Ryvu Therapeutics to present clinical and preclinical data on RVU120 at the 2024 European Hematology Association Congress

On May 12, 2024 Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, reported that it will present clinical and preclinical data from RVU120 at the 2024 European Hematology Association (EHA) (Free EHA Whitepaper) Congress (EHA) (Free EHA Whitepaper), June 13-16, Madrid, Spain (Press release, Ryvu Therapeutics, MAY 14, 2024, View Source [SID1234643265]).

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"At this year’s EHA (Free EHA Whitepaper) conference, we proudly unveil the latest advancements in RVU120 research and clinical development, illuminating its potential in addressing the unmet need in hematologic malignancies. RVU120 has demonstrated clinical activity as a single agent in 15 of 29 evaluable patients with AML and HR-MDS at tolerated doses. Furthermore, our preclinical data demonstrate the synergistic potential of RVU120 in combination with venetoclax, representing a promising strategy against resistance in AML. We are also excited about the emerging data in MPN models, where combination regimens with RVU120 could represent a new treatment paradigm." – Dr. Hendrik Nogai, Chief Medical Officer, Ryvu Therapeutics.

Based on these data and in line with prior guidance, Ryvu’s development plan for RVU120 includes four Phase II studies: the RIVER-52 study in patients with AML/HR-MDS as monotherapy, the RIVER-81 study in combination with venetoclax in patients with r/r AML, the POTAMI-61 study in patients with myelofibrosis, and the REMARK study, an investigator-initiated trial in patients with LR-MDS.

Poster highlights:

Abstract Title: RVU120, a first-in-class CDK8 inhibitor for the treatment of relapsed/refractory AML and high-risk MDS: preliminary results from two ongoing studies.

Number: 6466
Session date and time: Friday, June 14 (18:00-19:00 CEST)

The abstract includes data on 29 evaluable patients out of 38 total dosed patients.

In the Phase I trial CLI120-001, RVU120 shows promising clinical activity in patients with AML or HR-MDS. 15 out of 38 patients experienced clinical benefit with RBC transfusion independence and/or blast reduction. A complete remission (CR) was achieved in a patient with NPM1 and DNMT3A mutations as well as 3 marrow CRs in patients with HR-MDS. Relevant target inhibition was achieved at a dose of 110 mg or higher, supporting a recommended Phase II dose (RP2D) of 250mg. The follow-on Phase II RIVER-52 study investigates RVU120 as a monotherapy in patients with genetically defined AML or HR-MDS and is currently recruiting.

Abstract Title: Synergistic potential of RVU120, a first-in-class CDK8/CDK19 inhibitor, with venetoclax in AML: preclinical and initial clinical insights.

Abstract Number: 6720
Session date and time: Friday, June 14 (18:00-19:00 CEST)

The abstract presents updated preclinical data supporting the synergistic combination of RVU120 and venetoclax in AML, including RVU120’s potential to overcome resistance to venetoclax treatment. Ongoing preclinical research and an ongoing Phase II clinical study seek to refine patient stratification and enhance therapeutic outcomes, with initial safety profiles appearing favorable. The Phase II RIVER-81 study investigates RVU120 in combination with venetoclax in patients with AML and is currently recruiting.

Abstract Title: CDK8/19 Inhibition: A Promising Therapeutic Strategy in Myeloproliferative Neoplasms.

Poster Number: P1018

Session date and time: Friday, June 14 (18:00-19:00 CEST)

The presentation, prepared in collaboration with Prof. Raajit Rampal’s group from Memorial Sloan Kettering Cancer Center, includes the assessment of RVU120 as a monotherapy and in combination with JAK inhibitors and other drug candidates for the treatment of myeloproliferative neoplasms (MPN). Analysis of the effects from combinations of RVU120 with other compounds in vitro has revealed potentially new therapeutic options for patients with MPN sensitivity and resistance to ruxolitinib (RUX). In vivo data further support CDK8 inhibition as a potential novel therapeutic strategy in MPNs. Additional mechanistic work will potentially elucidate disease mechanisms and therapeutic action independent of JAK-STAT attenuation. This work has led to a Phase II clinical study (POTAMI-61) for RVU120 as a monotherapy in patients with myelofibrosis patients not eligible for RUX or in combination with RUX for those patients with myelofibrosis and suboptimal RUX response.

