Pivotal ZUMA-5 Study Demonstrates Overall Response Rate of 91% and a Complete Response rate of 77% in Patients Who Received Yescarta After Three or More Lines of Therapy

Kites Third Approved Cell Therapy Indication in Europe

SANTA MONICA, Calif.--(BUSINESS WIRE)--Kite, a Gilead Company (Nasdaq: GILD), today announces that the European Commission (EC) has approved its CAR T-cell therapy Yescarta (axicabtagene ciloleucel) for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after three or more lines of systemic therapy. Yescarta has maintained orphan medicinal product designation in this indication.

Patients with advanced relapsed or refractory follicular lymphoma have a high need for new treatment options, said Christi Shaw, CEO, Kite. This is the third approved indication for a Kite cell therapy in Europe, and we are pleased to enable more patients with different lymphomas greater access to this treatment innovation.

Follicular lymphoma is a form of non-Hodgkin lymphoma in which tumors grow slowly but can become more aggressive over time. FL is the second most common type of lymphoma globally and accounts for approximately 22% of all lymphomas diagnosed worldwide.In Europe, approximately 27,000 new cases are diagnosed each year.

Follicular lymphoma that has relapsed multiple times is a difficult-to-treat disease with an especially poor prognosis as only 20% of patients are still alive at five years after their second relapse, said Ibrahim Yakoub-Agha, MD, PhD, Head of the Hematopoietic Cell Transplantation and Cellular Therapy Unit, Lille University Hospital. Ninety-one percent of patients in the ZUMA-5 study responded to axicabtagene ciloleucel after three or more prior lines of therapy, and more than half of these were still in response two years later. This sign of durable remission is critical for patients who need options that can deliver long-term benefit.

Follicular lymphoma is often misunderstood as easy to treat or non life-threatening, even when it has reached a significantly advanced stage, said Nicola Mendelsohn, Founder and Chair of the Follicular Lymphoma Foundation (FLF). For patients with later-line relapsed or refractory disease, it is often very aggressive. Axicabtagene ciloleucel represents an important advance for a patient population in Europe with limited treatment options.

The approval is supported by data from the pivotal, single-arm Phase 2 ZUMA-5 international study in patients with relapsed or refractory FL who had received at least two prior lines of systemic therapy, including the combination of an anti-CD20 monoclonal antibody and an alkylating agent. Among patients who had received three or more lines of prior therapy (n=75), the overall response rate (ORR) was 91%, and the complete response (CR) rate was 77% at the 24-month analysis. The median duration of response (DoR) was 38.6 months, and the proportion of responders still in response at Month 24 was 56%.

Among all evaluable patients within ZUMA-5 (n=119), safety observations were consistent with the known safety profile for Yescarta. Grade 3 cytokine release syndrome (CRS) occurred in 6% of patients and neurologic events occurred 16% of patients. Most CRS cases (99%) of any grade resolved by the time of data cut-off and 60% of neurologic events were resolved within three weeks. The most significant and frequently occurring adverse events were CRS (77%), infections (59%) and encephalopathy (47%). For full details on the Special Warnings and Precautions for Use and Adverse Reactions (including appropriate management), please refer to the EU Summary of Product Characteristics (SmPC).

Additional data were shared separately during an oral presentation at the 2021 American Society of Hematology Meeting.

About Follicular Lymphoma

FL is a form of indolent non-Hodgkin lymphoma (iNHL) in which malignant tumors slowly grow but can become more aggressive over time, especially if they relapse. FL is the most common form of indolent non-Hodgkin lymphoma and the second most common type of lymphoma globally. It accounts for approximately 22% of all lymphomas diagnosed worldwide. Currently, there are limited options for the treatment of relapsed or refractory FL after two or more lines of therapy.

About ZUMA-5

ZUMA-5 is an ongoing, single-arm, open-label, international, multicentre trial evaluating 122 patients (18 years old) with relapsed or refractory follicular lymphoma (FL), who received at least two prior lines of systemic therapy, including the combination of an anti-CD20 monoclonal antibody and an alkylating agent. The primary endpoint was ORR, and secondary endpoints included CR rate, ORR and CR in patients who had received three or more lines of prior therapy, DoR, overall survival, progression-free survival and incidence of adverse events.

