First Patient Dosed in Phase 2 OASIS Trial of TNX-102 SL for Acute Stress Reaction Following Trauma
• Tonix Pharmaceuticals has initiated dosing in the Phase 2 OASIS trial evaluating TNX-102 SL for reducing acute stress reaction severity and acute stress disorder frequency following traumatic events.
• The investigator-initiated study, sponsored by UNC and supported by a $3 million DoD grant, will enroll approximately 180 motor vehicle collision survivors through emergency departments across the U.S.
• TNX-102 SL, a sublingual cyclobenzaprine formulation that has shown promise in improving sleep quality in PTSD patients, addresses a significant unmet need as no medications currently exist for immediate post-trauma treatment.
CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder
• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder.
• The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein.
• This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.
HHS and NIH Launch "Generation Gold Standard" Universal Vaccine Platform to Combat Pandemic Threats
• The U.S. Department of Health and Human Services and NIH have announced "Generation Gold Standard," a next-generation universal vaccine platform using beta-propiolactone-inactivated whole-virus technology.
• The government-owned platform aims to develop universal vaccines against multiple strains of influenza and coronaviruses, with clinical trials for universal influenza vaccines scheduled to begin in 2026.
• The BPL-1357 intranasal flu vaccine is currently in advanced trials and designed to block virus transmission—a capability absent in current vaccines—with FDA approval targeted for 2029.
FDA to Phase Out Animal Testing Requirement for Monoclonal Antibodies and Drugs
• The FDA announced it will phase out requirements for animal testing of monoclonal antibodies and other drugs, citing the availability of "more effective, human-relevant methods" for safety evaluation.
• The initiative aims to improve drug safety, accelerate evaluation processes, reduce R&D costs, and ultimately lower drug prices by implementing AI-based computational models, humanoid models, and real-world human data.
• This regulatory shift, enabled by the bipartisan FDA Modernization Act 2.0 of 2022, represents what FDA Commissioner Marty Makary calls "a paradigm shift in drug evaluation" that could expedite development of new treatments.
NIH Researchers Develop Eye Drops That Could Slow Progression of Retinitis Pigmentosa
• Researchers at NIH have developed eye drops containing small peptides derived from pigment epithelium-derived factor (PEDF) that successfully slowed vision loss in animal models of retinitis pigmentosa.
• The peptide formulations, particularly H105A, preserved up to 75% of photoreceptors in treated mice and maintained functional vision, while reaching the retina within 60 minutes of application without toxicity.
• While not a cure, these PEDF-based eye drops show promise for treating various degenerative retinal diseases, including retinitis pigmentosa and dry age-related macular degeneration, with researchers now planning human clinical trials.
MaineHealth Launches NIH-Funded Clinical Trials to Address Long COVID Symptoms
• MaineHealth is participating in two NIH RECOVER-ENERGIZE clinical trials testing interventions for long COVID symptoms, with enrollment continuing through July 2025 at their Scarborough research site.
• The first trial evaluates personalized cardiopulmonary rehabilitation for exercise intolerance, while the second tests structured pacing for post-exertional malaise (PEM), addressing two of the most debilitating long COVID symptoms.
• As one of 50 RECOVER study sites nationwide, MaineHealth's research aims to develop effective treatments for long COVID symptoms that significantly impact patients' quality of life.
Corvus Pharmaceuticals Initiates Phase 2 Trial of Soquelitinib for Rare Genetic Disease ALPS
• NIH/NIAID has launched a Phase 2 clinical trial of Corvus Pharmaceuticals' soquelitinib for autoimmune lymphoproliferative syndrome (ALPS), a rare genetic disease typically manifesting by age two.
• The trial will enroll up to 30 patients aged 16 or older with confirmed ALPS-FAS, evaluating soquelitinib's ability to reduce splenomegaly and lymph node volumes while improving cytopenias.
• Soquelitinib, an oral ITK inhibitor, aims to address immune dysregulation in T cells that characterizes ALPS, expanding its clinical development beyond ongoing trials in peripheral T cell lymphoma and atopic dermatitis.
