B-ALL

Bionano Announces Publication Showing that OGM Detects Over 1250 Structural Variants, including 56 Gene Fusions, in Pediatric Leukemia that were Missed by Whole Genome Sequencing

Retrieved on: 
Thursday, April 11, 2024
B-ALL, OGM, RNA, WGS, Genetic variation, Doctor of Philosophy, Research, Leukemia, Bone marrow, Biomarker, Genomics, Whole genome sequencing, Cancer, SVS, SAN, Vaccine

The study also used whole genome sequencing (WGS) for variant detection and evaluated the two approaches side-by-side and the potential benefit of integrating WGS and OGM to obtain a comprehensive analysis of genetic variation.

Key Points: 
  • The study also used whole genome sequencing (WGS) for variant detection and evaluated the two approaches side-by-side and the potential benefit of integrating WGS and OGM to obtain a comprehensive analysis of genetic variation.
  • The study concluded that OGM together with WGS can potentially identify new therapeutic targets and improve personalized medicine in pediatric leukemia and other cancers by providing a more complete view of genome variation, including structural variation.
  • The study findings showed that, of the 3,075 total SVs detected in the B-ALL samples, 1,255 were uniquely detected by OGM.
  • The study also found that WGS detected 66 gene fusions, and that OGM was able to detect an additional 56 fusions that were missed by WGS.

Vincerx Pharma Presents Positive Preliminary Phase 1 Data for VIP236 and Updates on Pipeline Progress at the American Association for Cancer Research (AACR) Annual Meeting 2024 

Retrieved on: 
Monday, April 8, 2024
Sarah Cannon Research Institute, B-cell leukemia, AML, B-ALL, Patient, Webcast, Neoplasm, Acute myeloid leukemia, Cancer, Syndrome, Spacecraft, Camptothecin, AACR, DLT, PALO, Antibody, Drug development, Safety, Infrared spectroscopy correlation table, MDS, Therapy, ADC, Drug resistance, Pharmaceutical industry

PALO ALTO, Calif., April 08, 2024 (GLOBE NEWSWIRE) -- Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, today presented positive preliminary Phase 1 data for VIP236 and updates on pipeline progress at the American Association for Cancer Research (AACR) Annual Meeting 2024.

Key Points: 
  • We are still in dose escalation and are starting to see tumor reduction after only two doses.
  • The study's main objective is to determine a safe dose and schedule for VIP236 for further clinical development.
  • As the study progresses through the dose escalation, Vincerx will present additional Phase 1 data for VIP943 on or around the 2024 European Hematology Association Annual Meeting in June 2024.
  • An archived replay of the webcast will be available on the Vincerx Investor Page website following the conclusion of the live event.

Autolus Therapeutics announces acceptance of Marketing Authorization Application (MAA) by the European Medicines Agency (EMA) for obecabtagene autoleucel (obe-cel) for Patients with Relapsed/refractory (r/r) Adult B-Cell Acute Lymphoblastic Leukemia (B-AL

Retrieved on: 
Tuesday, April 2, 2024
Certification, MAA, B-ALL, Patient, Orphan, ALL, EMA, GMP, European Medicines Agency, MHRA, Therapy, Priority, Marketing, FDA, BLA, PDUFA, Pharmaceutical industry

Obe-cel is Autolus’ lead investigational chimeric antigen receptor (CAR) T cell therapy, for the treatment of patients with relapsed/refractory (r/r) adult B-cell Acute Lymphoblastic Leukemia (ALL).

Key Points: 
  • Obe-cel is Autolus’ lead investigational chimeric antigen receptor (CAR) T cell therapy, for the treatment of patients with relapsed/refractory (r/r) adult B-cell Acute Lymphoblastic Leukemia (ALL).
  • The MAA submission was based on data from the pivotal Phase 2 FELIX study of obe-cel in adult r/r B-ALL.
  • Autolus’ Nucleus site has recently received the formal certification from the MHRA following a full inspection of the site in February 2024.
  • The MHRA issued two new GMP certificates to cover both clinical and commercial manufacture from the site.

