PPD

JUNE 20, 2025

However, recent advancements and strategic approaches in clinical trials offer hope that additional treatments are on the way. With the expiration of pirfenidone’s patent exclusivity in 2021, sponsors have renewed their focus on developing new therapies. That’s why minimizing patient burden is critical to IPF trial success.

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New Drug Approvals

MAY 8, 2025

MORE US10918622 US10695323 US10464905 US10342780 US12109193 Medical uses In the United States, pirtobrutinib is indicated to treat relapsed or refractory mantle cell lymphoma after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor. [1] 6] The same trial was used to assess safety and efficacy.

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New Drug Approvals

JUNE 29, 2025

1] [3] History The efficacy of taletrectinib to treat ROS1-positive non-small cell lung cancer was evaluated in participants with locally advanced or metastatic, ROS1-positive non-small cell lung cancer enrolled in two multi-center, single-arm, open-label clinical trials, TRUST-I (NCT04395677) and TRUST-II (NCT04919811). [3] 12 June 2025.

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New Drug Approvals

APRIL 18, 2025

2] History Crinecerfont’s approval is based on two randomized, double-blind, placebo-controlled trials in 182 adults and 103 children with classic congenital adrenal hyperplasia. [2] 2] In the first trial, 122 adults received crinecerfont twice daily and 60 received placebo twice daily for 24 weeks. [2] 1 December 2024.

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BioPharma Drive: Drug Pricing

JUNE 26, 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. Progress developing a treatment for leukemia led the company in 2021 to price a $177 million initial public offering.

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Disease Licensing Licensing Therapies 109

New Drug Approvals

APRIL 19, 2025

elimination half-life (min) cardio-selectivity (1/2) metabilization Landiolol 4 250 pseudocholinesterases Esmolol 9 30 ery-esterases Metoprolol 420 3 cytochrom P2D6 (Leber) History The beneficial effects of landiolol have been demonstrated in over sixty clinical trials (pubmed search -August 2018). 28 October 2021. February 2021).

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FDA Clinical Trials Treatment Trials 62

Codon

NOVEMBER 1, 2024

Although Amodei does acknowledge some real-world issues limiting scientific progress — such as the slow growth of organisms and tedious clinical trials — he mostly passes over the more general tools that will be required to accelerate research in the near term. This essay focuses on how we might do both, specifically for the cell.

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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. The SELECT trial set out to understand whether the drug has similar effect on patients without diabetes.

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Clinical Trials Trials Clinical Development Disease 52

BioPharma Drive: Drug Pricing

JULY 2, 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. Organon acquired the drug in its purchase of Forendo Pharma in 2021. You can unsubscribe at anytime.

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BioPharma Drive: Drug Pricing

JULY 9, 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. In 2021, Merck agreed to pay $11.5 You can unsubscribe at anytime. billion for Acceleron Pharma.

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The Pharma Data

JULY 3, 2025

The EIC Accelerator Programme is part of the broader Horizon Europe 2021–2027 Research and Innovation framework, which provides non-dilutive and equity funding to breakthrough, high-risk innovations with global market potential. These attributes are expected to make the therapy more accessible in routine clinical practice.

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New Drug Approvals

JULY 18, 2025

9] Azvudine is under investigation in clinical trial NCT04668235 (Study on Safety and Clinical Efficacy of AZVUDINE in COVID-19 Patients (Sars-cov-2 Infected)). It was approved in China in 2021 as a first-in-class treatment for human immunodeficiency virus (HIV). 2021, 6, 414. (9) 6] [7] Azvudine was first discovered in 2007. [8]

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RNA Clinical Trials Trials Virus 62

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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Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. Patients and caregivers also assess the benefits offered by different therapies, weighing the progression-free survival with their off-target effects. 3D rendering of Antibody Drug Conjugate Molecules.

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The Pharma Data

JANUARY 5, 2021

Sponsors of human gene therapy (GT) products for neurodegenerative diseases affecting adults and children should initiate first-in-human clinical trials in adults before undertaking pediatric trials, according to new draft guidance the FDA released last week. Crossover designs may also be considered in such trials.

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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.

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Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Hearing aids also have all sorts of deficits.

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Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

Chemical Biology and Drug Design

OCTOBER 10, 2023

million people worldwide were living with HIV in 2021. Several agents are in clinical trials, including apricitabine, racivir, elvucitabine, doravirine, dapivirine, and elsulfavirine. Abstract Human immunodeficiency virus (HIV) causes acquired immunodeficiency syndrome (AIDS), a lethal disease that is prevalent worldwide.

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FDA Approval Clinical Trials FDA Virus 100

The Pharma Data

JANUARY 11, 2021

Pfizer has started dosing in a phase 3 study evaluating its investigational gene therapy, PF-06939926, in young male patients with Duchenne muscular dystrophy (DMD). The trial plans to enroll 99 boys aged 4 to 7 years across 55 sites in 15 countries. PF-06939926 received Fast-Track designation from the FDA in October. Source link.

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Therapies Trials FDA Treatment 52

The Pharma Data

MAY 17, 2021

Sponsors using master protocols to test new drugs and therapies for treating or preventing COVID-19 should base their analyses on comparisons between control arm participants who were concurrently randomized, the FDA advised in a final guidance released yesterday. Read the guidance here: www.fdanews.com/05-17-21-COVID-19.pdf.

