KYV-101 has the potential to deliver durable, drug-free, disease-free remission in myasthenia gravis and set a new treatment standard for stiff person syndrome Innovative FDA-aligned KYSA-6 Phase 3 trial design for myasthenia gravis supports clear and rapid path to BLA Webcast today August 28th, at 11 am ET EMERYVILLE, Calif., Aug. 28, 2025 (GLOBE NEWSWIRE) -- Kyverna Therapeutics, Inc. (Nasdaq: KYTX), a clinical-stage biopharmaceutical company focused on developing cell therapies for patients with autoimm ...

Core Viewpoint - Lyell Immunopharma is advancing its lead CAR T-cell therapy, LYL314, which targets relapsed and/or refractory large B-cell lymphoma, showing promising clinical results and securing funding to support its development through mid-2027 [2][5][11]. Financial Results - For Q2 2025, Lyell reported a net loss of $42.7 million, a decrease from a net loss of $45.8 million in Q2 2024, primarily due to reduced stock-based compensation expenses [11][15]. - Cash, cash equivalents, and marketable securities as of June 30, 2025, were approximately $297 million, down from $384 million at the end of 2024, but expected to be sufficient to support operations into mid-2027 [13][19]. Clinical Development - LYL314 is currently in the pivotal PiNACLE trial for patients with relapsed and/or refractory large B-cell lymphoma in the third-line or later setting, with plans to initiate a second pivotal trial for the second-line setting by early 2026 [2][4][9]. - The Phase 1/2 trial data presented showed an overall response rate of 88% in the 3L+ cohort and 91% in the 2L cohort, with complete response rates of 72% and 64%, respectively [9]. Business Highlights - The company completed a private placement for gross proceeds of up to approximately $100 million, which will extend its cash runway and support the advancement of its clinical programs [5][8]. - LYL314 has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA, facilitating more frequent communication regarding its development [4][9]. Pipeline and Future Plans - Lyell is advancing a pipeline of next-generation CAR T-cell therapies, with plans to submit an Investigational New Drug (IND) application for a solid tumor candidate in 2026 [7][14]. - An update on the progress of the PiNACLE trial is expected in late 2025, with data anticipated to support a Biologics License Application submission to the FDA in 2027 [9].

Core Insights - CARsgen Therapeutics Holdings Limited has achieved a favorable outcome in opposition proceedings regarding its European patent EP3445407, which pertains to its GPC3-targeted CAR-T cell therapy [1][4] - A U.S.-based biotechnology company, the sole appellant among the original two opponents, has formally withdrawn its appeal against the EPO's decision to maintain the patent, making the decision final and binding [2][3] - The EPO granted the patent in 2022, and it was opposed by two parties in 2023, but the EPO upheld key claims related to the use of GPC3 CAR-T cell therapy for various cancers [3][4] Company Overview - CARsgen is a biopharmaceutical company focused on developing innovative CAR T-cell therapies to address unmet clinical needs, including hematologic malignancies, solid tumors, and autoimmune diseases [5] - The company has established comprehensive capabilities for CAR T-cell research and development, covering target discovery, preclinical research, clinical development, and commercial-scale production [5] - CARsgen aims to improve the safety profile, enhance efficacy in treating solid tumors, and reduce treatment costs, with a mission to become a global leader in providing innovative cell therapies [5]

Core Insights - Lyell Immunopharma, Inc. announced positive clinical data for LYL314, a CAR T-cell therapy for relapsed/refractory large B-cell lymphoma (LBCL), showing an 88% overall response rate and a 72% complete response rate in patients treated in the third- or later-line setting [1][3][4] Clinical Data Summary - In a Phase 1/2 trial, 25 patients in the 3L+ setting demonstrated an 88% overall response rate and a 72% complete response rate, with 71% of those achieving complete response remaining so at ≥ 6 months [1][3][4] - The efficacy evaluable population included 36 patients, with a median follow-up of 9 months for 3L+ patients and 5 months for 2L patients [2][3] - Among 51 CAR T-naive patients, 70% of those with primary refractory disease achieved a complete response [4] Safety Profile - The safety profile of LYL314 was manageable for outpatient administration, with no Grade ≥ 3 cytokine release syndrome reported and low rates of Grade 1 (22%) and Grade 2 (35%) adverse events [3][5] - Immune effector cell-associated neurotoxicity syndrome (ICANS) was reported in 6% (Grade 1), 2% (Grade 2), and 14% (Grade ≥ 3) of patients, with a median resolution time of 5 days [5] Ongoing Trials - The pivotal single-arm PiNACLE trial is currently underway, targeting approximately 120 patients with relapsed/refractory LBCL in the third- or later-line setting [6][12] - The trial aims to evaluate the overall response rate as the primary endpoint [12] Regulatory Designations - LYL314 has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA for the treatment of relapsed/refractory aggressive B-cell lymphoma [11] Manufacturing and Technology - LYL314 is designed to target B cells expressing either CD19 or CD20, utilizing a proprietary manufacturing process to enhance T-cell activity [10][9] - The Lyell LyFE Manufacturing Center™ has the capacity to produce over 1,200 CAR T-cell doses at full capacity [13]

