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Early-phase clinicaltrials, including phase 1 and some phase 2a studies, serve as a pivotal step for biotech companies, laying the foundation for a drug candidate’s journey to market.
Selecting the right CRO to partner with on your development program is a pivotal decision that can shape the trajectory of your product’s development. Our consultative approach ensures success by integrating research methodology, regulatory affairs strategy, and clinical operations.
In the trial, HU6 treatment also led to a substantial decline in liver, visceral and total body fat while preserving skeletal muscle mass. A Phase IIa trial was recently launched that enrolled obese subjects with HFpEF while a Phase IIb trial in obesity, including a subset of obese subjects with type 2 diabetes, is planned to begin next year.
Despite advancements in neuroscience and psychiatry, clinicaltrials for mental health conditions such as major depressive disorder (MDD), schizophrenia, bipolar disorder and generalized anxiety disorder face unique challenges. One of the main challenges lies in defining and selecting appropriate trial populations.
Clinicaldevelopment organizations face a wide array of challenges when it comes to data, many of which can impact the operational effectiveness of their clinicaltrials. In this whitepaper, experts from Revvity Signals explore how solutions like TIBCO® Spotfire® enable better, more streamlined studies.
In the trial, platelet response, defined as achieving at least 50,000 platelets per microliter or doubling baseline counts, was seen in 65 percent of patients on rilzabrutinib, compared to 33 percent on placebo. Participants received either rilzabrutinib or placebo for 24 weeks, followed by an open-label extension.
Global rare disease clinicaltrials face unique challenges due to small patient populations, complex logistics, diverse regulatory environments and cultural differences. ClinicalTrial Manager/Sr. These measures enhance recruitment, retention and overall trial success.
The surge in MASH cases stresses the need for early diagnosis, timely intervention and precise, reliable methodologies in clinicaltrials to evaluate new therapies effectively. Read on to gain insights from Medpace experts about advancements in imaging, histology and innovative techniques for MASH clinicaltrials.
Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinicaltrials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
Amidst a shifting clinical landscape characterized by increasingly complex trial designs and growing patient subpopulations, many contract research organizations (CROs) have adopted a “one-stop-shop” strategic approach. Moreover, these delays impact getting potentially lifesaving therapies to patients in need.
Biotechnology company Adcentrx Therapeutics has raised $38m in Series A+ financing to advance its pipeline of new antibody-drug conjugate (ADC) therapeutics into clinicaldevelopment. ADRX-070 is the company’s lead candidate and is anticipated to enter a first-in-human Phase I clinicaltrial in the second half of 2023.
On February 7, at a town hall organised to discuss clinicaltrial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases. GlobalData is the parent company of Pharmaceutical Technology.
Patients are the backbone of clinicaltrials, playing an essential role in the drug development process. This engagement is often less understood and is underutilized by sponsors, meaning a significant element of the trial and drug experience is missed during sponsor engagement with the FDA.
Last year, we witnessed a significant milestone in oncology clinicaldevelopment, with almost 50% of all development programs focused on oncology assets. To encourage further expansion and innovation in cancer studies, it’s critical to consider what contributes to success in an oncology trial.
Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.
This process can be daunting, but understanding how to manage feedback effectively is crucial for developing and ultimately gaining approval for new therapies, especially in oncology clinicaltrials.
In the ever-evolving landscape of pharmaceutical development, the complexity of early phase clinicaltrials is increasing. This surge in complexity is driven by escalating drug development costs, heightened competition and advanced scientific innovations. This is the storm that we find ourselves in the midst of.”
In clinical research, diversity is more than a prerequisite for a successful drug — it’s a critical component that enhances the validity and applicability of clinicaltrial outcomes. Diversity Action Plans have now moved from being a “nice-to-have” to a “must-have” in clinicaltrial designs.
The traditional clinicaltrials model is facing scrutiny. VivoSense , a pioneering company in the field of digital health technologies (DHTs) and digital endpoints for clinicaltrials, is aiming to enhance the efficiency and efficacy of clinical research. Integrating DHTs Across Drug Development Phases Dr.
Upstream Bio will use the funds to advance its UPB-101 into Phase II trials to treat asthma and chronic rhinosinusitis with nasal polyps (CRSwNP). It is also preparing for a swift transition to Phase III development. It targets the human thymic stromal lymphopoietin receptor to inhibit signalling.
It’s even being used to review investigator invoices and automate financial reconciliations—a monthly headache every contract research organization, sponsor, and clinicaltrials operator will be more than happy to resign to machines. MGI puts the value of this contribution to clinicaldevelopment at $15 billion to $25 b illion.
Stephanie Manson Brown, VP & Head of ClinicalDevelopment & Scientific Innovation of R&D at Allergan Aesthetics at AbbVie. She also spoke about clinicaltrials in the space, including Allergan Aesthetics’ commitment to increasing diversity in trials and developing products that do not cater to any one beauty standard.
Regulatory bodies such as the FDA oversee clinicaltrials to ensure that studies’ design, conduction, analysis, and reporting are per established guidelines and laws. Any delays or missteps in bioanalysis during a Phase I trial can derail the trajectory of a promising drug.
