Clinical Trial News
Study of Adjuvant Cemiplimab Versus Placebo After Surgery for High-Risk Cutaneous Squamous Cell Carcinoma
A clinical trial is investigating the efficacy of adjuvant Cemiplimab compared to placebo in patients who have undergone surgery for high-risk cutaneous squamous cell carcinoma (CSCC). The study focuses on patients meeting specific inclusion criteria, such as age, disease status, and functional status, while excluding those with certain other malignancies or autoimmune diseases.
Sandoz Receives FDA Approval for First Denosumab Biosimilars in US and Canada
- Sandoz received FDA approval for Jubbonti and Wyost, the first and only denosumab biosimilars approved in the United States and Canada, marking a significant milestone in biosimilar development.
- The biosimilars are approved as interchangeable with Amgen's reference medicines Prolia and Xgeva for all indications, including osteoporosis treatment and prevention of cancer-related bone complications.
- More than 10 million US adults aged 50 and over live with osteoporosis, with half of all women over 50 experiencing an osteoporotic fracture during their lifetime.
- The approvals are based on robust phase I and phase III clinical studies demonstrating no clinically meaningful differences from the reference medicines.
Highlighted Clinical Trials:
Jazz Pharmaceuticals Completes FDA Submission for Zanidatamab in HER2-Positive Biliary Tract Cancer
- Jazz Pharmaceuticals has completed its Biologics License Application (BLA) submission to the FDA for zanidatamab to treat HER2-positive metastatic biliary tract cancer (BTC).
- The BLA is supported by Phase 2b HERIZON-BTC-01 trial data, which showed a 41.3% confirmed objective response rate in previously treated patients.
- Zanidatamab, a HER2-targeted bispecific antibody, could become the first HER2-targeted therapy specifically approved for BTC in the U.S.
- A Phase 3 trial (HERIZON-BTC-302) is underway to confirm zanidatamab's efficacy in first-line advanced or metastatic HER2-positive BTC.
Highlighted Clinical Trials:
Jazz Pharmaceuticals
Posted 7/19/2024
Study Highlights Preference for Bladder-Sparing Treatments in High-Risk NMIBC Patients
A recent study reveals a strong preference among high-risk non-muscle invasive bladder cancer (NMIBC) patients for bladder-sparing treatments over radical cystectomy, primarily due to concerns over quality of life and the desire for bladder preservation. The TAR-200 monotherapy, a novel drug delivery system, is being explored as a potential treatment option.
Highlighted Clinical Trials:
Janssen Research & Development, LLC
Posted 12/18/2020
FDA Accepts Datopotamab Deruxtecan BLA for HR-Positive, HER2-Negative Metastatic Breast Cancer
- The FDA has accepted the BLA for datopotamab deruxtecan for treating HR-positive, HER2-negative metastatic breast cancer after prior systemic therapy.
- The application is based on the TROPION-Breast01 Phase III trial, which showed improved progression-free survival compared to chemotherapy.
- Datopotamab deruxtecan is a TROP2-directed DXd antibody drug conjugate developed by Daiichi Sankyo and AstraZeneca.
- The FDA's decision is expected in the first quarter of 2025, with ongoing trials assessing overall survival and further regulatory submissions planned.
Eflornithine Approved for High-Risk Neuroblastoma: EMA Approval Still Pending
- Eflornithine (Iwilfin) is FDA-approved for high-risk neuroblastoma in adults and children who have shown a partial response to prior therapies, including anti-GD2 immunotherapy.
- Clinical trials demonstrated that eflornithine reduces the risk of relapse, with a hazard ratio for event-free survival of 0.48 and a hazard ratio for overall survival of 0.32.
- As of March 2024, there is no active application for eflornithine's EMA approval, making its availability in Europe unlikely in the near future.
- Patients in Europe may potentially access eflornithine through clinical trials or via Named Patient Import regulations, requiring a prescription from their doctor.
Highlighted Clinical Trials:
Giselle Sholler
Posted 3/5/2015
FDA Approves Besremi, First Drug for Polycythemia Vera
- The FDA has approved Besremi (ropeginterferon alfa-2b), an interferon drug by PharmaEssentia, as the first drug therapy specifically for polycythemia vera.
- Besremi, administered as a bi-weekly subcutaneous injection, aims to reduce blood cell production by attaching to interferon alpha receptors in the bone marrow.
- Clinical trials showed 61% of patients achieved complete hematological response, with manageable side effects detailed in a black box warning for potential severe risks.
- This approval marks a significant advancement in treating polycythemia vera, a rare blood cancer affecting approximately 6,200 Americans annually.
IAG933, a Novel YAP/TAZ-TEAD Inhibitor, Shows Promise in Preclinical Cancer Models
- IAG933 directly disrupts the YAP/TAZ-TEAD protein-protein interaction, leading to YAP eviction from chromatin and reduced Hippo-mediated transcription.
- In preclinical models, IAG933 demonstrates deep tumor regression in Hippo-driven mesothelioma xenografts and shows efficacy in combination with other inhibitors in lung, pancreatic, and colorectal cancer.
- The inhibitor exhibits potent antiproliferative activity in Hippo-dependent cell lines, particularly mesothelioma, and demonstrates a more rapid and profound reduction in cell viability compared to allosteric TEAD inhibitors.
- Clinical evaluation of IAG933 is currently underway, marking a significant step in exploring its potential as a therapeutic agent for various solid tumors.
Highlighted Clinical Trials:
Novartis Pharmaceuticals
Posted 10/21/2021
Ikena Oncology
Posted 1/7/2022
Vivace Therapeutics, Inc
Posted 3/24/2021
FDA Approves Pyrukynd as First Treatment for Pyruvate Kinase Deficiency
- The FDA has approved mitapivat (Pyrukynd) as the first treatment for adults with pyruvate kinase (PK) deficiency, a rare inherited anemia.
- Pyrukynd, a small molecule, works by activating PK enzymes to improve red blood cell function, offering a new approach to managing this condition.
- Clinical trials demonstrated that Pyrukynd significantly increased hemoglobin levels in non-transfusion-dependent patients and reduced transfusion burden in regularly transfused patients.
- Agios Pharmaceuticals, now focused on rare genetic diseases, estimates that many PK deficiency cases remain undiagnosed, highlighting the need for increased awareness and testing.
Sanofi and Denali's RIPK1 Inhibitor Fails in Phase 2 ALS Trial
- Sanofi and Denali Therapeutics' investigational drug SAR443820 (DNL788), a RIPK1 inhibitor, did not meet the primary endpoint in a Phase 2 clinical trial for amyotrophic lateral sclerosis (ALS).
- The trial assessed the change in the ALS Functional Rating Scale-Revised (ALSFRS-R) as the primary outcome, with Sanofi planning to present detailed efficacy and safety results at a future scientific meeting.
- Despite the setback in ALS, the Phase 2 study of SAR443820 in multiple sclerosis (MS) is ongoing, and a 52-week open-label extension study in ALS will further evaluate the drug's safety and efficacy.
- The failure raises questions about RIPK1 inhibition as a therapeutic strategy for ALS, impacting other companies like Eli Lilly, which has a partnership with Rigel Pharmaceuticals for RIPK1-blocking molecules.