The final posters that will be presented on June 14th will contain additional information.

Investor Event:

Ryvu will host a webinar on Friday, May 17, at 3:00 PM CET with Professor Raajit Rampal, who will present the myelofibrosis landscape, the latest data and the potential of RVU120 in MF.

To join the webcast, please register here: View Source

Transgene provides business and financial update for Q1 2024

On May 14, 2024 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, reported a business update, including its financial position as of March 31, 2024 (Press release, Transgene, MAY 14, 2024, View Source [SID1234643264]).

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Key events and upcoming milestones

Transgene and its partner NEC reported updated TG4050 Phase I data at AACR (Free AACR Whitepaper) 2024 (see April 9 press release here), showing first clinical benefit for patients treated in the adjuvant setting for head and neck cancer, a patient population at high risk of relapse.

Almost all treated patients (16/17) developed a specific immune response against the selected personalized antigen targets. All patients who received TG4050 remain disease-free after a median follow-up of 18.6 months, comparing favorably to the observational arm which saw 3 out of 16 patients relapse during the same period.

These data provide a robust clinical proof of principle for the Company’s lead candidate.

Additional data on the 24-month median follow up of patients will be reported in H2 2024.

Following these promising data, the randomized Phase I trial will be expanded to a randomized Phase I/II trial in the adjuvant setting of head and neck cancer. The Phase II part is expected to start enrolling patients in Q2 2024 within the framework of an extended collaboration between Transgene and NEC.

TG4050 has potential applicability across a range of solid tumors where the medical need is still significant despite the existing therapeutic option including immunotherapies.

Across 2024, Transgene expects to communicate progress and significant results on all of its clinical stage assets.

TG4050 Randomized Phase I part of trial (head and neck):
– 24-month patient follow up H2 2024
Randomized Phase II part to start (head and neck) H1 2024
Preliminary work to launch additional Phase I trial 2024
TG4001 Randomized Phase II: topline results H2 2024
TG6050 Initial data from Phase I trial H2 2024
BT-001 Initial data from combination part of Phase I H2 2024

Operating revenue
Q1
In millions of euros 2024 2023
Government funding for research expenditures 1.6 1.5
Revenue from collaborative and licensing agreements 0.1 0.1
Other income – –
Operating revenue 1.7 1.6

During the first quarter of 2024, operating revenue mostly comprised government funding for research expenditures, which mainly consisted of accrual of 25% of the research tax credit expected for 2024 (€1.6 million in the first quarter of 2024 compared to €1.5 million for the same period in 2023).

Cash, cash equivalents and other financial assets

Cash, cash equivalents and other financial assets stood at €13.7 million as of March 31, 2024, compared to €15.7 million as of December 31, 2023. In the first quarter of 2024, Transgene’s net cash burn was €11.2 million, excluding Institut Mérieux’s credit facility, compared to €9.8 million for the
same period in 2023.

In March 2024, the Company signed an amendment to the current account advance agreement with Institut Mérieux (TSGH) raising the available amount to a maximum of €66 million. At the end of March 2024, Transgene has used €22.4 million. The credit facility will be available until the end of 2025 and Transgene is able to draw on and repay the facility at its discretion.

As a result, the Company confirms its financial visibility until Q4 2025, enabling the Company to reach important development milestones and deliver significant news flow on its portfolio.

Kite Data At EHA 2024 Demonstrate Positive Patient Outcomes And Advances In Delivery Of CAR T-Cell Therapy

On May 14, 2024 Kite, a Gilead Company (Nasdaq: GILD), reported 12 abstracts from its industry-leading Chimeric Antigen Receptor (CAR) T-cell therapy portfolio at the upcoming 2024 European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress, June 13-16, Madrid, Spain (Press release, Gilead Sciences, MAY 14, 2024, View Source [SID1234643263]).

