MedPath

Clinical Trial News

FDA Panel Supports Eflornithine for High-Risk Pediatric Neuroblastoma

• The FDA's Oncologic Drugs Advisory Committee (ODAC) voted 14-6 in favor of eflornithine to reduce relapse risk in pediatric high-risk neuroblastoma patients. • The vote was based on data from Study 3b, a single-arm trial with an external control arm from the ANBL0032 trial, showing improved event-free survival. • Eflornithine's safety profile includes manageable toxicities like hearing loss, with dose adjustments potentially mitigating adverse effects. • While some panel members expressed concerns about using an externally controlled trial, the overall consensus was that the benefits outweigh the risks.

Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission

NCT02395666CompletedPhase 2
Giselle Sholler
Posted 3/5/2015

Isotretinoin With or Without Dinutuximab, Aldesleukin, and Sargramostim Following Stem Cell Transplant in Treating Patients With Neuroblastoma

NCT00026312Active, Not RecruitingPhase 3
National Cancer Institute (NCI)
Posted 10/18/2001

Paradigm and Bristol Myers Squibb Collaborate to Enhance Clinical Trial Access and Efficiency

  • Paradigm and Bristol Myers Squibb are partnering to create a new clinical trial model focused on improving patient access, accelerating trial completion, and easing provider burden.
  • The collaboration aims to design trial protocols that align with the capabilities of community health providers, who serve diverse patient populations.
  • Paradigm's software platform will be leveraged to expand clinical research programs at community health organizations, offering clinical trials as a care option to more patients.
  • The initiative seeks to address the underrepresentation in clinical trials, where currently less than 5% of cancer patients participate, hindering the development and availability of new treatments.

Landmark UK Biobank Study Reveals 14,000 Novel Gene-Protein Interactions to Transform Drug Discovery

  • The Pharma Proteomics Project has mapped over 14,000 gene-protein associations in 54,000 individuals, with 81% being previously undiscovered, creating a groundbreaking dataset for drug development.
  • The study revealed significant findings about common health conditions, including elevated inflammatory proteins in depression patients, and demonstrated proteins' ability to predict biological markers accurately.
  • A collaborative effort between 13 pharmaceutical companies and UK Biobank will make this comprehensive proteomic data freely available to researchers worldwide, advancing precision medicine development.

Expanded Access Programs: Bridging the Gap Between Clinical Trials and Patient Need

  • Expanded Access Programs (EAPs) provide critical pathways for patients with severe conditions to access investigational drugs when clinical trials are not an option, operating under FDA oversight to ensure benefits outweigh risks.
  • EAPs serve multiple stakeholders: offering hope to patients, providing real-world data for researchers, and helping pharmaceutical companies build relationships with healthcare communities.
  • While EAPs face challenges including equity concerns and resource limitations, efforts are underway to streamline processes and improve accessibility while maintaining scientific and ethical standards.

Semaglutide Shows Sustained Benefits in Blood Sugar Control and Weight Loss Over Three Years

  • A recent study presented at the EASD demonstrates that semaglutide significantly improves blood sugar control and promotes weight loss over a three-year period.
  • The research, involving over 23,000 patients, showed a reduction in HbA1c levels and an average weight loss of 4.7 kilograms after six months of semaglutide treatment.
  • Semaglutide's long-term effectiveness highlights its potential as a valuable treatment option for managing type 2 diabetes and obesity, especially for those struggling with glycemic control.
  • Experts emphasize that while semaglutide offers significant benefits, it should be part of a comprehensive approach that includes lifestyle modifications and close monitoring by healthcare professionals.

Eli Lilly Acquires Point Biopharma for $1.4 Billion to Expand Radioligand Therapy Portfolio

Oncology
  • Eli Lilly announced the acquisition of Point Biopharma for $1.4 billion, paying $12.50 per share representing a 67% premium to strengthen its oncology pipeline with radioligand therapies.
  • The deal provides Lilly access to Point's lead asset PNT2002, a PSMA-targeted radioligand therapy in Phase III trials for metastatic castration-resistant prostate cancer with data expected in late 2023.
  • Point's pipeline includes PNT2003 for gastroenteropancreatic neuroendocrine tumors and earlier-stage assets, positioning Lilly to compete with established players like Novartis in the radioligand therapy space.
  • The acquisition represents Lilly's strategic entry into radiopharmaceuticals, which deliver targeted radiation directly to cancer cells while minimizing damage to healthy tissue.

