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Clinical Trial News

Decentralized Clinical Trials Show Promise in Remote Data Collection and Patient-Centric Research

  • A Phase I pharmacokinetic trial demonstrates the feasibility and benefits of decentralized clinical trials (DCTs) for remote data collection.
  • DCTs improve patient access, representation, and enrollment, while also enhancing the speed and quality of data collection.
  • Patient feedback indicates a preference for self-collection methods and remote consent, highlighting the potential for increased patient engagement.
  • Challenges remain in implementing DCTs, including the need for digital workflows, comprehensive data sets, and optimized patient recruitment.

Efficacy and Safety of Tanimilast in Asthmatics uNcontrolled on ICS-containinG backgrOund Maintenance Therapy

NCT06029595Active, Not RecruitingPhase 2
Chiesi Farmaceutici S.p.A.
Posted 11/26/2023

FDA Rejection of MDMA Therapy for PTSD Triggers Industry Reprioritization

  • The FDA rejected Lykos Therapeutics' MDMA-assisted therapy for PTSD, requesting an additional Phase III trial due to concerns about efficacy and safety.
  • The rejection has led some psychedelic drug developers to reassess their pipelines, with some prioritizing other assets over MDMA-based treatments.
  • Companies like Tactogen are focusing on novel compounds, while atai Life Sciences is prioritizing MDMA for social anxiety disorder, awaiting further developments with Lykos.
  • Experts highlight the high unmet need for effective PTSD treatments, with some advocating for the potential of psychedelic therapies despite regulatory hurdles.

Belantamab Mafodotin, Pomalidomide, and Dexamethasone Show Promise in Triple-Class Exposed Multiple Myeloma

  • A phase 1/2 trial evaluated belantamab mafodotin, pomalidomide, and dexamethasone (B-Pd) in patients with triple-class exposed relapsed/refractory multiple myeloma.
  • The combination therapy demonstrated an overall response rate of 59% in heavily pretreated patients, indicating notable efficacy.
  • The median progression-free survival was 11.5 months, suggesting a clinically meaningful benefit in this challenging patient population.
  • Ocular toxicities, a known side effect of belantamab mafodotin, were managed with dose modifications and did not preclude continued treatment.

Praxis Precision Medicines Sees Positive Analyst Outlook Following Pipeline Progress

  • Praxis Precision Medicines receives reiterated Buy rating and $120 price target from H.C. Wainwright after positive Phase 2 data for relutrigine and favorable reviews for PRAX-628.
  • The company's advancements in epilepsy portfolio, including pivotal readout for PRAX-628 and Essential3 trial progress, position it for significant growth in the next year.
  • EMBOLD Phase 2 study results showed a 46% reduction in motor seizures in DEE patients, with over 30% experiencing up to 28 days of seizure freedom.
  • Analysts from Oppenheimer and Guggenheim express optimism, raising price targets and reaffirming Buy ratings, highlighting the potential of Praxis Precision's relutrigine.

CAR-T Therapy Does Not Increase Risk of Secondary Cancers, Study Finds

  • A new study suggests CAR-T therapy does not elevate the risk of secondary malignancies compared to standard cancer treatments.
  • Researchers reviewed data from over 5,500 patients, finding similar rates of secondary cancers in CAR-T and non-CAR-T groups.
  • The study challenges current warning labels on CAR-T therapies, which may unnecessarily intimidate patients.
  • The findings indicate that the benefits of CAR-T therapy outweigh the minimal risk of developing T-cell malignancies.

Novo Nordisk's Oral Amycretin Shows Promising Weight Loss in Early Trials

  • Amycretin, an oral drug developed by Novo Nordisk, led to significant weight loss in overweight or obese individuals during a Phase 1 trial.
  • Participants taking amycretin experienced up to 13% body weight reduction over 12 weeks, surpassing the effects of a placebo.
  • The drug combines GLP-1 and amylin receptor agonism, offering a convenient pill form for obesity management.
  • Further trials are underway to assess the long-term safety and efficacy of amycretin for weight management.

