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Clinical Trial News

Fulcrum Therapeutics' Losmapimod Fails Phase 3 Trial in FSHD, Development Suspended

• Fulcrum Therapeutics' Phase 3 REACH trial of losmapimod in facioscapulohumeral muscular dystrophy (FSHD) did not meet its primary endpoint, showing no significant improvement in reachable workspace compared to placebo. • Secondary endpoints, including muscle fat infiltration and shoulder strength, also failed to achieve statistical significance in the losmapimod arm. • Fulcrum plans to suspend the losmapimod program and shift focus to pociredir for sickle cell disease and other early-stage programs. • The company's stock price plummeted following the announcement, raising concerns about its future prospects despite a solid cash position.

Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)

NCT05397470TerminatedPhase 3
Fulcrum Therapeutics
Posted 6/16/2022

Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT04003974CompletedPhase 2
Fulcrum Therapeutics
Posted 8/9/2019

Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)

NCT04264442TerminatedPhase 2
Fulcrum Therapeutics
Posted 2/13/2020

Biosimilar Switch in JIA Patients Shows Comparable Outcomes to Originator Maintenance

• A recent study indicates that switching children and young adults with juvenile idiopathic arthritis (JIA) from anti-TNF originators to biosimilars demonstrates similar disease activity control. • Drug persistence rates were comparable between patients who switched to biosimilars and those who remained on the originator anti-TNF therapy, suggesting similar long-term efficacy. • The study provides evidence supporting the safety and effectiveness of non-medical switching to biosimilars, offering reassurance to clinicians, patients, and families. • Injection-related issues were reported by some patients who discontinued biosimilars, with a portion switching back to the originator drug, highlighting the importance of patient support.

Aura Biosciences' Bel-Sar Shows Promise in Phase 2 for Early-Stage Choroidal Melanoma

• Bel-sar (AU-011) demonstrated an 80% tumor control rate in Phase 2 trial participants with early-stage choroidal melanoma. • The study showed a 90% visual acuity preservation rate among patients treated with bel-sar, offering a vision-sparing alternative to radiation. • The Phase 2 trial highlighted a favorable safety profile for bel-sar, with no serious adverse events reported during the study. • Aura Biosciences is advancing bel-sar into a Phase 3 trial, with potential to become a first-line treatment for choroidal melanoma.

Phase 2 Trial to Evaluate Belzupacap Sarotalocan (AU-011) Via Suprachoroidal Administration in Subjects With Primary Indeterminate Lesions and Small Choroidal Melanoma

NCT04417530CompletedPhase 2
Aura Biosciences
Posted 8/5/2020

Moderna Restructures Pipeline, Prioritizes Key Approvals, and Cuts R&D Spending

  • Moderna is reducing its research and development expenses by 20%, aiming for $16 billion between 2025 and 2028, to focus on profitability.
  • The company is prioritizing ten product approvals by 2027, including vaccines for COVID-19, flu, and RSV, while streamlining its pipeline.
  • Positive Phase 3 results were announced for Moderna's RSV vaccine in high-risk adults under 60, with plans to seek expanded FDA approval.
  • Moderna is delaying its break-even goal to 2028 and anticipates 2025 sales between $2.5 billion and $3.5 billion amid shifting market dynamics.

Zepbound Shows Greater Weight Loss Efficacy in Women, Study Finds

  • Tirzepatide, marketed as Zepbound, demonstrates consistent weight reduction in both women and men across multiple clinical trials.
  • Women experienced up to 25% reduction in initial body weight with tirzepatide, compared to 19% in men, indicating a sex-based difference.
  • The analysis pooled data from four trials involving nearly 3,000 women and 1,700 men with obesity, with or without type 2 diabetes.
  • While both sexes benefited, women reported higher rates of nausea and vomiting, but the overall safety profile remained similar.

Dimerix's Kidney Disease Drug Trial Reaches Key Pediatric Milestone

  • Dimerix has opened the first pediatric site in Mexico for its ACTION3 Phase 3 clinical trial, focusing on adolescent patients with focal segmental glomerulosclerosis (FSGS).
  • The FDA and EMA have approved the adult dosage of DMX-200 for adolescent patients aged 12-17, based on interim safety and pharmacokinetic data from the adult cohort.
  • Dr. Howard Trachtman, a pediatric nephrologist, has joined Dimerix's medical advisory board to support the ACTION3 study's expansion into pediatric patients.
  • Dimerix's ACTION3 Phase 3 clinical trial remains on track, with 116 patients out of the target 144 already randomized, and interim analysis expected around mid-2025.

Doherty Clinical Trials to Conduct Phase I Trial of Needle-Free Avian Influenza Vaccine

  • Doherty Clinical Trials initiates a Phase I clinical trial for a needle-free avian influenza vaccine developed by Vaxxas, assessing its safety and tolerability.
  • The trial utilizes Vaxxas' High-Density Microarray Patch (HD-MAP) technology, delivering the vaccine to immune cells just below the skin surface.
  • The study involves 258 healthy volunteers and aims to evaluate the potential for improved vaccine delivery, thermostability, and ease of self-administration.
  • This technology may offer a faster, broader vaccination response in future pandemics, reducing reliance on cold-chain refrigeration.

Neuraptive's NTX-001 Receives FDA Breakthrough Therapy Designation for Peripheral Nerve Injury

  • NTX-001, a drug-device combination by Neuraptive Therapeutics, has received Breakthrough Therapy Designation from the FDA for treating peripheral nerve injuries requiring repair.
  • The designation was granted based on promising Phase 2 NEUROFUSE study results, potentially accelerating NTX-001's development and review process.
  • Neuraptive is preparing to launch a Phase 3 pivotal program in early 2025, featuring a randomized, double-blind, placebo-controlled study.
  • Interim Phase 2 data showed significant improvement in MHQ total scores and reduced post-operative pain with NTX-001 compared to standard of care.

Alzheimer's Drugs May Work by Boosting Healthy Amyloid Beta Protein, Study Suggests

• New research suggests Alzheimer's drugs like lecanemab and donanemab may slow disease progression by increasing levels of healthy amyloid beta (Aβ42) protein in the brain. • The study challenges the prevailing theory that these drugs work solely by reducing toxic amyloid plaques, highlighting the potential importance of soluble Aβ42 in maintaining brain health. • Researchers analyzed data from nearly 26,000 patients, finding that increased levels of soluble Aβ42 correlated with slower Alzheimer's progression during treatment with these monoclonal antibodies. • The study authors propose alternative therapies focused on boosting soluble Aβ42 levels directly, rather than solely targeting amyloid plaques, which may have long-term risks.

CRISPR-Cas9 Corrects Dystrophin Duplications in DMD Patient Cells

  • Researchers used CRISPR-Cas9 to target intronic regions in the DMD gene in patient cells, aiming to delete duplicated regions.
  • The study confirmed restoration of the DMD open reading frame and rescued dystrophin expression after CRISPR-based deletion.
  • Off-target analysis revealed no significant unintended gene editing at predicted close-match off-target loci, enhancing safety profile.
  • This research presents a potential therapeutic avenue for DMD patients carrying duplications in exons 2-9, where no treatment exists.

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