Novavax COVID-19 Vaccine Receives FDA Emergency Use Authorization for 2023-2024 Formula

• The FDA has granted Emergency Use Authorization (EUA) for Novavax's COVID-19 vaccine (NVX-CoV2601) for individuals aged 12 and older. • The updated vaccine targets current variants, including XBB.1.5, XBB.1.16, and XBB.2.3, offering a protein-based, non-mRNA option. • Non-clinical data showed the vaccine induced neutralizing antibody responses and robust T-cell responses against emerging subvariants. • The Novavax vaccine is expected to be available at thousands of locations nationwide, providing a broader range of options for vaccination.

VALBIOTIS SA: Phase II/III REVERSE-IT Study to be Presented at 2023 EASD Congress

The Phase II/III REVERSE-IT study, conducted by Valbiotis in partnership with Nestlé Health Science, has been selected for an oral presentation at the 2023 European Association for the Study of Diabetes (EASD) congress. The study focuses on the efficacy of TOTUM•63, a non-drug active substance, in addressing early glycemic disorders.

FDA Greenlights Alzamend Neuro's Phase IIA Trial of Novel Lithium Therapy AL001 for Bipolar Disorder

  • Alzamend Neuro has received FDA clearance to proceed with a Phase IIA clinical trial of AL001, a next-generation lithium therapeutic for bipolar disorder type 1, with patient dosing expected in Q1 2024.
  • AL001 delivers lithium via a novel ionic cocrystal technology that may eliminate the need for therapeutic drug monitoring while providing improved brain distribution and reduced organ exposure compared to conventional lithium salts.
  • The development program may qualify for an expedited 505(b)(2) FDA approval pathway, potentially accelerating the availability of this improved treatment option for the approximately 7 million Americans affected by bipolar disorder.

Parkinson's Drug DNL-151 Phase III Trial Terminated; Lerodalcibep Shows Promise in Hypercholesterolemia

• Denali Therapeutics' DNL-151 Phase III trial for Parkinson's disease was terminated, leading to a 14-point decrease in its Likelihood of Approval (LoA). • LIB Therapeutics' lerodalcibep showed positive Phase III data in heterozygous familial hypercholesterolemia (HeFH), increasing its LoA by 11 points to 65%. • AstraZeneca's MEDI-7352 Phase IIb trial in osteoarthritis pain was completed, resulting in a six-point increase in its Phase Transition Success Rate (PTSR) to 45%. • Incyte's itacitinib saw its PTSR increase in cytokine release syndrome (CRS) after a Phase II trial completion, jumping by nine points to 60%.

A Study of Itacitinib for the Prevention of Cytokine Release Syndrome Induced by Immune Effector Cell Therapy

NCT04071366CompletedPhase 2
Incyte Corporation
Posted 2/7/2020

Study to Assess the Efficacy and Safety of LIB003 in HeFH Patients on Oral Lipid Therapy Needing Further LDL-C Reduction

NCT04797104CompletedPhase 3
LIB Therapeutics LLC
Posted 4/22/2021

A Study to Assess if BIIB122 Tablets Are Safe and Can Slow Worsening of Early-Stage Parkinson's Disease in Participants With Specific LRRK2 Genetic Variants Between the Ages of 30 and 80 Using the Movement Disorder Society-Unified Parkinson's Disease Rating Scale

NCT05418673TerminatedPhase 3
Biogen
Posted 8/26/2022

A Study of the Efficacy and Safety of MEDI7352 in Participants With Painful Osteoarthritis of the Knee

NCT04675034CompletedPhase 2
AstraZeneca
Posted 12/2/2020

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.