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However, as we note in that post, the design, timing of initiation, and timely conduct of confirmatory trials are also important considerations in FDAs determination of whether accelerated approval is appropriate. This blog post focuses on interpreting these new authorities with respect to timely conduct of confirmatory trials.
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome.
Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).
These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. Participants were classified as WHO Group 1 PAH, functional class (FC) II or III, indicating moderate to severe disease at either intermediate or high risk of clinical deterioration.
Clinical Trials: Phase 3 clinical trials have shown that nerandomilast can slow lung function decline in patients with IPF and PPF. Efficacy: The trials demonstrated that nerandomilast led to a smaller decline in forced vital capacity (FVC), a measure of lung function, compared to placebo. SCHEME 1H NMR (400 MHz, DMSO-D6) 1.57–1.84
It is becoming increasingly evident that generative artificial intelligence (GenAI) is a resourceful tool for helping pharmaceutical companies reduce manual tasks required by clinical trials. This is especially relevant with today’s heavier focus on enhancing personalised medicine via broader emerging scientific findings.
Phase 2 Study: Transformative Weight Loss and Glycemic Control The Phase 2 trial focused on evaluating the efficacy and safety of MariTide in individuals with obesity, both with and without Type 2 diabetes (T2D). The data revealed compelling weight loss outcomes , particularly when compared to placebo.
The trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options. The most common adverse events were mild to moderate in intensity and did not differ significantly from known safety patterns.
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.
Biogen Launches Global Phase 3 PROMINENT Trial Evaluating Felzartamab in Primary Membranous Nephropathy Biogen Inc. The trial is expected to complete in 2029. By selectively depleting these cells, felzartamab offers a promising, mechanism-driven approach to potentially halting disease progression.
has announced encouraging interim findings from the pivotal Phase 3 VISIONARY trial evaluating sibeprenlimab in adults with Immunoglobulin A nephropathy (IgAN), a rare and progressive autoimmune kidney disorder. Treatment-emergent adverse events (TEAEs) occurred in 76.3% in collaboration with Otsuka Pharmaceutical Co.,
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.
Aimee Shu, Executive Vice President of Endocrine & Rare Disease Medical Sciences and Chief Medical Officer at Ascendis Pharma, expressed optimism about the interim results. The Week 26 data from the COACH Trial now show that TransCon hGH may further enhance these benefits. mg/kg/week) during the trial.
Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.
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 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 Untitled Letter notes that the confirmatory trial for LYTGOBI is currently ongoing and has not been completed; therefore, clinical benefit of LYTGOBI has not yet been confirmed. As a single-arm trial (i.e., DCR is defined as the sum of complete response (CR), partial response (PR) and stable disease (SD).
This data includes results from two pivotal studies: Part C of the ongoing DEVOTE clinical trial and the final eight-year data from the NURTURE study. SMA, a rare and often devastating genetic neuromuscular disease, primarily affects motor neurons in the spinal cord, resulting in progressive muscle wasting and weakness.
EVEREST is the first-ever trial to demonstrate the superiority of Dupixent over Xolair on both nasal polyp endpoints and related symptoms in patients with co-existing asthma, while retaining a generally similar safety profile. Serious adverse events were somewhat lower in Dupixent (2%) than in omalizumab (4%).
The new data presented at EHA reflect the growing understanding that inhibiting this complex, in combination with standard-of-care treatments, may produce deep and durable responses in patients with poor-risk disease. Our aim is to bring forward a new era of treatment for a disease that desperately needs new and more effective options.”
Achieving the Primary Endpoint: Sotyktu Demonstrates Superiority Over Placebo The POETYK PsA-1 trial, officially designated as study IM011-054, enrolled patients with active PsA who had not received prior treatment with biologic disease-modifying antirheumatic drugs (bDMARDs).
Over 70% of new patients are diagnosed with stage IV disease, also called extensive stage disease (ES-SCLC). Tarlatamab triggered an ORR of ~40% in refractory (second-line or later) SCLC patients in the DeLLphi-301 clinical trial published in 2023. 4 patients (33%) had grade 4 adverse events. months (versus 20% ORR and 8.3
Cardiovascular Insights from the XYLO Trial One of the most notable late-breaking presentations was the Phase 4 XYLO study, which examined the cardiovascular benefits of switching patients from high-sodium, twice-nightly oxybate therapy to the same dosage of low-sodium Xywav over a six-week period. According to Dr. Richard J. Source link
Currently, treatment options for patients with relapsed or refractory disease remain limited, and prognosis is poor. Director of Lymphoma Research at Sarah Cannon Research Institute and principal study investigator for the trial. The most frequently encountered form within this group is diffuse large B-cell lymphoma (DLBCL).
Liso-cel achieved high, lasting response rates in patients with relapsed or refractory marginal zone lymphoma, underscoring its potential to significantly improve patient outcomes in this hard-to-treat disease,” said M. Neurologic events (NE) of any grade were documented in 33% of patients, with Grade 3 NEs in 4% and no Grade 4 or 5 NEs.
