The Pharma Data

JUNE 25, 2025

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).

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Drug Target Review

APRIL 7, 2025

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.

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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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Conversations in Drug Development Trends

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

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FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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The Pharma Data

JUNE 18, 2025

The presentations will also include new insights from Amgen’s Phase 3 FOURIER study of Repatha® (evolocumab) and the VESALIUS-REAL study exploring real-world lipid management practices. The ADA’s annual meeting is one of the most influential forums for showcasing scientific and clinical advancements in diabetes and metabolic disease research.

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Conversations in Drug Development Trends

APRIL 29, 2025

On the last day of February every year is Rare Disease Day , a dedicated day to celebrate the rare disease community, including patients, families, caregivers, clinicians, researchers, regulators, and more. The goal is to build community, share ideas and resources, and advance research and innovation.

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The Pharma Data

JUNE 18, 2025

The IDeate-Prostate01 trial marks a significant advancement in the B7-H3 ADC development program and underscores the shared commitment of both companies to addressing difficult-to-treat cancers. Addressing a Pressing Clinical Need in Prostate Cancer Prostate cancer remains one of the most prevalent malignancies among men worldwide.

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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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DrugBank

JULY 20, 2023

Rare diseases, therefore, present compelling opportunities for Drug Development. Since each disease is present in small groups of patients, this research is also fraught with complex challenges. We're excited to expand our data offering by launching a new Rare Disease add-on to complement our Clinical Trial dataset.

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The Pharma Data

JUNE 6, 2025

At this premier global meeting focused on thrombosis, hemostasis, and bleeding disorders, Sanofi will present compelling data across 18 scientific abstracts, including five oral presentations. In addition, two poster presentations will offer deeper insights into the LUNA 3 trial.

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Conversations in Drug Development Trends

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Operationalizing these trials requires proactive and flawless management at every stage.

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Chemical Biology and Drug Design

MAY 11, 2023

The figure depicts the neuroprotective role of crocin in Parkinson's disease. Abstract Parkinson's disease is among the most common forms of neurodegenerative illness, with present treatment being primarily symptomatic and frequently coming with substantial adverse effects.

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Broad Institute

OCTOBER 23, 2023

By Makenzie Kohler October 23, 2023 People of South Asian ancestry around the world have more than double the risk of developing cardiometabolic diseases like diabetes, heart attack, and stroke compared to other populations.

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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. The full dataset is expected to be presented at an upcoming international scientific conference, which will provide greater clarity on the magnitude of fenfluramine’s clinical benefits.

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The Pharma Data

JUNE 12, 2025

The data, presented at the annual European Alliance of Associations for Rheumatology (EULAR 2025) congress in Barcelona, Spain, show that dapirolizumab pegol resulted in significant and clinically meaningful improvements in disease activity while addressing a major symptom that profoundly impacts patients’ daily lives — fatigue.

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The Pharma Data

JUNE 6, 2025

has announced encouraging interim findings from the pivotal Phase 3 VISIONARY trial evaluating sibeprenlimab in adults with Immunoglobulin A nephropathy (IgAN), a rare and progressive autoimmune kidney disorder. in collaboration with Otsuka Pharmaceutical Co., Treatment-emergent adverse events (TEAEs) occurred in 76.3% in the placebo group.

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PPD

AUGUST 5, 2024

Pulmonary arterial hypertension (PAH) is a progressive and fatal lung disease that is caused or influenced by multiple factors. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success. Historically, the available drugs and U.S. None offers a cure for PAH.

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The Pharma Data

JUNE 23, 2025

Phase 2 Study: Transformative Weight Loss and Glycemic Control The Phase 2 trial focused on evaluating the efficacy and safety of MariTide in individuals with obesity, both with and without Type 2 diabetes (T2D). This schedule reflects the learnings from the Phase 1 PK-LDI trial and aims to maximize tolerability while sustaining efficacy.

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Drug Target Review

JULY 8, 2025

mCRPC is a particularly aggressive form of the disease, and patients currently have limited treatment options,” said Eleni. “As The preclinical data we presented in an oral presentation at AACR, demonstrate the broader anti-tumour activity of dual-targeting ABBV-969 compared to ADCs targeting either STEAP1 or PSMA.”

