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Clinical Trial News

EMA Panel Recommends Against Renewing Translarna Approval for Duchenne Muscular Dystrophy

  • The European Medicines Agency's CHMP has issued a negative opinion on renewing the conditional approval of Translarna for Duchenne muscular dystrophy (DMD).
  • PTC Therapeutics plans to request a re-examination of the decision, citing Translarna's established safety and efficacy profile in treating nonsense mutation DMD.
  • Translarna remains available in the EU while PTC seeks review, with a final decision expected after re-examination in January 2024.
  • The CHMP's decision was based on Phase 3 trial data that did not show significant improvement in the primary analysis population.

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

NCT03179631CompletedPhase 3
PTC Therapeutics
Posted 7/6/2017

Cloud-Based Data Analytics Emerges as Key Driver for Life Sciences Innovation and Drug Development

  • Life sciences organizations are facing unprecedented challenges in managing vast amounts of clinical, genomic, and image data, necessitating advanced analytics solutions for efficient drug development.
  • Cloud migration enables centralized data management and machine learning capabilities, significantly accelerating drug discovery processes and improving supply chain optimization.
  • Implementation of modern cloud-based platforms, supported by experienced partners, is revolutionizing critical business operations and enhancing decision-making across the pharmaceutical development lifecycle.

Axicabtagene Ciloleucel Shows Promise as Second-Line Therapy for Aggressive B-Cell Lymphoma

  • Axicabtagene ciloleucel (axi-cel) demonstrated a 71% complete metabolic response rate at 3 months in patients with relapsed or refractory aggressive B-cell lymphoma ineligible for stem cell transplant.
  • The study reported a manageable safety profile, with cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome being the most common adverse events.
  • At a median follow-up of 12 months, the estimated overall survival rate was 78.3%, suggesting a potential survival benefit with axi-cel in this patient population.
  • These findings support axi-cel as an effective second-line treatment option, particularly for older patients and those with comorbidities.

Alzheimer's Disease Treatment Advances: Lecanemab and Donanemab Offer New Hope

• Lecanemab (Leqembi) received full FDA approval in July 2023 and has shown success in slowing Alzheimer's progression, marking a significant milestone in treatment. • Eli Lilly's donanemab demonstrated a 35% reduction in cognitive decline by targeting amyloid protein, offering another promising treatment option for Alzheimer's. • These new drugs, while not cures, represent a crucial step forward, potentially leading to earlier diagnoses, increased awareness, and further research into Alzheimer's. • Challenges remain in ensuring broad access, managing costs, and establishing necessary infrastructure for diagnosis and treatment, particularly in low- and middle-income countries.

Moderna's Combo COVID-19 and Flu Vaccine Shows Promising Results in Early Trial

  • Moderna announced positive data from an early-stage study of its mRNA-based combination vaccine for COVID-19 and influenza, showing similar or stronger immune responses compared to standalone vaccines in older adults.
  • The company plans to begin Phase 3 trials of the combination vaccine later this year, with regulatory approval targeted for 2025, potentially simplifying the complicated vaccination schedule for respiratory diseases.
  • This development aligns with Moderna's broader respiratory franchise strategy, which expects $8-15 billion in sales by 2027 and includes successful Phase 3 results for standalone flu vaccine mRNA-1010.

Study of mRNA-1010 Seasonal Influenza Vaccine in Adults

NCT05827978CompletedPhase 3
ModernaTX, Inc.
Posted 4/17/2023

A Study of mRNA-1010 Seasonal Influenza Vaccine in Adults

NCT05415462CompletedPhase 3
ModernaTX, Inc.
Posted 6/6/2022

A Study of mRNA-1010 Seasonal Influenza Vaccine in Adults 50 Years Old and Older

NCT05566639CompletedPhase 3
ModernaTX, Inc.
Posted 9/14/2022

An Efficacy Study of Adjuvant Treatment With the Personalized Cancer Vaccine mRNA-4157 and Pembrolizumab in Participants With High-Risk Melanoma (KEYNOTE-942)

NCT03897881RecruitingPhase 2
ModernaTX, Inc.
Posted 7/18/2019

A Study to Evaluate the Safety and Efficacy of mRNA-1345 Vaccine Targeting Respiratory Syncytial Virus (RSV) in Adults ≥60 Years of Age

