SUMMIT, N.J., Oct. 14, 2021 /PRNewswire/ -- Seqirus, a global leader in influenza prevention, and a business of CSL Limited (ASX:CSL), today announced that The New England Journal of Medicine has published absolute efficacy data on the company's cell-based quadrivalent influenza vaccine (QIVc) from a randomized controlled trial (RCT) which met its primary endpoint.1 The study indicates that the seasonal influenza vaccine was effective and produced a sufficient immune response against influenza in children and adolescents 2 to <18 years of age over three influenza seasons in theSouthern (2017) and Northern (2017/18 and 2018/19) Hemispheres, compared to a non-influenza comparator.1 This represents the first absolute efficacy study of a cell-based influenza vaccine in children as young as two years of age.

"In this study, QIVc demonstrates absolute efficacy in children and adolescents, showing consistent benefit across three seasons and eight countries.1 This is particularly impactful giventhe disease burden in children as young as two years of age," said Jonathan Edelman, MD, Vice President, Clinical Development at Seqirus and study author. "These data add to a growing body of evidence supporting the fact that our differentiated, cell-based seasonal influenza vaccine can help provide effective protection against flu."

QIVc utilizes a cell-based influenza vaccine manufacturing process, an alternative to traditional egg-based manufacturing.2 Traditional egg-based vaccine production can cause the strain to mutate at several steps throughout the manufacturing process, which may lead to an antigenic mismatch between the circulating strains and the inactivated influenza strains contained within the seasonal influenza vaccine.2

Cell-based influenza vaccines are designed to produce an exact match to the World Health Organization (WHO)-selected influenza virus strains by avoiding egg-adapted changes, and therefore have the potential for greater vaccine effectiveness.2,3 Cell-based influenza vaccine technology may offer additional advantages over the standard influenza manufacturing process, including increased scalability and production speed in the event of an influenza pandemic.2

"Young children are at a higher risk than adults for serious influenza-related complications.4 We're particularly pleased with the results of this study because it supports the use of our differentiated, cell-based influenza vaccine technology as an effective means of influenza protection in children as young as two years old," said Gregg Sylvester, MD, Chief Medical Officer at Seqirus.

The results of this study supported the recent U.S. Food and Drug Administration (FDA) approval for an expanded age indication for use of QIVc in children 2 years of age.5 QIVc is marketed in the U.S. as FLUCELVAX QUADRIVALENT (Influenza Vaccine) and is currently under review by the FDA for an expanded age indication for children as young as six months of age.5

The Centers for Disease Control and Prevention (CDC) recommends annual seasonal influenza vaccination for everyone six months of age and older without contraindications as the best way to prevent seasonal influenza.6 It is estimated that more than 52,000 hospitalizations occurred in children younger than 18 years of age during the 2019/20 U.S. influenza season, and there were approximately over 434 influenza-related deaths in this age group.7

In the U.S., Seqirus operates a state-of-the-art cell-based manufacturing facility in Holly Springs, North Carolina, purpose-built in partnership with the Biomedical Advanced Research and Development Authority (BARDA) to increase cell-based vaccine manufacturing capacity and combat pandemic influenza threats.8 Last year, Seqirus announced plans to build a new, world-class cell-based manufacturing facility in Australia, which will be the only cell-based influenza vaccine manufacturing facility in the Southern Hemisphere.9

About the Study

This phase III/IV multi-center, randomized, observer-blind study was conducted across eight countries (Australia, Philippines, Thailand, Estonia, Finland, Lithuania, Poland, and Spain) over three influenza seasons Southern Hemisphere (2017) and Northern Hemisphere (2017/18 and 2018/19).1

The study was designed to demonstrate the efficacy and safety of QIVc in children 2 to <18 years of age, compared to a non-influenza comparator.1 A total of 4514 subjects (n= 2258 QIVc, n=2256 comparator (Menveo, meningococcal [Serogroup ACYW-135] conjugate vaccine)) were enrolled.1 The primary endpoint was the first occurrence of laboratory-confirmed influenza illness (by RT-PCR or viral culture) occurring between >14 days after last vaccination and the end of the influenza season.1 The influenza attack rate in the QIVc group was 175/2257 (7.8%; 6.5% to 10.2% over three seasons), compared with 364/2252 (16.2%; 15.2% to 17.4% over three seasons) cases in the control group.1

The efficacy of QIVc in children and adolescents against laboratory-confirmed influenza illness was 54.6% (95% CI 45.7 to 62.1), meeting the pre-specified endpoint for success and showing benefit across three seasons and eight countries.1

The safety profile of QIVc was comparable to the non-influenza comparator.1

About Seasonal Influenza

Influenza is a common, contagious seasonal respiratory disease that may cause severe illness and life- threatening complications in some people.10 Influenza can lead to clinical symptoms varying from mild to moderate respiratory illness to severe complications, hospitalization and in some cases, death.10 Because transmission of influenza viruses to others may occur one day before symptoms develop and up to 5 to 7 days after becoming sick, the disease can be easily transmitted to others.10 Estimates from the CDC report that during the 2019/20 influenza season, there were an estimated 405,000 influenza-related hospitalizations in the U.S.7 The CDC recommends annual vaccination for individuals aged 6 months and older, who do not have any contraindications.6 Since it takes about two weeks after vaccination for antibodies to develop in the body that help protect against influenza virus infection, it is recommended that people get vaccinated before influenza begins spreading in their community.6 The CDC recommends that people get vaccinated by the end of October.6 For non-pregnant adults, getting vaccinated too early (for example, in July or August), should be avoided, unless there is concern that later vaccination may not be possible, as it can be associated with reduced protection against influenza infection later in the flu season.6

About Seqirus

Seqirus is part of CSL Limited (ASX: CSL). As one of the largest influenza vaccine providers in the world, Seqirus is a major contributor to the prevention of influenza globally and a transcontinental partner in pandemic preparedness. With state-of-the-art production facilities in the U.S., the U.K. and Australia, and leading R&D capabilities, Seqirus utilizes egg, cell and adjuvant technologies to offer a broad portfolio of differentiated influenza vaccines in more than 20 countries around the world.

About CSL

CSL (ASX:CSL) is a leading global biotechnology company with a dynamic portfolio of life-saving medicines, including those that treat hemophilia and immune deficiencies, as well as vaccines to prevent influenza. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL including our two businesses, CSL Behring and Seqirus provides life- saving products to more than 100 countries and employs more than 27,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For more information about CSL Limited, visit http://www.csl.com.

For more information visit http://www.seqirus.com and http://www.csl.com.