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PPD

JUNE 24, 2024

In the realm of vaccine development, mega trials — studies enrolling 5,000 subjects or more — have been instrumental in the fight against many pathogens, including influenza, rotavirus, malaria, RSV and most recently in the rapid development of vaccines against COVID-19.

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PPD

MARCH 11, 2025

While individual rare diseases affect populations that are small in numbers, collectively they impact millions globally, posing significant health and research challenges. Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development.

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PPD

OCTOBER 9, 2023

“Patient-centric” is used frequently to describe today’s clinical trial and health care landscape. The industry applauds that point of view and strives to include a patient-centric approach in all aspects of clinical trial designs. Education on the disease state and regular reinforcement.

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Drug Target Review

JUNE 11, 2025

NGS has revolutionised genomic analysis, enabling the identification of disease-related genetic variants. Clinical genomics, powered by NGS, enables more precise target validation, improved patient stratification and optimised trial design, ultimately aiming to increase PoS. Highlighting data integration. This is an AI generated image.

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New Drug Approvals

JULY 9, 2025

1] Medical uses In the US, sunvozertinib is indicated for the treatment of adults with locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy. [1]

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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). Tarlatamab triggered an ORR of ~40% in refractory (second-line or later) SCLC patients in the DeLLphi-301 clinical trial published in 2023. The overall survival rate (OS) at 5 years post-diagnosis is about 3% for ES-SCLC.

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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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Conversations in Drug Development Trends

AUGUST 29, 2024

By: Nathan Chadwick, Therapeutic Strategy Lead, Rare Disease Over the last few years in clinical trials, particularly within the rare disease community, a notable shift is underway, where patients and caregivers are taking the lead in reaching out to clinical trial sites rather than the other way around.

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FDA Law Blog: Biosimilars

APRIL 13, 2025

The Untitled Letter notes that the confirmatory trial for LYTGOBI is currently ongoing and has not been completed; therefore, clinical benefit of LYTGOBI has not yet been confirmed. As a single-arm trial (i.e., DCR is defined as the sum of complete response (CR), partial response (PR) and stable disease (SD).

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The Pharma Data

JUNE 11, 2025

The findings were presented as part of a late-breaking abstract session (#LB0001) at the European Alliance of Associations for Rheumatology (EULAR) Congress 2025 , held June 11–14 in Barcelona, Spain. Minimal Disease Activity (MDA) : A composite measure reflecting low disease activity across multiple domains.

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PPD

MAY 18, 2023

For neurodegenerative disease research, the care partner, or caregiver, is equally important as the person with dementia. Even with stronger awareness of studies, drug developers must contend with the inherent lack of trust between the dementia community and clinical trials. There’s little time to lose.

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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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PPD

JANUARY 5, 2023

In an industry survey led by PPD, Thermo Fisher Scientific’s clinical research business, rare diseases emerged as a top therapeutic area in drug developers’ pipelines, with 39% of global biotech and biopharma leaders pursuing therapies in the broad rare disease space.

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SugarCone Biotech

MARCH 2, 2025

Where TGF- is present there is no or limited IFN- secretion by T cells and that means no PD-L1 expression within the tumor microenvironment, aka the TME. An emerging concept is to localize the inhibition of VEGF or the inhibition of TGF- within the disease microenvironment, e.g. in the context of cancer and the TME.

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SCIENMAG: Medicine & Health

MARCH 5, 2024

WHAT: A weekly injection of semaglutide was safe and reduced the amount of fat in the liver by 31% in people with HIV and metabolic dysfunction-associated steatotic liver disease (MASLD), according to a presentation today at the 2024 Conference on Retroviruses and Opportunistic Infections (CROI) in Denver.

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The Pharma Data

JUNE 12, 2025

The presentations highlight the ongoing progress and growing promise of the company’s innovative therapeutic platform, featuring its investigational oral CELMoD™ (Cereblon E3 Ligase Modulating Drugs) agents mezigdomide, iberdomide, and golcadomide, alongside its first-in-class, oral BCL6 (B-Cell lymphoma 6) ligand-directed degrader (LDD) BMS-986458.

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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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