About Yescarta

Please see full US Prescribing Information, including BOXED WARNING and Medication Guide.

YESCARTA is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:

U.S. IMPORTANT SAFETY INFORMATION

BOXED WARNING: CYTOKINE RELEASE SYNDROME AND NEUROLOGIC TOXICITIES

CYTOKINE RELEASE SYNDROME (CRS)

CRS, including fatal or life-threatening reactions, occurred. CRS occurred in 90% (379/422) of patients with non-Hodgkin lymphoma (NHL), including Grade 3 in 9%. CRS occurred in 93% (256/276) of patients with large B-cell lymphoma (LBCL), including Grade 3 in 9%. Among patients with LBCL who died after receiving YESCARTA, 4 had ongoing CRS events at the time of death. For patients with LBCL in ZUMA-1, the median time to onset of CRS was 2 days following infusion (range: 1-12 days) and the median duration was 7 days (range: 2-58 days). For patients with LBCL in ZUMA-7, the median time to onset of CRS was 3 days following infusion (range: 1-10 days) and the median duration was 7 days (range: 2-43 days). CRS occurred in 84% (123/146) of patients with indolent non-Hodgkin lymphoma (iNHL) in ZUMA-5, including Grade 3 in 8%. Among patients with iNHL who died after receiving YESCARTA, 1 patient had an ongoing CRS event at the time of death. The median time to onset of CRS was 4 days (range: 1-20 days) and the median duration was 6 days (range: 1-27 days) for patients with iNHL.

Key manifestations of CRS ( 10%) in all patients combined included fever (85%), hypotension (40%), tachycardia (32%), chills (22%), hypoxia (20%), headache (15%), and fatigue (12%). Serious events that may be associated with CRS include cardiac arrhythmias (including atrial fibrillation and ventricular tachycardia), renal insufficiency, cardiac failure, respiratory failure, cardiac arrest, capillary leak syndrome, multi-organ failure, and hemophagocytic lymphohistiocytosis/macrophage activation syndrome.

The impact of tocilizumab and/or corticosteroids on the incidence and severity of CRS was assessed in 2 subsequent cohorts of LBCL patients in ZUMA-1. Among patients who received tocilizumab and/or corticosteroids for ongoing Grade 1 events, CRS occurred in 93% (38/41), including 2% (1/41) with Grade 3 CRS; no patients experienced a Grade 4 or 5 event. The median time to onset of CRS was 2 days (range: 1-8 days) and the median duration of CRS was 7 days (range: 2-16 days). Prophylactic treatment with corticosteroids was administered to a cohort of 39 patients for 3 days beginning on the day of infusion of YESCARTA. Thirty-one of the 39 patients (79%) developed CRS and were managed with tocilizumab and/or therapeutic doses of corticosteroids with no patients developing Grade 3 CRS. The median time to onset of CRS was 5 days (range: 1-15 days) and the median duration of CRS was 4 days (range: 1-10 days). Although there is no known mechanistic explanation, consider the risk and benefits of prophylactic corticosteroids in the context of pre-existing comorbidities for the individual patient and the potential for the risk of Grade 4 and prolonged neurologic toxicities.

Ensure that 2 doses of tocilizumab are available prior to YESCARTA infusion. Monitor patients for signs and symptoms of CRS at least daily for 7 days at the certified healthcare facility, and for 4 weeks thereafter. Counsel patients to seek immediate medical attention should signs or symptoms of CRS occur at any time. At the first sign of CRS, institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids as indicated.