Intended Audience

This press release is issued from Seqirus USA Inc. in Summit, New Jersey, USA and is intended to provide information about our global business. Please be aware that information relating to the approval status and labels of approved Seqirus products may vary from country to country. Please consult your local regulatory authority on the approval status of Seqirus products.

Forward-Looking Statements

This press release may contain forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause our actual results, performance or achievements to be materially different from any future results, performances or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements.

FLUCELVAX QUADRIVALENT (Influenza Vaccine)IMPORTANT SAFETY INFORMATION

What is FLUCELVAX QUADRIVALENT (Influenza Vaccine)?

FLUCELVAX QUADRIVALENT is a vaccine that helps protect people aged 2 and older from the flu. Vaccination with FLUCELVAX QUADRIVALENT may not protect all people who receive the vaccine.

Who should not get FLUCELVAX QUADRIVALENT?

You should not get FLUCELVAX QUADRIVALENT if you have had a severe allergic reaction to any of the ingredients in the vaccine.

Before receiving FLUCELVAX QUADRIVALENT, tell your healthcare provider about all medical conditions, including if you:

What are the most common side effects of FLUCELVAX QUADRIVALENT?

Additional side effects seen in children include:

These are not all of the possible side effects of FLUCELVAX QUADRIVALENT. You can ask your healthcare provider for more information and for advice about any side effects that concern you.

To report SUSPECTED ADVERSE REACTIONS, contact Seqirus at 1855358-8966 or VAERS at 18008227967 or http://www.vaers.hhs.gov.

You are also encouraged to report negative side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch or call 1800FDA1088.

Before receiving this vaccine, please see the full US Prescribing Information for FLUCELVAX QUADRIVALENT. The information provided here does not include all that is known about FLUCELVAX QUADRIVALENT. To learn more, talk with your healthcare provider or pharmacist.

FLUCELVAX QUADRIVALENT is a registered trademark of Seqirus UK Limited or its affiliates.

All other trademarks referenced herein are the property of their respective owners.

USA-QIVc-21-0085

MEDIA CONTACTMaria Tortoreto+1 (201) 248-5208[emailprotected]

REFERENCES

1 Nolan T, Fortanier AC, Leav B, et al. Efficacy of a Cell-CultureDerived Quadrivalent Influenza Vaccine in Children. N Engl J Med. DOI: 10.1056/NEJMoa2024848.

2 Centers for Disease Control and Prevention (CDC). (2021). Cell-Based Flu Vaccines. Retrieved from: https://www.cdc.gov/flu/prevent/cell-based.htm. Accessed September 2021.

3 Rajaram, S., Boikos, C., Gelone, et al. (2020). Influenza Vaccines: The Potential Benefits of Cell-Culture Isolation and Manufacturing. Therapeutic Advances in Vaccines and Immunotherapy. DOI: 10.1177/2515135520908121.

4 CDC. (2021). Flu & Young Children. Retrieved from: https://www.cdc.gov/flu/highrisk/children.htm. Accessed September 2021.

5 FLUCELVAX QUADRIVALENT (Influenza Vaccine) [package insert]. Holly Springs, NC: Seqirus Inc; 2021.

6 CDC. (2021). Who Needs a Flu Vaccine and When. Retrieved from: https://www.cdc.gov/flu/prevent/vaccinations.htm. Accessed September 2021.

7 CDC. (2021). Estimated Influenza Illnesses, Medical visits, Hospitalizations, and Deaths in the United States 20192020 Influenza Season. Retrieved from: https://www.cdc.gov/flu/about/burden/2019- 2020.html. Accessed September 2021.

8 This project has been funded in whole or in part with Federal funds from the Office of the Assistant Secretary for Preparedness and Response, Biomedical Advanced Research and Development Authority, under contract numbers HHSO10020060001F2C, HHSO100200700030C, HHSO100200900101C and HHSO100201200003I.

9 Data on file. (2020). Seqirus USA Inc.

10 CDC. (2021). Key Facts about Influenza (Flu). Retrieved from: https://www.cdc.gov/flu/about/keyfacts.htm. Accessed September 2021.

SOURCE Seqirus

https://www.seqirus.com/

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The New England Journal of Medicine Publishes First-of-its-Kind Study on Cell-Based Quadrivalent Seasonal Influenza Vaccine (QIVc) Efficacy in...

BOSTON--(BUSINESS WIRE)--Ensoma, a gene therapy company advancing the future of medicine through precision in vivo engineering of blood and immune cells, today announced the appointment of Emile Nuwaysir, Ph.D., as president, chief executive officer and member of the companys board of directors. In conjunction with the CEO appointment, Paula Soteropoulos, executive chairman of Ensoma, will transition to the role of chairman of the board, and Kush M. Parmar, M.D., Ph.D., managing partner of 5AM ventures, will continue to serve as a member of the board.

Since launching Ensoma earlier this year, we have assembled a talented and diverse group of academic, scientific and clinical experts, initiated a strategic partnership with Takeda and have made significant progress on our platform and initial programs. Now, we are thrilled to welcome Emile to lead the team as we advance our platform and expand our strategic focus across multiple therapeutic areas ranging from rare genetic diseases to complex diseases of the immune system, such as oncology and autoimmunity disorders, said Ms. Soteropoulos. With more than 20 years of industry experience, a deep understanding of the gene and cell therapy landscape and as an entrepreneur with a track record for building successful life science companies from the ground up, Emile will be a tremendous asset to Ensoma as we seek to fully realize the power and promise of our platform to precisely edit and modify a patients own specific cells with a one-time in vivo curative medicine. We look forward to beginning our next chapter under Emiles leadership.

Dr. Nuwaysir is currently the chairman of the Alliance for Regenerative Medicine, an international advocacy organization comprised of 400+ member companies dedicated to advancing cell and gene medicines. He joins Ensoma from BlueRock Therapeutics, an independently operated subsidiary of Bayer Pharmaceuticals, where he was CEO and continues to serve as chairman of the companys board of directors. As the CEO of BlueRock from inception, he helped build a platform company at the intersection of cellular and genetic medicine, develop a pipeline of first-in-class therapeutic programs, and managed the companys acquisition and operation with Bayer. Prior to BlueRock, Dr. Nuwaysir served as president and chief operating officer of Cellular Dynamics International (CDI), A Fujifilm Company. He played a leading role in growing CDI and its stem cell therapy efforts from its inception, including its early efforts as a pre-revenue startup, to its successful IPO on Nasdaq and ultimate acquisition by Fujifilm. He was also president of Opsis Therapeutics Inc., CDI's retinal cell therapy subsidiary. Before CDI, Dr. Nuwaysir was the first employee of NimbleGen Systems, serving as a member of the senior management team that built a successful company and orchestrated a dual-track IPO process leading to the company's acquisition by Roche Diagnostics. After the acquisition of NimbleGen, Dr. Nuwaysir served as chief technical officer of the Roche NimbleGen subsidiary. Dr. Nuwaysir is also currently vice chairman of Invenra Inc., a multi-specifics antibody platform company he co-founded in 2011. He has held postdoctoral fellowships at the National Institutes of Health and the University of North Carolina at Chapel Hill. He holds a B.A. from the University of Delaware and a Ph.D. in molecular toxicology with a focus on oncology from the University of Wisconsin-Madison.