A successful gene therapy trialed at Michigan State University in dogs with an inherited eye disease is ready to be developed for clinical use in human patients with a rare condition called retinitis pigmentosa.
Eli Lilly shared new Phase II trial data showing that its experimental siRNA therapy, lepodisiran, leads to reductions in levels of lipoprotein(a) (Lp(a)) a genetically inherited risk factor for heart disease in adults with elevated levels of the marker. The 400 mg dose was the highest lepodisiran dose in the trial.
A new CTTI publication , published in Clinical Pharmacology & Therapeutics , reviews current applications of disease progression modeling (DPM) and opportunities to advance the awareness and value of DPM in clinicaltrials.
Author: Lona Sheeran, SVP, Clinical Operations, Early Phase At this year’s ClinicalTrials Nexus, I had the privilege of representing Worldwide ClinicalTrials as the sole CRO on a panel discussion: “Reversing the Conversation: What the ClinicalTrial Industry Really Wants from its Service Providers.”
In this episode, Ayesha spoke with Mario Marcondes, MD, PhD, VP Head of ClinicalDevelopment at Nektar Therapeutics , a company focused on the discovery and development of novel therapies for autoimmune disorders and cancer. Dr. Marcondes is a licensed and trained physician, specializing in oncology and immune oncology.
Rebyota’s safety and efficacy were analysed in the largest trial programme in the microbiome-based therapeutics field, including five trials enrolling over 1,000 subjects. The latest development is based on the findings from the clinical programme including the double-blind, randomised, placebo-controlled Phase III PUNCH CD3 trial.
The VLA1553-321 trial, conducted in Brazil, was the first to assess the vaccine in an endemic region. Importantly, the vaccine was well tolerated, with no safety concerns identified during the trial. Valneva is also advancing its investigational Lyme disease vaccine candidate, VLA15, currently in Phase II clinicaldevelopment.
Gildeuretinol is the first and only medicine in clinicaldevelopment to treat Stargardt disease by reducing vitamin A dimerization in the eye, without affecting normal vision. It decreased vitamin A dimerization by more than 80% and prevented blindness in a genetic animal model of the disease in preclinical trials.
BHV-8000’s mechanistic proof of concept was showed in several pre-clinical models. This year, Biohaven is expecting to advance the TYK2/JAK1 inhibitor into a Phase I trial. Hangzhou Highlightll will also receive up to $950m in development and commercial milestone payments and tiered royalties.
The post Novotech Sponsors Endpoints ASCO 2022 Expert Panel on Accelerating Oncology ClinicalTrials in China appeared first on Pharma Mirror Magazine. According to the Endpoints ASCO session: “China and the US are the world’s largest oncology research destinations, and the.
The FDA Oncology Center of Excellence (OCE) is committed to further enhancing oncology research and as a result, has recently implemented several projects aimed to revolutionize oncology clinicaltrials. However, Project Optimus emphasizes the importance of dose-finding studies in early clinicaldevelopment.
It is a third-generation, selective and structurally unique, small molecule CGRP receptor antagonist and is currently under clinicaldevelopment with oral and intranasal formulations. The regulator granted the approval based on two pivotal placebo-controlled, double-blind, randomised trials.
Memo Therapeutics AG will receive CHF 10.5m (US $11.4m) from the Swiss government to clinicallydevelop a SARS-CoV-2 antibody against COVID-19, allowing it to start Phase 1 studies in Q1, 2022.
It is currently being assessed in combination with Merck’s anti-PD-1 therapy, Keytruda, as an adjuvant treatment for high-risk melanoma patients in a Phase II clinicaltrial being conducted by Moderna. The company said that the data from the ongoing Phase II trial will be reported in the fourth quarter of this year.
SinoMab BioScience intends to commence a Phase I clinicaltrial in China to evaluate SM17’s safety profile. The company dosed the first healthy participant in a Phase I clinical first-in-human (FIH) clinicaltrial in June 2022.
The artificial intelligence-driven drug discovery company has dosed its first healthy volunteer in a trial for a candidate to treat idiopathic pulmonary fibrosis.
The approval is based on comprehensive data from the clinicaldevelopment programme of Roctavian, including two-year findings from the international Phase III GENEr8-1 clinicaltrial.
As the strategists for Worldwide ClinicalTrials’ rare disease sponsors, we design customized solutions that make a difference in achieving successful outcomes. This philosophy is encapsulated in our belief: “If you’ve seen one trial for a rare indication, you’ve seen one trial.”
TAK-755 is the first and only treatment in clinicaldevelopment that provides targeted replacement of ADAMTS13, addressing the underlying cause of the disease. “We The Phase III trial assessed TAK-755’s clinical benefit across several clinically relevant endpoints, against plasma-based therapies, in a randomised cross-over study.
In November 2023, at Outsourcing ClinicalTrials Dach in Zurich, our Executive Director, Oncology Strategy Lead, Matt Cooper , presented “Delivering Oncology Studies – Challenges and Considerations.” Umbrella Trial: Examines numerous drugs administered as individual drugs or combinations in a single tumor type.
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