that are purpose-fit to the variability in disease course that gMG patients experience, commented Wim Parys, M.D., Chief Medical Officer of argenx. Based on these data, we intend to submit a BLA for efgartigimod to the FDA before the end of the year, taking us one step closer to potentially making efgartigimod available to patients in 2021. All of us at argenx want to thank the patients and healthcare providers who participated in the ADAPT trial. ADAPT is the first pivotal trial of efgartigimod and these data further our confidence in its broad opportunity in other severe, IgG-mediated autoimmune diseases.

Additional ADAPT results, including secondary endpoints and prespecified analyses

Detailed data from the ADAPT trial will be submitted for presentation at a future medical meeting.

Phase 3 ADAPT Trial DesignThe Phase 3 ADAPT trial was a randomized, double-blind, placebo-controlled, multi-center, global trial evaluating the safety and efficacy of efgartigimod in patients with gMG. A total of 167 adult patients with gMG in North America, Europe and Japan enrolled in the trial and were treated. Enrolled patients had a confirmed gMG diagnosis and an MG-ADL total score of five or greater. Patients were on a stable dose of at least one gMG treatment prior to randomization, including acetylcholinesterase inhibitors, corticosteroids or nonsteroidal immunosuppressive drugs, and were required to remain on that stable dose throughout the primary trial. Patients were eligible to enroll in ADAPT regardless of antibody status, including patients with AChR antibodies (AChR-Ab+) and patients where AChR antibodies were not detected.

Patients were randomized in a 1:1 ratio to receive efgartigimod or placebo for a total of 26 weeks as part of the primary trial. ADAPT was designed to enable an individualized treatment approach with an initial treatment cycle followed by a variable number of subsequent treatment cycles. Treatment cycles consist of four infusions of efgartigimod (10mg/kg IV) or placebo at weekly intervals. Retreatment with additional treatment cycles was initiated according to clinical response. The primary endpoint was the number of AChR-Ab+ patients who achieved a response on the MG-ADL score defined by at least a two-point improvement for four or more consecutive weeks.

After the 26-week primary ADAPT trial, patients were eligible to roll-over into an open-label extension, ADAPT Plus.

About Efgartigimod

Efgartigimod is a first-in-class antibody fragment designed to reduce disease-causing immunoglobulin G (IgG) antibodies and block the IgG recycling process. Efgartigimod binds to the neonatal Fc receptor (FcRn), which is widely expressed throughout the body and plays a central role in rescuing IgG antibodies from degradation. Blocking FcRn reduces IgG antibody levels representing a logical potential therapeutic approach for several autoimmune diseases known to be driven by disease-causing IgG antibodies, including: myasthenia gravis (MG), a chronic disease that causes muscle weakness; pemphigus vulgaris (PV), a chronic disease characterized by severe blistering of the skin; immune thrombocytopenia (ITP), a chronic bruising and bleeding disease; and chronic inflammatory demyelinating polyneuropathy (CIDP), a neurological disease leading to impaired motor function.

About Myasthenia Gravis (MG)

MG is a rare and chronic autoimmune disease where IgG antibodies disrupt communication between nerves and muscles, causing debilitating and potentially life-threatening muscle weakness. More than 85% of people with MG progress to generalized MG (gMG) within 18 months, where muscles throughout the body may be affected, resulting in extreme fatigue and difficulties with facial expression, speech, swallowing and mobility. In more life-threatening cases, MG can affect the muscles responsible for breathing. Patients with confirmed AChR antibodies account for 80-90% of the total gMG population. There are approximately 65,000 people in the United States and 20,000 people in Japan living with the disease.

About argenxargenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx is translating immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx is evaluating efgartigimod in multiple serious autoimmune diseases, and cusatuzumab in hematological cancers in collaboration with Janssen. argenx is also advancing several earlier stage experimental medicines within its therapeutic franchises. argenx has offices in Belgium, the United States and Japan. For more information, visit http://www.argenx.com and follow us on LinkedIn at https://www.linkedin.com/company/argenx/.

SOURCE: argenx

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argenx Announces Positive Topline Results from Phase 3 ADAPT Trial of Efgartigimod in Patients with Generalized Myasthenia Gravis | Antibodies | News...

Immunology

Change in Orion Group Executive Management Board as of 1 June 2020: Professor Outi Vaarala appointed as Senior Vice President for Research and…

Outi Vaarala photo

Professor Outi Vaarala, Senior Vice President for Research and Development and member of the Executive Management Board of the Orion Group, as of 1 June 2020.

ORION CORPORATION STOCK EXCHANGE RELEASE 25 MAY 2020 AT 4.20 p.m. EEST

Change in Orion Group Executive Management Board as of 1 June 2020: Professor Outi Vaarala appointed as Senior Vice President for Research and Development

Professor Outi Vaarala, M.D., Ph.D. has been appointed Senior Vice President for Research and Development and member of the Executive Management Board of the Orion Group, as of 1 June 2020. Vaarala has held the position of Vice President, Oncology Research in Orions Research and Development as of 10 June 2019. Prior to Orion, Vaarala led drug discovery and development from target identification to clinical Phase 2 studies in Respiratory Inflammation and Autoimmunity therapy area, first at AstraZeneca, Sweden, and later at Medimmune/AstraZeneca, USA.

