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?

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Metabolic Stability Clinical Trials Small Molecule Therapies 92

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).

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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.

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Treatment Small Molecule Virus Pharmacokinetics 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

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 Therapies 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 Clinical Development Therapies 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.

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Drug Development Treatment Drugs FDA Approval 52

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

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.

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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

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.

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Clinical Trials Small Molecule Trials Therapies 40

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

New Drug Approvals

APRIL 18, 2025

2] The US Food and Drug Administration (FDA) granted the application for crinecerfont fast track , breakthrough therapy , orphan drug , and priority review designations. [2] New Drug Therapy Approvals 2024 (PDF). 2] In the first trial, 122 adults received crinecerfont twice daily and 60 received placebo twice daily for 24 weeks. [2]

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FDA FDA Approval Drugs Trials 62

New Drug Approvals

SEPTEMBER 13, 2024

14] Pharmacokinetics The oral bioavailability of aticaprant is 25%. [1] 19] As of 2016, aticaprant has reached phase II clinical trials as an augmentation to antidepressant therapy for treatment-resistant depression. [20] 14] Pharmacokinetics The oral bioavailability of aticaprant is 25%. [1] 2] Aticaprant is taken by mouth. [1]

Treatment 62

Treatment Clinical Trials Pharmacokinetics Pharmaceuticals 62

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

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): . . •. ?. . ?. . .

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Clinical Trials Clinical Development Trials Small Molecule 40

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.

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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

Broad Institute

OCTOBER 29, 2024

This small molecule reactivates the apoptosis cascade in tumor cells while sparing healthy cells in animal models. Previous clinical and laboratory studies on other MCL1 inhibitors have suggested that these molecules can impair heart cells, likely because of prolonged exposure to the compounds.

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PPD

APRIL 14, 2025

This expansion is creating opportunities for clinical trials related to a range of new therapy areas and their subpopulations. Thermo Fisher delivers everything needed for biologics, small molecule and viral vector manufacturing, all the way through validation and regulatory support.

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Clinical Trials Trials Clinical Development Disease 52

Alta Sciences

APRIL 24, 2025

Case Study: How Altasciences Overcame Pharmacological Challenges in a GLP-1 IND-Enabling Study pmjackson Thu, 04/24/2025 - 21:43 For emerging therapies with pronounced pharmacological effects, thoughtful study design is critical to ensuring reliable and interpretable data.

Small Molecule 52

Small Molecule Pharmacokinetics Treatment Compliance 52

DrugBank

FEBRUARY 26, 2025

This stagnation has created a perfect storm, threatening the effectiveness of current therapies and limiting the pipeline of new drugs. Advances in pharmacology and drug formulation have enabled researchers to refine these therapies and extend their utility.

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Therapies Pharmaceutical Companies Pharmacokinetics Treatment 52

The Pharma Data

JULY 2, 2023

The new approach in cancer therapy with innovative mechanism-of-action for ferroptosis induction A team of researchers led by Dr. Marcus Conrad from Helmholtz Munich discovered a novel anti-cancer drug, called icFSP1, which sensitizes cancer cells to ferroptosis.

Therapies 40

Therapies Small Molecule Pharmacokinetics Disease 40

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

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

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Clinical Trials Trials Pharmaceuticals Pharmaceutical Companies 52

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

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 52

Clinical Trials Treatment Trials Small Molecule 52

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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Biosimilars FDA FDA Approval Small Molecule 52

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 40

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

Alta Sciences

SEPTEMBER 27, 2024

Featuring two scenarios that explore the complexities of bioanalysis for immunomodulators, The Altascientist offers practical considerations for ensuring accurate bioanalysis, as well as pharmacokinetic, pharmacodynamic , and safety data in clinical trials. Each class of immunomodulator has a defined complexity and mechanism of action.

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Clinical Trials Trials Pharmacokinetics Small Molecule 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.

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Drugs Engineering Vaccine Treatment 52

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.

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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.

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Metabolite Tales Blog

JANUARY 22, 2025

Metabolism of 2024 FDA approved small molecules part 1 By Julia Shanu-Wilson Involvement of active metabolites The number of small molecules approved by the FDA in 2024 totaled 31 out of 50 NMEs with 56% (28) of these comprising small molecule therapies. Clin Pharmacokinet. Aprocitentan.

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Small Molecule FDA Approval FDA Pharmacokinetics 52

Drug Target Review

APRIL 25, 2025

3 These models are pivotal for offering key predictive insights into the clearance of metabolically stable small molecules, maintaining phase I and II metabolic activities for up to four weeks. 9,10 According to IQ-MPS recommendations, liver-on-chip systems can differentiate between toxic and non-toxic small molecule analogues.

Clinical Development 52

Clinical Development Small Molecule Drugs FDA Approval 52

New Drug Approvals

MAY 27, 2025

Cardiovascular and Pharmacokinetic Profiles of Intravenous Etripamil in Conscious Telemetered Cynomolgus Monkeys. ETRIPAMIL CAS 1593673-23-4 AS ACETATE 512.64 Milestone Pharmaceuticals Announces USAN Approval of Generic Name Etripamil for its Phase 2 Clinical Development Product for the Treatment of Paroxysmal Supraventricular Tachycardia. [3].

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Pharmaceuticals Small Molecule Pharmacokinetics Treatment 67