ASPET

SEPTEMBER 16, 2024

Glioblastoma (GBM) is a disease of the whole brain, with infiltrative tumor cells protected by an intact BBB. Acknowledging the potential for inter-species differences in pharmacokinetics, these data suggest that clinical translation of elimusertib in combination with temozolomide for treatment of GBM may be limited.

Pharmacogenetics 100

Pharmacogenetics DNA Treatment Pharmacokinetics 100

Elrig

MARCH 25, 2025

It will also cover recent advancements in broadening their application, including innovative approaches to ensure their safety and efficacy, the use of targeted delivery to reach disease-relevant tissues as well as success stories in bringing therapeutic oligonucleotides to the clinic.

RNA 59

RNA Bioinformatics Science Pharmacokinetics 59

ASPET

SEPTEMBER 16, 2024

Mitochondrial dysfunction is a hallmark of many genetic neurodegenerative diseases, but therapeutic options to reverse mitochondrial dysfunction are limited. These findings further support the therapeutic approach of targeting mitochondrial dysdynamism in neurodegeneration.

Small Molecule 100

Small Molecule Pharmacokinetics Engineering Disease 100

ASPET

JUNE 27, 2024

CB1 and CB2 agonists exhibit broad anti-inflammatory properties, suggesting their potential to treat inflammatory diseases. Secondary VAS and pharmacokinetic (PK) endpoints and adverse events were assessed. Background: Endocannabinoids, which are present throughout the central nervous system (CNS), can activate CB1 and CB2 receptors.

FDA Approval 100

FDA Approval Pharmacokinetics FDA Disease 100

ASPET

AUGUST 10, 2023

Pharmacokinetic half-lives ranged from 55 to 100 hours over the clinically relevant dose range, consistent with the expected half-life extension by glycoPEGylation. These results support future development of pegozafermin for the treatment of metabolic diseases, including nonalcoholic steatohepatitis and severe hypertriglyderidemia.

Cell Based Assays 100

Cell Based Assays Pharmacokinetics Regulations Treatment 100

DrugBank

SEPTEMBER 19, 2024

However, translating a promising therapeutic candidate into a successful oral medication presents pharmacokinetic and pharmacodynamic challenges.    Pharmacokinetics of Oral Drugs  Orally administered drugs undergo a series of biochemical processes that can affect their bioavailability and their clinical efficacy.

Pharmacokinetics 98

Pharmacokinetics Drugs Treatment Disease 98

Chemical Biology and Drug Design

MARCH 6, 2024

Abstract Targeting pro-inflammatory cytokines and their production is found to be of therapeutic benefit for the regulation of inflammation in various chronic autoimmune diseases. Computational analysis and in vitro studies corroborated the cytokines (TNF-α, IL-6 and IL-1β) inhibition potential of the new natural molecule polonilignan.

Pharmacokinetics 100

Pharmacokinetics Regulations Disease Production 100

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. IPF, unclassifiable interstitial lung diseases (uILDs), Long COVID respiratory complications and related sequelae) and lymphedema.

Pharmacokinetics 52

Pharmacokinetics Treatment Trials Production 52

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. James Miessler.

Disease 52

Disease Small Molecule Pharmacokinetics Licensing 52

ASPET

NOVEMBER 17, 2023

Quantitative pharmacokinetic models should therefore focus on OCT2/MATE when describing serum creatinine and creatinine clearance modulation by inhibitor drugs and genotype- or disease-related activity changes.

Pharmacogenetics 100

Pharmacogenetics Pharmacokinetics Disease Drugs 100

ASPET

AUGUST 29, 2023

A single subcutaneous dose of STAR-0215 ({greater than or equal to} 100 mg) was predicted to be active in patients for 3 months or longer, based on simulations using a minimal physiologically based pharmacokinetic (mPBPK) model.

Treatment 100

Treatment Pharmacokinetics Therapies Disease 100

Drug Target Review

NOVEMBER 1, 2023

2 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 1-5 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. Mediators of Inflammation. 2017;2017:1–15. Herr DR, Chew WS, Satish RL, Ong WY.

Clinical Trials 134

Clinical Trials Trials Small Molecule Disease 134

The Pharma Data

MARCH 22, 2021

Basel, 22 March 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced the decision to discontinue dosing in the Phase III GENERATION HD1 study of tominersen in manifest Huntington’s disease (HD). About Huntington’s disease. Survival ranges from approximately 10-20 years following motor onset of the disease.

