Drug Target Review

JUNE 11, 2025

Clinical genomics, powered by NGS, enables more precise target validation, improved patient stratification and optimised trial design, ultimately aiming to increase PoS. For example, MLMs can simultaneously explore genetic sequences and clinical data to improve crucial characteristics such as efficacy, safety and bioavailability.

Drugs

Drug Target Review

NOVEMBER 25, 2024

As the CEO of iOnctura, an innovative oncology biopharmaceutical company she co-founded in 2017, Catherine has played a key role in advancing the development of highly targeted small molecules aimed at revolutionising cancer treatment. Additionally, we are preparing to launch several other trials over the coming months.

Treatment

The Pharma Data

JUNE 25, 2025

In March 2025 , Regulus announced the successful completion of its Phase 1b multiple-ascending dose clinical trial for farabursen. These modalities offer a new layer of precision in targeting diseases that are difficult to address with conventional small molecules or biologics.

RNA

The Pharma Data

AUGUST 1, 2025

The committee highlighted the limited therapeutic options available for WM patients following BTK inhibitor therapy and acknowledged both the strong scientific rationale and the encouraging clinical data to date. BeOne Medicines is a global oncology company committed to discovering and developing transformative therapies for cancer patients.

Targeted Protein Degradation

Alta Sciences

JULY 3, 2025

Attendees were particularly drawn to our integrated solutions, which encompass everything from lead candidates advancing to IND-enabling programs to early-phase clinical trials to full-scale commercial manufacturing. Beyond the science, what’s exciting is how this technology opens doors. The response?

Small Molecule

Drug Target Review

JULY 18, 2025

Whether we are working with small molecules or larger biologics, our platform enables a precise design of innovative drug candidates with highly differentiated profiles.” Preparing for the clinic Preclinical success is just the beginning. “The

Medical Chemistry

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] 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]

Small Molecule

BioPharma Drive: Drug Pricing

JULY 8, 2025

Specially designed small molecule drugs can bring together target proteins with an enzyme that tags them with ubiquitin, thereby marking them for destruction. In their 2001 paper, Crews, Deshaies and others described how synthetic molecules could attach ubiquitins to unwanted cell parts. "It You can unsubscribe at anytime.

Targeted Protein Degradation

Drug Target Review

APRIL 7, 2025

Advancements in screening technologies for small-molecule drug discovery including cellular assays, computational screening, and biophysics-based methods enhanced by structural biology breakthroughs have improved screening hit rates and facilitated the identification of drug candidates for previously undruggable targets.

Fragment Screening

Broad Institute

OCTOBER 29, 2024

This small molecule reactivates the apoptosis cascade in tumor cells while sparing healthy cells in animal models. Apoptosis, or programmed cell death, is a natural end process for all cells. This rapid clearance minimizes the drug’s potential impact on healthy cells.

Drugs

The Pharma Data

JUNE 24, 2025

Notably, over 80% of Bayer’s major global multi-center clinical trials now include study sites in China, spanning early-phase development to late-stage pivotal trials. The company currently operates two global research and development (R&D) centers and four world-class production facilities across China.

Pharma R&D

New Drug Approvals

APRIL 2, 2025

1] [2] It is a non- opioid , small-molecule analgesic that works as a selective inhibitor of Na v 1.8 The efficacy of Journavx was evaluated in two randomized, double-blind, placebo- and active-controlled trials of acute surgical pain, one following abdominoplasty and the other following bunionectomy. ESI-MS m/z calc.475.0922,

FDA

Codon

JULY 28, 2025

That year, Terrence Flotte’s team at Johns Hopkins University used a natural strain of AAV, called AAV2, to introduce a cystic fibrosis transmembrane conductance regulator (CFTR) gene — defective in those with cystic fibrosis — into patients during a phase 1 clinical trial.

Immune Response

New Drug Approvals

JUNE 25, 2025

clinical candidate currently in Phase 2 development. Several glucokinase activators have advanced to clinical studies and demonstrated promising efficacy; however, many of these early candidates also revealed hypoglycemia as a key risk.

Small Molecule

New Drug Approvals

JULY 7, 2025

1] Pimicotinib is under investigation in clinical trial NCT05804045 (Study of Pimicotinib (ABSK021) for Tenosynovial Giant Cell Tumor (MANEUVER)). 1] Pimicotinib is under investigation in clinical trial NCT05804045 (Study of Pimicotinib (ABSK021) for Tenosynovial Giant Cell Tumor (MANEUVER)). 6] The U.S. 10 April 2024.

Small Molecule

New Drug Approvals

JULY 10, 2025

“Sebetralstat (KVD900): A Potent and Selective Small Molecule Plasma Kallikrein Inhibitor Featuring a Novel P1 Group as a Potential Oral On-Demand Treatment for Hereditary Angioedema” Journal of Medicinal Chemistry. Retrieved 9 July 2025 – via Business Wire. October 2022). 65 (20): 13629–13644. doi : 10.1021/acs.jmedchem.2c00921.

