Drug Target Review

NOVEMBER 1, 2023

ATX is mainly involved in phospholipidic metabolism and the production of extracellular lysophosphatidic acid (LPA) from lysophosphatidylcholine (LPC). Autotaxin has lysophospholipase D activity leading to tumor cell growth and motility by lysophosphatidic acid production. Cancer Metastasis Rev. 2018 Sep;37(2–3):509–18. 2017;2017:1–15.

Clinical Trials 134

Clinical Trials Trials Small Molecule Disease 134

The Pharma Data

DECEMBER 20, 2020

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 small molecule adopting an anti-virulence (non-antibiotic) approach to address the growing unmet medical needs of infections caused by Staphylococcus aureus.

Small Molecule 40

Small Molecule Clinical Trials Trials Treatment 40

The Pharma Data

NOVEMBER 17, 2020

The study demonstrated favorable proof-of-concept for LYT-100’s tolerability and pharmacokinetic (PK) profile, which will also enable twice-a-day (BID) dosing of LYT-100 in future studies. LYT-100 is PureTech’s most advanced wholly-owned product candidate. Photo: Business Wire). About LYT-100.

Pharmacokinetics 52

Pharmacokinetics Treatment Trials Production 52

New Drug Approvals

OCTOBER 12, 2024

ALZ-801 is a potent and orally available small-molecule β-amyloid (Aβ) anti-oligomer and aggregation inhibitor, valine-conjugated proagent of Tramiprosate with substantially improved PK properties and gastrointestinal tolerability compared with the parent compound. The product slowly crystallized. Clin Pharmacokinet.

Pharmacokinetics 62

Pharmacokinetics Small Molecule Disease Licensing 62

The Premier Consulting Blog

MAY 31, 2023

While the type, number, and design of these studies vary based on product-specific characteristics, IND-enabling packages submitted to the FDA generally include key information about the pharmacology, pharmacokinetics, and toxicology of the product. All these studies need to be performed under GLP.

Packaging 52

Packaging Pharmacokinetics Small Molecule Clinical Trials 52

The Premier Consulting Blog

JULY 31, 2024

Regulatory Pathway 505(b)(2) versus 505(b)(1) In the US, novel new small molecule drug products, including some peptides, are regulated, and approved by the FDA under the Federal Food, Drug, and Cosmetic Act (the “Federal FD&C Act”) under two key regulatory pathways: Section 505(b)(1) and Section 505(b)(2) NDAs.

Clinical Pharmacology 52

Clinical Pharmacology Drug Development Pharmacokinetics Drugs 52

The Premier Consulting Blog

AUGUST 8, 2024

On July 31, 2024, the US Food and Drug Administration (FDA) announced Fiscal Year 2025 (FY2025) Prescription Drug User Fee Amendments of 2022 (PDUFA VII) fee rates for the review of human drug and biological product applications along with prescription drug program fees. patients with renal, hepatic, or cardiovascular concerns).

Drugs 52

Drugs Pharmacokinetics FDA Production 52

Metabolite Tales Blog

FEBRUARY 16, 2024

The paper, written by a consortium of scientists from several big pharma companies, points out that discussions around multi labelling are happening with more frequency due to the increase in larger molecular weight small molecules being developed. Eur J Drug Metab Pharmacokinet 48 , 411–425 (2023). Drug Metab.

Small Molecule 52

Small Molecule Pharma Companies Pharmacokinetics Drugs 52

The Premier Consulting Blog

SEPTEMBER 11, 2024

The 505(b)(2) new drug application (NDA) pathway offers a unique opportunity for small molecule developers to bring innovative products to market more efficiently by leveraging existing data they do not own or have right of reference to. The new formulation resulted in a drug product that was very viscous. Human factors.

Drugs 52

Drugs Clinical Pharmacology Packaging Small Molecule 52

The Premier Consulting Blog

JANUARY 31, 2023

Of note, this guidance is specific to small molecule pharmaceuticals; similar guidance for biologics can be found in the ICH’s S6(R1) Preclinical Safety Evaluation for Biotechnology-Derived Pharmaceuticals. Pharmacokinetic and systemic exposure data is also important to consider.

Small Molecule 52

Small Molecule Pharmaceuticals FDA Production 52

Drug Target Review

JULY 12, 2023

Once a new drug target is identified, a proprietary deep learning artificial intelligence (AI) platform we call Fluency can rapidly identify small molecules that selectively bind to a target of interest. We are pleased that to date our lead product candidate has aligned DCI profiles as expected.

Biochemical Assays 98

Biochemical Assays Treatment Bioinformatics Disease 98

Practical Cheminformatics

JANUARY 8, 2024

It would be far more productive to privately or publicly fund an effort to generate large, relevant datasets and make the data publicly accessible. In addition to optimizing potency against a target of interest, we must design selective compounds with optimal pharmacokinetic profiles and a host of other desirable properties.

