Sygnature Discovery

NOVEMBER 29, 2023

A new treatment for G4-targeted solid tumors including pancreatic cancer has been administered to patients for the first time in a Phase 1a clinical trial. In January 2023, QN-302 received Orphan Drug Designation by the FDA for the potential treatment of pancreatic cancer. This deadly cancer has a 5-year survival rate of just 7%.

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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?

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Sygnature Discovery

NOVEMBER 29, 2023

A new treatment for G4-targeted solid tumors including pancreatic cancer has been administered to patients for the first time in a Phase 1a clinical trial. In January 2023, QN-302 received Orphan Drug Designation by the FDA for the potential treatment of pancreatic cancer. This deadly cancer has a 5-year survival rate of just 7%.

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Drug Discovery World

SEPTEMBER 9, 2024

Vesper Bio has completed a Phase I study for its lead candidate, VES001, a potentially disease-modifying treatment for frontotemporal dementia patients with mutations in the progranulin gene (FTD(GRN)). Frontotemporal dementia is the most common cause of dementia in people under the age of 60.

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New Drug Approvals

SEPTEMBER 10, 2024

g/mol 1929519-13-0 NBI-1065846 or TAK-041 Phase 2 (S)-2-(4-oxobenzo[d][1,2,3]triazin-3(4H)-yl)-N-(1-(4-(trifluoromethoxy)phenyl)ethyl)acetamide Zelatriazin ( NBI-1065846 or TAK-041 ) is a small-molecule agonist of GPR139. Zelatriazin, C 18 H 15 F 3 N 4 O 3 , 392.3 88 (8): 3872–3882. doi : 10.1111/bcp.15305. PMC 9544063. .;

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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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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. Across all doses evaluated, ANX1502 was generally well tolerated with mild to moderate treatment-emergent adverse events (TEAEs).

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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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Drug Discovery World

SEPTEMBER 26, 2022

Perhaps the biggest benefit drug repurposing brings is its potential to get treatments to patients faster than traditional means of drug discovery. This level of growth is unsurprising for an industry which will always look to the latest opportunities to drive science. . Benefits and barriers . Diving into data . References .

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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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Drug Target Review

JUNE 12, 2023

With 13 preclinical candidates and three AI-designed drugs currently undergoing clinical trials, Insilico is spearheading a revolution in cancer treatment and beyond. Can you provide a summary of the key findings and implications of the preclinical studies on ISM6331 for the treatment of advanced solid tumours?

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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.

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Drug Discovery World

JULY 11, 2024

The funding will help advance Mission’s potential disease-modifying treatment for Parkinson’s disease (PD), MTX325. 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.

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The Pharma Data

FEBRUARY 25, 2022

“Today’s announcement builds on our commitment to broaden access to essential, high-quality and cost-effective treatment options for patients living with certain chronic inflammatory conditions.”. Biosimilars play an important role in the treatment of autoimmune conditions.

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The Pharma Data

APRIL 11, 2023

Bayer is progressing novel research around its prostate cancer treatment darolutamide. 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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The Pharma Data

OCTOBER 27, 2020

Foghorn is anticipating filing an IND later this year for its lead candidate to begin a Phase I trial for the treatment of uveal melanoma, a cancer of the eye. . more than 3,600 new cases of anaplastic glioma are diagnosed each year with a median survival of just over three years despite treatment. Scorpion Therapeutics . In the U.S.,

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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. Dermavant’s tapinarof is one such friend. 8 This is not the only point of interest.

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The Pharma Data

AUGUST 21, 2020

When COVID-19 began to emerge – first in Asia, then in Europe – she could see that its relative ease transmitting from person to person, along with the lack of tests and preventions or treatments for infection, made the virus a credible threat to humanity. These will be screened against SARS-CoV-2.

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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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The Pharma Data

AUGUST 29, 2020

Phase II results are promising for treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder; 1,2 second Phase II study with LNP023 monotherapy in anti-C5 naïve PNH patients ongoing . All patients required RBC transfusions prior to starting LNP023 treatment. “In Peffault de Latour.

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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). .

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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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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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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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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 Pharma Data

NOVEMBER 4, 2020

CTP-543: An Investigational Treatment for Moderate to Severe Alopecia Areata. The presentation highlighted that in the open label extension study, treatment with CTP-543 shows continued maintenance of hair regrowth relative to the hair growth shown in the Company’s previously conducted Phase 2 trials in patients with alopecia areata.

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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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Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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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

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. The therapeutic dose of pirfenidone approved by the US Food and Drug Administration (FDA) for the treatment of IPF is 801 mg three times a day.

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The Pharma Data

NOVEMBER 11, 2020

Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for SLV213 for the treatment of COVID-19 and has dosed the first subjects in a Phase 1 clinical study. SLV213 is a novel, orally available, small molecule antiviral drug candidate that inhibits human host cell cysteine proteases to block viral entry.

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Drug Target Review

OCTOBER 4, 2023

Antibody-drug conjugates (ADCs) have been a groundbreaking approach to cancer treatment with their ability to deliver cytotoxic drugs directly to diseased cells while sparing healthy tissues. Groundbreaking strategies like proteolysis-targeting chimeric molecules (PROTACs) are also being explored.

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New Drug Approvals

SEPTEMBER 13, 2024

2] Like other kappa opioid antagonists currently under clinical investigation for the treatment of major depression, its efficacy may be compromised by the countervailing activation of pro-inflammatory cytokines in microglia within the CNS. [7] 14] Pharmacokinetics The oral bioavailability of aticaprant is 25%. [1] nM vs. 24.0

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Metabolite Tales Blog

DECEMBER 13, 2023

Conjugation of small molecule drugs to glucuronic acid is catalysed by several UGTs to frequently form N – and O -glucuronides. As in this example, most N -glucuronides of small molecule drugs are innocuous and possess no pharmacological activity. Tukey, Ryoichi Fujiwara. J Pharm Biomed Anal. Oncotarget.

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Drug Discovery World

DECEMBER 13, 2023

Custom antibodies can be developed for pharmacokinetic assays and biodistribution assays, supporting therapeutic development by giving researchers data about the behaviour of their novel therapeutic in vivo. Caplacizumab, for the treatment of a rare blood clotting disease, achieved FDA approval in 2019 2.

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Drug Discovery World

APRIL 12, 2023

Testing new modality drugs poses additional challenges The drug development pipeline is no longer predominantly composed of small molecule drugs. Such longitudinal studies enable adverse effects to be identified that would otherwise be missed, possibly until a drug reached the market, as was the case for Troglitazone.

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Drug Discovery World

JANUARY 20, 2023

Testing new modality drugs poses additional challenges The drug development pipeline is no longer predominantly composed of small molecule drugs. Such longitudinal studies enable adverse effects to be identified that would otherwise be missed, possibly until a drug reached the market, as was the case for Troglitazone.

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