Disease 52

Disease Clinical Trials Pharmacokinetics Treatment 52

The Pharma Data

DECEMBER 15, 2020

A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.

Disease 52

Disease Clinical Trials Small Molecule Trials 52

Drug Target Review

DECEMBER 12, 2023

We use hydrophilic linkers, which prevent ADC aggregation and generate highly stable ADCs, in combination with a unique attachment site on the antibody to create ADCs that retain pharmacokinetic properties similar to the original unconjugated antibody. This helps to maximize the targeted payload delivery to tumor cells.

Treatment 115

Treatment Engineering Pharmacokinetics Doctors 115

New Drug Approvals

OCTOBER 12, 2024

Alzheon licensed ALZ-801 from NeuroChem and is developing it for Alzheimer’s disease. ALZ-801 is an advanced and markedly improved candidate for the treatment of alzheimer’s disease. Clinical Pharmacokinetics and Safety of ALZ-801, a Novel Prodrug of Tramiprosate in Development for the Treatment of Alzheimer’s Disease.

Pharmacokinetics 62

Pharmacokinetics Small Molecule Disease Licensing 62

Drug Target Review

MARCH 17, 2025

By adjusting the activity of this receptor, these drugs can influence brain functions associated with mood, cognition and pain, positioning them as potential treatments for conditions like depression, cognitive disorders and neurological diseases. The drug’s pharmacokinetics (PK) and pharmacodynamics (PD) are closely linked.

Treatment 52

Treatment Licensing Licensing Science 52

New Drug Approvals

OCTOBER 14, 2024

Avenciguat can be used in research of chronic kidney disease (CKD) and diabetic kidney disease (DKD). Avenciguat (development name BI 685509 ) is a soluble guanylate cyclase activator developed by Boehringer Ingelheim for kidney disease, [1] [2] and cirrhosis. [3] 25 (8): 2218–2226. doi : 10.1111/dom.15099. PMID 37232058.

Pharmacokinetics 57

Pharmacokinetics Disease Trials Clinical Trials 57

Drug Target Review

DECEMBER 11, 2024

This shift in focus is especially critical in toxicology, where accurate target analysis plays a vital role in identifying toxic effects and ensuring patient safety, particularly as the field transitions from traditional drugs to the promising realm of biotherapeutics, especially for rare diseases.

International 52

International Laboratories Drugs Pharmacokinetics 52

The Pharma Data

NOVEMBER 26, 2020

(NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A.,

Disease 52

Disease Treatment Clinical Trials FDA 52

The Pharma Data

DECEMBER 29, 2020

(NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases , a business unit of Chiesi Farmaceutici S.p.A., mL/min/ 1.73m 2 /year.

Clinical Trials 52

Clinical Trials Disease Treatment Trials 52

DrugBank

OCTOBER 17, 2024

Computational chemistry and molecular modeling techniques can predict potential drug candidates' binding affinity and pharmacokinetic properties, enabling the selection of the most promising compounds for further development.

Drug Development 98

Drug Development Metabolic Stability Clinical Trials Drugs 98

The Pharma Data

NOVEMBER 5, 2021

About the study NCT03832114 is a Phase II, open- marker, two cohort,non-randomized study assessing the efficacity, safety and pharmacokinetics of iptacopan in cases with C3 glomerulopathy (C3G) ( cohort A) and cases who have experienced order transplantation and have posterior C3G rush in the transplanted organ ( cohort B).

Disease 52

Disease Treatment Pharmacokinetics FDA 52

LifeSciVC

APRIL 24, 2024

Target validation : Do we believe the target(s) plays a central role in disease biology and that modulation will modify disease? There is nothing more exciting than digging into a new target and trying to develop a thesis on whether modulation may be impactful in disease. Is the functionality of mutations known?

Production 117

Production Small Molecule Regulations Trials 117

The Pharma Data

OCTOBER 14, 2021

For all other classes evaluated in the analysis, including the broad range of MS therapies offered by Biogen, the antibody response to vaccination is consistent with the response of patients not being treated with an MS disease-modifying therapy (DMT). Cleveland Clinic, and a paid consultant for Biogen. Source link: [link].

Vaccine 52

Vaccine Therapies Immune Response Disease 52

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.

