Drug Discovery World

MARCH 15, 2024

The Phase Ib study examined the safety, tolerability and pharmacokinetics of twice-daily oral doses of ALX-001 in 32 heathy adult participants aged 50-80. The findings were presented at the AD/PD 2024 Conference in Lisbon. Study results show that ALX-001 was safe in cognitively normal older adults at all tested doses. “We

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Drug Discovery World

AUGUST 14, 2023

Research partners in the UK are progressing their orally available small molecule cancer immunotherapy drug into clinical development as a treatment for advanced solid tumours. HTL0039732 works by blocking signalling through a specific type of prostaglandin receptor, the prostaglandin E2 (PGE2)-type prostanoid receptor 4 (EP4).

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Drug Hunter

MAY 18, 2023

From a childhood in a small village in China’s northeast Shandong province to her work examining the drug metabolism and pharmacokinetics (DMPK) of antibody-drug conjugates (ADCs) and Bicycle toxin conjugates ® (BTCs), Dian has always looked to forge something new. It built my confidence to build something from scratch.” Proteomics!

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DrugBaron

FEBRUARY 7, 2022

Finding small molecule drugs is much harder than finding a needle in a haystack – discovering the right arrangement of atoms to bind precisely to a protein target to elicit a particular response is a problem of vast dimensionality. Yet the situation with small molecules is even worse.

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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.

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Drug Discovery World

JANUARY 4, 2024

ANX1502 is a first-in-kind oral, selective small molecule inhibitor that targets the active form of C1s responsible for propagating classical pathway activation in association with C1q. The post Positive Phase I results for first-in-kind small molecule complement inhibitor appeared first on Drug Discovery World (DDW).

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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.

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The Premier Consulting Blog

AUGUST 8, 2024

For example, pharmacokinetic (PK) data from a comparative BA study and PK modeling approaches (e.g., Typically, new drug development under the 505(b)(2) pathway requires less capital and time and has a higher success rate versus the 505(b)(1) pathway, where the Sponsor develops safety and effectiveness information from scratch.

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The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial.

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Metabolite Tales Blog

JANUARY 23, 2024

Metabolism of 2023 FDA Approved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. References [1] 2023 Novel Small Molecule FDA Drug Approvals. Health Sci Rep. 6(10):e1610.

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Alta Sciences

JANUARY 31, 2024

Pharmacokinetics, Pharmacodynamics and Toxicokinetics Demystified pmjackson Wed, 01/31/2024 - 14:55 Understanding the effects of a drug, and how it interacts with the body, and vice versa, is critical to ensure it is safe for human use. This is where pharmacokinetic (PK), pharmacodynamic (PD) , and toxicokinetic (TK) analyses step in.

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Drug Discovery World

SEPTEMBER 26, 2022

To do this, the Drug Repurposing Hub has developed an open-access library of over 6,000 compounds, which are being tested against disease models in an effort to find new treatments using small molecules – many of which have already been approved by the FDA. . Diving into data . Volume 23 – Issue 4, Fall 2022. References .

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Sygnature Discovery

NOVEMBER 29, 2023

The QN-302 Phase 1 clinical trial is a multi-site, open-label, dose-escalation and dose expansion, safety, pharmacodynamic and pharmacokinetic study of intravenous QN-302 in patients with advanced or metastatic solid tumors. A total of up to 36 patients will be enrolled in the dose escalation (Phase 1a) portion of the study.

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Drug Target Review

NOVEMBER 1, 2023

2,8-10 However, the number of compounds with satisfying pharmacokinetic parameters (PKD) are limited, with most unable to cross the blood-brain barrier or go sufficiently deep into a malignant tumour. Structure–Activity Relationships of Small Molecule Autotaxin Inhibitors with a Discrete Binding Mode. J Med Chem. J Med Chem.

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The Pharma Data

JULY 2, 2023

To this end, the team carefully evaluated hits from a screen of around ten thousand small molecule compounds and identified icFSP1 as a new in vivo effective drug. Although FSP1 has been considered as an attractive drug target for cancer therapy, in vivo efficacious FSP1 inhibitors have been lacking.

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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.