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Four presentations will highlight real-world experience with Yescarta (axicabtagene ciloleucel), including manufacturing experience for patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL) in second-line versus third-line treatment and beyond. The real-world manufacturing analysis investigated potential benefits of administering Yescarta in earlier lines of therapy by assessing manufacturing success rate and product characteristics.

Analyses exploring outpatient administration of Yescarta in R/R LBCL includes preliminary results of the Phase 2 ZUMA-24 study to evaluate the safety and efficacy of Yescarta outpatient administration compared to previous in-hospital clinical trials and real-world evidence. A real-world analysis on the use of Yescarta and Tecartus (brexucabtagene autoleucel) assess safety trends and hospitalization rates following treatment to further understand the feasibility of CAR T-cell therapy administration in the outpatient setting.

"Whether it’s continuing to evolve our industry-leading manufacturing capabilities or looking at different ways patients can obtain CAR T-cell therapy, such as in the outpatient setting, our goal is to improve patient outcomes and experience with our therapies," said Ibrahim Elhoussieny, Vice President, Medical Affairs, Kite. "These data will reflect our progress in helping CAR T reach more people with complex and hard-to-treat blood cancers."

Kite and its multiple myeloma (MM) partner Arcellx will share the clinical trial design for the pivotal Phase 3 iMMagine-3 trial, which will investigate the efficacy and safety of anitocabtagene autoleucel (anito-cel) versus standard of care in patients with R/R MM. In addition, Arcellx will highlight findings from the Phase 1 study of anito-cel in R/R MM in an oral presentation. Anito-cel is an BCMA CAR T investigational that utilizes a novel binder (or CAR) known as the D-Domain. Its small size (8kDa) facilitates high T-cell transduction and expression, resulting in more CAR positive cells and more CARs expressed per T-cell.

Table of Accepted Abstracts (all times CEST):

Abstract Details

Titles

Lymphomas

Poster P1425

Fri, June 14, 2024

6:00 – 7:00 PM CEST

Real-World Manufacturing Experience of Axicabtagene Ciloleucel for Patients with Relapsed or Refractory Large B-Cell Lymphoma Treated in Second Line versus Third Line of Therapy and Beyond

Jason R. Westin, Dalia Rangel, José G. Ramírez, David Myers, Laura Alquist, Clare Spooner, Harry W. Smith, Rhine R. Shen, Simone Filosto, and Frederick L. Locke.

Poster P1159

Fri, June 14, 2024

6:00 – 7:00 PM CEST

ZUMA-24 Preliminary Analysis: A Phase 2 Study of Axicabtagene Ciloleucel in the Outpatient Setting with Prophylactic Corticosteroids in Patients with Relapsed/Refractory Large B-Cell Lymphoma

Lori A. Leslie, John H. Baird, Ian W. Flinn, Michael Tees, Daanish Hoda, Abhinav Deol, Monica Mead, Brian McClune, Indumathy Vanadarajan, James Essell, Suzanne Fanning, Gary Simmons, William Clark, Aaron P. Rapaport, Tulio E. Rodriguez, Joshua N. Winters, Madison Davis, Harry M. Miao, Yan Zheng, Jenny J. Kim, and Olalekan O. Oluwole.

ePoster P2092

Fri, June 14, 2024

9:00 AM CEST

Complete Response at 6 Months as Associated with Overall Survival After 1L Chemoimmunotherapy for High-Risk Diffuse Large B-Cell Lymphoma: A Pooled Clinical Trial Analysis

Ran Reshef, Zhen-Huan Hu, Sarit Assouline, Corinne Haioun, Bertram Glass, Armin Ghobadi, Rahul Jain, Weixi Chen, Yiling Yang, Kelly Speth, Shilpa A. Shahani, Myrna Nahas, Elizabeth Shima Rich, Clare Spooner, Qinghua Song, Fang Sun, and Michael Dickinson.