I am simply thrilled to join Ensoma. The company is poised to deliver on the full promise of genetic medicine, with the ability to precisely engineer the cells of the hematopoietic system through a single, simple, in vivo process, said Dr. Nuwaysir. The elegance of the platform, combined with its unprecedented payload capacity, will enable applications of genetic medicine that are simply undreamed of today. We will create an entire new class of smart immune cells with the power to provide cures for hundreds of genetic diseases, as well as complex diseases like cancer and autoimmunity. I look forward to joining the team and to the possibility of a brighter future for patients in need. I want to thank Paula and Kush for their vision and important commitment in launching the company and look forward to working with them in their continuing board roles.

About EnsomaEnsoma believes the future of medicine lies within us. Our Engenious gene therapy platform is the first to precisely engineer any or all hematopoietic and immune cells with a one-time, off-the-shelf in vivo treatment. Engenious therapies have an unmatched capacity to deliver all modern genetic modification tools and set a new standard for versatile cell programming and control across a range of complex diseases. Ensoma is supported by top-tier investors, a strategic collaboration with Takeda and a passionate team committed to a bold, global vision for gene therapy. Ensoma is based in Boston. For more information, visit http://www.ensoma.com.

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Ensoma Appoints Cell and Gene Therapy Pioneer Emile Nuwaysir, Ph.D., as Chief Executive Officer - Business Wire

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Credit: UAB

BIRMINGHAM, Ala. Nine out of 10 patients with systemic lupus erythematosus the autoimmune disease also known as SLE are females. Black women have the highest concentration of disease severity, and they are two to three times more likely to suffer SLE than are white women. The disease usually strikes during childbearing years, ages 15 to 45.

To help Black women with SLE, the University of Alabama at Birmingham and two other institutions have received a three-year, $3 million Global Team Science Award from the Lupus Research Alliance. The research will focus on better understanding this difficult disease, where patients often respond differently to the same treatment and vary widely in the severity of their symptoms.

Ignacio Sanz, M.D., chief of Rheumatology at Emory University School of Medicine, leads the multidisciplinary team. His Emory colleagues, together with research teams at UAB and the Medical University of South Carolina, or MUSC, will focus specifically on African American patients, identifying the characteristics of the B cells and plasma cells that create attacking antibodies in the development and progression of severe lupus. The project will define how the different characteristics of these cells correspond to patients responses to treatment.

A key to this research by UAB, Emory and MUSC is their access to a unique cohort of Black SLE patients from the American South, using large cohorts composed of rural and urban patients who receive care at the three institutions.

At UAB, this includes the SLE Clinic, which is enriched for severe patients from Alabamas rural Black Belt and from Birminghams urban communities. At Emory, this includes the Georgians Organized Against Lupus cohort, the largest community-based Black SLE cohort in the nation, with urban and rural Black Belt patients who have high poverty and disproportionately poor outcomes. At MUSC, 30 percent of the MUSC SLE Program patients are from the Gullah cohort, a group unique in their low white admixture, their genetic homogeneity and a double familial prevalence of lupus. The Gullah are Blacks who live in the Lowcountry of Atlantic states from Georgia to North Carolina, both on the coastal plain and on the Sea Islands.

SLE results from harmful antibodies made by a patients immune system. These can attack any part of the body, including kidneys, brain, heart, lungs, blood, skin and joints. B cells are precursors to plasma cells, the cells that produce the damaging antibodies. The SLE B cell signature is particularly accentuated in Black patients with severe disease, yet major gaps in knowledge to understand the different types of SLE clinical disease still persist.

The researchers say knowledge gained from these studies will greatly enhance understanding of SLE pathogenesis and differences in clinical manifestations, outcomes and therapeutic responses. Moreover, this knowledge will enable clinical investigators to more precisely design and evaluate clinical studies and standard-of-care treatments, with the ultimate goal of safer and more effective personalized treatments.

UAB investigators for the award, Linking different B cell types to lupus symptoms, progression and response to therapy, are Fran Lund, Ph.D., Alex Rosenberg, Ph.D., Winn Chatham, M.D., and Troy Randall, Ph.D., all researchers in the UAB Marnix E. Heersink School of Medicine.

Lund is the Charles H. McCauley Professor of Microbiology and founding director of the UAB Immunology Institute, a cutting-edge and interdisciplinary hub for faculty, researchers, clinicians, health policy experts and educators to advance the study of immunology and improve human health through immune-based therapies. Chatham, a professor of medicine and the Louis W. Heck Clinical Scholar in Rheumatology, is director of the UAB SLE Clinic. Randall, a professor of medicine, holds the Meyer Foundation William J. Koopman, M.D., Endowed Chair in Immunology and Rheumatology. Rosenberg is an associate professor of microbiology.

In addition to Sanz and the UAB investigators, the study includes five other researchers at Emory and two at MUSC.

This Lupus Research Alliance grant is one of two $3 million grants that form the first Global Team Science Awards, totaling $6 million, the largest-ever grant from the alliance.

The other award, Identifying differences in genetic makeup to stratify children with SLE into specific groups, went to researchers at Boston Childrens Hospital, Toronto Hospital for Sick Children, Brigham and Womens Hospital, and the Australian National University.

The Global Team Science Award embodies the Lupus Research Alliances strategic priorities of defining lupus heterogeneity, enabling patient stratification, and fostering collaborative global research and technology teams, said Teodora Staeva, Ph.D., chief scientific officer at the alliance. These first two projects, selected from among many compelling proposals, examine important underlying mechanisms of the autoimmune response and point the way to potential new treatment approaches.

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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UAB researchers are part of a $3 million award to learn how to better treat lupus in Black patients - EurekAlert

Lupus, an autoimmune disease that can attack any part of the body, can be confounding because patients often respond differently to the same treatment, and they vary widely in the severity of their symptoms.