Core Insights - Johnson & Johnson announced promising results from a Phase 1b study of JNJ-90014496, a dual-targeting CAR T-cell therapy for relapsed or refractory large B-cell lymphoma, showing a 75-80% complete response rate among evaluable patients at the recommended Phase 2 dose [1][2] Company Overview - Johnson & Johnson is committed to advancing innovative therapies for patients with B-cell malignancies, emphasizing their long-term dedication to addressing unmet medical needs [2][4] - The company is expanding its pipeline of CAR T therapies through a collaboration with AbelZeta Inc. to develop next-generation CAR T-cell therapies [2][3] Industry Context - Large B-cell lymphoma, particularly diffuse large B-cell lymphoma, accounts for approximately 40% of all non-Hodgkin lymphoma cases globally, with an estimated 150,000 new cases diagnosed each year [3] - Current single-antigen-targeting CD19 CAR T therapies provide long-term remissions for only about 40% of patients, highlighting the need for innovative treatment options [2][3]

Company Overview - CARsgen Therapeutics Holdings Limited is focused on developing innovative CAR T-cell therapies to address unmet clinical needs in hematologic malignancies, solid tumors, and autoimmune diseases [3] - The company has established end-to-end capabilities for CAR T-cell research and development, including target discovery, preclinical research, clinical development, and commercial-scale production [3] - CARsgen aims to improve the safety profile, enhance efficacy in treating solid tumors, and reduce treatment costs of CAR T-cell therapies [3] Product Development - Satri-cel is an autologous CAR T-cell product candidate targeting Claudin18.2, with a primary focus on treating Claudin18.2-positive advanced gastric/gastroesophageal junction adenocarcinoma (G/GEJA) and pancreatic cancer [2] - The product has received Priority Review from the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA) for patients who have failed at least two prior lines of therapy [1] - Satri-cel has been granted Breakthrough Therapy Designation by the CDE in March 2025 and has received Orphan Drug designation from the U.S. FDA in September 2020 [2] Clinical Trials - Ongoing clinical trials for Satri-cel include a confirmatory Phase II trial for advanced G/GEJA in China, a Phase Ib trial for pancreatic cancer adjuvant therapy, and a Phase 1b/2 trial for advanced gastric or pancreatic adenocarcinoma in North America [2] - The company is also conducting investigator-initiated trials to explore the use of Satri-cel as a consolidation treatment following adjuvant therapy in patients with resected G/GEJA [2]

