Drug Target Review

JUNE 16, 2025

Unlike traditional pharmacokinetic (PK) studies that rely on plasma measurements alone, MSI, when combined with traditional histology, enables spatial mapping of drug distribution, metabolism and target engagement. Toxicological investigations play a key role in assessing drug safety and, importantly, inform development decisions.

Drugs

Broad Institute

OCTOBER 29, 2024

New drug triggers rapid cell death in cancer models By Karen Zusi-Tran October 29, 2024 Breadcrumb Home New drug triggers rapid cell death in cancer models BRD-810 inhibits the MCL1 protein and reactivates apoptosis in tumor cells, displaying therapeutic potential in animal models. The result was the compound named BRD-810.

Drugs

New Drug Approvals

JULY 2, 2025

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] 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]

Small Molecule

New Drug Approvals

JUNE 25, 2025

Several drugs in five major categories, each acting by different mechanisms, are available for treating hyperglycemia and subsequently, NIDDM (Moller, D. E., “New drug targets for Type 2 diabetes and the metabolic syndrome” Nature 414; 821 -827, (2001 )): (A) Insulin secretogogues, including sulphonyl-ureas (e.g.,

Small Molecule

New Drug Approvals

JULY 12, 2025

1] [2] [3] KIN-3248 is a small molecule 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. February 2022).

Production

New Drug Approvals

APRIL 18, 2025

2] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [4] 1] [2] Adverse effects The US Food and Drug Administration prescription label for crinecerfont has a warning for acute adrenal insufficiency or adrenal crisis. [2] Food and Drug Administration (FDA) (Press release). January 2025.

FDA

Alta Sciences

APRIL 24, 2025

Altasciences was chosen by a client for an IND-enabling study of a small-molecule GLP-1 receptor agonist in canines. The canines were dosed once daily for 28 days via tablets with a water flush to ensure administration.

Small Molecule

PPD

APRIL 14, 2025

The SELECT trial set out to understand whether the drug has similar effect on patients without diabetes. Participant fatigue in GLP-1 clinical trials The success of GLP-1 drugs in SELECT and other trials has biotech and biopharma companies racing to find which indications respond well to similar treatment.

Clinical Trials

New Drug Approvals

JULY 28, 2025

1] Unlike other diabetes drugs, it is intended to increase insulin sensitivity. [2] 133 This first-in-class, small molecule,oral,glucokinaseactivator(GKA)wasfirst approved in ChinainSeptember2022foradultpatientswithT2DMasa monotherapy and in combination with metformin (an antidiabetic medication).134 Drugs 2022, 82, 1745−1750.

Small Molecule

New Drug Approvals

JULY 21, 2025

Drugs 2021, 81, 1581−1585. (47) Safety, pharmacokinetics and pharmacodynamics of hetrombopag olamine, a novel TPO-R agonist, in healthy individuals. Hetrombopag Olamine , RAFUTROMBOPAG OLAMINE Hetrombopag diolamine SHR8735 olamine Hetrombopag ethanolamine SHR-8735 olamine 580.6 Developer Atridia; Jiangsu Hengrui Medicine Co.

Small Molecule

Metabolite Tales Blog

APRIL 10, 2024

Where data was publicly available, the routes of human metabolism for each of the drugs in this subset is listed in Table 1. Metabolites found in humans are also observed in monkeys, and all metabolites were found to possess <10% of the activity of the parent drug. of the administered radiolabel in the human ADME study.

Small Molecule

Metabolite Tales Blog

APRIL 4, 2023

Metabolism of 2022 FDA approved small molecule drugs part 2 Mixing it Up By Julia Shanu-Wilson In Part 1 of this topic we looked at metabolism of the small molecule drugs approved by the FDA in 2022 that were mediated by CYP3A4.

Small Molecule

Metabolite Tales Blog

JANUARY 23, 2024

Metabolism of 2023 FDA Approved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. Enzymes involved include CYP1A2, CYP2C8, CYP3A4, CYP4F2, and aldehyde oxidase (AOX).

Small Molecule

Alta Sciences

JANUARY 31, 2024

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.

Pharmacokinetics

DrugBank

OCTOBER 17, 2024

Data science has emerged as an innovative tool in the biopharmaceutical industry, leveraging the power of machine learning and artificial intelligence to drive innovation and efficiency across the entire drug development lifecycle.

Drug Development

Drug Hunter

MAY 18, 2023

Dian Su’s journey from chemistry and proteomics to the DMPK of drug conjugates Following a different path comes naturally to Dian Su, Senior Director of DMPK at Bicycle Therapeutics. In her small molecule DMPK research, proteomics also served to identify reactive metabolites (e.g., Proteomics!

Small Molecule

DrugBaron

FEBRUARY 7, 2022

Finding small molecule drugs is much harder than finding a needle in a haystack – discovering the right arrangement of atoms to bind precisely to a protein target to elicit a particular response is a problem of vast dimensionality. Yet the situation with small molecules is even worse.

Small Molecule

Drug Target Review

SEPTEMBER 25, 2024

While these approaches often produce encouraging initial results, the development of drug resistance remains a major obstacle for long-term patient survival. Classically, rational drug combinations are designed to target two nodes in the same oncogenic signalling pathway.

Small Molecule

The Pharma Data

JANUARY 19, 2021

Dr. Clark Cheng, Chief Medical Officer and Executive Director of Aptorum Group, commented, “The clearance of our CTA application for ALS-4 drug represents a significant milestone for the company and one of a number of targeted strategic goals for 2021. About ALS-4.

