Drug Target Review

APRIL 7, 2025

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.

Clinical Development 69

Clinical Development Clinical Trials Clinical Pharmacology Trials 69

SCIENMAG: Medicine & Health

JUNE 27, 2023

Credit: Insilico Medicine […]

Small Molecule 91

Small Molecule Trials Clinical Trials Drugs 91

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 145

Small Molecule Drugs Clinical Pharmacology Trials 145

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 64

Drugs Clinical Trials Drug Development Pharmaceuticals 64

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 57

Small Molecule Clinical Trials Trials Pharmacokinetics 57

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 62

Small Molecule Clinical Trials Licensing Licensing 62

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 130

Small Molecule Clinical Trials Trials Drugs 130

Drug Target Review

JULY 3, 2025

It is becoming increasingly evident that generative artificial intelligence (GenAI) is a resourceful tool for helping pharmaceutical companies reduce manual tasks required by clinical trials. Because LLMs are trained on extensive, internet-scale datasets, they can learn to identify contexts linking words and language.

Drug Development 64

Drug Development Drugs Animal Testing Engineering 64

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 52

Clinical Trials Biosimilars FDA Trials 52

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 128

Small Molecule Clinical Trials Trials Engineering 128

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 52

Treatment Small Molecule Clinical Trials Trials 52

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 98

Small Molecule Drugs Clinical Trials Therapies 98

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 52

Small Molecule Drug Development Pharmaceuticals Science 52

New Drug Approvals

JUNE 20, 2025

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

Small Molecule 62

Small Molecule Clinical Trials Trials Production 62

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 52

Small Molecule Clinical Trials Trials Pharmacokinetics 52

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 134

Clinical Trials Trials Small Molecule Disease 134

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 40

Clinical Trials Trials Small Molecule Clinical Research 40

Drug Target Review

JULY 6, 2023

1 TPD is expected to challenge undruggable proteins, which are highly difficult to target by conventional small molecules. 1 The concept of TPD was first demonstrated with a heterobifunctional small molecule to degrader a protein of interest in the early 2000s.

Targeted Protein Degradation 98

Targeted Protein Degradation Small Molecule Clinical Trials Science 98

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 52

RNA Clinical Trials Disease Small Molecule 52

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.

Drug Development 98

Drug Development Metabolic Stability Clinical Trials Drugs 98

Advarra

FEBRUARY 24, 2023

In most cases, the results you are really looking for and trying to prove in a trial can be complex. One area receiving increased focus from the Food and Drug Administration (FDA) are trial designs incorporating multiple endpoints to support efficacy. All key questions to consider and address before the therapy even leaves the lab.

Clinical Trials 52

Clinical Trials Trials FDA Therapies 52

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 40

Small Molecule Clinical Trials Trials Treatment 40

The Pharma Data

OCTOBER 25, 2020

NASDAQ: PTCT), today announced the initiation of a registration-directed Phase 2/3 clinical trial to evaluate vatiquinone (PTC743) in patients with mitochondrial epilepsy, the highly morbid condition of refractory seizures in children with inherited mitochondrial disease. . SOUTH PLAINFIELD, N.J. , Peltz , Ph.D.,

Clinical Trials 52

Clinical Trials Trials Small Molecule Regulations 52

The Pharma Data

SEPTEMBER 8, 2020

Phase III clinical trial programme initiated for investigational medicine fenebrutinib, designed to be a highly selective and reversible Bruton’s tyrosine kinase (BTK) inhibitor, in relapsing multiple sclerosis (RMS) and primary progressive MS (PPMS).

Clinical Trials 52

Clinical Trials Trials Disease FDA Approval 52

LifeSciVC

JANUARY 16, 2024

Small molecule GLP1s? Still may be some merit if they are clinical-stage and can achieve once-daily dosing without too steep a cut in efficacy or worsened adverse events vs. peptides, but be wary of the intensely competitive landscape. Additional trials (e.g.,

Small Molecule 143

Small Molecule Trials Therapies Disease 143

The Pharma Data

DECEMBER 5, 2020

.–(BUSINESS WIRE) December 5, 2020 — Syros Pharmaceuticals (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, today announced new clinical data from its Phase 2 trial evaluating SY-1425, its first-in-class selective retinoic acid receptor alpha (RAR?)

