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?

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PPD

NOVEMBER 11, 2024

Accelerate your drug development and clinical trial goals and benefit from our 360° CDMO and CRO solutions and expertise. Partner with us The post Redefining Acceleration of the Drug Development Journey appeared first on PPD.

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Fierce BioTech

MAY 27, 2025

Is it time to go beyond classic design in oncology trials? jgrcic Tue, 05/27/2025 - 11:55 Tue, 05/27/2025 - 11:55 Resource Type Whitepaper Promotion Start Tue, 05/27/2025 - 12:06 Use of healthy volunteers in first-in-human trials with oncology small molecules.

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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.

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SCIENMAG: Medicine & Health

JUNE 27, 2023

Credit: Insilico Medicine […]

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Metabolite Tales Blog

APRIL 10, 2024

Table 1: Small molecule 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.

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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.

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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.

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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. clinicaltrials.gov.

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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.

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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]

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Codon

FEBRUARY 13, 2025

Traditionally, scientists discover enzymes in nature and adapt them through trial and error. By training PLACER on structures stored in the Protein Data Bank, the team taught the model to predict atom arrangements that follow the physical and chemical rules governing protein-small molecule interactions.

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Drug Hunter

JUNE 26, 2023

This article compiles recent high-profile clinical readouts and related news with small molecules of general interest and structures where they are available. III trial ( NCT05099640 ; n=98, 7.5-60mg/kg Get ahead now by requesting a trial. III in Phenylketonuria sepiapterin (PTC923, CNSA-001) oral BH 4 precursor 7.5-60mg/kg

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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.

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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.

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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?

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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.

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The Pharma Data

JUNE 12, 2025

This stands in contrast to traditional small-molecule inhibitors, which typically aim to block a protein’s activity or binding. Bristol Myers Squibb’s platform includes a range of small-molecule degraders, which exploit the cell’s own disposal mechanisms — typically by sending the target protein to the proteasome for destruction.

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LifeSciVC

JANUARY 16, 2024

Small molecule GLP1s? Whether trial design, execution, or otherwise, drug development even where there is precedent is a challenging road and should not be taken for granted. Additional trials (e.g.,

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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.

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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.

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New Drug Approvals

JUNE 20, 2025

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

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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. […].

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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. Only in this way can AI reach its full potential in accelerating drug discovery and improving trial success rates. Highlighting data integration.

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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. References England Rare Diseases Action Plan 2022.

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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. 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.

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New Drug Approvals

JULY 6, 2025

It is a small molecule delivered orally. [1] mmol) was added tert-butyl piperidin-4-ylcarbamate (841 mg, 98% Wt, 1.2 mmol) and DIPEA (1.19 mL, 3 Eq, 10.3

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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.

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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.

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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.

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The Pharma Data

NOVEMBER 19, 2020

The Company is also aiming to leverage the highly conserved structure of the SARS-CoV-2 M pro protease as a basis for the design of novel oral small molecules against predicted future variants of SARS-CoV-2 and other related human viruses.

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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. Miller LM, Keune WJ, Castagna D, et al.

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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

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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.

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New Drug Approvals

SEPTEMBER 12, 2024

ZASTAPRAZAN 2133852-18-1 362.5 ZASTAPRAZAN 2133852-18-1 362.5 Zastaprazan can be used for the research of gastrointestinal inflammatory diseases or gastric acid-related diseases.

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Drug Target Review

SEPTEMBER 25, 2024

Applying our technology has enabled us to develop a new small molecule, 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 small molecule binds to the bromodomain of CBP/p300 in a highly specific manner.

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LifeSciVC

APRIL 24, 2024

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)?

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