Autolus Therapeutics Reports Full Year 2023 Financial Results and Business Updates

Retrieved on: 
Thursday, March 14, 2024
Nucleus, Bone marrow, BCMA, BioNTech, B-ALL, Patient, ALL, SLE, Engineering, Minocycline, European Medicines Agency, Therapy, Research, Canadian Aviation Regulations, Flow cytometry, ASH, General counsel, Immunotherapy, SVP, EMA, Legal Affairs, Trial of the century, GMP, SME, BLA, Nature Communications, Development, UCL, Biologics license application, Multiple myeloma, MIA, University College London, Senior, TRBC, GD2, ASCO, FDA, CD19, Conference call, Conference, MAA, EHA, ADS, MHRA, T cell, ACS Chemical Biology, PDUFA, Autoimmune disease, Pharmaceutical industry

“For now, we remain fully focused on preparing for a potential obe-cel launch and successfully transitioning Autolus to a commercial stage company.

Key Points: 
  • “For now, we remain fully focused on preparing for a potential obe-cel launch and successfully transitioning Autolus to a commercial stage company.
  • In February 2024, Autolus promoted Dr. Chris Williams to Chief Business Officer and Alex Driggs to Senior Vice President, Legal Affairs and General Counsel.
  • Dr. Leiderman brings extensive transactional and financial expertise, and Mr. Azelby brings more than 30 years of biopharmaceutical leadership and commercial experience to Autolus’ Board.
  • Financial Results for the Year Ended December 31, 2023

Juventas Cell Therapy Announces the Approval of Its First Cell Therapy Product - Inaticabtagene Autoleucel by China NMPA

Retrieved on: 
Friday, November 10, 2023
Patient, Hope, Survival, Principal, Trial of the century, NDA, New Drug Application, Hematology, NMPA, Juventas, Toxicity, CD19, Diagnosis, Injection, B-ALL, Vaccine

Beijing, China and Tianjin, China and Shanghai, China--(Newsfile Corp. - November 10, 2023) - Juventas Cell Therapy Ltd. announces China National Medical Product Administration (NMPA) has approved its New Drug Application (NDA) for Inaticabtagene Autoleucel (CNCT19 Injection), a CD19-tarrgeted autologous chimeric antigen receptor (CAR)-T cell therapy for adult relapsed and refractory B-cell acute lymphoblastic leukemia (r/r B-ALL).

Key Points: 
  • The approval of Inaticabtagene Autoleucel by NMPA delivers a breakthrough clinical option adult r/r B-ALL patients.
  • Beijing, China and Tianjin, China and Shanghai, China--(Newsfile Corp. - November 10, 2023) - Juventas Cell Therapy Ltd. announces China National Medical Product Administration (NMPA) has approved its New Drug Application (NDA) for Inaticabtagene Autoleucel (CNCT19 Injection), a CD19-tarrgeted autologous chimeric antigen receptor (CAR)-T cell therapy for adult relapsed and refractory B-cell acute lymphoblastic leukemia (r/r B-ALL).
  • Inaticabtagene Autoleucel is a CD19 CAR-T cell therapy product, featured with a unique CD19 scFv (HI19a) and advanced manufacturing processes.
  • Inaticabtagene Autoleucel is the first proprietary CD19-targeted CAR-T product invented in China, and the first commercialized CAR-T cell therapy product for B-ALL in China.

Autolus Therapeutics Presents Clinical Data Updates at the American Society of Hematology (ASH) Annual Meeting 2023

Retrieved on: 
Sunday, December 10, 2023
Associate, Food, Bone marrow, MRD, FDA, Royal College of Pathologists, CRi, Cancer Institute, Multiple myeloma, BCMA, EMD, Doctor of Philosophy, Society, Biologics license application, Therapy, ASH, EFS, University College London, Toxicity, CRS, Webcast, Patient, CD19, Safety, ALL, B-ALL, UCL, BLA, Pharmaceutical industry

Finally, in an oral presentation pre-clinical and Phase I clinical data from AUTO8, a BCMA/CD19 co-targeting CAR T cell candidate, evaluated in patients with refractory multiple myeloma.

Key Points: 
  • Finally, in an oral presentation pre-clinical and Phase I clinical data from AUTO8, a BCMA/CD19 co-targeting CAR T cell candidate, evaluated in patients with refractory multiple myeloma.
  • “The FELIX study, with 127 patients, is one of the largest CAR T cell studies in adults with r/r B-ALL.
  • A high proportion of patients responded, with many responses sustained, particularly in patients with low or intermediate disease-burden at lymphodepletion.
  • Cellular kinetic data shows high expansion and long-term persistence of CAR T cells in most responders.

