Clinical Trial News
COVID-19 Pandemic Reshapes Patient-Centric Clinical Trial Landscape in 2020
• The COVID-19 pandemic catalyzed unprecedented changes in clinical trials, with remote monitoring evolving from an optional feature to an essential requirement for study continuation.
• Clinical trial recruitment saw remarkable success during the pandemic, with studies like the RECOVERY Trial enrolling over 2,000 participants across 16 NHS sites in just three weeks.
• While the pandemic accelerated certain patient-centric practices, the urgency of COVID-19 research sometimes compromised patient engagement, revealing areas for improvement in the clinical trial ecosystem.
Palforzia: A New Hope for Children with Peanut Allergy
Palforzia, a treatment for peanut allergy in children aged 1 to 17, has shown promising results in clinical trials, enabling some patients to tolerate small amounts of peanut protein with only mild symptoms. The treatment involves a phased approach under medical supervision and requires a prescription. Despite potential side effects, the European Medicines Agency has concluded that the benefits of Palforzia outweigh its risks.
Interferon-Alpha Shows Promise in Reducing Myelofibrosis Risk and Mortality in Polycythemia Vera Patients
A recent study presented at the 2020 ASH Annual Meeting reveals that Interferon-alpha (IFN) significantly reduces the risk of myelofibrosis and improves overall survival in patients with polycythemia vera (PV), suggesting its potential as a long-term management strategy for both low- and high-risk PV patients.
SL-401 Shows Promise in Advanced Myeloproliferative Neoplasms, Including CMML
• SL-401, a targeted therapy directed at the interleukin-3 receptor (CD123), demonstrates encouraging activity in advanced, high-risk myeloproliferative neoplasms (MPN).
• The Phase II trial of SL-401 includes patients with chronic myelomonocytic leukemia (CMML), showing initial dosing is well-tolerated with no unexpected toxicities.
• Researchers aim to improve blood counts, reduce transfusion dependency, alleviate symptoms, and achieve overall and complete responses with SL-401 treatment.
• CD123, the target of SL-401, is overexpressed in MPN cells and microenvironmental immune cells, specifically plasmacytoid dendritic cells (pDCs), in the bone marrow.
SGLT2 Inhibitors Show Promising Results for Elderly with Type 2 Diabetes
A recent study utilizing the Korean National Health Insurance Service database compared the effectiveness and safety of SGLT2 inhibitors versus DPP-4 inhibitors in older adults with type 2 diabetes. Findings suggest SGLT2 inhibitors significantly reduce the risk of heart failure hospitalization, all-cause death, and stroke, despite higher risks of genital and urinary tract infections. The study highlights the need for careful consideration in prescribing SGLT2 inhibitors to very elderly patients due to diminished cardiovascular benefits.
COVID-19 Clinical Trial Landscape Analyzed for Gaps in Risk Factor Consideration and Older Adult Representation
• A comprehensive analysis of COVID-19 clinical trials reveals potential gaps in the consideration of underlying health conditions that may lead to severe illness.
• The study identifies exclusion criteria in COVID-19 trials that may implicitly exclude older adults, raising concerns about the generalizability of findings.
• Researchers systematically summarized various aspects of COVID-19 clinical studies to inform future trial designs and strategies for rapid pandemic response.
• The analysis leverages natural language processing to assess eligibility criteria and risk factor consideration in registered COVID-19 clinical studies.
Enhertu Nears EU Approval for HER2-Positive Breast Cancer Treatment
• Daiichi Sankyo and AstraZeneca's antibody-drug conjugate Enhertu received CHMP recommendation for HER2-positive metastatic breast cancer treatment in patients previously treated with anti-HER2 therapies.
• The drug has demonstrated significant efficacy in clinical trials, with tumor shrinkage observed in 61% of patients in the DESTINY-Breast01 trial, positioning it as the leading treatment in third and fourth-line settings.
• Enhertu's potential market reach extends beyond breast cancer to HER2-positive gastric and lung cancers, with projected peak sales of $4.5 billion.
Scottish Health Authority Rejects Daiichi Sankyo's Novel Cholesterol Drug Nilemdo
• The Scottish Medicines Consortium has declined to recommend Daiichi Sankyo's Nilemdo (bempedoic acid) for NHS Scotland, citing insufficient robustness in clinical and economic analyses.
• Nilemdo, approved by EMA and FDA for patients with high cholesterol who can't tolerate statins, launched in Germany with UK rollout planned for early next year.
• The rejection could impact the drug's projected $3 billion peak sales potential, especially if other health technology assessment agencies follow Scotland's decision.
uniQure's Etranacogene Dezaparvovec Shows Sustained Therapeutic Benefit in Hemophilia B Patients
• Two-year follow-up data from a Phase IIb study shows that etranacogene dezaparvovec maintains stable Factor IX (FIX) activity at therapeutic levels in hemophilia B patients.
• The gene therapy demonstrated a mean FIX activity of 44.2% of normal at two years post-administration, with reduced bleeding rates and FIX replacement therapy usage.
• Long-term data from a Phase I/II study of AMT-060, uniQure's first-generation gene therapy, shows sustained clinical benefits up to five years, including increased FIX activity.
• Regulatory submissions to the FDA and EMA for etranacogene dezaparvovec are anticipated to begin in the second half of 2021, incorporating data from the ongoing Phase III HOPE-B trial.
STRO-001 Shows Promise in Relapsed/Refractory B-Cell NHL
• STRO-001, a novel CD74-targeting antibody-drug conjugate (ADC), demonstrates preliminary anti-tumor activity in heavily pre-treated B-cell non-Hodgkin's lymphoma (NHL) patients.
• The Phase 1 study of STRO-001 shows it is well-tolerated, with mostly grade 1 or 2 treatment-emergent adverse events and no observed ocular or neuropathy toxicity.
• A complete response was observed in a DLBCL patient at the 0.075 mg/kg dose, and partial responses were seen in DLBCL patients at 0.65 mg/kg and 1.27 mg/kg.
• The dose escalation study is ongoing, with plans to enroll patients at higher dose levels of STRO-001 to further evaluate safety and efficacy.
Highlighted Clinical Trials:
Sutro Biopharma, Inc.
Posted 2/22/2018