Drug Target Review

OCTOBER 11, 2023

Most of these conditions are genetic in origin and the majority have no effective treatment. Symptoms were reversed in mouse models and a clinical trial is planned for later this year. That includes countless rare peripheral diseases, including many for which there are currently no treatments.

Disease 113

Disease Treatment Small Molecule Therapies 113

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. 6] The U.S.

Small Molecule 62

Small Molecule Clinical Trials Licensing Licensing 62

Drug Target Review

JUNE 11, 2025

Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments. Highlighting data integration.

Drugs 65

Drugs Clinical Trials Drug Development Pharmaceuticals 65

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

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

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. Additionally, treatment led to improvements in height-adjusted total kidney volume (htTKV) , an established clinical marker that correlates with disease progression in ADPKD.

RNA 52

RNA Clinical Trials Disease Small Molecule 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. Similarly, PROTACs can target and degrade overexpressed proteins, offering a way to overcome drug resistance, a common issue in cancer treatment.

Small Molecule 98

Small Molecule Drugs Clinical Trials Therapies 98

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. This is especially relevant with today’s heavier focus on enhancing personalised medicine via broader emerging scientific findings.

Drug Development 65

Drug Development Drugs Animal Testing Engineering 65

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

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.

Small Molecule 52

Small Molecule Clinical Trials Trials Pharmacokinetics 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

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

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

Drug Target Review

NOVEMBER 6, 2023

Advances in research and development and the utilisation of combination treatment strategies are revolutionising the haematology care landscape, but more must be done to address patients’ individual needs. 2 It is this complexity that necessitates powerful, targeted combination therapies.

Small Molecule 111

Small Molecule Therapies Treatment Disease 111

PPD

JUNE 27, 2024

In recent years, AA has followed in the footsteps of other dermatology indications — including psoriasis and atopic dermatitis — with growing research and clinical studies focusing on small molecules with broad cellular effects (JAK and PDE4 inhibitors) and cytokine-specific molecules antagonists (IL-23, Th2 and IL17) to treat the condition.

Treatment 52

Treatment Clinical Trials Clinical Research Small Molecule 52

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

Olympian Clinical Research

MAY 6, 2024

The journey toward effective treatments has been long and evolving, with recent breakthroughs offering new hope to those living with this challenging condition. From Coal Tar to Biologics: A Historical Perspective The history of psoriasis treatment is as old as the condition itself, with records dating back to ancient times.

Treatment 52

Treatment Small Molecule Clinical Research Immune Response 52

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

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). Roche’s Chief Medical Officer and Head of Global Product Development. “In

Clinical Trials 52

Clinical Trials Trials Disease FDA Approval 52

Drug Target Review

AUGUST 25, 2023

What makes RRx-001 a first-in-class hypoxia-activated therapeutic, and how does its dual mechanism of action differ from traditional cancer treatments? Most, if not all, traditional cancer treatments are single minded. AdAPT-001 is an adenovirus, agent of the common cold, currently in a Phase 2 clinical trial.

Small Molecule 98

Small Molecule Virus International Clinical Trials 98

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.,

Small Molecule 40

Small Molecule Licensing Licensing Treatment 40

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.

Clinical Trials 52

Clinical Trials Trials FDA Therapies 52

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

The Pharma Data

NOVEMBER 19, 2020

Structure-based design approach has identified potent compounds for further development as oral treatments for SARS-CoV-2 infection and related human coronaviruses. Program focused on inhibitors of SARS-CoV-2 main protease, a highly conserved protein essential for viral replication.

Small Molecule 40

Small Molecule Structure Based Drug Design (SBDD) Molecular Biology International 40

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. Frequent use of these drugs may lead to weight gain and may induce edema and anemia. (E)

Small Molecule 62

Small Molecule Treatment Regulations Drugs 62

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 2] Medical uses Suzetrigine is indicated for the treatment of moderate to severe acute pain in adults. [1] The FDA has long supported development of non-opioid pain treatment. Suzetrigine is taken by mouth. [1]

FDA 62

FDA Therapies Trials Treatment 62

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.

Small Molecule 60

Small Molecule Licensing Licensing Science 60

The Pharma Data

NOVEMBER 25, 2020

The approval of remdesivir ( Veklury ) for the treatment of patients hospitalized with COVID-19 met this legal and scientific standard. Background Randomized, controlled trials are the gold standard for evaluating the safety and effectiveness of drugs. Researchers evaluated the clinical status of subjects on Day 11.

FDA 52

FDA Treatment Clinical Trials Hospitals 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. . SUZHOU, China , Oct. SUZHOU, China , Oct.

Clinical Trials 40

Clinical Trials Trials Small Molecule Pharmaceutical Companies 40

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.

Trials 52

Trials Small Molecule Clinical Trials Treatment 52

Drug Target Review

JUNE 12, 2023

With 13 preclinical candidates and three AI-designed drugs currently undergoing clinical trials, Insilico is spearheading a revolution in cancer treatment and beyond. Can you provide a summary of the key findings and implications of the preclinical studies on ISM6331 for the treatment of advanced solid tumours?

Metabolic Stability 92

Metabolic Stability Clinical Trials Small Molecule Pharmacokinetics 92

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

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.

Small Molecule 52

Small Molecule Disease Therapies Drugs 52

KIF1A

JULY 22, 2023

If these mutations change the structure of mutant KIF1A to help it perform better, this begs the question: Can we identify small molecules that fix the structure of mutant R9Q/R11Q KIF1A? The authors found that fisetin treatment promoted synaptic and axonal growth, and partially rescued the R9Q worms’ symptoms!

Small Molecule 98

Small Molecule FDA Disease Treatment 98

The Pharma Data

JUNE 24, 2025

We look forward to this strategic alliance continuing to expand the frontiers of cutting-edge science and translating groundbreaking discoveries into transformative treatments for patients.” The company currently operates two global research and development (R&D) centers and four world-class production facilities across China.

Pharma R&D 40

Pharma R&D Pharmaceutical Companies Pharmaceuticals Science 40

The Pharma Data

DECEMBER 5, 2020

Syros will also present new translational data demonstrating that most RARA-positive newly diagnosed unfit AML patients have a monocytic disease phenotype that is highly correlated with resistance to upfront treatment with venetoclax and azacitidine. Syros expects to initiate the Phase 2 trial in the second half of 2021.

Clinical Trials 52

Clinical Trials Trials Small Molecule Treatment 52

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