NEUROLOGIC TOXICITIES

Neurologic toxicities (including immune effector cell-associated neurotoxicity syndrome) that were fatal or life-threatening occurred. Neurologic toxicities occurred in 78% (330/422) of all patients with NHL receiving YESCARTA, including Grade 3 in 25%. Neurologic toxicities occurred in 87% (94/108) of patients with LBCL in ZUMA-1, including Grade 3 in 31% and in 74% (124/168) of patients in ZUMA-7 including Grade 3 in 25%. The median time to onset was 4 days (range: 1-43 days) and the median duration was 17 days for patients with LBCL in ZUMA-1. The median time to onset for neurologic toxicity was 5 days (range:1- 133 days) and the median duration was 15 days in patients with LBCL in ZUMA-7. Neurologic toxicities occurred in 77% (112/146) of patients with iNHL, including Grade 3 in 21%. The median time to onset was 6 days (range: 1-79 days) and the median duration was 16 days. Ninety-eight percent of all neurologic toxicities in patients with LBCL and 99% of all neurologic toxicities in patients with iNHL occurred within the first 8 weeks of YESCARTA infusion. Neurologic toxicities occurred within the first 7 days of infusion for 87% of affected patients with LBCL and 74% of affected patients with iNHL.

The most common neurologic toxicities ( 10%) in all patients combined included encephalopathy (50%), headache (43%), tremor (29%), dizziness (21%), aphasia (17%), delirium (15%), and insomnia (10%). Prolonged encephalopathy lasting up to 173 days was noted. Serious events, including aphasia, leukoencephalopathy, dysarthria, lethargy, and seizures occurred. Fatal and serious cases of cerebral edema and encephalopathy, including late-onset encephalopathy, have occurred.

The impact of tocilizumab and/or corticosteroids on the incidence and severity of neurologic toxicities was assessed in 2 subsequent cohorts of LBCL patients in ZUMA-1. Among patients who received corticosteroids at the onset of Grade 1 toxicities, neurologic toxicities occurred in 78% (32/41), and 20% (8/41) had Grade 3 neurologic toxicities; no patients experienced a Grade 4 or 5 event. The median time to onset of neurologic toxicities was 6 days (range: 1-93 days) with a median duration of 8 days (range: 1-144 days). Prophylactic treatment with corticosteroids was administered to a cohort of 39 patients for 3 days beginning on the day of infusion of YESCARTA. Of those patients, 85% (33/39) developed neurologic toxicities, 8% (3/39) developed Grade 3, and 5% (2/39) developed Grade 4 neurologic toxicities. The median time to onset of neurologic toxicities was 6 days (range: 1-274 days) with a median duration of 12 days (range: 1-107 days). Prophylactic corticosteroids for management of CRS and neurologic toxicities may result in a higher grade of neurologic toxicities or prolongation of neurologic toxicities, delay the onset of and decrease the duration of CRS.

Monitor patients for signs and symptoms of neurologic toxicities at least daily for 7 days at the certified healthcare facility, and for 4 weeks thereafter, and treat promptly.

REMS

Because of the risk of CRS and neurologic toxicities, YESCARTA is available only through a restricted program called the YESCARTA and TECARTUS REMS Program which requires that: Healthcare facilities that dispense and administer YESCARTA must be enrolled and comply with the REMS requirements and must have on-site, immediate access to a minimum of 2 doses of tocilizumab for each patient for infusion within 2 hours after YESCARTA infusion, if needed for treatment of CRS. Certified healthcare facilities must ensure that healthcare providers who prescribe, dispense, or administer YESCARTA are trained in the management of CRS and neurologic toxicities. Further information is available at http://www.YescartaTecartusREMS.com or 1-844-454-KITE (5483).

HYPERSENSITIVITY REACTIONS

Allergic reactions, including serious hypersensitivity reactions or anaphylaxis, may occur with the infusion of YESCARTA.

SERIOUS INFECTIONS

Severe or life-threatening infections occurred. Infections (all grades) occurred in 45% of patients with NHL; Grade 3 infections occurred in 17% of patients, including Grade 3 infections with an unspecified pathogen in 12%, bacterial infections in 5%, viral infections in 3%, and fungal infections in 1%. YESCARTA should not be administered to patients with clinically significant active systemic infections. Monitor patients for signs and symptoms of infection before and after infusion and treat appropriately. Administer prophylactic antimicrobials according to local guidelines.

Febrile neutropenia was observed in 36% of all patients with NHL and may be concurrent with CRS. In the event of febrile neutropenia, evaluate for infection and manage with broad-spectrum antibiotics, fluids, and other supportive care as medically indicated.

