Tagged News
Philikos Initiates Phase 1/2 Trial of T-Guard for Diffuse Cutaneous Systemic Sclerosis
• Philikos has enrolled the first patient in a Phase 1/2 trial evaluating T-Guard for diffuse cutaneous systemic sclerosis (dcSSc), a severe autoimmune disorder with limited treatment options.
• The open-label study will assess safety and preliminary efficacy in 12 early-stage dcSSc patients whose disease remains refractory despite prior immunosuppressive therapy.
• T-Guard, administered as four infusions over one week, aims to provide a safer alternative to hematopoietic stem cell transplantation by selectively depleting disease-associated T cells and NK cells.
FDA Greenlights Promontory Therapeutics' Phase 3 Trial Design for PT-112 in Metastatic Prostate Cancer
• Promontory Therapeutics has successfully completed an End of Phase 2 meeting with the FDA, reaching agreement on key aspects of a registrational Phase 3 trial for PT-112 in metastatic castration-resistant prostate cancer.
• The FDA approved the proposed dosing regimen, patient population, study comparator, and endpoints, with an interim analysis provision that could allow for drug approval before study completion.
• Preliminary clinical outcomes from the Phase 2 trial of PT-112 will be presented at the upcoming ASCO 2025 Annual Meeting on June 2nd, following recent presentation of immune response biomarker data at AACR 2025.
Related Clinical Trials:
Promontory Therapeutics Inc.
Posted 7/1/2014
National Cancer Institute (NCI)
Posted 4/6/2022
Juvena Therapeutics Initiates First-in-Human Trial of JUV-161 for Muscle Regeneration in Myotonic Dystrophy
• Juvena Therapeutics has begun enrolling participants in the first human clinical trial of JUV-161, a novel fusion protein designed to enhance muscle regeneration for treating Myotonic Dystrophy Type 1 and sarcopenia.
• JUV-161, described as "insulin for muscle," works by restoring AKT signaling pathways that regulate muscle growth and metabolism, offering a unique approach compared to existing RNA-targeting or gene therapy strategies.
• The therapy was discovered using Juvena's proprietary JuvNET platform, which combines AI and stem cell secretome biology to identify therapeutic proteins with regenerative potential.
EMA Designates Allopurinol as First Orphan Drug for Marfan Syndrome
• The European Medicines Agency has designated allopurinol as the first orphan drug for Marfan syndrome, a rare connective tissue disease affecting approximately 7 in 100,000 people in the European Union.
• Researchers from the University of Barcelona, IDIBAPS, and CIBERER have demonstrated allopurinol's potential to halt and prevent aortic aneurysms in animal models, with international clinical trials in patients planned for the future.
• This repurposing of allopurinol, currently used for gout treatment, represents a significant advancement for Marfan syndrome patients who currently have no curative options beyond limited palliative treatments and high-risk surgical interventions.
AKANTIOR® Receives UK Marketing Authorization as First Approved Treatment for Acanthamoeba Keratitis
• SIFI's AKANTIOR® (polihexanide 0.08%) has received both Marketing Authorization and Promising Innovative Medicine designation from the UK's MHRA, marking it as the first approved treatment for Acanthamoeba keratitis.
• The approval confirms AKANTIOR's Orphan Drug Designation and New Active Substance status, recognizing its efficacy against an ultra-rare corneal infection that can lead to blindness if untreated.
• Following its European approval in August 2024, this UK authorization represents a significant advancement for patients with this devastating eye infection, with SIFI planning to file for NICE reimbursement by June 2025.
China Approves First Domestically Developed Enzyme Replacement Therapy for Gaucher Disease
• CANbridge Pharmaceuticals has received NMPA approval for velaglucerase-beta (Gaurunning), China's first domestically developed enzyme replacement therapy for Type I and III Gaucher disease in patients aged 12 and above.
• The pivotal clinical trial demonstrated statistically significant reductions in spleen volume at both 60 U/kg (P<0.0001) and 30 U/kg (P<0.001) doses, meeting its primary efficacy endpoint.
• Developed in collaboration with WuXi Biologics, Gaurunning represents a breakthrough in rare disease treatment in China, potentially improving accessibility and affordability for the estimated 3,000 Chinese Gaucher disease patients.
NICE Extends Access to Brineura for CLN2 Batten Disease While Seeking Long-term Solution
• NICE has secured continued access to cerliponase alfa (Brineura) for current patients and those starting treatment before December 2025, despite not recommending it for routine NHS use due to cost concerns.
• The enzyme replacement therapy, costing over £500,000 per patient annually, has demonstrated effectiveness in slowing CLN2 progression in the short term, but lacks sufficient long-term efficacy data.
• NICE, NHS England, and manufacturer BioMarin continue negotiations to reach a sustainable pricing agreement that could extend access to all future patients with this rare, life-limiting condition.
Ionis Reports Positive Phase 3 Results for Olezarsen in Familial Chylomicronemia Syndrome
• Ionis Pharmaceuticals' olezarsen met primary and key secondary endpoints in Phase 3 BALANCE trial for familial chylomicronemia syndrome (FCS), demonstrating significant triglyceride reduction.
• The antisense therapy showed a favorable safety profile with no major adverse events reported, potentially offering a new treatment option for this rare genetic disorder.
• Ionis plans to submit regulatory applications in the coming months, positioning olezarsen to potentially become the first approved therapy specifically targeting FCS.
Intellia's CRISPR Gene Therapy Shows Potential as Functional Cure for Hereditary Angioedema in Long-Term Study
• Intellia Therapeutics' NTLA-2002, an in vivo CRISPR-based gene editing therapy, demonstrated a 98% mean reduction in monthly hereditary angioedema attack rates across all patients, with follow-up extending beyond two years.
• Eight of ten patients remained completely attack-free following the initial 16-week observation period, with the longest attack-free duration reaching over 26 months and continuing.
• The single-dose treatment showed a favorable safety profile across all dose levels, with no serious adverse events reported, positioning NTLA-2002 as a potential functional cure for this rare genetic disease.
Related Clinical Trials:
Intellia Therapeutics
Posted 12/10/2021
First Patient Dosed in Phase 2b Trial of RT234, a Novel Inhaled Therapy for Pulmonary Arterial Hypertension
• Respira Therapeutics has dosed the first patient in its Phase 2b VIPAH-PRN trial evaluating RT234, an innovative dry powder inhaled formulation of vardenafil for on-demand symptom relief in PAH patients.
• RT234 represents a first-in-class approach for PAH, designed for "as needed" use to acutely improve exercise capacity and reduce breathlessness, unlike current maintenance therapies that don't address episodic symptoms.
• The multicenter dose-escalation study will assess RT234's ability to provide rapid symptom relief for patients with NYHA Functional Class II-III PAH symptoms, potentially addressing a significant unmet need in disease management.