NCT05127434Active, Not RecruitingPhase 2
ModernaTX, Inc.
Posted 11/17/2021

Acelyrin's Izokibep Fails to Meet Primary Endpoint in Phase 2b/3 Psoriasis Trial, Stock Plummets

• Acelyrin's stock price plummeted after its investigational drug, izokibep, failed to achieve the primary endpoint in a Phase 2b/3 clinical trial for moderate-to-severe psoriasis. • The trial results were described as "painfully disappointing" by analysts, leading to a significant reduction in the company's price target by Jefferies. • Despite the setback, all five analysts covering Acelyrin maintain a "strong buy" rating, though the consensus price target is now significantly higher than the current stock price. • Izokibep, a potentially best-in-class IL-17A inhibitor, showed promise in previous trials, but this outcome raises concerns about its efficacy in psoriasis.

Acelyrin's Izokibep Fails to Meet Primary Endpoint in Phase 2b/3 Hidradenitis Suppurativa Trial

• Acelyrin's izokibep, an IL-17A inhibitor, did not meet the primary endpoint of HiSCR75 in a Phase 2b/3 trial for hidradenitis suppurativa (HS). • The HiSCR75 response rate at week 16 was 39% for izokibep (160 mg weekly) and 29% for placebo, failing to demonstrate a statistically significant treatment effect (p=0.3278). • Despite the primary endpoint failure, post-hoc analyses showed stronger efficacy signals related to HiSCR100, indicating potential for complete disease resolution. • Acelyrin is continuing to investigate izokibep in an ongoing Phase 3 trial for HS, based on observed HiSCR100 results and previous psoriatic arthritis data.

Hidradenitis Suppurativa Study of Izokibep

NCT05905783TerminatedPhase 3
ACELYRIN Inc.
Posted 6/22/2023

Hidradenitis Suppurativa Phase 2b Pivotal Study of Izokibep

NCT05355805CompletedPhase 2
ACELYRIN Inc.
Posted 5/5/2022

Acelyrin's Izokibep Fails to Meet Primary Endpoint in Phase 2b Hidradenitis Suppurativa Trial

• Acelyrin's izokibep did not meet the primary endpoint of HiSCR75 at week 16 in a Phase 2b trial for moderate-to-severe Hidradenitis Suppurativa (HS). • The trial showed early HiSCR100 responses and a dose-effect favoring 160mg weekly dosing, with no safety or tolerability limitations observed. • Despite the setback, analysts remain bullish about Acelyrin's stock, maintaining a consensus rating of Strong Buy with unanimous Buys. • Acelyrin plans to continue evaluating 160 mg QW dosing in HS and other indications, including uveitis and Psoriatic Arthritis (PsA).

SKYRIZI® (risankizumab) Demonstrates Superiority Over STELARA® (ustekinumab) in Crohn's Disease Head-to-Head Trial

  • Risankizumab met the primary endpoint of non-inferiority for clinical remission at week 24 compared to ustekinumab in Crohn's disease patients who failed anti-TNF therapy.
  • The study also met the primary endpoint of superiority in endoscopic remission at week 48 with risankizumab compared to ustekinumab.
  • All secondary endpoints achieved statistical significance for superiority with risankizumab versus ustekinumab, reinforcing SKYRIZI's effectiveness.
  • The safety profile of risankizumab was consistent with previous studies, with no new safety risks identified in the SEQUENCE trial.

A Study Comparing Risankizumab to Placebo in Participants With Active Psoriatic Arthritis Including Those Who Have a History of Inadequate Response or Intolerance to Biologic Therapy(Ies)

NCT03671148Active, Not RecruitingPhase 3
AbbVie
Posted 3/7/2019

A Multicenter, Randomized, Double-Blind, Placebo Controlled Induction Study to Evaluate the Efficacy and Safety of Risankizumab in Participants With Moderately to Severely Active Ulcerative Colitis

NCT03398148CompletedPhase 2
AbbVie
Posted 3/7/2018

Study Comparing Intravenous (IV)/Subcutaneous (SC) Risankizumab to IV/SC Ustekinumab to Assess Change in Crohn's Disease Activity Index (CDAI) in Adult Participants With Moderate to Severe Crohn's Disease (CD)

NCT04524611Active, Not RecruitingPhase 3
AbbVie
Posted 9/30/2020

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