In immunosuppressed patients, including those who have received YESCARTA, life-threatening and fatal opportunistic infections including disseminated fungal infections (e.g., candida sepsis and aspergillus infections) and viral reactivation (e.g., human herpes virus-6 [HHV-6] encephalitis and JC virus progressive multifocal leukoencephalopathy [PML]) have been reported. The possibility of HHV-6 encephalitis and PML should be considered in immunosuppressed patients with neurologic events and appropriate diagnostic evaluations should be performed.

Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, can occur in patients treated with drugs directed against B cells, including YESCARTA. Perform screening for HBV, HCV, and HIV in accordance with clinical guidelines before collection of cells for manufacturing.

PROLONGED CYTOPENIAS

Patients may exhibit cytopenias for several weeks following lymphodepleting chemotherapy and YESCARTA infusion. Grade 3 cytopenias not resolved by Day 30 following YESCARTA infusion occurred in 39% of all patients with NHL and included neutropenia (33%), thrombocytopenia (13%), and anemia (8%). Monitor blood counts after infusion.

HYPOGAMMAGLOBULINEMIA

B-cell aplasia and hypogammaglobulinemia can occur. Hypogammaglobulinemia was reported as an adverse reaction in 14% of all patients with NHL. Monitor immunoglobulin levels after treatment and manage using infection precautions, antibiotic prophylaxis, and immunoglobulin replacement. The safety of immunization with live viral vaccines during or following YESCARTA treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least 6 weeks prior to the start of lymphodepleting chemotherapy, during YESCARTA treatment, and until immune recovery following treatment.

SECONDARY MALIGNANCIES

Secondary malignancies may develop. Monitor life-long for secondary malignancies. In the event that one occurs, contact Kite at 1-844-454-KITE (5483) to obtain instructions on patient samples to collect for testing.

EFFECTS ON ABILITY TO DRIVE AND USE MACHINES

Due to the potential for neurologic events, including altered mental status or seizures, patients are at risk for altered or decreased consciousness or coordination in the 8 weeks following YESCARTA infusion. Advise patients to refrain from driving and engaging in hazardous occupations or activities, such as operating heavy or potentially dangerous machinery, during this initial period.

ADVERSE REACTIONS

The most common non-laboratory adverse reactions (incidence 20%) in patients with LBCL in ZUMA-7 included fever, CRS, fatigue, hypotension, encephalopathy, tachycardia, diarrhea, headache, musculoskeletal pain, nausea, febrile neutropenia, chills, cough, infection with an unspecified pathogen, dizziness, tremor, decreased appetite, edema, hypoxia, abdominal pain, aphasia, constipation, and vomiting.

The most common adverse reactions (incidence 20%) in patients with LBCL in ZUMA-1 included CRS, fever, hypotension, encephalopathy, tachycardia, fatigue, headache, decreased appetite, chills, diarrhea, febrile neutropenia, infections with an unspecified, nausea, hypoxia, tremor, cough, vomiting, dizziness, constipation, and cardiac arrhythmias.

The most common non-laboratory adverse reactions (incidence 20%) in patients with iNHL in ZUMA-5 included fever, CRS, hypotension, encephalopathy, fatigue, headache, infections with an unspecified, tachycardia, febrile neutropenia, musculoskeletal pain, nausea, tremor, chills, diarrhea, constipation, decreased appetite, cough, vomiting, hypoxia, arrhythmia, and dizziness.

About Kite

Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, with manufacturing operations in North America and Europe. Kites singular focus is cell therapy to treat and potentially cure cancer. As the cell therapy leader, Kite has more approved CAR T indications to help more patients than any other company. For more information on Kite, please visit http://www.kitepharma.com. Follow Kite on social media on Twitter (@KitePharma) and LinkedIn.

About Gilead Sciences

Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including the ability of Kite and Gilead to initiate, progress or complete clinical trials within currently anticipated timelines or at all, and the possibility of unfavorable results from ongoing and additional clinical trials, including those involving Yescarta; the risk that physicians may not see the benefits of prescribing Yescarta for the treatment of FL; and any assumptions underlying any of the foregoing. These and other risks, uncertainties and other factors are described in detail in Gileads Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties, and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to Kite and Gilead, and Kite and Gilead assume no obligation and disclaim any intent to update any such forward-looking statements.

