The Pharma Data

JUNE 23, 2025

Study Overview and Population The HYPERION study was designed to assess the efficacy and safety of WINREVAIR when used in combination with standard background therapy, compared to placebo, in adult patients recently diagnosed with pulmonary arterial hypertension. It follows two earlier landmark studies: STELLAR and ZENITH.

Trials 40

Trials Therapies Treatment Disease 40

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. No chemotherapy regimen is used in the trial. Note one important difference in the trials is the inclusion of HPV-positive patients, which the Bicara trial will exclude.

Therapies 67

Therapies Clinical Trials Trials Clinical Development 67

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. There are several types of biomarkers to consider.

Clinical Development 69

Clinical Development Clinical Trials Clinical Pharmacology Trials 69

The Pharma Data

JUNE 13, 2025

The data were presented during the 2025 European Hematology Association (EHA) Congress (Abstract #S239). 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 40

Therapies Treatment Clinical Development Trials 40

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. These new 52-week data will be unveiled during a dedicated symposium scheduled for Monday, June 23, from 1:30 p.m. to 3:00 p.m.

Therapies 40

Therapies Clinical Trials Disease Trials 40

The Pharma Data

JUNE 18, 2025

The investigational ADC is built using Daiichi Sankyo’s proprietary DXd technology and represents one of the newest generation of targeted cancer therapies. Building on Early Clinical Success The Phase 3 IDeate-Prostate01 trial is based on encouraging results from the earlier IDeate-PanTumor01 Phase 1/2 study.

Trials 40

Trials Therapies Clinical Trials Treatment 40

The Pharma Data

JUNE 26, 2025

Held at a pivotal juncture for the company, the R&D Day offered an opportunity for BeOne’s executive and scientific leaders to present new clinical data, pipeline milestones, and strategic insights that reaffirm its mission: to build the world’s first next-generation oncology company.

Research 40

Research Clinical Development Clinical Trials Engineering 40

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.

Therapies 97

Therapies Clinical Trials Clinical Research Trials 97

Drug Target Review

JUNE 9, 2025

“This experience underscored the gaps in rare disease innovation, motivating me to lead Alltrna, a company pioneering engineered tRNA therapies to address a broad range of genetic diseases in a profound way unlike any other genetic modality.” While commonly used in oncology, the basket trial approach is novel for rare genetic diseases.

Disease 80

Disease Treatment Engineering Protein Production 80

Drug Target Review

JULY 8, 2025

Not only are they chasing new targets, they are also working to understand the biology behind resistance, discover biomarkers that matter and build smarter therapies from the ground up. While there are already therapies under investigation that focus on PSMA alone, none are concurrently targeting STEAP1.

Therapies 52

Therapies Science Drug Development Treatment 52

FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

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. In October, FDA announced seven new clinical trial grants awarded in fiscal year (FY) 2024 – including one for a Phase 3 trial – totaling $17.2

Clinical Development 69

Clinical Development Production Clinical Trials Treatment 69

The Pharma Data

JUNE 26, 2025

FDA Approves Streamlined Monitoring Requirements and REMS Program Removal for Bristol Myers Squibb’s CAR T Cell Therapies Breyanzi and Abecma, Marking Milestone Toward Expanding Access to Cancer Treatment In a significant regulatory development, Bristol Myers Squibb announced that the U.S.

FDA Approval 40

FDA Approval Therapies FDA Treatment 40

Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. CAR-NK cell trials only commenced in 2017 and most are still recruiting patients.

Therapies 118

Therapies Clinical Trials Cell Biology Trials 118

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 trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options.

Trials 40

Trials Treatment Therapies Pharmacokinetics 40

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. SCLC generally has a “hot” tumor microenvironment (TME) meaning that it is infiltrated by immune cells, including the T cells that are activated by immune checkpoint therapies.

Therapies 78

Therapies Clinical Trials Treatment Clinical Development 78

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.

Therapies 119

Therapies FDA FDA Approval Treatment 119

Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. While iPSC therapies hold great promise, several challenges remain, including poor infiltration of cytotoxic lymphocytes into solid tumours and insufficient cytotoxicity of myeloid cells.

Therapies 103

Therapies DNA Engineering Virus 103

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., in those receiving placebo—further underscoring the tolerability of this investigational therapy.

