MedPath

Tagged News

FDA Grants Orphan Drug Designation to Tigilanol Tiglate for Soft Tissue Sarcoma Treatment

Rare DiseaseOncology
  • The FDA has awarded orphan drug designation to tigilanol tiglate (Stelfonta), an intratumoral injection, for treating soft tissue sarcoma, a rare cancer affecting both adults and children.
  • The designation provides QBiotics with important regulatory incentives including tax credits, user fee exemptions, and potential seven years of market exclusivity following approval.
  • Tigilanol tiglate is currently being evaluated in a phase 2 trial at Memorial Sloan Kettering Cancer Center, with preliminary phase 1 data showing responses in 6 of 22 patients.
  • Soft tissue sarcomas comprise more than 80 subtypes with unfavorable prognosis in advanced stages, highlighting the urgent need for new treatment options in this rare disease area.

A Clinical Study to Investigate the Efficacy of Intratumoral Tigilanol Tiglate in Soft Tissue Sarcoma

NCT05755113RecruitingPhase 2
QBiotics Group Limited
Posted 4/13/2023

ADI-PEG20 Quadruples Three-Year Survival in Phase 3 Mesothelioma Trial

OncologyRare Disease
  • A phase 3 trial of ADI-PEG20 combined with chemotherapy increased median survival by 1.6 months and quadrupled three-year survival rates in 249 patients with pleural mesothelioma.
  • The drug works by depleting arginine levels in the bloodstream, starving tumor cells that cannot manufacture their own arginine due to missing ASS1 protein.
  • This represents the first successful combination therapy breakthrough for mesothelioma in 20 years, offering new hope for patients with one of the world's worst cancer survival rates.
  • The international ATOMIC-meso trial was conducted across 43 centers in five countries between 2017 and 2021, demonstrating the treatment was well tolerated with no new safety signals.

Bispecific Antibodies: Promising Advances Amid Adoption Challenges in Cancer Treatment

Drug ApprovalOncologyCAR-TMonoclonal Antibody
  • Bispecific antibodies represent a significant advancement in cancer immunotherapy, targeting both tumor antigens and immune cells to enhance cytotoxicity without requiring patient-derived cells like CAR-T therapy.
  • Despite clinical promise with nine FDA-approved bispecific antibodies, adoption faces challenges including transition between inpatient/outpatient settings, insurance coverage, adverse event management, and financial barriers in community settings.
  • Recent approvals of Mosunetuzumab, Glofitamab, and Epcoritamab have shown impressive response rates in relapsed/refractory indolent B-cell lymphomas, with manageable toxicity profiles when using step-up dosing strategies.

Signatures of Response and Resistance to Mosunetuzomab in Follicular Lymphomas (FL)

NCT05529524Completed
The Lymphoma Academic Research Organisation
Posted 11/7/2022

CAR-T Followed by Bispecific Antibodies

NCT04889716RecruitingPhase 2
Abramson Cancer Center at Penn Medicine
Posted 11/5/2021

Tazemetostat and Mosunetuzumab in Untreated Follicular Lymphoma

NCT05994235RecruitingPhase 2
Weill Medical College of Cornell University
Posted 11/1/2023

A Study Evaluating the Efficacy and Safety of Mosunetuzumab in Combination With Lenalidomide in Comparison to Rituximab in Combination With Lenalidomide With a US Extension of Mosunetuzumab in Combination With Lenalidomide in Participants With Follicular Lymphoma

NCT04712097Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 10/27/2021

Mosunetuzumab With or Without Polatuzumab Vedotin and Obinutuzumab for the Treatment of Untreated Indolent B-Cell Non-Hodgkin Lymphoma

NCT05169658CompletedPhase 2
University of Washington
Posted 3/23/2022

Study of Subcutaneous Epcoritamab in Combination With Intravenous Rituximab and Oral Lenalidomide (R2) to Assess Adverse Events and Change in Disease Activity in Adult Participants With Follicular Lymphoma

NCT05409066Active, Not RecruitingPhase 3
Genmab
Posted 9/20/2022

Glofit and Obin in Follicular Lymphoma and Marginal Zone Lymphoma

NCT05783596RecruitingPhase 2
Reid Merryman, MD
Posted 7/18/2023

Mosunetuzumab-Lenalidomide Versus Investigator Choices in Patients With Relapsed or Refractory Marginal Zone Lymphoma

NCT06006117RecruitingPhase 3
The Lymphoma Academic Research Organisation
Posted 9/5/2023

Safety and Efficacy Trial of Epcoritamab Combinations in Subjects With B-cell Non-Hodgkin Lymphoma (B-NHL)