Lupus, an autoimmune disease that can attack any part of the body, can be confounding because patients often respond differently to the same treatment, and they vary widely in the severity of their symptoms.Nine out of 10 patients with systemic lupus erythematosus the autoimmune disease also known as SLE are females. Black women have the highest concentration of disease severity, and they are two to three times more likely to suffer SLE than are white women. The disease usually strikes during childbearing years, ages 15 to 45.

To help Black women with SLE, theUniversity of Alabama at Birminghamand two other institutions have received a three-year, $3 million Global Team Science Award from the Lupus Research Alliance. The research will focus on better understanding this difficult disease, where patients often respond differently to the same treatment and vary widely in the severity of their symptoms.

Ignacio Sanz, M.D., chief of Rheumatology at Emory University School of Medicine, leads the multidisciplinary team. His Emory colleagues, together with research teams at UAB and the Medical University of South Carolina, or MUSC, will focus specifically on African American patients, identifying the characteristics of the B cells and plasma cells that create attacking antibodies in the development and progression of severe lupus. The project will define how the different characteristics of these cells correspond to patients responses to treatment.

A key to this research by UAB, Emory and MUSC is their access to a unique cohort of Black SLE patients from the American South, using large cohorts composed of rural and urban patients who receive care at the three institutions.

At UAB, this includes the SLE Clinic, which is enriched for severe patients from Alabamas rural Black Belt and from Birminghams urban communities. At Emory, this includes the Georgians Organized Against Lupus cohort, the largest community-based Black SLE cohort in the nation, with urban and rural Black Belt patients who have high poverty and disproportionately poor outcomes. At MUSC, 30 percent of the MUSC SLE Program patients are from the Gullah cohort, a group unique in their low white admixture, their genetic homogeneity and a double familial prevalence of lupus. The Gullah are Blacks who live in the Lowcountry of Atlantic states from Georgia to North Carolina, both on the coastal plain and on the Sea Islands.

SLE results from harmful antibodies made by a patients immune system. These can attack any part of the body, including kidneys, brain, heart, lungs, blood, skin and joints. B cells are precursors to plasma cells, the cells that produce the damaging antibodies. The SLE B cell signature is particularly accentuated in Black patients with severe disease, yet major gaps in knowledge to understand the different types of SLE clinical disease still persist.

The researchers say knowledge gained from these studies will greatly enhance understanding of SLE pathogenesis and differences in clinical manifestations, outcomes and therapeutic responses. Moreover, this knowledge will enable clinical investigators to more precisely design and evaluate clinical studies and standard-of-care treatments, with the ultimate goal of safer and more effective personalized treatments.

Fran Lund, Ph.D.UAB investigators for the award, Linking different B cell types to lupus symptoms, progression and response to therapy, are Fran Lund, Ph.D., Alex Rosenberg, Ph.D., Winn Chatham, M.D., and Troy Randall, Ph.D., all researchers in the UABMarnix E. Heersink School of Medicine.

Lund is the Charles H. McCauley Professor ofMicrobiologyand founding director of the UABImmunology Institute, a cutting-edge and interdisciplinary hub for faculty, researchers, clinicians, health policy experts and educators to advance the study of immunology and improve human health through immune-based therapies. Chatham, a professor ofmedicineand the Louis W. Heck Clinical Scholar inRheumatology, is director of the UAB SLE Clinic. Randall, a professor of medicine, holds the Meyer Foundation William J. Koopman, M.D., Endowed Chair in Immunology and Rheumatology. Rosenberg is an associate professor of microbiology.

In addition to Sanz and the UAB investigators, the study includes five other researchers at Emory and two at MUSC.

This Lupus Research Alliance grant is one of two $3 million grants that form the first Global Team Science Awards, totaling $6 million, the largest-ever grant from the alliance.

The other award, Identifying differences in genetic makeup to stratify children with SLE into specific groups, went to researchers at Boston Childrens Hospital, Toronto Hospital for Sick Children, Brigham and Womens Hospital, and the Australian National University.

The Global Team Science Award embodies the Lupus Research Alliances strategic priorities of defining lupus heterogeneity, enabling patient stratification, and fostering collaborative global research and technology teams, said Teodora Staeva, Ph.D., chief scientific officer at the alliance. These first two projects, selected from among many compelling proposals, examine important underlying mechanisms of the autoimmune response and point the way to potential new treatment approaches.

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UAB researchers are part of a $3 million award to learn how to better treat lupus in Black patients - News - The Mix

Every week there are numerous scientific studies published. Heres a look at some of the more interesting ones.

T-Cell Immune Response to COVID-19 Vaccines and Natural Infections

Much of the discussions and news reports about immune responses to vaccines and COVID-19 revolve around antibody levels. Much less has been said about T-cells, which provide longer-term protection. Researchers atGladstone Institutesconducteda detailed T-cell survey before and after COVID-19 immunization, which they published ineLife. They concluded that the Pfizer-BioNTech and Moderna mRNA vaccines create long-term populations of T-cells that recognize multiple SARS-COV-2 virus variants. They also found key differences in the T-cell responses in people who had COVID-19 infections before vaccination compared to people who had never been infected.

Overall, our data support the idea that vaccines are eliciting a very robust T-cell response in healthy individuals, said Nadia Roan, senior author of the study and Gladstone Associate Investigator. But they also suggest there may be some ways to improve them further, by getting more of the vaccine-elicited T-cells to park themselves in the respiratory tract.

Antibodies produced by B-cells quickly recognize viruses, target them, and prevent infection by destroying the viruses. T-cells, however, identify and destroy cells that are already infected. Antibodies are better at stopping initial infection, but T-cells typically last longer after an initial infection or vaccine. At that point they are better at fighting off disease in its early stages, which prevents severe symptoms. But T-cells are very diverse and difficult to analyze. Some subsets respond differently to infected cells and behave differently, while others have different functions within the overall T-cell immune response.

One key finding was that in people who had not been previously infected, the T-cell responses become stronger in quantity and quality after the second dose of the vaccine. But in people who had previously had COVID-19, there was not much of a change between the first and second vaccine dose.

Blood Biomarkers Provide Warning Signs of Dementia

Investigators at theGerman Center for Neurodegenerative Diseases (DZNE)identifiedmolecules in the blood that potentially warn of impending dementia. The research study included several university hospitals across Germany. The biomarkers were based on measuring levels of microRNAs. They say that the technique isnt ready yet for practical use, but they hope to develop a simple blood test. MicroRNAs have regulatory properties, influencing protein production and metabolism. In tests in humans, mice and cell cultures, they found three microRNAs whose levels were linked to mental performance. The three microRNAs also influence neuro-inflammation and neuroplasticity, including the ability of neurons to establish connections with each other.

