Drug Target Review

SEPTEMBER 25, 2024

BRAF and downstream mitogen-activated extracellular signal-regulated kinase (MEK) inhibitors are combined to treat BRAF-mutated melanoma, for example. Inhibiting KDM5A, a regulator of cell proliferation, poses another interesting approach.

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The Premier Consulting Blog

MAY 31, 2023

While the type, number, and design of these studies vary based on product-specific characteristics, IND-enabling packages submitted to the FDA generally include key information about the pharmacology, pharmacokinetics, and toxicology of the product. All these studies need to be performed under GLP.

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Drug Target Review

JUNE 6, 2023

However, the protein’s preclinical promise has not yet been replicated in clinical trials, where systemic administration is associated with dose-limiting toxicities and a narrow therapeutic index. Some approaches have already reached the clinical trial stage, with others not far behind.

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Drug Target Review

MARCH 17, 2025

Neuropsychiatric disorders, affecting millions worldwide, disrupt the brain’s intricate processes of mood regulation, cognition and behaviour. The drug’s pharmacokinetics (PK) and pharmacodynamics (PD) are closely linked. Neuropsychiatric treatment is on the verge of a major transformation.

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Drug Target Review

SEPTEMBER 19, 2023

puts an end to the previous mandate that all drugs need to be tested on animals prior to human clinical trials. This major shift to the orthodox tradition of using animal experiments in drug testing dates back the Aristotle’s time and cemented 80 years ago with initial federal mandate of drug safety regulation of 1938.

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The Pharma Data

JANUARY 19, 2021

The Phase 1 clinical trial is planned to be conducted in Canada and targeted to recruit up to 48 and 24 healthy volunteers for the single-ascending dose (SAD) and multiple- ascending dose (MAD) cohorts, respectively. About ALS-4. This press release is provided “as is” without any representation or warranty of any kind.

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The Pharma Data

DECEMBER 20, 2020

Pending Health Canada’s approval, the Phase 1 trial is designed to test the safety, tolerability and pharmacokinetics of ALS-4 in healthy volunteers. The primary objective of the trial is to evaluate the safety and tolerability of SAD and MAD of ALS-4 administered orally to healthy subjects. About ALS-4.

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The Pharma Data

DECEMBER 29, 2020

Phase III BRIDGE open-label, switch-over clinical trial met key objectives for safety and efficacy. Patients had the option to receive PRX-102 infusions in a home care setting based on infusion tolerability and country regulation. CARMIEL, Israel and BOSTON , Dec. About Fabry Disease. Galactosidase-A enzyme.

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The Pharma Data

DECEMBER 9, 2020

The open-label Phase 2a ‘AMBITION’ study is designed to assess safety, tolerability, pharmacokinetics and biomarker analyses for early assessments of efficacy of 75 mg and 225 mg CRV431, administered orally to F2 and F3 NASH patients (n=18/dosing group), once daily for 28 days.

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Drug Target Review

JULY 1, 2024

SRP-001 targets the central nervous system (CNS) by producing N-arachidonoylaminophenol (AM404) in the midbrain’s periaqueductal grey (PAG) region, crucial for pain sensation and regulation. In Phase I clinical trials, SRP-001 showed no serious adverse events among 56 healthy volunteers, highlighting its safety.

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Vial

MARCH 15, 2024

The decision to outsource to a CRO is more than a strategic move; outsourcing to a CRO has multiple benefits , including access to streamlined expertise and risk mitigation in clinical trials. Breakdown of CROs Roles Initially, CROs primarily concentrated on overseeing clinical trial site operations and data management.

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The Pharma Data

OCTOBER 25, 2020

The Company plans to initiate a Phase 2 trial within the next several months. “We An exploratory endpoint of this trial will be to determine the overall clinical improvement after drug administration using the Clinical Global Impression – Improvement Scale (“CGI-I”).

