PPD

JUNE 20, 2025

While two approved treatments, pirfenidone and nintedanib, slow disease progression, there currently is no treatment that reverses the effects or offers a cure for IPF. However, recent advancements and strategic approaches in clinical trials offer hope that additional treatments are on the way.

Clinical Development

The Pharma Data

JUNE 18, 2025

Discovered by Daiichi Sankyo, I-DXd is being co-developed with Merck (known as MSD outside of North America) as part of a broader strategic collaboration between the two companies. Addressing a Pressing Clinical Need in Prostate Cancer Prostate cancer remains one of the most prevalent malignancies among men worldwide.

Trials

PPD

NOVEMBER 11, 2024

Like many aspects of life in the 21st century, this era of scientific advancement and increased partnership is largely driven by next-generation technologies that are streamlining workflows, breaking down silos and empowering scientists to extract the most prescient insights from their data throughout the drug development process.

Drug Development

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. Both data sets now provide foundational insight into the design and direction of Amgen’s upcoming Phase 3 MARITIME clinical development program.

Trials

Drug Target Review

APRIL 30, 2025

It involves a dynamic and often unpredictable process where every stage, from target identification to clinical trials, generates vast amounts of data. Additionally, AI-driven predictive modelling can shorten the preclinical phase by simulating biological responses, leading to more targeted and efficient clinical trials.

Drugs

The Pharma Data

JUNE 11, 2025

Ionis Begins Pivotal Phase 3 REVEAL Study of ION582 in Angelman Syndrome, Dosing First Patient in Global Trial Ionis Pharmaceuticals , Inc. Currently, treatment options are limited to supportive care aimed at managing symptoms rather than targeting the underlying cause.

Trials

SugarCone Biotech

JUNE 19, 2025

For these patients, clinical onset means severe symptoms and a rapid decline in overall health. Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis. The DeLLphi-304 study was reported at ASCO earlier this month.

Therapies

Drug Target Review

JULY 29, 2025

Effective financial management is a cornerstone in the success of clinical trials, which are integral to the advancement of drug discovery. As the pharmaceutical and biotech industries continue to evolve, clinical trials become more complex, and the importance of robust financial oversight has never been clearer.

Clinical Trials

The Pharma Data

JUNE 26, 2025

The encouraging data builds on the foundation established by earlier studies and further strengthens the clinical case for MR-141 as a potential non-invasive, once-daily ophthalmic solution for millions affected by the age-related decline in near vision. According to the company, MR-141 met its primary endpoint with statistical significance.

Trials

Drug Target Review

JULY 24, 2025

Antibody therapies have transformed cancer treatment, yet their limits are becoming increasingly clear – particularly when tumours evolve, evade immune detection or develop resistance to existing drugs. The presence of p95HER2 – a shortened version of the receptor that still drives tumour growth – makes treatment even more difficult.

Immune Response

Drug Target Review

JUNE 11, 2025

Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments. Highlighting data integration. This is an AI generated image.

Drugs

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

PPD

MARCH 11, 2025

Developing treatments for individuals living with rare diseases is critical, but orphan drug development is laden with unique obstacles that necessitate innovative, multifaceted approaches. Moreover, in some rare disease trials, it is unethical to design a control group of patients with a placebo.

Drug Development

The Pharma Data

JUNE 25, 2025

In March 2025 , Regulus announced the successful completion of its Phase 1b multiple-ascending dose clinical trial for farabursen. Additionally, treatment led to improvements in height-adjusted total kidney volume (htTKV) , an established clinical marker that correlates with disease progression in ADPKD.

RNA

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. In the trial, participants were directly switched to Mim8 without a washout period.

Pharmacokinetics

The Pharma Data

JUNE 22, 2025

The results from the Phase I/II NXTAGE trial, announced during the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress in Washington, D.C., The trial is evaluating NXT007 in people with hemophilia A without factor VIII inhibitors who have not previously been treated with Hemlibra.

Clinical Trials

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.

Therapies

Drug Target Review

JULY 18, 2025

Immuno-oncology has transformed cancer treatment, but for many patients, tumours remain resistant to even the most advanced immune checkpoint inhibitors. DT-7012 retains its depletion activity even in high CCL1-concentrated environments – an essential property not shared by most competitor antibodies currently in clinical development.”

Medical Chemistry

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

MAY 23, 2025

As the biotech sector races to improve the tolerability of these revolutionary treatments, Poolbeg Pharma has a novel solution: an oral therapy, POLB 001, to block the development of CRS, by limiting inflammation without affecting the anti-cancer immune responses that are vital for effective immunotherapies.