Novel Drug VIP36 Targets CB1 Receptor for Pain Relief Without CNS Side Effects
• NIH-funded researchers have developed VIP36, a new drug targeting cannabinoid receptor type 1 (CB1) that shows promise for treating acute and chronic pain in animal models without harmful side effects.
• The drug overcomes previous CB1-targeting challenges by using computer modeling to create a "peripherally restricted" compound that minimizes central nervous system penetration and reduces tolerance development.
• This breakthrough could lead to non-addictive pain treatments and has broader implications for drug design targeting G-protein-coupled receptors involved in various physiological functions and disease states.
FDA Faces Turmoil: Leadership Changes and Operational Disruptions Raise Industry Concerns
• The FDA is experiencing significant disruption with approximately 1,000 employees laid off, though 300 may be rehired, while 30 facilities were initially slated for closure before some decisions were reversed.
• President Trump's FDA Commissioner nominee Marty Makary faced Senate questioning amid canceled vaccine advisory meetings, while other health agency nominees including Jay Bhattacharya (NIH) and David Weldon (CDC) generate controversy.
• Despite leadership turnover including the departure of CDER Director Patrizia Cavazzoni, industry analysts report minimal approval delays, with some suggesting new leadership may be more supportive of accelerated pathways for rare disease treatments.
Trump Administration Overhauls Healthcare Policy: Major Cuts to Medicaid, RFK Jr. Confirmed as HHS Secretary
• House Republicans narrowly pass budget resolution proposing $880 billion in Medicaid cuts over the next decade, with President Trump's endorsement despite previous promises to protect the program.
• Robert F. Kennedy Jr. confirmed as HHS Secretary in a 52-48 Senate vote, raising concerns over his controversial vaccine stance and unclear Medicare/Medicaid strategy.
• Trump administration terminates multiple health advisory committees, removes clinical trial diversity guidance, and reverses Biden-era drug pricing initiatives in sweeping policy changes.
Nektar and TrialNet Partner to Test Novel T-Cell Therapy Rezpegaldesleukin in Early-Stage Type 1 Diabetes
• Nektar Therapeutics and TrialNet have announced a collaboration to evaluate rezpegaldesleukin, a first-in-class T regulatory cell stimulator, in patients with new-onset type 1 diabetes.
• The Phase 2 randomized trial will enroll approximately 70 adults and children, measuring C-peptide preservation over 12 months to assess the drug's potential to slow disease progression.
• Rezpegaldesleukin works by expanding regulatory T cells to restore immune balance, targeting the underlying autoimmune mechanism of type 1 diabetes.
NIH Funding Cuts Threaten Cancer Research Progress and Early-Career Scientists
• Recent federal funding cuts to the National Institutes of Health (NIH) are creating significant challenges for cancer research institutions, potentially impacting clinical trials and treatment development.
• Early-career investigators and mid-career researchers are particularly vulnerable to these cuts, raising concerns about the long-term sustainability of academic cancer research over the next 5-10 years.
• The reduction in indirect costs funding is affecting research infrastructure maintenance, clinical trial operations, and forcing hiring freezes at major academic institutions.
HealthBio Launches FDA Fast-Track Trial for Long COVID Treatment Using Maraviroc-Atorvastatin Combination
• HealthBio initiates Phase III clinical trial testing a novel combination of Selzentry (maraviroc) and Lipitor (atorvastatin) for Long COVID treatment, involving 252 patients in a randomized, double-blind study.
• The company's approach targets vascular inflammation through CCR5 pathway modulation, supported by preliminary data showing promising results in over 12,000 treated patients.
• With $10M initial funding and FDA Fast-Track designation, the trial aims to address a condition affecting up to 23 million Americans and causing an estimated $3.7 trillion economic impact.
NIH Study: Single Dose of MEDI8852 Antibody Shows Complete Protection Against H5N1 Avian Flu in Primates
• A groundbreaking NIH study demonstrates that a single dose of MEDI8852, a broadly neutralizing antibody developed by MedImmune/AstraZeneca, provides complete protection against H5N1 avian influenza in macaques.