Clinical Trials 52

Clinical Trials Trials Small Molecule Treatment 52

The Pharma Data

JANUARY 10, 2021

SH ) submitted its pivotal phase III clinical trial application (IND) of national class I innovative drug Chiauranib to NMPA, for the treatment of Small Cell Lung Cancer (SCLC) as a single agent for the patients after 2nd-line systemic chemotherapy and recurrence afterwards. SHENZHEN, China , Jan.

Clinical Trials 52

Clinical Trials Treatment Trials Drugs 52

Drug Target Review

OCTOBER 11, 2023

Symptoms were reversed in mouse models and a clinical trial is planned for later this year. 5 This drug candidate is progressing towards the clinic, with phase 1 trials due to start within 18 months. Autophagy boosters, in contrast, are conventional small molecule drugs.

Disease 113

Disease Treatment Small Molecule Therapies 113

Broad Institute

NOVEMBER 2, 2023

As a result of this collaboration, Bayer’s mutant EGFR/HER2 inhibitor is currently in Phase I of clinical trials. This is the first reversible small molecule inhibitor targeting EGFR exon 20 insertion mutations and HER2 activating mutations to undergo clinical testing. Tags: Cancer Program Cancer

Therapies 105

Therapies Research Small Molecule Clinical Trials 105

Agency IQ

APRIL 19, 2024

Meet C3TI, FDA’s new clinical trial innovation hub On April 14, FDA announced that its Center for Drug Evaluation and Research (CDER) is launching a new clinical trial innovation hub, the CDER Center for Clinical Trial Innovation (C3TI). In particular, regulations in both the U.S.

Clinical Trials 40

Clinical Trials Trials Science FDA 40

The Pharma Data

JANUARY 13, 2021

PITTSBURGH–( BUSINESS WIRE )– Knopp Biosciences LLC today announced positive top-line results in a Phase 2 dose-ranging trial of the novel oral drug dexpramipexole in patients with moderate-to-severe eosinophilic asthma. The trial was conducted at 28 U.S. 14, 2021 11:00 UTC. study centers. ABOUT ORAL DEXPRAMIPEXOLE.

Trials 52

Trials Small Molecule Clinical Trials Treatment 52

The Pharma Data

OCTOBER 27, 2020

The objective of the Phase 3 clinical trial is to evaluate safety and effectiveness of 18 F-APN-1607 to differentiate patients with Mild Cognitive Impairment (MCI) and different stages of Alzheimer’s disease (AD) from healthy subjects. The proposed trial will enroll approximately 230 subjects with all receiving 18 F-APN-1607.

Clinical Trials 40

Clinical Trials Trials Small Molecule Pharmaceutical Companies 40

The Pharma Data

OCTOBER 19, 2020

is pleased to announce approval from Health Canada to proceed to a Phase 1 clinical trial with its lead asset, a long-acting anesthetic formulation for the treatment of post-surgical pain. Amaca gel has shown broad utility with an array of small-molecule drugs, biologics and cells for many different indications.

Clinical Trials 40

Clinical Trials Trials Small Molecule Production 40

Conversations in Drug Development Trends

APRIL 4, 2024

Stability Tests: For small molecule, only -20°C stability testing is required, while large molecule need both -20°C and -70°C tests if samples are intended for storage at these temperatures. The post Demystifying the ICH M10 Bioanalytical Method Validation Guidelines appeared first on Worldwide Clinical Trials.

Small Molecule 80

Small Molecule Clinical Trials Pharmaceuticals Pharmaceutical Companies 80

The Pharma Data

NOVEMBER 30, 2020

Biohaven acquires exclusive global rights to a portfolio of novel, small-molecule CGRP antagonists. The lead candidate, HTL0022562, has advanced through preclinical development demonstrating promising and differentiated properties for further investigation in human trials.

Small Molecule 40

Small Molecule Licensing Licensing Treatment 40

Drug Target Review

NOVEMBER 1, 2023

The mission of the CCDD is to discover novel small-molecule therapeutics for the treatment of cancer and progress them to hypothesis testing phase 1 clinical trials. I work in the Centre for Cancer Drug Discovery (CCDD) at The Institute of Cancer Research in London, which is an academic drug discovery centre.

Fragment Screening 96

Fragment Screening Biochemical Assays Small Molecule Biophysical Assays 96

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 52

Fragment Screening Drugs Biophysical Assays Small Molecule 52