TScan Therapeutics Presents Initial Phase 1 Clinical Results on TSC-100 and TSC-101 at the 65th American Society of Hematology Annual Meeting and Exposition

Retrieved on: 
Saturday, December 9, 2023
Patient, Associate, Immunosuppression, Malignancy, Exposition, Acute leukemia, MRD, Prognosis, TCR, Haematopoietic system, City, Society, Therapy, MDS, Cancer, Infrared spectroscopy correlation table, Conditional sentence, ASH, HCT, Syndrome, Transplant, Acute myeloid leukemia, Safety, Cell, ALL, AML, B-ALL, KOL, Pharmaceutical industry

“We are excited to present initial clinical data in our heme program, with six patients in our treatment arms and four patients in our control arm.

Key Points: 
  • “We are excited to present initial clinical data in our heme program, with six patients in our treatment arms and four patients in our control arm.
  • In contrast, these indicators were not achieved in any of the four control-arm patients.
  • After establishing the recommended Phase 2 dose, we plan to open expansion cohorts at that dose to further characterize safety and evaluate translational and efficacy endpoints.
  • We have designed TSC-100 and TSC-101 to address this unmet need and increase the success rate of transplantation.

Adaptive Biotechnologies and Collaborators to Present More than 30 Abstracts Demonstrating the Actionability of clonoSEQ® MRD Testing in Blood Cancer Patient Care and Drug Development at the 65th ASH Annual Meeting

Retrieved on: 
Tuesday, December 5, 2023
Sequence, ASCT, Skylark (rocket), NGS, MRD, Relapse, Durability, Haematopoietic system, Rituximab, Bortezomib, Minimal residual disease, CLL, ATLAS, Allotransplantation, B-ALL

Updated Results of the Phase I BALLI-01 Trial of UCART22 Process 2 (P2), an Anti-CD22 Allogeneic CAR-T Cell Product Manufactured By Cellectis Biologics, in Patients with Relapsed or Refractory (R/R) CD22+ B-Cell Acute Lymphoblastic Leukemia (B-ALL)

Key Points: 
  • Updated Results of the Phase I BALLI-01 Trial of UCART22 Process 2 (P2), an Anti-CD22 Allogeneic CAR-T Cell Product Manufactured By Cellectis Biologics, in Patients with Relapsed or Refractory (R/R) CD22+ B-Cell Acute Lymphoblastic Leukemia (B-ALL)
    Undetectable MRD Status in Patients with R/R CLL/SLL with Stable Disease after Lisocabtagene Maraleucel Treatment: Exploratory Analysis of the TRANSCEND CLL 004 Study
    Fixed-Duration Pirtobrutinib Combined with Venetoclax ± Rituximab in Relapsed/Refractory Chronic Lymphocytic Leukemia: Updated Results, Including MRD Data, from the BRUIN Phase 1b Study
    Impact of Sequence Uniqueness on MRD Monitoring in NGS Immunoglobulin Sequencing: An Analysis of Ig Loci Among >1200 Diffuse Large B-Cell Lymphoma Patients Tested By ClonoSEQ
    Epcoritamab SC Monotherapy Leads to Deep and Durable Responses in Patients with Relapsed or Refractory Follicular Lymphoma: First Data Disclosure from the Epcore NHL-1 Follicular Lymphoma Dose-Expansion Cohort
    Early Peripheral Blood Minimal Residual Disease Status By NGS in Patients with Newly Diagnosed Multiple Myeloma (MM) on a Phase 2 Trial Receiving Elotuzumab, Carfilzomib, Lenalidomide, and Dexamethasone (Elo-KRd)
    Primary Endpoint Analysis from a Response Adaptive Phase II Clinical Trial of Carfilzomib, Lenalidomide, Dexamethasone Plus Daratumumab (KRd-Dara) in Patients with Newly Diagnosed Multiple Myeloma (NDMM)
    Sequential T-Cell Engagement for Myeloma (“STEM”) Trial: A Phase 2 Study of Cevostamab Consolidation Following BCMA CAR T Cell Therapy
    A Phase II Study of Isatuximab, Once Weekly Carfilzomib, Lenalidomide, Dexamethasone, in Newly Diagnosed, Transplant-Eligible Multiple Myeloma (The SKylaRk Trial)
    Longitudinal Assessment of Minimal Residual Disease (MRD) in the ATLAS Randomized Phase 3 Trial of Post-Transplant Treatment with Carfilzomib, Lenalidomide, and Dexamethasone (KRd) Versus Lenalidomide (R) Alone in Patients with Newly Diagnosed Multiple Myeloma (NDMM)
    Final Analysis of a Phase 2 Trial of Daratumumab, Carfilzomib, Lenalidomide, and Dexamethasone in Newly Diagnosed Multiple Myeloma (NDMM) without Autologous Stem Cell Transplantation (ASCT)
    Phase II Trial of Daratumumab, Bortezomib, Lenalidomide and Dexamethasone in High-Risk Smoldering Multiple Myeloma
    Update for the "Watch" Registry, a Real-World Observational Study Using clonoSEQ® to Monitor MRD in Lymphoid Malignancies