Poster P1199

Fri, June 14, 2024

6:00 – 7:00 PM CEST

Real-World Outcomes of Axicabtagene Ciloleucel for Treatment of Relapsed or Refractory Large B-Cell Lymphoma in Canada

Christopher Lemieux, John Kuruvilla, Mona Shafey, Kelly Davidson, Kristjan Paulson, Brent Logan, Matthew Bye, Sue Li, Lieven Billen, Francis Nissen, Hai-Lin Wang, Jenny J. Kim, Harry Smith, Grace Lee, Zhen-Huan Hu, Marcelo C. Pasquini, and Kevin Hay.

Oral S334

Sat, June 15, 2024

4:30 – 5:45 PM CEST

The Cost-Effectiveness of Axicabtagene Ciloleucel versus Standard of Care as Second-Line Therapy in Patients with Large B-Cell Lymphoma in Sweden

Oskar Eklund, Viktor Hedlof Kanje, Yael A. Rodriguez-Guadarrama, Rob Blissett, Nathaniel J. Smith, Frank van Hees, Brett Doble, and Sachin Vadgama.

Poster P1217

Fri, June 14, 2024

6:00 – 7:00 PM CEST

Cost-effectiveness and Budget Impact Analyses of Axicabtagene Ciloleucel as Second-Line Therapy in Patients with Large B-Cell Lymphoma in Singapore

Francesca Lim, Tza-Kai Diong, Graeme Ball, Esther Chan, Mahendra Rai, Jatin Pruthi, Neha Deshpande and William Hwang.

ePoster P2088

Fri, June 14, 2024

9:00 AM CEST

Real-World Safety Outcomes of Axicabtagene Ciloleucel in Patients with Diffuse Large B-Cell Lymphoma and Follicular Lymphoma in Europe and United States: A Systematic Review and Meta-Analysis

Robin Sanderson, Javier Munoz, Francis Ayuk, Francis Nissen, Fang Sun, Eve Limbrick-Oldfield, David Wennersbusch, Grace Lee and Caron A. Jacobson.

Poster P1666

Fri, June 14, 2024

6:00 – 7:00 PM CEST

Treatment Preferences and Quality of Life in Patients with Relapsed/Refractory Follicular Lymphoma

John G. Gribben, Emmanuel Bachy, Markqayne Ray, Kathryn Krupsky, Kathleen Beusterien, Lewis Kopenhafer, Flor Mendez, Sara Beygi, Timothy Best, Graeme Ball, Kacper Perkowski, Oliver Will, Anik Patel and Paola Ghione.

Poster P1191

Fri, June 14, 2024

6:00 – 7:00 PM CEST

* In collaboration with Mayo Clinic

Updated Trends in Real-World Outpatient (OP) Administration of Axicabtagene Ciloleucel (Axi-Cel) and Brexucabtagene Autoleucel (Brexu-Cel) in Relapsed/Refractory (R/R) Non-Hodgkin Lymphoma (NHL)*

Radhika Bansal, Hil Hsu, Jonas Paludo, Brad Du, Andre De Menezes Silva Corraes, Patrick Johnston, Arushi Khurana, Yucai Wang, Christine Fu, Jenny J. Kim, Qinghua Song, James Wu, Fang Sun and Yi Lin.

Multiple Myeloma

E-publication only PB2724

To be published in the Online Abstract Book, a supplement of HemaSphere (EHA’s official journal)

*In collaboration with Kite partner Arcellx

iMMagine-3: A Phase 3, Randomized Study to Compare the Efficacy and Safety of Anitocabtagene Autoleucel (Anito-Cel) with Standard of Care in Patients with Relapsed/Refractory Multiple Myeloma (RRMM)

Tom Martin, Noopur S. Raje, Jesús San-Miguel, Krina Patel, Lucas McLoughlin, Christine Lui, Carolyn C. Jackson, Christopher R. Heery, Niels W.C.J. van de Donk, Jesús G. Berdeja and Maria-Victoria Mateo.

