Drug Target Review

JUNE 12, 2023

TEAD proteins are known to be very important in cancer progression TEAD proteins are known to be very important in cancer progression, and there are a number of therapies in development. What are the preclinical characteristics of ISM6331, including its efficacy, safety profile, and drug metabolism and pharmacokinetics (DMPK) properties?

Metabolic Stability 92

Metabolic Stability Small Molecule Clinical Trials Pharmacokinetics 92

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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Clinical Trials Treatment Trials Small Molecule 52

The Pharma Data

APRIL 11, 2023

Bayer is advancing its oncology R&D efforts in three scientific areas that have the potential to address unmet needs in cancer patients: next-generation Immuno-Oncology, Targeted Radionuclide Therapies and Precision Molecular Oncology. Data from all three areas of scientific focus will be showcased during this year’s meeting.

Research 40

Research Clinical Trials Small Molecule Pharmacokinetics 40

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.

Clinical Trials 40

Clinical Trials Treatment Trials Small Molecule 40

The Pharma Data

OCTOBER 27, 2020

Former Sarepta Therapeutics executive Bo Cumbo left to launch his new gene therapy company, AavantiBio, with a $107 million Series A. AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders. AavantiBio . Sirnaomics.

Small Molecule 52

Small Molecule Immune Response Pharmacokinetics Clinical Development 52

Drug Target Review

JULY 12, 2023

We also leverage existing clinical gene expression data from different subsets of molecularly profiled cancer patients, who differ in a certain aspect of disease or response to a particular therapy.

Biochemical Assays 98

Biochemical Assays Treatment Bioinformatics Disease 98

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.

Biosimilars 52

Biosimilars FDA FDA Approval Small Molecule 52

The Pharma Data

AUGUST 21, 2020

And then the long-term goal of the project is to develop new therapies for the SARS-CoV-2 virus at the root of COVID-19, as well as other coronaviruses which could cause future pandemics. Novartis has agreed to contribute a carefully curated library of about 1300 small molecules with antiviral activity to the CARE project.

Virus 52

Virus Small Molecule Licensing Licensing 52

Drug Discovery World

JULY 8, 2024

While mRNA vaccines and RNA interference therapies have garnered much attention, a new class of RNA therapeutics known as aptamers is gaining momentum, particularly to address acute diseases. The most developed RNAi is small interfering ribonucleic acid (siRNA)-based therapies.

RNA 148

RNA Small Molecule Vaccine FDA Approval 148

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. Cidara is currently advancing its dual CD73/PD-1 inhibitor DFC and CBO421 in preclinical studies.

Small Molecule 130

Small Molecule Biosimilars Pharmacokinetics Drugs 130

The Pharma Data

AUGUST 29, 2020

Oral, investigational complement factor B inhibitor LNP023 substantially improved hematological response as add-on therapy to eculizumab. To date, after at least six months of stable LNP023 add-on therapy, and at the investigators’ discretion, seven patients (70%) have discontinued eculizumab and remained on LNP023 as monotherapy.

Treatment 52

Treatment Disease Small Molecule Production 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

LifeSciVC

APRIL 24, 2024

and whether a molecule’s pharmacology can help to mitigate safety risk. Especially for oligo or small molecule discovery and development, it’s important to understand whether there are highly homologous sequences or proteins that may be impacted by a given therapeutic approach. in liver, in CNS)?

Production 117

Production Small Molecule Regulations Trials 117

The Pharma Data

NOVEMBER 4, 2020

Food and Drug Administration (FDA) Breakthrough Therapy and Fast Track Designations, is now in pivotal testing, and CTP-692 for schizophrenia is currently on track for topline data readout in the first quarter of 2021,” said Roger Tung, Ph.D., CTP-543 for moderate to severe alopecia areata, which received U.S.

Pharmaceuticals 52

Pharmaceuticals Clinical Trials Trials Small Molecule 52

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. Photo: Business Wire).

Pharmacokinetics 52

Pharmacokinetics Treatment Small Molecule Production 52

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. For some pharmaceutical companies, like AstraZeneca 3 , 30% of their early pipeline consists of new drug modalities. In a study by Long et al.,

Drug Development 130

Drug Development Drugs Small Molecule Immune Response 130

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. Moreover, custom antibodies are a key reagent in companion diagnostics for these novel therapeutics.

