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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Drug Target Review

MARCH 17, 2025

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. “We call this phenomenon ‘event-driven pharmacology.”

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The Pharma Data

JUNE 27, 2025

Biogen Unveils New Data Underscoring the Long-Term Benefits and Advancements of Nusinersen in Spinal Muscular Atrophy Treatment Biogen , a global leader in neuroscience, has announced compelling new data that reinforces the clinical value and therapeutic potential of nusinersen, a pioneering treatment for spinal muscular atrophy (SMA).

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The Pharma Data

JUNE 23, 2025

This study explored the effects of beginning treatment with lower starting doses of MariTide to improve tolerability while maintaining clinical benefit. These results were evaluated using the efficacy estimand, a statistical model that accounts for treatment adherence and protocol deviations.

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The Pharma Data

JUNE 12, 2025

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.

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The Pharma Data

JUNE 15, 2025

This is crucial information for guiding both patients and physicians in their treatment decisions.” All patients received their respective biologic therapy in addition to standard background treatment with mometasone furoate nasal spray (MFNS). Serious adverse events were somewhat lower in Dupixent (2%) than in omalizumab (4%).

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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., Furthermore, NXT007 was generally well tolerated across all dosing cohorts.

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FDA Law Blog: Biosimilars

APRIL 13, 2025

LYTGOBI was granted accelerated approval for the treatment of adult patients with previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma (iCCA) with FGFR2 gene fusions or rearrangements. DCR is defined as the sum of complete response (CR), partial response (PR) and stable disease (SD).

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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

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 characterized by early-onset, treatment-resistant seizures, beginning as early as six weeks of age, and profound global neurodevelopmental impairment.

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SugarCone Biotech

JUNE 19, 2025

Over 70% of new patients are diagnosed with stage IV disease, also called extensive stage disease (ES-SCLC). Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis. 4 patients (33%) had grade 4 adverse events.

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The Pharma Data

JUNE 13, 2025

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.

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The Pharma Data

JUNE 9, 2025

Food and Drug Administration (FDA) for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients aged seven years and older with narcolepsy. of participants reported treatment-emergent adverse events (TEAEs), all of which were mild or moderate and consistent with previous safety data on Xywav. Source link

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The Pharma Data

JUNE 11, 2025

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).

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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 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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FDA Law Blog: Drug Discovery

JANUARY 30, 2025

This target completion date is informed by the natural history of the disease, availability of alternative treatment, anticipated recruitment timeline, and the projected timeline for efficacy analysis(es); 2) the sponsors progress and plans for postapproval conduct of the trial provide sufficient assurance to expect timely completion of the trial.

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The Pharma Data

JUNE 6, 2025

Proteinuria reduction is a well-established surrogate marker for the progression of kidney disease, and this level of decline is considered both statistically significant and clinically meaningful in slowing disease progression toward end-stage kidney disease (ESKD). Treatment-emergent adverse events (TEAEs) occurred in 76.3%

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BioPharma Drive: Drug Pricing

JULY 8, 2025

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.

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The Pharma Data

JUNE 9, 2025

The COACH study is the first clinical investigation to assess the combined effects of two once-weekly treatments—TransCon® CNP (navepegritide) and TransCon® hGH (lonapegsomatropin)—in children with achondroplasia. TransCon CNP as a monotherapy has demonstrated the potential to transform the treatment of achondroplasia,” Dr. Shu stated.

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The Pharma Data

JUNE 15, 2025

The data, which encompass up to 3 years of follow-up after a single infusion of the treatment, were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, held from June 13–16 in Glasgow, United Kingdom. President and Chief Executive Officer of Intellia Therapeutics.

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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. of patients in the atogepant arm discontinued treatment because of AEs, versus a substantially higher 29.6% Specifically, only 12.1% among those taking topiramate. to 0.59; p <0.0001).

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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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Drug Target Review

APRIL 7, 2025

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.

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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

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 20, 2025

For some, however, disease management becomes even more precarious due to impaired hypoglycemia awareness and a heightened risk of severe hypoglycemic events (SHEs). Zimislecel was generally well tolerated, with most adverse events categorized as mild to moderate.

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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. Importantly, there were no treatment-related discontinuations due to adverse events (TEAEs).

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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). The ultimate aim is to enable the administration of ELEVIDYS safely while honoring its potential to provide a dramatic and durable therapeutic benefit for children battling this profoundly challenging disease.

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DrugBank

JUNE 18, 2025

As clinical development progresses, pharmaceutical companies must submit periodic updates to regulatory agencies, reporting on patient safety data, adverse events, and protocol modifications. This application is the foundation for obtaining approval to proceed with Phase I human trials.

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The Pharma Data

JUNE 27, 2025

mg/mL solution for injection) across the European Union (EU), allowing for extended treatment intervals of up to six months for individuals diagnosed with neovascular (wet) age-related macular degeneration (nAMD) and visual impairment due to diabetic macular edema (DME).

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The Pharma Data

JUNE 27, 2025

Originally developed for the treatment of type 2 diabetes, semaglutide has also been approved for chronic weight management in obese and overweight individuals, particularly those with weight-related health complications. Refer patients promptly to ophthalmology for evaluation if NAION is suspected.

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The Pharma Data

JULY 2, 2025

This marks a pivotal moment for the treatment of a rare and aggressive group of cancers—including gallbladder cancer (GBC) and cholangiocarcinoma (CCA)—which are often diagnosed at an advanced stage when curative surgical options are no longer feasible. Arndt Vogel , professor of gastroenterology at Hannover Medical School. EU, and Japan.

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The Pharma Data

JUNE 9, 2025

Among the most notable results: 28% reduction in the composite endpoint of all-cause mortality and recurrent cardiovascular events versus placebo. Adverse events (AEs), including serious and severe AEs, were reported at similar frequencies in both the treatment and placebo groups, underscoring the favorable safety profile of vutrisiran.

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New Drug Approvals

APRIL 11, 2025

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]

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The Pharma Data

JUNE 13, 2025

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.

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