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Clinical Trial News

Doxycycline Prophylaxis Shows Promise in Preventing Bacterial STIs Among High-Risk Populations

• Doxycycline prophylaxis demonstrates potential in preventing bacterial sexually transmitted infections (STIs), particularly among gay, bisexual, and other men who have sex with men. • Studies indicate that doxycycline prophylaxis can significantly reduce the incidence of syphilis and chlamydia, but further research is needed to confirm efficacy. • Ongoing studies are exploring optimal doxycycline dosage, regimen, and formulation, as well as long-term safety and the potential for antimicrobial resistance. • Experts emphasize the need for more robust data on community acceptability, behavioral risk compensation, and cost-effectiveness before broad recommendations can be made.

RNA-Targeted Drug Discovery Summit to Explore Breakthrough Small Molecule Therapeutics

  • Leading pharmaceutical companies and academic institutions unite at the 2nd RNA-Targeted Drug Discovery Summit to advance novel RNA-targeted small molecule therapeutics development.
  • Industry pioneers from Pfizer, Novartis, Bristol-Myers Squibb, and other major players will address critical challenges in RNA targeting, including binding specificity and drug-like properties.
  • The summit aims to transform previously "undruggable" RNA targets into viable therapeutic opportunities through innovative approaches and RNA-protein interaction studies.

Eptinezumab Shows Promise in Preventing Chronic Migraine

A phase 2b clinical trial demonstrates that eptinezumab, an intravenous anti-calcitonin gene-related peptide monoclonal antibody, is effective and well-tolerated for the preventive treatment of chronic migraine, paving the way for phase 3 trials.

FDA Approves Pexidartinib as First Treatment for Tenosynovial Giant Cell Tumour

  • Daiichi Sankyo's pexidartinib (Turalio) becomes the first FDA-approved therapy for tenosynovial giant cell tumour (TGCT), offering a new treatment option beyond surgery.
  • Phase III ENLIVEN trial demonstrated a 38% overall response rate with pexidartinib, including 15% complete responses and 23% partial responses, compared to zero response in placebo group.
  • The approval includes a boxed warning for potential liver toxicity, requiring careful monitoring through a Risk Evaluation and Mitigation Strategy (REMS) program.

Phase 3 Study of Pexidartinib for Pigmented Villonodular Synovitis (PVNS) or Giant Cell Tumor of the Tendon Sheath (GCT-TS)

NCT02371369CompletedPhase 3
Daiichi Sankyo
Posted 5/11/2015

Study to Evaluate Discontinuation and Re-Treatment in Participants With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib

NCT04526704CompletedPhase 4
Daiichi Sankyo
Posted 10/20/2020

Glembatumumab Vedotin Fails to Meet Efficacy Endpoint in Recurrent Osteosarcoma Phase II Trial

  • Glembatumumab vedotin (GV) was evaluated in a Phase II trial for patients aged 12-50 with relapsed or refractory osteosarcoma.
  • The primary endpoint of disease control at 4 months was not met, with only one partial response and two cases of stable disease observed.
  • The antibody-drug conjugate was generally well-tolerated, with rash being the most common grade III adverse event, and toxicities similar to previous studies.
  • No correlation was found between glycoprotein non-metastatic B (gpNMB) expression and response to glembatumumab vedotin in this patient population.

Glembatumumab Vedotin in Treating Patients With Recurrent or Refractory Osteosarcoma

NCT02487979CompletedPhase 2
National Cancer Institute (NCI)
Posted 2/16/2016

Genentech Expands Drug Discovery Portfolio with Three Strategic Platform Partnerships

  • Genentech has secured partnerships with Convelo Therapeutics, Sosei Heptares, and Skyhawk Therapeutics to enhance its small-molecule drug discovery capabilities across multiple therapeutic areas.
  • The Sosei Heptares collaboration, valued at up to $1 billion, focuses on GPCR drug targets, while Convelo's partnership aims to develop remyelination therapies for multiple sclerosis.
  • Skyhawk Therapeutics partnership will explore RNA-splicing modulation for cancer and neurodegenerative diseases, expanding Genentech's reach into novel therapeutic approaches.

FDA Approves Merck's Recarbrio for Complex Infections with Antimicrobial Stewardship Focus

  • The FDA has granted approval for Recarbrio, Merck's novel triple-drug antibiotic combining imipenem/cilastatin with relebactam, for treating complicated urinary tract and intra-abdominal infections.
  • Health authorities emphasize Recarbrio should be reserved as a last-resort treatment option, specifically for cases where limited or no alternative antibacterial drugs are available.
  • The drug targets resistant gram-negative bacteria including Pseudomonas and Klebsiella species, with relebactam designed to overcome carbapenem resistance mechanisms.

Medidata CEO Outlines Vision for Clinical Trial Evolution: Balancing Innovation with Regulatory Compliance

  • Medidata's leadership emphasizes the need for measured innovation in clinical trials, highlighting the delicate balance between rapid advancement and patient safety in pharmaceutical development.
  • The company is actively engaging with regulatory bodies like FDA and EMA to implement patient-centric approaches, including e-consent solutions and synthetic control arms to improve trial efficiency.
  • Industry transformation is being driven by regulatory demands for comprehensive data, payer focus on value-based outcomes, and the emergence of potentially curative compounds requiring nimbler development processes.

Alder BioPharmaceuticals Presents New Data on Eptinezumab's Clinical Profile for Migraine Prevention

Alder BioPharmaceuticals has unveiled new data from post-hoc analyses of its PROMISE-1 and PROMISE-2 Phase 3 clinical trials for eptinezumab, highlighting its potential in preventing migraines. The data, focusing on migraine-free months, severity, and quality of life improvements, supports eptinezumab's clinical profile. The findings were presented at the American Headache Society’s 61st Annual Scientific Meeting.

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