Clinical Trial News
Landmark Clinical Trials of 2024: Promising Advances in Cancer, Malaria, and Genetic Diseases
- AstraZeneca's trastuzumab deruxtecan shows a 73.3% intracranial response rate in breast cancer patients with brain metastases, offering a potential new treatment option.
- Oxford University's malaria vaccine, R21/Matrix-M, demonstrates a 78% efficacy rate in young African children, marking a significant improvement over existing vaccines.
- Verve Therapeutics' VERVE-101/VERVE-102 utilizes in vivo base-editing to deactivate the PCSK9 gene mutation, potentially providing a one-time treatment for high cholesterol.
- A Netherlands Cancer Institute study suggests that neoadjuvant ipilimumab and nivolumab may eliminate the need for surgery in stage III melanoma patients, with a 72% positive response.
Norgine Seeks Approval for Eflornithine in High-Risk Neuroblastoma via Project Orbis
- Norgine B.V. has submitted marketing authorization applications for eflornithine in Australia, Switzerland, and the United Kingdom for high-risk neuroblastoma (HRNB).
- The submissions are part of Project Orbis, an initiative to expedite the approval of innovative oncology products across multiple countries.
- Eflornithine is intended as an oral maintenance therapy to reduce the risk of relapse in pediatric and adult patients with HRNB.
- FDA approved eflornithine in December 2023 based on trials showing improved event-free and overall survival compared to standard of care.
Highlighted Clinical Trials:
Giselle Sholler
Posted 3/5/2015
National Cancer Institute (NCI)
Posted 10/18/2001
AstraZeneca's Voydeya Approved for PNH, Merck Advances KRAS Inhibitor
- The FDA has approved AstraZeneca's Voydeya (danicopan) as an add-on therapy for extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH).
- Merck's oral KRAS G12C inhibitor candidate, MK-1084, has entered phase III development for first-line treatment of metastatic non-small cell lung cancer (NSCLC).
- Jazz Pharmaceuticals has completed filing a regulatory application for zanidatamab, targeting HER2-positive metastatic biliary tract cancer (BTC).
Viatris Expands Pipeline with Idorsia Collaboration, Focuses on Cardiovascular and Autoimmune Therapies
- Viatris collaborates with Idorsia Ltd., acquiring rights to selatogrel for acute myocardial infarction and cenerimod for systemic lupus erythematosus.
- Selatogrel is a self-administered medicine for heart attack patients, complementing Viatris' cardiovascular expertise.
- Cenerimod, a novel immunology asset, targets systemic lupus erythematosus and may expand to other autoimmune indications.
- Viatris' Q4 2023 earnings missed estimates, but key brands like Yupelri and Lipitor showed growth, and new generics contributed $107 million in revenue.
Aldeyra Therapeutics Plans Reproxalap NDA Resubmission for Dry Eye Disease Following FDA Feedback
- Aldeyra Therapeutics plans to resubmit its NDA for reproxalap to treat dry eye disease after addressing FDA concerns.
- A new dry eye chamber study will be initiated in H1 2024 to assess ocular discomfort as the primary endpoint.
- The company expects to resubmit the NDA in H2 2024, with a potential six-month review period by the FDA.
- Aldeyra's cash reserves of $142.8 million are projected to fund operations beyond 2026, supporting ongoing clinical development.
Pfizer's RSV Vaccine Abrysvo Shows Promise in High-Risk Adults Under 60
- Pfizer's RSV vaccine, Abrysvo, demonstrated a robust immune response in high-risk adults aged 18-59, mirroring the response seen in older adults already approved for the shot.
- The company plans to seek expanded approval for Abrysvo to include this younger, high-risk population, potentially broadening access to RSV protection.
- The trial included adults with conditions like asthma, diabetes, and COPD, representing a significant portion of the adult population at increased risk from RSV.
- While the immune response was positive, some experts suggest the clinical benefit may vary, with the greatest impact for immunocompromised individuals.
Vertex Initiates Rolling NDA Submission for Suzetrigine in Acute Pain Management
- Vertex Pharmaceuticals has begun a rolling NDA submission to the FDA for suzetrigine (VX-548) for moderate-to-severe acute pain, with completion expected in Q2 2024.
- Phase III trials demonstrated suzetrigine's efficacy in reducing pain intensity across various surgical and non-surgical pain conditions, supporting the NDA submission.
- Vertex is also advancing suzetrigine in neuropathic pain, with phase III trials planned for diabetic peripheral neuropathy (DPN) in the second half of 2024.
- Suzetrigine holds FDA Fast Track and Breakthrough Therapy designations for acute pain and DPN, highlighting its potential to address unmet needs in pain management.
Amivantamab Plus Lazertinib Shows Promise in EGFR-Mutated NSCLC
- The MARIPOSA trial demonstrated that amivantamab plus lazertinib significantly improved progression-free survival (PFS) compared to osimertinib in treatment-naïve EGFR-mutated NSCLC patients.
- The combination therapy reduced the risk of progression or death by 30% and improved median PFS by 7.1 months compared to osimertinib alone.
- While the combination regimen resulted in higher rates of adverse events, they were mostly grade 1 or 2, suggesting the regimen may be suitable for younger, fitter patients.
- Amivantamab plus lazertinib also showed efficacy in patients with brain metastases, offering a potential new first-line treatment option without chemotherapy.
Highlighted Clinical Trials:
Janssen Research & Development, LLC
Posted 10/13/2020
Yuhan Corporation
Posted 2/15/2017
Janssen Research & Development, LLC
Posted 10/16/2019
FDA Panel Endorses Minimal Residual Disease (MRD) as Endpoint for Accelerated Approval in Multiple Myeloma
- The FDA's Oncologic Drugs Advisory Committee (ODAC) voted 12-0 in favor of using minimal residual disease (MRD) negativity as an endpoint for accelerated approval of multiple myeloma treatments.
- Meta-analyses of patient-level data demonstrate a strong correlation between MRD negativity at 12 months and improved progression-free survival (PFS) and overall survival (OS) in newly diagnosed multiple myeloma patients.
- While supporting MRD as an endpoint for accelerated approval, experts emphasize the need for continued monitoring of PFS, OS, and safety to confirm long-term clinical efficacy.
- The FDA acknowledged limitations in the meta-analyses, including variations in treatment types, assay standardization, and patient populations, highlighting the need for vigilant oversight.
Highlighted Clinical Trials:
Janssen Research & Development, LLC
Posted 2/16/2015
Intergroupe Francophone du Myelome
Posted 9/1/2015
Millennium Pharmaceuticals, Inc.
Posted 4/29/2013
Janssen Research & Development, LLC
Posted 8/29/2016
Janssen Research & Development, LLC
Posted 11/23/2017
Amgen
Posted 7/8/2013
FDA Grants Breakthrough Device Designation to Roche's Elecsys pTau217 Assay for Alzheimer's Diagnosis
- The FDA has granted Breakthrough Device Designation to Roche's Elecsys pTau217 assay, a blood test aiding in identifying amyloid pathology in individuals.
- This test, developed with Eli Lilly, aims to provide timely and accurate Alzheimer's diagnoses, potentially mitigating the disease's impact and facilitating clinical trial participation.
- Elecsys pTau217 measures pTau217, a biomarker distinguishing Alzheimer's from other neurodegenerative disorders, showing strong performance in research settings.
- If approved, the assay could significantly improve access to early diagnosis, addressing the growing need for additional diagnostic tools in Alzheimer's disease management.