Conversations in Drug Development Trends

JULY 8, 2024

By: Lisle Kingery, PhD, Therapeutic Strategy Lead, Neuroscience With depression rates at an all-time high and limited new treatments available, depression clinical trials are more important than ever. Treatment Treatment can take various forms, including therapy, medication, a combination of both, or a clinical trial.

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Advarra

MARCH 20, 2023

The Food and Drug Administration (FDA) recently released new guidance regarding cellular and gene therapy products, one of which may significantly impact early-phase clinical trials of such products. This article explores the implications of this guidance, including new approaches for studies focused on cellular or gene therapies.

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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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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.

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Broad Institute

NOVEMBER 6, 2024

Earlier this year, the researchers shared their first translational results, describing the immune cell response underlying colon inflammation in patients undergoing ICI therapy. These are home-run, breakthrough therapies, and quite an amazing success story. How did you go about launching this effort?

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Advarra

JUNE 20, 2023

Innovation Organizations conducting oncology clinical trials face challenges distinct from the rest of the research community. cell and gene therapies), with other therapeutic areas then pushing it further. Barriers to Enrollment Recent findings indicate significant barriers to enrolling patients remain in clinical trials.

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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? In most cases, the results you are really looking for and trying to prove in a trial can be complex.

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The Pharma Data

AUGUST 18, 2020

Poseida’s Phase 1 trial that used an investigational CAR T therapy for treating prostate cancer has been suspended due to a patient death. The trial, called P-PSMA-101, has been put on hold by the FDA with the company’s share prices plunging as much as 38% on Monday. Photo by FDA. ” Conor Kavanagh.

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DrugBank

OCTOBER 17, 2024

By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.

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The Pharma Data

AUGUST 30, 2021

Results from the Phase III EMPEROR-Preserved trial were presented today at the European Society of Cardiology Congress 2021 2 and published in The New England Journal of Medicine 1. 1 Trial participants were randomly assigned to empagliflozin 10 mg (n=2,997) or placebo (n=2,991) once daily.

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Alta Sciences

JANUARY 11, 2024

It is estimated that there are over 6,000 monogenic diseases, affecting over 350 million people worldwide; for these diseases, cell and gene therapy may provide hope for a cure. However, there are significant challenges associated with the successful development of these complex, leading-edge therapies. what is the altascientist?

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Advarra

JUNE 13, 2024

Recombinant DNA technologies and genetically modified biological agents are being adapted for a wide scope of therapeutic applications, and their use is becoming increasingly common in clinical trials. For example, a site conducting only protocols involving mRNA may be able to leverage a generic SOP covering any mRNA protocol at that site.

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The Pharma Data

JUNE 30, 2021

With a median follow-up of two years, the study met the primary endpoint of event-free survival (EFS; hazard ratio 0.398, p <0.0001). Event-free survival is defined as time from randomization to disease progression, commencement of new lymphoma therapy, or death from any cause.

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Vial

DECEMBER 5, 2023

Among others, NGS has led to the identification of disease-causing variants and novel drug targets and an improved understanding of complex biological events, e.g., the heterogeneity of tumors. Drug development The application of NGS has fueled the development of targeted therapies, precision medicine , and improved diagnostic methods.

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The Pharma Data

APRIL 22, 2023

UC Davis Health researchers have dosed the second participant in their clinical trial looking to identify a potential cure for HIV utilizing CAR T-cell therapy. The trial is the first-in-human clinical study investigating the duoCAR T-cell therapy for the treatment of HIV. The novel study uses immunotherapy.

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The Pharma Data

MAY 30, 2023

Pfizer Announces Positive Marstacimab Results from Pivotal Phase 3 Hemophilia A and B Trial (NYSE: PFE) today announced that the pivotal Phase 3 BASIS clinical trial ( NCT03938792 ) evaluating marstacimab has met its primary endpoints, having demonstrated statically significant and clinically meaningful effects.

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The Pharma Data

SEPTEMBER 1, 2020

Explore how emerging trends in clinical trial investigations are bringing patients closer to medicine making them more patient-centric. The patient experience is an important consideration in trial design. Download your copy today to find out how to digitally transform your clinical trials. Download Your Copy.

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The Pharma Data

APRIL 27, 2023

The trial met its primary endpoint and demonstrated clinically meaningful benefits across secondary endpoints 1. The APPOINT-PNH trial also showed clinically meaningful benefits for secondary endpoints 1. The most commonly reported adverse events (AEs) were infections (in 40.0% With iptacopan treatment, an estimated* 92.2%

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The Pharma Data

SEPTEMBER 2, 2021

(“Takeda”) today announced that the Phase 3 PANTHER (Pevonedistat-3001) study did not achieve pre-defined statistical significance for the primary endpoint of event-free survival (EFS). Takeda remains committed to conducting important research and transforming the lives of patients with cancer.”.

