Clinical Trial News
First Biologic Drug for Pulmonary Sarcoidosis in 50 Years Administered at Oxford Hospital
• Infliximab, a TNF-α inhibitor, has become the first new drug approved for pulmonary sarcoidosis in over 50 years, following NHS England commissioning with NICE support.
• The biologic therapy, administered to the first patient at Oxford's John Radcliffe Hospital, offers potential to reduce reliance on broad immunosuppressants that often cause significant side effects.
• Professor Ling-Pei Ho, who led the Oxford Sarcoidosis Service and chaired the initial NHS England working policy group, played a key role in securing the drug's approval for this rare condition affecting 1 in 10,000 UK residents.
Fezolinetant Shows Promising Body Composition Benefits for Menopausal Women in Phase 3 Trials
• Fezolinetant, a nonhormonal NK3R antagonist approved for treating menopausal vasomotor symptoms, demonstrated small but consistent reductions in waist circumference and body roundness index over 52 weeks while maintaining stable weight.
• The pooled analysis of three SKYLIGHT phase 3 trials involving 1,830 postmenopausal women showed decreases in waist circumference of approximately 0.88 cm with fezolinetant 45 mg, compared to only 0.15 cm with placebo.
• These findings suggest fezolinetant may offer additional cardiometabolic benefits beyond hot flash relief, potentially addressing central adiposity concerns in menopausal women who face increased risks for metabolic and cardiovascular conditions.
Cardioplexol™ Demonstrates Safety and Efficacy in Phase 3 Trial for Cardiac Surgery
• A pivotal phase 3 non-inferiority trial has confirmed the safety and efficacy of Cardioplexol™, a new low-volume cardioplegic solution, compared to standard Buckberg blood cardioplegia in cardiac surgeries.
• Cardioplexol™ showed significant advantages including faster cardiac arrest (11 vs 71 seconds), reduced cross-clamp time, lower defibrillation rates (10% vs 52%), and decreased need for post-operative inotropic support.
• Following successful clinical validation, Cardioplexol™ received marketing authorization in Switzerland in September 2023 and in 10 European countries in April 2024.
FDA Advisory Committee Rejects UroGen's Bladder Cancer Therapy UGN-102
• UroGen Pharma's stock plummeted approximately 47% after an FDA advisory committee voted against its bladder cancer therapy UGN-102 (mitomycin), citing concerns over study design.
• The FDA had repeatedly recommended a randomized trial design, noting that the Envision trial's lack of a concurrent control arm made primary endpoints difficult to interpret.
• Despite UroGen reporting an 80.6% probability of patients remaining in complete response at 18 months, the regulatory setback highlights challenges in developing alternatives to surgical intervention for bladder cancer.
Landmark VA Study Reveals Genomic Differences in Metastatic Prostate Cancer Across Racial Groups
• Researchers from Moffitt Cancer Center and partners analyzed genomic data from over 5,000 veterans with metastatic prostate cancer, finding significant biological differences between non-Hispanic Black and white patients.
• Non-Hispanic Black veterans showed higher rates of genomic alterations associated with immunotherapy benefit, while white veterans had more frequent mutations in DNA repair genes and androgen receptor pathways.
• Despite these biological differences, both groups demonstrated similar survival outcomes when given equal access to care, highlighting the potential of precision oncology to reduce healthcare disparities.
SP-102 Shows Clinically Meaningful Results for Sciatica Treatment in CLEAR Trial Analysis
• Scilex Holding Company presented post-hoc analysis of the C.L.E.A.R. trial demonstrating clinically meaningful safety and efficacy of SP-102 (SEMDEXA™) for lumbosacral radicular pain at the ASIPP Annual Meeting.
• The analysis showed clear separation between SP-102 and placebo in multiple endpoints, with enhanced results in the modified intent-to-treat population that received confirmed study drug.
• SP-102's viscous gel formulation was specifically designed as a safer alternative to current off-label products that carry warnings about potentially dangerous and life-threatening adverse events.
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.
BiaCure Secures $2.6M NIH Grant to Develop Novel High-Frequency Energy Treatment for Onychomycosis
• BiaCure Therapies has been awarded a $2.6 million SBIR Phase I grant from NIH to develop its high-frequency energy therapy system for treating onychomycosis, a condition affecting over 35 million Americans.
• The company's BiaPulse™ device delivers targeted high-frequency energy through a patient-friendly applicator, offering a non-invasive alternative to traditional oral antifungal medications without systemic side effects.
• The funding will support clinical efficacy studies in collaboration with dermatology and podiatry experts, potentially establishing a new standard of care for fungal nail infections.
AbelZeta's C-CAR168 Shows Promising Results in Treating Refractory Lupus Nephritis and Multiple Sclerosis
• Novel bispecific CAR-T therapy C-CAR168 targeting CD20/BCMA demonstrated positive safety profile with only low-grade cytokine release syndrome in early clinical trials for autoimmune diseases.
• Among four lupus nephritis patients reaching 6-month evaluation, all achieved SRI(4) response with two complete remissions, allowing discontinuation of immunosuppressants and steroids in most patients.
• Early efficacy signals were observed in a Secondary Progressive Multiple Sclerosis patient, including improved gait, reduced brain inflammation, and better functional assessment scores.
Highlighted Clinical Trials:
RenJi Hospital
Posted 3/20/2024
Alvotech Expands European Presence with Successful Nasdaq Stockholm Listing
• Alvotech's Swedish Depository Receipts (SDRs) offering was multiple times oversubscribed, attracting over 3,000 new shareholders and raising approximately SEK 39 million.
• The biosimilar developer's strategic expansion includes acquiring Xbrane's R&D operations at the Karolinska life-science hub, strengthening its research capabilities and establishing a footprint in Sweden's biotech sector.
• Trading of Alvotech's SDRs on Nasdaq Stockholm commenced on May 19, 2025, under the ticker "ALVO SDB," enhancing the company's access to Nordic and European investors and capital markets.