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Clinical Trial News

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.

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.

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.

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.

Study of STRO-001, an Anti-CD74 Antibody Drug Conjugate, in Patients With Advanced B-Cell Malignancies

NCT03424603CompletedPhase 1
Sutro Biopharma, Inc.
Posted 2/22/2018

Navigating Latin American Pharma Markets: Challenges and Opportunities Amid Regulatory Evolution

• Latin American pharmaceutical markets face significant barriers including bureaucratic complexities, political instability, and currency devaluation, leading some companies to exit the region entirely.
• COVID-19 has exposed healthcare system vulnerabilities in Latin America, disrupting supply chains and highlighting the need for innovative solutions like telemedicine, particularly in underserved areas.
• Regional governments are increasingly adopting sophisticated regulatory frameworks modeled after European standards, though strict price controls continue to impact medicine accessibility and market dynamics.

mRNA Vaccines: How Moderna and Pfizer-BioNTech Revolutionized COVID-19 Immunization

  • Moderna and Pfizer-BioNTech developed mRNA-based COVID-19 vaccines with efficacy rates exceeding 90%, representing a breakthrough in vaccine technology during the pandemic.
  • Both vaccines use lipid nanoparticle technology to deliver mRNA instructions that teach cells to produce the coronavirus spike protein, triggering a protective immune response without causing infection.
  • The vaccines require different storage conditions, with Pfizer's needing ultra-cold temperatures of -94°F (-70°C) while Moderna's remains stable at -4°F (-20°C), affecting global distribution logistics.

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