• The antibody targets a stable portion of the influenza virus protein, offering extended protection that could last weeks after administration, making it a promising preventive tool for potential H5N1 outbreaks.
• All macaques pre-treated with MEDI8852 survived H5N1 exposure with minimal or no symptoms, while untreated control subjects developed severe or fatal illness.
NIH Trial Shows Home-Based Peanut Butter Therapy Successfully Treats High-Threshold Peanut Allergies
• A groundbreaking NIH-sponsored trial demonstrated 100% success rate in helping children with high-threshold peanut allergies tolerate up to three tablespoons of peanut butter through gradual exposure therapy.
• The 18-month treatment protocol, using store-bought peanut butter with home-measured doses, offers a safe and accessible option for approximately 800,000 U.S. children with high-threshold peanut allergies.
• The study showed 68.4% of treated participants achieved sustained unresponsiveness to peanuts, compared to only 8.6% natural tolerance development in the avoidance group.
WashU's Novel Nasal COVID-19 Vaccine Advances to U.S. Phase 1 Clinical Trials
• FDA approves Ocugen's investigational new drug application for a nasal COVID-19 vaccine, developed at Washington University in St. Louis, advancing to Phase 1 clinical trials this spring.
• The innovative vaccine, designed to induce immunity in the upper respiratory tract, will be evaluated in 80 adults through both nasal spray and inhalation routes under NIAID sponsorship.
• Early animal studies demonstrated the vaccine's potential to prevent infection and viral transmission, offering advantages over traditional injectable COVID-19 vaccines.
Spear Bio and Beckman Coulter Receive FDA Breakthrough Device Designations for Alzheimer's Blood Tests
• Spear Bio's pTau 217 blood test received FDA Breakthrough Device Designation, offering a less invasive method for early Alzheimer's diagnosis.
• Beckman Coulter's Access p-Tau217/β-Amyloid 1-42 plasma ratio test also gained FDA Breakthrough Device Designation, aiding in identifying amyloid pathology.
• Both tests address the critical need for accessible, early Alzheimer's diagnosis, potentially improving patient outcomes and treatment access.
• These designations expedite the development and review process, bringing innovative diagnostic tools to market faster for Alzheimer's disease.
mRNA Cancer Vaccines Show Promise in Clinical Trials, Aiming for Personalized Immunotherapy
• Over 60 mRNA cancer vaccine candidates are in clinical trials, signaling a transformative shift in cancer treatment.
• BioNTech's BNT111, targeting melanoma-associated antigens, shows positive Phase 2 data when combined with cemiplimab.
• Personalized mRNA vaccines, like Moderna's mRNA-4157, are being explored in combination with PD-1 therapies for various cancers.
• The first commercial mRNA cancer vaccine is expected by 2029, driven by technological advancements and increased investment.
AI Clinical Trials Hit Record Numbers in 2024 as Pharma Giants Embrace Technology
• Clinical trials utilizing artificial intelligence reached unprecedented levels in 2024, with major pharmaceutical companies like Roche and AstraZeneca implementing AI for patient recruitment and trial management.
• AI applications are revolutionizing clinical trial processes by automating patient recruitment, optimizing eligibility criteria, and managing complex trial data in real-time.
• Despite patient concerns about reduced in-person interactions and potential medical errors, survey respondents across 11 countries recognize AI's potential to accelerate healthcare delivery.
Aurion Biotech's AURN001 Shows Promise in Phase 1/2 Trial for Corneal Edema
• Aurion Biotech's AURN001, an allogeneic cell therapy, demonstrated statistically significant improvement in visual acuity in patients with corneal edema in a Phase 1/2 trial.
• The high-dose AURN001 arm showed that 50% of patients achieved a ≥15-letter improvement in best-corrected visual acuity (BCVA) at 6 months, compared to 14.3% in the Y-27632-only arm.
• Key secondary endpoints, including change in BCVA and central corneal thickness, also showed statistically significant improvements in the high-dose AURN001 arm.
• AURN001 combines neltependocel (allogeneic human corneal endothelial cells) and Y-27632 (a rho kinase inhibitor) and is intended as a one-time anterior chamber injection.
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