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Clinical Trial News

ALS Clinical Trials: Masitinib and Edaravone Offer Hope After 20 Years of Failure

  • A review of ALS clinical trials over the past 20 years reveals a high failure rate, with riluzole remaining the only approved treatment for many years.
  • Masitinib, a tyrosine kinase inhibitor, has shown promise in Phase 3 trials by reducing neuroinflammation and slowing disease progression in ALS patients.
  • Edaravone, an antioxidant, received marketing authorization in Japan after demonstrating clinical efficacy in a Phase 3 trial with a specific ALS patient subgroup.
  • Both masitinib and edaravone represent potential new treatment options for ALS, though further research is needed to compare their efficacy and safety.

Phase 3 Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis

NCT01492686CompletedPhase 3
Mitsubishi Tanabe Pharma Corporation
Posted 12/1/2011

Masitinib in Combination With Riluzole for the Treatment of Patients Suffering From Amyotrophic Lateral Sclerosis (ALS)

NCT02588677CompletedPhase 2
AB Science
Posted 4/1/2013

Talampanel for Amyotrophic Lateral Sclerosis (ALS)

NCT00696332CompletedPhase 2
Teva Branded Pharmaceutical Products R&D, Inc.
Posted 9/1/2008

Efficacy and Safety Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis (ALS) Who Met Severity Classification III

NCT00415519CompletedPhase 3
Mitsubishi Tanabe Pharma Corporation
Posted 12/1/2006

Sandoz Marks Biosimilars Milestone with $1B Sales, Plans Five Major Launches by 2020

  • Sandoz achieved a significant milestone in 2016, surpassing $1 billion in biopharmaceutical sales and celebrating the 10th anniversary of Europe's first biosimilar commercialization with Omnitrope.
  • The company's biosimilar Zarxio has exceeded $100 million in sales and overtaken its reference product Neupogen in volume market share in the US market.
  • Sandoz is committed to launching five major biosimilars across EU and US markets by 2020, with ongoing development of complex proteins and monoclonal antibodies.

Novartis Leverages AI to Transform Drug Development Decision-Making Process

  • Novartis is implementing AI across drug discovery and development operations, using machine learning to analyze scientific literature and optimize molecule applications in disease treatment.
  • The pharmaceutical giant is utilizing AI to enhance clinical trial site selection by analyzing 10 years of historical trial data, moving away from subjective decision-making processes.
  • AI-powered natural language processing is being deployed to efficiently process over 1 million annual safety cases, accelerating regulatory reporting while maintaining human oversight.

NICE Rejects Alexion's Kanuma for Ultra-Rare LAL-D Treatment, Citing High Costs

• NICE has rejected Alexion's Kanuma (sebelipase alfa) for lysosomal acid lipase deficiency treatment, marking their second ultra-rare disease drug rejection in eight days.
• At £491,992 per patient annually, Kanuma becomes NICE's most expensive drug review to date, with the agency citing uncertain long-term benefits despite confidential discount offers.
• The decision affects approximately 25 patients in England with LAL-D, particularly impacting infants under six months who have no alternative treatment options.

Image Analysis Group and Alimentiv Form Strategic Partnership to Advance AI-Driven Gastroenterology Clinical Trials

AI
  • Image Analysis Group (IAG) and Alimentiv announced a strategic partnership to integrate IAG's DYNAMIKA AI platform into Alimentiv's comprehensive gastroenterology clinical trial services.
  • The collaboration introduces AI-driven histology endpoints and quantitative image analysis capabilities to enhance mucosal healing assessment and inflammation severity measurement in GI trials.
  • Alimentiv brings 30+ years of GI trial experience across 60+ countries, while IAG contributes proven AI technology validated through 700+ clinical trials since 2007.
  • The partnership aims to provide deeper insights into histologic changes and drug response at the cellular level, potentially accelerating gastroenterology drug development timelines.

A Study to Evaluate the Impact of Apremilast on Magnetic Resonance Imaging (MRI) Outcomes in Adults With Psoriatic Arthritis

NCT03783026CompletedPhase 4
Amgen
Posted 2/6/2019

MRD Negativity Predicts Superior Survival in Multiple Myeloma Patients

  • A meta-analysis of 14 studies involving 1,273 patients with multiple myeloma (MM) demonstrates that achieving minimal residual disease (MRD) negativity is associated with significantly better progression-free survival (PFS).
  • MRD-negative status also correlates with improved overall survival (OS) in MM patients, with a hazard ratio of 0.57 (95% CI: 0.46-0.71, P < .0001), indicating a substantial reduction in the risk of death.
  • The prognostic value of MRD status remains consistent regardless of the treatment type, suggesting that MRD assessment can be integrated into MM management to inform treatment decisions.
  • Achieving MRD negativity, even in patients who attain complete response (CR), predicts superior PFS and OS, establishing MRD status as a crucial endpoint in clinical trials and a surrogate marker for OS.

MabVax Therapeutics Expands Phase I Clinical Trial for Pancreatic Cancer Treatment

MabVax Therapeutics Holdings, Inc. has expanded its Phase I clinical trial for MVT-5873, a therapeutic antibody for pancreatic cancer, to include the HonorHealth Research Institute. The trial aims to evaluate the safety, tolerability, and pharmacokinetics of MVT-5873, both as a single agent and in combination with standard chemotherapy, in patients with metastatic pancreatic cancer.

Psilocybin Shows Promise in Reducing Depression and Anxiety in Cancer Patients

A study conducted by Johns Hopkins University School of Medicine found that a high dose of psilocybin, administered under supportive conditions, significantly decreased symptoms of depression and anxiety in cancer patients. These effects were sustained for up to six months, with participants reporting improved quality of life and decreased death anxiety.

Psychopharmacology of Psilocybin in Cancer Patients

NCT00465595CompletedPhase 2
Johns Hopkins University
Posted 4/1/2007

NICE Proposes Three-Tier Cost-Effectiveness Framework for Drug Evaluation in UK

• NICE has established a three-threshold system for drug cost-effectiveness, ranging from £20,000-30,000 per QALY for common diseases to £100,000 for ultra-orphan drugs.
• End-of-life treatments receive special consideration with a higher threshold of approximately £50,000 per QALY, reflecting society's prioritization of terminal care.
• The new £100,000 per QALY threshold for ultra-orphan drugs has sparked debate about evidence-based decision making and resource allocation in healthcare.

FDA Approves Rival Diabetes Combinations Xultophy and Soliqua for Type 2 Diabetes Management

  • The FDA has simultaneously approved two novel diabetes combination therapies: Novo Nordisk's Xultophy (insulin degludec/liraglutide) and Sanofi's Soliqua (insulin glargine/lixisenatide) for type 2 diabetes management.
  • Clinical trials demonstrated Xultophy's superior efficacy, with patients 4.5 times more likely to achieve glycemic targets without hypoglycemia compared to Lantus alone.
  • Both once-daily injectable combinations will enter the US market in early 2017, with Soliqua launching in January and Xultophy following in the first half of the year.

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