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Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. subsidiary of Merck KGaA, is the founder of radioligand therapy developer Actithera. You can unsubscribe at anytime.
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).
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. There are several types of biomarkers to consider.
Study Overview and Population The HYPERION study was designed to assess the efficacy and safety of WINREVAIR when used in combination with standard background therapy, compared to placebo, in adult patients recently diagnosed with pulmonary arterial hypertension. It follows two earlier landmark studies: STELLAR and ZENITH.
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. Among patients who had previously undergone two or more lines of therapy (n = 12), the ORR was 92% and the CRR was 75%.
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 trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options.
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. Because LLMs are trained on extensive, internet-scale datasets, they can learn to identify contexts linking words and language.
These results further validate the potential of Ascendis’ proprietary TransCon technology to deliver long-acting therapies that improve both efficacy and patient experience. The Week 26 data from the COACH Trial now show that TransCon hGH may further enhance these benefits. mg/kg/week) during the trial.
These pivotal findings were unveiled at the 85th Scientific Sessions of the American Diabetes Association (ADA) and were concurrently published in The New England Journal of Medicine , underscoring the significant promise of MariTide in the growing field of anti-obesity therapies.
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.
Otsuka Unveils Promising Phase 3 Results for Sibeprenlimab in IgA Nephropathy, Marking Significant Proteinuria Reduction and Advancing a Novel APRIL-Targeted Therapy Otsuka Pharmaceutical Development & Commercialization, Inc., Treatment-emergent adverse events (TEAEs) occurred in 76.3% in the placebo group.
While these therapies hold great promise for improving cancer treatment outcomes, their development presents significant challenges, especially in achieving the optimal balance between efficacy and safety. It also enables a more targeted approach to ADC optimisation, minimising the need for trial-and-error experimentation.
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.,
Details of the VEGA-3 Phase 3 Trial The VEGA-3 study is part of Viatris’ broader clinical development program to evaluate MR-141 for presbyopia. The trial was conducted as a randomized, placebo-controlled, double-blind study and enrolled a total of 545 adult participants.
In the trial, participants were directly switched to Mim8 without a washout period. Strong Safety Profile Reinforces Mim8’s Potential Mim8’s safety profile was a central focus of the FRONTIER5 trial. Throughout the 26-week study period, the investigational therapy was well-tolerated.
They have interesting patterns of expression in different cancer indications; thus, diverse therapies for attacking these targets have been developed. SCLC generally has a “hot” tumor microenvironment (TME) meaning that it is infiltrated by immune cells, including the T cells that are activated by immune checkpoint therapies.
The trial evaluated 125 patients with relapsed or refractory AML, as well as those who were newly diagnosed but ineligible for intensive chemotherapy — all with KMT2A-rearrangements or NPM1 mutations. Importantly, there were no cases of QTc prolongation of Grade 3 or greater, and only 6% of patients experienced Grade 1 prolongation.
As a proof of concept, a drug designed to target a newly discovered biological node is showing efficacy in treating rare genetic diseases in the kidney, the eye, and the brain and is now making its way to clinical trials in collaboration with a pharmaceutical partner. We will keep you in the loop on upcoming events and research highlights.
They have interesting patterns of expression in different cancer indications; thus, diverse therapies for attacking these targets have been developed. As we’ll see, ASCO 2025 was a breakout conference for B7-H3-targeted therapies. Advanced therapies targeting B7-H3 use different modalities, currently dominated by ADCs.
Biogen Launches Global Phase 3 PROMINENT Trial Evaluating Felzartamab in Primary Membranous Nephropathy Biogen Inc. The trial is expected to complete in 2029. This randomized, open-label study will compare felzartamab with tacrolimus, a commonly used immunosuppressant, in a cohort of approximately 180 patients worldwide.
a principal investigator for the TRANSCEND FL study and a lymphoma and cell therapy specialist at Memorial Sloan Kettering Cancer Center in New York. This high durability highlights the sustained Benefit provided by liso-cel in a population typically faced with poor prognosis after multiple lines of standard therapy.
