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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SCIENMAG: Medicine & Health

JUNE 27, 2023

Credit: Insilico Medicine […]

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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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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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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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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. This suited my personality and constant curiosity more than academic research.

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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

This milestone supports the transition of Aptorum Group to a clinical-stage company and reflects the potential of our scientific rigor and novel approach of our products. We are dedicated to delivering novel therapeutics in the field of growing unmet medical needs of infections starting with Staphylococcus aureus.”. About ALS-4.

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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. J Med Chem.

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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.

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The Pharma Data

DECEMBER 20, 2020

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. The secondary objective is to assess the pharmacokinetic profile of SAD and MAD of ALS-4 administered orally to healthy subjects. About ALS-4.

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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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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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Drug Target Review

OCTOBER 11, 2023

Symptoms were reversed in mouse models and a clinical trial is planned for later this year. Autophagy boosters, in contrast, are conventional small molecule drugs. However, it’s a very new technology and there are various technical challenges, including issues with delivery, that still need to be overcome.

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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.,

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Advarra

FEBRUARY 24, 2023

One area receiving increased focus from the Food and Drug Administration (FDA) are trial designs incorporating multiple endpoints to support efficacy. Developing the endpoint design and overall approach of a clinical trial program is mostly science, but there is a bit of art to it as well.

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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. small molecules continue to be enthusiastically funded even at the discovery stage.

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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).

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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. Vlad Coric , M.D.,

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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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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.

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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.

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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.

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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.

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Drug Target Review

AUGUST 25, 2023

However, in tissues where the 3 Hs are not present, the small molecule has anti-toxicity activity through 1) direct inhibition of the NLRP3 inflammasome, an inflammation starter and perpetuator, and 2) upregulation of the master antioxidant transcription factor, Nrf2.

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Drug Target Review

NOVEMBER 6, 2023

These regimens frequently include one or two small molecule inhibitors and immuno-oncology (IO) therapies, such as monoclonal antibodies and T-cell engagers. To bring us closer to curing cancer, a combination of effective drugs with non-overlapping mechanisms of action is required.

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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.

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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. SUZHOU, China , Oct. SUZHOU, China , Oct.

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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.

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Alta Sciences

FEBRUARY 10, 2025

Whether for a small or large molecule, we provide: Pharmacology, toxicology , and PK studies Analytical method development Formulation development and manufacturing Preclinical safety testing Clinical trial design Data reporting and documentation Want to know more before we get started?

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Broad Institute

OCTOBER 4, 2023

Project leaders Robert Manguso and Kathleen Yates, along with first author Hakimeh Ebrahimi Nik, discuss what makes the small molecule inhibitor so exciting. Learn more about the inhibitor. Learn more about TIDE, the project that identified the target.

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The Pharma Data

SEPTEMBER 29, 2020

. Sosei Heptares has received a $5m milestone payment from its strategic alliance partner Pfizer after the first subject in a clinical trial was dosed with a new drug candidate from the collaboration between the two companies.

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Drug Target Review

SEPTEMBER 11, 2024

How does morADC technology combine the properties of small molecules and monoclonal antibodies to enhance anti-aggregation effects for CNS applications? The morADC are able to cross the blood-brain barrier more efficiently and offer higher potency than individual parent molecules.

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The Pharma Data

JANUARY 13, 2021

The magnitude of change in blood AEC was consistent with the eosinophil depletion seen in previous clinical trials of dexpramipexole in other indications. In the EXHALE trial, eosinophil depletion in blood by oral dexpramipexole correlated with clinically important improvement in lung function as measured by pre-bronchodilator FEV1.

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The Pharma Data

DECEMBER 5, 2020

The trial will also evaluate the triplet as a salvage therapy in patients who don’t respond to venetoclax and azacitidine. Syros expects to initiate the Phase 2 trial in the second half of 2021. About Syros Pharmaceuticals Syros is redefining the power of small molecules to control the expression of genes.

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LifeSciVC

JULY 6, 2023

Although these results cannot prove that there is no possibility of finding a high-affinity small molecule binder of NBD1, they are discouraging…” It turns out the small molecules they were looking for are not only possible – they were ready for a dedicated team to push them ahead.

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The Premier Consulting Blog

NOVEMBER 20, 2024

For drugs with FDA Orphan Drug Designation (ODD), Breakthrough Therapy Designation (BTD), and EMA PRIority MEdicines designation (PRIME), all clinical, non-clinical, and CMC requirements are still required for regulatory approval, despite being on pathways for an expedited approval process.

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