Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.

Clinical Development

Pharma Manufacturing

JULY 23, 2025

The company saw positive momentum in mammalian, bioconjugates, and small molecules, offsetting lower sales from its cell and gene therapy and microbial businesses.

Small Molecule

Drug Target Review

JULY 3, 2025

New approach methodologies data In April, the US Food and Drug Administration (FDA) announced a plan to replace animal testing in the development of monoclonal antibodies and other therapies with validated “human-relevant” methods, including AI-based computational models evaluating toxicity, cellular lines and organoid toxicity.

Drug Development

PPD

NOVEMBER 11, 2024

As a result, biopharma and biotech companies working to bring their drug pipelines to the market require deep expertise from trusted partners to help deliver critical therapies for their patients. According to the United Nation’s 2024 World Population Prospects data, the global population of those 65 years old and over will grow from 0.8

Drug Development

The Pharma Data

JUNE 25, 2025

Novartis Finalizes Acquisition of Regulus Therapeutics, Strengthening Its Renal Disease Portfolio with Promising ADPKD Therapy Novartis AG, a leading global pharmaceutical company, has officially completed its acquisition of Regulus Therapeutics Inc. , a biotechnology firm known for its expertise in microRNA-targeting therapies.

RNA

Drug Target Review

DECEMBER 4, 2024

Confo Therapeutics , led by CEO Dr Cedric Ververken, is at the forefront of developing innovative GPCR-targeted therapies using its proprietary ConfoBody ® platform. In addition to generating agonistic antibodies, Confo is also successfully applying the ConfoBody/ConfoChimer technology to drugging GPCR with small molecules.

Small Molecule

Drug Target Review

JUNE 2, 2025

Bioconjugates are rapidly emerging as a key frontier in targeted therapies, particularly through Antibody-Drug Conjugates (ADCs) , which combine antibodies with cytotoxic drugs to deliver more precise and effective treatments. Very often, you have a payload that is extremely toxic, and then you require specially trained people to handle it.

Small Molecule

Alta Sciences

JULY 3, 2025

Because this transformation can significantly improve the stability, solubility, and overall performance of pharmaceutical compounds, especially when it comes to small molecules. Why does that matter? Beyond the science, what’s exciting is how this technology opens doors. The response? Ready to advance your next project?

Small Molecule

Drug Target Review

JULY 10, 2025

Before a compound can become a viable therapy, it must pass a series of tests – not just for potency, but for how it behaves in the body. Absorption, distribution, metabolism, excretion and toxicity – collectively known as ADMET – are among the most critical and challenging hurdles in small molecule drug discovery.

Metabolic Stability

Drug Target Review

JULY 18, 2025

Tackling resistance from a different angle: PAR2 While CCR8 is drawing attention, Domain is also exploring less conventional GPCRs like PAR2 – long known in inflammation and pain, but only recently linked to cancer therapy resistance.

Small Molecule

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.

Drugs

BioPharma Drive: Drug Pricing

JULY 8, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. Drugmakers are working to coopt this process with targeted therapies. You can unsubscribe at anytime.

Targeted Protein Degradation

The Pharma Data

JUNE 12, 2025

This small molecule inhibits the interaction between menin and its binding partners, thereby affecting the expression of genes that promote leukemia progression. Importantly, for newly diagnosed, intensive chemotherapy-ineligible patients, the ORR was even higher at 90% and the composite complete response rate was 75%.

Treatment

The Pharma Data

JUNE 12, 2025

This stands in contrast to traditional small-molecule inhibitors, which typically aim to block a protein’s activity or binding. Bristol Myers Squibb’s platform includes a range of small-molecule degraders, which exploit the cell’s own disposal mechanisms — typically by sending the target protein to the proteasome for destruction.

Targeted Protein Degradation

Drug Target Review

JUNE 16, 2025

Targeted drug delivery and therapeutic development MSI offers significant advantages for those developing small molecule therapeutics. 7 In immune therapies, MSI helps monitor immune-cell distribution and off-target effects, optimising therapeutic strategies and improving patient outcomes. For instance, Cheng et al.

Drugs

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. Trueline Therapeutics also tested the compound in a canine model and did not detect any markers of cardiac toxicity.

Drugs

The Pharma Data

JUNE 9, 2025

The acceptance, which follows the CDE’s grant of Priority Review status in May, represents an accelerated regulatory pathway for what may become the first approved systemic therapy for TGCT in China. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics Co.,

Treatment

The Pharma Data

JUNE 24, 2025

Oncology remains a core priority, reflecting the rising global burden of cancer and the need for more effective, targeted therapies. The goal is to transform laboratory discoveries into clinically relevant therapies that can reach patients faster and more efficiently. Source link

Pharma R&D

LifeSciVC

JULY 8, 2025

One team might be working on a cell therapy, another on RNA, another on small molecules. These organizations rarely follow a single asset path. They run portfolios across multiple programs, modalities, and often geographies. At the same time, external collaborators: CROs, CDMOs, academic groups, all operate on varied timelines.

Hospitals

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 The application received Breakthrough Therapy , Fast Track and Priority Review designations by the FDA. 11] The FDA granted the application for suzetrigine priority review , fast track , and breakthrough therapy designations. [2]

FDA

Sygnature Discovery

APRIL 7, 2025

Small molecule solutions Our strategy to deliver therapeutically relevant ADCs is based on our expertise in four key areas: Holistic development of novel antibody drug conjugates Our comprehensive antibody drug conjugate framework integrates in vitro and in vivo profiling to optimize potency, efficacy, and safety. Big ADC problem?