Xywav: A Low-Sodium Alternative with FDA Approval Xywav is a uniquely formulated, low-sodium oxybate therapy, and remains the only product of its kind approved by the U.S. of participants reported treatment-emergent adverse events (TEAEs), all of which were mild or moderate and consistent with previous safety data on Xywav.
By harnessing the full range of innovative technologies and taking advantage of an FSP partners extensive skills and experience, sponsors are able to bring their therapies to market more quickly and within budget even in the face of complicated global regulations and widely fluctuating workloads.
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 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 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. Among the 40 participants in Part C, adverse events (AEs) were reported in 37 individuals, with the majority categorized as mild to moderate in severity.
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.,
Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. Drugmakers are working to coopt this process with targeted therapies. You can unsubscribe at anytime.
The five-year results were derived from the Phase III Portal study, a long-term extension of the pivotal Archway trial, and presented at the American Society of Retina Specialists (ASRS) 2025 Annual Meeting held in Long Beach, California. Susvimo was designed to directly address this issue.
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.
This design was meant to enable clinician-researchers to gauge the ability of rilzabrutinib to control disease activity after the withdrawal of standard therapy. Safety Profile Consistent with Previous Studies : The safety profile of rilzabrutinib was well-understood from previous trials, with no new safety signals.
The data come from Phase 2 trials evaluating its investigational therapy VYVGART® (efgartigimod) for two hard-to-treat autoimmune conditions: idiopathic inflammatory myopathies (IIM or myositis) and Sjogren’s disease (SjD). Luc Truyen, Chief Medical Officer at argenx. No new safety concerns were identified.
With our presence at the event, we could showcase our core capabilities in drug development and manufacturing, as well as highlight our expanded portfolio of curated services. Events like this remind us why innovation thrives on collaboration. We're already looking forward to where these conversations will lead next.
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. Key results from the CLEVER trial include: A 60.5% Octavio Ramilo, Chair of the Department of Infectious Diseases at St.
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.
Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. The most common adverse events reported were insomnia as well as the sudden urge to urinate.
Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. Dive Insight: Vor hit reset in May, after nearly a decade of work on blood cancer therapies. Published Aug.
Importantly, the smaller size of the tablets, coupled with their film coating, is designed to make them easier to swallow than the current capsules—a meaningful benefit for patients on long-term therapy. In the United States, BRUKINSA has continued to demonstrate its clinical and commercial strength. Source link
Individuals with T1D must rely on lifelong insulin therapy to regulate blood glucose levels. For some, however, disease management becomes even more precarious due to impaired hypoglycemia awareness and a heightened risk of severe hypoglycemic events (SHEs). No serious adverse events were attributed to the treatment itself.
The event spotlighted significant advances in the company’s oncology portfolio, underscoring its growing stature as a transformative player in the global fight against cancer. To date, the company has conducted more than 170 clinical trials across 40 countries, enrolling over 25,000 patients.
The RedirecTT-1 trial shows the power of this novel combination approach as a potential treatment option for these patients, many of whom have limited options after multiple lines of therapy.” 20% had previously received BCMA-directed CAR-T therapy , adding further resistance to standard treatments. Among these: 84.4%
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).
Food and Drug Administration (FDA)-approved gene therapy for the treatment of Duchenne muscular dystrophy (DMD). These initiatives reflect a careful and comprehensive approach to patient safety — and a strong understanding of the delicate balance between delivering a potentially life-changing therapy and mitigating its associated risks.
TCEs offer a streamlined, off-the-shelf alternative to complex cell-based therapies such as chimeric antigen receptor (CAR) T-cells, simplifying manufacturing and distribution. Cytokine release syndrome (CRS) is a well-known risk for TCE therapies, characterised by a rapid systemic inflammatory response, fever and potential organ dysfunction.
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