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Clinicaltrials are expensive, slow and often limited by outdated design constraints. They offer patient-specific outcome predictions, generated using machine learning models trained on real historical clinical data. Accelerating timelines and de-risking development decisions across the pipeline.”
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.
Advanced strategies and tools are being implemented to establish the safety and efficacy of new therapeutic modalities, with the development of new biomarkers becoming one of the most relevant approaches for enhancing the precision and utility of early-stage studies.
Deep Dive Library Events Press Releases Topics Sign up Search Sign up Search Pharma Biotech FDA ClinicalTrials Deals Drug Pricing Gene Therapy An article from Dive Brief Actithera draws new investors to radiopharma drug pitch The four-year-old biotech raised about $75 million in a Series A round that involved nine venture capital firms.
While two approved treatments, pirfenidone and nintedanib, slow disease progression, there currently is no treatment that reverses the effects or offers a cure for IPF. However, recent advancements and strategic approaches in clinicaltrials offer hope that additional treatments are on the way.
In just two years, CTMC has advanced eight therapies into clinicaltrials, harnessing genetic engineering to enhance T-cell effectiveness in the fight against cancer. Ive been involved in therapeutics development for over 25 years, working with small, medium, and large biotech companies.
At the forefront of this transformation is Dr Ebrahim Delpassand, a nuclear medicine physician and the driving force behind RadioMedix (RMX), a radiopharmaceutical company focused on developing targeted diagnostics and therapies. The founding of RadioMedix was born from a recognition of unmet needs in cancer treatment.
Real-world data paired with machine learning is a game changer in drug development RWD is becoming increasingly important in influencing the drug development landscape, particularly when used to develop the natural history and patient journey through disease.
Like many aspects of life in the 21st century, this era of scientific advancement and increased partnership is largely driven by next-generation technologies that are streamlining workflows, breaking down silos and empowering scientists to extract the most prescient insights from their data throughout the drug development process.
Discovered by Daiichi Sankyo, I-DXd is being co-developed with Merck (known as MSD outside of North America) as part of a broader strategic collaboration between the two companies. Addressing a Pressing Clinical Need in Prostate Cancer Prostate cancer remains one of the most prevalent malignancies among men worldwide.
For these patients, clinical onset means severe symptoms and a rapid decline in overall health. Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis. The DeLLphi-304 study was reported at ASCO earlier this month.
This shift raises ethical questions around prioritizing supply for diabetes patients versus those seeking treatment for obesity or cosmetic weight loss. You can unsubscribe at anytime. The balance is complex, as obesity carries its own serious health risks. Informa PLCs registered office is 5 Howick Place, London SW1P 1WG. TechTarget, Inc.s
The encouraging data builds on the foundation established by earlier studies and further strengthens the clinical case for MR-141 as a potential non-invasive, once-daily ophthalmic solution for millions affected by the age-related decline in near vision. According to the company, MR-141 met its primary endpoint with statistical significance.
Neuropsychiatric treatment is on the verge of a major transformation. Historically, treatment options have been limited, with patients relying on daily medications that have minimal efficacy and troublesome side effects. “Zelquistinel is a positive modulator of NMDA receptors,” Donello explains.
Ionis Begins Pivotal Phase 3 REVEAL Study of ION582 in Angelman Syndrome, Dosing First Patient in Global Trial Ionis Pharmaceuticals , Inc. Currently, treatment options are limited to supportive care aimed at managing symptoms rather than targeting the underlying cause.
This study explored the effects of beginning treatment with lower starting doses of MariTide to improve tolerability while maintaining clinical benefit. Both data sets now provide foundational insight into the design and direction of Amgen’s upcoming Phase 3 MARITIME clinicaldevelopment program.
More than 7,000 rare diseases – conditions that affect fewer than 1 in 2,000 people – have been identified, and1 in 17 people will develop a rare disease in their lifetime, with children disproportionately affected. Most of these conditions are genetic in origin and the majority have no effective treatment.
It involves a dynamic and often unpredictable process where every stage, from target identification to clinicaltrials, generates vast amounts of data. Additionally, AI-driven predictive modelling can shorten the preclinical phase by simulating biological responses, leading to more targeted and efficient clinicaltrials.
Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments. Highlighting data integration. This is an AI generated image.
The results come from a open-label, proof-of-concept, Phase 2 study (NCT04520451) and highlight rilzabrutinib’s potential as a disease-changing treatment option for a condition that currently has limited and non-specific treatment options and involves substantial patient suffering due to its chronic and progressive course.
In March 2025 , Regulus announced the successful completion of its Phase 1b multiple-ascending dose clinicaltrial for farabursen. Additionally, treatment led to improvements in height-adjusted total kidney volume (htTKV) , an established clinical marker that correlates with disease progression in ADPKD.
Developingtreatments for individuals living with rare diseases is critical, but orphan drug development is laden with unique obstacles that necessitate innovative, multifaceted approaches. Moreover, in some rare disease trials, it is unethical to design a control group of patients with a placebo.