Trials 40

Trials Treatment FDA Therapies 40

PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

Therapies 105

Therapies FDA Clinical Trials Disease 105

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 64

Drugs Clinical Trials Drug Development Pharmaceuticals 64

Drug Target Review

FEBRUARY 1, 2024

Part of the challenge is that patients present with very different kinds of clinical phenotypes, meaning the populations are heterogeneous. This can present challenges when attempting to recruit an enriched patient population for clinical trials. This has been a hot button issue in neuroscience for as long as I can remember.

Therapies 104

Therapies Drug Development Disease Clinical Development 104

PPD

AUGUST 5, 2024

Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success.

Clinical Trials 98

Clinical Trials Trials Clinical Research Disease 98

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.

Trials 75

Trials Disease Clinical Trials Clinical Research 75

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 40

Trials Treatment Pharmacokinetics Clinical Trials 40

Conversations in Drug Development Trends

MAY 10, 2024

Over the past two decades, industry-sponsored clinical trials have targeted treatments for BPD, yielding some promising outcomes; however, broader psychiatric research often excludes BPD patients, a trend that extends to the emerging field of psychedelic studies.

Clinical Trials 95

Clinical Trials Trials Treatment Drug Development 95

New Drug Approvals

JULY 9, 2025

5] In China, it was conditionally approved in 2023 for the treatment of NSCLC and full approval is contingent on results of phase 3 clinical trials. [6] 7] Efficacy was evaluated in WU-KONG1B (NCT03974022), a multinational, open-label, dose randomization trial. [1] 1] History Sunvozertinib is being developed by Dizal Pharmaceutical. [5]

FDA 62

FDA FDA Approval Clinical Trials Trials 62

Drug Target Review

FEBRUARY 28, 2024

Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.

Therapies 105

Therapies Engineering Molecular Biology Science 105

The Pharma Data

JUNE 22, 2025

In the trial, participants were directly switched to Mim8 without a washout period. Strong Safety Profile Reinforces Mim8’s Potential Mim8’s safety profile was a central focus of the FRONTIER5 trial. Throughout the 26-week study period, the investigational therapy was well-tolerated.

Pharmacokinetics 40

Pharmacokinetics Therapies Treatment Trials 40

PPD

AUGUST 28, 2024

For pharmaceutical innovators and drug developers working to bring oncology therapies to market, patients are the “why” behind it all. For many patients, involvement in oncology clinical trials represents a last hope for an effective therapy. Researchers often employ cell therapy studies for oncology indications, and the U.S.

Trials 69

Trials Clinical Trials Drug Development Therapies 69

Conversations in Drug Development Trends

OCTOBER 29, 2024

By: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinical research, particularly in rare disease, represents a significant shift in the clinical trial landscape. Why Is There an Increase in Patient-Led Rare Disease Research?

Clinical Research 72

Clinical Research Disease Research Clinical Trials 72

PPD

MAY 18, 2023

Yet, four out of five care partners of people with neurodegenerative disorders have never been informed of clinical trial opportunities , according to a survey of more than 250 caregivers conducted by PPD, the clinical research business of Thermo Fisher Scientific. There’s little time to lose.

Clinical Trials 86

Clinical Trials Trials Clinical Research Doctors 86

PPD

MARCH 11, 2025

Breaking through research barriers Challenge #1: Small groups of patients Rare diseases impact a small number of individuals, making it difficult to recruit enough participants for clinical trials. Moreover, in some rare disease trials, it is unethical to design a control group of patients with a placebo.

Drug Development 52

Drug Development Clinical Trials Disease Trials 52

PPD

DECEMBER 16, 2024

Cost and complexity go hand-in-hand The rising costs and growing complexity in clinical trials are deeply linked, with patient recruitment, extended timelines and meeting regulatory demands emerging as some of the key drivers. Nearly half (49%) of clinical trial sponsors surveyed identified rising costs as their foremost concern in 2024.

Drug Development 52

Drug Development Clinical Trials Compliance Trials 52

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 40

Treatment Small Molecule Therapies Trials 40

BioPharma Drive: Drug Pricing

JUNE 23, 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. You can unsubscribe at anytime. Thus, these drugs best complement, rather than replace, healthy lifestyle interventions.

Treatment 130

Treatment Drugs Therapies FDA 130

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 40

Disease Treatment Trials Therapies 40

The Premier Consulting Blog

JANUARY 14, 2025

As the oncology landscape continues to evolve, the OCE aims to improve access to safe, effective, and cutting-edge therapies through streamlined regulatory processes and patient-centered approaches. These include Fast Track Designation, Breakthrough Therapy Designation, Priority Review Designation, and the Accelerated Approval Pathway (AAP).

Clinical Trials 52

Clinical Trials Therapies FDA Trials 52