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.

Disease

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.,

Clinical Trials

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. Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis.

Therapies

Drug Target Review

JULY 3, 2025

Given macro healthcare influences (eg, economic uncertainty, environmental changes) and the numerous available treatments for major diseases, drug developers may need to reassess their therapeutic strategies. This is especially relevant with today’s heavier focus on enhancing personalised medicine via broader emerging scientific findings.

Drug Development

The Pharma Data

JUNE 22, 2025

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. With new non-factor therapeutic options, many patients may feel uncertain about switching treatments.

Pharmacokinetics

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.

Treatment

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.”

Treatment

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. These data offer new hope to the CDD community, which has long struggled with a lack of effective seizure control therapies.

Trials

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.

Trials

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. Food and Drug Administration (FDA) for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients aged seven years and older with narcolepsy.

FDA Approval

The Pharma Data

JUNE 16, 2025

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.

Therapies

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.

Trials

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. The study successfully met its primary endpoint, showing that significantly fewer patients treated with atogepant discontinued therapy due to AEs compared to those on topiramate. In contrast, only 39.3%

Clinical Trials

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).

Treatment

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.

Treatment

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%).

Treatment

The Pharma Data

AUGUST 3, 2025

The findings offer significant hope for patients with nAMD—a leading cause of vision loss among adults over 60—by demonstrating that Susvimo can sustain visual acuity and disease control with just two treatments per year.

Treatment

SugarCone Biotech

JUNE 27, 2025

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.

Therapies

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.

Disease

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. Visit broad.io/L2C2023

Clinical Trials

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.

Trials

The Pharma Data

JUNE 11, 2025

The investigational therapy, an Fc-free anti-CD40L monoclonal antibody, is being developed for the treatment of systemic lupus erythematosus (SLE), a chronic autoimmune disease with limited treatment options and significant unmet need. Treatment-Emergent Adverse Events (TEAEs) : Occurred in 82.6%

Disease

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.

Targeted Protein Degradation

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.

Disease

The Pharma Data

JUNE 26, 2025

Specifically, the trial measured the percentage of participants who achieved a ≥3-line gain in Early Treatment Diabetic Retinopathy Study (ETDRS) chart readings at 12 hours post-dose on Day 8, while maintaining less than a 5-letter loss in BCDVA from baseline.

Trials

The Pharma Data

JUNE 15, 2025

According to the data, the combination demonstrated a high ORR and a strong depth of response in a patient population with limited treatment options and poor outcomes under standard care. were penta-drug refractory, having been exposed to additional lines of treatment. Head of Myeloma Unit at Tel-Aviv Sourasky Medical Center in Israel.

Therapies

The Pharma Data

JUNE 25, 2025

A Patient-Centered Innovation in B-Cell Cancer Treatment Zanubrutinib, marketed as BRUKINSA, is a next-generation Bruton’s tyrosine kinase (BTK) inhibitor approved for the treatment of various B-cell malignancies. In the United States, BRUKINSA has continued to demonstrate its clinical and commercial strength. Source link

Packaging

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. Safety Profile and Precautions Like all biologic therapies, ENFLONSIA comes with safety considerations.

FDA Approval

BioPharma Drive: Drug Pricing

JULY 14, 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. The most common adverse events reported were insomnia as well as the sudden urge to urinate.

Drugs

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.

Therapies

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.

RNA

The Pharma Data

JULY 2, 2025

Despite its impact, there are currently no FDA-approved therapies for PMN , and treatment options are limited to non-specific and often toxic agents such as chemotherapy or general immunosuppressants. Despite the severity of PMN, nearly one-third of patients fail to achieve remission with current treatment strategies.

Trials

The Pharma Data

JUNE 26, 2025

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. The event included detailed pipeline reviews, new data presentations, and forward-looking commentary on upcoming clinical readouts. Source link

Research

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).

Therapies

The Pharma Data

JUNE 15, 2025

Food and Drug Administration (FDA)-approved gene therapy for the treatment of Duchenne muscular dystrophy (DMD). For ambulatory patients — those retaining some ability to walk — there are no new treatment interruptions or regimen modifications at this time. ELEVIDYS is currently the only U.S.

Immune Response

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.

Drug Development

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.

Therapies