Oral S207

*In collaboration with Kite partner Arcellx

Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Patients with Relapsed and/or Refractory Multiple Myeloma: Results from at Least 1-Year Follow-up in All Patients*

Matthew Frigault, Jacalyn Rosenblatt, Binod Dhakal, Noopur Raje, Daniella Cook, Mahmoud Gaballa, Estelle Emmanuel-Alejandro, Danielle Nissen, Kamalika Bannerjee, Anand Rotte, Christopher Heery, David Avigan, Andrzej Jakubowiak and Michael Bishop.

Acute Lymphoblastic Leukemia

E-publication only PB3430

To be published in the Online Abstract Book, a supplement of HemaSphere (EHA’s official journal)

Sustained Cost-Effectiveness of Brexucabtagene Autoleucel for the Treatment of Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia in Patients Aged 26 Years or Older in Italy

Monia Marchetti, Cristina Dondoni, Tomas Spousta, Frank van Hees, Nate Smith and Brett Doble.

For more information, including a complete list of abstract titles at the meeting, please visit: View Source

About anitocabtagene autoleucel (anito-cel)

Anito-cel is a BCMA CAR T that utilizes a novel binder (or CAR) known as the D-Domain. Its small size (8kDa) facilitates high T-cell transduction and expression, resulting in more CAR positive cells and more CARs expressed per T-cell.

Anito-cel has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy designations by the U.S. Food and Drug Administration.

About Yescarta

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

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

Adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy.
Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.

Limitations of Use: YESCARTA is not indicated for the treatment of patients with primary central nervous system lymphoma.

Adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
U.S. IMPORTANT SAFETY INFORMATION

BOXED WARNING: CYTOKINE RELEASE SYNDROME, NEUROLOGIC TOXICITIES and SECONDARY HEMATOLOGICAL MALIGNANCIES

Cytokine Release Syndrome (CRS), including fatal or life-threatening reactions, occurred in patients receiving YESCARTA. Do not administer YESCARTA to patients with active infection or inflammatory disorders. Treat severe or life-threatening CRS with tocilizumab or tocilizumab and corticosteroids.
Neurologic toxicities, including fatal or life-threatening reactions, occurred in patients receiving YESCARTA, including concurrently with CRS or after CRS resolution. Monitor for neurologic toxicities after treatment with YESCARTA. Provide supportive care and/or corticosteroids, as needed.
T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including YESCARTA.
YESCARTA is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the YESCARTA and TECARTUS REMS Program.
CYTOKINE RELEASE SYNDROME (CRS)

CRS, including fatal or life-threatening reactions, occurred following treatment with YESCARTA. CRS occurred in 90% (379/422) of patients with non-Hodgkin lymphoma (NHL) receiving YESCARTA, including ≥ Grade 3 (Lee grading system1) CRS in 9%. CRS occurred in 93% (256/276) of patients with large B-cell lymphoma (LBCL), including ≥ Grade 3 CRS in 9%. Among patients with LBCL who died after receiving YESCARTA, four 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 to 12 days) and the median duration of CRS was 7 days (range: 2 to 58 days). For patients with LBCL in ZUMA-7, the median time to onset of CRS was 3 days following infusion (range: 1 to 10 days) and the median duration was 7 days (range: 2 to 43 days).

CRS occurred in 84% (123/146) of patients with indolent non-Hodgkin lymphoma (iNHL) in ZUMA-5, including ≥ Grade 3 CRS in 8%. Among patients with iNHL who died after receiving YESCARTA, one patient had an ongoing CRS event at the time of death. The median time to onset of CRS was 4 days (range: 1 to 20 days) and the median duration was 6 days (range: 1 to 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 (HLH/MAS).