Immune Response

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. “Zelquistinel is a positive modulator of NMDA receptors,” Donello explains.

Treatment

Drug Target Review

JUNE 28, 2025

. “The data show a high tumour uptake and a favourable safety profile… good efficacy and tumour reduction in mouse models and matching clinically-relevant DLL3 expression levels.” ” With an IND filing and first-in-human trials expected in 2025, MP0712 is moving steadily towards clinical evaluation.

Science

The Pharma Data

JUNE 24, 2025

The lead asset in this portfolio, daraxonrasib (RMC-6236) , is a multi-selective RAS(ON) inhibitor currently being evaluated in clinical trials for patients with non-small cell lung cancer (NSCLC) and colorectal cancer harboring KRAS mutations. Source link

Drug Development

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.

Disease

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. Joshua Jacobs, Medical Director, Allergy and Asthma Clinical Research, Inc.

Treatment

Conversations in Drug Development Trends

JULY 3, 2025

However, the FDA’s recent decision to remove the Risk Evaluation and Mitigation Strategy (REMS) requirements for certain approved Chimeric Antigen Receptor T-cell (CAR T) therapies could dramatically shift these numbers, making life-saving treatments more accessible to those in need.

Therapies

The Pharma Data

JULY 2, 2025

The decision follows an analysis of topline data from a Phase 1b clinical study, which demonstrated encouraging safety and efficacy results in patients with DOK7-CMS, one of the more severe and prevalent subtypes of this ultra-rare neuromuscular disorder. Detailed data from the trial will be shared at an upcoming medical conference.

Clinical Trials

The Pharma Data

JUNE 25, 2025

Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to riliprubart , an investigational immunology therapy developed by Sanofi, for the treatment of antibody-mediated rejection (AMR) in solid organ transplantation.

FDA

The Pharma Data

JUNE 12, 2025

Together, these data reflect a significant step forward in the treatment of hematologic malignancies — a group of aggressive, complex disorders for which many patients still face limited treatment options and poor outcomes — and demonstrate the potential for these novel mechanisms of action to make a profound difference in patient care.

Targeted Protein Degradation

The Pharma Data

JUNE 24, 2025

This significant financial infusion is designed to fuel Revolution Medicines’ ambitious clinical development and commercialization plans, with a particular focus on its groundbreaking RAS(ON) inhibitor programs for patients with RAS-addicted cancers.

Clinical Development

The Pharma Data

JUNE 6, 2025

These new findings reinforce the company’s growing leadership in rare hematologic diseases, with a focus on transformative treatments for immune thrombocytopenia (ITP) and hemophilia. In addition, two poster presentations will offer deeper insights into the LUNA 3 trial. In the U.S.,

Disease

Conversations in Drug Development Trends

JANUARY 21, 2025

Regulatory bodies such as the FDA oversee clinical trials to ensure that studies’ design, conduction, analysis, and reporting are per established guidelines and laws. Any delays or missteps in bioanalysis during a Phase I trial can derail the trajectory of a promising drug.

Clinical Trials

The Pharma Data

JUNE 26, 2025

This integrated strategy allows BeOne to move seamlessly from early discovery through proof-of-concept to pivotal clinical trials, reducing development timelines and enhancing scalability. To date, the company has conducted more than 170 clinical trials across 40 countries, enrolling over 25,000 patients.

Research

The Pharma Data

JUNE 24, 2025

We look forward to this strategic alliance continuing to expand the frontiers of cutting-edge science and translating groundbreaking discoveries into transformative treatments for patients.” The company currently operates two global research and development (R&D) centers and four world-class production facilities across China.

Pharma R&D

PPD

JULY 8, 2025

Balancing generative AI opportunities and risks Generative AI offers unparalleled opportunities for transforming various aspects of health care and clinical research. In clinical trials, generative AI accelerates trial designs through protocol optimization and enrollment forecasting, leading to faster and more effective studies.

Clinical Research

DrugBank

JUNE 18, 2025

Developing a new drug takes an average of 10–15 years and costs upwards of $2 billion, yet the majority of drug candidates fail before reaching regulatory approval. The failure rate in clinical trials exceeds 90%, often due to insufficient safety data, efficacy concerns, or regulatory non-compliance.

Drug Development

The Pharma Data

JUNE 22, 2025

Exelixis Reports Positive Phase 3 Results for Zanzalintinib and Atezolizumab Combo in Metastatic Colorectal Cancer Exelixis , has announced promising topline results from the STELLAR-303 phase 3 pivotal clinical trial, a major milestone in the development of its investigational therapy zanzalintinib.

Trials