Small Molecule

New Drug Approvals

JUNE 25, 2025

While this therapy can decrease blood glucose level, it has limited efficacy and tolerability, causes weight gain and often induces hypoglycemia. (B) 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.

Small Molecule

New Drug Approvals

MAY 26, 2025

Ervogastat CAS 2186700-33-2 Non-alcoholic Steatohepatitis (NASH) with Liver Fibrosis (FAST TRACK – U.S.) 2-[5-[(3-Ethoxy-2-pyridinyl)oxy]-3-pyridinyl]- N -[(3 S )-tetrahydro-3-furanyl]-5-pyrimidinecarboxamide ( S )-2-(5-((3-Ethoxypyridin-2-yl]oxy]pyridin-3-yl)- N -(tetrahydrofuran-3-yl)pyrimidine-5-carboxamide PF 06865571 BSOIY5AKQW 407.4

Small Molecule

Drug Target Review

NOVEMBER 7, 2024

In these conditions, the goal of therapy is typically to suppress or mitigate immune activity. Understanding the intricate role of PSGL-1 in immune-cell recruitment and activation is essential for developing targeted therapies aimed at modulating immune responses in various inflammatory and autoimmune disorders.

Disease

Alta Sciences

APRIL 24, 2025

Case Study: How Altasciences Overcame Pharmacological Challenges in a GLP-1 IND-Enabling Study pmjackson Thu, 04/24/2025 - 21:43 For emerging therapies with pronounced pharmacological effects, thoughtful study design is critical to ensuring reliable and interpretable data. Download a copy of this GLP-1 RA case study!

Small Molecule

New Drug Approvals

JULY 7, 2025

Food and Drug Administration (FDA) and the Center for Drug Evaluation (CDE) of NMPA granted pimicotinib breakthrough therapy designation (BTD) for the treatment of tenosynovial giant cell tumor patients that are not amenable to surgery in January 2023 and July 2022, respectively. [7] 6] The U.S. 15 : 53–66. doi : 10.2147/OTT.S345878.

Small Molecule

New Drug Approvals

APRIL 18, 2025

2] The US Food and Drug Administration (FDA) granted the application for crinecerfont fast track , breakthrough therapy , orphan drug , and priority review designations. [2] New Drug Therapy Approvals 2024 (PDF). 2] In the first trial, 122 adults received crinecerfont twice daily and 60 received placebo twice daily for 24 weeks. [2]

FDA

Drug Patent Watch

JANUARY 13, 2025

Oncology: Expanding Access to Life-Saving Treatments In the field of oncology, biosimilars have made significant inroads, particularly in supportive care and targeted therapies. We’re now able to offer biologic therapies to patients earlier in their disease course, potentially altering the long-term trajectory of their condition.

Biosimilars

New Drug Approvals

JULY 6, 2025

It is a small molecule delivered orally. [1] “Prospect of acromegaly therapy: molecular mechanism of clinical drugs octreotide and paltusotine” Nature Communications. mmol) was added tert-butyl piperidin-4-ylcarbamate (841 mg, 98% Wt, 1.2 mmol) and DIPEA (1.19 mL, 3 Eq, 10.3 14 (1): 962. Bibcode : 2023NatCo.14.962Z.

Small Molecule

New Drug Approvals

JUNE 9, 2025

HEXASODIUM PHYTATE cas 34367-89-0 myo -Inositol, 1,2,3,4,5,6-hexakis(dihydrogen phosphate) sodium salt (1:6) hexasodium;[2,3,4,5,6-pentakis[[hydroxy(oxido)phosphoryl]oxy]cyclohexyl] hydrogen phosphate free form RN: 83-86-3 C 6 H 12 Na 6 O 24 P 6 , 791.93

Clinical Trials

PPD

APRIL 14, 2025

This expansion is creating opportunities for clinical trials related to a range of new therapy areas and their subpopulations. Thermo Fisher delivers everything needed for biologics, small molecule and viral vector manufacturing, all the way through validation and regulatory support.

Clinical Trials

BioPharma Drive: Drug Pricing

JULY 14, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. It’ll also boost investments in U.S. drug production.

Production

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

BioPharma Drive: Drug Pricing

AUGUST 28, 2024

Under a collaboration with NextRNA Therapeutics, Bayer will access the biotech’s platform to target long, non-coding RNA interactions with small molecule drugs.

RNA

Covalent Modifiers

FEBRUARY 15, 2024

Herein, we use afatinib as a lead to undertake a structure-based drug design approach, aided by mass-spectrometry and x-ray crystallography, to develop DHC-156, a small molecule that more selectively binds brachyury and downmodulates it as potently as afatinib.

Small Molecule

Science Daily: Pharmacology News

NOVEMBER 30, 2023

Researchers have succeeded in restoring lost brain function in mouse models of stroke using small molecules that in the future could potentially be developed into a stroke recovery therapy.

Small Molecule

Covalent Modifiers

FEBRUARY 6, 2024

The most common adverse effects are hyperphosphatemia caused by FGFR1 inhibition and diarrhea due to FGFR4 inhibition, as current therapies are not selective among the FGFRs.

Small Molecule