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Clinical Trial News

UK Doctors Trial Freeze-Dried Fecal Microbiota Pills to Combat Antibiotic-Resistant Superbugs

  • UK researchers at Guy's and St Thomas' hospitals are testing "poo pills" containing freeze-dried donor feces to eliminate dangerous superbug infections from patients' intestinal tract.
  • The 41-patient pilot study showed promising early results, with donor bacteria persisting in patients' bowels for at least one month and competing with superbugs for resources in the gut.
  • The therapy aims to address antibiotic-resistant infections that kill approximately one million people annually by targeting the bowel, which serves as the largest reservoir of antibiotic resistance in humans.
  • If proven effective in larger trials, the treatment could be used for both prevention and treatment in high-risk patients, including those undergoing immunosuppressive therapies like cancer treatment or organ transplants.

Researchers Identify Two Drug Candidates for Ultra-Rare X-Linked Myopathy Using Zebrafish Model

Rare DiseaseDrug Discovery
  • Researchers have identified two potential drug candidates for treating X-linked myopathy with excessive autophagy (XMEA), an ultra-rare genetic disease documented in only 33 cases globally as of March 2024.
  • The study utilized zebrafish as a model organism to investigate XMEA, which is caused by mutations in the VMA21 gene and leads to progressive muscle weakness affecting the liver and heart.
  • The identified compounds showed promise in mitigating XMEA-related muscle degeneration, representing a significant step forward for a condition that currently lacks effective treatment options.
  • Further testing will be required to confirm the efficacy and safety of these drug candidates before potential clinical application in humans.

FDA Rejects Elamipretide for Rare Mitochondrial Disease Despite Patient Improvements

Rare DiseaseOrphan Drug
  • The FDA's Division of Cardiology and Nephrology rejected elamipretide's new drug application on May 29, 2025, despite reported improvements in patients with rare mitochondrial diseases.
  • Four-year-old Hope Filchak with Microphthalmia with Linear Skin Defects syndrome showed stabilized heart function and increased energy after 18 months of elamipretide treatment.
  • Stealth Biotherapeutics has reduced overhead by 30% and estimates it can continue manufacturing for only six to nine months without FDA approval.
  • The FDA's rejection was partly based on lack of improvement in six-minute walk tests, which experts argue is not an appropriate outcome measure for mitochondrial diseases.

New Zealand Universities Launch Clinical Trials Testing Psychedelics for Treatment-Resistant Mental Health Disorders

  • University of Auckland and University of Otago are conducting clinical trials investigating psychedelic drugs for treatment-resistant mood disorders, including an eight-week LSD microdosing study.
  • Researchers are testing whether psychedelics at specific dosages can produce changes in brain activity that may improve mental health outcomes for patients who haven't responded to conventional treatments.
  • The trials represent a significant shift in psychiatric research, exploring alternative therapeutic approaches for conditions like anxiety and depression that affect substantial portions of the population.
  • Study leaders emphasize the importance of controlled clinical settings and caution against attributing therapeutic effects solely to the psychedelic compounds without proper scientific evaluation.

UC Berkeley Awards $200,000 Venture Grant to HypO2Regen Therapeutics with Future Investment Option

  • UC Berkeley has awarded a $200,000 venture grant to HypO2Regen Therapeutics, a life sciences company, through its Life Sciences Entrepreneurship Center and Berkeley SkyDeck programs.
  • The funding consists of $100,000 from each program, with Berkeley SkyDeck retaining an option to invest an additional $100,000 in the future.
  • This dual-source funding demonstrates UC Berkeley's collaborative approach to fostering innovation and entrepreneurship in biotechnology and scientific research development.