NCT04663347Active, Not RecruitingPhase 1
Genmab
Posted 11/3/2020

Clarity's 64Cu-SAR-bisPSMA Shows Promise in Detecting Prostate Cancer Recurrence in COBRA Trial

Oncology
  • Initial results from Clarity Pharmaceuticals' Phase 1/2 COBRA trial demonstrate that 64Cu-SAR-bisPSMA safely and effectively detects prostate cancer lesions in patients with biochemical recurrence who had negative standard-of-care imaging.
  • The novel radiopharmaceutical identified lesions in up to 80% of patients on next-day imaging, with the number of detected lesions almost doubling compared to same-day imaging, highlighting a unique benefit of copper-64's longer half-life.
  • Clinicians reported they would change treatment plans for approximately 50% of patients based on 64Cu-SAR-bisPSMA scan results, with two-thirds of these patients subsequently receiving focal or systemic therapy.

64Cu-SAR-bisPSMA for Identification of Participants With Recurrence of Prostate Cancer (COBRA)

NCT05249127CompletedPhase 1
Clarity Pharmaceuticals Ltd
Posted 4/11/2022

Positron Emission Tomography (PET) Imaging of Participants With Confirmed Prostate Cancer Using 64Cu-SAR-bisPSMA (PROPELLER)

NCT04839367CompletedPhase 1
Clarity Pharmaceuticals Ltd
Posted 7/13/2021

64Cu-SAR-bisPSMA Positron Emission Tomography: A Phase 3 Study of Participants With Biochemical Recurrence of Prostate Cancer

NCT06970847RecruitingPhase 3
Clarity Pharmaceuticals Ltd
Posted 5/16/2025

Positron Emission Tomography Using 64Cu-SAR-bisPSMA in Participants With High-risk Prostate Cancer Prior to Radical Prostatectomy

NCT06056830RecruitingPhase 3
Clarity Pharmaceuticals Ltd
Posted 12/21/2023

Study on Olaparib Plus Abiraterone as First-line Therapy in Men With Metastatic Castration-resistant Prostate Cancer

NCT03732820Active, Not RecruitingPhase 3
AstraZeneca
Posted 10/31/2018

Kisqali Shows Enhanced Efficacy in Younger Breast Cancer Patients with 33% Risk Reduction

Oncology
  • Novartis' NATALEE trial subgroup analysis reveals Kisqali plus endocrine therapy reduced invasive disease risk by 33% in pre-menopausal early breast cancer patients at 44.2 months median follow-up.
  • Pre-menopausal and younger patients experienced greater risk reductions and fewer treatment discontinuations due to adverse events compared to post-menopausal patients.
  • A separate real-world analysis highlighted significant treatment disparities, showing Black patients with early breast cancer had worse outcomes despite meeting NATALEE eligibility criteria.
  • The findings underscore the importance of CDK4/6 inhibitor therapy for vulnerable populations, including younger and Black patients who face more aggressive disease characteristics.

A Trial to Evaluate Efficacy and Safety of Ribociclib With Endocrine Therapy as Adjuvant Treatment in Patients With HR+/HER2- Early Breast Cancer

NCT03701334Active, Not RecruitingPhase 3
Novartis Pharmaceuticals
Posted 12/7/2018

Dostarlimab Shows Superior Survival Outcomes Across Multiple Endometrial Cancer Molecular Subgroups in Phase 3 RUBY Trial

Oncology
  • The phase 3 RUBY trial demonstrated that dostarlimab combined with carboplatin and paclitaxel significantly improved progression-free survival and overall survival in patients with advanced or recurrent endometrial cancer across multiple molecular subgroups.
  • Patients with dMMR/MSI-H tumors showed the most dramatic benefit, with progression-free survival rates of 57.0% versus 10.2% in the placebo arm and overall survival rates of 83.3% versus 58.7%.
  • The TP53 mutation subgroup also demonstrated significant clinical benefit, with progression-free survival rates of 32.4% versus 17.8% and overall survival rates of 70.8% versus 33.2% in the placebo arm.
  • These findings support dostarlimab plus carboplatin/paclitaxel as a new standard of care for patients with newly diagnosed primary advanced or recurrent endometrial cancer.

