Drug Target Review

JUNE 5, 2025

Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.

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The Pharma Data

JUNE 12, 2025

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.

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The Pharma Data

JUNE 11, 2025

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.

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SugarCone Biotech

JUNE 19, 2025

They have interesting patterns of expression in different cancer indications; thus, diverse therapies for attacking these targets have been developed. Over 70% of new patients are diagnosed with stage IV disease, also called extensive stage disease (ES-SCLC). This leads us to Amgen’s T cell-engager therapeutic, tarlatamab.

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The Pharma Data

JUNE 22, 2025

Importantly, the therapy demonstrated a favorable safety profile, with no thromboembolic events reported so far. No thromboembolic events—an important safety consideration for all coagulation therapies—were reported. Engineered by Chugai Pharmaceutical Co., Engineered by Chugai Pharmaceutical Co.,

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The Pharma Data

JUNE 12, 2025

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.”

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Broad Institute

NOVEMBER 25, 2024

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.

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The Pharma Data

JUNE 27, 2025

SMA, a rare and often devastating genetic neuromuscular disease, primarily affects motor neurons in the spinal cord, resulting in progressive muscle wasting and weakness. 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.

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The Pharma Data

JUNE 27, 2025

The successful outcome of the GEMZ Phase 3 study underscores UCB’s broader strategy of addressing underserved rare neurological diseases and reinforces its commitment to investing in areas with high unmet medical need. These data offer new hope to the CDD community, which has long struggled with a lack of effective seizure control therapies.

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The Pharma Data

JUNE 16, 2025

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.

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The Pharma Data

JUNE 15, 2025

All patients received their respective biologic therapy in addition to standard background treatment with mometasone furoate nasal spray (MFNS). This further underscores the ability of Dupixent to provide comprehensive disease control in patients battling both upper and lower airway disease.

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The Pharma Data

JUNE 23, 2025

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.

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The Pharma Data

JUNE 9, 2025

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.

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The Pharma Data

JUNE 9, 2025

These results further validate the potential of Ascendis’ proprietary TransCon technology to deliver long-acting therapies that improve both efficacy and patient experience. Importantly, the combination therapy maintains a safety profile that aligns with what we’ve observed in monotherapy studies.” years in prior clinical studies.

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BioPharma Drive: Drug Pricing

JULY 8, 2025

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.

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The Pharma Data

JUNE 6, 2025

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.

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The Pharma Data

JUNE 11, 2025

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.

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The Pharma Data

JUNE 15, 2025

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.,

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The Pharma Data

JUNE 18, 2025

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.

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The Pharma Data

JUNE 9, 2025

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. A New Era in Infant RSV Protection Dr. Dean Y.

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Elrig

MARCH 25, 2025

Prof Rory Johnson, Associate Professor, University College Dublin, and Dr Shalini Andersson, Vice President Nucleic Acid Therapeutics, AstraZeneca will lead this years event focussed on drugging the undruggable.

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The Pharma Data

JUNE 15, 2025

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.

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The Pharma Data

JULY 2, 2025

Primary membranous nephropathy is a rare and serious autoimmune kidney disease characterized by the formation of autoantibodies that damage the glomerular basement membrane, often resulting in nephrotic syndrome —a condition marked by high levels of protein in the urine, severe swelling, and a significant risk of progression to kidney failure.

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The Pharma Data

JUNE 20, 2025

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.

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The Pharma Data

JUNE 15, 2025

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.

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The Pharma Data

JUNE 15, 2025

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.

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The Pharma Data

JUNE 27, 2025

The alert follows a detailed safety review and recommendation from the European Medicines Agency (EMA) , which concluded that non-arteritic anterior ischemic optic neuropathy (NAION) —a rare but serious eye condition resulting in permanent vision loss—should now be officially recognized as a very rare side effect of semaglutide therapy.

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DrugBank

JUNE 18, 2025

Before a therapy can be approved for patient use, it must undergo extensive clinical testing and strictly adhere to regulatory guidelines. As clinical development progresses, pharmaceutical companies must submit periodic updates to regulatory agencies, reporting on patient safety data, adverse events, and protocol modifications.

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The Pharma Data

JULY 2, 2025

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. Jazz holds commercialization rights in the U.S.,

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Drug Target Review

MARCH 17, 2025

However, a deeper understanding of brain function particularly the role of synaptic plasticity is now opening the door to innovative therapies. Event-driven pharmacology Donello highlights the growing recognition of synaptic plasticity’s crucial role in the biology of depression. .

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The Pharma Data

JUNE 9, 2025

With this green light, AMVUTTRA becomes the first and only RNAi therapy in the European Union approved for treating both the cardiomyopathic and neuropathic manifestations of transthyretin amyloidosis (ATTR). AMVUTTRA demonstrated statistically significant benefits across all 10 pre-specified primary and secondary endpoints.

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Alta Sciences

JULY 7, 2025

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).

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The Pharma Data

JUNE 18, 2025

The ADA’s annual meeting is one of the most influential forums for showcasing scientific and clinical advancements in diabetes and metabolic disease research. The therapy is administered via subcutaneous injection and is being studied for its ability to produce and sustain significant weight loss over extended periods. to 3:00 p.m.

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Advarra

NOVEMBER 14, 2024

The DSMB’s primary responsibilities include : Monitoring patient safety: Ensuring participants are not exposed to undue risk and adverse events are promptly identified and addressed. For example, consider a global Phase III, double-blind study, in a novel new therapy.

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The Pharma Data

JUNE 15, 2025

This update comes after two cases of fatal acute liver failure (ALF) were reported in non-ambulatory DMD patients who received Elevidys, a phenomenon that underscores the ongoing complexities and safety signals related to adeno-associated virus (AAV)-mediated gene therapy.

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LifeSciVC

JUNE 12, 2025

was based on experience building on the understanding that a single protein variant, could have markedly different outcomes for patients with chronic kidney disease. From the outset, our mission was clear: to discover, develop, and commercialize a new class of therapies that could impact a broad range of diseases, both rare and prevalent.

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The Pharma Data

JULY 3, 2025

In addition to confirming a significant improvement in overall survival, the PANOVA-3 study showed that TTFields therapy combined with standard chemotherapy significantly delayed both the onset of debilitating pain and the initiation of opioid use. TTFields therapy was well-tolerated, with no new safety concerns identified.

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