Clinical Trial News
Catalyst Biosciences Announces Positive Factor IX Clinical Data
Catalyst Biosciences, Inc. has announced positive clinical data from the first Cohort of its ongoing Phase 1/2 proof-of-concept clinical trial for CB 2679d, a next-generation coagulation Factor IX variant, in individuals with severe hemophilia B. The data shows CB 2679d is approximately 22 times more potent than current hemophilia B therapy, with a longer circulation time in the body.
Phase 2 Trial Shows High Efficacy of BOVen Regimen in Treating Chronic Lymphocytic Leukaemia
A phase 2 trial investigating the BOVen regimen (zanubrutinib, obinutuzumab, and venetoclax) for treating chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL) has shown promising results, with 89% of patients achieving undetectable minimal residual disease (MRD) in both peripheral blood and bone marrow. The study highlights the regimen's safety and efficacy, suggesting a potential new standard for first-line therapy in CLL/SLL patients.
Highlighted Clinical Trials:
Memorial Sloan Kettering Cancer Center
Posted 2/22/2019
Atezolizumab, Bevacizumab, and GEMOX Combination Shows Promise in Advanced Biliary Tract Cancer
- Atezolizumab, bevacizumab, and GEMOX (gemcitabine plus oxaliplatin) demonstrated efficacy and safety in treating advanced biliary tract cancer (BTC).
- The combination therapy achieved a 76.7% overall response rate (ORR) and a 90.0% disease control rate (DCR) in a study of 30 patients.
- Median progression-free survival (PFS) was 12.0 months, and the median overall survival (OS) was not reached during the study period.
- Alterations in the ALS2CL gene may serve as a potential predictive biomarker for the efficacy of this triple combination therapy.
Strategic Approaches to Securing Market Access for Orphan Drugs: Beyond HTA Approval
- Clinician engagement and education are crucial for orphan drug adoption, requiring targeted materials that effectively communicate disease burden and clinical benefits.
- Real-world evidence collection through independent registries plays a vital role in demonstrating orphan drug effectiveness, despite manufacturer funding challenges.
- Patient support networks, including home nurse visits and online platforms, can enhance treatment outcomes while helping justify high orphan drug costs to payers.
Medical Affairs Evolution: Eisai's Kirk Shepard Leads Transformation Towards Patient-Centric Pharma
- Medical Affairs has evolved from a bridge between R&D and commercial teams to become central in driving patient-centric, data-driven pharmaceutical development and decision-making.
- Kirk Shepard, Eisai's SVP of Global Medical Affairs Oncology, spearheads the Medical Affairs Professional Society (MAPS) to establish global standards and elevate the profession's impact.
- Medical Science Liaisons serve as crucial field representatives, gathering real-world insights from healthcare providers and facilitating evidence-based discussions with stakeholders.
Alicaforsen Shows Promise in Treating Inflammatory Bowel Disease
Alicaforsen, an ICAM-1 inhibitor, has demonstrated effectiveness in treating ulcerative colitis and shows potential for pouchitis, with ongoing phase 3 trials. Its safety profile in UC patients is favorable, and it has received orphan designation for pouchitis treatment.
FDA Grants Full Approval to Blinatumomab for B-cell Precursor ALL
The FDA has fully approved blinatumomab (Blincyto) for treating adults and children with relapsed/refractory B-cell precursor acute lymphoblastic leukemia (ALL), regardless of Philadelphia chromosome status. This approval is based on data from the phase III TOWER study, showing improved overall survival rates compared to standard chemotherapy.
Patient-Centric Approaches and Technology Reshape Clinical Trial Landscape, Industry Experts Report
- Only 39% of clinical trial sites achieve enrollment targets according to Tufts Center, highlighting critical recruitment challenges in drug development.
- Industry leaders from INC Research, DrugDev, and EMD Serono emphasize the importance of non-traditional partnerships and patient-focused technology solutions to improve trial participation.
- Experts advocate for incorporating patient feedback through advisory boards and focus groups, while cautioning that technology solutions must be tailored to specific patient populations' needs.
Manali Kamdar's Contributions to Lymphoma Treatment Research
Dr. Manali Kamdar, Clinical Director of Lymphoma Services at the University of Colorado Cancer Center, focuses on novel therapies for non-Hodgkin lymphoma, participating in significant clinical trials like TRANSFORM and TRANSCEND-NHL-001.
Highlighted Clinical Trials:
Juno Therapeutics, a Subsidiary of Celgene
Posted 1/6/2016
Soleno Therapeutics Gains FDA Support for DCCR Trial Design in Prader-Willi Syndrome
- Soleno Therapeutics received positive feedback from the FDA regarding its Phase III trial design for Diazoxide Choline Controlled-Release (DCCR) in Prader-Willi Syndrome (PWS).
- The FDA supports using change in hyperphagia score as the primary endpoint, without requiring a change in weight, compared to placebo.
- The FDA agreed to a shorter 3-4 month randomized, double-blind, placebo-controlled study, with long-term safety data collected separately.
- Soleno plans to finalize the Phase III trial design, aiming to enroll approximately 100 patients, with study initiation expected by the end of 2017.