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Alta Sciences

AUGUST 28, 2023

Altasciences Completes Successful Phase I Trial of Ischemix’ Novel Compound for Treatment of Traumatic Brain Injury (TBI) pmjackson Mon, 08/28/2023 - 14:04 Laval, Québec, August 30, 2023 – Altasciences is pleased to have completed a Phase I trial on Ischemix, Inc.’s We are proud to support Ischemix in advancing this vital new therapy.”

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The Pharma Data

MAY 27, 2022

Novartis today announced the US Food and Drug Administration (FDA) has granted accelerated approval for Kymriah ® (tisagenlecleucel) for the treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy.

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Broad Institute

NOVEMBER 6, 2024

The results also highlight possible therapeutic targets that bolster the rationale behind an ongoing clinical trial recently launched at MGH that is testing a drug for this kind of heart inflammation. This work provides a biological foundation for testing more targeted therapies for myocarditis due to an immune checkpoint inhibitor.

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The Pharma Data

AUGUST 30, 2021

Positive high-level results from the FoCus Phase III trial in Wilson disease showed ALXN1840 met the primary endpoint with a statistically significant improvement in daily mean copper mobilisation from tissues, demonstrating superiority compared with standard-of-care (SoC) treatments.

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The Pharma Data

JUNE 7, 2023

Rahway, NJ, USA), an anti-PD-1 therapy. Grade 3 or greater treatment-related adverse events (TRAEs) occurred in 31% of patients receiving doublet therapy and 58% of patients receiving triplet therapy. The percentage of ILD or pneumonitis events was similar across cohorts.

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The Pharma Data

DECEMBER 22, 2020

Approximately 74 patients are planned for this trial in Head and Neck Squamous Cell Carcinoma (HNSCC) and Cutaneous Squamous Cell Carcinoma (CuSCC). The trial will enroll three cohorts of patients within distinct populations – all with locally advanced, recurrent and/or metastatic HNSCC or locally advanced or metastatic CuSCC.

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Advarra

NOVEMBER 14, 2024

In an era where clinical trials are increasingly global, it’s more imperative than ever to leverage international expertise. Data and safety monitoring boards (DSMBs), also known as data monitoring committees (DMCs), play a critical role in overseeing a clinical trial’s safety and efficacy.

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The Pharma Data

SEPTEMBER 11, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for empagliflozin as an investigational treatment for adults with heart failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced. 2,3 A leading cause of hospitalization in the U.S. o Completion: 2020.

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The Pharma Data

JANUARY 9, 2021

(NASDAQ: BMRN) today announced positive topline results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. This is the largest global Phase 3 study to date for any gene therapy in any indication, with 134 participants.

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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. As a result, eliapixant may have the potential for safe and effective long-term use.

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The Pharma Data

AUGUST 4, 2020

Novartis is celebrating the success of its one-time CAR T therapy Kymriah (tisagenlecleucel) in patients with relapsed or refractory (r/r) follicular lymphoma (FL), revealing that the therapy met its primary its primary endpoint of complete response rate (CRR) in Phase 2 trials, according to an independent review committee.

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The Pharma Data

SEPTEMBER 27, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for empagliflozin as an investigational treatment for adults with coronary failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced. 2,3 a number one explanation for hospitalization within the U.S.

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Drug Target Review

OCTOBER 10, 2024

What are the risks of over-reliance on healthy donor material in early-stage research, and how can decision-makers ensure a balanced integration of diseased material to avoid potential gaps when transitioning to clinical trials? Diseased cells are likely to be more fragile or less abundant, making it difficult to obtain enough for therapy.

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Chemical Biology and Drug Design

MAY 11, 2023

Neuronal degeneration may arise due to a variety of pathological events, like inflammatory responses, neurotransmitter dysregulation, oxidative damage, mitochondrial malfunction, apoptosis, and genetic factors. Crocin, a naturally occurring carotenoid molecule, was found to have neuroprotective potential in the therapy of this disorder.

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FDA Law Blog: Drug Discovery

APRIL 23, 2023

This Draft Guidance focuses on the use of patient-level data from other clinical trials or from RWD sources. For example, objective response rate is often used as a single-arm trial endpoint in oncology given the established understanding that tumor shrinkage rarely occurs without an intervention.”

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Conversations in Drug Development Trends

FEBRUARY 28, 2023

Over the next few decades, that drug made its way through clinical trials, securing approval in 2007—just 36 months before Rob was diagnosed in 2010. If we don’t fund this research, we never have the chance for treatments, cures, or innovative therapies. At this event, they receive the funds to start their basic bench research.

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The Pharma Data

SEPTEMBER 12, 2020

Food and Drug Administration to expand the enrollment of their Phase 3 pivotal COVID-19 vaccine trial to up to approximately 44,000 participants which also allows for the enrollment of new populations. The pivotal trial is event-based and there are many variables that will ultimately impact read-out timing.

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The Pharma Data

JUNE 28, 2023

The study cohort includes 128 adult patients with relapsed or refractory (R/R) FL who received at least two or more lines of systemic therapy. “Together with our partner Genmab, these results may bring us one step closer to our goal of advancing a potential core therapy for patients with B-cell malignancies.” percent (1.6

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