The impact of tocilizumab and/or corticosteroids on the incidence and severity of CRS was assessed in two subsequent cohorts of LBCL patients in ZUMA-1. Among patients who received tocilizumab and/or corticosteroids for ongoing Grade 1 events (see Table 1), 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 to 8 days) and the median duration of CRS was 7 days (range: 2 to 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 at which point the patients were managed with tocilizumab and/or therapeutic doses of corticosteroids with no patients developing Grade 3 or higher CRS. The median time to onset of CRS was 5 days (range: 1 to 15 days) and the median duration of CRS was 4 days (range: 1 to 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 infusion of YESCARTA. Monitor patients at least daily for 7 days at the certified healthcare facility following infusion for signs and symptoms of CRS. Monitor patients for signs or symptoms of CRS for 4 weeks after infusion. 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 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 higher grade of neurologic toxicities or prolongation of neurologic toxicities, delay the onset 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 about the management of CRS and neurologic toxicities. Further information is available at 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 or higher infections occurred in 17% of patients, including ≥ Grade 3 or higher 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

Patients treated with YESCARTA may develop secondary malignancies. T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including YESCARTA. Mature T cell malignancies, including CAR-positive tumors, may present as soon as weeks following infusion, and may include fatal outcomes.

Monitor life-long for secondary malignancies. In the event that a secondary malignancy 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 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 pathogen 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 pathogen unspecified, tachycardia, febrile neutropenia, musculoskeletal pain, nausea, tremor, chills, diarrhea, constipation, decreased appetite, cough, vomiting, hypoxia, arrhythmia, and dizziness.

About Tecartus

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

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

Adult patients with relapsed or refractory mantle cell lymphoma (MCL).

This indication is approved under accelerated approval based on overall response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).
U.S. IMPORTANT SAFETY INFORMATION

BOXED WARNING: CYTOKINE RELEASE SYNDROME, NEUROLOGIC TOXICITIES and SECONDARY HEMATOLOGICAL MALIGNANCIES

Cytokine Release Syndrome (CRS), including life-threatening reactions, occurred in patients receiving Tecartus. Do not administer Tecartus to patients with active infection or inflammatory disorders. Treat severe or life-threatening CRS with tocilizumab or tocilizumab and corticosteroids.
Neurologic toxicities, including life-threatening reactions, occurred in patients receiving Tecartus, including concurrently with CRS or after CRS resolution. Monitor for neurologic toxicities after treatment with Tecartus. Provide supportive care and/or corticosteroids as needed.
T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies.
Tecartus is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Yescarta and Tecartus REMS Program.
Cytokine Release Syndrome (CRS), including life-threatening reactions, occurred following treatment with Tecartus. CRS occurred in 92% (72/78) of patients with ALL, including ≥ Grade 3 (Lee grading system) CRS in 26% of patients. Three patients with ALL had ongoing CRS events at the time of death. The median time to onset of CRS was five days (range: 1 to 12 days) and the median duration of CRS was eight days (range: 2 to 63 days) for patients with ALL.

Ensure that a minimum of two doses of tocilizumab are available for each patient prior to infusion of Tecartus. Following infusion, monitor patients for signs and symptoms of CRS daily for at least seven days at the certified healthcare facility, and for four 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 Events, including those that were fatal or life-threatening, occurred following treatment with Tecartus. Neurologic events occurred in 87% (68/78) of patients with ALL, including ≥ Grade 3 in 35% of patients. The median time to onset for neurologic events was seven days (range: 1 to 51 days) with a median duration of 15 days (range: 1 to 397 days) in patients with ALL. For patients with MCL, 54 (66%) patients experienced CRS before the onset of neurological events. Five (6%) patients did not experience CRS with neurologic events and eight patients (10%) developed neurological events after the resolution of CRS. Neurologic events resolved for 119 out of 134 (89%) patients treated with Tecartus. Nine patients (three patients with MCL and six patients with ALL) had ongoing neurologic events at the time of death. For patients with ALL, neurologic events occurred before, during, and after CRS in 4 (5%), 57 (73%), and 8 (10%) of patients; respectively. Three patients (4%) had neurologic events without CRS. The onset of neurologic events can be concurrent with CRS, following resolution of CRS or in the absence of CRS.