FDA

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). Resigratinib, KIN 3248 CAS 2750709-91-0 C 26 H 27 F 2 N 7 O 3 523.5

Production

New Drug Approvals

APRIL 18, 2025

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.

FDA

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.

Clinical Trials

New Drug Approvals

JUNE 9, 2025

Clinical trials: Clinical trials have demonstrated the safety and potential efficacy of hexasodium phytate in reducing hydroxyapatite crystallization in patients undergoing hemodialysis, providing a basis for its use in treating calciphylaxis.

Clinical Trials

BioPharma Drive: Drug Pricing

JULY 14, 2025

PCI helps biopharmaceutical companies manufacture and package drug products used in clinical trials as well as commercially. into a “ center of excellence ” that will produce a wide range of small molecules and biologic drugs, including protein-degrading “PROTACs” and antibody-drug conjugates. TechTarget, Inc.s

Production

New Drug Approvals

JUNE 20, 2025

MIVORILANER 1414642-93-5 Molecular Formula C22H17Cl2F6N3O3S Molecular Weight 588.35 g, 93%).

Small Molecule

Codon

JULY 17, 2025

Perkins’ observation drew little attention for a decade or so, until a group of Stanford doctors dug it up and ran a clinical trial to test the effects of DNP on overweight participants. The results of this trial, and its subsequent coverage in the press, kicked off a surge in demand for DNP.

Drugs

Science Daily: Pharmacology News

APRIL 8, 2025

Experimental drug NU-9 -- a small molecule compound approved by the U.S. Food and Drug Administration (FDA) for clinical trials for the treatment of amyotrophic lateral sclerosis (ALS) -- improves neuron health in animal models of Alzheimer's disease, according to a new study.

Small Molecule

Drug Hunter

APRIL 20, 2023

The molecule was the first allosteric TYK2 inhibitor to enter clinical trials, is the first JAK-family kinase inhibitor approved for psoriasis, and is also the first approved drug targeting a pseudokinase domain. MRTX1133 is currently in Ph I/II trials as an oral agent. #3: LP0200 has completed a Ph.

Small Molecule

Drug Hunter

MAY 31, 2023

This article compiles 20+ recent small molecules of general interest in the news in April 2023, with structures where they are available. I/II Data request a trial You don’t have time to read everything, but you can’t afford to fall behind. Get ahead now by requesting a trial. already a member?

Small Molecule

New Drug Approvals

JULY 21, 2025

A multicenter, randomized phase III trial of hetrombopag: a novel thrombopoietin receptor agonist for the treatment of immune thrombocytopenia. Hetrombopag Olamine , RAFUTROMBOPAG OLAMINE Hetrombopag diolamine SHR8735 olamine Hetrombopag ethanolamine SHR-8735 olamine 580.6 Xiong, J.; 2021, 14, 37. (50) 50) Shi, A.;

Small Molecule

New Drug Approvals

JULY 28, 2025

2] Dorzagliatin is under investigation in clinical trial NCT03173391 (Long-term Efficacy and Safety of HMS5552 in T2DM). 133 This first-in-class, small molecule,oral,glucokinaseactivator(GKA)wasfirst approved in ChinainSeptember2022foradultpatientswithT2DMasa monotherapy and in combination with metformin (an antidiabetic medication).134

Small Molecule

SCIENMAG: Medicine & Health

JUNE 27, 2023

Credit: Insilico Medicine […]

Trials

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. 2 However, when dosed at the MTD, ADCs display improved efficacy over small molecules in oncology trials. 3D rendering of Antibody Drug Conjugate Molecules.

Small Molecule

Covalent Modifiers

JULY 25, 2023

[link] Target validation remains a challenge in drug discovery, which leads to a high attrition rate in the drug discovery process, particularly in Phase II clinical trials. Consequently, new approaches to enhance target validation are valuable tools to improve the drug discovery process.

Small Molecule

ProRelix Research

NOVEMBER 23, 2022

Biological products have unlocked the potential for the management of several diseases such as cancers and autoimmune diseases for which treatment with small molecule, chemically synthesized drug molecules remain suboptimal. […].

Clinical Trials

Broad Institute

OCTOBER 4, 2023

By Allessandra DiCorato October 4, 2023 Credit: AbbVie The new small molecule inhibitor (green) sits inside the PTPN2 protein, where acidic sites are marked in red and basic sites are marked in blue. A new small-molecule drug candidate being tested in an early-stage clinical trial aims to improve patient responses to immunotherapy.

Small Molecule

Drug Target Review

NOVEMBER 1, 2023

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. J Med Chem.

Clinical Trials

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.

Small Molecule

DrugBank

JULY 24, 2024

These multifunctional small molecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. Multifunctional Small Molecules and Peptides Beyond PROTACs, there are other exciting tools in the new drug discovery toolbox.

Small Molecule

Alta Sciences

APRIL 17, 2024

Planning a First-In-Human Clinical Trial? blussier Wed, 04/17/2024 - 20:52 HTML Unique FIH Solutions for Small and Large Molecules Advancing your molecule to first-in-human (FIH) clinical trials is a major milestone.

Clinical Trials