Drugs 136

Drugs Small Molecule Bioinformatics Packaging 136

The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Genkyotex SA , a subsidiary of Calliditas Therapeutics AB (publ) (“Calliditas”) (Nasdaq OMX – CALTX; NASDAQ – CALT), today announced positive Phase 1 data demonstrating a favorable safety and pharmacokinetic profile of high-dose setanaxib, Genkyotex’s lead asset. STOCKHOLM , Jan.

Trials 40

Trials Clinical Trials Pharmacokinetics Small Molecule 40

The Pharma Data

DECEMBER 15, 2020

Small Molecule Inhibitors. NMD670 is a first-in-class small molecule inhibitor of the muscle specific chloride ion channel, the ClC-1 ion channel. The secondary outcome involves pharmacokinetic endpoints. Oral, Small Molecules. The primary outcome includes safety and tolerability endpoints.

Disease 52

Disease Clinical Trials Small Molecule Trials 52

The Pharma Data

AUGUST 29, 2020

About the Study NCT03439839 is a Phase II, multicenter, open-label, sequential 2-cohort trial to assess the safety, efficacy, tolerability and pharmacokinetics/pharmacodynamics of LNP023 in PNH patients (cohort 1: n=10) with active hemolysis despite treatment with eculizumab. You should not place undue reliance on these statements.

Treatment 52

Treatment Disease Small Molecule Production 52

The Pharma Data

OCTOBER 18, 2020

Several clinical sites are screening patients for the Phase 1 a/b multicenter, open-label, dose escalation study of safety, pharmacodynamics, and pharmacokinetics of CG-806 in ascending cohorts (3+3 design) to determine the maximum tolerated dose or recommended dose in patients with relapsed or refractory AML.

Clinical Trials 40

Clinical Trials Small Molecule Trials Therapies 40

The Pharma Data

APRIL 11, 2023

A focus of the presentation will be on the target engagement results and pharmacokinetics from its ongoing monotherapy dose-escalation study to explore a potential optimal dose and schedule to effectively inhibit AhR. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition.

Research 40

Research Clinical Trials Small Molecule Trials 40

The Pharma Data

NOVEMBER 11, 2020

Adding a new building, which will house up to 100 biologists by the end of the year, will bring together all the key functions for cutting-edge and high-performance small molecule discovery and up to commercial development. 100 co-owned product opportunities at clinical, pre-clinical and discovery stages (EVT Innovate).

Science 40

Science Small Molecule Pharmacokinetics Disease 40

Alta Sciences

APRIL 17, 2024

Our preclinical and bioanalytical experts have extensive experience in leading-edge technologies to support sponsors in the advancement of their gene therapy products. Gene Therapy resources Watch our on-demand webinar, “ Nonclinical Safety Assessment for Gene Therapy Products ”.

Drug Development 52

Drug Development Treatment FDA Approval Drugs 52

The Pharma Data

JANUARY 10, 2021

10, 2021 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create product candidates that address unmet medical needs, will provide an update on Vision 3×3 and the company’s 2021 key milestones at the 39 th Annual J.P. COPENHAGEN, Denmark, Jan.

Clinical Trials 40

Clinical Trials Clinical Development Small Molecule Trials 40

Agency IQ

JULY 26, 2024

FDA’s new guidance on postapproval manufacturing changes for biosimilars focuses on current practice, new dosage forms Meeting a biosimilar user fee commitment, the FDA is expanding on its recommendations for biosimilar and interchangeable product applicants asking the FDA for post-approval manufacturing changes. switching studies).

Biosimilars 40

Biosimilars Science FDA Production 40

Agency IQ

FEBRUARY 23, 2024

BY CHELSEY MCINTYRE, PHARMD , KARI OAKES | FEB 21, 2024 7:48 PM CST Background: Dose-finding in oncology Traditionally, early development of oncology products has focused on identifying the maximum tolerated dose (MTD) of an investigational product. Here, AgencyIQ offers some highlights from the panel discussions.

Science 40

Science FDA Trials Clinical Pharmacology 40

PPD

APRIL 14, 2025

Were also uniquely able to deliver a full suite of end-to-end offerings that include: Thermo Fisher Scientific manufacturing for production continuity from clinical trials to commercial scale.

Clinical Trials 52

Clinical Trials Trials Clinical Development Disease 52

Agency IQ

JUNE 9, 2023

Before a drug or biological product can be moved into clinical studies, sponsors must conduct nonclinical studies to demonstrate the product is reasonably safe and that its pharmacological effects and mechanisms of action are sufficiently characterized. Nonclinical studies encompass much more than just animal studies. Pivotal (i.e.,

FDA 40

FDA Science Small Molecule Immune Response 40

The Premier Consulting Blog

APRIL 25, 2023

In the US, new small molecule drugs are developed under the requirements of Sections 505(b)(1) and 505(b)(2) of the FFDCA. Due to concerns over manufacturing consistency and stability, the FDA required all oral levothyroxine manufacturers to obtain NDAs to continue marketing their products lawfully.