The most common neurologic events (>10%) were similar in MCL and ALL and included encephalopathy (57%), headache (37%), tremor (34%), confusional state (26%), aphasia (23%), delirium (17%), dizziness (15%), anxiety (14%), and agitation (12%). Serious events including encephalopathy, aphasia, confusional state, and seizures occurred after treatment with Tecartus.

Monitor patients daily for at least seven days for patients with MCL and at least 14 days for patients with ALL at the certified healthcare facility and for four weeks following infusion for signs and symptoms of neurologic toxicities and treat promptly.

REMS Program: Because of the risk of CRS and neurologic toxicities, Tecartus is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Yescarta and Tecartus REMS Program which requires that:

Healthcare facilities that dispense and administer Tecartus must be enrolled and comply with the REMS requirements. Certified healthcare facilities must have on-site, immediate access to tocilizumab, and ensure that a minimum of two doses of tocilizumab are available for each patient for infusion within two hours after Tecartus infusion, if needed for treatment of CRS.
Certified healthcare facilities must ensure that healthcare providers who prescribe, dispense, or administer Tecartus are trained in the management of CRS and neurologic toxicities. Further information is available at www.YescartaTecartusREMS.com or 1-844-454-KITE (5483).
Hypersensitivity Reactions: Serious hypersensitivity reactions, including anaphylaxis, may occur due to dimethyl sulfoxide (DMSO) or residual gentamicin in Tecartus.

Severe Infections: Severe or life-threatening infections occurred in patients after Tecartus infusion. Infections (all grades) occurred in 56% (46/82) of patients with MCL and 44% (34/78) of patients with ALL. Grade 3 or higher infections, including bacterial, viral, and fungal infections, occurred in 30% of patients with ALL and MCL. Tecartus should not be administered to patients with clinically significant active systemic infections. Monitor patients for signs and symptoms of infection before and after Tecartus infusion and treat appropriately. Administer prophylactic antimicrobials according to local guidelines.

Febrile neutropenia was observed in 6% of patients with MCL and 35% of patients with ALL after Tecartus infusion and may be concurrent with CRS. The febrile neutropenia in 27 (35%) of patients with ALL includes events of "febrile neutropenia" (11 (14%)) plus the concurrent events of "fever" and "neutropenia" (16 (21%)). 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, life-threatening and fatal opportunistic infections have been reported. The possibility of rare infectious etiologies (e.g., fungal and viral infections such as HHV-6 and progressive multifocal leukoencephalopathy) should be considered in 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. 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 Tecartus infusion. In patients with MCL, Grade 3 or higher cytopenias not resolved by Day 30 following Tecartus infusion occurred in 55% (45/82) of patients and included thrombocytopenia (38%), neutropenia (37%), and anemia (17%). In patients with ALL who were responders to Tecartus treatment, Grade 3 or higher cytopenias not resolved by Day 30 following Tecartus infusion occurred in 20% (7/35) of the patients and included neutropenia (12%) and thrombocytopenia (12%); Grade 3 or higher cytopenias not resolved by Day 60 following Tecartus infusion occurred in 11% (4/35) of the patients and included neutropenia (9%) and thrombocytopenia (6%). Monitor blood counts after Tecartus infusion.

Hypogammaglobulinemia: B cell aplasia and hypogammaglobulinemia can occur in patients receiving treatment with Tecartus. Hypogammaglobulinemia was reported in 16% (13/82) of patients with MCL and 9% (7/78) of patients with ALL. Monitor immunoglobulin levels after treatment with Tecartus and manage using infection precautions, antibiotic prophylaxis, and immunoglobulin replacement.

The safety of immunization with live viral vaccines during or following Tecartus treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least six weeks prior to the start of lymphodepleting chemotherapy, during Tecartus treatment, and until immune recovery following treatment with Tecartus.

