April 25th 2025
Xofluza (baloxavir marboxil) met its primary endpoint in the CENTERSTONE trial, reducing the odds of untreated household members contracting influenza virus by 32%.
In the Phase III SELECT-GCA trial, Rinvoq (upadacitinib) 15 mg with a 26-week glucocorticoid taper significantly improved sustained remission rates and reduced disease flares in patients with giant-cell arteritis, offering a promising glucocorticoid-sparing treatment option with a favorable safety profile.
Tolebrutinib did not show superiority over Aubagio (teriflunomide) in reducing relapse rates or MRI-detected inflammatory activity in relapsing multiple sclerosis, but early signals suggest it may have potential in slowing disability progression, warranting further long-term and comparative studies.
In the Phase III DESTINY-Breast09 trial, Enhertu (trastuzumab deruxtecan) and Perjeta (pertuzumab) significantly improved progression-free survival compared to the current standard of care in the first-line treatment of HER2-positive metastatic breast cancer, suggesting a potential new standard of care.
Leading the Technology and Innovation Front Through Rare Disease Research
April 18th 2025In this video interview, Derek Ansel, vice president, therapeutic strategy lead, rare disease, Worldwide Clinical Trials, discusses how artificial intelligence can aid in bringing therapies to rare disease patients faster.
FDA-Approved Gene Therapy Beqvez Shows Sustained Efficacy, Safety in Long-Term Hemophilia B Trial
Beqvez (fidanacogene elaparvovec), an FDA-approved one-time gene therapy for hemophilia B, demonstrated sustained factor IX expression, low bleeding rates, and a favorable safety profile over long-term follow-up.
Improving Relationships and Diversifying the Site Selection Process
April 17th 2025In this episode of the Applied Clinical Trials Podcast, Liz Beatty, co-founder and chief strategy officer, Inato, discusses a number of topics around site engagement including community-based sites, the role of technology in improving site/sponsor relationships, how increased operational costs are impacting the industry, and more.
Adding Farxiga (dapagliflozin) to standard care significantly reduced the risk of death or worsening heart failure in older patients with severe aortic stenosis following transcatheter aortic-valve implantation, with consistent benefits across patient subgroups and a favorable safety profile.
2025 Rally for Rare: Bringing the Rare Disease Community Together
April 16th 2025In this video interview, Derek Ansel, vice president, therapeutic strategy lead, rare disease, Worldwide Clinical Trials, highlights the Rally for Rare event and how it succeeded in providing the community a space for collaboration and innovation.
Unique Challenges in Rare Disease Research
April 14th 2025In this video interview, Derek Ansel, vice president, therapeutic strategy lead, rare disease, Worldwide Clinical Trials, discusses the most prominent challenges he is seeing in the space right now including competing research priorities and finding endpoints.
In the Phase III EXPECTS trial, patients experiencing mostly mild posterior circulation ischemic stroke who were ineligible for thrombectomy showed significantly better functional recovery at 90 days when treated with alteplase between 4.5 and 24 hours after stroke onset, without an increased risk of symptomatic intracranial hemorrhage.