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Clinical Trial News

Capivasertib Shows Promise in ER+/HER2- Metastatic Breast Cancer with PIK3CA Mutations

  • Capivasertib combined with fulvestrant demonstrates significant progression-free survival benefit in patients with AKT pathway-altered metastatic breast cancer, showing a median PFS of 7.3 months versus 3.1 months with placebo.
  • The phase 3 CAPItello-291 trial revealed capivasertib offers better tolerability compared to alpelisib, particularly regarding blood sugar management and rash occurrence in PIK3CA-mutated breast cancer.
  • Expert analysis suggests capivasertib as a preferred treatment option for PIK3CA-mutated breast cancer, with data showing an overall survival benefit with HR of 0.74 across all patients regardless of mutation status.

Capivasertib+Fulvestrant vs Placebo+Fulvestrant as Treatment for Locally Advanced (Inoperable) or Metastatic HR+/HER2- Breast Cancer

NCT04305496Active, Not RecruitingPhase 3
AstraZeneca
Posted 4/16/2020

Global Metabolic Disruption Discovered in SMA Patients: N-acetylneuraminic Acid Shows Treatment Potential

  • A case-control study reveals widespread metabolic disruptions in SMA patients, affecting nucleotide, lipid, carbohydrate, amino acid, and vitamin metabolism pathways.
  • Researchers identified 118 significantly altered metabolites in SMA patients' cerebrospinal fluid, with N-acetylneuraminic acid showing the most significant differentiation (P = .0000549).
  • The findings suggest N-acetylneuraminic acid could serve as a potential therapeutic agent for improving functional outcomes in SMA patients.

Apellis Faces Setback as EMA Rejects Pegcetacoplan, Mizuho Lowers Price Target

• Apellis Pharmaceuticals faces European market challenges as the EMA rejects pegcetacoplan (Syfovre) for regulatory approval, impacting potential revenue streams. • Mizuho Securities reduces Apellis' price target to $39, maintaining a Neutral rating due to the EMA's negative opinion and limited growth potential. • Despite the European setback, Apellis reports strong Q2 2024 growth, driven by robust sales of SYFOVRE and EMPAVELI in the United States. • Phase 3 VALIANT study shows Empaveli (pegcetacoplan) significantly reduces proteinuria in patients with rare kidney diseases, demonstrating potential beyond ophthalmology.

Biohaven Announces Topline Results of Troriluzole Trial in Spinocerebellar Ataxia

  • Biohaven's Study BHV4157-206-RWE assessed troriluzole's effectiveness in treating Spinocerebellar Ataxia (SCA) under FDA guidance.
  • Troriluzole is a novel glutamate modulator designed to reduce synaptic glutamate levels by enhancing glutamate uptake in glial cells.
  • The company will host a conference call on September 23 to discuss the topline data from the trial, providing further insights.
  • Biohaven is focused on developing treatments in immunology, neuroscience, and oncology, leveraging its drug development platforms.

Real-World Data Study of Troriluzole-Treated Patients With Spinocerebellar Ataxia (SCA) Compared to a Matched Natural History Control

NCT06529146Active, Not Recruiting
Biohaven Therapeutics Ltd.
Posted 3/18/2019

Novel Pan-Immune-Inflammation Nomogram Shows Promise in Predicting Epithelial Ovarian Cancer Outcomes

  • A new predictive model based on pan-immune-inflammation value (PIV) demonstrates strong accuracy in forecasting survival outcomes for epithelial ovarian cancer patients, with distinct differences between high and low PIV groups.
  • The study analyzed 576 Chinese patients, revealing significant survival differences - patients with low PIV showed better 3-year survival rates (76.71%) compared to those with high PIV (61.34%).
  • The nomogram successfully predicted both overall survival and progression-free survival, with area under the curve values reaching up to 0.839 in the training cohort.

Anlotinib Plus Penpulimab Shows Promise in Advanced Hepatocellular Carcinoma

  • The Phase III APOLLO study demonstrated that anlotinib combined with penpulimab significantly improved progression-free survival in patients with advanced hepatocellular carcinoma.
  • Overall survival was also significantly extended with the anlotinib-penpulimab combination compared to sorafenib, marking a notable advancement in HCC treatment.
  • The combination therapy showed a manageable safety profile, with treatment-related adverse events comparable to those observed with sorafenib alone.
  • Conducted in China, the study's findings suggest a potential new treatment option for HCC, particularly in populations with hepatitis B-related liver cancer.

Simvastatin Fails to Reduce Disability Progression in Phase 3 MS-STAT2 Trial for Secondary Progressive Multiple Sclerosis

  • The MS-STAT2 trial, a phase 3 study, evaluated simvastatin's effectiveness in slowing disability progression in patients with non-active secondary progressive multiple sclerosis (SPMS).
  • Results showed simvastatin did not significantly reduce disability progression compared to placebo over a 48-month period (HR 1.13, 95% CI 0.91-1.39, P = 0.26).
  • While simvastatin was safe and well-tolerated, the trial's primary and secondary outcomes did not demonstrate a benefit for SPMS, despite promising phase 2 results.
  • Researchers emphasize the trial's value in understanding progressive MS and enabling future large-scale clinical trials for emerging treatments.

Investigation of Simvastatin in Secondary Progressive Multiple Sclerosis

NCT00647348CompletedPhase 2
Imperial College London
Posted 1/1/2008

Multiple Sclerosis-Simvastatin Trial 2

NCT03387670Active, Not RecruitingPhase 3
University College, London
Posted 3/28/2018

UAMS Awarded $2.2 Million Grant to Investigate Efferocytosis in Retinopathy

  • The University of Arkansas for Medical Sciences (UAMS) has received a $2.2 million grant from the National Eye Institute (NEI).
  • The grant will fund research into how modulating the immune response through efferocytosis can benefit patients with retinopathy.
  • Dr. Abdel Fouda's lab will lead the study, exploring methods to enhance myeloid cell-mediated efferocytosis in treating retinopathy.
  • The research aims to develop new therapies for ischemic and trauma-induced retinopathy by understanding efferocytosis' role.

REDUCE-AMI Trial Re-evaluates Beta-Blocker Use Post-Myocardial Infarction

  • The REDUCE-AMI trial found no significant benefit in long-term beta-blocker treatment for patients with acute myocardial infarction (AMI) and preserved left ventricular ejection fraction (LVEF).
  • The study challenges current guidelines that widely recommend beta-blocker use post-AMI, regardless of LVEF, prompting a re-evaluation of treatment strategies.
  • Experts suggest a tailored approach to beta-blocker use, considering individual patient profiles, comorbidities, and potential side effects, especially in those with preserved LVEF.
  • The trial may lead to changes in clinical practice, with clinicians potentially reducing beta-blocker prescriptions in patients with preserved LVEF and no other indications.

CD Biopharma's CD-001 Receives FDA Clearance for Phase 1 Trial in Oncology

• CD Biopharma's CD-001, a bispecific fusion protein, has been cleared by the FDA for a Phase 1 clinical trial. • CD-001 targets PD-1-positive, CD8-positive T cells using an anti-PD-1 antibody and an engineered IL-21 mutant. • The Phase 1 trial will assess CD-001 as a potential treatment for solid tumors and hematologic malignancies. • Preclinical studies have demonstrated CD-001's effectiveness in mouse tumor models with good tolerance in non-human primates.

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