U.S. Prescribing Information for Yescarta including BOXED WARNING, is available at http://www.kitepharma.com and http://www.gilead.com .

Kite, the Kite logo, Yescarta, and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220628005521/en/

Jacquie Ross, Investorsinvestor_relations@gilead.com

Anna Padula, Mediaapadula@kitepharma.com

Source: Gilead Sciences, Inc.

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Kite's CAR T-cell Therapy Yescarta Granted European Marketing Authorization for the Treatment of Relapsed or Refractory Follicular Lymphoma - Gilead...

Summary: Researchers identified differences in isoforms that control Treg cells and how that affects the bodys immune system response.

Source: Indiana University

Researchers atIndiana University School of Medicineare learning more about how special regulatory T cells can impact the immune systems response and how those cells could be manipulated for potential treatments for food allergies and autoimmune diseases.

Ina study recently published inScience Immunology,researchers focused on regulatory T cells, or Treg cells, that regulate immune responses in the body and keep the immune system in order while fighting pathogens.

In some cases, the immune system becomes overly responsive, leading to autoimmune diseases, such as Type 1 diabetes or lupus, food allergies or other issues. Researchers were able to identify the differences in isoforms that control Treg cells and how that affects the bodys immune function.

There is a particular gene that controls this regulatory group of T cells, which controls immune response, saidBaohua Zhou, PhD, lead author of the study and associate professor of pediatrics forIU School of Medicine Department of Pediatrics.

Treg cells can help maintain the right balance to help the immune system not respond too strongly or too weakly.

The human gene FOXP3 produces two major isoforms through alternative splicinga longer isoform and a shorter isoform.

The two isoforms are naturally expressed in humans, but their differences in controlling regulatory T cell phenotype and functionality has been unclear. In this study, researchers showed patients expressing only the shorter isoform fail to maintain self-tolerance and develop issues like immunodeficiency, polyendocrinopathy and enteropathy X-linked (IPEX) syndrome.

They uncovered different functions of the FOXP3 isoforms to regulate Treg cells and immune homeostasis.

Now that we know the different functions of the isoforms, we hope to study how to change them, which could lead to new treatments for autoimmune diseases and allergies, Zhou said.

We could also potentially manipulate them to keep the body from responding improperly to diseases like cancer. If T reg cells are suppressing the antitumor response, can we change that?

Author: Christina GriffithsSource: Indiana UniversityContact: Christina Griffiths Indiana UniversityImage: The image is in the public domain

Original Research: Closed access.FOXP3 exon 2 controls Treg stability and autoimmunity by Baohua Zhou et al. Science Immunology

Abstract

FOXP3 exon 2 controls Treg stability and autoimmunity

Differing from the mouseFoxp3gene that encodes only one protein product, humanFOXP3encodes two major isoforms through alternative splicinga longer isoform (FOXP3 FL) containing all the coding exons and a shorter isoform lacking the amino acids encoded by exon 2 (FOXP3 E2).

The two isoforms are naturally expressed in humans, yet their differences in controlling regulatory T cell phenotype and functionality remain unclear.

In this study, we show that patients expressing only the shorter isoform fail to maintain self-tolerance and develop immunodeficiency, polyendocrinopathy, and enteropathy X-linked (IPEX) syndrome.

Mice withFoxp3exon 2 deletion have excessive follicular helper T (TFH) and germinal center B (GC B) cell responses, and develop systemic autoimmune disease with anti-dsDNA and antinuclear autoantibody production, as well as immune complex glomerulonephritis. Despite having normal suppressive function in in vitro assays, regulatory T cells expressing FOXP3 E2 are unstable and sufficient to induce autoimmunity when transferred intoTcrb-deficient mice.

Mechanistically, the FOXP3 E2 isoform allows increased expression of selected cytokines, but decreased expression of a set of positive regulators ofFoxp3without altered binding to these gene loci.

These findings uncover indispensable functions of the FOXP3 exon 2 region, highlighting a role in regulating a transcriptional program that maintains Tregstability and immune homeostasis.

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Important Factors for Regulating the Body's Immune Response - Neuroscience News

Stem Cell Therapy Market Is Expected To Reach USD455.61 Billion By 2027 At A CAGR Of 16 percent.

