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Clinical Trial News

EU Confirms Rejection of Apellis' Pegcetacoplan for Geographic Atrophy

  • The European Medicines Agency (EMA) has upheld its rejection of Apellis' pegcetacoplan (Syfovre) for treating geographic atrophy (GA).
  • The decision leaves millions of Europeans without a treatment option for this irreversible form of blindness, despite support from the retina community.
  • CHMP cited concerns that while Syfovre slows lesion growth, it doesn't translate to clinically meaningful benefits for patients' daily lives.
  • Apellis remains committed to expanding access to pegcetacoplan in the U.S. and other regions, where it is already approved.

FDA Approves AstraZeneca's Fasenra for Expanded EGPA Treatment

  • AstraZeneca's Fasenra has received FDA approval for expanded use in treating eosinophilic granulomatosis with polyangiitis (EGPA).
  • The approval is expected to boost AstraZeneca's revenue, building on an 18% increase in the first half of the year.
  • Fasenra's expanded indication offers a new treatment option for EGPA patients, addressing a critical unmet need.
  • AstraZeneca is focusing on launching at least 20 new molecular entities by the end of the decade, indicating a strong pipeline.

Beacon Therapeutics' AGTC-501 Shows Sustained Safety and Efficacy in X-Linked Retinitis Pigmentosa Trial

  • Beacon Therapeutics presented 36-month interim data from its Phase I/II Horizon trial of AGTC-501 for X-linked retinitis pigmentosa, showcasing a strong safety profile.
  • The trial results indicate that AGTC-501 continues to be well-tolerated, with no clinically significant safety events related to the gene therapy reported.
  • Promising efficacy signals and continued durability were observed up to 36 months post-dosing, which is encouraging for an early-stage trial.
  • Beacon is advancing AGTC-501 through Phase II and Phase II/III trials, utilizing an optimized dose and improved manufacturing process.

GPRC5D Emerges as Promising Target in Multiple Myeloma Clinical Trials

  • Recent clinical trials exploring GPRC5D-targeted therapies demonstrate significant potential for treating relapsed/refractory multiple myeloma patients.
  • The MajesTEC-1 and MagnetisMM-3 trials show encouraging long-term follow-up data, highlighting the durability of response with GPRC5D-directed treatments.
  • Researchers are actively investigating adverse event management strategies, particularly for CRS and ICANS, to optimize patient outcomes in GPRC5D therapy.

Paternal Use of Valproate Not Linked to Increased Birth Defect Risk, Study Suggests

  • A new review suggests that fathers taking the epilepsy drug valproate do not pose a significant risk of birth defects or developmental disorders in their children.
  • Researchers found scarce and inconsistent evidence linking paternal exposure to anti-seizure medications, including valproate, to adverse offspring outcomes compared to unexposed controls.
  • The study questions regulatory restrictions on valproate use for men, as it may lead to less effective medication choices and increased risks of disease and sudden unexpected death in epilepsy (SUDEP).
  • The authors emphasize the need for further research to investigate the potential reproductive implications of anti-seizure medication exposure in males.

Novel Strategies Emerge to Transform 'Cold' Tumors into Immunotherapy-Responsive 'Hot' Tumors

  • Only 20% of cancers currently respond well to immunotherapy, with "hot" tumors like melanoma and lung cancer showing better outcomes than "cold" tumors such as pancreatic and brain cancers.
  • Innovative approaches including bispecific antibodies, engineered T-cells, and combination therapies are showing promise in converting immunologically "cold" tumors into "hot" ones, potentially expanding immunotherapy's reach.
  • Recent breakthroughs include tarlatamab's approval for small cell lung cancer with 50% response rates in some groups, and promising results from neo-antigen vaccines in pancreatic cancer treatment.

CSPC Pharmaceutical Receives Approval for New Hypertension Drug Trials

  • CSPC Pharmaceutical has received approval to commence clinical trials for its novel hypertension drug, marking a significant step in its development program.
  • The new drug aims to address unmet needs in hypertension management, potentially offering an alternative treatment option for patients.
  • The approval allows CSPC Pharmaceutical to proceed with evaluating the drug's safety and efficacy in human subjects, advancing it through the clinical trial phases.

Yoga and Exercise Show Promise in Reducing Urinary Incontinence in Women

  • A recent study indicates that yoga and exercise can significantly reduce urinary incontinence episodes in women, offering a non-pharmacological alternative.
  • The research showed a 65% reduction in incontinence episodes among participants practicing yoga for 12 weeks, comparable to medication effectiveness.
  • Both yoga and standard exercise regimens demonstrated substantial improvements, highlighting the benefits of physical activity for bladder control.
  • The study emphasizes the accessibility and safety of yoga as a low-risk intervention for managing urinary incontinence and improving overall well-being.

Antidepressant Use for Pain Relief in Seniors Lacks Evidence, Posing Risks

  • A new review indicates limited evidence supports the use of antidepressants for pain relief in older adults, raising concerns about potential overuse.
  • Researchers found that seniors using antidepressants for pain had a higher risk of falls, dizziness, and injury compared to those using other methods.
  • International guidelines supporting antidepressants for chronic pain are primarily based on data from younger patients, potentially leading to misinformed prescribing decisions for seniors.
  • Duloxetine (Cymbalta) showed some efficacy for knee osteoarthritis pain over a few months, but benefits were small and must be weighed against risks.

Horsemanship Program Eases PTSD Symptoms in Combat Veterans

  • A recent study reveals that combat veterans participating in an eight-week horsemanship program experienced a reduction in PTSD symptoms and improved mental well-being.
  • The program involved veterans learning basic horse safety and behavior, grooming horses, and leading them, promoting present-moment focus and reducing hypervigilance.
  • Researchers observed decreased stress hormone concentrations in veterans and preliminary evidence of co-regulation between veterans and horses.
  • Alternative therapies like horsemanship are valuable due to high dropout rates from traditional PTSD treatments among military veterans.

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