Small Molecule 131

Small Molecule DNA Pharmacokinetics Engineering 131

Drug Target Review

JULY 12, 2023

These assays provide insights into the molecular mechanisms of disease biology and drug response, enabling the characterisation of gene expression profiles and deviations in diseased cells. Importantly, transcriptomics information shows that certain aspects of a disease change the gene expression profile.

Biochemical Assays 98

Biochemical Assays Treatment Bioinformatics Disease 98

The Pharma Data

JANUARY 19, 2021

a privately-held neuron regeneration therapeutics company, today announced the publication of results of their novel pharmacotherapy: NNI-362 to allosterically stimulate neural regeneration in human cultures and in vivo models of aging and disease. There is a great need for a disease-modifying treatment for the 5.3 CLARKSVILLE, Md.,

Disease 40

Disease Small Molecule Clinical Trials Therapies 40

Drug Target Review

SEPTEMBER 19, 2023

The chips can be modelled with primary cells, IPS derived stem cells or patient derived cells and potentiates to unlock molecular mechanisms that drive the disease pathophysiology, holding a greater promise. Human organs-on-chips for disease modelling, drug development and personalized medicine. Lab on a Chip. 2012;12(12):2156-.

Animal Testing 98

Animal Testing Clinical Trials Pharmaceutical Companies FDA 98

The Pharma Data

JUNE 17, 2022

Submission supported by comprehensive analytical and clinical data from new Phase I bridging pharmacokinetics study Adalimumab’s high-concentration 100 mg/mL formulation aims to provide an enhanced yet familiar experience for patients Submission builds on Sandoz’ well established biosimilar immunology portfolio in Europe.

Biosimilars 52

Biosimilars Pharmacokinetics FDA Approval Packaging 52

The Pharma Data

JUNE 7, 2022

. “The approval of RIABNI is an important advancement for adults living with moderate to severe rheumatoid arthritis, a chronic inflammatory joint disease, who now have access to a proven and affordable treatment option,” said Murdo Gordon, executive vice president of Global Commercial Operations at Amgen.

Biosimilars 52

Biosimilars FDA Approval FDA Pharmacokinetics 52

DrugBank

FEBRUARY 12, 2025

This approach capitalizes on prior investments in R&D, mitigates risk by leveraging established safety and pharmacokinetic profiles, and accelerates the delivery of treatments to patients. They discovered that cimetidine inhibits gene expression in tumor growth and metastasis, suggesting that it could effectively slow disease progression. 

Cell Based Assays 52

Cell Based Assays Drugs Pharmacokinetics Disease 52

The Pharma Data

JANUARY 31, 2021

.–( BUSINESS WIRE )– Pinteon Therapeutics, a biotechnology company focused on protecting neuronal health by targeting neurotoxic forms of tau, today announced results from its Phase 1 study of PNT001, a novel tau antibody that uniquely targets a toxic epitope known to drive neurodegenerative disease.

Pharmacokinetics 52

Pharmacokinetics Disease Treatment Therapies 52

PPD

JULY 29, 2024

There’s an added level of complexity when clinical trials need to enroll specialist patient populations, like rare diseases, pediatric patients or pregnant women, where doses need to be carefully selected to keep the subject out of harm’s way.

Clinical Research 98

Clinical Research Clinical Trials Research Trials 98

The Pharma Data

NOVEMBER 11, 2020

“As an orally bioavailable small molecule with broad antiviral activity, SLV213 could be a valuable treatment to meet today’s urgent need to fight COVID-19 as well other life-threatening infectious diseases, such as Chagas disease, Ebola virus disease, and Nipah virus infection.”. For more information, visit www.selvarx.com.

Treatment 52

Treatment Small Molecule Virus Pharmacokinetics 52

The Pharma Data

JULY 12, 2021

These data include results from a late-breaking presentation from a Phase 2a study evaluating the safety and pharmacokinetics (PK) of once-monthly (QM) oral islatravir for pre-exposure prophylaxis (PrEP) through 24 weeks. Safety and Pharmacokinetics of Islatravir in Study Participants with Severe Renal Insufficiency. Abstract 2361.

Pharmacokinetics 52

Pharmacokinetics Clinical Trials Trials Treatment 52

Drug Target Review

OCTOBER 24, 2023

As a biotech company, Microbiotica has leaned into harnessing and leveraging the natural biology of host-microbe interactions to treat human disease. What advantages does this offer in the field of precision medicine?

Treatment 122

Treatment Pharmaceuticals Pharmacokinetics Trials 122