Drug Target Review

APRIL 7, 2025

Involved in various physiological processes, such as vision, taste, smell, immune response and neurotransmission, GPCRs are activated by various molecules including hormones, neurotransmitters and environmental stimuli, which trigger a cascade of cellular events that help regulate bodily functions.

Fragment Screening

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. Nature Medicine, 28, 1656–1661 (2022) [link]

Drugs

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. They regulate lipid metabolism subsequently enhancing the response of these tissues to the actions of insulin.

Small Molecule

Drug Target Review

NOVEMBER 7, 2024

Agonist antibodies of immune checkpoint regulators These represent a groundbreaking class of immunotherapeutic agents that mimic the natural function of endogenous ligands by binding to specific cell-surface receptors. This approach augments the strength of achieving immune homeostasis.

Disease

Codon

JULY 28, 2025

The first of these components is the therapeutic genetic payload, or cargo, which is designed to correct or regulate the disease-causing mutation, whether that means offering a healthy copy of a mutated gene or delivering precision-editing tools like CRISPR. Equally vital is the delivery vector, which carries this payload to specific cells.

Immune Response

LifeSciVC

JULY 31, 2025

The CBM signalosome is made up of three key proteins (CARD11, BCL10 and MALT1) and regulates key pro-survival and proliferation pathways, including NFkB, JNK, mTORC1 and MYC. And importantly for patients, the small molecule modality would allow this combination to be orally administered, offering convenience for patients.

Small Molecule

Drug Patent Watch

JANUARY 13, 2025

As we delve into the intricate world of biosimilar market dynamics, we’ll explore how these complex molecules are reshaping treatment paradigms across diverse patient populations. Let’s explore how biosimilars are reshaping treatment paradigms across different therapeutic areas. from 2020 to 2025[1].

Biosimilars

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

government could do until the 1938 Federal Food, Drug, and Cosmetic Act gave authority to the FDA to regulate drugs. The molecule is little discussed outside of niche online bodybuilding communities , where some tout the substance as a quick way to lose body fat. Still, there wasn’t much the U.S. Today, few remember DNP.

Drugs

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

ASPET

AUGUST 5, 2024

The ability of small molecule lipophilic ligands to function as exogenous chaperones was examined using receptor-selective antagonists. Long term treatment leads to increases in receptor abundance and activity with no changes in mRNA supporting a role as pharmacological chaperones.

Small Molecule

The Pharma Data

NOVEMBER 17, 2020

Lead Pharma has entered into a collaboration and license agreement with Roche , hoping to lead the way in the development of oral small molecules for immune-mediated diseases like rheumatoid arthritis, psoriasis and inflammatory bowel diseases. . Our ambition is to develop life-changing treatments for patients.

Small Molecule

Metabolite Tales Blog

APRIL 4, 2023

Metabolism of 2022 FDA approved small molecule drugs part 2 Mixing it Up By Julia Shanu-Wilson In Part 1 of this topic we looked at metabolism of the small molecule drugs approved by the FDA in 2022 that were mediated by CYP3A4. Dermavant’s tapinarof is one such friend. 8 This is not the only point of interest.

Small Molecule

Drug Target Review

SEPTEMBER 25, 2024

BRAF and downstream mitogen-activated extracellular signal-regulated kinase (MEK) inhibitors are combined to treat BRAF-mutated melanoma, for example. Classically, rational drug combinations are designed to target two nodes in the same oncogenic signalling pathway.

Small Molecule

Drug Target Review

OCTOBER 6, 2023

This master regulator of the regulatory T cell (Treg) genome has opened new horizons in the fight against cancer, offering a precise and tailored solution to modulate the immune system’s response. Can you explain the role of steroid receptor coactivator-3 (SRC-3) in Tregs and how you are leveraging this discovery for 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

ASPET

DECEMBER 21, 2023

Further study revealed that DR3penA may mitigate pulmonary fibrosis by negatively regulating the PI3K/AKT pathway and MAPK pathway. The peptide DR3penA can be further developed for the treatment of multiple fibrotic lung diseases.

Biochemical Assays

Drug Target Review

JULY 13, 2023

Drug discovery is a complex and vital field that continually seeks to identify new therapeutic targets and develop effective treatments. They found that although the targets themselves may not be easily druggable due to their disordered nature, their ability to condense can be modulated using small molecules.

Disease

The Pharma Data

JANUARY 19, 2021

As part of Aptorum Group’s Acticule infectious disease platform, ALS-4 is a novel orally administered first-in-class small molecule that was developed based on an anti-virulence approach targeting Staphylococcus aureus including MRSA. About ALS-4.