Core Viewpoint - CARsgen Therapeutics announced the availability of research results from a confirmatory Phase II clinical trial of satricabtagene autoleucel ("satri-cel") for advanced gastric/gastroesophageal junction cancer, highlighting its potential as a new standard of care in this indication [1][9]. Group 1: Clinical Trial Overview - The Phase II trial (CT041-ST-01) was a multicenter, randomized controlled trial conducted in China, comparing satri-cel to standard of care in patients with CLDN18.2 positive advanced G/GEJC who had failed at least two prior treatments [2][4]. - The primary endpoint was progression-free survival (PFS) assessed by an Independent Review Committee (IRC), with overall survival (OS) as a key secondary endpoint [2][4]. Group 2: Patient Demographics and Treatment - A total of 156 patients were randomized (2:1) to receive either satri-cel (n=104) or treatment of physician's choice (TPC) (n=52), with a median of 2 prior systemic therapies in both arms [3][4]. - In the TPC arm, patients could receive subsequent satri-cel if they experienced disease progression or drug intolerance [3]. Group 3: Efficacy Results - In the intention-to-treat (ITT) population, the satri-cel arm showed a median PFS of 3.25 months compared to 1.77 months in the TPC arm, representing a 63% reduction in the risk of disease progression or death [5]. - The median OS for the satri-cel arm was 7.92 months versus 5.49 months for the TPC arm, indicating over a 30% reduction in mortality risk [5]. Group 4: Treatment Benefits in mITT Population - In the modified intention-to-treat (mITT) population, the median PFS was 4.37 months for satri-cel versus 1.84 months for TPC, showing a 70% reduction in risk of disease progression or death [6]. - The median OS in the mITT population was 8.61 months for satri-cel compared to 5.49 months for TPC, corresponding to a 40% reduction in mortality risk [6]. Group 5: Safety Profile - Satri-cel demonstrated a favorable safety profile, with only 4 cases of Grade 3 cytokine release syndrome (CRS) reported and no Grade 4-5 CRS events observed [8]. - No immune effector cell-associated neurotoxicity syndrome (ICANS) was reported [8]. Group 6: Significance of Findings - This trial represents the first confirmatory randomized controlled trial of CAR-T therapy in solid tumors, demonstrating significant PFS improvement and clinically meaningful OS benefits for patients with advanced G/GEJC [9]. - The results support the potential of satri-cel as a new standard of care for this patient population [9]. Group 7: About Satri-cel - Satri-cel is an autologous CAR T-cell product candidate targeting Claudin18.2, with ongoing trials for various indications including advanced gastric/gastroesophageal junction cancer and pancreatic cancer [10]. - The product has received Breakthrough Therapy Designation in China and Regenerative Medicine Advanced Therapy designation from the U.S. FDA, indicating its potential significance in treating advanced G/GEJC [10]. Group 8: About CARsgen Therapeutics - CARsgen Therapeutics is focused on developing innovative CAR T-cell therapies to address unmet clinical needs across various malignancies and diseases [11]. - The company has established comprehensive capabilities for CAR T-cell research and development, aiming to enhance safety and efficacy while reducing treatment costs [11].

Core Insights - Lyell Immunopharma, Inc. reported financial results and business highlights for Q1 2025, focusing on its lead clinical program LYL314, a CAR T-cell therapy for relapsed and/or refractory large B-cell lymphoma (LBCL) [1][2][3] Financial Performance - The company reported a net loss of $52.2 million for Q1 2025, an improvement from a net loss of $60.7 million in Q1 2024, primarily due to the absence of impairment expenses recognized in the prior year [8][18] - Cash, cash equivalents, and marketable securities totaled $330.1 million as of March 31, 2025, down from $383.5 million at the end of 2024, which is expected to support operations into 2027 [12][19] Clinical Development - LYL314 has received RMAT designation from the FDA, indicating its potential to address significant unmet needs in aggressive LBCL [4][5] - The company plans to present new clinical data for LYL314 at the International Conference on Malignant Lymphoma in June 2025, with pivotal trials expected to start in mid-2025 for the third- or later-line setting and early 2026 for the second-line setting [2][5][6] Research and Development - R&D expenses for Q1 2025 were $43.4 million, slightly up from $43.2 million in Q1 2024, driven by increased personnel costs related to workforce reductions [15][18] - The company is advancing a pipeline of next-generation CAR T-cell therapies, with a focus on overcoming T-cell exhaustion and enhancing durability in hostile tumor environments [7][13] Manufacturing Capabilities - The LyFE Manufacturing Center in Bothell, Washington, is now producing clinical supplies for LYL314 following successful technology transfer and FDA clearance [5][9] - The facility has the capacity to manufacture over 1,000 CAR T-cell therapy doses per year, supporting ongoing and planned pivotal trials [9][19]

Core Insights - Lyell Immunopharma, Inc. is advancing a pipeline of next-generation CAR T-cell therapies for cancer patients, with new clinical data for LYL314 to be presented at the ICML 2025 [1][2] - LYL314 is a dual-targeting CD19/CD20 CAR T-cell product candidate specifically for aggressive large B-cell lymphoma, having received FDA designations for advanced therapy and fast track [2] Company Overview - Lyell is a clinical-stage company focused on CAR T-cell therapies for hematologic malignancies and solid tumors, employing technologies to enhance CAR T-cell durability and efficacy in challenging tumor environments [3] Presentation Details - The presentation on LYL314 will occur on June 18, 2025, at 5:40 pm CEST, featuring Dr. Akil Merchant from Cedars-Sinai Medical Center [5]