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Sygnature Discovery

NOVEMBER 29, 2023

The QN-302 Phase 1 clinical trial is a multi-site, open-label, dose-escalation and dose expansion, safety, pharmacodynamic and pharmacokinetic study of intravenous QN-302 in patients with advanced or metastatic solid tumors. A total of up to 36 patients will be enrolled in the dose escalation (Phase 1a) portion of the study.

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Drug Target Review

JUNE 12, 2023

The current version of Chemistry42 uses over 40 generative models, including generative autoencoders and generative adversarial networks as well as both structure-based and ligand-based drug design to generate and optimise de novo small molecules.

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Molecular Design

FEBRUARY 7, 2023

The FDH is covered in the S2010 (see Box 1 and 2) and B2013 articles and, given that the targets of small molecule drugs tend to be intracellular, I’ll direct you to the excellent Smith & Rowland perspective on intracellular and intraorgan concentrations of drugs.

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The Pharma Data

FEBRUARY 25, 2022

The study met its primary goal by demonstrating pharmacokinetic equivalence in patients who switched multiple times between treatment with the two medicines. The company anticipates submitting study results for presentation at an upcoming medical congress.

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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.

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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. We hope it was a useful two-parter! We’re looking forward to the next crop! Br J Pharmacol.

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The Pharma Data

OCTOBER 27, 2020

Primmune aims to advance the development of their novel orally-administered, small molecule toll-like receptor 7 agonists as therapeutic-adjuvants for acute viral diseases and cancer. The company’s $27.4

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Drug Discovery World

JULY 11, 2024

MTX325 is a potent, selective, small molecule brain-penetrant USP30 inhibitor, which is designed to protect dopamine-producing neurons by improving mitochondrial quality and function. Fox Foundation for Parkinson’s Research (MJFF) and Parkinson’s UK.

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The Pharma Data

AUGUST 21, 2020

Novartis has agreed to contribute a carefully curated library of about 1300 small molecules with antiviral activity to the CARE project. We are also supporting the consortium’s preclinical safety and pharmacokinetics workstreams. These will be screened against SARS-CoV-2. And this is just one of our multistakeholder engagements.

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Drug Discovery World

APRIL 8, 2024

Alto Neuroscience has initiated a Phase II study to determine the pharmacodynamic effects of ALTO-203 in major depressive disorder (MDD) patients, as well as assess its safety, tolerability, and pharmacokinetics. To date, ALTO-203 has demonstrated positive emotional and cognitive effects in healthy participants after a single dose.

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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.

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Drug Discovery World

FEBRUARY 22, 2024

With no in-house laboratory space, the company works closely with multiple international contract research organisations (CROs), including Axxam, which carry out all of the Libra R&D, from compound screening assays, to in vivo pharmacokinetic/pharmacodynamic and ADME work, as well as in vitro and in vivo pre-clinical safety assessments.

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Drug Discovery World

DECEMBER 13, 2023

It demonstrated a differentiated in vitro activity profile across several assays compared with small molecule and antibody inhibitors of CD73 in clinical development. The company also presented data for CBO421, an AMP-competitive inhibitor of CD73.

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Drug Discovery World

OCTOBER 18, 2022

Enanta Pharmaceuticals, a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, has dosed the first subject in its Phase I clinical trial of EDP-323, a novel, oral L-protein inhibitor in development for the treatment of respiratory syncytial virus (RSV). . Context .

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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.

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Drug Discovery World

JULY 8, 2024

With the knowledge that RNA can adopt a diverse range of structural shapes, we can now develop therapeutics using RNAs 3D structure, similar to creating antibody therapies or small molecules that can target surfaces on proteins. These are critical attributes for acute care applications.

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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.

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The Pharma Data

JANUARY 19, 2021

The secondary objective is to assess the pharmacokinetic profile of SAD and MAD of ALS-4 administered orally to healthy subjects. The primary objective of the trial is to evaluate the safety and tolerability of SAD and MAD of ALS-4 administered orally to healthy subjects. About ALS-4.

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Drug Discovery World

NOVEMBER 3, 2022

VRG50635 is a small molecule inhibitor of PIKfyve, a novel therapeutic target for ALS discovered by CONVERGETM, Verge’s all-in-human, AI-powered platform. . Through the evaluation of more than 11.4

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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.

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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. Blood 2019;134(Suppl 1):3517. 3. Schubart A, et al.

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