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Disorders such as Alzheimer’s disease , primary glioblastoma and amyotrophic lateral sclerosis (ALS) all affect the CNS and are considered fatal, while more common conditions, including depression , strokes and epilepsy , require long-term treatments. This makes it difficult to identify relevant and effective treatments.
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).
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.
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.
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.
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 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.
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.
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.
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.
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.,
Immuno-oncology has transformed cancer treatment, but for many patients, tumours remain resistant to even the most advanced immune checkpoint inhibitors. This opens the door for a disease-modifying approach both as a monotherapy and in combination, expanding treatment options and offering new hope to millions of patients.”
Specially designed smallmolecule drugs can bring together target proteins with an enzyme that tags them with ubiquitin, thereby marking them for destruction. In their 2001 paper, Crews, Deshaies and others described how synthetic molecules could attach ubiquitins to unwanted cell parts. "It You can unsubscribe at anytime.
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.
This smallmolecule reactivates the apoptosis cascade in tumor cells while sparing healthy cells in animal models. Apoptosis, or programmed cell death, is a natural end process for all cells. This rapid clearance minimizes the drug’s potential impact on healthy cells.
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.
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]
g/mol O5ZD2TU2B7 FDA 7/3/2025, Ekterly, To treat acute attacks of hereditary angioedema N -[(3-fluoro-4-methoxypyridin-2-yl)methyl]-3-(methoxymethyl)-1-[[4-[(2-oxopyridin-1-yl)methyl]phenyl]methyl]pyrazole-4-carboxamide Sebetralstat , sold under the brand name Ekterly , is a medication used for the treatment of hereditary angioedema. [1]
CAR-T therapy (perhaps the most promising cancer treatment of the last several decades) is one example, in which scientists extract a patient’s blood cells, modify T cells so they can recognize and attack malignancies more effectively, and reinfuse them back into the body.
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.
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.
“Discovery of Ervogastat (PF-06865571): A Potent and Selective Inhibitor of Diacylglycerol Acyltransferase 2 for the Treatment of Non-alcoholic Steatohepatitis” Journal of Medicinal Chemistry. Ervogastat CAS 2186700-33-2 Non-alcoholic Steatohepatitis (NASH) with Liver Fibrosis (FAST TRACK – U.S.) 24 November 2022).
Formula C 27 H 29 Cl 2 FN 2 OS Crinecerfont , sold under the brand name Crenessity , is a medication used for the treatment of congenital adrenal hyperplasia. [1] 2] In the first trial, 122 adults received crinecerfont twice daily and 60 received placebo twice daily for 24 weeks. [2] 321839-75-2 Molecular Weight 519.50 1 December 2024.
1] [2] [3] KIN-3248 is a smallmolecule that targets and inhibits oncogenic fibroblast growth factor receptors (FGFRs). Discovery of KIN-3248, An Irreversible, Next Generation FGFR Inhibitor for the Treatment of Advanced Tumors Harboring FGFR2 and/or FGFR3 Gene Alterations. Tyhonas JS, et al. J Med Chem. Content Brief] [2].
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
Why it’s used: Hexasodium phytate is being investigated as a treatment for calciphylaxis, a serious complication of end-stage renal disease characterized by skin and tissue damage due to calcification of small blood vessels. Phytic acid is a major phosphorus storage compound of most seeds and cereal grains.
Initially approved for treatment of Type 2 diabetes and later for obesity studies in GLP-1 are now expanding in other indications such as obstructive sleep apnea (OSA), heart failure with preserved ejection fraction (HFpEF) and chronic kidney disease (CKD).
Perkins’ observation drew little attention for a decade or so, until a group of Stanford doctors dug it up and ran a clinical trial to test the effects of DNP on overweight participants. The results of this trial, and its subsequent coverage in the press, kicked off a surge in demand for DNP.
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.
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.
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.
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 ).
Hetrombopag olamine (6), an oral nonpeptide thrombopoietin receptor (TpoR)agonistdevelopedby JiangsuHengruiPharmaceutical, was approved in China in June2021 for treatment of adult patients with chronic primary immune thrombocytopenia (ITP) and severe aplastic anemiawhohave not responded well to other treatments.46Hetrombopag,
ALPHA.S)- Dorzagliatin(18)was developed by Hua Medicine as a treatment for diabetic kidney disease(DKD), type1diabetes mellitus(T1DM), and type2 diabetes mellitus (T2DM). 2] Dorzagliatin is under investigation in clinical trial NCT03173391 (Long-term Efficacy and Safety of HMS5552 in T2DM). .-(2-METHYLPROPYL)-2-OXO-, (.ALPHA.S)-
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.
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
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.
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.
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