Clinical Trial News
BATURA Trial: Airsupra Reduces Severe Asthma Exacerbation Risk by 47% Compared to Albuterol Alone
• AstraZeneca's Airsupra (albuterol/budesonide) demonstrated a 47% reduction in severe asthma exacerbation risk compared to albuterol alone in patients with mild asthma, according to the Phase IIIb BATURA trial.
• The anti-inflammatory rescue therapy reduced total systemic corticosteroid exposure by 63%, potentially decreasing risks associated with cumulative steroid use including diabetes, cardiovascular disease, and other adverse conditions.
• Results were so overwhelmingly positive that the Independent Data Monitoring Committee recommended early termination of the trial, suggesting a potential paradigm shift in asthma rescue treatment after 50 years of albuterol-only standard care.
Highlighted Clinical Trials:
AstraZeneca
Posted 5/20/2024
Bond Avillion 2 Development LP
Posted 9/2/2022
AstraZeneca
Posted 6/17/2024
FDA Expands Jivi Approval for Hemophilia A Treatment in Children Ages 7-12
• The FDA has approved Bayer's Jivi (antihemophilic factor recombinant, PEGylated-aucl) for pediatric patients aged 7 to 12 years with hemophilia A, expanding treatment options for this age group.
• The approval was based on data from the Alfa-PROTECT and PROTECT Kids studies, which demonstrated the safety and efficacy of Jivi in pediatric patients with severe hemophilia A.
• Jivi was previously approved in 2018 for adults and adolescents aged 12 and older, and can be used for on-demand treatment, perioperative management, and routine prophylaxis to reduce bleeding episodes.
Highlighted Clinical Trials:
Bayer
Posted 5/29/2013
Bayer
Posted 3/23/2022
FDA Grants Orphan Drug Designation to Repair Biotechnologies' mRNA Therapy for Rare Cholesterol Disorder
• The FDA has granted Orphan Drug Designation to REP-0003, a novel mRNA therapy developed by Repair Biotechnologies for treating homozygous familial hypercholesterolemia (HoFH), a rare condition causing accelerated atherosclerosis.
• REP-0003 works by selectively clearing harmful excess free cholesterol inside cells while preserving essential cholesterol, showing promising results in preclinical studies including plaque regression and improved liver function.
• The designation provides Repair Biotechnologies with seven years of market exclusivity upon approval, tax credits for clinical trials, waived FDA fees, and potential fast-track pathways as they prepare for first human trials in 2026.
Gradient's Pulmonary Artery Denervation System Receives FDA Breakthrough Designation for Pulmonary Hypertension
• Gradient Denervation Technologies' novel ultrasound-based pulmonary denervation system has received FDA Breakthrough Device Designation, creating an expedited review pathway for this potentially life-changing technology.
• The system treats pulmonary hypertension by ablating nerves around the pulmonary artery using therapeutic ultrasound in a minimally invasive procedure, aiming to reduce vascular resistance and decrease pulmonary pressures.
• The PreVail-PH2 Early Feasibility Study is currently enrolling patients with Group 2 Pulmonary Hypertension, a condition affecting up to two-thirds of heart failure patients worldwide with no currently approved therapies in the US.
CellCentric Secures $120 Million Series C Funding to Advance Oral Multiple Myeloma Treatment
• CellCentric has raised $120 million in Series C funding to advance inobrodib, a first-in-class oral p300/CBP inhibitor for multiple myeloma treatment.
• The funding will support a Phase II/III study with potential for accelerated approval, as well as trials combining inobrodib with bi-specific antibodies and in maintenance settings.
• Inobrodib has received FDA Fast Track and orphan drug designation, with promising efficacy and safety data presented at the American Society of Hematology Annual Meeting.
Prime Medicine Restructures: CEO Replaced, 25% Staff Cut, and Lead Program Halted
• Prime Medicine announced a major restructuring on Monday, replacing its CEO, laying off 25% of staff, and discontinuing its only clinical program despite previous scientific milestones.
• The company's decision reflects broader challenges in the gene editing sector, where numerous firms have reduced workforce or terminated programs during an extended industry downturn.
• This reorganization highlights the disconnect between promising early scientific data and commercial viability in the advanced therapeutic space, particularly for CRISPR-based technologies.
GT Biopharma Advances GTB-3650 Phase 1 Trial to Second Cohort Following Promising Immune Activation Signals
• GT Biopharma has successfully completed dosing in the first cohort of its Phase 1 trial for GTB-3650, a second-generation TriKE immunotherapy, with no safety or tolerability issues observed.
• Both patients in Cohort 1 demonstrated early biological signs of immune activation, including increased natural killer cell activity and expansion, supporting the therapy's mechanism of action.
• The trial, which will evaluate approximately 14 patients across seven dose cohorts, aims to treat relapsed or refractory CD33-expressing hematologic malignancies through enhanced NK cell-mediated cancer destruction.
Highlighted Clinical Trials:
Masonic Cancer Center, University of Minnesota
Posted 11/19/2024
Reunion Neuroscience Completes Patient Dosing in Phase 2 Trial of Novel Psychedelic for Postpartum Depression
• Reunion Neuroscience has completed patient dosing in its RECONNECT Phase 2 trial evaluating RE104, a short-duration psychedelic therapy for postpartum depression, with topline results expected in Q3 2025.
• The multicenter trial enrolled 84 adult female patients with moderate-to-severe PPD across 38 U.S. clinical sites, assessing the safety and efficacy of a single subcutaneous dose of RE104.
• RE104 is designed to deliver a shorter psychedelic experience (3-4 hours) compared to psilocybin or LSD, while maintaining similar intensity and a favorable safety profile based on Phase 1 results.
Highlighted Clinical Trials:
Reunion Neuroscience Inc
Posted 6/14/2024
Respira Therapeutics to Present Phase 2b Results of RT234 Inhalation Therapy for PAH at ATS 2025
• Respira Therapeutics will present late-breaking results from its Phase 2b trial of RT234, an inhaled vardenafil powder for pulmonary arterial hypertension, at the ATS 2025 International Conference on May 20.
• RT234 is being developed as the first as-needed (PRN) therapy for PAH patients to acutely improve exercise tolerance and reduce exertional symptoms, potentially enhancing daily activities and quality of life.
• The innovative treatment utilizes Respira's proprietary Axial Oscillating Sphere Dry Powder Inhaler technology to maximize drug delivery to the distal lung for optimal therapeutic effect.
Highlighted Clinical Trials:
Respira Therapeutics, Inc.
Posted 9/25/2020
Eascra Biotech Secures $100,000 MassVentures Grant for Novel Nanoparticle Drug Delivery Platform
• Eascra Biotech has received a $100,000 Stage I SBIR Targeted Technologies grant to advance their Janus Base Nanoparticles (JBNps) delivery platform for hard-to-reach tissues.
• The company has leveraged microgravity research, completing five missions on the International Space Station, becoming the first commercial entity to produce medical nanoparticles in space.
• Eascra's proprietary technology can deliver RNA therapeutics to challenging tissues like cartilage and solid tumors while maintaining mRNA stability at room temperature, eliminating cold chain requirements.