The Pharma Data

JUNE 12, 2025

Among the 85 relapsed or refractory patients, 36% had previously received a single line of treatment, 42% had undergone two lines, and 12% had gone through as many as three lines of prior therapy. Vice President of Early Clinical Development and Translational Research at Johnson & Johnson Innovative Medicine.

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The Pharma Data

JUNE 12, 2025

This design was meant to enable clinician-researchers to gauge the ability of rilzabrutinib to control disease activity after the withdrawal of standard therapy. We look forward to further evaluating rilzabrutinib in subsequent phases of its clinical development.” Fast Track and Orphan Drug Designations from FDA The U.S.

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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 15, 2025

Durable Benefit and Positive Clinical Outcomes “Today’s results underscore the promising potential of Intellia’s approach to genome-editing therapy — a one-time treatment that has been well tolerated and offers a highly differentiated, durable effect for patients suffering from a serious disease,” said John Leonard, M.D.,

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The Pharma Data

JUNE 26, 2025

This unmet need has driven the development of alternative pharmacological options, including topical eye drops like MR-141, which aim to improve near vision through non-surgical means. Details of the VEGA-3 Phase 3 Trial The VEGA-3 study is part of Viatris’ broader clinical development program to evaluate MR-141 for presbyopia.

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The Pharma Data

JUNE 11, 2025

(NASDAQ: BIIB) presented comprehensive new findings from their Phase 3 PHOENYCS GO clinical trial evaluating dapirolizumab pegol (DZP) at the 2025 Annual Congress of the European Alliance of Associations for Rheumatology (EULAR) in Barcelona, Spain. Treatment-Emergent Adverse Events (TEAEs) : Occurred in 82.6% in the SOC group.

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The Pharma Data

JUNE 26, 2025

a global oncology-focused biopharmaceutical company, delivered a comprehensive update to investors today during its highly anticipated Research and Development (R&D) Day. With more than 40 clinical and commercial-stage assets in active development, BeOne Medicines is demonstrating a scale of ambition matched by few in the field.

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DrugBank

JUNE 18, 2025

Drug development is a complex and highly regulated process. Before a therapy can be approved for patient use, it must undergo extensive clinical testing and strictly adhere to regulatory guidelines. Gilead’s remdesivir , an antiviral therapy for COVID-19, was given emergency use authorization under this program.

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Drug Target Review

MARCH 17, 2025

However, a deeper understanding of brain function particularly the role of synaptic plasticity is now opening the door to innovative therapies. Event-driven pharmacology Donello highlights the growing recognition of synaptic plasticity’s crucial role in the biology of depression.

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Treatment Licensing Licensing Science 52

Alta Sciences

JULY 7, 2025

ISSUE 44 — The Importance of Cytokine Release Assays to Derisk the Development of Immunomodulatory Drugs pmjackson Mon, 07/07/2025 - 21:31 New immunomodulatory drugs have the potential to cause serious immune-related adverse events, including cytokine release syndrome (CRS).

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DrugBank

JULY 14, 2025

Conversely, drug combinations that reduce the effectiveness of essential therapies can lead to therapeutic failure.   Challenges in Managing Drug-Drug Interactions in Clinical Development Predicting and managing DDI in clinical trials is one of the most complex challenges in drug development.

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BioPharma Drive: Drug Pricing

JUNE 11, 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. Among those who had gotten another such therapy, the rates were 52% and 62% in those trials.

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SugarCone Biotech

JULY 6, 2025

They have interesting patterns of expression in different cancer indications; thus, diverse therapies for attacking these targets have been developed. Each target and each therapeutic modality induces varying degrees of clinical efficacy, as well as causing toxicities. These activities are pro-oncogenic (reviewed here: [link] ).

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The Pharma Data

JULY 2, 2025

This trial was the first in its therapeutic area to use a composite primary endpoint consisting solely of major morbidity and mortality events , such as all-cause death, lung transplantation, or PAH-related hospitalization of at least 24 hours. Despite existing therapies, many PAH patients remain at substantial risk.

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The Pharma Data

JUNE 13, 2025

Food and Drug Administration (FDA) has approved KEYTRUDA® (pembrolizumab), its flagship anti-PD-1 therapy, for the treatment of adult patients with resectable, locally advanced head and neck squamous cell carcinoma (HNSCC) whose tumors express PD-L1 (Combined Positive Score [CPS] of 1 or greater) as determined by an FDA-approved test.

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The Pharma Data

JUNE 19, 2025

Grifols’ BT524 Fibrinogen Therapy Shows Promise in Phase 3 Trial Published in The Lancet’s eClinicalMedicine Grifols , a globally recognized healthcare company and a leader in the production of plasma-derived therapies, has marked a significant advancement in the treatment of acquired fibrinogen deficiency (AFD).

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Therapies Trials Clinical Trials Medical Schools 40

BioPharma Drive: Drug Pricing

JULY 10, 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. Since the drug’s U.S.

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Drugs Licensing Licensing Therapies 243

BioPharma Drive: Drug Pricing

JUNE 24, 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.

RNA 219

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PPD

APRIL 14, 2025

This expansion is creating opportunities for clinical trials related to a range of new therapy areas and their subpopulations. In a time-to-first-event analysis done at 40 months, the study found a 20% reduction in the incidence of nonfatal myocardial infarction, nonfatal stroke, or death from cardiovascular causes in this population.