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Drug Target Review

OCTOBER 3, 2024

3 This change signals a major shift away from animal use in drug safety regulation and encourages the use of alternative methods like organ-on-chip or other animal-free technologies. FDA no longer needs to require animal tests before human drug trials [Internet]. 1975 Aug;256(5517):495–7. Crescioli S, Kaplon H, Chenoweth A, et al.

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The Pharma Data

DECEMBER 15, 2020

The researchers regulated the abnormal immunological memory processes found in these patients. Scientists documented a significant depletion of long-lived plasma cells, a reduction of interferon type I activity, and a down-regulation of T-cell transcripts associated with chronic inflammation. Bioelectronic Platform.

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New Drug Approvals

OCTOBER 24, 2023

This approval was based on favorable results obtained from Pfizer’s Elevate UC Phase III registrational program, consisting of the Elevate UC 52 and Elevate UC 12 clinical trials, that investigates the efficacy of a 2-mg daily dose regimen of etrasimod, with a 32% and 26% remission rate observed in UC 52 and UC 12 trials respectively.

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The Pharma Data

APRIL 8, 2021

New 2-year findings from Part 2 of the Phase II/III FIREFISH trial show longer-term efficacy and safety of EVRYSDI in infants with symptomatic Type 1 SMA treated with EVRYSDI. P6: Neuromuscular Disorders and Clinical Trials. P6: Neuromuscular Disorders and Clinical Trials. Multiple Sclerosis (MS).

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The Pharma Data

OCTOBER 18, 2020

“We diligently and thoughtfully prepared for this trial,” said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer, “and we are grateful for the opportunity to treat relapsed or refractory AML patients with CG-806. Forward Looking Statements.

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The Pharma Data

APRIL 11, 2023

A focus of the presentation will be on the target engagement results and pharmacokinetics from its ongoing monotherapy dose-escalation study to explore a potential optimal dose and schedule to effectively inhibit AhR.

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Agency IQ

AUGUST 16, 2024

By Amanda Conti | Aug 13, 2024 10:00 PM CDT Regulatory context: Psychedelic regulation and drug development A growing body of evidence suggests that psychedelics may provide clinical benefit for certain purposes, especially mental health conditions. FDA accepted the Lykos NDA in February 2024, and the submission received priority review.

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Agency IQ

SEPTEMBER 15, 2023

BY AMANDA CONTI SEP 13, 2023 1:58 PM CDT Quick background on nonprescription drug regulation Nonprescription drugs, also known as over-the-counter (OTC) drugs, are regulated differently than traditional prescription drugs. The committee also made recommendations regarding pharmacokinetic and safety assessments.

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Agency IQ

DECEMBER 11, 2023

So far, the key takeaway was that both industry and regulators are still identifying best practices on setting enrollment targets in their research plans. The provisions in FDORA included a specific section on “Clinical Trial Diversity and Modernization,” (found at Subtitle F, Chapter 1 ).

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The Pharma Data

MAY 17, 2023

Additionally, pre-clinical data have shown fenebrutinib to be potent and highly selective, and it is the only reversible inhibitor currently in Phase III trials for MS. “I Fenebrutinib significantly reduced the total number of new gadolinium-enhancing T1 brain lesions compared to placebo, the primary endpoint of the trial (p=0.0022).

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The Pharma Data

DECEMBER 13, 2020

The STAMP-NAFLD trial was a 12-week, randomized, parallel group study in 120 presumed NASH patients with or without type 2 diabetes (T2DM). The Company additionally announced new preclinical results and plans for a Phase 2b trial focused on patients with noncirrhotic biopsy-proven NASH and coexisting prediabetes or T2DM. and in Europe.

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The Pharma Data

JULY 27, 2021

. “Over the last five years, Teneobio developed leading-edge expertise in efficiently engineering differentiated multispecific and bispecific therapeutics for numerous indications with potentially better safety, efficacy and pharmacokinetic profiles than the first generation of T-cell engagers. ” In June 2021, AbbVie Inc.