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.
The study marks a significant step forward in the treatment landscape for haemophilia A, demonstrating not only clinical safety and pharmacokinetic stability, but also strong patient preference for the Mim8 pen-injector delivery system. In the trial, participants were directly switched to Mim8 without a washout period.
Despite significant progress, there are more than 6,500 rare diseases without a single approved treatment. As a provider of end-to-end clinicaldevelopment solutions, we plan to leverage our expertise to refine drug-disease matches identified by Every Cure’s use of AI and data analysis.
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.
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.
The trial, which will explore the pharmacokinetics and safety of ATX01 in healthy volunteers, is due to start in January 2021. AlgoTherapeutix recently raised a 12M€ Series A that will fund the Phase 1 and 2 clinicaldevelopment of ATX01. CIPN is a leading cause for modification or interruption of chemotherapy.
Currently, treatment options for patients with relapsed or refractory disease remain limited, and prognosis is poor. Standard single-target CD19-directed CAR T-cell therapies typically enable long-term remissions in roughly 40% of patients, emphasizing a significant unmet need for new and more effective treatment strategies.
However, the FDA’s recent decision to remove the Risk Evaluation and Mitigation Strategy (REMS) requirements for certain approved Chimeric Antigen Receptor T-cell (CAR T) therapies could dramatically shift these numbers, making life-saving treatments more accessible to those in need.
The results from the Phase I/II NXTAGE trial, announced during the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C., The trial is evaluating NXT007 in people with hemophilia A without factor VIII inhibitors who have not previously been treated with Hemlibra.
As the biotech sector races to improve the tolerability of these revolutionary treatments, Poolbeg Pharma has a novel solution: an oral therapy, POLB 001, to block the development of CRS, by limiting inflammation without affecting the anti-cancer immune responses that are vital for effective immunotherapies.
The data were revealed during an oral presentation (S137) at the 2025 European Hematology Association (EHA) Congress in Vienna, reflecting ongoing progress in developing new treatment options for patients battling this aggressive hematologic malignancy.
Dapirolizumab Pegol Demonstrates Promise in Phase 3 SLE Trial, Showing Improvements in Fatigue and Disease Activity at EULAR 2025 UCB (Euronext Brussels: UCB) and Biogen Inc. The latest data from the PHOENYCS GO trial reinforce the potential of dapirolizumab pegol to improve disease outcomes in people with moderate-to-severe SLE.
To keep a pulse on this rapidly changing industry, the PPD clinical research business of Thermo Fisher Scientific surveyed 150 leaders in the biopharma and biotech industries for the second consecutive year. Differences in size, resources and agility have led the two groups to take different approaches to clinicaldevelopment.
Instead of the standard approach where studies are designed to address a primary focus, a parallel study method — a format that incorporates multiple endpoints and objectives, modifications, treatment arms and dose selections into a single protocol — is being rapidly adopted.
The decision follows an analysis of topline data from a Phase 1b clinical study, which demonstrated encouraging safety and efficacy results in patients with DOK7-CMS, one of the more severe and prevalent subtypes of this ultra-rare neuromuscular disorder. Detailed data from the trial will be shared at an upcoming medical conference.
Clissold — The trio of CDER, CBER, and CDRH released a new draft guidance titled “ Use of Data Monitoring Committees in ClinicalTrials ” that revises the 2006 guidance “Establishment and Operation of ClinicalTrial Data Monitoring Committees” and, when final, will replace the 2006 guidance.
1428321-10-1 Pritelivir mesylate is an antiviral drug currently under development, specifically targeting herpes simplex virus types 1 and 2 (HSV-1 and HSV-2). It is being investigated as a potential treatment for various herpes infections, including those resistant to traditional antivirals like acyclovir.
Bicara has focused ficerafusp alfa clinicaldevelopment on head and neck squamous cell carcinoma (HNSCC), adding pembrolizumab to separately block the PD-1 pathway. No chemotherapy regimen is used in the trial. Another clinical program that has making headlines in HNSCC is petosemtamab, an anti-EGFR x LGR5 bispecific antibody.
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.
This integrated strategy allows BeOne to move seamlessly from early discovery through proof-of-concept to pivotal clinicaltrials, reducing development timelines and enhancing scalability. To date, the company has conducted more than 170 clinicaltrials across 40 countries, enrolling over 25,000 patients.
FXR-targeted drugs have shown promise in late-stage clinicaltrials for non-alcoholic steatohepatitis. Cilofexor is one of few remaining FXR agonists in clinicaldevelopment. The farnesoid X receptor (FXR) is a nuclear receptor that controls bile acid, lipid, and cholesterol metabolism.
Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to riliprubart , an investigational immunology therapy developed by Sanofi, for the treatment of antibody-mediated rejection (AMR) in solid organ transplantation.
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