Science 148

Science Small Molecule Laboratories Engineering 148

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. For some pharmaceutical companies, like AstraZeneca 3 , 30% of their early pipeline consists of new drug modalities. In a study by Long et al.,

Drug Development 130

Drug Development Drugs Small Molecule Immune Response 130

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.”. About SLV213.

Treatment 52

Treatment Small Molecule Virus Pharmacokinetics 52

Alta Sciences

APRIL 17, 2024

Psychedelics Psychedelic therapy (or psychedelic-assisted therapy) refers to the use of psychedelic drugs, such as psilocybin, MDMA, LSD, ketamine, and ayahuasca, to treat mental disorders, especially those that have no effective treatments available or are treatment resistant.

Drug Development 52

Drug Development Treatment FDA Approval Drugs 52

Agency IQ

JUNE 9, 2023

However, this guideline is primarily intended to provide recommendations on evaluating the immune system’s response to small molecule drugs. The guidance contains recommendations for small molecule drugs, as well as any biological products that fall within CDER’s purview.

FDA 40

FDA Science Small Molecule Immune Response 40

The Pharma Data

JANUARY 19, 2021

This small molecule therapy is presently in Phase 1 clinical trial for mild to moderate Alzheimer’s disease (AD), which is supported by a NIA R01 grant in healthy aged volunteers. CLARKSVILLE, Md., 20, 2021 (GLOBE NEWSWIRE) — Neuronascent Inc. , There is a great need for a disease-modifying treatment for the 5.3

Disease 40

Disease Small Molecule Clinical Trials Pharmacokinetics 40

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. About CG-806.

Small Molecule 40

Small Molecule Clinical Trials Trials Therapies 40

Codon

APRIL 2, 2023

Read A one-time gene therapy injection for spinal muscular atrophy. Mice and human cells that were given the therapy had normal levels of the survival motor neuron protein, and no symptoms. Gene Therapy. Read A really simple way to isolate AAVs, which are a type of virus often used to deliver gene therapies into cells.

DNA 52

DNA RNA Engineering Licensing 52

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

The clinical responses were sustained by maintenance therapy with belimumab, an antibody to B-cell activating factor. Small Molecule Inhibitors. NMD670 is a first-in-class small molecule inhibitor of the muscle specific chloride ion channel, the ClC-1 ion channel. Oral, Small Molecules.

Disease 52

Disease Clinical Trials Small Molecule Trials 52

The Pharma Data

JANUARY 10, 2021

TransCon PTH: TransCon PTH is an investigational long-acting prodrug of parathyroid hormone (PTH) in development as a potential once-daily replacement therapy for adult hypoparathyroidism (HP): . . •. ?. . ?. . .

Clinical Trials 40

Clinical Trials Small Molecule Clinical Development Trials 40

Agency IQ

FEBRUARY 23, 2024

Additionally, this approach is generally no longer applicable to new treatments that are more targeted in their effects, such as immunotherapies and targeted small molecule therapies. This has spawned a movement focused on optimizing dose selection in oncology drug development.

Science 40

Science FDA Trials Clinical Pharmacology 40

The Pharma Data

SEPTEMBER 15, 2020

Of note, The Phase 1b study is a double-blind, placebo-controlled clinical trial evaluating the safety, tolerability and pharmacokinetics of PF-07304814, a phosphate prodrug that when administered intravenously is metabolized to the active compound PF-00835321, shown to be a very potent inhibitor of the SARS-Cov2 3CL protease in preclinical studies.

Vaccine 52

Vaccine Therapies Trials Clinical Trials 52

Agency IQ

DECEMBER 11, 2023

The side effects of some anticancer treatments can result in dose interruptions or de-escalations, or even outright discontinuation of an efficacious therapy. In 2015, the FDA, along with the American Association for Cancer Research (AACR), held a workshop on dose optimization for small molecules.

FDA 40

FDA Science Clinical Trials Trials 40

The Pharma Data

OCTOBER 14, 2020

Food and Drug Administration granted Breakthrough Therapy designation to France-based Inventiva’s experimental NASH treatment lanifibranor. has expanded its product portfolio to include Blueprint, a self-service, scientific visualization and analytics application for small molecule discovery. Dolmatics – U.K.-based

Clinical Trials 52

Clinical Trials Trials Pharmaceuticals Pharmaceutical Companies 52