Love Employee via Getty Images For decades now, scientists have been experimenting with a new way to target and destroy proteins linked to disease. It may also be a more efficient way to shut down known disease targets. Kymera Therapeutics is working on protein degraders for a wide range of immune diseases, and some cancers.
argenx Unveils Positive Phase 2 Results for Efgartigimod in Myositis and Sjogren’s Disease at EULAR 2025 argenx SE a global immunology company dedicated to advancing treatments for severe autoimmune diseases, presented encouraging new clinical data at the 2025 European Congress of Rheumatology (EULAR), held June 11–14 in Barcelona, Spain.
The double-blind treatment period lasted 24 weeks, during which both efficacy and adverse events (AEs) were closely monitored. Primary Endpoint Met: Improved Tolerability with Atogepant At the core of the TEMPLE study was the investigation of treatment discontinuation rates due to adverse events, a key measure of real-world drug tolerability.
This new monoclonal antibody therapy is designed to prevent lower respiratory tract disease caused by respiratory syncytial virus (RSV) in neonates and infants born during or entering their first RSV season. Dr. Octavio Ramilo, Chair of the Department of Infectious Diseases at St. Key results from the CLEVER trial include: A 60.5%
Durable Benefit and Positive Clinical Outcomes “Today’s results underscore the promising potential of Intellia’s approach to genome-editing therapy — a one-time treatment that has been well tolerated and offers a highly differentiated, durable effect for patients suffering from a serious disease,” said John Leonard, M.D.,
In an era where clinical trials are increasingly global, it’s more imperative than ever to leverage international expertise. Data and safety monitoring boards (DSMBs), also known as data monitoring committees (DMCs), play a critical role in overseeing a clinical trial’s safety and efficacy.
The challenge of GPCR drug discovery G protein-coupled receptors (GPCRs) are one of the most desirable and challenging target classes in drug discovery, as their mutation can lead to a wide range of diseases such as cancer, cardiovascular disorders and neurological conditions.
For some, however, disease management becomes even more precarious due to impaired hypoglycemia awareness and a heightened risk of severe hypoglycemic events (SHEs). The elimination of SHEs — combined with improved glycemic control and insulin independence — marks the successful achievement of the trial’s primary Phase 1/2 endpoint.
The study, which is the largest to date that focuses exclusively on this patient population, shows high overall response rates (ORR) alongside deep and durable responses, offering much-needed hope for individuals whose disease typically carries a poor prognosis. Head of Myeloma Unit at Tel-Aviv Sourasky Medical Center in Israel.
The updated results, presented during a significant oral presentation at the 30th European Hematology Association (EHA) 2025 Congress, highlight the strong activity and tolerability of this combination therapy in a population with high-risk disease. Importantly, there were no treatment-related discontinuations due to adverse events (TEAEs).
Importantly, both cases of ALF have been reported exclusively in non-ambulatory individuals with Duchenne muscular dystrophy — a population that typically bears a heavy disease burden — underscoring the need for careful and tailored approaches to their care. The FDA agrees with this cautious approach.
The failure rate in clinical trials exceeds 90%, often due to insufficient safety data, efficacy concerns, or regulatory non-compliance. Even drugs that complete clinical trials may face delays or rejections if submission documents are incomplete or do not align with regulatory expectations.
By adjusting the activity of this receptor, these drugs can influence brain functions associated with mood, cognition and pain, positioning them as potential treatments for conditions like depression, cognitive disorders and neurological diseases. “We call this phenomenon ‘event-driven pharmacology.”
A Groundbreaking Step in HER2-Targeted Therapy for BTC The EC’s decision was primarily driven by results from the Phase 2b HERIZON-BTC-01 trial , the largest of its kind in this population. The trial evaluated Ziihera as a monotherapy in 87 previously treated patients with HER2-positive BTC. formerly BeiGene, Ltd.)
1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] Fitusiran 1711.0g/mol, 1] It is an antithrombin-directed small interfering ribonucleic acid. [1]
These medications have become central components of chronic disease management globally, particularly in light of rising rates of obesity and type 2 diabetes. Wegovy® : A higher-dose injectable approved for long-term weight management in adults with obesity or overweight and related comorbidities. Source link
Grounded in HELIOS-B: A Landmark Phase 3 Study The EC’s decision was primarily based on positive results from the HELIOS-B Phase 3 trial , a pivotal, randomized, double-blind, placebo-controlled study that enrolled a broad and representative sample of patients living with ATTR-CM.
ISSUE 44 — The Importance of Cytokine Release Assays to Derisk the Development of Immunomodulatory Drugs pmjackson Mon, 07/07/2025 - 21:31 New immunomodulatory drugs have the potential to cause serious immune-related adverse events, including cytokine release syndrome (CRS).
Importantly, this will enable them to produce mRNA vaccines during periods between health crises, retaining trained personnel, technical expertise, and production capacity while retaining the ability to respond quickly in the event of future disease outbreaks.
Effective immediately, Roche is halting the administration of Elevidys to non-ambulatory DMD patients in both commercial and clinical trial settings. ” Duchenne muscular dystrophy is a rare, X-linked, degenerative muscle disease predominantly affecting boys.
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