Study of TSR-042, an Anti-programmed Cell Death-1 Receptor (PD-1) Monoclonal Antibody, in Participants With Advanced Solid Tumors

NCT02715284RecruitingPhase 1
Tesaro, Inc.
Posted 3/7/2016

A Study to Evaluate Dostarlimab Plus Carboplatin-paclitaxel Versus Placebo Plus Carboplatin-paclitaxel in Participants With Recurrent or Primary Advanced Endometrial Cancer

NCT03981796Active, Not RecruitingPhase 3
Tesaro, Inc.
Posted 7/15/2019

Merck and Daiichi Sankyo Form $22 Billion Alliance for Three Novel Antibody-Drug Conjugates

OncologyTargeted TherapyPrecision Medicine
• Merck will pay Daiichi Sankyo $4 billion upfront plus $1.5 billion in continuation payments, with potential additional milestone payments reaching a total of $22 billion.
• The collaboration focuses on three investigational antibody-drug conjugates: patritumab deruxtecan (HER3-DXd), ifinatamab deruxtecan (I-DXd), and raludotatug deruxtecan (R-DXd), targeting multiple solid tumors.
• Patritumab deruxtecan has received Breakthrough Therapy Designation for EGFR-mutated non-small cell lung cancer, with a biologics license application planned by March 2024.

HERTHENA-Lung01: Patritumab Deruxtecan in Subjects With Metastatic or Locally Advanced EGFR-mutated Non-Small Cell Lung Cancer

NCT04619004Active, Not RecruitingPhase 2
Daiichi Sankyo
Posted 2/2/2021

A Study of DS-6000a in Subjects With Advanced Renal Cell Carcinoma and Ovarian Tumors

NCT04707248Active, Not RecruitingPhase 1
Daiichi Sankyo
Posted 12/22/2020

Ifinatamab Deruxtecan (I-DXd) in Subjects With Pretreated Extensive-Stage Small Cell Lung Cancer (ES-SCLC)

NCT05280470Active, Not RecruitingPhase 2
Daiichi Sankyo
Posted 3/9/2022

Eli Lilly Acquires Point Biopharma for $1.4 Billion to Expand Radioligand Therapy Portfolio

Oncology
  • Eli Lilly announced the acquisition of Point Biopharma for $1.4 billion, paying $12.50 per share representing a 67% premium to strengthen its oncology pipeline with radioligand therapies.
  • The deal provides Lilly access to Point's lead asset PNT2002, a PSMA-targeted radioligand therapy in Phase III trials for metastatic castration-resistant prostate cancer with data expected in late 2023.
  • Point's pipeline includes PNT2003 for gastroenteropancreatic neuroendocrine tumors and earlier-stage assets, positioning Lilly to compete with established players like Novartis in the radioligand therapy space.
  • The acquisition represents Lilly's strategic entry into radiopharmaceuticals, which deliver targeted radiation directly to cancer cells while minimizing damage to healthy tissue.

Novel Targets and Strategic Sequencing: The Evolving Landscape of T-Cell Redirection Therapies in Multiple Myeloma

OncologyCAR-T
  • CAR T-cell therapies and bispecific antibodies targeting BCMA and GPRC5D are transforming multiple myeloma treatment, with ciltacabtagene autoleucel showing unprecedented efficacy with a median PFS of 34.9 months in heavily pretreated patients.
  • Disease progression rate is a critical factor in therapy selection, with rapidly progressing patients better suited for "off-the-shelf" bispecific antibodies, while patients with indolent disease may benefit more from CAR T-cell therapies despite longer manufacturing times.
  • Strategic sequencing of therapies is crucial, as prior BCMA-directed bispecific antibody treatment significantly reduces the efficacy of subsequent BCMA-targeted CAR T-cell therapy, highlighting the importance of preserving T-cell fitness and considering antigen loss.

A Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-cell Maturation Antigen (BCMA) in Participants With Multiple Myeloma

NCT04133636RecruitingPhase 2
Janssen Research & Development, LLC
Posted 11/7/2019

A Study of the Combination of Talquetamab and Teclistamab in Participants With Relapsed or Refractory Multiple Myeloma

NCT04586426Active, Not RecruitingPhase 1
Janssen Research & Development, LLC
Posted 12/15/2020

A Study of Talquetamab in Participants With Relapsed or Refractory Multiple Myeloma

NCT04634552RecruitingPhase 2
Janssen Research & Development, LLC
Posted 2/1/2021

A Study of Teclistamab in Participants With Relapsed or Refractory Multiple Myeloma

NCT04557098Active, Not RecruitingPhase 2
Janssen Research & Development, LLC
Posted 9/17/2020

Safety and Efficacy of Anti-BCMA/GPRC5D CAR-T Cell Therapy in Treating Relapsed and Refractory Multiple Myeloma(rr/MM)

NCT05509530RecruitingPhase 2
Xuzhou Medical University
Posted 5/1/2022

A Study of CAR-T Cells Targeting GPRC5D in the Treatment of r/r Multiple Myeloma

NCT05016778Active, Not RecruitingEarly Phase 1
Zhejiang University
Posted 6/8/2021

MCARH109 Chimeric Antigen Receptor (CAR) Modified T Cells for the Treatment of Multiple Myeloma

