MedPath

Clinical Trial News

India's Nafithromycin Offers New Hope Against Drug-Resistant Pneumonia

  • Nafithromycin, India's first indigenous antibiotic, targets drug-resistant community-acquired bacterial pneumonia (CABP) and offers a new treatment option.
  • Developed by BIRAC and Wockhardt, Nafithromycin boasts a three-day treatment regimen and is reportedly ten times more effective than azithromycin.
  • Clinical trials show a 96.7% cure rate with superior safety, minimal side effects, and broad-spectrum action against typical and atypical bacteria.
  • Awaiting final approval, Nafithromycin may be integrated into India's Ayushman Bharat scheme for increased accessibility and affordability.

GSK's Linerixibat Shows Promise for Cholestatic Pruritus in Phase III Trial

  • GSK's linerixibat met the primary endpoint in the Phase III GLISTEN study, demonstrating a statistically significant improvement in itch score for PBC patients.
  • The GLISTEN study evaluated linerixibat, a targeted inhibitor of the ileal bile acid transporter (IBAT), over a 24-week period compared to placebo.
  • Linerixibat has the potential to be the first therapy indicated for treating itch associated with PBC globally, addressing a significant unmet need.
  • Other companies like Gilead and Mirum are also developing treatments for PBC, reflecting the high unmet medical need in this rare autoimmune disease.

CAR-T Therapy Shows Promise in Refractory Autoimmune Diseases

  • Early clinical studies, including the CASTLE basket study, demonstrate the potential of CD19-directed CAR-T therapy in treating severe, refractory autoimmune diseases.
  • Favorable safety profiles and preliminary efficacy were observed in patients with systemic lupus erythematosus, systemic sclerosis, and myositis.
  • A faster manufacturing process for CAR-T therapies, reducing production time to 5 days, could enhance accessibility and reduce treatment costs.
  • Many patients experienced drug-free remission for an extended period, suggesting a potential for long-term disease control, though relapses can occur.

University Hospitals First in Northeast Ohio to Offer Donanemab for Alzheimer's

• University Hospitals is the first health system in Northeast Ohio to offer donanemab (Kisunla™), an FDA-approved treatment for Alzheimer's disease. • Donanemab targets and removes beta-amyloid plaques in the brain, potentially slowing cognitive and functional decline in early-stage Alzheimer's. • The treatment involves monthly IV infusions, with the duration varying from six to eighteen months based on patient response and monitoring for side effects. • Comprehensive evaluation, including medical history, cognitive tests, and brain imaging, is required to determine patient eligibility for donanemab therapy.

City of Hope Revolutionizes Cancer Care Through Patient-Centered Approach and Expanded Access

  • City of Hope is transforming cancer treatment by moving beyond traditional drug-focused approaches to precision medicine, reaching 7 million members through AccessHope and expanding to multiple locations across the United States.
  • The institution is addressing healthcare disparities by combating ageism in blood cancer treatment and implementing comprehensive geriatric assessments to ensure older patients receive appropriate care.
  • Through a $100 million gift, City of Hope launched the Cherng Family Center for Integrative Oncology, combining evidence-based Eastern and Western approaches to create new standards in supportive cancer care.

FDA Grants Accelerated Approval to Ziihera for HER2-Positive Biliary Tract Cancer

• The FDA has granted accelerated approval to Ziihera (zanidatamab) for adults with previously treated, unresectable, or metastatic HER2-positive biliary tract cancer (BTC). • Approval was based on a 52% objective response rate and a median duration of response of 14.9 months in the HERIZON-BTC-01 trial. • Continued approval may depend on verification of clinical benefit in the ongoing Phase 3 HERIZON-BTC-302 confirmatory trial. • Ziihera, developed by Jazz Pharmaceuticals, represents a new treatment option for patients with limited alternatives.

Greater Bay Area International Clinical Trial Institute Opens in Hong Kong

  • The Greater Bay Area International Clinical Trial Institute (GBAICTI) has officially opened in Hong Kong, aiming to boost biomedical technology development.
  • The GBAICTI will coordinate clinical trial resources across public and private sectors, streamlining workflows and fostering collaboration within the Greater Bay Area.
  • The institute plans to establish a Hong Kong Clinical Trial Digital Portal and a Clinical Trial Academy to enhance efficiency and talent development.
  • Strategic partnerships, including with the Hong Kong Genome Institute, will leverage local data and attract biomedical technology enterprises to the region.

Ocugen's OCU410ST Receives EMA Orphan Drug Designation for ABCA4-Associated Retinopathies

  • Ocugen's OCU410ST, a modifier gene therapy, has been granted orphan medicinal product designation by the EMA for treating ABCA4-associated retinopathies, including Stargardt disease.
  • This designation provides Ocugen with benefits such as protocol assistance, reduced regulatory fees, and potential market exclusivity in the European Union.
  • Preliminary data from the Phase 1/2 GARDian trial showed an 84% reduction in atrophic lesion growth in treated eyes compared to untreated eyes.
  • OCU410ST leverages an AAV delivery platform to deliver the RORA gene, targeting diverse physiological functions for photoreceptor development and maintenance.

Digital Experience Platforms Revolutionize Pharmaceutical Claims Management and HCP Engagement

  • Modern Digital Experience Platforms (DXPs) are transforming biopharma claims management by integrating MLR review processes with content creation, significantly reducing compliance risks and streamlining approvals.
  • The unified digital authoring approach enables automatic synchronization of claims across channels, ensuring healthcare professionals always receive up-to-date, compliant drug information through preferred digital channels.
  • Implementation of DXPs eliminates inefficient "swivel chair" processes, allowing pharmaceutical companies to streamline content delivery and enhance HCP engagement across multiple territories and brands.

mCSPC Prognosis Linked to AR, Neuroendocrine Markers, and Tumor Suppressor Gene Alterations

  • Analysis of the PEACE-1 trial reveals that AR and neuroendocrine marker expression can identify patient subgroups in metastatic castration-sensitive prostate cancer (mCSPC) with varying outcomes.
  • Patients with AR-high luminal tumors showed better prognoses, while those with neuroendocrine prostate cancer exhibited worse outcomes, particularly driven by neuroendocrine marker expression.
  • Alterations in TP53, PTEN, and RB1 tumor suppressor genes were prognostically significant, with patients having fewer than two alterations showing improved overall survival.
  • Neither AR nor neuroendocrine biomarkers predicted response to abiraterone acetate plus prednisone, leaving the need for predictive biomarkers for abiraterone benefit in mCSPC undetermined.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.