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Clinical Trial News

Biohaven's Taldefgrobep Alfa Shows Promise in Obesity Despite SMA Trial Setback

  • Biohaven's taldefgrobep alfa failed to meet the primary endpoint in a Phase 3 SMA trial, showing no statistically significant improvement in motor function compared to placebo.
  • Subgroup analysis revealed clinically meaningful improvements in motor function among Caucasian patients with measurable myostatin levels.
  • The trial demonstrated significant reductions in body fat mass and increases in lean muscle mass, prompting Biohaven to advance taldefgrobep alfa for obesity treatment.
  • Biohaven plans to initiate a Phase 2 placebo-controlled trial for taldefgrobep alfa in obesity, utilizing a user-friendly, self-administered autoinjector.

A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy

NCT05337553Active, Not RecruitingPhase 3
Biohaven Pharmaceuticals, Inc.
Posted 7/6/2022

Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

NCT03039686CompletedPhase 2
Hoffmann-La Roche
Posted 7/6/2017

Small Cell Lung Cancer Subtypes Identified, Paving Way for Personalized Treatment

  • Researchers have identified four distinct subtypes of small cell lung cancer (SCLC): SCLC-A, SCLC-N, SCLC-P, and SCLC-I, each with unique characteristics.
  • A blood-based assay has been developed to classify SCLC subtypes, offering a faster and less invasive alternative to traditional biopsies.
  • Immune checkpoint inhibitors show greater benefit for SCLC-I subtype patients, suggesting subtype-specific treatment approaches are warranted.
  • Clinical trials are planned to assign patients to therapies based on their SCLC subtype, comparing these personalized approaches to the standard of care.

Incyte Highlights New Tafasitamab Data in Follicular Lymphoma at ASH 2024

  • Incyte will present new data from its oncology portfolio at the 2024 American Society of Hematology (ASH) Annual Meeting in San Diego.
  • A late-breaking oral presentation will feature results from the Phase 3 inMIND study evaluating tafasitamab in relapsed or refractory follicular lymphoma (FL).
  • Incyte will host a virtual analyst and investor event on December 12, 2024, to discuss key data presentations from ASH.

A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease

NCT06388564RecruitingPhase 2
Incyte Corporation
Posted 10/11/2024

MAXPIRe: Study to Evaluate Axatilimab in Participants With Idiopathic Pulmonary Fibrosis (IPF)

NCT06132256RecruitingPhase 2
Syndax Pharmaceuticals
Posted 12/11/2023

ManagingLife Launches Digital Psychology Program for Post-Surgical Pain Management

  • ManagingLife introduced a digital psychology program integrated into its Manage My Pain platform, aimed at helping individuals manage post-surgical pain and reduce opioid dependence.
  • The program consists of four 15-minute self-guided modules based on Acceptance and Commitment Therapy (ACT) principles, designed to improve coping skills.
  • A randomized-controlled trial (RCT) led by Dr. Hance Clarke and Dr. Max Slepian has commenced to evaluate the intervention's effectiveness in reducing pain intensity and disability.
  • The digital intervention addresses the shortage of pain psychologists, offering a scalable and accessible solution for comprehensive pain management.

European Commission to Cover PharmaMar's Costs in Plitidepsin Case Against EMA

  • The General Court of the European Union ruled the Aplidin case as without object after the European Commission (EC) revoked its initial decision regarding plitidepsin marketing authorization.
  • The EC acknowledged a conflict of interest during plitidepsin's evaluation, involving an expert from the Scientific Advisory Group who was also a chief investigator at XNK Therapeutics, a company developing a rival product.
  • The EU General Court ordered the EC to bear its own costs and to pay PharmaMar's costs in proceedings before the General Court of the EU and the Court of Justice of the EU, highlighting unfairness in the initial evaluation.
  • The EMA will re-evaluate plitidepsin for Multiple Myeloma at its own discretion, and PharmaMar will demand impartiality, equality, transparency, and compliance during the reassessment process.

