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Clinical Trial News

Summit Therapeutics Secures $235 Million to Advance Ivonescimab Clinical Trials

  • Summit Therapeutics has raised $235 million through a private stock sale to fund clinical trials for ivonescimab, an investigational cancer treatment.
  • The financing involved the sale of over 10 million shares at $22.70 each, matching the closing stock price on the announcement day.
  • Major internal figures and leading biotech investors participated, signaling strong confidence in the company's lead drug candidate.
  • The funds will support the clinical development of ivonescimab and other corporate initiatives, with shares subject to SEC registration for resale.

Florey Institute to Accelerate MND Drug Discovery with Novel iPSC Screening Platform

  • The Florey Institute receives $5 million to advance drug screening for Motor Neurone Disease (MND) using a novel iPSC-based platform.
  • The platform uses iPSCs from over 100 MND patients to screen potential treatments by assessing their ability to keep motor neurons alive.
  • Initial screenings accurately mirrored clinical trial outcomes, showing 95% of previously tested drugs were ineffective, validating the platform's accuracy.
  • This technology aims to improve the efficiency of MND drug trials by filtering out ineffective drugs early, potentially revolutionizing drug discovery for sporadic MND.

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 Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)

NCT04264442TerminatedPhase 2
Fulcrum Therapeutics
Posted 2/13/2020

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

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.

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