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Clinical Trials Trials Clinical Development Disease 52

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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PPD

SEPTEMBER 16, 2024

ML is also used to identify predictors of response to a therapy or risk for adverse events (AEs) that can be used to inform value-based contracting or treatment strategies intended to minimize “wasted” use of a drug. Ready to learn more about convergence of real-world data and technology for clinical trials?

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Clinical Development Clinical Trials Clinical Research Drug Development 98

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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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.

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Treatment Drugs Therapies FDA 130

The Pharma Data

OCTOBER 21, 2020

and Shionogi Limited as shareholders, today announced the positive findings of a pooled analysis of COVID-19-related impacts across the investigational long-acting cabotegravir and rilpivirine clinical development programme. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days.

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Clinical Development Therapies Clinical Trials Treatment 52

The Pharma Data

JANUARY 27, 2021

. – Preclinical Data Underscore Treatment Potential for PBFT02 in Frontotemporal Dementia with Granulin (GRN) Mutations, a Devastating, Progressive Disorder Impacting Adults with No Approved Disease-Modifying Therapy Options. FTD is a debilitating form of early onset dementia that currently has no approved disease-modifying therapies. “We

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Therapies Treatment Clinical Trials FDA 52

LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. A permanent gene edit to produce a difficult-to-titrate supply of GLP1 in the liver?

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Small Molecule Trials Therapies Disease 143

Drug Target Review

AUGUST 24, 2023

Neutralising GDF-15 with CatalYm’s anti-GDF-15 antibody visugromab was shown to reverse its inhibitory effects and to re-sensitise tumours to anti-PD-1 treatment, achieving commensurate survival benefit of anti-GDF-15-anti-PD-1 combination therapy in vivo.

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Treatment Therapies Clinical Development Cell Biology 98

The Pharma Data

SEPTEMBER 16, 2020

executive vice president of Research and Development at Amgen, along with members of Amgen’s clinical development team and clinical investigators, will discuss Phase 1 data being presented on the Company’s investigational KRAS G12C inhibitor sotorasib (AMG 510). Reese , M.D., On Monday, Sept. PDT , David M.

Clinical Development 52

Clinical Development Therapies Disease Research 52

LifeSciVC

APRIL 24, 2024

There is a continuum of evidence for a given target – at one end are novel targets with some evidence of importance in disease, and at the other end are “de-risked” targets where the biology is precedented with an approved product or late-stage clinical asset(s). with gene editing or gene therapy, enzyme replacement therapy), agonism (e.g.,

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DrugBank

SEPTEMBER 26, 2024

While some clinical trials are necessary to confirm safety and efficacy in humans, these are typically smaller and shorter in duration compared to the extensive trials required for novel biopharmaceuticals. These molecules pose major scientific and regulatory hurdles due to their intricate structures and potential for immunogenicity.

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Advarra

FEBRUARY 24, 2023

How do you know your experimental therapy is working? But what does “working” really mean when efficacy means something subjective, like quality of life, reduced psychiatric events, or less symptoms? All key questions to consider and address before the therapy even leaves the lab.

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Clinical Trials Trials FDA Therapies 52

Conversations in Drug Development Trends

DECEMBER 20, 2023

Single-Arm Trial Design Single-arm trials have also become an increasingly common development strategy to support regulatory approval and allow patients expedited access to novel therapies, particularly in the accelerated access setting.

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Clinical Trials Trials Pharmacokinetics FDA 83

The Pharma Data

JANUARY 27, 2021

The two-hour event will cover various topics, including an overview of the Company’s board and executive leadership, its commitment to environment, social and governance (ESG) matters, and progress updates on the Company’s critical care programs, manufacturing and distribution approach, and commercial readiness plans. About MultiStem ®.

Clinical Trials 52

Clinical Trials Trials Therapies Production 52

The Pharma Data

MARCH 2, 2021

This new collaboration builds on the positive experience from previous partnerships with Gubra and combines Gubra’s proprietary streaMLine platform and expertise in obesity and peptide chemistry with Boehringer Ingelheim’s expertise in lead optimization and clinical development. population will have obesity. About Boehringer Ingelheim.

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Treatment Bioinformatics Clinical Development Therapies 52

The Pharma Data

SEPTEMBER 6, 2021

In the study, eliapixant demonstrated a positive benefit-risk profile, with the vast majority of adverse events considered mild or moderate. Discontinuation of the study due to adverse events occurred in 8% of patients treated with eliapixant. These results will guide us to advance our clinical development strategy of eliapixant.”.

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Trials Clinical Trials Treatment International 52

The Pharma Data

MAY 27, 2022

of patients refractory to their initial therapy and about one-third (33.1%) having received prior CAR T-cell therapy.¹ Cytokine release syndrome (CRS) was the most common adverse event occurring in 63.0% Incidence of Grade 3+ CRS was low (3.9%), with no Grade 5 events.¹. After a median follow-up of 12.6 months, 39.4%

Treatment 52

Treatment Therapies Clinical Trials Disease 52

Advarra

JUNE 20, 2023

Additionally, in 2021, a literature review estimated the average capitalized research and development (R&D) costs per new cancer medicine at between $944 million and $4.54 Typical clinical development timelines for anticancer drugs average an estimated 6.7 This means study startup times can vary greatly.

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