Stem Cell Population Essential for Bone Regeneration

Researchers at theUniversity of Tsukuba, Japan,identifieda subpopulation of mesenchymal stem cells that play a major role in bone healing. The stem cells are found in the bone marrow and express the marker CD73. When a bone fracture heals, it moves through a series of stages, including clotted blood forming at the fracture. This clot is replaced by fibrous tissue and cartilage, then by a hard bony callus. The bone is then remodeled, with regular bone replacing the hard callus. They found that the generation of the callus is critically dependent on recruiting MSCs from the surrounding tissue and bone marrow. They observed the CD73-positive MSCs migrating toward the fracture site and forming new cartilage and bone cells. When they grafted CD73-positive MSCs into the fracture, they noted enhanced healing processes.

Antiviral Molecule Prevents SARS-CoV-2 from Entering Cells

Scientists atWashington University School of Medicinein St. Louisdevelopeda compound that prevents the SARS-CoV-2 virus, which causes COVID-19, from entering cells. The compound is called MM3122 and has been studied in cell cultures and in mice. MM3122 targets a key human protein called transmembrane serine protease 2 (TMPRSS2), which coronaviruses use to enter and infect human cells. Once the virus attached onto a cell in the epithelia of the airway, the TMPRSS2 protein cuts the viral spike protein, which activates the spike protein to mediate fusion of the viral and cellular membranesstarting the infection process. In cell cultures, MM3122 protected cells from viral damage better than remdesivir,Gilead Sciences antiviral against COVID-19; and an acute safety assay in mice demonstrated that large doses of MM3122 given for seven days did not cause noticeable issues. The compound also was effective against SARS-CoV, the virus behind SARS, and MERS-CoV, the coronavirus that causes MERS. The researchers are now working with researchers at the NIH to test it in animal models of COVID-19. They are also working on an oral version of the injectable compound.

Specific Personality Traits Might Signal Pending Alzheimers

Researchers atFlorida State Universityfoundthat specific changes in the brain linked with Alzheimers disease are often visible earlier in people with personality traits associated with the disease. The research focused on two traits: neuroticism, or a predisposition for negative emotions, and conscientiousness, linked to a tendency to be careful, organized, goal-directed and responsible. They found that people with amyloid and tau deposits, proteins linked to Alzheimers disease in the brain, were identified in participants who scored higher in neuroticism levels and lower in conscientiousness. The study suggests that personality traits might help protect against Alzheimers and other brain diseases by delaying or preventing the neuropathology for people strong in conscientiousness and low in neuroticism.

Why We Overeat

Astudyfrom theUniversity of Washington School of Medicine/UW Medicinereported on the function of glutamatergic neurons in mice. These neurons communicate to the lateral habenula, a brain region associated with the pathophysiology of depression, and the ventral tegmental area, which is involved in motivation, reward and addiction. They found that when mice are eating, the neurons in the lateral habenula are more responsive than the neurons in the ventral tegmental area. They suggest that these neurons might play a bigger role in guiding feeding. In addition, they studied the influence of the leptin and ghrelin hormones, which are believed to regulate behavior via the mesolimbic dopamine system, part of the reward pathway. The research adds additional insight into satiety and why people or at least mice overeat.

We found these cells are not a monolithic group, and that different flavors of these cells do different things, said Garret Stuber, a joint UW professor of anesthesiology and pain medicine and pharmacology, the papers senior author.

Read more from the original source:
Research Roundup: T-Cell Immune Response to COVID-19 Vaccines and More - BioSpace

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Alicia Morgans: Hi, my name is Alicia Morgans, and I'm a GU Medical Oncologist at Dana-Farber Cancer Institute in Boston. I'm so excited to have here with me today, a good friend and colleague, Dr. Cora Sternberg, who is a Professor of Medicine and the Clinical Director of the Englander Institute for Precision Medicine at Weill Cornell Medical College, and Cornell University in New York City. Thank you so much for being here with me today, Dr. Sternberg.

Cora Sternberg: It's a pleasure to be here with you, Alicia.

Alicia Morgans: It is always my pleasure and it's always exciting to have you, especially to talk about some of the big trials you've been involved in. Today, we are going to talk about some work that you and your team have done looking at CTCs within the CARD study, which is, of course, information that you and the team presented at ESMO 2021. Before we dig into what you presented, can you remind all of us, briefly, what was the CARD study?

Cora Sternberg: Basically, the CARD study was looking at the sequencing of therapy in metastatic CRPC, and patients had already received either abiraterone or enzalutamide, and they were patients who actually did not respond for more than 12 months and they had also received docetaxel. They were randomized between cabazitaxel and then the other novel androgen receptor therapy. What was seen in that study was the patients who received cabazitaxel did much better than the patients who received enzalutamide or abiraterone. There was a difference in radiological progression-free survival and in overall survival. And this has been presented many, many times.

What was presented now at ESMO was looking at the circulating tumor cells in blood samples that were collected first at screening, and then during therapy, in a subset of the patients. What we saw was that we sent these CTCs to Epic Sciences for analysis. We looked at chromosomal instability, which was defined as having at least three CTCs with chromosome instability, and we also looked at patients having abnormalities that were associated with and looked more like neuroendocrine prostate cancer.

What we found in this study was that if patients had chromosomal instability, and if they either got worse or it didn't change, those patients on cabazitaxel did not benefit from cabazitaxel. The patients who were on hormonal therapy, no matter what they had, just did poorly anyway, whether or not they had chromosome instability or not. But it was very clear that the patients on cabazitaxel who had that chromosome instability did not do very well. Likewise, patients who had a neuroendocrine phenotype also did not benefit from cabazitaxel at all, not really very much. Patients really benefited who had CTCs that were without the chromosome instability and without a neuroendocrine phenotype. So this was an interesting study, it's retrospective, looking at the CTCs, and perhaps in the future, we could use this at screening to decide who should receive cabazitaxel and who should not.

Alicia Morgans: That is really fascinating, and I have to say, I'm a little surprised that the patients with chromosomal instability wouldn't necessarily be affected by chemotherapy. But it is what it is. And it's interesting because I think of these neuroendocrine, in particular, as being on a spectrum towards small-cell. There is a phase two study that is going on right now called CASCARA that is looking at the combination of cabazitaxel and carboplatin to see if that combination, then followed by abiraterone, may be a good way to capture more patients who have progression on chemotherapy. It seems like this might suggest that is potentially where we could go, although of course, neuroendocrine is not small-cell cancer, we know small-cell, again, on the spectrum can be affected by platinum.

