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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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.

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FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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PPD

OCTOBER 17, 2024

Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.

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ASPET

SEPTEMBER 29, 2023

Recent studies have yielded controversial results on the long-term effects of statin treatment on the risk of cardiovascular (CV) events. The incidence of first CV events was 6.0% Taken together, our data demonstrate that a 7-year stable control of LDL-C induces a forty percent reduction in the incidence of CV events.

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New Drug Approvals

JUNE 24, 2025

Safety: Diarrhea was the most frequent adverse event, but serious adverse events were balanced across treatment groups. FDA Designation: Nerandomilast received Breakthrough Therapy Designation from the FDA for the treatment of IPF. Nerandomilast CAS 1423719-30-5 C 20 H 25 ClN 6 O 2 S Molecular Weight 448.97

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Chemical Biology and Drug Design

JULY 26, 2023

Abstract Noncoding RNAs (ncRNAs) are engaged in key cell biological and pathological events, and their expression alteration is connected to cancer progression both directly and indirectly. However, there are several limitations, including delivery, uptake, and short half-life, in the application of ncRNAs in cancer treatment.

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ASPET

JUNE 30, 2023

Myeloproliferative Neoplasms (MPNs) are hematological malignancies that result from acquired driver mutations in hematopoietic stem cells (HSCs), causing overproduction of blood cells and an increased risk of thrombo-hemorrhagic events. This study aims to determine a personalized treatment strategy.

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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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FDA Law Blog: Drug Discovery

DECEMBER 6, 2024

Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. The draft guidance describes various methods to assess either cell distribution for cell therapy products or vector biodistribution for gene therapy products.

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The Pharma Data

JUNE 23, 2025

These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. According to Merck, the addition of WINREVAIR to background therapy resulted in a meaningful improvement in patient outcomes, particularly in delaying disease progression and reducing the risk of severe clinical events.

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PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. A gene-based treatment would have to alter many cells to exert a noticeable effect. Researchers have been working on developing gene therapy for DMD for decades. million DNA bases. Hyperbole or Hope?

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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.

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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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Chemical Biology and Drug Design

FEBRUARY 13, 2024

Some researchers proposed that the symptoms involved in AD (loss of memory, cognitive impairment, and amnesia) are the main event linked to the cholinergic neurotransmitter (acetylcholine). This review focuses on the last 10 year's literature search on huprine and its analogues for the treatment of AD.

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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 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.

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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.

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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. 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%

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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 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.

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Drug Target Review

AUGUST 6, 2024

Autoimmune diseases : Rheumatoid Arthritis : ADCs targeting specific immune cells or inflammatory mediators can provide more precise treatment options with potentially fewer side effects. Obesity : By targeting adipose tissue or specific metabolic pathways, ADCs could offer new treatments for obesity and related metabolic disorders.

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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 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 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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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

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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. 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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DrugBank

OCTOBER 17, 2024

By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.

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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.

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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

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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.

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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 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%

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Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options.

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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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Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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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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