Small Molecule

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. Pending Health Canada’s approval, the Phase 1 trial is designed to test the safety, tolerability and pharmacokinetics of ALS-4 in healthy volunteers. About ALS-4.

Small Molecule

FDA Law Blog: Biosimilars

JULY 11, 2023

But, as is inevitable, there were some disputes about whether certain products should have transitioned, including several that resulted in litigation against FDA for continuing to regulate given products as drugs rather than biologics. Thus, the threat of competition is enough to confer standing.

FDA

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

NOVEMBER 25, 2020

The approval of remdesivir ( Veklury ) for the treatment of patients hospitalized with COVID-19 met this legal and scientific standard. The odds of improvement with the 10-day treatment group when compared to those receiving only standard of care were numerically favorable, but not statistically significantly different. Source: FDA.

FDA

The Premier Consulting Blog

NOVEMBER 20, 2024

In the case of urgent or immediate public interest, process validation may be conducted concurrently with manufacturing the commercial small molecule or biologic product to expedite product availability for patients.

Small Molecule

KIF1A

JULY 1, 2023

This activity may be regulated by the K-loop, which is known to be important for KIF1A’s long-range movement. But we may find that we have a wealth of potential small molecule solutions for newly recognized disorders, just waiting to be rediscovered, and repositioned. Check out this #ScienceSaturday post to learn more.

Small Molecule

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. (Chipscreen Biosciences, Stock Symbol: 688321.SH

Clinical Trials

The Pharma Data

DECEMBER 15, 2020

FDA Approves Klisyri (tirbanibulin) for the Treatment of Actinic Keratosis on the Face or Scalp. NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, today announced that the U.S. BUFFALO, N.Y.,

FDA Approval

Drug Target Review

MAY 23, 2023

Therapies developed in recent decades have transformed the treatment of IBD, making hospitalisation and surgery less common. However, many patients respond poorly to corticosteroid treatment, or their immune system responds unfavourably to biological therapies, such as the development of autoimmune diseases.

Drugs

The Pharma Data

OCTOBER 25, 2020

Vatiquinone, developed from PTC’s Bio-e platform, is an investigational oral small molecule that inhibits 15-Lipoxygenase, a key enzyme that regulates oxidative stress and inflammation response pathways underpinning many neurological disease pathologies, including epilepsy. Peltz , Ph.D.,

Clinical Trials

The Pharma Data

JANUARY 26, 2021

Food and Drug Administration’s Office of Orphan Products Development has granted Orphan Drug Designation to Uttroside-B , a small molecule chemotherapeutic for the treatment of hepatocellular carcinoma (HCC), the most common form of liver cancer. (OTCQB: QBIO), announced today that the U.S. .

Treatment

Drug Target Review

AUGUST 10, 2023

Metastasis is the most lethal attribute of cancer cells and clinical decisions regarding treatment are based largely upon the likelihood of developing metastases. 1 The research, supported by the National Institutes of Health, centres on a cellular chaperone protein called GRP78, responsible for regulating protein folding within cells.

Cell Biology

New Drug Approvals

DECEMBER 16, 2023

1 Factor B is a positive regulator of the alternative complement pathway, where it activates C3 convertase and subsequently C5 convertase. 2 On December 6th, 2023, Iptacopan under the brand name Fabhalta was approved by the FDA for the treatment of adults with PNH. 1] It is taken by mouth. [1] Retrieved 6 September 2023. April 2019).

FDA

KIF1A

MAY 27, 2023

Lessard created and advanced KIF1A.ORG’s Therapeutic Acceleration Program, facilitating the creation of patient-derived cell lines, mouse models, and drug screening platforms that are the bedrock of our search for treatments. Read more to learn about the factors involved in incentivizing investment in rare disease treatments.

Science

The Pharma Data

OCTOBER 14, 2020

About VBL’s VB-600 Platform VBL is conducting two parallel drug development programs that are exploring the potential of MOSPD2 (motile sperm domain-containing protein 2), a protein that VBL has identified as a key regulator of cell motility, as a therapeutic target for inflammatory diseases and cancer.

Treatment

LifeSciVC

DECEMBER 6, 2022

That can be a good thing: for example, when determining inherited traits and regulating critical cell functions. IRF5 is a genetically validated Transcription Factor that is important in a variety of diseases and has always been considered undruggable by small molecules because it doesn’t have an active site.

Small Molecule

The Pharma Data

OCTOBER 27, 2021

Small molecule candidate Anle138b targets disease modification for multiple system atrophy and other neurological disorders. With the introduction of small-molecule medication, we open a new chapter in the fight against neurodegenerative diseases and have the chance to improve the lives of millions of patients drastically.”.

Licensing