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

The Pharma Data

JUNE 12, 2025

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.

Targeted Protein Degradation

The Pharma Data

JUNE 12, 2025

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.

Treatment

The Pharma Data

JUNE 9, 2025

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 small molecule developed by Abbisko Therapeutics Co.,

Treatment

Drug Target Review

JULY 18, 2025

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.”

Medical Chemistry

BioPharma Drive: Drug Pricing

JULY 8, 2025

Specially designed small molecule 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.

Targeted Protein Degradation

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

Broad Institute

OCTOBER 29, 2024

This small molecule 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.

Drugs

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

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

New Drug Approvals

JULY 10, 2025

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]

FDA

Codon

JULY 28, 2025

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.

Immune Response

New Drug Approvals

JUNE 25, 2025

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.

Small Molecule

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

New Drug Approvals

MAY 26, 2025

“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).

Small Molecule

New Drug Approvals

APRIL 18, 2025

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.

FDA

New Drug Approvals

JULY 12, 2025

1] [2] [3] KIN-3248 is a small molecule 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].

Production

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

Small Molecule

New Drug Approvals

JUNE 9, 2025

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.

Clinical Trials

PPD

APRIL 14, 2025

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).

Clinical Trials

Codon

JULY 17, 2025

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.

Drugs

Drug Hunter

JUNE 11, 2023

Small molecule 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 small molecules drugs of today look nothing like the molecules of the 1970s.

Small Molecule

Science Daily: Pharmacology News

APRIL 8, 2025

Experimental drug NU-9 -- a small molecule 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.

Small Molecule

Drug Hunter

APRIL 20, 2023

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.

Small Molecule

Drug Hunter

AUGUST 15, 2023

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 small molecule (BIIB122/DNL151) in Ph. I ( NCT04557800 ).

Small Molecule

New Drug Approvals

JULY 21, 2025

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,

Small Molecule

New Drug Approvals

JULY 28, 2025

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)-

Small Molecule

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule 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.

Small Molecule

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

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.

Small Molecule

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

Small Molecule

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

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

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

Drug Target Review

JULY 12, 2023

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?

Biochemical Assays

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

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.

Small Molecule