Conversations in Drug Development Trends

AUGUST 29, 2024

By: Nathan Chadwick, Therapeutic Strategy Lead, Rare Disease Over the last few years in clinical trials, particularly within the rare disease community, a notable shift is underway, where patients and caregivers are taking the lead in reaching out to clinical trial sites rather than the other way around.

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Advarra

OCTOBER 1, 2024

Managing clinical trial budgets efficiently is necessary for the success and sustainability of clinical research sites. Effective budget management not only ensures trials are financially viable but also maximizes return on investment (ROI). the impact and value of the data produced).

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FDA Law Blog: Drug Discovery

MARCH 7, 2024

Clissold — The trio of CDER, CBER, and CDRH released a new draft guidance titled “ Use of Data Monitoring Committees in Clinical Trials ” that revises the 2006 guidance “Establishment and Operation of Clinical Trial Data Monitoring Committees” and, when final, will replace the 2006 guidance.

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PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. The New Drug Duvyzat (givinostat), a type of drug called an HDAC inhibitor, has been in clinical trials to treat cancers and other disorders of the blood, Crohn’s disease, and a form of juvenile arthritis.

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On Medicine

SEPTEMBER 12, 2023

Considering such findings, the United Nations Office on Drugs and Crime (UNODC) combined the TRT intervention with parenting strategies designed to enhance the carer’s capacity to support children through life events such as disasters, conflicts, and displacement. TRT + Parenting was trialled in Lebanon with Syrian families.

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The Pharma Data

NOVEMBER 8, 2021

Bristol Myers Squibb moment blazoned the Phase 3 CheckMate-816 trial met the primary endpoint of bettered event-free survival (EFS) in cases with resectable stage IB to IIIAnon-small cell lung cancer (NSCLC). Bristol Myers Squibb thanks the cases and investigators who were involved in the CheckMate-816 clinical trial.

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Broad Institute

NOVEMBER 6, 2024

For some patients, this treatment results in striking and long-lasting remission — but for a subset, it triggers complications ranging from mild rash to potentially deadly brain and heart inflammation. We know a bit more in terms of treatments to try, but they don’t always work.

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Advarra

JUNE 20, 2023

Innovation Organizations conducting oncology clinical trials face challenges distinct from the rest of the research community. More targeted therapies may change which patients and cancers may benefit, since a given treatment may only impact very specific biomarkers and genetic profiles.

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The Pharma Data

OCTOBER 12, 2021

Trial met primary endpoint. The trial met the primary endpoint, with a cure of 600 mg of AZD7442 given by intramuscular (IM) injection reducing the threat of developing severe COVID-19 or death (from any cause) by 50 compared to placebo in rehabilitants who had been characteristic for seven days or lower.

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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

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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PPD

JULY 29, 2024

AI also has the potential to incorporate real-world data (RWD) obtained from electronic health records (EHRs), medical claims or other data sources to inform the design and optimization strategy of clinical trials.

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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. With iptacopan treatment, an estimated* 92.2% 100) achieved a 2 g/dL or more hemoglobin-level increase from baseline without the need for red blood cell transfusions after the 24-week core treatment period 1.

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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. This will enable a deeper understanding of cellular processes, disease mechanisms, and personalized treatment. Fountzilas et al.

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Broad Institute

NOVEMBER 6, 2024

Launched in 2017, this is a first-of-its-kind program in North America focused on improving the diagnosis, treatment, and understanding of serious immunotherapy complications, which can affect nearly every organ system.

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The Pharma Data

DECEMBER 1, 2020

Rapid and substantialimprovement in depressive symptoms achieved by 44% of patients at 2 weeks, 67% at 6 weeks (MADRS response), and sustained with long-term treatment. points at Week 6 (primary timepoint), with AXS-05 treatment. points at Week 6 (primary timepoint), with AXS-05 treatment. NEW YORK, Dec.

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The Pharma Data

NOVEMBER 2, 2020

(NASDAQ:ADIL; ADILW) , a clinical-stage biopharmaceutical company focused on the development of treatments for addictions, today provided an update on its landmark ONWARD Phase 3 pivotal trial. 40% of expected Trial patient screenings have been completed and the Trial is more than 25% enrolled relative to planned enrollment.

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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. The SOP should list these symptoms and any available prophylaxis for post-exposure treatment.

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The Pharma Data

JANUARY 17, 2021

The treatment could be administered in primary care centers to people who test positive for COVID-19, avoiding hospitalization due to the progression of the disease and complementing the vaccine in the early phase after vaccination. BARCELONA, Spain , Jan. 18, 2021 /PRNewswire/ — Grifols (MCE: GRF, MCE: GRF.P,

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The Pharma Data

DECEMBER 30, 2020

Long-term safety profile consistent with previously completed controlled trials. Treatment with AXS-07, rapidly, substantially, and durably relieved migraine pain and associated symptoms in this trial. Axsome remains on track to submit an NDA for AXS-07 in the acute treatment of migraine in the first quarter of 2021.