NCT04555551Active, Not RecruitingPhase 1
Memorial Sloan Kettering Cancer Center
Posted 9/8/2020

A Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-Cell Maturation Antigen (BCMA) in Participants With Relapsed or Refractory Multiple Myeloma

NCT03548207CompletedPhase 1
Janssen Research & Development, LLC
Posted 6/29/2018

A Study of CAR-T Cells Targeting Both BCMA and GPRC5D in Treatment of Relapsed or Refractory Multiple Myeloma

NCT05325801Not Yet RecruitingPhase 1
Zhejiang University
Posted 4/1/2022

A Study Comparing JNJ-68284528, a CAR-T Therapy Directed Against B-cell Maturation Antigen (BCMA), Versus Pomalidomide, Bortezomib and Dexamethasone (PVd) or Daratumumab, Pomalidomide and Dexamethasone (DPd) in Participants With Relapsed and Lenalidomide-Refractory Multiple Myeloma

NCT04181827Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 6/12/2020

MagnetisMM-3: Study Of Elranatamab (PF-06863135) Monotherapy in Participants With Multiple Myeloma Who Are Refractory to at Least One PI, One IMiD and One Anti-CD38 mAb

NCT04649359Active, Not RecruitingPhase 2
Pfizer
Posted 2/2/2021

A Study of Bortezomib, Lenalidomide and Dexamethasone (VRd) Followed by Cilta-cel, a CAR-T Therapy Directed Against BCMA Versus VRd Followed by Lenalidomide and Dexamethasone (Rd) Therapy in Participants With Newly Diagnosed Multiple Myeloma for Whom ASCT is Not Planned as Initial Therapy

NCT04923893Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 8/19/2021

A Study of MCARH109 and MCARH125 in People With Multiple Myeloma

NCT05431608Active, Not RecruitingPhase 1
Memorial Sloan Kettering Cancer Center
Posted 6/20/2022

Efficacy and Safety Study of bb2121 in Subjects With Relapsed and Refractory Multiple Myeloma

NCT03361748CompletedPhase 2
Celgene
Posted 12/13/2017

Safety and Efficacy of ALLO-715 BCMA Allogenic CAR T Cells in in Adults With Relapsed or Refractory Multiple Myeloma (UNIVERSAL)

NCT04093596Active, Not RecruitingPhase 1
Allogene Therapeutics
Posted 9/23/2019

Dose Escalation Study of Talquetamab in Participants With Relapsed or Refractory Multiple Myeloma

NCT03399799Active, Not RecruitingPhase 1
Janssen Research & Development, LLC
Posted 12/16/2017

A Study of Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) Followed by Ciltacabtagene Autoleucel Versus Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) Followed by Autologous Stem Cell Transplant (ASCT) in Participants With Newly Diagnosed Multiple Myeloma

NCT05257083RecruitingPhase 3
Stichting European Myeloma Network
Posted 10/10/2023

Efficacy and Safety Study of bb2121 Versus Standard Regimens in Subjects With Relapsed and Refractory Multiple Myeloma (RRMM)

NCT03651128Active, Not RecruitingPhase 3
Celgene
Posted 4/16/2019

Bispecific T-Cell Engagers Show Promise as Later-Line Therapies for Relapsed/Refractory DLBCL

CAR-TDrug ApprovalOncology
  • Two bispecific T-cell engagers, epcoritamab-bysp and glofitamab-gxbm, received FDA accelerated approval in 2023 for relapsed/refractory diffuse large B-cell lymphoma, offering new treatment options for heavily pretreated patients.
  • Both agents demonstrate remarkable activity with overall response rates above 50-60% and complete response rates around 40% in patients with CAR T-cell therapy failure, addressing a significant unmet medical need.
  • Epcoritamab offers convenient subcutaneous administration in outpatient settings with manageable cytokine release syndrome rates below 3%, while glofitamab provides fixed-duration therapy with durable responses lasting over one year after treatment completion.
  • These bispecific antibodies may challenge the paradigm that CAR T-cell therapy is the only curative option for relapsed/refractory patients, with emerging data suggesting potential for long-term disease control.

A Dose Escalation Study of Glofitamab (RO7082859) as a Single Agent and in Combination With Obinutuzumab, Administered After a Fixed, Single Pre-treatment Dose of Obinutuzumab in Participants With Relapsed/Refractory B-cell Non-hodgkin's Lymphoma

NCT03075696RecruitingPhase 1
Hoffmann-La Roche
Posted 2/21/2017

First-in-Human (FIH) Trial in Patients With Relapsed, Progressive or Refractory B-Cell Lymphoma

NCT03625037Active, Not RecruitingPhase 1
Genmab
Posted 6/26/2018

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.