Outi Vaarala is a medical doctor (University of Helsinki) and has a PhD in immunology. Outi Vaarala was appointed as a Professor of Pediatric Immunology, Linkoping University, Sweden, in 2000, and as a Professor of Autoimmune Diseases, Lund University, Sweden, in 2015.

Timo Lappalainen, President and CEO, says:

Orions Research and Development is in an interesting phase. Our operations have been developed into a recognized world-class organization that has also achieved remarkable results. Outi Vaarala has excellent background and experience from various leadership positions in Research and Development in the international pharmaceutical industry as well as a long experience of immunology and remarkablecareer in the academy. She has published more than 200 peer-reviewed scientific articles in internationally renowned scientific journals. I warmly welcome Outi as a member of Orions Executive Management Board.

Outi Vaarala says:

During the past year when leading Oncology Research team at Orion, I have recognised the fabulous opportunities in Orion based on the knowledge and expertise across the organisation. This makes me excited about this new challenge ahead me. Co-operation and international networking is a key to success because nobody can develop drugs alone. Finland and Orion have a lot to offer for the proprietary drug discovery, and at the same time international networking is a crucial part of the operations of Orions Research and Development. In my new position, I want to emphasise this.

The CV of Outi Vaarala and a photo of her are attached.

Orion Corporation

Timo Lappalainen Olli HuotariPresident and CEO Senior Vice President, Corporate Functions

Contact person:

Timo Lappalainen, President and CEO, phone +358 50966 3692

Contact person for media:Terhi Ormio, Vice President, Communications, phone +358 50966 4646, terhi.ormio@orion.fi

APPENDIX:

The CV of Outi Vaarala

Outi Vaarala

MD, University of Helsinki, 1990 PhD in immunology, University of Helsinki, 1991Professor of Pediatric Immunology, Linkoping University, Sweden, 2000 Professor of Autoimmune Diseases, Lund University, Sweden, 2015

Born 1962

Senior Vice President for Research and Development, Orion Corporation as of 1 June 2020

Career

Positions of trust2005-2014 Permanent expert at the European Medicines Agency (EMA)

PublicationsMore than 200 peer-reviewed scientific articles in internationally renowned scientific journals (as in Science Translational Medicine, Cell Host Microbes, JAMA, New England Journal of Medicine, the Lancet, and Journal of Immunology).

Publisher:Orion CorporationCommunicationsOrionintie 1A, FI-02200 Espoo, Finlandhttp://www.orion.fi/en http://www.twitter.com/OrionCorpIR

Orion is a globally operating Finnish pharmaceutical company a builder of well-being. Orion develops, manufactures and markets human and veterinary pharmaceuticals and active pharmaceutical ingredients. The company is continuously developing new drugs and treatment methods. The core therapy areas of Orion's pharmaceutical R&D are central nervous system (CNS) disorders, oncology, Finnish heritage rare diseases and respiratory diseases for which Orion develops inhaled Easyhaler pulmonary drugs. Orion's net sales in 2019 amounted to EUR 1,051 million and the company had about 3,300 employees at the end of the year. Orion's A and B shares are listed on Nasdaq Helsinki.

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Change in Orion Group Executive Management Board as of 1 June 2020: Professor Outi Vaarala appointed as Senior Vice President for Research and...

Immunology

RAPT Therapeutics to Present at the Jefferies Virtual Global Healthcare Conference – GlobeNewswire

SOUTH SAN FRANCISCO, Calif., May 26, 2020 (GLOBE NEWSWIRE) -- RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage immunology-based biopharmaceutical companyfocused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in oncology and inflammatory diseases, today announced that Brian Wong, M.D., Ph.D., President and CEO, will present a corporate update and company overview at the Jefferies 2020 Healthcare Conference on Tuesday, June 2nd at 9:30 a.m. Eastern Time.

A live webcast and audio archive of the presentation may be accessed on the RAPT Therapeutics website athttps://investors.rapt.com/events-and-presentations. Please connect to the website 10 minutes prior to the presentation to ensure adequate time for any software downloads that may be necessary to listen to the webcast.

AboutRAPT Therapeutics, Inc.RAPT Therapeutics is a clinical stage immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in oncology and inflammatory diseases. Utilizing its proprietary discovery and development engine, the Company is developing highly selective small molecules designed to modulate the critical immune drivers underlying these diseases. RAPT has discovered and advanced two unique drug candidates, FLX475 and RPT193, each targeting C-C motif chemokine receptor 4 (CCR4), for the treatment of cancer and inflammation, respectively. The Company is also pursuing a range of targets, including hematopoietic progenitor kinase 1 (HPK1) and general control nonderepressible 2 (GCN2)), that are in the discovery stage of development.

Media Contact:Angela Bittingmedia@rapt.com(925) 202-6211

Investor Contact:Sylvia Wheelerswheeler@wheelhouselsa.com

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