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FDA Trials Therapies Treatment 52

The Pharma Data

JANUARY 27, 2021

Pfizer’s and Myovant Sciences’ investigational once-daily relugolix combination therapy (relugolix 40 mg plus estradiol 1 mg and norethindrone acetate 0.5 mg) helped reduce menstrual pain and nonmenstrual pelvic pain in women with endometriosis, according to findings from a phase 3 trial. Data from the study found that 84.8

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Science Therapies Treatment Trials 52

The Pharma Data

MARCH 11, 2021

Eli Lilly and Company (NYSE: LLY) today announced that data from programs across its oncology portfolio and pipeline will be presented at the 2021 American Association for Cancer Research (AACR) Annual Meeting, to be held virtually April 10-15, 2021. LY3484356, an oral SERD, is currently being studied in a Phase 1/2 clinical trial.

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Research Clinical Trials Trials FDA Approval 52

The Pharma Data

SEPTEMBER 29, 2021

Presentations: 535P, 536P, 534P, 104P, 1534P, 444P, 630P, LBA26, 623P, 603P, 593P, 594P, 1309P, 606P, 590P, 826O, 530P, LBA58, 1520O, 48P, 508TiP, 505TiP, 509TiP, 1438TiP Bayer will present new data from its established portfolio of approved oncology therapies at the upcoming ESMO Congress 2021 from September 16-21, 2021.

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Research Clinical Trials Therapies Trials 52

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Therapies Clinical Trials FDA Production 52

The Pharma Data

AUGUST 1, 2021

to $6.14; Raises 2021 Adjusted Diluted EPS Guidance Range from $12.37 Based upon the momentum of our business, we are raising our full year 2021 EPS guidance and believe AbbVie is very well positioned for the long term.” ” Second-Quarter Results. Worldwide net revenues were $13.959 billion, an increase of 33.9

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FDA Treatment Trials Disease 52

The Pharma Data

APRIL 7, 2021

today announced new data from its expansive neuroscience portfolio will be presented at the 2021 American Academy of Neurology (AAN) Annual Meeting, to be held virtually from April 17-22. . Key AbbVie abstracts and presentation details for the 2021 AAN Annual Meeting program are outlined below. Clinical Trials Plenary Session.

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Clinical Trials Treatment Trials Disease 52

The Pharma Data

MARCH 20, 2021

Precision imaging analytics technology known as radiomics will see increased adoption in 2021 and will be incorporated into drug development strategies and clinical trials management, according to Rose Higgins, CEO of HealthMyne.

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Clinical Trials Drug Development Trials Drugs 52

The Pharma Data

JANUARY 19, 2021

With this kind of a start, 2021 may be scorching. TCR 2 is developing novel T-cell therapies for solid tumors and hematological cancers. TC-210 is currently in a Phase I/II trial for mesothelin-positive non-small cell lung cancer (NSCLC), ovarian cancer, malignant pleural/peritoneal mesothelioma, and cholangiocarcinoma.

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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 Innovation Organizations conducting oncology clinical trials face challenges distinct from the rest of the research community.

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Trials Clinical Trials FDA Approval Therapies 52

The Pharma Data

APRIL 8, 2021

today announced that new data for its approved and investigational medicines for the treatment of neurological disorders will be presented at the 73rd American Academy of Neurology (AAN) Annual Meeting being held virtually April 17-22, 2021. Following U.S. Roche’s Chief Medical Officer and Head of Global Product Development.

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Clinical Trials Trials Disease Therapies 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. Additional trials (e.g.,

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The Pharma Data

JULY 6, 2021

Sanofi highlights scientific innovations in the field of rare blood disorders at I STH 2021. New research from first-in-class marketed and investigational therapies in hemophilia, immune thrombocytopenia and acquired thrombotic thrombocytopenic purpura will be presented. ePoster Abstract # OC48.2

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Therapies Pharmacokinetics Treatment RNA 52

KIF1A

APRIL 25, 2024

She is scheduled to be the 2nd patient in the world to receive ASO therapy for KAND. Timeline: December 2021 – We met with Dr Chung in NYC and discussed and applied to n-Lorem. March 2023 – We met with n-Lorem and found out Susannah’s ASO (1st KAND patient to receive ASO therapy for KAND) would work for Sloane.

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Therapies FDA Approval FDA Treatment 52

National Institute on Drug Abuse: Nora's Blog

MARCH 6, 2025

Advancing reduction of drug use as an endpoint in addiction treatment trials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.&

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The Pharma Data

JANUARY 3, 2021

04, 2021 (GLOBE NEWSWIRE) — Passage Bio , Inc. Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that U.S. GM1 is a rare and often life-threatening CNS disorder with no approved disease-modifying therapies available.

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Therapies FDA Treatment Clinical Trials 52

The Pharma Data

OCTOBER 14, 2021

The results, which demonstrate that patients treated with Biogen’s portfolio of MS therapies mount an effective antibody response to COVID-19 vaccination, are being presented at the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) virtual meeting, October 13-15, 2021.

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Vaccine Therapies Immune Response Disease 52

The Pharma Data

JANUARY 27, 2021

– Second Product Candidate Expected to Enter Clinic in First Half of 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. PHILADELPHIA, Jan.

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