Experimental Blood Test Shows Promise in Predicting COPD and Lung Disease Risk

  • A novel blood test analyzes 32 proteins to predict the likelihood of rapid lung function decline, offering a potential tool for early intervention.
  • Individuals with higher test scores showed an 84% increased risk of COPD and an 81% increased chance of mortality from respiratory diseases.
  • The test demonstrated a 17% increased risk of needing hospital care for respiratory issues and a 10% increased risk of treatable respiratory symptoms.
  • Further clinical trials are needed to validate the test before it can be implemented in clinical practice and approved by the FDA.

enGene Appoints Dr. Raj Pruthi as SVP to Advance EG-70 in Urologic Oncology

  • enGene Holdings Inc. has appointed Dr. Raj Pruthi as Senior Vice President, Urologic Oncology and Clinical Development, to support the advancement of EG-70.
  • Dr. Pruthi will focus on the clinical development of EG-70, a non-viral, intravesical genetic medicine, across various urologic applications.
  • EG-70 is currently in a pivotal study for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC), addressing a critical unmet need.
  • Dr. Pruthi will engage with the urological community to further the development and understanding of EG-70's potential benefits.

FDA Approves Cobenfy, a Novel Schizophrenia Treatment Targeting Cholinergic Receptors

  • The FDA has approved Cobenfy (xanomeline and trospium chloride) as the first new class of drug for schizophrenia in over 30 years, offering a novel approach to treatment.
  • Cobenfy targets muscarinic receptors, unlike traditional antipsychotics that focus on dopamine, potentially reducing side effects like weight gain and movement disorders.
  • Clinical trials demonstrated Cobenfy significantly reduced schizophrenia symptoms compared to placebo, marking a transformative moment in managing this challenging condition.
  • Expected to launch in late October, Cobenfy offers a new option for adults with schizophrenia, with ongoing studies exploring its potential in Alzheimer's psychosis and other conditions.

A Study to Evaluate Safety and Tolerability of CVL-231 (Emraclidine) in Adult Participants With Schizophrenia

NCT05443724Active, Not RecruitingPhase 2
AbbVie
Posted 9/2/2022

Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-1117568 in Adults With Schizophrenia

NCT05545111RecruitingPhase 2
Neurocrine Biosciences
Posted 10/4/2022

A Trial of 15 and 30 mg Doses of CVL-231 (Emraclidine) in Participants With Schizophrenia

NCT05227703CompletedPhase 2
AbbVie
Posted 6/30/2022

A Study to Assess Safety and Efficacy of KarXT in Adult Patients With Schizophrenia

NCT03697252CompletedPhase 2
Karuna Therapeutics
Posted 9/18/2018

A Trial of 10 and 30 mg Doses of CVL-231 (Emraclidine) in Participants With Schizophrenia

NCT05227690CompletedPhase 2
AbbVie
Posted 6/30/2022

DRP-104 (Glutamine Antagonist) in Combination With Durvalumab in Patients With Advanced Stage Fibrolamellar Carcinoma (FLC)

NCT06027086RecruitingPhase 1
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Posted 2/12/2024

A Study to Assess Efficacy and Safety of KarXT in Acutely Psychotic Hospitalized Adult Patients With Schizophrenia (EMERGENT-3)

NCT04738123CompletedPhase 3
Karuna Therapeutics
Posted 4/6/2021

A Study to Assess Efficacy and Safety of KarXT in Acutely Psychotic Hospitalized Adult Patients With Schizophrenia (EMERGENT-2)

NCT04659161CompletedPhase 3
Karuna Therapeutics
Posted 12/16/2020

A Study to Assess Efficacy and Safety of Adjunctive KarXT in Subjects With Inadequately Controlled Symptoms of Schizophrenia

NCT05145413CompletedPhase 3
Karuna Therapeutics
Posted 11/12/2021

HARMONIE Trial Reimagines Commercial Clinical Trial Design with Nirsevimab

  • The HARMONIE trial investigated the impact of nirsevimab on hospitalizations related to respiratory syncytial virus (RSV) in infants.
  • Conducted across France, Germany, and the UK, the trial adopted a pragmatic approach to assess nirsevimab's effectiveness.
  • Professor Saul Faust will present the trial's innovative design and execution at an upcoming PCT Grand Rounds session.
  • The study aims to provide insights into optimizing the design and delivery of commercial clinical trials.

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