BridgeBio's Attruby Gains Analyst Confidence with Promising Launch Outlook

• BridgeBio's Attruby receives positive analyst ratings due to its early FDA approval for ATTR-CM and potential for widespread adoption. • Attruby's competitive pricing, 10% lower than tafamidis, and favorable insurance coverage are expected to drive market penetration. • Analyst surveys indicate strong interest in Attruby, with over 30% of first-line patients likely to be treated with the drug. • BridgeBio is also advancing gene therapy programs for Canavan disease and Limb-girdle Muscular Dystrophy, diversifying its pipeline.

Cassava Sciences' Simufilam Fails in Phase 3 Alzheimer's Trial, Program Halted

  • Cassava Sciences' Phase 3 trial of simufilam in mild-to-moderate Alzheimer's disease did not meet its co-primary endpoints, assessing cognition and function at 52 weeks.
  • The company has decided to discontinue its second Phase 3 trial, ReFocus-ALZ, and the Open Label Extension study following the disappointing results.
  • Simufilam had been under scrutiny due to allegations of data manipulation, leading to a $40 million settlement with the SEC over misleading statements.
  • The failure represents a setback for Alzheimer's research and is particularly disheartening for patients who participated in the trials, highlighting the challenges in developing effective treatments.

NIH's TrialGPT AI Algorithm Streamlines Clinical Trial Matching

  • The NIH has developed TrialGPT, an AI algorithm designed to accelerate matching potential volunteers to clinical trials listed on ClinicalTrials.gov.
  • TrialGPT identifies relevant clinical trials, explains how individuals meet enrollment criteria, and ranks trials by relevance and eligibility.
  • A study showed TrialGPT achieved nearly the same accuracy as clinicians in assessing patient eligibility, while reducing screening time by 40%.
  • The AI tool aims to improve clinical trial enrollment, expedite medical research, and reduce barriers to participation for underrepresented populations.

JNJ-2113 Shows Promise for Psoriasis, Infigratinib for Achondroplasia, and Gildeuretinol Gains FDA Support for Stargardt Disease

  • Phase 3 trials of JNJ-2113 demonstrate potential as a first-in-class oral peptide targeting the IL-23 receptor for moderate-to-severe plaque psoriasis in patients aged 12 years and older.
  • Infigratinib shows statistically significant increases in annualized height velocity and improved body proportionality in children with achondroplasia in a Phase 2 study.
  • Gildeuretinol receives Rare Pediatric Disease and Fast Track designations from the FDA for Stargardt disease, an inherited condition causing vision loss in children and young adults.

Study of Infigratinib in Children with Achondroplasia

NCT04265651CompletedPhase 2
QED Therapeutics, Inc.
Posted 3/10/2020

A Study of JNJ-77242113 in Adolescent and Adult Participants With Moderate to Severe Plaque Psoriasis

NCT06095115Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 10/12/2023

A Study of JNJ-77242113 for the Treatment of Participants With Plaque Psoriasis Involving Special Areas (Scalp, Genital, and/or Palms of the Hands and the Soles of the Feet)

NCT06095102Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 10/12/2023

Merck's Winrevair Shows Promise in Pulmonary Hypertension; Cassava's Alzheimer's Drug Fails Trial

• Merck's Winrevair significantly reduced the risk of death, lung transplantation, or disease-related hospitalizations in pulmonary arterial hypertension patients, meeting its Phase 3 trial endpoint. • Cassava Sciences' experimental treatment, sumifilam, failed to slow cognitive or functional decline in Alzheimer's patients compared to placebo in a Phase 3 study. • The FDA approved BridgeBio's Attruby for transthyretin amyloid cardiomyopathy (ATTR-CM), demonstrating a 42% reduction in mortality and cardiovascular hospitalizations. • Research indicates a potential link between Merck's asthma drug, Singulair, and severe mental health issues, reinforcing previous FDA warnings.

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