Clinical Trial News
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.
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.
Recludix's STAT6 Inhibitor Shows Promising Efficacy in Preclinical Asthma Model, Matching Biologic Treatments
• Recludix Pharma's novel STAT6 inhibitor demonstrated efficacy comparable to anti-IL-4/IL-13 antibody treatments in preclinical asthma models, with significant dose-dependent reduction of airway inflammation.
• The reversible, non-degrading inhibitor achieved durable target modulation without degrading STAT6 protein, potentially avoiding hematologic safety concerns associated with JAK inhibitors.
• Recludix is collaborating with Sanofi to advance this potential first-in-class oral therapy for type 2 inflammatory diseases including asthma, COPD, and atopic dermatitis into clinical trials.
Cordis Launches 10,000-Patient Global Registry for SELUTION SLR Drug-Eluting Balloon
• Cordis has initiated the SELUTION Global Coronary Registry, which will track real-world outcomes of their sirolimus-eluting balloon in up to 10,000 patients worldwide for five years.
• The registry complements five ongoing randomized trials and builds on positive peripheral experience data, positioning SELUTION SLR as a next-generation technology that delivers sustained drug release without permanent implants.
• A distinguished steering committee of international interventional cardiologists will oversee the registry, including Dr. Ajay Kirtane from Columbia University as chairman.
Rani Therapeutics and Chugai Pharmaceutical Partner to Advance Oral Antibody Delivery Technology
• Rani Therapeutics has entered a research agreement with Chugai Pharmaceutical to evaluate the application of its RaniPill® oral delivery technology for Chugai's antibodies against undisclosed targets.
• Initial analysis confirms RaniPill® delivery demonstrated comparable bioavailability to subcutaneous injection for both molecules studied, potentially transforming how biologics are administered.
• The collaboration leverages Rani's expertise in oral biologics delivery with Chugai's antibody engineering capabilities, aiming to address the challenge of oral administration for complex biological drugs.
Fujifilm Validates Novel Peptide-Oligonucleotide Conjugates for Targeted Cancer Cell Delivery
• Fujifilm has successfully validated peptide-oligonucleotide conjugates that selectively target cancer cells, utilizing cyclic peptides from their proprietary library containing trillions of variants.
• The company developed a cyclic peptide that binds strongly to integrins overexpressed on cancer cell surfaces with a low dissociation constant of 1.6 nM, enabling higher cellular uptake compared to non-conjugated oligonucleotides.
• This breakthrough addresses delivery challenges in oligonucleotide-based therapies and will be presented at TIDES USA 2025 in San Diego, highlighting Fujifilm's expansion of global drug discovery services for peptide therapeutics.
Prime Medicine Reports Breakthrough Clinical Data for First Prime Editing Therapy in Chronic Granulomatous Disease
• Prime Medicine's PM359, the first Prime Editing therapy administered to humans, demonstrated rapid restoration of NADPH oxidase activity in a patient with Chronic Granulomatous Disease, exceeding therapeutic thresholds.
• A single infusion of PM359 achieved 66% DHR positivity by Day 30, significantly above the 20% threshold believed to be potentially curative, with faster engraftment than existing gene editing technologies.
• The therapy showed an encouraging safety profile with no serious adverse events related to PM359, marking a significant milestone for Prime Editing technology as a potential one-time curative treatment for genetic diseases.
Ainos Reports Promising Interim Results for VELDONA in Feline Chronic Gingivostomatitis Treatment
• Ainos's low-dose oral interferon therapy VELDONA has shown significant anti-inflammatory effects in cats with Feline Chronic Gingivostomatitis, with inflammation score improvements ranging from 10.5% to 44.4% in initial trial participants.
• The treatment demonstrated a notable steroid-sparing effect, allowing complete discontinuation of steroid therapy in two cases and dose reduction in another, potentially addressing a critical unmet need in veterinary medicine.
• With no significant side effects observed, VELDONA positions Ainos to potentially capture a share of the global pet dental market, projected to reach $13 billion by 2030.
Ruxoprubart Shows Promising Phase II Results as Novel Monotherapy for Paroxysmal Nocturnal Hemoglobinuria
• NovelMed's Ruxoprubart demonstrated significant efficacy in a Phase II trial for treatment-naïve PNH patients, meeting all primary endpoints including complete transfusion avoidance and increased hemoglobin levels.
• The drug's selective inhibition of the Alternative Pathway without affecting the Classical Pathway offers a potentially safer profile than existing treatments, which often carry Black Box warnings for infection risk.
• With FDA Orphan Drug Designation already secured and plans to file for Breakthrough Therapy Designation, Ruxoprubart is positioned as a potential best-in-class therapy for PNH and other complement-mediated disorders.