Maximize Market Research has published a report on theStem Cell Therapy Marketthat provides a detailed analysis for the forecast period of 2022 to 2027.

Stem Cell Therapy Market Scope:

The report provides comprehensive market insights for industry stakeholders, including an explanation of complicated market data in simple language, the industrys history and present situation, as well as expected market size and trends. The research investigates all industry categories, with an emphasis on key companies such as market leaders, followers, and new entrants. The paper includes a full PESTLE analysis for each country. A thorough picture of the competitive landscape of major competitors in the Stem Cell Therapy market by goods and services, revenue, financial situation, portfolio, growth plans, and geographical presence makes the study an investors guide.

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Stem Cell Therapy Market Overview:

Stem cells, which are the most important in the body, exist in both humans and animals. Stem cells, which may multiply and grow into almost any cell type in the body, are employed in surgery and medicine. There are two types of stem cells: adult stem cells and embryonic stem cells. Embryonic stem cells are stem cells derived from human embryos (ESCs). They are pluripotent, which means they can develop into practically any type of cell in the body. Regenerative medicine or cornerstone treatment are other terms for stem cell therapy. Regenerative medicines can restore cells and replace those that have been damaged or killed.

Mesenchymal stem cells may penetrate and integrate into different organs, heal cardiovascular, lung, and spinal cord injuries, and ameliorate the condition of autoimmune illnesses, liver disorders, and bone and cartilage diseases. Stem cells are an effective therapy option for infections induced by inflammation, immune system failure, or tissue degradation.

Stem Cell Therapy MarketDynamics:

The use of stem cells in regenerative medicine, notably in dermatology, is likely to drive significant growth in the global Stem Cell Therapy Market during the forecasted period. Additionally, increased oncology use, as a result of a large number of pipeline medications under development for the treatment of tumors or malignancies, would move the market ahead. The stem cell business is expected to flourish in the future as the number of regenerative medicine clinics increases. Moreover, the rising prevalence of chronic diseases has assisted the growth of the stem cell treatment sector.

Long work hours, a lack of physical activity, and unhealthy eating and drinking habits all lead to the development of chronic diseases and need stem cell therapy. Moreover, the growing death rate from chronic diseases throughout the world is expected to propel the worldwide Stem Cell Therapy Market ahead. Additionally, the growing popularity of personalized pharmaceuticals is driving the worldwide Stem Cell Therapy Market. Researchers have identified new procurement strategies that can be used to generate personalized pharmaceuticals.

Because stem cells are generated by killing human embryos, they raise several ethical concerns. Human embryos are recognized as potential life, and eliminating them, even if they can save a human life, is considered immoral. Concerns about using embryonic stem cells to develop stem cell therapies are restricting the global market growth.

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Stem Cell Therapy MarketRegional Insights:

The market for stem cell treatment was dominated by North America, Asia Pacific, and Europe. This geographical segments significant share of the stem cell therapy market can be attributed to increased public-private financing and research grants for producing safe and effective stem cell treatment products, as well as the growing number of clinical trials, as well as North Americas major share of the stem cell therapy market with increased sales of stem cell therapy.

Stem Cell Therapy MarketSegmentation:

By Treatment:

By Therapeutic Application:

By Cell Source:

By End users:

Stem Cell Therapy Market Key Competitors:

About Maximize Market Research:

Maximize Market Research is a multifaceted market research and consulting company with professionals from several industries. Some of the industries we cover include medical devices, pharmaceutical manufacturers, science and engineering, electronic components, industrial equipment, technology and communication, cars and automobiles, chemical products and substances, general merchandise, beverages, personal care, and automated systems. To mention a few, we provide market-verified industry estimations, technical trend analysis, crucial market research, strategic advice, competition analysis, production and demand analysis, and client impact studies.

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Stem Cell Therapy Market Is Expected To Reach USD 455.61 Billion By 2027 At A CAGR Of 16 percent By Forecast 2027 Says Maximize Market Research (MMR)...