Serve You Rx Adds Two Ustekinumab Biosimilars to Formulary, Offering Up to 96% Cost Savings

  • Serve You Rx has added two newly approved biosimilars to Stelara (ustekinumab) - Otulfi and Yesintek - to its Biosimilar Advantage Formulary for treating Crohn's disease, ulcerative colitis, plaque psoriasis, and psoriatic arthritis.
  • The biosimilars offer substantial cost savings of up to 96% compared to Stelara's annual cost of up to $152,000, representing significant opportunities for self-funded employers and health-plan sponsors to manage healthcare costs.
  • Otulfi will be dispensed exclusively through Mark Cuban Cost Plus Drugs via Waltz Health's network, while Yesintek will be available through Serve You Rx's specialty pharmacy network powered by Waltz Health.
  • Both medications will be supported by robust copay assistance programs to ensure patient access without financial barriers while maintaining the same therapeutic benefits as the reference drug.

Empagliflozin Demonstrates Kidney Safety Across Multiple Patient Populations, New Pooled Analysis Shows

  • A pooled analysis of four major trials involving 23,340 participants confirms empagliflozin reduces acute kidney injury risk by 27% and large serum creatinine increases by 19%.
  • The kidney protective effects remain consistent regardless of predicted eGFR dip size or baseline disease severity markers across patients with diabetes, heart failure, and chronic kidney disease.
  • Secondary analysis of the EMPACT-MI trial reveals empagliflozin preserves kidney function in heart attack patients, with stable kidney function after two years compared to significant worsening in placebo patients.
  • Researchers conclude that acute eGFR dips following empagliflozin initiation are benign events that should not deter clinicians from prescribing this therapy to eligible patients.

EMPACT-MI: A Study to Test Whether Empagliflozin Can Lower the Risk of Heart Failure and Death in People Who Had a Heart Attack (Myocardial Infarction)

NCT04509674CompletedPhase 3
Boehringer Ingelheim
Posted 12/16/2020

IL-12-Modified mRNA Vaccines Demonstrate Enhanced T Cell Activation in University of Pennsylvania Study

  • Researchers from the University of Pennsylvania, including the School of Veterinary Medicine, Perelman School of Medicine, and Children's Hospital of Philadelphia, have developed modified mRNA vaccines incorporating cytokine IL-12 that significantly enhance CD8+ T cell responses.
  • The study published in Science Immunology shows that IL-12-modified vaccines elicit stronger cellular immunity compared to traditional mRNA formulations, potentially reducing vaccination frequency and dosage requirements.
  • This advancement could improve vaccine effectiveness against infectious diseases and cancer, with potential applications for HIV and avian influenza vaccines.
  • The collaborative research demonstrates how cytokine biology integration may lead to a new generation of vaccines with enhanced protective capabilities and reduced dose-related side effects.

FDA Approves Moderna's Next-Generation COVID-19 Vaccine mRNA-1283 with Enhanced Efficacy

Drug Approval
  • The FDA has approved Moderna's new COVID-19 vaccine mRNA-1283 (mNEXSPIKE), marking the first authorization under updated federal guidelines prioritizing vaccination for adults 65 and older.
  • In a Phase 3 trial of over 11,000 participants, mRNA-1283 demonstrated 9.3% higher relative vaccine efficacy compared to Moderna's original vaccine, with 13.5% increased efficacy in adults 65 and older.
  • The vaccine showed a comparable safety profile with fewer local side effects and will be available for the 2025-2026 respiratory virus season in the U.S.
  • COVID-19 vaccination remains available for children 6 months and older through shared clinical decision-making, reflecting ongoing federal adjustments to immunization recommendations.

Zhengye Biotechnology Completes $6 Million IPO as Chinese Veterinary Vaccine Manufacturer Enters US Markets

  • Zhengye Biotechnology raised $6 million in its initial public offering on January 7th, 2025, issuing 1.5 million shares at $4.00 per share with Kingswood Capital Partners as underwriter.
  • The Chinese veterinary vaccine manufacturer focuses on livestock vaccine research, development, manufacturing and sales, primarily serving the Chinese market.
  • ZYBT stock has gained 21.6% since its IPO debut, trading at $5.91 as of early January 2025, with a trading range between $3.50 and $14.30.
  • The company's lock-up period expires on July 7th, 2025, after which insiders and major shareholders will be permitted to sell their shares.

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