Small Molecule

Practical Cheminformatics

JANUARY 8, 2024

Picking up where we left off in Part I , this post covers several other ML in drug discovery topics that interested me in 2023. Most of the LLM activity in the drug discovery space in 2023 was reported as preprints from academic groups. Most of the drug discovery examples were underwhelming. Here’s the structure of Part II.

Drugs

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. ALS-4 is a novel small molecule adopting an anti-virulence (non-antibiotic) approach to address the growing unmet medical needs of infections caused by Staphylococcus aureus.

Small Molecule

The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule 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.

Disease

LifeSciVC

APRIL 24, 2024

Is a novel target at the inflection point where enough evidence is available to suggest it may prove to be a compelling drug? and whether a molecule’s pharmacology can help to mitigate safety risk. In order to start building a case for or against a target, I like to start with genetics – first human and then mouse. in liver, in CNS)?

Production

The Premier Consulting Blog

MAY 31, 2023

As a cornerstone of the drug development process, nonclinical investigational new drug (IND)-enabling studies are essential for supporting first-in-human (FIH) dosing for novel therapeutics. Pharmacology A typical IND-enabling package includes information on the primary, secondary, and safety pharmacology of the drug.

Packaging

Drug Target Review

NOVEMBER 1, 2023

3 Another recent development is the implication of ATX in neurological diseases, 6,7 as ATX levels are related to metabolic disorders in Alzheimer disease, and thus might be an interesting biomarker and drug target for this devasting pathology. Rational Design of Autotaxin Inhibitors by Structural Evolution of Endogenous Modulators.

Clinical Trials

New Drug Approvals

OCTOBER 12, 2024

ALZ-801 is a potent and orally available small-molecule β-amyloid (Aβ) anti-oligomer and aggregation inhibitor, valine-conjugated proagent of Tramiprosate with substantially improved PK properties and gastrointestinal tolerability compared with the parent compound. Clin Pharmacokinet. 2016 ; Abushakra et al., Hey JA, et al.

Pharmacokinetics

The Pharma Data

NOVEMBER 17, 2020

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. The therapeutic dose of pirfenidone approved by the US Food and Drug Administration (FDA) for the treatment of IPF is 801 mg three times a day.

Pharmacokinetics

The Premier Consulting Blog

JULY 31, 2024

In this blog, we explain the role of clinical pharmacology in drug development and demonstrate how the right strategy can accelerate development under the US Food and Drug Administration (FDA) 505(b)(1) and 505(b)(2) New Drug Application (NDA) pathways.

Clinical Pharmacology

Drug Target Review

JUNE 12, 2023

This molecule shows immense potential as a pan-TEAD inhibitor, targeting advanced solid tumours. TEAD proteins are crucial for tumour progression and drug resistance, making them an attractive focus for therapeutic interventions. ISM6331 is a Pan-TEAD inhibitor with novel scaffold with good IP space, discovered by Chemistry42.

Metabolic Stability

The Premier Consulting Blog

AUGUST 8, 2024

On July 31, 2024, the US Food and Drug Administration (FDA) announced Fiscal Year 2025 (FY2025) Prescription Drug User Fee Amendments of 2022 (PDUFA VII) fee rates for the review of human drug and biological product applications along with prescription drug program fees.

Drugs

The Premier Consulting Blog

SEPTEMBER 11, 2024

The 505(b)(2) new drug application (NDA) pathway offers a unique opportunity for small molecule developers to bring innovative products to market more efficiently by leveraging existing data they do not own or have right of reference to. Since there was no change in drug substance, the sponsor was able to reference the DMF.

Drugs

The Pharma Data

NOVEMBER 11, 2020

Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for SLV213 for the treatment of COVID-19 and has dosed the first subjects in a Phase 1 clinical study. While SLV213 can be dosed orally or intravenously, Selva is first advancing it as an oral drug candidate for COVID-19. About SLV213.

Treatment

Metabolite Tales Blog

FEBRUARY 16, 2024

This results in cleavage of the drug to two metabolites, M1 and M9, with only M1 retaining the 14C label. of total drug related material in plasma. Biotransformation studies which reveal significant cleavage of a drug could thus trigger a route involving dual radiolabelling. Eur J Drug Metab Pharmacokinet 48 , 411–425 (2023).

Small Molecule

Drug Target Review

JULY 12, 2023

These assays provide insights into the molecular mechanisms of disease biology and drug response, enabling the characterisation of gene expression profiles and deviations in diseased cells. This platform can help identify and optimise pharmacologic properties of new drugs.

Biochemical Assays

The Premier Consulting Blog

JANUARY 31, 2023

Food and Drug Administration (FDA) adopted an addendum to the guidance titled “ S1B(R1) Testing for Carcinogenicity of Pharmaceuticals ,” which had previously been finalized by the International Council for Harmonization (ICH). Pharmacokinetic and systemic exposure data is also important to consider. On November 1, 2022, the U.S.

Small Molecule

Metabolite Tales Blog

DECEMBER 13, 2023

N -glucuronidation: the human element By Julia Shanu-Wilson In our last blog of the year, we look at why N -glucuronidation of drugs is important in human drug metabolism. Glucuronidation is the most common phase II reaction observed in the metabolism of drugs in humans. In fact, rodents lack a human UGT1A4 homologue gene [3].

Small Molecule

New Drug Approvals

SEPTEMBER 10, 2024

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 49 (2): 121–132. doi : 10.1124/dmd.120.000246. 120.000246.

Clinical Pharmacology