Cora Sternberg: I'm not really sure what this means, because this is the neuroendocrine phenotype in the CTCs. And as you know from the work by Ana Aparicio at MD Anderson, cabazitaxel and carboplatin are a good combination, it's a combination that we use in patients with neuroendocrine prostate cancer. So I'm not really sure what to make of this. I guess we're just selecting out a poor part of the patient group. These were patients with adenocarcinoma who had cells that showed this neuroendocrine phenotype, and the cabazitaxel alone in those patients had lower response rates and just didn't do as well as those who didn't have it. So I think we're probably just selecting out a poor phenotype. Not to say that other trials will not work.

Alicia Morgans: Absolutely. I really appreciate that and I just think it's interesting that we learn more and more from the analyses we do in the trials that we run, and I really love that you are doing this biomarker work to help us pull all that we can from this data and really inform patient decisions. If you had to sum it up and give a message to listeners, what would that be?

Cora Sternberg: Well, I think the phenotypic diversity that you can find in circulating tumor cells just shows that there are many different mechanisms of resistance to therapies, to chemotherapy, and even to endocrine therapy, and I think that we should look more into that and look at those mechanisms of resistance, and perhaps CTCs is one way of doing that.

Alicia Morgans: I could not agree more. I appreciate your continued devotion to enlightening the field, and of course, the work that you do, and all the patients that you help. We appreciate, of course, your expertise as well tonight, so thank you so much for your time.

Cora Sternberg: Thank you, Alicia.

Read more:
Therapy Resistance Identified in Circulating Tumor Cell Morphologic Sub-Types Present Prior to Treatment in the CARD Trial - Cora Sternberg - UroToday

Malignant Mesothelioma is a rare growth of mesothelial cells strongly associated with asbestos exposure. Major pipeline molecules include durvalumab, pembrolizumab, tremelimumab, nintedanib, ADI-PEG 20, and MesoPher. Additionally, the development of novel therapies is going to introduce advanced treatment options.

Las Vegas, USA, Oct. 13, 2021 (GLOBE NEWSWIRE) -- Propulsion of Malignant Mesothelioma Pipeline as Novel and Extensive Therapies Enter the Treatment Domain, Anticipates DelveInsight

Malignant Mesothelioma is a rare growth of mesothelial cells strongly associated with asbestos exposure. Major pipeline molecules include durvalumab, pembrolizumab, tremelimumab, nintedanib, ADI-PEG 20, and MesoPher. Additionally, the development of novel therapies is going to introduce advanced treatment options.

DelveInsights Malignant Mesothelioma Pipeline Insight 2021 report offers exhaustive global coverage of available, marketed, and pipeline therapies in different phases of clinical development, key companies working to advance the pipeline space, and future growth potential of the Malignant Mesothelioma pipeline domain.

Some of the crucial points taken from the Malignant Mesothelioma Pipeline report:

DelveInsights Malignant Mesothelioma Pipeline analysis depicts the space with 25+ active players working to develop 25+ pipeline therapies.

Major pharmaceutical companies that are developing potential drug candidates to improve the Malignant Mesothelioma treatment scenario include Momotaro-Gene, Polaris Group, Amphera, Vivace Therapeutics, MedImmune, TCR2 Therapeutics, Atara Biotherapeutics, Inhibrx, Eisai, Targovax, Merck & Co, Bayer HealthCare, SELLAS Life Sciences Group, CanBas, FKD Therapies, Virttu Biologics, MolMed, Ys Therapeutics, LIPAC Oncology, TCR2 Therapeutics, Hutchison Medipharma Limited, Clovis Oncology, Sanofi, Merck & Co., PharmaMar, Targovax and many others.

Key Malignant Mesothelioma pipeline therapies such as rAd-IFN, Pembrolizumab, ADI-PEG 20, MesoPher, MTG201, HMPL-453, SAR444245, Rucaparib, TC 210, iCasp9M28z T-cell, INBRX-109, VT3989, LEITP-1009, YS110, Gavo-cel, iCasp9M28z T-cell, Lurbinectedin, ONCOS-102 and others are under investigation in different phases of clinical trials.

Targovax initiated a randomized, phase I/II clinical trial combining ONCOS-102 with Pem-Cis in 31 patients, indicating that ONCOS-102 can activate the immune system and increase tumour infiltrating T-cells into Malignant Pleural Mesothelioma.

Sellas Life Sciences Group is evaluating Galinpepimut-S (GPS) for the treatment of Malignant Mesothelioma. It is an immunotherapy that targets the Wilms Tumor 1 (WT1) protein which is present and over-expressed in an array of hematologic malignancies and solid tumors.

LIPAC is developing a new Liposomal Enhanced IntraThoracic Paclitaxel (LEITP) for the treatment of Malignant Pleural Mesothelioma utilizing the LiPax technology. Preclinical studies with LEITP-1009 are underway.

In July 2021, PharmaMar announced that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has given its positive opinion for Orphan Drug Designation to Zepzelca (lurbinectedin) for the treatment of Malignant Mesothelioma.

UV1 is a peptide-based vaccine inducing a specific T cell response against the universal cancer antigen telomerase.

Momotaro-Gene is evaluating MTG201, a novel investigational gene therapy with unique dual mechanisms of action capable of addressing a range of cancers. A Phase II clinical trial is being conducted at the Baylor College of Medicine in Houston, Texas.

Track which potential therapy is anticipated to take charge of Malignant Mesothelioma pipeline in the forthcoming years by requesting a sample @ Malignant Mesothelioma Emerging Therapies and Forecast

Story continues

The Malignant Mesothelioma pipeline report lays down detailed profiles of the pipeline assets, inactive and dormant assets, comparative analysis of clinical and non-clinical stage Malignant Mesothelioma products, comprehensive assessment of driving and restraining factors, along with the opportunities and risks in the Malignant Mesothelioma pipeline landscape.

Malignant Mesothelioma Overview

Malignant Mesothelioma is a rare, incurable, asbestos-related cancer. It mainly affects the lining of the lung and chest cavity (pleura) or lining of the abdomen (peritoneum). In rare cases, mesothelioma tumors can grow in the linings of the heart (pericardium) or testes (tunica vaginalis).

There are 4 types of mesothelioma, each affecting a different area in the body. The four types are pleural mesothelioma, peritoneal mesothelioma, pericardial mesothelioma, and testicular mesothelioma.