Neurona has initiated dosing of a first-ever regenerative human cell therapy candidate in a Phase 1/2 clinical trial for epilepsy

Pioneering cell therapy approach could revolutionize the treatment of drug-resistant focal epilepsy

SAN FRANCISCO, June 29, 2022 (GLOBE NEWSWIRE) -- Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for the treatment of neurological disorders, today announced the first patient dosed in a Phase 1/2 clinical trial of its lead program, NRTX-1001, in a first-in-human epilepsy study. An estimated three million Americans have epilepsy, and 25 to 35 percent live with ongoing seizures despite dozens of approved drugs on the market, which means that there is a huge unmet medical need in this community. NRTX-1001 is a regenerative neural cell therapy delivered as a single dose and designed to provide long-term secretion of gamma-aminobutyric acid (GABA), a key inhibitory neurotransmitter, to repair hyper-excitable neural networks associated with mesial temporal lobe epilepsy (MTLE), the most common form of focal epilepsy in adults.

Administration of NRTX-1001 to the first patient in our clinical trial for MTLE represents a huge milestone for Neurona and the neurology field, said Cory R. Nicholas, Ph.D. Neuronas president and chief executive officer. This promising program is the result of many years of work, dedication, and innovation by the Neurona team and its collaborators as well as key funding secured by grants received from the California Institute for Regenerative Medicine. We are very excited to take this next step, bringing a first-in-class, novel regenerative treatment to people living with chronic focal seizures. I would like to thank the excellent team at SUNY Upstate Medical University who treated the first patient, as well as our other clinical sites across the country who are part of this multicenter study.

Neuronas regenerative cell therapy approach has the potential to provide a single-administration, non-destructive alternative for the treatment of drug-resistant focal epilepsy, Currently, people with mesial temporal lobe epilepsy who are not responsive to anti-seizure medications have few options, such as an invasive surgery that removes or destroys the affected brain tissue. said Harish Babu, M.D., Ph.D., assistant professor of neurosurgery at SUNY Upstate Medical University and the surgeon who administered the first dose of NRTX-1001.

The objective of NRTX-1001 is to add cells that have the potential to repair the circuits that are damaged in epilepsy and thus reduce seizure activity. said Robert Beach, M.D., Ph.D. professor of neurology at SUNY Upstate Medical University. We are delighted to work with the Neurona team to evaluate the potential of this exciting new therapeutic approach.

About Neuronas Clinical Trial of NRTX-1001 for Mesial Temporal Lobe Epilepsy (MTLE)Neuronas multicenter, Phase 1/2 clinical trial is designed to evaluate the safety and efficacy of a single administration of NRTX-1001 for drug-resistant MTLE. The first stage of the trial is an open-label dose-escalation study in up to 10 people with MTLE. Patients treated with a single infusion of NRTX-1001 cells will be monitored for safety, tolerability, neural cell viability, and effects on their epilepsy disease symptoms. Patient recruitment is underway at epilepsy centers across the United States. For more information, please visit http://www.clinicaltrials.gov (NCT05135091). The first part of the clinical trial is supported by a recently announced $8.0 million grant from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355).

About NRTX-1001NRTX-1001 is a regenerative neural cell therapy derived from human pluripotent stem cells. The fully-differentiated neural cells, called interneurons, secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). Delivered as a one-time dose, the human interneurons are intended to integrate and innervate on-target, providing long-term GABAergic inhibition to repair hyper-excitable neural networks.

About Mesial Temporal Lobe Epilepsy (MTLE)MTLE primarily affects the internal structures of the temporal lobe, where seizures often begin in a structure called the hippocampus. MTLE is the most common type of focal epilepsy in adults. For people resistant to anti-seizure drugs, epilepsy surgery, where the damaged temporal lobe is surgically removed or ablated by laser, can be an option. However, the current surgical options are not available or effective for all, are tissue-destructive, and can have significant adverse effects.

About NeuronaNeuronas regenerative cell therapies have single-dose curative potential. Neurona is developing off-the-shelf, allogeneic neuronal, glial, and gene-edited cell therapy candidates that are designed to provide long-term repair of dysfunctional neural networks for multiple neurological disorders. For more information about Neurona, visit http://www.neuronatherapeutics.com

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Neurona Therapeutics Announces Initial Subject Dosed in First Clinical Trial of Regenerative Human Cell Therapy, NRTX-1001, in Adults with...