FDA 52

FDA Animal Testing Pharmacokinetics Pharmaceuticals 52

New Drug Approvals

SEPTEMBER 29, 2024

link] 01 Aug 2022 Cortexyme is now called Quince Therapeutics You need to be a logged in or subscribed to view this content This small molecule is an orally available protease inhibitor targeting the lysine proteases of the periodontal pathogen Porphyromonas gingivalis. In the same study, they show that in mice, oral P.

Small Molecule 62

Small Molecule DNA Disease Trials 62

DrugBank

FEBRUARY 26, 2025

In 2020, MIT researchers demonstrated the potential of AI by discovering salicin , a novel antibiotic that targets bacterial energy production. Our platform offers an extensive database of drug interactions, molecular targets, and pharmacokinetics, providing researchers with the tools to identify and optimize effective compounds.

Therapies 52

Therapies Pharmaceutical Companies Pharmacokinetics Treatment 52

The Pharma Data

FEBRUARY 25, 2022

The PAS was supported by positive topline data from the REFLECTIONS B538-12 study which evaluated multiple switches between treatment with ABRILADA and its reference product, Humira, both of which were administered with methotrexate in adult patients with moderate to severe rheumatoid arthritis (RA).

Biosimilars 52

Biosimilars FDA FDA Approval Small Molecule 52

The Pharma Data

OCTOBER 14, 2020

has expanded its product portfolio to include Blueprint, a self-service, scientific visualization and analytics application for small molecule discovery. Talking Medicine uses advanced AI to provide pharmaceutical companies with real-time data intelligence. Dolmatics – U.K.-based based Dotmatics Ltd.

Clinical Trials 52

Clinical Trials Trials Pharmaceuticals Therapies 52

Agency IQ

DECEMBER 11, 2023

BY RACHEL COE, MSC | DEC 6, 2023 5:27 PM EST The push and pull: Trends in oncology After the creation of the Accelerated Approval Pathway, several policies in the early 2000s were put in place with the goal of getting cancer products to patients faster. OCE has also been busy this year.

FDA 40

FDA Science Clinical Trials Trials 40

Codon

APRIL 2, 2023

Read Safety and pharmacokinetics of escalating doses of neutralising monoclonal antibody CAP256V2LS administered with and without VRC07-523LS in HIV-negative women in South Africa (CAPRISA 012B): a phase 1, dose-escalation, randomised controlled trial. Read Small-molecule aptamer for regulating RNA functions in mammalian cells and animals.

DNA 52

DNA RNA Engineering Licensing 52

DrugBank

FEBRUARY 5, 2025

Conversely, conjugation involves chemically attaching the drug to the nanoparticle surface, allowing for targeted delivery and improved pharmacokinetics. These ligands can be antibodies, peptides, aptamers, or small molecules.

Drugs 52

Drugs Engineering Vaccine Treatment 52

The Pharma Data

SEPTEMBER 15, 2020

Three of the nine boys were dosed with gene therapy product that was manufactured using the commercial manufacturing process developed at Pfizer’s facility in Sanford, North Carolina. The company announced the initiation of its Phase 1b clinical trial to evaluate the safety of a novel investigational therapeutic for COVID-19, PF-07304814.

Vaccine 52

Vaccine Therapies Trials Clinical Trials 52

Drug Target Review

OCTOBER 4, 2023

Groundbreaking strategies like proteolysis-targeting chimeric molecules (PROTACs) are also being explored. 6 Combining the effect of payloads with different mechanisms of action – an approach that revolutionised small molecule chemotherapy – also holds the promise of enhanced therapeutic activity for ADCs.

Small Molecule 126

Small Molecule Drugs Immune Response Targeted Protein Degradation 126

Drug Target Review

DECEMBER 4, 2024

In addition to generating agonistic antibodies, Confo is also successfully applying the ConfoBody/ConfoChimer technology to drugging GPCR with small molecules. VHH-based therapies have been clinically-validated many times with multiple products in numerous indications, including for long-term dosing.

Pharmacokinetics 122

Pharmacokinetics Small Molecule Therapies Immune Response 122

Metabolite Tales Blog

NOVEMBER 13, 2024

In vitro studies in rat liver microsomes looking at production of M1 revealed a 4.2 Several marketed drugs contain this ring system, for which oxadiazole ring reductive cleavage products have been reported [3]. References [1] Pharmacokinetics, disposition, and biotransformation of the cardiac myosin inhibitor aficamten in humans.

Pharmacokinetics 59

Pharmacokinetics Small Molecule Production Drugs 59