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Agency IQ

AUGUST 11, 2023

The company highlights several applications relevant to life sciences regulation, including pharmacovigilance and quality assurance and regulatory compliance management. A core focus of BIMO is on clinical data integrity and uncovering mistakes or outright clinical trial fraud.

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The Pharma Data

AUGUST 29, 2020

About the Study NCT03439839 is a Phase II, multicenter, open-label, sequential 2-cohort trial to assess the safety, efficacy, tolerability and pharmacokinetics/pharmacodynamics of LNP023 in PNH patients (cohort 1: n=10) with active hemolysis despite treatment with eculizumab.

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Agency IQ

FEBRUARY 23, 2024

On the first day, discussants looked at how to tackle first-in human trials, weighed the importance of the maximum tolerated dose, and looked at the evolving concept of the clinical utility index. Read AgencyIQ’s analysis of the draft guidance document here , and the two FDA-ASCO meetings here and here.]

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Agency IQ

AUGUST 9, 2024

estimated prevalence of the condition of interest, any relevant pharmacokinetic or pharmacogenomic data, demographic factors) and how they intend to meet those goals, as well as operational and process factors such as how they should be submitted and how the “action plan” may be updated over time. What about combination products?

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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. As of mid-2024, this includes at least 650 Phase I-IV trials, with about 430 of those already ongoing.

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Alta Sciences

DECEMBER 31, 2024

The issue also features a drug-drug interaction case study, in which the effects of two doses of a sponsors investigational product on the pharmacokinetics of multiple oral doses of clopidogrel, and a single dose of warfarin in healthy adult subjects were evaluated. The Altascientist, Issue 7, pg.

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The Pharma Data

JULY 21, 2021

An overview of the islatravir treatment and prevention development program is available here , which includes our two Phase 3 IMPOWER trials evaluating islatravir as once-monthly oral PrEP across diverse populations of people who may benefit from additional HIV-1 prevention options. Phase 2a Oral Study Results for Investigational Islatravir.

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Alta Sciences

JULY 31, 2024

How Improving Diversity Can Benefit Clinical Trials pmjackson Wed, 07/31/2024 - 19:19 In July 2024, the U.S. Food and Drug Administration (FDA) published a draft guidance to ensure greater diversity in clinical trials, which is expected to become a final guidance by June 2025.

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The Pharma Data

DECEMBER 28, 2020

The AMBITION trial is the first placebo-controlled study of CRV431 in NASH patients with evidence of moderate-to-severe fibrosis. The primary objectives of the AMBITION trial are to assess safety and tolerability of CRV431, as well as to delineate pharmacokinetics. To date, there are no approved drugs to treat NASH.

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Agency IQ

DECEMBER 11, 2023

Previously, burdensome side effects may have been written off as a necessary part of treatment and benefits measured in additional weeks or months of survival time; today, though, patients, clinicians, and regulators expect more. These issues are especially apparent in treatments that are intended for chronic use.

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DrugBank

FEBRUARY 26, 2025

Quorum sensing , a process by which bacteria communicate to regulate group behaviors, is one promising target. Clinical trials are currently evaluating bacteriophage therapy for MDR infections, including MRSA and Pseudomonas aeruginosa. Regulators are also adapting to the urgency of the antibiotic resistance crisis.

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FDA Law Blog: Drug Discovery

JANUARY 7, 2024

A PMR is a study “that sponsors are required to conduct under one or more statutes or regulations,” whereas a PMC is a study “that a sponsor has agreed to conduct, but that are not required by a statute or regulation” (see FDA Webpage, Postmarketing Requirements and Commitments: Introduction ).

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New Drug Approvals

SEPTEMBER 29, 2024

Findings Two Phase 1 trials of atuzaginstat were completed by June 2019. The pharmacokinetic profiles of COR388 in AD and controls were reported to be similar. A Phase 2/3 trial (GAIN) evaluating a 48-week course of COR388 in 643 people with mild to moderate AD began in April 2019. This trial involves 93 sites in the U.S.

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