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It is a smallmolecule with a dual mechanism of action, acting as both a complete estrogen receptor antagonist and a selective estrogen receptor degrader (SERD). Clinical Trials: Palazestrant is currently in clinical trials, including Phase 1/2 and Phase 3 studies, for the treatment of ER+, HER2- metastatic breast cancer.
A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome. Diagnostic biomarkers typically confirm or establish a diagnosis and are often used in selecting patient populations for clinical trials.
Orforglipron ( LY-3502970 ) is an oral, non-peptide, small-molecule GLP-1 receptor agonist developed as a weight loss drug by Eli Lilly and Company. [1] 1] [3] Clinical trials The results of Phase I safety and Phase II ascending-dose clinical trials enrolling people with obesity or type 2 diabetes were published in 2023. [4]
This smallmolecule reactivates the apoptosis cascade in tumor cells while sparing healthy cells in animal models. Previous clinical and laboratory studies on other MCL1 inhibitors have suggested that these molecules can impair heart cells, likely because of prolonged exposure to the compounds.
2011, 2 , 828-839 DOI: 10.1039/C1MD00116G [link] [link] Glucokinase is a key regulator of glucose homeostasis and smallmolecule activators of this enzyme represent a promising opportunity for the treatment of Type 2 diabetes. clinical candidate currently in Phase 2 development.
1] [2] [3] KIN-3248 is a smallmolecule that targets and inhibits oncogenic fibroblast growth factor receptors (FGFRs). The safety, tolerability, pharmacokinetics, and preliminary efficacy of KIN-3248 are currently being evaluated in adults with advanced tumors harboring FGFR2 and/or FGFR3 gene alterations.
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.
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.
Table 1: Smallmolecule drugs approved by the FDA in 2023 with reported involvement of phase II mechanisms In vitro : In vivo differences Incubation of the SGLT2 (sodium-glucose co-transporter-2) inhibitor bexagliflozin in human liver microsomes points to metabolism through both oxidation and glucuronidation to 6 main metabolites.
Pharmacokinetics, Pharmacodynamics and Toxicokinetics Demystified pmjackson Wed, 01/31/2024 - 14:55 Understanding the effects of a drug, and how it interacts with the body, and vice versa, is critical to ensure it is safe for human use. This is where pharmacokinetic (PK), pharmacodynamic (PD) , and toxicokinetic (TK) analyses step in.
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.
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.
2 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 1-5 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. J Med Chem. 2017 Mar 9;60(5):2006–17. Miller LM, Keune WJ, Castagna D, et al.
Pending Health Canada’s approval, the Phase 1 trial is designed to test the safety, tolerability and pharmacokinetics of ALS-4 in healthy volunteers. ALS-4 is a novel smallmolecule adopting an anti-virulence (non-antibiotic) approach to address the growing unmet medical needs of infections caused by Staphylococcus aureus.
Applying our technology has enabled us to develop a new smallmolecule, TT125-802, and to assign a new role to the epigenetic regulator CBP/p300 as a novel master regulator of non-oncogene resistance. This orally available smallmolecule binds to the bromodomain of CBP/p300 in a highly specific manner.
In the absence of a clinical trial result or FDA label to point to, how does one create the case and target product profile (TPP) around a new target? and whether a molecule’s pharmacology can help to mitigate safety risk. Human and mouse genetics can inform not only efficacy but also safety. in liver, in CNS)?
Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a smallmolecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial.
Years later, a subgroup analysis of the trial data indicated a potential positive effect in participants who carried two copies of ApoE4 ( Abushakra et al., Clinical Pharmacokinetics and Safety of ALZ-801, a Novel Prodrug of Tramiprosate in Development for the Treatment of Alzheimer’s Disease. Clin Pharmacokinet.
The study demonstrated favorable proof-of-concept for LYT-100’s tolerability and pharmacokinetic (PK) profile, which will also enable twice-a-day (BID) dosing of LYT-100 in future studies. Photo: Business Wire). Multiple ascending dose and food effect study results.
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.
With 13 preclinical candidates and three AI-designed drugs currently undergoing clinical trials, Insilico is spearheading a revolution in cancer treatment and beyond. What are the preclinical characteristics of ISM6331, including its efficacy, safety profile, and drug metabolism and pharmacokinetics (DMPK) properties?
18, 2021 /PRNewswire/ — Genkyotex SA , a subsidiary of Calliditas Therapeutics AB (publ) (“Calliditas”) (Nasdaq OMX – CALTX; NASDAQ – CALT), today announced positive Phase 1 data demonstrating a favorable safety and pharmacokinetic profile of high-dose setanaxib, Genkyotex’s lead asset. STOCKHOLM , Jan.
In November and December, several large pharmas held “AI Day” presentations featuring LLM applications for clinical trial data analysis. These information retrieval capabilities have many applications, from writing computer code and collating clinical trial results to summarizing papers on a specific topic.
SetPoint Medical received FDA Investigational Device Exemption (IDE) approval for a multicenter, double-blind, randomized, sham-controlled pivotal trial that will enroll up to 250 patients at 40 clinical trial sites in the U.S. SmallMolecule Inhibitors. The secondary outcome involves pharmacokinetic endpoints.