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Advarra

FEBRUARY 24, 2023

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. All key questions to consider and address before the therapy even leaves the lab.

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Conversations in Drug Development Trends

FEBRUARY 28, 2023

95% of rare diseases don’t have a treatment approved and won’t anytime soon. Miraculously, there was an approved treatment. 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. Uplifting Athletes (UA) is on a mission to change that.

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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). Pevonedistat is a NEDD8-activating enzyme (NAE) inhibitor that leads to cancer cell death by disrupting protein homeostasis. Source link: [link].

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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

AUGUST 10, 2023

As one of the more recent fields to emerge in the clinical space, psychedelics have gained significant traction as a potential treatment for various neuropsychiatric diseases. Psychedelic Research at Worldwide Since 2017, Worldwide Clinical Trials have supported a myriad of sponsors leading the way in psychedelic research programs.

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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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Drug Target Review

JULY 1, 2024

As a non-opioid, SRP-001 also eliminates abuse potential, positioning it as a safer and effective drug candidate for the treatment of acute and neuropathic pain and migraine headache. In Phase I clinical trials, SRP-001 showed no serious adverse events among 56 healthy volunteers, highlighting its safety.

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The Pharma Data

JANUARY 3, 2021

United Kingdom Medicines and Healthcare Products Regulatory Agency authorized Clinical Trial Application. With these important regulatory clearances for our first-in-human clinical trial for INZ-701 in subjects with ENPP1 deficiency, we have transitioned from a research-stage to a clinical-stage company. “We

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The Pharma Data

SEPTEMBER 6, 2021

The primary efficacy outcome was met showing a statistically significant reduction in the 24-hour cough count (average hourly cough frequency based on 24-hour sound recordings) of up to 27% for eliapixant 75 mg twice daily over placebo after 12 weeks of treatment.

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The Pharma Data

NOVEMBER 12, 2021

COMET-TAIL Phase III data demonstrated that intramuscular administration of sotrovimab was non-inferior and offered similar efficacy to intravenous administration for high-risk populations The trial enrolled participants during the Delta variant wave of the pandemic in the US. In the IM administration (500 mg) arm of the trial, there was a2.7

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The Pharma Data

AUGUST 30, 2021

ALXN1840 demonstrated approximately three times greater copper mobilisation from tissues than standard-of-care treatments. The trial enrolled 214 patients, including treatment-naïve participants and those who have been on SoC therapy for an average of ten or more years.

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The Pharma Data

DECEMBER 30, 2020

Axsome Therapeutics reported positive data from the long-term, open-label Phase III MOVEMENT trial of AXS-07 for the treatment of acute migraine. The MOVEMENT trial evaluated long-term safety of the drug for up to 12 months in patients with migraine attacks. The most common adverse events were nausea, dizziness, and vomiting.

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The Pharma Data

JUNE 7, 2023

1,2 While first-line treatment with immune checkpoint inhibitors with or without chemotherapy has improved outcomes for patients with NSCLC without AGAs, like EGFR or ALK, most patients eventually experience disease progression. 6 There are currently no TROP2 directed ADCs approved for the treatment of lung cancer.

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The Pharma Data

APRIL 28, 2022

Novo Nordisk today announced headline results from the ONWARDS 2 trial, a phase 3a, 26-week efficacy and safety treat-to-target trial investigating once-weekly insulin icodec vs insulin degludec in 526 people with type 2 diabetes switching from daily insulin. events per patient year exposed to once-weekly insulin icodec and 0.27

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The Pharma Data

MARCH 29, 2022

Application based on CheckMate -816, the only Phase 3 trial to demonstrate improved event-free survival and pathologic complete response with an immunotherapy-based combination in the neoadjuvant setting of NSCLC. Opdivo -based treatments have shown clinical benefit in four Phase 3 trials in earlier stages of cancer.

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The Pharma Data

JANUARY 17, 2021

Aruvant has chosen Lonza to help develop and manufacture ARU-1801 for its upcoming pivotal trial. As we advance our gene therapy through clinical trials, this partnership signifies a critical milestone in the development pathway.” Once complete, Lonza will deliver cGMP material supply for the pivotal clinical trial.

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Quanticate

AUGUST 2, 2024

Real-world evidence (RWE) is changing clinical drug development, bridging the gap between controlled clinical trial environments and the complexities of everyday patient experiences.

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