This site uses cookies to improve your experience. To help us insure we adhere to various privacy regulations, please select your country/region of residence. If you do not select a country, we will assume you are from the United States. Select your Cookie Settings or view our Privacy Policy and Terms of Use.
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Used for the proper function of the website
Used for monitoring website traffic and interactions
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Strictly Necessary: Used for the proper function of the website
Performance/Analytics: Used for monitoring website traffic and interactions
Smallmolecule drugs make up most of the drugs we take conveniently as pills, including painkillers like ibuprofen (Advil), antibiotics like penicillin and amoxicillin, or cholesterol-lowering drugs like atorvastatin (Lipitor). The smallmolecules drugs of today look nothing like the molecules of the 1970s.
We asked the global drug discovery community to nominate and vote on their favorite molecule from 2022, and the results are in. The 2022 winner, with the most overall votes across the ten finalist molecules , is BMS’ oral, deuterated allosteric TYK2 inhibitor, deucravacitinib, the first new treatment for plaque psoriasis in nearly a decade.
Experimental drug NU-9 -- a smallmolecule 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.
Clinical advancement of LRRK2 inhibitors was initially stalled by concerns about on-target lung findings in primates, but these were ameliorated by a Merck/Genentech/Pfizer/MJFF study showing that these lung changes were reversible, and Biogen/Denali has currently a smallmolecule (BIIB122/DNL151) in Ph. I ( NCT04557800 ).
It is a smallmolecule with a dual mechanism of action, acting as both a complete estrogen receptor antagonist and a selective estrogen receptor degrader (SERD). This means it can block estrogen receptor activity and also degrade the receptor itself, potentially offering a more effective treatment approach. ribociclib).
However, getting essential treatments to patients quickly and safely requires more than just technological innovation. A global network with local expertise To ensure the delivery of treatments to patients worldwide, biotech and biopharma companies also need partners that can provide comprehensive solutions from a geographic perspective.
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 smallmolecules aimed at revolutionising cancer treatment. Additionally, we are preparing to launch several other trials over the coming months.
These therapies have broadened treatment options for patients to expand beyond the more traditional smallmolecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options. 3D rendering of Antibody Drug Conjugate Molecules.
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.
A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome. Susceptibility or risk biomarkers can detect the likelihood of a patient developing a disease or medical condition, which is crucial for treatments that are most effective before the onset of symptoms.
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.
Together, these data reflect a significant step forward in the treatment of hematologic malignancies — a group of aggressive, complex disorders for which many patients still face limited treatment options and poor outcomes — and demonstrate the potential for these novel mechanisms of action to make a profound difference in patient care.
Table 1: Smallmolecule 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.
This article compiles recent high-profile clinical readouts and related news with smallmolecules 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
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. This is an AI generated image.
A New Treatment Option for a Debilitating Joint Tumor TGCT is a rare, debilitating condition that primarily affects young and middle-aged adults, often striking individuals in their prime working years. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational smallmolecule developed by Abbisko Therapeutics Co.,
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.
By Allessandra DiCorato October 4, 2023 Credit: AbbVie The new smallmolecule 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.
These multifunctional smallmolecules 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.
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.
The data were revealed during an oral presentation (S137) at the 2025 European Hematology Association (EHA) Congress in Vienna, reflecting ongoing progress in developing new treatment options for patients battling this aggressive hematologic malignancy.
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.
Molecular-level biochemical assays like transcriptomics, genomics and proteomics have emerged as valuable tools for identifying potential targets in cancer treatment through deep cyclic inhibition (DCI). How does the DCI mechanism compare to the design of other drugs for cancer treatment?
Biological products have unlocked the potential for the management of several diseases such as cancers and autoimmune diseases for which treatment with smallmolecule, chemically synthesized drug molecules remain suboptimal. […].
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.
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 smallmolecule adopting an anti-virulence (non-antibiotic) approach to address the growing unmet medical needs of infections caused by Staphylococcus aureus.
4 Another related and well described non-oncogene resistance mechanism is the histological transformation of EGFR-mutated non-small cell lung cancer (NSCLC) to small-cell lung cancer upon treatment with an EGFR inhibitor. This orally available smallmolecule binds to the bromodomain of CBP/p300 in a highly specific manner.
It is a smallmolecule 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
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.
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 smallmolecule inhibitor targeting EGFR exon 20 insertion mutations and HER2 activating mutations to undergo clinical testing. Tags: Cancer Program Cancer
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.
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.
Orelabrutinib is a Phase 2 oral smallmolecule Bruton’s tyrosine kinase inhibitor with high selectivity and the ability to cross the blood-brain barrier InnoCare to receive a $125 million upfront payment and is eligible to receive potential development and commercial milestone payments. Biogen Inc.
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 smallmolecules with broad cellular effects (JAK and PDE4 inhibitors) and cytokine-specific molecules antagonists (IL-23, Th2 and IL17) to treat the condition.
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. President and CEO of Knopp. “We study centers.
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.
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.
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]
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.
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.
Consequently, a GK activator may provide therapeutic treatment for NIDDM and associated complications, inter alia, hyperglycemia, dyslipidemia, insulin resistance syndrome, hyperinsulinemia, hypertension, and obesity. clinical candidate currently in Phase 2 development. For example, U.S. Patent publication No.
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.
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.
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.
We organize all of the trending information in your field so you don't have to. Join 15,000+ users and stay up to date on the latest articles your peers are reading.
You know about us, now we want to get to know you!
Let's personalize your content
Let's get even more personalized
We recognize your account from another site in our network, please click 'Send Email' below to continue with verifying your account and setting a password.
Let's personalize your content