This smallmolecule therapy is presently in Phase 1 clinical trial for mild to moderate Alzheimer’s disease (AD), which is supported by a NIA R01 grant in healthy aged volunteers. CLARKSVILLE, Md., 20, 2021 (GLOBE NEWSWIRE) — Neuronascent Inc. , About Neuronascent, Inc.
g/mol 1929519-13-0 NBI-1065846 or TAK-041 Phase 2 (S)-2-(4-oxobenzo[d][1,2,3]triazin-3(4H)-yl)-N-(1-(4-(trifluoromethoxy)phenyl)ethyl)acetamide Zelatriazin ( NBI-1065846 or TAK-041 ) is a small-molecule agonist of GPR139. Zelatriazin, C 18 H 15 F 3 N 4 O 3 , 392.3 88 (8): 3872–3882. doi : 10.1111/bcp.15305. PMC 9544063.
Of note, this guidance is specific to smallmolecule pharmaceuticals; similar guidance for biologics can be found in the ICH’s S6(R1) Preclinical Safety Evaluation for Biotechnology-Derived Pharmaceuticals. Pharmacokinetic and systemic exposure data is also important to consider.
For example, pharmacokinetic (PK) data from a comparative BA study and PK modeling approaches (e.g., For example, pharmacokinetic (PK) data from a comparative BA study and PK modeling approaches (e.g., patients with renal, hepatic, or cardiovascular concerns). patients with renal, hepatic, or cardiovascular concerns).
Foghorn is anticipating filing an IND later this year for its lead candidate to begin a Phase I trial for the treatment of uveal melanoma, a cancer of the eye. . The company currently has over 10 programs in its pipeline. Scorpion Therapeutics . Orbus Therapeutics . The $71 million funding includes an initial financing of $32.5
Once a new drug target is identified, a proprietary deep learning artificial intelligence (AI) platform we call Fluency can rapidly identify smallmolecules that selectively bind to a target of interest. We have so far observed extraordinary PK C max levels and a mean half-life of approximately two hours in our Phase I trial to date.
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.
“We diligently and thoughtfully prepared for this trial,” said William G. The Company’s smallmolecule cancer therapeutics pipeline includes products designed to provide single agent efficacy and to enhance the efficacy of other anti-cancer therapies and regimens without overlapping toxicities. Rice, Ph.D.,
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Initiated CTP-543 THRIVE-AA1 Phase 3 Trial in November 2020. The THRIVE-AA1 Phase 3 trial is a randomized, double-blind, placebo-controlled clinical trial of CTP-543 to evaluate hair regrowth using the Severity of Alopecia Tool (SALT) after 24 weeks of dosing in approximately 700 adult patients with moderate to severe alopecia areata.
However, the CYP51 inhibitor posaconazole (an antifungal medication) showed poor efficacy in a clinical trials for chronic Chagas disease. Does this mean that CYP51 is a bad target? The quick answer is “maybe but maybe not” because we can’t really tell whether the lack of efficacy is due to irrelevance of the target or inadequate exposure.
2] As of July 2022, it is in phase 3 clinical trials for major depressive disorder. [2] hours following a single dose of 10 mg, which supported the 4 mg to 25 mg dosages that aticaprant is being explored at in clinical trials. [13] 14] Pharmacokinetics The oral bioavailability of aticaprant is 25%. [1] nM vs. 24.0 nM vs. 24.0
Development of TransCon CNP is progressing as planned with the recent initiation by VISEN Pharmaceuticals of a second phase 2 trial in patients with achondroplasia, the ACcomplisH China Trial, which provides for dose expansion at an effective dose determined from the ACcomplisH Trial.
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Fifteen oligonucleotides have so far received market authorization in different countries, and several others are being test in clinical Phase I to III trials. Several smallmolecule GLP-1R agonists, such as oral orforglipron, are in late-stage clinical development.
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. Blood 2019;134(Suppl 1):3517. 3. Schubart A, et al.
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. This has spawned a movement focused on optimizing dose selection in oncology drug development.
In switching studies , a two-arm trial design is commonly employed. in equipment) for products that are more complex or aren’t as well-characterized, such as antibody drug conjugates or recombinant DNA products, will be considered higher risk than they would if the change was made to the production of standard, smallmolecule products.
link] 01 Aug 2022 Cortexyme is now called Quince Therapeutics You need to be a logged in or subscribed to view this content This smallmolecule is an orally available protease inhibitor targeting the lysine proteases of the periodontal pathogen Porphyromonas gingivalis. This trial involves 93 sites in the U.S. and Europe.
A new treatment for G4-targeted solid tumors including pancreatic cancer has been administered to patients for the first time in a Phase 1a clinical trial. This latest development follows the US Food and Drug Administration (FDA) giving clearance for QN-302 in July 2023 to proceed to this initial clinical trial stage.
However, nonclinical studies are not conducted exclusively before human trials begin; they usually continue throughout the early phases of clinical trials to further assess a product’s performance on different metabolic pathways and at different doses.
Clinical trials are currently evaluating bacteriophage therapy for MDR infections, including MRSA and Pseudomonas aeruginosa. Developing Antimicrobial Peptides and Biomolecules In addition to traditional small-molecule antibiotics, researchers are exploring the potential of antimicrobial peptides (AMPs) and other biomolecules.
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