Secondary Malignancies may develop. T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies. Mature T cell malignancies, including CAR-positive tumors, may present as soon as weeks following infusion, and may include fatal outcomes. 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 Tecartus infusion. Advise patients to refrain from driving and engaging in hazardous activities, such as operating heavy or potentially dangerous machinery, during this period.

Adverse Reactions: The most common non-laboratory adverse reactions (≥ 20%) were fever, cytokine release syndrome, hypotension, encephalopathy, tachycardia, nausea, chills, headache, fatigue, febrile neutropenia, diarrhea, musculoskeletal pain, hypoxia, rash, edema, tremor, infection with pathogen unspecified, constipation, decreased appetite, and vomiting. The most common serious adverse reactions (≥ 2%) were cytokine release syndrome, febrile neutropenia, hypotension, encephalopathy, fever, infection with pathogen unspecified, hypoxia, tachycardia, bacterial infections, respiratory failure, seizure, diarrhea, dyspnea, fungal infections, viral infections, coagulopathy, delirium, fatigue, hemophagocytic lymphohistiocytosis, musculoskeletal pain, edema, and paraparesis.

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

Autolus Therapeutics to Present Three Clinical Data Updates on obecabtagene autoleucel (obe-cel) in relapsed/refractory (r/r) B-Cell acute lymphoblastic leukemia (ALL) patients at the 2024 European Hematology Association (EHA) Congress

On May 14, 2024 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported the online publication of three abstracts submitted to the European Hematology Association (EHA) (Free EHA Whitepaper) Congress, to be held June 13-16, 2024 (Press release, Autolus, MAY 14, 2024, View Source [SID1234643262]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Oral presentation:

Title: obecabtagene autoleucel in adult relapsed/refractory B Cell acute lymphoblastic leukemia: Survival and potential impact of CAR-T cell persistence and stem cell transplantation in the FELIX study
Session Title: s419 Acute lymphoblastic leukemia – Clinical 1: Immunotherapy: antibodies and CAR-T cells
Session date and time: Friday, June 14 from 14:45 – 16:00 CEST
Session room: N104
Final Abstract Code: S114
Presenting Author: Dr. Claire Roddie
Poster presentations:

Title: obecabtagene autoleucel (obe-cel, AUTO1) for relapsed/refractory adult B-Cell acute lymphoblastic leukemia (R/R B-ALL): The impact of inotuzumab-containing bridging therapy on treatment outcomes
Session Title: Poster session
Session date and time: Friday, June 14 from 18:00 – 19:00 CEST
Final Abstract Code: P418
Presenting Author: Dr. Jae H. Park

Title: Droplet digital PCR and flow cytometry sensitivity for measuring CAR-T cell kinetics in adult patients with relapsed/refractory B Cell acute lymphoblastic leukemia treated with obecabtagene autoleucel
Session Title: Poster session
Session date and time: Friday, June 14 from 18:00 – 19:00 CEST
Final Abstract Code: P1469
Presenting Author: Dr. Claire Roddie

Akebia Therapeutics to Present at the 2nd Annual HCW Bioconnect Investor Conference

On May 14, 2024 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, reported that John Butler, Chief Executive Officer, will participate in a fireside chat at the 2nd Annual HCW Bioconnect Investor Conference on Monday, May 20th, 2024 at 3:00 p.m. ET (Press release, Akebia, MAY 14, 2024, View Source/news-releases/news-release-details/akebia-therapeutics-present-2nd-annual-hcw-bioconnect-investor" target="_blank" title="View Source/news-releases/news-release-details/akebia-therapeutics-present-2nd-annual-hcw-bioconnect-investor" rel="nofollow">View Source [SID1234643261]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The 2nd Annual HCW Bioconnect Investor Conference will take place in New York, NY on May 20, 2024.

A webcast of the presentation can be accessed through the "Investors" section of Akebia’s website at View Source for 30 days following the conference.