Explore more about Malignant Mesothelioma pipeline therapeutics assessment @ Malignant Mesothelioma Pipeline Assessment

Malignant Mesothelioma Pipeline Drugs

Drug

Company

Phase

MoA

RoA

rAd-IFN

Trizell Ltd

III

IFNA2B expression stimulants

NA

Pembrolizumab

Merck & Co

III

Programmed cell death-1 receptor antagonists

Intravenous

ADI-PEG 20

Polaris Pharmaceuticals

II/III

Arginine deiminase replacements

Intramuscular

MesoPher

Amphera

II/III

Immunostimulant

Intradermal

MTG201

Momotaro-Gene

II

DKK3 expression modulators

Intratumoural

HMPL-453

Hutchison Medipharma Limited

II

Type 1, 2, 3 1 fibroblast growth factor receptor antagonists

Oral

SAR444245

Sanofi

II

Interleukin-2 replacements

Intravenous

Rucaparib

Clovis Oncology

II

Poly(ADP-ribose) polymerase 1, 2, 3 inhibitors

Oral

TC 210

TCR2 Therapeutics

I/II

Immunologic cytotoxicity; T lymphocyte replacements

NA

iCasp9M28z T-cell

Bellicum Pharmaceuticals

I/II

Immunologic cytotoxicity; Programmed cell death-1 receptor antagonists; T lymphocyte replacements

NA

INBRX-109

Inhibrx

I

TRAIL receptor 2 agonists

Parenteral

VT3989

Vivace Therapeutics

I

Transcription factor inhibitors

Oral

LEITP-1009

LIPAC Oncology

Preclinical

Proto-oncogene protein c-bcl-2 inhibitors

NA

Request for Sample to know more @ Malignant Mesothelioma Pipeline Analysis and Key Companies

Malignant Mesothelioma Therapeutics Assessment

The Malignant Mesothelioma Pipeline report proffers an integral view of the Malignant Mesothelioma emerging novel therapies segmented by Stage, Product Type, Route of Administration, Molecule Type, and Mechanism of Action.

By Product Type

Mono

Combination

Mono/Combination

By Stage

Follow this link:
Propulsion of Malignant Mesothelioma Pipeline as Novel and Extensive Therapies Enter the Treatment Domain, Anticipates DelveInsight - Yahoo Finance

The Brain & Behavior Research Foundation named Justin Wolter, PhD, postdoc in the Neuroscience Research Center, as a recipient of the Young Investigator Award.

Justin Wolter, PhD, a postdoctoral researcher in the labs of Jason Stein, PhD, and Mark Zylka, PhD, at the UNC Neuroscience Research Center, the UNC Department of Genetics, and the UNC Department of Cell Biology and Physiology, was named a recipient of the 2021 Young Instigator Award by the Brain & Behavior Research Foundation (BBRF). The award is for $70,000 over two years.

In his research at the UNC School of Medicine, Wolter aims to understand the molecular and cellular mechanisms of neurodevelopmental diseases. With the BBRF award, he will establish a resource to systematically identify genetic interactions between high-risk autism genes and common genetic variation. This project will build upon work in which Wolter established a cell culture-based approach to conduct genome wide association studies in primary human neural progenitor cells.

Wolter will establish a pilot library of genetically diverse induced pluripotent stem cell (iPSC) lines to explore how common and rare genetic variation interact to influence risk and resilience in a genetically defined subtype of autism.

In 2020, Wolter was first author of a Nature paper from the Zylka lab showing how to use the gene-editing technology CRISPR-Cas9 as part of a potential gene therapy approach to treating Angelman syndrome, an autism spectrum disorder.

Initiated in 1987, the BBRF Young Investigator Grant program provides support for the most promising young scientists conducting neurobiological and psychiatric research. This program facilitates innovative research through support of early-career basic, translational and clinical investigators.

This year, the Foundations Scientific Council, led by Herbert Pardes, MD, and comprised of 176 world-renowned scientists with expertise in every area of brain research, reviewed more than 780 applications and selected the 150 meritorious research projects. Many of the Young Investigator grantees are pursuing basic research projects. Others are specifically focusing on new ideas for therapies, diagnostic tools, and technologies. These research projects will provide future insights and advances that will help move the fields of psychiatry and neuroscience forward.

Read more here:
Wolter Earns Young Investigator Award | Newsroom - UNC Health and UNC School of Medicine

Credit: The University of British Columbia

Peter Zandstra was attracted to British Columbia from Ontario by a unique opportunity at the University of British Columbiathe opportunity to achieve the vision of a world where biology and technologywork seamlessly to transform health and healthcare for everyone.

When Zandstra joined UBC in 2017, he founded BCs first and only School of Biomedical Engineering (SBME@UBC) and set to work building a world-leading hub for cutting-edge biomedical engineering education, innovation and research to help bring new treatments to patients faster.

The University of British ColumbiaBritish Columbias burgeoning biotech sector has leaped onto the world stage, says Zandstra, director of SBME@UBC. Its an ecosystem where people, technologies, facilities and stakeholders from hospitals, universities and industry are coming together like never before. SBME@UBC is becoming the community home for this ecosystem and the place where the next generation of talent and technologies will emerge.

Among the top stem cell researchers in Canada, Zandstra and his team pioneered the field of stem cell bioengineering, the scientific foundation to many emerging cellular therapies. By applyingengineering principles to stem cell biology, Zandstras team develops cell-based therapies for leukemia and other blood diseases.

In less than five years at UBC, Zandstra has successfully brought together more than 80 faculty to form a talented and diverse community of teaching, research and clinical expertise to develop new ways to manage and treat long-standing medical challenges such as Alzheimers disease, cancer, diabetes, heart disease and spinal cord injury. These bioengineers work across biological scalesfrom molecular and cellular engineering to human systems physiologyto build interdisciplinary, tailor-made solutions to medical problems that previously could not be addressed using conventional approaches.

Zandstras next priority: to build a state-of-theart, purpose-designed home for SBME@UBC.

Innovation only happens when we collaborate, says Zandstra. It requires bringing together the full breadth of our knowledge, technology and talent under one roof. Imagine a living laba fully creative environment without technological, scientific or academic boundary.

With construction scheduled to start next year and the grand opening only two years away, philanthropic support is urgently needed to make this building a reality.

Community leaders like you have the opportunity to name spaces ranging from the building itself to the atrium, lecture theatres, teaching labs, design studios and core research facilities in the new building.

Your donation to UBC will directly support the construction of the new building for SBME@UBCa launchpad for medical innovation that will transform healthcare and the economy of the future.

This is your chance to put your name on the future of medical innovation in British Columbia.

PLEASE MAKE A GIFT TODAY.To help change the future of medicine, please contact Violetta Yan, associate director of development, at 604-822-8079 or violetta.yan@ubc.ca.