New Analyses Presented at ASH 2023 Support the Potential Long-Term Response and Safety of Kite’s Tecartus® in Patients With Aggressive Blood Cancers

Retrieved on: 
Tuesday, December 12, 2023
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, Hospitals, Clinical Trials, Hackensack University Medical Center, Safety, Cytokine release syndrome, Senior, City, ASH, Aes, CRS, Haematopoietic system, Sepsis, Patient, Abstract, Thrombocytopenia, Blinatumomab, The James Cancer Hospital, Division, SCT, Acute lymphoblastic leukemia, Neutropenia, ORR, Encephalopathy, B-ALL, Infection, Survival, Death, CD20, Doctor of Philosophy, Society, Abstract (summary), OS, Confidence interval, TP53, LD, Cytopenia, Pharmaceutical industry, Nursing, Hematology, Brexucabtagene autoleucel, Lymphoma, MD

“The clinical results and real-world evidence presented at ASH clearly support the potential for long-term response and safety of Tecartus in aggressive blood cancers for which patients have limited treatment options,” said Frank Neumann, MD, PhD, Senior Vice President, Global Head of Clinical Development, Kite.

Key Points: 
  • “The clinical results and real-world evidence presented at ASH clearly support the potential for long-term response and safety of Tecartus in aggressive blood cancers for which patients have limited treatment options,” said Frank Neumann, MD, PhD, Senior Vice President, Global Head of Clinical Development, Kite.
  • The median OS for patients with complete response (CR) (n=46) was 58.7 months.
  • Efficacy and safety outcomes for 23 patients with R/R MCL enrolled in ZUMA-18, a multicenter, open-label, expanded-access study of Tecartus, were also presented.
  • An analysis of a CIBMTR observational database of R/R MCL patients receiving Tecartus from 84 U.S. centers was presented.

Innate Pharma Shares Efficacy and Safety Phase 1 /2 Results of NK Cell Engager SAR443579 / IPH6101 Developed by Sanofi at ASH 2023

Retrieved on: 
Monday, December 11, 2023
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, FDA, Clinical Trials, Euronext Paris, Safety, Bone marrow, Acute myeloid leukemia, Sanofi, Patient, Fast track (FDA), Hematopoietic stem cell transplantation, IPH, Hematology, B-ALL, Cancer, Partnership, AML, American Society of Hematology, Acute lymphoblastic leukemia, Society, IPHA, CD123, NK, Pharmaceutical industry

SAR443579 has FDA Fast Track Designation for the treatment of acute myeloid leukemia.

Key Points: 
  • SAR443579 has FDA Fast Track Designation for the treatment of acute myeloid leukemia.
  • As of July 5, 2023, 43 patients (42 R/R AML and 1 HR-MDS) across 8 dose levels between 10 – 6000 μg/kg/dose were available for analysis.
  • “We believe this investigational NK cell engager may be important to patients with AML who, too often, have limited therapeutic options available to them.
  • Our updated results help drive Sanofi R&D teams around the world to continue efforts to develop impactful treatments for diseases with high unmet medical needs.”