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Clinical Trial News

University at Buffalo Opens FOXG1 Research Center to Advance Gene Therapy for Rare Disorder

  • The University at Buffalo (UB) has inaugurated the FOXG1 Research Center (FRC) to focus on developing treatments for FOXG1 syndrome and related neurodevelopmental disorders.
  • Researchers at the FRC are developing a viral gene therapy showing promise in preclinical studies, with potential human clinical trials planned for as early as spring 2026.
  • The center is supported by the FOXG1 Research Foundation, which has raised over $7 million to advance research and clinical trials for FOXG1 syndrome.
  • The FOXG1 Research Center aims to leverage AI to assist children with speech and language disorders, alongside developing effective treatments for FOXG1 syndrome.

Thrombolytic Science Receives FDA Clearance for mproUK Clinical Trial

  • Thrombolytic Science (TSI) has received FDA clearance for its Investigational New Drug application for mproUK, a recombinant fibrinolytic pro-enzyme, confirming its safety profile.
  • The Phase II trial in the UK for myocardial infarction is set to commence following MHRA approval, building on promising Phase II results from the DUMAS trial in stroke patients in the Netherlands.
  • mproUK offers a potentially safer and more effective reperfusion option for life-threatening thrombotic conditions, with the possibility of earlier blood flow re-establishment.
  • The treatment approach combines a mini bolus of r-tPA followed by a low dose of mproUK, showing promise in clinical studies with reduced bleeding risk and rethrombosis.

FDA's Approval of Kisunla Faces Scrutiny Over Advisory Committee Conflicts of Interest

  • An investigation reveals that a significant number of FDA advisory committee members who recommended Kisunla's approval had financial ties to Eli Lilly and other companies developing anti-amyloid drugs.
  • Concerns are raised about the potential influence of these conflicts on the drug approval process and the public's trust in the FDA's decisions, especially after the controversy surrounding Aduhelm.
  • Experts question whether the benefits of Kisunla outweigh the risks of brain swelling and bleeding, despite the advisory committee's unanimous recommendation for approval.
  • The FDA's practice of granting waivers for committee members with financial conflicts is under scrutiny, with calls for stricter guidelines to bolster public confidence in the agency.

FDA Approves Bimzelx for Psoriatic Arthritis and Spondyloarthritis; New Eczema Treatment Ebgliss

  • The FDA has approved Bimzelx (bimekizumab-bkzx) for psoriatic arthritis, non-radiographic axial spondyloarthritis, and ankylosing spondylitis, targeting IL-17A and IL-17F to reduce inflammation.
  • Eli Lilly's Ebgliss (lebrikizumab-lbkz) has been approved by the FDA for moderate-to-severe eczema in patients 12 and older, showing skin clearance and itch relief in studies.
  • A new study indicates that osteoporosis and fracture rates have significantly decreased in rheumatoid arthritis patients over the past 24 years due to advances in treatments like biologics.
  • Research suggests that moderate coffee consumption (3 cups/day) may lower the risk of heart disease, stroke, and type 2 diabetes due to antioxidants and anti-inflammatory agents.

Evaluation of the Efficacy and Safety of Lebrikizumab (LY3650150) in Moderate to Severe Atopic Dermatitis (ADvocate1)

NCT04146363CompletedPhase 3
Eli Lilly and Company
Posted 9/24/2019

Evaluation of the Efficacy and Safety of Lebrikizumab (LY3650150) in Moderate to Severe Atopic Dermatitis

NCT04178967CompletedPhase 3
Eli Lilly and Company
Posted 10/29/2019

Celltrion's Ustekinumab Biosimilar SteQeyma Approved in Europe; Zymfentra Access Expands

  • The European Commission approved Celltrion's SteQeyma, an ustekinumab biosimilar referencing Stelara, for treating chronic inflammatory diseases across gastroenterology, dermatology, and rheumatology.
  • Celltrion USA partnered with Cigna Healthcare and Express Scripts to broaden patient access to Zymfentra (infliximab-dyyb) for ulcerative colitis and Crohn's disease.
  • MedImpact Healthcare Systems is expanding access to biosimilars referencing Humira (adalimumab), including Simlandi, for patients with Crohn's disease and rheumatoid arthritis.
  • The Association for Accessible Medicines held a policy briefing addressing patent-related barriers hindering patient access to biosimilars and generic medicines.

Eyenovia Launches Clobetasol Propionate Ophthalmic Suspension for Post-Ocular Surgery Inflammation and Pain

  • Eyenovia has commercially launched clobetasol propionate ophthalmic suspension 0.05% in the U.S. for treating post-operative inflammation and pain after eye surgery.
  • Clobetasol, a potent steroid, offers a convenient twice-daily dosing regimen, distinguishing it from many other post-surgical eye drops.
  • Phase 3 study results presented at AAO 2024 demonstrated rapid inflammation and pain relief with clobetasol compared to placebo, along with improved visual acuity.
  • Market research indicates strong interest from ophthalmic surgeons in prescribing clobetasol due to its efficacy, safety profile, and simplified distribution model.

PolarCool Submits FDA De Novo Application for PolarCap System to Treat Concussions

  • PolarCool has submitted a De Novo application to the FDA for its PolarCap System, aiming for market approval in the U.S.
  • The U.S. market for concussion treatment is valued at approximately SEK 3.4 billion annually, presenting a significant opportunity for PolarCool.
  • Clinical results from a 5-year study support the De Novo application, positioning PolarCap as a novel treatment for concussions.
  • PolarCool anticipates a full-scale investment in the U.S. market in 2025-2026, with an employee already stationed in the U.S. to prepare for the launch.

GAO Report Highlights Incomplete Reporting in DOD and VA Biomedical Research

  • A recent GAO report reveals that the Departments of Defense (DOD) and Veterans Affairs (VA) dedicated approximately $20 billion and $10 billion, respectively, to biomedical R&D from 2019 to 2023.
  • The study found inconsistencies in disclosing DOD support in biomedical patents, with 559 out of 3,078 patents lacking correct DOD award numbers.
  • The GAO report indicates that the majority of DOD- and VA-funded clinical trials registered between 2014 and 2023 had late or missing results, hindering transparency and access to crucial scientific information.

New HER2 Test Could Expand Trastuzumab Deruxtecan Eligibility for Lung Cancer Patients

• A novel, more sensitive test for HER2 protein in lung cancer tissues has been developed, potentially expanding the number of patients eligible for trastuzumab deruxtecan (T-DXd). • The new assay detected elevated HER2 levels in 63% of lung cancer samples, a significant increase compared to the 2% identified by current standard tests. • This advancement could lead to more personalized treatment approaches, matching patients with targeted therapies based on their unique biomarker profiles. • Researchers are working on similar tests for other cancer types, paving the way for broader application of biomarker-driven cancer treatments.

Intrathecal Pain Pump Significantly Improves Quality of Life for Pancreatic Cancer Patients

  • An intrathecal pain pump delivers medication directly to the spinal cord, offering potent pain relief with lower doses compared to oral medications for pancreatic cancer patients.
  • A patient at MD Anderson Cancer Center experienced significant improvements in quality of life, including increased activity levels and reduced pain, after receiving an intrathecal pain pump.
  • The pain pump delivers non-opioid medication, addressing both tumor pain and neuropathy, allowing the patient to engage in daily activities and enjoy a fuller life.
  • This treatment option is particularly beneficial for patients with constant cancer pain who seek an improved quality of life, warranting a discussion with their healthcare provider.

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