Connect:

Twitter | @sbme_ubcInstagram | @ubcsbmeWebsite | bme.ubc.ca

Created by BCBusiness in partnership with The University of British Columbia

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Peter Zandstra Wants to Change the Future of Medicine - BCBusiness

NEW YORK - BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, will present findings from a multicenter, open label clinical trial of NurOwn in progressive multiple sclerosis. The study, 'Phase 2 Safety and Efficacy Study of Intrathecal MSC-NTF cells in Progressive Multiple Sclerosis,' will be delivered in an oral presentation today at the fully digital 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS).

The Phase 2 clinical trial was designed to evaluate intrathecal administration of NurOwn (autologous MSC-NTF cells) in participants with progressive MS. The study achieved the primary endpoint of safety and tolerability. It demonstrated a reduction of neuroinflammatory biomarkers and an increase in neuroprotective biomarkers in the cerebrospinal fluid (CSF) and consistent improvement across MS functional outcome measures, including measures of walking, upper extremity function, vision and cognition.

'We were pleased that this study demonstrated safety, preliminary evidence of efficacy and relevant biomarker outcomes in patients with progressive multiple sclerosis, in an area of high unmet need,' said Jeffrey Cohen, M.D., Director of Experimental Therapeutics at the Cleveland Clinic Mellen Center for MS and principal investigator for the trial. 'These results should be confirmed in a randomized placebo-controlled trial.'

The study was sponsored by Brainstorm Cell Therapeutics with additional financial support for biomarker analyses from the National Multiple Sclerosis Society Fast-Forward Program. It was conducted at four U.S. MS centers of excellence: Cleveland Clinic Mellen Center for MS, Cleveland; Icahn School of Medicine at Mount Sinai, New York; Keck School of Medicine of the University of Southern California, Los Angeles; and Stanford University School of Medicine, Palo Alto, Calif.

'We very much appreciate the tremendous collaboration among many premier organizations, for their generous sharing of expertise, support and data, which enabled the important balance between scientific rigor and ethical treatment of progressive MS participants in the trial,' said Ralph Kern, M.D., MHSc., President and Chief Medical Officer, Brainstorm Cell Therapeutics. 'We are holding discussions with key MS experts, and seeking guidance from the FDA to determine next steps for the development of NurOwn in progressive MS.'

'The National MS Society is pleased to support the biomarker portion of this study through our commercial funding program Fast Forward,' said Mark Allegretta, Ph.D., Vice President, Research. 'We're encouraged to see evidence that the biomarker analysis showed proof of concept for detecting neuroprotection and reduced inflammation.'

About the trial

The Phase 2 open-label study evaluated the safety and efficacy of intrathecal administration of autologous MSC-NTF cells in patients with primary or secondary progressive MS. The primary study endpoint was safety and tolerability. Secondary efficacy endpoints included: timed 25-foot walk (T25FW); 9-Hole Peg Test (9-HPT); Low Contrast Letter Acuity (LCLA); Symbol Digit Modalities Test (SDMT); 12 item MS Walking Scale (MSWS-12); as well as cerebrospinal fluid (CSF) and blood biomarkers. Clinical efficacy outcomes were compared with matched (n=48) participants in the Comprehensive Longitudinal Investigation of Multiple Sclerosis (CLIMB) registry, Tanuja Chitnis, MD Brigham and Women's Hospital and the Ann Romney Center for Neurologic Diseases, and 255 patient randomized double blind placebo controlled NN-102 SPRINT-MS Study, courtesy NIH/NINDS, PI: Robert J. Fox, MD, MS, FAAN, Cleveland Clinic, CTR: NCT01982942. Baseline characteristics from these two cohorts were similar allowing for comparison of efficacy results, comparisons with SPRINT-MS were with the placebo arm of this study.

Mean age of participants was 47 years, 56% were female, and mean baseline EDSS score was 5.4. 18 participants were treated, 16 (80%) received all 3 treatments and completed the entire study; 2 study discontinuations were due to procedure-related adverse events. No deaths or treatment-related adverse events due to worsening of MS were observed.

In responder analyses, 14% and 13% of MSC-NTF treated participants showed at least a 25% improvement in T25FW and 9-HPT (combined hands) respectively, compared to 5% and 0% in matched CLIMB patients and 9% and 3% in SPRINT. Twenty-seven percent (27%) showed at least an 8-letter improvement in LCLA (binocular, 2.5% threshold) and 67% showed at least a 3-point improvement in SDMT, compared to 6% and 18% in CLIMB and 13% and 35% in SPRINT, respectively.

Mean improvements of +0.10 ft/sec in T25FW and -0.23 sec in 9-HPT (combined hands), were observed in MSC-NTF treated participants, compared to a mean worsening of -0.07 ft/sec and +0.49 sec in CLIMB and -0.06 ft/sec and +0.28 sec in SPRINT, respectively. MSC-NTF treated participants showed a mean improvement of +3.3 letters in LCLA (binocular, 2.5% threshold) and 3.8 points in SDMT, compared to a mean worsening of -1.07 letters in LCLA (binocular, 2.5% threshold) and mean improvement of +0.10 in SDMT, in CLIMB and -0.6 and -0.1 in SPRINT. In addition the MSFC-4 Composite Z-score of T25W, 9-HPT, SDMT and LCLA showed a 0.18 point improvement in MSC-NTF treated participants, while CLIMB and SPRINT showed decreases of -0.02 and -0.05.

Furthermore, 38% of treated patients showed at least a 10-point improvement in the MSWS-12 a patient reported outcome that evaluates the impact of MS on walking function, whereas this outcome was not evaluated in CLIMB or SPRINT.

CSF biomarkers obtained at 3 consecutive time points, showed increases in neuroprotective molecules (VEGF, HGF, NCAM-1, Follistatin, Fetuin-A) and decreases in neuroinflammatory biomarkers (MCP-1, SDF-1, sCD27 and Osteopontin).

About NurOwn

The NurOwn technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS).

For more information, visit the company's website at http://www.brainstorm-cell.com.

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future NurOwn manufacturing and clinical development plans, constitute 'forward-looking statements' and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as 'may,' 'should,' 'would,' 'could,' 'will,' 'expect,' 'likely,' 'believe,' 'plan,' 'estimate,' 'predict,' 'potential,' and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, the prospects for regulatory approval of BrainStorm's NurOwn treatment candidate, the initiation, completion, and success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture, or to use third parties to manufacture, and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

Contact:

Investor

Eric Goldstein

LifeSci Advisors, LLC

Phone: +1 (646) 791-9729

E: egoldstein@lifesciadvisors.com

Original post:
BrainStorm Cell Therapeutics : Phase 2 Clinical Trial Data of NurOwn in Progressive MS Will Be Presented at the 37th Congress of the European...